Haploidentical transplantation in high‐risk pediatric leukemia: A retrospective comparative analysis on behalf of the Spanish working Group for bone marrow transplantation in children (GETMON) and the Spanish Grupo for hematopoietic transplantation (GETH)

A total of 192 pediatric patients, median age 8.6 years, with high‐risk hematological malignancies, underwent haploidentical stem cell transplantation (haplo‐HSCT) using post‐transplantation cyclophosphamide (PT‐Cy), or ex vivo T cell‐depleted (TCD) graft platforms, from January 1999 to December 201...

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Veröffentlicht in:American journal of hematology 2020-01, Vol.95 (1), p.28-37
Hauptverfasser: Pérez‐Martínez, Antonio, Ferreras, Cristina, Pascual, Antonia, Gonzalez‐Vicent, Marta, Alonso, Laura, Badell, Isabel, Fernández Navarro, José María, Regueiro, Alexandra, Plaza, Mercedes, Pérez Hurtado, Jose María, Benito, Ana, Beléndez, Cristina, Couselo, José Miguel, Fuster, José Luis, Díaz‐Almirón, Mariana, Bueno, David, Mozo, Yasmina, Marsal, Julia, Gómez López, Alicia, Sisinni, Luisa, Heredia, Cristina Díaz, Díaz, Miguel Ángel
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container_issue 1
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container_title American journal of hematology
container_volume 95
creator Pérez‐Martínez, Antonio
Ferreras, Cristina
Pascual, Antonia
Gonzalez‐Vicent, Marta
Alonso, Laura
Badell, Isabel
Fernández Navarro, José María
Regueiro, Alexandra
Plaza, Mercedes
Pérez Hurtado, Jose María
Benito, Ana
Beléndez, Cristina
Couselo, José Miguel
Fuster, José Luis
Díaz‐Almirón, Mariana
Bueno, David
Mozo, Yasmina
Marsal, Julia
Gómez López, Alicia
Sisinni, Luisa
Heredia, Cristina Díaz
Díaz, Miguel Ángel
description A total of 192 pediatric patients, median age 8.6 years, with high‐risk hematological malignancies, underwent haploidentical stem cell transplantation (haplo‐HSCT) using post‐transplantation cyclophosphamide (PT‐Cy), or ex vivo T cell‐depleted (TCD) graft platforms, from January 1999 to December 2016 in 10 centers in Spain. Some 41 patients received an unmanipulated graft followed by PT‐Cy for graft‐vs‐host disease (GvHD) prophylaxis. A total of 151 patients were transplanted with CD3‐depleted peripheral blood stem cells (PBSCs) by either CD34+ selection, CD3+CD19+ depletion, TCRαβ+CD19+ depletion or CD45RA+ depletion, added to CD34+ selection for GvHD prophylaxis. The PBSCs were the only source in patients following ex vivo TCD haplo‐HSCT; bone marrow was the source in 9 of 41 patients following PT‐CY haplo‐HSCT. Engraftment was achieved in 91.3% of cases. A donor younger than 30 years, and the development of chronic GvHD were positive factors influencing survival, whereas positive minimal residual disease (MRD) before transplant and lymphoid disease were negative factors. The probability of relapse increased with lymphoid malignancies, a donor killer‐cell immunoglobulin‐like receptor (KIR) haplotype A and positive MRD pretransplant. No difference was found in overall survival, disease‐free survival or relapse incidence between the two platforms. Relapse is still of concern in both platforms, and it should be the focus of future efforts. In conclusion, both platforms for haplo‐HSCT were effective and could be utilized depending on the comfort level of the center.
doi_str_mv 10.1002/ajh.25661
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Some 41 patients received an unmanipulated graft followed by PT‐Cy for graft‐vs‐host disease (GvHD) prophylaxis. A total of 151 patients were transplanted with CD3‐depleted peripheral blood stem cells (PBSCs) by either CD34+ selection, CD3+CD19+ depletion, TCRαβ+CD19+ depletion or CD45RA+ depletion, added to CD34+ selection for GvHD prophylaxis. The PBSCs were the only source in patients following ex vivo TCD haplo‐HSCT; bone marrow was the source in 9 of 41 patients following PT‐CY haplo‐HSCT. Engraftment was achieved in 91.3% of cases. A donor younger than 30 years, and the development of chronic GvHD were positive factors influencing survival, whereas positive minimal residual disease (MRD) before transplant and lymphoid disease were negative factors. The probability of relapse increased with lymphoid malignancies, a donor killer‐cell immunoglobulin‐like receptor (KIR) haplotype A and positive MRD pretransplant. No difference was found in overall survival, disease‐free survival or relapse incidence between the two platforms. Relapse is still of concern in both platforms, and it should be the focus of future efforts. 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Some 41 patients received an unmanipulated graft followed by PT‐Cy for graft‐vs‐host disease (GvHD) prophylaxis. A total of 151 patients were transplanted with CD3‐depleted peripheral blood stem cells (PBSCs) by either CD34+ selection, CD3+CD19+ depletion, TCRαβ+CD19+ depletion or CD45RA+ depletion, added to CD34+ selection for GvHD prophylaxis. The PBSCs were the only source in patients following ex vivo TCD haplo‐HSCT; bone marrow was the source in 9 of 41 patients following PT‐CY haplo‐HSCT. Engraftment was achieved in 91.3% of cases. A donor younger than 30 years, and the development of chronic GvHD were positive factors influencing survival, whereas positive minimal residual disease (MRD) before transplant and lymphoid disease were negative factors. The probability of relapse increased with lymphoid malignancies, a donor killer‐cell immunoglobulin‐like receptor (KIR) haplotype A and positive MRD pretransplant. No difference was found in overall survival, disease‐free survival or relapse incidence between the two platforms. Relapse is still of concern in both platforms, and it should be the focus of future efforts. 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No difference was found in overall survival, disease‐free survival or relapse incidence between the two platforms. Relapse is still of concern in both platforms, and it should be the focus of future efforts. In conclusion, both platforms for haplo‐HSCT were effective and could be utilized depending on the comfort level of the center.</abstract><cop>Hoboken, USA</cop><pub>John Wiley &amp; Sons, Inc</pub><pmid>31625177</pmid><doi>10.1002/ajh.25661</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0002-6436-9195</orcidid><oa>free_for_read</oa></addata></record>
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source Wiley-Blackwell Journals; MEDLINE; Wiley Online Library Journals; EZB-FREE-00999 freely available EZB journals
subjects Bone marrow
Bone marrow transplantation
Bone Marrow Transplantation - methods
Bone Marrow Transplantation - mortality
CD19 antigen
CD3 antigen
CD34 antigen
CD45RA antigen
Child
Comparative analysis
Cyclophosphamide
Cyclophosphamide - therapeutic use
Female
Graft Survival
Graft vs Host Disease - prevention & control
Graft-versus-host reaction
Hematologic Neoplasms - therapy
Hematology
Hematopoietic Stem Cell Transplantation - methods
Hematopoietic Stem Cell Transplantation - mortality
Hematopoietic stem cells
Humans
Leukemia - mortality
Leukemia - therapy
Lymphocyte Depletion
Lymphocytes T
Male
Minimal residual disease
Pediatrics
Pediatrics - methods
Peripheral blood
Prophylaxis
Recurrence
Retrospective Studies
Spain
Stem cell transplantation
Survival Analysis
T cell receptors
Transplantation, Haploidentical
Transplants & implants
title Haploidentical transplantation in high‐risk pediatric leukemia: A retrospective comparative analysis on behalf of the Spanish working Group for bone marrow transplantation in children (GETMON) and the Spanish Grupo for hematopoietic transplantation (GETH)
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