Romiplostim in the management of severe aplastic anemia: a comprehensive clinical review

Severe aplastic anemia (SAA) is a rare bone marrow failure disorder with high morbidity and mortality, particularly in patients ineligible for hematopoietic stem cell transplantation. While immunosuppressive therapy (IST) remains standard first-line treatment, a significant proportion of patients fail to respond or relapse. Thrombopoietin receptor agonists (TPO-RAs) have emerged as promising therapeutic options to enhance hematopoiesis. Among them, romiplostim has shown increasing efficacy across multiple treatment settings. To comprehensively review the clinical efficacy, safety, and optimal use of romiplostim in the treatment of SAA, based on data from clinical trials, real-world studies, and pediatric case series. A systematic literature search was conducted in PubMed, Web of Science, and Scopus through March 2026 using keywords related to "romiplostim" and "severe aplastic anemia." Studies included prospective trials, retrospective cohorts, and mechanistic studies reporting hematologic response, transfusion independence, and adverse events in SAA patients treated with romiplostim. Romiplostim demonstrated consistent efficacy across pediatric and adult populations in 19 studies involving more than 390 patients with severe aplastic anemia. Hematologic response rates ranged from 41% to 95%, with trilineage recovery observed in up to 55% of patients. High-dose regimens (10-20 µg/kg/week) and early initiation were associated with superior outcomes, particularly in eltrombopag-refractory cases. Long-term use was well tolerated, with low rates of clonal evolution or serious toxicity. Romiplostim is an effective and safe therapeutic option in SAA, particularly in patients who are refractory to or intolerant of standard therapies. Its integration into frontline and salvage regimens offers the potential for durable trilineage responses and transfusion independence. Further studies are needed to define its role in combination strategies and optimal dosing and duration of therapy.