Non-invasive positive pressure ventilation (NPPV) can improve survival in ALS patients with advanced respiratory impairment, but it is not known if it is beneficial earlier in the disease course. A retrospective cohort study of patients with ALS was performed comparing survival from time of diagnosis in subjects who started NPPV use when their FVC was >or=65% predicted (Early NPPV) with subjects who started NPPV when their FVC was below 65% predicted (Standard NPPV). The Early group (n = 25) and the Standard group (n = 67) were similar except for pulmonary function (mean FVC in Early NPPV group = 74.3+/-10.1% predicted and 48.3+/-11.3 in Standard group, p<0.001). The median time from ALS diagnosis to death was significantly longer in the Early NPPV group (2.7 years vs. 1.8 years, p = 0.045). This remained significant after adjustment for potential confounding factors (H.R. = 0.55, 95% CI 0.31-0.98). Survival from time of diagnosis was nearly one year longer in the Early group. Until more definitive data are available from randomized trials, our findings suggest that clinicians either encourage earlier use of NPPV or use more sensitive tests for respiratory muscle impairment than upright FVC.

We confirmed earlier findings showing that BMAB is poorly tolerated. While mean pain scores were not significantly different between the study arms, secondary analyses suggest that viewing a nature scene while listening to nature sounds is a safe, inexpensive method that may reduce pain during BMAB. This approach should be considered to alleviate pain during invasive procedures.

Retrospective studies are important in ALS but require markers of disease severity to enable risk adjustment and to allow fair comparisons between patient groups. The ALSFRS-R could be used as such a measure. This study aimed to determine if accurate ALSFRS-R scores could be generated by reviewing clinic notes. Five investigators reviewed 100 de-identified clinic notes to generate estimated ALSFRS-R scores. These scores were compared to ALSFRS-R scores completed by patients within three months of the clinic note. The retrospective ALSFRS-R scores did not differ significantly from the actual scores (mean retrospective score 38.7+/-5 vs. actual score 38.4+/-6, p =0.5). The intra-class correlation coefficient between actual and retrospective scores confirmed reasonable agreement (rho = 0.53, p <0.001). Bland Altman analysis also confirmed good agreement between the actual and retrospective scores. This study indicates that ALSFRS-R scores can be accurately reproduced from information in clinic notes and should be considered as a marker of disease severity for use in retrospective studies.