藥費是國際推薦療法的1/15，療效卻高出12個百分點,。一種以中國特色的藥物為基礎的急性白血病治療方案日前得到國際主流學術刊物的認可并發(fā)表,，可望為全球白血病患者帶來更多福音,。
《柳葉刀—腫瘤學》雜志5月9日在線發(fā)表了中國17家知名血液學?？坡?lián)合完成的一項研究。這項研究證實,，以一種名為高三尖杉酯堿的中國特色藥物為基礎,，我國醫(yī)療機構目前推廣使用的一種治療方案，可以大幅提高急性髓細胞白血病的治療效果,。
早在上個世紀70年代,，中國的醫(yī)學界就開始嘗試使用高三尖杉酯堿治療白血病。這種生物酯堿提取自中國的三尖杉屬植物,，具有明顯的抗癌作用,。在近20年的臨床研究中，醫(yī)學科研人員發(fā)現(xiàn),，以該藥物為基礎的一種簡稱為ＨＡＡ的治療方案,，對治療急性髓細胞白血病效果明顯。
5年前,，包括浙江大學醫(yī)學院附屬第一醫(yī)院,、上海瑞金醫(yī)院、天津血液病研究所在內的中國17所最權威的血液病研究和治療機構共同開展了一項多中心的隨機對照臨床研究,。研究人員對來自全國各地的高達620位急性髓細胞白血病患者進行了臨床治療調查和隨訪,。發(fā)現(xiàn)其療效大幅優(yōu)于目前普遍采用的國際推薦的ＤＡ治療方案,。
研究負責人金潔教授介紹，目前國際推薦使用的ＤＡ方案是過去40多年來,，西方發(fā)達國家在數(shù)百個臨床試驗基礎上得出的標準治療方案。雖有一定的療效,，但尋找更新,、更有效的治療手段仍是全球面臨的難題。“臨床數(shù)據(jù)顯示,，中國方案治療毒性小,，疾病完全緩解率達到73％，3年病情穩(wěn)定率為35％,，效果均明顯高于國際推薦方案12個百分點,。”
相比國際推薦方案，中國方案不僅療效更優(yōu),，還能大幅降低治療費用,。使用中國的治療方案，一個療程化療藥費支出僅為2000元左右,，而國際推薦的方案需要30000元,。中國工程院院士陳賽娟評價認為，此舉將有效減輕患者,、特別是醫(yī)療保障程度較低的發(fā)展中國家患者的醫(yī)療負擔,，從而挽救更多患者的生命。
在經(jīng)過嚴格的論文評估后,，《柳葉刀·腫瘤學》雜志不僅準予發(fā)表,，還專門邀請了美國ＭＤ Ａｎｄｅｒｓｏｎ癌癥中心白血病專家法哈德·洛凡迪教授為該論文撰寫評論,。洛凡迪教授在評論中稱,，相比國際標準ＤＡ方案，來自中國的方案因更好的治療效果而“受到醫(yī)生和患者的歡迎”,。（生物谷Bioon.com）
生物谷推薦英文摘要：
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.
