Survey offers look at life, treatment priorities of those living with ALS

Patient-reported measures could improve ALS interventions, scientists say

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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People with amyotrophic lateral sclerosis (ALS) report significant changes in lifestyle after their diagnosis, but still have an acceptable quality of life and low stress levels, according to a survey conducted across nine European countries.

When asked about new drug development, participants most commonly prioritized treatments that slow disease progression or help with breathing or mobility. Also, there may be a need to better educate patients about the scales used to monitor disease progression or certain symptoms.

The study, “IMPACT-ALS: summary of results from a European survey of people living with ALS,” was published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.

The majority of scales used to monitor people with ALS tend to focus more on the clinical aspect of the disease, such as the motor symptoms it causes and how fast the disease is progressing. But the actual patient experience is often left out.

Thus, to better understand the functional burden of ALS and ensure that patient experiences contribute to the development of novel therapies, researchers developed the IMPACT-ALS European survey.

The survey collected the experiences of people living with ALS across nine European countries participating in the European Network to Cure of ALS: United Kingdom, Ireland, Germany, France, Spain, Italy, Belgium, Netherlands, and Sweden.

It involved similar questions from a previous U.S. IMPACT-ALS survey, which were adapted and translated into the main languages of the participating countries.

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Questionnaire covered demographics, treatments, living with ALS

The questionnaire included 36 questions grouped in four sections. The first group of questions collected data regarding demographics such as age, gender, employment, marital status, education, and country of residence. The second and third collected information about the disease and treatments, and the fourth was focused on living with ALS, including recent symptoms, fears, and stress levels.

A total of 857 patients, mostly men (57%), responded to the survey. The majority of responses were from Italy, and participation rates ranged from 0.2%-6.3% based on ALS prevalence in each country. About two-thirds of patients were in the 55-74 age range.

In total, 91% of participants answered questions regarding their symptoms and ALS diagnosis. In half, the first symptoms had appeared two to three years before the survey and the formal diagnosis had been issued 1.5-2 years earlier. The most common first symptom was weakness in the arms or legs (60%), followed by falling and speech problems.

About two-thirds knew which ALS type they had: 58% of patients had sporadic ALS and 6.4% familial ALS, while 35% who did not know their disease form.

Also, while genetic testing is now recommended for all with the neurodegenerative condition, less than half of the respondents underwent such testing. The most frequent mutations were detected in the SOD1 (15%) and C9orf72 (24%) genes.

More than half of patients did not know about the ALS Functional Rating Scale – Revised (ALSFRS-R), the researchers noted. This is a validated measure of disease progression that essentially assesses a person’s ability to perform activities of daily living such as buttoning a shirt, grabbing a pen, turning in bed, or speaking clearly.

For the patients who knew their ALSFRS-R score, the median score was higher — indicating less severe disease and a greater ability to perform daily functions — among patients with the most recent diagnosis than in those with a longer-established diagnosis (37 vs. 31).

ALS symptoms included fatigue, speech issues, depression

In the two weeks preceding the survey, the most common symptoms were limb weakness, fatigue, and speech problems. Other symptoms such as pain, depression or mood changes, apathy, and thinking difficulties were also present in at least 1 in 10 patients.

Patients also often reported issues with physical activities in that period, such as trouble walking (67%), difficulty managing buttons or keys (64%), or problems rising from a chair (62%).

Still, most patients reported low to medium levels of stress in the two weeks prior to the survey and moderate quality of life in the two days prior.

There were certain fears related to the future in about 82% of patients, although fear was less common in people diagnosed for longer (75.3%). The most common fears were leaving family too soon, dying from respiratory failure, having an injury, needing invasive ventilation, or experiencing social isolation due to communication impairments.

Participants also reported changes in their life since the diagnosis. In general, they spent less time on most daily activities, and more on the internet, reading, and communicating with family and friends.

Treatment options detailed

In terms of treatment, 12% required a feeding tube and 22% needed noninvasive ventilatory support. For mobility, more than half attended physical therapy, about one-third did regular exercise, and some required manual or motorized wheelchairs or wheeled walkers.

When asked about what they valued more about current and new treatments, most patients wanted therapies that could stop disease progression. Other treatments that were highly sought were those that could improve breathing and mobility.

“To enable the delivery of care that meets the needs of [people with ALS], further work is required to focus on commonly reported symptoms as well as those symptoms which patients want treating the most,” the researchers wrote. “The findings we report from this survey support the increased use of [patient-reported outcome measures] in clinical trials to promote the development of patient focused interventions to improve quality of life.”