Psychometric properties of the FACT-M questionnaire in patients with Merkel cell carcinoma

Abstract

BackgroundNo validated disease-specific questionnaires exist to capture health-related quality of life (HRQoL) in patients with Merkel cell carcinoma (MCC). The Functional Assessment of Cancer Therapy – Melanoma (FACT-M) is validated in patients with melanoma, which shares many similarities with MCC. This paper reports the psychometric properties of the FACT-M in the metastatic MCC population.MethodsData were collected as part of a single-arm, open-label, multicenter trial involving patients with metastatic MCC who had failed at least one previous line of chemotherapy. FACT-M and EQ-5D were administered at baseline, Week 7, Week 13, and Week 25. An optional interview was administered at the same time points. MCC-specific FACT-M scores were derived following a combined quantitative and qualitative approach. Reliability and construct validity of original and additional MCC-specific FACT-M scores were assessed at baseline. Capacity to detect change in tumor size was assessed from baseline to Week 7. Minimally important differences (MIDs) were computed using distribution and anchor-based methods.ResultsBaseline assessments were available in 70 patients (mean age: 70 years; 74.3% male); 19 patients were interviewed at baseline. Additional MCC-specific scores were as follows: Physical Function score (six items), Psychological Impact score (six items), and MCC summary score (12 items). FACT-M original and additional MCC-specific scores both demonstrated acceptable psychometric properties: high reliability (Cronbach’s alpha: 0.81–0.96), good convergent validity (correlations above 0.4 observed for 88% of items of the Melanoma surgery scale, 75% of items of the Melanoma scale, and 100% of items of the other FACT-M domains). Some evidence of floor/ceiling effects and poor discriminant ability was found. Higher scores (better HRQoL) on all FACT-M domains were observed in patients with better functioning (assessed by ECOG performance score), supporting clinical validity. Despite the small sample for responsiveness analysis (n = 37), the majority of FACT-M scores showed sensitivity to changes in tumor size at Week 7 with small to moderate effect sizes. MIDs were consistent with previously reported values in the literature for FACT-M domains.ConclusionsFACT-M is suitable to capture HRQoL in patients with metastatic MCC, thus making it a potential candidate for assessing HRQoL in MCC trials.Trial registrationThis study is a post-hoc analysis conducted on data collected in Part A of the JAVELIN Merkel 200 trial. This trial was registered on 2 June 2014 with ClinicalTrials.gov as NCT02155647.

Nonprogression with Avelumab Treatment Associated with Gains in Quality of Life in Metastatic Merkel Cell Carcinoma

“…MCC and melanoma are both aggressive skin malignancies; despite some differences between MCC and melanoma, including worse prognosis for MCC, the content of the FACT-M seems appropriate to assess QoL in patients with MCC. This was further confirmed by a thorough psychometric validation of the FACT-M in the JAVELIN Merkel 200 trial population [29]. Avelumab has been shown to achieve meaningful clinical benefit in mMCC with an ORR of 33.0% (95% CI: 23.3-43.8), and Kaplan-Meier estimated proportion of responses with at least 6 months' duration was 93% (95% CI: 74-98).…”

“…The number of patients who completed the EQ-5D (FACT-M) questionnaire was 54 (53) at week 7, 43 (42) at week 13 and 32 (31) This is considered a clinically meaningful improvement on this QoL score, assuming that the minimally important difference (MID) of the FACT-M obtained from melanoma patients is also valid for the MCC population [29,36]. Correlation coefficients calculated between tumor size change and change in EQ-5D VAS score were small (ρ = -0.188), indicating no clear association.…”

Patient-reported outcome instruments used in immune-checkpoint inhibitor clinical trials in oncology: a systematic review

“…From (Table S5). Only one publication [40] re-validated the instrument in the study population and the psychometric results have been published elsewhere [42]. Overall, most of the publications reported differences between groups in terms of "clinically meaningful deterioration", "clinically meaningful improvement", "clinically relevant", "stable", "minimal change", "statistically significant", or "significant difference" (Table S5).…”

Patient Experiences with Avelumab in Treatment-Naïve Metastatic Merkel Cell Carcinoma: Longitudinal Qualitative Interview Findings from JAVELIN Merkel 200, a Registrational Clinical Trial

“…At the time of the start of this trial, there was no quantitative instruments specific to MCC, hence a melanoma-specific questionnaire, FACT-M, was administered to patients. Data from the collected qualitative interviews alongside the trial were used to support its psychometric validation [34]. A mixed-method research approach using the qualitative interview data together with the questionnaire quantitative data and clinical data to provide a wholistic perspective on patient experience has been previously published using part A data [17].…”