Pegvisomant treatment in patients with acromegaly in clinical practice: The French ACROSTUDY

Objective. - To monitor long-term pegvisomant treatment of patients with acromegaly in routine clinical practice. Patients and methods. - The French ACROSTUDY is part of the global ACROSTUDY, an observational post-authorization safety surveillance study of acromegaly treatment with pegvisomant. Results. - The median duration of follow-up of the 292 included patients was 5.2 years. Overall 272 (93%) patients received somatostatin analogues before initiation of pegvisomant. The most prescribed initial dose of pegvisomant (after possible administration of a loading dose) was 10 mg/day and, starting from the 2nd year, the median dose was 20 mg/day. Serum IGF-1 concentration decreased as soon as pegvisomant was started and after 5 years there was a 62% mean decrease in serum IGF-1 concentration. The percentage of patients with serum IGF-1 concentration within normal ranges (for age and sex) of the local laboratory shifted from 11% at start of pegvisomant to 43% at 6 months and 63% after 5 years. The last available imaging (242 patients) showed an increased or decreased tumor size in 4 and 10% of patients, respectively. Mean weight increased by 3 kg over the 5-year period (P<10(-3)). Mean fasting blood glucose significantly decreased over time (P<0.05), while HbA1c level remained unchanged. Tolerance profile was generally good and similar to that described in clinical studies. Conclusion. - This analysis showed a significant decrease in IGF-1 levels throughout the follow-up period, and confirmed that pegvisomant treatment is safe in acromegaly. The results of this interim analysis remain to be confirmed by the final analysis. (C) 2015 Elsevier Masson SAS. All rights reserved.