Faculty Collaboration Database

Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. J Clin Oncol 2013 May 01;31(13):1669-76

Date

04/03/2013

Pubmed ID

23547077

Pubmed Central ID

PMC3635221

DOI

10.1200/JCO.2012.45.9719

Scopus ID

2-s2.0-84884540764 (requires institutional sign-in at Scopus site)   61 Citations

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 List

Ayas M, Saber W, Davies SM, Harris RE, Hale GA, Socie G, LeRademacher J, Thakar M, Deeg HJ, Al-Seraihy A, Battiwalla M, Camitta BM, Olsson R, Bajwa RS, Bonfim CM, Pasquini R, Macmillan ML, George B, Copelan EA, Wirk B, Al Jefri A, Fasth AL, Guinan EC, Horn BN, Lewis VA, Slavin S, Stepensky P, Bierings M, Gale RP

Author

Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Child
Child, Preschool
Cytogenetic Analysis
Fanconi Anemia
Female
Hematopoietic Stem Cell Transplantation
Humans
Male
Middle Aged
Myelodysplastic Syndromes
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Risk Factors
Survival Analysis
Transplantation Conditioning
Transplantation, Homologous
Treatment Outcome
Young Adult