Project description:Purpose This systematic literature review investigated whether extended dosing intervals (EDIs) of pharmacological acromegaly treatments reduce patient burden and costs compared with standard dosing, while maintaining effectiveness. Methods MEDLINE/Embase/the Cochrane Library (2001–June 2021) and key congresses (2018–2021) were searched and identified systematic literature review bibliographies reviewed. Included publications reported on efficacy/effectiveness, safety and tolerability, health-related quality of life (HRQoL), and patient-reported and economic outcomes in longitudinal/cross-sectional studies in adults with acromegaly. Interventions included EDIs of pegvisomant, cabergoline, and somatostatin receptor ligands (SRLs): lanreotide autogel/depot (LAN), octreotide long-acting release (OCT), pasireotide long-acting release (PAS), and oral octreotide; no comparator was required. Results In total, 35 publications reported on 27 studies: 3 pegvisomant monotherapy, 11 pegvisomant combination therapy with SRLs, 9 LAN, and 4 OCT; no studies reported on cabergoline, PAS, or oral octreotide at EDIs. Maintenance of normal insulin-like growth factor I (IGF-I) was observed in ≥ 70% of patients with LAN (1 study), OCT (1 study), and pegvisomant monotherapy (1 study). Achievement of normal IGF-I was observed in ≥ 70% of patients with LAN (3 studies) and pegvisomant in combination with SRLs (4 studies). Safety profiles were similar across EDI and standard regimens. Patients preferred and were satisfied with EDIs. HRQoL was maintained and cost savings were provided with EDIs versus standard regimens. Conclusions Clinical efficacy/effectiveness, safety, and HRQoL outcomes in adults with acromegaly were similar and costs lower with EDIs versus standard regimens. Physicians may consider acromegaly treatment at EDIs, especially for patients with good disease control. Supplementary Information The online version contains supplementary material available at 10.1007/s11102-022-01285-1. Plain language summary Acromegaly is a rare disease where the body makes too much growth hormone. It is usually caused by a benign pituitary tumor. Excess growth hormone causes body parts such as the hands, feet, head, and heart to grow larger than normal. Organs including the heart can work less well, leading to other health conditions. Treatment aims to improve signs and symptoms, decrease tumor size, and normalize growth hormone levels. However, if surgery does not cure acromegaly, it can require ongoing, life-long treatment. Treatment can cause frequent side effects and impact daily life. Increasing time between medication doses could ease the burden of acromegaly treatment and reduce costs. We searched scientific literature for evidence on less frequent dosing (known as extended dosing intervals) when treating acromegaly, investigating efficacy/effectiveness, safety, well-being, and costs. We searched for evidence on treatments including: lanreotide autogel/depot, octreotide long-acting release, oral octreotide, pasireotide long-acting release, cabergoline, and pegvisomant. In people with stable acromegaly at standard dosing of lanreotide autogel, octreotide long-acting release, and pegvisomant, less frequent dosing generally did not compromise treatment effectiveness and kept growth hormones at normal levels. Furthermore, monthly treatments with lanreotide autogel or octreotide long-acting release alone are sometimes not enough to normalize hormone levels. In these cases, adding pegvisomant with less frequent dosing generally helped to stabilize disease. Generally, patient quality of life did not deteriorate with less frequent dosing, and satisfaction remained high. Furthermore, patients preferred less frequent dosing compared to standard dosing, which was also shown to reduce treatment costs.
| S-EPMC9708130 | biostudies-literature
