Blood Res.  2022 Jun;57(2):144-151. 10.5045/br.2022.2021137.

Safety and efficacy of nilotinib in adult patients with chronic myeloid leukemia: a post-marketing surveillance study in Korea

Affiliations
  • 1Department of Hematology-Oncology, Chonnam National University Hwasun Hospital, Hwasun, Korea
  • 2Department of Hematology-Oncology, School of Medicine, Kyungpook National University, Daegu, Korea
  • 3Department of Internal Medicine, 3 University of Ulsan College of Medicine, Asan Medical Center, Seoul, Korea
  • 4Department of Internal Medicine, Seoul National University Hospital, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea
  • 5Division of Hematology, Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea
  • 6Department of Internal Medicine, Inje University Busan Paik Hospital, Busan, Korea
  • 7Division of Hematology, Department of Internal Medicine, Korea University Anam Hospital, Seoul, Korea
  • 8Division of Hematology, Department of Internal Medicine, College of Medicine, Chungnam National Univeristy, Daejeon, Korea
  • 9Division of Hematology, Department of Internal Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea
  • 10Department of Hematology and Oncology, Ewha Womans University College of Medicine, Seoul, Korea
  • 11Division of Hematology, Gachon University Gil Medical Center, Gachon University College of Medicine, Incheon, Korea
  • 12Department of Internal Medicine, Inje University, Sanggye-Paik Hospital, Seoul, Korea
  • 13Department of Internal Medicine, Yeungnam University College of Medicine, Daegu, Korea
  • 14Division of Hematology-Oncology, Department of Internal Medicine, Daegu Catholic University Medical Center, Daegu, Korea
  • 15Department of Hematology-Oncology, Wonkwang University School of Medicine, Iksan, Korea
  • 16Division of Hematology-Oncology, Department of Internal Medicine, Pusan National University Yangsan Hospital, Yangsan, Korea
  • 17Department of Internal Medicine, Gyeongsang Institute of Health Sciences, Gyeongsang National University College of Medicine and Gyeongsang National University Changwon Hospital, Changwon, Korea
  • 18Department of Internal Medicine, Hallym University Sacred Heart Hospital, Anyang, Korea
  • 19Department of Internal Medicine, Korea University Ansan Hospital, Ansan, Korea
  • 20Department of Internal Medicine, Dong-A University College of Medicine, Busan, Korea
  • 21Division of Oncology-Hematology, Department of Internal Medicine, National Health Insurance Service Ilsan Hospital, Goyang, Korea
  • 22Division of Oncology-Hematology, Department of Internal Medicine, Kosin University College of Medicine, Busan, Korea
  • 23Department of Internal Medicine, Korea University Guro Hospital, Seoul, Korea
  • 24Department of Internal Medicine, Chungbuk National University College of Medicine, Cheongju, Korea
  • 25Department of Internal Medicine, Inje University College of Medicine, Haeundae Paik Hospital, Busan, Korea
  • 26Department of Internal Medicine, Kangbuk Samsung Hospital, Sungkyunkwan University School of Medicine, Seoul, Korea
  • 27Novartis Korea Ltd., Seoul, Korea
  • 28Department of Hematology, Uijeongbu Eulji Medical Center, Leukemia Omics Research Institute, Eulji University Uijeongbu Campus, Uijeongbu, Korea

Abstract

Background
Nilotinib is a tyrosine kinase inhibitor approved by the Ministry of Food and Drug Safety for frontline and 2nd line treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). This study aimed to confirm the safety and efficacy of nilotinib in routine clinical practice within South Korea.
Methods
An open-label, multicenter, single-arm, 12-week observational post-marketing surveillance (PMS) study was conducted on 669 Korean adult patients with Ph + CML from December 24, 2010, to December 23, 2016. The patients received nilotinib treatment in routine clinical practice settings. Safety was evaluated by all types of adverse events (AEs) during the study period, and efficacy was evaluated by the complete hematological response (CHR) and cytogenetic response.
Results
During the study period, AEs occurred in 61.3% (410 patients, 973 events), adverse drug reactions (ADRs) in 40.5% (271/669 patients, 559 events), serious AEs in 4.5% (30 patients, 37 events), and serious ADRs in 0.7% (5 patients, 8 events). Furthermore, unexpected AEs occurred at a rate of 6.9% (46 patients, 55 events) and unexpected ADRs at 1.2% (8 patients, 8 events). As for the efficacy results, CHR was achieved in 89.5% (442/494 patients), and minor cytogenetic response or major cytogenetic response was achieved in 85.8% (139/162 patients).
Conclusion
This PMS study shows consistent results in terms of safety and efficacy compared with previous studies. Nilotinib was well tolerated and efficacious in adult Korean patients with Ph + CML in routine clinical practice settings.

Keyword

Nilotinib; Tyrosine kinase inhibitor; Chronic myeloid leukemia; Philadelphia chromosome positive; Post-marketing surveillance

Figure

  • Fig. 1 Study flow chart.Abbreviation: CBC, complete blood count.

  • Fig. 2 Distribution of AE incidence by diagnosis.Abbreviations: AE, adverse events; AP, accelerated phase; CP, chronic phase.

  • Fig. 3 The incidence of adverse events (AEs) by categories. (A) AEs have been classified and evaluated along five severity categories: mild, moderate, severe, life threatening consequences, and death. (B) Action taken due to AEs has been evaluated along six categories as: none, permanent discontinuation, temporary discontinuation, dose reduction, dose escalation, and not applicable. (C) The incidence of AEs by sex. (D) The incidence of AEs by age.


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