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Transplant results in adults with Fanconi anaemia

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Transplant results in adults with Fanconi anaemia. / Bierings, Marc; Bonfim, Carmem; Peffault De Latour, Regis; Aljurf, Mahmoud; Mehta, Parinda A; Knol, Cora; Boulad, Farid; Tbakhi, Abdelghani; Esquirol, Albert; McQuaker, Grant; Sucak, Gulsan A; Othman, Tarek B; Halkes, Constantijn J M; Carpenter, Ben; Niederwieser, Dietger; Zecca, Marco; Kröger, Nicolaus; Michallet, Mauricette; Risitano, Antonio M; Ehninger, Gerhard; Porcher, Raphael; Dufour, Carlo; EBMT SAA WP.

in: BRIT J HAEMATOL, Jahrgang 180, Nr. 1, 01.2018, S. 100-109.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

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, Porcher, R, Dufour, C & EBMT SAA WP 2018, 'Transplant results in adults with Fanconi anaemia', BRIT J HAEMATOL, Jg. 180, Nr. 1, S. 100-109. https://doi.org/10.1111/bjh.15006

APA

Bierings, M., Bonfim, C., Peffault De Latour, R., Aljurf, M., Mehta, P. A., Knol, C., Boulad, F., Tbakhi, A., Esquirol, A., McQuaker, G., Sucak, G. A., Othman, T. B., Halkes, C. J. M., Carpenter, B., Niederwieser, D., Zecca, M., Kröger, N., Michallet, M., Risitano, A. M., ... EBMT SAA WP (2018). Transplant results in adults with Fanconi anaemia. BRIT J HAEMATOL, 180(1), 100-109. https://doi.org/10.1111/bjh.15006

Vancouver

Bierings M, Bonfim C, Peffault De Latour R, Aljurf M, Mehta PA, Knol C et al. Transplant results in adults with Fanconi anaemia. BRIT J HAEMATOL. 2018 Jan;180(1):100-109. https://doi.org/10.1111/bjh.15006

Bibtex

@article{815e7aca92d34e8fa1833743af2c41c6,
title = "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.",
keywords = "Journal Article",
author = "Marc Bierings and Carmem Bonfim and {Peffault De Latour}, Regis and Mahmoud Aljurf and Mehta, {Parinda A} and Cora Knol and Farid Boulad and Abdelghani Tbakhi and Albert Esquirol and Grant McQuaker and Sucak, {Gulsan A} and Othman, {Tarek B} and Halkes, {Constantijn J M} and Ben Carpenter and Dietger Niederwieser and Marco Zecca and Nicolaus Kr{\"o}ger and Mauricette Michallet and Risitano, {Antonio M} and Gerhard Ehninger and Raphael Porcher and Carlo Dufour and {EBMT SAA WP}",
note = "{\textcopyright} 2017 John Wiley & Sons Ltd.",
year = "2018",
month = jan,
doi = "10.1111/bjh.15006",
language = "English",
volume = "180",
pages = "100--109",
journal = "BRIT J HAEMATOL",
issn = "0007-1048",
publisher = "Wiley-Blackwell",
number = "1",

}

RIS

TY - JOUR

T1 - Transplant results in adults with Fanconi anaemia

AU - Bierings, Marc

AU - Bonfim, Carmem

AU - Peffault De Latour, Regis

AU - Aljurf, Mahmoud

AU - Mehta, Parinda A

AU - Knol, Cora

AU - Boulad, Farid

AU - Tbakhi, Abdelghani

AU - Esquirol, Albert

AU - McQuaker, Grant

AU - Sucak, Gulsan A

AU - Othman, Tarek B

AU - Halkes, Constantijn J M

AU - Carpenter, Ben

AU - Niederwieser, Dietger

AU - Zecca, Marco

AU - Kröger, Nicolaus

AU - Michallet, Mauricette

AU - Risitano, Antonio M

AU - Ehninger, Gerhard

AU - Porcher, Raphael

AU - Dufour, Carlo

AU - EBMT SAA WP

N1 - © 2017 John Wiley & Sons Ltd.

PY - 2018/1

Y1 - 2018/1

N2 - 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.

AB - 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.

KW - Journal Article

U2 - 10.1111/bjh.15006

DO - 10.1111/bjh.15006

M3 - SCORING: Journal article

C2 - 29094350

VL - 180

SP - 100

EP - 109

JO - BRIT J HAEMATOL

JF - BRIT J HAEMATOL

SN - 0007-1048

IS - 1

ER -