IRCT20221129056655N1 IRCT 2023-06-01 Shiraz University of Medical Sciences Romiplostim in treatment-refractory acquired aplastic anemia Assessment of efficacy and safety of Romiplostim in immunosuppressive-refractory children with severe acquired aplastic anemia 2023-06-22 anticipated 24 Complete https://irct.ir/trial/70398 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Single, Purpose: Treatment. 2-3 Acquired aplastic anemia. Intervention group: Romiplostim is administered subcutaneously at a fixed dose of 10 mcg/kg weekly for 4 weeks (weeks 1-4) followed by titrated dose steps of 10, 15, and 20 mcg/kg once weekly up to 27 weeks (weeks 5-27). The romiplostim dose is adjusted depending on platelet response and toxicity. The dose is increased by one step every 4 weeks until a platelet response is achieved. If the platelet count is >200 ≥ 109/L, the dose is reduced by one step. The romiplostim dose is tapered with the intent to discontinue when trilineage hematopoiesis is achieved. Trilineage hematopoiesis is defined as a platelet count of >50 × 109/L, hemoglobin concentration of >10 g/dL and neutrophil count of >1×109/L maintained for 8 weeks with the same romiplostim dose without transfusion.. Yes - There is a plan to make this available What will be shared: Patients' data will be shared anonymously. When: The data will be accessible after the results are published To whom: Academic investigators will have access to the data upon their request. Conditions: Researchers can use data for research purposes with the permission of the principal investigator. Where to obtain: Send an e-mail to the PI to receive the data. How to obtain: The request will be sent to the Ethics Committee of the University. If there is no ethical concern, they will be sent to them. Comments: