Key Points
• Conclusion/Relevance: “Among patients with glucocorticoid-refractory or -dependent chronic GVHD, ruxolitinib led to significantly greater overall response, failure-free survival, and symptom response. The incidence of thrombocytopenia and anemia was greater with ruxolitinib.”
• In the current Phase 3 open-label (REACH3), randomized trial, researchers assessed the efficacy/safety of ruxolitinib (Jakafi) at 10 mg BID vs 10 commonly used alternatives in patients in 329 patients aged 12 years and older with moderate or severe glucocorticoid-refractory or glucocorticoid-dependent chronic GVHD. The overall response at week 24 was 49.7% in the experimental group vs 25.6% in the control group. The safety profile aligned with expectations.
• Results of the current trial demonstrated the superiority of ruxolitinib vs other second-line treatments, such as ibrutinib and extracorporeal photopheresis. Overall response with the agent was higher at week 24 despite organs involved. There was also a greater “best overall” response (i.e., at any time); longer duration of response; and greater failure-free survival with ruxolitinib.
• “The absence of a strong end point, such as glucocorticoid-free remission, and the presence of confounders, including concomitant treatments, make determination of the effect on glucocorticoid dose over time with ruxolitinib as compared with commonly used therapies difficult,” the authors wrote. “However, patients treated with ruxolitinib had consistent reductions in glucocorticoid dose over time, suggesting a glucocorticoid-sparing effect, a finding in line with previous observations.”
• According to the authors, an open-label study design was required to attenuate bias. The researchers analyzed responses via the newest NIH criteria.