The Value of Patient-Reported Outcomes in Choosing ITP Therapies

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.