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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 Publisher Pubmed



Pagliuca S1 ; Peffault De Latour R1 ; Volt F2, 3 ; Locatelli F4 ; Zecca M5 ; Dalle JH6 ; Comoli P5 ; Vettenranta K7 ; Diaz MA8 ; Reuven O9 ; Bertrand Y10 ; Diaz De Heredia C11 ; Nagler A12 ; Ghavamzadeh A13 Show All Authors
Authors
  1. Pagliuca S1
  2. Peffault De Latour R1
  3. Volt F2, 3
  4. Locatelli F4
  5. Zecca M5
  6. Dalle JH6
  7. Comoli P5
  8. Vettenranta K7
  9. Diaz MA8
  10. Reuven O9
  11. Bertrand Y10
  12. Diaz De Heredia C11
  13. Nagler A12
  14. Ghavamzadeh A13
  15. Sufliarska S14
  16. Lawson S15
  17. Kenzey C2, 3
  18. Rocha V2, 16
  19. Dufour C17
  20. Gluckman E2, 3
  21. Passweg J18
  22. Ruggeri A2, 3, 19
Show Affiliations
Authors Affiliations
  1. 1. Hopital Saint-Louis—Assistance Publique-Hopitaux de Paris, Paris, France
  2. 2. Eurocord, Hopital Saint Louis, Paris, France
  3. 3. Monacord, Monacord, Centre Scientifique de Monaco, Monaco, United States
  4. 4. Oncoematologia Pediatrica e Medicina Trasfusionale IRCCS Ospedale Pediatrico Bambino Gesu, Universita di Pavia, Rome, Italy
  5. 5. Oncoematologia Pediatrica and Cell Factory, Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy
  6. 6. Pediatric Haematology Immunology, Robert Debre Hospital, France and Paris Diderot University, Paris, France
  7. 7. University of Helsinki Children's Hospital, Helsinki, Finland
  8. 8. Nino Jesus Children's Hospital, Madrid, Spain
  9. 9. Department of Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel
  10. 10. Institut d'Hematologie et Oncologie Pediatrique, CHU Lyon, Lyon, France
  11. 11. Servicio de Hematologia y Oncologia Pediatrica, Hospital Universitario Vall d'Hebron, Barcelona, Spain
  12. 12. Hematology Division, Chaim Sheba Medical Center and Tel Aviv University, Tel-Hashomer, Ramat-Gan, Israel
  13. 13. Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran
  14. 14. Bone Marrow Transplantation Unit, Comenius University Children's Hospital, Bratislava, Slovakia
  15. 15. Department of Haematology, Birmingham Children's Hospital, Birmingham, United Kingdom
  16. 16. Churchill Hospital, Oxford University, Oxford, United Kingdom
  17. 17. Hematology Unit, Giannina Gaslini Children's Hospital, Genoa, Italy
  18. 18. Klinik fur Hamatologie Bereich Innere Medizin Universitatsspital Basel, Basel, Switzerland
  19. 19. Service d'Hematologie et Therapie Cellulaire, Hopital Saint-Antoine, Paris, France

Source: Biology of Blood and Marrow Transplantation Published:2017


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. © 2017 The American Society for Blood and Marrow Transplantation
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