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Intrabone infusion for allogeneic umbilical cord blood transplantation in children

Bone Marrow Transplantation volume 56, pages 1937–1943 (2021)Cite this article

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Abstract

Umbilical cord blood transplantation (UCBT) has been used to treat malignant and non-malignant diseases. UCBT offers the advantages of easy procurement and acceptable partial HLA mismatches, but also shows delayed hematopoietic and immunological recoveries. We postulated that an intrabone (IB) infusion of cord blood could provide a faster short- and long-term engraftment in a pediatric population with malignant and non-malignant hematologic diseases. We conducted this phase I-II single arm, exploratory clinical trial (NCT01711788) from 2012 to 2016 in a single center. Fifteen patients aged from 1.9 to 16.4 years received an IB UCBT. Median time to neutrophils and platelet recoveries were 18 days (range: 13-36 days) and 42 days (range: 26-107 days), respectively. Rate of severe acute GVH grade was low, with only one patient with grade III aGVH. Relapse occurred in 5 patients (38.5%) and TRM occurred in 1 patient. This leads to 6 years EFS and OS of 66.7% and 80% respectively. In conclusion, IB UCBT is safe and well-tolerated in children and hematological recovery compared similarly to the results obtained with IV UCBT.

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Fig. 1: Survival of the 15 patients included in the trial.

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Authors and Affiliations

  1. Cell Therapy and Transplant Program, Division of Hematology-Oncology, Ste-Justine University Hospital Center, Montreal, QC, Canada

    Stephanie Vairy, Isabelle Louis, Johanne Richer, Pierre Teira, Sonia Cellot, Michel Duval & Henrique Bittencourt

  2. Centre Hospitalier de l’Université de Montreal, Montreal, QC, Canada

    Marie-France Vachon

  3. Division of Anesthesia, Ste-Justine University Hospital Center, Montreal, QC, Canada

    Edith Villeneuve

  4. Division of Immunology, Ste-Justine University Hospital Center, Montreal, QC, Canada

    Elie Haddad

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  1. Stephanie Vairy
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Correspondence to Henrique Bittencourt.

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Vairy, S., Louis, I., Vachon, MF. et al. Intrabone infusion for allogeneic umbilical cord blood transplantation in children. Bone Marrow Transplant 56, 1937–1943 (2021). https://doi.org/10.1038/s41409-021-01275-0

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