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Long-term treatment of acromegalic patients resistant to somatostatin analogues with the GH receptor antagonist pegvisomant: its efficacy in relation to gender and previous radiotherapy

Endocrinology Divisions¹ Hospital Universitario de la Princesa, 28006 Madrid² Hospital Universitario Puerta de Hierro, 28222 Majadahonda³ Hospital Universitario La Paz, 28046 Madrid, Universidad Autónoma de Madrid, Spain⁴ Hospital Clinic, Universidad de Barcelona, 8036 Barcelona, Spain⁵ Hospital Clinico San Carlos, 28040 Madrid, Universidad Complutense de Madrid, Spain⁶ Complejo Hospitalario Universitario de Santiago de Compostela, Universidad de Santiago de Compostela, 15706 Santiago de Compostela, Spain⁷ Centro de Investigación Biomédica en Red, área temática Fisiopatología Obesidad y Nutrición (CiberObn), Instituto de Salud Carlos III, 15706 Santiago de Compostela, Spain

(Correspondence should be addressed to M Marazuela; Email: mmarazuela.hlpr{at}madrid.salud.org)

Context: Pegvisomant is an effective treatment for somatostatin analogue-resistant acromegaly, but the determinants defining the response to this treatment are largely unknown.

Objective: To investigate the efficacy of pegvisomant treatment in resistant acromegalic patients (e.g. serum IGF1 at least 1.25xupper normal limit) in a clinical setting and the factors conditioning this response.

Design and setting: A retrospective cross-sectional study performed in six Spanish University hospitals from 2004 to 2007.

Patients: Forty-four acromegalic patients (61.4% female, mean age: 49±14), 95% of whom had undergone pituitary surgery and 61% having received pituitary radiotherapy. The mean follow-up was 22.7±11.2 months.

Main outcome measures: IGF1 levels reflected treatment efficacy, and the influence of gender, age, weight, previous radiotherapy and duration of treatment was assessed.

Results: IGF1 normalisation was achieved in 84% of the patients. Male gender (P<0.05), previous irradiation (P<0.05) and the treatment duration (r=0.364, P<0.02) were associated with a better response to pegvisomant therapy. There was a significant decrease in HbA1c (P<0.001) and in the mean insulin dose (P<0.01) in acromegalic diabetic patients. Although 25% of patients experienced mild adverse events, pegvisomant was only withdrawn in four patients due to side effects (two cases of tumour growth, one liver dysfunction and one headache).

Conclusions: Long-term pegvisomant is a very effective therapy in resistant acromegaly. Male gender and prior radiotherapy influence the therapeutic response rate.

This article has been cited by other articles:

				I. Bernabeu, C. Alvarez-Escola, C. Quinteiro, T. Lucas, M. Puig-Domingo, M. Luque-Ramirez, P. de Miguel-Novoa, E. Fernandez-Rodriguez, I. Halperin, L. Loidi, et al. The Exon 3-Deleted Growth Hormone Receptor Is Associated with Better Response to Pegvisomant Therapy in Acromegaly J. Clin. Endocrinol. Metab., January 1, 2010; 95(1): 222 - 229. [Abstract] [Full Text] [PDF]

				T. Brue, F. Castinetti, F. Lundgren, M. Koltowska-Haggstrom, P. Petrossians, and on behalf of all ACROSTUDY investigators Which patients with acromegaly are treated with pegvisomant? An overview of methodology and baseline data in ACROSTUDY Eur. J. Endocrinol., November 1, 2009; 161(suppl_1): S11 - S17. [Abstract] [Full Text] [PDF]