South Australian Health and Medical Research Institute, Division of Haematology and Centre for Cancer Biology, South Australia Pathology, University of Adelaide, Adelaide, Australia;
Princess Margaret Hospital, Leukemia Clinic, Blood and Marrow Transplant Centre, Apheresis Unit, Toronto, ON, Canada;
Blood. 2014 Jul 31;124(5):729-36. doi: 10.1182/blood-2013-12-544015. Epub 2014 Jun 19.
Patients in complete cytogenetic response (CCyR) with detectable BCR-ABL1 after ≥2 years on imatinib were randomized to nilotinib (400 mg twice daily, n = 104) or continued imatinib (n = 103) in the Evaluating Nilotinib Efficacy and Safety in clinical Trials-Complete Molecular Response (ENESTcmr) trial. By 1 and 2 years, confirmed undetectable BCR-ABL1 was achieved by 12.5% vs 5.8% (P = .108) and 22.1% vs 8.7% of patients in the nilotinib and imatinib arms, respectively (P = .0087). Among patients without molecular response 4.5 (BCR-ABL1(IS) ≤0.0032%; MR(4.5)) and those without major molecular response at study start, MR(4.5) by 2 years was achieved by 42.9% vs 20.8% and 29.2% vs 3.6% of patients in the nilotinib and imatinib arms, respectively. No patient in the nilotinib arm lost CCyR, vs 3 in the imatinib arm. Adverse events were more common in the nilotinib arm, as expected with the introduction of a new drug vs remaining on a well-tolerated drug. The safety profile of nilotinib was consistent with other reported studies. In summary, switching to nilotinib enabled more patients with chronic myeloid leukemia in chronic phase (CML-CP) to sustain lower levels of disease burden vs remaining on imatinib. This trial was registered at www.clinicaltrials.gov as #NCT00760877.
在伊马替尼治疗≥2 年后达到完全细胞遗传学缓解（CCyR）且可检测到 BCR-ABL1 的患者中，随机分配至尼洛替尼（400mg，每日 2 次，n = 104）或继续伊马替尼治疗（n = 103），这是在Evaluating Nilotinib Efficacy and Safety in clinical Trials-Complete Molecular Response（ENESTcmr）试验中进行的。在 1 年和 2 年时，尼洛替尼和伊马替尼组分别有 12.5%和 5.8%（P =.108）以及 22.1%和 8.7%的患者达到确认的不可检测 BCR-ABL1（P =.0087）。在无分子学反应的患者中（BCR-ABL1（IS）≤0.0032%；MR（4.5））和无分子学反应的起始时主要分子学反应的患者中，在 2 年时，尼洛替尼组有 42.9%和 29.2%的患者分别达到 MR（4.5），而伊马替尼组分别为 20.8%和 3.6%。尼洛替尼组无患者丧失 CCyR，而伊马替尼组有 3 例。预期引入新药会导致比继续使用耐受良好的药物更常见的不良反应。尼洛替尼的安全性特征与其他已报道的研究一致。总之，与继续使用伊马替尼相比，转换为尼洛替尼使更多慢性髓性白血病慢性期（CML-CP）患者能够维持较低水平的疾病负担。该试验在 www.clinicaltrials.gov 上注册为 #NCT00760877。
