Patient-Reported Outcome Measures and Validation Data Used in Melanoma Research and Routine Practice: A Systematic Review.

Importance: Patient-reported outcome measures (PROMs) are increasingly used in melanoma research and to guide clinical practice; however, the validation of these PROMs for use in melanoma populations is unknown.

Objective: To conduct a systematic review and construct an evidence gap map to identify PROMs that have been used in melanoma research and clinical practice and related melanoma-specific validation data.

Evidence review: MEDLINE, Embase, Web of Science Index Medicus, CINAHL, the Cochrane Central Register of Controlled Trials, and PsychINFO were searched in January 2025 including any studies that used a PROM to evaluate outcomes of patients with melanoma published from January 1, 2010, onward to identify instruments relevant to the current era of melanoma diagnosis and treatment.

Findings: Of 30 895 abstracts screened, 136 articles detailing 124 studies were included in this review; a total of 32 784 participants were included. There were 52 cross-sectional studies (41%), 31 randomized clinical trials (25%), 23 longitudinal studies (19%), 8 pre-post studies (6%), 6 cohort studies (5%), 1 retrospective analysis (1%), 1 phase 4 trial (1%), 1 protocol (1%), and 1 quasi-experimental trial (1%). A total of 61 studies (49%) included a melanoma treatment, with immunotherapy being the most common (24 [39%]), followed by surgery (7 [11%]), and chemotherapy (7 [11%]). These 124 studies used 110 unique PROMs, with patient emotional/psychological well-being (28 [25%]), health-related quality of life (21 [19%]), and patient self-functioning, efficacy, and coping strategies (20 [18%]) being the most common outcome categories. Only 17 PROMs (15%) had melanoma-specific validation data available, of which only the Functional Assessment of Cancer Therapy-Melanoma questionnaire had data available for all psychometric variables of interest.

Conclusions and relevance: In this systematic review, PROMs used in melanoma research and clinical practice were heterogenous, with nearly as many unique instruments identified as studies that used them. Furthermore, few instruments had melanoma-specific validation data available. Future research should aim to address the gaps in melanoma-specific validation data of commonly used PROMs through psychometric evaluation studies to increase researchers' and clinicians' confidence in the performance and accuracy of these measures in melanoma populations.