自体造血干细胞移植治疗难治性炎症性肠病10例回顾与随访

目的 回顾自体造血干细胞移植(HSCT)治疗10例炎症性肠病(IBD)患者的疗效及安全性.方法 2004年1月至2006年8月,采用HSCT治疗9例糖皮质激素及免疫抑制剂治疗无效的克罗恩病(CD)患者及1例Truelove临床重型溃疡性结肠炎(UC)患者.CD患者中2例CD活动指数(CDAD大于450(严重型),6例CDAI为150～450(活动型).1例UC患者为全结肠炎型.经环磷酰胺(CTX)及粒细胞刺激因子动员后,采集患者外周血于细胞,行CD34+细胞分选并置液氮保存,2周后行CTX及抗淋巴细胞球蛋白预处理,将解冻的干细胞回输.结果 HSCT后3和12个月时分别有5例和1例患者CDAI<150(完全缓解).2例术后CDAI分值下降但未达缓解标准;完全缓解者症状消失,血液检查指标正常,体重明显增加(5～20 kg).平均随访16.1个月,复发4例,除1例病情严重外,余均较术前病情减轻;5例患者获长期缓解.1例UC患者术后10个月无复发症状,血液检查指标均正常,但肠镜复查示无明显改善.与HSCT治疗相关的并发症有干细胞回输期间与白细胞减少相关的发热及感染、术后脱发,1例乙型肝炎病毒(HBV)携带者术后转氨酶水平及HBV-DNA滴度增高.结论 HSCT可改善其他方法治疗无效的难治性IBD,近期疗效满意,部分患者可获较长期的缓解,治疗期间无严重不良反应,但不能阻止病情复发,且不能改善患者肠道病理改变.

Retrospective and follow-up study on autologous haematopoietic stem cell transplantation in ten patients with refractory inflammatory bowel disease

Objective To investigate the safety and efficacy of autologous haematopoietic stem cell transplantation (HSCT) in treatment of patients with refractory inflammatory bowel disease (IBD). Methods Ten patients with active moderate-severe IBD [nine with Crohn's disease (CD) and one with ulcerative disease (UC)] were treated with HSCT from January 2004 to August 2006.Among 9 CD patients,the CD active inedx(CDAl) of 2 patients were above 450 (severe),6 patients were 150-450 (active).One patient was suffered from severe UC(whole colon).The stem cells were collected from the patients who intravenously received cycloptlosphamide (2.0 g/m2 ) and granulocyte colony-stimulating factor (5 μg · kg-1 · d-1 ).The CD34+ cells were isolated and cryo-preserved.After 2 weeks,the HSCT was carried out.Results In 9 patients with CD,clinical complete remission (CDAI<150) was achieved in 5 and 1 patients at 3 and 12 months after HSCT.The CDAI of other 2 patients decreased but not reached clinical complete remission.The patients were followed up of 16.1 months (ranged 10-33 months).The disease relapsed in 4 patients and complete remission in 5 patients.However,no improvement was observed under repeated eolonoscopy in 1 patient with UC who had not relapsed in 10 months.HSCT-related side effects included neutropenia caused fever,infection,etc.One HBV-carrier developed asymptomatie increase of liver enzymes and HBV-DNA copies after HSCT.Conclusions Autologous HSCT can be conducted safely and is well tolerated in patients with refractory IBD.It can induce clinical remission in most of the patients,although endoscopic remission may not be achieved,and relapses can not be avoided in some patients.