Clinical benefits of autologous haematopoietic stem cell transplantation in type 1 diabetes patients

Abstract Type 1 diabetes (T1D) is characterized by severe damage to pancreas islet function through immunological attack; therefore, it is also called ‘insulin-dependent diabetes'. The present study aimed to evaluate the safety and clinical efficacy of autologous haematopoietic stem cell transplantation (AHSCT) in adolescent patients with newly diagnosed T1D. A phase-II prospective, parallel-assignment, non-randomized trial was conducted from March 2008 to December 2011 with 40 T1D patients, of whom 20 received AHSCT therapy and 20 were treated only with insulin injections. Of these patients, 14 (70%) in the AHSCT group became insulin-independent for 1.5 to 48 months compared with only one patient in the Insulin group. Of these 14 AHSCT patients, 11 relapsed within a median time of 19.5 (range 5.5–1) months and resumed insulin use. By the end of the 4-year follow-up, the difference in daily insulin dosages between the AHSCT and Insulin groups had become smaller (0.49±0.32IU/kg/day vs. 0.79±0.18IU/kg/day, respectively; P