TY - JOUR
T1 - Update on the ESCEO recommendation for the conduct of clinical trials for drugs aiming at the treatment of sarcopenia in older adults
AU - Reginster, Jean Yves
AU - Beaudart, Charlotte
AU - Al-Daghri, Nasser
AU - Avouac, Bernard
AU - Bauer, Jürgen
AU - Bere, Nathalie
AU - Bruyère, Olivier
AU - Cerreta, Francesca
AU - Cesari, Matteo
AU - Rosa, Mario Miguel
AU - Cooper, Cyrus
AU - Cruz Jentoft, Alfonso J.
AU - Dennison, Elaine
AU - Geerinck, Anton
AU - Gielen, Evelien
AU - Landi, Francesco
AU - Laslop, Andrea
AU - Maggi, Stefania
AU - Prieto Yerro, María Concepción
AU - Rizzoli, René
AU - Sundseth, Hildrun
AU - Sieber, Cornel
AU - Trombetti, Andrea
AU - Vellas, Bruno
AU - Veronese, Nicola
AU - Visser, Marjolein
AU - Vlaskovska, Mila
AU - Fielding, Roger A.
N1 - Funding Information:
The ESCEO Working Group was funded by ESCEO. The ESCEO receives unrestricted education Grants to support its educational and scientific activities from non-governmental organisations, not-for-profit organisations, non-commercial or corporate partners. The choice of topics, participants, content and agenda of the Working Group as well as the writing, editing, submission and reviewing of the manuscript are under the sole responsibility of the ESCEO, without any influence from third parties.
Publisher Copyright:
© 2020, The Author(s).
PY - 2021/1
Y1 - 2021/1
N2 - Background: In 2016, an expert working group was convened under the auspices of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) and formulated consensus recommendations for the conduct of clinical trials for drugs to prevent or treat sarcopenia. Aims: The objective of the current paper is to provide a 2020 update of the previous recommendations in accordance with the evidence that has become available since our original recommendations. Methods: This paper is based on literature reviews performed by members of the ESCEO working group and followed up with face to face meetings organized for the whole group to make amendments and discuss further recommendations. Results: The randomized placebo-controlled double-blind parallel-arm drug clinical trials should be the design of choice for both phase II and III trials. Treatment and follow-up should run at least 6 months for phase II and 12 months for phase III trials. Overall physical activity, nutrition, co-prescriptions and comorbidity should be recorded. Participants in these trials should be at least 70-years-old and present with a combination of low muscle strength and low physical performance. Severely malnourished individuals, as well as bedridden patients, patients with extremely limited mobility or individuals with physical limitations clearly attributable to the direct effect of a specific disease, should be excluded. Multiple outcomes are proposed for phase II trials, including, as example, physical performance, muscle strength and mass, muscle metabolism and muscle-bone interaction. For phase III trials, we recommend a co-primary endpoint of a measure of functional performance and a Patient Reported Outcome Measure. Conclusion: The working group has formulated consensus recommendations on specific aspects of trial design, and in doing so hopes to contribute to an improvement of the methodological robustness and comparability of clinical trials. Standardization of designs and outcomes would advance the field by allowing better comparison across studies, including performing individual patient-data meta-analyses, and different pro-myogenic therapies.
AB - Background: In 2016, an expert working group was convened under the auspices of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) and formulated consensus recommendations for the conduct of clinical trials for drugs to prevent or treat sarcopenia. Aims: The objective of the current paper is to provide a 2020 update of the previous recommendations in accordance with the evidence that has become available since our original recommendations. Methods: This paper is based on literature reviews performed by members of the ESCEO working group and followed up with face to face meetings organized for the whole group to make amendments and discuss further recommendations. Results: The randomized placebo-controlled double-blind parallel-arm drug clinical trials should be the design of choice for both phase II and III trials. Treatment and follow-up should run at least 6 months for phase II and 12 months for phase III trials. Overall physical activity, nutrition, co-prescriptions and comorbidity should be recorded. Participants in these trials should be at least 70-years-old and present with a combination of low muscle strength and low physical performance. Severely malnourished individuals, as well as bedridden patients, patients with extremely limited mobility or individuals with physical limitations clearly attributable to the direct effect of a specific disease, should be excluded. Multiple outcomes are proposed for phase II trials, including, as example, physical performance, muscle strength and mass, muscle metabolism and muscle-bone interaction. For phase III trials, we recommend a co-primary endpoint of a measure of functional performance and a Patient Reported Outcome Measure. Conclusion: The working group has formulated consensus recommendations on specific aspects of trial design, and in doing so hopes to contribute to an improvement of the methodological robustness and comparability of clinical trials. Standardization of designs and outcomes would advance the field by allowing better comparison across studies, including performing individual patient-data meta-analyses, and different pro-myogenic therapies.
KW - Clinical trial
KW - Drug registration
KW - Guidelines
KW - Recommendations
KW - Sarcopenia
KW - Treatment
UR - http://www.scopus.com/inward/record.url?scp=85088870274&partnerID=8YFLogxK
U2 - 10.1007/s40520-020-01663-4
DO - 10.1007/s40520-020-01663-4
M3 - Review article
C2 - 32737844
AN - SCOPUS:85088870274
SN - 1594-0667
VL - 33
SP - 3
EP - 17
JO - Aging Clinical and Experimental Research
JF - Aging Clinical and Experimental Research
IS - 1
ER -