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Efficacy and Safety of Romiplostim in Treating Thrombocytopenia after Hematopoietic Stem Cell Transplantation in Pediatric Patients: A Single-Arm Clinical Trial
Plain-language guide to PMID 41500465 (2026): randomized and multicenter clinical trial study of Romiplostim in 47 patients.
Key Facts
- PMID
- 41500465
- Journal
- Transplantation and cellular therapy
- Year
- 2026
- Study type
- Clinical Trial
- Authors
- Karimizadeh Z et al. (7 authors)
- DOI
- 10.1016/j.jtct.2025.12.1003
What This Study Did, in Plain Language
What Was Tested
The paper reports a study of Romiplostim, published as a clinical trial.
This prospective single-arm clinical trial enrolled 47 pediatric patients (median age 7 yr, range 0.42 to 17 yr) with prolonged isolated thrombocytopenia (n = 14) or secondary failure of platelet recovery (SFPR; n = 33) post-HSCT.
Secondary endpoints included erythroid/neutrophil responses, safety (National Cancer Institute Common Terminology Criteria for Adverse Events v5.0), graft-versus-host disease (GVHD), and 6-mo overall survival (OS)/event-free survival (EFS).
Who Took Part
According to the abstract, 47 patients were involved.
By PubMed's age tags, the people studied were infants, young children, children and teenagers, both men and women.
How the Study Was Run
The researchers set it up as a randomized and multicenter study.
The patients were allocated by chance, so the Romiplostim group and the comparison group should start out alike.
Running at several centres spread the Romiplostim study over different clinics and patient groups.
What Was Measured
This prospective single-arm clinical trial enrolled 47 pediatric patients (median age 7 yr, range 0.42 to 17 yr) with prolonged isolated thrombocytopenia (n = 14) or secondary failure of platelet recovery (SFPR; n = 33) post-HSCT.
Secondary endpoints included erythroid/neutrophil responses, safety (National Cancer Institute Common Terminology Criteria for Adverse Events v5.0), graft-versus-host disease (GVHD), and 6-mo overall survival (OS)/event-free survival (EFS).
PubMed files the paper under topics including Thrombopoietin, Receptors, Fc, Recombinant Fusion Proteins, Hematopoietic Stem Cell Transplantation, Thrombocytopenia, Receptors, Thrombopoietin and Platelet Count.
The authors tagged it with the keywords Thrombopoietin receptor agonist.
What This Means in Plain Language
The paper is a randomized and multicenter study of Romiplostim in 47 patients.
The publication is from 2026 (Transplantation and cellular therapy), a journal based in United States.
More context: the Romiplostim compound page, all human trials.
Our Notes on the Compound
Romiplostim: A peptide-antibody fusion (peptibody) that activates the thrombopoietin receptor to raise platelet counts; sold as Nplate.
Related Pages
Source: PubMed record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.