Daily prednisone treatment in duchenne muscular dystrophy in southwest china

Muscle and Nerve

Volumn 52, Issue 6, 2015, Pages 1001-1007

  Hu, Jun ^a,b,c,d,e   Ye, Yuanzhen ^b,c,d,e   Kong, Min ^b,c,d,e   Hong, Siqi ^b,c,d,e   Cheng, Li ^b,c,d   Wang, Qiao ^b,c,d,e   Qin, Jiaqiang ^b,c,d,e   Zou, Lin ^b,c,d,e   Jiang, Li ^b,c,d,e  

^a FUJIAN MEDICAL UNIVERSITY   (China)

^b BEIJING UNIVERSITY OF POSTS AND TELECOMMUNICATIONS   (China)

^c CHONGQING KEY LABORATORY OF PEDIATRICS   (China)

^d Technology Cooperation Center for Child Development and Disorders   (China)

^e CHONGQING MEDICAL UNIVERSITY   (China)

Author keywords

Children; Duchenne muscular dystrophy; Efficacy; Prednisone; Safety

Indexed keywords

PLACEBO; PREDNISONE; ANTIINFLAMMATORY AGENT; CREATINE KINASE;

ACNE; ARTICLE; BODY HEIGHT; BODY MASS; BODY WEIGHT; CHILD; CHINA; CLINICAL EVALUATION; CONTROLLED STUDY; CUSHINGOID SYNDROME; DIASTOLIC BLOOD PRESSURE; DRUG EFFICACY; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; FOLLOW UP; FUNCTIONAL STATUS; HAIR GROWTH; HUMAN; INCREASED APPETITE; MAJOR CLINICAL STUDY; MALE; MUSCLE FUNCTION; MUSCLE STRENGTH; PRESCHOOL CHILD; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; SIDE EFFECT; ULTRASOUND; WEIGHT GAIN; DRUG EFFECTS; ECHOGRAPHY; FEMALE; METABOLISM; MUSCULAR DYSTROPHY, DUCHENNE; NONPARAMETRIC TEST; PSYCHOLOGY; QUESTIONNAIRE; RETROSPECTIVE STUDY; SKELETAL MUSCLE; TREATMENT OUTCOME;

ANTI-INFLAMMATORY AGENTS; CHILD; CHILD, PRESCHOOL; CHINA; CREATINE KINASE; FEMALE; FOLLOW-UP STUDIES; HUMANS; MALE; MUSCLE STRENGTH; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; PREDNISONE; QUALITY OF LIFE; RETROSPECTIVE STUDIES; STATISTICS, NONPARAMETRIC; SURVEYS AND QUESTIONNAIRES; TREATMENT OUTCOME;

EID: 84955210449     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.24665     Document Type: Article

References (26)

1. Emery AE. Population frequencies of inherited neuromuscular diseases-a world survey. Neuromuscul Disord 1991; 1: 19-29.
2. Kunkel LM, Hejtmancik JF, Caskey CT, Speer A, Monaco AP, Middlesworth W, et al. Analysis of deletions in DNA from patients with Becker and Duchenne muscular dystrophy. Nature 1986; 322: 73-77.
3. Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Florence J, et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy. Neurology 1989; 39: 475-481.
4. Mendell JR, Moxley RT, Griggs RC, Brooke MH, Fenichel GM, Miller JP, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 1989; 320: 1592-1597.
5. Griggs RC, Moxley RT III, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, et al. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Arch Neurol 1991; 48: 383-388.
6. Bäckman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord 1995; 5: 233-241.
7. Beenakker EA, Fock JM, van Tol MJ, Maurits NM, Koopman HM, Brouwer OF, et al. Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial. Arch Neurol 2005; 62: 128-132.
8. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev 2008; 23: CD003725.
9. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9: 77-93.
10. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010; 9: 177-189.
11. Moxley RT III, Ashwal S, Pandya S, Connolly A, Florence J, Mathews K, et al. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005; 64: 13-20.
12. Florence JM, Pandya S, King WM, Robison JD, Signore LC, Wentzell M, et al. Clinical trials in Duchenne dystrophy. Standardization and reliability of evaluation procedures. Phys Ther 1984; 64: 41-45.
13. Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve 1983; 6: 91-103.
14. Hu J, Jiang L, Hong S, Cheng L, Kong M, Ye Y. Reliability and validity of the Chinese version of the pediatric quality of life inventory™ (PedsQL™) 3.0 neuromuscular module in children with Duchenne muscular dystrophy. Health Qual Life Outcomes 2013; 11: 47.
15. Jansen M, van Alfen N, Nijhuis van der Sanden MW, van Dijk JP, Pillen S, de Groot IJ. Quantitative muscle ultrasound is a promising longitudinal follow-up tool in Duchenne muscular dystrophy. Neuromuscul Disord 2012; 22: 306-317.
16. Maurits NM, Beenakker EA, van Schaik DE, Fock JM, van der Hoeven JH. Muscle ultrasound in children: normal values and application to neuromuscular disorders. Ultrasound Med Biol 2004; 30: 1017-1027.
17. Scholten RR, Pillen S, Verrips A, Zwarts MJ. Quantitative ultrasonography of skeletal muscles in children: normal values. Muscle Nerve 2003; 27: 693-698.
18. Pillen S, Verrips A, van Alfen N, Arts IM, Sie LT, Zwarts MJ. Quantitative skeletal muscle ultrasound: diagnostic value in childhood neuromuscular disease. Neuromuscul Disord 2007; 17: 509-516.
19. Iannaccone ST, Hynan LS, Morton A, Buchanan R, Limbers CA, Varni JW, et al. The PedsQL in pediatric patients with Spinal Muscular Atrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Generic Core Scales and Neuromuscular Module. Neuromuscul Disord 2009; 19: 805-812.
20. Uzark K, King E, Cripe L, Spicer R, Sage J, Kinnett K, et al. Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. Pediatrics 2012; 130: e1559-e1566.
21. Moxley RT III, Pandya S, Ciafaloni E, Fox DJ, Campbell K. Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. J Child Neurol 2010; 25: 1116-1129.
22. Urganci N, Arapoğlu M, Serdaroğlu P, Nuhoğlu A. Incidental raised transaminases: a clue to muscle disease. Ann Trop Paediatr 2006; 26: 345-348.
23. Kim HK, Laor T, Horn PS, Racadio JM, Wong B, Dardzinski BJ. T2 mapping in Duchenne muscular dystrophy: distribution of disease activity and correlation with clinical assessments. Radiology 2010; 255: 899-908.
24. Zatz M, Rapaport D, Vainzof M, Passos-Bueno MR, Bortolini ER, Pavanello Rde C, et al. Serum creatine-kinase (CK) and pyruvate-kinase (PK) activities in Duchenne (DMD) as compared with Becker (BMD) muscular dystrophy. J Neurol Sci 1991; 102: 190-196.
25. Malm C, Nyberg P, Engstrom M, Sjodin B, Lenkei R, Ekblom B, et al. Immunological changes in human skeletal muscle and blood after eccentric exercise and multiple biopsies. J Physiol 2000; 529: 243-262.
26. Zaidman CM, Connolly AM, Malkus EC, Florence JM, Pestronk A. Quantitative ultrasound using backscatter analysis in Duchenne and Becker muscular dystrophy. Neuromuscul Disord 2010; 20: 805-809.