Treatment Outcome of Haematopoietic Stem Cell Transplant in Fanconi Anaemia: Experience from a Low- and Middle-Income Country.
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OBJECTIVE: To determine the outcome of haematopoietic stem cell transplantation (HSCT) in Fanconi anaemia (FA) patients. STUDY DESIGN: Retrospective observational study. Place and Duration of the Study: Department of Clinical Haematology, Armed Forces Bone Marrow Transplant Centre, CMH Rawalpindi, Pakistan, from February 2009 to December 2023. METHODOLOGY: The data of 41 cases of FA undergoing HSCT were analysed retrospectively, from February 2009 to December 2023. Cases transformed into myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML) were excluded. All cases of FA under 18 years of age having fully HLA-matched HSCT were included. A non-myeloablative conditioning regimen consisting of Flu120/Cy30/ATG20 was used. Descriptive statistics were obtained for harvest source, complications, graft success, and survival. RESULTS: The study included 27 (65.9%) boys and 14 (34.1%) girls with a mean age of 10.3 ± 3.1 years. Bone marrow as a stem cell source was harvested in 33 (80.5%) cases. Cyclosporin-induced hypertension was documented in 36 (87.8%) cases. The cumulative incidence of acute GVHD and chronic GVHD was 34.1% and 32.3%, respectively. Six (14.6%) patients had graft failure, three had primary graft failure, and three had secondary graft failure. At a median follow-up of 63.8 ± 46.1 months, the overall survival (OS) rate was 73.2% and the disease-free survival (DFS) rate was 73.2%. CONCLUSION: Overall survival of more than 70% is promising but inferior to that in developed countries. Graft failure, infectious complications, and GVHD remain major challenges requiring improvement in the developing countries. KEY WORDS: Haematopoietic stem cell transplantation, Fanconi anaemia, Pakistan.