Evaluating Ruxolitinib for Chronic Graft-versus-Host Disease

Treatment with ruxolitinib appeared to be safe and effective in patients with chronic graft-versus-host disease (GVHD), according to results from a retrospective analysis published in Leukemia Research.

Approximately half of patients with GVHD treated with steroids will require additional therapy due to a lack of sustained response, the authors wrote. Ruxolitinib has recently been approved by the US Food and Drug Administration for the treatment of acute GvHD.

The researchers reviewed efficacy and safety outcomes in a cohort of 36 patients diagnosed with moderate (25%) or severe (75%) steroid-refractory or steroid-dependent chronic GVHD. Cutaneous GVHD was the most frequent presentation.

Of this cohort, patients began ruxolitinib after a median of three previous lines of therapy (range, 1-11) for a median of 8.6 months (range, 1-51.6). Response evaluation was performed at three and six months.

The overall response rate (ORR) at three months was 59%, which included a complete response rate (CRR) of 9% and a partial response rate (PRR) of 50%. At six months, the ORR was 62 % (CRR=15%; PR=46 %).

Regarding safety, no other permanent discontinuations due to adverse events were documented. Cutaneous, oral, genital, and ocular GVHD significantly improved after treatment, the authors reported. Rates of two-year overall survival and two-year transplant-related mortality were 74% and 19% respectively. They also observed that ruxolitinib was associated with a significant reduction of steroid dose.

Based on these findings, “ruxolitinib was confirmed to be a safe and effective option as salvage treatment also for advanced stages of chronic GVHD,” the authors concluded. “Longer follow-up is needed to evaluate durability of response. Prospective analyses on larger cohorts are ongoing.”