Abstract

To describe real-world treatment patterns and steroid usage as well as the steroid prescription alignment to clinical guidelines, prior to the use of thrombopoietin receptor agonists (TPO-RAs) in patients with immune thrombocytopenia (ITP) in Germany. The IMS® LRx prescription database was used to identify patients with at least one TPO-RA (romiplostim or eltrombopag) prescription between 7/2016 and 6/2019 in Germany. ITP treatments received prior to TPO-RA therapy were assessed. In total 3,553 patients were included in the analysis (2,752 eltrombopag, 801 romiplostim). Overall, 48% of patients were male and 57% of patients were >60 years when initiating TPO-RA. Oral steroids were the most commonly used therapy before TPO-RAs, in 2,289 of patients (64%; 69% eltrombopag, 48% romiplostim), followed by intravenous immunoglobulins (IVIg) in 249 of patients (7%; 6% eltrombopag, 10% romiplostim). Rituximab or other ITP treatments (e.g. azathioprine, mycophenolate mofetil, cyclophosphamide, dapsone) were rarely used before TPO-RAs. Rituximab was used in 2% of patients (1% eltrombopag, 4% romiplostim), other prior treatments in 5% of patients (5% eltrombopag, 6% romiplostim). Mean total days of steroid use prior to TPO-RAs was 169 days (median 105 days, maximum 540 days) and mean cumulative steroid (prednisolone equivalent) dose before TPO-RA therapy was 3,328 mg (median 2,522 mg, maximum 39,702 mg). Oral steroids were the most commonly used medication before TPO-RA therapy, reflecting their continued importance in the management of newly diagnosed ITP. However, treatment duration and cumulative steroid dose exceeded current guideline recommendations (ie from the American Society of Hematology 2019) and international consensus on ITP treatment. This over-use of steroids has the potential to lead to serious side effects and negatively impact on mental health, mood, sleep or weight gain for patients.

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