Symptom management in amyotrophic lateral sclerosis: We can do better

Patients with amyotrophic lateral sclerosis (ALS) may receive the erroneous message, either from their physicians or through the lay press, that their disease is untreatable. The error lies in the misinterpretation of treatment as cure. There are many diseases we cannot cure. Treatment in the form of symptom management has long been a key role of physicians, nurses, and other health care providers, as noted by Hippocrates more than 2,000 years ago: “Cure sometimes, treat often, comfort always.” A cure for ALS remains elusive. Disease modifying therapies can slow progression or prolong survival but cannot improve strength or function. Thus, as they have since ALS was initially described by Charcot in 1869, clinicians have directed their efforts toward optimizing quality of life (QOL) for persons with ALS and their families through symptom management. In doing so, a multidisciplinary approach to ALS care prolongs survival and provides improved QOL compared with conventional care. The study presented by Nicholson and colleagues in this issue provides insight into the current state of symptom management of a cohort of ALS patients. Data on the presence and severity of these symptoms, the degree to which symptoms were bothersome, and the efficacy of symptom management were collected. About 31% of those who were contacted responded. The mean disease duration was 5 years. In order of decreasing prevalence, patients cited fatigue (90%), muscle stiffness (84%), muscle cramps (74%), shortness of breath (66%), sleep difficulty (60%), pain (59%), anxiety (55%), depression (52%), increased saliva (52%), constipation (51%), pseudobulbar affect (38%), loss of appetite (37%), and weight loss (29%). Fatigue was not only the most frequently reported symptom but also the least frequently treated one. It is striking that so many other symptoms commonly encountered in ALS were found to have low treatment prevalence: weight loss 22%, pseudobulbar affect 29%, sialorrhea 32%, muscle stiffness 39%, pain 44%, and dyspnea 49%. If symptom management is the best care we have to offer our patients, then it is concerning that these percentages are so low. Are we missing opportunities to improve the quality of the lives of our patients and, if so, why? Mental health care fares no better. Specifically, this study also calls attention to the relatively high prevalence of anxiety and depression, consistent with the overall significant psychological morbidity noted in patients with ALS. Several years ago, a call was issued for further research into psychological intervention. However, treatment prevalence of anxiety and depression in this recent large survey was only about 50%, suggesting that the need for effective psychological intervention remains, to a great degree, unmet. Why does this continue to be true? Can some of the blame be attributed to a lack of awareness of these symptoms on the part of physicians? This seems unlikely. Attention to the need for symptomatic treatment of patients with ALS is not new. The American Academy of Neurology issued practice parameters in 2009, identifying a number of areas in which symptomatic treatment is important. Many of the symptoms cited at that time appear prominently in the article by Nicholson and colleagues. It is notable that the high prevalence of fatigue in ALS patients is not a new finding. It can worsen over the course of the disease and has been shown to improve with modafinil therapy. Is the gap in providing adequate symptom management due to lack of evidence for the efficacy of such treatments? Most likely, this is at least partially the case. The 2009 American Academy of Neurology guidelines note varying degrees of evidence for symptomatic treatments. Then and now, double-blind, placebo-controlled studies were available for only a few such treatments, such as dextromethorphan plus quinidine for pseudobulbar affect and botulinum toxin for sialorrhea. The lack of high-quality clinical trials for the treatment Correspondence to: S. A. Goutman; e-mail: sgoutman@med.umich.edu