Combined Ianalumab and Eltrombopag Therapy in Immune Thrombocytopenia: Insights from the VAYHIT2 Trial

VAYHIT2: Ianalumab plus eltrombopag extends response durability in previously treated primary ITP, delaying treatment failure and increasing six‑month sustained platelet responses.

The randomized Phase III trial showed the combination produced a longer median time to treatment failure and higher rates of sustained platelet response at six months versus placebo plus eltrombopag, expanding second‑line options to pair B‑cell suppression with thrombopoietic support.

Until now, relapsed or refractory ITP was typically managed with TPO‑receptor agonists, splenectomy, or repeat immunosuppression. By combining a B‑cell–targeting antibody with eltrombopag, the strategy aims to reduce autoantibody generation while sustaining platelet production and to decrease chronic dependence on daily TPO‑RA therapy after first‑line steroid failure.

VAYHIT2 enrolled roughly 150 adults with previously treated primary ITP and used time to treatment failure as its primary endpoint; key secondaries included six‑month durable platelet response and patient‑reported fatigue. These endpoints address bleeding risk, need for rescue therapy, and quality‑of‑life impacts that matter in routine ITP care.

The VAYHIT2 trial noted an approximate 2.8‑fold longer median time to treatment failure and higher six‑month sustained response rates (about 62% vs 39%) with ianalumab plus eltrombopag versus placebo plus eltrombopag.

The study also noted transient neutropenia occurred more often with ianalumab without a corresponding increase in overall infection rates, and recorded lower fatigue scores at 24 weeks.

Practically, extended response durability will allow clinicians to space platelet monitoring during stable remission while maintaining close early surveillance for treatment‑emergent cytopenias. Reasonable monitoring is baseline full blood count, weekly neutrophil checks for the first month after initiating ianalumab, then monthly peripheral counts during maintenance and whenever tapering eltrombopag.