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日本研究组对注射亮丙瑞林的最新研究结果

日本研究组对注射亮丙瑞林的最新研究结果

技术组病友黑-阿米奇推荐了一篇日本研究组新发表的有关利用亮丙瑞林治疗肯尼迪病的论文摘要。我们不逐句翻译了,大致翻译一下,主要内容不会遗漏。

Long-term treatment with leuprorelin for spinal and bulbar muscular atrophy: natural history-controlled study

Atsushi Hashizume等9人

Abstract

Objective To evaluate the prognosis and progression of spinal and bulbar muscular atrophy (SBMA), a rare X-linked motor neuron disorder caused by trinucleotide repeat expansion in the AR (androgen receptor) gene, after long-term androgen suppression with leuprorelin acetate treatment.

Methods In the present natural history-controlled study, 36 patients with SBMA treated with leuprorelin acetate for up to 84 months (leuprorelin acetate-treated group; LT group) and 29 patients with SBMA with no specific treatment (non-treated group; NT group) were analysed. Disease progression was evaluated by longitudinal quantitative assessment of motor functioning using the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), and the modified Norris score. In addition, we selected two major clinical endpoint events, namely the occurrence of pneumonia requiring hospitalisation and death, to evaluate disease prognosis following long-term leuprorelin acetate treatment.

Results In our analysis of the longitudinal disease progression using the random slope model, we observed a significant difference in theALSFRS-R total score, the Limb Norris Score, and the Norris Bulbar Score (p=0.005, 0.026 and 0.020, respectively ), with the LT group exhibiting a slower per-12-months decline compared with the NT group. As for the event analysis, the prognosis of the LT group was better in comparison to the NT group as for the event-free survival period (p=0.021).

Conclusion Long-term treatment with leuprorelin acetate appears to delay the functional decline and suppress the incidence of pneumonia and death in subjects with SBMA.

译文:

  选择了36位肯尼迪病人接受了84个月(7年)的亮丙瑞林注射,同时选择了29个肯尼迪病人不给予亮丙瑞林的注射,以进行病情进展的对比。发现,每年都能观察到注射组比未注射组病情发展减慢。经过84个月的注射后,利用随机斜率法进行了注射组与不注射组的对比。对比采用了ALSFRS-R总分数,Limb Norris分数, 和 Norris Bulbar 分数 (p=0.005, 0.026 and 0.020)对比的方法。(我们不是专业人员,不必关心这种分数)。不仅观察到注射组病情发展的减慢,而且观察到亮丙瑞林的注射能抑制肯尼迪病人肺炎和死亡的发生率。


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1 楼 | 2017-10-24 | 回复
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zhang雨过天晴 一切随风 冰山上的来客 WY 忘忧草3264
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没有全部翻译,但主要内容给出了。
手机网页 2 楼 | 2017-10-24 |回复
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日本的卫生机构正式批准亮丙瑞林为治疗肯尼迪病的药物。但美国。欧洲仍没有响应,看来对这个药疗效的看法还是有分歧。
手机网页 3 楼 | 2017-10-25 |回复
老师,辛苦了
谢谢给我们带来最新的情报
手机网页 4 楼 | 2017-10-25 |回复
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ding

5 楼 | 07-07 |回复

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日本的卫生机构正式批准亮丙瑞林为治疗肯尼迪病的药物。但美国。欧洲仍没有响应,看来对这个药疗效的看法还是有分歧。

感谢老师,您又增加了我对抗KD的信心[微笑]
手机网页 6 楼 | 07-08 |回复
有病友在用亮丙瑞林吗?疗效到底如何啊?
手机网页 7 楼 | 07-11 |回复
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