Development of the Multiple Myeloma Symptom and Impact Questionnaire: A New Patient-Reported Outcome Instrument to Assess Symptom and Impacts in Patients With Multiple Myeloma

• Published in: Value in health 2021-12, Vol.24 (12), p.1807-1819
• Main Authors: Gries, Katharine S., Fastenau, John, Seo, Caroline, Potrata, Barbara, Iaconangelo, Charles, Serrano, Daniel
• Format: Article
• Language: English
• Subjects: Aged; Aged, 80 and over; Clinical outcomes; Clinical research; Clinical trials; Cognitive ability; Concepts; content validity; Debriefing; Elicitation; Female; Humans; Interviews; Male; Medical diagnosis; Middle Aged; Multiple myeloma; Multiple Myeloma - physiopathology; Multiple Myeloma - psychology; Myeloma; Patient Reported Outcome Measures; patient-reported outcome instrument; Patients; Qualitative Research; Questionnaires; Saturation; Studies; Surveys and Questionnaires; Validity
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2021.06.010

This study aimed to develop and assess the content validity of a patient-reported outcomes (PROs) instrument to measure symptoms and impacts experienced by patients with active multiple myeloma (MM). The PRO instrument was developed using an iterative, mixed-methods approach. The list of concepts was generated based on a review of existing evidence (qualitative studies and literature) and post hoc psychometric evaluations of 2 PRO instruments in 3 clinical trials. A total of 30 adult patients with MM from the United States participated in hybrid concept elicitation/cognitive debriefing interviews to validate the content validity of the newly developed PRO instrument. Translatability assessment was completed in 8 languages. The item generation process resulted in 17 symptom and 9 impact concepts for evaluation. The concept elicitation interviews and analysis were based on the first 25 participants; evidence of saturation was observed. The cognitive debriefing interviews and analysis were based on the last 23 participants across 4 waves of interviews. On the basis patient feedback, 10 items were removed, and 1 item was added to the PRO instrument. The translatability assessment resulted in 1 minor revision. The multiple myeloma symptom and impact questionnaire (MySIm-Q) includes 11 symptom and 6 impact concepts, organized within 8 hypothesized subdomains, with each concept measured using a 5-point verbal rating scale and a 7-day recall period. The MySIm-Q instrument was developed using rigorous and mixed methodology and with direct input from patients who received a diagnosis of MM. The MySIm-Q has good content validity and is culturally relevant for use in global clinical trials.