Matched unrelated donor HSCT for thalassemia major using treosulphan based conditioning protocol for children: A single center experience from India

Abstract

Hematopoietic stem cell transplantation (HSCT) is the only potentially curative treatment for patients with thalassemia major. However, only 30-35% of patients have a suitable HLA-matched family donor (MFD). We report herewith our experience with matched unrelated donor (MUD) HSCT using a treosulfan based conditioning protocol, from a tertiary center in India. The cohort consisted of 25 children (18 boys and 7 girls) with transfusion-dependent thalassemia major who were transplanted using matched or minimally mismatched unrelated donors over four years with a median follow-up of 12 months (range 2–36 months). The median age of the cohort at HSCT was five years. None of the children transplanted had graft rejection. Reactivation of cytomegaloviral infection occurred in 32% of children. Grade II-IV acute graft versus host disease (aGvHD) occurred in 60% of the children. Of these, two (8%), succumbed to aGvHD. Chronic extensive GvHD was not observed in any of the children until the last follow-up. The probability of survival at a mean of 34.3 months (CI 31.2–37.4 months) for those who underwent a fully HLA-matched unrelated donor HSCT was 95%. This data shows that if the donor selection is based on strict compatibility criteria, MUD-HSCT can offer a cure to children with thalassemia major, with outcomes similar to the MFD-HSCT.