Baccarani Michele, Castagnetti Fausto, Gugliotta Gabriele, Rosti Gianantonio
Department of Hematology and Oncology "L. and A. Seràgnoli", University of Bologna, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Ann Hematol. 2015 Apr;94 Suppl 2:S141-7. doi: 10.1007/s00277-015-2322-2. Epub 2015 Mar 27.
Several guidelines and recommendations on the management of chronic myeloid leukemia (CML) have been prepared by several scientific societies. The European LeukemiaNet (ELN) appointed a panel of experts who submitted their recommendations to peer-reviewed scientific journals in 2006, 2009, and 2013. Here, we make a critical review of the last, 2013, ELN recommendations, concerning the use of the five available tyrosine kinase inhibitors (TKIs), the evaluation of cytogenetic and molecular response, and the strategy of treatment. Three TKIs (imatinib, nilotinib, dasatinib) are recommended first-line. Bosutinib and ponatinib are available second-line; ponatinib is particularly indicated in case of the T315I mutation. Achieving an optimal response, not only for survival but also for a deeper, stable, treatment-free remission, requires a BCR-ABL transcripts level ≤ 10 % at 3 months, ≤ 1 % at 6 months, ≤ 0.1 % at 1 year, and ≤ 0.01 % later on. Molecular monitoring must include mutational analysis in every case of failure. A successful treatment of accelerated and blastic phase requires TKIs, and in many cases also allogeneic stem cell transplantation.
多个科学学会已经制定了若干关于慢性髓性白血病（CML）管理的指南和建议。欧洲白血病网（ELN）指定了一个专家小组，该小组于2006年、2009年和2013年将其建议提交给同行评审的科学期刊。在此，我们对ELN 2013年的最新建议进行批判性回顾，内容涉及五种可用酪氨酸激酶抑制剂（TKIs）的使用、细胞遗传学和分子反应的评估以及治疗策略。三种TKIs（伊马替尼、尼洛替尼、达沙替尼）被推荐作为一线用药。博舒替尼和普纳替尼可作为二线用药；普纳替尼特别适用于T315I突变的情况。要实现最佳反应，不仅关乎生存，还关乎更深、更稳定的无治疗缓解，这要求在3个月时BCR-ABL转录本水平≤10%，6个月时≤1%，1年时≤0.1%，之后≤0.01%。分子监测在每个失败病例中都必须包括突变分析。加速期和急变期的成功治疗需要TKIs，在许多情况下还需要异基因干细胞移植。
