Romiplostim 250microgram powder for solution for injection vials

Background Pediatric primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia and an increased risk of bleeding. Conventional therapies, while effective in some cases, are often limited by suboptimal response rates and significant adverse effects with prolonged use. Thrombopoietin receptor agonists (TPO-RAs), including recombinant human thrombopoietin (rhTPO), romiplostim, and eltrombopag, have emerged as promising therapeutic alternatives for pediatric ITP. However, a comprehensive comparison of their efficacy and safety profiles remains lacking. Objective To conduct a systematic review and network meta-analysis to evaluate and compare the efficacy and safety of rhTPO, romiplostim, and eltrombopag in the treatment of pediatric ITP. Methods A systematic literature search was performed across PubMed, Embase, Cochrane Library, and other relevant databases. Seven randomized controlled trials (RCTs) involving a total of 375 pediatric ITP patients were included. Direct meta-analysis and Bayesian network meta-analysis were employed to assess overall response rates (ORR) and the incidence of serious adverse events (SAEs). The Surface Under the Cumulative Ranking Curve (SUCRA) was utilized to rank the interventions based on their efficacy and safety. Results Direct meta-analysis demonstrated that romiplostim (OR = 17.57, 95% CI: 4.90–63.03), eltrombopag (OR = 5.34, 95% CI: 2.50–11.39), and rhTPO (OR = 5.32, 95% CI: 2.03–13.96) were all significantly more effective than placebo in achieving ORR (P < 0.001). In terms of SAEs, romiplostim was associated with a higher risk (OR = 3.79, 95% CI: 0.66–21.85), whereas eltrombopag (OR = 0.68, 95% CI: 0.23–2.03) and rhTPO (OR = 0.28, 95% CI: 0.01–7.17) exhibited more favorable safety profiles. Network meta-analysis ranked romiplostim (SUCRA = 0.96) as the most efficacious intervention, followed by eltrombopag (0.52) and rhTPO (0.52). For safety, rhTPO (SUCRA = 0.78) ranked highest, followed by eltrombopag (0.66), while romiplostim (0.12) was associated with the highest risk. Conclusion Romiplostim exhibits superior efficacy in the management of pediatric ITP but necessitates vigilant monitoring for potential adverse effects, including bone marrow fibrosis. rhTPO, with its favorable safety profile, is particularly well-suited for acute bleeding scenarios. Eltrombopag offers a balanced combination of oral convenience and safety, making it an optimal choice for long-term therapy. Clinical decision-making should be guided by individual patient factors, including bleeding risk, treatment adherence, and drug accessibility. Future research should prioritize head-to-head comparative trials and long-term follow-up studies to further refine therapeutic strategies and optimize outcomes in pediatric ITP.

Abstract licence: CC BY

A World Health Organisation panel previously recommended the use of hematopoietic cytokines to manage H-ARS within 24 h of exposure to ⩾2 Gy radiation dose, a recommendation that has been endorsed by hematologists and oncologists with expertise in radiation management. Nevertheless, no state-of-the-art consensus has been reached regarding categorical selection of cytokines for emergency scenarios involving accidental exposures where implementation of planned and/or extended countermeasures is certain or likely (International Nuclear and Radiological Event Scale levels 5, 6 and 7) or an exposure from a detonated nuclear weapon. A systematic review of the published literature was conducted (422 citations identified, 391 of which were screened) in non-human primates 9 meeting inclusion criteria), and in reviews of human cases treated with cytokines in a search of the MEDLINE database (1970-present), websites/official publications of major national and international organisations and radiation societies, cytokine reviews and full prescribing information. In contrast to filgrastim, pegfilgrastim and romiplostim, sargramostim augments the differentiation and proliferation of multiple lymphohematopoietic lineages. NHP survival benefits without the support of blood products was reported with sargramostim or pegfilgrastim plus romiplostim. Four cytokine reviews met criteria for summarising published reports of 63 human cases that included at least one case meeting inclusion criteria. Cytokine efficacy was documented when administered at up to 96 h after NHP exposure for sargramostim and at 24 h but not 48 h after exposure for filgrastim, pegfilgrstim or pegfilgrastim plus romiplostim. Ease of use favoured pegfilgrastim (administered weekly x2) and romiplostim (administered once), compared to filgrastim and sargramostim (administered daily x5 and x14, respectively). Formal assessment of the published evidence is urgently needed to provide categorical guidance regarding cytokine use for patient management, and to public health officials involved in establishing a national or shared regional radiation stockpile for immediate use in a mass casualty radiological/nuclear (R/N) emergency.

Abstract licence: CC BY

This was a randomized multicenter single‐blinded active‐controlled equivalence Phase III study evaluating the efficacy and safety of the biosimilar of romiplostim (GP40141) compared to the reference drug Nplate in patients with persistent or chronic immune thrombocytopenia (ITP).

Abstract licence: CC BY-NC