Remote digital assessment of amyotrophic lateral sclerosis functional rating scale – a multicenter observational study

Figures & data

Figure 1 Sample characteristics. To evaluate the feasibility of remote digital assessment the ALSFRS-R, the total number of patients receiving treatment at the participating ALS centers was of interest. Therefore, an estimate of these patients was made – at the beginning of the observation, added by the patients who annually entered treatment at these centers (estimated multicenter cohort). Only a subpopulation of the multicenter cohort fulfilled the inclusion criteria (below) and contributed to this observational study (total study cohort). Among the total study cohort, two main sub-cohorts emerged: The first cohort included patients who allowed the secondary use of the existing data of clinical assessment of the ALSFRS-R (Clinic-ALSFRS-R cohort) but did not complete a rating via a computer or the mobile app. For this cohort, demographic and clinical data were collected and the Clinic-ALSFRS-R data were analyzed. The second main sub-cohort included patients who performed a remote digital assessment (Remote-ALSFRS-R cohort), either on a computer (Co-ALSFRS-R cohort) or on a mobile application (App-ALSFRS-R cohort), or on both. In a distinct cohort, other persons acted as patients’ deputy and realized the rating (PD-ALSFRS-R). Within the PD-ALSFRS-R cohort, no distinction was made between computer or mobile app use. Therefore, preferences of relatives regarding computer or app use were not analyzed. n: number of patients.

Table 1 Demographic and clinical characteristics of participants.

Characteristics	Classification	Total cohort, n = 1,893	Clinic-ALSFRS-R cohort^a, n = 922	Remote-ALSFRS-R cohort^b, n = 971	p-value^c
Sex	female, % (n)	39.2 (743)	42.6 (393)	36.0 (350)	0.004
	male, % (n)	60.8 (1,150)	57.4 (529)	64.0 (621)
Age	at onset, years, mean (SD, R)	60.1 (12.1, 18.2–88.1)	61.2 (12.6, 18.2-88.1)	58.6 (11.1, 25.4–87.3)	<0.001
	at time of first assessment, years, mean (SD, R)	62.8 (11.5, 19.7–90.2)	64.0 (12.4, 19.7-90.2)	61.7 (10.5, 26.7–88.2)	<0.001
	at time of last assessment, years, mean (SD, R)	64.5 (11.4, 20.2–95.8)	66.6 (12.0, 20.2-95.8)	62.5 (10.5, 27.0–88.2)	<0.001
Disease duration	at time of last assessment, months, mean (SD, R)	53.5 (52.3, 2–472)	61.9 (58.5, 2-472)	41.9 (39.5, 2–240)	<0.001
Disease progression	mean (SD, R)	0.69 (0.64, 0.01–6.00)	0.64 (0.62, 0.01–6.00)	0.74 (0.66, 0.01–4.75)	0.007
King's clinical stage^d	Stage 1, % (n)	12.1 (229)	10.7 (99)	13.4 (130)	0.234
	Stage 2, % (n)	20.6 (390)	20.6 (190)	20.6 (200)
	Stage 3, % (n)	35.1 (665)	36.0 (332)	34.3 (333)
	Stage 4, % (n)	32.3 (609)	32.6 (301)	31.7 (308)

^aEncompasses patients with assessment of the ALSFRS-R during clinic visits (Clinic-ALSFRS-R).

^bEncompasses patients with remote digital assessment of the ALSFRS-R (Remote-ALSFRS-R cohort). Remote assessment of the ALSFRS-R was performed using a computer (Co-ALSFRS-R) or the mobile “ALS-App” (App-ALSFRS-R).

^cDifference in frequencies between different groups were assessed by Chi-squared test or Mann-Whitney U test and between-metric data by t-test, a p value < 0.05 was considered significant. Significant differences were compared between the Clinic-ALSFRS-R cohort and Remote-ALSFRS-R cohort.

^dFour cohorts were differentiated according to King's clinical stage that was reached by any given patient. King's clinical stage 1 translated to the involvement of one clinical region. Stage 2 and 3 was reached with the involvement of a second and third clinical region, respectively. Stage 4 referred to nutritional or respiratory failure, as defined by the use of percutaneous endoscopic gastrostomy (PEG) or ventilation therapy (noninvasive or invasive), respectively.(24).

Abbreviations: n: number of participants; SD: standard deviation; R: range; ALSFRS-R: Amyotrophic Lateral Sclerosis Functional Rating Scale Revised.

Table 2 Number of patients with assessments and assessments per patient per year.

	Total number of assessments of ALSFRS-R	Number of patients with ALSFRS-R assessments	Number of patients with at least two ALSFRS-R assessments	Number of patients with at least four ALSFRS-R assessments per year	Observation period in years^a	Frequency of ALSFRS-R assessments per atient per year^a
	n	n (%)	n (%)	n (%)	Mean (SD, R)	Mean (SD, R)
Clinic-ALSFRS-R	4,626 (50.7)	922 (48.5)	648 (70.3)	430 (46.6)	1.4 (0.9, 0.1–3.3)	4.1 (2.4, 1.3–24.0)
Remote-ALSFRS-R	4,506 (49.3)	971 (51.3)	620 (63.8)	521 (53.7)	1.1 (1.0, 0.1–4.3)	9.6 (8.3, 0.8–96.0)
Co-ALSFRS-R	3,288 (36.0)	857 (45.3)	502 (58.6)	407 (47.5)	1.1 (0.9, 0.1–4.3)	7.9 (7.6, 0.8–96.0)
App-ALSFRS-R	1,127 (12.3)	234 (12.4)	147 (62.8)	139 (59.4)	0.6 (0.4, 0.1–1.5)	14.6 (9.0, 2.2–48.0)
PD-ALSFRS-R	91 (1.0)	36 (1.9)	17 (47.2)	12 (33.3)	0.8 (0.9, 0.1–2.9)	10.7 (9.9, 1.6–36.0)
Total	9,132 (100)	1,893 (100)	1,268 (67.0)	951 (50.2)	1.2 (0.7, 0.1–4.3)	6.8 (6.7, 0.8–96.0)

The assessment periods differed between studied cohorts: Clinic-ALSFRS-R from August 2017 to December 2020; Remote-ALSFRS-R: August 2017 to December 2021, Co-ALSFRS-R: August 2017 to December 2021; App-ALSFRS-R: May 2020 to December 2021, PD-ALSFRS-R: August 2017 to November 2021.

Abbreviations: n: number of patients; ALSFRS-R: ALS Functional Ratings Scale Revised; Clinic-ALSFRS-R: assessment of ALSFRS-R during clinic visits; Remote-ALSFRS-R: remote digital assessment of the ALSFRS-R; Co-ALSFRS-R: computer ALSFRS-R; PD-ALSFRS-R: patients’ deputy ALSFRS-R; SD: standard deviation; R: range.

^apatients with at least two assessments were included.

Table 3 Results of ALSFRS-R ratings in relation to type of assessment.

	Number of patients with ALSFRS-R assessments^a, n (%)	ALSFRS-R score of first assessment^a, mean (SD, R)	ALSFRS-R score of last assessment^a, mean (SD, R)
Clinic-ALSFRS-R	648 (51.1)	31.7 (12.0, 0–48)	25.2 (11.9, 0–48)
Remote-ALSFRS-R	620 (48.9)	36.2 (8.6, 1–48)	27.7 (10.5, 0–47)
Co-ALSFRS-R	502 (39.6)	36.2 (8.5, 1–48)	27.8 (10.5, 0–48)
App-ALSFRS-R	147 (11.6)	35.9 (8.2, 3–48)	30.5 (9.8, 4–47)
PD-ALSFRS-R	17 (1.9)	30.8 (10.2, 14–45)	20.4 (8.3, 6–38)

The assessment periods differed between studied cohorts: Clinic-ALSFRS-R from August 2017 to December 2020; Remote-ALSFRS-R: August 2017 to December 2021, Co-ALSFRS-R: August 2017 to December 2021; App-ALSFRS-R: May 2020 to December 2021, PD-ALSFRS-R: August 2017 to November 2021.

Abbreviations: n: number of patients; ALSFRS-R: ALS Functional Ratings Scale Revised; Clinic-ALSFRS-R: assessment of ALSFRS-R during clinic visits; Remote-ALSFRS-R: remote digital assessment of the ALSFRS-R; Co-ALSFRS-R: computer ALSFRS-R; PD-ALSFRS-R: Patients’ deputy ALSFRS-R; SD: standard deviation; R: range.

^aPatients with at least two assessments were included.

Figure 2 Frequency of ALSFRS-R ratings per year. Frequency of ALSFRS-R were obtained in the cohorts of clinical assessment of the ALSFRS-R (Clinic-ALSFRS-R) as compared to remote self-assessment using a computer (Co-ALSFRS-R) or mobile application (App-ALSFRS-R), or both. Significant differences were assessed by t-test. A p-value <0.05 was considered significant. Abbreviations: n: number of patients; ALSFRS-R: ALS Functional Ratings Scale Revised.

Balendra R, Jones A, Jivraj N, Knights C, Ellis CM, Burman R, et al. Estimating clinical stage of amyotrophic lateral sclerosis from the ALS functional rating scale. Amyotroph Lateral Scler Frontotemporal Degener. 2014; 15:279–84.

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Data availability statement

The data that support the findings of this study are available from the corresponding author upon reasonable request.