Bousvaros A, Kirschner B S, Werlin S L, Parker-Hartigan L, Daum F, Freeman K B, Balint J P, Day A S, Griffiths A M, Zurakowski D, Ferry G D, Leichtner A M
Combined Program in Pediatric Gastroenterology and Nutrition, Boston Children's Hospital, Boston, Massachusetts 02115, USA.
J Pediatr. 2000 Dec;137(6):794-9. doi: 10.1067/mpd.2000.109193.
To evaluate the efficacy of oral tacrolimus as an induction agent in steroid-refractory severe colitis.
Open-label, multicenter trial of oral tacrolimus in patients with severe colitis. Patients not responding to conventional therapy received tacrolimus, 0.1 mg/kg/dose given twice a day, and the dosage was adjusted to achieve blood levels between 10 and 15 ng/mL. Response was defined as improvement in a number of clinical parameters (including abdominal pain, diarrhea, rectal bleeding, and cessation of transfusions). Patients who responded by 14 days continued to receive tacrolimus, and 6-mercaptopurine or azathioprine was added as a steroid-sparing agent 4 to 6 weeks after the tacrolimus was instituted.
Fourteen patients were enrolled in the study. One patient elected to withdraw after 48 hours. Of the 13 remaining, 9 (69%) responded and were discharged. Tacrolimus was continued for 2 to 3 months in the responders, except for 1 patient who was given tacrolimus for 11 months. After 1 year of follow-up, only 5 (38%) patients were receiving maintenance therapy; the other 4 responders had undergone colectomy.
Although tacrolimus is effective induction therapy for severe ulcerative or Crohn's colitis, fewer than 50% of patients treated will successfully achieve a long-term remission.
评估口服他克莫司作为激素难治性重症结肠炎诱导治疗药物的疗效。
一项关于口服他克莫司治疗重症结肠炎患者的开放标签、多中心试验。对传统治疗无反应的患者接受他克莫司治疗，剂量为0.1mg/kg/次，每日2次，并调整剂量以使血药浓度达到10至15ng/mL。反应定义为多项临床参数（包括腹痛、腹泻、直肠出血和输血停止）有所改善。在14天内有反应的患者继续接受他克莫司治疗，并在开始使用他克莫司4至6周后加用6-巯基嘌呤或硫唑嘌呤作为激素节约剂。
14名患者纳入研究。1名患者在48小时后选择退出。其余13名患者中，9名（69%）有反应并出院。有反应的患者继续使用他克莫司2至3个月，1名患者使用他克莫司11个月。随访1年后，只有5名（38%）患者接受维持治疗；其他4名有反应的患者接受了结肠切除术。
虽然他克莫司是重症溃疡性结肠炎或克罗恩结肠炎的有效诱导治疗药物，但接受治疗的患者中不到50%能成功实现长期缓解。
