Autologous hematopoietic stem cell transplantation modulates immunocompetent cells and improves β-cell function in Chinese patients with new onset of type 1 diabetes

J Clin Endocrinol Metab. 2012 May;97(5):1729-36. doi: 10.1210/jc.2011-2188. Epub 2012 Mar 14.


Context: Autologous hematopoietic stem cell transplantation (AHSCT) has the potential to induce clinical remission in patients with newly diagnosed type 1 diabetes.

Objective: The objective of the study was to examine the impact of AHSCT on lymphocytes and pancreatic β-cell function.

Design: This was a nonrandomized, open-label prospective study.

Patients and interventions: Thirteen patients with new onset of type 1 diabetes, 10 of them with diabetic ketoacidosis, were subjected to AHSCT with cryopreserved CD34(+) progenitor cells and followed up for 31-54 months.

Main outcome measures: The numbers of different subsets of lymphocytes and the levels of serum cytokines, islet antibodies, C-peptide, and plasma glycosylated hemoglobin were longitudinally measured.

Results: The numbers of different subsets of lymphocytes, except for CD8(+) T cells, in the patients before AHSCT were significantly lower than those in controls. However, all lymphocytes gradually recovered after AHSCT, accompanied by decreased levels of serum autoantibodies, IL-1, IL-17, and TNF-α. After AHSCT, 11 of 13 patients required significantly reduced doses of insulin for adequate glycemic control, accompanied by reduced levels of glycosylated hemoglobin but increased C-peptide concentrations. Three patients achieved exogenous insulin independence for 7-54 months. The survival of remaining β-cells was associated positively with the preexisting β-cell function but negatively with preexisting autoantibodies (P < 0.05). The numbers of infused CD34(+) cells were positively correlated with the concentrations of serum IL-10, IL-4, TGF-β, and fasting C-peptide but negatively correlated with the levels of serum TNF-α and insulin doses after AHSCT (P < 0.05).

Conclusion: AHSCT modulated lymphocytes and preserved β-cell function in Chinese patients with new onset of type 1 diabetes and diabetic ketoacidosis.

Trial registration: NCT01341899.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Asian Continental Ancestry Group
  • Blood Glucose / metabolism
  • Child
  • Diabetes Mellitus, Type 1 / blood
  • Diabetes Mellitus, Type 1 / physiopathology
  • Diabetes Mellitus, Type 1 / surgery*
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Insulin / blood
  • Insulin-Secreting Cells / physiology*
  • Male
  • Prospective Studies
  • Transplantation, Autologous
  • Treatment Outcome
  • Young Adult


  • Blood Glucose
  • Insulin

Associated data