학술논문
Lean tissue mass measurements by dual-energy X-ray absorptiometry and associations with strength and functional outcome measures in facioscapulohumeral muscular dystrophy.
Document Type
Academic Journal
Author
Wang LH; University of Washington, Department of Neurology, WA, USA. Electronic address: leowang@uw.edu.; Leung DG; Kennedy Krieger Institute, The Johns Hopkins School of Medicine, MD, USA.; Wagner KR; Kennedy Krieger Institute, The Johns Hopkins School of Medicine, MD, USA.; Lowry SJ; Seattle Children's Research Institute, WA, USA.; McDermott MP; University of Rochester Medical Center, Department of Biostatistics and Computational Biology, NY, USA; University of Rochester Medical Center, Department of Neurology, NY, USA.; Eichinger K; University of Rochester Medical Center, Department of Neurology, NY, USA.; Higgs K; University of Kansas Medical Center, Department of Neurology, KS, USA.; Walker M; University of Kansas Medical Center, Department of Neurology, KS, USA.; Lewis L; University of Rochester Medical Center, Department of Biostatistics and Computational Biology, NY, USA.; Martens WB; University of Rochester Medical Center, Department of Biostatistics and Computational Biology, NY, USA.; Mul K; Radboud University, Nijmegen, Netherlands.; Sansone VA; The NEMO Clinical Center, Neurorehabilitation Unit, University of Milan, Department of Neurology, Milan, Italy.; Shieh P; University of California, Los Angeles, CA, USA.; Elsheikh B; The Ohio State University, OH, USA.; LoRusso S; The Ohio State University, OH, USA.; Butterfield RJ; University of Utah, UT, USA.; Johnson N; Virginia Commonwealth University, VA, USA.; Preston MR; University of Washington, Department of Neurology, WA, USA.; Messina C; Galeazzi Institute, Radiology Department, University of Milan, Italy.; Carraro E; The NEMO Clinical Center, Neurorehabilitation Unit, University of Milan, Department of Neurology, Milan, Italy.; Tawil R; University of Rochester Medical Center, Department of Neurology, NY, USA.; Statland J; University of Kansas Medical Center, Department of Neurology, KS, USA.
Source
Publisher: Pergamon Press Country of Publication: England NLM ID: 9111470 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2364 (Electronic) Linking ISSN: 09608966 NLM ISO Abbreviation: Neuromuscul Disord Subsets: MEDLINE
Subject
Language
English
Abstract
Facioscapulohumeral muscular dystrophy (FSHD) is a slowly progressive disease of skeletal muscle. Dual energy X-ray absorptiometry (DEXA) is a widely available, cost-effective and sensitive technique for measuring whole body and regional lean tissue mass and has been used in prior clinical trials in neuromuscular diseases. The Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD (ReSolve) study is a prospective, longitudinal, observational multisite study. We obtained concurrent DEXA scans and functional outcome measurements in 185 patients with FSHD at the baseline visit. We determined the associations between lean tissue mass in the upper and lower extremities and corresponding clinical outcome measures. There were moderate correlations between upper and lower extremity lean tissue mass and their corresponding strengths and function. Lean tissue mass obtained by DEXA scan may be useful as a biomarker in future clinical trials in FSHD.
Competing Interests: Declaration of Competing Interest LHW reports consultancy/advisory board for Fulcrum Therapeutics, Argenx, Avidity, Ultragenyx, PepGen, data safety monitoring board for Scholar Rock. KE has served on advisory boards or has received consulting fees from Biogen, Acceleron, PTC, Ionis Pharmaceuticals, Fulcrum Therapeutics, Dyne Therapeutics, Avidity Biosciences and Roche. RNT reports consultancy with Fulcrum Therapeutics, Arrowhead, Dyne Therapeutics, Acceleron, MT Pharma, miRecule, and Roche Pharma. JS has served as consultant for Arrowhead, ML Bio, MT Pharma, Epic Bio, Armatus; served on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics, Avidity, Fulcrum Therapeutics, Roche. PS has served as consultant for Sarepta, Genentech, Biogen, Alexion, Argenx, UCB; served on speakers’ bureaus for CSL Behring, Grifols, Alexion, Biogen, Genentech, Argenx, Catalyst. KRW is an employee of F. Hoffmann-La Roche Ltd. BE received research funding from Biogen, Genentech, Alexion, Pharnext, and Viela Bio and served as a consultant for Biogen, Genentech, and Argenx. NJ has received grant funding from NINDS (R01NS104010), NCATS (21TR003184), CDC (U01DD001242–02) and the FDA (7R01FD006071–02). He receives royalties from the CCMDHI and the CMTHI; receives research funds from Dyne, AveXis, Takeda, Sanofi Genzyme, Pepgen, Vertex Pharmaceuticals, Fulcrum Therapeutics, Sarepta, and AskBio; has provided consultation for Takeda, Pepgen, Arthex, AveXis, AMO Pharma, Fulcrum Therapeutics, Dyne, Avidity, and Vertex Pharmaceuticals. KM reports consultancy for Avidity Biosciences. RJB reports consultancy/advisory board for Aavanti Bio, Scholar Rock, Avexis, Pfizer, Reata, and Sarepta Therapeutics. SLO, MRP, MD have no disclosures.
(Copyright © 2023. Published by Elsevier B.V.)
Competing Interests: Declaration of Competing Interest LHW reports consultancy/advisory board for Fulcrum Therapeutics, Argenx, Avidity, Ultragenyx, PepGen, data safety monitoring board for Scholar Rock. KE has served on advisory boards or has received consulting fees from Biogen, Acceleron, PTC, Ionis Pharmaceuticals, Fulcrum Therapeutics, Dyne Therapeutics, Avidity Biosciences and Roche. RNT reports consultancy with Fulcrum Therapeutics, Arrowhead, Dyne Therapeutics, Acceleron, MT Pharma, miRecule, and Roche Pharma. JS has served as consultant for Arrowhead, ML Bio, MT Pharma, Epic Bio, Armatus; served on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics, Avidity, Fulcrum Therapeutics, Roche. PS has served as consultant for Sarepta, Genentech, Biogen, Alexion, Argenx, UCB; served on speakers’ bureaus for CSL Behring, Grifols, Alexion, Biogen, Genentech, Argenx, Catalyst. KRW is an employee of F. Hoffmann-La Roche Ltd. BE received research funding from Biogen, Genentech, Alexion, Pharnext, and Viela Bio and served as a consultant for Biogen, Genentech, and Argenx. NJ has received grant funding from NINDS (R01NS104010), NCATS (21TR003184), CDC (U01DD001242–02) and the FDA (7R01FD006071–02). He receives royalties from the CCMDHI and the CMTHI; receives research funds from Dyne, AveXis, Takeda, Sanofi Genzyme, Pepgen, Vertex Pharmaceuticals, Fulcrum Therapeutics, Sarepta, and AskBio; has provided consultation for Takeda, Pepgen, Arthex, AveXis, AMO Pharma, Fulcrum Therapeutics, Dyne, Avidity, and Vertex Pharmaceuticals. KM reports consultancy for Avidity Biosciences. RJB reports consultancy/advisory board for Aavanti Bio, Scholar Rock, Avexis, Pfizer, Reata, and Sarepta Therapeutics. SLO, MRP, MD have no disclosures.
(Copyright © 2023. Published by Elsevier B.V.)