近期，北京大学血液病研究所黄晓军教授课题组在恶性血液病诊疗方面取得的一系列创新性研究成果，三篇学术论文发表在国际肿瘤学顶级期刊《Journal of Clinical Oncology》(影响因子18.038)，国际血液学顶级期刊《Blood》(影响因子9.06)、《Leukemia》(影响因子10.164)。
急性早幼粒白血病是一种起病凶险的恶性血液病，单纯依赖化疗患者复发率高，总体生存较差。北京大学血液病研究所组织全国七家血液病中心开展口服砷剂（复方黄黛片）和静脉砷剂（亚砷酸）治疗急性早幼粒细胞白血病的多中心前瞻性随机对照试验，口服砷剂和静脉砷剂组患者3年总体存活率分别达到99.1%和96.6%，在国际上首次通过前瞻临床试验证实口服砷剂和静脉砷剂具有相似的疗效和安全性。这项研究成果有希望促成急性早幼粒白血病从住院治疗到门诊治疗的重大革命，改善患者预后、提高生活质量并降低医疗费用。该论文第一作者为北京大学血液病研究所主鸿鹄副主任医师（参看链接1）。
伴有t(8；21)的急性髓性白血病是一种常见恶性血液病，国际权威指南认为该类型疾病属于预后良好类型，首选大剂量化疗治疗，而非异基因造血干细胞移植。黄晓军课题组发现，如果仅依赖大剂量化疗，复发率达45%-50%，因此需要早期识别高危复发患者并采取更为有效的治疗。课题组利用实时定量PCR动态监测微小残留病，据此建立新的分层治疗体系。对于低危病人选择大剂量化疗，而对于高危病人选择异基因造血干细胞移植，使复发率从45%-50%下降到15%，5年生存率由50%-65%提高到82.7%，达到了目前国际最好的疗效。国际急性髓性白血病协作组（AMLSG）主席、Annals of Hematology主编Arnold Ganser教授等国际一线专家对该研究成果寄予厚望，表示将根据此项研究结果积极推进相应国际指南的修订。该论文第一作者为北京大学血液病研究所主鸿鹄副主任医师（参看链接2）。
慢性粒细胞白血病（慢粒）分为慢性期、加速期、急变期，其治疗选择一直是恶性血液病领域研究的热点。黄晓军课题组2012发现造血干细胞移植治疗加速期高危慢粒患者较伊马替尼治疗具有显著的生存优势。国际上慢粒慢性期的治疗选择一直缺乏循证医学证据级别较高的临床研究。黄晓军课题组在国际上首次通过前瞻临床试验证实，新诊断慢粒慢性期患者伊马替尼治疗较HLA相合造血干细胞移植具有生存优势，此项成果进一步丰富了慢粒诊疗体系，推动了慢粒治疗最优策略选择。该论文第一作者为北京大学血液病研究所江倩主任医师（参看链接3）。
黄晓军课题组继2012年在造血干细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疗研究领域也开始引领国际先进水平。这些系列研究得到国家自然科学基金重点项目、国家“863”、“973”基金、教育部长江学者奖励计划、“科技北京”百名领军人才、北京市重点实验室阶梯计划、北京大学-清华大学生命科学联合中心等项目的资助。（生物谷Bioon.com）
生物谷推荐英文摘要：
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
