title: Hurler syndrome with severe complication in post-bone marrow transplantation course: life threatening interstitial pneumonitis and hypertension.
abstract: Bone marrow transplantation (BMT) was performed in a 3 year old patient with Hurler syndrome. The post-BMT course was complicated by interstitial pneumonitis and severe hypertension, which were life threatening. The patient responded well to therapy and recovered. BMT in this patient resulted in significant clinical improvement in the signs and symptoms of Hurler syndrome. Biochemical improvement, including elevated alpha-L-iduronidase activity in white blood cells and decreased urinary glycosaminoglycan excretion was significant. However, skeletal and neurological impairment were not improved. We conclude that BMT for Hurler syndrome should be performed at an earlier stage, before severe neuronal damage has occurred. Moreover, BMT is a high risk procedure and there will always be a possibility that life threatening complications will occur, as in our case.
authors.affiliation: Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan.
authors.forename: T
authors.initials: T
authors.lastname: Ohashi
authors.forename: T
authors.initials: T
authors.lastname: Tahara
authors.forename: K
authors.initials: K
authors.lastname: Fujisawa
authors.forename: Y
authors.initials: Y
authors.lastname: Hoshi
authors.forename: F
authors.initials: F
authors.lastname: Ito
authors.forename: N
authors.initials: N
authors.lastname: Suzuki
authors.forename: Y
authors.initials: Y
authors.lastname: Eto
authors.forename: K
authors.initials: K
authors.lastname: Maekawa
mesh_terms: D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D006801:Humans; D006973:Hypertension / Q000209:etiology*; D017563:Lung Diseases, Interstitial / Q000209:etiology*; D008297:Male; D008059:Mucopolysaccharidosis I / Q000601:surgery*; D011183:Postoperative Complications