Single-joint outcome measures: preliminary validation of patient-reported outcomes and physical examination.
OBJECTIVE
To assess the validity, responsiveness, and reliability of single-joint outcome measures for determining target joint (TJ) response in patients with inflammatory arthritis.


METHODS
Patient-reported outcomes (PRO), consisting of responses to single questions about TJ global status on a 100-mm visual analog scale (VAS; TJ global score), function on a 100-mm VAS (TJ function score), and pain on a 5-point Likert scale (TJ pain score) were piloted in 66 inflammatory arthritis subjects in a phase 1/2 clinical study of an intraarticular gene transfer agent and compared to physical examination measures (TJ swelling, TJ tenderness) and validated function questionnaires (Disabilities of the Arm, Shoulder and Hand scale, Rheumatoid Arthritis Outcome Score, and the Health Assessment Questionnaire). Construct validity was assessed by evaluating the correlation between the single-joint outcome measures and validated function questionnaires using Spearman's rank correlation. Responsiveness or sensitivity to change was assessed through calculating effect size and standardized response means (SRM). Reliability of physical examination measures was assessed by determining interobserver agreement.


RESULTS
The single-joint PRO were highly correlated with each other and correlated well with validated functional measures. The TJ global score exhibited modest effect size and modest SRM that correlated well with the patient's assessment of response on a 100-mm VAS. Physical examination measures exhibited high interrater reliability, but correlated less well with validated functional measures and the patient's assessment of response.


CONCLUSIONS
Single-joint PRO, particularly the TJ global score, are simple to administer and demonstrate construct validity and responsiveness in patients with inflammatory arthritis. (ClinicalTrials.gov identifier NCT00126724).
Targeted Genetics Corporation, Seattle, Washington, USA.
Alison E
AE
Heald
Edward J
EJ
Fudman
Pervin
P
Anklesaria
Philip J
PJ
Mease
D000203:Activities of Daily Living; D000328:Adult; D000368:Aged; D015535:Arthritis, Psoriatic / Q000503:physiopathology*; D001172:Arthritis, Rheumatoid / Q000503:physiopathology*; D004185:Disability Evaluation; D005260:Female; D006304:Health Status; D006801:Humans; D007596:Joints / Q000503:physiopathology*; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care; D010147:Pain Measurement / Q000592:standards; D010358:Patient Participation / Q000379:methods*; D010808:Physical Examination; D011788:Quality of Life; D015203:Reproducibility of Results; D012680:Sensitivity and Specificity; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires / Q000592:standards