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Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: Safety and efficacy
Journal article   Peer reviewed

Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: Safety and efficacy

D.J. Kuter, J.B. Bussel, A. Newland, R.I. Baker, R.M. Lyons, J. Wasser, J-F Viallard, G. Macik, M. Rummel, K. Nie, …
British Journal of Haematology, Vol.161(3), pp.411-423
2013
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Abstract

Romiplostim was effective, safe, and well-tolerated over 6 – 12 months of continuous treatment in Phase 3 trials in patients with immune thrombocytopenia (ITP). This report describes up to 5 years of weekly treatment with romiplostim in 292 adult ITP patients in a long-term, single-arm, open-label study. Outcome measures included adverse events (including bleeding, thrombosis, malignancy, and reticulin/fibrosis), platelet response (platelet count > 50 9 10 9 per litre), and the proportion of patients requiring rescue treatments. Treatment – related serious adverse events were infrequent and did not increase with longer treatment. No new classes of adverse events emerged. Thrombotic events occurred in 6 5% of patients and were not associated with platelet count. Median platelet counts of 50 – 200 9 10 9 per litre were maintained with stable doses of romiplostim (mean 5 – 8 l g/kg; generally self-administered at home) throughout the study. A platelet response was achieved at least once by 95% of patients, with a platelet response maintained by all patients on a median 92% of study visits. There was a low rate of bleeding and infrequent need for rescue treatments. In conclusion, this study demonstrated that romiplostim was safe and well-tolerated over 614 patient-years of exposure in ITP patients, and that efficacy was maintained with stable dosing for up to 5 years of continuous treatment.

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Collaboration types
Industry collaboration
Domestic collaboration
International collaboration
Citation topics
1 Clinical & Life Sciences
1.103 Blood Disorders
1.103.1225 Immune Thrombocytopenia
Web Of Science research areas
Hematology
ESI research areas
Clinical Medicine
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