Update: The Care of the Patient with Amyotrophic Lateral Sclerosis

1. The ALS Association is proud to host this American Academy of Neurology presentation on ALS practice guidelines.

2. Report of the Quality Standards Subcommittee of the American Academy of Neurology R. G. Miller, MD, FAAN; C. E. Jackson, MD, FAAN; E. J. Kasarskis, MD, PhD, FAAN; J. D. England, MD, FAAN; D. Forshew, RN; W. Johnston, MD; S. Kalra, MD; J. S. Katz, MD; H. Mitsumoto, MD, FAAN; J. Rosenfeld, MD, PhD, FAAN; C. Shoesmith, MD, BSc; M. J. Strong, MD; S. C. Woolley, PhD Update: The Care of the Patient with Amyotrophic Lateral Sclerosis

3. Presentation Objectives • To describe the process of making an evidence-based guideline • To present the findings on and recommendations for care of people with amyotrophic lateral sclerosis (ALS) • Drug, symptom, nutritional, and breathing therapies • Multidisciplinary care in specialized ALS clinics • Behavioral and thinking problems

4. Practice Guidelines Evidence-based statements to helpdoctors and patients make smart decisions about health care

5. AAN Guidelines Are Evidence-based Consensus-based Evidence-based ?

6. AAN Guideline Process • Clinical Question • Evidence • Conclusions • Recommendations

7. Clinical Questions • The first step in developing guidelines is to askimportant clinical questions. • Questions look at areas of medicine where practice varies because of doctors’ understanding, beliefs, or attitudes. • Questions must be answerable with published data. • Answering the question must have the potential to improve care/outcomes.

8. Rating the Evidence Weak Strong Randomized Masked Study SingleCase Report Class I Class II Class IIIClass IV Level A B  C  U

9. AAN Level of Recommendations • Strong evidence = more than one high-quality scientific study • Good evidence = at least one high-quality scientific study or two or more studies of a lesser quality • Weak evidence = the studies, while supportive, are weak in design or strength of the findings • Not enough evidence = either different studies have come to conflicting results or there are no studies of reasonable quality

10. Introduction What is ALS? • ALS is a disease of weakening muscles. It is also called Lou Gehrig’s disease. The cause is unknown. • In ALS, certain nerve cells in the brain and spinal cord become damaged. The affected cells are called motor neurons. These nerve cells send signals to the muscles in your arms and legs, and to the muscles for speaking, swallowing, and breathing.

11. Introduction Is there a cure for ALS? Will I die from it? • ALS is serious. However, several therapies are now available to make life longer and easier. • ALS progresses quickly and eventually leads to death. Most people with ALS die within three to five years from the onset of their first symptoms. However, 20% of people live longer.

12. Drug Therapies for Lengthening Life Are there any medications I can take to help me live longer? • As of January 2010, riluzole is the only medication approved by the US Food and Drug Administration (FDA) to treat ALS. To a modest degree, riluzole slows the rate at which the disease worsens. • Strong evidence shows that riluzole can prolong survival by two to three months. Experts suggest a greater benefit when riluzole is started early in the course of the disease. • Riluzole is very safe but quite expensive. • People who are newly diagnosed with ALS should discuss riluzole with their neurologists.

13. Multidisciplinary Care My doctor says my ALS will greatly affect my health and how I live my life. How should I go about getting the care I need? • There is good evidence that visiting a multidisciplinary clinic can help people with ALS live longer and get the best possible care for their condition. • Weak evidence shows that people with ALS who visit a multidisciplinary clinic have better quality of life.

14. Multidisciplinary Care My doctor says my ALS will greatly affect my health and how I live my life. How should I go about getting the care I need?, cont. • These clinics are called multidisciplinary clinics because they provide a central location with many services that can help meet the needs of people with ALS. • These services include sessions with a neurologist or rehabilitation specialist experienced in treating ALS, as well as a nurse case manager. These people will work to coordinate your care.

15. Multidisciplinary Care My doctor says my ALS will greatly affect my health and how I live my life. How should I go about getting the care I need?, cont. • You also will meet with several other types of specialists, each of whom will focus on a particular area of care if you eventually need it. • A physical therapist will work with you on toning muscles and maintaining range of motion and mobility. He or she also will assist you with choosing devices that help with movement.

16. Multidisciplinary Care My doctor says my ALS will greatly affect my health and how I live my life. How should I go about getting the care I need?, cont. • An occupational therapist will help you and your caregiver with performing daily activities and adapting your home environment to meet your changing needs. • A speech pathologist will help you develop different ways of communicating and give advice to make swallowing easier.

17. Multidisciplinary Care My doctor says my ALS will greatly affect my health and how I live my life. How should I go about getting the care I need?, cont. • A dietitian will guide you with nutritionand maintaining your weight. • For help with managing your breathing, you will work with a respiratory therapist. • A social worker will work with you on how to cope with the disease and will identify additional resources to help you make the needed changes to your daily routine.

18. Nutritional Therapies Will having trouble swallowing affect my health? • When you have problems swallowing, eating becomes very difficult. It is common to begin to choke and accidentally breathe in food you are trying to eat. • Eventually weight loss and dehydration (not enough fluid in your system) can occur.

19. Nutritional Therapies How can this problem be treated? • It is important to manage your swallowing problem so that you maintain good nutrition. Changing the texture of your food, taking smaller bites, and tucking your chin slightlywhile you swallow can help. • You can also use a feeding tube called a percutaneous endoscopic gastrostomy (PEG) tube. These types of tubes are inserted through the skin into the stomach. or radiologically inserted gastrostomy (RIG) tube. • There is good evidence that a PEG or RIG tube will likely help stabilize body weight and prolong survival.

20. Nutritional Therapies Do vitamin and nutritional supplements help people with ALS live longer? • There is not enough evidence to show if vitamin or nutritional supplements help lengthen the lives of people with ALS. • Specifically, studies of creatine, CoQ10, and Vitamin E (taken in high doses) do not show a benefit.

21. Drug Therapies for Symptoms Are there any medications I can take to treat my symptoms? • There are medications available that can be very helpful in treating some symptoms of ALS. • One symptom, uncontrolled laughing or crying, can be embarrassing and hard to manage. • A person with ALS at times may cry excessively or laugh longer than expected at something that is only mildly funny.

22. Drug Therapies for Symptoms Are there any medications I can take to treat my symptoms?, cont. • There is good evidence that a combination of the medications dextromethorphan (DM) and quinidine (Q) will lessen episodes of uncontrolled laughing or crying—both how severe they are and how often they happen. • The side effects associated with DM and Q are dizziness, nausea, and sleepiness, so some people stop taking them. This combination of medications is currently awaiting FDA approval.

23. Drug Therapies for Symptoms Are there any medications I can take to treat my symptoms?, cont. • Another symptom, drooling, happens because of swallowing problems. • Drooling can be embarrassing, and trouble with swallowing the drool can be a health risk. The excess saliva can be accidentally breathed in, which in some cases contributes to pneumonia. • There are several medications used to dry up the drool, but none of them has been studied for use in ALS.

24. Drug Therapies for Symptoms Are there any medications I can take to treat my symptoms?, cont. • There is good evidence that injections of the medication botulinum toxin type B (BTxB) can help with drooling. The medication is injected into the glands near the jaw that make saliva. • No serious side effects have been reported from use of BTxB.

25. Non-Drug Therapies for Symptoms Are there other ways of treating these symptoms? • You can get radiation therapy for drooling if BTxB does not give relief. • There is weak evidence that small amounts of radiation therapy might help. The side effects of this therapy are sore throat, nausea, and reddening of the skin. • Ask your doctor whether radiation therapy would be a good choice for you.

26. Non-Drug Therapies for Symptoms Are there other ways of treating these symptoms?, cont. • Also, services such as physical therapy, occupational therapy, and speech and language therapy can help you live better and more fully with your symptoms.

27. Non-Drug Therapies for Symptoms There doesn’t seem to be much evidence for therapies for symptoms. Why? • In this guideline update, the authors were able to review only those studies that were specific to ALS. At this time, there are no studies available on use of these therapies in people with ALS.

28. Behavioral and Thinking Problems Is ALS a purely physical disease? • ALS is mainly thought of as a physical disease. Many people with ALS have problems only with weakness of voluntary muscles. • However, some people with ALS also develop problems with thinking or behavior changes, or both. • For a small number of people with ALS, the effects on the brain can lead to a form of dementia.

29. Behavioral and Thinking Problems Is ALS a purely physical disease?, cont. • The dementia that can occur with ALS progresses slowly and leads to changes in social behavior, loss of the ability to express feelings clearly and appropriately, and loss of the ability to reflect on things deeply. • Other problems in this type of dementia are language problems and poor self-care.

30. Behavioral and Thinking Problems Is ALS a purely physical disease?, cont. • Because these problems with thinking ability and behavior changes usually appear slowly, you may not notice them until they have become serious. • It is important to try to detect and manage these problems as early as possible. • Some people with ALS who develop these types of problems might not be willing to use important therapies that may prolong their survival or improve their quality of life.

31. Behavioral and Thinking Problems Is ALS a purely physical disease?, cont. • There are tests available that screen for behavioral or thinking problems in people with ALS. • Good evidence shows that people with ALS should be screened for possible problems with thinking ability or behavioral change.

32. Behavioral and Thinking Problems Is ALS a purely physical disease?, cont. • If you are screened for a problem with thinking ability and the results show some problems, your doctor may suggest further testing. • Talk to your doctor about whether such tests would be useful for you. Also ask your doctor about therapies that may help you and your caregivers to manage these changes.

33. Breathing Therapies How can problems with breathing be treated? • Breathing problems develop in people with ALS because of weakening of the muscles that help with breathing. • Treating breathing problems is very important. • The most common cause of death from ALS occurs from weakening of breathing muscles.

34. Breathing Therapies How can problems with breathing be treated?, cont. • It can be hard to know if you have a breathing problem because you may not notice it until it becomes serious. • Talk to your doctor about available tests to detect a breathing problem.

35. Breathing Therapies How can problems with breathing be treated?, cont. • If your doctor discovers that you have a breathing problem, he or she may recommend that you use an assisted-breathing device, also known as a noninvasive ventilation (NIV) device. • Good evidence shows that using an NIV device will likely lengthen survival and slow the development of breathing problems. • There is weak evidence that shows use of an NIV device will also improve quality of life.

36. Breathing Therapies How can problems with breathing be treated?, cont. • Muscle weakness that affects breathing can also affect the ability to cough. If your cough is weak, you might have trouble clearing mucus and other fluids from your throat, which may lead to a chest infection. • Assisted-coughing devices, also known as mechanical insufflation/exsufflation (MIE) devices, are available to help with coughing. • Weak evidence shows that using an MIE device may help clear fluids from the throat, especially during a chest infection.

37. Conclusions • There has been some progress in evaluating new therapies for people with ALS. • More high-quality studies are available on multidisciplinary clinics, riluzole, breathing and nutrition, and therapies for some symptoms (laughing and crying behaviors, drooling). • Unfortunately, many of these important treatments are not being used to their full potential. • What’s more, there is still a great need for more and better research in many areas.

38. Future Research • Lithium carbonate • Larger studies are in progress on whether lithium slows the rate at which the disease worsens and whether lithium lengthens survival. • Nutrition • Studies are needed on the best timing for inserting a feeding tube and for beginning antioxidant and supplement use.

39. Future Research • Respiratory Management • Evaluation is needed on when to start use of an NIV (breathing) device: impact on quality of life and survival. • Comparison of the best techniques is needed for clearing phlegm and saliva.

40. Future Research • Breaking the News • Measures are needed on how and when to disclose an ALS diagnosis. • Evaluation is needed on neurologists’ and patients’ attitudes toward strategies for breaking the news. • Studies are needed on the effects of different strategies on patient satisfaction, preserving hope, and outcomes. • Multidisciplinary Clinic • Evaluation is needed on factors essential to benefits, best visit frequency, and economic concerns.

41. Future Research • Symptom Management • Studies are needed on medication treatment for spasticity, cramps, constipation, drooling, laughing and crying behaviors, pain, depression, anxiety, fatigue, and exercise. • Behavioral and Thinking Problems • Evaluation is needed on diagnosis and research criteria, screening tests, and the history of and treatments for these problems.

42. Future Research • Communication • Evaluation is needed on communication strategies for people with weakness of their muscles for speech. • Studies are needed that compare different communication strategies. • Palliative Care • Studies are needed on end-of-life symptom management, advanced directives, hospice, and spiritual care.

43. Acknowledgments The authors thank Gary Gronseth, MD; Thomas Getchius; Valerie Cwik, MD; Larry Brower; and Sid Valo for contributions to the practice parameters; Christina Metzler and Barbara Phillips, MS, OTR/L, for their contributions to the patient summary versions of the guidelines; and Sharon J. Matland, RN, MBA, for her contributions to both the practice parameters and the patient summary versions.

44. References For a complete list of references, please access the full guidelines at www.aan.com/guidelines

45. Questions/Comments

46. Thank you for your participation!

47. The ALS Association thanks the American Academy of Neurology and all of today’s presenters and participants.