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美国加州大学洛杉矶分校肯尼迪病研究诊所发给肯尼迪病人的一封信谈及注射亮丙瑞林的观点

技术组黑-阿米其检索到 美国加州大学洛杉矶分校肯尼迪病研究诊所发给所有在册肯尼迪病人的一封信.我们将其中的主要内容翻译如下: 


  From the medical side we have a new consultant, Dr Carlo Rinaldi, who has many years’ experience in KD.  Carlo runs a research group at Oxford University studying ways to develop new treatments for KD. Dr Helen Devine, a registrar, has returned from maternity leave and is joining the clinic alongside her PhD using stem cells to study KD.

   Results from Japan using testosterone-lowering drug

   Many of you have asked us about the results from a clinical trial recently published by colleagues in Japan.

    Background: Our bodies naturally produce testosterone.  In people with KD, this testosterone binds to faulty androgen receptors – and that causes damage to the nerves and muscles.  Gen Sobue’s KD lab in Japan wanted to see if less testosterone would mean less activity of the androgen receptor – and therefore less damage. There is already a drug called Leuroprelin that makes men produce less testosterone.  A team lead by the scientist Atsushi Hashizume ran a long-term trial giving Leuroprelin to some people with KD in order to reduce the levels of testosterone in their bodies, to see if they stayed healthier than others who were not given the drug.  The scientists chose people who were similar in terms of age, length of disease and CAG repeat length. The first results were published in 2009 and actually showed that there was no clear benefit to people with KD after 18m of treatment.

  Results from this study: Treatment was continued with Leuroprelin after the original study end and now the effect of the drug after up to 11.5 years in the longest-term patients has been published. Encouragingly, the researchers found that, over the observed time period, the people who were given Leuroprelin were less likely to develop pneumonia requiring hospitalisation, and they had a slower progression of disease.

Our view: The positive finding of this study is that reducing the action of testosterone can impact on the disease course of KD. On the cautionary side, however, the benefits are modest, and the drug, when taken chronically, has some side-effects.

In summary, although Leuprorelin may not prove to be clearly beneficial for people with KD, these results show that modifying testosterone can have an impact on disease, which brings optimism for future drug therapies.


   (美国加州大学洛杉矶分校肯尼迪病研究诊所给所有注册肯尼迪病人发了一封信 .信中无关的内容我们就不翻译了(主要是欢迎病人和他们联系,询问各种问题).只将重要的内容翻译如下)


    从医疗方面,我们来了一个新的顾问,卡罗博士(Carlo Rinald).他有多年研究KD的经验。卡罗同时在牛津大学办了一个研究小组,研究开发KD新的治疗方法。另外,Helen Devine博士,一位注册员,已经休完产假回来了,她正在和卡罗博士一起加入诊所,用干细胞研究KD。

   

 日本使用降睾酮药物的结果:

 你们中的许多人问我们,最近日本的同行发表的一个临床试验的结果如何。这个临床试验简述如下:

  背景:我们的身体每天都自然地产生睾酮。KD病人患病原因是因为他们的睾丸激素与雄激素受体发生了失调,从而导致神经和肌肉不断地,缓慢受损。日本的专家Sobue在日本的KD实验室,想看看睾酮的减少是否意味着雄激素受体的活性降低,从而减少这种损伤。由于有一种叫亮丙瑞林的药物能使男性产生更少的睾酮,Sobue和科学家Atsushi Hashizume领导的一个研究小组进行了一项长期试验,给一些患有KD的人注射亮丙瑞林,以降低他们体内的睾酮水平,看看他们是否比没有使用这种药物的病人病情发展变得缓慢。科学家选择了在年龄、疾病长度和CAG重复长度方面相似的人。第一项结果发表于2009年,实际上显示经过1800万次治疗后,KD患者并没有得到明显的益处。

   本研究结果:在初始研究结束后,继续使用亮丙瑞林治疗.现在该药物在长期患者中已经进行了长达11.5年注射,其效果已经发表。令人鼓舞的是,研究人员发现,在观察的时间段内,给予亮丙瑞林注射的患者疾病进展变得较慢,而且,重病人不太可能发展成需要住院的肺炎.

   我们的观点:这项研究的积极发现是确定了减少睾酮的作用可以影响KD的病程。然而,需要警示的是,注射的益处是适度的,并且长期使用该药物会产生一些副作用。

综上所述,虽然亮丙瑞林不能证明对KD患者有非常明显的益处,但这一研究结果表明改变睾酮可以影响疾病,这给未来的药物研究和治疗带来了较乐观的前景。


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1 楼 | 2018-09-22 | 回复
5 +1
江苏_勿忘我 期盼 zhang雨过天晴 閑雲㙒鶴 maxwu
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谢谢分享

2 楼 | 2018-09-22 |回复

让人很纠结的选择...

3 楼 | 01-17 |回复
谢谢阿米其和刘老师的资料分享,从资料上看,“给予亮丙瑞林注射的患者疾病进展变得较慢,而且,重病人不太可能发展成需要住院的肺炎.”即只是疾病进展变得较慢,并没有达到我们想要的结果。
手机网页 4 楼 | 01-19 |回复

用亮丙瑞林,我不喜欢

5 楼 | 01-26 |回复
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