Are We Achieving Pharmacological Disease Control in Acromegaly?

Abstract

Acromegaly is a rare endocrine disorder, associated with significant morbidity and mortality due to the harmful effects of prolonged exposure to increased levels of growth hormone (GH) and its effector, insulin-like growth factor-1 (IGF-1). The most common cause of acromegaly is a pituitary adenoma, for which surgical resection is usually the first choice treatment. In cases where surgical resection is not possible, or where the patient declines surgery, somatostatin analogues (SSAs) are used as first-line medical therapy. Other therapeutic options include dopamine antagonists and the GH receptor antagonist – pegvisomant. In addition, considerable current research is investigating the clinical utility of combined therapies. Disease control is defined in terms of reduction of GH and IGF-1 normalisation and reduction in mortality levels to those seen in the general population. Reported disease control rates of acromegaly are highly variable and it has been reported that treatment efficacy in clinical practice is considerably lower than the success rates reported by reference centres. There is therefore a substantial need for improved disease management strategies for acromegaly.