Up-front Treatment With Romiplostim in Children With Acquired Bone Marrow Failure: A Single Institutional Pediatric Case Series. Issue 3 (April 2021)

• Record Type: Journal Article
• Authors: Al-Huniti, Ahmad; Rathi, Nityam; Modi, Arun; Bhagavathi, Sharathkumar; Mitten, Roxane; Sharathkumar, Anjali A.
• Is Part Of: Journal of pediatric hematology/oncology. Volume 43:Issue 3(2021)
• Publication Date: 2021-04
• Subjects: aplastic anemia -- myelodysplastic syndrome -- TPO-receptor agonist -- romiplostim; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000001810
• Languages: English
• ISSNs: 1077-4114

Abstract

Abstract : Background: Thrombopoietin receptor agonists are emerging as a therapeutic option for patients with aplastic anemia (AA) and myelodysplastic syndrome (MDS). We report our experience of treating children with AA/MDS with romiplostim, thrombopoietin receptor agonist. Observations: Three children (AA, 2; MDS, 1) received romiplostim treatment at a median dose of 10 μg/kg/week (starting dose: 5 μg/kg/wk; 2.5 μg/kg/wk increment). Trilineage hematopoietic recovery occurred at a median of 13 weeks (range: 13 to 16 wk) without adverse events. Hematopoiesis continued to improve after therapy discontinuation (median follow-up: 2.8 y; range: 0.5 to 3.0). Conclusion: Our experience supports the short-term safety and efficacy of romiplostim in children with AA/MDS.