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.

Keywords:
Growth hormone, Acromegaly, Pegvisomant, Somatostatin analogues, Pituitary surgery, Observational study
1.
Colao A, Pivonello R, Auriemma RS, De Martino MC, Bidlingmaier M, Briganti F, Tortora F, Burman P, Kourides IA, Strasburger CJ, Lombardi G: Efficacy of 12-month treatment with the GH receptor antagonist pegvisomant in patients with acromegaly resistant to long-term, high-dose somatostatin analog treatment: effect on IGF-I levels, tumor mass, hypertension and glucose tolerance. Eur J Endocrinol 2006;154:467–477.
2.
Trainer PJ, Drake WM, Katznelson L, Freda PU, Herman-Bonert V, van der Lely AJ, Dimaraki EV, Stewart PM, Friend KE, Vance ML, Besser GM, Scarlett JA, Thorner MO, Parkinson C, Klibanski A, Powell JS, Barkan AL, Sheppard MC, Malsonado M, Rose DR, Clemmons DR, Johannsson G, Bengtsson BA, Stavrou S, Kleinberg DL, Cook DM, Phillips LS, Bidlingmaier M, Strasburger CJ, Hackett S, Zib K, Bennett WF, Davis RJ: Treatment of acromegaly with the growth hormone-receptor antagonist pegvisomant. N Engl J Med 2000;342:1171–1177.
3.
van der Lely AJ, Hutson RK, Trainer PJ, Besser GM, Barkan AL, Katznelson L, Klibanski A, Herman-Bonert V, Melmed S, Vance ML, Freda PU, Stewart PM, Friend KE, Clemmons DR, Johannsson G, Stavrou S, Cook DM, Phillips LS, Strasburger CJ, Hackett S, Zib KA, Davis RJ, Scarlett JA, Thorner MO: Long-term treatment of acromegaly with pegvisomant, a growth hormone receptor antagonist. Lancet 2001:358:1754–1759.
4.
Trainer PJ: ACROSTUDY: an overview. Horm Res 2007;68(suppl 5):68–69.
5.
Strasburger CJ, Buchfelder M, Droste M, Mann K, Stalla GK, Saller B: Experience from the German Pegvisomant Observational Study. Horm Res 2007;68(suppl 5):70–73.
6.
Schreiber I, Buchfelder M, Droste M, Forssmann K, Mann K, Saller B, Strasburger CJ: Treatment of acromegaly with the GH receptor antagonist pegvisomant in clinical practice: safety and efficacy evaluation from the German Pegvisomant Observational Study. Eur J Endocrinol 2007;156:75–82.
© 2009 S. Karger AG, Basel
2009
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