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Long-term outcomes in patients with congenital adrenal hyperplasia treated with hydrocortisone modified-release hard
Wiebke Arlt1,2, Aude Brac de la Perrière3, Angelica L Hirschberg4,5
1MRC Laboratory of Medical Sciences (MRC LMS), London W12 0HS, United Kingdom.
Background:
Hydrocortisone modified-release hard capsules (MRHC, development name Chronocort) replace the physiological overnight cortisol rise and improve the biochemical control of congenital adrenal hyperplasia (CAH).
Aim:
This study aims to evaluate long-term safety, tolerability, and efficacy of MRHC.
Methods:
This is an open-label follow-on study.
Results:
Ninety-one patients with classic CAH, mean age 37 years, 68% female, 32% male, entered the study and 22 discontinued. Median treatment duration was 4 years (range 0.2-5.8). Median hydrocortisone dose at study entry was 30 mg/day and reduced to 20 mg/day after 24 weeks and stayed stable thereafter until 48 months (P < .0001). Disease control improved on MRHC for the steroid disease markers serum 17-hydroxyprogesterone (17OHP) (P < .03) and androstenedione (A4) (P < .002). After 4 years, the majority of patients had a 17OHP < 4-fold upper limit of normal (ULN) (71%) and an A4
Conclusions:
Modified-release hard capsule treatment resulted in hydrocortisone dose reduction followed by a stable dose with improved biochemical control associated with fertility. Biochemical control could be reliably monitored by a single blood sample taken between 09:00 and 13:00 h. The incidence of adrenal crises was below that reported previously in patients with CAH.
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