Departments of Leukemia and Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
J Clin Oncol. 2010 Jan 20;28(3):392-7. doi: 10.1200/JCO.2009.25.4896. Epub 2009 Dec 14.
Although most patients with chronic myeloid leukemia (CML) in chronic phase respond well to front-line therapy with imatinib, some patients do not achieve the desirable end point, and others may eventually lose response or are intolerant.
Patients with newly diagnosed CML in chronic phase were treated with nilotinib 400 mg twice daily on an empty stomach as initial therapy.
Among 51 patients in chronic phase observed for at least 3 months, 50 (98%) achieved a complete cytogenetic remission (CCyR), and 39 (76%) achieved a major molecular response (MMR). Responses occurred rapidly, with 96% of patients achieving CCyR by 3 months and 98% achieving CCyR by 6 months. The projected event-free survival at 24 months is 90%, and all patients are alive after a median follow-up time of 17 months. Grade >or= 3 neutropenia occurred in 12% of patients, and thrombocytopenia in occurred 11%. Nonhematologic toxicity was usually grade 1 to 2 and manageable. The actual median dose at 12 months was 800 mg (range, 200 to 800 mg).
Nilotinib is an effective option for the initial management of CML in early chronic phase, producing high rates of CCyR and MMR, with most patients reaching these responses early during their therapy.
虽然大多数慢性髓性白血病（CML）慢性期患者对伊马替尼一线治疗有良好反应，但仍有部分患者无法达到理想终点，还有部分患者最终可能会失去反应或不耐受。
新诊断为 CML 慢性期的患者接受尼洛替尼 400 mg 每日两次空腹作为初始治疗。
在至少观察 3 个月的 51 例慢性期患者中，50 例（98%）达到完全细胞遗传学缓解（CCyR），39 例（76%）达到主要分子缓解（MMR）。反应迅速，96%的患者在 3 个月时达到 CCyR，98%的患者在 6 个月时达到 CCyR。24 个月时无事件生存率为 90%，中位随访 17 个月后所有患者均存活。12%的患者发生>或=3 级中性粒细胞减少症，11%的患者发生血小板减少症。非血液学毒性通常为 1 级至 2 级，且易于管理。实际中位剂量为 12 个月时为 800 mg（范围为 200 至 800 mg）。
尼洛替尼是早期慢性期 CML 初始治疗的有效选择，可产生高 CCyR 和 MMR 率，大多数患者在治疗早期即可达到这些反应。
