ESPE Abstracts

Standard management of Adrenal Insufficiency (AI) in children is with hydrocortisone (HC). Under or over-treating impacts on growth and quality of life. Until recently, treatment involved dissolving 10mg HC tablets in water; this is time consuming and may give inaccurate doses. We have offered HC granules (Alkindi) to all new patients ≤6-years since January 2019 and 22 patients who were currently dissolving HC tablets. We have reviewed our patients’ experience with Alkindi and monitored growth and safety data over this period. Questionnaires were sent to families of children with AI who started HC ≤6-years, asking about treatment satisfaction, stress-dosing and hospital admissions. We also collected growth data from hospital e-records, including parental heights. Thirty-six patients (≤6 years; 26M, 10F) with AI started/switched to Alkindi between January 2019 and March 2022. 21 were diagnosed with congenital adrenal hyperplasia, 13 combined pituitary hormone deficiency and 2 neonatal adrenal haemorrhage. Questionnaires were received from 26/34 patients (1 moved away; 1 had a recent bereavement) - 76% response rate. Alkindi has also been used in 3 older patients (aged 10-13 years) requiring smaller doses (not included in growth data). All 11 patients who started on Alkindi at diagnosis continue. 16/20 (80%) patients who switched from dissolved HC to Alkindi remain on this, 4/20 (20%) did not tolerate the granules. A further 5 patients, with unsafe swallow, were not offered Alkindi. Patient satisfaction with their current treatment was high (96%). Families on Alkindi commented on accuracy of dosing and ease of use. In the last 6-months, stress dosing varied from 0-7 occasions (average 4 episodes/patient/year), with no significant difference between Alkindi and HC. Only 1 patient was admitted with an adrenal crisis (used Alkindi routinely, but stress-dosed with HC). For patients on Alkindi from diagnosis, height SDS remained stable (-0.68 baseline; +0.01 at 1-year; -0.36 at 2-years; parental +0.81). In those that switched to Alkindi, their height SDS moved closer to parental SDS (-0.62 baseline; -0.20 at 1-year; +0.17 at 2-years; parental +0.15). This is the largest single-centre report of Alkindi administration over the longest time-period, demonstrating high acceptability for most AI patients. All patients on Alkindi from diagnosis tolerate it well; 20% of those switching to Alkindi returned to dissolved HC. Growth data, based on height SDS scores, is encouraging. No safety issues with increased stress dosing or adrenal crisis have been identified. On-going follow up will continue to monitor long-term growth.