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Transplant results in adults with Fanconi anaemia.

Abstract：

:The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16 years of age when diagnosed with FA, and underwent transplantation at a median age of 23 years. Time between diagnosis and transplant was shortest (median 2 years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76-87%), acute graft-versus-host disease (GvHD) grade II-IV in 22% (95% CI 16-28%) and the incidence of chronic GvHD at 96 months was 26% (95% CI 20-33). Non-relapse mortality at 96 months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58 months. Patients transplanted after 2000 had improved survival (84% at 36 months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.

journal_name

Br J Haematol

journal_title

British journal of haematology

authors

Bierings M,Bonfim C,Peffault De Latour R,Aljurf M,Mehta PA,Knol C,Boulad F,Tbakhi A,Esquirol A,McQuaker G,Sucak GA,Othman TB,Halkes CJM,Carpenter B,Niederwieser D,Zecca M,Kröger N,Michallet M,Risitano AM,Ehninger G

doi

10.1111/bjh.15006

subject

Has Abstract

pub_date

2018-01-01 00:00:00

pages

100-109

issue

1

eissn

0007-1048

issn

1365-2141

journal_volume

180

pub_type

杂志文章,多中心研究

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