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口服他克莫司治疗儿童激素抵抗型溃疡性结肠炎。

Oral tacrolimus for pediatric steroid-resistant ulcerative colitis.

机构信息

Pediatric Gastroentrology and Nutrition Unit, Hospital Materno Infantil, Málaga, Spain.

出版信息

J Crohns Colitis. 2014 Jan;8(1):64-9. doi: 10.1016/j.crohns.2013.03.006. Epub 2013 Apr 9.

Abstract

BACKGROUND

Ulcerative colitis (UC) occurring during childhood is generally extensive and is associated with severe flares that may require intravenous steroid treatment. In cases of corticosteroid resistance is necessary to introduce a second-line treatment to avoid or delay surgery.

AIMS

To describe the efficacy and safety of oral tacrolimus for the treatment of severe steroid-resistant UC.

METHODS

We performed a retrospective study that included all patients under age 18 suffering from severe steroid-resistant UC treated with oral tacrolimus during the period January 1998 to October 2012 and with a follow-up period after treatment of 24 months or more.

RESULTS

A total of ten patients were included. The age at baseline was 9.4±4.9 years, and the time from diagnosis was 1.3 months (IQR, 1-5.7). Seven of the patients were in their first flare of disease. All of them received an oral dose of 0.12 mg/kg/day of tacrolimus divided in two doses. Trough plasma levels of tacrolimus were maintained between 4 and 13 ng/ml. Response was seen in 5/10 patients at 12 months, colectomy was eventually performed in 60% of patients during the follow-up period.

CONCLUSIONS

Tacrolimus is useful in inducing remission in patients with severe steroid-resistant UC, preventing or delaying colectomy, and allowing the patient and family to prepare for a probable surgery. Tacrolimus may also be used as a treatment bridge for corticosteroid-dependent patients until the new maintenance therapy takes effect.

摘要

背景

儿童时期发生的溃疡性结肠炎(UC)通常为广泛性的,与严重的发作有关,可能需要静脉注射类固醇治疗。在皮质类固醇抵抗的情况下,有必要引入二线治疗以避免或延迟手术。

目的

描述口服他克莫司治疗严重类固醇抵抗性 UC 的疗效和安全性。

方法

我们进行了一项回顾性研究,纳入了 1998 年 1 月至 2012 年 10 月期间接受口服他克莫司治疗且治疗后随访时间超过 24 个月的所有年龄在 18 岁以下的患有严重类固醇抵抗性 UC 的患者。

结果

共纳入 10 例患者。基线时的年龄为 9.4±4.9 岁,从诊断到开始治疗的时间为 1.3 个月(IQR,1-5.7)。7 例患者为首次疾病发作。所有患者均接受 0.12mg/kg/天的口服他克莫司治疗,分两次给药。他克莫司的谷浓度维持在 4 至 13ng/ml 之间。12 个月时,5/10 例患者出现缓解,随访期间 60%的患者最终行结肠切除术。

结论

他克莫司可有效诱导严重类固醇抵抗性 UC 患者缓解,预防或延迟结肠切除术,并使患者和家属为可能的手术做好准备。他克莫司也可作为依赖皮质类固醇的患者的治疗桥梁,直至新的维持治疗生效。

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