Ianalumab plus Eltrombopag Extends Time to Treatment Failure in Previously Treated Immune Thrombocytopenia: Phase 3 VAYHIT2 Trial
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A phase 3 clinical trial found that ianalumab, a novel B-cell targeting agent, significantly extended the time patients with immune thrombocytopenia stayed free from bleeding complications or treatment failure when added to standard therapy. In the VAYHIT2 trial, patients who received four monthly infusions of ianalumab plus the standard oral platelet-boosting drug eltrombopag went more than 13 months without needing rescue treatment, compared to fewer than 5 months for those on eltrombopag plus placebo. These findings, published in the New England Journal of Medicine, suggest ianalumab may offer a new approach to changing the course of ITP rather than just managing symptoms.
Abstract
Immune thrombocytopenia (ITP) is an autoimmune disorder in which the immune system destroys platelets, causing platelet counts to fall below 50,000 per microliter in most affected patients and increasing the risk of serious bleeding. While first-line treatment with glucocorticoids and antibody infusions can stabilize platelet counts, high relapse rates and long-term steroid side effects represent significant unmet needs. Ianalumab is a novel anti-BAFF receptor (BAFFR) antibody that eliminates pathogenic B cells, the immune cells responsible for producing the platelet-destroying antibodies central to ITP pathology.
VAYHIT2 was a phase 3, randomized, double-blind, placebo-controlled trial that enrolled 152 adults with primary ITP who had failed first-line corticosteroid treatment across 24 countries. Patients received the thrombopoietin receptor agonist eltrombopag for 16 to 24 weeks, paired with four once-monthly infusions of ianalumab at 9 mg/kg, ianalumab at 3 mg/kg, or placebo. The primary endpoint was time to treatment failure, defined as the time to a bleeding event requiring rescue therapy or initiation of a new ITP treatment after completing study therapy.
The median time to treatment failure was 13.0 months in the high-dose ianalumab group, compared to 4.7 months in the placebo group. The lower-dose ianalumab group had too few events to calculate a median, indicating a durably high proportion of event-free patients. Stable platelet responses at six months were achieved in 62.0% of high-dose patients and 56.9% of low-dose patients, compared to 39.2% in the placebo group. Transient neutropenia occurred more often with ianalumab but did not translate into higher rates or severity of infections. These results support ianalumab as a disease-modifying therapy capable of extending durable disease control in previously treated ITP.