Bone marrow transplantation for diamond-blackfan anemia

Vivek Roy; Waleska S. Pérez; Mary Eapen; Judith C.W. Marsh; Marcelo Pasquini; Ricardo Pasquini; Mahmoud M. Mustafa; Christopher N. Bredeson; S. E. Ball; B. M. Camitta; R. P. Gale; T. G. Gross; G. A. Hale; J. T. Horan; J. M. Lipton; C. M. Niemeyer; P. J. Orchard

doi:10.1016/j.bbmt.2005.05.005

Bone marrow transplantation for diamond-blackfan anemia

Vivek Roy, Waleska S. Pérez, Mary Eapen, Judith C.W. Marsh, Marcelo Pasquini, Ricardo Pasquini, Mahmoud M. Mustafa, Christopher N. Bredeson, S. E. Ball, B. M. Camitta, R. P. Gale, T. G. Gross, G. A. Hale, J. T. Horan, J. M. Lipton, C. M. Niemeyer, P. J. Orchard

Pediatrics - Blood/Marrow Transplant

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

58 Scopus citations

Abstract

Patients with Diamond-Blackfan anemia (DBA) who are unresponsive to or intolerant of corticosteroids, experience treatment failure with other treatments, develop additional cytopenias or clonal disease, or opt for curative therapy are often treated with allogeneic bone marrow transplantation. We studied the transplantation outcomes of 61 DBA patients whose data were reported to the International Bone Marrow Transplant Registry between 1984 and 2000. The median age was 7 years (range, 1-32 years). Among 55 patients with available transfusion information, 35 (64%) had received ≥20 units of blood before transplantation. Most patients (67%) received their bone marrow grafts from an HLA-matched related donor. The median time to neutrophil recovery was 17 days (range, 10-119 days) and to platelet recovery was 23 days (range, 9-119 days). Five patients did not achieve neutrophil engraftment. The 100-day mortality was 18% (95% confidence interval, 10%-29%). Grade II to IV acute graft-versus-host disease occurred in 28% (range, 17%-39%) and chronic graft-versus-host disease in 26% (range, 15%-39%). The 3-year probability of overall survival was 64% (range, 50%-74%). In univariate analysis, a Karnofsky score ≥90 and transplantation from an HLA-identical sibling donor were associated with better survival. These data suggest that allogeneic bone marrow transplantation is effective for the treatment of DBA. Transplantation before deterioration of the performance status and from an HLA-identical sibling donor may improve survival.

Original language	English (US)
Pages (from-to)	600-608
Number of pages	9
Journal	Biology of Blood and Marrow Transplantation
Volume	11
Issue number	8
DOIs	https://doi.org/10.1016/j.bbmt.2005.05.005
State	Published - Aug 2005

Bibliographical note

Funding Information:
Supported by Public Health Service grant no. U24-CA76518 from the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and the National Heart, Lung and Blood Institute; Agency for Healthcare Research and Quality; and grants from Aetna; AIG Medical Excess; Allianz Life/Life Trac; American Red Cross; American Society of Clinical Oncology; Amgen, Inc.; an anonymous donation to the Medical College of Wisconsin; AnorMED, Inc.; Aventis Pharmaceuticals; Baxter Healthcare Corp.; Baxter Oncology; Berlex Laboratories, Inc.; Biogen IDEC, Inc.; Blue Cross and Blue Shield Association; The Lynde and Harry Bradley Foundation; BRT Laboratories, Inc.; Cedarlane Laboratories Ltd.; Celgene Corp.; Cell Pathways; Cell Therapeutics, Inc.; CelMed Biosciences; Centocor, Inc.; Cubist Pharmaceuticals; Dynal Biotech ASA; Edwards Lifesciences RMI; Endo Pharmaceuticals, Inc.; Enzon Pharmaceuticals, Inc.; ESP Pharma; Excess, Inc.; Fujisawa Healthcare, Inc.; Gambro BCT, Inc.; Genzyme; GlaxoSmithKline, Inc.; Human Genome Sciences; ICN Pharmaceuticals, Inc.; ILEX Oncology; Kirin Brewery Company; Ligand Pharmaceuticals, Inc.; Eli Lilly and Company; Nada and Herbert P. Mahler Charities; Merck & Company; Millennium Pharmaceuticals; Miller Pharmacal Group; Milliman USA, Inc.; Miltenyi Biotec; The Irving I. Moskowitz Foundation; National Leukemia Research Association; National Marrow Donor Program; NeoRx Corporation; Novartis Pharmaceuticals, Inc.; Novo Nordisk Pharmaceuticals; Ortho Biotech, Inc.; Osiris Therapeutics, Inc.; PacifiCare Health Systems; Pall Medical; Pfizer U.S. Pharmaceuticals; Pharmametrics; Pharmion Corp.; Protein Design Labs; QOL Medical; Roche Laboratories; Schering AG; StemCyte, Inc.; StemCell Technologies, Inc.; Stemco Biomedical; StemSoft Software, Inc.; SuperGen, Inc.; Sysmex; THERAKOS, a Johnson & Johnson Co.; University of Colorado Cord Blood Bank; Upside Endeavors; ViaCell, Inc.; ViaCor Biotechnologies; WB Saunders Mosby Churchill; Wellpoint Health Network; and Zymogenetics, Inc. The contents of this article are solely the responsibility of the authors and do not necessarily represent the official views of the National Cancer Institute.

Keywords

Bone marrow transplantation
Congenital anemia
Diamond-Blackfan anemia

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Roy, V., Pérez, W. S., Eapen, M., Marsh, J. C. W., Pasquini, M., Pasquini, R., Mustafa, M. M., Bredeson, C. N., Ball, S. E., Camitta, B. M., Gale, R. P., Gross, T. G., Hale, G. A., Horan, J. T., Lipton, J. M., Niemeyer, C. M., & Orchard, P. J. (2005). Bone marrow transplantation for diamond-blackfan anemia. Biology of Blood and Marrow Transplantation, 11(8), 600-608. https://doi.org/10.1016/j.bbmt.2005.05.005

Roy, V, Pérez, WS, Eapen, M, Marsh, JCW, Pasquini, M, Pasquini, R, Mustafa, MM, Bredeson, CN, Ball, SE, Camitta, BM, Gale, RP, Gross, TG, Hale, GA, Horan, JT, Lipton, JM, Niemeyer, CM & Orchard, PJ 2005, 'Bone marrow transplantation for diamond-blackfan anemia', Biology of Blood and Marrow Transplantation, vol. 11, no. 8, pp. 600-608. https://doi.org/10.1016/j.bbmt.2005.05.005

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abstract = "Patients with Diamond-Blackfan anemia (DBA) who are unresponsive to or intolerant of corticosteroids, experience treatment failure with other treatments, develop additional cytopenias or clonal disease, or opt for curative therapy are often treated with allogeneic bone marrow transplantation. We studied the transplantation outcomes of 61 DBA patients whose data were reported to the International Bone Marrow Transplant Registry between 1984 and 2000. The median age was 7 years (range, 1-32 years). Among 55 patients with available transfusion information, 35 (64%) had received ≥20 units of blood before transplantation. Most patients (67%) received their bone marrow grafts from an HLA-matched related donor. The median time to neutrophil recovery was 17 days (range, 10-119 days) and to platelet recovery was 23 days (range, 9-119 days). Five patients did not achieve neutrophil engraftment. The 100-day mortality was 18% (95% confidence interval, 10%-29%). Grade II to IV acute graft-versus-host disease occurred in 28% (range, 17%-39%) and chronic graft-versus-host disease in 26% (range, 15%-39%). The 3-year probability of overall survival was 64% (range, 50%-74%). In univariate analysis, a Karnofsky score ≥90 and transplantation from an HLA-identical sibling donor were associated with better survival. These data suggest that allogeneic bone marrow transplantation is effective for the treatment of DBA. Transplantation before deterioration of the performance status and from an HLA-identical sibling donor may improve survival.",

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note = "Funding Information: Supported by Public Health Service grant no. U24-CA76518 from the National Cancer Institute, the National Institute of Allergy and Infectious Diseases, and the National Heart, Lung and Blood Institute; Agency for Healthcare Research and Quality; and grants from Aetna; AIG Medical Excess; Allianz Life/Life Trac; American Red Cross; American Society of Clinical Oncology; Amgen, Inc.; an anonymous donation to the Medical College of Wisconsin; AnorMED, Inc.; Aventis Pharmaceuticals; Baxter Healthcare Corp.; Baxter Oncology; Berlex Laboratories, Inc.; Biogen IDEC, Inc.; Blue Cross and Blue Shield Association; The Lynde and Harry Bradley Foundation; BRT Laboratories, Inc.; Cedarlane Laboratories Ltd.; Celgene Corp.; Cell Pathways; Cell Therapeutics, Inc.; CelMed Biosciences; Centocor, Inc.; Cubist Pharmaceuticals; Dynal Biotech ASA; Edwards Lifesciences RMI; Endo Pharmaceuticals, Inc.; Enzon Pharmaceuticals, Inc.; ESP Pharma; Excess, Inc.; Fujisawa Healthcare, Inc.; Gambro BCT, Inc.; Genzyme; GlaxoSmithKline, Inc.; Human Genome Sciences; ICN Pharmaceuticals, Inc.; ILEX Oncology; Kirin Brewery Company; Ligand Pharmaceuticals, Inc.; Eli Lilly and Company; Nada and Herbert P. Mahler Charities; Merck & Company; Millennium Pharmaceuticals; Miller Pharmacal Group; Milliman USA, Inc.; Miltenyi Biotec; The Irving I. Moskowitz Foundation; National Leukemia Research Association; National Marrow Donor Program; NeoRx Corporation; Novartis Pharmaceuticals, Inc.; Novo Nordisk Pharmaceuticals; Ortho Biotech, Inc.; Osiris Therapeutics, Inc.; PacifiCare Health Systems; Pall Medical; Pfizer U.S. Pharmaceuticals; Pharmametrics; Pharmion Corp.; Protein Design Labs; QOL Medical; Roche Laboratories; Schering AG; StemCyte, Inc.; StemCell Technologies, Inc.; Stemco Biomedical; StemSoft Software, Inc.; SuperGen, Inc.; Sysmex; THERAKOS, a Johnson & Johnson Co.; University of Colorado Cord Blood Bank; Upside Endeavors; ViaCell, Inc.; ViaCor Biotechnologies; WB Saunders Mosby Churchill; Wellpoint Health Network; and Zymogenetics, Inc. The contents of this article are solely the responsibility of the authors and do not necessarily represent the official views of the National Cancer Institute. ",

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AU - Pasquini, Ricardo

AU - Mustafa, Mahmoud M.

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AU - Camitta, B. M.

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AU - Gross, T. G.

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N2 - Patients with Diamond-Blackfan anemia (DBA) who are unresponsive to or intolerant of corticosteroids, experience treatment failure with other treatments, develop additional cytopenias or clonal disease, or opt for curative therapy are often treated with allogeneic bone marrow transplantation. We studied the transplantation outcomes of 61 DBA patients whose data were reported to the International Bone Marrow Transplant Registry between 1984 and 2000. The median age was 7 years (range, 1-32 years). Among 55 patients with available transfusion information, 35 (64%) had received ≥20 units of blood before transplantation. Most patients (67%) received their bone marrow grafts from an HLA-matched related donor. The median time to neutrophil recovery was 17 days (range, 10-119 days) and to platelet recovery was 23 days (range, 9-119 days). Five patients did not achieve neutrophil engraftment. The 100-day mortality was 18% (95% confidence interval, 10%-29%). Grade II to IV acute graft-versus-host disease occurred in 28% (range, 17%-39%) and chronic graft-versus-host disease in 26% (range, 15%-39%). The 3-year probability of overall survival was 64% (range, 50%-74%). In univariate analysis, a Karnofsky score ≥90 and transplantation from an HLA-identical sibling donor were associated with better survival. These data suggest that allogeneic bone marrow transplantation is effective for the treatment of DBA. Transplantation before deterioration of the performance status and from an HLA-identical sibling donor may improve survival.

AB - Patients with Diamond-Blackfan anemia (DBA) who are unresponsive to or intolerant of corticosteroids, experience treatment failure with other treatments, develop additional cytopenias or clonal disease, or opt for curative therapy are often treated with allogeneic bone marrow transplantation. We studied the transplantation outcomes of 61 DBA patients whose data were reported to the International Bone Marrow Transplant Registry between 1984 and 2000. The median age was 7 years (range, 1-32 years). Among 55 patients with available transfusion information, 35 (64%) had received ≥20 units of blood before transplantation. Most patients (67%) received their bone marrow grafts from an HLA-matched related donor. The median time to neutrophil recovery was 17 days (range, 10-119 days) and to platelet recovery was 23 days (range, 9-119 days). Five patients did not achieve neutrophil engraftment. The 100-day mortality was 18% (95% confidence interval, 10%-29%). Grade II to IV acute graft-versus-host disease occurred in 28% (range, 17%-39%) and chronic graft-versus-host disease in 26% (range, 15%-39%). The 3-year probability of overall survival was 64% (range, 50%-74%). In univariate analysis, a Karnofsky score ≥90 and transplantation from an HLA-identical sibling donor were associated with better survival. These data suggest that allogeneic bone marrow transplantation is effective for the treatment of DBA. Transplantation before deterioration of the performance status and from an HLA-identical sibling donor may improve survival.

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Bone marrow transplantation for diamond-blackfan anemia

Abstract

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