Tantravahi Srinivas K, Guthula Raga S, O'Hare Thomas, Deininger Michael W
Division of Hematology and Hematologic Malignancies, Huntsman Cancer Hospital, The University of Utah, Salt Lake City, UT, 84112, USA.
Huntsman Cancer Institute, The University of Utah, Salt Lake City, 84112, USA.
Curr Hematol Malig Rep. 2017 Oct;12(5):495-505. doi: 10.1007/s11899-017-0409-7.
BCR-ABL1 tyrosine kinase inhibitors (TKIs) have improved the prognosis of chronic phase chronic myeloid leukemia (CP-CML) to an extent that survival is largely determined by non-CML mortality. Monitoring for minimal residual disease by measuring BCR-ABL1 messenger RNA is a key component of CML management. CP-CML patients who achieve a stable deep molecular response may discontinue (TKIs) with an ~ 50% chance of entering treatment-free remission (TFR). So far discontinuation of TKIs has largely been limited to clinical trials, but is on the verge of becoming a part of wider clinical practice. Careful patient selection, dense molecular monitoring, and prompt reinstitution of treatment in the event of relapse are all vital to reproduce the same level of success. Much effort has been dedicated to identifying therapeutic strategies to eliminate CML stem cells and enable to TFR in more patients. Unfortunately, despite promising preclinical data, as yet, none of the various approaches have entered clinical practice.
BCR-ABL1酪氨酸激酶抑制剂（TKIs）已在一定程度上改善了慢性期慢性髓性白血病（CP-CML）的预后，以至于生存率很大程度上由非CML死亡率决定。通过测量BCR-ABL1信使核糖核酸来监测微小残留病是CML管理的关键组成部分。实现稳定深度分子反应的CP-CML患者停用（TKIs）后有大约50%的机会进入无治疗缓解期（TFR）。到目前为止，TKIs的停用主要限于临床试验，但即将成为更广泛临床实践的一部分。仔细的患者选择、密集的分子监测以及复发时迅速恢复治疗对于取得同样的成功水平都至关重要。人们付出了很多努力来确定消除CML干细胞并使更多患者实现TFR的治疗策略。不幸的是，尽管临床前数据很有前景，但到目前为止，各种方法都尚未进入临床实践。
