Perrotti Danilo, Silvestri Giovannino, Stramucci Lorenzo, Yu Justine, Trotta Rossana
Marlene and Stewart Greenebaum Cancer Center, University of Maryland School of Medicine, Baltimore, MD 21201, United States.
Curr Drug Targets. 2017;18(4):377-388. doi: 10.2174/1389450117666160615074120.
The use of imatinib, second and third generation ABL tyrosine kinase inhibitors (TKI) (i.e. dasatinib, nilotinib, bosutinib and ponatinib) made CML a clinically manageable and, in a small percentage of cases, a cured disease. TKI therapy also turned CML blastic transformation into a rare event; however, disease progression still occurs in those patients who are refractory, not compliant with TKI therapy or develop resistance to multiple TKIs. In the past few years, it became clear that the BCRABL1 oncogene does not operate alone to drive disease emergence, maintenance and progression. Indeed, it seems that bone marrow (BM) microenvironment-generated signals and cell autonomous BCRABL1 kinase-independent genetic and epigenetic alterations all contribute to: i. persistence of a quiescent leukemic stem cell (LSC) reservoir, ii. innate or acquired resistance to TKIs, and iii. progression into the fatal blast crisis stage. Herein, we review the intricate leukemic network in which aberrant, but finely tuned, survival, mitogenic and self-renewal signals are generated by leukemic progenitors, stromal cells, immune cells and metabolic microenvironmental conditions (e.g. hypoxia) to promote LSC maintenance and blastic transformation.
伊马替尼、第二代和第三代ABL酪氨酸激酶抑制剂(TKI)(即达沙替尼、尼洛替尼、博舒替尼和普纳替尼)的使用使慢性粒细胞白血病成为一种临床上可控制的疾病,并且在一小部分病例中可治愈。TKI治疗还使慢性粒细胞白血病的急变转化成为罕见事件;然而,在那些难治、不依从TKI治疗或对多种TKI产生耐药性的患者中,疾病仍会进展。在过去几年中,很明显BCRABL1致癌基因并非单独发挥作用来驱动疾病的发生、维持和进展。事实上,骨髓(BM)微环境产生的信号以及细胞自主的BCRABL1激酶非依赖性遗传和表观遗传改变似乎都导致:i. 静止白血病干细胞(LSC)库的持续存在,ii. 对TKI的固有或获得性耐药,以及iii. 进展到致命的急变期。在此,我们综述了复杂的白血病网络,其中白血病祖细胞、基质细胞、免疫细胞和代谢微环境条件(如缺氧)产生异常但精细调节的存活、促有丝分裂和自我更新信号,以促进LSC的维持和急变转化。