Lean tissue mass measurements by dual-energy X-ray absorptiometry and associations with strength and functional outcome measures in facioscapulohumeral muscular dystrophy

Leo H Wang; Doris G Leung; Kathryn R Wagner; Sarah J Lowry; Michael P McDermott; Katy Eichinger; Kiley Higgs; Michaela Walker; Leann Lewis; William B Martens; Karlien Mul; Valeria A Sansone; Perry Shieh; Bakri Elsheikh; Samantha LoRusso; Russell J Butterfield; Nicholas Johnson; Matthew R Preston; Carmelo Messina; Elena Carraro; Rabi Tawil; Jeff Statland; ReSolve Investigators of the FSHD CTRN

doi:10.1016/j.nmd.2023.06.008

Lean tissue mass measurements by dual-energy X-ray absorptiometry and associations with strength and functional outcome measures in facioscapulohumeral muscular dystrophy

Neuromuscul Disord. 2023 Sep;33(9):63-68. doi: 10.1016/j.nmd.2023.06.008. Epub 2023 Jun 21.

Authors

Leo H Wang¹, Doris G Leung², Kathryn R Wagner², Sarah J Lowry³, Michael P McDermott⁴, Katy Eichinger⁵, Kiley Higgs⁶, Michaela Walker⁶, Leann Lewis⁷, William B Martens⁷, Karlien Mul⁸, Valeria A Sansone⁹, Perry Shieh¹⁰, Bakri Elsheikh¹¹, Samantha LoRusso¹¹, Russell J Butterfield¹², Nicholas Johnson¹³, Matthew R Preston¹⁴, Carmelo Messina¹⁵, Elena Carraro⁹, Rabi Tawil⁵, Jeff Statland⁶; ReSolve Investigators of the FSHD CTRN

Affiliations

¹ University of Washington, Department of Neurology, WA, USA. Electronic address: leowang@uw.edu.
² Kennedy Krieger Institute, The Johns Hopkins School of Medicine, MD, USA.
³ Seattle Children's Research Institute, WA, USA.
⁴ University of Rochester Medical Center, Department of Biostatistics and Computational Biology, NY, USA; University of Rochester Medical Center, Department of Neurology, NY, USA.
⁵ University of Rochester Medical Center, Department of Neurology, NY, USA.
⁶ University of Kansas Medical Center, Department of Neurology, KS, USA.
⁷ University of Rochester Medical Center, Department of Biostatistics and Computational Biology, NY, USA.
⁸ Radboud University, Nijmegen, Netherlands.
⁹ The NEMO Clinical Center, Neurorehabilitation Unit, University of Milan, Department of Neurology, Milan, Italy.
¹⁰ University of California, Los Angeles, CA, USA.
¹¹ The Ohio State University, OH, USA.
¹² University of Utah, UT, USA.
¹³ Virginia Commonwealth University, VA, USA.
¹⁴ University of Washington, Department of Neurology, WA, USA.
¹⁵ Galeazzi Institute, Radiology Department, University of Milan, Italy.

PMID: 37400350
PMCID: PMC10527411 (available on 2024-09-01)
DOI: 10.1016/j.nmd.2023.06.008

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.

Keywords: Facioscapulohumeral muscular dystrophy; Imaging markers; Leg strength; Patient reported outcome.

Publication types

Research Support, U.S. Gov't, P.H.S.
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Absorptiometry, Photon / methods
Humans
Muscle, Skeletal
Muscular Dystrophy, Facioscapulohumeral* / diagnostic imaging
Outcome Assessment, Health Care
Prospective Studies

Abstract

Publication types

MeSH terms

Grants and funding