Hematopoietic Stem Cell Transplantation in Thalassemia Patients: a Jordanian Single Centre Experience

Beta thalassemia major is the commonest inherited hematological disorder worldwide which needs lifelong sufficient supportive management. Hematopoietic stem Cell transplantation (HSCT) is the only curative treatment available till now. To evaluate the outcome of children who underwent allogenic hematopoietic stem Cell transplantation as a curative approach for Thalassemia Major, treated at Queen Rania AL- Abdullah children Hospital (QRCH). A retrospective review of the medical files was conducted for all children (< 15 years) who had thalassemia major and received HSCT between January, 2010 and January, 2019. The following variables were studied for all patients: age , gender, Pesaro classifications, the count of infused raw bone marrow stem cell (CD34), engraftment time, outcome and complications. A total of 34 children were transplanted for thalassemia major, at an average of 4 cases per year. All underwent allogenic raw bone marrow transplantation from matched related donors. Thirteen patients (38.2%) were males and twenty one (61.2%) were females. The age ranged between 2 and 15 years, with a median age of 6.5 years. According to Pesaro classification, 31 patients were class 2 (91.2%) and 3 patients were class 3 (8.8%) while no single case met the criteria for class 1 Pesaro classification. The median CD34 count was 3.5 million/Kg of recipient weight (range, 1.5*10 -7*10 /kg). The median time for neutrophil engraftment was 15.5 days. At a median follow up of 5 years (range 1- 9.5), 33 patients were alive. One patient died before 100 days post transplantation due to grade IV acute gastrointestinal Graft Versus Host Disease (GVHD). Three patients had secondary graft failure (8.8%). Six patients (17.5 %) developed mild grade 1-2 skin GVHD while another patient developed hemorrhagic cystitis due to BK virus and cytomegalovirus (CMV) which reactivated simultaneously, and was successfully managed. The outlook for Thalassemia major has dramatically changed after HSCT, with a considerable success in Jordan and results comparable to international data.