Journal article
Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome
Journal of pediatric hematology/oncology, Vol.46(5), pp.252-261
07/2024
DOI: 10.1097/MPH.0000000000002891
PMID: 38787686
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 <21 y) with newly diagnosed and relapsed/refractory severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS). Data were collected from an observational study and a single arm interventional pilot study. The safety outcome was treatment-related adverse events (AEs). Efficacy was evaluated by complete hematopoietic response (CHR) at week 24. Romiplostim was commenced at 5 µg/kg/week, with dose escalation of 2.5 µg/kg/week (maximum, 20 µg/kg/dose) based on platelet response. Romiplostim was continued until CHR was observed. Ten subjects (SAA, 9 [IST, 4; without IST, 5]; MDS, 1) completed the study (median age: 9.2 y). Median romiplostim dose was 10 µg/kg/week (range: 5 to 17.5 µg/kg/week). The cumulative incidence of CHR was 70.4% (95% CI, 20.2%-92.6%). Among 21 AEs (Grade 1 to 3), 3 were attributed to romiplostim. At a median posttherapy follow-up of 10.9 months (range: 0.7 to 77.5), no clonal evolution, bone marrow fibrosis or mortality was reported. This proof-of-concept study provides data about short-term safety, tolerability, and preliminary efficacy of romiplostim (±IST) for treatment of pediatric SAA/MDS.
Details
- Title: Subtitle
- Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome
- Creators
- Anjali Sharathkumar - University of Iowa, Hematology/OncologyJamie Carr - University of IowaDavid Claassen - Stead Family Department of Pediatrics, Carver College of MedicineSergei Syrbu - University of IowaSharathkumar Bhagavathi - University of IowaAhmad Al-Huniti - Mayo ClinicArunkumar Modi - University of Arkansas for Medical SciencesMelissa Bates - University of IowaSarah L Mott - University of Iowa Hospitals and Clinics
- Resource Type
- Journal article
- Publication Details
- Journal of pediatric hematology/oncology, Vol.46(5), pp.252-261
- DOI
- 10.1097/MPH.0000000000002891
- PMID
- 38787686
- NLM abbreviation
- J Pediatr Hematol Oncol
- eISSN
- 1536-3678
- Language
- English
- Electronic publication date
- 05/24/2024
- Date published
- 07/2024
- Academic Unit
- Stead Family Department of Pediatrics; Pathology; Hematology/Oncology; Fraternal Order of Eagles Diabetes Research Center; Health, Sport, and Human Physiology ; Internal Medicine
- Record Identifier
- 9984630760102771
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