PSYCHOMETRIC ANALYSES OF PATIENT-REPORTED OUTCOME INSTRUMENTS FOR AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE

Author(s)

Cole JC*1;Cheng R1;Oberdhan D2;Krasa HB2, Hays RD3 1Covance Market Access Services, Inc., San Diego, CA, USA, 2Otsuka Pharmaceutical Development & Commercialization, Inc., Rockville, MD, USA, 3University of California at Los Angeles, Los Angeles, CA, USA

OBJECTIVES: Patient-reported disease burden in Autosomal Dominant Polycystic Kidney Disease (ADPKD) has not been rigorously documented.  This study provides initial evaluation of the psychometric properties of two new ADPKD-targeted instruments: ADPKD Impact Scale (ADPKD-IS) and ADPKD Urinary Impact Scale (ADPKD-UIS). METHODS: US-English versions of ADPKD-IS and ADPKD-UIS were administered to 702 adults with ADPKD as part of an observational study.  Confirmatory factor analysis (CFA) was conducted to test the fit of previously developed conceptual frameworks.  Next, item response theory (IRT) and classical psychometrics were examined, including item- and scale-level reliability and convergent validity (with the SF-12v2 and Brief Pain Inventory – Short Form (BPI-SF)).  RESULTS: CFA results for both instruments demonstrated good fit between data and conceptual frameworks (ADPKD-IS domains of physical impact, fatigue, and emotional impact and ADPKD-UIS domains of urinary urgency burden, urinary frequency burden, and nocturia burden): ADPKD-IS = confirmatory fit index (CFI)/nonnormed fit index (NNFI) > 0.95, root mean square error of approximation (RMSEA) <0.06; ADPKD-UIS = CFI/NNFI > 0.99, RMSEA <0.08.  IRT- and item-level analyses revealed no misclassified items with their intended scales or problems with skew or inappropriate heterogeneity of variance throughout the response scale.  Internal consistency reliability for all domains ranged from the mid .80s to mid .90s.  Convergent validity of the ADPKD-IS/ADPKD-UIS domains was supported by correlations with the SF-12 (PCS/MCS) and BPI-SF domains (mid .40s to mid .60s). CONCLUSIONS: The ADPKD IS/ADPKD-UIS instruments demonstrated prespecified psychometric criteria.  Whereas longitudinal evaluation of these measures is needed, the current study provides encouraging results for these new ADPKD-specific measures of patient burden.

Conference/Value in Health Info

2013-05, ISPOR 2013, New Orleans, LA, USA

Value in Health, Vol. 16, No. 3 (May 2013)

Code

PUK18

Topic

Patient-Centered Research

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes

Disease

Urinary/Kidney Disorders

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