ALS patients differ on treatment choices in later phases of disease
Two new studies analyzing treatment decisions in late-stage amyotrophic lateral sclerosis (ALS) patients shed light onto treatments aimed to extend the duration and quality of life in this progressively debilitating neuromuscular disorder. Researchers from the Perelman School of Medicine at the University of Pennsylvania found that waiting until the last minute to receive one treatment resulted in not living long enough to experience the benefits. In a separate study, Penn researchers uncovered polarized preferences among patients regarding the value of an expensive, marginally effective disease-modifying drug. The research will be presented at the American Academy of Neurology’s 64th Annual Meeting in New Orleans.
ALS, commonly known as Lou Gehrig’s disease, is rare, affecting approximately 30,000 Americans. In later stages of the disease, it paralyzes ALS patients’ bodies, while minds often stay sharp. Along with other treatments and supportive therapies used in later stages of the disease, many patients receive a feeding tube to ensure nourishment can be obtained when muscles are impaired.
One Penn Medicine study demonstrates that ALS patients who have feeding tubes placed before an emergency situation strikes fare better. Those having surgeries in non-emergent settings were much less likely to die within one month after surgery, compared to ALS patients receiving their feeding tubes under duress. Median survival after the feeding tube surgery was 6 months overall and longer for patients undergoing non-emergent versus emergent placement (7 months vs. 4 months). In addition, mortality rates were worse for patients having procedures done at hospitals that did not regularly perform feeding tubes placement in ALS patients.
“Timing is crucial for placement of feeding tubes in ALS patients,” said the lead author of both studies, Amy Tsou, MD, MSc, a fellow in Neurology and a Robert Wood Johnson VA Clinical Scholar. “We’ve shown that waiting too long can be detrimental and happens too often. In general, it is important for clinicians and patients to proactively prepare and reevaluate treatment decisions as ALS patients enter into different phases of the disease.”
In a second study, researchers found polarized treatment preferences regarding Riluzole, the first FDA approved treatment to slow ALS. Patients had sharply polarized preferences about this expensive treatment, which modestly prolongs length of life of ALS patients. In a survey of 98 patients with ALS or Motor Neuron Disease, nearly two-thirds of the patients ranked Riluzole as either the most important (30 percent) or least important (33 percent) treatment option.
What is ALS?
Amyotrophic lateral sclerosis (ALS), often referred to as “Lou Gehrig’s Disease,” is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed.
A-myo-trophic comes from the Greek language. “A” means no or negative. “Myo” refers to muscle, and “Trophic” means nourishment–“No muscle nourishment.” When a muscle has no nourishment, it “atrophies” or wastes away. “Lateral” identifies the areas in a person’s spinal cord where portions of the nerve cells that signal and control the muscles are located. As this area degenerates it leads to scarring or hardening (“sclerosis”) in the region.
As motor neurons degenerate, they can no longer send impulses to the muscle fibers that normally result in muscle movement. Early symptoms of ALS often include increasing muscle weakness, especially involving the arms and legs, speech, swallowing or breathing. When muscles no longer receive the messages from the motor neurons that they require to function, the muscles begin to atrophy (become smaller). Limbs begin to look “thinner” as muscle tissue atrophies.
“It is important to ask patients how they value their treatments, as in this case, we learned that patients who are older and looking for a high quality of life, valued this drug considerably more than people with impaired walking ability, who instead preferred supportive therapies like adaptive equipment,” said Leo McCluskey, MD, professor of Neurology and director of the Penn ALS Center. “Overall, medical care providers should work with patients to discuss treatment options throughout the progression of the disease to ensure a high quality of end-of-life care.”
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The two posters will be presented during the American Academy of Neurology’s 64th Annual Meeting:
What are the symptoms?
The first sign of ALS is often weakness in one leg, one hand, the face, or the tongue. The weakness slowly spreads to both arms and both legs. This happens because as the motor neurons slowly die, they stop sending signals to the muscles. So the muscles don’t have anything telling them to move. Over time, with no signals from the motor neurons telling the muscles to move, the muscles get weaker and smaller.
Over time, ALS also causes:
- Muscle twitching.
- Trouble using your hands and fingers to do tasks.
- Problems with speaking, swallowing, eating, walking, and breathing.
ALS doesn’t cause numbness, tingling, or loss of feeling.
Respiratory problems are the most common serious complication of ALS. As the muscles in the throat and chest weaken, swallowing, coughing, and breathing problems tend to get worse. Pneumonia, Pulmonary embolism, lung failure, and heart failure (probably caused by weak breathing) are the most common causes of death.
How is ALS diagnosed?
It can be hard for your doctor to tell if you have ALS. It may not be clear that you have the disease until symptoms get worse or until your doctor has done more testing. To find out if you have ALS, your doctor will do a physical exam and will ask you about your symptoms and past health. You will also have tests that show how your muscles and nerves are working.
Monday, April 23, 2012 2:00 PM: [P01.102] Predictors of 30 Day Mortality after Gastrostomy in Amyotrophic Lateral Sclerosis Amy Tsou, Judith Long, Leo McCluskey, Philadelphia, PA, Lauren Elman, Jason Karlawish, Philadelphia, PA
Tuesday, April 24, 2012 2:00 PM: [P03.173] Measuring Value: Patient Preferences for Riluzole in Amyotrophic Lateral Sclerosis (ALS) Amy Tsou, Judith Long, Leo McCluskey, Philadelphia, PA, Lauren Elman, Katelin Hoskins, Mark Cary, Jason Karlawish, Philadelphia, PA
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Kim Menard
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University of Pennsylvania School of Medicine