Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes: A Report From Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation

Simona Pagliuca, Régis Peffault de Latour, Fernanda Volt, Franco Locatelli, Marco Zecca, Jean-Hugues Dalle, Patrizia Comoli, Kim Vettenranta, Miguel Angel Diaz, Or Reuven, Yves Bertrand, Cristina Diaz de Heredia, Arnon Nagler, Ardeshir Ghavamzadeh, Sabina Sufliarska, Sarah Lawson, Chantal Kenzey, Vanderson Rocha, Carlo Dufour, Eliane GluckmanJakob Passweg, Annalisa Ruggeri

Research output: Contribution to journalArticle

Abstract

Cord blood transplantation (CBT) from HLA-identical siblings is an attractive option for patients with bone marrow failure (BMF) syndrome because of the low risk of graft-versus-host disease (GVHD) and the absence of risk to the donor. We analyzed outcomes of 117 patients with inherited or acquired BMF syndrome who received CBT from a related HLA-identical donor in European Society for Blood and Marrow Transplantation centers between 1988 and 2014. Ninety-seven patients had inherited and 20 patients acquired BMF syndrome. Eighty-two patients received a single cord blood (CB) unit, whereas 35 patients received a combination of CB and bone marrow cells from the same donor. Median age at CBT was 6.7 years, and median follow-up was 86.7 months. The cumulative incidence function (CIF) of neutrophil recovery was 88.8% (95% CI, 83.1% to 94.9%), 100-day CIF of grades II to IV acute GVHD was 15.2%, and 7-year CIF of chronic GVHD was 14.5%. Overall survival at 7 years was 87.9% (95% CI, 80.8% to 92.6%), 89% for inherited and 81% for acquired BMF syndromes (P = .66). Results of this study are consistent with outcomes of bone marrow transplantation shown by previous series in the same setting and indicate that in pediatric patients with BMF syndrome, CBT from an HLA-identical sibling donor is associated with excellent long-term outcomes and that collection of CB unit at birth of a new sibling is strongly recommended.

Original languageEnglish
Pages (from-to)1939-1948
Number of pages10
JournalBiology of Blood and Marrow Transplantation
Volume23
Issue number11
DOIs
Publication statusPublished - Nov 2017

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Aplastic Anemia
Fetal Blood
Siblings
Transplantation
Bone Marrow
Graft vs Host Disease
Tissue Donors
Incidence
Recovery of Function
Bone Marrow failure syndromes
Bone Marrow Transplantation
Bone Marrow Cells
Neutrophils
Parturition
Pediatrics
Survival

Keywords

  • Journal Article

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Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes : A Report From Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. / Pagliuca, Simona; Peffault de Latour, Régis; Volt, Fernanda; Locatelli, Franco; Zecca, Marco; Dalle, Jean-Hugues; Comoli, Patrizia; Vettenranta, Kim; Diaz, Miguel Angel; Reuven, Or; Bertrand, Yves; Diaz de Heredia, Cristina; Nagler, Arnon; Ghavamzadeh, Ardeshir; Sufliarska, Sabina; Lawson, Sarah; Kenzey, Chantal; Rocha, Vanderson; Dufour, Carlo; Gluckman, Eliane; Passweg, Jakob; Ruggeri, Annalisa.

In: Biology of Blood and Marrow Transplantation, Vol. 23, No. 11, 11.2017, p. 1939-1948.

Research output: Contribution to journalArticle

Pagliuca, S, Peffault de Latour, R, Volt, F, Locatelli, F, Zecca, M, Dalle, J-H, Comoli, P, Vettenranta, K, Diaz, MA, Reuven, O, Bertrand, Y, Diaz de Heredia, C, Nagler, A, Ghavamzadeh, A, Sufliarska, S, Lawson, S, Kenzey, C, Rocha, V, Dufour, C, Gluckman, E, Passweg, J & Ruggeri, A 2017, 'Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes: A Report From Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation', Biology of Blood and Marrow Transplantation, vol. 23, no. 11, pp. 1939-1948. https://doi.org/10.1016/j.bbmt.2017.08.004
Pagliuca, Simona ; Peffault de Latour, Régis ; Volt, Fernanda ; Locatelli, Franco ; Zecca, Marco ; Dalle, Jean-Hugues ; Comoli, Patrizia ; Vettenranta, Kim ; Diaz, Miguel Angel ; Reuven, Or ; Bertrand, Yves ; Diaz de Heredia, Cristina ; Nagler, Arnon ; Ghavamzadeh, Ardeshir ; Sufliarska, Sabina ; Lawson, Sarah ; Kenzey, Chantal ; Rocha, Vanderson ; Dufour, Carlo ; Gluckman, Eliane ; Passweg, Jakob ; Ruggeri, Annalisa. / Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes : A Report From Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. In: Biology of Blood and Marrow Transplantation. 2017 ; Vol. 23, No. 11. pp. 1939-1948.
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T1 - Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes

T2 - A Report From Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation

AU - Pagliuca, Simona

AU - Peffault de Latour, Régis

AU - Volt, Fernanda

AU - Locatelli, Franco

AU - Zecca, Marco

AU - Dalle, Jean-Hugues

AU - Comoli, Patrizia

AU - Vettenranta, Kim

AU - Diaz, Miguel Angel

AU - Reuven, Or

AU - Bertrand, Yves

AU - Diaz de Heredia, Cristina

AU - Nagler, Arnon

AU - Ghavamzadeh, Ardeshir

AU - Sufliarska, Sabina

AU - Lawson, Sarah

AU - Kenzey, Chantal

AU - Rocha, Vanderson

AU - Dufour, Carlo

AU - Gluckman, Eliane

AU - Passweg, Jakob

AU - Ruggeri, Annalisa

N1 - Copyright © 2017 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

PY - 2017/11

Y1 - 2017/11

N2 - Cord blood transplantation (CBT) from HLA-identical siblings is an attractive option for patients with bone marrow failure (BMF) syndrome because of the low risk of graft-versus-host disease (GVHD) and the absence of risk to the donor. We analyzed outcomes of 117 patients with inherited or acquired BMF syndrome who received CBT from a related HLA-identical donor in European Society for Blood and Marrow Transplantation centers between 1988 and 2014. Ninety-seven patients had inherited and 20 patients acquired BMF syndrome. Eighty-two patients received a single cord blood (CB) unit, whereas 35 patients received a combination of CB and bone marrow cells from the same donor. Median age at CBT was 6.7 years, and median follow-up was 86.7 months. The cumulative incidence function (CIF) of neutrophil recovery was 88.8% (95% CI, 83.1% to 94.9%), 100-day CIF of grades II to IV acute GVHD was 15.2%, and 7-year CIF of chronic GVHD was 14.5%. Overall survival at 7 years was 87.9% (95% CI, 80.8% to 92.6%), 89% for inherited and 81% for acquired BMF syndromes (P = .66). Results of this study are consistent with outcomes of bone marrow transplantation shown by previous series in the same setting and indicate that in pediatric patients with BMF syndrome, CBT from an HLA-identical sibling donor is associated with excellent long-term outcomes and that collection of CB unit at birth of a new sibling is strongly recommended.

AB - Cord blood transplantation (CBT) from HLA-identical siblings is an attractive option for patients with bone marrow failure (BMF) syndrome because of the low risk of graft-versus-host disease (GVHD) and the absence of risk to the donor. We analyzed outcomes of 117 patients with inherited or acquired BMF syndrome who received CBT from a related HLA-identical donor in European Society for Blood and Marrow Transplantation centers between 1988 and 2014. Ninety-seven patients had inherited and 20 patients acquired BMF syndrome. Eighty-two patients received a single cord blood (CB) unit, whereas 35 patients received a combination of CB and bone marrow cells from the same donor. Median age at CBT was 6.7 years, and median follow-up was 86.7 months. The cumulative incidence function (CIF) of neutrophil recovery was 88.8% (95% CI, 83.1% to 94.9%), 100-day CIF of grades II to IV acute GVHD was 15.2%, and 7-year CIF of chronic GVHD was 14.5%. Overall survival at 7 years was 87.9% (95% CI, 80.8% to 92.6%), 89% for inherited and 81% for acquired BMF syndromes (P = .66). Results of this study are consistent with outcomes of bone marrow transplantation shown by previous series in the same setting and indicate that in pediatric patients with BMF syndrome, CBT from an HLA-identical sibling donor is associated with excellent long-term outcomes and that collection of CB unit at birth of a new sibling is strongly recommended.

KW - Journal Article

U2 - 10.1016/j.bbmt.2017.08.004

DO - 10.1016/j.bbmt.2017.08.004

M3 - Article

C2 - 28797779

VL - 23

SP - 1939

EP - 1948

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

IS - 11

ER -