Long-term management of children with neuromuscular disorders

Jornal de Pediatria

Volumn 85, Issue 5, 2009, Pages 379-384

  Strehle, Eugen Matthias ^a  

^a NORTH TYNESIDE GENERAL HOSPITAL   (United Kingdom)

Author keywords

Children; Follow up; Management; Myopathy; Neuromuscular disorders; Neuropathy

Indexed keywords

3,4 DIAMINOPYRIDINE; ALUMINUM HYDROXIDE; ANTIBIOTIC AGENT; BISPHOSPHONIC ACID DERIVATIVE; CALCIUM; CHOLINESTERASE INHIBITOR; DEFLAZACORT; DESMOPRESSIN; DOCUSATE SODIUM; EPHEDRINE; IBUPROFEN; IMMUNOSUPPRESSIVE AGENT; LACTULOSE; MACROGOL; MAGNESIUM CARBONATE; MELATONIN; MODAFINIL; OXYBUTYNIN; PAMIDRONIC ACID; PARACETAMOL; PREDNISOLONE; PYRIDOSTIGMINE; RANITIDINE; RISEDRONIC ACID; SALBUTAMOL; SENNA EXTRACT; STEROID; VITAMIN D;

BECKER MUSCULAR DYSTROPHY; BEHAVIOR DISORDER; BRACE; CARDIOMYOPATHY; CAST APPLICATION; CATARACT; CHEST INFECTION; CHILD; COGNITIVE DEFECT; CONGENITAL MYASTHENIC SYNDROME; CONSTIPATION; CONTRACTURE; CUSHINGOID SYNDROME; DAYTIME SOMNOLENCE; DIURNAL ENURESIS; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; FRACTURE; GASTROESOPHAGEAL REFLUX; GROWTH DISORDER; HEREDITARY SPINAL MUSCULAR ATROPHY; HUMAN; HYPERTENSION; INCREASED APPETITE; JAW OSTEONECROSIS; LEG CRAMP; LEG PAIN; LONG TERM CARE; LOWER RESPIRATORY TRACT INFECTION; MUSCLE STRENGTH; MYASTHENIA GRAVIS; MYOPATHY; MYOTONIC DYSTROPHY; NEUROMUSCULAR DISEASE; NEUROPATHY; NOCTURNAL ENURESIS; OSTEOPENIA; OSTEOPOROSIS; PHYSIOTHERAPY; PRACTICE GUIDELINE; QUALITY OF LIFE; REVIEW; SCOLIOSIS; SIDE EFFECT; SLEEP DISORDER; SPINAL MUSCULAR ATROPHY; SPINE STABILIZATION; SPLINTING; STRETCHING EXERCISE; THYMECTOMY; TRANSCUTANEOUS NERVE STIMULATION; TREATMENT PLANNING; WEIGHT GAIN;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; FEMALE; GREAT BRITAIN; HUMANS; INFANT; INFANT, NEWBORN; MALE; NEUROMUSCULAR DISEASES; YOUNG ADULT;

EID: 70350452114     PISSN: 00217557     EISSN: None     Source Type: Journal    
DOI: 10.2223/JPED.1929     Document Type: Review

References (59)

1. Strehle EM. Food for thought: autophagic vacuolar myopathies. Arch Dis Child. 2009;94:567-569
2. Jain Foundation Inc. Orchestrating a cure for LGMD2B/Miyoshi [website]. https://www.jain-foundation.org/projects.php. Access: 09/03/2009.
3. Manzur AY, Kinali M, Muntoni F. Update on the management of Duchenne muscular dyst rophy. Arch Dis Chi ld. 2008;93:986-990
4. Eagle M, Bourke J, Bullock R, Gibson M, Mehta J, Giddings D, et al. Managing Duchenne muscular dystrophy - the additive effect of spinal surgery and home nocturnal ventilation in improving survival. Neuromuscul Disord. 2007;17:470-475 (Pubitemid 46795560)
5. Iannaccone ST. Modern management of spinal muscular atrophy. J Child Neurol. 2007;22:974-978 (Pubitemid 47308311)
6. Muscular Dystrophy Campaign [website]. http://www.musculardystrophy.org/ how-we-help-you/for-professionals/clinical-databases. Access: 09/03/2009.
7. Steffensen B, Hyde S, Lyager S, Mattsson E. Validity of the EK scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy. Physiother Res Int. 2001;6:119-134
8. Bakker JP, de Groot IJ, Beckerman H, de Jong BA, Lankhorst GJ. The effects of knee-ankle-foot orthoses in the treatment of Duchenne muscular dystrophy: review of the literature. Clin Rehabil. 2000;14:343-359 (Pubitemid 30495202)
9. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008;(1):CD003725.
10. Ciafaloni E, Moxley RT. Treatment options for Duchenne muscular dystrophy. Curr Treat Options Neurol. 2008;10:86-93.
11. Kinali M, Beeson D, Pitt MC, Jungbluth H, Simonds AK, Aloysius A, Cockerill H, Davis T, Palace J, Manzur AY, Jimenez-Mallebrera C, Sewry C, Muntoni F, Robb SA. Congenital myasthenic syndromes in childhood: diagnostic and management challenges. J Neuroimmunol. 2008;201-202:6-12.
12. Forsyth R, Newton R. Paediatric Neurology. Oxford: University Press, 2007.
13. Angelozzi C, Borgo F, Tiziano FD, Martella A, Neri G, Brahe C. Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells. J Med Genet. 2008;45:29-31. (Pubitemid 351158155)
14. Pane M, Staccioli S, Messina S, D'Amico A, Pelliccioni M, Mazzone ES, et al. Daily salbutamol in young patients with SMA type II. Neuromuscul Disord. 2008;18:536-540
15. Main M, Mercuri E, Haliloglu G, Baker R, Kinali M, Muntoni F. Serial casting of the ankles in Duchenne muscular dystrophy: can it be an alternative to surgery? Neuromuscul Disord. 2007;17:227-230
16. Burns J, Ryan MM, Ouvrier RA. Evolution of foot and ankle manifestations in children with CMT1A. Muscle Nerve. 2009;39:158-166
17. Cheuk DK, Wong V, Wraige E, Baxter P, Cole A, N'Diaye T, et al. Surgery for scoliosis in Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2007;(1):CD005375.
18. Mullender M, Blom N, De Kleuver M, Fock J, Hitters W, Horemans A, et al. A Dutch guideline for the treatment of scoliosis in neuromuscular disorders. Scoliosis. 2008;3:14.
19. Kotwicki T, Jozwiak M. Conservative management of neuromuscular scoliosis: personal experience and review of literature. Disabil Rehabil. 2008;30:792-798 (Pubitemid 351586976)
20. King WM, Ruttencutter R, Nagaraja HN, Matkovic V, Landoll J, Hoyle C, et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology. 2007;68:1607-1613 (Pubitemid 46717986)
21. Houde S, Filiatrault M, Fournier A, Dube J, D'Arcy S, Berube D, et al. Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up. Pediatr Neurol. 2008;38:200-206 (Pubitemid 351221232)
22. Russell RG. Biphosphonates: mode of action and pharmacology. Pediatrics. 2007;119 Suppl 2:S150-62.
23. Mok CC, Tong KH, To CH, Siu YP, Ma KM. Risedronate for the prevention of bone mineral density loss in patients receiving high-dose glucocorticoids: a randomized double-blind placebo-controlled trial. Osteoporos Int. 2008;19:357-364
24. Cartsos VM, Zhu S, Zavras AI. Biphosphonate use and the risk of adverse jaw outcomes: a medical claims study of 714,217 people. J Am Dent Assoc. 2008;139:23-30.
25. Chahine C, Cheung MS, Head TW, Schwartz S, Glorieux FH, Rauch F. Tooth extraction socket healing in pediatric patients treated with intravenous pamidronate. J Pediatr. 2008;153:719-720
26. D'Angelo MG, Bresolin N. Cognitive impairment in neuromuscular disorders. Muscle Nerve. 2006;34:16-33.
27. Young HK, Barton BA, Waisbren S, Portales Dale L, Ryan MM, Webster RI, et al. Cognitive and psychological profile of males with Becker muscular dystrophy. J Child Neurol. 2008;23:155-162
28. Hendriksen JG, Vles JS. Neuropsychiatric disorders in males with Duchenne muscular dystrophy: frequency rate of attention-deficit hyperactivity disorder (ADHD), autism spectrum disorder, and obsessive-compulsive disorder. J Child Neurol. 2008;23:477-481
29. Quera Salva MA, Blumen M, Jacquette A, Durand MC, Andre S, De Villiers M, et al. Sleep disorders in childhood-onset myotonic dystrophy type 1. Neuromuscul Disord. 2006;16:564-570 (Pubitemid 44382732)
30. Kumar R. Approved and investigational uses of modafinil: an evidence based review. Drugs. 2008;68:1803-1839
31. Auer-Grumbach M. Hereditary sensory neuropathy type I. Orphanet J Rare Dis. 2008;3:7.
32. Hayes J, Veyckemans F, Bissonnette B. Duchenne muscular dystrophy: an old anesthesia problem revisited. Paediatr Anaesth. 2008;18:100-106
33. Decruynaere C, Thonnard JL, Plaghki L. How many response levels do children distinguish on faces scales for pain assessment? Eur J Pain. In press 2009.
34. Engel JM, Kartin D, Jaffe KM. Exploring chronic pain in youths with Duchenne Muscular Dystrophy: a model for pediatric neuromuscular disease. Phys Med Rehabil Clin N Am. 2005;16:1113-1124 (Pubitemid 41415708)
35. Clinch J, Eccleston C. Chronic musculoskeletal pain in children: assessment and management. Rheumatology (Oxford). 2009;48:466-474
36. Finsterer J, Stöllberger C. Primary myopathies of the heart. Scand Cardiovasc J. 2008;42:9-24.
37. Sovari AA, Bodine CK, Farokhi F. Cardiovascular manifestations of myotonic dystrophy-1. Cardiol Rev. 2007;15:191-194
38. Finsterer J, Stöllberger C. Atrial fibrillation/flutter in myopathies. Int J Cardiol. 2008;128:304-310
39. Duboc D, Meune C, Pierre B, Wahbi K, Eymard B, Toutain A, et al. Perindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years' follow-up. Am Heart J. 2007;154:596-602. (Pubitemid 47268252)
40. Ogata H, Ishikawa Y, Ishikawa Y, Minami R. Beneficial effects of beta-blockers and angiotensin-converting enzyme inhibitors in Duchenne muscular dystrophy. J Cardiol. 2009;53:72-78
41. Johnson MC, Schneider CJ, Beck AM. Management of systemic hypertension in children and adolescents: an update. Curr Treat Options Cardiovasc Med. 2007;9:381-390 (Pubitemid 47497000)
42. United Kingdom. Department of Health. [website]. http://www.dh.gov.uk/en/ AdvanceSearchResult/index.htm?searchTerms=green+book. Access: 09/03/2009.
43. Bushby K, Bourke J, Bullock R, Eagle M, Gibson M, Quinby J. The multidisciplinary management of Duchenne muscular dystrophy. Curr Paediatr. 2005;15:292-300. (Pubitemid 41558088)
44. Kang SW. Pulmonary rehabilitation in patients with neuromuscular disease. Yonsei Med J. 2006;47:307-314 (Pubitemid 43984156)
45. Homnick DN. Mechanical insufflation-exsufflation for airway mucus clearance. Respir Care. 2007;52:1296-1305
46. Robert D, Argaud L. Clinical review: long-term noninvasive ventilation. Crit Care. 2007;11:210.
47. Ozsancak A, D'Ambrosio C, Hill NS. Nocturnal noninvasive ventilation. Chest. 2008;133:1275-1286
48. Aloysius A, Born P, Kinali M, Davis T, Pane M, Mercuri E. Swallowing difficulties in Duchenne muscular dystrophy: indications for feeding assessment and outcome of videofluoroscopic swallow studies. Eur J Paediatr Neurol. 2008;12:239-245 (Pubitemid 351539070)
49. Engvall M, Sjögreen L, Kjellberg H, Robertson A, Sundell S, Kiliaridis S. Oral health in children and adolescents with myotonic dystrophy. Eur J Oral Sci. 2007;115:192-197
50. Pijpers MA, Tabbers MM, Benninga MA, Berger MY. Currently recommended treatments of childhood constipation are not evidence based: a systematic literature review on the effect of laxative treatment and dietary measure. Arch Dis Child. 2009;94:117-131
51. Philpot J, Bagnall A, King C, Dubowitz V, Muntoni F. Feeding problems in merosin deficient congenital muscular dystrophy. Arch Dis Child. 1999;80:542-547 (Pubitemid 29264368)
52. Messina S, Pane M, De Rose P, Vasta I, Sorleti D, Aloysius A, et al. Feeding problems and malnutrition in spinal muscular atrophy type II. Neuromuscul Disord. 2008;18:389-393
53. Garrood P, Eagle M, Jardine PE, Bushby K, Straub V. Myoglobinuria in boys with Duchenne muscular dystrophy on corticosteroid therapy. Neuromuscul Disord. 2008;18:71-73 (Pubitemid 351173835)
54. Aboumousa A, Hoogendijk J, Charlton R, Barresi R, Herrmann R, Voit T, et al. Caveolinopathy - new mutations and additional symptoms. Neuromuscul Disord. 2008;18:572-578
55. Van Adel BA, Tarnopolsky MA. Metabolic myopathies: update 2009. J Clin Neuromuscul Dis. 2009;10:97-121.
56. Ellsworth P, Caldamone A. Pediatric voiding dysfunction: current evaluation and management. Urol Nurs. 2008 28:249-257
57. Coulthard MG, Lambert HJ, Keir MJ. Do systemic symptoms predict the risk of kidney scarring after urinary tract infection? Arch Dis Child. 2009;94:278-281
58. Peter NG, Forke CM, Ginsburg KR, Schwarz DF. Transition from pediatric to adult care: internists' perspectives. Pediatrics. 2009;123:417-423
59. Wagner KR. Approaching a new age in Duchenne muscular dystrophy treatment. Neurotherapeutics. 2008;5:583-591