Saydam Guray, Haznedaroglu Ibrahim C, 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, Akkaynak Diyar Z, Akin Gulnur, İlhan Osman
a Department of Internal Medicine , Ege University Medical Faculty Hospital , Izmir , Turkey.
b Department of Internal Medicine , Hacettepe University Medical Faculty Hospital , Ankara , Turkey.
Hematology. 2018 Feb 27:1-7. doi: 10.1080/10245332.2018.1444919.
Nilotinib is a BCR-ABL1 tyrosine kinase inhibitor approved for the treatment of patients with chronic myeloid leukemia in chronic phase (CML-CP). This study was the first prospective evaluation of the efficacy and safety of nilotinib in Turkish patients with newly diagnosed CML-CP. The primary endpoint of the study was the rate of major molecular response (MMR; BCR-ABL1 ≤ 0.1% on the International Scale [BCR-ABL1]) by 12 months.
Patients with newly diagnosed CML-CP were treated with nilotinib 300 mg twice daily. This analysis was based on the first 12 months of follow-up in a 24-month study. This study is registered with ClinicalTrials.gov (NCT01274351).
Of 112 patients enrolled, 66.1% (80% CI, 59.7-72.0%) achieved MMR and 22.3% achieved a deep molecular response of MR (BCR-ABL1 ≤0.0032%) by 12 months. During the first year of treatment, one patient progressed to blast crisis and two patients died. Safety results were consistent with previous studies. Most adverse events (AEs) were grade 1/2. Most frequently reported nonhematologic AEs of any grade were elevations in bilirubin, alanine aminotransferase, and triglycerides.
These results support the use of nilotinib 300 mg twice daily as a standard-of-care treatment option for patients with newly diagnosed CML-CP with low and intermediate risk.
尼洛替尼是一种获批用于治疗慢性期慢性髓性白血病（CML-CP）患者的BCR-ABL1酪氨酸激酶抑制剂。本研究是对尼洛替尼在土耳其新诊断CML-CP患者中的疗效和安全性进行的首次前瞻性评估。该研究的主要终点是12个月时的主要分子反应率（MMR；国际量表[BCR-ABL1]上BCR-ABL1≤0.1%）。
新诊断的CML-CP患者接受尼洛替尼每日两次、每次300 mg的治疗。该分析基于一项为期24个月研究的前12个月随访。本研究已在ClinicalTrials.gov注册（NCT01274351）。
在112名入组患者中，66.1%（80%CI，59.7-72.0%）在12个月时达到MMR，22.3%达到MR深度分子反应（BCR-ABL1≤0.0032%）。在治疗的第一年，1例患者进展为急变期，2例患者死亡。安全性结果与既往研究一致。大多数不良事件（AE）为1/2级。任何级别的最常报告的非血液学AE是胆红素、丙氨酸转氨酶和甘油三酯升高。
这些结果支持将每日两次、每次300 mg的尼洛替尼作为新诊断的低危和中危CML-CP患者的标准治疗选择。
