Department of endocrine and metabolic diseases, Ruijin hospital, Shanghai Jiao-Tong university, school of medicine, Shanghai Key laboratory for endocrine tumours, Shanghai clinical centre for endocrine and metabolic diseases, Shanghai institute of endocrine and metabolic diseases and Shanghai E-institute for endocrinology, Shanghai, China.
Department of haematology, Ruijin hospital, Shanghai Jiao-Tong university, school of medicine, Shanghai, China.
Diabetes Metab. 2018 Sep;44(4):341-345. doi: 10.1016/j.diabet.2017.12.006. Epub 2017 Dec 22.
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<0.01). C-peptide levels increased significantly at 3 months in both groups and later decreased, with the insulin group showing more rapid deterioration. Most of the adverse events in the AHSCT group were transplantation complications. Our data suggest that AHSCT treatment was well tolerated and slowed deterioration of islet β-cell function while significantly decreasing daily insulin dosages. However, because of the high relapse rate, more information on longer-term outcomes is needed before AHSCT can be routinely considered for T1D patients.
although this was a non-randomized clinical study, this phase-II trial demonstrated the beneficial effects of AHSCT in patients with newly diagnosed T1D by increasing C-peptide levels and inducing insulin independence, while showing its safety and good tolerability compared with conventional intensive insulin therapy. Thus, these results are helpful for increasing our understanding of the use of haematopoietic stem cell therapy in the treatment of T1D and for evaluating whether it can become more widespread in future.
1 型糖尿病（T1D）的特征是由于免疫攻击导致胰岛功能严重受损；因此，它也被称为“胰岛素依赖型糖尿病”。本研究旨在评估自体造血干细胞移植（AHSCT）在新诊断为 T1D 的青少年患者中的安全性和临床疗效。这是一项 2008 年 3 月至 2011 年 12 月进行的 II 期前瞻性、平行分组、非随机临床试验，共纳入 40 例 T1D 患者，其中 20 例接受 AHSCT 治疗，20 例仅接受胰岛素注射治疗。在 AHSCT 组中，14 例（70%）患者在 1.5 至 48 个月时实现了胰岛素非依赖性，而胰岛素组中仅 1 例患者实现了胰岛素非依赖性。在这 14 例 AHSCT 患者中，11 例在中位时间 19.5（范围 5.5-1）个月时复发并恢复使用胰岛素。在 4 年随访结束时，AHSCT 组和胰岛素组之间的每日胰岛素剂量差异变小（0.49±0.32IU/kg/day 与 0.79±0.18IU/kg/day，分别；P<0.01）。两组患者的 C 肽水平在 3 个月时均显著升高，随后下降，胰岛素组下降更快。AHSCT 组的大多数不良事件为移植并发症。我们的数据表明，AHSCT 治疗耐受性良好，可减缓胰岛β细胞功能的恶化，同时显著减少每日胰岛素剂量。然而，由于复发率较高，在 AHSCT 可常规用于 T1D 患者之前，还需要更多关于长期结果的信息。
尽管这是一项非随机临床研究，但这项 II 期试验通过增加 C 肽水平和诱导胰岛素独立性，证明了 AHSCT 对新诊断为 T1D 的患者的有益效果，同时与传统强化胰岛素治疗相比，显示出其安全性和良好的耐受性。因此，这些结果有助于我们更好地了解造血干细胞治疗在 T1D 治疗中的应用，并评估其是否在未来更广泛应用。
