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Establishing how much improvement in lung function and distance walked is clinically important for adult patients with Pompe disease.

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第一作者： Aglina,Lika

第一单位： Department of Pediatrics, Centre for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Centre, Rotterdam, The Netherlands.;Department of Biostatistics, Erasmus MC University Medical Centre, Rotterdam, The Netherlands.;Department of Epidemiology, Erasmus MC University Medical Centre, Rotterdam, The Netherlands.

作者： Aglina,Lika ^[1] ; Eleni-Rosalina,Andrinopoulou ^[2] ; Nadine A M E,van der Beek ^[3] ; Dimitris,Rizopoulos ^[2] ; Ans T,van der Ploeg ^[4] ; Michelle E,Kruijshaar ^[4]

作者单位： Department of Pediatrics, Centre for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Centre, Rotterdam, The Netherlands.;Department of Biostatistics, Erasmus MC University Medical Centre, Rotterdam, The Netherlands.;Department of Epidemiology, Erasmus MC University Medical Centre, Rotterdam, The Netherlands. ^[1] Department of Biostatistics, Erasmus MC University Medical Centre, Rotterdam, The Netherlands.;Department of Epidemiology, Erasmus MC University Medical Centre, Rotterdam, The Netherlands. ^[2] Department of Neurology, Centre for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Centre, Rotterdam, The Netherlands. ^[3] Department of Pediatrics, Centre for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Centre, Rotterdam, The Netherlands. ^[4]

关键词 6‐minute walk test Pompe disease anchor‐based method clinically meaningful threshold forced vital capacity minimal clinically important difference minimal important change