药费是国际推荐疗法的1/15，疗效却高出12个百分点。一种以中国特色的药物为基础的急性白血病治疗方案日前得到国际主流学术刊物的认可并发表，可望为全球白血病患者带来更多福音。
《柳叶刀—肿瘤学》杂志5月9日在线发表了中国17家知名血液学专科联合完成的一项研究。这项研究证实，以一种名为高三尖杉酯碱的中国特色药物为基础，我国医疗机构目前推广使用的一种治疗方案，可以大幅提高急性髓细胞白血病的治疗效果。
早在上个世纪70年代，中国的医学界就开始尝试使用高三尖杉酯碱治疗白血病。这种生物酯碱提取自中国的三尖杉属植物，具有明显的抗癌作用。在近20年的临床研究中，医学科研人员发现，以该药物为基础的一种简称为ＨＡＡ的治疗方案，对治疗急性髓细胞白血病效果明显。
5年前，包括浙江大学医学院附属第一医院、上海瑞金医院、天津血液病研究所在内的中国17所最权威的血液病研究和治疗机构共同开展了一项多中心的随机对照临床研究。研究人员对来自全国各地的高达620位急性髓细胞白血病患者进行了临床治疗调查和随访。发现其疗效大幅优于目前普遍采用的国际推荐的ＤＡ治疗方案。
研究负责人金洁教授介绍，目前国际推荐使用的ＤＡ方案是过去40多年来，西方发达国家在数百个临床试验基础上得出的标准治疗方案。虽有一定的疗效，但寻找更新、更有效的治疗手段仍是全球面临的难题。“临床数据显示，中国方案治疗毒性小，疾病完全缓解率达到73％，3年病情稳定率为35％，效果均明显高于国际推荐方案12个百分点。”
相比国际推荐方案，中国方案不仅疗效更优，还能大幅降低治疗费用。使用中国的治疗方案，一个疗程化疗药费支出仅为2000元左右，而国际推荐的方案需要30000元。中国工程院院士陈赛娟评价认为，此举将有效减轻患者、特别是医疗保障程度较低的发展中国家患者的医疗负担，从而挽救更多患者的生命。
在经过严格的论文评估后，《柳叶刀·肿瘤学》杂志不仅准予发表，还专门邀请了美国ＭＤ Ａｎｄｅｒｓｏｎ癌症中心白血病专家法哈德·洛凡迪教授为该论文撰写评论。洛凡迪教授在评论中称，相比国际标准ＤＡ方案，来自中国的方案因更好的治疗效果而“受到医生和患者的欢迎”。（生物谷Bioon.com）
生物谷推荐英文摘要：
The Lancet Oncology DOI： 10.1016/S1470-2045(13)70152-9
Homoharringtonine-based induction regimens for patients with de-novo acute myeloid leukaemia: a multicentre, open-label, randomised, controlled phase 3 trial
Jie Jin etc.
Background
Homoharringtonine-based induction regimens have been widely used in China for patients with acute myeloid leukaemia. However, their efficacy has not been tested in a multicentre randomised controlled trial in a large population. We assessed the efficacy and safety of homoharringtonine-based induction treatment for management of newly diagnosed acute myeloid leukaemia.
Methods
This open-label, randomised, controlled, phase 3 study was done in 17 institutions in China between September, 2007, and July, 2011. Untreated patients aged 14—59 years with acute myeloid leukaemia were randomly assigned (by a computer-generated allocation schedule without stratification) to receive one of three induction regimens in a 1:1:1 ratio: homoharringtonine 2 mg/m2 per day on days 1—7, cytarabine 100 mg/m2 per day on days 1—7, and aclarubicin 20 mg/day on days 1—7 (HAA); homoharringtonine 2 mg/m2 per day on days 1—7, cytarabine 100 mg/m2 per day on days 1—7, and daunorubicin 40 mg/m2 per day on days 1—3 (HAD); or daunorubicin 40—45 mg/m2 per day on days 1—3 and cytarabine 100 mg/m2 per day on days 1—7 (DA). Patients in complete remission were offered two cycles of intermediate-dose cytarabine (2 g/m2 every 12 h on days 1—3). The primary endpoints were the proportion of patients who achieved complete remission after two cycles of induction treatment and event-free survival in the intention-to-treat population. The trial is registered in the Chinese Clinical Trial Register, number ChiCTR-TRC-06000054.
Findings
We enrolled 620 patients, of whom 609 were included in the intention-to-treat analysis. 150 of 206 patients (73%) in the HAA group achieved complete remission versus 125 of 205 (61%) in the DA group (p=0·0108); 3-year event-free survival was 35·4% (95% CI 28·6—42·2) versus 23·1% (95% CI 17·4—29·3; p=0·0023). 133 of 198 patients (67%) in the HAD group had complete remission (vs DA, p=0·20) and 3-year event-free survival was 32·7% (95% CI 26·1—39·5; vs DA, p=0·08). Adverse events were much the same in all groups, except that more patients in the HAA (12 of 206 [5·8%]) and HAD (13 of 198 [6·6%]) groups died within 30 days than in the DA group (two of 205 [1%]; p=0·0067 vs HAA; p=0·0030 vs HAD).
Interpretation
A regimen of homoharringtonine, cytarabine, and aclarubicin is a treatment option for young, newly diagnosed patients with acute myeloid leukaemia.
Funding
Chinese National High Tech Programme, Key Special Research Foundation of the Ministry of Science and Technology of China, National Nature Science Foundation of China, National Clinical Key Specialty Construction Project.
