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Recommendations for the conduct of clinical trials for drugs to treat or prevent sarcopenia

Recommendations for the conduct of clinical trials for drugs to treat or prevent sarcopenia
Recommendations for the conduct of clinical trials for drugs to treat or prevent sarcopenia
Purpose

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.
Clinical trials, sarcopenia, public health, preventative health care, frailty
1594-0667
1-12
Reginster, J.Y.
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Cooper, C.
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Rizzoli, R.
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Kanis, J.A.
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Appelboom, G.
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Bautmans, I.
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Bischoff-Ferrari, H.A.
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Boers, M.
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Brandi, M.L.
4147f967-9c22-494d-ae35-c8141161413d
Bruyere, O.
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Cherubini, A.
d5bf21fa-0629-49c1-865b-461debf45d60
Flamion, B.
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Fielding, B.
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Gasparik, A.I.
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Van Loon, L.
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McCloskey, E.
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Mitlak, B.H.
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Pilotto, A.
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Reiter-Niesert, S.
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Rolland, Y.
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Tsouderos, Y.
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Visser, M.
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Cruz-Jentoft, A.J.
dd510566-9cd6-4243-a83c-31e1e0206e70
Reginster, J.Y.
4083b457-5347-4ece-a53e-af19c8868c42
Cooper, C.
e05f5612-b493-4273-9b71-9e0ce32bdad6
Rizzoli, R.
2214fb77-8fb7-4c0b-bfc4-9f8d3cace5d7
Kanis, J.A.
8da04a36-08a7-4310-b4b4-a6d432439587
Appelboom, G.
1759d35d-a0ca-4541-9edc-24372a032cc2
Bautmans, I.
8f7e6327-892b-4462-8b27-96bd2f6c8f53
Bischoff-Ferrari, H.A.
4540486a-8ebe-40e7-97d3-f3dafcde84ff
Boers, M.
1e4a9620-354d-4b5a-a978-4de78fd3f6c3
Brandi, M.L.
4147f967-9c22-494d-ae35-c8141161413d
Bruyere, O.
9c455ea4-7f77-407f-ab5a-ecb571db8245
Cherubini, A.
d5bf21fa-0629-49c1-865b-461debf45d60
Flamion, B.
f5efa657-827d-4b5f-8457-0a05365c63f4
Fielding, B.
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Gasparik, A.I.
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Van Loon, L.
070f7461-b4ef-48c2-8937-0551c3e9dba3
McCloskey, E.
5211de37-303a-42f8-b24b-00c475264f78
Mitlak, B.H.
49ce3fa7-4b82-426e-9618-812f3a08126b
Pilotto, A.
14cf5528-3d77-4c13-8693-59f08f2a5f30
Reiter-Niesert, S.
a28560e4-6ea8-419d-9958-dadcc149c07d
Rolland, Y.
2baa5a50-58f6-4527-88d4-3bb20e05f886
Tsouderos, Y.
1cf1fc63-d05f-41de-98f8-01ae38bddfa3
Visser, M.
5b26c37b-3677-4606-9a46-9c06ce3cad8b
Cruz-Jentoft, A.J.
dd510566-9cd6-4243-a83c-31e1e0206e70

Reginster, J.Y., Cooper, C., Rizzoli, R., Kanis, J.A., Appelboom, G., Bautmans, I., Bischoff-Ferrari, H.A., Boers, M., Brandi, M.L., Bruyere, O., Cherubini, A., Flamion, B., Fielding, B., Gasparik, A.I., Van Loon, L., McCloskey, E., Mitlak, B.H., Pilotto, A., Reiter-Niesert, S., Rolland, Y., Tsouderos, Y., Visser, M. and Cruz-Jentoft, A.J. (2015) Recommendations for the conduct of clinical trials for drugs to treat or prevent sarcopenia. Aging Clinical and Experimental Research, 1-12. (doi:10.1007/s40520-015-0517-y). (PMID:26717937)

Record type: Article

Abstract

Purpose

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.

Text
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More information

Accepted/In Press date: 6 December 2015
e-pub ahead of print date: 30 December 2015
Keywords: Clinical trials, sarcopenia, public health, preventative health care, frailty
Organisations: Faculty of Medicine

Identifiers

Local EPrints ID: 385427
URI: http://eprints.soton.ac.uk/id/eprint/385427
ISSN: 1594-0667
PURE UUID: 1130d371-0dc9-4283-97e7-acbeb732b34d
ORCID for C. Cooper: ORCID iD orcid.org/0000-0003-3510-0709

Catalogue record

Date deposited: 19 Jan 2016 15:46
Last modified: 18 Mar 2024 02:45

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Contributors

Author: J.Y. Reginster
Author: C. Cooper ORCID iD
Author: R. Rizzoli
Author: J.A. Kanis
Author: G. Appelboom
Author: I. Bautmans
Author: H.A. Bischoff-Ferrari
Author: M. Boers
Author: M.L. Brandi
Author: O. Bruyere
Author: A. Cherubini
Author: B. Flamion
Author: B. Fielding
Author: A.I. Gasparik
Author: L. Van Loon
Author: E. McCloskey
Author: B.H. Mitlak
Author: A. Pilotto
Author: S. Reiter-Niesert
Author: Y. Rolland
Author: Y. Tsouderos
Author: M. Visser
Author: A.J. Cruz-Jentoft

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