Outcomes with frontline nilotinib treatment in Turkish patients with newly diagnosed Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase

ABSTRACT

Objective: 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^IS]) by 12 months.

Methods: 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.

Results and Conclusions: Of 112 patients enrolled, 66.1% (80% CI, 59.7–72.0%) achieved MMR and 22.3% achieved a deep molecular response of MR^4.5 (BCR-ABL1^IS ≤ 0.0032%) by 12 months. During the first year of treatment, 1 patient progressed to blast crisis and 2 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.

KEYWORDS:

• BCR-ABL1
• chronic myeloid leukemia
• molecular response
• nilotinib
• tyrosine kinase inhibitor

Declaration of interest

In collaboration with the study investigators, the study sponsor (Novartis Pharmaceuticals Corporation) participated in the design of the study and the data analyses and interpretation.

G Saydam has received honoraria from Novartis, Celgene, Bristol-Myers Squibb, and Gilead; served on speakers bureaus for Novartis and Bristol-Myers Squibb, and served as a consultant for Novartis. IC Haznedaroglu has received research funding from, and served on advisory committees for, Novartis. AS Yavuz has served on advisory committees for Novartis. DZ Akkaynak and IM Dag are employees of Novartis. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Writing assistance was utilised in the preparation of this manuscript; it was funded by Novartis and carried out by Karen Kaluza, PhD, and Joy Loh, PhD (both of ArticulateScience LLC).

Additional information

Funding

This study was funded by Novartis Pharmaceuticals Corporation.