GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©): Psychometric properties and clinically meaningful thresholds as assessed in the phase 3 DeFi randomized controlled clinical trial Journal Article

   

Authors:	Gounder, M. M.; Atkinson, T. M.; Bell, T.; Daskalopoulou, C.; Griffiths, P.; Martindale, M.; Smith, L. M.; Lim, A.
Article Title:	GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©): Psychometric properties and clinically meaningful thresholds as assessed in the phase 3 DeFi randomized controlled clinical trial
Abstract:	Purpose: The GODDESS© tool was developed to assess Desmoid Tumor/Aggressive Fibromatosis (DT/AF) symptom severity and impact on patients’ lives. This study evaluated GODDESS©’s cross-sectional and longitudinal measurement properties. Methods: The Phase 3, randomized placebo-controlled, DeFi study (NCT03785964) of nirogacestat in DT/AF was used to assess GODDESS©’s reliability, construct validity, responsiveness, and estimate of meaningful change thresholds (MCTs). Other patient-reported outcome (PRO) measures included Patient Global Impression of Severity (PGIS) in DT/AF symptoms, EORTC QLQ-C30, Brief Pain Inventory Short Form, and PROMIS Physical Function short-form 10a v2.0 plus 3 items. Results: DeFi participants (N = 142) had a median age of 34 years (range: 18–76) and were mostly female (64.8%), with extra-abdominal (76.8%) or intra-abdominal tumors (23.2%). The GODDESS© symptom/impact scales showed internal consistency at baseline, cycles 4 and 7 (Cronbach’s α > 0.70) and test–retest reliability (intra-class correlation coefficient > 0.85). GODDESS© scales correlated moderately to highly with PRO measures capturing similar content and differentiated among PGIS and Eastern Cooperative Oncology Group groups. GODDESS© scales detected improvement over time. For the total symptom score, a 1.30-point decrease was estimated as the within-person MCT and a 1.00-point decrease as the between-group MCT. For the physical functioning impact score, estimated within- and between-group MCTs were 0.60-point and 0.50-point decreases, respectively. Few participants exhibited symptom worsening. Conclusion: GODDESS© was found to be reliable, valid, responsive, and interpretable as a clinical trial endpoint in the pooled sample of DT/AF patients. Estimated MCTs can be used to define responders and assess group-level differences in future, unblinded, efficacy analyses. Trial registration number and registration date: NCT03785964; December 24, 2018. © 2023, The Author(s).
Keywords:	adolescent; adult; controlled study; human tissue; aged; middle aged; young adult; major clinical study; fatigue; placebo; reproducibility; reproducibility of results; quality of life; pain; nausea; randomized controlled trial; psychology; cancer research; abdominal pain; questionnaire; muscle weakness; cross-sectional study; cross-sectional studies; construct validity; test retest reliability; anxiety; phase 3 clinical trial; patient-reported outcomes; double blind procedure; psychometrics; longitudinal study; internal consistency; psychometry; desmoid tumor; avoidance behavior; rare diseases; aggressive fibromatosis; hopelessness; fibromatosis, aggressive; swelling; anger; patient-reported outcome; brief pain inventory; psychometric properties; abdominal tumor; humans; human; male; female; article; musculoskeletal disease assessment; surveys and questionnaires; ecog performance status; european organization for research and treatment of cancer quality of life questionnaire core 30; disease symptom assessment; nirogacestat; gounder desmoid tumor research foundation desmoid symptom impact scale; patient global impression of severity
Journal Title:	Quality of Life Research
Volume:	32
Issue:	10
ISSN:	0962-9343
Publisher:	Springer
Date Published:	2023-10-01
Start Page:	2861
End Page:	2873
Language:	English
DOI:	10.1007/s11136-023-03445-7
PUBMED:	37347393
PROVIDER:	scopus
PMCID:	PMC10474203
DOI/URL:	https://www.scopus.com/inward/record.uri?eid=2-s2.0-85162699776&doi=10.1007%2fs11136-023-03445-7&partnerID=40&md5=2e4b1f7be1d5147f38151a630f86ddaa
Notes:	Article -- MSK Cancer Center Support Grant (P30 CA008748) acknowledged in PubMed and PDF -- Source: Scopus

https://synapse.mskcc.org/synapse/works/has_related_data_chk/210069