Clinical efficacy and safety of ruxolitinib in treatment of chronic graft versus host disease

Objective To study the clinical efficacy and safety of ruxolitinib in the treatment of chronic graft versus host disease (cGVHD). Methods We collected the clinical data of 42 cGVHD patients after receiving allogeneic hematopoietic stem cell transplantation from July 1, 2016 to February 1, 2019 in our hospital. There were 2 specific treatment regimens. For the patients diagnosed with the skin, mouth, eyes, joints or genital tract involvement, 5 mg/d ruxolitinib was given (when the patient weighs less than 25 kg, or ruxolitinib is administered in combination with a potent CYP3A4 inhibitor or fluconazole, the total dose is halved) on the basis of the original immunosuppressant therapy. For those diagnosed with lung, liver or gastrointestinal tract involvement, 5 mg/d ruxolitinib and 1 mg/(kg·d) methylprednisolone were given on the basis of the original immunosuppressant therapy. The clinical efficacy was evaluated in 1 and 3 month after treatment. Peripheral blood test, coagulation function, copy number of human cytomegalovirus and chest CT scanning were performed every week. Results Among the 42 cGVHD patients, overall response rates (ORR) were 85.7% (36/42) and 95.2% (40/42) at 1 month and 3 months after treatment, respectively. In the process of this study, 6 patients developed pulmonary infection, and 4 patients experienced bleeding events. There were 12 patients having human cytomegalovirus and epstein-barr virus infection or the virus copy number being continued to rise. Bone marrow suppression at grade of Ⅰ~Ⅱ presented in 8 patients. During the follow-up period of 4 (1~19) months, no one patient was lost, and 4 patients (9.5%, 4/42) died, due to leukemia recurrence, pulmonary infection and poor control of cGVHD. Conclusion Ruxolitinib has significant efficacy in the treatment of cGVHD, with low adverse reactions and preventable and tolerable effects