期刊
DIABETES
卷 63, 期 9, 页码 3041-3046出版社
AMER DIABETES ASSOC
DOI: 10.2337/db14-0295
关键词
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资金
- Italian Scientists and Scholars of North America Foundation-Fondazione Marche Fellowship
- JDRF Career Development Award
- American Society of Nephrology Career Development Grant
- American Diabetes Association Mentor-Based Fellowship Award
- Translational Research Program grant from Boston Children's Hospital
- Harvard Stem Cell Institute grant (Diabetes Program) [DP-0123-12-00]
- Italian Ministry of Health [RF-2010-2303119, RF-2010-2314794, RF-FSR-2008-1213704]
Type 1 diabetes (T1D) is one of the major autoimmune diseases affecting children and young adults worldwide. To date, the different immunotherapies tested have achieved insulin independence in <5% of treated individuals. Recently, a novel hematopoietic stem cell (HSC)-based strategy has been tested in individuals with new-onset T1D. The aim of this study was to determine the effects of autologous nonmyeloablative HSC transplantation in 65 individuals with new-onset T1D who were enrolled in two Chinese centers and one Polish center, pooled, and followed up for 48 months. A total of 59% of individuals with T1D achieved insulin independence within the first 6 months after receiving conditioning immunosuppression therapy (with antithymocyte globulin and cyclophosphamide) and a single infusion of autologous HSCs, and 32% remained insulin independent at the last time point of their follow-up. All treated subjects showed a decrease in HbA(1c) levels and an increase in C-peptide levels compared with pretreatment. Despite a complete immune system recovery (i.e., leukocyte count) after treatment, 52% of treated individuals experienced adverse effects. Our study suggests the following: 1) that remission of T1D is possible by combining HSC transplantation and immunosuppression; 2) that autologous nonmyeloablative HSC transplantation represents an effective treatment for selected individuals with T1D; and 3) that safer HSC-based therapeutic options are required.
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