Longitudinal assessment of noninvasive positive pressure ventilation adjustments in ALS patients
ABSTRACT The absence of data guiding optimal titration of noninvasive positive pressure ventilation (NIPPV) over time in ALS patients may contribute to the under-prescribing of NIPPV. We conducted a retrospective, single-center, chart review assessment of NIPPV pressure settings used for symptomatic treatment of ALS patients to determine NIPPV adjustments, and to compare survival between those who were tolerant and intolerant to NIPPV. All subjects were started on nocturnal NIPPV at 8 and 3 cm H2O inspiratory and expiratory pressure, respectively. Of the 18 tolerant subjects identified, 4 (22%) had no NIPPV pressure changes before death; 8 (44%), 1 change; 4 (22%), 2 changes; 1 (6%), 3 changes; and 1 (6%), 5 changes. Most pressure changes occurred during the first year of NIPPV initiation. The maximum pressure needed for comfort by any patient in this study was 19/5 cm H2O, while 4 (22%) found the original 8/3 cm H2O settings to be sufficient until death. Subjects in the tolerant group had better survival, when adjusting for age and site of symptom onset (bulbar versus limb), with a hazard ratio of 0.23 [95% confidence interval: 0.10, 0.54]. The current data suggest that ALS patients who are tolerant to NIPPV typically need at least one upward change in pressure settings. Tolerance to relatively low NIPPV inspiratory pressures is associated with improved survival.
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ABSTRACT: Clinical trials in amyotrophic lateral sclerosis have significantly evolved over the last decade. New outcome measures have been developed that have reduced the sample size requirement as compared with survival studies. There has been increasing recognition that dose-ranging studies are crucial to full evaluation of experimental agents. While the requirements of late stage trials have not changed, many new designs have been suggested for earlier phase development. While no design achieves the perfect balance of sensitivity and efficiency, clinical trialists continue to work toward the goals of smaller and shorter trials so that more compounds can be studied concurrently.Physical Medicine and Rehabilitation Clinics of North America 09/2008; 19(3):495-508, ix. DOI:10.1016/j.pmr.2008.02.002 · 1.09 Impact Factor
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ABSTRACT: To prospectively investigate causes of death and the circumstances surrounding death in 302 patients with amyotrophic lateral sclerosis (ALS). The functional status of patients immediately before death was also determined. Information was obtained from neurologists at ALS centres, patients' files, and, when deaths occurred outside a medical facility, attending physicians. Most patients (63%) died in a medical facility. The most frequently reported cause of death was respiratory failure (77%), including terminal respiratory insufficiency (58%), pneumonia (14%), asphyxia due to a foreign body (3%) and pulmonary embolism (2%). Ten per cent of patients died from other causes: post-surgical or traumatic conditions (5%), cardiac causes (3.4%), suicide (1.3%) and sudden death of unknown origin (0.7%). The cause of death could not be determined in 13% of cases (6% inside a medical facility and 25% outside). At the time of death, only 55% of patients were receiving riluzole, 33% were undergoing non-invasive ventilation, 3% had a tracheotomy and 37% a gastrostomy. The information provided by this study helps to improve our understanding of the natural history of the disease and may help optimize the quality of care we can offer patients at the end of life.European Journal of Neurology 12/2008; 15(11):1245-51. DOI:10.1111/j.1468-1331.2008.02307.x · 3.85 Impact Factor
- Sleep Medicine Clinics 09/2010; 5(3):485-505. DOI:10.1016/j.jsmc.2010.05.002