Abstract
ROMILOSTIM FOR TRTEATMENT OF CHILDREN AND YOUNG ADULTS WITH SEVERE APLASTIC ANEMIA AND MYELODYPLASTIC SYNDROME: A SINGLE CENTRE PILOT STUDY
EJC paediatric oncology, Vol.6(Suppl 1), 100365
12/2025
DOI: 10.1016/j.ejcped.2025.100365
Abstract
Background: Severe aplastic anemia (SAA) is a rare but life-threatening condition caused by immune mediated destruction of hematopoietic stem cells. Current standard of care consists of matched sibling donor hematopoietic transplantation and immune suppressive therapy (IST). Thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, have shown promise in inducing trilineage hematopoiesis in patients with SAA.
Methods: 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.
Conclusions: 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
- ROMILOSTIM FOR TRTEATMENT OF CHILDREN AND YOUNG ADULTS WITH SEVERE APLASTIC ANEMIA AND MYELODYPLASTIC SYNDROME: A SINGLE CENTRE PILOT STUDY
- Creators
- Anjali Sharathkumar - University of IowaDavid Claassen - University of IowaSarah Bell - University of Iowa Carver College of Medicine, Internal Medicine, Iowa City, USA
- Resource Type
- Abstract
- Publication Details
- EJC paediatric oncology, Vol.6(Suppl 1), 100365
- DOI
- 10.1016/j.ejcped.2025.100365
- ISSN
- 2772-610X
- eISSN
- 2772-610X
- Publisher
- Elsevier Ltd
- Language
- English
- Date published
- 12/2025
- Academic Unit
- Stead Family Department of Pediatrics; Hematology/Oncology
- Record Identifier
- 9985024266502771
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