A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel

Table 5 Gross motor function using GMFM-88 for Atidarsagene versus standard care in Fumagalli et al. [13]

MLD type	Symptom status	Follow-up (years)	Atidarsagene		Standard care		Comparison between OTL and standard care
MLD type	Symptom status	Follow-up (years)	N	% GMFM-88 total score	N	% GMFM-88 total score	Comparison between OTL and standard care
LI	Pre-symp	2	11	73.1	9	7.6	65.6% (95% CI 48.9–82.3)	p < 0.001 in favour of Atidarsagene *
LI	Pre-symp	3	10	74.3	12	2.8	71.5% (95% CI 50.3–92.7)	p < 0.001 in favour of Atidarsagene*
EJ	Mixed	2	10	78.7	11	36.7	42.0% (95% CI 12.3–71.8)	p = 0.036 in favour of Atidarsagene*
EJ	Mixed	3	10	72.9	12	16.3	56.7% (95% CI 33.7%–79.6)	p < 0.001 in favour of Atidarsagene*

Mixed refers to populations with a mixture of pre-symptomatic and symptomatic patients
ANCOVA analysis of covariance model, CI confidence interval, EJ early juvenile MLD, GMFM-88 gross motor function measure-88 items, LI late infantile, LS least squares, MD mean difference, MLD metachromatic leukodystrophy; mth month; N total number analysed; pre-symp pre-symptomatic
GMFM-88 consists of 88 questions organised into 5 domains: (a) lying and rolling; (b) sitting; (c) crawling and kneeling; (d) standing; and (e) walking, running, and jumping. Each of the 88 questions is scored from 0 to 3 (maximum number of points = 264). Scores of each dimension are expressed as a percentage of the maximum score for that dimension, and a total score is obtained by averaging the percentage scores across the 5 dimensions, with 0% corresponding to loss of all voluntary movement. The GMFM score is also related to age; by the age of 60 months, most healthy children will achieve their maximum score, approximating 100%
*GMFM-88 data at Year 2 and 3 were analysed using an ANCOVA model fitting age and treatment (Atidarsagene or NHx) and testing the null hypothesis that the difference was 10%. Age was fitted in months for the LI group

ISSN: 1750-1172