title: Psychometric evaluation of a patient-reported symptom assessment tool for adults with haemophilia (the HAEMO-SYM).
abstract: In patients with haemophilia, repeated bleeding events result in significant comorbid conditions that can degrade health-related quality of life. Clinician-reported symptom measures are available for use in patients with haemophilia A or B; however, there has not been a validated patient-reported symptom evaluation instrument available for haemophilia to date. The objective of this study was to develop and evaluate a self-report instrument, the HAEMO-SYM, for measuring symptom severity in patients with haemophilia. Eighty-four haemophilic subjects from Canada and the USA were enrolled and completed the HAEMO-SYM, SF-36, and Health Assessment Questionnaire-Functional Disability Index (HAQ-FDI). Four-week reproducibility was evaluated in 72 stable subjects. Construct validity was assessed by correlating subscale scores with the SF-36, HAQ-FDI, a coping questionnaire and clinical scores. The final 17-item HAEMO-SYM has two subscales: pain and bleeds. Internal consistency reliability was good (Cronbach's alphas, 0.86-0.94) and test-retest reliability was good (Intraclass Correlation Coefficients, 0.75-0.94). HAEMO-SYM subscale scores were significantly correlated with SF-36 scores (P < 0.05 for all except HAEMO-SYM Pain and SF-36 Mental Health), HAQ-FDI scores (P < 0.05 for all but HAEMO-SYM Bleeds with HAQ-FDI Hygiene and Reach), Gilbert scale (P < 0.01), coping (P < 0.05) and global pain (P < 0.001). Mean HAEMO-SYM scores varied significantly in groups defined by severity, HIV status and treatment regimen. Greater symptom severity was associated with more severe disease, HIV-positive status and prophylaxis treatment. The results of this study suggest that the HAEMO-SYM, a haemophilia-specific symptom severity instrument, has good reliability and provides evidence that supports construct validity in patients with haemophilia.
authors.affiliation: The Center for Health Outcomes Research at UBC, Bethesda, MD, USA.
authors.forename: A
authors.initials: A
authors.lastname: Rentz
authors.forename: E
authors.initials: E
authors.lastname: Flood
authors.forename: R
authors.initials: R
authors.lastname: Butler
authors.forename: B
authors.initials: B
authors.lastname: Christie
authors.forename: P
authors.initials: P
authors.lastname: Giangrande
authors.forename: P
authors.initials: P
authors.lastname: McCusker
authors.forename: J
authors.initials: J
authors.lastname: Wasserman
authors.forename: E
authors.initials: E
authors.lastname: Gorina
mesh_terms: D000203:Activities of Daily Living / Q000523:psychology*; D000328:Adult; D002170:Canada; D003431:Cross-Cultural Comparison; D006467:Hemophilia A / Q000150:complications / Q000523:psychology* / Q000628:therapy; D006801:Humans; D008297:Male; D011594:Psychometrics / Q000379:methods; D011788:Quality of Life / Q000523:psychology*; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D014481:United States