Curr Treat Options Neurol (2014) 16:270 DOI 10.1007/s11940-013-0270-5

NEUROMUSCULAR DISORDERS (SA RUDNICKI, SECTION EDITOR)

Respiratory and Nutritional Support in Amyotrophic Lateral Sclerosis Namita A. Goyal, MD* Tahseen Mozaffar, MD Address *Department of Neurology, University of California, Irvine, CA, USA Email: [email protected]

Published online: 4 January 2014 * Springer Science+Business Media New York 2014

This article is part of the Topical Collection on Neuromuscular Disorders Keywords Amyotrophic lateral sclerosis I ALS I Respiratory support I Nutritional support I Respiratory insufficiency I Noninvasive ventilation I Percutaneous endoscopic gastrostomy I Enteral nutrition I Diaphragmatic pacing ABBREVIATIONS AAN American Academy of Neurology I ALS Amyotrophic lateral sclerosis I BMI Body mass index I DPS Diaphragmatic pacing stimulators I EN Enteral nutrition I FVC Forced vital capacity I HFCWO Highfrequency chest wall oscillation I IPAP Inspiratory airway pressure I MIE Mechanical insufflation/exsufflation I MIP Maximal inspiratory pressure I MIF Maximal mouth-inspiratory force I NIPPV Noninvasive positive pressure ventilation I PCEF Peak cough expiratory flow I Pdi Transdiaphragmatic pressure I PEG Percutaneous endoscopic gastrostomy I RIG Radiologically inserted gastrostomy I SNIP Sniff nasal inspiratory pressure I SVC Slow vital capacity

Opinion statement Amyotrophic lateral sclerosis (ALS) is an uncommon and almost invariably fatal neurodegenerative disease. There is no known cure for ALS, and only one disease-modifying therapy is currently approved. In the absence of robust pharmacologic treatment options, the value of nutritional and respiratory support in the management of the disease should not be underestimated. The primary causes of morbidity and mortality in ALS are complications from dysphagia, leading to malnutrition and respiratory insufficiency, and these require focused therapeutic attention. This article reviews the current evidence for nutritional and respiratory support in the management of ALS patients.

Introduction Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition characterized by progressive weakness and amyotrophy due to selective motor neuron loss in

the spinal cord, brainstem, and motor cortex, resulting in significant disability and eventually death, with a median survival of three to five years [1]. As the cause of the

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disease is still unknown, there is no cure for ALS, although a number of disease-modifying therapies are available. Only one drug, riluzole, that has shown modest benefit has been approved by the Food and Drug Administration, and was the subject of a practice advisory published by the American Academy of Neurology (AAN) in 1997 and again in 2012 [2, 3]. The mainstay of therapy, therefore, is symptomatic multidisciplinary management. Several advances have been made in ad-

dressing the complex issues that arise in ALS, including respiratory insufficiency, dysarthria, dysphagia, weight loss, functional decline, and psychosocial stressors. The primary causes of morbidity and mortality in ALS are complications from dysphagia, leading to malnutrition and respiratory insufficiency, and these require focused therapeutic attention. In this article we address the management of respiratory and nutritional care in patients with ALS.

Respiratory management The diagnosis and management of respiratory insufficiency in ALS is critical, as this feature is present in almost all ALS cases at some stage of the disease [4••]. Respiratory failure is the most common cause of death in ALS [5], and therefore a decline in respiratory function is an important negative prognostic indicator [6]. Reduced ventilation results in part from respiratory muscle weakness, secondary to progressive lower motor neuron degeneration of the phrenic nerve, which innervates the diaphragm. Respiratory muscle weakness is defined as the inability of respiratory muscles to generate normal levels of pressure and airflow during inspiration and expiration [7]. This leads to respiratory insufficiency, which is defined as inadequate pulmonary ventilation resulting in impairment of gas exchange, causing carbon dioxide retention, hypoxemia, and eventually respiratory failure [7, 8]. Respiratory complications in ALS patients result from diaphragmatic weakness, difficulty clearing respiratory secretions, ineffective cough, inability to handle oropharyngeal secretions, and sleep-disordered breathing caused by nocturnal hypoventilation [9–11]. Randomized and controlled data have indicated that treatment of respiratory insufficiency in ALS patients with long-term noninvasive ventilation (NIV) appears to improve survival [12, 13] and quality of life [14], which may contribute to a slower rate of pulmonary function decline [15, 16]. In light of this data, in both 1999 and 2009, the AAN ALS evidence-based practice parameters recommended management of respiratory insufficiency with NIV for ALS patients with significant respiratory muscle weakness [3, 4]. However, despite these recommendations, respiratory therapies remain underutilized in the U.S., with only 21 % of the 5,600 patients in the ALS Care Database study receiving NIV [17]. The percentage of patients who had respiratory parameters that would support NIV is unknown.

Pulmonary tests to detect respiratory insufficiency Vital capacity Forced vital capacity (FVC) is the most commonly used respiratory measurement in ALS [18]. FVC is a significant predictor of survival, and G50 % of the predicted value has been shown to be associated with poor prognosis

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[19]. Slow vital capacity (SVC) is a variant of vital capacity measurement and may reflect a more accurate representation of diaphragmatic weakness in advanced cases [20]. Supine FVC may be a better predictor of diaphragm weakness than erect FVC [21], as the diaphragm is the main inspiratory muscle in the supine position [22]. FVC closely correlates with transdiaphragmatic pressure (Pdi), and a supine FVCG75 % reliably predicts an abnormally low Pdi, [23] indicating diaphragmatic weakness and thus respiratory insufficiency. Although commonly used, FVC is not an ideal test of respiratory muscle strength in ALS. A decline in FVC may not be noted until there is considerable muscle weakness, in part due to the sigmoid shape of the lung pressure-volume curve, which causes the VC measurement to be insensitive to modest changes [24]. Additionally, patients with bulbar weakness have difficulty making a tight seal around the mouthpiece, resulting in an inaccurate reflection of their respiratory muscle strength. Similarly, limitations due to the presence of bulbar weakness apply to alternative tests of respiratory muscle strength, such as the maximal inspiratory pressure (MIP), also called the maximal mouth-inspiratory force (MIF), and the maximal mouth-expiratory force [25].

Sniff nasal inspiratory pressure The maximal sniff nasal inspiratory pressure (SNIP) does not require a seal around the mouthpiece when the test is performed, and thus has the advantage of being able to be used in patients with bulbar dysfunction [20]. The SNIP has been shown to correlate well with diaphragmatic strength [26] and is sensitive to changes in respiratory muscle strength [27]. In a prospective observational study of 98 patients with ALS, a SNIP of G40 cm H2O correlated with nocturnal hypoxia and had a higher sensitivity of predicting 6-month mortality compared with VC of G50 % (97 versus 58 %, respectively) [25]. When the SNIP in ALS patients was G30 cm, the median survival was 3 months [25].

Other measures of respiratory insufficiency Nocturnal polysomnographic recording of oxygen desaturation is a useful marker of respiratory dysfunction. Nocturnal desaturations G90 % for 1 cumulative minute is a more sensitive indicator of nocturnal hypoventilation than either FVC or MIP [28]. Hypercapnia, or elevated carbon dioxide levels, may be measured using end-tidal carbon dioxide monitoring (a noninvasive measurement of exhaled CO2) or in arterialized venous blood using a noninvasive transcutaneous earlobe monitor, allowing for a simple and efficient screening for respiratory failure. A recent study in ALS patients measured the partial pressure of carbon dioxide in each of 40 patients, comparing the method of measurement of transcutaneous earlobe monitoring to that obtained by an arterialized earlobe capillary blood sample, and found minimal differences in the two methods, suggesting that noninvasive transcutaneous carbon dioxide monitoring is a useful tool in evaluating for signs of respiratory insufficiency in ALS patients [29]. The sniff Pdi is effective in detecting

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Curr Treat Options Neurol (2014) 16:270 hypercapnia (earlobe blood gas CO2 tension 96 kPa [normal G6]) and correlates with the apnea-hypopnea index on polysomnography [26]. Peak cough expiratory flow (PCEF) is a commonly used measure of cough effectiveness. A mean PCEF 9337 L/min indicates a greater chance of survival at 18 months [30].

Noninvasive ventilation Noninvasive positive pressure ventilation (NIPPV) is a therapeutic option in ALS treatment that has been shown to improve the quality of life in those that can tolerate its use [31] and which suggests prolonged survival [12]. Current guidelines recommend that NIPPV should be initiated in ALS patients when there is evidence of respiratory decline based on the following criteria: vital capacity G50 %, SNIP G40 cm H2O, MIPG-60 cm, abnormal nocturnal oximetry (G90 % for one cumulative minute), or symptomatic hypercapnia [4, 25, 32]. “Early” intervention with NIPPV and use of NIPPV 94 hours/day have been associated with greater survival, with a slower rate of FVC decline in comparison to patients using the device G4 hours/day [13, 16]. While studies indicate that NIPPV is beneficial in patients with normal to moderately impaired bulbar function, there is no evidence to suggest that NIPPV prolongs survival in patients with severe bulbar impairment, although quality of life, including sleep-related symptoms, may be improved [4, 12, 33]. NIPPV should be initiated early in patients with respiratory compromise, as time and patience is often required, with regular review and adjustment of settings by respiratory therapists to achieve optimal use. NIPPV compliance and benefits are often driven by patient tolerance of the face mask, which is especially worsened when there is a sense of claustrophobia. Several different face masks are available to choose from, and patients should be encouraged not to forgo treatment simply because they are not comfortable with the initial mask [34]. A small dose of an anxiolytic (benzodiazepine) may also help patients adjust to NIPPV. Other factors that adversely affect the NIPPV tolerability include the presence of bulbar symptoms, with increased secretions [35] and presence cognitive impairment [36]. Auto-titrating NIV devices are relatively new alternative to the standard bilevel PAP. In auto-titrating NIV, clinicians can specify a target tidal volume and a range for the maximum and minimum inspiratory airway pressure (IPAP). Examples of auto-titrated NIV include the Trilogy mechanical ventilator (Philips Respironics), BiPAP AVAPS (Philips Respironics), and the VPAP Auto (ResMed). In theory, the auto-titrated NIV is superior to the standard bilevel NIV with respect to NIV tolerance, as only the IPAP is required to support the target tidal volume. However, a randomized crossover study comparing the two types of devices in patients with neuromuscular

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disease showed that auto-titrated NIV provided less ventilatory support and was not associated with improved patient self-reported tolerance [37]. The data thus far have failed to indicate a clear advantage of auto-titrated versus standard NIV for respiratory insufficiency in ALS patients. Furthermore, benefits of volume ventilation, such as in the Trilogy and similar devices, over standard bilevel positive-pressure ventilation have not been demonstrated.

Diaphragmatic pacing The role of diaphragmatic pacing stimulators (DPS) in ALS has also been under consideration as a way to postpone the need for invasive mechanical ventilation. DPS involves intramuscular electrode implantation to provide maximal contraction of the diaphragm. The purpose of DPS is to maintain diaphragm strength, with the goal of prolonging time to tracheostomy [38]. A small nonrandomized study of 16 ALS patients showed an improvement in FVC rate of decline from 2.4 % per month before surgery to 0.9 % per month after surgery [38]. While early results are favorable, the value of DPS in ALS patients remains uncertain given the limited data available, and larger randomized controlled trials are needed before DPS is used in routine clinical care. ClinicalTrials.gov indicates that trials NCT01938495 and NCT01605006 are currently ongoing to evaluate the benefit of DPS in ALS patients . The FDA has given Humanitarian Device Exemption approval for DPS, and therefore the device does not require scientific evidence of efficacy to be marketed. However, the manufacturer’s application must contain sufficient information for the FDA to determine that the benefit to health outweighs the risk of injury or illness from use of the device [34].

Invasive ventilation It is important that ALS patients are well-informed about the course of their disease from the time of diagnosis onward. Formal discussions regarding tracheostomy with mechanical ventilation and advanced directives regarding end of life are optimal when the patient is stable, well before respiratory failure develops. Tracheostomy with invasive mechanical ventilation can be considered when the goal is long-term survival. The use of tracheostomy ventilation in ALS patients is varies widely by country, from 1.4–15 % of referring centers in the United States, to a majority of patients in Japan [39, 40]. One U.S. study showed a mean annual cost of home ventilation of approximately $150,000 [39]. Emergent tracheostomy is initiated for symptoms of respiratory failure when patients are intolerant to NIV, often due to the inability to clear respiratory secretions in the setting of severe bulbar weakness [34] or in the setting of respiratory failure in those patients without advanced directives [41] who were less likely to receive information about impending respiratory failure [42]. Semi-urgent elective tracheostomy with mechanical ventilation should be considered when daytime oxygen saturation is G95 % despite

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Curr Treat Options Neurol (2014) 16:270 maximal NIV therapy [43]. It is reasonable for an ALS clinician to initiate a discussion regarding elective tracheostomy when 1) the patient uses NIV 912 hours in a 24-hour time period, or 2) the patient is intolerant to NIV and FVC G50 % or symptoms of dyspnea are present [34]. Although invasive mechanical ventilation avoids death from respiratory failure and prolongs life, it is not associated with indefinite survival [34]. Median survival ranges from G12 months to 37 months when tracheostomy is initiated for respiratory failure, with the most common cause of death being respiratory tract infection [41, 44–46]. With disease progression, as many as 50–70 % of ALS patients with tracheostomy have minimal communication ability, including loss of eye movement, and risk becoming essentially “locked-in” [47, 48]. As such, it is imperative that the wishes of patients are known prior to loss of communication and that their advance directives outline when and if a decision for withdrawing ventilation is to be made.

Respiratory interventions for secretion clearance Preventive management strategies for airway secretion clearance should be undertaken prior to the need for mechanical ventilation. Education in aspiration risk-reduction techniques and secretion management may help prepare the patient and may avoid acute crises. Treatment of tenacious secretions with mucolytic agents (such as guaifenesin or N-acetylcysteine), nebulized saline with beta-receptor antagonists (metoprolol or propranolol), or anticholinergic bronchodilators (such as ipratropium), as well as adequate hydration, are often helpful [32]. Excessive secretions may be managed with hyoscine or scopolamine patches, amitriptyline, or glycopyrrolate. When these agents are ineffective in the management of sialorrhea, botulinum toxin injections of the salivary or parotid glands or even low-dose radiation therapy to the salivary glands may help [32]. Expiratory muscle weakness may lead to ineffective cough, retained upper airway secretions, and subsequent pulmonary tract infection [4]. Suctioning secretions and assistive devices such as a cough insufflator [mechanical insufflation/exsufflation (MIE)] are recommended when the peak cough expiratory flow (PCEF) falls below 270 L/min [49]. High-frequency chest wall oscillation (HFCWO) is an alternative approach for clearing airway secretions that has been shown to be effective in cystic fibrosis patients [50, 51], but there is insufficient data to support its benefit in patients with ALS. A small study in 9 ALS patients showed no benefit in the rate of FVC decline or in survival [52]. Importantly, oxygen therapy should not be used in patients with early evidence of respiratory failure, as it suppresses the hypoxic drive and increases the risk of hypercapnia [20].

Nutritional support Malnutrition in ALS Malnutrition is a frequent finding in ALS, with prevalence varying between 16 % and 53 % depending on the factors considered and the mode of pre-

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sentation of the disease [53]. Dysphagia, present in more than 81 % of patients with advanced ALS [54], increases the risk for inadequate caloric and fluid intake, increases the risk of aspiration, and leads to worsening of weakness and fatigue [3, 55]. The loss of 910 % of body weight or a body mass index (BMI) G18.5 kg/m2 are supportive indices of malnutrition and negative predictors of survival in ALS patients [53]. Significant evidence suggests that low BMI and malnutrition negatively affect the disease course and survival in patients with ALS, with a 7-fold increase in mortality in malnourished ALS patients [56–59]. Deterioration of nutritional status in ALS results from multifactorial causes. including muscle atrophy, hypophagia secondary to loss of autonomy and dysphagia, and hypermetabolism [60]. Strategies in preventing malnutrition have a positive impact on survival and quality of life [61, 62]. The AAN practice parameters and European ALS Consortium suggest that proactive nutrition management and education is the standard of care [4, 32], and recommend regular nutrition assessments every 3 months [3]. Additionally, increasing attention is being given to dietary intervention in the treatment of ALS, such as evaluating the role of a high-fat, high-calorie diet. Studies in the mutant superoxide dismutase-1 (SOD1) mouse model of ALS have shown that a high-fat diet slowed disease progression [63, 64] and, conversely, that caloric restriction reduced survival [65]. A recent large multicenter randomized clinical trial assessed the potential efficacy of a high-fat versus high-calorie versus optimal nutrition diet in ALS patients, the results of which have not yet been published (ClinicalTrials.gov NCT00983983). Early in the ALS disease course, when dysphagia is not present or is mild so that it does not impair physiologic eating, monthly monitoring of food and fluid intake to maintain adequate nutrition and weight stability is sufficient [60]. When dysphagia occurs, initial strategies focus on diet modification to soft semisolid textured foods, with thicker liquids to help prevent risk of aspiration [66, 67]. If these modifications cannot ensure adequate nutrition, and weight loss progresses despite intervention (BMI G20 kg/m2 or 95–10 % body weight loss) [53], artificial nutrition becomes an essential consideration [53, 68, 69].

Enteral nutrition Enteral nutrition (EN) is an alternative long-term medical treatment of choice in ALS patients for delivering nutrition, typically given at home or in nursing homes, and which requires adequate enteral access. Percutaneous endoscopic gastrostomy (PEG) is the most commonly used enteral access for medium- to long-term EN [69]. The AAN practice parameters suggest PEG placement should be performed while FVC is 950 % of predicted value, as the risk of PEG placement increases when FVC falls below 50 % of predicted [4]. An alternative to PEG is the radiologically inserted gastrostomy (RIG) that is performed through percutaneous access with local anesthesia and

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Curr Treat Options Neurol (2014) 16:270 fluoroscopic guidance. In comparison to PEG, the RIG procedure has been associated with lower rates of complications and higher rates of technical success, even in patients with significant respiratory insufficiency [70, 71]. The primary disadvantages noted in RIG have included risk of obstruction due to the small diameter of the tube as well as dislocation of the tube [72]. Several studies have shown that PEG is effective in stabilizing body weight/ body mass index [56, 73–76] and in prolonging survival in ALS [75, 77].

Parenteral nutrition and nasogastric/jejunal feeding Parenteral nutrition (PN) is rarely indicated in ALS patients. PN is considered as an alternative method only when consent to EN is denied or EN is contraindicated from an inability to access or use the gastrointestinal tract due to malabsorption, dysmotility, or enterocutaneous fistulas. An observational study comparing EN to PN showed similar survival times in ALS patients with respiratory insufficiency; however, PN was 6 times more expensive than EN [78]. Nasogastric or nasojejunal feeding is not recommended for longterm EN due to patient discomfort, mechanical complications [69], and risk of worsening respiratory insufficiency [78].

Alternative therapies: vitamins and nutritional supplements Alternative therapies such as high-dose vitamins, minerals, and other dietary supplements (also called nutraceuticals), adjunct to traditional therapies, are becoming increasing popular amongst ALS patients, with up to 80 % use, according to the literature [44, 79]. In one study, a telephone survey of ALS patients reported the use of 23 different nutritional supplements and functional foods [80]. Despite the wide use of supplements, there is limited evidence to suggest an impact on altering disease progression. Two studies evaluating creatine at 10 g/day and 5 g/ day failed to alter survival or rate of functional decline of ALS patients [81, 82]. Alpha-tocopherol (vitamin E) given concurrently with riluzole failed to slow the rate of functional decline or impact overall survival [83, 84]. Despite a lack of evidence of significant benefit of dietary supplements, antioxidants are generally well-tolerated, without serious adverse effects [85], which may offer some degree of hope and thereby improve the quality of life for the ALS patient.

Compliance with Ethics Guidelines Conflict of Interest Namita A. Goyal and Tahseen Mozaffar declare that they have no conflict of interest. Human and Animal Rights and Informed Consent This article does not contain any studies with human or animal subjects performed by any of the authors.

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References and Recommended Reading 1. 2.

3.

4.

5. 6.

7.

8.

9.

10.

11.

12.

13.

14.

Rowland, Shneider N. Amyotrophic lateral sclerosis. N Engl J Med. 2001;344(22):1688–700. Miller R, Mitchell J, Moore D. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane Database Syst Rev. 2012;3:CD001447. Miller R, Rosenberg J, Gelinas D, et al. Practice parameter: the care of the patient with amyotrophic lateral sclerosis (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology: ALS Practice Parameters Task Force. Neurology. 1999;52(7):1311–23. Miller R, Jackson C, Kasarskis E, 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(15):1218–26. Corcia P, Meininger V. Management of amyotrophic lateral sclerosis. Drugs. 2008;68(8):1037–48. Chiò A, Logroscino G, Hardiman O, et al. Prognostic factors in ALS: A critical review. Amyotroph Lateral Scler. 2009;10(5–6):310–23. Bergofsky E. Respiratory failure in disorders of the thoracic cage. Am Rev Respir Dis. 1979;119(4):643– 69. De Troyer A, Borenstein S, Cordier R. Analysis of lung volume restriction in patients with respiratory muscle weakness. Thorax. 1980;35(8):603–10. Bach J. Amyotrophic lateral sclerosis: prolongation of life by noninvasive respiratory AIDS. Chest. 2002;122(1):92–8. Hadjikoutis S, Wiles C. Respiratory complications related to bulbar dysfunction in motor neuron disease. Acta Neurol Scand. 2001;103(4):207–13. Mustfa N, Moxham J. Respiratory muscle assessment in motor neurone disease. QJM. 2001;94(9):497– 502. Bourke S, Tomlinson M, Williams T, et al. 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(2):140–7. Pinto A, de Carvalho M, Evangelista T, et al. Nocturnal pulse oximetry: a new approach to establish the appropriate time for non-invasive ventilation in ALS patients. Amyotroph Lateral Scler Other Motor Neuron Disord. 2003;4(1):31–5. Bourke S, Bullock R, Williams T, et al. Noninvasive ventilation in ALS: indications and effect on quality of life. Neurology. 2003;61(2):171–7.

15.

16.

17.

18.

19.

20. 21.

22.

23.

24.

25.

26.

27.

Carratù P, Spicuzza L, Cassano A, 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. Kleopa K, Sherman M, Neal B, et al. Bipap improves survival and rate of pulmonary function decline in patients with ALS. J Neurol Sci. 1999;164(1):82–8. McKim D, Road J, Avendano M, et al. Home mechanical ventilation: a Canadian Thoracic Society clinical practice guideline. Can Respir J. 2011;18(4):197–215. Melo J, Homma A, Iturriaga E, et al. Pulmonary evaluation and prevalence of non-invasive ventilation in patients with amyotrophic lateral sclerosis: a multicenter survey and proposal of a pulmonary protocol. J Neurol Sci. 1999;169(1–2):114–7. Czaplinski A, Yen A, Appel S. Forced vital capacity (FVC) as an indicator of survival and disease progression in an ALS clinic population. J Neurol Neurosurg Psychiatry. 2006;77(3):390–2. Hardiman O. Management of respiratory symptoms in ALS. J Neurol. 2011;258(3):359–65. Varrato J, Siderowf A, Damiano P, et al. Postural change of forced vital capacity predicts some respiratory symptoms in ALS. Neurology. 2001;57(2):357–9. Baumann F, Henderson R, Morrison S, et al. Use of respiratory function tests to predict survival in amyotrophic lateral sclerosis. Amyotroph Lateral Scler. 2010;11(1–2):194–202. Lechtzin N, Wiener C, Shade D, et al. Spirometry in the supine position improves the detection of diaphragmatic weakness in patients with amyotrophic lateral sclerosis. Chest. 2002;121(2):436– 42. Fitting J, Paillex R, Hirt L, et al. Sniff nasal pressure: a sensitive respiratory test to assess progression of amyotrophic lateral sclerosis. Ann Neurol. 1999;46(6):887–93. Morgan R, 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(3):269–74. Lyall R, Donaldson N, Polkey M, et al. Respiratory muscle strength and ventilatory failure in amyotrophic lateral sclerosis. Brain. 2001;124(Pt 10):2000– 13. Stefanutti D, Benoist M, Scheinmann P, et al. Usefulness of sniff nasal pressure in patients with neuromuscular or skeletal disorders. Am J Respir Crit Care Med. 2000;162(4 Pt 1):1507–11.

270, Page 10 of 12 28.

29.

30.

31.

32.

33.

34.

35.

36.

37.

38.

39.

40.

41.

Jackson C, Rosenfeld J, Moore D, et al. A preliminary evaluation of a prospective study of pulmonary function studies and symptoms of hypoventilation in ALS/MND patients. J Neurol Sci. 2001;191(1– 2):75–8. Rafiq M, Bradburn M, Proctor A, et al. Using transcutaneous carbon dioxide monitor (TOSCA 500) to detect respiratory failure in patients with amyotrophic lateral sclerosis: a validation study. Amyotroph Lateral Scler. 2012;13(6):528–32. Chaudri M, Liu C, Hubbard R, et al. Relationship between supramaximal flow during cough and mortality in motor neurone disease. Eur Respir J. 2002;19(3):434–8. Mustfa N, Walsh E, Bryant V, et al. The effect of noninvasive ventilation on ALS patients and their caregivers. Neurology. 2006;66(8):1211–7. Andersen P, Borasio G, Dengler R, et al. Good practice in the management of amyotrophic lateral sclerosis: clinical guidelines. An evidence-based review with good practice points EALSC Working Group Amyotroph Lateral Scler. 2007;8(4):195–213. Radunovic A, Annane D, Rafiq M, et al. Mechanical ventilation for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2013;3:CD004427. Gruis K, Lechtzin N. Respiratory therapies for amyotrophic lateral sclerosis: a primer. Muscle Nerve. 2012;46(3):313–31. Gruis K, Brown D, Schoennemann A, et al. Predictors of noninvasive ventilation tolerance in patients with amyotrophic lateral sclerosis. Muscle Nerve. 2005;32(6):808–11. Olney R, Murphy J, Forshew D, et al. The effects of executive and behavioral dysfunction on the course of ALS. Neurology. 2005;65(11):1774–7. Jaye J, Chatwin M, Dayer M, et al. Autotitrating versus standard noninvasive ventilation: a randomised crossover trial. Eur Respir J. 2009;33(3):566–71. Onders R, Carlin A, Elmo M, et al. Amyotrophic lateral sclerosis: the Midwestern surgical experience with the diaphragm pacing stimulation system shows that general anesthesia can be safely performed. Am J Surg. 2009;197(3):386–90. Moss A, Casey P, Stocking C, et al. Home ventilation for amyotrophic lateral sclerosis patients: outcomes, costs, and patient, family, and physician attitudes. Neurology. 1993;43(2):438–43. Borasio G, Gelinas D, Yanagisawa N. Mechanical ventilation in amyotrophic lateral sclerosis: a crosscultural perspective. J Neurol. 1998;245 Suppl 2:S7– 12. Vianello A, Arcaro G, Palmieri A, et al. Survival and quality of life after tracheostomy for acute respiratory failure in patients with amyotrophic lateral sclerosis. J Crit Care. 2011;26(3):

Curr Treat Options Neurol (2014) 16:270 42.

43.

44.

45.

46.

47.

48.

49.

50.

51.

52.

53.

54.

55.

Kaub-Wittemer D, Steinbüchel N, Wasner M, et al. Quality of life and psychosocial issues in ventilated patients with amyotrophic lateral sclerosis and their caregivers. J Pain Symptom Manage. 2003;26(4):890–6. Bach J, Bianchi C, Aufiero E. Oximetry and indications for tracheotomy for amyotrophic lateral sclerosis. Chest. 2004;126(5):1502–7. Bradley W, Anderson F, Gowda N, et al. Changes in the management of ALS since the publication of the AAN ALS practice parameter 1999. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004;5(4):240–4. Chiò A, Calvo A, Ghiglione P, et al. Tracheostomy in amyotrophic lateral sclerosis: a 10-year populationbased study in Italy. J Neurol Neurosurg Psychiatry. 2010;81(10):1141–3. Lo Coco D, Marchese S, La Bella V, et al. The amyotrophic lateral sclerosis functional rating scale predicts survival time in amyotrophic lateral sclerosis patients on invasive mechanical ventilation. Chest. 2007;132(1):64–9. Hayashi H, Oppenheimer E. ALS patients on TPPV: totally locked-in state, neurologic findings and ethical implications. Neurology. 2003;61(1):135–7. Rabkin J, Albert S, Tider T, et al. Predictors and course of elective long-term mechanical ventilation: A prospective study of ALS patients. Amyotroph Lateral Scler. 2006;7(2):86–95. Sancho J, Servera E, Díaz J, et al. Efficacy of mechanical insufflation-exsufflation in medically stable patients with amyotrophic lateral sclerosis. Chest. 2004;125(4):1400–5. Arens R, Gozal D, Omlin K, et al. Comparison of high frequency chest compression and conventional chest physiotherapy in hospitalized patients with cystic fibrosis. Am J Respir Crit Care Med. 1994;150(4):1154–7. Warwick W, Hansen L. The long-term effect of highfrequency chest compression therapy on pulmonary complications of cystic fibrosis. Pediatr Pulmonol. 1991;11(3):265–71. Chaisson K, Walsh S, Simmons Z, et al. A clinical pilot study: high frequency chest wall oscillation airway clearance in patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler. 2006;7(2):107– 11. Piquet MA. [Nutritional approach for patients with amyotrophic lateral sclerosis]. Rev Neurol (Paris). 2006;162(Spec No 2):4S177-4S187. Traynor B, Codd M, Corr B, et al. Incidence and prevalence of ALS in Ireland, 1995-1997: a population-based study. Neurology. 1999;52(3):504– 9. Borasio G, Shaw P, Hardiman O, et al. Standards of palliative care for patients with amyotrophic lateral sclerosis: results of a European survey. Amyotroph

Curr Treat Options Neurol (2014) 16:270

56.

57.

58.

59. 60.

61.

62.

63.

64.

65.

66. 67.

68.

69.

70.

Lateral Scler Other Motor Neuron Disord. 2001;2(3):159–64. Desport J, Preux P, Truong C, et al. Nutritional assessment and survival in ALS patients. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000;1(2):91–6. Desport J, Preux P, Truong T, et al. Nutritional status is a prognostic factor for survival in ALS patients. Neurology. 1999;53(5):1059–63. Heffernan C, Jenkinson C, Holmes T, et al. Nutritional management in MND/ALS patients: an evidence based review. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004;5(2):72–83. Kasarskis E, Neville H. Management of ALS: nutritional care. Neurology. 1996;47(4 Suppl 2):S118–20. Atsuta N, Watanabe H, Ito M, et al. Age at onset influences on wide-ranged clinical features of sporadic amyotrophic lateral sclerosis. J Neurol Sci. 2009;276(1–2):163–9. Mitsumoto H, Rabkin J. Palliative care for patients with amyotrophic lateral sclerosis: "prepare for the worst and hope for the best". JAMA. 2007;298(2):207–16. Park J, Kang S-W. Percutaneous radiologic gastrostomy in patients with amyotrophic lateral sclerosis on noninvasive ventilation. Arch Phys Med Rehabil. 2009;90(6):1026–9. Mattson M, Cutler R, Camandola S. Energy intake and amyotrophic lateral sclerosis. Neuromolecular Med. 2007;9(1):17–20. Dupuis L, Oudart H, René F, et al. Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high-energy diet in a transgenic mouse model. Proc Natl Acad Sci U S A. 2004;101(30):11159–64. Hamadeh M, Rodriguez M, Kaczor J, et al. Caloric restriction transiently improves motor performance but hastens clinical onset of disease in the Cu/Znsuperoxide dismutase mutant G93A mouse. Muscle Nerve. 2005;31(2):214–20. Langmore S. Issues in the management of dysphagia. Folia Phoniatr Logop. 1999;51(4–5):220–30. Perry A, Anderson K, Lean R, et al. Elevation of the soft palate in speech and swallowing in normal female participants and females with motor neuron disease: an innovative procedure for measuring palatal elevation. Int J Lang Commun Disord. 2002;37(2):197–214. Cameron A, Rosenfeld J. Nutritional issues and supplements in amyotrophic lateral sclerosis and other neurodegenerative disorders. Curr Opin Clin Nutr Metab Care. 2002;5(6):631–43. Katzberg H, Benatar M. Enteral tube feeding for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2011;1:CD004030. Allen J, Chen R, Ajroud-Driss S, et al. Gastrostomy tube placement by endoscopy versus radiologic

Page 11 of 12, 270

71.

72.

73.

74.

75.

76.

77.

78.

79.

80.

81.

82.

83.

methods in patients with ALS: a retrospective study of complications and outcome. Amyotroph Lateral Scler Frontotemporal Degener. 2013;14(4):308–14. 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(2):88–93. McLoughlin R, Gibney R. Fluoroscopically guided percutaneous gastrostomy: tube function and malfunction. Abdom Imaging. 1994;19(3):195–200. Chiò A, Finocchiaro E, Meineri P, et al. Safety and factors related to survival after percutaneous endoscopic gastrostomy in ALS. ALS Percutaneous Endoscopic Gastrostomy Study Group. Neurology. 1999;53(5):1123–5. Desport J-C, Torny F, Lacoste M, et al. Hypermetabolism in ALS: correlations with clinical and paraclinical parameters. Neurodegener Dis. 2005;2(3–4):202–7. Mazzini L, Corrà T, Zaccala M, et al. Percutaneous endoscopic gastrostomy and enteral nutrition in amyotrophic lateral sclerosis. J Neurol. 1995;242(10):695–8. Mitsumoto H, Davidson M, Moore D, et al. Percutaneous endoscopic gastrostomy (PEG) in patients with ALS and bulbar dysfunction. Amyotroph Lateral Scler Other Motor Neuron Disord. 2003;4(3):177– 85. Shaw A, Ampong M-A, Rio A, et al. Survival of patients with ALS following institution of enteral feeding is related to pre-procedure oximetry: a retrospective review of 98 patients in a single centre. Amyotroph Lateral Scler. 2006;7(1):16–21. 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(1):42–6. Rosenfeld J, Ellis A. Nutrition and dietary supplements in motor neuron disease. Phys Med Rehabil Clin N Am. 2008;19(3):573. Körner S, Hendricks M, Kollewe K, et al. Weight loss, dysphagia and supplement intake in patients with amyotrophic lateral sclerosis (ALS): impact on quality of life and therapeutic options. BMC Neurol. 2013;13:84. Groeneveld G, Veldink J, van der Tweel I, et al. A randomized sequential trial of creatine in amyotrophic lateral sclerosis. Ann Neurol. 2003;53(4):437– 45. Shefner J, Cudkowicz M, Schoenfeld D, et al. A clinical trial of creatine in ALS. Neurology. 2004;63(9):1656–61. Desnuelle C, Dib M, Garrel C, et al. A double-blind, placebo-controlled randomized clinical trial of alpha-tocopherol (vitamin E) in the treatment of

270, Page 12 of 12

84.

amyotrophic lateral sclerosis. ALS riluzole-tocopherol Study Group Amyotroph Lateral Scler Other Motor Neuron Disord. 2001;2(1):9–18. Graf M, Ecker D, Horowski R, et al. High dose vitamin E therapy in amyotrophic lateral sclerosis as

Curr Treat Options Neurol (2014) 16:270

85.

add-on therapy to riluzole: results of a placebo-controlled double-blind study. J Neural Transm. 2005;112(5):649–60. Orrell R. Understanding the causes of amyotrophic lateral sclerosis. N Engl J Med. 2007;357(8):822–3.

Respiratory and nutritional support in amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is an uncommon and almost invariably fatal neurodegenerative disease. There is no known cure for ALS, and only one...
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