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Table 8 NCV for HSCT patients

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

MLD type

Symptom status

Follow-up timepoint after HSCT

Outcome definition

n/N (%)

Source

LI

Mixed

NR

No. with worsening NCV^

1/3 (33.3%)~

Boucher [21]—R

LI

Mixed

NR

No. with worsening NCV*

5/8 (62.5%)

Martin [23]—R

J

Mixed

NR

No. with worsening NCV^

10/15 (66.7%)~

Boucher [21]—R

J

Mixed

NR

No. with worsening NCV *

4/12 (33.3%)

Martin [23]—R

LI

Mixed

NR

No. with stabilised NCV^

2/3 (66.7%)~

Boucher [21]—R

LI

Mixed

NR

No. with NCV stabilised*^

3/8 (37.5%)

Martin [23]—R

J

Mixed

NR

No. with stabilised NCV^

5/15 (33.3%)~

Boucher [21]—R

J

Mixed

NR

No. with NCV stabilised*^

8/12 (66.7%)

Martin [23]—R

  1. LI late infantile MLD, J juvenile MLD, HSCT haemopoietic stem cell transplantation, MLD metachromatic leukodystrophy, NCV nerve conduction velocity, n number of patients with the outcome, N total number of patients assessed, NR not reported, P prospective, R retrospective
  2. ^Not defined
  3. ~Data were only reported in a figure from these results were calculated