Due to copyright issues, an electronic copy of the thesis must be ordered from the faculty. For the faculty to have time to process the order, the order must be received by the faculty at the latest 2 days before the public defence. Orders received later than 2 days before the defence will not be processed. After the public defence, please address any inquiries regarding the thesis to the candidate.
Trial Lecture – time and place
See Trial Lecture.
Adjudication committee
- First opponent: Senior Consultant Marianne Ifversen, Rigshospitalet Copenhagen, Denmark
- Second opponent: Professor Håkon Reikvam, University of Bergen, Norway
- Third member and chair of the evaluation committee: Professor II Marianne Grønlie Guren, University of Oslo
Chair of the Defence
Professor emeritus Klaus Beiske, University of Oslo
Principal Supervisor
Professor II Ellen Ruud, University of Oslo
Summary
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative but intensive treatment for patients without other options. While survival has improved, the burden of late effects remains considerable, particularly among those treated in childhood, adolescence, or young adulthood (CAYA). This study explored long-term outcomes after CAYA HSCT in Norway, focusing on the prevalence and impact of chronic graft-versus-host disease (GVHD), as well as the cost-effectiveness of HSCT.
We conducted a nationwide, cross-sectional study of survivors who underwent HSCT before age 30, were ≥16 at inclusion, and survived ≥5 years post-transplant. A complementary cohort study included all eligible CAYA HSCT patients treated in Norway between 1974 and 2009. Chronic GVHD was assessed using the 2014 National Institutes of Health (NIH) criteria.
At a median of 17 years post-HSCT, one third had active chronic GVHD, commonly affecting eyes and mouth. Severe cases were linked to higher symptom burden and reduced quality of life.
Dry eye disease (DED), a common ocular manifestation of GVHD, affected over half of the survivors, with systemic chronic GVHD increasing DED risk more than fourfold. Diagnostic comparisons showed that criteria from the International Chronic Ocular GVHD Consensus Group better distinguished ocular GVHD from general DED than the NIH criteria.
Cost-utility analysis estimated the cost per quality-adjusted life year at €9053—well below Norway’s accepted threshold—confirming HSCT as cost-effective. Over 35 years, the use of unrelated donors, peripheral blood stem cells, and reduced-intensity conditioning increased. Among transplanted patients, 45% died during follow-up; 5- and 10-year survival was 60% and 58%, respectively.
In conclusion, chronic GVHD remains a major late complication with significant long-term consequences. Our findings support tailored follow-up and provide essential input for future guidelines and health policy.
Additional information
Contact the research support staff.