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.

Em homens, macroprolactinomas predominam em relação aos microprolactinomas e têm maior invasividade que nas mulheres. O tratamento clínico é a primeira opção tanto em macro como em microadenomas, independente do sexo. Comparamos apresentação clínica, níveis de prolactina, invasividade neurorradiológica e resposta da prolactinemia em 42 homens com prolactinomas, 23 com terapia clínica (grupo 1) e 19 que também utilizaram tratamento cirúrgico e/ou radioterápico (grupo 2). Os dados obtidos foram submetidos a análise estatística utilizando-se os testes de qui-quadrado ou exato de Fisher para comparações de proporções. Para comparar médias foi aplicado o teste t de Student ou, na ausência de distribuição normal ou com número pequeno de eventos, o não paramétrico de Mann-Whitney. O nível de significância adotado foi de 5% (p<0,05). Os grupos foram similares para idade (p=0,23), período entre primeiro sintoma e diagnóstico (p=0,82), níveis de prolactina pré tratamento (p=0,41) e proporção de macroadenomas invasivos (p=0,096). Ocorreu percentual significativamente maior de cefaléia (p=0,009), deficit visual (p=0,025), tempo de terapia (p=0,007) e período de acompanhamento (p=0,0005) no grupo 2. Variações dos níveis de prolactina antes e após terapia não apresentaram diferença significativa nos grupos (p=0,49), nem o percentual de normalização da prolactina após tratamento (p=0,20). Concluímos, enfatizando a importância do diagnóstico precoce do prolactinoma em homens, tendo em vista a morbidade demonstrada, e reforçamos a necessidade do uso do agonista dopaminérgico como opção terapêutica inicial independente do tamanho do adenoma.

Macroprolactinomas predominate in males in comparison to microprolactinomas, with greater trend to invasiveness than in females. The clinical treatment has been the first option to prolactinomas, in both macro and microadenomas, irrespective the sex. We compared clinical presentation, prolactin levels, neuroradiologic invasiveness and prolactinemia response of 23 men with prolactinomas subjected to clinical therapy (group 1) with 19 who went also through surgical and/or radiotherapic treatment (group 2). The statistical analysis was done by the tests of chi-square or exact of Fisher, in order to compare proportions, and by t of Student or Mann-Whitney, in order to compare means. The level of significance adopted was 5% (p<0.05). The two groups were similar regarding age (p=0.23), period between start of the first symptom and diagnosis (p=0.82), prolactin levels before treatment (p=0.41) and invasive macroadenomas proportion (p=0.096). There was significantly greater percentage of headache (p=0.009) and visual deficit (p=0.025) in group 2, as well as the drug usage (p=0.007) and observation (p=0.0005) periods were superior in this group. The variations of prolactin levels before and after therapy (p=0.49) as well as the percentage of prolactin normalization (p=0.20) did not show any significant difference when comparing the two groups. We conclude, emphasizing the relevance of precocious prolactinoma diagnostic in men, because of the demonstrated morbidity. We strengthen the use of dopamine agonist as the first therapeutic option irrespective the adenoma size.