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Year : 2018  |  Volume : 9  |  Issue : 4  |  Page : 120-125

Hematopoietic stem cell transplantation in children with hemoglobinopathies: A single-center experience

1 Department of Oncology, King Faisal Specialist Hospital and Research Centre, Jeddah, Saudi Arabia
2 King Faisal Specialist Hospital and Research Center, Jeddah, Saudi Arabia
3 Ibn Sina National College, Jeddah, Saudi Arabia

Correspondence Address:
Dr. Muhammad Matloob Alam
Department of Oncology, King Faisal Specialist Hospital and Research Centre, P. O. Box 40047, Jeddah 21499
Saudi Arabia
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Source of Support: None, Conflict of Interest: None

DOI: 10.4103/joah.joah_32_18

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INTRODUCTION: Hematopoietic stem cell transplantation (HSCT) is a curative treatment for children with serious hematological disorders including thalassemia major (TM) and sickle cell disease (SCD). This study aimed to determine the frequencies of transplant-related morbidity and outcome in our tertiary health-care facility. MATERIALS AND METHODS: We retrospectively analyzed the clinical, laboratory, and outcome data of 32 cases of hemoglobinopathies (TM = 19 and SCD = 13) who underwent bone marrow (BM) transplantation between 2005 and 2016. SPSS was used for statistical analysis, and Kaplan–Meier curve was constructed for overall survival (OS) and event-free survival. RESULTS: The median age of the study population at the time of transplant was 7.8 (range 2–14 years). Male-to-female ratio was 1:1. All patients were received allogeneic full matched related donor transplantation. Conditioning regimen was Bu/Cy/antithymocyte globulin (ATG) for SCD and Flu/Bu/Cy/ATG for TM. Source of stem cell in all patients was bone marrow and median stem cell dose CD 34+ was 5.26 (range 2.9–9.9) × 106/kg. All patients were received cyclosporin and methotrexate for graft versus host disease (GVHD) prophylaxis. Acute and chronic GVHD were observed in 10 (31.25%) and 2 (6.3%) cases, respectively. The median time to absolute neutrophil count (ANC) engraftment was 22 days (range: 12–27), and the median time to platelet engraftment was 32 days (range: 18–45) in 28 patients and the remaining four patients had delayed platelet engraftment (62–90 days). Cytomegalovirus (CMV) reactivation was observed in 18 (56.3%) cases; majority were asymptomatic (n = 17/18; 94.4%). Three-year event-free survival (EFS) and OS were 90.6% and 93.7%, respectively, with median duration of follow-up 33.72 (± 25.3) months. CONCLUSION: The results of the pediatric HSCT for hemoglobinopathies at our institution are excellent and are comparable to published reports from international collaborative groups.

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