Treatment Options for Patients With Chronic Graft vs Host Disease

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My name is Meilin Diaz-Paez, and I am a family certified nurse practitioner. I currently work at the University of Miami Sylvester Comprehensive Cancer Center with the Division of Transplantation & Cellular Therapy. I have been a nurse practitioner with transplant for about seven years and spent an additional seven years as a registered nurse at the bedside.

What treatment options currently exist for patients with chronic GVHD?

Currently, we use calcineurin inhibitors such as tacrolimus, sirolimus, mycophenolate, corticosteroids, extracorporeal photophoresis (which is actually a procedure, not a medication), sunitinib, selemucidal, and many more. We use a lot of extracorporeal photophoresis at the University of Miami, and the newer agents like belumosudil and ruxolitinib have been gaining a lot of traction lately.

Can you discuss the ROCKstar study, including its participants and key findings?

The ROCKstar study was a pivotal phase 2 multicenter open-label randomized study. It evaluated two dosages for belumosudil: 200 milligram once a day, and 200 milligram twice a day. It included allogeneic stem cell transplant recipients aged 12 years and older. Patients had to have had active chronic GVHD and had 2 to 5 prior lines of systemic therapy at the time systemic therapy was indicated.

Its primary endpoint was to evaluate the best overall response at any time during the 12 months of enrollment. They also looked at secondary endpoints, which evaluated safety, duration of response, time to response, effect on quality of life, effect on corticosteroid dosage and calcium neuron inhibitor dosages, failure-free survival, and overall survival rate.

Results showed a 75% overall response rate. Time to response was as early as 4 weeks; 63% of responses were observed between 4 to 8 weeks, and 94% of responses were observed by week 24 or approximately 6 months into treatment.

Sixty-two percent of responders did not experience death or new systematic therapy initiation for more than 12 months after response. The failure-free survival rate was 73% at 6 months and 57% at 12 months after their response.

Fifty-two percent of patients reported improvement in quality of life, and this was evaluated with the Lee Symptom Scale scores. Sixty-four percent of patients saw reductions in their corticosteroid dose, and 20% were actually able to stop their corticosteroid doses altogether. Forty-two percent were able to reduce the dose in the calcium neuron inhibitor therapy, and 17% were able to discontinue calcium neuron inhibitor therapy altogether.

Permanent discontinuation due to adverse effects occurred in about 18% of patients, and the most common adverse effect was nausea. There were no reports of cytomegalovirus infection and cytopenias grade 3 or higher were reported in less than 4% of patients. Infection grade 3 or 4 was noted in about 16% of patients, 4% being viral and 4% being bacterial.

How can these findings on belumosudil be applied in real-world clinical practice?

Patients need to be started promptly on effective treatment to block the inflammatory and fibrotic process that we see in chronic GVHD, while at the same time not suppressing the immune system further, which increases the risk for infections.

Patients with progressive chronic GVHD not responding to 2 lines of therapy are candidates for belumosudil early in their disease progress, which is important.

This transcript has been edited for clarity.