Abstract
PurposeThe purpose of this study was to evaluate the psychometric properties of the PROMIS-Physical Function (PF) and Worst Stiffness Numeric Rating Scale (NRS) among patients with tenosynovial giant cell tumors (TGCT).MethodsMeasurement properties of the customized lower extremity (LE) and upper extremity (UE) PROMIS-PF scales and Worst Stiffness NRS were assessed using data from the Phase 3 ENLIVEN trial (n = 120). Anchor- and distribution-based analyses were utilized to derive a responder threshold for meaningful change over time. The Patient Global Rating of Concept (PGRC)-Physical Functioning and Patient Global Impression of Change (PGIC)-Stiffness served as anchors. Responsiveness and responder threshold analyses were from baseline to week 25.ResultsCronbach’s alpha values for internal consistency reliability were 0.93 and 0.91 for the PROMIS-PF LE and UE, respectively. Test-retest reliability intra-class correlation coefficients were > 0.75 for both instruments. Convergent validity for both instruments was supported by moderate to strong correlations (≥0.30) with the Brief Pain Inventory and EQ-5D. Known-groups validity was established between subgroups stratified by pain level (p < 0.05). Responsiveness was supported by evaluating change scores among different levels of change in PGRC-Physical Functioning and PGIC-Stiffness (overall F values < 0.001). Triangulation of responder definition analyses resulted in a threshold of ≥3 for the PROMIS-PF and ≥ 1 for the Worst Stiffness NRS.ConclusionThis study is the first to establish the psychometric properties of patient-reported outcome measures in TGCT. The evidence demonstrates that the PROMIS-PF and Worst Stiffness NRS have good reliability, validity, and responsiveness, and provides guidance for the interpretation of meaningful change.
Highlights
Tenosynovial giant cell tumors (TGCT) are rare nonmalignant neoplasms that involve the synovium or tendon sheath
Individual unidimensional Confirmatory factor analysis (CFA) models were fit for the nine Patient-Reported Outcomes Measurement Information System (PROMIS)-Physical Function (PF) items that overlapped between the upper extremity (UE) and LE scales, and the 13 PROMIS-PF lower extremity items
Data from 104 LE and UE subjects were included for the model that included the 9 items that overlapped across tumor location, the factor loadings ranged from 0.609– 0.881, with the exception of item PFA16R1 (“Are you able to dress yourself, including tying shoelaces and buttoning up your clothes?”), which had a factor loading of 0.394
Summary
Tenosynovial giant cell tumors (TGCT) are rare nonmalignant neoplasms that involve the synovium or tendon sheath. They typically present in young and middleaged adults of both sexes, and result in functional limitations, morbidity, and decreased quality of life (QOL) [21]. In the recently completed ENLIVEN Phase 3 trial (NCT02371369), pexidartinib, a novel, orally active, small molecule receptor tyrosine kinase inhibitor has demonstrated efficacy in reducing tumor size and improving functional outcomes [18]. Through qualitative work completed in preparation of the ENLIVEN trial, which included interviews with both patients and clinicians, it was demonstrated that physical functioning and stiffness were important treatment outcomes to patients with TGCT [9]
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