Long-term use of continuous subcutaneous hydrocortisone infusion therapy in patients with congenital adrenal hyperplasia
| Autor: | Ninet Sinaii, Ashwini Mallappa, Aikaterini A. Nella, Deborah P. Merke, Chia Ying Liu, Ashley F. Perritt, Steven J. Soldin, Alexander Ling, Parag Kumar, Hamsini Rao, Verena Gounden |
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| Rok vydání: | 2018 |
| Předmět: |
Adult
Male medicine.medical_specialty Hydrocortisone Proton Magnetic Resonance Spectroscopy Endocrinology Diabetes and Metabolism Urology 030209 endocrinology & metabolism Article Body Mass Index 03 medical and health sciences 0302 clinical medicine Endocrinology Infusion therapy Bone Density Internal medicine medicine Adrenal insufficiency Humans Adrenal adenoma Congenital adrenal hyperplasia Androstenedione hirsutism Adrenal Hyperplasia Congenital business.industry medicine.disease 030220 oncology & carcinogenesis Quality of Life Lean body mass Female business Biomarkers medicine.drug |
| Zdroj: | Clinical Endocrinology. 89:399-407 |
| ISSN: | 0300-0664 |
| DOI: | 10.1111/cen.13813 |
| Popis: | 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 (HRQoL) 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 HRQoL, 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 HRQoL 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. |
| Databáze: | OpenAIRE |
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