Abstract
Objective: To investigate whether intravenous enzyme replacement therapy (ERT) benefits cognitive function in patients with mucopolysaccharidosis type IH (Hurler syndrome) undergoing hematopoietic cell transplantation (HCT). Study design: Data were obtained for 9 children treated with HCT + ERT (ERT group) and 10 children treated with HCT only (no-ERT group) from neuropsychologic evaluations before HCT and at 1-year and 2-year post-HCT follow-up. Results: At 2 years after HCT, children in the ERT group lost 9.19 fewer IQ points per year compared with children in the no-ERT group (P = .031). Furthermore, the ERT group improved in nonverbal problem solving and processing, whereas the no-ERT group declined, resulting in a difference of 9.44 points per year between the 2 groups (P < .001). Conclusion: ERT in association with HCT enhances cognitive outcomes, providing new evidence that ERT is a valuable addition to the standard transplantation protocol. Although the mechanism responsible for this improved outcome is unknown, both direct benefits and indirect effects must be considered.
Original language | English (US) |
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Pages (from-to) | 375-380.e1 |
Journal | Journal of Pediatrics |
Volume | 162 |
Issue number | 2 |
DOIs | |
State | Published - Feb 2013 |
Keywords
- CNS
- Central nervous system
- CsA
- Cyclosporine
- ELC
- ERT
- Early Learning Composite
- Enzyme replacement therapy
- HCT
- Hematopoietic cell transplantation
- IV
- Intravenous
- MPS
- Mucopolysaccharidosis