Bone marrow transplant for a girl with bone marrow failure and cerebral palsy

作者：Kodama Yuichi; Okamoto Yasuhiro^*; Shinkoda Yuichi; Tanabe Takayuki; Nishikawa Takuro; Yamaki Yuni; Kurauchi Koichiro; Kawano Yoshifumi

来源：Pediatrics International, 2014, 56(3): 424-426.

DOI：10.1111/ped.12297

• bone marrow transplantation
• cerebral palsy
• congenital bone marrow failure
• mental retardation

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.

• 出版日期2014-6