Medical therapies for amyotrophic lateral sclerosis-related respiratory decline: an appraisal of needs, opportunities and obstacles

References

• Lyall RA, Donaldson N, Fleming T, Wood C, Newsom-Davis I, Polkey MI, et al. A prospective study of quality of life in ALS patients treated with noninvasive ventilation. Neurology. 2001;57:153–6.
   PubMed Web of Science ®Google Scholar
• Mustfa N, Walsh E, Bryant V, Lyall RA, Addington-Hall J, Goldstein LH, et al. The effect of noninvasive ventilation on ALS patients and their caregivers. Neurology. 2006;66:1211–7.
   PubMed Web of Science ®Google Scholar
• Bourke SC, Shaw PJ, Gibson GJ. Respiratory function vs sleep-disordered breathing as predictors of QOL in ALS. Neurology. 2001;57:2040–4.
   PubMed Web of Science ®Google Scholar
• Miller RG, Mitchell JD, Moore DH. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane Database Syst Rev. 2012;3:CD001447.
   PubMed Web of Science ®Google Scholar
• Vu M, Tortorice K, Zacher J, Dong D, Hur K, Zhang R, et al. Assessment of use and safety of edaravone for amyotrophic lateral sclerosis in the veterans affairs health care system. JAMA Netw Open. 2020;3:e2014645.
   PubMed Web of Science ®Google Scholar
• Ng L, Khan F, Young CA, Galea M. Symptomatic treatments for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2017;1:CD011776.
   PubMed Web of Science ®Google Scholar
• Petrov D, Mansfield C, Moussy A, Hermine O. ALS clinical trials review: 20 years of failure. Are we any closer to registering a new treatment? Front Aging Neurosci. 2017;9:68.
   PubMed Web of Science ®Google Scholar
• Bach JR. Amyotrophic lateral sclerosis: prolongation of life by noninvasive respiratory AIDS. Chest. 2002;122:92–8.
   PubMed Web of Science ®Google Scholar
• Pinto AC, Evangelista T, Carvalho M, Alves MA, Sales Luís ML. Respiratory assistance with a non-invasive ventilator (Bipap) in MND/ALS patients: survival rates in a controlled trial. J Neurol Sci. 1995;129:19–26.
   PubMed Web of Science ®Google Scholar
• Kleopa KA, Sherman M, Neal B, Romano GJ, Heiman-Patterson T. Bipap improves survival and rate of pulmonary function decline in patients with ALS. J Neurol Sci. 1999;164:82–8.
   PubMed Web of Science ®Google Scholar
• Newsom-Davis IC, Lyall RA, Leigh PN, Moxham J, Goldstein LH. The effect of non-invasive positive pressure ventilation (NIPPV) on cognitive function in amyotrophic lateral sclerosis (ALS): a prospective study. J Neurol Neurosurg Psychiatry. 2001;71:482–7.
   PubMed Web of Science ®Google Scholar
• Miller RG, Jackson CE, Kasarskis EJ, England JD, Forshew D, Johnston W, 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;73:1218–26.
   PubMed Web of Science ®Google Scholar
• EFNS Task Force on Diagnosis and Management of Amyotrophic Lateral Sclerosis, Andersen PM, Abrahams S, Borasio GD, de Carvalho M, et al. EFNS guidelines on the clinical management of amyotrophic lateral sclerosis (MALS)—revised report of an EFNS task force. Eur J Neurol. 2012;19:360–75.
   PubMed Web of Science ®Google Scholar
• Georges M, Golmard J-L, Llontop C, Shoukri A, Salachas F, Similowski T, et al. Initiation of non-invasive ventilation in amyotrophic lateral sclerosis and clinical practice guidelines: single-centre, retrospective, descriptive study in a national reference centre. Amyotroph Lateral Scler Frontotemporal Degener. 2017;18:46–52.
   PubMed Web of Science ®Google Scholar
• Heiman-Patterson TD, Cudkowicz ME, De Carvalho M, Genge A, Hardiman O, Jackson CE, et al. Understanding the use of NIV in ALS: results of an international ALS specialist survey. Amyotroph Lateral Scler Frontotemporal Degener. 2018;19:331–41.
   PubMed Web of Science ®Google Scholar
• Motor neurone disease: assessment and management; 2016. Available at: https://www.nice.org.uk/guidance/ng42/evidence/full-guideline-pdf-2361774637.
   Google Scholar
• Pinto S, de Carvalho M. SVC is a marker of respiratory decline function, similar to FVC, in patients with ALS. Front Neurol. 2019;10:109.
   PubMed Web of Science ®Google Scholar
• Pinto S, de Carvalho M. Comparison of slow and forced vital capacities on ability to predict survival in ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2017;18:528–33.
   PubMed Web of Science ®Google Scholar
• Calvo A, Vasta R, Moglia C, Matteoni E, Canosa A, Mattei A, et al. Prognostic role of slow vital capacity in amyotrophic lateral sclerosis. J Neurol. 2020;267:1615–21.
   PubMed Web of Science ®Google Scholar
• de Carvalho M, Costa J, Pinto S, Pinto A. Percutaneous nocturnal oximetry in amyotrophic lateral sclerosis: periodic desaturation. Amyotroph Lateral Scler. 2009;10:154–61.
   PubMed Web of Science ®Google Scholar
• Velasco R, Salachas F, Munerati E, Pradat PF, Lacomblez L, Orvoen EF, et al. Nocturnal oxymetry in patients with amyotrophic lateral sclerosis: role in predicting survival. Rev Neurol. 2002;158:575–8.
   PubMed Web of Science ®Google Scholar
• Pinto A, de Carvalho M, Evangelista T, Lopes A, Sales Luís ML. Nocturnal pulse oximetry: a new approach to establish the appropriate time for noninvasive ventilation in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler. 2003;4:31–5.
   PubMed Web of Science ®Google Scholar
• Jenkins JAL, Sakamuri S, Katz JS, Forshew DA, Guion L, Moore D, et al. Phrenic nerve conduction studies as a biomarker of respiratory insufficiency in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17:213–20.
   PubMed Web of Science ®Google Scholar
• Pinto S, Turkman A, Pinto A, Swash M, de Carvalho M. Predicting respiratory insufficiency in amyotrophic lateral sclerosis: the role of phrenic nerve studies. Clin Neurophysiol. 2009;120:941–6.
   PubMed Web of Science ®Google Scholar
• Pinto S, Pinto A, de Carvalho M. Phrenic nerve studies predict survival in amyotrophic lateral sclerosis. Clin Neurophysiol. 2012;123:2454–9.
   PubMed Web of Science ®Google Scholar
• ARTP COVID-19 Guideline. Available at: https://www.artp.org.uk/write/MediaUploads/Standards/COVID19/ARTP_COVID-19_endemic__guidance_Vers_5.6_final.pdf. Accessed December 14, 2020.
   Google Scholar
• Jacobs TL, Brown DL, Baek J, Migda EM, Funckes T, Gruis KL, et al. Trial of early noninvasive ventilation for ALS: a pilot placebo-controlled study. Neurology. 2016;87:1878–83.
   PubMed Web of Science ®Google Scholar
• Carratù P, Spicuzza L, Cassano A, Maniscalco M, Gadaleta F, Lacedonia D, et al. Early treatment with noninvasive positive pressure ventilation prolongs survival in amyotrophic lateral sclerosis patients with nocturnal respiratory insufficiency. Orphanet J Rare Dis. 2009;4:10.
   PubMed Web of Science ®Google Scholar
• Hardiman O. Management of respiratory symptoms in ALS. J Neurol. 2011;258:359–65.
   PubMed Web of Science ®Google Scholar
• Prell T, Ringer TM, Wullenkord K, Garrison P, Gunkel A, Stubendorff B, et al. Assessment of pulmonary function in amyotrophic lateral sclerosis: when can polygraphy help evaluate the need for non-invasive ventilation? J Neurol Neurosurg Psychiatry. 2016;87:1022–6.
   PubMed Web of Science ®Google Scholar
• Dal Bello-Haas V, Florence JM. Therapeutic exercise for people with amyotrophic lateral sclerosis or motor neuron disease. Cochrane Database Syst Rev. 2013;2013:CD005229.
   Google Scholar
• Pinto S, Swash M, Carvalho M. Respiratory exercise in amyotrophic lateral sclerosis. Amyotroph Lateral Scler. 2012;13:33–43.
   PubMed Web of Science ®Google Scholar
• Bourke SC, Tomlinson M, Williams TL, Bullock RE, Shaw PJ, John Gibson G. Effects of non-invasive ventilation on survival and quality of life in patients with amyotrophic lateral sclerosis: a randomised controlled trial. Lancet Neurol. 2006;5:140–7.
   PubMed Web of Science ®Google Scholar
• Radunovic A, Annane D, Rafiq MK, Mustfa N. Mechanical ventilation for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2017;3:CD004427.
   Google Scholar
• Vitacca M, Montini A, Lunetta C, Banfi P, Bertella E, De Mattia E, et al. Impact of an early respiratory care programme with non-invasive ventilation adaptation in patients with amyotrophic lateral sclerosis. Eur J Neurol. 2018;25:556-e33.
   PubMed Web of Science ®Google Scholar
• Vitacca M, Banfi P, Montini A, Paneroni M. Does timing of initiation influence acceptance and adherence to NIV in patients with ALS? Pulmonology. 2020;26:45–8.
   PubMed Web of Science ®Google Scholar
• Berlowitz DJ, Howard ME, Fiore JF, Vander Hoorn S, O'Donoghue FJ, Westlake J, et al. Identifying who will benefit from non-invasive ventilation in amyotrophic lateral sclerosis/motor neurone disease in a clinical cohort. J Neurol Neurosurg Psychiatry. 2016;87:280–6.
   PubMed Web of Science ®Google Scholar
• Lechtzin N, Scott Y, Busse AM, Clawson LL, Kimball R, Wiener CM, et al. Early use of non-invasive ventilation prolongs survival in subjects with ALS. Amyotroph Lateral Scler. 2007;8:185–8.
   PubMed Web of Science ®Google Scholar
• Ando H, Williams C, Angus RM, Thornton EW, Chakrabarti B, Cousins R, et al. Why don’t they accept non-invasive ventilation? Insight into the interpersonal perspectives of patients with motor neurone disease. Br J Health Psychol. 2015;20:341–59.
   PubMed Web of Science ®Google Scholar
• Aboussouan LS, Khan SU, Banerjee M, Arroliga AC, Mitsumoto H. Objective measures of the efficacy of noninvasive positive-pressure ventilation in amyotrophic lateral sclerosis. Muscle Nerve. 2001;24:403–9.
   PubMed Web of Science ®Google Scholar
• Lo Coco D, Marchese S, Pesco MC, La Bella V, Piccoli F, Lo Coco A. Noninvasive positive-pressure ventilation in ALS: predictors of tolerance and survival. Neurology. 2006;67:761–5.
   PubMed Web of Science ®Google Scholar
• Thakore NJ, Lapin BR, Pioro EP, Aboussouan LS. Variation in noninvasive ventilation use in amyotrophic lateral sclerosis. Neurology. 2019;93:e306–16.
   Web of Science ®Google Scholar
• Cousins R, Ando H, Thornton E, Chakrabarti B, Angus R, Young C. Determinants of accepting non-invasive ventilation treatment in motor neurone disease: a quantitative analysis at point of need. Health Psychol Behav Med. 2013;1:47–58.
   PubMedGoogle Scholar
• Galvin M, Corr B, Madden C, Mays I, McQuillan R, Timonen V, et al. Caregiving in ALS – a mixed methods approach to the study of burden. BMC Palliat Care. 2016;15:81.
   PubMed Web of Science ®Google Scholar
• Soofi AY, Bellow-Haas VD. Understanding the struggles for informal caregivers caring for people with amyotrophic lateral sclerosis. Ann Neurodegen Dis. 2018;3:1027.
   Google Scholar
• Torheim H, Gjengedal E. How to cope with the mask? Experiences of mask treatment in patients with acute chronic obstructive pulmonary disease-exacerbations. Scand J Caring Sci. 2010;24:499–506.
   PubMed Web of Science ®Google Scholar
• Martin NH, Landau S, Janssen A, Lyall R, Higginson I, Burman R, et al. Psychological as well as illness factors influence acceptance of non-invasive ventilation (NIV) and gastrostomy in amyotrophic lateral sclerosis (ALS): a prospective population study. Amyotroph Lateral Scler Frontotemporal Degener. 2014;15:376–87.
   PubMed Web of Science ®Google Scholar
• Gruis KL, Brown DL, Schoennemann A, Zebarah VA, Feldman EL. Predictors of noninvasive ventilation tolerance in patients with amyotrophic lateral sclerosis. Muscle Nerve. 2005;32:808–11.
   PubMed Web of Science ®Google Scholar
• Moore A, Young CA, Hughes DA. Health utilities and costs for motor neurone disease. Value Health. 2019;22:1257–65.
   PubMed Web of Science ®Google Scholar
• Beghi E, Chiò A, Couratier P, Esteban J, Hardiman O, Logroscino G, et al. The epidemiology and treatment of ALS: focus on the heterogeneity of the disease and critical appraisal of therapeutic trials. Amyotroph Lateral Scler. 2011;12:1–10.
   PubMed Web of Science ®Google Scholar
• Wilson IB, Cleary PD. Linking clinical variables with health-related quality of life. A conceptual model of patient outcomes. JAMA. 1995;273:59–65.
   PubMed Web of Science ®Google Scholar
• Young CA, Ealing J, McDermott C, Williams T, Al-Chalabi A, Majeed T, et al. The relationships between symptoms, disability, perceived health and quality of life in amyotrophic lateral sclerosis/motor neuron disease. Amyotroph Lateral Scler Frontotemporal Degener. 2019;20:317–27.
   PubMed Web of Science ®Google Scholar
• Mayo NE, Moriello C, Asano M, van der Spuy S, Finch L. The extent to which common health-related quality of life indices capture constructs beyond symptoms and function. Qual Life Res. 2011;20:621–7.
   PubMed Web of Science ®Google Scholar
• Simmons Z, Felgoise SH, Bremer BA, Walsh SM, Hufford DJ, Bromberg MB, et al. The ALSSQOL: balancing physical and nonphysical factors in assessing quality of life in ALS. Neurology. 2006;67:1659–64.
   PubMed Web of Science ®Google Scholar
• Franchignoni F, Mora G, Giordano A, Volanti P, Chiò A. Evidence of multidimensionality in the ALSFRS-R Scale: a critical appraisal on its measurement properties using Rasch analysis. J Neurol Neurosurg Psychiatry. 2013;84:1340–5.
   PubMed Web of Science ®Google Scholar
• Yorke J, Moosavi SH, Shuldham C, Jones PW. Quantification of dyspnoea using descriptors: development and initial testing of the Dyspnoea-12. Thorax. 2010;65:21–6.
   PubMed Web of Science ®Google Scholar
• Chiò A, Calvo A, Moglia C, Mazzini L, Mora G, PARALS Study Group. Phenotypic heterogeneity of amyotrophic lateral sclerosis: a population based study. J Neurol Neurosurg Psychiatry. 2011;82:740–6.
   PubMed Web of Science ®Google Scholar
• Moore A, Young CA, Hughes DA. Mapping ALSFRS-R and ALSUI to EQ-5D in patients with motor neuron disease. Value Health. 2018;21:1322–9.
   PubMed Web of Science ®Google Scholar
• Chiò A, Mazzini L, Mora G. Disease-modifying therapies in amyotrophic lateral sclerosis. Neuropharmacology. 2020;167:107986.
   PubMed Web of Science ®Google Scholar
• Gysels MH, Higginson IJ. The lived experience of breathlessness and its implications for care: a qualitative comparison in cancer, COPD, heart failure and MND. BMC Palliat Care. 2011;10:15.
   PubMed Web of Science ®Google Scholar