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Bone marrow transplant for a girl with bone marrow failure and cerebral palsy
Author(s) -
Kodama Yuichi,
Okamoto Yasuhiro,
Shinkoda Yuichi,
Tanabe Takayuki,
Nishikawa Takuro,
Yamaki Yuni,
Kurauchi Koichiro,
Kawano Yoshifumi
Publication year - 2014
Publication title -
pediatrics international
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.49
H-Index - 63
eISSN - 1442-200X
pISSN - 1328-8067
DOI - 10.1111/ped.12297
Subject(s) - medicine , girl , bone marrow transplant , bone marrow transplantation , bone marrow , cerebral palsy , bone marrow failure , pediatrics , pathology , physical medicine and rehabilitation , haematopoiesis , stem cell , psychology , developmental psychology , biology , genetics
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 .