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: ClinicalTrials.gov NCT01341899.