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Cord blood transplant via NiCord reduces time to neutrophil, platelet recovery
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SALT LAKE CITY — NiCord single unit expanded umbilical cord blood transplantation decreased time to neutrophil and platelet recovery, according to results of a phase 2 study presented at the BMT Tandem Meetings.
“Regardless of where the stem cells are coming from, hematopoietic transplant is very risky. There is a period of extreme vulnerability in the first 3 months which can continue up to 6 months,” Mitchell E. Horwitz, MD, professor of medicine at Duke University School of Medicine, told HemOnc Today. “Cord blood has historically had a long period of vulnerability [and] what these data show is that the period of vulnerability can be reduced by reducing the time it takes for blood counts to recover.”
NiCord (Gamida Cell) is an ex vivo-expanded, cryopreserved graft derived from an entire cord blood unit.
Horwitz and colleagues sought to determine if NiCord could be used as a standalone graft and increase the number of hematopoietic stem and progenitor cells within the cord blood graft to overcome delayed or failed engraftment after adult umbilical cord blood transplantation.
The analysis included 36 patients (median age, 44 years) who underwent HSCT at 10 clinical sites throughout the United States, European Union and Singapore. All patients received myeloablative conditioning with a total body irradiation-based (n = 14) or chemotherapy-only (n = 21) regimen. Graft-versus-host disease prophylaxis included mycophenolate mofetil and tacrolimus or cyclosporine.
Cumulative incidence of NiCord-derived neutrophil engraftment served as the primary endpoint.
Results showed NiCord processing led to a median 33-fold (range, 20-52) increase in CD34-positive cells relative to those reported by the cord blood bank prior to cryopreservation, which resulted in a median CD34-positive cell dose of 6.3 x 106/kg (range, 1.4-14.9).
The cumulative incidence of engraftment was 94% at day 42. Two patients experienced secondary graft failure; one at day 40 due to human herpesvirus 6 infection, and another at day 260 due to lethal adenovirus infection.
Neutrophil engraftment occurred at a median of 11 days (95% CI, 9-13). Platelet engraftment occurred at a median 34 days (95% CI, 32-42).
“We showed there is a 10- to 12-day reduction in time to blood count recovery compared [with] standard stem cell transplant,” Horwitz said.
Researchers observed full donor whole blood chimerism among 97% of engrafted patients by day 100 posttransplant.
The incidence of acute GVHD was 44% for grades 2 to 4 and 11% for grades 3 or 4.
At 1-year posttransplant, cumulative incidence of chronic GVHD was 40% and moderate to severe chronic GVHD was 10%. The 100-day incidence of grade 2 to 3 bacterial (n = 9) or grade 3 fungal (n = 0) infections was 23.9%.
Over a median follow-up of 14 months (range, 1-35), nonrelapse mortality rates were 12.8% at 6 months and 20.3% at 2 years. At 2 years, the cumulative incidence of relapse was 33%.
OS at 2 years was 51.2% (95% CI, 32.9-66.8).
No unique limitations in using NiCord were observed throughout the study, Horwitz said.
“Cord blood has unique properties not requiring a match, so it gives patients who don’t have matched donors an option for transplant,” Horwitz said. “These data show that umbilical cord blood transplantation using NiCord technology could be an excellent first option for adult patients without a matched adult donor.”
Other key takeaway messages from the data include the ability to expand and grow stem cells outside the body, the time to blood count recovery is unprecedented regardless of what the graft is derived from, and the ability to develop and deliver these expanded cord blood grafts to sites around the world.
“There was a question on whether this type of thing is feasible, and the study demonstrates it is, in fact, feasible,” Horwitz said. – by Melinda Stevens
Reference:
Horwitz ME, et al. Abstract 49. Presented at: BMT Tandem Meetings; Feb. 21-25, 2018; Salt Lake City.
Disclosures: Horwitz reports research funding from Gamida Cell. Please see the abstract for all other authors’ relevant financial disclosures.