REACH5 final analysis: Ruxolitinib in pediatric treatment-naïve/steroid-refractory cGvHD

Results from the final analysis of the phase II REACH5 study (NCT03774082), evaluating ruxolitinib + corticosteroids (CS) in pediatric patients (aged 2–18 years; N = 45) with treatment-naïve or steroid-refractory moderate-to-severe chronic graft-versus-host disease (cGvHD), were published in Haematologica by Locatelli et al. The REACH5 study design and interim results have been previously reported on the GvHD Hub.

Key data: At a median follow-up of 36 months, the best overall response (BOR) rate was 84.4% (90% confidence interval [CI], 72.8–92.5; complete response [CR], 31.1%; partial response [PR], 53.3%). The median duration of response (DoR), failure-free survival (FFS), and overall survival (OS) were not reached. The 12‑month and 36‑month OS rates were 84.2% (95% CI, 69.7–92.2) and 74.9% (95% CI, 59.3–85.3), respectively. Non-relapse mortality (NRM) was reported in 20.0% of patients. The overall CS‑free response rate was 42.2% (90% CI, 29.7–55.5). Grade ≥3 adverse events (AEs) were reported in 68.9% of patients, while no new safety concerns emerged with longer ruxolitinib exposure.

Key learning: These results demonstrate the efficacy and manageable safety profile of ruxolitinib in pediatric patients with treatment-naïve or steroid-refractory moderate-to-severe cGvHD.