Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes

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 journalArticlepeer-review


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
Issue number11
Publication statusPublished - Nov 2017


  • Journal Article


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