Romiplostim in ITP Treatment – Data from German Clinical Practice
Real-world clinical practice data from Germany confirm the beneficial effect and safety profile of romiplostim in patients with immune thrombocytopenia (ITP), both newly diagnosed, persistent, and chronic.
Management of ITP
Primary immune thrombocytopenia (ITP) is an autoimmune disease characterized by a low platelet count (< 100 × 109/L). The essence of the disease is antibody-mediated increased degradation and impaired production of platelets. The definition of ITP also includes the duration of symptoms, categorizing it into newly diagnosed (less than 3 months), persistent (up to 12 months from initial symptoms), and chronic ITP.
Current guidelines for first-line treatment include corticosteroids and intravenous immunoglobulins (IVIG). In the second-line treatment, newer drugs such as thrombopoietin receptor agonists (TPO-RA), like romiplostim, are now included. Numerous clinical observations show that romiplostim may lead to sustained responses even in newly diagnosed or persistent ITP patients who are resistant to corticosteroids. An integrated analysis of 9 studies suggests that romiplostim had a higher likelihood of achieving treatment-free response (TFR) in patients with ITP duration of less than 1 year compared to those with longer disease duration.
Additional clinical experiences are essential for creating recommendations for the optimal use of modern treatment options in ITP patients. Data from routine clinical practice in Germany, published in Acta Haematologica, can aid in this endeavor.
Post hoc Data Analysis
This is a post hoc analysis of a prospective German multicenter observational study in adults who received at least one dose of romiplostim. Data were collected for almost two years, focusing on the overall platelet response (≥ 1× platelet count ≥ 50 × 109/l within 2−24 weeks of starting romiplostim treatment) and sustained platelet response (≥ 75% of measurements showing ≥ 50 × 109/l platelets during 14−24 weeks). Information on adverse events associated with treatment was also gathered.
Results
Data from 96 ITP patients (18 newly diagnosed, 25 persistent, and 53 chronic) were analyzed. During the 2−24 weeks follow-up, overall platelet response was achieved in 100% of patients with newly diagnosed or persistent ITP and 96.2% of chronic ITP patients. Similarly, sustained platelet response was attained in 88.2% of newly diagnosed ITP patients, 65% of persistent ITP patients, and 69.4% of chronic ITP patients.
Regarding observed adverse events, during the 2-year follow-up, they were noted in 24−35.8% of patients across all treatment phases. The most common were fatigue (6.5−14.3%), dizziness (7.9−14.3%), nausea (7.1−10.9%), diarrhea, and vomiting. No thrombotic adverse events were observed. Bone marrow fibrosis was described in 2 chronic ITP patients. In total, 4% of patients discontinued treatment due to adverse events.
Conclusion
The study from real-world clinical practice confirmed the favorable safety profile of romiplostim. It also illustrates its good effect when initiated earlier, supporting its administration in newly diagnosed ITP patients and those with persistent ITP resistant to first-line therapy.
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Source: Reiser M., Josten K. M., Dietzfelbinger H. et al. Romiplostim for primary immune thrombocytopenia in routine clinical practice: results from a multicenter observational study in Germany. Acta Haematol 2022; 145 (4): 394–403, doi: 10.1159/000521689.
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