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.
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: Bahareh Ajami; Jami L Bennett; Charles Krieger; Wolfram Tetzlaff; Fabio M V Rossi Journal: Nat Neurosci Date: 2007-11-18 Impact factor: 24.884
Authors: P J Orchard; C Milla; E Braunlin; T DeFor; K Bjoraker; B R Blazar; C Peters; J Wagner; J Tolar Journal: Bone Marrow Transplant Date: 2009-11-09 Impact factor: 5.483
Authors: J Cox-Brinkman; J-J Boelens; J E Wraith; A O'meara; P Veys; F A Wijburg; N Wulffraat; R F Wynn Journal: Bone Marrow Transplant Date: 2006-05-22 Impact factor: 5.483
Authors: Su Han Lum; Karolina M Stepien; Arunabha Ghosh; Alexander Broomfield; Heather Church; Jean Mercer; Simon Jones; Robert Wynn Journal: J Inherit Metab Dis Date: 2017-03-10 Impact factor: 4.982
Authors: Elsa G Shapiro; Igor Nestrasil; Kyle Rudser; Kathleen Delaney; Victor Kovac; Alia Ahmed; Brianna Yund; Paul J Orchard; Julie Eisengart; Gregory R Niklason; Julian Raiman; Eva Mamak; Morton J Cowan; Mara Bailey-Olson; Paul Harmatz; Suma P Shankar; Stephanie Cagle; Nadia Ali; Robert D Steiner; Jeffrey Wozniak; Kelvin O Lim; Chester B Whitley Journal: Mol Genet Metab Date: 2015-06-17 Impact factor: 4.797
Authors: Agnes H Chen; Paul Harmatz; Igor Nestrasil; Julie B Eisengart; Kelly E King; Kyle Rudser; Alexander M Kaizer; Alena Svatkova; Amy Wakumoto; Steven Q Le; Jacqueline Madden; Sarah Young; Haoyue Zhang; Lynda E Polgreen; Patricia I Dickson Journal: Mol Genet Metab Date: 2019-11-30 Impact factor: 4.797
Authors: Arunabha Ghosh; Weston Miller; Paul J Orchard; Simon A Jones; Jean Mercer; Heather J Church; Karen Tylee; Troy Lund; Brian W Bigger; Jakub Tolar; Robert F Wynn Journal: Mol Genet Metab Date: 2016-01-27 Impact factor: 4.797
Authors: A S Kunin-Batson; E G Shapiro; K D Rudser; C A Lavery; K J Bjoraker; S A Jones; R F Wynn; A Vellodi; J Tolar; P J Orchard; J E Wraith Journal: JIMD Rep Date: 2016-01-30