Heghine Khachatryan: Phase III Trial Results Support BAFF-R Inhibition in Corticosteroid-Refractory ITP
Heghine Khachatryan, Editor-in-Chief of Hemostasis Today, Head of Hemophilia and Thrombosis Center at Yeolyan Hematology and Oncology Center, shared a post on LinkedIn:
“ISTH 2026 | Day 2
One of the most compelling advances presented at ISTH 2026 was the Phase III VAYHIT2 trial evaluating ianalumab, a first-in-class monoclonal antibody targeting the BAFF/BAFF-R pathway, in combination with eltrombopag for adults with primary immune thrombocytopenia (ITP).
Unlike conventional approaches that primarily stimulate platelet production, ianalumab directly addresses the underlying autoimmune pathogenesis of ITP through a dual mechanism: B-cell depletion via antibody-dependent cellular cytotoxicity (ADCC) and BAFF receptor blockade, thereby suppressing autoreactive B-cell activation and survival.
The study demonstrated that combining ianalumab with eltrombopag resulted in rapid platelet recovery, higher and more durable response rates, and significantly prolonged time to treatment failure compared with eltrombopag alone.
Importantly, therapeutic benefit persisted even after eltrombopag tapering and discontinuation, suggesting a sustained immunomodulatory effect rather than a transient increase in platelet counts.
These findings represent a significant step toward disease-modifying therapy in chronic primary ITP, highlighting the potential to move beyond symptomatic platelet support and toward durable immune reprogramming.
If confirmed in long-term follow-up and integrated into future treatment algorithms, BAFF-R–targeted therapy may redefine the management of patients with corticosteroid-refractory or relapsed ITP.
Scientific advances such as these reinforce the rapid evolution of precision immunotherapy in benign hematology and illustrate how a deeper understanding of disease biology is translating into more durable and clinically meaningful patient outcomes.
Hemostasis Today.”
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