FDA Approves Ibrutinib in Pediatric Patients With Chronic GVHD


Based on results of the phase 1/2 iMAGINE trial, the FDA has approved ibrutinib for pediatric patients with chronic graft-versus-host disease.

The FDA has approved ibrutinib (Imbruvica) as an oral suspension or in the form of capsules and tablets for pediatric patients 1 year or older with chronic graft-versus-host-disease (GVHD) who have experience with 1 or more prior lines of failed therapy, according to a press release from the FDA.

Results from the approval were based on the phase 1/2 iMAGINE trial (NCT03790332) which enrolled patients ages 1 to less than 22 years old with moderate-to-severe GVHD (n = 47). The overall response rate through week 25 was 60% (95% CI, 44%-74%), and the median duration of response was 5.3 months (95% CI, 2.8-8.8). From first response through events of either death or new systemic therapies for chronic GVHD, the median time was 14.8 months (95% CI, 4.6-not evaluable).

Overall, patients were excluded if they had single organ genitourinary involvement as the only manifestation of chronic GVHD. The median patient age in the population was 13 years (range, 1-19), with 70% of patients being male.

For patients 12 years or older, the recommended dose of ibrutinib is 420 mg orally once daily, and 240 mg/m2 once daily for those under 12. Patients can continue treatment until progression, recurrence, or unacceptable toxicity.

Common adverse effects included anemia, musculoskeletal pain, pyrexia, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.


FDA approves ibrutinib for pediatric patients with chronic graft versus host disease, including new oral suspension. News Release. FDA. August 24, 2022. Accessed August 24, 2022. https://bit.ly/3cgRdJ8

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