Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

Molecular Genetics and Metabolism

Volumn 117, Issue 3, 2016, Pages 373-377

  Ghosh, Arunabha ^a,c   Miller, Weston ^b   Orchard, Paul J ^b   Jones, Simon A ^a   Mercer, Jean ^a   Church, Heather J ^a   Tylee, Karen ^a   Lund, Troy ^b   Bigger, Brian W ^c   Tolar, Jakub ^b   Wynn, Robert F ^d  

^a ST MARY S HOSPITAL   (United Kingdom)

^b UNIVERSITY OF MINNESOTA   (United States)

^c UNIVERSITY OF MANCHESTER   (United Kingdom)

^d ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

Enzyme replacement therapy; Haematopoietic stem cell transplantation; Hurler syndrome; Mucopolysaccharidosis type i; Outcome

Indexed keywords

ALEMTUZUMAB; BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; GLYCOSAMINOGLYCAN; LARONIDASE; THIOTEPA; THYMOCYTE ANTIBODY; LEVO IDURONIDASE;

ACUTE GRAFT VERSUS HOST DISEASE; ARTICLE; CHILD; CHRONIC GRAFT VERSUS HOST DISEASE; ENGRAFTMENT; ENZYME REPLACEMENT; FEMALE; FOLLOW UP; GRAFT FAILURE; HEALTH CARE FACILITY; HEART MUSCLE CONTRACTILITY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HURLER SYNDROME; INFANT; MAJOR CLINICAL STUDY; MALE; MORTALITY; NONMYELOABLATIVE CONDITIONING; OVERALL SURVIVAL; PRESCHOOL CHILD; PRIORITY JOURNAL; URINE LEVEL; CLINICAL TRIAL; GRAFT VS HOST DISEASE; MUCOPOLYSACCHARIDOSIS I; MULTICENTER STUDY; SURVIVAL ANALYSIS; TIME FACTOR; TREATMENT OUTCOME; URINE;

CHILD, PRESCHOOL; ENZYME REPLACEMENT THERAPY; FEMALE; FOLLOW-UP STUDIES; GLYCOSAMINOGLYCANS; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IDURONIDASE; INFANT; MALE; MUCOPOLYSACCHARIDOSIS I; SURVIVAL ANALYSIS; TIME FACTORS; TREATMENT OUTCOME;

EID: 84959544523     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2016.01.011     Document Type: Article

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