Investigators sought to characterize first-line and subsequent therapies for immune thrombocytopenia (ITP) used in Australia, noting that access to recommended second-line therapies, including rituximab and thrombopoietin agonists (TPO-A) is restricted in the country. The authors identified a wide range of second-line agents with differing efficacy and safety profiles, with rituximab and TPO-A demonstrating the strongest efficacy.
The analysis, published in Research and Practice in Thrombosis and Haemostasis, included 322 retrospective patients with ITP treated at 8 participating centers across Australia between 2013 and 2020. Mean age at diagnosis was 48.8 ± 22.6 years, and 58.3% of patients were women. Researchers compared data and frequency among centers using descriptive statistics, pivot tables, and paired t tests.
Effective Second-Line Options Exist, but Australians Lack Access
The ITP diagnosis was primary in 72% of patients and secondary in 28%, and 95% of patients had first-line treatment with 1 or more of prednisolone (76%), dexamethasone (15%), or intravenous immunoglobulin (48%). Notably, patients with secondary ITP were less steroid-dependent (72% vs 76%) and required less second-line therapy (47% vs 58%).
More than half (56%) the cohort received 1 or more second-line agents, and the mean number of second-line therapies per patient was 1.9 ± 1.2. The most commonly used treatments were rituximab, eltrombopag, and splenectomy, which yielded the highest rates of complete response at 60.3%, 72.1%, and 71.8%, respectively. The authors noted that long-term corticosteroids and splenectomy had the least favorable side effects.
Ultimately, the study’s authors judged that effective second-line therapies for ITP are available, but they reemphasized that access to these agents is limited in Australia and there is a lack of direct comparison studies. Consequently, treatment choices are largely based on the experience of individual providers.