Philippe Chanson1, Thierry Brue2, Brigitte Delemer3, Philippe Caron4, Françoise Borson-Chazot5, Hichem Zouater6. 1. Université Paris-Saclay, Université Paris-Sud, Unité Mixte de Recherche S1185, Faculté de Médecine Paris-Sud, 63, rue Gabriel-Péri, 94276 Le Kremlin-Bicêtre, France; Service d'endocrinologie et des maladies de la reproduction, Assistance Publique-Hôpitaux de Paris, hôpital de Bicêtre, 78, rue du Général-Leclerc, 94275 Le Kremlin-Bicêtre, France. Electronic address: philippe.chanson@bct.aphp.fr. 2. Unité Mixte de Recherche 7286, Aix-Marseille University, Centre de Recherche en Neurobiologie et Neurophysiologie de Marseille (CRN2M), Centre National de la Recherche Scientifique, Faculté de Médecine de Marseille, 13284 Marseille, France; Department of Endocrinology, Centre de Référence des Maladies Rares d'Origine Hypophysaire, Assistance Publique-Hôpitaux de Marseille (APHM), Hôpital de la Conception, 13005 Marseille, France. 3. Service d'endocrinologie, diabète et nutrition, Hôpital Robert-Debré, CHU de Reims, avenue du Général-Koenig, 51092 Reims Cedex, France. 4. Service d'Endocrinologie, Maladies métaboliques et Nutrition, Pôle Cardio-Vasculaire et Métabolique, CHU Larrey, 24, chemin de Pouvourville, 31059 Toulouse cedex 09, France. 5. Hospices Civils de Lyon, Fédération d'Endocrinologie du pôle Est, Lyon, France. 6. Pfizer France, Paris, France.
Abstract
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 10mg/day and, starting from the 2nd year, the median dose was 20mg/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.
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 10mg/day and, starting from the 2nd year, the median dose was 20mg/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.
Authors: M Ragonese; S Grottoli; P Maffei; A Alibrandi; M R Ambrosio; G Arnaldi; A Bianchi; S Puglisi; M C Zatelli; L De Marinis; E Ghigo; A Giustina; F Maffezzoni; C Martini; L Trementino; S Cannavo Journal: J Endocrinol Invest Date: 2017-10-28 Impact factor: 4.256
Authors: Maria Fleseriu; Dagmar Führer-Sakel; Aart J van der Lely; Laura De Marinis; Thierry Brue; Joli van der Lans-Bussemaker; Judith Hey-Hadavi; Cecilia Camacho-Hubner; Michael P Wajnrajch; Srinivas Rao Valluri; Andrew Anthony Palladino; Roy Gomez; Roberto Salvatori Journal: Eur J Endocrinol Date: 2021-08-27 Impact factor: 6.664