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Long‐term use of continuous subcutaneous hydrocortisone infusion therapy in patients with congenital adrenal hyperplasia
Author(s) -
Mallappa Ashwini,
Nella Aikaterini A.,
Sinaii Ninet,
Rao Hamsini,
Gounden Verena,
Perritt Ashley F.,
Kumar Parag,
Ling Alexander,
Liu ChiaYing,
Soldin Steven J.,
Merke Deborah P.
Publication year - 2018
Publication title -
clinical endocrinology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.055
H-Index - 147
eISSN - 1365-2265
pISSN - 0300-0664
DOI - 10.1111/cen.13813
Subject(s) - congenital adrenal hyperplasia , medicine , hirsutism , adrenal insufficiency , androstenedione , quality of life (healthcare) , hydrocortisone , endocrinology , cosyntropin , lean body mass , androgen , pediatrics , adrenocorticotropic hormone , hormone , obesity , polycystic ovary , insulin resistance , body weight , nursing
Summary Background In a phase 2 short‐term (6 months) study of patients with congenital adrenal hyperplasia ( CAH ), continuous subcutaneous hydrocortisone infusion ( CSHI ) was found to be a safe, effective and well‐tolerated method of replacing cortisol with improved disease and patient‐related outcomes. Objective To evaluate the safety and efficacy of long‐term CSHI . Design Single‐centre, open‐label, phase 2 extension study. Patients Five adults with classic CAH . Measurements Biomarkers of disease control, metabolic indices and health‐related quality‐of‐life ( HRQ oL) estimates. Results Six of eight patients chose to continue on long‐term CSHI therapy. Compared to baseline, eighteen months of CSHI resulted in decreased ( P = 0.043) 0700‐hour ACTH , 17‐hydroxyprogesterone, androstenedione and progesterone; increased whole‐body lean mass ( P = 0.024); and improved HRQ oL, especially symptoms of adrenal insufficiency ( P = 0.003). Findings at six and eighteen months did not differ, and improvements achieved in androgen control, lean body mass and HRQ oL after 6 months of CSHI were maintained at eighteen months. The hydrocortisone dose appeared to decrease with time [6 vs 18 months: 38.3 ± 8.8 vs 33.6 ± 12.2 mg/day ( P = 0.062)], especially in women receiving oral contraceptives. Reduction of testicular adrenal rest and adrenal size observed at 6 months remained stable. In one patient, an adrenal adenoma continually decreased over time. Subjective improvement in hirsutism was reported. Conclusions Long‐term use of CSHI is a safe and well‐tolerated treatment option in a select set of adults with classic CAH . Improvements observed short term in disease control and subjective health status continued long term.