5 years later - overview of the data collected in ACROSTUDY - different treatment regimens, database structure, basic strategies and safety.

Abstract

ACROSTUDY is an observational registry intended to collect safety and efficacy data on pegvisomant therapy. 792 patients have been enrolled, of whom 83% had commenced pegvisomant prior to recruitment. The mean follow-up is 1.66 years with the mean duration of pegvisomant therapy 3.31 years representing 2625 patient years of treatment. 90% of patients were on once daily pegvisomant, and 67% were on monotherapy. Disappointingly IGF-I was normalised in less than 70% of patients, furthermore in 80% of patients with an elevated IGF-I the daily dose of pegvisomant was 20 mg or less. 56 Serious Adverse Events (SAEs) were reported, 13 of which were related to pegvisomant. 276 Adverse Events (AEs) were reported, of which 56 were considered related to pegvisomant. The AEs most frequently attributed to pegvisomant were disturbed liver function tests and injection site reactions. MRI imaging was available in 684 patients. 411 patients had at least one MRI on pegvisomant compared to a baseline. In 31 patients a decrease in tumour size has been reported, of whom 20 had previously received radiotherapy. An increase in tumour size has been reported and confirmed in 22 patients. In 11 patients there was contradictory data on tumour size, while in 6 patients central review of the films failed to confirm increase in tumour size. In conclusion the safety data are generally reassuring while the IGF-I normalisation rate is disappointing which probably reflects a failure of dose titration. Further effort is needed to understand the reasons for the failure of dose titration.