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

Table 10 MRI Severity (Loes) scores for HSCT versus standard care reported in van Rappard [17]

MLD type	Symptom status	Follow-up (years)	HSCT		Standard care
MLD type	Symptom status	Follow-up (years)	N	Median MRI Loes score (range)*	N	Median MRI Loes score (range)*
LI	NR/unclear	Baseline	2	Median 3 (range 2–3)	6	Median 11 (range 4–17)
LI	NR/unclear	Follow-up^	2	Median 14 (range 8–20)	0	NA
LI	NR/unclear	Baseline	7	Median 6 (range 0–20)	19	Median 15 (range 4–22)
LI	NR/unclear	Follow-up~	7	Median 11 (range 0–25)	4	Median 23 (range 19–26)
J	NR/unclear	Baseline	5	Median 8 (range 0–20)	13	Median 17 (range 12–22)
J	NR/unclear	Follow-up^#	5	Median 10 (range 0–25)	4	Median 23 (range 19–26)

Higher scores indicate a deterioration in neurological function as assessed by MRI; baseline = pre-HSCT or at diagnosis for standard care; follow-up = post-HSCT or at latest assessment for standard care
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, NA not applicable
*Not reported in the paper but calculated from individual patient data reported in the paper
^Average follow-up 35 months (range 10–60)
^~Average follow-up 60 months (range 10–127)
^#Average follow-up 69 months (range 11–127)

ISSN: 1750-1172