title: Bone marrow transplant for a girl with bone marrow failure and cerebral palsy.
abstract: Bone marrow transplantation (BMT) has been used with increasing frequency to treat congenital bone marrow failure syndrome (CBMFs) successfully. Decision to perform BMT, however, is difficult in the case of comorbidity because of regimen-related toxicities. We describe here a child with CBMFs, severe cerebral palsy (CP) at Gross Motor Function Classification System level V and mental retardation (MR) who was transfusion dependent despite various medications. She underwent BMT from an HLA-1 locus-mismatched unrelated donor. Although engraftment was successful, no neurological improvement was seen 5 years after BMT. While CBMFs patients who have CP and MR could undergo transplantation safely, they may not benefit neurologically from BMT.
authors.affiliation: Department of Pediatrics, Kagoshima University Graduate School of Medical and Dental Sciences, Kagoshima, Japan.
authors.forename: Yuichi
authors.initials: Y
authors.lastname: Kodama
authors.forename: Yasuhiro
authors.initials: Y
authors.lastname: Okamoto
authors.forename: Yuichi
authors.initials: Y
authors.lastname: Shinkoda
authors.forename: Takayuki
authors.initials: T
authors.lastname: Tanabe
authors.forename: Takuro
authors.initials: T
authors.lastname: Nishikawa
authors.forename: Yuni
authors.initials: Y
authors.lastname: Yamaki
authors.forename: Koichiro
authors.initials: K
authors.lastname: Kurauchi
authors.forename: Yoshifumi
authors.initials: Y
authors.lastname: Kawano
mesh_terms: D000741:Anemia, Aplastic; D001855:Bone Marrow Diseases; D000080983:Bone Marrow Failure Disorders; D016026:Bone Marrow Transplantation; D002547:Cerebral Palsy / Q000150:complications*; D002675:Child, Preschool; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000601:surgery*; D006801:Humans; D008607:Intellectual Disability / Q000150:complications
keywords: bone marrow transplantation; cerebral palsy; congenital bone marrow failure; mental retardation