Research ArticleArticle
The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis: A Randomized, Double-blind, Placebo-controlled Feasibility Study
Saunya Dover, Samantha Stephens, Jane E. Schneiderman, Eleanor Pullenayegum, Greg D. Wells, Deborah M. Levy, Jo-Anne Marcuz, Kristi Whitney, Andreas Schulze, Ingrid Tein and Brian M. Feldman
The Journal of Rheumatology August 2020, jrheum.191375; DOI: https://doi.org/10.3899/jrheum.191375
Saunya Dover
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Samantha Stephens
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Jane E. Schneiderman
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Eleanor Pullenayegum
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Greg D. Wells
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Deborah M. Levy
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Jo-Anne Marcuz
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Kristi Whitney
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Andreas Schulze
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Ingrid Tein
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Brian M. Feldman
Funding for this work was provided by The Pedano Family Juvenile Dermatomyositis Research Grant of The Myositis Association and The Rare Disease Foundation. Study drugs were supplied by AlzChem. The funders were not involved in any aspects of study design, data collection, data analysis or interpretation, writing of the report or the decision to publish the report. S. Dover, MSc, Child Health Evaluative Sciences, The Hospital for Sick Children; S. Stephens, PhD, Neurosciences and Mental Health, The Hospital for Sick Children; J.E. Schneiderman, PhD, RKin, CEP, Clinical Research Services, Research Institute, The Hospital for Sick Children, and Faculty of Kinesiology and Physical Education, University of Toronto; E. Pullenayegum, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management and Evaluation, the Dalla Lana School of Public Health, University of Toronto; G.D. Wells, PhD, Translational Medicine, The Hospital for Sick Children; D.M. Levy, MD, MS, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children; J.A. Marcuz, MScPT, K. Whitney, MSc, BScPT, Division of Rheumatology, The Hospital for Sick Children, and Department of Rehabilitation, The Hospital for Sick Children; A. Schulze, MD, PhD, Clinical and Metabolic Genetics, The Hospital for Sick Children; I. Tein, MD, FRCPC, Division of Neurology, Department of Pediatrics and Genetics and Genome Biology Program, The Hospital for Sick Children, and Department of Laboratory Medicine and Pathobiology, University of Toronto; B.M. Feldman, MD, MSc, FRCPC, Child Health Evaluative Sciences, The Hospital for Sick Children, and Institute of Health Policy, Management & Evaluation, the Dalla Lana School of Public Health, University of Toronto, and Department of Pediatrics, Faculty of Medicine, University of Toronto, and Division of Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada. A. Schulze reports grants from Aglea BioTherapeutics during the conduct of the study. I. Tein reports grants from Myositis Association during the conduct of the study; grants from Foundation for Prader Willi Research, grants from Cure JM Foundation, grants from Connaught Global Challenge Award, University of Toronto, grants from Canadian Institutes of Health Research Planning and Dissemination Grant outside the submitted work. The remaining authors have no conflicts to disclose. Address correspondence to Dr. B.M. Feldman, The Hospital for Sick Children Research Institute, 555 University Ave., Toronto, ON M5G 1X8, Canada. Email: brian.feldman@sickkids.ca. Accepted for publication July 13, 2020.
Abstract
Objective To evaluate the feasibility of studying creatine in juvenile dermatomyositis ( JDM). Secondary objectives were to determine the effect of creatine on muscle function and metabolism, aerobic capacity, fatigue, physical activity, and quality of life (QOL), as well as its safety.
Methods We conducted a 6-month, double-blind, randomized, multiple-baseline design; patients were assigned to creatine or placebo. Feasibility was assessed using attended study visits, completed study procedures, and adherence. Muscle function, aerobic capacity, and muscle strength were assessed with standardized exercise tests. Muscle metabolism was assessed using a 31-Phosphorus Magnetic Resonance Spectroscopy protocol. Fatigue, physical activity, and QOL were assessed by questionnaires. Statistical significance was estimated using a randomization (permutation) test. Changes in outcome measures taken at baseline and end-of-study were calculated using paired t-tests.
Results Median (range) adherence to the study drug was 88.5% (20.5–95.5%) and the proportion of subjects with 80% adherence or higher was 76.9%. There were no missed study visits. There were no statistically significant changes in muscle function, strength, aerobic capacity, disease activity, fatigue, physical activity, or QOL while subjects were receiving creatine compared to placebo. There were statistically significant adaptations in muscle metabolism (e.g., decrease in change in muscle pH following exercise, and decrease in phosphate/phosphocreatine ratio) at the end-of-study compared to baseline. There were no significant adverse effects.
Conclusion Creatine supplementation in children with JDM is feasible to study, and is safe and well-tolerated; it may lead to improvements in muscle metabolism.
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In this issue
The Journal of Rheumatology
Vol. 48, Issue 4
1 Apr 2021
The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis: A Randomized, Double-blind, Placebo-controlled Feasibility Study
Saunya Dover, Samantha Stephens, Jane E. Schneiderman, Eleanor Pullenayegum, Greg D. Wells, Deborah M. Levy, Jo-Anne Marcuz, Kristi Whitney, Andreas Schulze, Ingrid Tein, Brian M. Feldman
The Journal of Rheumatology Aug 2020, jrheum.191375; DOI: 10.3899/jrheum.191375
The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis: A Randomized, Double-blind, Placebo-controlled Feasibility Study
Saunya Dover, Samantha Stephens, Jane E. Schneiderman, Eleanor Pullenayegum, Greg D. Wells, Deborah M. Levy, Jo-Anne Marcuz, Kristi Whitney, Andreas Schulze, Ingrid Tein, Brian M. Feldman
The Journal of Rheumatology Aug 2020, jrheum.191375; DOI: 10.3899/jrheum.191375
