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Br J Haematol. 2018 Jan;180(1):100-109. doi: 10.1111/bjh.15006. Epub 2017 Nov 2.

Transplant results in adults with Fanconi anaemia.

Author information

1
Department of Haematology and Stem Cell Transplantation, Utrecht University Children's Hospital, Utrecht, The Netherlands.
2
Federal University of Parana, Curitiba, Brazil.
3
BMT Unit, French Reference Centre for Aplastic Anaemia, Hospital St Louis, Paris, France.
4
King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.
5
Division of Bone Marrow Transplant and Immune Deficiency, Cincinnati Children's Hospital Medical Centre, Cincinnati, OH, USA.
6
EBMT Data Office, Leiden, The Netherlands.
7
Paediatric Bone Marrow Transplant Service, Memorial Sloan Kettering, New York, NY, USA.
8
King Hussein Cancer Centre, Amman, Jordan.
9
Hospital Santa Creu I Sant Pau, Barcelona, Spain.
10
Beatson West of Scotland Cancer Centre, Glasgow, UK.
11
Gazi Universitesi, Ankara, Turkey.
12
Centre National de Greffe de Moelle, Tunis, Tunesia.
13
Department of Haematology, Leiden University Medical Centre, Leiden, The Netherlands.
14
University College London Hospital, London, UK.
15
Haematology and Oncology, University of Leipzig, Leipzig, Germany.
16
Paediatric Haematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
17
University Hospital Eppendorf, Hamburg, Germany.
18
Centre Hospitalier Lyon Sud, Lyon, France.
19
University of Napoli, Naples, Italy.
20
Universitatsklinikum Dresden, Dresden, Germany.
21
University Paris Descartes, Paris, France.
22
Istituto Giannina Gaslini, Genova, Italy.

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.

KEYWORDS:

Fanconi anaemia; allogeneic transplant; inborn bone marrow failure syndrome; myelodysplasia

PMID:
29094350
DOI:
10.1111/bjh.15006
[Indexed for MEDLINE]

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