Yilmaz Musa, Abaza Yasmin, Jabbour Elias
Department of Hematology and Oncology, Baylor College of Medicine, Houston, TX, USA.
Curr Hematol Malig Rep. 2015 Jun;10(2):145-57. doi: 10.1007/s11899-015-0254-5.
With the discovery of Philadelphia chromosome, understanding of chronic myeloid leukemia (CML) pathobiology has tremendously increased. Development of tyrosine kinase inhibitors (TKI) targeting the BCR/ABL1 oncoprotein has changed the landscape of the disease. Today, the expected survival of CML patients, if properly managed, is likely to be similar to the general population. Imatinib is the first-approved TKI in CML treatment, and for several years, it was the only option in the frontline setting. Four years ago, second-generation TKIs (nilotinib and dasatinib) were approved as alternative frontline options. Now, clinicians are faced the challenge of making decision for which TKI to chose upfront. Second-generation TKIs have been demonstrated to induce deeper and faster responses compared to imatinib; however, none of three TKIs have been shown to have a clear survival advantage, they all are reasonable options. In contrast, when considering therapy in individual patients, the case may be stronger for a specific TKI. Co-morbidities of the patient and side effect profile of the TKI of interest should be an important consideration in decision making. At present, the cost nilotinib or dasatinib is not remarkably different from imatinib. However, patent for imatinib is expected to expire soon, and it will be available as a generic. Clinicians, then, need to weigh the advantages some patients gain with nilotinib or dasatinib in the frontline setting against the difference in cost. Whatever TKI is chosen as frontline, intolerance, non-compliance, or treatment failure should be recognized early as a prompt intervention increases the chance of achieving best possible response.
随着费城染色体的发现,对慢性髓性白血病(CML)病理生物学的认识有了极大的提高。针对BCR/ABL1癌蛋白的酪氨酸激酶抑制剂(TKI)的开发改变了该疾病的治疗格局。如今,如果管理得当,CML患者的预期生存期可能与普通人群相似。伊马替尼是CML治疗中首个获批的TKI,并且在数年时间里,它是一线治疗的唯一选择。四年前,第二代TKI(尼罗替尼和达沙替尼)获批作为替代一线治疗选择。现在,临床医生面临着决定 upfront选择哪种TKI的挑战。与伊马替尼相比,第二代TKI已被证明能诱导更深、更快的反应;然而,这三种TKI均未显示出明显的生存优势,它们都是合理的选择。相比之下,在考虑个体患者的治疗时,对于特定的TKI可能有更充分的理由。患者的合并症以及所关注的TKI的副作用情况应是决策中的重要考虑因素。目前,尼罗替尼或达沙替尼的成本与伊马替尼并无显著差异。然而,伊马替尼的专利预计很快到期,届时它将作为仿制药上市。那么,临床医生需要权衡一些患者在一线治疗中使用尼罗替尼或达沙替尼所获得的优势与成本差异。无论选择哪种TKI作为一线治疗,都应尽早识别不耐受、不依从或治疗失败情况,因为及时干预会增加实现最佳反应的机会。