IMPORTANCE OF PATIENT EDUCATION IN THE UTILIZATION OF LEE SYMPTOM SCALES IN PATIENTS WITH CGVHD FOR ANALYSIS OF SYMPTOM CONTROL.

Chronic graft versus host disease (cGVHD) remains the leading cause of non-relapse mortality in patients post-allogeneic transplant. Novel agents are becoming available for patients with cGVHD, increasing the need for a personalized approach and evaluation of symptom control to guide treatment decisions. The National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in cGVHD has proposed use of the Lee Symptom Scale (LSS) in clinical trials to measure efficacy. Patient understanding of this questionnaire is essential to accurately capture data, minimize the impact of survey fatigue, and evaluate evidence of symptom improvement. Here we provide methods utilized to educate patients enrolled in SNDX-6352-0503 evaluating the use of axatilimab in patients with cGVHD. SNDX-6352-0503 is a Phase 1/2 study assessing the safety/efficacy of axatilimab in patients with active cGVHD despite ≥2 prior lines of systemic therapy. Key secondary objectives included improvement in LSS from baseline. LSS was assessed day one of each cycle and evaluates symptoms via 30-questions clustered by organ systems routinely affected by cGVHD. Total scores range from 0-120 and a 7-point improvement indicates clinical benefit. Study coordinators worked closely with participants to collect LSS data, used verbal instructions to educate on LSS value and addressed the sense of survey burden. Paper forms were provided with instructions to consider changes in their symptoms in the past 30-days. Ample time and a quiet space were provided with coordinators readily available to provide necessary clarifications. Areas typically requiring clarification included questions related to unintended weight loss and scoring of skin changes. Forms were then collected and scanned for completeness. The FDA and other regulatory bodies require an assessment of patient-reported symptoms when evaluating a new agent. Patients must receive appropriate education in the importance of the LSS questionnaire and completion to enable effective and reliable evaluation of novel agents in cGVHD. Applying these methods decreased questionnaire fatigue and led to the collection of a robust data set illustrating a 7-point improvement in 50% (n=19/38) of pts and contributing to axatilimab's continued development. AGAVE-201 and other studies in cGVHD will continue to utilize the original or modified LSS to assess symptom control, highlighting the importance of patient education in the utilization of these tools. [ABSTRACT FROM AUTHOR]