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阻止糖尿病发展:自体非清髓性干细胞移植

Stopping diabetes in its tracks: autologous non-myeloablative stem cell transplantation.

作者信息

Fousteri Georgia, Hayek Alberto, von Herrath Matthias

机构信息

La Jolla Institute for Allergy and Immunology, 9420 Athena Circle, La Jolla, CA 92037, USA.

出版信息

Regen Med. 2007 Sep;2(5):845-51. doi: 10.2217/17460751.2.5.845.

Abstract

A recent report in this year's April issue of Journal of the American Medical Association describes an unprecedented success in delaying insulin dependence in patients with recent-onset Type 1 diabetes after non-myeloablative immune suppression with cyclophosphamide and antithymocyte globulin followed by autologous stem cell transplantation. In this study, 14 out of 15 patients became insulin-independent, which lasted up to 35 months. Concomitantly, C-peptide levels increased substantially compared with preintervention values. Treatment of autoimmune disorders, and in particular Type 1 diabetes, constitutes a complex balancing act between suppressing autoaggressive responses strongly and permanently enough, while circumventing much-feared long-term side effects from chronic immunosuppression. This clinical Phase I/II trial is relevant to fine-tuning interventive protocols and contributing to the further development of suitable combination therapies to prevent and treat Type 1 diabetes.

摘要

今年4月号的《美国医学会杂志》上最近发表的一篇报告描述了一项前所未有的成功案例,即对近期发病的1型糖尿病患者进行非清髓性免疫抑制,使用环磷酰胺和抗胸腺细胞球蛋白,随后进行自体干细胞移植,成功延缓了胰岛素依赖。在这项研究中,15名患者中有14名不再依赖胰岛素,这一情况持续了长达35个月。与此同时,与干预前的值相比,C肽水平大幅上升。自身免疫性疾病的治疗,尤其是1型糖尿病的治疗,是一种复杂的平衡行为,既要足够强烈和持久地抑制自身攻击性反应,又要避免慢性免疫抑制带来的令人担忧的长期副作用。这项临床I/II期试验对于微调干预方案以及推动预防和治疗1型糖尿病的合适联合疗法的进一步发展具有重要意义。

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