RESUMEN
The disease course in a young girl with chronic immune thrombocytopenia (ITP) at the initial age of 19 months, treated with eltrombopag, was evaluated retrospectively and is presented as a case record and discussed against the background of the available literature. A stable response and reduction in clinical symptoms, over several years and without frequent dose changes, was achieved. Bleeding symptoms were mild to moderate and occurred particularly frequently in combination with low platelet counts. Raising the platelet count, in turn, was accompanied by a decreased bleeding tendency. Eltrombopag was tolerated well. No new safety signals were observed during the treatment. Based on a follow-up of more than 2.5 years, our case confirms that a child with chronic ITP can benefit from treatment with eltrombopag in the regular care setting. We assume that early treatment with a thrombopoietin receptor agonist could save many children from repeated and lengthy hospitalizations with intravenous immunoglobulins and prolonged administration of corticosteroids.
RESUMEN
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.
