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HLA-matched sibling hematopoietic stem cell transplantation for fanconi anemia: comparison of irradiation and nonirradiation containing conditioning regimens. Biol Blood Marrow Transplant 2008 Oct;14(10):1141-1147 PMID: 18804044 PMCID: PMC2581414

Pubmed ID

18804044

DOI

10.1016/j.bbmt.2008.06.020

Abstract

Related to the underlying DNA repair defect that is the hallmark of Fanconi anemia (FA), preparatory regimen-related toxicities have been obstacles to hematopoietic cell transplantation (HCT). In an attempt to decrease the risk and severity of regimen-related toxicities, nonirradiation regimens have been explored. The aim of this study is to compare outcomes after irradiation and nonirradiation regimens in 148 FA patients and identify risk factors impacting upon HCT outcomes. Hematopoietic recovery, acute and chronic graft-versus-host disease (aGVHD, GVHD), and mortality were similar after irradiation and nonirradiation regimens. In both groups of recipients aged >10 years, prior use of androgens and cytomegalovirus seropositivity in either the donor or recipient were associated with higher mortality. With median follow-ups >5 years, the 5-year probability of overall survival, adjusted for factors impacting overall mortality was 78% and 81% after irradiation and nonirradiation regimens, P = .61. In view of the high risk of cancer and other radiation-related effects on growth and development, these results support the use of nonirradiation preparatory regimens. As the peak time for developing solid tumors after HCT is 8 to 9 years, longer follow-up is required before definitive statements can be made regarding the impact of nonirradiation regimens on cancer risk.

Author List

Pasquini R, Carreras J, Pasquini MC, Camitta BM, Fasth AL, Hale GA, Harris RE, Marsh JC, Robinson AJ, Zhang MJ, Eapen M, Wagner JE

Authors

Bruce M. Camitta MD Clinical Professor in the Medicine department at Medical College of Wisconsin
Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Marcelo C. Pasquini MD, MS Associate Professor in the Medicine department at Medical College of Wisconsin
Mei-Jie Zhang PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin




Scopus

2-s2.0-51649094272   41 Citations

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

Adolescent
Adult
Fanconi Anemia
Female
Graft vs Host Disease
Hematopoiesis
Hematopoietic Stem Cell Transplantation
Histocompatibility Testing
Humans
Male
Radiotherapy
Registries
Retrospective Studies
Risk Factors
Siblings
Survival Analysis
Transplantation Conditioning
Treatment Outcome
jenkins-FCD Prod-353 9ccd8489072cb19f5b9f808bb23ed672c582f41e