Abstract B066: Bridging patient reported outcomes and CTCAE to improve supportive care and trial design: The PROptimise initiative

Abstract Introduction The Common Terminology Criteria for Adverse Events (CTCAE) dictionary is used to define and grade adverse events (AEs) in phase I/II trials. SSPedi is a validated 15-symptom questionnaire for pediatric oncology patients ages 2 to 18 years old (https://www.sungresearch.com/symptom-assessment-tools). Here, we describe our reconciliation approach, performed between SSPedi items and CTCAE terms, to allow correlation of PRO with reported AEs. We hypothesize that we can improve sensitivity in capturing participant’s symptoms using patient reported outcome (PRO) measures within an early phase pediatric precision oncology basket trial. Methods In the setting of the international “Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer” (OPTIMISE) study (NCT06208657), symptom assessment through SSPedi questionnaires is implemented as an additional patient-centered measure, on a voluntary basis at every scheduled visit. A team of early phase trial investigators, clinical research associates and psychologists reconciled SSPedi items with CTCAE v5.0 terms by conceptual analysis. Results We identified 3 groups of SSPedi items: (a) SSPedi item and CTCAE term are identical (i.e. SSPedi item is “feeling tired” and CTCAE term is “fatigue”); (b) SSPedi term is identical to the CTCAE term, but this item could also be reconciled with several causative diagnoses. For example, SSPedi item “headache” matches with CTCAE term “headache” but also with: “superior vena cava syndrome”, “cytokine release syndrome”, “CSF leakage”, “meningismus”, and “reversible posterior leukoencephalopathy syndrome”; (c) SSPedi item corresponds to several related CTCAE terms of similar relevance. For example, SSPedi item “changes in how your body or face looks” could be reconciled with the following CTCAE terms: “weight gain”, “weight loss”, “cushingoid” and all terms within “skin and subcutaneous tissue disorders”. We generated two reconciliation tables matching each SSPedi item with one or more AE terms: a light version including primary matches, and an extended version including all matches. Those reconciliation tables will be used to describe association between reported SSPedi items and reported AEs, at the cohort or sub-cohort level within the OPTIMISE basket study. Conclusion SSPedi is a patient-centered and child-friendly tool which may improve symptom detection in phase I/II clinical trial participants. With this reconciliation effort, we provide a framework to study the association between SSPedi items and CTCAE terms within the OPTIMISE early phase basket study. Our final objective will be to show how PRO can complement CTCAE in capturing patient symptoms and experience. Citation Format: Gabriel Revon-Rivière, Kate Hetherington, Aiman Siddiqi, Lily Wong, Laura G. Mitchell, Denise Connolly, Daniel Morgenstern, David S Ziegler, Sarah Cohen-Gogo. Bridging patient reported outcomes and CTCAE to improve supportive care and trial design: The PROptimise initiative [abstract]. In: Proceedings of the AACR Special Conference in Cancer Research: Advances in Pediatric Cancer Research; 2024 Sep 5-8; Toronto, Ontario, Canada. Philadelphia (PA): AACR; Cancer Res 2024;84(17 Suppl):Abstract nr B066.