一种廉价、可预防老年人视觉丧失的药物被证明安全而有效,但目前没有获得批准。研究发表在《英国医学杂志》上。
贝伐单抗(阿瓦斯丁)被准许用于治疗肠癌,但它作为一种非常廉价的药物被广泛替代兰尼 (Lucentis) 用来预防湿性老年性黄斑变性(AMD),比较这两种药物的几项大型研究目前正在进行中。
尽管当前这项研究没有包括兰尼(在实验开始时,该药没有被批准使用),但研究人员赞成在一些通过预算限制患者使用兰尼的医疗机构使用这种药物。世界上大多数国家,或者对湿性老年性黄斑变性没办法治疗或者治疗效果不好,适当使用具有成本低高效益的阿瓦斯丁,将会对减少因该病而引发的失明的发病率产生直接的影响。
湿性老年性黄斑变性是导致北美洲和欧洲50岁以上老年人视力丧失的主要原因。视力丧失是由异常损害和血管渗漏导致的眼睛后面光感细胞逐渐丧失造成的。这些患者不是失明,但实际上会发现,他们不能阅读、驾驶和从事细微、敏锐、需要中央视觉的工作。
2006年,英国三家眼科中心的研究人员开始着手研究阿瓦斯丁相比英国国民保健系统(NHS)所采用的治疗来说,是否是一种安全有效、可以治疗湿性老年性黄斑变性的药物。
研究人员对131名年龄在50岁以上、患有AMD的患者随机进行为期6周的阿瓦斯丁注射,或者进行标准的治疗(NHS批准的三种不同治疗的一种),并在治疗的开始和一年以后对他们的视敏度进行测量。一年后,阿瓦斯丁治疗组有32%的患者在基础视敏度基础上增加了15个字母,而常规治疗组只有3%。在阿瓦斯丁治疗组中,视敏度损失少于15个字母的比例(91%)明显高于标准治疗组(67%)。相比标准治疗组下降了9.7个字母来说,阿瓦斯丁治疗组在平均注射阿瓦斯丁7次情况下他们的平均视敏度提高了 7个字母,并且从开始出现改善的第18周一直持续到第54周。阿瓦斯丁治疗伴随着较低的严重不良反应。
这些研究结果证明,在研究初期,对AMD进行6周的阿瓦斯丁注射,其效果优于现有的标准治疗。这项研究为阿瓦斯丁治疗湿性老年性黄斑变性提供了第一手证据。
英国皇家维多利亚医院教授乌莎 查克拉瓦蒂在随后的评论中说,虽然这一试验填补了证据上的空白,并明确证明阿瓦斯丁好于过去的治疗方法,但还不能告诉我们阿瓦斯丁是否会像兰尼那样有效。她还警告说,“在进行这两种药物比较的大型随机研究,公布他们的研究结果之前,不鼓励不按说明使用阿瓦斯丁。”(生物谷Bioon.net)
生物谷推荐原文出处:
BMJ 2010;340:c2459, doi: 10.1136/bmj.c2459 (Published 10 June 2010)
Bevacizumab for neovascular age related macular degeneration (ABC Trial): multicentre randomised double masked study
Adnan Tufail, Praveen J Patel, Catherine Egan, Philip Hykin, Lyndon da Cruz, Zdenek Gregor, Jonathan Dowler, Mohammed A Majid, Clare Bailey, Quresh Mohamed, Robert Johnston, Catey Bunce, Wen Xing, and ABC Trial Investigators
1 Moorfields Eye Hospital NHS Foundation Trust, London EC1V 2PD, 2 Bristol Eye Hospital, Bristol, BSL 2LX, 3 Gloucestershire Eye Department, Cheltenham General Hospital, Cheltenham GL53 7AN
Objectives To evaluate the efficacy and safety of intravitreous bevacizumab injections for the treatment of neovascular age related macular degeneration.
Design Prospective, double masked, multicentre, randomised controlled trial.
Setting Three ophthalmology centres in the United Kingdom.
Participants 131 patients (mean age 81) with wet age related macular degeneration randomised 1:1 to intervention or control.
Interventions Intravitreous bevacizumab (1.25 mg, three loading injections at six week intervals followed by further treatment if required at six week intervals) or standard treatment available at the start of the trial (photodynamic treatment with verteporfin for predominantly classic type neovascular age related macular degeneration, or intravitreal pegaptanib or sham treatment for occult or minimally classic type neovascular age related macular degeneration).
Main outcome measures Primary outcome: proportion of patients gaining 15 letters of visual acuity at one year (54 weeks). Secondary outcomes: proportion of patients with stable vision and mean change in visual acuity.
Results Of the 131 patients enrolled in the trial, five patients did not complete the study because of adverse events, loss to follow-up, or death. In the bevacizumab group, 21 (32%) patients gained 15 or more letters from baseline visual acuity compared with two (3%) in the standard care group (P<0.001); the estimated adjusted odds ratio was 18.1 (95% confidence interval 3.6 to 91.2) and the number needed to treat was 4 (3 to 6). In addition, the proportion of patients who lost fewer than 15 letters of visual acuity from baseline was significantly greater among those receiving bevacizumab treatment (91% (59) v 67% (44) in standard care group; P<0.001). Mean visual acuity increased by 7.0 letters in the bevacizumab group with a median of seven injections compared with a decrease of 9.4 letters in the standard care group (P<0.001), and the initial improvement at week 18 (plus 6.6 letters) was sustained to week 54. Among 65 patients treated with bevacizumab, there were no cases of endophthalmitis or serious uveitis related to the intervention. All end points with respect to visual acuity in the study eye at 54 weeks favoured bevacizumab treatment over standard care.
Conclusions Bevacizumab 1.25 mg intavitreous injections given as part of a six weekly variable retreatment regimen is superior to standard care (pegaptanib sodium, verteporfin, sham), with low rates of serious ocular adverse events. Treatment improved visual acuity on average at 54 weeks.