Measuring function in advanced ALS: validation of ALSFRS-EX extension items.

Area of Science:

• Neurology
• Rehabilitation Medicine
• Biomedical Engineering

Background:

• Patients with amyotrophic lateral sclerosis (ALS) often require assistive technology, enabling survival beyond traditional functional assessment limits.
• There is a critical need to monitor functional status in advanced ALS, especially for those with severe impairment or locked-in syndrome.

Purpose of the Study:

• To develop and pilot new items for the ALSFRS-R scale to enhance sensitivity in assessing physical function at lower levels.
• To improve the assessment of patients with advanced ALS, particularly those using assistive technology.

Main Methods:

• Utilized an online patient community (PatientsLikeMe) for people with ALS (PALS) to generate new scale items.
• Surveyed PALS with advanced disease to create and pilot potential additions to the ALSFRS-R.
• Collected baseline data from 326 PALS, with follow-up retests at 1 week (n=169) and 3 months (n=218).

Main Results:

• Ten new scale items were generated from patient input.
• The new items were added to the existing ALSFRS-R for data collection.
• Test-retest reliability was assessed over 1 week and 3 months.

Conclusions:

• Three new items were selected that align with the ALSFRS-R's factor structure.
• Selected items assess finger manipulation of devices, facial emotional expression, and indoor mobility.
• Further real-world validation is required to confirm the utility of the new ALSFRS-EX scale.