Brazilian multicenter study on pegvisomant treatment in acromegaly

• Autor(a) principal: Boguszewski, Cesar L.
• Data de Publicação: 2019
• Outros Autores: Huayllas, Martha Katherine P, Vilar, Lucio, Naves, Luciana Ansaneli, Ribeiro-Oliveira Junior, Antonio, Soares, Beatriz Santana, Czepielewski, Mauro Antonio, Abucham, Julio, Correa-Silva, Silvia Regina, Bronstein, Marcello Delano, Jallad, Raquel Soares, Duarte, Felipe Gaia, Musolino, Nina Rosa, Kasuki, Leandro, Gadelha, Monica Roberto
• Tipo de documento: Artigo
• Idioma: eng
• Título da fonte: Repositório Institucional da UnB
• Texto Completo: https://repositorio.unb.br/handle/10482/36696; https://doi.org/10.20945/2359-3997000000159; http://orcid.org/0000-0001-7285-7941; http://orcid.org/0000-0002-1042-5083; http://orcid.org/0000-0003-4815-6963; http://orcid.org/0000-0002-3363-3803; http://orcid.org/0000-0002-5686-7899; http://orcid.org/0000-0002-6114-5786; http://orcid.org/0000-0001-5083-5776; http://orcid.org/0000-0003-4804-1525; http://orcid.org/0000-0003-2405-0013; http://orcid.org/0000-0002-0113-5201; http://orcid.org/0000-0003-0477-1892; http://orcid.org/0000-0002-3495-1301; http://orcid.org/0000-0003-1562-4476; http://orcid.org/0000-0003-1339-3192; http://orcid.org/0000-0002-9250-3558
• topic: Acromegalia; Somatotropina; Pegvisomanto; Estudo multicêntrico

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.