Hematopoietic Stem Cell Transplantation for Fanconi Anemia: A Single Center Experience from India

Abstract

Hematopoietic stem cell transplantation (HSCT) is the only treatment option for the hematological manifestations of Fanconi anemia (FA). Fludarabine based reduced intensity conditioning regimens have helped in improving outcomes significantly in FA patients. We retrospectively analyzed the outcomes of FA patients who underwent allogeneic HSCT at BLK Superspeciality Hospital, New Delhi from June 2011 to September 2019. Twenty FA patients underwent 23 transplants at our center. Overall survival and disease free survival were 65% and 50%, respectively at a median of 23 months. Overall mortality was 30%. HSCT for FA is a feasible option even in developing countries although children present late to transplant centers after multiple transfusions and infections.

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Abbreviations

FA:

Fanconi anaemia

HSCT:

Hematopoietic stem cell transplant

AML:

Acute myeloid leukemia

DEB:

Diepoxybutane

MMC:

Mitomycin-C

OS:

Overall survival

DFS:

Disease free survival

EBMT:

European Bone Marrow Transplant

GvHD:

Graft versus host disease

CMV:

Cytomegalovirus

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Correspondence to Divya Doval.

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Doval, D., Choudhary, D., Sharma, S.K. et al. Hematopoietic Stem Cell Transplantation for Fanconi Anemia: A Single Center Experience from India. Indian J Hematol Blood Transfus 36, 565–568 (2020). https://doi.org/10.1007/s12288-020-01254-3

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Keywords

  • Fanconi anemia
  • HSCT
  • Matched sibling donor