Table 6 Gross motor function (GMFM-MLD score or GMFC-MLD) for HSCT versus standard care
Treatment | MLD type | Symptom status | Time point | Outcome definition | n/N (%) | Source |
|---|---|---|---|---|---|---|
HSCT | LI | Mixed | Last available follow-for each patient^ | No. with worsening GMFM score | 2/2 (100%) | Boucher [21]—R |
LI | NR/unclear | Last available follow-for each patient^ | No. with worsening GMFM score | 2/2 (100%) | van Rappard [17]—R | |
LI to J | Mixed | Last available follow-for each patient^ | No. with worsening GMFM score | 13/16 (81.25%) | Boucher [21]—R | |
J | NR/unclear | Last available follow-for each patient^ | No. with worsening GMFM score | 2/5 (40%) | van Rappard [17]—R | |
J | Mixed | Last available follow-for each patient^ | No. with worsening GMFM score | 11/14 (78.6%) | Boucher [21]—R | |
J | NR/unclear | Last available follow-for each patient^ | No. with no change in GMFM score | 3/14 (21.4%) | van Rappard [17]—R | |
J | Mixed | 10 years** | Progression to GMFC-MLD level 5a | 8/20 (40%) | Groeschel [16]—R* | |
Standard care | LI | NR/unclear | Last available follow-for each patient^ | No. with no change in GMFM score | 2/6 (33.3%) | van Rappard [17]—R |
LI | NR/unclear | Last available follow-for each patient^ | No. with worsening GMFM score | 4/6 (66.7%) | van Rappard [17]—R | |
J | NR/unclear | Last available follow-for each patient^ | No. with no change in GMFM score | 12/12 (100%) | van Rappard [17]—R | |
J | Mixed | 10 years** | Progression to GMFC-MLD level 5a | 28/41 (68.29%) | Groeschel[16]—R* |