Endocrine Abstracts

Introduction: Pegvisomant (PEG) is an efficient treatment for acromegaly but the recommendation for this treatment is less active due to its high cost. In Romania it is reimbursed in doses up to 30 mg s.c/day or as 40 or 80 mg/week in combination with a somatostatin analog (SSA).

Design: Retrospective analysis of 18 consecutive patients treated with PEG for acromegaly (either as monotherapy, or in combination with SSA and/or cabergoline (CAB), or both consecutively. All patients had a macroadenoma, underwent surgery, all had residual disease, 17 underwent radiotherapy. In the study were included 12 women, 6 men, mean age 44.5 years old (range: 19–70). Before commencing PEG, patients had an elevated IGF‐I level: mean 875.3 ± 397.3 ng/ml. The mean duration of the PEG treatment was 3 ± 1.6 years.

Results: 14 patients had PEG in monotherapy, mean dose 16 ± 5.2 mg/day (112 mg/week). From these, 8 normalised IGF1, 2 of them were disease free after RT and PEG therapy. In 4 patients optimal response was not achieved with 30 mg PEG/day. From 6 patients with combined therapy: SSA + PEG or SSA + PEG + CAB (mean PEG dose 58.6 ± 24 mg/week), 2 patients normalised IGF1 (33%). Adding CAB decreased IGF1 levels but did not normalise them. In the whole group 4 patients (22%) had side effects: a tumor increase with 2.5–8 mm in 2 patients, after 8 and 24 months, respectively, and elevated transaminases in 2 patients (1 of these tolerated a lower PEG dose + SSA, the other one stopped PEG therapy).

Conclusions: Pegvisomant is an effective treatment for acromegaly (in 55% in our series), if the dose is well titrated and tolerated during treatment. Special attention is needed for the potential side effects (noted in 22% of our series).