Acromegaly: a review of current medical therapy and new drugs on the horizon

Acromegaly is a disease that results from a growth hormone (GH)-secreting pituitary tumor. Clinically, the disease is characterized by excessive skeletal growth, soft tissue enlargement with disfigurement, and increased risk of cardiovascular death. The goals of treatment are the removal or reduction of the tumor mass via surgery and normalization of GH secretion. Another treatment goal is the preservation of normal pituitary function if possible. Transsphenoidal surgery by an experienced neurosurgeon is usually the first line of therapy, especially for small tumors. Surgeon expertise is crucial for outcome, with dedicated pituitary surgeons having better results. However, overall cure rates remain low because patients with these tumors usually present at an incurable stage. Therefore, medical therapy to control excess GH secretion plays a significant role in a large proportion of patients with acromegaly who are not cured by surgery or other forms of therapy, such as radiotherapy, and/or are awaiting the effects of radiotherapy. If surgery is not curative, lifelong monitoring and the control of excess GH is usually necessary by a care team experienced in handling this chronic disease. In the past decade major progress has occurred in the development of highly specific and selective pharmacological agents that have greatly facilitated more aggressive management of active acromegaly. Treatment approach should be individualized and take into consideration a patient's tumor size and location, symptoms, comorbid conditions, and preferences. Because a surgical cure can be difficult to achieve, all patients, even those with what seems to be a clinically and biochemically inactive disease, should undergo long-term biochemical testing and pituitary MR imaging. (DOI: 10.3171/2010.7.FOCUS10154)