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

Seo Yeon Ahn, Sang Kyun Son, Gyu Hyung Lee, Inho Kim, June Won Cheong, Won Sik Lee, Byung Soo Kim, Deog Yeon Jo, Chul Won Jung, Chu Myoung Seong, Jae Hoon Lee, Young Jin Yuh, Min Kyoung Kim, Hun Mo Ryoo, Moo Rim Park, Su Hee Cho, Hoon Gu Kim, Dae Young Zang, Jinny Park, Hawk KimSeryeon Lee, Sung Hyun Kim, Myung Hee Chang, Ho Sup Lee, Chul Won Choi, Jihyun Kwon, Sung Nam Lim, Suk Joong Oh, Inkyung Joo, Dong Wook Kim

Research output: Contribution to journalArticlepeer-review

1 Citation (Scopus)


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.

Original languageEnglish
Pages (from-to)144-151
Number of pages8
JournalBlood Research
Issue number2
Publication statusPublished - 2022 Jun 1
Externally publishedYes


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

ASJC Scopus subject areas

  • Hematology


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