A Novel Treatment Strategy for Immune Thrombocytopenia

For patients with steroid-refractory immune thrombocytopenia (ITP), eltrombopag — an oral thrombopoietin agonist (TPO-A) — is a key second-line therapy; however, long-term efficacy can be limited. Because TPO-As stimulate platelet production but do not address immune system dysregulation in ITP, combination therapies targeting both pathways are logical to pursue.

Here, investigators tested whether combining eltrombopag with all-trans retinoic acid (ATRA), which has immune-modulating effects, would improve treatment durability. They randomized 96 adults with glucocorticoid-resistant or relapsed ITP and a platelet count <30 × 10⁹/l to receive eltrombopag plus ATRA or eltrombopag alone.