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®.


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