[Effectiveness and safety of ibrutinib for steroid-dependent/refractory chronic graft-versus-host disease: a...

We conducted a single-center retrospective study to evaluate the effectiveness and safety of ibrutinib (IBR) for steroid-dependent/refractory chronic graft-versus-host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation. In the 21 patients assessed, IBR was initiated at a median of 31 months after cGVHD diagnosis. Most patients (71.4%) had severe cGVHD according to the NIH criteria. The median dose of prednisolone was 0.18 (range: 0.01-0.54) mg/kg/day. With a median follow-up of 924 days for survivors and a median IBR treatment duration of 427 days, 2-year overall survival and failure-free survival rates were 56.3% (95% confidence interval [CI]: 32.6-74.5%) and 18.8% (95%CI: 4.9-39.7%). At 6 months, partial response and stable disease (SD) rates among evaluable patients were 19.0% and 28.6%. The most common grade 3 adverse event was infection (52.3%), and IBR was discontinued in 76.2% of patients, most frequently due to infection (38.1%). Despite a higher proportion of severe cases compared to the pivotal trial, approximately half of the patients achieved SD or better at 6 months. The high rates of hospitalization and drug discontinuation due to infection underscore the importance of careful infection management during IBR therapy.