Q&A: Robert G. Miller Discusses New ALS Guidelines

October 5, 2009


Two new AAN guidelines—"The Care of the Patient with Amyotrophic Lateral Sclerosis: Drug, Nutritional, and Respiratory Therapies (an evidence-based review)," and "The Care of the Patient with Amyotrophic Lateral Sclerosis: Multidisciplinary Care, Symptom Management, and Cognitive/Behavioral Impairment (an evidence-based review)"—were published in the October 13, 2009, issue of Neurology® (2009; 73:1218-1233). AAN.com asked Robert G. Miller, MD, FAAN, to discuss how the guidelines were developed. He spoke with AAN.com Practice & Technology Editor, Neil A. Busis, MD, FAAN.

AAN.com: What is the purpose of these AAN clinical practice guidelines?

Miller: To systematically examine the evidence on managing patients with Amyotophic Lateral Sclerosis (ALS) that has become available since the guidelines were first published in 1999, and to make recommendations for treatments that are evidence based, rather than based on opinion or even consensus. The first editions of the guidelines identified both the areas with strong evidence for ALS treatment and the areas for which strong evidence is lacking.

AAN.com: Who is the target audience?

Miller: Neurologists and other clinicians who manage persons with ALS, but there is also a lay version for patients and families.

AAN.com: What are the main conclusions of these new guidelines?

Miller: Many treatments are now available that will help persons with ALS live longer and easier lives. Evaluation of the adherence to the first editions of the guidelines showed underutilization of practices for which there was strong evidence. Many of these practices were studied in the last decade, but now with stronger evidence about benefit, we hope treatments will be more widely used.

AAN.com: How should neurologists use these guidelines in clinical practice?

Miller: They should serve as a tool to help clinicians guide decision-making for patients regarding key therapeutic decisions in the course of ALS. The guidelines indicate where there is strong evidence for certain treatments, as well as areas that have not been adequately studied.

Guidelines should be used with consideration of each patient's circumstances. Guidelines provide information that should enhance disease management, particularly when the use of recommendations deriving from strong studies is available.

AAN.com: One recommendation is "Specialized multidisciplinary clinic referral should be considered for patients with ALS to optimize health care delivery and prolong survival." What services should be available in such clinics?

Miller: The evidence supporting multidisciplinary clinics has been much improved since the first editions of the ALS guidelines. Such clinics should have a rigorous approach to diagnosis, in order to provide an expert opinion regarding the diagnosis, prognosis, and treatment plan, usually by a neurologist. Some clinics have a rehabilitation specialist experienced in treating ALS, and most have a nurse case manager. These people will work to coordinate care. The clinic services typically include those of a physical therapist, who focuses on maintaining range of motion and mobility. Occupational therapist services are also provided, which help with performing daily activities and adapting the home environment. A speech pathologist is often available to help develop different ways of communicating and give advice to make swallowing easier. Further, many clinics staff dietitians, whose purpose is to examine nutrition, as well as employing respiratory therapists to help with breathing. A social worker is often available to help cope with the disease and to identify additional resources for psychosocial issues.

AAN.com: Another recommendation is "Screening for cognitive and behavioral impairment should be considered in patients with ALS," as many patients with ALS demonstrate cognitive impairment, which in some cases meets criteria for dementia. Please comment on non-motor aspects of ALS. How has our understanding of the clinical manifestations of this disease changed recently?

Miller: There are genetic links between frontotemporal dementia and ALS. Fortunately, the full syndrome of frontotemporal dementia is found in only five percent of patients with ALS. However, cognitive and behavioral impairment, indicating frontal or executive dysfunction, may be found in a much higher proportion of patients. These abnormalities may be subtle and require vigilance for the clinician to detect them. Sensitive screening tests for clinic use are available.

AAN.com: Is there an "expiration date" on these guidelines? If so, when will they be updated?

Miller: They will be reexamined annually in order to consider an update. When there are significant advances in therapeutics, they will be updated.

AAN.com: What are the most important unanswered questions regarding the care of the patient with ALS?

Miller: We desperately need new therapeutics, both pharmacological and physical, to alter the relentless progression of the disease. In this regard, new genes have been discovered that may lead to better animal models for screening promising therapies. While we are waiting for the cure, we need a better understanding of how to optimize management of persons with ALS:

  1. We need to better understand why helpful evidence-based therapies (e.g., multidisciplinary clinics, riluzole, noninvasive ventilation [NIV], percutaneous endoscopic gastrostomy [PEG]) are underutilized.
  2. Optimal timing for NIV and PEG deserves further study, as do more effective methods of optimizing NIV in patients with bulbar dysfunction.
  3. Studies are needed to examine symptomatic treatments (e.g., cramps, spasticity, depression, insomnia, terminal dyspnea).
  4. We need a better understanding of the diagnosis, natural history, and treatment of cognitive and behavioral impairment in ALS.

A full listing of the ALS management areas identified as needing further research can be found in the updated ALS practice parameters.

Additional Resources

Author Disclosures

Dr. Miller serves on the editorial board for the ALS Journal; received a speaker honorarium from the AANEM; served as a consultant to Celgene, Knopp Neurosciences Inc., Teva Pharmaceutical Industries Ltd., Taiji Biomedical Inc., Sanofi-Aventis, Novartis, and Neuraltus; and receives research support from the NIH (R01 NS 44887 [PI]) and the Muscular Dystrophy Association (PI).

Within the past 24 months, Dr. Busis received personal compensation for his service as AAN.com Practice and Technology Editor, as well as for speaking at AAN and AANEM courses. In addition, he served as Editorial Advisor for Neurology Coding Alert, and has provided medical-legal consulting services for coding issues.