Understanding Gaps and Limitations in Current ITP Therapies

Individuals living with immune thrombocytopenia (ITP) face significant challenges with existing treatment options that extend beyond bleeding prevention. Current standard therapies, including corticosteroids, present considerable tolerability issues for patients, such as elevated blood sugar, high blood pressure, sleep disruption, and cognitive impairment. Rituximab requires intravenous administration and can leave patients vulnerable to infections through prolonged immunosuppression, while fostamatinib causes hypertension, diarrhea, and potential bleeding complications.

Beyond these safety concerns, current ITP treatments fail to provide sustained platelet responses for many patients. Intravenous immunoglobulin and anti-D therapies offer only temporary improvements without long-term durability. This lack of sustained efficacy creates ongoing management challenges for individuals managing this chronic autoimmune condition.

Quality of life represents a critical unmet need that existing therapies inadequately address. Patients with ITP experience fatigue, reduced daily functioning, and gender-specific health impacts that persist even when platelet counts improve. For individuals with diabetes, corticosteroid-induced hyperglycemia poses particular risks, while those at risk for blood clots face complicated decisions regarding thrombopoietin receptor agonists. Premenopausal women specifically struggle with quality-of-life impacts that current treatments fail to address, highlighting the urgent need for therapies that target both disease mechanisms and patient wellbeing comprehensively.