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EBMT 2021 | Overcoming challenges of UCB transplantation

Guillermo Sanz, MD, PhD, University Hospital La Fe, Valencia, Spain, discusses the advantages and drawbacks of umbilical cord blood (UCB) transplantation and the history of omidubicel at EBMT 2021. UBC transplantation has demonstrated a lower relapse risk in higher-risk patients with acute leukemia, including those who are measurable residual disease (MRD) positive before transplantation. The biggest drawback associated with UBC transplantation is delayed engraftment due to low numbers of progenitor cells. Prof. Sanz describes how omidubicel was developed to overcome these challenges. Omidubicel consists of hematopoietic stem cells expanded in the presence of nicotinamide and hematopoietic cytokines, to enable a high number of progenitor cells and retention of differentiation potential, functionality, and phenotype. This interview took place during the 47th Annual Meeting of the European Group for Blood and Marrow Transplantation (EBMT) 2021.

Transcript (edited for clarity)

Umbilical cord blood transplantation has several advantages over adult unrelated transplantation. And they include first, that they are readily available for transplantation, that they allow a greater HLA-match disparity between the donor and the recipient, and also, they have demonstrated a lower relapse risk in higher risk patients with acute leukemia, including those with a minimal residual disease positive before the transplant...

Umbilical cord blood transplantation has several advantages over adult unrelated transplantation. And they include first, that they are readily available for transplantation, that they allow a greater HLA-match disparity between the donor and the recipient, and also, they have demonstrated a lower relapse risk in higher risk patients with acute leukemia, including those with a minimal residual disease positive before the transplant.

The major drawback of umbilical cord blood transplantation is the very delayed engraftment of umbilical cord blood cells. And this is due to the low progenitor cell content in the graft, and this results in a greater transplant-related mortality and morbidity, and also in a higher use of hospital resources.

We thought as one possibility to overcome these issues would be to use standard cord blood cells. And that’s what did in this omidubicel clinical trial comparing omidubicel, that is a three-week expanded CB unit in the presence of nicotinamide and other hematopoietic cytokines that allows a very high number of progenitor cells while retaining the differentiation capability of the cells and maintaining both the functionality and the phenotype of the stem cells.

And this is why we started this randomized Phase III clinical trial comparing omidubicel expanded cord blood cells with the standard of care cord blood transplantation.

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Disclosures

Prof. Guillermo Sanz, MD, PhD, has received research funding from Celgene/BMS, Gamida Cell and Novartis; has received speaker’s fees from Takeda, has received honoraria from Celgene/BMS; has fulfilled a consulting or advisory role for AbbVie, Amgen, Boehringer-Ingelheim, Celgene/BMS, Helsinn Healthcare, Janssen, Novartis, Roche and Takeda, and has received travel and/or accommodation expenses from Celgene/BMS, Gilead, Roche and Takeda.

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