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We describe key measurement properties of the MySIm-Q instrument using CARTITUDE-4 data.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methodology<\/jats:title>\n                    <jats:p>The phase 3 CARTITUDE-4 (NCT04181827) trial compares ciltacabtagene autoleucel with standard-of-care regimens in patients with lenalidomide-refractory MM after 1\u20133 lines of therapy. Following US Food and Drug Administration guidance on the use of PRO measures in clinical trials, the reliability, as well as aspects of construct validity, convergent and discriminant validity of the MySIm-Q symptom and impact scores (2 separate concepts) were assessed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>In total, 361 patients completed MySIm-Q assessments. Internal consistency results met the predefined threshold (McDonald\u2019s \ua7b7 coefficient\u2009&gt;\u20090.7; \ua7b7=0.87 for total symptom scores, \ua7b7=0.79 for total impact scores), while test-retest reliability was slightly below this threshold (intraclass correlation coefficient [ICC](2,1)\u2009&gt;\u20090.70; ICC\u2009=\u20090.67 and 0.65, respectively). Known-groups validity of total symptom and total impact scores was established through multiple hypotheses. Factor scores estimated from the confirmatory factor analysis model were highly correlated with simple observed scores calculated in symptom and impact scores. Item-level convergent and discriminant validity was supported for all and nearly all items, respectively. Domain convergent and discriminant validity for total scores were largely met.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>These results demonstrate that the MySIm-Q yields reliable and valid scores based on a number of evidentiary sources, supporting its use as a fit-for-purpose PRO instrument in clinical outcome assessments for patients with MM.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registration<\/jats:title>\n                    <jats:p>\n                      CARTITUDE-4: ClinicalTrials.gov ID: NCT04181827. Date of registration: 27 November 2019. URL:\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/www.clinicaltrials.gov\/study\/NCT04181827\" ext-link-type=\"uri\">https:\/\/www.clinicaltrials.gov\/study\/NCT04181827<\/jats:ext-link>\n                      .\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s41687-026-01034-z","type":"journal-article","created":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T13:44:23Z","timestamp":1773409463000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Validation of the Multiple Myeloma Symptom and Impact Questionnaire (MySIm-Q) in patients with multiple myeloma who were enrolled in the CARTITUDE-4 trial"],"prefix":"10.1186","volume":"10","author":[{"given":"Roberto","family":"Mina","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne K.","family":"Mylin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hisayuki","family":"Yokoyama","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hila","family":"Magen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Winfried","family":"Alsdorf","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leyla","family":"Shune","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Iris","family":"Isufi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon J.","family":"Harrison","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Urvi A.","family":"Shah","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9","family":"De Champlain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva G.","family":"Katz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katharine S.","family":"Gries","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jordan M.","family":"Schecter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nikoletta","family":"Lendvai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Slaughter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolina","family":"Lonardi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William","family":"Deraedt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Octavio","family":"Costa Filho","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nitin","family":"Patel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erika","family":"Florendo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kai Fai","family":"Ho","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lionel","family":"Karlin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katja","family":"Weisel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,3,13]]},"reference":[{"key":"1034_CR1","first-page":"417","volume":"22","author":"K Jordan","year":"2014","unstructured":"Jordan K, Proskorovsky I, Lewis P, Ishak J, Payne K, Lordan N, Kyriakou C, Williams CD, Peters S, Davies FE (2014) Effect of general symptom level, specific adverse events, treatment patterns, and patient characteristics on health-related quality of life in patients with multiple myeloma: results of a European, multicenter cohort study. 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An independent ethics committee or institutional review board at each site approved the protocol before study-related procedures commenced.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"All patients provided written informed consent before enrollment into the CARTITUDE-4 study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"R Mina reported a consulting role, advisory role, or honoraria for Amgen, BMS\/Celgene, GSK, Janssen, Menarini-Stemline, Pfizer, Sanofi, and Takeda. H Yokoyama reported honoraria for Astellas. W Alsdorf reported honoraria, research funding, or travel expenses for Affimed, BioNTech, GSK, Immatics, and Janssen. L Shune reported a consulting role or advisory role for MS and J&J. I Isufi reported a consulting role for AbbVie, ADC Therapeutics, Beam Therapeutics, Genmab, Gilead, and Incyte as well as holding equity in Gilead. SJ Harrison reported consulting roles, advisory roles, honoraria, research funding, or speaker\u2019s bureau for AbbVie, Amgen, Celgene\/BMS, Eusa, F. Hoffmann-La Roche Ltd.\/Genentech, Inc., GSK, HaemaLogiX, Janssen, Novartis, and Terumo BCT. UA Shah reported receiving support in part from National Institutes of Health\/National Cancer Institute Cancer Center grant P30CA008748; research funding support from Celgene\/BMS and Janssen to the institution; and personal fees from Janssen Biotech, Sanofi, BMS, and i3Health outside the submitted work. AD Champlain, EG Katz, KS Gries, Dr Lendvai, and A Slaughter have equity and stock options in Janssen and are employed by Janssen. JM Schecter has stock and other ownership interests in Johnson & Johnson and is currently employed by Janssen. C Lonardi and W Deraedt are employed by Janssen. OC Costa Filho and N Patel have equity in Legend Biotech USA Inc. and are employed by Legend Biotech USA Inc. E Florendo is employed by Legend Biotech USA Inc. K Fai Ho reported a consulting role for Janssen. L Karlin reported a consulting role or honoraria for AbbVie, Amgen, Celgene, GSK, Janssen, Sanofi, and Takeda. K Weisel reported a consulting role, honoraria, or research funding for AbbVie, Amgen, Adaptive Biotech, AstraZeneca, BeiGene, BMS\/Celgene, GSK, Janssen, Karyopharm, Menarini, Novartis, Oncopeptides, Pfizer, Roche, Sanofi, Stemline, and Takeda. No other disclosures were reported.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"64"}}