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

Saydam G., HAZNEDAROĞLU İ. C., KAYNAR L., Yavuz A. S., ALİ R., GÜVENÇ B., ...More

EXPERT OPINION ON PHARMACOTHERAPY, vol.17, no.14, pp.1851-1858, 2016 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Review
  • Volume: 17 Issue: 14
  • Publication Date: 2016
  • Doi Number: 10.1080/14656566.2016.1219338
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.1851-1858
  • Keywords: BCR-ABL1, chronic myeloid leukemia, molecular response, nilotinib, tyrosine kinase inhibitor
  • Istanbul Medipol University Affiliated: No


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-ABL1IS]) 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 MR4.5 (BCR-ABL1IS ≤ 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.