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BACKGROUNDIt has been indicated that autologous hematopoietic stem cell transplantation (AHST) is a promising treatment to adults with type 1 diabetes, however, the application of AHST therapy to children with type 1 diabetes still needs more data. The aim of this study was to assess the clinical effect of immune intervention combined with AHST and conventional insulin therapy in the treatment of children with newly diagnosed type 1 diabetes.

METHODSThis 1:2 matched case-control study was comprised of 42 children who were newly diagnosed with type 1 diabetes in the Department of Endocrinology, Beijing Children's Hospital from 2009-2010. The case group included 14 patients, who were treated with AHST within the first 3 months after being diagnosed with diabetes at request of their parents during 2009-2010. The control group included 28 patients with newly diagnosed type 1 diabetes at the same period of hospitalization. We compared the baseline and follow-up data of them, including ketoacidosis onset, clinical variables (glycosylated hemoglobin (HbA1c), insulin dosage and serum C-peptide).

RESULTSThe clinical characteristics of the patients was comparable between the case group and the control group. At 6-12 months ((10.7±4.2) months) after AHST treatment, we found 11 patients in the case group did not stop the insulin therapy, three cases stopped insulin treatment for 2, 3 and 11 months, respectively. No diabetic ketoacidosis (DKA) occurred after transplantation in all the patients in the case group. HbA1c in the control group was significant lower than that in the case group (P < 0.01), while the insulin dosage and serum C-peptide were not significant different between the two groups (P > 0.05). In order to eliminate the honeymoon effect, we performed final follow-up at the 3-5 years ((4.2±1.8) years) after AHST treatment, and found that HbA1c in the control group was still lower than that in the case group (P < 0.01); however, the insulin dosage and serum C-peptide were not significantly different between the two groups (P > 0.05). Moreover, the insulin dosage was not significant different from baseline to follow-up period in the case group.

CONCLUSIONAHST treatment showed no advantage in effectiveness in children with newly diagnosed type 1 diabetes, both in insulin dose and long term blood glucose control.

Adolescent ; Case-Control Studies ; Child ; Child, Preschool ; Diabetes Mellitus, Type 1 ; drug therapy ; therapy ; Female ; Hematopoietic Stem Cell Transplantation ; Humans ; Hypoglycemic Agents ; therapeutic use ; Infant ; Insulin ; therapeutic use ; Male ; Transplantation, Autologous ; Treatment Outcome