The hunt for a cure: modern medical approaches for immune thrombocytopenia

Splenectomy and stem cell transplant have historically provided long-term remissions that seemingly cure a subset of patients with immune thrombocytopenia (ITP). Dozens of modern medical therapies with better safety profiles have revolutionized ITP treatment and several can induce sustained response off treatment (SROT) and delay relapse, providing hope for disease modification and cure.

In this review, we consider how ITP cure might be defined, review key pathophysiologic mechanisms relevant for disease modification, discuss historical approaches, and explore modern therapies that hold promise. Articles were identified based on the authors' expertise. This review on potential ITP cure focuses on pathophysiologic studies that shed light on determinants of immunologic complexity, persistence of autoimmune memory, and treatment resistance or refractoriness and clinical studies that report on long-term remission and relapse rates, prioritizing clinical trials and large retrospective studies, where available.

While SROT and deep immunologic disruption are possible with modern therapies, it remains unknown whether this translates to a meaningful alteration of ITP trajectory. As the field strives for cure, identifying predictive biomarkers, clinical trials incorporating longer follow-up, better understanding of the pathophysiologic underpinnings of autoimmune memory, and balancing patient-focused considerations of risk and toxicity are critical.