FDA Expands Ibrutinib Indications to Chronic Graft-vs-Host Disease

On August 2, 2017, the U.S. Food and Drug Administration (FDA) approved ibrutinib (Imbruvica) for the treatment of adult patients with chronic graft-vs-host disease after failure of one or more lines of systemic therapy. This is the first FDA-approved therapy for the treatment of chronic graft-vs-host disease.

Trial Details

Approval was based on Study PCYC-1129-CA, an open-label, multicenter, single-arm clinical trial enrolling 42 patients with chronic graft-vs-host disease after failure of first-line corticosteroid therapy and requiring additional therapy. The majority of patients (88%) had at least two organs involved at baseline. The most common organs involved were mouth (86%), skin (81%), and gastrointestinal tract (33%).

Patients received ibrutinib orally at 420 mg once daily. Investigator-assessed overall response rate was 67%, or 28 patients (95% confidence interval [CI] = 51%–80%). The median time to response coinciding with the first scheduled response assessment was 12.3 weeks (range = 4.1–42.1 weeks).

Responses were seen in all organs involved with graft-vs-host disease (skin, mouth, gastrointestinal tract, and liver). Responses lasting 5 months or longer were observed in 48% of the patients (n = 20).

The most common adverse reactions (≥ 20%) were fatigue, bruising, diarrhea, thrombocytopenia, stomatitis, muscle spasms, nausea, hemorrhage, anemia, and pneumonia. Atrial fibrillation occurred in one patient (Grade 3). Treatment was discontinued due to adverse reactions in 24% of the patients. The most common adverse reactions leading to discontinuation were fatigue and pneumonia. Adverse reactions leading to dose reduction occurred in 26% of patients.

The FDA previously approved ibrutinib for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma with 17p deletion, Waldenström’s macroglobulinemia, marginal zone lymphoma, and mantle cell lymphoma.

The recommended dose of ibrutinib for chronic graft-vs-host disease is 420 mg taken orally once daily (three 140-mg capsules once daily).

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.




By continuing to browse this site you permit us and our partners to place identification cookies on your browser and agree to our use of cookies to identify you for marketing. Read our Privacy Policy to learn more.