A longitudinal study of creatine kinase and creatinine levels in Duchenne muscular dystrophy

被引:5
作者
Zygmunt, Alexander M. M. [1 ,2 ,5 ,6 ]
Wong, Brenda L. L. [3 ]
Horn, Paul S. S. [1 ,2 ]
Lambert, Joshua [4 ]
Bange, Jean E. E. [1 ,2 ]
Rybalsky, Irina [1 ,2 ]
Chouteau, Wendy [1 ,2 ]
Tian, Cuixia [1 ,2 ]
机构
[1] Univ Cincinnati, Coll Med, Div Neurol, Cincinnati, OH USA
[2] Cincinnati Childrens Hosp, Med Ctr, Cincinnati, OH USA
[3] Univ Massachusetts Chan Med Sch, Dept Pediat, Worcester, MA USA
[4] Univ Alabama, Dept Psychol, Tuscaloosa, AL USA
[5] Univ Cincinnati, Div Neurol, Coll Med, 3333 Burnet Ave, Cincinnati, OH 45229 USA
[6] Cincinnati Childrens Hosp, Med Ctr, 3333 Burnet Ave, Cincinnati, OH 45229 USA
关键词
biomarkers; creatine kinase; creatinine; Duchenne muscular dystrophy; natural history; SERUM ENZYME; MUSCLE MASS; BOYS; DMD; GOLODIRSEN; BIOMARKERS; DISEASE;
D O I
10.1002/mus.27760
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Introduction/AimsManagement of Duchenne muscular dystrophy (DMD) has entered an era featuring novel treatments. Trackable noninvasive biomarkers could improve disease progression monitoring and drug effect detection. Our aim in this study was to measure changes in selected noninvasive biomarkers and assess their relationship to age and motor function. MethodsWe retrospectively studied 555 patients with DMD who had at least 12 months of treatment of glucocorticoids and were not enrolled in trials of potential disease-modifying therapies. We extracted biomarker data of serum creatine kinase (CK), serum creatinine (Cr), urine Cr, and urine Cr/urine osmolality (osm), as well as functional data for age at loss of ambulation and Functional Motor Scale (FMS) values from patients' clinical records. Data were analyzed using linear mixed-model analyses. ResultsCK, serum Cr, urine Cr, and urine Cr/urine osm all decreased with declining motor function. CK consistently decreased and FMS score consistently worsened with age without clear inflection points. There was an increased odds ratio for LOA with lower values of CK, serum Cr, urine Cr, and urine Cr/urine osm, most notably for urine Cr. DiscussionAlthough individual biomarker values are challenging to directly apply clinically, our study has demonstrated that trends over time may complement functional measures in the assessment of individuals with DMD. Future studies could elucidate predictive utility of these biomarkers in assessing motor function changes in DMD.
引用
收藏
页码:138 / 145
页数:8
相关论文
共 45 条
[1]   The 100-meter timed test: Normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials [J].
Alfano, Lindsay N. ;
Miller, Natalie F. ;
Berry, Katherine M. ;
Yin, Han ;
Rolf, Kimberly E. ;
Flanigan, Kevin M. ;
Mendell, Jerry R. ;
Lowes, Linda P. .
NEUROMUSCULAR DISORDERS, 2017, 27 (05) :452-457
[2]  
[Anonymous], 2023, Neurology, V100, P936, DOI 10.1212/WNL.0000000000012406
[3]   Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history [J].
Arora, Harneet ;
Willcocks, Rebecca J. ;
Lott, Donovan J. ;
Harrington, Ann T. ;
Senesac, Claudia R. ;
Zilke, Kirsten L. ;
Daniels, Michael J. ;
Xu, Dandan ;
Tennekoon, Gihan I. ;
Finanger, Erika L. ;
Russman, Barry S. ;
Finkel, Richard S. ;
Triplett, William T. ;
Byrne, Barry J. ;
Walter, Glenn A. ;
Sweeney, H. Lee ;
Vandenborne, Krista .
MUSCLE & NERVE, 2018, 58 (05) :631-638
[4]   Influence of muscle mass and physical activity on serum and urinary creatinine and serum cystatin C [J].
Baxmann, Alessandra Calabria ;
Ahmed, Marion Souza ;
Marques, Natalia Cristina ;
Menon, Viviane Barcellos ;
Pereira, Aparecido Bernardo ;
Kirsztajn, Gianna Mastroianni ;
Heilberg, Ita Pfeferman .
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2008, 3 (02) :348-354
[5]   DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study [J].
Bello, Luca ;
Morgenroth, Lauren P. ;
Gordish-Dressman, Heather ;
Hoffman, Eric P. ;
McDonald, Craig M. ;
Cirak, Sebahattin .
NEUROLOGY, 2016, 87 (04) :401-409
[6]   Histological effects of givinostat in boys with Duchenne muscular dystrophy [J].
Bettica, Paolo ;
Petrini, Stefania ;
D'Oria, Valentina ;
D'Amico, Adele ;
Catteruccia, Michela ;
Pane, Marika ;
Sivo, Serena ;
Magri, Francesca ;
Brajkovic, Simona ;
Messina, Sonia ;
Vita, Gian Luca ;
Gatti, Barbara ;
Moggio, Maurizio ;
Puri, Pier Lorenzo ;
Rocchetti, Maurizio ;
De Nicolao, Giuseppe ;
Vita, Giuseppe ;
Comi, Giacomo P. ;
Bertini, Enrico ;
Mercuri, Eugenio .
NEUROMUSCULAR DISORDERS, 2016, 26 (10) :643-649
[7]  
Burch Peter M, 2015, J Neuromuscul Dis, V2, P241
[8]   Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping A Phase 2 Randomized Clinical Trial [J].
Clemens, Paula R. ;
Rao, Vamshi K. ;
Connolly, Anne M. ;
Harper, Amy D. ;
Mah, Jean K. ;
Smith, Edward C. ;
McDonald, Craig M. ;
Zaidman, Craig M. ;
Morgenroth, Lauren P. ;
Osaki, Hironori ;
Satou, Youhei ;
Yamashita, Taishi ;
Hoffman, Eric P. .
JAMA NEUROLOGY, 2020, 77 (08) :982-991
[9]   The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD [J].
Duong, Tina ;
Canbek, Jennifer ;
Birkmeier, Marisa ;
Nelson, Leslie ;
Siener, Catherine ;
Fernandez-Fernandez, Alicia ;
Henricson, Erik ;
McDonald, Craig M. ;
Gordish-Dressman, Heather .
JOURNAL OF NEUROMUSCULAR DISEASES, 2021, 8 (06) :939-948
[10]   204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands [J].
Ferlini, Alessandra ;
Flanigan, Kevin M. ;
Lochmuller, Hanns ;
Muntoni, Francesco ;
't Hoen, Peter A. C. ;
McNally, Elizabeth .
NEUROMUSCULAR DISORDERS, 2015, 25 (02) :184-198