Journal Article

Psychometric evaluation of the hepatitis C virus patient-reported outcomes (HCV-PRO) instrument: validity, responsiveness, and identification of the minimally important difference in a phase 2 clinical trial

Dietz, Birgitta, Gooch, Katherine, Anderson, Roger T., Revicki, Dennis A., Erickson, Pennifer, Baran, Robert W.

2014

Overview

Purpose To describe the psychometric properties and identify the minimally important difference (MID) of the hepatitis C virus patient-reported outcomes (HCV-PRO) instrument. Chronic HCV infection and associated treatments negatively affect PROs of function and well-being. Methods In a phase 2 trial, HCV-infected patients received direct-acting antivirals (DAAs) for 12 weeks with peg-interferon/ribavirin (peg-IFN/RBV) for 48 weeks, or placebo plus peg-IFN/RBV. The HCV-PRO total score, SF-36 PCS and MCS scores, EQ-5D-3L, and EQ VAS were measured at baseline, week 8, end of DAA treatment (EODT), end of peg-IFN/RBV treatment (EOT), and posttreatment week 24 (SVR24). Convergent validity of the HCV-PRO was assessed by Pearson's correlation coefficients. Discriminant validity was assessed by analyzing mean HCV-PRO total scores by EQ-5D anxiety/depression and pain/discomfort domain scores (none vs. some) and presence/absence of depression or fatigue adverse events. MID was identified through effect size (ES) and receiver-operating characteristic (ROC) curve analyses (HCV-PRO response vs. SF-36 PCS/MCS and EQ VAS MID thresholds). Results In 74 patients (22 % female; 81 % White; 51 % ≥50 years), correlations (0.64–0.96) between HCV-PRO total scores, SF-36 PCS/MCS scores, and EQ VAS scores at all time points supported convergent validity. HCV-PRO total scores were reduced to 10–30 points in patients impaired by depression, pain, or fatigue symptoms. Impact of peg-IFN/RBV regimen on HCV-PRO ES increased over time (EODT −0.76; EOT −0.93). ES and ROC curve analyses indicated an MID of −10 points. Conclusion The HCV-PRO was valid and responsive in the population studied. An MID of −10 points represented a threshold of clinical significance for the HCV-PRO.

• Publisher: Springer,Springer International Publishing,Springer Nature B.V
• Subject: Adolescent / Adult / Aged / Aged, 80 and over / Antibodies / Antiviral Agents - adverse effects / Antiviral Agents - therapeutic use / Antiviral drugs / Antivirals / Anxiety - complications / Biopsy / Clinical outcomes / Clinical trials / Data Interpretation, Statistical / Depression - complications / Depressive disorders / Discriminants / Drug Therapy, Combination / Fatigue / Female / Health Status Indicators / Hepacivirus / Hepacivirus - classification / Hepacivirus - drug effects / Hepatitis / Hepatitis C / Hepatitis C virus / Hepatitis C, Chronic - drug therapy / Hepatitis C, Chronic - psychology / Humans / Infections / Interferon Type I - adverse effects / Interferon Type I - therapeutic use / Liver / Male / Medicine / Medicine & Public Health / Middle Aged / Patient Outcome Assessment / Patients / Placebos / Protease inhibitors / Psychometrics / Psychometrics - standards / Public Health / Quality of Life / Quality of Life Research / Quantitative psychology / Questionnaires / Reproducibility of Results / Ribavirin - adverse effects / Ribavirin - therapeutic use / Severity of Illness Index / Sociology / Surveys and Questionnaires / Treatment Outcome / Validity / Well being / Wellbeing / Young Adult