Patients with nonmalignant diseases undergoing hematopoietic cell transplantation using treosulfan-based conditioning can achieve a 2-year survival of 90%, according to a prospective multi-center trial of 31 transplants. All patients engrafted, and day-100 transplant-related mortality was 0%. At a median follow-up of 24 months, 2-year survival was 90%. The cumulative incidences of grades III-IV acute GVHD at day 100 and chronic GVHD at 2 years were 10% and 21%, respectively. The authors concluded that a treosulfan-based conditioning regimen “is effective at establishing donor engraftment with low toxicity and improved survival in patients with nonmalignant diseases and support the need for future disease-specific clinical trials.”
Burroughs LM, et al. Biol Blood Marrow Transplant