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Table 3 Top 5 most frequently reported important criteria to choose 'YES' or 'NO' for HSCT

From: Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

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
  1. 1 Also stated as 'low IQ' or 'CNS-involvement'.