HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens

Ricardo Pasquini; Jeanette Carreras; Marcelo C. Pasquini; Bruce M. Camitta; Anders L. Fasth; Gregory A. Hale; Richard E. Harris; Judith C. Marsh; Anibal J. Robinson; Mei Jie Zhang; Mary Eapen; John E. Wagner

doi:10.1016/j.bbmt.2008.06.020

HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens

Ricardo Pasquini, Jeanette Carreras, Marcelo C. Pasquini, Bruce M. Camitta, Anders L. Fasth, Gregory A. Hale, Richard E. Harris, Judith C. Marsh, Anibal J. Robinson, Mei Jie Zhang, Mary Eapen, John E. Wagner

Research output: Contribution to journal › Article › peer-review

54 Scopus citations

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.

Original language	English (US)
Pages (from-to)	1141-1147
Number of pages	7
Journal	Biology of Blood and Marrow Transplantation
Volume	14
Issue number	10
DOIs	https://doi.org/10.1016/j.bbmt.2008.06.020
State	Published - Oct 2008

Bibliographical note

Funding Information:
Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U01HL069294 from NHLBI and NCI; a contract HHSH234200637015C with Health Resources and Services Administration (HRSA/DHHS); 2 grants (N00014-06-1-0704 and N00014-08-1-0058) from the Office of Naval Research; and grants from AABB; Aetna; American Society for Blood and Marrow Transplantation; Amgen, Inc.; Anonymous donation to the Medical College of Wisconsin; Association of Medical Microbiology and Infectious Disease Canada; Astellas Pharma US, Inc.; Baxter International, Inc.; Bayer HealthCare Pharmaceuticals; BloodCenter of Wisconsin; Blue Cross and Blue Shield Association; Bone Marrow Foundation; Canadian Blood and Marrow Transplant Group; Celgene Corporation; CellGenix, GmbH; Centers for Disease Control and Prevention; ClinImmune Labs; CTI Clinical Trial and Consulting Services; Cubist Pharmaceuticals; Cylex Inc.; CytoTherm; DOR BioPharma, Inc.; Dynal Biotech, an Invitrogen Company; Enzon Pharmaceuticals, Inc.; European Group for Blood and Marrow Transplantation; Gambro BCT, Inc.; Gamida Cell, Ltd.; Genzyme Corporation; Histogenetics, Inc.; HKS Medical Information Systems; Hospira, Inc.; Infectious Diseases Society of America; Kiadis Pharma; Kirin Brewery Co., Ltd.; Merck & Company; The Medical College of Wisconsin; MGI Pharma, Inc.; Michigan Community Blood Centers; Millennium Pharmaceuticals, Inc.; Miller Pharmacal Group; Milliman USA, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Nature Publishing Group; New York Blood Center; Novartis Oncology; Oncology Nursing Society; Osiris Therapeutics, Inc.; Otsuka Pharmaceutical Development & Commercialization, Inc.; Pall Life Sciences; PDL BioPharma, Inc; Pfizer Inc; Pharmion Corporation; Saladax Biomedical, Inc.; Schering Plough Corporation; Society for Healthcare Epidemiology of America; StemCyte, Inc.; StemSoft Software, Inc.; Sysmex; Teva Pharmaceutical Industries; The Marrow Foundation; THERAKOS, Inc.; Vidacare Corporation; Vion Pharmaceuticals, Inc.; ViraCor Laboratories; ViroPharma, Inc.; and Wellpoint, Inc.

Keywords

Conditioning regimen
Fanconi anemia
Graft-versus-host disease
Matched sibling donor
Survival

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1016/j.bbmt.2008.06.020

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Pasquini, R., Carreras, J., Pasquini, M. C., Camitta, B. M., Fasth, A. L., Hale, G. A., Harris, R. E., Marsh, J. C., Robinson, A. J., Zhang, M. J., Eapen, M., & Wagner, J. E. (2008). HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens. Biology of Blood and Marrow Transplantation, 14(10), 1141-1147. https://doi.org/10.1016/j.bbmt.2008.06.020

HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia : Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens. / Pasquini, Ricardo; Carreras, Jeanette; Pasquini, Marcelo C. et al.

In: Biology of Blood and Marrow Transplantation, Vol. 14, No. 10, 10.2008, p. 1141-1147.

Research output: Contribution to journal › Article › peer-review

Pasquini, R, Carreras, J, Pasquini, MC, Camitta, BM, Fasth, AL, Hale, GA, Harris, RE, Marsh, JC, Robinson, AJ, Zhang, MJ, Eapen, M & Wagner, JE 2008, 'HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens', Biology of Blood and Marrow Transplantation, vol. 14, no. 10, pp. 1141-1147. https://doi.org/10.1016/j.bbmt.2008.06.020

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title = "HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens",

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.",

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author = "Ricardo Pasquini and Jeanette Carreras and Pasquini, {Marcelo C.} and Camitta, {Bruce M.} and Fasth, {Anders L.} and Hale, {Gregory A.} and Harris, {Richard E.} and Marsh, {Judith C.} and Robinson, {Anibal J.} and Zhang, {Mei Jie} and Mary Eapen and Wagner, {John E.}",

note = "Funding Information: Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement U24-CA76518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U01HL069294 from NHLBI and NCI; a contract HHSH234200637015C with Health Resources and Services Administration (HRSA/DHHS); 2 grants (N00014-06-1-0704 and N00014-08-1-0058) from the Office of Naval Research; and grants from AABB; Aetna; American Society for Blood and Marrow Transplantation; Amgen, Inc.; Anonymous donation to the Medical College of Wisconsin; Association of Medical Microbiology and Infectious Disease Canada; Astellas Pharma US, Inc.; Baxter International, Inc.; Bayer HealthCare Pharmaceuticals; BloodCenter of Wisconsin; Blue Cross and Blue Shield Association; Bone Marrow Foundation; Canadian Blood and Marrow Transplant Group; Celgene Corporation; CellGenix, GmbH; Centers for Disease Control and Prevention; ClinImmune Labs; CTI Clinical Trial and Consulting Services; Cubist Pharmaceuticals; Cylex Inc.; CytoTherm; DOR BioPharma, Inc.; Dynal Biotech, an Invitrogen Company; Enzon Pharmaceuticals, Inc.; European Group for Blood and Marrow Transplantation; Gambro BCT, Inc.; Gamida Cell, Ltd.; Genzyme Corporation; Histogenetics, Inc.; HKS Medical Information Systems; Hospira, Inc.; Infectious Diseases Society of America; Kiadis Pharma; Kirin Brewery Co., Ltd.; Merck & Company; The Medical College of Wisconsin; MGI Pharma, Inc.; Michigan Community Blood Centers; Millennium Pharmaceuticals, Inc.; Miller Pharmacal Group; Milliman USA, Inc.; Miltenyi Biotec, Inc.; National Marrow Donor Program; Nature Publishing Group; New York Blood Center; Novartis Oncology; Oncology Nursing Society; Osiris Therapeutics, Inc.; Otsuka Pharmaceutical Development & Commercialization, Inc.; Pall Life Sciences; PDL BioPharma, Inc; Pfizer Inc; Pharmion Corporation; Saladax Biomedical, Inc.; Schering Plough Corporation; Society for Healthcare Epidemiology of America; StemCyte, Inc.; StemSoft Software, Inc.; Sysmex; Teva Pharmaceutical Industries; The Marrow Foundation; THERAKOS, Inc.; Vidacare Corporation; Vion Pharmaceuticals, Inc.; ViraCor Laboratories; ViroPharma, Inc.; and Wellpoint, Inc. ",

year = "2008",

month = oct,

doi = "10.1016/j.bbmt.2008.06.020",

language = "English (US)",

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AU - Camitta, Bruce M.

AU - Fasth, Anders L.

AU - Hale, Gregory A.

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AU - Eapen, Mary

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AB - 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.

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KW - Matched sibling donor

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HLA-Matched Sibling Hematopoietic Stem Cell Transplantation for Fanconi Anemia: Comparison of Irradiation and Nonirradiation Containing Conditioning Regimens

Abstract

Bibliographical note

Keywords

UN SDGs

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Find It

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