Dr. Kurtzberg on Long-term OS for Remestemcel-L in Pediatric Steroid-Refractory aGVHD

Joanne Kurtzberg, MD, Jerome S. Harris Distinguished professor of Pediatrics, professor of Pediatrics, professor of Pathology, Core Faculty in Innovation & Entrepreneurship, member, Department of Pediatrics, Duke School of Medicine, the Duke Cancer Institute, Affiliate of the Duke Regeneration Center, discusses the long-term survival outcomes from an observational cohort study evaluating remestemcel-L in pediatric patients with steroid-refractory acute graft-vs-host disease (aGVHD).

The observational study included patients who were enrolled on the phase 3 MSB-GVHD001 trial (NCT02336230) who received at least 1 dose of remestemcel-L and had data reported to the Center for International Blood & Marrow Transplant Research (CIBMTR). Findings presented at the 2023 Transplantation and Cellular Therapy Meetings showed that the 4-year overall survival (OS) rate was 48.7% (95% CI, 35.1%-62.3%). The 1-, 2-, and 3-year OS rates were 62.7%, 50.8%, and 48.7%, respectively. Moreover, patients with grade IV/D aGVHD, which is the most severe form, experienced a 4-year OS rate of 48% and compared favorably with previously published results for patients in this subgroup, Kurtzberg says.

OS served as the primary end point of the observational study, and investigators also sought to compare the survival data for this population with CIBMTR data, Kurtzberg continues. This was done to ensure comparator data came from a neutral database that collects patient data from all transplant center, Kurtzberg notes.

In the original MSB-GVHD001 trial, 54 patients were treated, and the primary end point was overall response rate at 28 days, Kurtzberg says. However, in the observational cohort, 51 of the 54 original patients were available for follow-up and were analyzed, Kurtzberg concludes.