Ruxolitinib Shows Promise as a Safe, Effective Treatment for Steroid-Refractory GVHD

Ruxolitinib therapy is a potentially effective and safe treatment for patients with steroid-refractory graft-versus-host disease (GVHD), according to findings from a systematic review and meta-analysis published in PloS One.

The study aimed to assess the efficacy of ruxolitinib for the treatment of steroid-refractory GVHD and analyze its adverse effects.

“[GVHD], a cause of morbidity and mortality is treated with corticosteroids. However, patients with steroid-refractory GVHD after HSCT have a poor prognosis,” explained Meng-Yun Zhang, MD, School of Clinical Medicine, Guizhou Medical University, Guiyang, GuiZhou Province, China.

The team used the Cochrane Central Register of Controlled Trials, EMBASE, PubMed, and Web of Science databases to identify randomized controlled trials (RCTs) and non-RCTs of ruxolitinib-based therapy in patients with steroid-refractory GVHD. Outcomes included overall response rate, survival, and adverse effects.

The Methodological Index for Non-randomized Studies and the Cochrane collaboration risk-of-bias tool were used to assess methodological quality. In addition, tunnel plots, Egger's test, and the trim and fill method were used to assess publication bias.

A total of 1470 studies were identified, of which 19 studies (17 non-RCTs, 2 RCTs) involving 1358 patients met the inclusion criteria. Survival rates at the longest follow-up in non-RCTs, were 57.5% (95% confidence interval [CI], 46.9 to 67.4) and 80.3% (95% CI, 69.7 to 87.9) for acute GVHD and chronic GVHD, respectively.

In non-RCTs, the overall response was 74.9% (95% CI, 66.6 to 81.8; I2 = 49%) in acute GVHD and 73.1% (95% CI, 62.5 to 81.6; I2 = 49%) in chronic GVHD.

In acute GVHD, the response rates were gastrointestinal, 61.4% to 90.2%; skin, 52.5% to 80.6%; and liver, 41.8% to 71.8%. In chronic GVHD, the response rates were gastrointestinal, 30.1% to 70.4%; skin, 30.1% to 84.4%; lung, 27% to 83%; and mouth 3.5% to 98.1%.

In addition, Dr Zhang and colleagues noted that a lower acute GVHD grade and moderate chronic GVHD were associated with a better clinical response. Common adverse events were cytopenia and infectious complications.

“Our systematic review and meta-analysis indicated that ruxolitinib therapy could be a potentially effective and safe treatment for patients with steroid-refractory GVHD,” they concluded.

Source:

Zhang MY, Zhao P, Zhang Y, Wang JS. Efficacy and safety of ruxolitinib for steroid-refractory graft-versus-host disease: Systematic review and meta-analysis of randomised and non-randomised studies. PloS One. 2022;17(7):e0271979. doi:10.1371/journal.pone.0271979