Saydam Guray, Haznedaroglu Ibrahim Celalettin, Kaynar Leylagul, Yavuz Akif S, Ali Ridvan, Guvenc Birol, Akay Olga M, Baslar Zafer, Ozbek Ugur, Sonmez Mehmet, Aydin Demet, Pehlivan Mustafa, Undar Bulent, Dagdas Simten, Ayyildiz Orhan, Akin Gülnur, Dag Ilkiz M, Ilhan Osman
a Ege University Medical Faculty Hospital , Department of Internal Medicine , Izmir , Turkey.
b HacettepeUniversity Medical Faculty Hospital , Department of Internal Medicine , Ankara , Turkey.
Hematology. 2018 Dec;23(10):771-777. doi: 10.1080/10245332.2018.1498167. Epub 2018 Jul 11.
This report presents final results (24 months of follow-up) from the first prospective, national study of frontline nilotinib in chronic myeloid leukemia (CML) patients in Turkey.
Patients with newly diagnosed Philadelphia chromosome-positive CML in chronic phase (CML-CP; N = 112) received nilotinib 300 mg twice daily. The primary endpoint, which was the cumulative rate of major molecular response (MMR; BCR-ABL1 ≤ 0.1% on the International Scale [BCR-ABL1]) by 12 months, was previously reported (66.1% [80% CI, 59.7%-72.0%]). ClinicalTrials.gov identifier NCT01274351 Results: By 24 months, 83.0% of patients achieved MMR, and 50.9% achieved MR (BCR-ABL1 ≤0.0032%). Safety results at 24 months were consistent with those at 12 months. No additional deaths or disease progressions to accelerated phase/blast crisis were observed between 12 and 24 months.
Treatment with nilotinib 300 mg twice daily for 2 years provided high MMR with a good safety/tolerability profile in newly diagnosed CML-CP patients in Turkey. Assessment of MMR across time points showed increasing rates through 18 months, after which as lower rate of increase was observed. The safety profile of nilotinib 300 mg twice daily with 24 months of follow-up was similar to that observed at 12 months, and no new safety concerns were identified. These efficacy and safety findings are consistent with the results from the 12-month analysis of this study and from previous nilotinib studies. These findings support nilotinib as an option for frontline treatment of CML-CP.
Frontline nilotinib treatment provided sustained efficacy, with good tolerability, over 24 months in newly diagnosed CML-CP patients.
本报告展示了土耳其针对慢性髓性白血病（CML）患者进行的一线尼洛替尼前瞻性全国性研究的最终结果（24个月随访）。
新诊断为慢性期费城染色体阳性CML（CML-CP；N = 112）的患者每日两次服用300毫克尼洛替尼。主要终点是12个月时主要分子反应（MMR；国际量表上BCR-ABL1≤0.1% [BCR-ABL1]）的累积发生率，此前已有报告（66.1% [80%CI，59.7%-72.0%]）。ClinicalTrials.gov标识符NCT01274351 结果：到24个月时，83.0%的患者实现了MMR，50.9%的患者实现了MR（BCR-ABL1≤0.0032%）。24个月时的安全性结果与12个月时一致。在12至24个月期间未观察到额外死亡或疾病进展至加速期/急变期。
在土耳其新诊断的CML-CP患者中，每日两次服用300毫克尼洛替尼治疗2年可提供高MMR，且安全性/耐受性良好。对各时间点MMR的评估显示，直至18个月发生率不断上升，此后上升速率降低。每日两次服用300毫克尼洛替尼并随访24个月的安全性概况与12个月时观察到的相似，未发现新的安全问题。这些疗效和安全性发现与本研究12个月分析结果以及先前尼洛替尼研究结果一致。这些发现支持将尼洛替尼作为CML-CP一线治疗的选择。
一线尼洛替尼治疗在新诊断的CML-CP患者中24个月内提供了持续疗效，且耐受性良好。
