Table 3 Overview of current practices countries where HSCT for MLD is available
N (%) | |
|---|---|
Total number of countries | 23 (100%) |
Centralization of HSCT care | |
No | 6 (22%) |
1 center | 11 (48%) |
2 centers | 3 (13%) |
3 centers | 4 (17%) |
Referral by (pediatric) neurologist | 21 91%) |
Urgency of transplantation | |
Never | 3 (13%) |
In all cases* | 10(43%) |
Only in near-symptomatic patients* | 11(48%) |
Routinely testing of siblings | |
No | 1 (4%) |
All siblings | 19 (83%) |
Only younger siblings | 1 (4%) |
Other/no established routine | 2 (9%) |
Centralized clinical decision-making in one or two centers | 9 (39%) |
Eligible MLD subtypes | |
Late-infantile | 9(39%) |
Early-juvenile | 19(82%) |
Late-juvenile | 18(78%) |
Adult | 14(61%) |
Aspects that are taken into account | |
Severity of symptoms | 22 (96%) |
Donor availability | 17 (74%) |
Total IQ as criterion | |
No | 7 (30%) |
> 60 | 1 (4%) |
> 70 | 3 (13%) |
> 75 | 1 (4%) |
80 | 4 (17%) |
85 | 6 (26%) |
> 90 | 2 (9%) |
GMFC-MLD as criterion | |
No | 9 (39%) |
1 | 4 (17%) |
< 2 | 10 (43%) |
< 3 | 2 (9%) |
MLD MRI severity score as criterion | |
No | 9 (39%) |
< 7 | 5 (22%) |
< 17 | 9 (39%) |
Total number of units^ | 20 (100%) |
Preferred source of CD34 + cells | |
BM | 8 (40%) |
BM; UCB | 3 (15%) |
BM, UCB, PBSC | 1 (5%) |
BM; PBSC | 2 (10%) |
UCB | 3 (15%) |
PBSC | 1 (5%) |
Other | 2 (10%) |
Conditioning regimen | |
Bu-Flu | 9 (45%) |
Bu-Cy | 2 (10%) |
Treosulfan-based | 4 (20% |
Reduced intensity | 2 (10%) |
Other | 1 (5%) |
Screening for carrier status in familial donors | 15 (75%) |