Eighteen-year follow-up of the first Italian MPSI patient treated with bone marrow transplantation

Bone Marrow Transplantation

Volumn 41, Issue 10, 2008, Pages 905-906

  Messina, C ^a   Rampazzo, A ^a   Cesaro, S ^a   Monciotti, C ^a   Gasparotto, N ^a   Tomanin, R ^a   Scarpa, Maurizio ^a  

^a UNIVERSITY OF PADOVA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; GLYCOSAMINOGLYCAN; LEVO IDURONIDASE; STEROID;

ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ADULT; BODY HEIGHT; BONE MARROW TRANSPLANTATION; CASE REPORT; CHILD; CHIMERA; CORNEA TRANSPLANTATION; ECHOCARDIOGRAPHY; FEMALE; FOLLOW UP; GRAFT VERSUS HOST REACTION; HEART DISEASE; HEART EJECTION FRACTION; HEART LEFT VENTRICLE FAILURE; HUMAN; HURLER SYNDROME; ITALY; LETTER; LIVER BIOPSY; PRIORITY JOURNAL; QUALITY OF LIFE; SEXUAL MATURATION; SPINE STABILIZATION; TENDON SURGERY;

ADOLESCENT; ADULT; BONE MARROW TRANSPLANTATION; CHILD, PRESCHOOL; FEMALE; FOLLOW-UP STUDIES; HUMANS; MUCOPOLYSACCHARIDOSIS I;

EID: 44449129111     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1705988     Document Type: Letter

References (4)

1. Neufeld EF, Muenzer J. The Mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds). The Metabolic and Molecular Bases of Inherited Disease, 8th edn, vol. III. McGraw-Hill: New York, 2001, pp 3421-3452.
2. Hobbs JR, Hugh-Jones K, Barrett AJ, Byrom N, Chambers D, Henry K et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981; 2: 709-712.
3. Peters C, Steward CG. Hematopoietic cell transplantation for inherited metabolic diseases: An overview of outcomes and practice guidelines. Bone Marrow Transplant 2003; 31: 229-239.
4. Wraith JE. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother 2005; 6: 489-506.