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

Table 7 NCV 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	Mean NCV index	N	Mean ncv index	Comparison between OTL and standard care
Total NCV score
LI	Pre-symp	2	9	− 7.6	10	− 13.3	Treatment difference 5.8 (95% CI 2.4–9.1	p = 0.004 in favour of Atidarsagene
LI	Pre-symp	3	6	− 8.3	10	− 11.5	Treatment difference 3.2 (95% CI 1.0–5.3)	p = 0.010 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 ML, LI late infantile, LS least squares, MD mean difference, MLD metachromatic leukodystrophy, mth month, N total number analysed, pre-symp pre-symptomatic

ISSN: 1750-1172