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Table 4 Top 5 most frequently reported complicating issues 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

Complicating for 'YES' for HSCT
1. Developmental delay1
2. Older age
3. Cardiomyopathy
4. ERT prior to HSCT desirable
5. Uncertain (CNS) prognosis
Complicating for 'NO' for HSCT
1. ERT prior to HSCT might improve symptoms
2. Developmental delay1
3. Long-term course unknown
4. Unknown role for HSCT in milder phenotype
5. Early diagnosis
  1. 1 Also stated as 'low IQ' or 'CNS-involvement'.