Allogeneic cord blood mesenchymal stem cell transplantation for treating Emery-Dreifus muscular dystrophy in one case
10.3969/j.issn.1673-8225.2009.27.041
- VernacularTitle:异基因脐血间充质干细胞移植治疗Emery-Dreifuss型肌营养不良1例
- Author:
Yu LIAO
- Publication Type:Journal Article
- From:
Chinese Journal of Tissue Engineering Research
2009;13(27):5393-5396
- CountryChina
- Language:Chinese
-
Abstract:
A male patient, aged 32 years, weighing 38 kg, was recruited at the Department of Neurology, Wuxi Second Hospital, Nanjing Medical University in February 2008. The patient had general fatigue and amyotrophy for 18 years, and recruited in the hospital 3 days following burst dizziness and vomiting. Following genetic analysis and muscle biopsy, the patient was diagnosed as having Emery-Dreifus muscular dystrophy and cerebellar infarction. HLA compatible cord blood mesenchymal stem cell (CB-MSC) transplantation was performed. The grafted CB-MSCs were from Shanghai Cord Blood Bank. The donor was HIA all-identical, Rh blood type-identical, ABO blood type-incompatible. The patient received preconditioning of busulfan + cyclophosphamide + rabbit antithymocyte globulin. The patient then received peripheral venous administration of 40 mL CB-MSCs, 10 drops/minute, with total nucleated cell number of 31.98x108. 5 weeks after transplantation, symptoms of dizziness, swallowing difficulty and regurgitation of fluid were markedly alleviated. His weight increased by 4 kg and he recovered of hand coordination to handling computer operation. 6 months following transplantation, serum creatine phosphokinase decreased from 35.79 μkat/L, before transplantation to 9.55 μkat/L; serum creatine phosphokinase isoenzyme reduced from 18.20 μkat/L before transplantation to 4.78 μkat/L. Myoglobin decreased from 413.50 μg/L to 213.20 μg/L. Functional independence measure scores increased from 71 points to 101 points. Immunological rejection or tumorigenesis was not identified. CB-MSC transplantation can evidently decrease serum creatine phosphokinase level within a short period, and improve motor function in Emery-Dreifus muscular dystrophy patient. CB-MSC transplantation is beneficial for Emery-Dreifus muscular dystrophy, and short-term safety is reliable.
