PROMIS Overview: Development of New Tools for Measuring Health-related Quality of Life and Related Outcomes in Patients with Chronic Diseases

PROMIS Overview: Development of New Tools for Measuring Health-related Quality of Life and Related Outcomes in Patients with Chronic Diseases William Riley, Ph.D. National Heart, Lung, and Blood Institute David Cella, Ph.D. Northwestern University Feinberg School of Medicine on behalf of the PROMIS Network

Brief History of PROMIS NIH Roadmap Initiative to: Develop and evaluate PRO item banks for a range of chronic disease outcomes research Create a computerized adaptive testing system (CAT) for efficient administration Make these resources available to clinical researchers PROMIS I (FY2005-2009) 6 research sites and a Statistical Coordinating Center Develop measures for 5 core HRQL domains: pain, fatigue, emotional distress, physical function, and social well-being PROMIS II: (FY2010 2013) 12 research sites and three centers Develop new domains & validate PROMIS I measures in a variety of clinical populations & in children, minorities, persons with disabilities

Progress to Date Over 30 PROMIS research protocols Nearly 40,000 participants > 1,500 in qualitative research > 35,000 in quantitative research More than 9,000 children More than 2,000 proxy reports More than 25,000 adults More than 4,000 Spanish-speaking adults and children 30 released item banks to date (21 adult, 9 child) Over 100 publications to date Web-based Administration and Scoring System (Assessment Center)

Why PROMIS? Don t we have enough patient report measures already? Cacophony of PROs for any given health concept Varying quality, complexity, and response burden Difficult to compare findings across studies or conditions on a single metric Most current PROs were developed prior to: Current qualitative item development standards Application of Item Response Theory to health measures Widespread use of computerized administration

How is PROMIS Different? Rigorous Item Development

PROMIS Item Development Extant review of existing items (legacy items) Patient focus groups to insure content saturation Development of new and derived items Binning and winnowing of items Revisions for readability, consistency, and translatability Cognitive interviews (with iteration) to insure each item is understood and responded to as intended

How is PROMIS Different? Utilization of Item Response Theory (IRT) and Computer Adaptive Testing (CAT) Psychometric properties at the item level allow for item banks that can be flexibly administered and continuously refined Crosswalk with existing scales for comparability Improved efficiency (less respondent burden) Automated administration, scoring and reporting Tailored targeting of items Item administration based on prior responses

Item Response Theory (IRT): Category Response Curves In the past 7 days, I felt unhappy. 1.0 0.8 Never Rarely Some times Always Often 0.6 P 0.4 0.2 In the past 7 days, I felt I had no reason for living. P 0.0 1.0 0.8 0.6 very mild Depressive Symptoms severe -3.00-2.00-1.00 0.00 1.00 2.00 3.00 Never Some times Rarely Always Often 0.4 0.2 0.0-3.00-2.00-1.00 0.00 1.00 2.00 3.00 very mild Depressive Symptoms severe

Simulate Measure = 48 Item Meas SE 1 37 21 2 40 12 3 42 9 4 44 8 5 45 7 6 46 7 7 47 6 8 48 6 0 10 20 30 40 50 60 70 80 90 100 9 47 5 10 48 5

Fatigue Item Bank Chemotherapy trial Rheumatoid Arthritis trial Parkinson s Disease trial Items 1-10 CAT Items 6-12 Diabetes trial Items 2, 4, 9, 13 CHF trial Items 1-5 Same metric, same meaning

How is PROMIS Different? Improved Measurement Precision Psychometric properties at the item level allow for item banks that can be flexibly administered and continuously refined Improved efficiency (less respondent burden) Automated administration, scoring and reporting Tailored targeting of items to severity level Item administration based on prior responses Improved measurement precision (reliability)

Measurement Precision (standard error) for Physical Function 0.5 PF items for SF-12 0.4 0.3 0.2 PF-10 items from SF-36 HAQ CAT 10 items Full Bank SE = 0.32 rel = 0.90 SE = 0.22 rel = 0.95 0.1 rheumatoid arthritis patients representative sample 0 10 20 30 40 50 60 70 80-4 -3-2 -1 0 +1 +2 normed theta values Rose et al, J Clin Epidemiol 2007 (accepted)

Comparison of Measurement Precision Full-length Item Bank vs. Legacy vs. CAT vs. Short-form 0.6 0.5 0.4 4-item SF36/Vitality 4-item CAT 13-item FACIT-Fatigue 13-item CAT 98-item Bank Standard Error 0.3 0.2 SE=0.32 (r=0.90) SE=0.22 (r=0.95) 0.1 0-2.5-1.5-0.5 0.5 1.5 2.5 No Fatigue Severe Fatigue

PROMIS Domain Framework Physical Health Symptoms Function Self-Reported Health Mental Health Affect Behavior Cognition Social Health Relationships Function

PROMIS Current Physical Health Banks Adult Pediatric Pain Behavior Pain Interference Pain Interference Physical Health Fatigue Physical Function Sleep Disturbance Sleep-related Impairment Sexual Function Fatigue Upper Extremity Function Mobility Asthma Impact

PROMIS Current Mental Health Banks Adult Pediatric Anxiety Anxiety Mental Health Depression Anger Illness Impact Negative Illness Impact Positive Applied Cognition - Concerns Applied Cognition - Abilities Depression Anger

PROMIS Current Social Health Banks Adult Ability to Participate in Roles & Activities Pediatric Peer Relationships Social Health Satisfaction with Roles & Activities Companionship Emotional Suppor t Informational Suppor t Instrumental Suppor t Social Isolation

PROMIS Language Availability and Translations in Progress Subdomain Bank - Calibrated ENG items Un-calibrated ENG items Short-form Anger (29) SPA, GER (partial bank), POR, MCHI, SPA (14) SPA, MCHI Anxiety (29) SPA, GER (partial bank), POR, MCHI, SPA (19) SPA, MCHI Depression (28) SPA, GER (partial bank), POR, MCHI SPA (25) SPA, CZE, FRE, GER, HUN, ITA, POL, MCHI Fatigue (95) SPA, GER (partial bank), POR, MCHI SPA (22) SPA, CZE, FRE, GER, HUN, ITA, POL, MCHI Pain Behavior (39) SPA, GER (partial bank), POR, MCHI n/a SPA, MCHI Pain Interference (41) SPA, GER (partial bank), POR, MCHI SPA (6) SPA, MCHI Physical Function (124) SPA, GER (partial bank), POR, MCHI, SPA (15) SPA, MCHI Sleep Disturbance (27) SPA, MCHI n/a SPA, MCHI, BUL, GER, HUN, JPN, LAT, LIT, POL, RUS, SLK, UKR Sleep-Related Impairment (16) SPA, MCHI n/a SPA, MCHI Social Satisfaction_DSA (12) SPA, GER (partial bank), POR, MCHI n/a SPA, MCHI Social Satisfaction_Roles (14) SPA, GER (partial bank), POR, MCHI n/a SPA, MCHI Social Function SPA SPA, MCHI Social Support SPA SPA, MCHI Social Isolation SPA Social Satisfaction SPA Global (10) SPA, POR, MCHI (one item) n/a SPA, POR Pediatric * (156) SPA, POR SPA, POR (1) SPA, POR *Pediatric items: Anger (Short-Form), Anxiety, Depression, Fatigue, Pain Interference, Peer Relationships, Mobility, Upper Extremity, and Asthma Selected adult items (5) from Fatigue, Pain Impact, Physical Function, Sleep/Wake, and Social-Satisfaction with Roles banks are also available in Danish, Dutch, French, German, Italian and Norwegian.