Effectiveness of ibrutinib in the management of chronic GVHD

Objectives: Chronic graft-versus-host disease (cGVHD) represents a significant adverse event that may ensue following allogeneic hematopoietic stem cell transplantation (Allo-HSCT). In patients resistant to corticosteroids, which is the first-line treatment for cGVHD, ibrutinib is being evaluated as a potential treatment option. In this study, we aimed to share the findings of our multicenter study regarding the outcomes of ibrutinib treatment in patients with corticosteroid-resistant cGVHD who had previously received multiple systemic therapies.

Material and methods: A retrospective analysis was conducted to examine the clinical characteristics and outcomes of patients who received ibrutinib treatment for corticosteroid-resistant cGVHD after Allo-HSCT.

Results: A total of 24 patients diagnosed with cGVHD who received ibrutinib treatment were included in the study. The median age of the patients was 34.5 (20-67). The included patients were followed for a median of 6 (1-30) months. All patients had stem cells collected from the peripheral blood. Fifty percent of the patients had multiple organ involvement, while the other 50 % had single organ involvement. The most frequently affected organs were skin and liver. On average, patients received four (3-5) lines of systemic therapy before ibrutinib treatment. At week 24 of ibrutinib treatment, 10 patients (41.7 %) had a complete response, and 10 patients (41.7 %) had a partial response; at week 48, 8 patients (33.3 %) had a complete response, and 10 patients (41.7 %) had a partial response. The most common hematological side effect after ibrutinib treatment was thrombocytopenia in 5 out of 24 patients, while the most common non-hematological side effect was CMV infection in 6 out of 24 patients.

Conclusion: In patients with corticosteroid-resistant cGVHD, ibrutinib treatment has been demonstrated to be an efficacious option exhibiting an elevated overall response rate and a tolerable side effect profile.