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

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 .