070723 cp perinopril-us

Perindopril reduces mortality in Duchenne myopathy A team of researcher-clinicians coordinated by Professor Denis Duboc (Cochin Hospital, AP-HP, René Descartes University Paris V) and Doctor Henri-Marc Bécane (Myology Institute, AFM) have published the results of a study into the 10-year follow-up of children affected with Duchenne myopathy treated with perindopril. The study shows a significant reduction of mortality in these children. Moreover, the results seem to underline perindopril’s overall role of protector, particularly of the muscle function. Supported by the AFM thanks to Téléthon donations, this work is published in the American Heart Journal. It integrates into the AFM-backed clinical research activities for better patient management as well as research for the development of innovative therapies. The results – published in the review American Heart Journal – are the fruit of a very long-term study: 10 years of following up the administration of an angiotensin converting enzyme inhibitor (perindopril) in cardiomyopathy prevention. In Duchenne myopathy the cardiac muscle is inevitably affected, and is responsible for the fatal outcome in approximately half the cases. Thus, fighting this cardiac damage is essential in maintaining patients’ life expectancy. It was in this context that this long-term clinical research project was undertaken in 10 clinical centres in France. The study involved 57 children aged from 10 to 13 years and lasted a total of ten years, of which three were in double blind against placebo. A first group of 28 patients were given daily treatment with perindopril and a second group of 29 patients a placebo. In 2005 the first results of the follow-up at the 5-year point in the clinical study showed the preventive role of perindopril for the appearance of cardiac muscle failure. At the end of the study it was thus shown that perindopril reduced the mortality of these children – in the treatment group 26 out of the 28 patients are still alive as against 19 out of 29 in the control group. As well as its role of protector of the cardiac muscle, perindopril seems also to have an effect on the diaphragm and the inter-costal muscles. The earlier perindopril treatment is begun, the more this protection is effective. Moreover, according to a very recent study carried out on an animal model, the blocking of angiotensin 2 action – the pharmalogical target of perindopril – has an anti-fibrosant effect on the muscle as well as its effect on cardiac hemodynamics. This confirms the relevance of the forthcoming therapeutic trial with perindopril on much younger children as yet unaffected by fibrosis. This trial is supported by the AFM and Servier Laboratories. This class of drugs is usually used to treat high blood pressure and heart failure, but curatively – in other words, once the dysfunction is certified. The results published today give weight to the recommendation – made by the AFM and Professor Denis Duboc to the specialist authorities – that perindopril be used preventively on children from the age of 10 years. The objective of the clinical research supported by the AFM is to improve the medical management of patients at the respiratory, cardiac and orthopaedic levels. The research also optimises treatments through a better understanding of the physiopathological mechanisms of the disease. Thanks to this support over the last 20 years, more than ten years’ life expectancy has been gained for Duchenne myopathy patients. Further information: Périndopril preventive treatment on mortality in Duchenne muscular dystrophy : 10 years follow-up. Denis Duboc1, Christophe Meune1, Bertrand Pierre1, Karim Wahbi1, Bruno Eymard2, Annick Toutain3, Carole Berard4, Guy Vaksmann5, Simon Weber1, Henri-Marc Becane2. American Heart Journal (on line). 1 Department of Cardiology, Cochin Hospital, AP-HP, Paris V René Descartes University, Paris. 2 Myology Institute, Pitié-Salpêtrière Hospital, Paris. 3 Department of Genetics, Bretonneau University Hospital, Tours. 4 Department of Pediatric rehabilitation, Lyon-Sud Hospital, Lyon. 5 Department of Pediatrics, Cardiology Hospital, Lille. More information about the disease: Association Française contre les Myopathies www.afm-france.org Tél. : 0 810 811 088 (local call rate) Researcher contacts: Hôpital Cochin (AP-HP) Denis Duboc, Karim Wahbi - 01 58 41 16 52 Press contacts: AFM – Delphine Carvalho, Mathilde Maufras, 01 69 47 28 28, presse@afm.genethon.fr AP-HP – Service de presse, 01 40 27 37 22, service.presse@sap.aphp.fr

Source: http://www.institut-myologie.org/anglais/e_upload/pdf/070723_cp_perinopril-us.pdf

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