Gastroenterology Department, Hospital Clínic de Barcelona, CIBEREHD, Institut d'investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Barcelona, Spain.
Hematology Department-HSCT Unit, Hospital Clínic de Barcelona, Barcelona, Spain.
J Crohns Colitis. 2017 Oct 1;11(10):1161-1168. doi: 10.1093/ecco-jcc/jjx054.
Haematopoietic stem cell transplantation [HSCT] is considered a therapeutic option for patients with severe Crohn's disease [CD] unresponsive to currently available therapies.
Autologous HSCT was considered for CD patients with active disease, unresponsive or intolerant to approved medications and unsuitable for surgery. After HSCT, patients were closely followed up every 6 weeks during the first 2 years and every 6 months thereafter up to 5 years. Colonoscopy and/or magnetic resonance imaging were performed at Months 6, 12, 24, and 48 after HSCT.
From December 1, 2007 to December 31, 2015, 37 CD patients were assessed for HSCT. Of these, 35 patients [13 within the ASTIC trial] underwent mobilisation. Six patients did not complete the transplant for various reasons and 29 patients were finally transplanted. Patients were followed up during a median of 12 months [6-60]. At 6 months, 70% of patients achieved drug-free clinical remission (Crohn's Disease Index of Severity [CDAI] < 150). The proportion of patients in drug-free remission (CDAI < 150, Simple Endoscopic activity Score [SES]-CD < 7] was 61% at 1 year, 52% at 2 years, 47% at 3 years, 39% at 4 years, and 15% at 5 years. Patients who relapsed were re-treated and 80% regained clinical remission. Six out of the 29 [21%] required surgery. One patient died due to systemic cytomegalovirus infection 2 months after transplant.
HSCT is a salvage therapy for patients with extensive and refractory CD. Although relapse occurs in a majority of patients within 5 years after transplant, drug responsiveness is regained and clinical remission achieved in 80% of cases.
造血干细胞移植（HSCT）被认为是对目前可用治疗方法无反应或不耐受的严重克罗恩病（CD）患者的一种治疗选择。
对于活动期、对已批准的药物无反应或不耐受且不适合手术的 CD 患者，考虑进行自体 HSCT。HSCT 后，在前 2 年内每 6 周、此后 5 年内每 6 个月对患者进行密切随访。在 HSCT 后第 6、12、24 和 48 个月进行结肠镜检查和/或磁共振成像。
从 2007 年 12 月 1 日至 2015 年 12 月 31 日，对 37 例 CD 患者进行了 HSCT 评估。其中，13 例患者在 ASTIC 试验中接受了动员。由于各种原因，有 6 例患者未完成移植，最终有 29 例患者进行了移植。患者中位随访时间为 12 个月[6-60]。在 6 个月时，70%的患者达到无药物临床缓解（克罗恩病严重程度指数[CDAI]＜150）。无药物缓解（CDAI＜150、简单内镜活动评分[SES]-CD＜7）的患者比例在 1 年时为 61%、2 年时为 52%、3 年时为 47%、4 年时为 39%、5 年时为 15%。复发患者进行了再治疗，80%的患者恢复了临床缓解。29 例患者中有 6 例[21%]需要手术。1 例患者在移植后 2 个月因全身巨细胞病毒感染死亡。
HSCT 是广泛和难治性 CD 患者的挽救性治疗。尽管大多数患者在移植后 5 年内复发，但 80%的患者对药物有反应并获得临床缓解。
