Interventions for preventing and treating cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy

Cochrane Database of Systematic Reviews

Volumn 2018, Issue 10, 2018, Pages

  Bourke, John P ^a   Bueser, Teofila ^b   Quinlivan, Rosaline ^c  

^a FREEMAN HOSPITAL   (United Kingdom)

^b KING'S COLLEGE LONDON   (United Kingdom)

^c NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANGIOTENSIN RECEPTOR ANTAGONIST; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; EPLERENONE; GROWTH HORMONE; IDEBENONE; LISINOPRIL; LOSARTAN; PERINDOPRIL; ANTIHYPERTENSIVE AGENT; CARDIOVASCULAR AGENT; HUMAN GROWTH HORMONE; PLACEBO; UBIQUINONE;

ALLERGIC REACTION; BECKER MUSCULAR DYSTROPHY; CONGESTIVE CARDIOMYOPATHY; DETERIORATION; DRUG SAFETY; DRUG WITHDRAWAL; DUCHENNE MUSCULAR DYSTROPHY; EVIDENCE BASED MEDICINE; FOLLOW UP; HEART DISEASE; HEART FUNCTION; HEART LEFT VENTRICLE EJECTION FRACTION; HEART PROTECTION; HUMAN; INTERVENTION STUDY; META ANALYSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; OUTCOME ASSESSMENT; PATIENT CARE; PREMEDICATION; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RISK ASSESSMENT; SIDE EFFECT; SYSTEMATIC REVIEW; TREATMENT DURATION; X LINKED DILATED CARDIOMYOPATHY; ADOLESCENT; ADULT; ANALOGS AND DERIVATIVES; CARDIOMYOPATHY; CHILD; COMPLICATION; CONTROLLED CLINICAL TRIAL (TOPIC); DISEASE EXACERBATION; DRUG EFFECT; HEART STROKE VOLUME; MALE; YOUNG ADULT;

ADOLESCENT; ADULT; ANGIOTENSIN RECEPTOR ANTAGONISTS; ANGIOTENSIN-CONVERTING ENZYME INHIBITORS; ANTIHYPERTENSIVE AGENTS; CARDIOMYOPATHIES; CARDIOMYOPATHY, DILATED; CARDIOVASCULAR AGENTS; CHILD; DISEASE PROGRESSION; EPLERENONE; HUMAN GROWTH HORMONE; HUMANS; LISINOPRIL; LOSARTAN; MALE; MUSCULAR DYSTROPHY, DUCHENNE; NON-RANDOMIZED CONTROLLED TRIALS AS TOPIC; PERINDOPRIL; PLACEBOS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; STROKE VOLUME; UBIQUINONE; YOUNG ADULT;

EID: 84965073258     PISSN: None     EISSN: 1469493X     Source Type: Journal    
DOI: 10.1002/14651858.CD009068.pub3     Document Type: Review

References (137)

1. Allen HD, Flanigan KM, Thrush PT, Viollet-Callendret L, Dvorchik I, Yin H, et al. A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in Duchenne muscular dystrophy. PLOS Currents Muscular Dystrophy 2013;5:1-13. [PMCID: PMC3871420; PUBMED: 24459612]9293899
2. NCT01982695 . Cardiomyopathy in DMD: lisinopril vs. losartan [Compare efficacy of the angiotensin converting enzyme inhibitor (ACEI) lisinopril with angiotensin II receptor antagonist losartan (ARB) for the cardiomyopathy of Duchenne muscular dystrophy]. clinicaltrials.gov/ct2/show/study/NCT01982695 First received: 29 October 2013; results first received 14 August 2015. 9293900
3. Buyse GM, Goemans N, van den Hauwe M, Thijs D, de Groot IJM, Schara U, et al. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a randomized placebo-controlled trial. Neuromuscular Disorders 2011;21:396-405. [PUBMED: 21435876]9293902
4. Cittadini A, Ines Comi L, Longobardi S, Rocco Petretta V, Casaburi C, Passamano L, et al. A preliminary randomized study of growth hormone administration in Becker and Duchenne muscular dystrophies. European Heart Journal 2003;24(7):664-72. [PUBMED: 12657225]9293904
5. Duboc D, Meune C, Lerebours G, Devaux J-Y, Vaksmann G, Bécane H-M. Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy. Journal of the American College of Cardiology 2005;45(6):855-7. [PUBMED: 15766818]9293906
6. Raman S, Hor K, Mazur W, Halnon N, Kissel J, He X, et al. Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial. Lancet Neurology 2015;14(2):153-61. [PUBMED: 25554404]9293908
7. Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, et al. PTC124-GD-007-DMD STUDY GROUP. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle & Nerve 2014;50(4):477-87. 9293910
8. Duboc D, Meune C, Pierre B, Wahbi K, Eymard B, Toutain T, et al. Perindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years follow-up. American Heart Journal 2007;154(3):596-602. [PUBMED: 17719312]9293912
9. Folkers K, Wolaniuk J, Simonsen R, Morishita M, Vadhanavikit S. Biochemical rationale and the cardiac response of patients with muscle disease to therapy with coenzyme Q10. Proceedings of the National Academy of Sciences of the United States of America 1985;82(13):4513-16. 9293914
10. Ishikawa Y, Back J, Ishikawa Y, Minami R. A management trial for Duchenne cardiomyopathy. American Journal of Physical Medicine & Rehabilitation 1995;74:345-50. 9293916
11. Kajimoto H, Ishigaki K, Okumura K, Tomimatsu H, Nakazawa M, Saito K, et al. Beta-blocker therapy for cardiac dysfunction in patients with muscular dystrophy. Circulation Journal 2006;70(8):991-4. 9293918
12. Matsumura T, Tamura T, Kuru S, Kikuchi Y, Mitsuru K. Carvedilol can prevent cardiac events in Duchenne muscular dystrophy. Internal Medicine 2010;49(14):1357-63. 9293920
13. Mendell J, Rodino-Klapac L, Sahenk Z, Roush K, Bird L, Lowes LP, et al. Eteplirsen Study Group. Eteplirsen for the treatment of DMD. Annals of Neurology 2013;74(5):637-47. 9293922
14. Rhodes J, Margossian R, Darras BT, Colan SD, Jenkins KJ, Geva T, Powell AJ. Safety and efficacy of carvedilol therapy for patients with dilated cardiomyopathy secondary to muscular dystrophy. Pediatric Cardiology 2008;29(2):343-51. 9293924
15. Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, et al. Safety and efficacy of Drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised placebo controlled phase II study. Lancet Neurology 2014;13(10):987-96. 9293926
16. EUCTR2008-007236-18-IT . Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of cardiac magnetic resonance study. clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007236-18 (first received 11 December 2008). 9293928
17. Leung DG, Herzka DA, Thompson WR, He B, Bibat G, Tennekoon G, et al. Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy. Annals of Neurology 2014;76(4):541-9. 9293930
18. NCT01168908 . Revatio for heart disease in Duchenne muscular dystrophy and Becker muscular dystrophy (REVERSE-DBMD). clinicaltrials.gov/show/NCT01168908 (first received 23 July 2010). 9293931
19. IRCT2015070223018N1 . Effect of Q10 coenzyme in improving cardiac function [A study on the effect of Q10 coenzyme via tissue doppler method in improving cardiac function in patients with early Duchenne cardiomyopathy aged 6-10 years]. en.irct.ir/trial/19746 (first received 11 August 2015). 9293933
20. Salehi F, Zeinaloo A, Riasi HR, Shamloo AS. Effectiveness of Coenzyme Q10 on echocardiographic parameters of patients with Duchenne muscular dystrophy. Electronic Physician 2017;9(3):3896-3904. 9293934
21. NCT01603407 . Finding the optimum regimen for Duchenne muscular dystrophy (FOR-DMD) [Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen]. https://clinicaltrials.gov/ct2/show/NCT01603407 (first received 23 May 2012). 9293936
22. ISRCTN50395346 . A double-blind randomised multi-centre, placebo-controlled trial of combined angiotensin converting enzyme-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with Duchenne muscular dystrophy without echo-detectable left ventricular dysfunction. isrctn.com/ISRCTN50395346 (first received 13 August 2007). 9293938
23. NCT00606775 . The preventive efficacy of carvedilol on cardiac dysfunction in Duchenne muscular dystrophy [Carvedilol for the prevention of minor cardiac damage and cardiac function in Duchenne muscular dystrophy]. clinicaltrials.gov/show/NCT00606775 (first received 5 February 2008). 9293940
24. NCT00819845 . Ramipril versus carvedilol in Duchenne and Becker patients [Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of cardiac magnetic resonance study]. clinicaltrials.gov/show/NCT00819845 (first received 9 January 2009). 9293942
25. NCT01126697 . Clinical trial of coenzyme Q10 and lisinopril in muscular dystrophies [PITT0908:clinical trial of coenzyme Q10 and lisinopril in muscular dystrophies]. clinicaltrials.gov/show/NCT01126697 (first received 20 May 2010). 9293944
26. NCT01350154 . Effect of modulating the nNOS system on cardiac, muscular and cognitive function in Becker muscular dystrophy patients [Does modulation of the nNOS system in patients with muscular dystrophy and defect nNOS signalling affect cardiac, muscular or cognitive function?]. clinicaltrials.gov/show/NCT01350154 (first received 9 May 2011). 9293946
27. NCT01648634 . Nebivolol for the prevention of left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy (NEBIDYS) [A randomized, double-blind, placebo-controlled, multi-center study to examine the effect of nebivolol, a beta-blockade drug, for the prevention of ventricular systolic dysfunction in patients with Duchenne muscular dystrophy]. clinicaltrials.gov/show/NCT01648634 (first received 24 July 2012). 9293948
28. NCT02354352 . Therapeutic potential for aldosterone inhibition in Duchenne muscular dystrophy. https://clinicaltrials.gov/ct2/show/NCT02354352 (first received 21 August 2018). 9293950
29. NCT02432885 . Myocardial fibrosis progression in Duchenne and Becker muscular dystrophy - ACE inhibitor therapy trial [Myocardial fibrosis progression in Duchenne and Becker muscular dystrophy - angiotensin-converting-enzyme (ACE) inhibitor therapy]. clinicaltrials.gov/show/NCT02432885 (first received 4 May 2015). 9293952
30. NCT02485938 . HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) (HOPE) [A randomized, open-label study of the safety and efficacy of multi-vessel intracoronary delivery of allogeneic cardiosphere-derived cells in patients with cardiomyopathy secondary to Duchenne muscular dystrophy]. clinicaltrials.gov/show/NCT02485938 (first received 30 June 2015). 9293954
31. NCT03340675 . Oral ifetroban in subjects with Duchenne muscular dystrophy (DMD) [A randomized, double-blind, placebo-controlled, multiple dose study to determine the safety, pharmacokinetics and efficacy of oral ifetroban in subjects with Duchenne muscular dystrophy (DMD)]. clinicaltrials.gov/show/NCT03340675 (first received 13 November 2017). 9293956
32. NCT03406780 . A study of CAP-1002 in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (HOPE-2) [A phase 2, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of intravenous delivery of allogeneic cardiosphere-derived cells in subjects with Duchenne muscular dystrophy]. https://clinicaltrials.gov/ct2/show/NCT03406780 (first received 23 January 2018). 9293958
33. NCT03439670 . A study to assess the efficacy and safety of vamorolone in boys with Duchenne muscular dystrophy (DMD) [A phase IIb randomized, double-blind, parallel group, placebo- and active-controlled study with double-blind extension to assess the efficacy and safety of vamorolone in ambulant boys with Duchenne muscular dystrophy (DMD)]. https://clinicaltrials.gov/ct2/show/NCT03439670 (first received 24 July 2018). 9293960
34. Aartsma-Rus A, Van Ommen GJ, Kaplan JC. Innovating therapies for muscle diseases. Handbook of Clinical Neurology 2013;113:1497-501.
35. Abdel-Salam Z, Rayan M, Saleh A, Abdel-Barr MG, Hussain M, Nammas W. I(f) current inhibitor ivabradine in patients with idiopathic dilated cardiomyopathy: impact on the exercise tolerance and quality of life. Cardiology Journal 2014;22(2):227-32.
36. Abraham WT, Aggarwal S, Prabhu SD, Cecere R, Pamboukian SV, Bank AJ, et al. C-Pulse Trial Group. Ambulatory extra-aortic counterpulsation in patients with moderate to severe chronic heart failure. JACC: Heart Failure 2014;2(5):526-33.
37. Adorisio R, Catarutti N, D'Amico A, Chinali M, Baban A, Iorio FS, et al. Heart rate reduction strategy with Ivabradine in reducing acute heart failure in Duchenne dilated cardiomyopathy. European Heart Journal 2017;38(Suppl 1):5275.
38. Andrikopoulos G, Kourouklis S, Trika C, Tzeis S, Rassias I, Papademetriou C, et al. Cardiac resynchronisation therapy in Becker muscular dystrophy. Hellenic Journal of Cardiology 2013;54(3):227-9.
39. Angelini C, Fanin M, Freda MP, Martinello F, Miorin M, Melacini P, et al. Prognostic factors in mild dystrophinopathies. Journal of the Neurological Sciences 1996;142(1-2):70-8.
40. Backman E, Nylander E. The heart in Duchenne muscular dystrophy: a non-invasive study. European Heart Journal 1992;13(9):1239-44.
41. Barber B, Andrews J, Lu Z, West N, Meaney J, Price E, et al. Oral corticosteroids and onset of cardiomyopathy in Duchenne muscular dystrophy. Journal of Pediatrics 2013;163(4):1080-4.
42. Becker PE, Kiener F. Eine neue X-chromasomale Muskeldystrophie. Archiv fur Psychiatrie und Nervenkrankheiten 1955;193:427-48.
43. Bies RD, Friedman D, Roberts R, Perryman MB, Caskey CT. Expression and localization of dystrophin in human cardiac Purkinje fibres. Circulation 1992;86(1):147-53.
44. Bigggar WD, Harris VA, Eliasoph L, Alman B. Long term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscular Disorders 2006;16(4):249-55.
45. Black MC, Schumer EM, Rogers M, Trivedi J, Slaughter MS. Sunshine Heart C-Pulse: device for NYHA III and ambulatory class IV heart failure. Future Cardiolology 2016;12(5):521-31.
46. Bleeker GB, Kaandorp TA, Lamb HJ, Boersma E, Steendijk P, de Roos A, et al. Effect of posterolateral scar tissue on clinical and echocardiographic improvement after cardiac resynchronization therapy. Circulation 2006;113(7):969-76.
47. Burnett H, Earley A, Voors AA, Senni M, McMurray JJ, Deschaseaux C, et al. Thirty years of evidence on the efficacy of drug treatments for chronic heart failure with reduced ejection fraction: a network meta-analysis. Circulation. Heart Failure 2017;10(1):e003529.
48. Bushby KMD, Thambyayah M, Gardner-Medwin D. Prevalence and incidence of Becker muscular dystrophy. Lancet 1991;337(8748):1022-4.
49. Bushby K, Goodship JA, Nicholson L, Johnson M, Haggerty ID, Gardner-Medwin D. Variability in clinical, genetic and protein abnormalities in manifesting carriers of Duchenne and Becker muscular dystrophy. Neuromuscular Disorders 1993;3(1):57-64.
50. Casazza F, Brambilla G, Salvato A, Morandi L, Gronda E, Bonacina E. Cardiac transplantation in Becker muscular dystrophy. Journal of Neurology 1988;235(8):496-8.
51. Cevik C, Nugent K, Perez-Verdia A, Fish RD. Prophylactic implantation of cardioverter defibrillator in idiopathic non-ischaemic cardiomyopathy for the primary prevention of death: a narrative review. Clinical Cardiology 2010;33(5):254-60.
52. Cicoira M, Zanolla L, Franceschini L, Rossi A, Golia G, Zeni P, et al. Relation of aldosterone 'escape' despite angiotensin-converting enzyme inhibitor administration to impaired exercise capacity in chronic congestive heart failure secondary to ischaemic or idiopathic dilated cardiomyopathy. American Journal of Cardiology 2002;89(4):403-7.
53. Corrado G, Lissoni A, Beretta S, Terenghi L, Tadeo G, Foglia-Manzillo G, et al. Prognostic value of echocardiograms, ventricular late potentials, ventricular arrhythmias and left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy. American Journal of Cardiology 2002;89(7):838-41.
54. Danialou G, Comtois AS, Dudley R, Karpati G, Vincent G, Des Rosiers C, et al. Dystrophin-deficient cardiomyocytes are abnormally vulnerable to mechanical stress-induced contractile failure and injury. FASEB Journal 2001;15(9):1655-7.
55. De Kermadec JM, Bécane HM, Chénard A, Tertrain F, Weiss Y. Prevalence of left ventricular systolic dysfunction in Duchenne muscular dystrophy: an electrocardiographic study. American Heart Journal 1994;127(3):618-23.
56. De Visser M, de Voogt WG, la Rivière GV. The heart in Becker muscular dystrophy, fascioscapulohumeral muscular dystrophy and Bethlem myopathy. Muscle & Nerve 1992;15(5):591-6.
57. Donofrio PD, Challa VR, Hackshaw BT, Mills SA, Cordell R. Cardiac transplant in a patient with muscular dystrophy and cardiomyopathy. Archives of Neurology 1989;46(6):705-7.
58. Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R, Bushby K. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscular Disorders 2002;12(10):926-9.
59. Emery AEH, Skinner R. Clinical studies in benign (Becker type) X-linked muscular dystrophy. Clinical Genetics 1976;10(4):189-201.
60. Emery AEH, Muntoni F. Duchenne Muscular Dystrophy. 3rd Edition. Oxford: Oxford Medical Publications, 2003.
61. Fararolo R, Peradejord M, Berlotti A, Diez M, Favaloro L, Gomez C, et al. Results of heart transplantation: 16 years experience in a center in Argentina. Transplantation Proceedings 2010;42(1):321-3.
62. Fayssoil A, Orlikowski D, Nardi O, Annane D. Complete atrioventricular block in Duchenne muscular dystrophy. Europace 2008;10(11):1351-2.
63. Ferlini A, Sewry C, Melis MA, Mattedu A, Muntoni F. X-linked dilated cardiomyopathy and the dystrophin gene. Neuromuscular Disorders 1999;9(5):339-46.
64. Finserer J, Stöllberger C. The heart in human dystrophinopathies. Cardiology 2003;99(1):1-19.
65. Gardner RJ, Bobrow M, Roberts RG. The identification of point mutations in Duchenne muscular dystrophy patients by using reverse transcription PCR and the protein truncation test. American Journal of Human Genetics 1989;57(2):311-20.
66. Globus JH. The pathological findings in the heart muscle in progressive muscular dystrophy. Archives of Neurology and Psychiatry 1923;9(1):59-72.
67. McMaster University (developed by Evidence Prime, Inc.). GRADEpro GDT: GRADEpro Guideline Development Tool. Version accessed 20 June 2018. Hamilton (ON): McMaster University (developed by Evidence Prime, Inc.), 2015.
68. Griffin JL, Williams HJ, Sang E, Clarke K, Rae C, Nicholson JK. Metabolic profiling of genetic disorders: a multi-tissue (1)H nuclear magnetic resonance spectroscopic and pattern recognition study into dystrophic tissue. Analytical Biochemistry 2001;293(1):16-21.
69. Heran BS, Musini VM, Bassett K, Taylor RS, Wright JM. Angiotensin receptor blockers for heart failure. Cochrane Database of Systematic Reviews 2012, Issue 4. [DOI: 10.1002/14651858.CD003040.pub2]
70. Heymsfield SB, McNish T, Perkins JV, Felner JM. Sequence of cardiac changes in Duchenne muscular dystrophy. American Heart Journal 1978;95(3):283-94.
71. Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 (updated March 2011). The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
72. Holloway SM, Wilcox DE, Wilcox A, Dean JC, Berg JN, Goudie DR, et al. Life expectancy and death from cardiomyopathy amongst carriers of Duchenne and Becker muscular dystrophy in Scotland. Heart 2008;94(5):633-6.
73. Hoogerwaard EM, De Voogt WG, Wilde AA, Van der Wouw PA, Bakker E, Van Ommen GJ, et al. Evolution of cardiac abnormalities in Becker muscular dystrophy over a 13-year period. Journal of Neurology 1997;244(10):657-63.
74. Hoogerwaard EM, Van der Wouw PA, Wilde AA, Bakker E, Ippel PF, Oosterwijk JC, et al. Cardiac involvement in carriers of Duchenne and Becker muscular dystrophy. Neuromuscular Disorders 1999;9(5):347-51.
75. Hor KN, Wansapura JP, Al-Khalidi HR, Gottliebson WM, Taylor MD, Czosek RJ, et al. Presence of mechanical dyssynchrony in Duchenne muscular dystrophy. Journal of Cardiovascular Magnetic Resonance 2011;13(1):12. [DOI: 10.1186/1532-429X-13-12]
76. Hussein G, Mansour L, Ghafor H, Mostafa F, Fawaz L. Short-term effects of corticosteroid therapy on cardiac and skeletal muscles in muscular dystrophies. Journal of Investigative Medicine 2014;62(6):875-9.
77. Iodice F, Testa G, Averardi M, Brancaccio G, Amodeo A, Cogo P. Implantation of a left ventricular assist device as a destination therapy in Duchenne muscular dystrophy patients with end stage cardiac failure: management and lessons learned. Neuromuscular Disorders 2015;25(1):19-23.
78. Ishigaki C, Patria SI, Nishio H, Yoshioka A, Matsuo M. Early cardiac failure in a child with Becker muscular dystrophy is due to an abnormally low amount of dystrophin transcript lacking exon 13. Acta Paediatrica Japonica 1997;39(6):685-9.
79. Kamakura K, Kawai M, Arahata K, Koizuma H, Watanabe K, Sugita HA. A manifesting carrier of Duchenne muscular dystrophy with severe myocardial symptoms. Journal of Neurology 1990;237(8):483-5.
80. Koenig M, Beggs AH, Moyer M, Scherpf S, Heindrich K, Bettecken T, et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Human Genetics 1989;45(4):498-506.
81. Kono T, Ogimoto A, Nishimura K, Yorozuva T, Okura T, Higaki J. Cardiac resynchronisation therapy in a young patient with Duchenne muscular dystrophy. International Medical Case Reports Journal 2015;21(8):173-5.
82. Kuru S, Tanahashi T, Matsumoto S, Kitamura T, Konagava M. Complete atrioventricular block in Duchenne muscular dystrophy. Rinsho Shinkeigaku 2012;52(9):684-7.
83. Kwon HW, Kwon BS, Kim GB, Chae JH, Park JD, Bae EJ, et al. The effect of enalapril and carvedilol on left ventricular function in middle childhood and adolescent patients with muscular dystrophy. Korean Circulation Journal 2012;42(3):184-91.
84. Lane RJ, Gardner-Medwin D, Roses AD. Electrocardiographic abnormalities in carriers of Duchenne muscular dystrophy. Neurology 1980;30(5):497-506.
85. Lanza GA, Dello Russo A, Giglio V, De Luca L, Messano L, Santini C, et al. Impairment of cardiac autonomic function in patients with Duchenne muscular dystrophy: relationship to myocardial and respiratory function. American Heart Journal 2001;141(5):808-12.
86. Malhotra SB, Hart KA, Klamut HJ, Thomas NS, Bodrug SE, Burghes AH, et al. Frame-shift deletions in patients with Duchenne and Becker muscular dystrophy. Science 1988;242(4879):755-9.
87. Matsumara T, Tamura T, Kuru S, Kikuchi Y, Kewai M. Carvedilol can prevent cardiac events in Duchenne muscular dystrophy. Internal Medicine 2010;49:1357-63.
88. Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY. Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database of Systematic Reviews 2016, Issue 5. [DOI: 10.1002/14651858.CD003725.pub4]
89. Melacini P, Fanin M, Danieli GA, Villanova C, Martinello F, Miorin M, et al. Myocardial involvement is very frequent in patients affected with subclinical Becker's muscular dystrophy. Circulation 1996;94(12):3168-75.
90. Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Annals of Neurology 2012;71(3):304-13.
91. Menke A, Jockusch H. Decreased osmotic stability of dystrophin-less muscle cells from the mdx mouse. Nature 1991;349(6304):69-71.
92. Meune C, Pascal O, Bécane HM, Héloire F, Christoforou D, Laforet P, et al. Reliable detection of early myocardial dysfunction by tissue Doppler echocardiography in Becker muscular dystrophy. Heart 2004;90(8):947-8.
93. Miyoshi K. Echocardiographic evaluation of fibrous replacement in the myocardium of patients with Duchenne muscular dystrophy. British Heart Journal 1991;66(6):452-5.
94. Mori K, Manabe T, Nii M, Hayabuchi Y, Kuroda Y, Tatara K. plasma levels of Natriuretic peptides and echocardiographic parameters in Duchenne's muscular dystrophy. Paediatric Cardiology 2002;23(2):160-6.
95. Mori K, Hayabuchi Y, Inoue M, Suzuki M, Sakata M, Nakagawa R, et al. Myocardial strain imaging for early detection of cardiac involvement in patients with Duchenne's progressive muscular dystrophy. Echocardiography 2007;24(6):598-608.
96. Mukoyama M, Kondo K, Hizawa K, Nishitani H. Life spans of Duchenne muscular dystrophy patients in the hospital care programme in Japan. Journal of the Neurological Sciences 1987;81(2-3):155-8.
97. Muntoni F, Di Lenarda A, Porcu M, Sinagra G, Mateddu A, Marrosuo G, et al. Dystrophin gene abnormalities in two patients with idiopathic dilated cardiomyopathy. Heart 1997;78(6):608-12.
98. Muntoni F. Cardiomyopathy in muscular dystrophies. Current Opinion in Neurology 2003;16(5):577-83.
99. Nigro G, Comi LI, Limongelli FM, Giugliano MA, Politano L, Petretta V. Prospective study of X-linked progressive muscular dystrophy in Campania. Muscle & Nerve 1983;6(4):253-62.
100. Nigro G, Comi L, Politano L, Bain RJ. The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy. International Journal of Cardiology 1990;26(3):271-7.
101. Nigro G, Comi LI, Politano L, Limongelli FM, Nigro V, De Rimini ML, et al. Evaluation of the cardiomyopathy in Becker muscular dystrophy. Muscle & Nerve 1995;18(3):283-91.
102. Nigro G, Nigro G, Politano L, Santangelo L, Petretta VR, Passamano L, et al. Is the value of QT dispersion a valid method to foresee the risk of sudden death? A study in Becker patients. Heart 2002;87(2):156-7.
103. Nikolíc G. Dominant R wave in lead V1. Heart & Lung 1998;27(5):352-3.
104. Nolan MA, Jones OD, Pedersen RL, Johnston HM. Cardiac assessment in childhood carriers of Duchenne and Becker muscular dystrophies. Neuromuscular Disorders 2003;13(2):129-32.
105. Oldfors A, Eriksson BO, Kyllerman M, Martinsson T, Wahlstrom J. Dilated cardiomyopathy and the dystrophin gene: an illustrated review. British Heart Journal 1994;72(4):344-8.
106. Papa AA, D'Ambrosio P, Petillo R, Palladino A, Politano L. Heart transplantation in patients with dystrophinopathic cardiomyopathy: review of the literature and personal series. Intractable & Rare Diseases Research 2017;6(2):95-101.
107. Perloff JK, Henze E, Schelbert HR. Alterations in regional myocardial metabolism, perfusion, and wall motion in Duchenne muscular dystrophy studied by radionuclide imaging. Circulation 1984;69(1):33-42.
108. Phillips M, Quinlivan R. Calcium antagonists for Duchenne muscular dystrophy. Cochrane Database of Systematic Reviews 2008, Issue 4. [DOI: 10.1002/14651858.CD004571.pub2]
109. Ponikowski P, Voors AA, Anker SD, Bueno H, Cleland JGF, Coats AJS, et al. 2016 ESC Guidelines for the diagnosis and treatment of acute and chronic heart failure. The Task Force for the diagnosis and treatment of acute and chronic heart failure of the European Society of Cardiology (ESC). Developed with the special contribution of the Heart Failure Association (HFA) of the ESC. European Journal of Heart Failure 2016;18(8):891-975.
110. Quinlivan R, Ball J, Dunckley M Thomas DJ, Flinter F, Morgan-Hughes J. Becker muscular dystrophy presenting with complete heart block in the sixth decade. Journal of Neurology 1995;242(6):398-400.
111. Quinlivan RM, Lewis P, Marsden P, Dundas R, Robb SA, Baker E. Cardiac function, metabolism and perfusion in Duchenne and Becker muscular dystrophy. Neuromuscular Disorders 1996;6(4):237-46.
112. The Nordic Cochrane Centre, The Cochrane Collaboration. Review Manager (RevMan). Version 5.3. Copenhagen: The Nordic Cochrane Centre, The Cochrane Collaboration, 2014.
113. Ryan TD, Jefferies JL, Sawnani H, Wong BL, Gardner A, Del Corral M, et al. Implantation of the HeartMate II and HeartWare left ventricular assist devices in patients with Duchenne muscular dystrophy: lessons learned from the first applications. ASAIO Journal 2014;60(2):246-8.
114. Sakata C, Sunohara N, Nonaka I, Arahata K, Sugita H. A case of Becker muscular dystrophy presenting cardiac failure as the initial symptom. Rinsho Shinkeigaku 1990;30(2):210-3.
115. Schram G, Fournier A, Leduc H, Dahdah N, Therien J, Vanasse M, et al. All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy. Journal of the American College of Cardiology 2013;61(9):948-54.
116. Silva MC, Meira ZM, Gurgel Giannetti J, da Silva MM, Campos AF, Barbosa Mde M, et al. Myocardial delayed enhancement by magnetic resonance imaging in patients with muscular dystrophy. Journal of the American College of Cardiology 2007;49(18):1874-9.
117. Silversides CK, Webb GD, Harris VA, Biggar DW. Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy. American Journal of Cardiology 2003;91(6):769-72.
118. Soslow J, Xu M, Slaughter J, Stanley M, Crum K, Markham LW, et al. Evaluation of echocardiographic measures of left ventricular function in patients with Duchenne muscular Dystrophy. Journal of the American Society of Echocardiography 2016;29(10):983-91.
119. Spurney CF, McCaffrey F, Morgenroth L, Ghelani SJ, Gordish-Dressman H, Arrieta A, et al. Feasibility and reproducibility of echocardiographic measures in children with muscular dystrophies. Journal of the American Society of Echocardiography 2015;28(8):999-1008.
120. Stabile C, Taylor M, Mazur W, Cripe LH, King E, Pratt J, et al. Left ventricular non-compaction in Duchenne muscular dystrophy. Journal of Cardiovascular Magnetic Resonance 2013;15(1):67-75.
121. Steare SE, Dubowitz V, Benatar A. Subclinical cardiomyopathy in Becker muscular dystrophy. British Heart Journal 1992;68(3):304-8.
122. Swedberg K, Komajda M, Böhm M, Borer JS, Ford I, Dubost-Brama A, et al. Ivabradine and outcomes in chronic heart failure (SHIFT): a randomised placebo-controlled study. Lancet 2010;376(9744):874-85. [DOI: 10.1016/S0140-6736(10)61198-1]
123. Takenaka A, Yokota M, Iwase M, Miyaguchi K, Hayashi H, Saito H. Discrepancy between systolic and diastolic dysfunction of the left ventricle in patients with Duchenne muscular dystrophy. European Heart Journal 1993;14(5):669-76.
124. Tanaka H, Nishi S, Katanasako H. Natural course of cardiomyopathy in Duchenne muscular dystrophy. Japanese Circulation Journal 1979;43(11):974-84.
125. Towbin JA, Hejtmancik JF, Brink P, Gelb B, Zhu XM, Chamberlain JS, et al. X-linked dilated cardiomyopathy. Molecular genetic evidence of linkage to the Duchenne muscular dystrophy (dystrophin) gene at the Xp21 locus. Circulation 1993;87(6):1854-65.
126. Turley A, Raja SG, Salhiyyah K, Nagarajan K. Does cardiac resynchronisation therapy improve survival and quality of life in patients with end-stage heart failure?. Interactive Cardiovascular and Thoracic Surgery 2008;7(6):141-6.
127. Varni JW, Seid M, Rode CA. The PedsQL: measurement model for the pediatric quality of life inventory. Medical Care 1999;37(2):126-39. [PUBMED: 10024117]
128. Vincent KA, Carr AJ, Walburn J, Scott DL, Rose MR. Construction and validation of a quality of life questionnaire for Neuromuscular Disease (INQol). Neurology 2007;68(13):1051-7.
129. Viollet L, Thrush PT, Flanigan KM, Mendell JR, Allen HD. Effects of angiotensin-converting enzyme inhibitors and/or beta blockers on the cardiomyopathy in Duchenne muscular dystrophy. American Journal of Cardiology 2012;110(1):98-102.
130. Wagner K, Lechtzin N, Judge D. Current treatment of adult Duchenne muscular dystrophy. Biochemica et Biophysica Acta 2007;1772:229-237.
131. Ware JE Jr, Kosinski M, Bjorner JB, Turner-Bowker DM, Gandek B, Maruish ME. User's Manual for the SF-36v2TM Health Survey. 2nd Edition. Lincoln, RI: Quality Metric Incorporated, 2007.
132. Wasala NB, Bostick B, Yue Y, Duan D. Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy. Human Molecular Genetics 2013;22(13):2634-41.
133. Wu R, Gupta S, Brown R, Yancy C, Wald J, Kaiser P, et al. Clinical outcomes after cardiac transplantation in muscular dystrophy patients. Journal of Heart and Lung Transplantation 2010;29(4):432-8.
134. Yamamoto S, Matsushima H, Suzuki A, Sotobata I, Indo T, Matsuoka Y. A comparative study of thallium-201 single-photon emission computed tomography and electrocardiography in Duchenne and other types of muscular dystrophy. American Journal of Cardiology 1988;61(10):838-43.
135. Zannad F, Alla F, Dousset B, Perez A, Pitt B. Limitation of excessive extracellular matrix turnover may contribute to survival benefit of spironolactone therapy in patients with congestive heart failure: insights from the randomized aldactone evaluation study (RALES). Rales Investigators. Circulation 2000;102(6):2700-6.
136. Quinlivan R, Chikermane A, Bourke JP. Prevention and treatment for cardiac complications in Duchenne and Becker muscular dystrophy. Cochrane Database of Systematic Reviews 2011, Issue 4. [DOI: 10.1002/14651858.CD009068]
137. Quinlivan R, Bourke JP, Bueser T. Prevention and treatment for cardiac complications in Duchenne and Becker muscular dystrophy and X-linked dilated cardiomyopathy. Cochrane Database of Systematic Reviews 2012, Issue 9. [DOI: 10.1002/14651858.CD009068.pub2]