Tobias Gedde-Dahl

• Rinaldetti, S.; Chepeleva, M.; Hedblom, A.; Sticht, C.; Muciek, M. & Panayiotidis, P. [Vis alle 28 forfattere av denne artikkelen] (2026). Comprehensive molecular characterization of treatment-free remission and molecular relapse in chronic myeloid leukemia patients: the EURO-SKI Biomarker Study. Leukemia. ISSN 0887-6924. doi: 10.1038/s41375-026-03001-5. Fulltekst i vitenarkiv Vis sammendrag

  The European Stop Kinase Inhibitors (EURO-SKI) trial was launched to investigate the discontinuation of tyrosine kinase inhibitors (TKIs) in patients with chronic myeloid leukemia (CML). The molecular mechanisms underlying sustained treatment-free remission (TFR) and loss of TFR, are still poorly understood. To address this, we performed whole transcriptome gene expression analyses coupled with NanoString nCounter-based absolute gene quantification. Gene expression was assessed in peripheral blood leukocytes from 240 CML patients on the final day of TKI intake (training sample: n = 122, validation sample: n = 118) as well as from 10 healthy controls. To identify TKI-specific mechanisms, transcriptomic data from an external dataset of 96 TKI-naïve CML patients was incorporated into our analyses. TFR patients showed an activation of GATA1, KLF1 and MYBL1 regulons characteristic for erythroid progenitor cells. TFR patients maintain persistent communication between innate (natural killer and dendritic cells) and adaptive immunity (e.g., CD8+, CD4+, and γδ T lymphocytes), mirroring patterns observed in healthy controls. This intercellular communication is disrupted in patients with loss of TFR. Furthermore, we identified an FLT3 threshold that distinguished two patient groups with significantly different probabilities of TFR loss. Leveraging mechanisms that reestablish or reinforce the communication between innate and adaptive immunity could be pivotal in achieving durable TFR.

• Pati, Niladri Bhusan; Chung, Brian K.; Holm, Kristian; Sætre, Frank Ove; Reims, Henrik Mikael & Aasebø, Anders Theodor Michaelsen [Vis alle 12 forfattere av denne artikkelen] (2026). Ultra long-lived plasma cells in the human small intestine produce microbiota-reactive IgA antibodies. Beneficial Microbes. ISSN 1876-2883. s. 1–13. doi: 10.1163/18762891-bja00120. Fulltekst i vitenarkiv
• Myhre, Anders Eivind; Meyer-Myklestad, Malin Holm; Gullaksen, Hanne Hestdal; Søgaard, Ole S.; Tolstrup, Martin & Salgado, Maria [Vis alle 51 forfattere av denne artikkelen] (2026). Long-term HIV-1 remission achieved through allogeneic haematopoietic stem cell transplant from a CCR5Δ32/Δ32 sibling donor. Nature Microbiology. 11, s. 1374–1386. doi: 10.1038/s41564-026-02304-8. Fulltekst i vitenarkiv Vis sammendrag

  Only few cases of human immunodeficiency virus (HIV) remission have been reported after allogeneic haematopoietic stem cell transplantation (HSCT), mostly involving stem cell donors with the homozygous CCR5Δ32 (CCR5Δ32/Δ32) mutation, which confers resistance to CCR5-tropic HIV-1. Here we report the case of a 63-year-old man in off-treatment HIV remission, 5 years after HSCT with a CCR5Δ32/Δ32 sibling donor for myelodysplastic syndrome. In-depth clinical characterization including virological and immunological analyses of peripheral blood, gut and bone marrow samples revealed that full donor chimerism was achieved. Antiretroviral therapy was discontinued after 24 months, and 48 months after HSCT, no intact HIV DNA was detected in blood or gut biopsies. Replication-competent virus and HIV-specific T cell responses were absent, and HIV antibody responses showed a gradual decline. Full donor chimerism in the gut, which is the primary viral reservoir, underscores the likelihood of a cure.

• Iversen, Per Ole; Gedde-Dahl, Tobias; Tjønnfjord, Geir Erland & Ali, Maryan Mohamed (2025). Quality of life the first year after allogeneic haematopoietic stem cell transplantation—A randomized controlled trial examining extracorporeal photophoresis to prevent graft‐versus‐host disease. British Journal of Haematology. ISSN 0007-1048. 207(5), s. 2183–2186. doi: 10.1111/bjh.70119. Fulltekst i vitenarkiv
• Villalba, Marta; Ferhat, Allain-Thibeault; Gedde-Dahl, Tobias; Socie, Gerard; Huynh, Anne & Blau, Igor Wolfgang [Vis alle 27 forfattere av denne artikkelen] (2025). Impact of donor selection in adverse-risk AML undergoing hematopoietic cell transplantation: A study from the EBMT Acute Leukemia Working Party. Bone Marrow Transplantation. ISSN 0268-3369. 61(2), s. 187–196. doi: 10.1038/s41409-025-02751-7. Fulltekst i vitenarkiv
• Skøyeneie, Sigrid Cecilie Tinglum; Fauske, Lena; Wik, Hilde Skuterud; Tjønnfjord, Geir Erland; Gedde-Dahl, Tobias & Myhre, Anders Eivind (2025). Patients’ Experiences With Hospital at Home Following Allogenic Hematopoietic Stem Cell Transplantation: A Qualitative Study. Cancer Nursing. ISSN 0162-220X. doi: 10.1097/NCC.0000000000001504. Fulltekst i vitenarkiv Vis sammendrag

  Background Hospital at Home (HaH) models aim to deliver hospital-level care to patients in a home setting. In the context of allogeneic hematopoietic stem cell transplantation (HSCT), HaH is intended to improve patients’ quality of life and reduce symptom burden. However, limited research exists on patients’ perceptions of this innovative care model. Objective This study aimed to explore how patients experienced HaH following HSCT. Methods A qualitative exploratory research design was employed, utilizing semistructured individual interviews. Sixteen patients who received HaH care following HSCT were interviewed. Reflexive thematic analysis was used to identify themes representing patterns of meaning across the dataset. Results The following 3 themes were identified: (1) actively working through the illness experience: participants needed to engage in active coping mechanisms, emphasizing the crucial role of their informal caregivers, (2) recognition of normality in the illness situation: experiencing autonomy and living in a homelike environment enhanced their sense of normality, and (3) fundamental aspects of daily life that promote recovery: psychological and emotional benefits of the private setting, including improved rest, calmness, relaxation, increased physical activity, and the opportunities to engage in familiar daily activities, facilitated recovery in ways that differed from the traditional hospital setting. Conclusion This study suggests that HaH positively influences recovery and well-being among HSCT patients and has the potential to improve patient outcomes. Implications for Practice HaH offers a viable alternative to traditional hospitalization, promoting a more personalized approach to care.

• Loukili, Noureddine Henoun; Gras, Luuk; Koster, Linda; Blaise, Didier; Gedde-Dahl, Tobias & Maertens, Johan [Vis alle 23 forfattere av denne artikkelen] (2025). Conditioning intensity in myelodysplastic patients aged ≥ 50 years undergoing allogeneic hematopoietic cell transplantation (allo-HCT): a study on behalf of the chronic malignancies working party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 60(11), s. 1487–1495. doi: 10.1038/s41409-025-02682-3. Fulltekst i vitenarkiv Vis sammendrag

  Reduced intensity conditioning (RIC) is usually used for patients with myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (allo-HCT), particularly in the elderly or those with comorbidities. The impact of conditioning intensity on patients’ outcome remains controversial with clinicians’ subjective opinion/ experience remaining a major guide in choosing the intensity. Here, we compare RIC versus MAC in a large EBMT retrospective study in MDS patients aged ≥50 years undergoing allo-HCT between 2014 and 2018. Among the 1393 included patients, 922 (66%) were males, and the median age at transplant was 62.8 (50.0–77.9) years. The majority of patients (n = 884; 64.3%) had MDS with excess blasts. IPSS-R recorded was very low/low (n = 598, 43%), intermediate (n = 352, 25%), and high/very high (n = 443, 32%). Karnofsky index was ≥90 in 916 (69.3%) patients, and HCT-CI ≥ 3 in 292(27.3%) patients. A RIC regimen was used in 1053 (75.5%) patients. Median follow-up was 27.9 months (IQR: 26.4–30.6). Both uni- and multi-variable analyses did not show any significant association between conditioning intensity and outcomes. This study highlights a lack of association between RIC/MAC regimens and outcomes in allo-HCT MDS patients. Our results support the recently published systematic review and meta-analysis, where evidence for using one conditioning regimen over another remains weak.

• Ru, Yuhua; Chen, Jia; Ferhat, Allain Thibeault; Labussière-Wallet, Hélène; Gedde-Dahl, Tobias & Kröger, Nicolaus [Vis alle 14 forfattere av denne artikkelen] (2025). Similar Outcome With Haploidentical, Matched Sibling, or Matched Unrelated Donor Hematopoietic Cell Transplantation for Adult Patients With Adverse-Risk TP53-Mutated Acute Myeloid Leukemia in First Remission: A Comparative Study From the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. American Journal of Hematology. ISSN 0361-8609. 100(12), s. 2220–2229. doi: 10.1002/ajh.70069. Fulltekst i vitenarkiv
• Bazarbachi, Ali; Galimard, Jacques Emmanuel; Dalle, Iman Abou; Socié, Gérard; Versluis, Jurjen & Wu, Depei [Vis alle 20 forfattere av denne artikkelen] (2025). Challenging the Adverse Label: Diverse Outcomes of ELN 2022 Adverse Cytogenetic Subgroups in Acute Myeloid Leukemia Patients Allografted in First Remission: From EBMT ALWP. American Journal of Hematology. ISSN 0361-8609. 100(8), s. 1374–1386. doi: 10.1002/ajh.27726. Fulltekst i vitenarkiv
• Pagano, Livio; Maschmeyer, Georg; Lamoth, Frederic; Blennow, Ola; Xhaard, Alienor & Spadea, Manuela [Vis alle 51 forfattere av denne artikkelen] (2025). Primary antifungal prophylaxis in hematological malignancies. Updated clinical practice guidelines by the European Conference on Infections in Leukemia (ECIL). Leukemia. ISSN 0887-6924. 39(7), s. 1547–1557. doi: 10.1038/s41375-025-02586-7. Fulltekst i vitenarkiv Vis sammendrag

  At the 10th European Conference on Infections in Leukaemia (ECIL), the guidelines for antifungal prophylaxis in pediatric and adult patients with hematological malignancies (HM) were updated and some changes introduced. Regarding acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients undergoing remission induction chemotherapy, a B-II grading has been assigned to isavuconazole, micafungin, and caspofungin, based on non-randomized studies that have shown efficacy in preventing invasive fungal diseases (IFD). Regarding high-risk MDS patients treated with azacytidine, prophylaxis with posaconazole during the first four cycles of treatment is supported in the literature. Prophylaxis is not indicated in patients treated for myeloproliferative neoplasms (NPM), acute lymphoid leukemia (ALL), and Hodgkin lymphoma (HL). For patients with chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma (NHL), prophylaxis is not generally indicated. For patients with multiple myeloma (MM), prophylaxis is not indicated and the limited epidemiological data available do not support the use of prophylaxis in subjects treated with bispecific antibodies. For patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT), no substantial changes were made, apart from the addition of isavuconazole with grading B-II in the post-engraftment period. In patients undergoing auto-HSCT, antifungal prophylaxis is not indicated. Previous ECIL guidelines did not include CAR-T cells. The expert panel proposes to endorse the use of anti-mold prophylaxis in high-risk patients during pre-infusion and post-infusion, while in low-risk patients, anti-yeast prophylaxis can be recommended (B-II). For pediatric hematology patients, based on newly published data, caspofungin received a B-I grading as mold-active prophylaxis. Moreover, patients with ALL with insufficient treatment response during induction therapy, and children older than 12 y.o are now considered at high risk for IFD and are recommended to receive antifungal prophylaxis.

• Ye, Yishan; Labopin, Myriam; Yakoub-Agha, Ibrahim; Socié, Gérard; Blaise, Didier & Gedde-Dahl, Tobias [Vis alle 21 forfattere av denne artikkelen] (2025). Superior GVHD-Free, Relapse-Free Survival for Haploidentical Transplant With PTCy Than Matched Unrelated Donor for AML Patients Transplanted in Second Complete Remission: A Study From the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. American Journal of Hematology. ISSN 0361-8609. 100(10), s. 1760–1771. doi: 10.1002/ajh.70008. Fulltekst i vitenarkiv
• Nagler, Arnon; Ferhat, Allain Thibeault; Kayser, Sarah; Eder, Matthias; Kröger, Nicolaus & Stelljes, Matthias [Vis alle 18 forfattere av denne artikkelen] (2025). Prognostic significance of cytogenetic risk score in patients with secondary acute myeloid leukemia undergoing allogeneic stem cell transplantation from HLA-matched unrelated donors: a study from the ALWP /EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 60(9), s. 1218–1227. doi: 10.1038/s41409-025-02620-3. Fulltekst i vitenarkiv
• Iacobelli, Simona; Schönland, Stefan; Koster, Linda; Blaise, Didier; Nicholson, Emma & Broers, A. E.C. [Vis alle 25 forfattere av denne artikkelen] (2025). Outcomes following different upfront stem cell transplantation strategies for multiple myeloma: a statistical perspective on behalf of the Chronic Malignancies Working Party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 60(10), s. 1361–1368. doi: 10.1038/s41409-025-02675-2. Fulltekst i vitenarkiv
• Skøyeneie, Sigrid Cecilie Tinglum; Wik, Hilde Skuterud; Tjønnfjord, Geir Erland; Fauske, Lena; Gedde-Dahl, Tobias & Solvang, Grethe [Vis alle 7 forfattere av denne artikkelen] (2025). Perceptions of safety in hospital at home following allogeneic haematopoietic stem cell transplantation: A qualitative study. European Journal of Oncology Nursing. ISSN 1462-3889. 80. doi: 10.1016/j.ejon.2025.103044. Fulltekst i vitenarkiv
• Güven, Mustafa; Peczynski, Christophe; Boreland, William; Blaise, Didier; Latour, Régis Peffault de & Yakoub-Agha, Ibrahim [Vis alle 29 forfattere av denne artikkelen] (2025). The impact of ABO compatibility on allogeneic hematopoietic cell transplantation outcomes: a contemporary and comprehensive study from the transplant complications working party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 60(7), s. 956–963. doi: 10.1038/s41409-025-02580-8. Fulltekst i vitenarkiv
• Prata, Pedro Henrique; Eikema, Dirk Jan; Piepenbroek, Brian; Kulasekararaj, Austin; Drexler, Beatrice & Tjon, Jennifer M.L. [Vis alle 25 forfattere av denne artikkelen] (2025). Hematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms: the EBMT Severe Aplastic Anemia Report. Haematologica. ISSN 0390-6078. 111(1), s. 168–176. doi: 10.3324/haematol.2024.287205. Fulltekst i vitenarkiv
• Nagler, Arnon; Kayser, Sarah; Ferhat, Allain Thibeault; Kröger, Nicolaus; Eder, Matthias & Socié, Gérard [Vis alle 21 forfattere av denne artikkelen] (2025). Non-T-Depleted Haploidentical Transplantation Compared to Allogeneic Transplantation From Matched Siblings or Unrelated Donors in Patients With Secondary AML in First Complete Remission: A Study From the ALWP/EBMT. American Journal of Hematology. ISSN 0361-8609. 101(2), s. 281–290. doi: 10.1002/ajh.70164. Fulltekst i vitenarkiv
• Vydra, Jan; Ferhat, Allain Thibeault; Kröger, Nicolaus; Gedde-Dahl, Tobias; Eder, Matthias & Schroeder, Thomas [Vis alle 19 forfattere av denne artikkelen] (2025). Posttransplant cyclophosphamide versus antithymocyte globulin in patients with cardiovascular comorbidity undergoing allogeneic hematopoietic cell transplantation for acute myeloid leukaemia in first complete remission from unrelated donors: a retrospective matched-pair analysis from the ALWP of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-025-02766-0. Fulltekst i vitenarkiv
• Busterud, Kari; Remberger, Mats; Bentsen, Pål Tore; Jerm, Marianne Brenn; Gedde-Dahl, Tobias & Kittang, Astrid Marta Olsnes [Vis alle 7 forfattere av denne artikkelen] (2025). Allogeneic stem cell transplantation for acute myeloid leukaemia 2005–22. Tidsskrift for Den norske legeforening (Tidsskriftet). ISSN 0029-2001. 145(12). doi: 10.4045/tidsskr.25.0001. Fulltekst i vitenarkiv Vis sammendrag

  Bakgrunn: Allogen stamcelletransplantasjon er den eneste kurative behandlingen ved en rekke blod- og benmargssykdommer. Formålet med denne undersøkelsen er å beskrive hvordan allogen stamcelletransplantasjon ved akutt myelogen leukemi benyttes i Norge og undersøke eventuelle ulikheter mellom de fire helseregionene. Materiale og metode: I perioden 1.1.2005 til 31.12.2022 ble 2 979 personer diagnostisert med akutt myelogen leukemi i Norge. Vi identifiserte alder, remisjonsstatus, donorkarakteristika, overlevelse og transplantasjonsrelatert dødelighet hos pasientene som ble behandlet med allogen stamcelletransplantasjon. Resultater: I studieperioden gjennomgikk 674/2 979 (22,6 %) av personene med påvist akutt myelogen leukemi allogen stamcelletransplantasjon. Det var et økende antall behandlinger over tid: fra 14 transplantasjoner i 2005 til 67 i 2020. Behandlingsresultatene ble progressivt bedre: femårs overlevelse økte fra 48 % i perioden 2005–10, til 63 % i perioden 2017–22. Median alder hos pasientene fra Helse Midt (48 år) og Helse Vest (50 år) var lavere enn hos pasientene fra Helse Sør-Øst (54 år) og Helse Nord (60 år). Det var ingen forskjeller mellom helseregionene når det gjaldt kjønn, remisjonsstatus og bruk av familiedonor og ubeslektet donor. Fortolkning: Allogen stamcelletransplantasjon er i dag en sentral del av behandlingen ved akutt myelogen leukemi. Stadig flere pasienter behandles med allogen stamcelletransplantasjon uten at behandlingsresultatene forringes.

• Esteve, Jordi; Nagler, Arnon; Labopin, Myriam; Versluis, Jurjen; Sanz, Jaime & Gedde-Dahl, Tobias [Vis alle 17 forfattere av denne artikkelen] (2025). Allogeneic Hematopoietic Cell Transplantation in Patients With Acute Myeloid Leukemia With Myelodysplasia-Related Genetic Features: Relevance of the Genetic Underlying Category. A Retrospective Analysis on Behalf of the Acute Leukemia Working Party of the EBMT. American Journal of Hematology. ISSN 0361-8609. 100(6), s. 954–962. doi: 10.1002/ajh.27647. Fulltekst i vitenarkiv
• Frøen, Hege; Brodtkorb, Marianne; Holte, Harald; Kolstad, Arne; Myhre, Anders Eivind & Remberger, Mats [Vis alle 8 forfattere av denne artikkelen] (2025). Allogen stamcelletransplantasjon for pasienter med lymfom 2011–21. Tidsskrift for Den norske legeforening (Tidsskriftet). ISSN 0029-2001. 145(3). doi: 10.4045/tidsskr.24.0443. Fulltekst i vitenarkiv
• Mishra, Vinod Kumar; Gedde-Dahl, Tobias; Remberger, Mats; Solvang, Grethe; Selvaag, Kristin Mill Lien & Fosseng, Arne [Vis alle 10 forfattere av denne artikkelen] (2025). Hospital at Home Following Allogeneic Hematopoietic Stem Cell Transplantation: An Economic Analysis. Healthcare. doi: 10.3390/healthcare13141648. Fulltekst i vitenarkiv Vis sammendrag

  Background: Advanced home care is becoming increasingly common for cancer patients and serves as a viable alternative to inpatient hospital care. The transition to home care is driven by both the rising costs of healthcare and evidence indicating better quality of care. This study aims to compare the costs of hospital-at-home treatment and in-hospital care for patients undergoing allo-HSCT. Methods: The cost analysis was conducted as a case–control study comparing the costs of allo-HSCT at home (HaH) to the costs of allo-HSCT for patients receiving in-hospital care (INH). The cost evaluation was conducted from the hospital’s perspective, which means that costs incurred outside the hospital setting were not included. Post-procedural costs for the first year after allo-HSCT included all readmissions and outpatient visits at Oslo University Hospital. Results: The cost for the peritransplant period could be reduced by up to 33% by treating allo-HSCT recipients at home instead of in the hospital. During the study period, 24% of the allo-HSCT recipients were treated at home, but our data from 2021 and 2022 indicate that at least a third of the patients scheduled for allo-HSCT are candidates for HaH. Conclusions: The findings demonstrate that patients in advanced home care experience significantly lower total costs compared to those receiving in-hospital treatment.

• Skøyeneie, Sigrid Cecilie Tinglum; Wik, Hilde Skuterud; Tjønnfjord, Geir Erland; Fauske, Lena; Gedde-Dahl, Tobias & Myhre, Anders Eivind (2025). Family Caregivers’ Experiences of Hospital at Home Following Allogeneic Haematopoietic Stem Cell Transplantation: A Qualitative Study. European Journal of Cancer Care. ISSN 0961-5423. 2025(1). doi: 10.1155/ecc/7043910. Fulltekst i vitenarkiv Vis sammendrag

  Purpose: The use of Hospital at Home (HaH) is expanding globally, including in the context of allogeneic haematopoietic stem cell transplantation (HSCT), as an innovative care model that delivers hospital-equivalent care to patients in a home setting. The HaH model designed for HSCT relies on informal caregivers, such as next of kin or friends. Given the limited research on caregivers’ perceptions of HaH, this study aimed to explore how informal caregivers of patients undergoing HSCT experienced HaH. Method: Using a phenomenological interpretive approach, semistructured individual interviews were conducted with 14 family caregivers. Data were analysed using reflexive thematic analysis. Results: The analysis identified three themes: (1) On the inside as a caregiver, (2) Variation in caregiver burden and (3) Appreciating normality. Caregivers valued the opportunity to be physically present and involved in caring for the patient during HSCT. Although most had to undertake an active caregiving role, they reported varying levels of involvement and distress. Caregivers also highlighted the sensation of normality that HaH fostered, emphasising that maintaining a familiar routine in the home setting was less exhausting than traditional hospital care. Conclusion: This study found that family caregivers appreciated being physically present in HaH, experiencing a sensation of being ‘on the inside’ and closely sharing the patient’s treatment journey, in contrast to during hospitalisation. The findings revealed diverse experiences of caregiving and associated distress, underscoring the importance of adequately preparing caregivers for their role and ensuring appropriate support.

• Giebel, Sebastian; Labopin, Myriam; Peric, Zinaida; Passweg, Jakob; Blaise, Didier & Salmenniemi, Urpu [Vis alle 19 forfattere av denne artikkelen] (2024). Impact of the Type of Tyrosine Kinase Inhibitor (imatinib or dasatinib) Used Before allo-HCT on Outcome of Patients with Philadelphia-Positive Acute Lymphoblastic Leukemia. A Study on Behalf of the Acute Leukemia Working Party of the EBMT. Transplantation and Cellular Therapy (TCT). ISSN 2666-6375. 31(1). doi: 10.1016/j.jtct.2024.07.016. Fulltekst i vitenarkiv
• Fageräng, Beatrice Linnea Christina; Cyranka, Leon; Schjalm, Camilla; Ekholt, Karin; Larsen, Carina Sandem & Heinzelbecker, Julia [Vis alle 14 forfattere av denne artikkelen] (2024). The function of the complement system remains fully intact throughout the course of allogeneic stem cell transplantation. Frontiers in Immunology. 15. doi: 10.3389/fimmu.2024.1422370. Fulltekst i vitenarkiv Vis sammendrag

  Introduction: Hematopoietic stem cell transplantation (HSCT) is associated with immune complications and endothelial dysfunction due to intricate donor-recipient interactions, conditioning regimens, and inflammatory responses. Methods: This study investigated the role of the complement system during HSCT and its interaction with the cytokine network. Seventeen acute myeloid leukemia patients undergoing HSCT were monitored, including blood sampling from the start of the conditioning regimen until four weeks post-transplant. Clinical follow-up was 200 days. Results: Total complement functional activity was measured by WIELISA and the degree of complement activation by ELISA measurement of sC5b-9. Cytokine release was measured using a 27-multiplex immuno-assay. At all time-points during HSCT complement functional activity remained comparable to healthy controls. Complement activation was continuously stable except for two patients demonstrating increased activation, consistent with severe endotheliopathy and infections. In vitro experiments with post-HSCT whole blood challenged with Escherichia coli, revealed a hyperinflammatory cytokine response with increased TNF, IL-1β, IL-6 and IL-8 formation. Complement C3 inhibition markedly reduced the cytokine response induced by Staphylococcus aureus, Aspergillus fumigatus, and cholesterol crystals. Discussion: In conclusion, HSCT patients generally retained a fully functional complement system, whereas activation occurred in patients with severe complications. The complement-cytokine interaction indicates the potential for new complement-targeting therapeutic strategies in HSCT.

• Ye, Yishan; Labopin, Myriam; Gérard, Socié; Yakoub-Agha, Ibrahim; Blau, Igor Wolfgang & Aljurf, Mahmoud [Vis alle 22 forfattere av denne artikkelen] (2024). Lower relapse incidence with haploidentical versus matched sibling or unrelated donor hematopoietic cell transplantation for core-binding factor AML patients in CR2: A study from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. American Journal of Hematology. ISSN 0361-8609. doi: 10.1002/ajh.27342. Fulltekst i vitenarkiv
• Chalandon, Yves; Eikema, Diderik-Jan; Moiseev, Ivan; Ciceri, Fabio; Koster, Linda & Vydra, Jan [Vis alle 23 forfattere av denne artikkelen] (2024). Unrelated donor transplantation with posttransplant cyclophosphamide vs ATG for myelodysplastic neoplasms. Blood Advances. ISSN 2473-9529. 8(18), s. 4792–4802. doi: 10.1182/bloodadvances.2024013468. Fulltekst i vitenarkiv
• Scheid, Christof; Eikema, Diderik-Jan; Gelder, Michel van; Salmenniemi, Urpu; Maertens, Johan & Passweg, Jakob [Vis alle 30 forfattere av denne artikkelen] (2024). Does IPSS-R downstaging before transplantation improve the prognosis of patients with myelodysplastic neoplasms? Blood. ISSN 0006-4971. doi: 10.1182/blood.2023022273. Fulltekst i vitenarkiv
• Grønvold, Bjørn Christer Linder; Ali, Maryan Mohamed; Myklebust, Tor Åge; Lenartova, Andrea; Remberger, Mats & Abrahamsen, Ingerid Weum [Vis alle 10 forfattere av denne artikkelen] (2024). Allogeneic stem cell transplant recipients surviving at least 2 years without relapse: outcome and risk factors. eJHaem. 5(1), s. 117–124. doi: 10.1002/jha2.842. Fulltekst i vitenarkiv Vis sammendrag

  Outcomes of 2-year survivours undergoing allo-haematopoietic stem cell transplantation at Oslo University Hospital were retrospectively assessed with the objectives of identification of risk factors for late death as possible means for precautionary measures and interventions to improve long-term survival. 421 patients with haematological malignancy, transplanted between 2005 and 2019, alive and free of disease after 2 years were included with data reported from The OUS-HSCT registry. Median follow-up was 6.2 years (2.016.1), and 232 patients (55%) were observed for minimum 5 years. The probability of being alive 5 and 10 years after HSCT was 86% and 76%. Primary risk factors for late death included initial diagnosis of age ≥ 60 years, chronic lymphocytic leukaemia (CLL), previous blood stream- or invasive fungal infection (BSI, IFI), and chronic graft-versus-host disease (cGVHD). Transplant-related mortality (TRM) and relapse at 5 years were 9.0% and 7.7%, respectively. Two factors were associated with the latter: cytomegalovirus (CMV) seronegative donor and CLL. Compared with the age- and gender-matched Norwegian general population, life expectancy was lower for each disease, except for CML. The prospect for the long-term survival is good for 2-year survivors of the allogeneic hematopoietic stem cell transplantation. However, life expectancy remains inferior to the age- and gender-matched general population. Optimising prophylaxis and treatment for chronic GVHD, BSI and IFI are needed along with the improved adherence to guidelines for early detection of secondary malignancies. Measures to improve immune reconstitution, possibly the microbiota, and the use of CMV seropositive donors regardless of recipient sero-status may be warranted and should be addressed in further studies.

• Mohty, Razan; Bazarbachi, Abdul Hamid; Labopin, Myriam; Esteve, Jordi; Kröger, Nicolaus & Cornelissen, Jan J. [Vis alle 23 forfattere av denne artikkelen] (2024). Isocitrate dehydrogenase (IDH) 1 and 2 mutations predict better outcome in patients with acute myeloid leukemia undergoing allogeneic hematopoietic cell transplantation: a study of the ALWP of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-024-02384-2. Fulltekst i vitenarkiv
• Moukalled, Nour; Labopin, Myriam; Versluis, Jurjen; Socié, Gérard; Blaise, Didier & Salmenniemi, Urpu [Vis alle 19 forfattere av denne artikkelen] (2024). Complex karyotype but not other cytogenetic abnormalities is associated with worse posttransplant survival of patients with nucleophosmin 1-mutated acute myeloid leukemia: A study from the European Society for Blood and Marrow Transplantation Acute Leukemia Working Party. American Journal of Hematology. ISSN 0361-8609. doi: 10.1002/ajh.27187. Fulltekst i vitenarkiv
• Styczynski, Jan; Tridello, Gloria; Knelange, Nina; Wendel, Lotus; Ljungman, Per & Mikulska, Malgorzata [Vis alle 35 forfattere av denne artikkelen] (2024). Adenovirus infections after allogeneic hematopoietic cell transplantation in children and adults: a study from the Infectious Diseases Working Party of the European Society for Blood and Marrow Transplantation. Bone Marrow Transplantation. ISSN 0268-3369. s. 1402–1412. doi: 10.1038/s41409-024-02361-9. Fulltekst i vitenarkiv
• Flygt, Hjalmar; Söderlund, Stina; Richter, Johan; Saussele, Susanne; Koskenvesa, Perttu & Stenke, Leif [Vis alle 20 forfattere av denne artikkelen] (2024). Treatment-free remission after a second TKI discontinuation attempt in patients with Chronic Myeloid Leukemia re-treated with dasatinib – interim results from the DAstop2 trial. Leukemia. ISSN 0887-6924. doi: 10.1038/s41375-024-02145-6. Fulltekst i vitenarkiv
• Brune, Mats; Kiss, Thomas; Anderson, Harald; Nicklasson, Malin; Delage, Robert & Finke, Jürgen [Vis alle 19 forfattere av denne artikkelen] (2024). Reduced Intensity transplantation vs chemotherapy in CR1. A prospective, pseudorandomized study in 50–70 year old AML patients. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-024-02408-x. Fulltekst i vitenarkiv
• Penack, Olaf; Abouqateb, Mouad; Peczynski, Christophe; Boreland, William; Kröger, Nicolaus & Stelljes, Matthias [Vis alle 18 forfattere av denne artikkelen] (2024). ATG or post-transplant cyclophosphamide to prevent GVHD in matched unrelated stem cell transplantation? Leukemia. ISSN 0887-6924. doi: 10.1038/s41375-024-02225-7. Fulltekst i vitenarkiv
• Hamed, Rama Al; Labopin, Myriam; Wu, Depei; Gedde-Dahl, Tobias; Aljurf, Mahmoud & Forcade, Edouard [Vis alle 23 forfattere av denne artikkelen] (2024). Allogeneic stem cell transplantation in de novo core-binding factor acute myeloid leukemia in first complete remission: data from the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-024-02373-5. Fulltekst i vitenarkiv
• McDonald, George B.; Landsverk, Ole J. B.; McGovern, Dermot P.B.; Aasebø, Anders Theodor Michaelsen; Paulsen, Vemund & Haritunians, Talin [Vis alle 14 forfattere av denne artikkelen] (2024). Allogeneic bone marrow transplantation for patients with treatment-refractory Crohn's Disease. Heliyon. 10:e24026(1), s. 1–10. doi: 10.1016/j.heliyon.2024.e24026. Fulltekst i vitenarkiv Vis sammendrag

  Background & aims Durable remissions of Crohn's Disease (CD) have followed myeloablative conditioning therapy and allogeneic marrow transplantation. For patients with treatment-refractory disease, we used reduced-intensity conditioning to minimize toxicity, marrow from donors with low Polygenic Risk Scores for CD as cell sources, and protracted immune suppression to lower the risk of graft-versus-host disease (GVHD). Our aim was to achieve durable CD remissions while minimizing transplant-related complications. Methods DNA from patients and their HLA-matched unrelated donors was genotyped and Polygenic Risk Scores calculated. Donor marrow was infused following non-myeloablative conditioning. Patient symptoms and endoscopic findings were documented at intervals after transplant. Results We screened 807 patients, 143 of whom met eligibility criteria; 2 patients received allografts. Patient 1 had multiple complications and died at day 332 from respiratory failure. Patient 2 had resolution of CD symptoms until day 178 when CD recurred, associated with persistent host chimerism in both peripheral blood and intestinal mucosa. Withdrawal of immune suppression was followed by dominant donor immune chimerism in peripheral blood and resolution of CD findings. Over time, mucosal T-cells became donor-dominant. At 5 years after allografting, Patient 2 remained off all medications but had mild symptoms related to a jejunal stricture that required stricturoplasty at 6 years. At 8 years, she remains stable off medications. Conclusions The kinetics of immunologic chimerism after allogeneic marrow transplantation for CD patients depends on the intensity of the conditioning regimen and the magnitude of immune suppression. One patient achieved durable improvement of her previously refractory CD only after establishing donor immunologic chimerism in intestinal mucosa. Her course provides proof-of-principal for allografting as a potential treatment for refractory CD, but an immunoablative conditioning regimen should be considered for future studies.

• Nagler, Arnon; Ngoya, Maud; Galimard, Jacques-Emmanuel; Labopin, Myriam; Blau, Igor Wolfgang & Kröger, Nicolaus [Vis alle 21 forfattere av denne artikkelen] (2024). Comparable relapse incidence after unrelated allogeneic stem cell transplantation with post-transplant cyclophosphamide versus conventional anti-graft versus host disease prophylaxis in patients with acute myeloid leukemia: A study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. American Journal of Hematology. ISSN 0361-8609. doi: 10.1002/ajh.27383. Fulltekst i vitenarkiv
• Arrieta-Bolaños, Esteban; Burg, Lars L. J. van der; Gedde-Dahl, Tobias; Robin, Marie; Salmenniemi, Urpu & Kröger, Nicolaus [Vis alle 20 forfattere av denne artikkelen] (2024). Directionality of HLA-DP permissive mismatches improves risk prediction in HCT for acute leukemia and MDS. Blood. ISSN 0006-4971. doi: 10.1182/blood.2024024351. Fulltekst i vitenarkiv
• Montoro, Juan; Eikema, Dirk-Jan; Tuffnell, Joe; Potter, Victoria; Kalwak, Krzysztof & Halkes, Constantijn J. M. [Vis alle 27 forfattere av denne artikkelen] (2024). Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT. Blood. ISSN 0006-4971. doi: 10.1182/blood.2024024173. Fulltekst i vitenarkiv
• Arrieta-Bolaños, Esteban; Bonneville, Edouard F.; Crivello, Pietro; Robin, Marie; Gedde-Dahl, Tobias & Salmenniemi, Urpu [Vis alle 26 forfattere av denne artikkelen] (2024). Human Leukocyte Antigen Mismatching and Survival in Contemporary Hematopoietic Cell Transplantation for Hematologic Malignancies. Journal of Clinical Oncology. ISSN 0732-183X. doi: 10.1200/JCO.24.00582. Fulltekst i vitenarkiv
• Nagler, Arnon; Labopin, Myriam; Swoboda, Ryszard; Schroeder, Thomas; Hamladji, Rose-Marie & Griskevicius, Laimonas [Vis alle 21 forfattere av denne artikkelen] (2024). Post-transplant cyclophosphamide, calcineurin inhibitor, and mycophenolate mofetil compared to anti-thymocyte globulin, calcineurin inhibitor, and methotrexate combinations as graft-versus-host disease prophylaxis post allogeneic stem cell transplantation from sibling and unrelated donors in patients with acute myeloid leukemia: a study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-024-02284-5. Fulltekst i vitenarkiv
• Loke, Justin; Labopin, Myriam; Craddock, Charles; Socié, Gérard; Gedde-Dahl, Tobias & Blaise, Didier [Vis alle 23 forfattere av denne artikkelen] (2024). Prognostic impact of number of induction courses to attain complete remission in patients with acute myeloid leukemia transplanted with either a matched sibling or human leucocyte antigen 10/10 or 9/10 unrelated donor: An Acute Leukemia Working Party European Society for Blood and Marrow Transplantation study. Cancer. ISSN 0008-543X. doi: 10.1002/cncr.35308. Fulltekst i vitenarkiv Vis sammendrag

  For the majority of patients with acute myeloid leukemia (AML) an allogeneic stem cell transplant (SCT) in first complete remission (CR) is preferred. However, whether the number of courses required to achieve CR has a prognostic impact is unclear. It is unknown which factors remain important in patients requiring more than one course of induction to attain remission. Methods This Acute Leukaemia Working Party study from the European Society for Blood and Marrow Transplantation identified adults who received an allograft in first CR from either a fully matched sibling or 10/10 or 9/10 human leucocyte antigen (HLA)-matched unrelated donor (HLA-A, HLA-B, HLA-C, HLA-DR, or HLA-DQ). Univariate and multivariate analyses were undertaken to identify the prognostic impact of one or two courses of induction to attain CR. Results A total of 4995 patients were included with 3839 (77%) patients attaining a CR following one course of induction chemotherapy (IND1), and 1116 patients requiring two courses (IND2) to attain CR. IND2 as compared to IND1 was a poor prognostic factor in a univariate analysis and remained so in a multivariate Cox model, resulting in an increased hazard ratio of relapse (1.38; 95% confidence interval [CI], 1.16–1.64; p = .0003) and of death (1.27; 95% CI, 1.09–1.47; p = .002). Adverse prognostic factors in a multivariate analysis of the outcomes of patients requiring IND2 included age, FLT3-ITD, adverse cytogenetics, and performance status. Pretransplant measurable residual disease retained a prognostic impact regardless of IND1 or IND2. Conclusion Initial response to chemotherapy as determined by number of courses to attain CR, retained prognostic relevance even following SCT in CR.

• Guièze, Romain; Eikema, Diderik-Jan; Koster, Linda; Schetelig, Johannes; Sengeloev, Henrik & Passweg, Jakob [Vis alle 26 forfattere av denne artikkelen] (2024). Allogeneic hematopoietic stem-cell transplantation for patients with Richter transformation: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-024-02256-9. Fulltekst i vitenarkiv
• Penack, Olaf; Abouqateb, Mouad; Peczynski, Christophe; Boreland, William; Gülbas, Zafer & Gedde-Dahl, Tobias [Vis alle 18 forfattere av denne artikkelen] (2024). PTCy versus ATG as graft-versus-host disease prophylaxis in mismatched unrelated stem cell transplantation. Blood Cancer Journal. 14(1). doi: 10.1038/s41408-024-01032-8. Fulltekst i vitenarkiv Vis sammendrag

  There is an increased risk of GVHD and of non-relapse mortality (NRM) after allogeneic stem cell transplantations (alloSCT) when mismatched unrelated donors (MMUD) are used. In Europe, it is standard practice to use rabbit anti-thymocyte globulin (rATG) to reduce the high NRM and GVHD risks after MMUD alloSCT. As an alternative to rATG, post-transplantation Cyclophosphamide (PTCy) is in increasing clinical use. It is currently impossible to give general recommendations regarding preference for one method over another since comparative evidence from larger data sets is lacking. To improve the evidence base, we analyzed the outcome of rATG vs. PTCy prophylaxis in adult patients with hematologic malignancies undergoing first peripheral blood alloSCT from MMUD (9/10 antigen match) between Jan 2018 and June 2021 in the database of the European Society for Blood and Marrow Transplantation (EBMT). We performed multivariate analyses using the Cox proportional-hazards regression model. We included 2123 patients in the final analyses (PTCy, n = 583; rATG, n = 1540). p values and hazard ratios (HR) presented here are multivariate outcomes. Two years after alloSCT we found a lower NRM in the PTCy group of 18% vs. 24.9% in the rATG group; p = 0.028, HR 0.74. Overall survival in the PTCy cohort was higher with 65.7% vs. 55.7% in the rATG cohort; p < 0.001, HR 0.77. Progression-free survival was also better in the PTCy patients with 59.1% vs. 48.8% when using rATG; p = 0.001, 0.78. The incidences of chronic GVHD and acute GVHD were not significantly different between the groups. We found significantly lower NRM as well as higher survival in recipients of peripheral blood alloSCTs from MMUD receiving PTCy as compared to rATG. The results of the current analysis suggest an added value of PTCy as GVHD prophylaxis in MMUD alloSCT.

• Holland, Petter; Istre, Mette Stausland; Ali, Maryan Mohamed; Gedde-Dahl, Tobias; Buechner, Jochen & Wildhagen, Mari [Vis alle 12 forfattere av denne artikkelen] (2024). Epigenetic aging of human blood cells is influenced by the age of the host body. Aging Cell. ISSN 1474-9718. 23(5), s. 1–5. doi: 10.1111/acel.14112. Fulltekst i vitenarkiv
• Nagler, Arnon; Labopin, Myriam; Mielke, Stephan; Passweg, Jakob; Blaise, Didier & Gedde-Dahl, Tobias [Vis alle 20 forfattere av denne artikkelen] (2023). Matched related versus unrelated versus haploidentical donors for allogeneic transplantation in AML patients achieving first complete remission after two induction courses: a study from the ALWP/EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-01980-y. Fulltekst i vitenarkiv
• Spyridonidis, Alexandros; Labopin, Myriam; Gedde-Dahl, Tobias; Ganser, Arnold; Stelljes, Matthias & Craddock, Charles [Vis alle 30 forfattere av denne artikkelen] (2023). Validation of the transplant conditioning intensity (TCI) index for allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-02139-5. Fulltekst i vitenarkiv Vis sammendrag

  Abstract The intensity of the conditioning regimen given before allogeneic hematopoietic cell transplantation (allo-HCT) can vary substantially. To confirm the ability of the recently developed transplant conditioning intensity (TCI) score to stratify the preparative regimens of allo-HCT, we used an independent and contemporary patient cohort of 4060 transplant recipients with acute myeloid leukemia meeting inclusion criteria from the discovery study (allo-HCT in first complete remission, matched donor), but who were allografted in a more recent period (2018–2021) and were one decade older (55–75 years, median 63.4 years), we assigned them to a TCI category (low n  = 1934, 48%; intermediate n  = 1948, 48%, high n  = 178, 4%) according to the calculated TCI score ([1–2], [2.5–3.5], [4–6], respectively), and examined the validity of the TCI category in predicting early non-relapse mortality (NRM), 2-year NRM and relapse (REL). In the unadjusted comparison, the TCI index provided a significant risk stratification for d100 and d180 NRM, NRM and REL risk. In the multivariate analysis adjusted for significant variables, there was an independent association of TCI with early NRM, NRM and REL. In summary, we confirm in contemporary treated patients that TCI reflects the conditioning regimen related morbidity and anti-leukemic efficacy satisfactorily and across other established prognostic factors.

• Giannakopoulou, Eirini Chrysovalantou; Lehander, Madeleine; Culleton, Stina Virding; Yang, Weiwen; Li, Yingqian & Kärpänen, Terhi Hannele [Vis alle 29 forfattere av denne artikkelen] (2023). A T cell receptor targeting a recurrent driver mutation in FLT3 mediates elimination of primary human acute myeloid leukemia in vivo. Nature Cancer. 4(10). doi: 10.1038/s43018-023-00642-8. Fulltekst i vitenarkiv Vis sammendrag

  Abstract Acute myeloid leukemia (AML), the most frequent leukemia in adults, is driven by recurrent somatically acquired genetic lesions in a restricted number of genes. Treatment with tyrosine kinase inhibitors has demonstrated that targeting of prevalent FMS-related receptor tyrosine kinase 3 (FLT3) gain-of-function mutations can provide significant survival benefits for patients, although the efficacy of FLT3 inhibitors in eliminating FLT3-mutated clones is variable. We identified a T cell receptor (TCR) reactive to the recurrent D835Y driver mutation in the FLT3 tyrosine kinase domain (TCR FLT3D/Y ). TCR FLT3D/Y -redirected T cells selectively eliminated primary human AML cells harboring the FLT3 D835Y mutation in vitro and in vivo. TCR FLT3D/Y cells rejected both CD34 + and CD34 − AML in mice engrafted with primary leukemia from patients, reaching minimal residual disease-negative levels, and eliminated primary CD34 + AML leukemia-propagating cells in vivo. Thus, T cells targeting a single shared mutation can provide efficient immunotherapy toward selective elimination of clonally involved primary AML cells in vivo.

• Nagler, Arnon; Labopin, Myriam; Kröger, Nicolaus; Schroeder, Thomas; Gedde-Dahl, Tobias & Eder, Matthias [Vis alle 14 forfattere av denne artikkelen] (2023). The role of anti-thymocyte globulin in allogeneic stem cell transplantation (HSCT) from HLA-matched unrelated donors (MUD) for secondary AML in remission: a study from the ALWP /EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-02095-0. Fulltekst i vitenarkiv
• Hernández-Boluda, Juan Carlos; Eikema, Diderik-Jan; Koster, Linda; Kröger, Nicolaus; Robin, Marie & Witte, Moniek de [Vis alle 26 forfattere av denne artikkelen] (2023). Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: a study of the Chronic Malignancies Working Party of EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-02094-1. Fulltekst i vitenarkiv
• Geelen, Inge G.P.; Gullaksen, Stein-Erik; Ilander, Mette M.; Olssen-Strömberg, Ulla; Mustjoki, Satu & Richter, Johan [Vis alle 15 forfattere av denne artikkelen] (2023). Switching from imatinib to nilotinib plus pegylated interferon-α2b in chronic phase CML failing to achieve deep molecular response: clinical and immunological effects. Annals of Hematology. ISSN 0939-5555. doi: 10.1007/s00277-023-05199-1. Fulltekst i vitenarkiv
• Vo, Camilla Dao; Myhre, Anders Eivind; Abrahamsen, Ingerid Weum; Remberger, Mats; Mattsson, Jonas & Fløisand, Yngvar [Vis alle 10 forfattere av denne artikkelen] (2023). Allogen stamcelletransplantasjon hos voksne 2015–21. Tidsskrift for Den norske legeforening (Tidsskriftet). ISSN 0029-2001. 143(4). doi: 10.4045/tidsskr.22.0521. Fulltekst i vitenarkiv Vis sammendrag

  BAKGRUNN Allogen stamcelletransplantasjon er eneste kurative behandling for flere maligne og ikke-maligne blodsykdommer, og er assosiert med risiko for alvorlige komplikasjoner. De siste årene er det innført flere endringer med mål om å redusere behandlingsrelaterte komplikasjoner. Denne retrospektive studien gjennomgår kvalitetsindikatorer for pasienter som gjennomgikk transplantasjon i perioden 2015–21. MATERIALE OG METODE Studien inkluderte 589 voksne pasienter som ble behandlet med allogen stamcelletransplantasjon for første gang ved Oslo universitetssykehus fra mai 2015 til mai 2021. Tre toårsperioder er sammenliknet med deskriptive metoder. RESULTATER Fra 2015 til 2021 økte antall årlige førstegangstransplantasjoner fra 85 til 113. Ettårsoverlevelse økte fra 68 % i første toårsperiode til 74 % i andre periode og til 82 % i siste periode. Det var en reduksjon i både akutt og kronisk transplantat-mot-vert-sykdom, og andelen pasienter i live ett år etter transplantasjonen uten transplantat-mot-vert-sykdom eller residiv økte fra 42 % til 60 % i studieperioden. FORTOLKNING Siden 2015 er antall transplantasjoner økt samtidig som overlevelsen er forbedret og risikoen for komplikasjoner lavere.

• Poiré, Xavier; Labopin, Myriam; Polge, Emmanuelle; Ganser, Arnold; Socié, Gérard & Gedde-Dahl, Tobias [Vis alle 17 forfattere av denne artikkelen] (2023). Allogeneic hematopoietic cell transplantation for acute myeloid leukemia with hyperdiploid complex karyotype. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-02167-1. Fulltekst i vitenarkiv
• Hamed, Rama Al; Ngoya, Maud; Galimard, Jacques-Emmanuel; Sengeloev, Henrik; Gedde-Dahl, Tobias & Kulagin, Aleksandr [Vis alle 18 forfattere av denne artikkelen] (2023). Unrelated or haploidentical allogeneic hematopoietic cell transplantation in second complete remission for acute myeloid leukemia—Improved outcomes over time: A European Society for Blood and Marrow Transplantation Acute Leukemia Working Party study. Cancer. ISSN 0008-543X. doi: 10.1002/cncr.34843. Fulltekst i vitenarkiv
• Gjærde, Lars Klingen; Ruutu, Tapani; Peczynski, Christophe; Boreland, William; Kröger, Nicolaus & Blaise, Didier [Vis alle 20 forfattere av denne artikkelen] (2023). The impact of pre-transplantation diabetes and obesity on acute graft-versus-host disease, relapse and death after allogeneic hematopoietic cell transplantation: a study from the EBMT Transplant Complications Working Party. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-02154-6. Fulltekst i vitenarkiv Vis sammendrag

  Abstract Obesity and diabetes can modulate immune responses, which may impact allogeneic HCT outcomes and GvHD. From the EBMT registry, we included 36,539 adult patients who underwent allogeneic HCT for a hematological malignancy between 2016 and 2020. Of these, 5228 (14%) had obesity (BMI ≥ 30 kg/m 2 ), 1415 (4%) had diabetes (requiring treatment with insulin or oral hypoglycemics), and 688 (2%) had obesity + diabetes pre-transplantation. Compared with patients without diabetes or obesity, the hazard ratio (HR) of grade II–IV acute GvHD was 1.00 (95% confidence interval [CI] 0.94–1.06, p  = 0.89) for patients with obesity, 0.95 (CI 0.85–1.07, p  = 0.43) for patients with diabetes, and 0.96 (CI 0.82–1.13, p  = 0.63) for patients with obesity + diabetes. Non-relapse mortality was higher in patients with obesity (HR 1.08, CI 1.00–1.17, p  = 0.047), diabetes (HR 1.40, CI 1.24–1.57, p  &lt; 0.001), and obesity + diabetes (HR 1.38, CI 1.16–1.64, p  &lt; 0.001). Overall survival after grade II–IV acute GvHD was lower in patients with diabetes (HR 1.46, CI 1.25–1.70, p  &lt; 0.001). Pre-transplantation diabetes and obesity did not influence the risk of developing acute GvHD, but pre-transplantation diabetes was associated with poorer survival after acute GvHD.

• Sanz, Jaime; Labopin, Myriam; Pabst, Thomas; Versluis, Jurjen; Gorkom, Gwendolyn Van & Meijer, Ellen [Vis alle 17 forfattere av denne artikkelen] (2023). Etoposide plus cytarabine versus cyclophosphamide or melphalan in busulfan-based preparative regimens for autologous stem cell transplantation in adults with acute myeloid leukemia in first complete remission: a study from the Acute Leukemia Working Party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-023-02075-4. Fulltekst i vitenarkiv
• Ali, Maryan Mohamed; Gedde-Dahl, Tobias; Osnes, Liv T. N.; Perrier, Flavie; Veierød, Marit Bragelien & Tjønnfjord, Geir Erland [Vis alle 7 forfattere av denne artikkelen] (2023). Extracorporeal Photopheresis as Graft-versus-Host Disease Prophylaxis: A Randomized Controlled Trial. Transplantation and Cellular Therapy (TCT). ISSN 2666-6375. 29(6), s. 364.e1–364.e11. doi: 10.1016/j.jtct.2023.02.023. Fulltekst i vitenarkiv Vis sammendrag

  Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the sole curative option for many patients diagnosed with hematologic malignancies. A major obstacle is graft-versus-host disease (GVHD), causing significant morbidity and mortality. Extracorporeal photopheresis (ECP) is an increasingly applied treatment for GVHD, owing in part to its favorable safety profile. In contrast, reports on the use of ECP to prevent GVHD are rare, and randomized controlled trials (RCTs) are lacking. We conducted an RCT to assess whether ECP applied post-transplantation could prevent the development of GVHD within the first year of transplantation. We enrolled 157 patients (age 18 to 74 years) with a hematologic malignancy undergoing their first allo-HSCT, randomized as 76 to the intervention group and 81 to the control group. ECP was initiated directly on engraftment and was planned twice weekly for 2 weeks, then once weekly for 4 weeks. GVHD, relapse, and death were analyzed by Cox regression analysis. During the first year, 45 patients in the intervention group and 52 control patients developed GVHD (hazard ratio [HR], .82; 95% confidence interval [CI], .55 to 1.22; P = .32). There were no differences in acute or chronic GVHD or its organ distribution in this intention-to-treat RCT. A per-protocol analysis revealed a significant difference in GVHD between the intervention group (per-protocol; n = 39 of 76) and the control group (n = 77), 46% versus 68%, respectively (HR, .47; 95% CI, .27 to .80; P = .006). Relapse occurred in 15 patients in the intervention group and in 11 control patients (HR, 1.38; 95% CI, .64 to 3.01; P = .42). GVHD-free relapse-free survival, event-free survival, overall survival, and nonrelapse mortality did not differ significantly between the 2 study groups. There also was no significant difference in immune reconstitution between the 2 groups. This first intention-to-treat RCT investigating ECP as GVHD prophylaxis in allo-HSCT for hematologic malignancy does not support the use of ECP as an adjunct to standard drug-based GVHD prophylaxis.

• Bazarbachi, Ali; Labopin, Myriam; Gedde-Dahl, Tobias; Remenyi, Peter; Forcade, Edouard & Kröger, Nicolaus [Vis alle 18 forfattere av denne artikkelen] (2023). Improved Posttransplant Outcomes in Recent Years for AML Patients with FLT3-ITD and Wild-type NPM1: A Report from the EBMT Acute Leukemia Working Party. Clinical Cancer Research. ISSN 1078-0432. 29(21), s. 4441–4448. doi: 10.1158/1078-0432.CCR-23-0954. Fulltekst i vitenarkiv
• Federico, Lorenzo; Tvedt, Tor Henrik Anderson; Gainullin, Murat; Osen, Julie Røkke; Chaban, Viktoriia & Lund, Kathrine Persgård [Vis alle 19 forfattere av denne artikkelen] (2023). Robust spike-specific CD4⁺ and CD8⁺ T cell responses in SARS-CoV-2 vaccinated hematopoietic cell transplantation recipients: a prospective, cohort study. Frontiers in Immunology. 14. doi: 10.3389/fimmu.2023.1210899. Fulltekst i vitenarkiv Vis sammendrag

  Poor overall survival of hematopoietic stem cell transplantation (HSCT) recipients who developed COVID-19 underlies the importance of SARS-CoV-2 vaccination. Previous studies of vaccine efficacy have reported weak humoral responses but conflicting results on T cell immunity. Here, we have examined the relationship between humoral and T cell response in 48 HSCT recipients who received two doses of Moderna’s mRNA-1273 or Pfizer/BioNTech’s BNT162b2 vaccines. Nearly all HSCT patients had robust T cell immunity regardless of protective humoral responses, with 18/48 (37%, IQR 8.679-5601 BAU/mL) displaying protective IgG anti-receptor binding domain (RBD) levels (&amp;gt;2000 BAU/mL). Flow cytometry analysis of activation induced markers (AIMs) revealed that 90% and 74% of HSCT patients showed reactivity towards immunodominant spike peptides in CD8 + and CD4 + T cells, respectively. The response rate increased to 90% for CD4 + T cells as well when we challenged the cells with a complete set of overlapping peptides spanning the entire spike protein. T cell response was detectable as early as 3 months after transplant, but only CD4 + T cell reactivity correlated with IgG anti-RBD level and time after transplantation. Boosting increased seroconversion rate, while only one patient developed COVID-19 requiring hospitalization. Our data suggest that HSCT recipients with poor serological responses were protected from severe COVID-19 by vaccine-induced T cell responses.

• Garcia-Horton, Alejandro; Cyriac, Sunu L.; Gedde-Dahl, Tobias; Fløisand, Yngvar; Remberger, Mats & Mattsson, Jonas [Vis alle 7 forfattere av denne artikkelen] (2022). Patient age and donor HLA matching can stratify allogeneic hematopoietic cell transplantation patients into prognostic groups. European Journal of Haematology. ISSN 0902-4441. 109(6), s. 672–679. doi: 10.1111/ejh.13850. Fulltekst i vitenarkiv
• Nagler, Arnon; Galimard, Jacques-Emmanuel; Labopin, Myriam; Blaise, Didier; Arcese, William & Trisolini, Silvia Maria [Vis alle 15 forfattere av denne artikkelen] (2022). Autologous stem cell transplantation (ASCT) for acute myeloid leukemia in patients in first complete remission after one versus two induction courses: A study from the ALWP of the EBMT. Cancer Medicine. doi: 10.1002/cam4.5039. Fulltekst i vitenarkiv Vis sammendrag

  Background Achieving complete remission (CR) is the main goal in AML treatment and a prerequisite for successful autologous stem cell transplantation (ACT). Methods Comparing results of peripheral blood ACT in patients with AML in CR1 attained following 1 versus 2 chemotherapy courses transplanted in 2000–2019. Results Patients 1532 (84%) with one and 293 (16%) patients with two induction chemotherapies courses (a total of 1825 patients) were included in the study. Follow-up was 7.9 (95% CI: 7.4–8.4) and 7.7 (95% CI: 7.0–8.6) years (p = 0.8). Time from diagnosis to ACT was 4.7 (range, 3.9–5.8) versus 5.7 (range, 4.7–7.1) months (p < 0.001), respectively. Leukemia free survival (LFS) and overall survival (OS) at 5 years were inferior for patients achieving CR1 with 2 versus 1 course of chemotherapy: 26.6% versus 41.7% (HR = 1.42 [95% CI: 1.22–1.66], p < 0.001) and 36.2% versus 53.3%, (HR = 1.48 [95% CI: 1.25–1.75], p < 0.001), and 5-year relapse incidence (RI) was higher: 67.2% versus 52.3%, (HR = 1.46 [95% CI: 1.25–1.72], p < 0.001). Five-year non-relapse mortality (NRM) was 6.2% versus 6.0% for patients with 2 versus 1 chemotherapy courses, and did not differ significantly (HR = 1.31 [95% CI: 0.81–2.10], p = 0.27). Conclusions LFS and OS were inferior and relapse rate was higher in AML patients who received two inductions chemotherapy courses to reach CR1 before being autografted. AML patients who required 2 induction courses to achieve remission, may be offered allogeneic transplantation rather than an autologous one in an attempt to reduce their high RI and improve outcomes.

• Nagler, Arnon; Ngoya, Maud; Jacques-Emmanuel, Galimard; Labopin, Myriam; Kröger, Nicolaus & Socié, Gerard [Vis alle 19 forfattere av denne artikkelen] (2022). Trends in outcome of transplantation in patients with secondary acute myeloid leukemia: an analysis from the Acute Leukemia Working Party (ALWP) of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-022-01825-0. Fulltekst i vitenarkiv
• Albert, Michael H.; Sirlait, Tiarlan; Eikema, Dirk-Jan; Bakunina, Katerina; Wehr, Claudia & Suarez, Felipe [Vis alle 51 forfattere av denne artikkelen] (2022). Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study. Blood. ISSN 0006-4971. 140. doi: 10.1182/blood.2022015506. Fulltekst i vitenarkiv
• Swan, Dawn; Hayden, Patrick J.; Eikema, Dirk-Jan; Koster, Linda; Sauer, Sandra & Blaise, Didier [Vis alle 25 forfattere av denne artikkelen] (2022). Trends in autologous stem cell transplantation for newly diagnosed multiple myeloma: Changing demographics and outcomes in European Society for Blood and Marrow Transplantation centres from 1995 to 2019. British Journal of Haematology. ISSN 0007-1048. 197(1), s. 82–96. doi: 10.1111/bjh.18025. Fulltekst i vitenarkiv
• Nagler, Arnon; Labopin, Myriam; Craddock, Charles; Socié, Gerard; Yakoub-Agha, Ibrahim & Gedde-Dahl, Tobias [Vis alle 15 forfattere av denne artikkelen] (2022). Cytogenetic risk classification maintains its prognostic significance in transplanted FLT3-ITD mutated acute myeloid leukemia patients: On behalf of the acute leukemia working party/European society of blood and marrow transplantation. American Journal of Hematology. ISSN 0361-8609. 97(3), s. 274–282. doi: 10.1002/ajh.26442. Fulltekst i vitenarkiv
• Tvedt, Tor Henrik Anderson; Rose-John, Stefan; Tsykunova, Galina; Ahmed, Aymen B; Gedde-Dahl, Tobias & Ersvær, Elisabeth [Vis alle 7 forfattere av denne artikkelen] (2022). IL-6 Responsiveness of CD4⁺ and CD8⁺ T Cells after Allogeneic Stem Cell Transplantation Differs between Patients and Is Associated with Previous Acute Graft versus Host Disease and Pretransplant Antithymocyte Globulin Therapy. Journal of Clinical Medicine. 11(9). doi: 10.3390/jcm11092530. Fulltekst i vitenarkiv Vis sammendrag

  Graft-versus-host disease (GVHD), one of the most common and serious complications after allogeneic stem cell transplantation, is mediated by allocative T cells. IL-6 mediates both pro- and anti-inflammatory effects and modulates T cell response through classical signaling and trans-signaling. We investigated the effects on the mTOR and JAK/STAT pathways after various types of IL-6 signaling for circulating T cells were derived from 31 allotransplant recipients 90 days post-transplant. Cells were stimulated with IL-6 alone, hyper-IL-6 (trans-signaling), IL-6+IL-6 receptor (IL-6R; classical + trans-signaling) and IL-6+IL-6R+soluble gp130-Fc (classical signaling), and flow cytometry was used to investigate the effects on phosphorylation of AKT (Thr308), mTOR (Ser2442), STAT3 (Ser727) and STAT3 (Tyr705). CD3+CD4+ and CD3+C8+ T cells responded to classical and trans IL-6 stimulation with increased STAT3 (Tyr705) phosphorylation; these responses were generally stronger for CD3+CD4+ cells. STAT3 (Tyr705) responses were stronger for patients with previous acute GVHD; CD3+CD4+ cells from GVHD patients showed an additional STAT3 (Ser727) response, whereas patients without acute GVHD showed additional mTOR (Ser2448) responses. Furthermore, treatment with antithymocyte globulin as a part of GVHD prophylaxis was associated with generally weaker STAT3 (Tyr705) responses and altered STAT3 (Ser727) responsiveness of CD3+CD4+ cells together with increased mTOR (Ser2448) responses for the CD3+CD8+ cells. Thus, early post-transplant CD3+CD4+ and CD3+ CD8+ T cell subsets differ in their IL-6 responsiveness; this responsiveness is modulated by antithymocyte globulin and differs between patients with and without previous acute GVHD. These observations suggest that allotransplant recipients will be heterogeneous with regard to the effects of post-transplant IL-6 targeting.

• Huuhtanen, Jani; Ilander, Mette; Yadav, Bhagwan; Dufva, Olli M.J.; Lähteenmäki, Hanna & Kasanen, Tiina [Vis alle 26 forfattere av denne artikkelen] (2022). IFN-α with dasatinib broadens the immune repertoire in patients with chronic-phase chronic myeloid leukemia. Journal of Clinical Investigation. ISSN 0021-9738. 132(17). doi: 10.1172/JCI152585. Fulltekst i vitenarkiv Vis sammendrag

  In chronic myeloid leukemia (CML), combination therapies with tyrosine kinase inhibitors (TKIs) aim to improve the achievement of deep molecular remission that would allow therapy discontinuation. IFN-α is one promising candidate, as it has long-lasting effects on both malignant and immune cells. In connection with a multicenter clinical trial combining dasatinib with IFN-α in 40 patients with chronic-phase CML (NordCML007, NCT01725204), we performed immune monitoring with single-cell RNA and T cell receptor (TCR) sequencing (n = 4, 12 samples), bulk TCRβ sequencing (n = 13, 26 samples), flow cytometry (n = 40, 106 samples), cytokine analyses (n = 17, 80 samples), and ex vivo functional studies (n = 39, 80 samples). Dasatinib drove the immune repertoire toward terminally differentiated NK and CD8+ T cells with dampened functional capabilities. Patients with dasatinib-associated pleural effusions had increased numbers of CD8+ recently activated effector memory T (Temra) cells. In vitro, dasatinib prevented CD3-induced cell death by blocking TCR signaling. The addition of IFN-α reversed the terminally differentiated phenotypes and increased the number of costimulatory intercellular interactions and the number of unique putative epitope-specific TCR clusters. In vitro IFN-α had costimulatory effects on TCR signaling. Our work supports the combination of IFN-α with TKI therapy, as IFN-α broadens the immune repertoire and restores immunological function.

• Aasebø, Anders Theodor Michaelsen; Gedde-Dahl, Tobias; Reims, Henrik Mikael; Bækkevold, Espen Sønderaal & Jahnsen, Frode Lars (2022). Calprotectin Expressing Donor-Derived Macrophages Increase in Acute Gastrointestinal Graft-Versus-Host Disease. Transplantation and Cellular Therapy (TCT). ISSN 2666-6375. 28(5), s. 248.e1–248.e8. doi: 10.1016/j.jtct.2022.01.028. Fulltekst i vitenarkiv
• Kharfan-Dabaja, Mohamed A.; Labopin, Myriam; Bazarbachi, Ali; Ciceri, Fabio; Finke, J. & Bruno, Benedetto [Vis alle 29 forfattere av denne artikkelen] (2021). Comparing outcomes of a second allogeneic hematopoietic cell transplant using HLA-matched unrelated versus T-cell replete haploidentical donors in relapsed acute lymphoblastic leukemia: a study of the Acute Leukemia Working Party of EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 56(9), s. 2194–2202. doi: 10.1038/s41409-021-01317-7. Fulltekst i vitenarkiv
• Nagler, Arnon; Labopin, Myriam; Dholaria, Bhagirathbhai; Wu, Depei; Choi, Goda & Aljurf, Mahmoud [Vis alle 24 forfattere av denne artikkelen] (2021). Graft-versus-Host Disease Prophylaxis with Post-Transplantation Cyclophosphamide versus Cyclosporine A and Methotrexate in Matched Sibling Donor Transplantation. Transplantation and Cellular Therapy (TCT). ISSN 2666-6375. doi: 10.1016/j.jtct.2021.11.013. Fulltekst i vitenarkiv
• Ali, Maryan Mohamed; Grønvold, Bjørn Christer Linder; Remberger, Mats; Abrahamsen, Ingerid Weum; Myhre, Anders Eivind & Tjønnfjord, Geir Erland [Vis alle 8 forfattere av denne artikkelen] (2021). Addition of Anti-thymocyte Globulin in Allogeneic Stem Cell Transplantation With Peripheral Stem Cells From Matched Unrelated Donors Improves Graft-Versus-Host Disease and Relapse Free Survival. Clinical Lymphoma, Myeloma & Leukemia. ISSN 2152-2650. 21(9), s. 598–605. doi: 10.1016/j.clml.2021.05.003. Fulltekst i vitenarkiv Vis sammendrag

  Anti-thymocyte globulin (ATG) is commonly used to prevent graft-versus-host disease (GvHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). To evaluate the impact of ATG as part of the GvHD prophylaxis in our institution, we report the outcome of 415 patients with matched unrelated donors (MUD) transplanted for hematological malignancies with or without ATG from 2005 to 2019 at Oslo University Hospital, Norway. The following groups were compared: (1) 154 patients transplanted with peripheral blood stem cells (PBSC) without ATG 2005-2014. (2) 137 patients transplanted with bone marrow stem cells (BMSC) 2005-2019. (3) 124 patients transplanted with PBSC and ATG (PBSC + ATG) 2014-2019. Three years survival was similar in the groups, 61% following allografting with PBSC, 54% with BMSC, and 59% with PBSC + ATG. Acute GvHD grade III-IV was 14%, 14%, and 7%; chronic GvHD was 81%, 32, and 26%; and extensive cGvHD 44%, 15%, and 6% in the corresponding groups. Both acute and chronic GvHD were significantly reduced in the PBSC + ATG-versus the PBSC group (p < 0.05 and p < 0.001 respectively).Transplant-related mortality (TRM) was 33%, 25%, and 17% (p = 0.18). Graft versus host disease and relapse free survival (GRFS) at 3 years was 43 %, 43%, and 64% in the groups. Adding ATG to the GvHD prophylaxis regimen of MUD allo-HSCT with PBSC resulted in a substantial reduction of both acute and chronic GvHD without compromising the disease control, reflected in a superior 3 years GRFS.

• Richter, Johan; Lübking, Anna; Söderlund, Stina; Lotfi, Kourosh; Markevärn, Berit & Själander, Anders [Vis alle 19 forfattere av denne artikkelen] (2021). Molecular status 36 months after TKI discontinuation in CML is highly predictive for subsequent loss of MMR—final report from AFTER-SKI. Leukemia. ISSN 0887-6924. 35, s. 2416–2418. doi: 10.1038/s41375-021-01173-w. Fulltekst i vitenarkiv
• Jørgensen, Silje Fjellgård; Buechner, Jochen; Myhre, Anders Eivind; Galteland, Eivind; Spetalen, Signe & Kulseth, Mari Ann [Vis alle 19 forfattere av denne artikkelen] (2021). A Nationwide Study of GATA2 Deficiency in Norway-the Majority of Patients Have Undergone Allo-HSCT. Journal of Clinical Immunology. ISSN 0271-9142. doi: 10.1007/s10875-021-01189-y. Fulltekst i vitenarkiv Vis sammendrag

  Purpose: GATA2 deficiency is a rare primary immunodeficiency that has become increasingly recognized due to improved molecular diagnostics and clinical awareness. The only cure for GATA2 deficiency is allogeneic hematopoietic stem cell transplantation (allo-HSCT). The inconsistency of genotype-phenotype correlations makes the decision regarding "who and when" to transplant challenging. Despite considerable morbidity and mortality, the reported proportion of patients with GATA2 deficiency that has undergone allo-HSCT is low (~ 35%). The purpose of this study was to explore if detailed clinical, genetic, and bone marrow characteristics could predict end-point outcome, i.e., death and allo-HSCT. Methods: All medical genetics departments in Norway were contacted to identify GATA2 deficient individuals. Clinical information, genetic variants, treatment, and outcome were subsequently retrieved from the patients' medical records. Results: Between 2013 and 2020, we identified 10 index cases or probands, four additional symptomatic patients, and no asymptomatic patients with germline GATA2 variants. These patients had a diverse clinical phenotype dominated by cytopenia (13/14), myeloid neoplasia (10/14), warts (8/14), and hearing loss (7/14). No valid genotype-phenotype correlations were found in our data set, and the phenotypes varied also within families. We found that 11/14 patients (79%), with known GATA2 deficiency, had already undergone allo-HSCT. In addition, one patient is awaiting allo-HSCT. The indications to perform allo-HSCT were myeloid neoplasia, disseminated viral infection, severe obliterating bronchiolitis, and/or HPV-associated in situ carcinoma. Two patients died, 8 months and 7 years after allo-HSCT, respectively. Conclusion: Our main conclusion is that the majority of patients with symptomatic GATA2 deficiency will need allo-HSCT, and a close surveillance of these patients is important to find the "optimal window" for allo-HSCT. We advocate a more offensive approach to allo-HSCT than previously described.

• Flygt, Hjalmar; Söderlund, Stina; Stentoft, Jesper; Richter, Johan; Koskenvesa, Perttu & Mustjoki, Satu [Vis alle 18 forfattere av denne artikkelen] (2021). Long-term tolerability and efficacy after initial PegIFN-α addition to dasatinib in CML-CP: Five-year follow-up of the NordCML007 study. European Journal of Haematology. ISSN 0902-4441. s. 1–7. doi: 10.1111/ejh.13699. Fulltekst i vitenarkiv Vis sammendrag

  Objectives Treatment-free remission (TFR) has emerged as a treatment goal in chronic myeloid leukemia in the chronic phase (CML-CP). Attempts to increase proportion of patients achieving TFR include combination of tyrosine kinase inhibitors (TKI) and other drugs. Interferon-α in addition to TKI has shown promising efficacy but with dose-dependent toxicity and discontinuations. NordCML007 was initiated to study the efficacy and safety of low dose pegylated IFN-α (PegIFN-α) in combination with dasatinib (DAS) in CML-CP. Methods Forty patients with newly diagnosed CML-CP were given DAS upfront. After month 3 (M3) 15 μg/wk of PegIFN-α was added and increased to 25 μg/wk from M7 until M15. DAS treatment was continued and adverse events and BCR-ABL1 qRT-PCR values were reported yearly after M24. Results from M1 to M18 have previously been published, and here we present long-term data. Results After 5 years of follow-up, there were no suspected unexpected serious adverse reactions, no increase in serosal effusions, no disease progressions and no CML-related deaths. Rates of MR3.0 (MMR), MR4.0 and MR4.5 were 84.6%, 64.1% and 51.3% respectively at M60, and 95% of patients reached MMR at some point during the study. Conclusion Initial addition of PegIFN-α to DAS shows good long-term efficacy without increased toxicity.

• Waidhauser, Johanna; Labopin, Myriam; Esteve, Jordi; Kröger, Nicolaus; Cornelissen, Jan & Gedde-Dahl, Tobias [Vis alle 17 forfattere av denne artikkelen] (2021). Allogeneic stem cell transplantation for AML patients with RUNX1 mutation in first complete remission: a study on behalf of the acute leukemia working party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 56(10), s. 2445–2453. doi: 10.1038/s41409-021-01322-w. Fulltekst i vitenarkiv
• Remberger, Mats; Tjønnfjord, Geir Erland; Abrahamsen, Ingerid Weum; Ali, Maryan Mohamed; Myhre, Anders Eivind & Gedde-Dahl, Tobias [Vis alle 7 forfattere av denne artikkelen] (2021). Superior Graft-versus-Host Disease-Free Relapse-Free Survival in Matched Unrelated Donor Hematopoietic Stem Cell Transplantation with Anti-Thymocyte Globulin (ATG) Compared to Matched Related Donor without ATG. Transplantation and Cellular Therapy (TCT). ISSN 2666-6375. 27(7), s. 621.e1–621.e3. doi: 10.1016/j.jtct.2021.03.019. Fulltekst i vitenarkiv
• Giebel, Sebastian; Labopin, Myriam; Sobczyk-Kruszelnicka, Malgorzata; Stelljes, Matthias; Byrne, Jenny L. & Fegueux, Nathalie [Vis alle 100 forfattere av denne artikkelen] (2021). Total body irradiation + fludarabine compared to busulfan + fludarabine as “reduced-toxicity conditioning” for patients with acute myeloid leukemia treated with allogeneic hematopoietic cell transplantation in first complete remission: a study by the Acute Leukemia Working Party of the EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 56(2), s. 481–491. doi: 10.1038/s41409-020-01050-7. Fulltekst i vitenarkiv
• Cesaro, Simone; Tridello, Gloria; Knelange, Nina Simone; Blijlevens, Nicole; Martin, Murray & Snowden, JA [Vis alle 29 forfattere av denne artikkelen] (2021). Impact of early candidemia on the long-term outcome of allogeneic hematopoietic stem cell transplant in non-leukemic patients: an outcome analysis on behalf of IDWP–EBMT. Bone Marrow Transplantation. ISSN 0268-3369. 56(7), s. 1563–1572. doi: 10.1038/s41409-021-01212-1. Fulltekst i vitenarkiv
• Andersen, Anders Mikal; Bergan, Stein; Gedde-Dahl, Tobias; Buechner, Jochen & Vethe, Nils Tore (2021). Fast and reliable quantification of busulfan in blood plasma using two-channel liquid chromatography tandem mass spectrometry: Validation of assay performance in the presence of drug formulation excipients. Journal of Pharmaceutical and Biomedical Analysis. ISSN 0731-7085. 203, s. 1–11. doi: 10.1016/j.jpba.2021.114216. Fulltekst i vitenarkiv Vis sammendrag

  A fast and reliable method based on two-channel liquid chromatography coupled to tandem mass spectrometry was developed and successfully validated for quantification of busulfan. The drug vehicle polyethylene glycol 400 was quantified simultaneously in patient samples. The sample preparation consisted of simple protein precipitation using a mixture of methanol and zinc sulphate containing busulfan-d8 as internal standard. Chromatographic separation was performed on a short biphenyl column (30 mm × 3.0 mm, 5 μm particles) using a step gradient from 30 % to 85 % methanol, ensuring co-elution of the analyte and internal standard. Quantification was performed using the mass transition of 264.1 > 151.1 for busulfan and 272.1 > 159.1 for the internal standard. Using only 20 μL of plasma sample, the lower limit of quantification was 25 ng/mL. Signal to noise ratio at the lower limit of quantification exceeded 300. The assay performance was not adversely affected by matrix effects originating from drug formulation excipients or other sample components. The coefficient of variation was ≤4 % and the mean accuracy 101–108 % across the calibration range 25–5 000 ng/mL. Chromatographic run time was 2 min and 8 s, allowing an effective run-time of 1 min and 10 s when using two alternating LC-channels. The assay has been implemented in routine practice with accreditation according to the ISO 15189 standard, and performs well in external quality control assessments. We present for the first time that shortly after an IV infusion of busulfan, the plasma levels of polyethylene glycol 400 may be in the range of 400−800 mg/L. The presence of these levels of detergent in patient samples may have detrimental effects on assay performance in LC–MS/MS, not limited to busulfan assays. This may be a concern for any LC–MS/MS analysis performed on samples collected within the first 24 h after an IV infusion of busulfan.

• Tvedt, Tor Henrik Anderson; Skaarud, Kristin Aneta Joan; Tjønnfjord, Geir Erland; Gedde-Dahl, Tobias; Iversen, Per Ole & Bruserud, Øystein (2020). The systemic metabolic profile early after allogeneic stem cell transplantation: Effects of adequate energy support administered through enteral feeding tube. Biology of Blood and Marrow Transplantation. ISSN 1083-8791. 26(2), s. 380–391. doi: 10.1016/j.bbmt.2019.10.005. Fulltekst i vitenarkiv Vis sammendrag

  Patients undergoing allogeneic stem cell transplantation usually require nutritional support. There is no consensus on whether enteral support through tube feeding should be preferred. A recent randomized study could not detect any difference between enteral and parenteral feeding with regard to post-transplant outcomes, whereas 2 retrospective studies described an association between enteral feeding and a favorable post-transplant outcome. We compared pre- and post-transplant plasma metabolomic profiles for 10 patients receiving mainly enteral nutritional support and 10 patients receiving mainly parenteral support. Samples were collected before conditioning and 3 weeks post-transplant; 824 metabolites were analyzed using mass spectrometry. The pretransplant metabolite profiles showed a significant overlap between the 2 groups. Post-transplant samples for both patient groups showed an increase of secondary bile acids and endocannabinoids, whereas reduced levels were seen for food preservatives, plasmalogens, and retinol metabolites. The main post-transplant differences between the groups were decreased levels of fatty acids and markers of mitochondrial activation in the control group, indicating that these patients had insufficient energy intake. A significant effect was also seen for heme/bilirubin metabolism for the parenteral support. To conclude, allotransplant recipients showed altered metabolic profiles early after transplantation; this was mainly due to the conditioning/transplantation/reconstitution, whereas the type of nutritional support had minor effects.

• Divito, Sherrie J.; Aasebø, Anders Theodor Michaelsen; Matos, Tiago R.; Hsieh, Pei-Chen; Collin, Matthew & Elco, Christopher P. [Vis alle 25 forfattere av denne artikkelen] (2020). Peripheral host T cells survive hematopoietic stem cell transplantation and promote graft-versus-host disease. Journal of Clinical Investigation. ISSN 0021-9738. 130(9), s. 4624–4636. doi: 10.1172/JCI129965. Fulltekst i vitenarkiv
• Tadele, Dagim Shiferaw; Robertson, Joseph; Crispin, Richard; Herrera, Carmen; Chlubnová, Markéta & Piechaczyk, Laure Isabelle [Vis alle 14 forfattere av denne artikkelen] (2020). A cell competition-based small molecule screen identifies a novel compound that induces dual c-Myc depletion and p53 activation. Journal of Biological Chemistry. ISSN 0021-9258. 296. doi: 10.1074/jbc.RA120.015285. Fulltekst i vitenarkiv Vis sammendrag

  Breakpoint Cluster Region-Abelson kinase (BCR–Abl) is a driver oncogene that causes chronic myeloid leukemia and a subset of acute lymphoid leukemias. Although tyrosine kinase inhibitors provide an effective treatment for these diseases, they generally do not kill leukemic stem cells (LSCs), the cancer-initiating cells that compete with normal hematopoietic stem cells for the bone marrow niche. New strategies to target cancers driven by BCR–Abl are therefore urgently needed. We performed a small molecule screen based on competition between isogenic untransformed cells and BCR–Abl-transformed cells and identified several compounds that selectively impair the fitness of BCR–Abl-transformed cells. Interestingly, systems-level analysis of one of these novel compounds, DJ34, revealed that it induced depletion of c-Myc and activation of p53. DJ34-mediated c-Myc depletion occurred in a wide range of tumor cell types, including lymphoma, lung, glioblastoma, breast cancer, and several forms of leukemia, with primary LSCs being particularly sensitive to DJ34. Further analyses revealed that DJ34 interferes with c-Myc synthesis at the level of transcription, and we provide data showing that DJ34 is a DNA intercalator and topoisomerase II inhibitor. Physiologically, DJ34 induced apoptosis, cell cycle arrest, and cell differentiation. Taken together, we have identified a novel compound that dually targets c-Myc and p53 in a wide variety of cancers, and with particularly strong activity against LSCs.

• Halaburda, Kazimierz; Labopin, Myriam; Mailhol, Audrey; Socié, Gérard; Craddock, Charles & Aljurf, Mahmoud [Vis alle 18 forfattere av denne artikkelen] (2020). Allogeneic stem cell transplantation in second complete remission for core binding factor acute myeloid leukemia: A study from the acute leukemia working party of the european society for blood and marrow transplantation. Haematologica. ISSN 0390-6078. 105(6), s. 1723–1730. doi: 10.3324/haematol.2019.222810. Fulltekst i vitenarkiv
• Wiktor-Jedrzejczak, W.; Drozd-Sokolowska, J; Eikema, DJ; Hoek, J; Potter, M & Wulf, G [Vis alle 32 forfattere av denne artikkelen] (2019). EBMT prospective observational study on allogeneic hematopoietic stem cell transplantation in T-prolymphocytic leukemia (T-PLL). Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-019-0448-x. Fulltekst i vitenarkiv
• Søraas, Arne Vasli; Matsuyama, Mieko; Lima, Marcos de; Wald, David; Buechner, Jochen & Gedde-Dahl, Tobias [Vis alle 12 forfattere av denne artikkelen] (2019). Epigenetic age is a cell-intrinsic property in transplanted human hematopoietic cells. Aging Cell. ISSN 1474-9718. 18(2). doi: 10.1111/acel.12897. Fulltekst i vitenarkiv Vis sammendrag

  The age of tissues and cells can be accurately estimated by DNA methylation analysis. The multitissue DNA methylation (DNAm) age predictor combines the DNAm levels of 353 CpG dinucleotides to arrive at an age estimate referred to as DNAm age. Recent studies based on short‐term observations showed that the DNAm age of reconstituted blood following allogeneic hematopoietic stem cell transplantation (HSCT) reflects the age of the donor. However, it is not known whether the DNAm age of donor blood remains independent of the recipient's age over the long term. Importantly, long‐term studies including child recipients have the potential to clearly reveal whether DNAm age is cell‐intrinsic or whether it is modulated by extracellular cues in vivo. Here, we address this question by analyzing blood methylation data from HSCT donor and recipient pairs who greatly differed in chronological age (age differences between 1 and 49 years). We found that the DNAm age of the reconstituted blood was not influenced by the recipient's age, even 17 years after HSCT, in individuals without relapse of their hematologic disorder. However, the DNAm age of recipients with relapse of leukemia was unstable. These data are consistent with our previous findings concerning the abnormal DNAm age of cancer cells, and it can potentially be exploited to monitor the health of HSCT recipients. Our data demonstrate that transplanted human hematopoietic stem cells have an intrinsic DNAm age that is unaffected by the environment in a recipient of a different age.

• Passweg, Jakob Robert; Labopin, Myriam; Christopeit, Maximillian; Cornelissen, Jan J.; Pabst, Thomas & Soçie, Gérard J. [Vis alle 19 forfattere av denne artikkelen] (2019). Postremission consolidation by autologous hematopoietic cell transplantation (HCT) for acute myeloid leukemia in first complete remission (CR) and negative implications for subsequent allogeneic HCT in second CR: A study by the acute leukemia working party of the European Society for Blood and Marrow Transplantation (EBMT). Biology of Blood and Marrow Transplantation. ISSN 1083-8791. s. 1–6. doi: 10.1016/j.bbmt.2019.11.021. Fulltekst i vitenarkiv Vis sammendrag

  After autologous hematopoietic cell transplantation (HCT) in the first complete remission (CR1), patients with acute myeloid leukemia (AML) may relapse and undergo allogeneic HCT in the second complete remission (CR2). The aim of this study was to analyze the outcome of allogeneic HCT performed in CR2 comparing patients with prior consolidation by autologous HCT versus patients with chemotherapy consolidation. Included were 2619 adults with allogeneic HCT in CR2 from 2000 to 2017 with (n = 417) or without (n = 2202) prior autologous HCT. Patient groups were not entirely comparable; patients with prior autologous HCT were younger, had less often a favorable cytogenetic profile, had more commonly donors other than matched siblings, and more often received reduced-intensity conditioning. In multivariate analysis, nonrelapse mortality risks in patients with prior autologous HCT were 1.34 (1.07 to 1.67; P = .01) after adjustment for age, cytogenetic risk, transplant year, donor, conditioning intensity, sex matching, interval diagnosis-relapse, and relapse-allogeneic HCT as compared with chemotherapy consolidation. Similarly, risks of events in leukemia-free survival and graft-versus-host disease, relapse-free survival were higher with prior autologous HCT, 1.17 (1.01 to 1.35), P = .03 and 1.18 (1.03 to 1.35), P = .02, respectively. Risk of death was also higher, 1.13 (0.97 to 1.32), P = .1, but this was not significant. Postremission consolidation with autologous HCT for AML in CR1 increases toxicity of subsequent allogeneic HCT in CR2.

• Gilleece, Maria H; Labopin, Myriam; Savani, Bipin N; Yakoub-Agha, Ibrahim; Socié, Gérard & Gedde-Dahl, Tobias [Vis alle 16 forfattere av denne artikkelen] (2019). Allogeneic haemopoietic transplantation for acute myeloid leukaemia in second complete remission: a registry report by the Acute Leukaemia Working Party of the EBMT. Leukemia. ISSN 0887-6924. 34(1), s. 87–99. doi: 10.1038/s41375-019-0527-4. Fulltekst i vitenarkiv

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• Kopland, Marthe; Ottersen, Vigdis Skinnemoen; Dahle, Dag Olav; Gedde-Dahl, Tobias & Dalgaard, Jakob (2025). En mann i 50-årene med akutt nyresvikt, anemi og trombocytopeni. Tidsskrift for Den norske legeforening (Tidsskriftet). ISSN 0029-2001. 145(1). doi: 10.4045/tidsskr.24.0231. Fulltekst i vitenarkiv
• Flygt, Hjalmar; Söderlund, Stina; Richter, Johan; Saussele, Susanne; Koskenvesa, Perttu & Stenke, Leif [Vis alle 20 forfattere av denne artikkelen] (2024). Correction to: Treatment-free remission after a second TKI discontinuation attempt in patients with Chronic Myeloid Leukemia re-treated with dasatinib – interim results from the DAstop2 trial (Leukemia, (2024), 10.1038/s41375-024-02145-6). Leukemia. ISSN 0887-6924. doi: 10.1038/s41375-024-02184-z. Fulltekst i vitenarkiv
• Ye, Yishan; Labopin, Myriam; Chen, Jia; Wu, Depei; Gedde-Dahl, Tobias & Blaise, Didier [Vis alle 21 forfattere av denne artikkelen] (2024). Lower relapse incidence with HAPLO versus MSD or MUD HCTs for AML patients with KMT2A rearrangement: a study from the Global Committee and the ALWP of the EBMT. Blood Cancer Journal. 14(1). doi: 10.1038/s41408-024-01072-0. Fulltekst i vitenarkiv
• Blum, Sabine; Chalandon, Yves; Labopin, Myriam; Finke, Jürgen; Gedde-Dahl, Tobias & Othman, Tarek Ben [Vis alle 15 forfattere av denne artikkelen] (2024). Incidence and outcome of central nervous system relapse after hematopoietic stem cell transplantation in patients suffering from acute myeloid leukemia and acute lymphoblastic leukemia: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Haematologica. ISSN 0390-6078. 109(7), s. 2346–2350. doi: 10.3324/haematol.2023.284858. Fulltekst i vitenarkiv
• Drozd-Sokolowska, Joanna; Gras, Luuk; Koster, Linda; Martino, Rodrigo; Salas, María Queralt & Salmenniemi, Urpu [Vis alle 26 forfattere av denne artikkelen] (2024). Autologous hematopoietic cell transplantation for T-cell prolymphocytic leukemia: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT. Haematologica. ISSN 0390-6078. 109(5), s. 1608–1613. doi: 10.3324/haematol.2023.284359. Fulltekst i vitenarkiv
• Vethe, Nils Tore; Andersen, Anders Mikal; Gedde-Dahl, Tobias; Buechner, Jochen & Bergan, Stein (2024). Therapeutic Drug Monitoring of Intravenous Busulfan and Analytical Challenges due to the Drug Formulation Excipient PEG 400: Letter to the Editor. Therapeutic Drug Monitoring. ISSN 0163-4356. 46(3), s. 416–417. doi: 10.1097/FTD.0000000000001209. Fulltekst i vitenarkiv
• Ruggeri, Annalisa; Wreede, Liesbeth C. de; Müller, Carlheinz R.; Crivello, Pietro; Bonneville, Edouard F. & Petersdorf, Effie W. [Vis alle 170 forfattere av denne artikkelen] (2023). Integrating biological HLA-DPB1 mismatch models to predict survival after unrelated hematopoietic cell transplantation. Haematologica. ISSN 0390-6078. 108(2), s. 645–652. doi: 10.3324/haematol.2021.280055. Fulltekst i vitenarkiv
• Rørvik, Synne Dragesund; Abrahamsen, Ingerid Weum; Myhre, Anders Eivind; Vo, Camilla Dao; Meling, Eva Ødegaard & Bruserud, Øystein [Vis alle 8 forfattere av denne artikkelen] (2023). Kronisk transplantat-mot-vertsykdom. Tidsskrift for Den norske legeforening (Tidsskriftet). ISSN 0029-2001. 143(4), s. 337–341. doi: 10.4045/tidsskr.22.0525. Fulltekst i vitenarkiv Vis sammendrag

  Kronisk transplantat-mot-vert-sykdom er en senkomplikasjon etter allogen stamcelletransplantasjon, og medfører kronisk inflammasjon og fibrose i forskjellige organer på grunn av feil regulering av donors immunceller. Sykdommen kan forekomme i alle organer, men sees hyppigst i hud, øyne, munnhule, gastrointestinaltraktus, genitalia, lunger, muskler, fascier og ledd. Kronisk transplantat-mot-vert-sykdom er assosiert med betydelig sykelighet og dødelighet, og behandling krever tett samarbeid mellom forskjellige deler av spesialisthelsetjenesten. Vi gir i denne artikkelen en klinisk oversikt over kronisk transplantat-mot-vert-sykdom basert på ikke-systematiske litteratursøk og egne kliniske erfaringer.

• Horn, Morten Andreas; Myhre, Anders Eivind; Prescott, Trine; Aasly, Jan; Sundal, Christina Heidemann & Gedde-Dahl, Tobias (2022). Prophylactic Allogeneic Hematopoietic Stem Cell Therapy for CSF1R-Related Leukoencephalopathy. Movement Disorders. ISSN 0885-3185. 37(5), s. 1108–1109. doi: 10.1002/mds.29011. Fulltekst i vitenarkiv
• Nagler, Arnon; Ngoya, Maud; Galimard, Jacques-Emmanuel; Labopin, Myriam; Kröger, Nicolaus & Socié, Gerard [Vis alle 19 forfattere av denne artikkelen] (2022). Correction: Trends in outcome of transplantation in patients with secondary acute myeloid leukemia: an analysis from the Acute Leukemia Working Party (ALWP) of the EBMT (Bone Marrow Transplantation, (2022), 10.1038/s41409-022-01825-0). Bone Marrow Transplantation. ISSN 0268-3369. doi: 10.1038/s41409-022-01853-w. Fulltekst i vitenarkiv
• Abrahamsen, Ingerid Weum; Grønvold, Bjørn Christer; Inderberg, Else Marit; Mensali, Nadia; Mattsson, Jonas & Gedde-Dahl, Tobias (2020). Posttransplantation Lymphoproliferative Disease Treated by Retransplantation. Case Reports in Immunology. ISSN 2090-6609. 2020. doi: 10.1155/2020/9403123. Fulltekst i vitenarkiv Vis sammendrag

  Epstein–Barr virus- (EBV-) induced posttransplantation lymphoproliferative disease (PTLD) is a life-threatening complication following allogeneic stem cell transplantation. *e main risk factor is anti-thymocyte globulin (ATG). Patients who fail first-line treatment with rituximab have a poor prognosis. *ough adoptive transfer of EBV-specific T cells is a potentially effective option, it is not readily available. In this case report, the patient developed PTLD following transplantation for aplastic anemia using ATG as part of the conditioning. He failed rituximab treatment and developed graft failure. We were aware that the stem cell donor had a recent EBV infection prior to transplantation, whereas the patient most likely was EBV negative before transplant. We describe our strategy to meet the patient’s urgent need for EBV-specific Tcells, as well as new hematopoietic stem cells. *e same donor was used for a second transplant, using peripheral blood stem cells. *e conditioning used was thiotepa/busulfan/fludarabin with a single dose of cyclophosphamide after transplant as graft-versus-host disease (GVHD) prophylaxis. *e EBV DNA levels fell when conditioning was started, and have been undetectable since day +15 and remained so till 18 months after transplantation. *e patient is doing well. *is case reports successful use of cyclophosphamide after transplantation as GVHD prophylaxis, preserving virus-specific immunity.

• Grønvold, Bjørn Christer Linder; Ali, M.M.; Remberger, M.; Mattsson, J.; Egeland, Torstein & Lundin, Katrin [Vis alle 13 forfattere av denne artikkelen] (2020). HLA-DPB1 mismatch reduce relapse and improve survival in T-cell replete unrelated donor allogeneic stem cell transplantation. Bone Marrow Transplantation. ISSN 0268-3369. 55(8), s. 1658–1661. doi: 10.1038/s41409-020-0827-3. Fulltekst i vitenarkiv

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