Real-world experiences with subcutaneous therapy in adults with primary immune thrombocytopenia: A dual cross-sectional survey study of patients and clinicians in Italy.

BACKGROUND: Thrombopoietin receptor agonists (TPO-RAs) are frequently prescribed as second-line therapy for immune thrombocytopenia (ITP). Approved TPO-RAs have different characteristics that can influence doctor-patient shared decision-making. OBJECTIVES: To gain insight into how ITP and its treatment with subcutaneous TPO-RA are perceived by Italian adults with ITP and by treating hematologists. DESIGN: Observational, cross-sectional survey. METHODS: Italian national surveys were conducted in adults with ITP receiving subcutaneous TPO-RA treatment for ≥ 3 months, and separately in hematologists with extensive experience treating ITP. The ITP-PAQ questionnaire captured the impact of ITP and treatments. The patient survey assessed self-reported treatment adherence, setting of TPO-RA administration, administration autonomy, and collected demographic and disease-related data. Methodologic limitations include potential selection bias, absence of a comparison group, and reliance on patient-reported adherence. RESULTS: Between December 2023 and February 2024, the surveys were completed by 50 clinicians and 78 patients distributed across Italy; mean patient age 55.5 (±13.7) years, mean disease duration 2.4 (±2.1) years. Patients were receiving subcutaneous ITP treatment for median 7 (minimum 3-maximum 50) months. The mean total ITP-PAQ score was 70.1±8.5, with approximately 80% of patients reporting little or no impact of ITP on overall quality of life. Most patients (51%) self-administered subcutaneous therapy at home; 27% went to hospital. Overall self-reported adherence was 97%. After the first months of treatment, 90% of patients reported being "extremely satisfied"/"satisfied" (68%/22%). This was maintained at survey, when 95% patients were "extremely satisfied"/"satisfied" (72%/23%). Satisfaction was highest with home self-administration (p=0.09). There were no significant differences (90% confidence level) in patients' and clinicians' responses regarding the main advantages of weekly administration. CONCLUSION: Both patients and specialists indicated that the high satisfaction may be associated with perceived advantages of weekly TPO-RA subcutaneous administration, which may be associated with improved adherence to therapy and reduced medication burden.