Long-term efficacy and safety of pegvisomant in combination with long-acting somatostatin analogs in acromegaly

S. J.C.M.M. Neggers, S. E. Franck, F. W.M. De Rooij, A. H.G. Dallenga, R. M.L. Poublon, R. A. Feelders, J. A.M.J.L. Janssen, M. Buchfelder, L. J. Hofland, J. O.L. Jørgensen, A. J. Van Der Lely

Onderzoeksoutput: Bijdrage aan tijdschriftArtikelpeer review

99 Citaten (Scopus)


Background: Treatment for acromegaly patients with long-acting somatotropin release-inhibiting factor (LA-SRIF) often does not result in complete normalization of IGF-1. Addition of pegvisomant (PEGV), a GH receptor antagonist, could improve this; however, the literature has not described long-term follow-up.

Objective: To assess long-term efficacy and safety of this combined treatment in the largest current single-center cohort of patients, from 2004-2013.

Design: Acromegaly patients were treated for at least 6 months with a high-dose LA-SRIF. To patients with persistently elevated IGF-1 levels (>1.2 × upper limit of normal) or poor quality of life, PEGV was added as one weekly injection.

Results: The patients (n = 141) were treated with PEGV and LA-SRIFs for a median period of 4.9 years (range, 0.5-9.2). Efficacy, defined as the lowest measured IGF-1 level during treatment, was 97.0%. The median PEGV dose to achieve this efficacy was 80 mg weekly (interquartile range, 60-120 mg). Combination treatment-related adverse events were recorded in 26 subjects (18.4%). Pituitary tumor size increase was observed in one patient. Injection-site reactions were observed in four subjects. In 19 patients (13.5%), transiently elevated liver transaminases of more than three times the upper limit of normal were observed, of which 83% occurred within the first year of combination treatment. Eight patients died, at a mean age of 71 years; none of them were considered treatment-related.

Conclusions: The combination treatment with LA-SRIFs and PEGV was effective in 97% of the patients, it appears to be a safe medical treatment and it reduces the required dose of PEGV.

Originele taal-2Engels
Pagina's (van-tot)3644-3652
Aantal pagina's9
TijdschriftJournal of Clinical Endocrinology and Metabolism
Nummer van het tijdschrift10
StatusGepubliceerd - 1 okt. 2014
Extern gepubliceerdJa


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