Bone marrow is the primary hematopoietic organ, which is involved in multiple malignant diseases including acute and chronic leukemia, multiple myeloma, myelodysplastic syndromes, and bone metastases from solid tumors. These malignancies affect normal homeostasis and reshape the bone marrow microenvironment. There are limited treatment options for them because of their inevitable aggravation. The current systemic administration of anticancer agents is difficult to achieve ideal therapeutic dose to suppress tumor growth at bone marrow diseased sites, and is always associated with a high incidence of relapse and severe side effects. The limitations of current treatments urge scientists to develop bone marrow targeted drug delivery systems intended for the treatment of diseased bone marrow, which can improve the efficacy of therapeutic agents and reduce their dose-limiting systemic side effects on healthy tissues. In this review we first present the current opinions on bone marrow vasculature, as well as the molecular and structural interactions between tumor cells and the diseased bone marrow. In the second part, we highlight the different design rationales and strategies of bone marrow delivery systems and their therapeutic applications for the treatment of malignancies inside the bone marrow.

骨髓是主要的造血器官，涉及多种恶性疾病，包括急性和慢性白血病，多发性骨髓瘤，骨髓增生异常综合症以及实体瘤引起的骨转移。这些恶性肿瘤会影响正常的体内平衡并重塑骨髓微环境。由于它们不可避免的加重，它们的治疗选择有限。当前的全身性抗癌剂给药难以达到理想的治疗剂量来抑制骨髓病变部位的肿瘤生长，并且总是与复发率高和严重的副作用有关。当前治疗方法的局限性促使科学家开发用于治疗患病骨髓的靶向骨髓的药物输送系统，可以提高治疗剂的功效，并减少其对健康组织的剂量限制性全身性副作用。在这篇综述中，我们首先介绍有关骨髓脉管系统的最新观点，以及肿瘤细胞与患病骨髓之间的分子和结构相互作用。在第二部分中，我们重点介绍了骨髓输送系统的不同设计原理和策略及其在骨髓内恶性肿瘤治疗中的治疗应用。