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

Table 9 MRI Severity (Loes) scores for Atidarsagene versus standard care in Fumagalli et al. [13]

MLD type	Symptom status	Follow-up (years)	Atidarsagene		Standard care		Comparison between atidarsagene and standard care
MLD type	Symptom status	Follow-up (years)	N	LS mean change in MRI Loes from baseline (SD)*	N	LS mean change in MRI Loes from baseline (SD)*	Comparison between atidarsagene and standard care
LI	Pre-symp	2	9	2.4	15	15.3	MD − 12.9 (95% CI − 16.2 to − 9.7)	p < 0.001 in favour of Atidarsagene versus standard care control
LI	Pre-symp	3	8	3.6	9	21.7	MD -18.1 (95% CI − 21.1 to − 15.0)	p < 0.001 in favour of Atidarsagene versus standard care control
EJ	Mixed	2	10	9.4	11	17.9	MD − 8.5 (95% CI − 14.7 to − 2.3)	p = 0.010
EJ	Mixed	3	9	10.1	12	20.5	MD − 10.4 (95% CI − 17.0 to − 3.8)	p = 0.004 in favour of Atidarsagene versus standard care control

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, LI late infantile, LS least squares, MD mean difference, MLD metachromatic leukodystrophy, mth month, pre-symp pre-symptomatic, N total number analysed
*LS mean calculated using ANCOVA model fitting age and treatment (Atidarsagene or NHx). Age was fitted in mths for the LI group

ISSN: 1750-1172