Up-to 5-year efficacy of pasireotide in a patient with Cushing's disease and pre-existing diabetes: literature review and clinical practice considerations

Purpose Pasireotide is a multi-receptor-targeted somato- statin analogue approved in the EU and in the US for the treatment of adults with Cushing’s disease (CD). Pasireo- tide has a safety profile similar to other somatostatin ana- logues with the exception of hyperglycemia. In this report and literature review, the current understanding of pre- dicting a positive treatment response to pasireotide in CD and the management of diabetes mellitus (DM) during pasireotide treatment are discussed and analyzed. Case presentation We report a case of a 55-year-old woman with CD and DM who benefitted from long-term pasireotide. The patient, who was enrolled in a phase III trial of the drug, showed early clinical improvements with pasireotide [900 lg subcutaneously twice daily (bid)] but was classified as a non-responder as urinary free cortisol (UFC) levels, were not normalized. Continuation of pa- sireotide for 12 months at an increased dose (1,200 lg bid) normalized UFC levels and restored cortisol rhythm. The initial deterioration in her blood glucose was managed with insulin and metformin; however, after 12 months’ treat- ment with pasireotide her DM was well controlled with oral hypoglycemic agents. Five years later, the patient is still receiving pasireotide (300 lg bid) with no loss of clinical or biochemical efficacy and with continued gly- cemic control. Conclusions This case presentation indicates that uncon- trolled UFC levels during the first few months of pasireotide treatment as well as worsening of glycemic control in patients with CD and DM are not always predictive of the efficacy and tolerability and appears to support the long- term continuation of pasireotide.