Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade

Neuromuscular Disorders - Tập 16 - Trang 249-255 - 2006
W.D. Biggar1,2,3, V.A. Harris1, L. Eliasoph1,3, B. Alman1,2,3
1Bloorview Kids Rehab, 150 Kilgour Road, Toronto, Ont., Canada M4G IR8
2Hospital for Sick Children, Toronto, Ont., Canada
3University of Toronto, Toronto, Ont., Canada

Tài liệu tham khảo

McDonald, 1995, Profiles of neuromuscular diseases. Duchenne muscular dystrophy, Am J Phys Med Rehabil, 74, S70, 10.1097/00002060-199509001-00003 Brooke, 1983, Clinical investigation in duchenne dystrophy: 2. Determination of the ‘power’ of therapeutic trials based on the natural history, Muscle Nerve, 6, 91, 10.1002/mus.880060204 Emery, 1993 Dubowitz, 1995 Angelini, 1986, Natural history of muscular dystrophy inferred from a multicenter trial and the results of a dietetic trial, Minerva Pediatr, 38, 645 Rideau, 1981, Respiratory function in the muscular dystrophies, Muscle Nerve, 4, 155, 10.1002/mus.880040213 Griggs, 1981, Evaluation of pulmonary function in neuromuscular disease, Arch Neurol, 38, 9, 10.1001/archneur.1981.00510010035004 Polgar, 1971, Pulmonary function testing in children Hahn, 1997, Clinical implications of maximal respiratory pressure determinations for individuals with Duchenne muscular dystrophy, Arch Phys Med Rehabil, 78, 1, 10.1016/S0003-9993(97)90001-0 Baydur, 1990, Decline in respiratory function and experience with long-term assisted ventilation in advanced Duchenne's muscular dystrophy, Chest, 97, 884, 10.1378/chest.97.4.884 Wilkins, 1976, The patterns of spinal deformity in Duchenne muscular dystrophy, J Bone Joint Surg Am, 58, 24, 10.2106/00004623-197658010-00004 Nigro, 1990, The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy, Int J Cardiol, 26, 271, 10.1016/0167-5273(90)90082-G Nigro, 1997, 42nd ENMC Sponsored international workshop: X-linked cardiomyopathies. 21–23 June 1996, Naarden, The Netherlands, Neuromuscul Disord, 7, 397, 10.1016/S0960-8966(97)87156-8 Nigro G. Cardiomyopathies associated with muscular dystrophies. In: Engel AG, Franzini-Armstrong C, editors. Myology: basic and clinical. 3rd ed. New York: McGraw-Hill; 2004 [chapter 45]. Eagle, 2002, Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation, Neuromuscul Disord, 12, 926, 10.1016/S0960-8966(02)00140-2 Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev 2004;(2): CD003725. Wong, 2002, Corticosteroids in Duchenne muscular dystrophy: a reappraisal, J Child Neurol, 17, 183, 10.1177/088307380201700306 Campbell, 2003, Deflazacort for the treatment of Duchenne Dystrophy: a systematic review, BMC Neurol, 3, 7, 10.1186/1471-2377-3-7 Moxley, 2005, 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, 64, 13, 10.1212/01.WNL.0000148485.00049.B7 Bushby, 2004, Report on the 124th ENMC international workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2–4 April 2004, Naarden, The Netherlands, Neuromuscul Disord, 14, 526, 10.1016/j.nmd.2004.05.006 Biggar, 2001, Deflazacort treatment of Duchenne muscular dystrophy, J Pediatr, 138, 45, 10.1067/mpd.2001.109601 Silversides, 2003, Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy, Am J Cardiol, 91, 769, 10.1016/S0002-9149(02)03429-X Alman, 2004, Steroid treatment and the development of scoliosis in males with duchenne muscular dystrophy, J Bone Joint Surg Am, 86-A, 519, 10.2106/00004623-200403000-00009 Biggar, 2004, Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols, Neuromuscul Disord, 14, 476, 10.1016/j.nmd.2004.05.001 Quinones, 1974, Echocardiographic assessment of left ventricular function with special reference to normalized velocities, Circulation, 50, 42, 10.1161/01.CIR.50.1.42 Stamm, 1982, Two-dimensional echocardiographic measurement of left ventricular ejection fraction: prospective analysis of what constitutes an adequate determination, Am Heart J, 104, 136, 10.1016/0002-8703(82)90651-2 Gutgesell, 1977, Evaluation of left ventricular size and function by echocardiography. Results in normal children, Circulation, 56, 457, 10.1161/01.CIR.56.3.457 SPSS. Version 12.0.1. SPSS 12.0 command syntax reference; 2003. Schara, 2001, Long-term steroid therapy in Duchenne muscular dystrophy-positive results versus side effects, J Clin Neuromuscul Dis, 2, 179, 10.1097/00131402-200106000-00002 Dubrovsky, 1999, Deflazacort treatment and respiratory function in Duchenne muscular dystrophy, Neurology, 52A, 544 Biggar WD, Harris VA, Campbell KA, Vajsar J. Improved pulmonary function in boys with Duchenne muscular dystrophy when Deflazacort treatment is started in the second decade. WMS; September 2005. Ahmad, 1978, Echocardiographic assessment of left ventricular function in Duchenne's muscular dystrophy, Br Heart J, 40, 734, 10.1136/hrt.40.7.734 D'Orsogna, 1988, Cardiomyopathy of Duchenne muscular dystrophy, Pediatr Cardiol, 9, 205, 10.1007/BF02078410 De Kermadec, 1994, Prevalence of left ventricular systolic dysfunction in Duchenne muscular dystrophy: an echocardiographic study, Am Heart J, 127, 618, 10.1016/0002-8703(94)90672-6 Farah, 1980, Echocardiographic evaluation of left ventricular function in Duchenne's muscular dystrophy, Am J Med, 69, 248, 10.1016/0002-9343(80)90385-X Ramaciotti, 2002, Assessment of cardiac function in adolescents with Duchenne muscular dystrophy: importance of neurohormones, J Child Neurol, 17, 191, 10.1177/088307380201700307 Perloff, 1967, The distinctive electrocardiogram of Duchenne's progressive muscular dystrophy. An electrocardiographic–pathologic correlative study, Am J Med, 42, 179, 10.1016/0002-9343(67)90017-4 Perloff, 1984, Alterations in regional myocardial metabolism, perfusion, and wall motion in Duchenne muscular dystrophy studied by radionuclide imaging, Circulation, 69, 33, 10.1161/01.CIR.69.1.33 Yamamoto, 1988, A comparative study of thallium-201 single-photon emission computed tomography and electrocardiography in Duchenne and other types of muscular dystrophy, Am J Cardiol, 61, 836, 10.1016/0002-9149(88)91076-4 Buyse, 2003, Idebenone treatment in Friedreich's ataxia: neurological, cardiac, and biochemical monitoring, Neurology, 60, 1679, 10.1212/01.WNL.0000068549.52812.0F Markham, 2004, The evaluation of ventricular function by cardiac magnetic resonance imaging in neuromuscular disorders, Neuromuscul Disord, 14, 577 Alman, 1999, Pelvic obliquity after fusion of the spine in duchenne muscular dystrophy, J Bone Joint Surg Br, 81, 821, 10.1302/0301-620X.81B5.9383 Weimann, 1983, Surgical stabilization of the spine in duchenne muscular dystrophy, Spine, 8, 776, 10.1097/00007632-198310000-00015 Mayfield, 1981, Spine deformity subsequent to acquired childhood spinal cord injury, J Bone Joint Surg Am, 63, 1401, 10.2106/00004623-198163090-00006 Lancourt, 1981, Paralytic spinal deformity following traumatic spinal-cord injury in children and adolescents, J Bone Joint Surg Am, 63, 47, 10.2106/00004623-198163010-00006 Zata, 1986, RTB Benign Duchenne muscular dystrophy in a patient with growth hormone deficiency; a five year follow-up, Am J Genet, 24, 567, 10.1002/ajmg.1320240323 Zatz, 1988, Relation between height and clinical course in Duchenne muscular dystrophy, Am J Med Genet, 29, 405, 10.1002/ajmg.1320290223