Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis

  1. Paolo Fiorina1,2
  1. 1Nephrology Division, Boston Children's Hospital, Harvard Medical School, Boston, MA
  2. 2Transplant Medicine, IRCCS San Raffaele Hospital, Milan, Italy
  3. 3Department of Internal Diseases, Diabetology and Endocrinology, Central Hospital, Ministry of Interior Affairs and Administration, Warsaw, Poland
  4. 4Department of Endocrinology, Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland
  5. 5Division of Endocrinology, The Affiliated Drum Tower Hospital of Nanjing University, Nanjing, Jiangsu, People’s Republic of China
  6. 6Shangai Jiao Tong University School of Medicine, Shanghai, People’s Republic of China
  7. 7Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, Warsaw, Poland
  1. Corresponding author: Paolo Fiorina, paolo.fiorina{at}


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 HbA1c 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.

  • Received February 20, 2014.
  • Accepted April 8, 2014.

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