Characterization and treatment of immune thrombocytopenia in Europe: a qualitative observational study. Issue 1 (1st January 2021) - Pogna, Edgar A.
ABSTRACT: Background: Immune thrombocytopenia (ITP) is a rare disease, characterized by increased platelet destruction/suboptimal platelet production, leading to thrombocytopenia and risk of severe bleeding events. Methods: Interviews with 23 physicians and 12 payors, a survey with 113 physicians and validation using published data were used to define the current treatment paradigm and healthcare resource utilization and to determine the costs associated with managing acute bleeds in six European countries (Germany, Spain, France, Italy, Netherlands, UK). The study estimated a prevalence of 9 to 10 per 100, 000 adults in 2020 across all six countries (disease severity split: 34% mild, 32% moderate, 33% severe (due to rounding up some values might not sum up to 100%). Results: Physician feedback showed that most patients with ITP (60%) received first-line treatment or were monitored by their physician; ∼75% of patients relapsed within 3–4 months. Thrombopoietin-receptor agonists (TPO-RAs) and rituximab were used to achieve disease stabilization in patients who relapse; 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 bleeding events. Conclusion: Physicians would welcome earlier use of TPO-RAs to help maintain long-term control of ITP bleeds and potentially reduce both hospitalization and therapy costs.