Brazilian multicenter study on pegvisomant treatment in acromegaly

Abstract

Objective: Investigate the therapeutic response of acromegaly patients to pegvisomant (PEGV) in a real-life, Brazilian multicenter study. Subjects and methods: Characteristics of acromegaly patients treated with PEGV were reviewed at diagnosis, just before and during treatment. All patients with at least two IGF-I measurements on PEGV were included. Efficacy was defined as any normal IGF-I measurement during treatment. Safety data were reviewed. Predictors of response were determined by comparing controlled versus uncontrolled patients. Results: 109 patients [61 women; median age at diagnosis 34 years; 95.3% macroadenomas] from 10 Brazilian centers were studied. Previous treatment included surgery (89%), radiotherapy (34%), somatostatin receptor ligands (99%), and cabergoline (67%). Before PEGV, median levels of GH, IGF-I and IGF-I % of upper limit of normal were 4.3 µg/L, 613 ng/mL, and 209%, respectively. Pre-diabetes/diabetes was present in 48.6% and tumor remnant in 71% of patients. Initial dose was 10 mg/day in all except 4 cases, maximum dose was 30 mg/day, and median exposure time was 30.5 months. PEGV was used as monotherapy in 11% of cases. Normal IGF-I levels was obtained in 74.1% of patients. Glycemic control improved in 56.6% of patients with pre-diabetes/diabetes. Exposure time, pre-treatment GH and IGF-I levels were predictors of response. Tumor enlargement occurred in 6.5% and elevation of liver enzymes in 9.2%. PEGV was discontinued in 6 patients and 3 deaths unrelated to the drug were reported. Conclusions: In a real-life scenario, PEGV is a highly effective and safe treatment for acromegaly patients not controlled with other therapies.

Pegvisomant in Acromegaly and Gigantism

“…In the phase III study, injection site reactions were reported in six patients (5%) receiving pegvisomant [11]. In the Brazilian real-life study, it was reported in 4.6% of patients [14]. Pain from injection was reported in 2.7% patients.…”

“…In the phase III study, one patient with 15 mg/day of pegvisomant withdrew due to persistent headaches [11]. In the Brazilian real-life study, headaches were reported in two cases (1.8%) [14].…”

“…IGF1 normalisation was obtained in 74% of patients with a median dose of 15 mg/day when used alone and 10 mg/day in combined therapy. In this study, there were three response predictors: exposure time, GH pretreatment, and IGF1 pretreatment ( Table 1) [14].…”

“…In the Brazilian and Argentinian cohort, elevation of liver enzymes was reported in 9% of cases and was responsible for pegvisomant discontinuation in 1 and 5 patients, respectively ( Table 1) [14,33].…”

Pegvisomant for acromegaly: does it always works?

“…T wo studies published in this present issue of the Archives of Endocrinology and Metabolism, by Natalia Garcia Basavilbaso (1) and coworkers, as well as the one by Cesar Boguszewski and coworkers (2), describe the treatment response in real-life of pegvisomant (PEGV) therapy in acromegaly patients, including aspects of safety and efficacy.…”