Efficacy and safety of pasireotide treatment in acromegaly: A systematic review and single arm meta-analysis

Acromegaly is a rare chronic endocrine disorder associated with significant comorbidities. Many patients fail to achieve biochemical control with current medical therapies, including surgery and first-generation somatostatin ligands (fg-SRLs). We aimed to perform a systematic review and single-arm meta-analysis to evaluate the efficacy of the multi-receptor somatostatin ligand pasireotide in patients with active or uncontrolled acromegaly.

We systematically searched PubMed, Embase, and Cochrane databases for studies that assessed the efficacy of pasireotide in patients with acromegaly and reported the outcomes of (1) biochemical control and its composite indicators; (2) normalized IGF-1 level and (3) low GH level. For the statistical analysis, we used R software.

We included nine studies with a total of 590 patients: four clinical trials and five observational cohorts. 82.2% of the overall population consisted of inadequately controlled acromegaly patients. After a follow-up of 12 months, the pooled biochemical control rate was 26.50% (95% CI 14.87–42.66). The prevalence of normalized IGF-1 and low GH levels was 36.27% (95% CI 29.15–43.39) and 34.76% (95% CI 24.58–44.95), respectively. Additionally, biochemical response rates were sustained throughout the extension phase of these studies. In a pooled analysis including four studies with extension phase results, the prevalence of biochemical control rate was 29.03% (95% CI 11.49–46.58) with 76 events out of 281 patients. The most commonly reported adverse events were gastrointestinal disturbances in 31.26% (95% CI 7.44–72.01) and hyperglycemia in 29.55% (95% CI 21.80–37.29) of patients. The incidence of new-onset diabetes mellitus significantly increased after pasireotide treatment, with a rate of 23.36% (95% CI 19.58–27.13).

Pasireotide demonstrates biochemical control in patients with active or uncontrolled acromegaly. Although a high rate of hyperglycemic adverse events and diabetes mellitus related to the treatment were observed, most of them were manageable.