Treatment approaches in motor neurone disease

Current Opinion in Neurology

Volumn 29, Issue 5, 2016, Pages 581-591

  Dharmadasa, Thanuja ^a   Matamala, José M ^a   Kiernan, Matthew C ^a  

^a UNIVERSITY OF SYDNEY   (Australia)

Author keywords

Management; Motor neurone disease; Neuroprotection; Quality of life; Survival

Indexed keywords

CENTRAL NERVOUS SYSTEM AGENTS; GENE THERAPY AGENT; NEW DRUG; RILUZOLE; NEUROPROTECTIVE AGENT;

DISEASE MANAGEMENT; EVIDENCE BASED PRACTICE; GENE THERAPY; HUMAN; INTERPERSONAL COMMUNICATION; MOTOR NEURON DISEASE; MULTICENTER STUDY (TOPIC); NONHUMAN; NUTRITION; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PSYCHIATRIC TREATMENT; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); RESPIRATION CONTROL; REVIEW; STEM CELL TRANSPLANTATION; TERMINAL CARE; DISEASE EXACERBATION; EVIDENCE BASED MEDICINE; NONINVASIVE VENTILATION;

DISEASE MANAGEMENT; DISEASE PROGRESSION; EVIDENCE-BASED MEDICINE; HUMANS; MOTOR NEURON DISEASE; NEUROPROTECTIVE AGENTS; NONINVASIVE VENTILATION; QUALITY OF LIFE;

EID: 84979707895     PISSN: 13507540     EISSN: 14736551     Source Type: Journal    
DOI: 10.1097/WCO.0000000000000369     Document Type: Review

References (79)

1. Rooney J, Byrne S, Heverin M, et al. A multidisciplinary clinic approach improves survival in ALS: a comparative study of ALS in Ireland and Northern Ireland. J Neurol Neurosurg Psychiatry 2015; 86:496-501.
2. Van den Berg JP, Kalmijn S, Lindeman E, et al. Multidisciplinary ALS care improves quality of life in patients with ALS. Neurology 2005; 65:1264-1267.
3. Traynor BJ, Alexander M, Corr B, et al. Effect of a multidisciplinary amyotrophic lateral sclerosis (ALS) clinic on ALS survival: a population based study, 1996-2000. J Neurol Neurosurg Psychiatry 2003; 74:1258-1261.
4. Pradas J, Finison L, Andres PL, et al. The natural history of amyotrophic lateral sclerosis and the use of natural history controls in therapeutic trials. Neurology 1993; 43:751-755.
5. Miller RG, Jackson CE, Kasarskis EJ, et al. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: drug, nutritional, and respiratory therapies (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology 2009; 73:1218-1226.
6. Corcia P, Pradat P-F, Salachas F, et al. Causes of death in a postmortem series of ALS patients. Amyotroph Lateral Scler 2008; 9:59-62.
7. Polkey MI, Green M, Moxham J. Measurement of respiratory muscle strength. Thorax 1995; 50:1131-1135.
8. Morgan RK, McNally S, Alexander M, et al. Use of Sniff nasal-inspiratory force to predict survival in amyotrophic lateral sclerosis. Am J Respir Crit Care Med 2005; 171:269-274.
9. Hannan LM, Dominelli GS, Chen Y-W, et al. Systematic review of noninvasive positive pressure ventilation for chronic respiratory failure. Respir Med 2014; 108:229-243.
10. Kleopa KA, Sherman M, Neal B, et al. Bipap improves survival and rate of pulmonary function decline in patients with ALS. J Neurol Sci 1999; 164:82-88.
11. Radunovic A, Annane D, Rafiq MK, et al. Mechanical ventilation for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev 2013; (3):CD004427.
12. Pinto A, de Carvalho M, Evangelista T, et al. Nocturnal pulse oximetry: a new approach to establish the appropriate time for noninvasive ventilation in ALS patients. Amyotroph Lateral Scler Other Motor Neuron Disord 2003; 4:31-35.
13. Lechtzin N, Scott Y, Busse AM, et al. Early use of noninvasive ventilation prolongs survival in subjects with ALS. Amyotroph Lateral Scler 2007; 8:185-188.
14. Georges M, Moré lot-Panzini C, Similowski T, et al. Noninvasive ventilation reduces energy expenditure in amyotrophic lateral sclerosis. BMC Pulm Med 2014; 14:17.
15. Gruis KL, Brown DL, Lisabeth LD, et al. Longitudinal assessment of noninvasive positive pressure ventilation adjustments in ALS patients. J Neurol Sci 2006; 247:59-63.
16. Gonzalez-Bermejo J, Morelot-Panzini C, Arnol N, et al. Prognostic value of efficiently correcting nocturnal desaturations after one month of noninvasive ventilation in amyotrophic lateral sclerosis: a retrospective monocentre observational cohort study. Amyotroph Lateral Scler Frontotemporal Degener 2013; 14:373-379.
17. Vandenberghe N, Vallet A-E, Petitjean T, et al. Absence of airway secretion accumulation predicts tolerance of noninvasive ventilation in subjects with amyotrophic lateral sclerosis. Respir Care 2013; 58:1424-1432.
18. Chiò A, Ilardi A, Cammarosano S, et al. Neurobehavioral dysfunction in ALS has a negative effect on outcome and use of PEG and NIV. Neurology 2012; 78:1085-1089.
19. Gruis KL, Brown DL, Schoennemann A, et al. Predictors of noninvasive ventilation tolerance in patients with amyotrophic lateral sclerosis. Muscle Nerve 2005; 32:808-811.
20. Bourke SC, Tomlinson M, Williams TL, et al. Effects of noninvasive ventilation on survival and quality of life in patients with amyotrophic lateral sclerosis: a randomised controlled trial. Lancet Neurol 2006; 5:140-147.
21. Sancho J, Servera E, Díaz JL, et al. Home tracheotomy mechanical ventilation in patients with amyotrophic lateral sclerosis: causes, complications and 1-year survival. Thorax 2011; 66:948-952.
22. Bach JR. Amyotrophic lateral sclerosis. Communication status and survival with ventilatory support. Am J Phys Med Rehabil 1993; 72:343-349.
23. Sancho J, Servera E, Bañ uls P, et al. Prolonging survival in amyotrophic lateral sclerosis: efficacy of noninvasive ventilation and uncuffed tracheostomy tubes. Am J Phys Med Rehabil 2010; 89:407-411.
24. Onders RP, Elmo M, Kaplan C, et al. Final analysis of the pilot trial of diaphragm pacing in amyotrophic lateral sclerosis with long-term follow-up: diaphragm pacing positively affects diaphragm respiration. Am J Surg 2014; 207:393-397.
25. McDermott CJ, Bradburn MJ, Maguire C, et al. Safety and efficacy of diaphragm pacing in patients with respiratory insufficiency due to amyotrophic lateral sclerosis (DiPALS): a multicentre, open-label, randomised controlled trial. Lancet Neurol 2015; 14:883-892.
26. McDermott CJ, Shaw PJ. Diagnosis and management of motor neurone disease. BMJ 2008; 336:658-662.
27. Zoing MC, Burke D, Pamphlett R, et al. Riluzole therapy for motor neurone disease: an early Australian experience (1996-2002). J Clin Neurosci 2006; 13:78-83.
28. Miller RG, Mitchell JD, Moore DH. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane database Syst Rev 2012; (3):CD001447.
29. Traynor BJ, Alexander M, Corr B, et al. An outcome study of riluzole in amyotrophic lateral sclerosis-a population-based study in Ireland, 1996-2000. J Neurol 2003; 250:473-479.
30. Zoccolella S, Beghi E, Palagano G, et al. Riluzole and amyotrophic lateral sclerosis survival: a population-based study in southern Italy. Eur J Neurol 2007; 14:262-268.
31. Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med 1994; 330:585-591.
32. Cassiman D, Thomeer M, Verbeken E, et al. Hypersensitivity pneumonitis possibly caused by riluzole therapy in ALS. Neurology 2003; 61:1150-1151.
33. Kiernan MC, Vucic S, Cheah BC, et al. Amyotrophic lateral sclerosis. Lancet 2011; 377:942-955.
34. Spataro R, Ficano L, Piccoli F, et al. Percutaneous endoscopic gastrostomy in amyotrophic lateral sclerosis: effect on survival. J Neurol Sci 2011; 304:44-48.
35. Wills A-M, Hubbard J, Macklin EA, et al. Hypercaloric enteral nutrition in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet 2014; 383:2065-2072.
36. Ahmed RM, Irish M, Piguet O, et al. Amyotrophic lateral sclerosis and frontotemporal dementia: distinct and overlapping changes in eating behaviour and metabolism. Lancet Neurol 2016; 15:332-342.
37. Jesse S, Thal DR, Ludolph AC. Thiamine deficiency in amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry 2015; 86:1166-1168.
38. Dupuis L, Corcia P, Fergani A, et al. Dyslipidemia is a protective factor in amyotrophic lateral sclerosis. Neurology 2008; 70:1004-1009.
39. Kasarskis EJ, Berryman S, Vanderleest JG, et al. Nutritional status of patients with amyotrophic lateral sclerosis: relation to the proximity of death. Am J Clin Nutr 1996; 63:130-137.
40. Desport JC, Preux PM, Truong TC, et al. Nutritional status is a prognostic factor for survival in ALS patients. Neurology 1999; 53:1059-1063.
41. Desport JC, Preux PM, Truong CT, et al. Nutritional assessment and survival in ALS patients. Amyotroph Lateral Scler Other Motor Neuron Disord 2000; 1:91-96.
42. Desport J-C, Torny F, Lacoste M, et al. Hypermetabolism in ALS: correlations with clinical and paraclinical parameters. Neurodegener Dis 2005; 2:202-207.
43. NCGC. Motor neurone disease: assessment and management. London Natl Inst Heal Care Excell (UK) 2016.
44. Chiò A, Galletti R, Finocchiaro C, et al. Percutaneous radiological gastrostomy: a safe and effective method of nutritional tube placement in advanced ALS. J Neurol Neurosurg Psychiatry 2004; 75:645-647.
45. Chavada G, El-Nayal A, Lee F, et al. Evaluation of two different methods for per-oral gastrostomy tube placement in patients with motor neuron disease (MND): PIG versus PEG procedures. Amyotroph Lateral Scler 2010; 11:531-536.
46. Laasch H-U, Wilbraham L, Bullen K, et al. Gastrostomy insertion: comparing the options-PEG, RIG or PIG? Clin Radiol 2003; 58:398-405.
47. Verschueren A, Monnier A, Attarian S, et al. Enteral and parenteral nutrition in the later stages of ALS: an observational study. Amyotroph Lateral Scler 2009; 10:42-46.
48. Thornton FJ, Fotheringham T, Alexander M, et al. Amyotrophic lateral sclerosis: enteral nutrition provision-endoscopic or radiologic gastrostomy? Radiology 2002; 224:713-717.
49. Desport J-C, Mabrouk T, Bouillet P, et al. Complications and survival following radiologically and endoscopically-guided gastrostomy in patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord 2005; 6:88-93.
50. Cobble M. Language impairment in motor neurone disease. J Neurol Sci 1998; 160 (Suppl):S47-S52.
51. Veaux C, Yamagishi JKS. Towards personalized synthesized voices for individuals with vocal disabilities: voice banking and reconstruction. SLPAT 2013 (4th Workshop on Speech and Language Processing for Assistive Technologies), 2013, 107-111.
52. Saz OHT. Asynchronous factorisation of speaker and background with feature transforms in speech recognition. Proceedings of the 14th Annual Conference of the International Speech Communication Association (Interspeech), 2013; 1238-1242.
53. Yamagishi J, Veaux C, King SE. Speech synthesis technologies forindividuals with vocal disabilities: voice banking and reconstruction. Acoust Sci Tech 2012; 33:1-5.
54. Lillo P, Savage S, Mioshi E, et al. Amyotrophic lateral sclerosis and frontotemporal dementia: a behavioural and cognitive continuum. Amyotroph Lateral Scler 2012; 13:102-109.
55. Burrell JR, Halliday GM, Kril JJ, et al. The frontotemporal dementia-motor neuron disease continuum. Lancet 2016. pii: S0140-6736(16)00737-746.
56. Boxer AL, Knopman DS, Kaufer Di, et al. Memantine in patients with frontotemporal lobar degeneration: a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol 2013; 12:149-156.
57. Pressman PS, Miller BL. Diagnosis and management of behavioral variant frontotemporal dementia. Biol Psychiatry 2014; 75:574-581.
58. Lillo P, Mioshi E, Hodges JR. Caregiver burden in amyotrophic lateral sclerosis is more dependent on patients' behavioral changes than physical disability: a comparative study. BMC Neurol 2012; 12:156.
59. Atassi N, Cook A, Pineda CME, et al. Depression in amyotrophic lateral sclerosis. Amyotroph Lateral Scler 2011; 12:109-112.
60. Rabkin JG, Goetz R, Factor-Litvak P, et al. Depression and wish to die in a multicenter cohort of ALS patients. Amyotroph Lateral Scler Frontotemporal Degener 2015; 16:265-273.
61. Caga J, Turner MR, Hsieh S, et al. Apathy is associated with poor prognosis in amyotrophic lateral sclerosis. Eur J Neurol 2016; 23:891-897.
62. Kurt A, Nijboer F, Matuz T, et al. Depression and anxiety in individuals with amyotrophic lateral sclerosis: epidemiology and management. CNS Drugs 2007; 21:279-291.
63. Connolly S, Galvin M, Hardiman O. End-of-life management in patients with amyotrophic lateral sclerosis. Lancet Neurol 2015; 14:435-442.
64. Jackson CE, McVey AL, Rudnicki S, et al. Symptom management and end-oflife care in amyotrophic lateral sclerosis. Neurol Clin 2015; 33:889-908.
65. Neudert C, Oliver D, Wasner M, et al. The course of the terminal phase in patients with amyotrophic lateral sclerosis. J Neurol 2001; 248:612-616.
66. Andersen PM, Abrahams S, Borasio GD, et al. EFNS guidelines on the clinical management of amyotrophic lateral sclerosis (MALS)-revised report of an EFNS task force. Eur J Neurol 2012; 19:360-375.
67. Martin J, Turnbull J. Lasting impact in families after death from ALS. Amyotroph Lateral Scler Other Motor Neuron Disord 2001; 2:181-187.
68. Shefner JM, Wolff AA, Meng L, et al. A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener 2016; 1-10.
69. Wainger BJ, Kiskinis E, Mellin C, et al. Intrinsic membrane hyperexcitability of amyotrophic lateral sclerosis patient-derived motor neurons. Cell Rep 2014; 7:1-11.
70. Dubreuil P, Letard S, Ciufolini M, et al. Masitinib (AB1010), a potent and selective tyrosine kinase inhibitor targeting KIT. PLoS One 2009; 4:e7258.
71. Kaji R, Kuzuhara S, Iwasaki Y, et al. Ultra-high dose methylcobalamin (E0302) prolongs survival of ALS: report of 7 years' randomised double-blind, phase 3 clinical trial (ClinicalTrials.gov NCT00444613). Neurology 2015; 84 (Suppl):7060.
72. Yoshida H, Yanai H, Namiki Y, et al. Neuroprotective effects of edaravone: a novel free radical scavenger in cerebrovascular injury. CNS Drug Rev 2006; 12:9-20.
73. Abe K, Itoyama Y, Sobue G, et al. Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler Frontotemporal Degener 2014; 15:610-617.
74. Williams JR, Trias E, Beilby PR, et al. Copper delivery to the CNS by CuATSM effectively treats motor neuron disease in SODG93A mice co-expressing the copper-chaperone-for-SOD. Neurobiol Dis 2016; 89:1-9.
75. Li W, Lee M-H, Henderson L, et al. Human endogenous retrovirus-K contributes to motor neuron disease. Sci Transl Med 2015; 7: 307ra153.
76. Lunn JS, Sakowski SA, Feldman EL. Concise review: stem cell therapies for amyotrophic lateral sclerosis: recent advances and prospects for the future. Stem Cells 2014; 32:1099-1109.
77. Richard J-P, Maragakis NJ. Induced pluripotent stem cells from ALS patients for disease modeling. Brain Res 2014; 1607:15-25.
78. Payne NL, Sylvain A, O'Brien C, et al. Application of human induced pluripotent stem cells for modeling and treating neurodegenerative diseases. N Biotechnol 2015; 32:212-228.
79. Scarrott JM, Herranz-Martín S, Alrafiah AR, et al. Current developments in gene therapy for amyotrophic lateral sclerosis. Expert Opin Biol Ther 2015; 15:935-947.