造血系统恶性肿瘤(hematopoietic malignancy,HM)除了传统治疗方法外，各种生物治疗方法也发挥着重要作用。其中依据白血病分化障碍的特点，可以采用不同诱导分化剂进行定向诱导分化，即在不同诱导分化剂的作用下，定向诱导分化为粒系细胞、单核/巨噬细胞和DC细胞等。其次，针对HM发病机制中的关键致病基因、蛋白质或细胞膜抗原分子等，可以设计新型靶向抗体等药物。再有，鉴于白血病细胞分化障碍与DNA甲基化和染色体组蛋白乙酰化、去乙酰化异常有关，部分学者在研究白血病相关基因表观遗传学调控基础上，探讨了DNA甲基化和组蛋白乙酰化调节药物的治疗作用。另外，针对特异性肿瘤抗原或肽的肿瘤疫苗、细胞因子及其受体介导的靶向治疗，以及相对靶向性的细胞载体治疗都呈现出较好的应用前景。因此，无论是定向诱导分化，抑或是针对关键基因和分子的靶向药物，都为HM的治疗提供了新的手段，它们单独或者与其他治疗方法的联合使用，将明显提高HM的治疗效果。

In addition to the conventional therapeutic strategies, biotherapy also plays an important part in the treatment of hematopoietic malignancy (HM). According to the differentiation disorders of leukemia cells, HM should be treated by different differentiation inducers, which may induce them to differentiate into granulocytes, monocytes or DCs. Secondly, based on the key disease-associated genes, proteins, and cell surface antigen molecules in the pathogenesis of HM, new anti-HM drugs such as target antibody can be designed. Thirdly, based on the relationship of abnormalities of DNA methylation, chromosomes histone acetylation and histone deacetylation with the differentiation disorder of leukemia cells, some researchers studied the epigenetic modification of leukemia-associated genes and the therapeutic effects of anti-HM drugs regulating DNA methylation and histone acetylation. Last, tumor antigen or peptide specific tumor vaccines, cytokine/receptors-mediated target therapy, and partially-targeted cell vector-based therapy also have potential clinical applications. In conclusion, the differentiation inducers, key genes and molecule-targeted drugs and other target therapy agents all provide new ways for the treatment of HM; these agents used alone or in combination with other therapies will greatly enhance the treatment outcomes of HM.

国家自然科学基金资助项目（No. 30771103，No. 30810444）；国家“十一五”重大新药创制科技重大专项子课题（No. 2009ZX09503-010）；山东省科技攻关重大项目（No. 2007GG2002023）