ArticleTherapeutic advances in hematology2026
Real-world experiences with subcutaneous therapy in adults with primary immune thrombocytopenia: A dual cross-sectional survey study of patients and clinicians in Italy.
Article in Therapeutic advances in hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
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.
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