BTK participates in a variety of signal transduction of innate and adaptive immunity, and has an important role in cell proliferation, differentiation and apoptosis. The impact of BTK inhibitors on hematological malignancies and autoimmune diseases has been well studied. This project was undertaking by Qilu Hospital of Shandong University in China. In order to report the efficacy and safety of the selective BTK inhibitor Orelabrutinib in the management of refractory ITP.
The investigators are undertaking a prospective, open-lable, multicentre trial of 40 refractory ITP adult patients in China. Eligible participants will receive Orelabrutinib in 50 mg po. qd, every 4 weeks for one cycle and it will be given 3 cycles. For non-responders who were well tolerated at 12 weeks of follow-up, the treatment could be extended to 6 cycles. The treatment will be discontinued after 6 months without blood index reaction. In order to report the efficacy and safety of Orelabrutinib in the management of refractory ITP, platelet count, bleeding and other symptoms will be evaluated before and after treatments. Adverse events are also recorded throughout the study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Orelabrutinib 50mg po qd, every four weeks for one cycle. It will be given three cycles.
Initial overall response to Orelabrutinib
Complete response was defined as a platelet count of 100×10⁹ cells per L or higher and an absence of bleeding. Partial response was defined as a platelet count of 30×10⁹ cells per L or higher, but less than 100×10⁹ cells per L, and at least a doubling of the baseline platelet count and an absence of bleeding. Platelet counts were confirmed on two separate occasions at least 7 days apart when defining complete response or partial response. No response was defined as a platelet count of less than 30×10⁹ cells per L, or less than two-times increase from baseline platelet count, or bleeding.
Time frame: 14 days after the first dose of Orelabrutinib
Sustained overall response to Orelabrutinib
Complete response was defined as a platelet count of 100×10⁹ cells per L or higher and an absence of bleeding. Partial response was defined as a platelet count of 30×10⁹ cells per L or higher, but less than 100×10⁹ cells per L, and at least a doubling of the baseline platelet count and an absence of bleeding. Platelet counts were confirmed on two separate occasions at least 7 days apart when defining complete response or partial response. No response was defined as a platelet count of less than 30×10⁹ cells per L, or less than two-times increase from baseline platelet count, or bleeding.
Time frame: A response lasting for at least 6 months without any additional intervention specific to primary immune thrombocytopenia was defined as a sustained response
Time to response
Time from treatment initiation to achieve a complete response or a partial response
Time frame: An average of 6 months
Duration of response
Time from achievement of a complete response or a partial response to the loss of response (platelet count \<30×10⁹ cells per L; measured on two occasions more than 1 day apart or the presence of bleeding).
Time frame: through study completion, an average of 1 year
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Therapy associated adverse events
Potential adverse events: leukopenia (report in number \* 10\^9/L, time of occurrence and duration); nausea and diarrhea (report in frequency); infection (report pathogens and infectious diseases).
Time frame: Up to 1 year