THEME 8 RESPIRATORY AND NUTRITIONAL MANAGEMENT

P146 EVALUATION OF RESTING ENERGY EXPENDITURE IN AMYOTROPHIC LATERAL SCLEROSIS PATIENTS: COMPARISON BETWEEN METABOLIC MULTISENSOR ARMBAND AND INDIRECT CALORIMETRY

GALLETTI R¹, CHIÒ A², SABET D¹, FADDA M¹, MANCINO MV¹, RIVETTI M¹, CALVO A², FINOCCHIARO E¹, BOGGIO BD¹

¹Department of Clinical Nutrition, San Giovanni Battista Hospital, Turin, Italy, ²Department of Neuroscience, University of Turin and San Giovanni Battista Hospital, Turin, Italy

E-mail address for correspondence: [email protected]

Keywords: malnutrition, resting energy expenditure, indirect calorimetry

Background: Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease associated with dysphagia and consequent malnutrition. When oral feeding fails, enteral nutrition should be considered as an alternative or supplemental route for oral nutrition. It is therefore extremely important to strictly monitor patients’ nutritional status.

Objectives: The aim of our study was to validate the measurement of Resting Energy Expenditure (REE) with a new tool called the Metabolic Multisensor Armband (SWA), using Indirect Calorimetry (IC) as the gold standard. We also measured body composition with a Body Impedence Analyzer (BIA), because we wanted to test the hypothesis that patients with ALS, while presenting a reduction of Fat-Free Mass (FFM), show an increase of metabolism.

Methods: After overnight fasting, we made a measurement of REE for 30 minutes using both IC (Calorimeter Delta Trak Datex Ohmeda) and SWA (SenseWear Pro2 Armband); then we measured body composition by BIA (Impedenzometry Tetrapolar Akern 101) and we calculated Fat Free Mass (FFM) and Fat Mass (FM). The results are presented as means and standard deviation; comparison between methods was assessed using the Bland-Altman test. Values were compared with Student's t test for paired data and with linear regression. A P level <0.05 was considered significant. The total number of patients was 39, 17 male and 22 female; 12 had bulbar onset and 27 spinal onset.

Results: The patients’ mean age was 62.3 (±7.6) years, BMI 24.2 (±3.9), protein intake 62.5 (±13.7) g; nutritional blood tests were: total protein 7.2 (±0.4) g/dL; albumin 4.5 (±0.3) g/dL; transferrin 244 (±24.6) mg/dL; total cholesterol 190 (±36.3) mg/dL. The mean REE measured with DT was 1256 (±253) and measured with SWA was 1286 (±228) (P = 0.64). The two methods were highly comparable according to linear regression (DT vs SWA r² 0.957; P < 0.001). The comparison with Bland Altman test demonstrates that SWA underestimated REE by about −28.3 calories compared to DT. Body composition differed from normality (taking into consideration patients’ gender and age), with an increase of FM (59% in males and 60% in females) and a decrease of FFM (35% in males and 75% in females).

Conclusions: REE measurement with SWA was revealed to be accurate in predicting energy needs in patients with ALS at a non-advanced stage of disease. In our series, REE did not differ from normal controls.

P147 SAFETY OF HOME PARENTERAL NUTRITION IN PATIENTS WITH ALS: A FRENCH NATIONAL SURVEY

ABDELNOUR-MALLET M¹, VERSCHUEREN A², GUY N³, SORIANI M-H⁴, CHALBI M¹, GORDON P¹, SALACHAS F¹, BRUNETEAU G¹, LE FORESTIER N¹, LENGLET T¹, DESNUELLE C⁴, CLAVELOU P³, POUGET J², MEININGER V¹, PRADAT P-F¹

¹APHP, CHU Pitié-Salpêtrière, Paris, France, ²CHU La Timone, Marseille, France, ³CHU Montpied, Clermont-Ferrand, France, ⁴CHU Hôpital Archet, Nice, France

E-mail address for correspondence: [email protected]

Keywords: home parenteral nutrition, safety, radiologic inserted gastrostomy

Background: Gastrostomy is the most common alternative feeding device used in patients with amyotrophic lateral sclerosis (ALS) when oral intakes become inadequate. Percutaneous endoscopic gastrostomy (PEG) placement is not always possible in cases of severe respiratory failure. We carried out a retrospective multicentre study to assess the safety of home parenteral nutrition (HPN) in patients with ALS.

Patients and methods: We reviewed the case records of patients from French ALS centres treated with HPN by central venous catheter (CVC) using an implantable port between January 2005 and October 2009.

Results: Seventy-three patients received HPN for a total of 11908 catheter days. Twenty-seven patients experienced a total of 37 CVC related complications resulting in an incidence rate of 3.11 CVC complications /1000 catheter days, including 1.93 septic complications and 1.09 mechanical complications per 1000 catheter days. Metabolic complications were frequent but without serious consequences or mortality. The use of the catheter for intravenous therapies in addition to HPN was identified as a risk factor associated with a high rate of septicaemia (relative risk (RR) = 2.54, confidence interval CI (1.56;4.14), P = 0.04)

Discussion and conclusion: HPN is an alternative procedure to PEG in advanced ALS patients. The incidence of complications appears to be comparable to data from the literature on HPN in other diseases. A prospective study comparing HPN and radiologically inserted gastrostomy (RIG) would allow comparison of the relative risk-benefit and survival of these procedures. The relation of timing of CVC and RIG placement and the occurrence of complications should also be investigated.

P148 BELIEFS AROUND PERCUTANEOUS ENDOSCOPIC GASTROSTOMY TUBE PLACEMENT IN ALS PATIENTS

HOSKINS K, DAHODWALA N, ELMAN L, GOLASZEWSKI A, PALOVCAK M, MCCLUSKEY L

University of Pennsylvania, Philadelphia, PA, United States

E-mail address for correspondence: [email protected]

Keywords: percutaneous endoscopic gastrostomy tube, attitudes, decision-making

Background: The progressive muscle weakness characteristic of ALS may make swallowing and eating difficult or impossible. Though patients may qualify for placement of a percutaneous endoscopic gastrostomy tube (PEG) based on impaired swallowing, weight loss, and deteriorating vital capacity, some elect not to undergo or to delay the procedure.

Objectives: We explored the attitudes and concerns of ALS patients considering PEG placement to better understand the decision-making process.

Methods: With guidance from patient and caregiver groups, experts from various clinical disciplines collaborated to create a 32 question survey assessing factors related to the decision-making process, PEG placement procedure, stress related to eating, quality of life, caregiver involvement, body image, and role of a PEG in prolonging life. Patients with a predicted forced vital capacity of 50% or less, weight loss greater than or equal to 5–10% body mass, and/or difficulty swallowing both liquids and solids were educated about the risks/benefits of PEG placement. Thirty-five consenting patients completed our survey. We assessed responses with a bivariate statistical analysis comparing subjects who replied that they were either very likely or likely to choose a PEG (Group 1) to those who were very unlikely, unlikely, or not sure (Group 2).

Results: Seventy percent of respondents classified themselves as very likely or likely to choose a PEG and comprised Group 1. Group 1 expressed little concern that a PEG would impede their ability to eat/drink by mouth. They were neutral or had little concern regarding post-op pain, infection, and mortality. Group 1 demonstrated significantly more stress related to eating, drinking liquids, and taking medications than Group 2, believing that PEG placement would reduce the stress of eating (P = 0.0002) and drinking (P = 0.0007). Group 1 also believed that a PEG would significantly reduce choking, as well as reduce caregiver concerns. Sixty-eight percent of participants believed that a PEG would reduce risk of aspiration pneumonia. Over half believed that a PEG would increase survival. Though both groups’ quality of life means were comparable, Group 2 expressed greater concern that a PEG would extend their suffering with ALS. Group 1 believed that a PEG would likely improve their quality of life (P = 0.0001).

Discussion and conclusions: Current difficulty with eating, drinking, and taking medications plus the beliefs that a PEG would reduce the stress of eating and drinking, reduce patient and caregiver concerns, reduce choking on solids and liquids, as well as increase quality of life were all significant factors in the likelihood of electing PEG placement. Patients demonstrated misconceptions about the risks of aspiration pneumonia and the role of PEG in survival. More information is needed on the role of caregivers in the decision-making process. These issues should be incorporated into discussions surrounding possible PEG placement.

P149 NUTRITION AND RESPIRATION: COMBINING GASTROSTOMY WITH DIAPHRAGM PACING FOR IMPROVED SURVIVAL

ONDERS R, KATIRJI B, SCHILZ R, PONSKY J, ELMO M, KAPLAN C

University Hospitals Case Medical Center, Cleveland, Ohio, United States

E-mail address for correspondence: [email protected]

Keywords: gastrostomy, diaphragm pacing, PEG

Background: Since 1980 when the percutaneous endoscopic gastrostomy (PEG) was first described its role in ALS/MND was soon identified to help overcome weight loss and dehydration from dysphagia. Current AAN practice parameters state that PEG should be considered to stabilize weight and prolong survival. One of the major risk factors of PEG placement is further compromising respiratory function, which leads to a 30 day mortality rate approaching 25%. Diaphragm Pacing (DP) has over 10 years of use in spinal cord injured patients with pure upper motor neuron (UMN) involvement and PEGs. This report outlines the experience of DP and PEG in the combined UMN and lower motor neuron (LMN) involvement of the diaphragm in ALS/MND.

Objective: Analyze safety, utility and long term outcome of patients having simultaneous DP and PEG placement or utilizing DP during subsequent PEG placement.

Methods: Subgroup analysis of all patients who had DP and PEG placement during prospective, nonrandomized, controlled, pilot, and pivotal trials under IRB and/or FDA approval at a single institution as previously described.

Results: From 2005 until 2010, 17 patients had simultaneous DP and PEG and an additional 9 patients had PEG placement after DP implantation. The average age was 54 with 20 males and 6 females. Two patients were excluded: one due to an abdominal co-morbidity and one lost to follow-up. The FVC at either DP and PEG or PEG with DP was 53% predicted. There was no 30 day mortality. There were no peri-operative pulmonary infections. The 6 month survival from PEG placement with DP is 88% and the one year survival 73%. The average post PEG follow-up to survival in patients who have diaphragm pacing is 29 months.

Discussion: Sedation given during PEG placement can suppress the respiratory drive in ALS/MND patients leading to posterior lobe atelectasis and increased risk of respiratory infections. This was identified by reading the continuous diaphragm EMG activity of the implanted electrodes during a PEG showing significant periods of diaphragm apnea which was overcome with DP use. By utilizing DP we showed an increase in respiratory compliance by 18% which decreases the work of breathing and makes recovery from the procedure easier. ALS/MND patients have an increased respiratory instability leading to apneas with oxygen therapy or pain medication that may be needed from the PEG. DP maintains respiratory drive in these patients and therefore decreases those risks. These factors may explain the improved survival compared to Forbes who reported 25% 30 day mortality and a year survival rate of 23% for PEG alone.

Conclusion: PEG with DP can be safely implanted simultaneously with an improved survival compared to historical experience of PEG alone.

P150 POSITIVE CLINICAL RESULTS OF DIAPHRAGM PACING IN ALS/MND WITH CHRONIC HYPOVENTILATION AND UPPER MOTOR NEURON RESPIRATORY DEFICITS WITH INTACT LOWER MOTOR NEURON PHRENIC MOTOR FUNCTION

ONDERS R¹, KATIRJI B¹, SO Y², KATZ J³, MILLER R³, NEWMAN D⁴, SIMPSON E⁵, APPEL S⁵, BOYLAN K⁶, MARAGAKIS N⁷, LECHTZIN N⁷, ROTHSTEIN J⁷

¹University Hospitals Case Medical Center, Cleveland, Ohio, United States, ²Stanford University Medical Center, Palo Alto, California, United States, ³California Pacific Medical Center, San Francisco, California, United States, ⁴Henry Ford Hospital, Detroit, Michigan, United States, ⁵Methodist Neurological Institute, Houston, Texas, United States, ⁶Mayo Clinic, Jacksonville, Florida, United States, ⁷John Hopkins Medical Center, Baltimore, Maryland, United States

E-mail address for correspondence: [email protected]

Keywords: respiratory failure, diaphragm pacing, phrenic nerve

Background: Alternative therapies to help with respiration are needed in ALS/MND with a majority of deaths occurring in a median of three years from symptom onset from respiratory issues. In the United States, the Food and Drug Administration (FDA) has a separate pathway, humanitarian use device (HUD) designation, for approval of medical devices that affect fewer than 4000 patients per year. Safety and ‘probable benefit’ are analysed to allow access for patients with an orphan disease. The surgical safety and use of diaphragm pacing has been reported in ALS/MND. This report analyses the HUD designation subgroup of the DP database for whom diaphragm pacing will have long term probable benefit.

Objective: To report on the safety and probable benefit of DP in a post-hoc sub-group analysis of ALS/MND patients implanted in a prospective, nonrandomized, controlled, multicenter, interventional trial with a lead-in design and 9 month post -implantation treatment period with follow-up until death.

Methods: Subjects were included for analysis that had intact phrenic nerves as demonstrated by phrenic nerve conduction studies or clear visualization of diaphragm movement under fluoroscopy, who also had evidence of chronic hypoventilation by FVC < 50% or MIP <60 cm, H 2 O or PCO 2 > 45 mmHg.

Results: Eighty six subjects out of the total 145 subject enrolment from 3/2005 to 1/2009 met the inclusion criteria for analysis. Four patients were further excluded from analysis due to co-morbidities (2) and being lost to follow-up (2). The remaining patients have provided survival follow-up data with over 120 cumulative total years of use with no patients stopping use of the device due to adverse device effects. Subject demographics include average age 58, 67% male, median onset to implantation 33 months, 33% bulbar onset, 80% riluzole use, 76% use of NIV, ALSFRS-r at implant 28 and FVC at implant 59% predicted. In the patients who had declining FVC during lead in there was a positive improvement in the decline of 1.14% per month (95% CI 0.29, 1.97, P < 0.01). There was an observed 30% improvement in the rate of decline of ALSFRS-r post implantation though not statistically significant except when analyzing the respiratory sub-score alone in which there was an improvement. Twelve patients had pre and post implant sleep studies showing a significant improvement in sleep quality. Forty patients have died and 11 more have had a tracheostomy with full time ventilation. None were related to device use. The one year tracheostomy-free survival rate from implant is 70% with a median of 20 months. The median tracheostomy-free survival from symptom onset is 58 months.

Conclusion: The DPS system can be safely implanted and utilized in ALS patients improving survival. The degree of intact nerve function to the diaphragm is the key factor in providing a successful symptomatic therapy.

P151 NON-INVASIVE VENTILATION (NIV) AND DIAPHRAGM PACING: DP AUGMENTS AND IMPROVES THE EFFECTIVENESS OF NIV IN ALS/MND LEADING TO AN IMPROVED SURVIVAL

LECHTZIN N¹, MARAGAKIS N¹, ROTHSTEIN J¹, ONDERS R², KATIRJI B², SO Y³, KATZ J⁴, MILLER R⁴, NEWMAN D⁵, SIMPSON E⁶, APPEL S⁶, BOYLAN K⁷

¹John Hopkins Medical Center, Baltimore, Maryland, United States, ²University Hospitals Case Medical Center, Cleveland, Ohio, United States, ³Stanford University Medical Center, Palo Alto, California, United States, ⁴California Pacific Medical Center, San Francisco, California, United States, ⁵Henry Ford Hospital, Detroit, Michigan, United States, ⁶Methodist Neurological Institute, Houston, Texas, United States, ⁷Mayo Clinic, Jacksonville, Florida, United States

E-mail address for correspondence: [email protected]

Keywords: non-invasive ventilation, diaphragm pacing, NIPPV

Background: Non-invasive ventilation (NIV) can improve survival in patients with ALS/MND. Positive pressure ventilation however may suppress diaphragm function leading to atrophy and conversion to less functional fast twitch Type IIb from slow twitch Type I diaphragm muscle fibers (1). This report analyzes the prospective diaphragm pacing pivotal trial results with a retrospective cohort study of NIV use in ALS (2).

Objective: Compare NIV use alone to NIV and DP conditioning in ALS/MND.

Methods: The entire Lechtzin et al's data set that was used for a publication was made available so that a more direct comparison to the prospective, controlled, multicenter, inter-ventional DP trial with lead-in and post -implantation treatment periods could be made. Analysis of inclusion criteria was then limited to FVC < 65% predicted and >45% predicted to correlate with the DP study inclusion criteria. The DP patients analyzed also all met the criteria for chronic hypoventilation of FVC < 50% or MIP <60 cm, H 2 O or PCO 2 > 45 mmHg. All patients in both groups needed to be using NIV.

Results: Forty four patients in the DP group met the analysis inclusion criteria of the 145 subjects enrolled with 106 implantations from 3/2005 to 3/2009. Forty three patients from the Lechtzin database met the criteria from a 298 patient database from 1998–2005. The DP/NIV group was younger than the NIV alone group (59±9 vs. 64±9, P < 0.05) and had a lower ALSFRSr score(28±8 vs. 32±7, P < 0.05) while the NIV alone group had a higher riluzole use (93% vs. 70%, P < 0.01) with more males (75% vs. 53%, P < 0.05). There was no statistical difference between DP/NIV and NIV alone group in the remainder of the comparison: bulbar onset (31% vs. 20%); G-tube use (61% vs. 53%); onset to diagnosis (17±19 vs. 15±11 months); diagnosis to first respiratory therapy (15±13 vs. 11±7 months); or FVC - % predicted (54±6 vs. 55±6). In the DP/NIV group patients initially started using DP for five 30 minute sessions a day and then increased based on patient presumed benefits. During diaphragm EMG analysis of implanted electrodes while the patient was on NIV, if NIV suppressed diaphragm activity the patients used DP whenever utilizing NIV. In the DP/NIV group the median survival from therapy was 20.7 months ±1.8 while in the NIV alone group it was 11.9±2.2 (P < 0.001). The median survival from diagnosis was 37.5±3.5 in DP/NIV group and only 21.4±5.8 in NIV alone group (P < 0.001).

Conclusion: Although limited by the retrospective analysis, the results suggest a significant augmentive effect of DP pacing to NIV leading to improved survival with no safety concerns with simultaneous usage. The most likely reason for this is prevention of diaphragm activity suppression with DP use.

References:

• Levine S, Nguyen T, Taylor N, et al. NEJM 2008;358:1327–35.
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• Lechtzin N, Scott Y, Busse A, et al. ALS 2007;8:185–8.
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P152 THE PREDICTIVE VALUE OF THE PHRENIC NERVE MOTOR RESPONSE FOR NON-INVASIVE VENTILATION

PINTO S¹, SCOTTO M², PINTO A^1,3, SWASH M^1,4, DE CARVALHO M^1,5

¹Neuromuscular Unit, Instituto de Medicina Molecular, Lisbon, Portugal, ²Department of Mathematics, University of Aveiro, Aveiro, Portugal, ³Department of Physical and Rehabilitation Medicine, Hospital de Santa Maria, Lisbon, Portugal, ⁴Department of Neurology, Royal London Hospital, Queen Mary School of Medicine, University of London, London, United Kingdom, ⁵Department of Neurosciences, Lisbon, Portugal

E-mail address for correspondence: [email protected]

Keywords: non-invasive ventilation, phrenic nerve motor response, hazard ratio predictive value

Introduction: Respiratory insufficiency is the main cause of death in Amyotrophic Lateral Sclerosis (ALS), but it is usually a late event. Non-invasive ventilation (NIV) improves survival and quality of life in ALS. We have evaluated the predictive value of several clinical features and respiratory tests indicating the need for NIV in our ALS population.

Methods: We evaluated 460 consecutive ALS patients followed in our Neuromuscular Unit over the last 7 years. The inclusion criteria were: follow-up in our unit; clinical progression; age at disease onset between 20 and 80 years; disease duration < 18 months at diagnosis; phrenic nerve motor response (mean value of bilateral peak-to-peak amplitude -Mean PhrenAmpl) and predicted forced vital capacity (FVC). FVC and Mean PhrenAmpl were tested ≤ 2 months after diagnosis. Patients with other medical conditions, in particular respiratory disorders, were excluded. Criteria to adapt patients to NIV were respiratory symptoms associated with low FVC or abnormal nocturnal oxymetry. We tested the hazard ratio for predictive value for NIV adaptation of the following features and measurements: presentation (bulbar vs spinal form); age at onset; disease duration at diagnosis; ALS-FRS; FVC; Mean PhrenAmpl. We applied Kaplan-Meier survival curves and Cox proportional-hazards regression analyses.

Results: We included 138 ALS patients, 59 women, 57 with bulbar-onset, age at disease onset 61.2±10.9 yrs (mean±SD) and disease duration of 10.94±4.5 months. ALS-FRS was 31.45±4.5 (from 14 to 39), FVC 84.5±23.6% and Mean PhrenAmpl 0.49±0.25 mV (from 0 to 1.28) at study entry. All patients were followed before and after NIV The following covariates were predictive of NIV: late onset (P = 0.027), low ALS-FRS (P = 0.027), low FVC (P = 0.006), and low Mean PhrenAmpl (P < 0.001). For each unit increment in ALS-FRS, FVC and Mean PhrenAmpl there was a relative risk decrease of 3.6% (CI:0.4–6.7%), 1.2% (CI:0.3–2%) and 86.3% (CI:65.5–94.6%), respectively. For each year increment in age at onset there was a relative risk increase of 1.02% (1.0–1.04%). Stratifying for the region of onset showed that Mean PhrenAmpl was significant for both groups (P < 0.001 and P = 0.071 for spinal and bulbar-onset patients, respectively), low ALS-FRS for spinal-onset patients (P = 0.022) and late age at onset (P = 0.021) for bulbar-onset subjects.

Discussion: Mean PhrenAmpl is a non-volitional respiratory test which is strongly predictive of the need for NIV in ALS patients, independently of the region of onset. These results indicate that motor unit loss in the diaphragm is a main factor determining respiratory symptoms in ALS. This test should be more extensively applied in this disease.

P153 A RANDOMIZED CROSSOVER TRIAL OF EARLY NONINVASIVE VENTILATION FOR ALS

LECHTZIN N, CLAWSON L, KIMBALL R, WIENER C

Johns Hopkins University School of Medicine, Baltimore, MD, United States

E-mail address for correspondence: [email protected]

Keywords: ventilation, clinical trial

Background: Respiratory complications are a frequent cause of death in ALS. Noninvasive ventilation (NIV) is the standard therapy for individuals with ALS and severe respiratory muscle impairment and has been shown to improve survival and quality of life. Several studies suggest that early use of NIV may slow the decline in lung function and improve survival.

Objectives: To determine if use of NIV in ALS can slow decline in forced vital capacity (FVC).

Methods: This was a randomized cross-over trial in which patients with a FVC > 60% of predicted were assigned to a three month period on NIV and a three month period off NIV The goal was to enroll 66 patients.

Results: Sixteen patients entered this study. The mean age was 56.8±11 years, 82% were male, 88% were Caucasian, and 29% had bulbar onset disease. The ALSFRS score at baseline was 41.1 ±5. The mean FVC at study entry was 3.57±0.96 L (91.1% predicted. The mean FEV1 was 2.85±0.77 L (85.7% predicted). The mean maximal inspiratory pressure was −70 cm H₂O and the mean arterial PCO₂ was 40.1 ±4 mmHg. While there were no significant effects of NIV on gas exchange or quality of life, there was a trend toward less change in FVC on NIV. During the period on NIV the FVC increased by 0.1 liters but decreased by 0.2 liters off of NIV (P = 0.07). The PaCO₂ increased by 1.4 mmHg on NIV and it decreased by 0.44 mmHg off NIV (P = 0.44). Quality of life score were similar during the period on and off of NIV. On NIV the physical component of the SF-12 decreased by 3.5 points and off NIV the score increased by 3.8 points (0.17). The mental component of the SF12 increased by 2.2 points while on NIV and it decreased by 3.4 points during the period off NIV (P = 0.37).

Discussion: This study was severely limited by poor enrollment. Additionally the duration on and off of NIV was only three months. The subjects studied had good normal lung function and gas exchange at the start of the study and did not decline appreciably during the 6 months of the study. While there was a suggestion that FVC improved during the period on NIV, this was not statistically significant and therefore this study is negative with respect to its primary outcome.

Conclusions: This study showed that people with ALS and normal lung function can use NIV but given the small sample size we are unable to draw any firm conclusion about potential benefits of NIV in this population. A larger study should be conducted to answer this important clinical question.

P154 NURSE ROLE IN USE OF NIPPV COMPLIANCE AND EFFICACY DATA

ARMSTRONG J, CASEY P, ALLEN J, HELLER S, AJROUD-DRISS S, SIDDIQUE T, SUFIT R, WOLFE L

Northwestern University, Chicago, IL, United States

E-mail address for correspondence: [email protected]

Keywords: non-invasive ventilation, nursing role for non-invasive ventilation, efficacy data for non-invasive ventilation

Background: The use of non-invasive positive air pressure ventilation (NIPPV) has benefits for people with ALS (1). Both quality of life, as well as quantity of life can be improved. However, there is not an ALS standard of care for management of NIPPV therapy, nor do we have a definition for the role of nursing regarding triage and therapy. NIPPV devices contain internal pressure monitors (pneumotachographs) that measure parameters of both compliance (numbers of hours used) and efficacy (minute ventilation, leak, respiratory rate, tidal volume, and apnea/hypopnea index). This data is available on current commercial home NIPPV devices. The role of the nurse coordinator, neurologist and/or pul-monologist monitoring this data has not been described in the literature.

Objectives: Review data collected from home NIPPV devices. Identify the tasks associated with review of this data as part of the function of the role of the nurse coordinator.

Methods: Compliance and efficacy data was obtained from a retrospective review of people with ALS using NIPPV. The routine for collecting the data is a minimum of three months, averages monthly. Changes to the device settings were made accordingly to physician interpretation and more frequently with reported symptoms. This information included how often data was obtained and how frequently that data influ-enced changes in therapy. Therapy was managed by the nurse coordinator under the direction of the Pulmonologist in the ALS Specialty Center.

Results: Review of the therapy decisions regarding NIPPV when made considering symptoms in question and recent triage history between patient and nurse. Case presentations will explain how simple problems can be managed (ie mask fitting causing high leak reading) to complex problems (ie trending high minute ventilation with increasing c/o shortness of breath led to investigating for pulmonary embolism).

Discussions and conclusions: Compliance and efficacy data interpretation can assist healthcare professionals in maximizing NIPPV therapy for people with ALS. Managing the shallow breathing index has proven helpful. Monitoring trends in the data can be useful and supports collection of serial data. Patients may benefit from monitoring data at a minimum of every three months, or as frequently as symptoms are reported.

Reference:

• Kleopa KA, Sherman M, Neal B. Journal of the Neurological Sciences. 1999;164:82–8.
   PubMed Web of Science ®Google Scholar

P155 PATTERNS OF ADHERENCE TO NON INVASIVE VENTILATION IN MOTOR NEURON DISEASE PATIENTS: A PROSPECTIVE STUDY

ANDO H¹, CHAKRABARTI B², ANGUS R¹, THORNTON E³, YOUNG C^1,3

¹Walton Centre for Neurology and Neurosurgery, Liverpool, United Kingdom, ²University Hospital Aintree, Liverpool, United Kingdom, ³University of Liverpool, Liverpool, United Kingdom

E-mail address for correspondence: [email protected]

Keywords: NIV, adherence, noncompliance

Background: Non-invasive ventilation (NIV) is increasingly being offered to Motor Neuron Disease (MND) patients. Studies report benefit in terms of survival with intervention and adherence to ventilation.

Objectives: In a longitudinal study patients were assessed both physiologically and psychologically prior to initiation of NIV and 3 monthly until death. Part of the aim was to map usage of NIV.

Methods: Thirty-five (24 male) MND patients were enrolled; 4 died before NIV was established and 3 remain under monitoring. Of 28 patients offered NIV, 11 declined and 17 were established on treatment. Ten patients (9 male, mean age 60 years) have 6 or more months follow up on ventilation. Six had bulbar symptoms at NIV commencement. All had evidence of nocturnal hypoventilation as assessed by continuous pulse oximetry - criterion used: greater than 30 minutes < 90% oxygen saturation. Adherence to NIV was assessed by analysis of the ‘memory card’ storage device in the ventilator at 4–6 months post initiation and at 10–12 months. Patients were recommended to use the treatment initially at night during sleep and for a minimum of 4 hours.

Results: At 4–6 months post initiation, the overall cohort used NIV for >4 hours nightly for a mean 65% (SD 32%) of nights with 3/10 patients using NIV >4 hours nightly for >90% nights. Bulbar patients were deemed less compliant than non-bulbar patients (54% nights v 81% nights>4 hour use). By 10–12 months, the remaining cohort (n = 7) used NIV for >4 hours nightly for a mean 78% (SD 36%) of nights with 4/7 patients using NIV >4 hours nightly for >90% nights; not significantly different compared with use at 4–6 months; P = 0.16. Bulbar patients were again deemed less compliant than non-bulbar patients (68% nights v 92% nights >4 hour use). Only 2 patients used the treatment every night.

Discussion: Use of NIV increases with disease progression, however our data shows that adherence to NIV is significantly less than recommended, despite patients being aware we were assessing use of NIV Electronic recording allows assessment of usage and for noncompliance to be identified, allowing the issue to be discussed with patients. There is a need to understand and address the issues determining noncompliance with NIV if adherence is to be addressed and patients are to realise the full benefit of this intervention.

Conclusion: Adherence to NIV is poor in these MND patients and at a level that is of clinical significance.

P156 THE LIVED EXPERIENCE OF PATIENTS WITH MOTOR NEURON DISEASE WHO DECLINED OR DID NOT TOLERATE NON-INVASIVE VENTILATION

ANDO H¹, WILLIAMS C⁴, ANGUS R², THORNTON E³, CHAKRABARTI B², PIGGIN L³, YOUNG C^1,3

¹Walton Centre for Neurology and Neurosurgery, Liverpool, United Kingdom, ²University Hospital Aintree, Liverpool, United Kingdom, ³University of Liverpool, Liverpool, United Kingdom, ⁴Hope University, Liverpool, United Kingdom

E-mail address for correspondence: [email protected]

Keywords: NIV, treatment acceptability

Background: Non-invasive ventilation (NIV) is known to be an effective intervention in motor neuron disease (MND) and is increasingly being offered to patients. The main benefits of NIV in MND patients are prolonged survival and resolution of symptoms of nocturnal respiratory insufficiency. However, it has been acknowledged that some patients do not tolerate a trial of NIV, while other patients decline a trial of NIV when offered.

Objectives: To follow a cohort of MND patients prospec-tively in order to determine what psychological issues are important in patients declining or failing to tolerate NIV, particularly those issues which contribute to treatment failure.

Methods: Thirty five (24 male) MND patients were enrolled, 4 died before NIV was established and 3 remain under monitoring. Of 28 patients offered NIV, 17 were established on NIV whereas 11 (8 male, average age 74.1yrs) have declined or failed to tolerate NIV: 7 intolerants, 3 early refusers without trial, and 1 late stage decliner. Of these 8 had limb onset and 3 bulbar; however 9 had bulbar symptoms at the time of trial-ling NIV. Semi-structured interviews were carried out with all the patients. The interviews were analysed using interpretative phenomenological analysis (IPA). One patient with only one interview was excluded from the analysis as the interview provided insufficient information to contribute. Triangulation by a respiratory physician and 2 psychologists was carried out on the interviews from 10 patients to examine emergent themes.

Results: Several key themes emerged: 1) personal perceptions of NIV consequence, 2) maintenance of self identity, 3) negotiation of the disease symptoms and 4) external influ-ences. The first theme addresses perceived negative outcomes of NIV, consequently generating reluctance towards NIV, yet the issue appeared to be closely related to the second theme: self identity. Self identity was observed to have become vulnerable with MND as a result of physical deterioration and the change in patients’ perception of self was further challenged by NIV. The third theme focuses on a discrepancy between the physician's recommendations and the patient's perceived need. The last theme addresses negative impact of experiences with healthcare services in the patient's decision-making. Similar themes were identified in those who declined and those who were intolerant of NIV after a trial of treatment.

Discussion: Thirty nine per cent of patients who were offered NIV either declined or did not tolerate the treatment. Subjective perception of MND and its impact on identity, and negative experience of healthcare services, are factors for decision-making regarding NIV use.

Conclusions: A psychological dynamic relating to NIV use was explored constructively using IPA. Understanding the implications of NIV for individuals is important if treatment is to be offered optimally and sensitively.

P157 ORAL SECRETION SCALE SCORE IN AMYOTROPHIC LATERAL SCLEROSIS PATIENTS IS ASSOCIATED WITH TOLERANCE OF NONINVASIVE POSITIVE PRESSURE VENTILATION, NEED FOR HOSPICE OR TRANSITION TO TRACHEAL POSITIVE PRESSURE VENTILATION AND SURVIVAL

CAZZOLLI P¹, BROOKS BR², LEWARSKI J³, MCKIM D⁴, CHATBURN R⁵

¹ALS Care Project, Canton, OH, United States, ²Carolinas Medical Center Department of Neurology, Charlotte, NC, United States, ³Invacare Corporation, Elyria, OH, United States, ⁴Ottawa Rehabilitation Centre, Ottawa, ON, Canada, ⁵Cleveland Clinic Foundation, Cleveland, OH, United States

E-mail address for correspondence: [email protected]

Keywords: noninvasive positive pressure ventilation, oral secretions, hospice care

Background: Noninvasive Positive Pressure Ventilation (NPPV) may significantly prolong survival in ALS patients but severe bulbar impairment limits its use. A clinimetric scale (4 = normal automatic saliva swallow; no drooling; 3 = automatic saliva swallow decreased; infrequent drooling; 2 = conscious saliva swallow required, drooling upright-leaning forward, lip-blotting 4–6 /hr; 1 = difficult conscious secretion swallowing, frequent drooling any position, lip-blotting 12–30 /hr, intermittent suctioning; 0 = constant drooling requiring constant lip blotting, regular suctioning) quantifying ability to clear oral secretions from the airway was developed based on a set of ALS patients followed in the home setting.

Objective: To develop and validate an Oral Secretion Scale (OSS) to predict tolerance of NPPV, the need for hospice (end-of-life) care, when to transition to Tracheal Positive Pressure Ventilation (TPPV), if desired, and survival of NPPV users with ALS.

Design and methods: In this observational study, 157 consecutive ALS patients were followed prospectively at the start of NPPV and for up to 40 months during subsequent home visits. Median survival was compared using Kruskal-Wallis One Way ANOVA; P values < 0.05 were considered significant.

Results: At the start of NPPV, 86/157 (55%) patients had no impairment (OSS score = 4). In 22/157 (14%) NPPV was attempted during acute respiratory failure but failed in 80% of those patients with OSS scores of 0 or 1. Median survival of 135/157 patients who continued on NPPV was significantly (P = 0.002) longer (27 months) in those with higher OSS score (= 4) and shorter (8 months) in those with lower OSS score (= 1). In 49/135 patients who stopped NPPV, median OSS score (=2) at the start of NPPV was significantly (P < 0.05) higher than the median OSS score (=1) when NPPV was no longer tolerated or when NPPV was stopped due to death, or transition to TPPV. Patients with an OSS score (= 4) remain on NPPV more than 3 times longer than patients with OSS score (=1).

Conclusions: OSS score was associated with tolerance of NPPV, when to initiate TPPV and survival. OSS score of 1 reliably signaled the intolerance of NPPV and/or the need for transition to tracheostomy ventilation or hospice care.

P158 ORAL SECRETIONS SCALE – TEST-RE-TEST RELIABILITY PILOT STUDY IN A JAPANESE AMYOTROPHIC LATERAL SCLEROSIS POPULATION

NAKAYAMA Y¹, OGURA A¹, MATSUDA C¹, SHIMIZU T¹, CAZZOLLI P², BROOKS BR³

¹Tokyo Metropolitan Institute for Neuroscience, Tokyo, Japan, ²The ALS/Neuromuscular Education Project, Ohio, United States, ³Carolinas Neuromuscular/ALS-MDA Center, Charlotte, United States

E-mail address for correspondence: [email protected]

Keywords: oral secretions scale, reliability, NIV

Background: The Oral Secretions Scale (OSS) permits identification of oral secretion burden that may limit usefulness of non-invasive positive pressure ventilation (NPPV).

Objective: Investigate the Test-Re-Test Reliability of OSS as a parameter to evaluate oral secretions in a Japanese ALS patient cohort.

Methods: In the OSS, saliva retention and swallowing are evaluated using 5 grades (normal, mild, moderate, severe, and most severe). ALS patients (5 males and 10 females) with a mean duration of disease 6.4±5.2 (standard deviation) years (range 1–16 years) participated. NPPV was employed in 5/15 patients. Dysphagia was present in 12/15 patients (9/12 had gastrostomy tube placement). An intermittent suctioning machine was applied in 6/15, and a low-pressure continuous suctioning machine in 4/15. In this study, we examined the consistency rate and inter-rater reliability (kappa coefficient) of the scores obtained by 4 nurses separately observing and rating 15 ALS patients. The study was 90% powered to detect a Kappa: 0.90 or above (almost perfect correlation).

Results: For the total 15 patient dataset Kappa was 0.66 with fixed-marginal kappa: 0.55 and free-marginal kappa: 0.57. In 4 patients scoring 4 and 2 patients scoring 0, the results of evaluation were consistent among the 4 nurses. In 1 patient scoring 4/3 and 6 patients scoring 3/2 and 2 patients scoring 2/1 or 1/0, there was only a 1 point difference among them. However, when classifying the subjects as those with a score of 2 or higher and those with 1 or lower, there was only one difference in the results of the evaluation. The subset kappa coefficient ranged from 0.64 to 0.73. The results of evaluation were less consistent when oral ingestion was possible in the presence of dysphagia, and when the grade of saliva retention/leakage depended on the posture and time.

Conclusions: OSS score reliability among 4 evaluators and 15 patients was 0.66 (substantial correlation). However, when classifying the subjects as those with a score of 2 or higher and those with 1 or lower, there was perfect correlation with only one difference in the results of evaluation among the 4 raters for the OSS score rating. These results for a 5-category scale indicate excellent inter-rater reliability of the OSS score rating among multiple independent raters.

Reference:

• Cazzolli PA, Brooks BR, Lewarski JS, et al. American Academy of Neurology Annual Meeting, Toronto, April 10–17, 2010; Abstract.
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P159 SUPINE VITAL CAPACITY REVISITED

RUDNICKI S¹, INGERSOLL E², ARCHIBALD D², CUDKOWICZ M³, GRIBKOFF V², BOZIK M², THE KNS-760704-CL201 STUDY GROUP⁴

¹University of Arkansas Medical Center, Little Rock, AR, United States, ²Knopp Neurosciences Inc., Pittsburgh, PA, United States, ³Massachusetts General Hospital, Boston, MA, United States, ⁴Multiple locations, United States

E-mail address for correspondence: [email protected]

Keywords: vital capacity, dyspnea, orthopnea

Background: Increases in the difference between percent predicted vital capacity (VC) in supine (sVC) compared to upright (uVC) position may be associated with dyspnea and orthopnea in ALS (1). As reference, the mean ΔVC (uVC minus sVC) in healthy controls is 7.5 + 5.7%; mean ΔVCs of >13.2% and >18.9%, therefore, are >1 and >2 standard deviations (SD) greater than the mean for healthy controls, respectively (2).

Objectives: To determine if epidemiologic factors are associated with a greater ΔVC, if ΔVC changes over time, and how ΔVC is related to dyspnea and orthopnea.

Methods: As part of a Phase 2 study of dexpramipexole (KNS-760704; [6R]-4,5,6,7-tetrahydro-N6-propyl-2,6-ben-zothiazole-diamine) in ALS (KNS-760704-CL201), uVC and sVC were performed at Baseline and Week 12; the ALSFRS-R was also administered. Dyspnea and orthopnea reports are based on ALSFRS-R data. Results are reported here across treatment groups.

Results: Of 102 subjects enrolled in the study, 99 had uVC and sVC data at Baseline, 94 had data at Week 12, and 91 had data at both visits (analysis population). Baseline uVC was 90.0 + 14.2% while the Baseline sVC was 83.5 + 17.4% (P < 0.001, paired t-test). Week 12 uVC was 79.8 + 19.7% and sVC was 75.9 + 21.6 (P = 0.009, paired t-test). ΔVC at Baseline was 7.0 + 8.6%, which decreased to 3.8 + 11.0% (P = 0.007, paired t-test) at Week 12. At Baseline, there was no relationship between the ΔVC and age, disease duration, site of disease onset, uVC, or BMI. Baseline ΔVC for 78 subjects with no dyspnea was 6.2 + 8.0% and for 13 subjects with dyspnea was 11.8 + 10.7% (P = 0.027). Twenty-nine subjects reported dyspnea at Week 12 and 62 subjects were dyspnea free; ΔVC for those with dyspnea was 4.9 + 11.6% and 3.4 + 10.8% for those without (P = 0.55, ns). At Baseline, only 3/99 subjects had orthopnea. At Week 12, 78 subjects denied orthopnea (ΔVC 3.5 + 11.3%) and 13 reported orthopnea (ΔVC 6.0 + 9.1%) (P = 0.45, ns). At Week 12 only 3 subjects had a ΔVC of >18.9%; 2/3 had dyspnea and 0/3 had orthopnea. A ΔVC of >13.2% was observed in 12 subjects at Week 12; dyspnea occurred in 6 of these 12 subjects and in 23/79 subjects with ΔVC <13.2% (P = 0.267, ns, chi square with continuity correction). Orthopnea occurred in 4/12 subjects with ΔVC >13.2% at Week 12, and in 9/79 subjects with a ΔVC <13.2% (P = 0.115, ns, chi square with continuity correction).

Conclusions: The relationship between ΔVC, dyspnea, and orthopnea may be less robust than previously thought. This may be in part related to the tendency of the ΔVC to lessen over time, and that some decline in VC when supine is normal.

References:

• Verrato J, et al. Neurology 2001;57(2):357–9.
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P160 THE BEHAVIOR OF SLEEP QUALITY IN PATIENTS WITH POST-POLIO SYNDROME ASSOCIATED WITH FATIGUE

SILVA T^1,2, OLIVEIRA A^1,2, QUADROS A^1,2, MOREIRA G¹, PRADELLA-HALLINAN M¹, TUFIK S¹

¹UNIFESP - EPM, São Paulo, Brazil, ²ABRELA, São Paulo, Brazil

E-mail address for correspondence: [email protected]

Keywords: post-polio syndrome, sleep, fatigue

Background: Post-polio syndrome represents new neuro-muscular symptoms in patients with previous poliomyelitis. These symptoms include new weakness, fatigue, joint and muscle pain, sleep disturbances and intolerance to cold.

Objective: To analyze the behavior of sleep quality and sleep-related disordered breathing (SRDB) in patients with a history of poliomyelitis and with current post-polio syndrome (PPS) associated with fatigue.

Material and method: We prospectively studied 60 consecutive patients with PPS. All 60 patients were accompanied by our team for a 1-year period at Federal University of Sao Paulo. Our team abstracted the features of acute polio and PPS. For sleep evaluation we applied the polysomnography exam, Epworth sleepiness scale (ESS), Stanford sleepiness scale (SSS), mini-sleep questionnaire (MSQ), as well as fatigue severity scale (FSS), analogical visual scale (AVS) for pain, and Barthel scale for daily living activities.

Results: The features of PPS were fatigue, new weakness, hyper somnolence, and musculoskeletal pain. Fatigue evaluation revealed two groups: with fatigue (group F, N = 48) and without fatigue (WF, N = 12). Twenty percent of our 60 patients were using continuous positive airway pressure (CPAP) for treatment of obstructive sleep apnea (OSA). The mean of OSA was 20.2 and 15.3 per hour in groups F and WF, respectively. The mean apnea/hypopnea index (AH) was 7.1 and 5.2 per hour, periodic limb movements (PLM) index was 5.0 and 6.6 per hour in patients in these respective study groups. The overall mean age at onset of symptoms of PPS was 47 years, and the mean latent period after acute polio was 46 years. Hyper-somnolence was the commonest SRDB symptom, present in 33 of the 60 patients. Pain was referred for 33% of patients in group WF, and 75% in group F. Snoring was noted in 81% of patients in group F, and 25% in group WF. The ability for activities of daily living was good in 50 of the 60 patients.

Conclusion: PPS patients with and without fatigue do not differ in quality of sleep. Therefore, bad quality of sleep seems to be a new symptom, independent of others, for patients with PPS.