The First FDA-Approved Treatment for Graft Versus Host Disease - Iibrutinib

On 2 August 2017, the United States Drug Agency, FDA, approved ibrutinib (trade name Imbruvica) for adults with chronic graft versus host disease (kGVHH) after failure of one or more systemic treatments.

Ibrutinib Is the First FDA-Approved Treatment for Graft Versus Host Disease.

FDAhad previously approved ibrutinib for chronic lymphocytic leukemia/small lymphocytic lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma with 17p deletion, Waldenstrom macroglobulinemia, marginal zone lymphoma and mantle cell lymphoma. Ibrutinib is among the drugs with the most FDA approvals for different cancers to date.

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• What is graft versus host disease, and how is it treated?

Approval was based on an open-label, multicenter, single-arm study enrolling 42 people with chronic graft versus host disease who needed additional treatment after first-line corticosteroid ( cortisone ) treatment failed. Most participants (%88) initially had at least two involved organs, most commonly the mouth (%86), skin (%81) and gastrointestinal tract (%33).

Patients received 420 mg oral ibrutinib once daily. Overall response rate was reported as % 67 (28 patients). Average time to response, coinciding with the first planned response assessment, was 12.3 weeks (range 4.1-42.1 weeks). Responses occurred in all organs involved by the disease (skin, mouth, gastrointestinal system and liver). Responses lasting at least five months occurred in % 48 of patients (20 patients).

The most common side effects (≥%20) were fatigue, diarrhea, low numbers of platelets, the cells that stop bleeding (thrombocytopenia), with associated bruising, mouth sores (mucositis), muscle spasms, nausea, bleeding, anemia and pneumonia. One patient reportedly developed a heart rhythm disorder. Treatment was discontinued in %24 because of side effects, most commonly fatigue and pneumonia. Side effects leading to dose reduction occurred in %26 of patients.

For graft versus host disease, the recommended ibrutinib dose is 420 mg orally once daily (three 140 mg capsules once daily).

You May Be Interested In:

• What is ibrutinib, and how does it work?