PMON66 A Systematic Literature Review to Evaluate Extended Dosing Intervals in the Pharmacological Management of Acromegaly

Abstract

Abstract Background Acromegaly is a rare, lifelong disorder characterized by excess growth hormone (GH) and insulin-like growth factor 1 (IGF-1). Extended dosing intervals (EDIs) of pharmacological treatments may reduce patient burden and costs compared with standard dosing. This systematic literature review (SLR) investigated outcomes in patients with acromegaly receiving treatment at EDIs. Methods MEDLINE/Embase/the Cochrane Library (2001–June 2021) and key congresses (2018–2021) were searched for relevant literature; identified SLR bibliographies were also reviewed. A dual reviewer process was utilized. Included publications reported clinical effectiveness, safety, humanistic, and economic outcomes in longitudinal/cross-sectional studies in adult patients with acromegaly. Interventions included EDIs of lanreotide autogel (LAN), octreotide long-acting release (OCT), and pasireotide (all administered less often than every four weeks), oral octreotide (less than twice daily), pegvisomant (PEG; less than once daily), and cabergoline (less than twice weekly), with no comparator required. PROSPERO 2021: CRD42021278922. Results In total, 35 publications reported on 27 studies: 14 on PEG, 9 on LAN, and 4 on OCT as monotherapies or combination therapies at EDIs. No identified studies assessed EDIs of oral octreotide, pasireotide, or cabergoline. At Baseline, disease control and treatment history differed across studies. As compared with Baseline, treatment at EDIs resulted in reduced IGF-1 levels in 12/16 studies assessing LAN, OCT, or PEG (7–104 patients treated at EDIs) and GH levels in 5/6 studies (LAN/OCT; 15–32 patients). Normalized IGF-1 and/or GH, using study definitions, was achieved/maintained in 70%–100% of patients in 12/13 studies (LAN/OCT/PEG; 15–124 patients). Proportions of patients experiencing adverse events (n=4 studies reporting overall events; LAN/OCT/PEG; 8–96 patients) or discontinuing treatment (n=9; LAN/OCT/PEG; 7–124 patients) were similar across EDI and standard regimens. Health-related quality of life (HRQoL) improvement from Baseline was reported in 9/10 studies after treatment at EDIs (LAN/OCT/PEG; 7–109 patients); HRQoL did not deteriorate versus standard regimens. Patients preferred EDIs (2/2 studies; LAN/OCT; 8–112 patients), and satisfaction with EDIs was high (2/2 studies; LAN/OCT; 13–109 patients). Substantially reduced costs were observed with EDIs versus standard regimens in 3/3 studies (9–23 patients) with LAN (120 mg every 6 versus 4 weeks), OCT (20–30 mg every 6–12 versus 4 weeks), and PEG (40–160 mg weekly versus 15–60 mg daily, both with LAN/OCT). Conclusion Clinical, safety, and HRQoL outcomes in patients with acromegaly were similar and costs lower with EDIs versus standard regimens. Satisfaction was high with EDIs. Limitations, including variation in study design and endpoints, and small, heterogenous patient populations, precluded conducting meta-analyses. Physicians should consider EDIs where appropriate. Further clinical research may substantiate reduced patient and healthcare system burden with EDIs versus standard regimens. Study funded by Ipsen. Medical writing support provided by Costello Medical. Presentation: Monday, June 13, 2022 12:30 p.m. - 2:30 p.m.