272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16-18 June 2023, Hoofddorp, The Netherlands

被引:33
作者
Lilleker, James B. [1 ,2 ]
Naddaf, Elie [3 ]
Saris, Christiaan G. J. [4 ]
Schmidt, Jens [5 ,6 ,7 ,8 ]
de Visser, Marianne [9 ]
Weihl, Conrad C. [10 ,11 ]
机构
[1] Northern Care Alliance NHS Fdn Trust, Manchester Ctr Clin Neurosci, Manchester Acad Hlth Sci Ctr, Salford, England
[2] Univ Manchester, Div Musculoskeletal & Dermatol Sci, Manchester, England
[3] Dept Neurol, Mayo Clin, Rochester, MN USA
[4] Radboud Univ Nijmegen, Donders Inst Brain Cognit & Behav, Dept Neurol, Med Ctr, Nijmegen, Netherlands
[5] Brandenburg Med Sch MHB, Neuromuscular Ctr, Dept Neurol & Pain Treatment, Univ Hosp, Rudersdorf, Germany
[6] Brandenburg Med Sch MHB, Ctr Translat Med, Immanuel Klin Rudersdorf, Univ Hosp, Rudersdorf, Germany
[7] Fac Hlth Sci Brandenburg, Brandenburg Med Sch Theodor Fontane, Rudersdorf, Germany
[8] Univ Med Ctr Gottingen, Dept Neurol, Gottingen, Germany
[9] Univ Amsterdam, Dept Neurol, Amsterdam UMC, Amsterdam Neurosci, Amsterdam, Netherlands
[10] Washington Univ, Neuromuscular Div, Sch Med, St Louis, MO USA
[11] Washington Univ, Dept Neurol, Sch Med, Box 8111 660 S Euclid Ave, St Louis, MO 63110 USA
关键词
Inclusion body myositis; Diagnostic criteria; Outcome measures; Clinical trials; CYTOSOLIC 5'-NUCLEOTIDASE 1A; IDIOPATHIC INFLAMMATORY MYOPATHIES; DOUBLE-BLIND; PHYSICAL-ACTIVITY; SKELETAL-MUSCLE; PREVALENCE; TDP-43; AUTOANTIBODIES; MITOCHONDRIAL; ACCUMULATION;
D O I
10.1016/j.nmd.2024.03.001
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Since the publication of the 2013 European Neuromuscular Center (ENMC) diagnostic criteria for Inclusion Body Myositis (IBM), several advances have been made regarding IBM epidemiology, pathogenesis, diagnostic tools, and clinical trial readiness. Novel diagnostic tools include muscle imaging techniques such as MRI and ultrasound, and serological testing for cytosolic 5' -nucleotidase-1A antibodies. The 272nd ENMC workshop aimed to develop new diagnostic criteria, discuss clinical outcome measures and clinical trial readiness. The workshop started with patient representatives highlighting several understudied symptoms and the urge for a timely diagnosis. This was followed by presentations from IBM experts highlighting the new developments in the field. This report is composed of two parts, the first part providing new diagnostic criteria on which consensus was achieved. The second part focuses on the use of outcome measures in clinical practice and clinical trials, highlighting current limitations and outlining the goals for future studies.
引用
收藏
页码:36 / 51
页数:16
相关论文
共 98 条
[1]  
ADAMS R. D., 1965, TRANS AMER NEUROL ASS, V90, P213
[2]   Atypical presentations of inclusion body myositis: Clinical characteristics and long-term outcomes [J].
Alamr, Mazen ;
Pinto, Marcus, V ;
Naddaf, Elie .
MUSCLE & NERVE, 2022, 66 (06) :686-693
[3]   Diagnosing and managing dysphagia in inclusion body myositis: a systematic review [J].
Ambrocio, Kevin Renz ;
Garand, Kendrea L. ;
Roy, Bhaskar ;
Bhutada, Ankita M. ;
Malandraki, Georgia A. .
RHEUMATOLOGY, 2023, 62 (10) :3227-3244
[4]   Anti-Valosin-Containing Protein (VCP/p97) Autoantibodies in Inclusion Body Myositis and Other Inflammatory Myopathies [J].
Amlani, Adam ;
Choi, May Y. ;
Buhler, Katherine A. ;
Hudson, Marie ;
Tarnopolsky, Mark ;
Brady, Lauren ;
Schmeling, Heinrike ;
Swain, Mark G. ;
Stingl, Cory ;
Reed, Ann ;
Fritzler, Marvin J. .
ACR OPEN RHEUMATOLOGY, 2023, 5 (01) :10-14
[5]   Comparison of weakness progression in inclusion body myositis during treatment with methotrexate or placebo [J].
Badrising, UA ;
Maat-Schieman, MLC ;
Ferrari, MD ;
Zwinderman, AH ;
Wessels, JAM ;
Breedveld, FC ;
van Doorn, PA ;
van Engelen, BGM ;
Hoogendijk, JE ;
Höweler, CJ ;
de Jager, AE ;
Jennekens, FGI ;
Koehler, PJ ;
de Visser, M ;
Viddeleer, A ;
Verschuuren, JJ ;
Wintzen, AR .
ANNALS OF NEUROLOGY, 2002, 51 (03) :369-372
[6]  
Badrising Umesh A, 2017, J Neuromuscul Dis, V4, P327, DOI 10.3233/JND-170234
[7]   Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial [J].
Benveniste, Olivier ;
Hogrel, Jean-Yves ;
Belin, Lisa ;
Annoussamy, Melanie ;
Bachasson, Damien ;
Rigolet, Aude ;
Laforet, Pascal ;
Dzangue-Tchoupou, Gaelle ;
Salem, Joe-Elie ;
Nguyen, Lee S. ;
Stojkovic, Tanya ;
Zahr, Noel ;
Hervier, Baptiste ;
Landon-Cardinal, Oceane ;
Behin, Anthony ;
Guilloux, Edith ;
Reyngoudt, Harmen ;
Amelin, Damien ;
Uruha, Akinori ;
Mariampillai, Kuberaka ;
Marty, Benjamin ;
Eymard, Bruno ;
Hulot, Jean-Sebastien ;
Greenberg, Steven A. ;
Carlier, Pierre G. ;
Allenbach, Yves .
LANCET RHEUMATOLOGY, 2021, 3 (01) :E40-E48
[8]   213th ENMC International Workshop: Outcome measures and clinical trial readiness in idiopathic inflammatory myopathies, Heemskerk, The Netherlands, 18-20 September 2015 [J].
Benveniste, Olivier ;
Rider, Lisa G. .
NEUROMUSCULAR DISORDERS, 2016, 26 (08) :523-534
[9]   Amyloid deposits and inflammatory infiltrates in sporadic inclusion body myositis: the inflammatory egg comes before the degenerative chicken [J].
Benveniste, Olivier ;
Stenzel, Werner ;
Hilton-Jones, David ;
Sandri, Marco ;
Boyer, Olivier ;
van Engelen, Baziel G. M. .
ACTA NEUROPATHOLOGICA, 2015, 129 (05) :611-624
[10]   Long-term observational study of sporadic inclusion body myositis [J].
Benveniste, Olivier ;
Guiguet, Marguerite ;
Freebody, Jane ;
Dubourg, Odile ;
Squier, Waney ;
Maisonobe, Thierry ;
Stojkovic, Tanya ;
Leite, Maria Isabel ;
Allenbach, Yves ;
Herson, Serge ;
Brady, Stefen ;
Eymard, Bruno ;
Hilton-Jones, David .
BRAIN, 2011, 134 :3176-3184