Title: Hematopoietic stem cell transplantation in thalassemia patients: A Jordanian single centre experience

Abstract

Introduction: 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. Aim: To evaluate the outcome of children who underwent allogenic hematopoietic stem Cell transplantation as a curative approach for Thalassemia Major. Settings: Queen Rania AL- Abdullah children Hospital (QRCH). Methods: 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. Results: 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 crieteria for class 1 Pesaro classification. The median CD34 count was 3.5 million /Kg of recipient weight (range, 1.5*106-7*106 /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 . Conclusion: The outlook for Thalassemia major has dramatically changed after HSCT, with a considerable success in Jordan and results comparable to international data.