Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome.

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.

• Authors: Julie B Eisengart, Kyle D Rudser, Jakub Tolar, Paul J Orchard, Teresa Kivisto, Richard S Ziegler, Chester B Whitley, Elsa G Shapiro
• Journal: The Journal of pediatrics (J Pediatr) Vol. 162 Issue 2 Pg. 375-80.e1 (Feb 2013) ISSN: 1097-6833 [Electronic] United States
• PMID: 22974573 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2013 Mosby, Inc. All rights reserved.

Topics

• Cognition
• Enzyme Replacement Therapy
• Female
• Hematopoietic Stem Cell Transplantation
• Humans
• Infant
• Male
• Mucopolysaccharidosis I (surgery)
• Prospective Studies
• Treatment Outcome