Disease management of patients with immune thrombocytopenia—results of a representative retrospective survey in Germany

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.

Treatment patterns in adults with immune thrombocytopenia before, during and after use of thrombopoietin receptor agonists: a longitudinal prescription database study from Germany

“…In the periods before, during and after TPO-RA treatment, oral corticosteroids were prescribed in 64.4% (18-month period), 43.4% (median of approximately 9 months) and 36.1% (median of approximately 14 months) of all patients, respectively. In support of our data, a retrospective analysis of data (gathered via questionnaires) from 26 German hematology centers reported that corticosteroids were the most commonly prescribed first-line treatment for ITP received by 45% of patients (41% underwent a watch and wait approach); 36% and 28% of patients received corticosteroids in the second and third line, respectively [21]. Thus, our study builds upon published data, using a robust and generalizable statutory healthcare prescription database covering approximately 90% of the German population.…”

“…While the preferred sequencing of ITP treatment is well defined on an international and German level, a translational delay from empirical guidelines to realworld clinical practice is well recognized. Indeed, contrary to guidelines, persistent overuse of corticosteroids has been observed [19][20][21]. The present study evaluated treatment patterns prior to, during and after the use of TPO-RAs in adult patients with ITP in the German healthcare setting.…”

Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia: Italian Delphi-based consensus recommendations

“…10 In addition, in a German survey of 1023 patients, 62% of patients receiving steroids as first-line treatment therapy received them for up to 6 months, and 29% received them for more than a year during second-and even third-line treatment. 11 In many patients, ITP becomes persistent or chronic, and second-line treatment may thus be warranted. 5,12,13 In this regard, it should be considered that limiting corticosteroids to the first line of therapy allows earlier transition to secondline therapy.…”

“…10 In addition, in a German survey of 1023 patients, 62% of patients receiving steroids as first-line treatment therapy received them for up to 6 months, and 29% received them for more than a year during second- and even third-line treatment. 11…”

Romiplostim for Primary Immune Thrombocytopenia in Routine Clinical Practice: Results from a Multicentre Observational Study in Germany

“…Our study sample was representative of the general German ITP population, as shown by comparison to patients in a national survey (2016–2017) [ 25 ]. In both study cohorts, over 50% of patients had chronic ITP (>12 months); corticosteroids and IVIg were the most frequently used treatments prior to romiplostim initiation.…”