A phase 3, single-arm, prospective study of remestemcel-L, ex vivo culture-expanded adult human mesenchymal stromal cells for the treatment of pediatric patients who failed to respond to steroid treatment for acute graft-versus-host disease
The primary report of MSB-GVHD001: seven in ten children with steroid-refractory acute graft-versus-host disease responded to remestemcel-L by day 28, well above the 45 percent historical rate, and responders were far more likely to be alive at six months.
Overview
Phase 3, prospective, single-arm, multicentre study (NCT02336230) in 54 children with primary steroid-refractory acute graft-versus-host disease who were naive to other immunosuppressant therapies for it, treated with remestemcel-L at 2 million cells per kilogram twice weekly for 4 weeks.
Remestemcel-L significantly improved day 28 overall response rate compared with the prespecified control value of 45 percent (70.4 percent versus 45 percent, P = .0003). The response was sustained through day 100, with complete response rising from 29.6 percent at day 28 to 44.4 percent at day 100. Overall survival was 74.1 percent at day 100 and 68.5 percent at day 180. Day 28 response predicted survival: 86.8 versus 47.1 percent at day 100 (P = .0001) and 78.9 versus 43.8 percent at day 180 (P = .003) for responders and nonresponders. No infusion-related toxicities or other safety concerns were identified.
- Day 28 overall response 70.4% against a prespecified control of 45% (P = .0003); complete response 29.6% at day 28 and 44.4% at day 100.
- Overall survival 74.1% at day 100 and 68.5% at day 180.
- Survival at day 180 78.9% in day 28 responders vs 43.8% in nonresponders (P = .003); no infusion-related toxicities.
This is the study behind the December 2024 US approval of remestemcel-L (Ryoncil), the first mesenchymal stromal cell therapy approved in the United States, for children whose acute graft-versus-host disease has not settled on steroids. The label reports the same 54 children as a 70 percent response.
- Single arm, judged against a historical 45% response rate rather than a concurrent control.
- 54 children; the registry full analysis set counts 55.
- Do not confuse with the adult placebo-controlled remestemcel-L study (Kebriaei 2020), a different trial.
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