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沙利度胺成功治疗遗传性出血性毛细血管扩张症合并的胃肠道血管发育异常出血

Successful treatment of bleeding gastro-intestinal angiodysplasia in hereditary haemorrhagic telangiectasia with thalidomide.

作者信息

Alam Mohamed Aftab, Sami Sarmad, Babu Sathish

机构信息

Department of Gastroenterology/General Medicine, Scarborough General Hospital, Scarborough, North Yorkshire, UK.

出版信息

BMJ Case Rep. 2011 Nov 8;2011:bcr0820114585. doi: 10.1136/bcr.08.2011.4585.

DOI:10.1136/bcr.08.2011.4585
PMID:22674103
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3214210/
Abstract

Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant disorder characterised by epistaxis, cutaneous telangiectasia and visceral arterio-venous malformations (AVMs). It affects approximately one in 5000 people. Control of sustained and repeated haemorrhages from telangiectasias in the nose and gut in patients who may be transfusion dependent is clinically challenging. After repeated endoscopic coagulations, multiple lesions often recur at other sites of gastro-intestinal tract, where endoscopic therapy or surgical resection is not possible. Hormonal therapy has been employed for more than 50 years but has recently been shown to be ineffective. Thalidomide, with its antiangiogenic mechanism of action, seems to be promising drug as a treatment option where other modalities have been unsuccessful. In this article, the authors discuss a novel treatment of bleeding gastro-intestinal angiodysplasia.

摘要

遗传性出血性毛细血管扩张症(HHT)是一种常染色体显性疾病,其特征为鼻出血、皮肤毛细血管扩张和内脏动静脉畸形(AVM)。该病影响约五千分之一的人群。对于可能依赖输血的患者,控制鼻部和肠道毛细血管扩张引起的持续性反复出血在临床上具有挑战性。经过反复内镜下凝血治疗后,多个病变常于胃肠道其他无法进行内镜治疗或手术切除的部位复发。激素治疗已应用50多年,但最近已证明无效。沙利度胺具有抗血管生成作用机制,在其他治疗方式均未成功的情况下,似乎是一种有前景的治疗选择药物。在本文中,作者讨论了一种治疗出血性胃肠道血管发育异常的新方法。

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本文引用的文献

1
Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia.沙利度胺可刺激血管成熟,减少遗传性出血性毛细血管扩张症患者的鼻出血。
Nat Med. 2010 Apr;16(4):420-8. doi: 10.1038/nm.2131. Epub 2010 Apr 4.
2
Lenalidomide to control gastrointestinal bleeding in hereditary haemorrhagic telangiectasia: potential implications for angiodysplasias?
Br J Haematol. 2009 Jul;146(2):220-2. doi: 10.1111/j.1365-2141.2009.07730.x. Epub 2009 May 12.
3
Antiestrogen therapy for hereditary hemorrhagic telangiectasia: a double-blind placebo-controlled clinical trial.遗传性出血性毛细血管扩张症的抗雌激素治疗:一项双盲安慰剂对照临床试验。
Laryngoscope. 2009 Feb;119(2):284-8. doi: 10.1002/lary.20065.
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Angiogenesis and vascular malformations: antiangiogenic drugs for treatment of gastrointestinal bleeding.血管生成与血管畸形:用于治疗胃肠道出血的抗血管生成药物
World J Gastroenterol. 2007 Dec 7;13(45):5979-84. doi: 10.3748/wjg.v13.45.5979.
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Hormonal and antihormonal therapy for epistaxis in hereditary hemorrhagic telangiectasia.遗传性出血性毛细血管扩张症鼻出血的激素及抗激素治疗
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Thalidomide for treatment of severe intestinal bleeding.沙利度胺用于治疗严重肠道出血。
Gut. 2004 Apr;53(4):609-12. doi: 10.1136/gut.2003.029710.
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Rendu-Osler-Weber disease: experience with 56 patients.朗杜-奥-韦氏病:56例患者的诊治经验
Ann Ital Med Int. 2002 Jul-Sep;17(3):173-9.
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Genetic epidemiology of hereditary hemorrhagic telangiectasia in a local community in the northern part of Japan.日本北部某当地社区遗传性出血性毛细血管扩张症的遗传流行病学
Hum Mutat. 2002 Feb;19(2):140-8. doi: 10.1002/humu.10026.
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Efficacy of unusually high doses of tranexamic acid for the treatment of epistaxis in hereditary hemorrhagic telangiectasia.超大量氨甲环酸治疗遗传性出血性毛细血管扩张症鼻出血的疗效
N Engl J Med. 2001 Sep 20;345(12):926. doi: 10.1056/NEJM200109203451216.
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Increased expression of angiogenic factors in human colonic angiodysplasia.人类结肠血管发育异常中血管生成因子表达增加。
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