FDA approved ibrutinib for (cGVHD) 1 year aged patients

Pediatric patients aged 1 year and above with chronic graft-versus-host disease (cGVHD) can now benefit from ibrutinib following the FDA approval. The FDA green light for ibrutinib was granted based on the efficacy results of the open-label, multicenter, single-arm trial, iMAGINE(NCT03790332). The 47 participating patients (aged 1 to less than 22 years with moderate or severe cGVHD) required additional therapy after failure of one or more lines of systemic therapy.

The main efficacy outcome was the overall response rate (ORR) through the Week 25 measure. According to the 2014 NIH Consensus Development Project Response Criteria, ORR included complete or partial responses. By Week 25 ORR was 60% (95% CI: 44, 74). The median duration of response was 5.3 months (95% CI: 2.8, 8.8), and the median time from first response to death or new systemic therapies for cGVHD was 14.8 months (95% CI: 4.6, not evaluable).

According to iMAGINE trial, the most common (≥20%) adverse reactions, including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.