Skip to main content

Table 4 Quality of life measures and cognitive screening measures in ALS

From: Qualitative measures that assess functional disability and quality of life in ALS

Tool

Description

Strengths

Limitations

Recommendations for best use in a clinical trial

ALSAQ-40 [52, 53]

40-item disease specific HRQoL. Many items similar to ALSFRS-R

A validated ALS-specific HRQoL measure developed in accordance with FDA PRO guidance

40 items may be too impractical for use in a clinical trial due to increased patient burden

Use as secondary endpoint

Use in addition to a function measure to provide extra sensitivity in more severe disease

ALSAQ-5 [54]

5-item measure generated from the ALSAQ-40

Provides a quick measure of HRQoL with minimal patient burden

Shorter instrument lacks measurement precision of larger measures

Use when HRQoL is an exploratory endpoint

May be a valid alternative to the ALSAQ-40 (i.e., reduced patient burden, costs)

ALSSQOL-20 (ALSSQOL-SF) [55]

20-item disease specific measure of global QOL reduced from the larger ALSQOL

Short administration time suitable for clinical use

Use is better in clinical care

Focus is more on global QoL than HRQoL

Only 2 bulbar items

Use in an interventional study to determine treatment effect if combined with an HRQoL measure

EQ-5D

Self-report [56,57,58]

5-items on mobility, self-care, usual activities, pain/discomfort, anxiety/depression plus a self-report of current health

Quick, easy to complete

Well validated measure very commonly used in clinical trials

Quick snapshot of patient’s current state of health but not specific to ALS

Use when a health economics outcome is required or if general HRQoL is a secondary or exploratory endpoint

WHOQOL-BREF [59]

Recently validated, self-reported 26-item generic measure of QoL. Domains include physical health, social relationships, and environment

Quick, easy to complete

High compliance reported in ALS

Available in 19 different languages

High reliability and construct validity

Enables comparisons between ALS conditions and general population

Interval level measurement (Rasch)

Social domain showed an unsatisfactory fit to the Rasch model

Generic measure, may not capture QoL items specific to ALS patients

Limited use data in ALS patients

Limited longitudinal validity data

Use for parametric analysis and for comparison with other conditions or general populations

Neuro-QoL [24]

Short form measures of functioning, ADLS

Self/proxy report, 6–8 items

Very well developed and validated function and ADL measures (FDA PRO guidance) in a population of patients with neurodegenerative diseases including ALS

Not feasible to include all the individual measures to assess overall functioning

Limited validity data in ALS specific populations

Another HRQoL option to the ALSAQ-20

Select the appropriate function / symptom measure for highest treatment impact

Secondary or exploratory measure of HRQOL to supplement a primary function measure

PROMIS-10

Global Health [24, 60]

Self-reported short form generic Global Health measure of mental and physical health

Well developed and validated

Simple, easy to administer

Provides mental and physical health scores

May be too general for use in an ALS population when other disease specific HRQoL measures exist

Limited validity data in ALS specific populations

Use for quick global measure of HRQoL rather than disease specific for ALS (secondary or exploratory measure)

Can be used in collaboration with NeuroQoL measures (fatigue)

ALS Cognitive Behavior Screen (ALS-CBS) [63,64,65]

Clinician assessed, 10-item cognitive section and 18-item, caregiver-rated behavioral section to identify patients who have cognitive or behavioral changes suggestive of frontal temporal dementia (FTD)

Validated against neuropsychological tests

Can distinguish between cognitively impaired and non-impaired patients

Accommodates decline in motor functioning

Caregiver/self-reported behavior section

Administration time < 10 min

Requires health professional for assessment of cognitive section which incurs added expense

Test–retest not established

Does not assess language or social cognition

Behavioral component may not capture mild behavioral change

Use as initial screening of cognitive or behavioral changes within the population that might affect assessment of disease severity and progression and potentially confound response to therapy

Edinburgh Cognitive and Behavioral ALS Screen (ECAS) [61, 66,67,68]

ECAS-cognitive screen comprises 16 items divided into ALS-specific subscale and a non-ALS-specific subscale. ECAS-behavioral screen includes 5 domains of behavior

Validated against a battery of neuropsychological tests

Can provide early identification of cognitive and behavioral changes

Accommodates decline in motor functioning

Assesses language and social cognition, may be suitable for patients with bulbar disability

Guidelines prefer that a neuropsychologist administer or supervise ECAS administration (added expense)

45 min + administration time

Behavioral component requires interviewing the caregiver

May not be feasible in clinical trials due to increased patient burden

Use for screening cognitive or behavioral changes that might affect assessment of ALS disease severity and progression and potentially confound response to therapy

Use when decline in functioning in more severe ALS patients may be confounded by neuropsychological impairment that could be related to FTD