Thrombopoietin receptor agonists in chronic primary immune thrombocytopenia: Efficacy of eltrombopag and its role as an alternative to splenectomy
DOI:
https://doi.org/10.5281/zenodo.21796810Keywords:
immune thrombocytopenia, eltrombopag, thrombopoietin receptor agonists, splenectomy, sustained platelet response, chronic ITPAbstract
Background: Primary immune thrombocytopenia (ITP) is an autoimmune disorder that becomes chronic in 60%–70% of adults. First-line treatments such as corticosteroids are associated with high relapse rates and serious adverse effects with prolonged use. Thrombopoietin receptor agonists (TPO-RAs), such as oral eltrombopag, emerged as a therapeutic innovation with a dual thrombopoietic and immunomodulatory mechanism. The aim of this review was to evaluate the efficacy of eltrombopag in achieving a sustained platelet response and its role as an alternative to splenectomy. Methods: An integrative review was conducted following the PRISMA 2020 protocol. A systematic search was performed in four electronic databases, covering evidence from January 2020 to March 2026. Eligibility criteria (PICO framework) focused on adults with chronic/persistent ITP (duration ≥ 3 months, platelets < 30 × 10⁹/L), comparing TPO-RAs with placebo, standard treatment or splenectomy. Outcomes included platelet response (≥ 50 × 10⁹/L), treatment-free sustained remission (TFSR) and splenectomy rate. Results: Fifty-eight studies with more than 18,500 participants were included. Eltrombopag demonstrated statistically significant superiority over placebo for platelet response (RR = 3.42; P < 0.0001), with overall response rates of up to 91% in long-term real-world studies (> 12 months). Induction of TFSR was observed in 25%–53% of patients after gradual discontinuation. Adoption of TPO-RAs was associated with a reduction in the frequency of splenectomies. The safety profile showed hepatic dysfunction (8.2%–22.4%) and thrombotic events (2.2%–3.2%), without a significant increase relative to placebo. Conclusion: Eltrombopag consistently achieves a sustained platelet response superior to placebo and can induce TFSR. Its dual mechanism (thrombopoietic and immunomodulatory) supports its position as a preferred medical alternative to splenectomy in chronic ITP refractory to first-line therapy. Future research priorities include identification of TFSR biomarkers and economic analyses in low-resource health systems.
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Copyright (c) 2026 Anna Clara Peres Luz, Meury Nakano Araújo Marques, Francine Aquino de Carvalho Almeida, Eliaquim Frison Hartmann, Leonardo de Castro Souza Filho, Breno Alexandre Rocha Oliveira, Brenda Heloise Padovani Alves, Paola Britos (Autor/a)
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