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ABSTRACT - Recent years have seen several advances in the management of endocrine diseases. These include novel drugs developed as a consequence of better understanding of the pathophysiology of endocrine conditions, as well as improved delivery methods for existing drugs. In this article, we summarise recent studies evaluating several drugs used in the treatment of endocrine disorders.
KEY WORDS: endocrine diseases, pharmacotherapy
Introduction
Control of hormone hypersecretion and replacement of hormone deficiencies are the mainstay of treatment in endocrinology. The ultimate goal of treatment is to reduce the longterm morbidity and mortality associated with hormone hypoor hypersecretion and to improve the quality of life. Here, we review the evidence for the efficacy and safety of some of the recent drugs used in the pharmacotherapy of several endocrine diseases.
Cushing's disease
The primary treatment for Cushing's disease is surgical removal of the adrenocorticotropic hormone (ACTH)-secreting pituitary tumour. Medical control of hypercortisolism is required when surgery is contraindicated, in preparation for surgery and in cases of persistence or recurrence of hypercortisolism following surgery. Medication commonly used includes ketoconazole and metyrapone. Ketoconazole blocks the first step (sidechain cleavage) and, to a lesser extent, the last step in cortisol biosynthesis (ie conversion of 11-deoxycortisol to cortisol) by inhibiting 11 beta-hydroxylase. Metyrapone inhibits the last step in cortisol biosynthesis. Two medical treatments targeting the pituitary corticotroph adenoma have recently shown potential benefit in the treatment of Cushing's disease.
Cabergoline
Dopamine (D)2 receptors have been identified in corticotroph tumours. Treatment with cabergoline (a D2 agonist) is associated with a reduction in 24-h urinary free cortisol (UFC) in patients with Cushing's disease.1,2 In one study, cabergoline normalised 24-h UFC after six months in 25% of patients with Cushing's disease unsuccessfully treated by surgery at doses ranging from 2 to 3 mg/week.1 In another study, normalisation of UFC was achieved in approximately 37% of patients with Cushing's disease within 3-6 months. In this study, 30% of patients had sustained normalisation of UFC after a mean of 37 months, with a mean dose of 2.1 mg/week of cabergoline.2 Close follow-up is necessary for dose adjustments. However, UFC is not a good marker of cortisol dynamics and normalisation of UFC does not mean that the patient is necessarily in remission. Plasma cortisol concentrations...