CAHQL: A Patient-Reported Outcome Instrument to Assess Health-Related Quality of Life in Congenital Adrenal Hyperplasia
An erratum to this article was published (J Clin Endocrinol Metab, 13 Aug 2026 ↗)
Authors' conclusion
CAHQL is the first validated PRO instrument to capture disease-specific HRQoL outcomes in CAH. In addition to its anticipated use in the clinical setting, the instrument could be used to assess the efficacy of novel treatments in development.
Study context
- Population
- Patients >16 years old with classic congenital adrenal hyperplasia (CAH)validation sample
- Participants
- 69 patients
- Outcome
- CAHQL instrument structure, internal consistency and validity
Methodological detailsFDA guidelines for patient-reported outcome (PRO) instrument development, patient and parent interviews, expert feedback, cognitive interviews, Edited after cognitive interviews with 6 patients, Preliminary instrument from interviews with 12 patients (aged 16 to 68 years) with CAH and 3 parents, and feedback from 4 endocrinologists
- FDA guidelines for PRO instrument development
- Patient and parent interviews
- Expert feedback
- Cognitive interviews
- Edited after cognitive interviews with 6 patients
- Preliminary instrument from interviews with 12 patients (aged 16 to 68 years) with CAH and 3 parents, and feedback from 4 endocrinologists
Performance
As reported in the abstract
Final questionnaire: questions within 7 domains (General Health, Adrenal Insufficiency, Glucocorticoid Excess, Physical Functioning, Mental Health and Cognition, Social Functioning, Sexual Functioning)
44
Internal consistency (Cronbach's alpha), acceptable
≥ .6
Validity vs non-CAH-specific validated health-related quality of life (HRQoL) and PRO instruments (r), acceptable
−0.350 to 0.866
Original publication
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