| Summary: | Cushing disease is a rare disease caused by persistent exposure to excess glucocorticoid due to abnormal secretion of adrenocorticotrophic hormone (ACTH) from a pituitary adenoma. Major clinical signs and symptoms include obesity, hypertension and cardiovascular disease, glucose intolerance, dyslipidemia, fatigue and muscle weakness, various dermatologic manifestations, neuropsychological changes, bone loss, and limited immune function. Cushing disease is associated with a decrease in quality of life (QoL) and increased mortality primarily due to cardiovascular complications. First-line treatment is surgical resection of the pituitary tumour; however, remission is not always achieved and even when it is, up to 25% of patients will experience recurrence in the long term. Despite poor evidence of efficacy and significant safety concerns, several drugs that have not been approved by Health Canada to treat Cushing disease have been used in these patients in clinical practice. Pasireotide is a somatostatin analogue that binds with high affinity to several subtypes of somatostatin receptors that are over-expressed by ACTH-producing adenomas involved in Cushing disease. Pasireotide has a Health Canada indication for the treatment of adult patients with Cushing disease for whom surgery is not an option or for whom surgery has failed, as long as clinical benefit is derived. The maximum recommended dose is 0.9 mg twice daily. The manufacturer has requested that pasireotide be reimbursed for the treatment of patients with Cushing disease for whom medical therapy is appropriate. The objective of this report was to perform a systematic review of the beneficial and harmful effects of pasireotide for the treatment of Cushing disease in adult patients for whom surgery is not an option or for whom surgery has failed
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