Kyrcz-Krzemień Sławomira, Helbig Grzegorz, Torba Karolina, Koclęga Anna, Krawczyk-Kuliś Małgorzata
Hematology. 2016 Jan;21(1):42-5. doi: 10.1179/1607845415Y.0000000049. Epub 2015 Sep 2.
Multiple sclerosis (MS) is a T-cell-mediated chronic inflammatory disorder of the central nervous system. Several agents have been approved for the treatment of MS; however, their efficacy is limited and short term. Autologous hematopoietic stem cell (HSC) transplantation may remain an encouraging option for some MS patients who failed prior conventional treatment. Objective To assess the safety and effectiveness of HSCs mobilization in patients with MS.
Thirty-nine patients (20 females and 19 males) with relapsing-remitting MS at median age of 40 years (range: 25-63) were included in this study. As a stem cell mobilization, they received either granulocyte colony-stimulating factor (G-CSF) alone (10 µg/kg s.c. daily; n = 1) or cyclophosphamide (CY; 2.0 g/m(2) i.v. on days 1-2) followed by G-CSF (n = 38).
The median number of mobilized HSCs per kg was 6.32 × 10(6) (range: 2.64-26.3 × 10(6)). One apheresis was sufficient for collection of HSCs in 30 out of 39 MS patients (77%). Two aphereses were required for seven patients, three for one and four for one (17, 3, and 3%; respectively). Side effects of HSCs mobilization have been reported for eight patients (30%) and they were following: Staphylococcus epidermidis bacteremia (n = 1), fever of unknown origin (n = 3), diarrhea (n = 3), and headache (n = 1).
Mobilization using CY and/or G-CSF resulted in effective mobilization in all MS patients. This procedure was found to be safe. No fatal outcome has been reported.
多发性硬化症（MS）是一种由T细胞介导的中枢神经系统慢性炎症性疾病。已有多种药物被批准用于治疗MS；然而，它们的疗效有限且维持时间短。对于一些先前传统治疗失败的MS患者，自体造血干细胞（HSC）移植可能仍是一个令人鼓舞的选择。目的是评估MS患者中HSC动员的安全性和有效性。
本研究纳入了39例复发缓解型MS患者（20例女性和19例男性），中位年龄为40岁（范围：25 - 63岁）。作为干细胞动员，他们要么单独接受粒细胞集落刺激因子（G - CSF）（10μg/kg皮下注射，每日一次；n = 1），要么先接受环磷酰胺（CY；2.0g/m²静脉注射，第1 - 2天），随后接受G - CSF（n = 38）。
每千克动员的HSC中位数为6.32×10⁶（范围：2.64 - 26.3×10⁶）。39例MS患者中有30例（77%）一次单采就足以收集HSC。7例患者需要两次单采，1例需要三次，1例需要四次（分别为17%、3%和3%）。已报告8例患者（30%）出现HSC动员的副作用，具体如下：表皮葡萄球菌菌血症（n = 1）、不明原因发热（n = 3）、腹泻（n = 3）和头痛（n = 1）。
使用CY和/或G - CSF进行动员在所有MS患者中均能有效动员。该程序被发现是安全的。未报告有致命结局。
