Abstract | INTRODUCTION: METHODS AND ANALYSIS: Randomised treatment assignment is 2:1 for eltrombopag versus standard first-line management and is stratified by age and by prior treatment. The primary endpoint of the study is platelet response, defined as ≥3 of 4 weeks with platelets >50×109/L during weeks 6-12 of therapy. Secondary outcomes include number of rescue therapies needed during the first 12 weeks, proportion of patients who do not need ongoing treatment at 12 weeks and 6 months, proportion of patients with a treatment response at 1 year, and number of second-line therapies used in weeks 13-52, as well as changes in regulatory T cells, iron studies, bleeding, health-related quality of life and fatigue. A planned sample size of up to 162 randomised paediatric patients will be enrolled over 2 years at 20 sites. ETHICS AND DISSEMINATION: The study has been approved by the centralised Baylor University Institutional Review Board. The results are expected to be published in 2023. TRIAL REGISTRATION NUMBER: NCT03939637.
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Authors | Kristin A Shimano, Rachael F Grace, Jenny M Despotovic, Ellis J Neufeld, Robert J Klaassen, Carolyn M Bennett, Clement Ma, Wendy B London, Cindy Neunert |
Journal | BMJ open
(BMJ Open)
Vol. 11
Issue 8
Pg. e044885
(08 27 2021)
ISSN: 2044-6055 [Electronic] England |
PMID | 34452956
(Publication Type: Clinical Trial Protocol, Journal Article, Research Support, Non-U.S. Gov't)
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Copyright | © Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. |
Chemical References |
- Benzoates
- Hydrazines
- Pyrazoles
- eltrombopag
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Topics |
- Benzoates
(therapeutic use)
- Child
- Clinical Trials, Phase III as Topic
- Humans
- Hydrazines
(therapeutic use)
- Platelet Count
- Purpura, Thrombocytopenic, Idiopathic
(drug therapy)
- Pyrazoles
- Quality of Life
- Randomized Controlled Trials as Topic
- Treatment Outcome
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