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Long-term treatment with pegvisomant for acromegaly: a 10-year experience.

Abstract：

BACKGROUND:Efficacy of the GH-receptor antagonist pegvisomant (PEG) has differed between preclinical and observational studies mainly due to dose adjustment and IGF-I normalization criteria. An escape phenomenon has also been described, but its definition and underlying causes have not been fully established. OBJECTIVE:To re-evaluate the outcomes of long-term PEG in a series of previously published patients and analyse the escape phenomenon. METHODS:We reviewed all patients with acromegaly resistant to SSA in whom PEG was started as monotherapy, who had been included in a previous publication. We prospectively evaluated 64 (56·3% women) from six tertiary care referral hospitals in Spain, for whom data as of June 2014 were available. Escape to PEG was defined as confirmed loss of biochemical control (IGF-I >1·2xULN), after at least 6 months of previous control with a stable dose of PEG. RESULTS:Patients were followed up for 13·0 (5·9-34·8) years since diagnosis, and 9·0 (4·1-10·4) years since the first administration of PEG. Fifty-one (89·5%) patients had an adequate IGF-I control at the last follow-up visit, 9 of them without treatment. Tumour growth was reported in 6 of 64 cases (9·4%), none of whom had received prior radiotherapy (P = 0·011). Seven patients died during follow-up. We found 16 escapes in 10 patients (15·6%). We identified potential underlying causes in 9 cases (tumour regrowth, previous treatment modifications, concomitant menopause and change in testosterone administration). The reason was unknown in 7 escapes, which occurred in 6 patients (9·4%). All patients, except one, achieved subsequent biochemical control after treatment adjustment. CONCLUSIONS:We reassure the efficacy and safety of long-term PEG. An escape phenomenon may occur, but it can be overcome by adjusting therapy.

journal_name

Clin Endocrinol (Oxf)

journal_title

Clinical endocrinology

authors

Ramos-Leví AM,Bernabeu I,Álvarez-Escolá C,Aller J,Lucas T,de Miguel P,Rodríguez-Cañete L,Sampedro-Núñez MA,Halperin I,Puig-Domingo M,Marazuela M

doi

10.1111/cen.12993

subject

Has Abstract

pub_date

2016-04-01 00:00:00

pages

540-50

issue

eissn

0300-0664

issn

1365-2265

journal_volume

pub_type

杂志文章,多中心研究

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Long-term treatment with pegvisomant for acromegaly: a 10-year experience.