ACROSTUDY: Status Update on 469 Patients

Abstract

Background: ACROSTUDY is an international noninterventional surveillance study open to all patients with acromegaly treated with the growth hormone antagonist pegvisomant. This web-based registry contains data reflecting actual clinical care of patients, and it is being used to monitor the long-term safety and efficacy of pegvisomant therapy for acromegaly. Since ACROSTUDY was launched in 2004, there has been an increase in cumulative patient recruitment from 116 patients in 2005 to over 500 by mid-2008. As of May 2008, over 300 centers in 10 countries have contributed data from 469 patients. At the time of inclusion, 84% of patients had already been treated with pegvisomant. Of the 469 patients, the majority had received somatostatin analogue treatment (63% had received octreotide and 34% had received lanreotide); 20% reported no prior medical treatment. Overall, 74% had prior transsphenoidal surgery, 3% had undergone craniotomy and 35% had received radiation therapy (including stereotactic radiosurgery). Interestingly, 67% were treated with pegvisomant alone at study start, 4% took pegvisomant with a dopamine agonist, 26% with a somatostatin analogue and 3% took pegvisomant with both types of analogues. The starting dose of pegvisomant was 10 mg/day for 65% of patients treated with subcutaneous daily injections, and the mean insulin-like growth factor I (IGF-I) concentration at baseline was 526 ng/ml. Annual assessments since study launch in 2004 found 62–78% of patients had normal IGF-I levels after 1–4 years of pegvisomant treatment at mean doses of 18.7–22 mg/day. Adverse events were reported for 13% of patients, including 6 serious adverse events considered possibly related to the drug. An increase in tumor size relative to the size at start of pegvisomant therapy was reported for 24 of the 469 patients (5.1%). Liver function test results more than twice the upper limit of normal were observed in 7.7%. Conclusions: Further analyses of the ACROSTUDY database will provide a better understanding of pegvisomant treatment in clinical practice.