Abstract
In Europe, ruxolitinib is the first approved treatment for corticosteroid-refractory/-dependent acute or chronic graft-versus-host disease (aGvHD/cGvHD). This retrospective, non-interventional study evaluated the real-world efficacy and safety of ruxolitinib in 56 adult aGvHD patients treated with ruxolitinib from January 2019 through August 2021 in Finland. The primary endpoint was best overall response rate (ORR) at any time. The main secondary endpoints were the time to response and loss of response, overall survival (OS), and corticosteroid discontinuation. The follow-up lasted until death/August 2022. The ORR was 91% (95% CI: 83.5–98.5; complete response [CR], 69.6%; partial response [PR], 21.4%). The median time to best response was 28 days (95% CI: 21–38). The median time to loss of response due to aGvHD progression, cGvHD, or a relapse-related death was 8.8 months (95% CI: 3.3–not reached). The most common cause of discontinuation was the achievement of response (64.3%). Two-thirds of the corticosteroid-treated patients discontinued corticosteroids before the end of follow-up; one-third were on a median dose of 0.2 mg/kg (IQR: 0.1–0.5) at the end of follow-up. The three-year OS was 64.1% (95% CI: 48.2–76.3). Ruxolitinib appears effective and safe in real-world practice. The presented data is in line with the results of clinical trials.