Assessing the effectiveness of the TURALIO (pexidartinib) risk evaluation and mitigation strategy program: patient knowledge, attitudes, and behavior survey (2020–2024)

Background Treatment of tenosynovial giant cell tumor with TURALIO (pexidartinib) is associated with serious hepatotoxicity risk. To ensure its safe use, a Risk Evaluation and Mitigation Strategy (REMS) program was implemented, incorporating structured education, mandatory enrollment, and liver function monitoring. The assessment of patient and caregiver knowledge, attitudes, and behaviors (KAB) is a central component of evaluating REMS effectiveness.Methods Four waves of TURALIO Patient KAB Survey were conducted between 2020 and 2024 among REMS-enrollees. Surveys assessed understanding of two Key Risk Messages (KRMs): (KRM 1) hepatotoxicity risk and symptom recognition, and (KRM 2) liver monitoring requirements before and during treatment. Response rates and demonstrated understanding of KRMs were summarized descriptively.Results Across four waves of this voluntary survey, 239 participants completed KAB surveys. Survey response rates ranged from approximately one-third to one-half of invited participants across waves. Correct response rates for individual knowledge items were consistently high. Demonstrated understanding of hepatotoxicity risk remained at ≥80% (the predefined threshold) across waves (87.5% in Wave 1; 80.0% in Wave 4). Understanding liver monitoring requirements improved over time, reaching 100% in Wave 3. Combined demonstrated understanding of KRM1 and KRM2 was <80% in three survey waves, particularly in Wave 2 (73.4%). Demonstrated understanding of KRMs remained generally stable despite evolving patient demographics and treatment duration over time.Conclusion Across KAB Survey waves, patients receiving TURALIO demonstrated sustained knowledge of hepatotoxicity risk and monitoring requirements supporting the effectiveness of the tREMS program in ensuring the safe real-world use of pexidartinib.