Open AccessCord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome
Author(s) -
Susan Staba,
Maria L. Escolar,
Michele D. Poe,
Young Kim,
Paul Martin,
Paul Szabolcs,
June Allison-Thacker,
Susan Wood,
David A. Wenger,
Pablo Rubinstein,
John J. Hopwood,
William Krivit,
Joanne Kurtzberg
Publication year - 2004
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa032613
Subject(s) - hurler syndrome , medicine , busulfan , transplantation , cord blood , total body irradiation , mucopolysaccharidosis type i , mucopolysaccharidosis i , cyclophosphamide , mucopolysaccharidosis , surgery , graft versus host disease , gastroenterology , hematopoietic stem cell transplantation , disease , enzyme replacement therapy , chemotherapy
Hurler's syndrome (the most severe form of mucopolysaccharidosis type I) causes progressive deterioration of the central nervous system and death in childhood. Allogeneic bone marrow transplantation before the age of two years halts disease progression and prolongs life, but many children lack a bone marrow donor. We investigated the feasibility of using cord-blood transplants from unrelated donors and a myeloablative preparative regimen that did not involve total-body irradiation in young children with Hurler's syndrome.
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