Irradiation-free, T-cell replete haploidentical transplant for Fanconi anaemia, weighing the benefits

Br J Haematol. 2022 Dec;199(5):639-641. doi: 10.1111/bjh.18446. Epub 2022 Sep 19.

Abstract

The optimal haploidentical haematopoietic cell transplant approach for Fanconi anaemia (FA) patients is not well established, given the rarity of the disease, the increased sensitivity to DNA-damaging agents and the high risk of severe graft-versus-host disease (GVHD). The report by Xu et al. suggests that excellent engraftment and short-term survival can be achieved in FA patients without irradiation, but their retrospective cohort was plagued by a high rate of severe GVHD. Our commentary explores the outcomes in T-cell replete haploidentical haematopoietic cell transplant and ponders whether elimination of total body irradiation in FA patients is the best method if it limits the ability to safely administer post-transplant cyclophosphamide. Commentary on: Xu et al. Unmanipulated haploidentical haematopoietic cell transplantation with radiation-free conditioning in Fanconi anaemia: A retrospective analysis from the Chinese Blood and Marrow Transplantation Registry Group. Br J Haematol. 2022;199:401-410.

Keywords: Fanconi anaemia; T-cell replete haploidentical transplant; irradiation-free.

MeSH terms

  • Cyclophosphamide / therapeutic use
  • Fanconi Anemia* / therapy
  • Graft vs Host Disease* / etiology
  • Graft vs Host Disease* / prevention & control
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Retrospective Studies
  • T-Lymphocytes
  • Transplantation Conditioning / methods
  • Transplantation, Haploidentical

Substances

  • Cyclophosphamide