Strategies Shift Beyond Traditional Management of Immune Thrombocytopenia

Immune thrombocytopenia (ITP) treatment has evolved rapidly as researchers have gained a deeper understanding of the disease's underlying pathophysiology. In this video, Adam Cuker, MD, chief of the Section of Hematology at the University of Pennsylvania, and Amit Mehta, MD, a hematologist/oncologist practicing in Raleigh, North Carolina, discuss how that evolving understanding has reshaped care for patients with ITP.

Both physicians trace the field's progression from rituximab (Rituxan) and thrombopoietin receptor agonists to newer targeted options, including the SYK inhibitor fostamatinib (Tavalisse) and the BTK inhibitor rilzabrutinib (Wayrilz), with additional mechanisms—anti-CD38 agents, complement inhibitors, BAFF receptor inhibitors, neonatal FC receptor antagonists, and even chimeric antigen receptor T-cell therapy—in development. They describe how this expanding toolkit allows treatment to be individualized around a patient's thromboembolism risk, quality-of-life impact, and preferred route of administration.

On sequencing therapies for patients with chronic or difficult-to-treat ITP, Cuker acknowledges that treatment selection today still relies largely on trial and error, whereas Mehta frames the lack of a single standard sequence as an opportunity to tailor care to each patient's individual history and goals.

Both physicians also point to a shift in how treatment response is measured. Citing the 2021 I-WISH study, Mehta notes that hematologists have historically underestimated the fatigue patients experience with chronic ITP by roughly 50%, and describes incorporating more direct conversations about energy level, work impact, and psychological health into his practice. Cuker discusses treatment goals extending beyond platelet count to include tolerability, convenience, and the emerging concept of sustained response off treatment.

Looking ahead, the physicians identify several priorities for the field: developing biomarkers to guide treatment selection, studying whether therapies can safely be tapered or combined, improving management of ITP during pregnancy, and building better laboratory tools to track disease activity. Both close with a shared message for community hematologists that patients with chronic ITP now have more effective options than ever, and physicians should not settle for suboptimal symptom control when better-tailored treatment may be available.