Comparative analysis of three treatment approaches for primary immune thrombocytopenic purpura; prednisolone with thrombopoietin, prednisolone alone, and pulse methylprednisolone; a randomized clinical trial

Introduction: Idiopathic thrombocytopenic purpura (ITP) is a hematological disorder characterized by thrombocytopenia (a reduced platelet count) without an identifiable clinical etiology. Various treatment options are available, with glucocorticoids being the primary recommendation. Although recombinant human thrombopoietin (rhTPO) has shown promise in limited studies, data remain insufficient to determine the optimal treatment approach. Objectives: This study compared the therapeutic efficacy of prednisolone+rhTPO, prednisolone alone, and pulse methylprednisolone alone in treating primary ITP. Patients and Methods: This open-label, parallel, randomized clinical trial included 75 patients diagnosed with primary ITP. Participants were randomly assigned to one of three equal treatment groups: pulse methylprednisolone (2–3 mg/kg intravenously, n=25), oral prednisolone (1 mg/kg daily, n = 25), and rhTPO+oral prednisolone (250 μg rhTPO weekly via subcutaneous injection + 0.5 mg/kg daily, n = 25). All patients were followed for seven months, and treatment outcomes, including complete response, partial response, stable response, and adverse events, were compared across the groups. Results: The mean age of the participants was 43.12±11.22 years, and 54.67% were female. Platelet counts showed a significant upward trend in all three groups, with the most pronounced increase observed in the prednisolone + rhTPO group (P<0.001). After seven months, the prednisolone + rhTPO group achieved the highest platelet count. The complete and sustained response rates were 76% and 72% in the pulse methylprednisolone group, 52% and 48% in the oral prednisolone group, and 84% and 88% in the prednisolone+rhTPO group, respectively (P<0.05). The relapse rate of platelet decline was 4% in the prednisolone+rhTPO group, 16% in the pulse methylprednisolone group, and 36% in the oral prednisolone group (P=0.016). Adverse drug events did not significantly differ between groups (P>0.05). Conclusion: The combination of prednisolone+rhTPO provides superior therapeutic efficacy compared to traditional monotherapies, demonstrating higher response rates and a lower relapse rate. Trial Registration: The trial was registered in the Iranian Clinical Trial Registry (IRCT) under the registration number IRCT20230717058813N1 (https://irct.behdasht.gov.ir/trial/71792) and was approved by the ethics committee under the ethical code IR.SBMU.MSP.REC.1402.180.