LAUSR

Abstract The balance between therapy effectiveness and economic efficiency in chronic immune thrombocytopenia (ITP) becomes more confusing. This single-center open label randomized controlled trial evaluates the effectiveness and safety of hydroxychloroquine in chronic ITP patients against other affordable second-line treatments. It is registered under number (NCT03229746) at Clinical Trials.gov. 120 patients were recruited and randomly allocated to three arms of hydroxychloroquine, vincristine, and azathioprine equally. Platelet counts of more than 100 × 109/L were interpreted as complete response (CR), while response (R) was determined as platelet counts ranging from 30 × 109/L to less than 100 × 109/L with the doubling of the pretreatment platelet count. Overall response (OR) was defined to include both CR and R. Patients were monitored every 6 weeks for a total of 24 weeks. The population baseline characteristics regarding age, sex, duration of the disease, baseline platelets count, and presence of antinuclear antibodies ANA or antiplatelet antibodies were similar among tested groups. There was a significant difference in the overall response between hydroxychloroquine (80.6%) and azathioprine (55.9%) (p-value <0.05). This difference was not significant between hydroxychloroquine and vincristine group (63.2%) (p-value = 0.09). This study proves that hydroxychloroquine can contribute to the therapy of chronic ITP especially as an affordable and well-tolerated drug.