Corey S. Cutler, MD, MPH, medical director of the Stem Cell Transplant Program, Dana-Farber Cancer Institute, discusses the current treatment options for patients with chronic graft-versus-host-disease (GVHD) and highlighted some upcoming research.

Corticosteroids remains the mainstay of therapy in chronic GVHD, says Cutler. However, 50% to 60% of patients who receive steroids will become refractory within the first year of therapy, so there is another FDA-approved treatment available, which is the BTK inhibitor Ibrutinib (Imbruvica). This agent, which is approved for patients with steroid-refractory chronic GVHD, demonstrated a relatively impressive response rate around 60% in a small open-label phase II clinical trial.

Beyond ibrutinib, Cutler says there is no approved third-line option for patients with chronic GVHD. The most promising agent, however, is KD025, a ROCK2 inhibitor. He expects to hear more about this agent in the coming years, but a recent randomized phase II trial showed responses in approximately 65% of heavily pretreated patients. Cutler says experts anxiously await the final results of this study, and hopefully there will be an FDA approval to follow as well.

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