Real-World Management of Patients With Immune Thrombocytopenia in Italy: A Retrospective Database Study

OBJECTIVES: Immune thrombocytopenia (ITP) is an autoimmune disease characterized by a low platelet count. Newly diagnosed patients are traditionally managed with corticosteroids (CS), but guidelines discourage long-term steroid therapy. In patients with steroid-dependency or unresponsiveness, other therapy options are rituximab, thrombopoietin receptor agonists (TPO-RA), and splenectomy. The present real-world analysis described the ITP adult population in Italy, focusing on its epidemiology, patients’ therapeutic management, and healthcare costs.

METHODS: Administrative databases of Italian healthcare entities covering about 12,000,000 residents were used to identify patients with ITP through specific exemption or hospitalization codes or TPO-related prescriptions between Jan-2015 and Sep-2022.

RESULTS: Total 2,891 ITP adults were included (mean age 57.6 years, 43% males). Prevalence rates of ITP rose over time, from 128/1,000,000 people in 2015 to 273.7/1,000,000 people in 2022. The analysis of treatment patterns revealed that 9% of patients were not actively treated for ITP. Among treated patients, 57.8% received CS only, while 42.2% received also TPO-RA or fostamatinib. The total duration of treatment with CS was 11.1-14.0 months, while 69.7% of the cycles lasted longer than 3 months. At least one therapy course lasting more than 3 months was received by more than half of the population (51.7%). Total splenectomy was performed in 4% of patients. Overall healthcare costs/patient at 1-year follow-up was €10,608, mostly driven by drug expenses (€6,883, of which €5,571 for ITP-related drugs), followed by hospitalizations (€2973 of which €1,657 for ITP-related hospitalizations), and outpatient specialist services (€751.5). Patients with splenectomy showed higher total costs than those without splenectomy (€19,320 and €10,243, respectively), one-third for hospitalization expenses.

CONCLUSIONS: Despite the advent of novel treatment options, part of patients with ITP receive long-term corticosteroid therapy. These findings suggest that ITP therapeutic management still needs to be optimized, also in view of a better allocation of healthcare resources.