Al-Rayess, Heba2024-01-052024-01-052021-08https://hdl.handle.net/11299/259535University of Minnesota M.S. thesis. August 2021. Major: Clinical Research. Advisor: Kyriakie Sarafoglou. 1 computer file (PDF); iv, 28 pages.BACKGROUND. Young children with CAH require small doses (0.1-1.25 mg) of hydrocortisone (HC) to control excess androgen production and to avoid the negative effects of overtreatment. The smallest commercially available HC formulation, before the recent FDA approval of HC granules, was a scored 5 mg tablet. The options to achieve small doses were limited to using a pharmacy-compounded suspension, which the CAH Clinical PracticeGuidelines recommends against, or splitting tablets into quarters or eighths, or dissolving tablets into water. METHODS. Cross sectional chart review of 130 children with classic CAH treated with tablets versus a pharmacy-compounded alcohol-free hydrocortisone suspension to compare growth, weight, skeletal maturation, total daily HC dose, and total HC exposure over the first four years of life. RESULTS. No significant differences were found in height, weight, or BMI z-scores at 4 years, and in predicted adult height, before or after adjusting for age at diagnosis and sex. Bone age z- scores averaged 2.78 SDs lower for patients treated with HC suspension compared to HC tablets after adjusting for sex and age at diagnosis (p<0.001). The suspension group received 30.4% lower (p<0.001) average cumulative HC doses by their 4th birthday. CONCLUSIONS. Our data indicates that treatment with alcohol-free HC suspension decreased androgen exposure as shown by lower bone age z-scores, allowed lower average and cumulative daily HC dose compared to HC tablets, and generated no significant differences in SDS in growth parameters in children with CAH at 4 years of age. Long-term outcomes of treating children with CAH with smaller HC doses during childhood need to be studied.enCongenital adrenal hyperplasiaGrowthHydrocortisoneThesis or Dissertation