OBJECTIVES: ITP is a rare disease, characterized by increased platelet destruction/suboptimal platelet production; patients with ITP have an increased risk of bleeds and disease management is complex. We aimed to evaluate the current treatment paradigm for chronic primary ITP and assess impact on healthcare resource utilization in six European countries (DE, ES, FR, IT, NL, UK).

METHODS: Interviews were conducted with 23 physicians and 12 payors (June/July 2020) supplemented by a separate survey of 113 physicians and further validation using published data. These data were used to build a flow model of patient management, by therapy line and severity of platelet depletion, and to determine the resource utilization and costs associated with managing acute bleeds.

RESULTS: From the responses, it was estimated that across all six countries, ~27,000 adults were diagnosed with ITP in 2020 (prevalence, split by disease severity: ~34% mild, ~32% moderate, ~33% severe). Physicians reported that most patients with ITP (60%) were receiving first-line treatment or monitored by their physician; second-/third-line treatment accounted for 40%. Approximately 75% of patients receiving first-line treatment (corticosteroids most common) relapse within 3–4 months; disease stabilization is achieved with thrombopoietin-receptor agonists (TPO-RAs) and rituximab. Patients could switch to an alternative TPO-RA to control symptoms, manage side-effects or improve adherence. The costs of rescue therapies and hospital services (e.g. surgery and admissions) accounted for the majority of healthcare resources to manage bleeds. Serious and life-threatening bleeds accounted for ~40% of all bleeds, which cost approximately €16,000–24,000 and €28,000–41,000 per event, respectively. Consistent with recent ASH guidelines, physicians favored the use of TPO-RAs earlier in the treatment pathway to avoid costly bleeds.

CONCLUSIONS: Physician interviews/survey indicated that following a decline in response, switching TPO-RA can help maintain long-term control of ITP bleeds, which could potentially reduce both hospitalization and therapy costs.