Quality of Life in children and young people with congenital adrenal hyperplasia—UK nationwide multicenter assessment

Lawrence, N. R. et al. (2024) Quality of Life in children and young people with congenital adrenal hyperplasia—UK nationwide multicenter assessment. Journal of Clinical Endocrinology and Metabolism, 109(1), e336-e346. (doi: 10.1210/clinem/dgad405) (PMID:37439248)

Abstract

Context Quality of life (QoL) has been inconsistently reported in children and young people (CYP) with congenital adrenal hyperplasia (CAH). Objective Assess QoL in CYP with CAH in the UK alongside biometric and androgen profiles. Design To define the evidence base for health care delivery, we conducted a cross-sectional study in CYP with CAH in the UK. Questionnaire results were compared with normative data and between groups, and modelled for association with sex, height, weight, body mass index, or steroid biomarkers of CAH control. Setting Tertiary care in 14 UK centers. Patients Results from 104 patients, 55% female, mean age 12.7 years (SD 3.0), paired responses from parents. Interventions Strengths and Difficulties questionnaire (SDQ) and pediatric QoL questionnaire. Main Outcome Measure Total QoL scores as assessed by SDQ and a pediatric QoL questionnaire in comparison to normative data. Results Total scores were worse in parents than normative data, but similar in patients. Patient QoL was rated better in social functioning but worse in emotional, school, and peer domains by patients, and worse in total scores and domains of peer problems, and psychosocial, emotional, and school functioning by parents. Parents consistently scored QoL of their children lower than their child. Larger height-SD score and lower weight-SD score were associated with better QoL. Girls with lower steroid biomarkers had worse SDQ scores. Conclusions In CYP with CAH, reduced height, increased weight, and hormonal biomarkers consistent with overtreatment were associated with worse QoL; addressing these problems should be prioritized in clinical management.

• Item Type: Articles
• Additional Information: This work was supported by the National Institute for Health and Care Research rare disease translational research collaboration (NIHR RD TRC), the Chief Scientist Office of Scotland, and Diurnal Ltd.
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Ahmed, Professor Syed Faisal
• Authors: Lawrence, N. R., Bacila, I., Dawson, J., Mahdi, S., Alvi, S., Cheetham, T. D., Crowne, E., Das, U., Dattani, M. T., Davies, J. H., Gevers, E., Krone, R. E., Patel, L., Randell, T., Ryan, F. J., Keevil, B., Ahmed, S. F., and Krone, N. P.
• College/School: College of Medical Veterinary and Life Sciences > School of Medicine, Dentistry & Nursing
• Journal Name: Journal of Clinical Endocrinology and Metabolism
• Publisher: Oxford University Press
• ISSN: 0021-972X
• ISSN (Online): 1945-7197
• Published Online: 13 July 2023