Allogeneic stem cell transplantation with omidubicel in sickle cell disease.
Abstract
Many patients with sickle cell disease (SCD) do not have HLA-matched related donors
for hematopoietic stem cell transplantation (HSCT). Unrelated cord blood (UCB) is
an alternative graft option but is historically associated with high graft failure
rates, with inadequate cell dose a major limitation. Omidubicel is a nicotinamide-based,
ex vivo-expanded UCB product associated with rapid engraftment in adults with hematologic
malignancies. We hypothesized that increasing the UCB cell dose with this strategy
would lead to improved engraftment in pediatric patients undergoing myeloablative
HSCT for SCD. We report the outcomes of a phase 1/2 study in 13 patients with severe
SCD who received omidubicel in combination with an unmanipulated UCB graft and 3 who
received a single omidubicel graft. Grafts were minimally matched with patients at
4 of 6 HLA alleles. Median age at transplant was 13 years. A median CD34+ expansion
of ∼80-fold was observed in omidubicel and led to rapid neutrophil engraftment (median,
7 days). Long-term engraftment was derived from the unmanipulated graft in most of
the double cord blood recipients. Two of the 3 single omidubicel recipients also had
sustained engraftment. Incidence of acute graft-versus-host disease (GVHD) was high,
but resolved in all surviving patients. Event-free survival in the double cord group
was 85% (median follow-up 4 years). All 3 patients in the single cord group were alive
at 1 year after transplantation. Ex vivo expansion of UCB with omidubicel supports
engraftment in patients with SCD. This approach to decreasing the incidence of GVHD
should be optimized for general use in patients with SCD. This study was registered
at www.clinicaltrials.gov as #NCT01590628.
Type
Journal articleSubject
HumansAnemia, Sickle Cell
Graft vs Host Disease
Transplantation Conditioning
Cord Blood Stem Cell Transplantation
Hematopoietic Stem Cell Transplantation
Adult
Child
Permalink
https://hdl.handle.net/10161/24710Published Version (Please cite this version)
10.1182/bloodadvances.2020003248Publication Info
Parikh, Suhag; Brochstein, Joel A; Galamidi, Einat; Schwarzbach, Aurélie; & Kurtzberg,
Joanne (2021). Allogeneic stem cell transplantation with omidubicel in sickle cell disease. Blood advances, 5(3). pp. 843-852. 10.1182/bloodadvances.2020003248. Retrieved from https://hdl.handle.net/10161/24710.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
Collections
More Info
Show full item recordScholars@Duke
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina

Articles written by Duke faculty are made available through the campus open access policy. For more information see: Duke Open Access Policy
Rights for Collection: Scholarly Articles
Works are deposited here by their authors, and represent their research and opinions, not that of Duke University. Some materials and descriptions may include offensive content. More info