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Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy
- Authors: Kuter D.J.1, Bussel J.B.2, Newland A.3, Baker R.I.4, Lyons R.M.5, Wasser J.6, Viallard J.7, Macik G.8, Rummel M.9, Nie K.10, Jun S.10
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Affiliations:
- Massachusetts General Hospital, Boston, MA
- Division of Hematology, Departments of Pediatrics and Medicine, Weill Medical College of Cornell University, New York, NY, USA
- Department of Haematology, The Royal London Hospital, Whitechapel, London, UK
- Centre for Thrombosis and Haemophilia, Royal Perth Hospital, Murdoch University, Perth, WA, Australia
- Cancer Care Centers South Texas / US Oncology, San Antonio, TX
- Division of Hematology-Oncology, Department of Medicine, University of Connecticut School of Medicine, Farmington, CT, USA
- Universitй Victor Segalen Bordeaux 2, Hфpital Haut-Lйvкque, CHU de Bordeaux, Pessac, France
- University of Virginia, Charlottesville, VA, USA
- Klinikum der Justus-Liebig-Universität, Giessen, Germany
- Amgen Inc., Thousand Oaks, CA, USA
- Issue: Vol 9, No 3 (2014)
- Pages: 47-62
- Section: PHARMACOTHERAPY
- Published: 30.10.2014
- URL: https://oncohematology.abvpress.ru/ongm/article/view/123
- DOI: https://doi.org/10.17650/1818-8346-2014-9-3-47-62
- ID: 123
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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 × 109 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 × 109 per litre were maintained
with stable doses of romiplostim (mean 5–8 μg / 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 welltolerated 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.
About the authors
D. J. Kuter
Massachusetts General Hospital, Boston, MA
Author for correspondence.
Email: kuter.david@MGH.harvard.edu
Russian Federation
J. B. Bussel
Division of Hematology, Departments of Pediatrics and Medicine, Weill Medical College of Cornell University, New York, NY, USARussian Federation
A. Newland
Department of Haematology, The Royal London Hospital, Whitechapel, London, UKRussian Federation
R. I. Baker
Centre for Thrombosis and Haemophilia, Royal Perth Hospital, Murdoch University, Perth, WA, AustraliaRussian Federation
R. M. Lyons
Cancer Care Centers South Texas / US Oncology, San Antonio, TXRussian Federation
J. Wasser
Division of Hematology-Oncology, Department of Medicine, University of Connecticut School of Medicine, Farmington, CT, USARussian Federation
J.-F. Viallard
Universitй Victor Segalen Bordeaux 2, Hфpital Haut-Lйvкque, CHU de Bordeaux, Pessac, FranceRussian Federation
G. Macik
University of Virginia, Charlottesville, VA, USARussian Federation
M. Rummel
Klinikum der Justus-Liebig-Universität, Giessen, GermanyRussian Federation
K. Nie
Amgen Inc., Thousand Oaks, CA, USARussian Federation
S. Jun
Amgen Inc., Thousand Oaks, CA, USARussian Federation
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