Ianalumab plus Eltrombopag in Immune Thrombocytopenia

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Source: NEJM

Original: https://www.nejm.org/doi/full/10.1056/NEJMoa2515168?af=R&rss=currentIssue...

Published: 2025-12-09T12:30:00Z

The phase III study VAYHIT2 evaluated the efficacy of the combination of ianalumab and eltrombopag in adult patients with primary immune thrombocytopenia (ITP) unresponsive to corticosteroid therapy. Ianalumab is a monoclonal antibody that targets the BAFF receptor and works in two ways: it induces B cell depletion and blocks their function and survival. Patients received ianalumab in two doses (3 mg/kg or 9 mg/kg) or placebo, always in combination with the platelet-stimulating agent eltrombopag. The primary objective was to increase the time to treatment failure (TTF), i.e. to a decrease in the safe level of platelets during and after treatment. The results showed that the addition of ianalumab to eltrombopag significantly prolonged TTF compared with placebo plus eltrombopag. Patients treated with ianalumab also achieved higher rates of sustained improvement in platelet counts. The safety profile of the combination was favorable and matched expectations from previous studies. This combined treatment represents a promising option for patients with ITP resistant to standard steroid therapy[1][2][4].