Meaningful efficacy measures and trial endpoints in patients with advanced melanoma being considered for adjuvant systemic therapy: a prospective patient survey study

In this prospective, survey-based study conducted from May 2023 to February 2025 at the Juravinski Cancer Center, patient preferences and perceptions of clinically meaningful outcomes in the adjuvant treatment of resected stage IIB-IV melanoma were evaluated. Sixty-four eligible patients with resected stage IIB-IV melanoma who were considering adjuvant systemic therapy completed an online survey assessing their understanding and prioritization of treatment outcomes, thresholds for meaningful clinical benefit, and willingness to accept toxicity risks. Overall survival (OS) and quality of life (QoL) emerged as the most important endpoints, prioritized by 37 and 31% of respondents respectively, with patients choosing surveillance tending to prioritize QoL while those on active treatment favoured OS. Willingness to accept disease-free/progression-free benefit in the absence of an OS benefit declined as the risk of potential side effects increased, even among patients who ultimately chose active treatment. Patients considered a reduction in recurrence risk from 27 to 10% over 2 years (stage IIB-IIC) and from 50 to 20% over 5 years (stage III-IV) to be clinically meaningful, accepting a median 10-12.5% risk of significant side effects, and desired a minimum cancer-free interval of 4.75 years. These findings suggest that while OS remained a primary concern, many melanoma patients placed high value on QoL and were cautious about initiating adjuvant therapies without demonstrable survival benefit. Reluctance to accept hypothetical treatment with potential toxicity contrasted with those who proceeded with treatment. This highlighted the importance of incorporating patient preferences and risk tolerance into shared decision-making and trial design.