ESPE Abstracts (2018) 89 P-P2-003

Glucocorticoid Replacement Regimens in the Treatment of 21-hydroxylase Deficiency Congenital Adrenal Hyperplasia: A Systematic Cochrane Review

Sze Nga,b & Karolina Stepienc


aSouthport and Ormskirk Hospitals NHS Trust, Southport, UK; bUniversity of Liverpool, Liverpool, UK; cSalford Royal NHS Foundation Trust, Salford, UK


Background: Congenital adrenal hyperplasia (CAH) is an autosomal recessive condition which leads to glucocorticoid deficiency. During childhood, the main aims of treatment are to prevent adrenal crisis and to achieve normal stature, optimal adult height and to undergo normal puberty. In adults, the aims of treatment are to prevent adrenal crisis, ensure normal fertility and to avoid long-term consequences of glucocorticoid use. Current treatment regimens for CAH with glucocorticoids cannot optimally replicate normal physiological cortisol level. Overtreatment or undertreatment of CAH is often reported in individuals who may be treated with different steroid treatment regimens. There is no current standard treatment for CAH and physicians often customise treatment for each individual using various regimens. It remains unclear which treatment regimen is most effective.

Objectives: This Cochrane review aims to determine the efficacy and safety of different glucocorticoid replacement regimens in the treatment of CAH due to 21-hydroxylase deficiency in children and adults.

Methods: We included any RCT or quasi-RCT comparing different glucocorticoid replacement regimens in the treatment of CAH due to 21-hydroxylase deficiency in children and adults. The authors independently searched and extracted data. Data from different interventions were analysed separately. GRADE was used to assess the quality of the evidence.

Results: The initial search identified 297 records which identified 20 publications for further examination. After screening full texts of 20 selected papers, we included five RCTs with 101 people with CAH due to 21-hydroxylase deficiency. The number of participants in each trial varied from 6 to 44 with participants’ ages ranging from 1.2 to 21 years. They received different glucocorticoid replacement regimens such as frequency in the day or different forms of glucocorticoids and were followed up for between six and 12 months. Although 17OHP and androstenedione are frequently used to monitor treatment, there is a high amount of variability in the measurements which hampers usefulness of these tests. Overall, we judged trials to be moderate to high risk of bias; lack of methodological detail led to ’unclear’ risk of bias judgements across many of the domains.

Conclusions: There are limited trials to date which compare the efficacy and safety of different glucocorticoid replacement regimens in the treatment of CAH in children and adults. This review addressed a diverse range treatment regimens with many trials at high or unclear risk of bias. There is insufficient evidence to indicate which glucocorticoid replacement regimen results in better outcomes.

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