Table 3 Top 5 most frequently reported important criteria to choose 'YES' or 'NO' for HSCT
Important for 'YES' for HSCT |
|---|
1. Developmental delay1 |
2. Young age |
3. Severe phenotype |
4. Kyphosis |
5. Likely to benefit |
Important for 'NO' for HSCT |
1. Milder phenotype |
2. Older age |
3. Normal development |
4. Developmental delay1 |
5. Mainly joint stiffness/late diagnosis |