Bone marrow transplantation from HLA-identical siblings as treatment for myelodysplasia

Allogeneic hematopoietic stem cell transplantation is the only curative therapy for myelodysplasia (MDS). To identify factors influencing transplantation outcome, we studied 452 recipients of HLA-identical sibling transplants for MDS from 1989 to 1997, reported to the International Bone Marrow Trans...

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Veröffentlicht in:Blood 2002-09, Vol.100 (6), p.1997-2004
Hauptverfasser: Sierra, Jorge, Pérez, Waleska S., Rozman, Ciril, Carreras, Enric, Klein, John P., Rizzo, J. Douglas, Davies, Stella M., Lazarus, Hillard M., Bredeson, Christopher N., Marks, David I., Canals, Carmen, Boogaerts, Marc A., Goldman, John, Champlin, Richard E., Keating, Armand, Weisdorf, Daniel J., de Witte, Theo M., Horowitz, Mary M.
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container_end_page 2004
container_issue 6
container_start_page 1997
container_title Blood
container_volume 100
creator Sierra, Jorge
Pérez, Waleska S.
Rozman, Ciril
Carreras, Enric
Klein, John P.
Rizzo, J. Douglas
Davies, Stella M.
Lazarus, Hillard M.
Bredeson, Christopher N.
Marks, David I.
Canals, Carmen
Boogaerts, Marc A.
Goldman, John
Champlin, Richard E.
Keating, Armand
Weisdorf, Daniel J.
de Witte, Theo M.
Horowitz, Mary M.
description Allogeneic hematopoietic stem cell transplantation is the only curative therapy for myelodysplasia (MDS). To identify factors influencing transplantation outcome, we studied 452 recipients of HLA-identical sibling transplants for MDS from 1989 to 1997, reported to the International Bone Marrow Transplant Registry. Patients with treatment-related MDS or unclassified MDS were excluded. Median age was 38 years (range, 2-64 years). Sixty percent had refractory anemia with excess blasts (n = 136) or with excess blasts in transformation (n = 136). Conditioning regimens included total body irradiation in 199 (44%) cases. Marrow was T-cell depleted for 58 (13%) transplants. Cumulative incidences of neutrophil engraftment, grades II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were 91% (95% confidence interval [CI], 88%-93%), 36% (95% CI, 31%-40%), and 39% (95% CI, 33%-44%), respectively. Three-year transplantation-related mortality (TRM), relapse, disease-free survival, and overall survival rates were 37% (95% CI, 32%-42%), 23% (95% CI, 19%-27%), 40% (95% CI, 36%-45%), and 42% (95% CI, 37%-47%), respectively. Multivariate analyses showed that young age and platelet counts higher than 100 × 109/L at transplantation were associated with lower TRM and higher disease-free and overall survival rates. Relapse incidence was higher in patients with high percentages of blasts in the marrow at transplantation or presentation, with high International Prognostic Scoring System scores at diagnosis, and with T-cell–depleted transplants. These findings indicate that transplantation from an HLA-identical sibling offers the possibility of long-term, disease-free survival to patients with MDS. Best candidates are younger patients with a low percentage of blasts and preserved platelet counts.
doi_str_mv 10.1182/blood.V100.6.1997
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Douglas ; Davies, Stella M. ; Lazarus, Hillard M. ; Bredeson, Christopher N. ; Marks, David I. ; Canals, Carmen ; Boogaerts, Marc A. ; Goldman, John ; Champlin, Richard E. ; Keating, Armand ; Weisdorf, Daniel J. ; de Witte, Theo M. ; Horowitz, Mary M.</creator><creatorcontrib>Sierra, Jorge ; Pérez, Waleska S. ; Rozman, Ciril ; Carreras, Enric ; Klein, John P. ; Rizzo, J. Douglas ; Davies, Stella M. ; Lazarus, Hillard M. ; Bredeson, Christopher N. ; Marks, David I. ; Canals, Carmen ; Boogaerts, Marc A. ; Goldman, John ; Champlin, Richard E. ; Keating, Armand ; Weisdorf, Daniel J. ; de Witte, Theo M. ; Horowitz, Mary M.</creatorcontrib><description>Allogeneic hematopoietic stem cell transplantation is the only curative therapy for myelodysplasia (MDS). To identify factors influencing transplantation outcome, we studied 452 recipients of HLA-identical sibling transplants for MDS from 1989 to 1997, reported to the International Bone Marrow Transplant Registry. Patients with treatment-related MDS or unclassified MDS were excluded. Median age was 38 years (range, 2-64 years). Sixty percent had refractory anemia with excess blasts (n = 136) or with excess blasts in transformation (n = 136). Conditioning regimens included total body irradiation in 199 (44%) cases. Marrow was T-cell depleted for 58 (13%) transplants. Cumulative incidences of neutrophil engraftment, grades II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were 91% (95% confidence interval [CI], 88%-93%), 36% (95% CI, 31%-40%), and 39% (95% CI, 33%-44%), respectively. Three-year transplantation-related mortality (TRM), relapse, disease-free survival, and overall survival rates were 37% (95% CI, 32%-42%), 23% (95% CI, 19%-27%), 40% (95% CI, 36%-45%), and 42% (95% CI, 37%-47%), respectively. Multivariate analyses showed that young age and platelet counts higher than 100 × 109/L at transplantation were associated with lower TRM and higher disease-free and overall survival rates. Relapse incidence was higher in patients with high percentages of blasts in the marrow at transplantation or presentation, with high International Prognostic Scoring System scores at diagnosis, and with T-cell–depleted transplants. These findings indicate that transplantation from an HLA-identical sibling offers the possibility of long-term, disease-free survival to patients with MDS. Best candidates are younger patients with a low percentage of blasts and preserved platelet counts.</description><identifier>ISSN: 0006-4971</identifier><identifier>EISSN: 1528-0020</identifier><identifier>DOI: 10.1182/blood.V100.6.1997</identifier><identifier>PMID: 12200358</identifier><language>eng</language><publisher>Washington, DC: Elsevier Inc</publisher><subject>Adolescent ; Adult ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Biological and medical sciences ; Bone Marrow Transplantation - adverse effects ; Bone Marrow Transplantation - immunology ; Bone Marrow Transplantation - mortality ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Child ; Child, Preschool ; Disease-Free Survival ; Graft vs Host Disease ; Hematologic and hematopoietic diseases ; Hematologic Diseases - complications ; Hematologic Diseases - mortality ; Hematologic Diseases - therapy ; Histocompatibility ; Histocompatibility Testing ; Humans ; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis ; Medical sciences ; Middle Aged ; Myelodysplastic Syndromes - complications ; Myelodysplastic Syndromes - mortality ; Myelodysplastic Syndromes - therapy ; Nuclear Family ; Prospective Studies ; Retrospective Studies ; Risk ; Survival Analysis ; Transfusions. Complications. Transfusion reactions. 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Douglas</creatorcontrib><creatorcontrib>Davies, Stella M.</creatorcontrib><creatorcontrib>Lazarus, Hillard M.</creatorcontrib><creatorcontrib>Bredeson, Christopher N.</creatorcontrib><creatorcontrib>Marks, David I.</creatorcontrib><creatorcontrib>Canals, Carmen</creatorcontrib><creatorcontrib>Boogaerts, Marc A.</creatorcontrib><creatorcontrib>Goldman, John</creatorcontrib><creatorcontrib>Champlin, Richard E.</creatorcontrib><creatorcontrib>Keating, Armand</creatorcontrib><creatorcontrib>Weisdorf, Daniel J.</creatorcontrib><creatorcontrib>de Witte, Theo M.</creatorcontrib><creatorcontrib>Horowitz, Mary M.</creatorcontrib><title>Bone marrow transplantation from HLA-identical siblings as treatment for myelodysplasia</title><title>Blood</title><addtitle>Blood</addtitle><description>Allogeneic hematopoietic stem cell transplantation is the only curative therapy for myelodysplasia (MDS). To identify factors influencing transplantation outcome, we studied 452 recipients of HLA-identical sibling transplants for MDS from 1989 to 1997, reported to the International Bone Marrow Transplant Registry. Patients with treatment-related MDS or unclassified MDS were excluded. Median age was 38 years (range, 2-64 years). Sixty percent had refractory anemia with excess blasts (n = 136) or with excess blasts in transformation (n = 136). Conditioning regimens included total body irradiation in 199 (44%) cases. Marrow was T-cell depleted for 58 (13%) transplants. Cumulative incidences of neutrophil engraftment, grades II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were 91% (95% confidence interval [CI], 88%-93%), 36% (95% CI, 31%-40%), and 39% (95% CI, 33%-44%), respectively. Three-year transplantation-related mortality (TRM), relapse, disease-free survival, and overall survival rates were 37% (95% CI, 32%-42%), 23% (95% CI, 19%-27%), 40% (95% CI, 36%-45%), and 42% (95% CI, 37%-47%), respectively. Multivariate analyses showed that young age and platelet counts higher than 100 × 109/L at transplantation were associated with lower TRM and higher disease-free and overall survival rates. Relapse incidence was higher in patients with high percentages of blasts in the marrow at transplantation or presentation, with high International Prognostic Scoring System scores at diagnosis, and with T-cell–depleted transplants. These findings indicate that transplantation from an HLA-identical sibling offers the possibility of long-term, disease-free survival to patients with MDS. Best candidates are younger patients with a low percentage of blasts and preserved platelet counts.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Biological and medical sciences</subject><subject>Bone Marrow Transplantation - adverse effects</subject><subject>Bone Marrow Transplantation - immunology</subject><subject>Bone Marrow Transplantation - mortality</subject><subject>Bone marrow, stem cells transplantation. Graft versus host reaction</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Disease-Free Survival</subject><subject>Graft vs Host Disease</subject><subject>Hematologic and hematopoietic diseases</subject><subject>Hematologic Diseases - complications</subject><subject>Hematologic Diseases - mortality</subject><subject>Hematologic Diseases - therapy</subject><subject>Histocompatibility</subject><subject>Histocompatibility Testing</subject><subject>Humans</subject><subject>Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis</subject><subject>Medical sciences</subject><subject>Middle Aged</subject><subject>Myelodysplastic Syndromes - complications</subject><subject>Myelodysplastic Syndromes - mortality</subject><subject>Myelodysplastic Syndromes - therapy</subject><subject>Nuclear Family</subject><subject>Prospective Studies</subject><subject>Retrospective Studies</subject><subject>Risk</subject><subject>Survival Analysis</subject><subject>Transfusions. Complications. Transfusion reactions. 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Douglas</creator><creator>Davies, Stella M.</creator><creator>Lazarus, Hillard M.</creator><creator>Bredeson, Christopher N.</creator><creator>Marks, David I.</creator><creator>Canals, Carmen</creator><creator>Boogaerts, Marc A.</creator><creator>Goldman, John</creator><creator>Champlin, Richard E.</creator><creator>Keating, Armand</creator><creator>Weisdorf, Daniel J.</creator><creator>de Witte, Theo M.</creator><creator>Horowitz, Mary M.</creator><general>Elsevier Inc</general><general>The Americain Society of Hematology</general><scope>6I.</scope><scope>AAFTH</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>7X8</scope></search><sort><creationdate>20020915</creationdate><title>Bone marrow transplantation from HLA-identical siblings as treatment for myelodysplasia</title><author>Sierra, Jorge ; Pérez, Waleska S. ; Rozman, Ciril ; Carreras, Enric ; Klein, John P. ; Rizzo, J. Douglas ; Davies, Stella M. ; Lazarus, Hillard M. ; Bredeson, Christopher N. ; Marks, David I. ; Canals, Carmen ; Boogaerts, Marc A. ; Goldman, John ; Champlin, Richard E. ; Keating, Armand ; Weisdorf, Daniel J. ; de Witte, Theo M. ; Horowitz, Mary M.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-e289t-94248269edf1941e32cc66fdcaeb047e044a1dd38a1b6f7fe33fcd35e60a9a173</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2002</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</topic><topic>Biological and medical sciences</topic><topic>Bone Marrow Transplantation - adverse effects</topic><topic>Bone Marrow Transplantation - immunology</topic><topic>Bone Marrow Transplantation - mortality</topic><topic>Bone marrow, stem cells transplantation. 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subjects Adolescent
Adult
Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy
Biological and medical sciences
Bone Marrow Transplantation - adverse effects
Bone Marrow Transplantation - immunology
Bone Marrow Transplantation - mortality
Bone marrow, stem cells transplantation. Graft versus host reaction
Child
Child, Preschool
Disease-Free Survival
Graft vs Host Disease
Hematologic and hematopoietic diseases
Hematologic Diseases - complications
Hematologic Diseases - mortality
Hematologic Diseases - therapy
Histocompatibility
Histocompatibility Testing
Humans
Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis
Medical sciences
Middle Aged
Myelodysplastic Syndromes - complications
Myelodysplastic Syndromes - mortality
Myelodysplastic Syndromes - therapy
Nuclear Family
Prospective Studies
Retrospective Studies
Risk
Survival Analysis
Transfusions. Complications. Transfusion reactions. Cell and gene therapy
Transplantation, Isogeneic - adverse effects
Transplantation, Isogeneic - immunology
Transplantation, Isogeneic - mortality
title Bone marrow transplantation from HLA-identical siblings as treatment for myelodysplasia
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