Clinical observation of neural stem cells in treatment of 4 patients with methylmalonic acidemia secondary to brain injury
YANG Hui
QU Suqing
LIU Weipeng
DU Kan
YANG Yinxiang
LUAN Zuo
Abstract：Objective To observe the neural stem cell therapeutic effect with methylmalonic acidemia(MMA) secondary to brain injury.Methods From April 2008 to October 2010, 4 patients with methylmalonic acidemia secondary to brain injury admitted to our hospital .All the patients re-ceived the neural stem cell transplantation via ventricle puncture positioned by MRI , and all the pa-tients were given a follow-up at 1 month and 6 month after the transplantation with Gesell scales assessment .Results Three of the four patients got improvements in motor and fine skills , visual and intellectual reactions after the transplantation within 1 month.In the following 6 months the conti-nuing improvements could be observed after the transplantation among the 3 patients.Cell therapy did not appear irreversible adverse events .Conclusion Neural stem cell therapy is safe and reliable , it can improve the clinical manifestations in children with methylmalonic academia secondary to brain injury in a short time .The efficacy can sustain to 6 months after the transplantation .On the basis of the treatment for the cause , the neural stem cell transplantation provides a new method of treatment for methylmalonic acidemia secondary to brain injury .But the long-term effect needs a further follow-up observation .
Keywords：Methylmalonic acidemiaBrain injuryCell transplantationNeural stem cells
Publication Date：2014-01-01
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：4( 212-215 )