The Value of Patient-Reported Outcomes in Choosing ITP Therapies

EP: 1.Understanding Immune Thrombocytopenia: Pathophysiology, Classification, and Differentiation

EP: 2.Diagnostic and Patient Education Strategies in ITP

EP: 3.Managing Bleeding Risk in Patients with ITP

EP: 4.Living with ITP: Impact of Fatigue and Other Symptoms on Patients’ Quality of Life

EP: 5.Tailoring Care in ITP Through a Health Equity Lens

EP: 6.Looking Ahead in ITP: Addressing Unmet Needs and Embracing Innovation

EP: 7.Key Clinical Signs and Symptoms Used in Diagnosis of Immune Thrombocytopenia

EP: 8.Clinical Decision-Making in ITP: Timing Treatment Initiation and Therapy Goals

EP: 9.Current Treatment Algorithm for Management of Immune Thrombocytopenia

EP: 10.Addressing the Limitations of Current ITP Therapies

EP: 11.Risks versus Benefits: ITP Treatment Side Effects and the Influence on Patients’ Quality of Life

EP: 12.Defining Treatment Success, Failure, and When to Reassess Patients’ Management of ITP

EP: 13.Navigating Refractory ITP: Clinical Insights and Treatment Strategies

EP: 14.Potential Impact of Emerging Therapies on Patients Living with ITP

EP: 15.Emerging Agents Reshaping the ITP Landscape

EP: 16.Understanding Gaps and Limitations in Current ITP Therapies

EP: 17.LUNA 3 Trial Evaluation: Understanding BTK Inhibition in ITP Management

EP: 18.Defining the Role of Rilzabrutinib in ITP Management

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EP: 19.The Value of Patient-Reported Outcomes in Choosing ITP Therapies

EP: 20.Practical Considerations for ITP Therapy Selection and Future Research

Historical ITP treatment experience reveals a troubling disconnect: Rising platelet counts with conventional therapies frequently fail to improve how patients feel and function daily. This gap between laboratory improvement and lived experience has left many individuals with ITP managing adequate platelet numbers while continuing to struggle with fatigue, reduced activity tolerance, and impaired quality of life. LUNA 3’s inclusion of comprehensive patient-reported outcome measures as prospectively defined end points represents a paradigm shift toward evaluating treatments through the patient’s perspective.

Rilzabrutinib demonstrated significant improvements across all quality-of-life domains assessed in LUNA 3, with particular benefits in fatigue—the symptom most commonly reported as debilitating by individuals living with ITP. These improvements appeared as early as week 5, providing relatively rapid relief for patients accustomed to chronic exhaustion. The correlation between quality-of-life enhancement and treatment suggests that rilzabrutinib’s mechanism—reducing the pro-inflammatory state driving ITP pathophysiology—addresses root causes of symptom burden beyond platelet destruction alone.

The broad tolerability and oral administration of rilzabrutinib remove practical barriers that compromise treatment adherence for many patients. Unlike intravenous infusions requiring clinic visits or injectable medications necessitating weekly appointments, individuals can manage rilzabrutinib therapy independently through pharmacy pickup and twice-daily oral dosing. This convenience factor proved clinically relevant in real-world scenarios where patients discontinued effective injectable therapies due to visit burden. For individuals balancing ITP management with work, family responsibilities, and other life demands, the autonomy of oral therapy combined with meaningful symptom improvement and favorable safety represents a comprehensive advancement in patient-centered ITP care.