The Outcomes of Allogenic Bone Marrow Transplantation for BThalassemia Major Patients: Single-Centre Experience

Abstract

Objectives: The Primary objective of this study is to assess the outcome of Allogenic Bone Marrow Transplantation for B- Thalassemia Major (B-TM) Patients at KFSHD, also to compare our local results with international outcomes, and to identify the complications of this modality of treatment in our cohort. Design: This is a retrospective descriptive study. It includes all patients’ less16 year transfusion-dependent, b-TM patients who underwent matched-related donor (MRD) allogeneic stem cell transplantation at KFSHD, pediatric hematology/ oncology department between January 2013 and December 2020. Total number of cohort is 21 patients. Methods: After obtaining the IRB approval, all the data and information were retrieved from the patients’ hard and electronic medical records and then computerized using a Microsoft Excel sheet. Computerized data exported to (SPSS) program updated version 24 (IBM Corp., Armonk, NY, USA) which was used for analysis of the data. Results: The results of the 21 patients with b-TM who underwent MRD allogenic BMT at KFSHD highlighted the role of MRD/BMT as curative therapy of b-TM with no increase in transplantation-related complications when compared with similar results. In our study, SOS rate was found to be 33.3%, GVHD was 23.8% but non-invasive infections were 76.2%, which is high but not influential. The overall survival (OS) was 90.5% and the disease-free survival (DFS) was 95.2%. Conclusion: Because of the perfect, promising figures of survival for stem cell transplantation in b-thalassemia major (b-TM) patients during the last decades and the noticeable improvement in the quality of their life after that, and since our study showed similar good comparable outcome and survival rates with other international centers, we strongly recommend early transplantation for b-TM if a suitable donor is available in an experienced BMT center. The overall survival (OS) was 90.5% and the disease-free survival (DFS) was 95.2%, which are comparable outcomes with other international similar centers.