Literature DB >> 19187736

Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.

Robert F Wynn1, Jean Mercer, Joanne Page, Trevor F Carr, Simon Jones, J Edmond Wraith.   

Abstract

We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning.

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Year:  2009        PMID： 19187736     DOI： 10.1016/j.jpeds.2008.07.004

Source DB:  PubMed          Journal:  J Pediatr        ISSN： 0022-3476            Impact factor:   4.406


  23 in total

1.  Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.

Authors:  Muhammad Ameer Saif; Brian W Bigger; Karen E Brookes; Jean Mercer; Karen L Tylee; Heather J Church; Denise K Bonney; Simon Jones; J Ed Wraith; Robert F Wynn
Journal:  Haematologica       Date:  2012-02-27       Impact factor: 9.941

2.  Enzyme replacement in neuronal storage disorders in the pediatric population.

Authors:  Erika F Augustine; Jonathan W Mink
Journal:  Curr Treat Options Neurol       Date:  2013-10       Impact factor: 3.598

3.  Combined Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation in Mucopolysacharidosis Type VI.

Authors:  D Sillence; K Waters; S Donaldson; P J Shaw; C Ellaway
Journal:  JIMD Rep       Date:  2011-09-06

Review 4.  Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.

Authors:  Madeleine Taylor; Shaukat Khan; Molly Stapleton; Jianmin Wang; Jing Chen; Robert Wynn; Hiromasa Yabe; Yasutsugu Chinen; Jaap Jan Boelens; Robert W Mason; Francyne Kubaski; Dafne D G Horovitz; Anneliese L Barth; Marta Serafini; Maria Ester Bernardo; Hironori Kobayashi; Kenji E Orii; Yasuyuki Suzuki; Tadao Orii; Shunji Tomatsu
Journal:  Biol Blood Marrow Transplant       Date:  2019-02-14       Impact factor: 5.742

Review 5.  Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.

Authors:  R Kohan; I A Cismondi; A M Oller-Ramirez; N Guelbert; Tapia V Anzolini; G Alonso; S E Mole; Dodelson R de Kremer; Noher I de Halac
Journal:  Curr Pharm Biotechnol       Date:  2011-06       Impact factor: 2.837

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

Authors:  Julie B Eisengart; Kyle D Rudser; Jakub Tolar; Paul J Orchard; Teresa Kivisto; Richard S Ziegler; Chester B Whitley; Elsa G Shapiro
Journal:  J Pediatr       Date:  2012-09-10       Impact factor: 4.406

7.  Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.

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

8.  Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice.

Authors:  Alexander Langford-Smith; Fiona L Wilkinson; Kia J Langford-Smith; Rebecca J Holley; Ana Sergijenko; Steven J Howe; William R Bennett; Simon A Jones; Je Wraith; Catherine Lr Merry; Robert F Wynn; Brian W Bigger
Journal:  Mol Ther       Date:  2012-05-01       Impact factor: 11.454

Review 9.  Therapeutic liver repopulation for phenylketonuria.

Authors:  Cary O Harding; K M Gibson
Journal:  J Inherit Metab Dis       Date:  2010-05-22       Impact factor: 4.982

10.  Receiving enzyme replacement therapy for a lysosomal storage disorder: a preliminary exploration of the experiences of young patients and their families.

Authors:  R Freedman; M Sahhar; L Curnow; J Lee; H Peters
Journal:  J Genet Couns       Date:  2013-03-28       Impact factor: 2.537
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