低剂量白细胞介素 2 治疗 Wiskott-Aldrich 综合征患者的 I 期临床试验。
Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome.
机构信息
Division of Allergy and Immunology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
The Children's Hospital of Philadelphia, Philadelphia, PA, United States.
出版信息
Clin Immunol. 2017 Jun;179:47-53. doi: 10.1016/j.clim.2017.02.001. Epub 2017 Feb 14.
BACKGROUND
Low dose IL-2 can restore the function of T and NK cells from Wiskott-Aldrich (WAS) patients. However, the safety of in vivo IL-2 in WAS is unknown.
OBJECTIVES
A phase-I study to assess safety of low dose IL-2 in WAS.
METHODS
Patients received 5 daily subcutaneous IL-2 injections, every 2months, for three courses. A "3+3" dose escalation method was used.
RESULTS
6 patients received the 0.5millionunits/m/day dose without serious adverse events. However, 2 of 3 patients receiving the 1millionunits/m/day dose developed thrombocytopenia requiring platelet transfusions. A statistically significant platelet increase occurred in patients receiving the 0.5millionunits/m/day dose. A trend toward higher T, B and NK cell numbers and higher T regulatory cell percentages was observed.
CONCLUSION
We have identified a safe IL-2 dose for WAS patients. Additional trials are indicated to study the efficacy of this immunostimulant as a therapy for WAS.
背景
低剂量白细胞介素 2（IL-2）可以恢复威斯科特-奥尔德里奇综合征（WAS）患者 T 和 NK 细胞的功能。然而，体内应用 IL-2 在 WAS 中的安全性尚不清楚。
目的
一项评估低剂量 IL-2 在 WAS 中安全性的 I 期研究。
方法
患者接受 5 天的每日皮下 IL-2 注射，每 2 个月一次，共三个疗程。采用“3+3”剂量递增法。
结果
6 名患者接受 0.5 百万单位/m/天剂量，未发生严重不良事件。然而，3 名接受 1 百万单位/m/天剂量的患者中有 2 名发生血小板减少症，需要血小板输注。接受 0.5 百万单位/m/天剂量的患者血小板明显增加。观察到 T、B 和 NK 细胞数量增加和 T 调节细胞百分比升高的趋势。
结论
我们已经确定了 WAS 患者的安全 IL-2 剂量。需要进一步的试验来研究这种免疫刺激剂作为 WAS 治疗方法的疗效。
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