Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome

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Abstract

Thrombopoietin receptor agonists (TPO-RAs) induce trilineage hematopoiesis under conditions with acquired hematopoietic failure. We evaluated safety, tolerability, and preliminary efficacy of a TPO-RA, romiplostim (Nplate), with or without standard-of-care immunosuppressive therapy (± IST) for children (ages

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Sharathkumar, A., Carr, J., Claassen, D., Syrbu, S., Bhagavathi, S., Al-Huniti, A., … Mott, S. L. (2024). Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome. Journal of Pediatric Hematology/Oncology, 46(5), 252–261. https://doi.org/10.1097/MPH.0000000000002891

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