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<p>Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-compatible family member or unrelated donor is the only way to restore normal haematopoiesis in Fanconi anaemia (FA) patients. During the last two decades, outcomes of HSCT for FA patients have significantly improved. In this study, we compare the outcomes using non-irradiation-containing conditioning regimens for FA patients transplanted from HLA-matched siblings. Fifteen patients with FA received 16 MSD HSCT for FA between January 2017 and January 2023. Fifteen were transplanted for aplastic anaemia. 1 patient had evidence of evolution to myeloid malignancy. Conditioning used was Fludarabine-cyclophosphamide- horse ATG in all HSCT except in one patient where a second transplant was done, and Fludarabine-Busulphan-ATG was used. GVHD prophylaxis was cyclosporine-methotrexate in all the cases. The median CD34 dose given was 5.2 X 106 /kg. The median time for neutrophil engraftment was 14 days and platelet engraftment was 18 days. The incidence of grade II-IV acute graft-versus-host disease (GVHD) was 6.25 % and chronic GVHD was 12.5% at 2-year follow-up. Four patients (25%) had CMV viremia, 1 had BK viremia, 1 had EBV viremia, 2 had culture-proven sepsis and 15 developed mucositis. Of the 15, 14 are alive at the last follow-up. Overall survival at a median follow-up of 2 years was 93.7 % and EFS of 81.2%. None of the survivors had evidence of a cytogenetic clone, MDS or leukaemia or documentation of secondary malignancy at 2 years post-transplant noted. The overall survival rates are on the rise, and long-term morbidity in the form of chronic GVHD and secondary malignancies remains a formidable setback to a successful HSCT, but our studies showed better overall survival with lower GVHD rates.</p>

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Matched Sibling Hematopoietic Stem Cell Transplantation for Pediatric Fanconi Anemia: Experience from A Tertiary Care Centre in India

  • 期刊论文

  • 发表日期：

    2026年1月27日

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  • Swati Bhayana,
  • Sohini Chakraborty,
  • Shrinidhi Nathany,
  • Arun Danewa,
  • Neha Rastogi Panda,
  • Sunisha Arora,
  • Parminder Pal Singh,
  • Surbhi Pokhariyal,
  • Rahul Bhargava,
  • Vikas Dua

摘要

Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-compatible family member or unrelated donor is the only way to restore normal haematopoiesis in Fanconi anaemia (FA) patients. During the last two decades, outcomes of HSCT for FA patients have significantly improved. In this study, we compare the outcomes using non-irradiation-containing conditioning regimens for FA patients transplanted from HLA-matched siblings. Fifteen patients with FA received 16 MSD HSCT for FA between January 2017 and January 2023. Fifteen were transplanted for aplastic anaemia. 1 patient had evidence of evolution to myeloid malignancy. Conditioning used was Fludarabine-cyclophosphamide- horse ATG in all HSCT except in one patient where a second transplant was done, and Fludarabine-Busulphan-ATG was used. GVHD prophylaxis was cyclosporine-methotrexate in all the cases. The median CD34 dose given was 5.2 X 106 /kg. The median time for neutrophil engraftment was 14 days and platelet engraftment was 18 days. The incidence of grade II-IV acute graft-versus-host disease (GVHD) was 6.25 % and chronic GVHD was 12.5% at 2-year follow-up. Four patients (25%) had CMV viremia, 1 had BK viremia, 1 had EBV viremia, 2 had culture-proven sepsis and 15 developed mucositis. Of the 15, 14 are alive at the last follow-up. Overall survival at a median follow-up of 2 years was 93.7 % and EFS of 81.2%. None of the survivors had evidence of a cytogenetic clone, MDS or leukaemia or documentation of secondary malignancy at 2 years post-transplant noted. The overall survival rates are on the rise, and long-term morbidity in the form of chronic GVHD and secondary malignancies remains a formidable setback to a successful HSCT, but our studies showed better overall survival with lower GVHD rates.