Assessment of medication adherence in children and adults with congenital adrenal hyperplasia and the impact of knowledge and self‐management

Abstract

Background Congenital adrenal hyperplasia (CAH) is caused by a deficiency of one of the enzymes required for cortisol biosynthesis. The disease is classified as either classic (severe phenotype), subdivided into simple virilizing (SV) and salt‐wasting (SW), or non‐classic (NC) CAH. The treatment regime involves life‐long glucocorticoid replacement, especially in classic phenotype. Objectives We aimed to assess medication adherence, endocrine knowledge and self‐management in patients with CAH and to compare patients’ and physicians’ assessments of medication adherence. Methods A prospective cross‐sectional study of 108 patients with CAH (52 children and 56 adults) and 45 parents/caregivers. Two adherence measures were used, a self‐reported questionnaire named Adherence Starts with Knowledge (ASK‐12) with a cut‐off level > 22 defined as poor adherence rate, and an assessment by a physician based on growth rate, 17‐hydroxyprogesterone profile, and medical history, ranked using a five‐point Likert scale. Measurements of the patients’/parents’ knowledge and self‐management were performed using Endocrine Society Clinical Practice Guidelines. Results Self‐reported medication adherence was good with 74% of the participants reported good adherence with higher adherence in patients with the SW form. The highest endocrine knowledge and self‐management were found in parents compared with children and adults with classic CAH. There was 30% discordance between the assessments by a physician and the self‐reported ASK‐12 scores independent of the severity of CAH. Conclusion Patients and endocrinologists reported high medication adherence, however, discordance was found in 30% of the studied patients. Patients with the more severe form of CAH had higher adherence rates and demonstrated good endocrine knowledge/self‐management.

The impact of adherence and therapy regimens on quality of life in patients with congenital adrenal hyperplasia

“…An important clinical question to consider when treating patients with CAH is how much adherence is enough to obtain the full treatment benefits of cortisol 27 . It is well known today that patients living with a chronic disorder commonly have a medication adherence of around 50%–70% 12,14 and nonadherence has been shown to contribute to poor health outcomes including impaired QoL 11 . This is in line with our results with a poor adherence rate to therapy regimens as a key factor for impaired QoL in adolescents and adults with CAH.…”

“…They were recruited from the Department of Paediatric Endocrinology at Astrid Lindgren Children's Hospital, Department of Endocrinology and the Department of Gynecology and Reproductive Medicine at Karolinska University Hospital, all in Stockholm, Sweden. A detailed description of the cohort has been previously published 14 …”

“…A detailed description of the cohort has been previously published. 14 In total, 54%, 23% and 23% of participants had the SW, SV and NC phenotypes, respectively. Seventy-seven percent had the classic form of CAH (SW and SV phenotypes).…”

“…Regular practical CAH training sessions with the aim of improving patients' understanding of the benefits of medication as well as increasing their knowledge of the high-risk nature of their illness are possible factors that have contributed to our results. 14,33 Fluctuating cortisol and adrenal androgen levels may have an impact on QoL and several studies have shown different results regarding treatment regimens. 28,29 Additionally, patients with impaired QoL, poor disease control and low adherence rates are sometimes prescribed a more potent glucocorticoid with a longer half-life, which makes it difficult to understand the relationship between cause and effect.…”

Increased Prevalence of Fractures in Congenital Adrenal Hyperplasia: A Swedish Population-based National Cohort Study

“…Androgen excess in CAH may contribute to fewer fractures due to its protective role, but this effect of androgens is difficult to tease out due to the interplay of many additional confounding factors. Poor glucocorticoid adherence increases androgen concentrations, and this may increase BMD, while good adherence may result in low androgen concentrations ( 42 , 43 ) and impaired BMD ( 25 ). Increased adrenal androgen concentrations in males with CAH, on the other hand, may decrease the total testosterone concentration due to inhibition of gonadotropins ( 44 ).We did not find an increased fracture rate in patients with the NC phenotype, which may be explained by prolonged androgen exposure before diagnosis, at least in females.…”