The National Institute for Health and Clinical Excellence has declined to recommend eltrombopag to treat chronic idiopathic thrombocytopenic purpura.

In draft guidance issued June 10, reviewers for NICE, the clinical effectiveness agency for England and Wales, cited the drug’s high cost and a lack of clarity as to the benefits eltrombopag could provide, compared with current treatments.

Eltrombopag (Revolade, GlaxoSmithKline) is an oral medication that works by stimulating platelet production; it has marketing authorization in the United Kingdom for use in ITP patients who have undergone a splenectomy and are not responding to standard treatments.

NICE reviewers considered the results of a manufacturer-sponsored randomized, placebo-controlled controlled trial of 197 adults with ITP and platelet counts of less than 30,000/mcL (median 16,000/mcL) who had received prior treatment, including splenectomy. Patients in the eltrombopag arm had platelet counts of between 52,000 and 91,000/mcL after 6 months, and had significantly fewer episodes of bleeding; 59% were able to cease taking their other treatments. Patients receiving eltrombopag were eight times more likely to achieve an overall response of increased platelet counts of 50,000-400,000/mcL.

The NICE reviewers, while persuaded that eltrombopag was effective in improving platelet counts, nonetheless said they were concerned that the nonsplenectomized patients included in the trial “did not necessarily have contraindications to splenectomy, and may not therefore adequately represent the population for whom eltrombopag is licensed.” They also cited a “lack of direct evidence comparing the adverse effects of eltrombopag with active comparator treatments.”

The cost of the drug – between £22,020.00 and £33,030.00 per treatment year per patient – made it too expensive to recommend, NICE concluded. It was the second GlaxoSmithKline drug nixed this month because of its price; on June 9 the agency announced it would not recommend lapatinib, a drug used to treat an aggressive form of breast cancer.

NICE’s guidance on eltrombopag is not final; comments close at the end of June.

Copyright (c) 2009 Elsevier Global Medical News. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

英国国家卫生与临床优化研究所(NICE)拒绝推荐艾曲波帕(eltrombopag)用于慢性特发性血小板减少性紫癜(ITP)的治疗。

 

在6月10日发布的指南草案中，英格兰和威尔士临床疗效评价机构NICE的评审专家指出，与现有的治疗相比，艾曲波帕的价格高昂且效益尚欠明晰。

 

艾曲波帕 (Revolade，葛兰素史克)是一种通过刺激血小板生成而发挥作用的口服治疗药物；在英国，该药已获批上市用于脾切除术后经标准治疗无效的ITP患者。

 

NICE的评审专家对一项由生厂商资助的随机安慰剂对照试验的结果进行了分析。该试验纳入了197例先前曾接受过治疗(包括脾切除术)且血小板计数低于30,000/μl (中位值16,000/μl)的ITP患者。6个月后，艾曲波帕组患者的血小板计数介于52,000~91,000/μl，并且出血的发作次数显著少于安慰剂组；其中59%的患者能够停用其之前接受的其他治疗。接受艾曲波帕治疗的患者获得总体应答(即血小板计数增加至50,000~400,000/μl)的几率是安慰剂组的8倍。

 

NICE的评审专家虽然相信艾曲波帕可有效改善血小板计数，但同时也指出，他们担心试验所纳入的非脾切除患者“不一定都有接受脾切除术的禁忌证， 因此可能无法充分代表已获许可的艾曲波帕治疗人群。” 评审专家还指出，该试验“缺乏针对艾曲波帕与阳性对照药物不良反应的直接比较证据。”

 

NICE 总结道，该药的价格偏高，1名患者1年的治疗费用高达22,020 ~ 33,030英镑，价格过于昂贵而不适于推荐。这是葛兰素史克本月第2个因药价问题而惨遭拒绝的药物；6月9日，NICE宣布不会推荐拉帕替尼(lapatinib)，这是一种用于治疗侵袭性乳腺癌的药物。

 

NICE关于艾曲波帕的指南尚未最终定论；评审将于6月底结束。

 

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