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结节性硬化症突破性疗法:西罗莫司凝胶

2018-04-23 MedSci MedSci原创

结节性硬化症(TSC)是一种罕见的顽固性疾病,TSC是由TSC1(hamartin)和TSC2(tuberin)基因中的突变引起的遗传性常染色体显性疾病。TSC1和TSC2肿瘤抑制基因中的突变引起mTORC1的超活化,能够在众多器官如脑,皮肤,肾脏和肺中发展为错构瘤的肿瘤性病变。

结节性硬化症(TSC)是一种罕见的顽固性疾病,TSC是由TSC1hamartin)和TSC2tuberin)基因中的突变引起的遗传性常染色体显性疾病。TSC1TSC2肿瘤抑制基因中的突变引起mTORC1的超活化,能够在众多器官如脑,皮肤,肾脏和肺中发展为错构瘤的肿瘤性病变。

TSC的症状包括癫痫、学习障碍、发育迟缓、自闭症和错构瘤,出血、继发性细菌感染、疼痛和与病变相关的功能紊乱导致患者极度痛苦。在各种症状中,超过90%的TSC患者出现皮肤问题,例如面部血管纤维瘤,出于美容原因严重危害他们的生活质量。然而,目前已确立的疗法仅限于手术治疗,这对于无法进行全身麻醉的儿童患者是难以实现的。

尽管西罗莫司(雷帕霉素)的全身给药能够降低TSC中的肿瘤体积,然而全身治疗可能会造成不良影响。因此,由Mari Wataya-Kaneda领导的研究小组继续研究和开发mTORC1抑制剂的局部凝胶作为安全的治疗剂,考虑局部使用西罗莫司制剂以增强有效成分的吸收。这种外用西罗莫司凝胶是目前唯一可局部应用于TSC皮肤损伤的药物,具有安全、无痛、易于使用等众多优点。


原始出处:

https://www.europeanpharmaceuticalreview.com/news/74377/sirolimus-gel-tuberous-sclerosis-complex

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    2018-04-25 lishizhe
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    2018-04-24 明月清辉

    谢谢分享.学习了

    0

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    2018-04-24 1209e435m98(暂无昵称)

    学习了.谢谢分享

    0

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研究背景•套细胞淋巴瘤(MCL)是一种侵袭性B细胞淋巴瘤,预后极差。虽然治疗不断进步,依然没有国际公认的RRMCL的治疗方案。•西罗莫司在欧洲批准用于RRMCL单药治疗,单药伊布替尼也批准用于治疗复发套细胞淋巴瘤,两者之间疗效是否存在差异目前尚无研究。研究目的•本研究目的是进行一项随机、对照、开放标签、多中心、3期研究,比较伊布替尼和西罗莫司在rr MCL患者(包含≥1种既往含利妥昔单抗化疗方案者

JACC:西罗莫司缓解心脏移植后免疫反应更佳

既往有报道称西罗莫司(SRL)在缓解心脏移植后的移植物血管病(CAV)方面的效果要优于钙调神经磷酸酶抑制剂(CNI),然而缺乏远期的疗效报道。本研究旨在评估用SRL代替CNI作为维持治疗心脏移植后的CAV远期的有效性和安全性。本研究纳入了402例心脏移植后的患者,这些患者有的仅接受CNI治疗(n = 134),有的用SRL代替了CNI作为一级免疫抑制预防治疗(n = 268),CAV通过冠脉血管内

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