Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia

Mouhab Ayas, Wael Saber, Stella M. Davies, Richard E. Harris, Gregory A. Hale, Gerard Socie, Jennifer Le-Rademacher, Monica Thakar, H. Joachim J. Deeg, Amal Al-Seraihy, Minoo Battiwalla, Bruce M. Camitta, Richard Olsson, Rajinder S. Bajwa, Carmem M. Bonfim, Ricardo Pasquini, Margaret L. MacMillan, Biju George, Edward A. Copelan, Baldeep Wirk & 9 others Abdullah Al Jefri, Anders L. Fasth, Eva C. Guinan, Biljana N. Horn, Victor A. Lewis, Shimon Slavin, Polina Stepensky, Marc Bierings, Robert Peter Gale

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Abstract

Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.

Original languageEnglish (US)
Pages (from-to)1669-1676
Number of pages8
JournalJournal of Clinical Oncology
Volume31
Issue number13
DOIs
StatePublished - May 1 2013
Externally publishedYes

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Fanconi Anemia
Myelodysplastic Syndromes
Cell Transplantation
Chromosome Aberrations
Leukemia
Survival
Transplantation
Bone Marrow
Tissue Donors
Dilatation and Curettage
Graft vs Host Disease
Neutrophils
Transplants

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. / Ayas, Mouhab; Saber, Wael; Davies, Stella M.; Harris, Richard E.; Hale, Gregory A.; Socie, Gerard; Le-Rademacher, Jennifer; Thakar, Monica; Deeg, H. Joachim J.; Al-Seraihy, Amal; Battiwalla, Minoo; Camitta, Bruce M.; Olsson, Richard; Bajwa, Rajinder S.; Bonfim, Carmem M.; Pasquini, Ricardo; MacMillan, Margaret L.; George, Biju; Copelan, Edward A.; Wirk, Baldeep; Jefri, Abdullah Al; Fasth, Anders L.; Guinan, Eva C.; Horn, Biljana N.; Lewis, Victor A.; Slavin, Shimon; Stepensky, Polina; Bierings, Marc; Gale, Robert Peter.

In: Journal of Clinical Oncology, Vol. 31, No. 13, 01.05.2013, p. 1669-1676.

Research output: Contribution to journalArticle

Ayas, M, Saber, W, Davies, SM, Harris, RE, Hale, GA, Socie, G, Le-Rademacher, J, Thakar, M, Deeg, HJJ, Al-Seraihy, A, Battiwalla, M, Camitta, BM, Olsson, R, Bajwa, RS, Bonfim, CM, Pasquini, R, MacMillan, ML, George, B, Copelan, EA, Wirk, B, Jefri, AA, Fasth, AL, Guinan, EC, Horn, BN, Lewis, VA, Slavin, S, Stepensky, P, Bierings, M & Gale, RP 2013, 'Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia', Journal of Clinical Oncology, vol. 31, no. 13, pp. 1669-1676. https://doi.org/10.1200/JCO.2012.45.9719
Ayas, Mouhab ; Saber, Wael ; Davies, Stella M. ; Harris, Richard E. ; Hale, Gregory A. ; Socie, Gerard ; Le-Rademacher, Jennifer ; Thakar, Monica ; Deeg, H. Joachim J. ; Al-Seraihy, Amal ; Battiwalla, Minoo ; Camitta, Bruce M. ; Olsson, Richard ; Bajwa, Rajinder S. ; Bonfim, Carmem M. ; Pasquini, Ricardo ; MacMillan, Margaret L. ; George, Biju ; Copelan, Edward A. ; Wirk, Baldeep ; Jefri, Abdullah Al ; Fasth, Anders L. ; Guinan, Eva C. ; Horn, Biljana N. ; Lewis, Victor A. ; Slavin, Shimon ; Stepensky, Polina ; Bierings, Marc ; Gale, Robert Peter. / Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. In: Journal of Clinical Oncology. 2013 ; Vol. 31, No. 13. pp. 1669-1676.
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title = "Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia",
abstract = "Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78{\%} and 85{\%} of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26{\%} (95{\%} CI, 19{\%} to 35{\%}) and 12{\%} (95{\%} CI, 7{\%} to 19{\%}), respectively. Survival probabilities at 1, 3, and 5 years were 64{\%} (95{\%} CI, 55{\%} to 73{\%}), 58{\%} (95{\%} CI, 48{\%} to 67{\%}), and 55{\%} (95{\%} CI, 45{\%} to 64{\%}), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69{\%} [95{\%} CI, 57{\%} to 80{\%}] v 39{\%} [95{\%} CI, 26{\%} to 53{\%}], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78{\%} [95{\%} CI, 64{\%} to 90{\%}] v 34{\%} [95{\%} CI, 20{\%} to 50{\%}], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67{\%} [95{\%} CI, 52{\%} to 81{\%}] v 43{\%} [95{\%} CI, 27{\%} to 59{\%}], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.",
author = "Mouhab Ayas and Wael Saber and Davies, {Stella M.} and Harris, {Richard E.} and Hale, {Gregory A.} and Gerard Socie and Jennifer Le-Rademacher and Monica Thakar and Deeg, {H. Joachim J.} and Amal Al-Seraihy and Minoo Battiwalla and Camitta, {Bruce M.} and Richard Olsson and Bajwa, {Rajinder S.} and Bonfim, {Carmem M.} and Ricardo Pasquini and MacMillan, {Margaret L.} and Biju George and Copelan, {Edward A.} and Baldeep Wirk and Jefri, {Abdullah Al} and Fasth, {Anders L.} and Guinan, {Eva C.} and Horn, {Biljana N.} and Lewis, {Victor A.} and Shimon Slavin and Polina Stepensky and Marc Bierings and Gale, {Robert Peter}",
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TY - JOUR

T1 - Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia

AU - Ayas, Mouhab

AU - Saber, Wael

AU - Davies, Stella M.

AU - Harris, Richard E.

AU - Hale, Gregory A.

AU - Socie, Gerard

AU - Le-Rademacher, Jennifer

AU - Thakar, Monica

AU - Deeg, H. Joachim J.

AU - Al-Seraihy, Amal

AU - Battiwalla, Minoo

AU - Camitta, Bruce M.

AU - Olsson, Richard

AU - Bajwa, Rajinder S.

AU - Bonfim, Carmem M.

AU - Pasquini, Ricardo

AU - MacMillan, Margaret L.

AU - George, Biju

AU - Copelan, Edward A.

AU - Wirk, Baldeep

AU - Jefri, Abdullah Al

AU - Fasth, Anders L.

AU - Guinan, Eva C.

AU - Horn, Biljana N.

AU - Lewis, Victor A.

AU - Slavin, Shimon

AU - Stepensky, Polina

AU - Bierings, Marc

AU - Gale, Robert Peter

PY - 2013/5/1

Y1 - 2013/5/1

N2 - Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.

AB - Purpose Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed. Patients and Methods We analyzed data on 113 patients with FA with cytogenetic abnormalities (n 54), MDS (n 45), or acute leukemia (n 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007. Results Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( v 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P .001). In transplantations from HLA-matched related donors (n 82), younger patients ( v 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P .03) had superior 5-year survival. Conclusion Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.

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DO - 10.1200/JCO.2012.45.9719

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JF - Journal of Clinical Oncology

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