Abstract

Clinical research has resulted in an improvement of treatment options for patients with immune thrombocytopenia (ITP) over the last years. However, only few data exist on the real-life management of patients with ITP. To expand the knowledge, a multicenter, national survey was undertaken in 26 hematology practices distributed all over Germany. All patients with a diagnosis of ITP were documented using questionnaires, irrespective of the diagnosis date over a period of 2 years. Overall, data of 1023 patients were evaluated with 56% of patients being older than 60 years. Seventy-nine percent of the patients had chronic (> 12 months), 16% persistent (> 3–12 months), and 5% newly diagnosed (0–3 months) ITP. In 61% of cases, the disease lasted 3 or more years before survey documentation started. Main strategies applied as first-line therapy consisted of steroids in 45% and a “watch and wait” approach in 41% of patients. During second- and third-line strategies, treatment with steroids decreased (36% and 28%, respectively), while treatment modalities such as TPO-RAs increased (19% and 26%, respectively). As expected, patients with a low platelet count and thus a higher risk for bleeding and mortality received treatment (esp. steroids) more frequently during first line than those with a higher platelet count. Up to a third of patients were treated with steroids for more than a year. Overall, our study provides a cross-section overview about the current therapeutic treatment landscape in German ITP patients. The results will help to improve therapeutic management of ITP patients.

Highlights

  • Immune thrombocytopenia (ITP) is an autoimmune disorder, characterized by transient or persistent decrease of the platelet count [1,2,3]

  • The 26 participating hematology practices provided all available data of their ITP patients by means of a questionnaire designed for the retrospective data collection

  • The data of 1023 patients treated in 26 hematology practices distributed all over Germany were evaluated, giving a strong picture of real-world ITP treatment

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Summary

Introduction

Immune thrombocytopenia (ITP) is an autoimmune disorder, characterized by transient or persistent decrease of the platelet count (less than 100 × 109/l) [1,2,3]. Patients with ITP have no clinical symptoms and the diagnosis is due to a routine blood analysis. ITP is diagnosed because of mild bleeding symptoms, with severe bleeding being a relatively rare event [5]. The diagnosis of ITP is established only after exclusion of secondary causes of thrombocytopenia, like infections, autoimmune or myeloid disorders, as there are no diagnostic tests to confirm ITP [3]. While the 2011 guidelines of the American Society of Hematology (ASH) suggest treatment for newly diagnosed adult patients with a platelet count < 30 × 109/l irrespective of

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