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长效奥曲肽成功用于治疗儿童难治性慢性胃肠道出血。

Successful use of long-acting octreotide for intractable chronic gastrointestinal bleeding in children.

作者信息

O'Meara Marie, Cicalese Maria P, Bordugo Andrea, Ambrosi Alessandro, Hadzic Nedim, Mieli-Vergani Giorgina

机构信息

*Paediatric Liver, GI and Nutrition Centre †Department of Pharmacy, King's College Hospital NHS Foundation Trust, London, UK ‡Department of Paediatrics, S. Maria degli Angeli Hospital, Pordenone §Università Vita-Salute, San Raffaele Hospital ||San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), San Raffaele Scientific Institute, Milan, Italy.

出版信息

J Pediatr Gastroenterol Nutr. 2015 Jan;60(1):48-53. doi: 10.1097/MPG.0000000000000540.

Abstract

BACKGROUND AND AIMS

Octreotide reduces splanchnic blood flow and is effective in controlling gastrointestinal bleeding (GIB) caused by portal hypertension. Monthly long-acting octreotide (OCT-LAR) with an efficacy and safety profile similar to subcutaneous daily administration presents an attractive option for long-term therapy. We report our experience with OCT-LAR for severe/recurrent GIB in children with portal hypertension secondary to chronic liver disease or portal vein thrombosis who were unresponsive to standard interventions.

METHODS

A total of 9 patients, 7 boys, who received OCT-LAR between 2000 and 2009 were studied retrospectively (median age at first bleeding 21 months, range 1 month-14.5 years). The dose (2.5-20 mg intramuscularly monthly) was extrapolated from that used in adult acromegaly and neuroendocrine tumours (10-60 mg/mo). Response to treatment was assessed by comparing the number of bleeding events, hospital admissions for acute bleeding, and number of blood units required during the year before and year after starting OCT-LAR.

RESULTS

OCT-LAR led to a reduction in the number of bleeding episodes in all of the children and to cessation of bleeding in 7. Two children listed for transplantation because of severe GIB were removed from the list. No serious adverse effects immediately attributable to OCT-LAR were observed. One child developed growth hormone deficiency and hypothyroidism during a prolonged period of treatment with subcutaneous octreotide before commencing OCT-LAR.

CONCLUSIONS

OCT-LAR can control severe intractable recurrent GIB in children with portal hypertension. Prospective randomised controlled trials and pharmacokinetic studies are indicated to establish the optimum dose and length of treatment of OCT-LAR and confirm its efficacy and long-term safety in children.

摘要

背景与目的

奥曲肽可减少内脏血流量,对控制门静脉高压引起的胃肠道出血(GIB)有效。长效奥曲肽(OCT-LAR)每月注射一次,其疗效和安全性与每日皮下注射相似,是长期治疗的一个有吸引力的选择。我们报告了在慢性肝病或门静脉血栓形成继发门静脉高压的儿童中,使用OCT-LAR治疗严重/复发性GIB的经验,这些儿童对标准干预措施无反应。

方法

对2000年至2009年间接受OCT-LAR治疗的9例患者(7例男孩)进行回顾性研究(首次出血时的中位年龄为21个月,范围为1个月至14.5岁)。剂量(每月2.5 - 20 mg肌肉注射)是根据成人肢端肥大症和神经内分泌肿瘤使用的剂量(10 - 60 mg/月)推算而来。通过比较开始使用OCT-LAR前一年和后一年的出血事件数量、急性出血的住院次数以及所需的血液单位数量来评估治疗反应。

结果

OCT-LAR使所有儿童的出血发作次数减少,7例出血停止。2例因严重GIB列入移植名单的儿童被从名单中移除。未观察到立即归因于OCT-LAR的严重不良反应。1例儿童在开始使用OCT-LAR之前,在长期皮下注射奥曲肽治疗期间出现生长激素缺乏和甲状腺功能减退。

结论

OCT-LAR可控制门静脉高压儿童的严重顽固性复发性GIB。需要进行前瞻性随机对照试验和药代动力学研究,以确定OCT-LAR的最佳剂量和治疗时长,并证实其在儿童中的疗效和长期安全性。

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