Outcome of children with acute leukemia given HLA-haploidentical HSCT after ab T-cell and B-cell depletion

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Abstract

© 2017 by The American Society of Hematology. Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-haploidentical relative (haplo-HSCT) is a suitable option for children with acute leukemia (AL) either relapsed or at high-risk of treatment failure. We developed a novel method of graft manipulation based on negative depletion of ab T and B cells and conducted a prospective trial evaluating the outcome of children with AL transplanted with this approach. Eighty AL children, transplanted between September 2011 and September 2014, were enrolled in the trial. All children were given a fully myeloablative preparative regimen. Anti–T-lymphocyte globulin from day 25 to 23 was used for preventing graft rejection and graft-versus-host disease (GVHD); no patient received any posttransplantation GVHD prophylaxis. Two children experienced primary graft failure. The cumulative incidence of skin-only, grade 1-2 acute GVHD was 30%; no patient developed extensive chronic GVHD. Four patients died, the cumulative incidence of nonrelapse mortality being 5%, whereas 19 relapsed, resulting in a 24% cumulative incidence of relapse. With a median follow-up of 46 months for surviving patients, the 5-year probability of chronic GVHD-free, relapse-free survival (GRFS) is 71%. Total body irradiation–containing preparative regimen was the only variable favorably influencing relapse incidence and GRFS. The outcomes of these 80 patients are comparable to those of 41 and 51 children given transplantation from an HLA-identical sibling or a 10/10 allelic-matched unrelated donor in the same period. These data indicate that haplo-HSCT after ab T- and B-cell depletion represents a competitive alternative for children with AL in need of urgent allograft. This trial was registered at www.clinicaltrials.gov as #NCT01810120.
Original languageEnglish
Pages (from-to)677-685
Number of pages9
JournalBlood
Volume130
Issue number5
DOIs
Publication statusPublished - Aug 3 2017

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T-cells
Hematopoietic Stem Cell Transplantation
Stem cells
Grafts
Leukemia
B-Lymphocytes
Graft vs Host Disease
Cells
T-Lymphocytes
Recurrence
Incidence
Transplants
Unrelated Donors
Survival
Globulins
Graft Rejection
Lymphocytes
Treatment Failure
Allografts
Siblings

Cite this

@article{01b40b64eb6e4960825f0cd2600bf250,
title = "Outcome of children with acute leukemia given HLA-haploidentical HSCT after ab T-cell and B-cell depletion",
abstract = "{\circledC} 2017 by The American Society of Hematology. Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-haploidentical relative (haplo-HSCT) is a suitable option for children with acute leukemia (AL) either relapsed or at high-risk of treatment failure. We developed a novel method of graft manipulation based on negative depletion of ab T and B cells and conducted a prospective trial evaluating the outcome of children with AL transplanted with this approach. Eighty AL children, transplanted between September 2011 and September 2014, were enrolled in the trial. All children were given a fully myeloablative preparative regimen. Anti–T-lymphocyte globulin from day 25 to 23 was used for preventing graft rejection and graft-versus-host disease (GVHD); no patient received any posttransplantation GVHD prophylaxis. Two children experienced primary graft failure. The cumulative incidence of skin-only, grade 1-2 acute GVHD was 30{\%}; no patient developed extensive chronic GVHD. Four patients died, the cumulative incidence of nonrelapse mortality being 5{\%}, whereas 19 relapsed, resulting in a 24{\%} cumulative incidence of relapse. With a median follow-up of 46 months for surviving patients, the 5-year probability of chronic GVHD-free, relapse-free survival (GRFS) is 71{\%}. Total body irradiation–containing preparative regimen was the only variable favorably influencing relapse incidence and GRFS. The outcomes of these 80 patients are comparable to those of 41 and 51 children given transplantation from an HLA-identical sibling or a 10/10 allelic-matched unrelated donor in the same period. These data indicate that haplo-HSCT after ab T- and B-cell depletion represents a competitive alternative for children with AL in need of urgent allograft. This trial was registered at www.clinicaltrials.gov as #NCT01810120.",
author = "Franco Locatelli and Franco Locatelli and Pietro Merli and Daria Pagliara and {Li Pira}, Giuseppina and Michela Falco and Daniela Pende and Roberto Rondelli and Barbarella Lucarelli and Brescia, {Letizia Pomponia} and Riccardo Masetti and Milano, {Giuseppe Maria} and Valentina Bertaina and Mattia Algeri and Pinto, {Rita Maria} and Luisa Strocchio and Raffaella Meazza and Lavinia Grapulin and Rupert Handgretinger and Alessandro Moretta and Alice Bertaina and Lorenzo Moretta",
year = "2017",
month = "8",
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doi = "10.1182/blood-2017-04-779769",
language = "English",
volume = "130",
pages = "677--685",
journal = "Blood",
issn = "0006-4971",
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TY - JOUR

T1 - Outcome of children with acute leukemia given HLA-haploidentical HSCT after ab T-cell and B-cell depletion

AU - Locatelli, Franco

AU - Locatelli, Franco

AU - Merli, Pietro

AU - Pagliara, Daria

AU - Li Pira, Giuseppina

AU - Falco, Michela

AU - Pende, Daniela

AU - Rondelli, Roberto

AU - Lucarelli, Barbarella

AU - Brescia, Letizia Pomponia

AU - Masetti, Riccardo

AU - Milano, Giuseppe Maria

AU - Bertaina, Valentina

AU - Algeri, Mattia

AU - Pinto, Rita Maria

AU - Strocchio, Luisa

AU - Meazza, Raffaella

AU - Grapulin, Lavinia

AU - Handgretinger, Rupert

AU - Moretta, Alessandro

AU - Bertaina, Alice

AU - Moretta, Lorenzo

PY - 2017/8/3

Y1 - 2017/8/3

N2 - © 2017 by The American Society of Hematology. Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-haploidentical relative (haplo-HSCT) is a suitable option for children with acute leukemia (AL) either relapsed or at high-risk of treatment failure. We developed a novel method of graft manipulation based on negative depletion of ab T and B cells and conducted a prospective trial evaluating the outcome of children with AL transplanted with this approach. Eighty AL children, transplanted between September 2011 and September 2014, were enrolled in the trial. All children were given a fully myeloablative preparative regimen. Anti–T-lymphocyte globulin from day 25 to 23 was used for preventing graft rejection and graft-versus-host disease (GVHD); no patient received any posttransplantation GVHD prophylaxis. Two children experienced primary graft failure. The cumulative incidence of skin-only, grade 1-2 acute GVHD was 30%; no patient developed extensive chronic GVHD. Four patients died, the cumulative incidence of nonrelapse mortality being 5%, whereas 19 relapsed, resulting in a 24% cumulative incidence of relapse. With a median follow-up of 46 months for surviving patients, the 5-year probability of chronic GVHD-free, relapse-free survival (GRFS) is 71%. Total body irradiation–containing preparative regimen was the only variable favorably influencing relapse incidence and GRFS. The outcomes of these 80 patients are comparable to those of 41 and 51 children given transplantation from an HLA-identical sibling or a 10/10 allelic-matched unrelated donor in the same period. These data indicate that haplo-HSCT after ab T- and B-cell depletion represents a competitive alternative for children with AL in need of urgent allograft. This trial was registered at www.clinicaltrials.gov as #NCT01810120.

AB - © 2017 by The American Society of Hematology. Allogeneic hematopoietic stem cell transplantation (HSCT) from an HLA-haploidentical relative (haplo-HSCT) is a suitable option for children with acute leukemia (AL) either relapsed or at high-risk of treatment failure. We developed a novel method of graft manipulation based on negative depletion of ab T and B cells and conducted a prospective trial evaluating the outcome of children with AL transplanted with this approach. Eighty AL children, transplanted between September 2011 and September 2014, were enrolled in the trial. All children were given a fully myeloablative preparative regimen. Anti–T-lymphocyte globulin from day 25 to 23 was used for preventing graft rejection and graft-versus-host disease (GVHD); no patient received any posttransplantation GVHD prophylaxis. Two children experienced primary graft failure. The cumulative incidence of skin-only, grade 1-2 acute GVHD was 30%; no patient developed extensive chronic GVHD. Four patients died, the cumulative incidence of nonrelapse mortality being 5%, whereas 19 relapsed, resulting in a 24% cumulative incidence of relapse. With a median follow-up of 46 months for surviving patients, the 5-year probability of chronic GVHD-free, relapse-free survival (GRFS) is 71%. Total body irradiation–containing preparative regimen was the only variable favorably influencing relapse incidence and GRFS. The outcomes of these 80 patients are comparable to those of 41 and 51 children given transplantation from an HLA-identical sibling or a 10/10 allelic-matched unrelated donor in the same period. These data indicate that haplo-HSCT after ab T- and B-cell depletion represents a competitive alternative for children with AL in need of urgent allograft. This trial was registered at www.clinicaltrials.gov as #NCT01810120.

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U2 - 10.1182/blood-2017-04-779769

DO - 10.1182/blood-2017-04-779769

M3 - Article

VL - 130

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EP - 685

JO - Blood

JF - Blood

SN - 0006-4971

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