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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: The European Group for Blood and Marrow Transplantation experience
Blood, Volume 122, No. 26, Year 2013
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Description
Although allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for patients with Fanconi anemia (FA), published series mostly refer to single-center experience with limited numbers of patients. We analyzed results in 795 patients with FA who underwent first HSCT between May 1972 and January 2010. With a 6-year median follow-up, overall survival was 49% at 20 years (95% confidence interval, 38-65 years). Better outcome was observed for patients transplanted before the age of 10 years, before clonal evolution (ie, myelodysplastic syndrome or acute myeloid leukemia), from a matched family donor, after a conditioning regimen without irradiation, the latter including fludarabine. Chronic graft-versus-host disease and secondary malignancy were deleterious when considered as time-dependent covariates. Age more than 10 years at time of HSCT, clonal evolution as an indication for transplantation, peripheral blood as source of stem cells, and chronic graft-versus-host disease were found to be independently associated with the risk for secondary malignancy. Changes in transplant protocols have significantly improved the outcome of patients with FA, who should be transplanted at a young age, with bone marrow as the source of stem cells. © 2013 by The American Society of Hematology.
Authors & Co-Authors
de Latour, Régis Peffault
France, Paris
Hôpital Saint-louis
Porcher, Raphaël
France, Paris
Hôpital Saint-louis
Dalle, Jean Hugues H.
France, Paris
Hôpital Robert-debré Ap-hp
Aljurf, Mahmoud Deeb
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Korthof, Elisabeth T.
Netherlands, Leiden
Leids Universitair Medisch Centrum
Svahn, Johanna
Italy, Genoa
Istituto Giannina Gaslini
Willemze, Roelof
Netherlands, Leiden
Leids Universitair Medisch Centrum
Barrenetxea, Cristina
Spain, Barcelona
Hospital Universitari Vall D'hebron
Mialou, Valérie
France, Lyon
Institut D'hématologie et Oncologie Pédiatrique
Soulier, Jean
France, Paris
Hôpital Saint-louis
Ayas, Mouhab Fakhreddine
Saudi Arabia, Riyadh
King Faisal Specialist Hospital and Research Centre
Oneto, Rosi
Italy, Genoa
Irccs San Martino Polyclinic Hospital
Bacigalupo, Andrea A.
Italy, Genoa
Irccs San Martino Polyclinic Hospital
Marsh, Judith C.W.
United Kingdom, London
King's College London
Peters, Christina
Austria, Vienna
St Anna Kinderspital, Vienna
Socié, Gérard A.
France, Paris
Hôpital Saint-louis
France, Paris
Université Paris Cité
Dufour, Carlo
Italy, Genoa
Istituto Giannina Gaslini
Statistics
Citations: 186
Authors: 17
Affiliations: 11
Identifiers
Doi:
10.1182/blood-2013-01-479733
ISSN:
00064971
Research Areas
Cancer
Study Design
Cohort Study