title: Recommendations for the conduct of clinical trials for drugs to treat or prevent sarcopenia.
abstract: OBJECTIVE
  Sarcopenia is an age-related muscle condition which is frequently a precursor of frailty, mobility disability and premature death. It has a high prevalence in older populations and presents a considerable social and economic burden. Potential treatments are under development but, as yet, no guidelines support regulatory studies for new drugs to manage sarcopenia. The objective of this position paper is therefore to suggest a set of potential endpoints and target population definitions to stimulate debate and progress within the medico-scientific and regulatory communities.
  
  
  METHODS
  A multidisciplinary expert working group was hosted by the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis, which reviewed and discussed the recent literature from a perspective of clinical experience and guideline development. Relevant parallels were drawn from the development of definition of osteoporosis as a disease and clinical assessment of pharmaceutical treatments for that indication.
  
  
  RESULTS
  A case-finding decision tree is briefly reviewed with a discussion of recent prevalence estimations of different relevant threshold values. The selection criteria for patients in regulatory studies are discussed according to the aims of the investigation (sarcopenia prevention or treatment) and the stage of project development. The possible endpoints of such studies are reviewed and a plea is made for the establishment of a core outcome set to be used in all clinical trials of sarcopenia.
  
  
  CONCLUSIONS
  The current lack of guidelines for the assessment of new therapeutic treatments for sarcopenia could potentially hinder the delivery of effective medicines to patients at risk.
authors.affiliation: Department of Public Health, Epidemiology and Health Economics, University of Liege, CHU Sart Tilman B23, 4000, Liège, Belgium.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Reginster
authors.affiliation: MRC Lifecourse Epidemiology Unit, University of Southampton, Southampton, UK.|NIHR Musculoskeletal Biomedical Research Unit, University of Oxford, Oxford, UK.
authors.forename: Cyrus
authors.initials: C
authors.lastname: Cooper
authors.affiliation: Service of Bone Diseases, Geneva University Hospitals and Faculty of Medicine, Geneva, Switzerland.
authors.forename: René
authors.initials: R
authors.lastname: Rizzoli
authors.affiliation: Centre for Metabolic Bone Diseases, and Centre for Integrated Research in Musculoskeletal Ageing, University of Sheffield, Sheffield, UK.
authors.forename: John A
authors.initials: JA
authors.lastname: Kanis
authors.affiliation: Columbia University Medical Center, The Neurological Institute, New York, USA.
authors.forename: Geoff
authors.initials: G
authors.lastname: Appelboom
authors.affiliation: Gerontology and Frailty in Ageing Research Department, Vrije Universiteit Brussel (VUB), Brussels, Belgium.
authors.forename: Ivan
authors.initials: I
authors.lastname: Bautmans
authors.affiliation: Department of Geriatrics, University Hospital of Zurich, Zurich, Switzerland.
authors.forename: Heike A
authors.initials: HA
authors.lastname: Bischoff-Ferrari
authors.affiliation: Department of Epidemiology and Biostatistics; and Amsterdam Rheumatology and Immunology Center, VU University Medical Center, Amsterdam, Netherlands.
authors.forename: Maarten
authors.initials: M
authors.lastname: Boers
authors.affiliation: Department of Internal Medicine, University of Florence, Florence, Italy.
authors.forename: Maria Luisa
authors.initials: ML
authors.lastname: Brandi
authors.affiliation: Department of Public Health, Epidemiology and Health Economics, University of Liege, CHU Sart Tilman B23, 4000, Liège, Belgium. olivier.bruyere@ulg.ac.be.
authors.forename: Olivier
authors.initials: O
authors.lastname: Bruyère
authors.affiliation: Geriatrics and Geriatric Emergency Care, IRCCS-INRCA, Ancona, Italy.
authors.forename: Antonio
authors.initials: A
authors.lastname: Cherubini
authors.affiliation: Research Unit of Molecular Physiology (URPHYM), NARILIS, University of Namur, Namur, Belgium.
authors.forename: Bruno
authors.initials: B
authors.lastname: Flamion
authors.affiliation: Nutrition, Exercise Physiology and Sarcopenia Laboratory, Jean Mayer USDA Human Nutrition Research Center on Aging at Tufts University, Boston, USA.
authors.forename: Roger A
authors.initials: RA
authors.lastname: Fielding
authors.affiliation: Department of Public Health and Health Management, University of Medicine and Pharmacy of Tirgu Mures, Tirgu Mures, Romania.
authors.forename: Andrea Ildiko
authors.initials: AI
authors.lastname: Gasparik
authors.affiliation: NUTRIM School for Nutrition, Toxicology and Metabolism, Maastricht University, Maastricht, Netherlands.
authors.forename: Luc
authors.initials: L
authors.lastname: Van Loon
authors.affiliation: Centre for Metabolic Bone Diseases, and Centre for Integrated Research in Musculoskeletal Ageing, University of Sheffield, Sheffield, UK.
authors.forename: Eugene
authors.initials: E
authors.lastname: McCloskey
authors.affiliation: Lilly Research Laboratories, Eli Lilly and Company, Indianapolis, USA.
authors.forename: Bruce H
authors.initials: BH
authors.lastname: Mitlak
authors.affiliation: Department of OrthoGeriatrics, Rehabilitation and Stabilization, Frailty Area, E.O. Galliera Hospital, NR-HS, Genoa, Italy.
authors.forename: Alberto
authors.initials: A
authors.lastname: Pilotto
authors.affiliation: , Bonn, Germany.
authors.forename: Suzanne
authors.initials: S
authors.lastname: Reiter-Niesert
authors.affiliation: Gérontopôle of Toulouse, University of Toulouse III, CHU Purpan, Toulouse, France.
authors.forename: Yves
authors.initials: Y
authors.lastname: Rolland
authors.affiliation: Institut de Recherches Internationales Servier, Suresnes, France.
authors.forename: Yannis
authors.initials: Y
authors.lastname: Tsouderos
authors.affiliation: Department of Health Sciences, VU University, Amsterdam, Netherlands.|Department of Nutrition and Dietetics, Internal Medicine, VU University Medical Center, Amsterdam, Netherlands.
authors.forename: Marjolein
authors.initials: M
authors.lastname: Visser
authors.affiliation: Servicio de Geriatría, Hospital Universitario Ramón y Cajal, Madrid, Spain.
authors.forename: Alfonso J
authors.initials: AJ
authors.lastname: Cruz-Jentoft
mesh_terms: D000368:Aged; D002986:Clinical Trials as Topic / Q000379:methods; D006801:Humans; D054539:Medication Therapy Management; D017410:Practice Guidelines as Topic; D012107:Research Design; D055948:Sarcopenia / Q000175:diagnosis / Q000188:drug therapy / Q000209:etiology / Q000517:prevention & control
keywords: Clinical trials; Frailty; Preventative health care; Public health; Sarcopenia