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Reduced dose prednisone plus eltrombopag for the treatment of newly diagnosed adult primary immune thrombocytopenia: A prospective, multicentre, phase 2 clinical trial

Year: 2025

Type: article

Abstract: Reduced dose prednisone plus eltrombopag for the treatment of newly diagnosed adult primary immune thrombocytopenia: A prospective, multicentre, phase 2 clinical trialTo the Editor, Primary immune thrombocytopenia (ITP) is an autoimmune disorder involving low platelet (PLT) counts and impaired bone marrow megakaryopoiesis.First-line therapy for newly diagnosed (ND) ITP with PLT ≤30 × 10 9 /L employs corticosteroids (CIS), intravenous immunoglobulin or anti-D immunoglobulin. 1 While high-dose CIS induces rapid responses, prolonged use causes significant toxicity, prompting guideline-recommended tapering to ≤5 mg/ day prednisone-equivalent within 6-8 weeks.2,3 However, frequent relapses post-CIS withdrawal often necessitate second-line therapies like thrombopoietin receptor agonists (TPO-RAs).Eltrombopag (EPAG), the first oral non-peptide TPO-RAs, achieved ~80% response in chronic ITP patients.Chinese guidelines recommend 25 mg initial dosing, 4 with dose-dependent efficacy: 22.22% (25 mg), 45.45% (50 mg) and 65.52% (75 mg) response rates.5 Nevertheless, although the approval of multiple generic versions has substantially reduced treatment costs, dose escalation and maintenance therapy may still impose financial burdens, especially in developing countries, highlighting the need for cost-effective protocols that ensure rapid CIS withdrawal, sustained responses, reduction of toxicity and reduced relapse.Thus, we conducted a multicentre, prospective phase 2 clinical trial to evaluate the efficacy of reduced-dose prednisone (RD-Pred) plus low-dose eltrombopag (LD-EPAG) in ND, treatment-naïve ITP patients.The study was registered at chictr. org. cn (#ChiCTR2200058259).Eligible patients were ND ITP, ≥18 years of age, without previous therapy, baseline PLT <30 × 10 9 /L or PLT <50 × 10 9 /L with significant bleeding symptoms.Participants were excluded if they had positive serology for HIV, hepatitis B or C, inherited or congenital thrombocytopenia or secondary thrombocytopenia.This study was approved by the ethical committee of the First Affiliated Hospital of Zhejiang Chinese Medical University (2022-KL-014-01), which was performed in accordance with the Declaration of Helsinki.The study included three phases: (1) core treatment period (8 weeks), in which patients received a fixed dose of RD-Pred (weight ≤ 50 kg, 20 mg/day; weight > 50 kg, 0.4 mg/kg/day) for 3 weeks, followed by a gradual taper to the target dose within 8 weeks.LD-EPAG

Source: British Journal of Haematology

DOI: 10.1111/bjh.70168