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Brazilian multicenter study on pegvisomant treatment in acromegaly

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...

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Autores principales: Boguszewski, Cesar L., Huayllas, Martha Katherine P, Vilar, Lucio, Naves, Luciana Ansaneli, Ribeiro-Oliveira, 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Sociedade Brasileira de Endocrinologia e Metabologia 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10528655/
https://www.ncbi.nlm.nih.gov/pubmed/31365632
http://dx.doi.org/10.20945/2359-3997000000159
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author Boguszewski, Cesar L.
Huayllas, Martha Katherine P
Vilar, Lucio
Naves, Luciana Ansaneli
Ribeiro-Oliveira, 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
author_facet Boguszewski, Cesar L.
Huayllas, Martha Katherine P
Vilar, Lucio
Naves, Luciana Ansaneli
Ribeiro-Oliveira, 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
author_sort Boguszewski, Cesar L.
collection PubMed
description 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.
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spelling pubmed-105286552023-09-28 Brazilian multicenter study on pegvisomant treatment in acromegaly Boguszewski, Cesar L. Huayllas, Martha Katherine P Vilar, Lucio Naves, Luciana Ansaneli Ribeiro-Oliveira, 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 Arch Endocrinol Metab Original Article 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. Sociedade Brasileira de Endocrinologia e Metabologia 2019-07-11 /pmc/articles/PMC10528655/ /pubmed/31365632 http://dx.doi.org/10.20945/2359-3997000000159 Text en https://creativecommons.org/licenses/by/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Original Article
Boguszewski, Cesar L.
Huayllas, Martha Katherine P
Vilar, Lucio
Naves, Luciana Ansaneli
Ribeiro-Oliveira, 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
Brazilian multicenter study on pegvisomant treatment in acromegaly
title Brazilian multicenter study on pegvisomant treatment in acromegaly
title_full Brazilian multicenter study on pegvisomant treatment in acromegaly
title_fullStr Brazilian multicenter study on pegvisomant treatment in acromegaly
title_full_unstemmed Brazilian multicenter study on pegvisomant treatment in acromegaly
title_short Brazilian multicenter study on pegvisomant treatment in acromegaly
title_sort brazilian multicenter study on pegvisomant treatment in acromegaly
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10528655/
https://www.ncbi.nlm.nih.gov/pubmed/31365632
http://dx.doi.org/10.20945/2359-3997000000159
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