Quality of Life in Children and Young People With Congenital Adrenal Hyperplasia-UK Nationwide Multicenter Assessment

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.Clinical Trials Registration Number: SCH/15/088.

Keywords: 17-OH progesterone; androstenedione; congenital adrenal hyperplasia; hyperandrogenism; quality of life.