近期，北京大學(xué)血液病研究所黃曉軍教授課題組在惡性血液病診療方面取得的一系列創(chuàng)新性研究成果，三篇學(xué)術(shù)論文發(fā)表在國(guó)際腫瘤學(xué)頂級(jí)期刊《Journal of Clinical Oncology》(影響因子18.038),，國(guó)際血液學(xué)頂級(jí)期刊《Blood》(影響因子9.06)、《Leukemia》(影響因子10.164),。
急性早幼粒白血病是一種起病兇險(xiǎn)的惡性血液病,，單純依賴化療患者復(fù)發(fā)率高，總體生存較差。北京大學(xué)血液病研究所組織全國(guó)七家血液病中心開展口服砷劑（復(fù)方黃黛片）和靜脈砷劑（亞砷酸）治療急性早幼粒細(xì)胞白血病的多中心前瞻性隨機(jī)對(duì)照試驗(yàn),，口服砷劑和靜脈砷劑組患者3年總體存活率分別達(dá)到99.1%和96.6%,，在國(guó)際上首次通過(guò)前瞻臨床試驗(yàn)證實(shí)口服砷劑和靜脈砷劑具有相似的療效和安全性。這項(xiàng)研究成果有希望促成急性早幼粒白血病從住院治療到門診治療的重大革命,，改善患者預(yù)后,、提高生活質(zhì)量并降低醫(yī)療費(fèi)用。該論文第一作者為北京大學(xué)血液病研究所主鴻鵠副主任醫(yī)師（參看鏈接1）,。
伴有t(8,；21)的急性髓性白血病是一種常見惡性血液病，國(guó)際權(quán)威指南認(rèn)為該類型疾病屬于預(yù)后良好類型,，首選大劑量化療治療,，而非異基因造血干細(xì)胞移植。黃曉軍課題組發(fā)現(xiàn),，如果僅依賴大劑量化療,，復(fù)發(fā)率達(dá)45%-50%，因此需要早期識(shí)別高危復(fù)發(fā)患者并采取更為有效的治療,。課題組利用實(shí)時(shí)定量PCR動(dòng)態(tài)監(jiān)測(cè)微小殘留病,，據(jù)此建立新的分層治療體系。對(duì)于低危病人選擇大劑量化療,，而對(duì)于高危病人選擇異基因造血干細(xì)胞移植,，使復(fù)發(fā)率從45%-50%下降到15%，5年生存率由50%-65%提高到82.7%,，達(dá)到了目前國(guó)際最好的療效,。國(guó)際急性髓性白血病協(xié)作組（AMLSG）主席、Annals of Hematology主編Arnold Ganser教授等國(guó)際一線專家對(duì)該研究成果寄予厚望,，表示將根據(jù)此項(xiàng)研究結(jié)果積極推進(jìn)相應(yīng)國(guó)際指南的修訂,。該論文第一作者為北京大學(xué)血液病研究所主鴻鵠副主任醫(yī)師（參看鏈接2）。
慢性粒細(xì)胞白血?。＃┓譃槁云?、加速期、急變期,，其治療選擇一直是惡性血液病領(lǐng)域研究的熱點(diǎn),。黃曉軍課題組2012發(fā)現(xiàn)造血干細(xì)胞移植治療加速期高危慢粒患者較伊馬替尼治療具有顯著的生存優(yōu)勢(shì),。國(guó)際上慢粒慢性期的治療選擇一直缺乏循證醫(yī)學(xué)證據(jù)級(jí)別較高的臨床研究,。黃曉軍課題組在國(guó)際上首次通過(guò)前瞻臨床試驗(yàn)證實(shí)，新診斷慢粒慢性期患者伊馬替尼治療較HLA相合造血干細(xì)胞移植具有生存優(yōu)勢(shì),，此項(xiàng)成果進(jìn)一步豐富了慢粒診療體系,，推動(dòng)了慢粒治療最優(yōu)策略選擇。該論文第一作者為北京大學(xué)血液病研究所江倩主任醫(yī)師（參看鏈接3）。
黃曉軍課題組繼2012年在造血干細(xì)胞移植研究中取得系列重要成果后,，本年度在急性白血病化療研究領(lǐng)域也開始引領(lǐng)國(guó)際先進(jìn)水平,。這些系列研究得到國(guó)家自然科學(xué)基金重點(diǎn)項(xiàng)目、國(guó)家“863”,、“973”基金,、教育部長(zhǎng)江學(xué)者獎(jiǎng)勵(lì)計(jì)劃、“科技北京”百名領(lǐng)軍人才,、北京市重點(diǎn)實(shí)驗(yàn)室階梯計(jì)劃,、北京大學(xué)-清華大學(xué)生命科學(xué)聯(lián)合中心等項(xiàng)目的資助。（生物谷Bioon.com）
生物谷推薦英文摘要：
Journal of Clinical Oncology doi：10.1200/JCO.2013.48.8312
Oral Tetra-Arsenic Tetra-Sulfide Formula Versus Intravenous Arsenic Trioxide As First-Line Treatment of Acute Promyelocytic Leukemia： A Multicenter Randomized Controlled Trial
Hong-Hu Zhu,， De-Pei Wu,， Jie Jin， Jian-Yong Li,， Jun Ma,， Jian-Xiang Wang， Hao Jiang,， Sai-Juan Chen and Xiao-Jun Huang？
Purpose This randomized,， multicenter,， phase III noninferiority trial was designed to test the efficacy and safety of an oral tetra-arsenic tetra-sulfide (As4S4) –containing formula named the Realgar-Indigo naturalis formula (RIF) compared with intravenous arsenic trioxide (ATO) as both induction and maintenance therapies for newly diagnosed acute promyelocytic leukemia (APL).
Patients and Methods In all， 242 patients with APL were randomly assigned (1：1) to oral RIF (60 mg/kg) or ATO (0.16 mg/kg) combined with all-trans retinoic acid (ATRA,； 25 mg/m2) during induction therapy. After achieving complete remission (CR),， all patients received three courses of consolidation chemotherapy and maintenance treatment with sequential ATRA followed by either RIF or ATO for 2 years. The primary end point was the rate of disease-free survival (DFS) at 2 years， which was assessed for noninferiority with a 10% noninferiority margin.
Results The median follow-up time was 39 months. DFS at 2 years was 98.1% (106 of 108) in the RIF group and 95.5% (107 of 112) in the ATO group. The DFS difference was 2.6% (95% CI,， ,？3.0% to 8.0%). The lower limit of the 95% CI of DFS difference was greater than the ？10% noninferiority margin,， confirming noninferiority (P < .001). No significant differences were noted between the RIF and ATO groups with regard to the CR rate (99.1% v 97.2%,； P = .62) or the overall survival at 3 years (99.1% v 96.6%； P = .18). The rates of adverse events were similar in the two groups.
Conclusion Oral RIF plus ATRA is not inferior to intravenous ATO plus ATRA as first-line treatment of APL and may be considered as a routine treatment option for appropriate patients.
2. MRD-directed risk stratification treatment may improve outcomes of t(8;21) AML in the first complete remission: results from the AML05 multicenter trial. Blood. 2013 May 16;121(20):4056-62.
http://www.ncbi.nlm.nih.gov/pubmed/23535063
3. Imatinib results in better outcomes than HLA-identical sibling transplants in young persons with newly diagnosed chronic-phase chronic myelogenous leukemia. Leukemia. 2013 May 23. [Epub ahead of print]
http://www.ncbi.nlm.nih.gov/pubmed/23698276
