No abstract

Background Riluzole is approved for the treatment of amyotrophic lateral sclerosis in most countries. Questions persist about its clinical utility because of high cost and modest e icacy. Objectives To examine the e icacy of riluzole in prolonging survival and in delaying the use of surrogates (tracheostomy and mechanical ventilation) to sustain survival, and to assess the e ect of riluzole upon functional health.

The type and quality of end-of-life care varies greatly in ALS; the time to initiate end-of-life care is not defined, and decision making is hampered by logistical and financial barriers. There has been no systematic review of these issues in ALS. The goals of this initiative are to: 1) improve end-of-life care for patients with ALS and families based on what limited evidence is available; 2) increase awareness, interest, and debate on the end-of-life care in ALS; and 3) identify areas needed for new prospective clinical research. The ALS Peer Workgroup reviewed the literature and 1) identified the current state of knowledge, 2) analysed the gaps in care, and 3) provided recommendations for standard of care and future research. It was shown that areas of investigation are needed on the incorporation of an interdisciplinary approach to care in ALS that includes: psychosocial evaluation and spiritual care; the use of validated instruments to assess patient and caregiver quality of life; and the establishment of proactive caregiver programs. Several public policy changes that will improve coverage for medical care, hospice, and caregiver costs are also reviewed. More clinical evidence is needed on how to provide optimal end-of-life care specifically in ALS.

In spite of emerging evidence of therapeutic benefit from non-invasive positive pressure ventilation (NPPV), only a minority of ALS patients use this therapy. We examined factors which correlate with use of NPPV in ALS patients. Data were analyzed from the ALS CARE Database on the use of NPPV in patients with FVC less than 50% of predicted and probable or definite ALS based on modified El Escorial criteria. Of the 403 eligible patients, 146 (36%) used NPPV. NPPV compliance was strongly correlated with symptoms of dyspnea and orthopnea as well as with the use of other therapies including PEG tubes, augmentative speech devices, and riluzole. Male gender and household income >$80,000 were also associated with higher NPPV use. There was no correlation between age, race, type of insurance, forced vital capacity, duration of symptoms, ALSFRS-R, caregiver burden or quality of life with the use of NPPV. These data suggest that the factors which are most closely associated with NPPV utilization are symptomatic orthopnea and dyspnea. The findings may be useful in designing prospective studies to examine the factors which might explain the underutilization of NPPV and the optimal use of this treatment.

Multidisciplinary care in ALS is associated with longer survival, improved quality of life, and reduced hospital admissions, but there are no published data on institutional costs associated with multidisciplinary ALS care at U.S. centers. We prospectively examined institutional costs, adherence to AAN Practice Parameters and patient satisfaction in multidisciplinary ALS clinics at 18 U.S. ALS centers. Centers reported patient volumes; direct costs for staff salary/benefits, supplies and equipment; and institutional non-salary and overhead costs over a three-month period. In 1117 patients seen during this period, mean age was 61.5 years (range 25-91 years), 56% were male, and mean ALSFRS-R score was 29. Mean total salary/benefit cost per clinic day for all providers was $2964 (range $1692-$5236 across centers). Mean salary/benefit cost per patient per clinic was $507 (range $258-$806 across centers). Differences among centers in reporting non-salary costs prevented meaningful analysis. Practice parameter adherence and patient satisfaction were high. This prospective collaborative study demonstrates the direct financial burden of evidence-based multidisciplinary ALS care in the U.S.; more refined non-salary and overhead cost data are needed to evaluate the full cost impact of care. These data may be useful in supporting evidence-based models of patient centered care for ALS.