Transplant results in adults with Fanconi anaemia
Por:
Bierings, M, Bonfim, C, De Latour, RP, 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, Kroger, N, Michallet, M, Risitano, AM, Ehninger, G, Porcher, R, Dufour, C, EBMT SAA WP
Publicada:
1 ene 2018
Resumen:
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 16years of age when diagnosed with FA, and underwent transplantation at a median age of 23years. Time between diagnosis and transplant was shortest (median 2years) 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 96months was 26% (95% CI 20-33). Non-relapse mortality at 96months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58months. Patients transplanted after 2000 had improved survival (84% at 36months), 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.
Filiaciones:
Bierings, M:
Univ Utrecht, Childrens Hosp, Dept Haematol & Stem Cell Transplantat, Utrecht, Netherlands
Bonfim, C:
Univ Fed Parana, Curitiba, Parana, Brazil
De Latour, RP:
Hosp St Louis, French Reference Ctr Aplast Anaemia, BMT Unit, Paris, France
Aljurf, M:
King Faisal Specialist Hosp & Res Ctr, Riyadh, Saudi Arabia
Mehta, PA:
Cincinnati Childrens Hosp Med Ctr, Div Bone Marrow Transplant & Immune Deficiency, Cincinnati, OH 45229 USA
Knol, C:
EBMT Data Off, Leiden, Netherlands
Boulad, F:
Paediat Bone Marrow Transplant Serv, Mem Sloan Kettering, New York, NY USA
Tbakhi, A:
King Hussein Canc Ctr, Amman, Jordan
Esquirol, A:
Hosp Santa Creu I Sant Pau, Barcelona, Spain
McQuaker, G:
Beatson West Scotland Canc Ctr, Glasgow, Lanark, Scotland
Sucak, GA:
Gazi Univ, Ankara, Turkey
Othman, TB:
Ctr Natl Greffe Moelle, Tunis, Tunisia
Halkes, CJM:
Leiden Univ, Med Ctr, Dept Haematol, Leiden, Netherlands
Carpenter, B:
Univ Coll London Hosp, London, England
Niederwieser, D:
Univ Leipzig, Haematol & Oncol, Leipzig, Germany
Zecca, M:
Fondazione IRCCS Policlin San Matteo, Paediat Haematol Oncol, Pavia, Italy
Kroger, N:
Univ Hosp Eppendorf, Hamburg, Germany
Michallet, M:
Ctr Hosp Lyon Sud, Lyon, France
Risitano, AM:
Univ Napoli, Naples, Italy
Ehninger, G:
Univ Klinikum Dresden, Dresden, Germany
Porcher, R:
Univ Paris, Paris, France
Dufour, C:
Ist Giannina Gaslini, Genoa, Italy
Bronze
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