In a meta-analysis published in PLoS One, researchers evaluated the safety and efficacy of the novel Janus kinase inhibitor, ruxolitinib, for the treatment of steroid-refractory graft-versus-host disease (GVHD) after hematopoietic stem cell transplantation (HSCT). According to the study’s lead author, Meng-Yun Zhang, ruxolitinib therapy was potentially more effective in patients with low-grade disease and gastrointestinal involvement.
The researchers reviewed the Cochrane, EMBASE, PubMed, and Web of Science databases for relevant articles published by March 2021. Their final analysis included 19 studies—two randomized controlled trials (RCT) and 17 non-RCTs—with a combined cohort of 1,358 eligible patients. The outcomes of interest were overall response rate, survival outcomes, and adverse events.
Ruxolitinib Safety and Efficacy in SR-GVHD
At the longest follow-up point in the non-RCTs, the acute GVHD (aGVHD) and chronic GVHD (cGHVD) populations had survival rates of 57.5% (95% confidence interval [CI], 46.9-67.4) and 80.3% (95% CI, 69.7-87.9), respectively. Additionally, aGVHD and cGVHD had overall ruxolitinib response rates of 74.9% (95% CI, 66.6-81.8; I2 = 49%) and 73.1% (95% CI, 62.5-81.6; I2 = 49%), respectively, in the non-RCTs.
According to the analysts, the reported response rates based on aGVHD disease location were as follows:
- Gastrointestinal: 61.4-90.2%
- Skin: 52.5-80.6%
- Liver: 41.8-71.8%
Comparatively, cGVHD had the following response rates:
- Gastrointestinal: 30.1-70.4%
- Skin: 30.1-84.4%
- Lung: 27.0-83.0%
- Mouth: 3.5-98.1%
Patients with lower-grade aGVHD or moderate cGVHD were also associated with an improved ruxolitinib therapy response, the authors noted. Cytopenia and infectious complications were common adverse effects.
Overall, Zhang and colleagues felt that their analysis supported the safety and efficacy of ruxolitinib in the treatment of patients with steroid-refractory acute or chronic GVHD.
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