Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with hereditary ataxia
| Autor: | Min Zhang, Xiang Hu, Ning Zhao, Chun-Zhen Li, Matthew Gandjian, Wan-Zhang Yang, Shan Jiang, Yun Zhang, Thomas E. Ichim, Shu Jiang, You-Xiang Sheng, SC Cho, Fang Wu, Shao-Hui Li, Guo-Jian Shu, Ying Tang |
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| Rok vydání: | 2011 |
| Předmět: |
Adult
Male medicine.medical_specialty Ataxia Cell Transplantation lcsh:Medicine Immunoglobulins Gastroenterology Umbilical cord Peripheral blood mononuclear cell General Biochemistry Genetics and Molecular Biology Young Adult T-Lymphocyte Subsets Internal medicine medicine Clinical endpoint Humans Adverse effect Aged Spinocerebellar Degenerations Medicine(all) medicine.diagnostic_test business.industry Lumbar puncture Biochemistry Genetics and Molecular Biology(all) Research lcsh:R General Medicine Middle Aged Fetal Blood Transplantation medicine.anatomical_structure Treatment Outcome Berg Balance Scale Immunology Leukocytes Mononuclear Female medicine.symptom business |
| Zdroj: | Journal of Translational Medicine Journal of Translational Medicine, Vol 9, Iss 1, p 65 (2011) |
| ISSN: | 1479-5876 |
| Popis: | Background The differential diagnosis for hereditary ataxia encompasses a variety of diseases characterized by both autosomal dominant and recessive inheritance. There are no curative treatments available for these neurodegenerative conditions. This open label treatment study used human umbilical cord blood-derived mononuclear cells (CBMC) combined with rehabilitation training as potential disease modulators. Methods 30 patients suffering from hereditary ataxia were treated with CBMCs administered systemically by intravenous infusion and intrathecally by either cervical or lumbar puncture. Primary endpoint measures were the Berg Balance Scale (BBS), serum markers of immunoglobulin and T-cell subsets, measured at baseline and pre-determined times post-treatment. Results A reduction of pathological symptoms and signs was shown following treatment. The BBS scores, IgG, IgA, total T cells and CD3+CD4 T cells all improved significantly compared to pre-treatment values (P < 0.01~0.001). There were no adverse events. Conclusion The combination of CBMC infusion and rehabilitation training may be a safe and effective treatment for ataxia, which dramatically improves patients' functional symptoms. These data support expanded double blind, placebo-controlled studies for these treatment modalities. |
| Databáze: | OpenAIRE |
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