Real world data in acromegaly a retrospective chart audit

Program: Abstracts - Orals, Featured Poster Presentations, and Posters
Session: SUN 281-290-Comparative Effectiveness/Health Outcomes/Quality Improvement/Patient or Provider Education/Endocrine Emergencies
Sunday, June 16, 2013: 1:45 PM-3:45 PM
Expo Halls ABC (Moscone Center)

Poster Board SUN-283
Diego Ferone*1, Anna Forsythe2, William Henry Ludlam2, Roberta Rondena3, Cherry T. Thomas2 and Monica Gadelha4
1Centre of Excellence for Biomedical Research, University of Genoa, Genoa, Italy, 2Novartis Pharmaceuticals Corporation, East Hanover, NJ, 3Novartis Farma SpA, Italy, 4Federal University of Rio de Janeiro, Rio de Janeiro, Brazil
Introduction: The ideal strategy and the role of the different therapeutic options in acromegaly remain unclear. This research focuses on observing treatment options, and the respective level of disease control in acromegaly.

Methods: A retrospective chart audit was conducted in US, France, Italy, and Brazil (September 2012). 97 endocrinologists completed structured case report forms for the 4 most recently-seen patients (2 with somatostatin analogues [SSAs], 2 with pegvisomant [PegV] or PegV+SSAs), N=380; those on the last choice of medical therapy ≥6 months were included in the analysis (N=335). Patient demographics, comorbidities, treatment history, insulin-like growth factor type 1 (IGF-1) and symptoms were recorded. Control of IGF-1 was defined as IGF-1≤1ULN,

Results: Of the 335 medically treated patients (mean age 51 yrs; 52% men), 193 (57.6%) were treated with SSAs, 116 (34.6%) with PegV, and 26 (7.8%) with PegV+SSAs. 208 of 335 patients (62%) were treated with surgery prior to medical therapy (74% US, 63% Brazil, 54% France, 51% Italy), 127 (38%) received medical therapy with (n=6) or without (n=121) radiation. 124 (37%) of all study patients had IGF-1>ULN (44% US, 48% Brazil, 31% France, 41% Italy); 67 (20%) had IGF-1>1.5xULN (19% US, 28% Brazil, 21% France, 15% Italy) despite treatments. 63 of 208 (30%) patients treated with surgery prior to medical therapy and 61 of 127 (48%) treated with medical therapy had IGF-1>ULN. 59% of SSA-treated patients had IGF-1≤ULN vs. 67% of patients on PegV, and 73% of patients on PegV+SSAs. The rates of acromegaly symptoms and comorbidities were similar among patients treated with SSAs and PegV (overall 65% hypertension, 31% type 2 diabetes, 23% arthritis, 48% headache, 51% fatigue, 43% joint pain, 15% paresthesia and 24% perspiration). The rates of acromegaly symptoms were higher in patients with IGF-1>ULN: 50% vs.46% headache, 53% vs. 49% fatigue, 47% vs. 41% joint pain, 23% vs. 10% paresthesia* and 32% vs. 19% perspiration (*p<0.05).

Conclusions: Medical therapy remains an integral component of acromegaly management. Despite the different therapeutic strategies, including treatment with different drugs with stable dose for at least 6 months, 37% of all medically treated patients remain with  IGF-1>ULN.

Disclosure: DF: Medical Advisory Board Member, Novartis Pharmaceuticals, Medical Advisory Board Member, Eli Lilly & Company, Speaker, Novartis Nutrition, Inc., Speaker, Ipsen. AF: Employee, Novartis Pharmaceuticals. WHL: Employee, Novartis Pharmaceuticals. RR: Employee, Novartis Pharmaceuticals. CTT: Employee, Novartis Pharmaceuticals. MG: Medical Advisory Board Member, Novartis Pharmaceuticals, , Novartis Pharmaceuticals, Investigator, Novartis Pharmaceuticals, Investigator, Pfizer, Inc., Speaker, Pfizer, Inc., Speaker, Ipsen.

*Please take note of The Endocrine Society's News Embargo Policy at

Sources of Research Support: This study was sponsored by Novartis Pharmaceuticals Corporation