TY - JOUR
T1 - Long-term efficacy and safety of pegvisomant in combination with long-acting somatostatin analogs in acromegaly
AU - Neggers, S. J.C.M.M.
AU - Franck, S. E.
AU - De Rooij, F. W.M.
AU - Dallenga, A. H.G.
AU - Poublon, R. M.L.
AU - Feelders, R. A.
AU - Janssen, J. A.M.J.L.
AU - Buchfelder, M.
AU - Hofland, L. J.
AU - Jørgensen, J. O.L.
AU - Van Der Lely, A. J.
N1 - Publisher Copyright:
Copyright © 2014 by the Endocrine Society.
PY - 2014/10/1
Y1 - 2014/10/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=84907610860&partnerID=8YFLogxK
U2 - 10.1210/jc.2014-2032
DO - 10.1210/jc.2014-2032
M3 - Article
C2 - 24937542
AN - SCOPUS:84907610860
SN - 0021-972X
VL - 99
SP - 3644
EP - 3652
JO - Journal of Clinical Endocrinology and Metabolism
JF - Journal of Clinical Endocrinology and Metabolism
IS - 10
ER -