P1 LONG TERM INVASIVE VENTILATION THROUGH TRACHEOSTOMY IN ADVANCED ALS: CAROLINAS NEUROMUSCULAR/ALS-MDA CENTER EXPERIENCE
BRAVVER E1,2, DESAI U1,2, LINDBLOM S3,2, WILLIAMS N1, LANGFORD V1, BOCKENEK W4,2, BROOKS BR1,2
1Carolinas Medical Center, Department of Neurology, Charlotte, North Carolina, USA, 2University of North Carolina School of Medicine – Charlotte Campus, Charlotte, North Carolina, USA, 3Carolinas Medical Center, Department of Internal Medicine, Charlotte, North Carolina, USA, 4Carolinas Medical Center, Department of Physical Medicine and Rehabilitation, Charlotte, North Carolina, USA
Email address for correspondence: [email protected]
Keywords: invasive ventilation, tracheostomy, survival
Background: Amyotrophic lateral sclerosis (ALS) is a life-limiting disease with 50% patients dying within 3 years and 90% within 5 years after diagnosis. Although invasive ventilation through tracheostomy (IVTT) may significantly extend the survival in ALS patients with respiratory insufficiency, it is not commonly recommended due to perceived poor quality of life, high cost of care, and clinicians’ personal biases.
Objectives: Identify life expectancy, quality of life, and specific challenges of ALS IVTT patient management imposed on caregivers, physicians, and multidisciplinary clinic staff.
Methods: We audited medical records of IVTT patients followed at Carolinas Neuromuscular/ALS-MDA in the Department of Neurology at the Carolinas Medical Center during the past 3 years. We reviewed several patients’ factors: gender, race, age at onset, clinical manifestation site, time between symptom onset and tracheostomy, time after tracheostomy, and total ALS FRS-R score and respiratory ALS FRS-R subscore.
Results: 42 IVTT patients evaluated at CMC ALS Clinic since beginning of 2007 were identified: 26/42(60%) males, 16/42(40%) females; 7/42(17.5%) African Americans, 2/42(5%) Hispanics, and 33/40(77.5%) Caucasians. 12/42(29%) patients had bulbar, 16/42(38%) upper extremity, 11/42(26%) lower extremity, 2/42(5%) respiratory, and 1/42(2%) PLS onset. The age of symptom onset was 26-68(mean 53.6) years; the age at tracheostomy procedure ranged 26-72(mean 56.6) years. The time between symptom onset and tracheostomy procedure was 0.3-15 (mean 3.4) years. Time after tracheostomy in 19 living patients was 0.7-13(mean 3.9) years; time between tracheostomy and death in 22 deceased patients was 0.4-6.4(mean 2.44) years. 40/42(95%) patients had PEG tubes. 24/42(57%) had ALS FRS-R 0-3; 10/42(24%) had ALS FRS-R 4-10; 1/42(2%) had ALS FRS-R of 36; no ALS FRS-R was available for 7/42(17%). 4/42(10%) patients developed true locked in state. Only one IVTT patient requested hospice assisted withdrawal of life support.
Discussion: IVTT prolongs survival in ALS patients with respiratory failure but it brings a great financial and care burden. There have been only a few studies that have investigated the outcome of ALS patients receiving treatment with IVTT, and there is little knowledge about the clinical characteristics of the ALS patients with long term IVTT. 42/300(14%) of all ALS patients followed at CMC ALS Center during 2007-2011 were on IVTT. More than half patients had ALS FRS-R 0-3. Our experience stresses the importance of careful planning of medical interventions, and providing counseling for patients and families in making decisions about initiation as well as withdrawal of ventilatory support.
Conclusions: IVTT is an effective measure to prolong survival in ALS. More studies are needed to evaluate the quality of life in long term IVTT ALS patients. Timely initiation of discussions about end of life care to facilitate decision-making process with patients and their caregivers is an important part of multidisciplinary care.
P2 THREE-MONTH SURVIVAL AFTER AN ICU ADMISSION FOR ACUTE RESPIRATORY FAILURE IN AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND PROGNOSTIC FACTORS
MAYAUX J1, DUGUET A1,2, GONZALEZ-BERMEJO J1,2, MORELOT-PANZINI C1,2, DORSETT J1, SALACHAS F3, SIMILOWSKI T1,2
1Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Respiratory and Intensive Care Medicine, Paris, France, 2Université Paris 6 Pierre et Marie Curie, ER10upmc, Paris, France, 3Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Neurology and ALS Center, Paris, France
Email address for correspondence: [email protected]
Keywords: respiratory insufficiency, intensive care, acute
Background: The main complication of ALS is chronic respiratory failure due to muscle weakness. Non-invasive ventilation (NIV) improves survival whilst maintaining quality of life. However, during an episode of acute respiratory distress, the decision of whether or not to admit the patient to the ICU can be a difficult one to make.
Objective: To describe the survival of patients with ALS 3 months after an ICU stay and identify prognostic risk factors.
Methods: A descriptive, observational, retrospective monocentric study in the ICU of the Department of Respiratory and Intensive Care Medicine of a 1600-bed teaching hospital, participating to the ALS reference center in Paris, France. All the first ICU stays of ALS patients were analyzed, except when they were pre-programmed (e.g. for tracheostomy or at risk gastrostomy). The variables analysed fell within three categories: 1) disease description and neurologic status within the 3 months preceding the admission; 2) presence or not of tracheotomy or of non-invasive ventilatory support (NIV) before the ICU stay; 3) ICU stay description. The analysis was first conducted in the whole population, and then restricted to the NIV group.
Results: Between 1st January 2000 and 30th June 2009, 111 admissions of ALS patients were recorded. Of these, 90 corresponded to the inclusion criteria, in 66 men and 24 women. The overall 3 months survival was 50%. It was not associated with any neurological descriptor, including the existence of a bulbar involvement. A prior history of domiciliary ventilation did not affect 3-month mortality either. Age and the severity of respiratory acidosis at admission were independent predictors of the 3-months mortality according to the multivariate analysis of the data. A tracheotomy either before or during the ICU stay was associated with better survival. These results were identical in the “prior NIV” subgroup. One third of the patients were alive after one year.
Conclusion: In this retrospective cohort, age and respiratory acidosis were the only factors that predicted the risk of death at 3 months. The lack of predictive value of the bulbar involvement came as a surprise but could be the result of a strong referral bias. The median survival following the admission of an ALS patient to the ICU is short (median of 4 months), but one third of the patients are still alive at one year. This indicates that systematically censoring ICU admission in ALS patients, even if they suffer from established respiratory insufficiency, is unwarranted.
P3 ANALYSIS OF THE END OF LIFE SITUATION IN 115 CONSECUTIVE ALS PATIENTS IN THE PAST NINE YEARS
OGINO M, KITAMURA E, MIYAKAWA S, UTINO A, URANO Y, NAGAI M, OGINO Y
Kitasato University Department of Neurology, Sagamihara, Kanagawa, Japan
Email address for correspondence: [email protected]
Keywords: NPPV, end of life, morphine
Background: Recently ALS treatment has been changing in Japan due to the wide use of NPPV and the use of morphine.
Objectives: To clarify the change of the end of life situation by the spread of NPPV and the use of morphine.
Methods: In 115 cases who died by ALS from April, 2002, we examined the use of NPPV, use of morphine and situation of the end of life.
We compared two periods : from April 2002 to March 2005 (1st period); and from April 2005 to March 2011 (2nd period).
Results: Among eight patients using permanent mechanical ventilation, six of them died of infection and 2 of them died by accident. In the remaining 107 cases, there were 30 cases in the 1st period and 77 cases in the 2nd period.
NPPV was used in 5 cases (17%) in the 1st period and 44 cases (57%) in the 2nd period, morphine was used in 1 case (3%) and 37 cases (48%), and sudden death occurred in 9 cases (30%) and 10 cases (13%), respectively. No case of sudden death used NPPV and morphine. Almost all of the patients who used NPPV needed to use morphine. Almost all of the patients who used morphine died peacefully, except for one patient who needed terminal sedation.
Discussion: It was reported that approximately 50% of ALS patients died suddenly before NPPV use spread. In this study sudden death was obviously much higher in 1st period when NPPV was not popular as compared to 2nd period. We assume that the use of NPPV avoids sudden death even in the patients who had bulbar symptoms. NPPV prolongs one's life, then patients come to feel dyspnea for a longer period than without NPPV. We should not hesitate to use morphine especially for cases with NPPV. Further investigation is needed to clarify whether NPPV is useful to prevent sudden death even for the patients who have severe bulbar symptoms and mild respiratory dysfunction which does not satisfy the criteria for indication of NPPV.
Conclusion: NPPV improves not only QOL and the life expectancy but also prevents sudden death, therefore the patient can spend the end period with their family. Since neurologists see ALS patient until the end stage in Japan, to make that period meaningful, we need to become proficient in using NPPV and morphine.
P4 AN EXPLORATORY MIXED METHODS STUDY OF PATIENTS WITH MOTOR NEURONE DISEASE WHO ARE ON NON-INVASIVE VENTILATION
ANDO H1, CHAKRABARTI B2, ANGUS R2, THORNTON E3, YOUNG C4
1Hope University, Liverpool, UK, 2University Hospital Aintree, Liverpool, UK, 3University of Liverpool, Liverpool, UK, 4Walton Centre for Neurology and Neurosurgery, Liverpool, UK
Email address for correspondence: [email protected]
Keywords: NIV, longitudinal
Background: Non-invasive ventilation (NIV) has been recommended as a treatment option for some patients with motor neurone disease (MND) because of its positive impact on survival. However, previous studies do not provide insight into how patients’ perceptions of NIV may evolve over time, and how this influences NIV use.
Objectives: To investigate longitudinally how NIV is experienced by MND patients while their condition deteriorates.
Methods: Five MND patients (male = 4, mean age = 59 years) were serially interviewed every three months. Interviews were transcribed verbatim and analysed using interpretative phenomenological analysis (IPA). These patients were part of a wider study, the data presented focuses on all participants for whom we have at least four post NIV interviews, covering more than 12 months of NIV treatment (mean = 13 months), along with ventilator interaction data.
Results: The IPA identified three main themes: physical-psychological relationship, coping style effects and the impact of the terminal prognosis. The first theme explains how physical benefits of NIV were related to psychological benefits, while no relationship between negative physical aspects of NIV and psychological effects was observed. The second theme focuses on participants’ coping styles and their experience of NIV; positive experience of NIV was reported among patients with active coping styles, who also expressed their positive attitude towards life, while an avoidant and resistant attitude towards NIV was found with a patient who displayed the same attitude towards the illness. The final concept describes how the sense of death or the sense of hopelessness both provoked by the perception of MND, prompted a reinforced or a reluctant engagement with NIV, respectively. The ventilator interaction data identified regular use of NIV by four participants (mean NIV use time = 9 hrs 20 mins), who had positive coping styles, and irregular use by one participant (mean NIV use time = 2 hrs 30 mins), who had a negative coping style and in whom MND induced feelings of hopelessness.
Discussion: Generally, patients displayed psychological benefits from NIV and these appeared to increase over time. However, individuals may be resistant to NIV because they perceive a hopeless future and due to their coping styles. The study suggests that the individual's experience of NIV is determined by their interpretation of the illness and its perceived impact on the future, which is in turn influenced by their coping styles and their attitude towards life. The qualitative interpretations were in agreement with the pattern of participants’ NIV use.
Grant from Motor Neurone Disease Association UK.
P5 HOW DOES THE “REAL-LIFE” PRACTICE OF NON-INVASIVE VENTILATION IN AMYOTROPHIC LATERAL SCLEROSIS AT A LARGE REFERENCE CENTER COMPARE WITH CONSENSUS EXPERT RECOMMENDATIONS?
KARTAS S1, MORELOT-PANZINI C1,2, GOLMARD J-L4, DORSETT J1, MEININGER V3, SIMILOWSKI T1,2, GONZALEZ-BERMEJO J1,2
1Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Respiratory and Intensive Care Medicine, Paris, France, 2Université Paris 6 Pierre et Marie Curie, ER10upmc, Paris, France, 3Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Neurology and ALS center, Paris, Paris, France, 4Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Biostatistics, Paris, France
Email address for correspondence: [email protected]
Keywords: registry, intermittent positive-pressure ventilation, respiratory insufficiency
Background: Chronic respiratory failure due to respiratory muscle weakness is frequent in amyotrophic lateral sclerosis (ALS) and a leading cause of death. Non-invasive ventilation (NIV) is a recognized standard of care that alleviates symptoms and prolongs survival. Criteria for starting NIV in ALS derive from expert recommendations, but the evidence behind them is weak, and how realistic they are in practice is unknown.
Objective: To describe the practice of the main French tertiary referral centre for ALS with regard to NIV initiation in reference with the corresponding French expert consensus guidelines.
Methods: The French national register of ALS tertiary referral centres (17 centres in France) contains data on 5410 patients followed up in the Paris centre since 2004. Among these, 594 patients have been started on NIV. We extracted the main criterion identified by the clinicians as the reason to initiate NIV from the database (among symptoms, PaCO2, VC, Pimax, SNIP and the percentage of night time spent with a SpO2 < 90%) and described the patients accordingly.
Results: Symptoms were the main reason for NIV initiation (39%; only reason in 6 cases), followed by hypercapnea (28%). Functional respiratory impairment rarely came first (Pimax or SNIP in 3%; VC in 2%; nocturnal desaturation 3%). 10% were ventilated due to acute respiratory insufficiency. Sixty-five patients (11%) were ventilated without demonstrating any of the consensus criteria for starting NIV. The main reason for 4% of the patients was a high blood level of HCO3−.
At the time of NIV initiation, ninety percent of the patients reported symptoms (effort dyspnoea, dyspnoea at rest, orthopnoea, nocturnal arousals, daytime somnolence or morning headaches). Paradoxical respiration was observed in 47% of patients and use of accessory muscles in 70%. Average VC was 52 + 22%, average Pimax was 56 + 33%, average SNIP was 41 + 28, average nocturnal desaturation time was 32 + 33 % of the time spent <90%.
Conclusion: At the time of starting NIV, our patients were very symptomatic and often hypercapnic, and had functional characteristics suggesting that NIV would have been started earlier if guidelines had been applied rigorously. The main hypothesis to explain these observations is an insufficient resource allocation to the respiratory management of ALS, in spite of the fact that our center has 3 full time respiratory physicians devoted to this activity and the highest national proportion of ALS patients receiving NIV.
In collaboration with the ALS/NMD group of Société de Pneumologie en Langue Française
P6 EARLY TREATMENT WITH NONINVASIVE POSITIVE PRESSURE VENTILATION PROLONGS SURVIVAL IN AMYOTROPHIC LATERAL SCLEROSIS PATIENTS
LI W, LI Z, WU Q, YE C, LU X, ZHEN Z, YAO X
Department of Neurology, the First Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China
Email address for correspondence: [email protected]
Keywords: noninvasive positive pressure ventilation, pulmonary ventilation function, forced vital capacity
Objective: To investigate 1 year survival of ALS patients with FVC < 75% treated with NPPV, compared to a well-matched population of ALS patients, who refused or were intolerant to NPPV.
Methods: We investigated 60 consecutive ALS patients who underwent pulmonary function tests. Twenty-eight patients presented a FVC > 75% and served as control group, 33 patients presented a FVC < 75% and showed nocturnal respiratory insufficiency, requiring NPPV; 14 patients who were treated with NPPV constituted group 1, while 12 patients who refused or were intolerant constituted group 2.
Results: Increased survival rate at 1 year in patients with FVC < 75% treated with NPPV, as compared to those who refused or could not tolerate NPPV (P < 0.05), but showed no difference with the control(P > 0.05). The median rate of decline in FVC% was slower in NPPV patients than in patients who did not use NPPV.
Conclusions: Early treatment with NPPV delays the development of the patient's condition and prolongs survival and reduces decline of FVC% in ALS.
P7 HOME BASED UNATTENDED SLEEP STUDY AS AN INTEGRAL COMPONENT OF MULTIDISCIPLINARY ALS CLINIC
DESAI U1,2, LANGFORD V1,2, BRAVVER E1, BROOKS BR1
1Carolinas Neuromuscular/ALS-MDA Center, Sleep Disorders Clinic, CMC, Charlotte, NC, USA, 2Sleep Disorders Clinic, CMC, Charlotte NC, USA, 3Neurology and Sleep Center, CMC, Matthews, NC, USA
Email address for correspondence: [email protected]
Keywords: sleep disordered breathing, respiratory insufficiency, NIV
Background: The most frequent terminal event in Amyotrophic Lateral Sclerosis (ALS) is respiratory insufficiency (RI) (1). RI becomes evident earlier during sleep rather than daytime (2) with resultant fatigue, excessive daytime sleepiness and poor quality of life (3). Early diagnosis of RI is important because noninvasive ventilation is a standard of care in ALS with increased survival related to number of ventilator use hours (1).
Objectives: To capture SDB (sleep disordered breathing) causing RI and identify patients needing NIV at their earliest possible physician-patient interface by a home based unattended sleep study, as an integral component of a multidisciplinary ALS clinic.
Methods: Peripheral arterial tonometry (PAT) is a noninvasive technique to measure changes in blood flow to the finger, an area nearly exclusively regulated by adrenergic innervation. PAT signal changes in response to airway obstruction and arousals. Algorithms of periodicity, duration, heart rate and desaturation (4,5) indirectly estimate AHI (Apnea Hypopnea Index) by identifying surges of sympathetic activation (autonomic arousals) occurring at termination of respiratory events. Advantages include home based setting, simplicity of equipment, reduced cost and it incorporates Actigraphy calculating total sleep time providing ‘events per hour in sleep’ rather ‘events per hour in bed’ (6). We used Watch PAT100 (Itamar Medical) which had showed good correlation between AHI measured with simultaneous PAT100 and standard laboratory PSG setting (rho = 0.88).
Results: 15 patients were studied over 3 months. Sitting and supine FVC (Forced Vital Capacity), NIF (Negative Inspiratory Force), sleep efficiency, AHI (Apnea Hypopnea Index), RDI (Respiratory Disturbance Index) and ODI (Oxygen Desaturation Index) were reviewed. 9 were males (60%), 6 females (40%). 3/15 (20%) showed sitting FVC < 50% qualifying for NIV. 12/15 (80%) showed sitting FVC > 50%. One showed supine FVC < 50%. Out of 11/15 (73.3%) who could not qualify for NIV based on FVC > 50% (sitting or supine) or NIF (< -60); 7/11 (63.6%) showed elevated AHI (> 5), 2/11 (18.1%) showed elevated RDI, 9/11 (81.8%) showed elevated AHI and/or RDI (> 5) and qualified for NIV. Out of 11/15 (73.3%) who could not qualify for NIV based on FVC > 50% (sitting and supine); 8/11 (72.7%) showed nocturnal O2 desaturation nadir < 90%, while all these 8 patients showed diurnal resting O2 > 90%.
Discussion and conclusions: ALS patients have difficulty tolerating multichannel sleep laboratory studies because of weakness, reduced mobility, secretions, dysarthria hampering communication with technologist in addition to “first night effect”. An alternative approach is home based unattended sleep study by peripheral arterial tonometry; an easy and cost effective method (8) to identify patients early for NIV as standard diurnal respiratory measures like FVC and NIF can undermine degree of RI. We propose that unattended home based sleep studies should be an integral part of a multidisciplinary ALS clinic.
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P8 THE SURVIVAL OF AMYOTROPHIC LATERAL SCLEROSIS PATIENTS PLACED UNDER NON INVASIVE VENTILATION (NIV) DOES NOT DEPEND ON THE VENTILATORY MODE USED (VOLUME VS. PRESSURE NIV)
GONZALEZ-BERMEJO J1,2, MORELOT-PANZINI C1,2, DORSETT J1, SERVERA E4, SALACHAS F3, SIMILOWSKI T1,2, SANCHO J4
1Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Respiratory and Intensive Care Medicine, Paris, France, 2Université Paris 6 Pierre et Marie Curie, ER10upmc, Paris, France, 3Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of neurology and ALS center, Paris, France, 4Department of Respiratory Medicine, Hospital Clinico Universitario, Universitat de Valencia, Valencia, Spain
Email address for correspondence: [email protected]
Keywords: intermittent positive ventilation, volumetric ventilation, barometric ventilation
Background: Non-invasive ventilation (NIV) has become a universally accepted standard of care in amyotrophic lateral sclerosis (ALS) when the disease-related respiratory muscle weakness is responsible for chronic respiratory failure. NIV alleviates symptoms and prolongs survival. Whether or not these benefits depend on the ventilatory mode used to deliver NIV is not precisely known.
Objective: To compare survival in two separate cohorts of ALS patients treated at two distinct European tertiary referral centres, with pressure preset NIV in one case and volume preset NIV in the other case (p-NIV and v-NIV, respectively).
Methods: Retrospective comparison of 62 patients receiving v-NIV (Valencia, Spain) with 82 patients receiving p-NIV (Paris, France), in terms of their anthropometric characteristics, neurological data, respiratory variables, and 5 years survival. Of note, the two centers follow the same clinical management guidelines, the only important difference being their choice of ventilatory mode.
Results:: There was no statistically significant difference between the two groups at the time of NIV initiation, regarding age (62 ± 9 vs 64 ± 11 years in the p-NIV vs v-NIV group, respectively), vital capacity (52 ± 24 vs 55 ± 19 % pred), Pimax (47 ± 25 vs 42 ± 25 cmH20), PaCO2 (51 ± 9 vs 53 ± 9 mmHg) and total night time spent with a SpO2 < 90% (TST90%, 35 ± 30 vs 38 ± 33 %). The Norris bulbar score was lower in the p-NIV group (27 ± 10 vs 24 ± 5, p < 0.05). The median survival from NIV onset was 15 ± 4 months in the v-NIV cohort, vs. 17 ± 4 months in the p-NIV cohort (p = 0.4). Restricting the analysis to patients with a bulbar onset of the disease (n = 23 in the v-NIV cohort, n = 9 in the p-NIV cohort, p < 0.05), who are often said to be more difficult to ventilate, provided similar results (8 ± 2 months in the v-NIV cohort, vs. 9 ± 1 months in the p-NIV cohort, p = 0.27). A non-significant trend toward a better survival was observed in the bulbar onset patients receiving v-NIV after the first year of NIV. A multivariate analysis conducted on the population as a whole (n = 144) identified usual prognostic factors for survival, namely an older age (HR = 1.03, p = 0.01), vital capacity (HR = 0.5, p = 0.01) and a bulbar onset of the disease (HR = 0.4, p = 0. 008). In addition, this analysis also identified PaCO2 under NIV as an independent prognostic factor (HR = 1.1, p = 0.009), irrespective of the ventilatory mode used.
Conclusion: p-NIV and v-NIV provide similar survival in ALS patients with chronic respiratory insufficiency. Failure to lower PaCO2 with NIV is an independent prognostic factor. This suggests that an adequate NIV is more important than the ventilatory mode used to deliver it.
P9 IMPACT OF THE QUALITY OF NON-INVASIVE VENTILATION ON THE ONE-YEAR MORTALITY OF PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS
GONZALEZ-BERMEJO J1,2, MORELOT-PANZINI C1,2, ARNOL N4, DORSETT J1, MEININGER V3, KRAOUA S1, SALACHAS F3, SIMILOWSKI T1,2
1Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Respiratory and Intensive Care Medicine, Paris, France, 2Université Paris 6 Pierre et Marie Curie, ER10upmc, Paris, France, 3Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Neurology and ALS Center, Paris, France, 4Biostatistics, AGIRaDOM, Grenoble, France
Email address for correspondence: [email protected]
Keywords: intermittent positive pressure, home ventilation, nocturnal monitoring
Background: Non-invasive ventilation (NIV) is the standard of care of ALS-related chronic respiratory insufficiency due to respiratory muscle weakness. It improves quality of life and improves survival. There are data to suggest that this benefit is lost in when NIV is ill-tolerated by patients.
Objectives: To evaluate the impact of the quality of NIV on the one-year survival of ALS patients.
Methods: We analysed survival rates during the first year following the introduction of NIV in 82 patients with ALS. The quality of NIV was evaluated at one month. Patients who had a SpO2 < 90% for more than 5% of the time were considered inadequately ventilated and constituted Group 1. They were compared with a Group 2 made of the patients not exhibiting nocturnal desaturations, in terms of 1) symptoms; 2) sleep nocturnal polygraphic recordings (Reslink®, Resmed, Sydney, Australia); 3) arterial blood gases.
Results: Group 1 comprised 42 patients (51%). Group 1 and Group 2 were generally similar, without significant differences in terms of variables known to be associated with poor prognosis (older age, bulbar onset, vital capacity and its decline rhythm, rapid functional decline, ALSFRS). Inadequate ventilation was due to air leaks (56%), upper airway obstruction (24%) and other causes (20%). The one-year survival was significantly better in Group 2 than in Group 1 (12 vs 10.5 months, p = 0.002). In a multivariate analysis, an inadequate NIV at one month appeared as an independent predictor of mortality (HR = 2.32, p = 0.029), in addition to the body mass index (HR = 0.09, p = 0.001), vital capacity (HR = 0.97, p = 0.010), bulbar onset (HR = 4.31, p = 0.002) and a rapid disease progression (HR = 3.55, p = 0.014).
Conclusions: Poor quality of ventilation at one month is an independent risk factor for mortality at one year in ALS patients.
P10 DEPRESSED VENTILATORY DRIVE WITH GLOTTIS CLOSURE: A NEWLY RECOGNISED CAUSE OF NIV INTOLERANCE IN AMYOTROPHIC LATERAL SCLEROSIS
ATTALI V1,2, MORELOT-PANZINI C1,2, COLLET J-M4, KARTAS S1, SALACHAS F4, SIMILOWSKI T1,2, GONZALEZ-BERMEJO J1,2
1Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Respiratory and Intensive Care Medicine, Paris, France, 2Université Paris 6 Pierre et Marie Curie, ER10upmc, Paris, France, 3Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Neurology and ALS Center, Paris, France, 4Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Stomatology, Paris, France
Email address for correspondence: [email protected]
Keywords: obstructive apnea, intermittent positive-pressure ventilation, respiratory insufficiency
Background: Non invasive ventilation (NIV) can be rendered ineffective by various mechanisms: 1) air leaks; 2) airway obstruction; 3) reduced ventilatory drive; 4) patient-ventilator asynchrony. Depressed ventilatory drive with glottis closure can occur during sleep in response to hyperventilation. It has been described in normal subjects, but never in ALS patients and never as a cause of NIV inefficiency.
Objectives: To describe and elucidate the occurrence of depressed ventilatory drive with glottis closure in ALS patients unable to tolerate NIV and presenting with signs of airway obstruction.
Methods: All the ALS patients treated with NIV in the Paris ALS reference centre (France) are followed up with nocturnal monitoring every 3 months (automatic polygraphy, Reslink™ Resmed, Sydney Australia). A full in-lab sleep polygraphy is performed under NIV when episodes of airway obstruction are retained as the main cause of a NIV inefficiency. We therefore reviewed the 2010 database of our sleep center to identify episodes of depressed ventilatory drive with glottis closure. They were defined as an event (desaturation or/ad arousal) due to an interrupted or reduced inspiratory flow associated with a decrease in ventilatory drive (Gonzalez-Bermejo, Thorax 2011).
Results: Of 1528 nocturnal monitorings performed in 2010, 213 were carried out on ALS patients. Fifteen of these demonstrated depressed ventilatory drive with glottis closure. The absence of hypocapnea ruled out a hyperventilation secondary to the NIV. The impact of this phenomenon was significant with nocturnal desaturations in 9 patients (12 % (1-18) of the recording time) and arousals in 6 patients. We observed that the phenomenon occurred during waking hours in two patients, thereby allowing bronchoscopic visualisation. The lower jaw was seen to fall, the tongue slid backwards and the epiglottis fell onto the glottis which, in all, created a total closure of the upper airway. This obstruction was accompanied by a complete absence of respiratory movements. Changing NIV settings failed to solve the problem. Mandibular advancement with NIV provided short-term improvements.
Conclusion: Depressed ventilatory drive with glottis closure should be suspected in ALS patients when airway obstruction occurs under NIV. How mandibular advancement can correct this problem is currently being investigated.
P11 LONG-TERM EVOLUTION OF AMYOTROPHIC LATERAL SCLEROSIS PATIENTS UNDERGOING CHRONIC DIAPHRAGM CONDITIONING: A 2 YEAR FOLLOW-UP OF THE FRENCH ALS-PHRENIC PACING COHORT
MORELOT-PANZINI C1,2, TANGUY M-L3, DORSETT J1, SALACHAS F4, BRUNETEAU G4, PRADAT P-F4, MEININGER V4, SIMILOWSKI T1,2, GONZALEZ-BERMEJO J1,2
1Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Respiratory and Intensive Care Medicine, Paris, France, 2Université Paris 6 Pierre et Marie Curie, ER10upmc, Paris, France, 3Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Biostatistics, Paris, France, 4Assistance Publique – Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, Department of Biostatistics, Paris, France
Email address for correspondence: [email protected]
Keywords: intermittent positive ventilation, phrenic nerve pacing, diaphragm
Background: The effects of diaphragm conditioning through phrenic nerve stimulation with a laparoscopically implanted intramuscular stimulator (NeurRxDP4, Synapse Biomedical, Oberlin, OH, USA) on the decline of vital capacity (VC) in ALS have been tested during a prospective, non-controlled, non-randomized, multicenter pivotal trial. Preliminary information suggest that diaphragm conditioning can slow down VC decline in certain cases and can improve sleep. Final results are pending. This study involved a 9 months follow-up, beyond which the patients and their caregivers were free to continue using phrenic stimulation. Among the patients enrolled at the French center of this study (n = 18, the last one has been implanted in March 2009), 17 patients continued to pace their diaphragm after completion of the study follow-up (for more than 6h/day in 16 cases, less than 1 hour in one) and one stopped.
Objective: To assess the status of ALS patiens still using their phrenic stimulator 2 years after the completion of the initial study.
Methods: Comparison of the survival observed in the implanted patients (n = 18, and 11 with an onset of disease after 2006) with survival data gathered prospectively at our center since 2006 (onset of disease after 2006, n = 756). The effect of stimulation on the survival of patients was assessed using a Cox regression model with stimulation as time- dependent variable.
Results: At the time of implantation, the implanted patients were 66 + 7.71, 55 % were men, 9% had had a bulbar onset. Their ALSRS-r total score was 31 + 6, with riluzole use in 100% of cases, and non-invasive ventilation use in 50%. As of April 2011, 3 patients (all implanted during the last trimester of 2008) were still alive, 2 with NIV at a stable dose of 8 hours/night, 1 without NIV. The hazard ratio for death at the 2 years time point was 5.46 for the non-implanted patients vs. the implanted ones (95% CI 0.732; 40.788, p = 0.09).
Conclusion: Diaphragm conditioning with phrenic stimulation might prolong survival and prevent the progression of ventilator dependency in ALS. These outcomes must be studied in a randomized controlled manner.
With an initial grant from ARSla (Association pour la recherche sur la SLA) and AFM (Association française contre les myopathies)
P12 CLINICAL IMPLICATION OF PARAMETERS USED FOR EVALUATING EXPIRATORY MUSCLE FUNCTION IN NMD
CHOI WA1, KANG SW1, BAEK JH1, KIM DH2
1Department of Rehabilitaiton Medicine and Rehabilitation Institute of Muscular Disorder,Yonsei University College of Medicine, Seoul, Republic of Korea, 2Department of Rehabilitation Medicine, Wonkwang University School of Medicine and Hospital, Iksan, Jeonbuk, Republic of Korea
Email address for correspondence: [email protected]
Keywords: expiratory muscle, peak cough flow, peak expiratory flow
Background: Pulmonary complication caused by respiratory muscle weakness comprises major mortality in neuromuscular disease (NMD). Expiratory muscle weakness not only results in a reduction of coughing capacity but also induces a failure of clearing airway secretions. Therefore, assessment of expiratory muscle function is important to infer coughing capacity of NMD patients.
Objectives: The aim of this study is to evaluate clinical utility of several parameters used for assessing expiratory muscle function; peak expiratory flow (PF), peak cough flow (PCF), and maximal expiratory pressure (MEP).
Methods: Thirty amyotrophic lateral sclerosis (ALS) patients with bulbar impairment and 31 Duchenne muscular dystrophy (DMD) patients were included in this study. 53 age-matched healthy subjects were included as a control group. Control group A were age-matched with DMD, and control group B were age-matched with ALS patients. PF and PCF were obtained with a peak flow meter. MEP was measured by a respiratory pressure meter with mouth piece.
Results: Mean MEP in DMD and ALS was 23.8 ± 14.6% and 18.2 ± 15.1% of normal predicted values respectively. Mean PF and PCF was 147.4 ± 46.1 L/min and 205.6 ± 54.9 L/min in DMD, 125.5 ± 46.0 L/min and 153.3 ± 59.1 L/min in ALS, 490.3 ± 55.2 L/min and 1674.1 ± 59.0 L/min in control group A, and 435.6 ± 52.5 L/min and 636.7 ± 62.8 L/min in control group B respectively. Patient groups showed significantly lower values of PF and PCF than control group (p < 0.05). PF and PCF were positively correlated with MEP both in DMD (r2 = 0.888, r2 = 0.458, p < 0.01) and ALS patients (r2 = 0.997, r2 = 0.456, p < 0.01). The mean PCF-PF difference calculated by a formula of PCF-PF/PF × 100 was 46.2 ± 27.1% in control group A, 47.5 ± 16.3% in control group B, 41.9 ± 15.2% in DMD, and 21.5 ± 7.1% in ALS with bulbar impairment respectively. The PCF-PF difference in ALS with bulbar impairment was significantly lower compared to values of control group (p < 0.05).
Discussion and conclusions: Both of PCF and PF were significantly decreased in NMD patient group, and both parameters positively correlated with MEP. The results of this study implicate that these parameters can be useful for assessing expiratory muscle weakness, especially for patients presenting difficulty in measuring MEP. In addition, PCF-PF difference can be applied as a valuable parameter to detect bulbar impairment.
P13 THE EFFECT OF POSTURE ON MAXIMAL PEAK COUGH FLOW VALUES IN INDIVIDUALS WITH ALS
CLEARY S, HOPPER T, CUST S, KALRA S, JOHNSTON W
University of Alberta, Edmonton, Alberta, Canada
Email address for correspondence: [email protected]
Keywords: coughing, swallowing, positioning
Background: Peak cough flow (PCF) is a surrogate measure of cough strength and capacity and is measured using a spirometer or hand-held PCF meter. According to American Thoracic Society (ATS) standards, patients should be seated fully upright during pulmonary function testing. However, the effect of sitting angle on PCF in patients with ALS has not been studied systematically.
Objective: The following research questions were of interest: 1) Is there a difference in PCF during coughing when patients with ALS sit with 90 degrees flexion as compared to 80 degrees flexion? 2) What degree of forward flexion do patients with ALS naturally assume while swallowing?
Methods: Ten patients with ALS participated in the pilot study. They ranged in age from 39-75 years with an average time since diagnosis of 18.8 months and a mean total ALSFRS-R score of 27.9. To answer the first research question, we used a within-group, pre-test post-test design. Measures of PCF were collected with a Vernier digital spirometer and respiratory chest strap while the patient was seated in a modified chair which included a fixed Goneometer attachment and a moveable back support to place patients in each of the two angles of flexion, (90 and then 80 degrees). A digital laser was used to validate the point at which the particular degree of forward flexion was reached. The dependent measure was the maximum of three coughs. For the second research question, patients were observed eating pudding over 10 swallows, while seating in the modified chair. The dependent measure was the average degree of forward flexion at the onset of swallowing.
Results: Research Question 1: The mean PCF at 90 degrees (239.4, SD = 166.1) was less than the mean PCF at 80 degrees (267.6; SD = 151.1); however, this difference was not statistically significant (Wilcoxon Signed Ranks, z = 21.68; p = 0.08). Seven out of 10 participants showed a pattern of greater PCF when seated at 80 versus 90 degrees. Research Question 2: The mean degree of flexion during eating and swallowing was 85.1 degrees. Eight of the 10 participants had a forward flexion of less than 90 degrees; 2/10 had a forward flexion greater than 90 degrees.
Conclusions: The pilot nature of this study precludes firm conclusions about the effects of forward flexion angle on PCF during coughing. However, the preliminary findings are the foundation for future research with larger samples. The finding that 8/10 of the participants sat slightly forward while swallowing, suggests a natural tendency to compensate for weakness and improve airway protection through postural adjustment. Theoretical and clinical implications will be discussed.
P14 THE VOICE ANALYSIS AND MANOMETRIC EVALUATION OF SWALLOWING IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS
TOMIK J1, TOMIK B2, GAJEC S1, SKLADZIEN J1, WIATR M1, STREK P1, OLES K1, SZCZUDLIK A2
1ENT Department Medical College Jagiellonian University, Krakow, Poland, 2Neurological Department Medical College Jagiellonian University, Krakow, Poland
Email address for correspondence: [email protected]
Keywords: manometry, voice analysis, movement disorders
Background: Patients with bulbar onset present firstly with speech disorders, swallowing problems and dysphonia. The type and dynamic of voice disturbances can be assessed via perceptual, videostroboscopy and acoustic analyses. The swallowing problems in ALS patients are frequently estimated using manometric parameters.
Objective: The aim of the study was to analyse the phonation function of the larynx and the disturbances of the oro-pharyngeal swallowing phase of dysphagia in ALS patients.
Material and methods: Thirty two ALS patients were diagnosed in the Department of Neurology. There were 19 (59.4%) male and 13 (40.6%) female patients, aged between 26 and 78 (mean: 59.9 ± 11.4 years). Complete phoniatric and manometric examinations were performed three times: at the ‘start point’ of the research, and then 6 and 12 months later in ENT Department. The videostroboscopy (VSS) examinations of larynx demonstrated on range of voice, symmetricity and amplitude of vocal fold's vibrations, mobility and closure of vocal folds. The maximal phonation time (MPT) and acoustic parameters such as: average fundamental frequency (Fo), Jitter, Shimmer as well as noise-to-harmonic ratio (NHR) were discovered. Manometric examinations of pharyngeal segment were carried out by using the esophageal ballon-based method with 4 balloon transducers. The following manometric parameters were analysed: the maximal contractions of the base of tongue (CBT), the UES resting pressure (RP); hypopharyngeal suction pump (HSP) as well as the oropharyngeal, pharyngeal and hypopharyngeal transit time (OTT, PTT, HTT, respectively) and velocity for bolus.
Results: Significant weakness of CBT (p = 0.007), decrease of HSP (p = 0.006) and decrease of velocity of bolus transit (p = 0.001) were particularly marked between the first and the third examination. MPT was shorter (p < 0.001) in women during all three examinations. The amplitude of vocal fold movement was shortened in all tests (p < 0.001), vocal fold vibration was irregular in all patients during the third test (p < 0.001) and we observed incomplete vocal fold closure in all subjects in second and third examinations. Jitter and Shimmer increased (p < 0.001) especially in women. The value of vFo did not differ whereas NHR parameter was significantly higher in three tests, but only in women.
Discussion: This study shows that change on VSS (especially amplitude of movement, vibration and closure of vocal fold) was the most frequent observed in bulbar ALS cases. The voices of these patients were characterized with abnormal parameters of acoustic analysis and very short MPT. The results obtained from manometric examinations confirm progression of swallowing disorders.
Conclusion: The manometric and phoniatric examination are useful and supportive methods in the analysis of voice and swallowing disturbances in ALS patients.
P15 THE IMPACT OF PERCUTANEOUS ENDOSCOPIC GASTROSTOMY ON SURVIVAL IN PATIENTS WITH MOTOR NEURONE DISEASE: RESULTS FROM AN AUSTRALIAN MND SERVICE
LABRA J, ALLEN C, MORRISON S
St Joseph's Hospital, Auburn, NSW, Australia
Email address for correspondence: [email protected]
Keywords: PEG, area of onset, survival
Background: Up to 80% of patients with Motor Neurone Disease (MND) will develop swallowing difficulties. This can lead to dehydration, malnutrition and aspiration pneumonia. The option of percutaneous endoscopic gastrostomy (PEG) is often presented with the intention of providing a safer and more comfortable means of nutrition and hydration, improving quality of life and prolonging survival. However, the impact of PEG on survival continues to be debated.
Though there have been no randomised controlled trials (RCTs) looking at the impact of PEG on survival or quality of life, the evidence available shows a trend towards positive outcomes in both of these areas. Furthermore, there has been little research comparing the survival impact of PEG in bulbar- versus limb-onset MND.
Objectives: Our main aim was to compare survival of patients with and without PEG, suffering from bulbar- versus limb-onset MND.
Methods: A retrospective review of all deceased patients in the St Joseph's Hospital Multidisciplinary MND Service database from July 2006 to April 2011 was conducted. Sixty-six subjects were classified into four groups: bulbar-onset with PEG (n = 25); bulbar-onset without PEG (n = 5); limb-onset with PEG (n = 13); and limb-onset without PEG (n = 20). Survival times from the date of diagnosis until death were then compared.
Results: For bulbar-onset patients, those without PEG survived an average of 10 months post-diagnosis (range 1.5 2 48 months), compared to those with PEG who survived an average of 15 months post-diagnosis (range 4 2 32 months). Interestingly, we found the opposite in limb-onset patients, where those without a PEG survived an average of 23 months (range 1.5 – 72 months), while those with PEG survived on average only 18 months post-diagnosis (range 3 2 36 months).
Discussion and conclusions: A retrospective review of 66 deceased patients with MND showed an average five month survival advantage for bulbar-onset patients with PEG, compared to those without PEG. This outcome was predicted, as the presence of a PEG enables nutrition and hydration to be maintained until there is onset of respiratory failure.
For patients with limb-onset MND the opposite was found, with an average five month survival disadvantage in those with PEG. Anecdotal analysis of the limb-onset patients suggested factors contributing to a poorer outcome include delayed patient acceptance of PEG insertion and earlier onset and more aggressive progression of dysphagia in those who underwent PEG.
Larger studies comparing PEG survival advantages among the various types of MND are required. It would be beneficial if these studies included information regarding time of onset and severity of dysphagia for all patients. Furthermore, research into the reasons for delayed patient acceptance of PEG, as well as the effect of PEG on quality of life would be valuable.
P16 RELEVANCE OF NUTRITION ON SURVIVAL OF PATIENTS WITH MOTOR NEURONE DISEASE
STANICH P1, CASTRO I1,2, LEICO ODA A1, NAKAO F1, DOS SANTOS SALVIONI C1, MESQUITA E SILVA T1, VITAL DE CARVALHO E1, DE SOUZA BULLE OLIVEIRA A1
1University Federal of Sao Paulo, São Paulo, Brazil, 2University of São Paulo, São Paulo, Brazil
Email address for correspondence: [email protected]
Keywords: survival analysis, percutaneous endoscopic gastrostomy, nutritional assessment
Aims: To evaluate the effect of nutrition on survival of patients with Motor Neurone Disease (MND) and present the predictor variables for indications of nutritional therapy, percutaneous endoscopic gastrostomy (PEG).
Methods: It was a retrospective longitudinal cohort study, from 2000 to 2008, and the sample consisted of 128 patients with MND. Clinical, nutritional and respiratory variables were analysed. Analyses were conducted by adopting the survival as the dependent variable. The survival curve was evaluated by Kaplain-Meier. The variables that had a significance level of 20% (p < 0.20) were selected for the proportional regression model of Cox.
Results: One hundred and eleven patients underwent gastrostomy, and 59 limb onset (ALS) and 52 with bulbar onset (PBP). Malnutrition was present in 32% of the population before PEG, most frequently in patients with limb onset. The survival time after PEG was 10.5 months for patients with PBP and 16 months for ALS (p < 0.05). Variables associated with survival were: early indication in the PEG, for ALS and PBP; reduction of FVC% and BMI before PEG (hazard ratio of 0.254, p = 0.007) for patients with limb onset and exclusion of oral feeding and tracheostomy (hazard ratio of 0.345, p = 0.014) for patients with bulbar onset.
Conclusions: Early insertion of percutaneous endoscopic gastrostomy, from the time of diagnosis was a protective factor for patient survival. Malnutrition was a bad prognostic factor, especially for patients with limb onset. Nutritional surveillance for disease progression may improve results when the goal is to increase the survival of patients with MND/ALS.
P17 SELF-ASSESSMENT OF THE DAILY FOOD INTAKE IN ALS VIA AN APPLICATION ON A MOBILE DEVICE
HOLM T, MAIER A, LINKE P, MÜNCH C, MEIER R, LEIMEISTER JM, PRINZ A, STEINFURTH L, MAIER T
Charité University Hospital, Berlin, Germany
Email address for correspondence: [email protected]
Keywords: self-assessment, food intake, nutrition
Background: In the course of the disease an undesirable weight loss is common in ALS patients. Nutritional status is an important prognostic factor for survival in ALS. The early detection of alterations in the food intake as well as changes in weight is essential for these patients. Commonly a nutrition consultation is performed after patients develop swallowing difficulties or suffer from weight loss. The nutritionist anamnestically determines a retrospective dietary protocol by conducting a standardized interview to evaluate the daily oral food intake and the daily energy imbalance.
Objectives: To examine the recording of the actual nutritional intake in ALS via a web-based application on a tablet computer.
Methods: In a prospective, controlled study, patients recorded the oral food intake via an Internet-based nutrition application by using a touchscreen tablet computer in their home care environment. The application requires internet connection to store the data on a server located in the virtual and physical secured environment of the research facility. Based on the established “quartered plate method” the web application shows different options of meals, portions and durations. Patients assess the portion and duration of every single meal according to a full plate compared to their normal food intake. During the inclusion visit the nutritionist calculates the individual mean daily food intake of every participant and estimates individual portions. The self-assessment should be done at three predetermined days per week over a period of three months.
Results:We already included 10 patients. Every patient is provided with a tablet computer with the installed application and a nutrition consultation was performed. The intuitive user interface and the simple usability improve the compliance especially in patients with manual deficits.
Discussion: Web applications on tablet computers or on smartphones are well known by a wide range of internet users. To our knowledge we present the first web application for measuring the daily caloric intake in ALS. The study establishes the methodical feasibility and clinical tolerability of a web application for monitoring the daily food intake in ALS patients. Based on this technique, patients are able to record the nutritional intake between the outpatient visits; we suggest that use in clinical practice enables the early detection of changes for nutritional intervention.
P18 INTERNET-BASED SELF-ASSESSMENT OF APPETITE IN ALS
HOLM T, MAIER A, LINKE P, MÜNCH C, STEINFURTH L, JANZEN C, MEIER R, MEYER T
Charité University Hospital, Berlin, Germany
Email address for correspondence: [email protected]
Keywords: self-assessment, loss of appetite, nutrition
Background: An undesirable loss of weight over the course of the disease is common in ALS patients. Underlying causes are malnutrition, hypermetabolism, cachexia as well as a multifactorial reduction of appetite. The established ALS Functional Rating Scale, ALS FRS-R, records symptoms of dysphagia and manual deficits upon intake of food. The loss of appetite as a therapeutically relevant cause of undesirable weight loss is not reflected in the ALS FRS-R.
Objectives: In the context of a controlled study, we examined for the first time the symptom of loss of appetite in ALS by means of a specific self-assessment score.
Methods: In a prospective, controlled and stratified study, patients conducted a symptom-oriented self-assessment of appetite. An SQL database structure was developed for the technical realisation. Via encoded connections, a questionnaire is generated via the front-end of a web server featuring a specially secured operating system. Patients have access to a protected online portal via the URL www.ALShome.de. The internet-based self-assessment is realised by using the Council on Nutrition Appetite Questionnaire (CNAQ). This self-assessment questionnaire comprises 8 questions. Patients answer by using a 5 point scale. The score is a sum of these 8 items with a maximum of 40 points. Lower scores indicate deterioration in appetite, if the patient scores 28 or less a predicted weight loss of at least 5% within the next six months occurs. Besides the assessment of appetite we looked for an association between ALS related symptoms and a reduction of appetite.
Results: Fifty seven patients, 38 (67%) males and 19 (33%) females were included in this self-assessment trial. During the first self-rating 53% obtained the critical score. Patients with dyspnea more often displayed a critical loss of appetite, we detected 29 patients suffering from mild to severe dyspnea, of these 58.6% displayed the critical CNAQ Score, whereas only 31.8% of 22 asymptomatic patients were affected. The mean CNAQ Score in patients with dyspnea was 26.9, in patients without dyspnea 29.7 points.
Discussion: In our present internet based self-assessment study we first investigated loss of appetite in ALS patients. The collected data suggest that already 50% of the patients suffer from severe loss of appetite. Early ascertainment of indications for nutrition management including ecotrophological consultation, supplementary nutrition and pharmacotherapy represent essential objectives in the treatment of undesirable weight loss.