Caring for the ALS patient today: Are we under‐utilizing our available clinical tools?

Twenty-five years following the identification of the first known gene to cause amyotrophic lateral sclerosis (ALS), the ongoing treatment and care of these patients continues to remain a challenge. The expectations of recent scientific gains are tempered by the lack of an effective treatment to significantly alter the course of this disease. The ALS patient remains unique with respect to the complex variability of clinical presentation, as evidenced by the bulbar versus spinal site of disease onset, the recognized contiguous spread with nonlinear rate of progression, the functional and psychological impact on everyday life, and often most decidedly, the underlying patient expectations for a curative disease altering therapy. Research on novel stem cell and gene therapies has provided renewed hope for an effective treatment, yet until this has been achieved, how do we continue to provide the most optimal care? As these patients routinely turn to off-label compounds and testimonial trials, we as clinicians need to provide the necessary medical guidance to permit a sound framework for hope. This begins with a proper understanding and active clinical integration of the recognized and validated interventions known to influence the course of this disease. Clinicians must become familiar with the established markers of disease progression and the timely initiation of these interventions to assist the patient in maintaining the highest level of functional ability. Are we, however, underutilizing the currently available interventions needed to maximally impact the life quality and disease course in these patients? As bulbar and respiratory compromise are major sources of morbidity and mortality that ultimately face the ALS patient, an emphasis on properly assessing these systems with clinically reliable and validated measures, applicable within a busy ALS clinic, would appear essential. Recognized pulmonary measures such as forced vital capacity (FVC) and maximum inspiratory pressure, routinely administered in the clinic, may simply not be enough. The literature is replete with studies assessing the effects of respiratory compromise in ALS, yet in a recently published review of the respiratory measures routinely followed in ALS, survival was shown to be significantly influenced by the use of multiple standard respiratory parameters, including the rate of decline in slow vital capacity the timely initiation of noninvasive ventilation (NIV), measures of nocturnal pulse oximetry, maximal esophageal pressure, and phrenic nerve motor amplitudes. Additionally, reliable objective measures of bulbar disease progression available within our clinics include speech system (speaking rate and sentence intelligibility) and subsystem (articulatory, phonatory, respiratory, and resonance) analysis, instrumentation based testing (pause analysis), tongue strength measures (Iowa Oral Performance Instrument, IOPI), voice recordings used for longitudinal speech assessments, and validated patient report scales assessing bulbar function (Center for Neurologic Study Bulbar Function Scale--CNS-BFS). A recent multicenter survey, however, highlighted the inconsistent variability in the usage of individual bulbar assessment tools within the ALS clinic setting. In this issue of the Journal, Matsuda et al. assessed the prognostic significance of the cough peak flow (CPF) rate of decline in ALS. They studied 34 ALS patients over 8 years, comparing CPF decline to the rates of decline of FVC, body mass index, and the revised ALS Functional Rating Scale (ALSFRS-R), following these patients to NIV, tracheostomy, or death. Citing a recent study that 32% of autopsied ALS patients who died from respiratory failure had evidence of an aspiration pneumonia, and understanding that an ineffective cough often leads to aspiration, the authors chose to study CPF rate of decline as a measure of both bulbar and respiratory function in ALS, and as a means of assessing effective coughing and airway clearance. The study identified significant correlations between the decline rate of CPF and (1) overall survival, (2) the bulbar score of the ALSFRS-R, and (3) FVC. Those patients with annual CPF decline rates of 25% or greater showed a much shorter survival than those with decline rates <25%. The study concluded that the CPF decline rate was significantly correlated with the progression of See article on pages 168–173 in this issue.