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<p>This phase 1/2 study investigated the association between genetic characteristics and outcomes for NS-87/CPX-351 in Japanese patients with high-risk acute myeloid leukemia. Blood samples collected from 29 patients were analyzed using a 70-gene next-generation sequencing panel. The most frequently mutated genes were <i>TP53</i> (44.8%), <i>TET2</i> (24.1%), <i>DNMT3A</i> (13.8%), and <i>NRAS</i> (13.8%). The rates of composite complete remission (CRc; complete remission [CR] or CR with incomplete hematologic recovery [CRi]) were comparable between patients with and without mutations in <i>TP53, TET2</i>, <i>DNMT3A</i>, and <i>NRAS</i> (<i>P</i> = 0.571 for all). Notably, patients with <i>TP53</i> mutations had a similar CRc rate (69.2% vs. 56.3%), but shorter overall survival (OS), event-free survival (EFS), and relapse-free survival (RFS) compared to patients with wild-type <i>TP53</i> (median OS: 7.43 vs. 18.18&#xa0;months; <i>P</i> = 0.108, median EFS: 2.43 vs. 6.28&#xa0;months; <i>P</i> = 0.012, median RFS: 1.48 vs. 10.19&#xa0;months; <i>P</i> = 0.012). In conclusion, no gene mutations directly associated with the efficacy of NS-87/CPX-351 were found. While NS-87/CPX-351 achieved remission even in patients with <i>TP53</i> mutations, relapse risk was higher in these patients. Therefore, it is advisable to consider treatment strategies such as early transplantation after achieving remission with NS-87/CPX-351, especially in patients with <i>TP53</i> mutations.</p>

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Association between gene mutations and outcomes in Japanese high-risk AML patients: a phase 1/2 study of NS-87/CPX-351

  • 期刊论文

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  • 发表日期：

    2025年2月27日

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  • Hideki Makishima,
  • Taisuke Mikasa,
  • Kento Isogaya,
  • Toshihiro Miyamoto,
  • Takuji Yamauchi,
  • Akira Yokota,
  • Masahiro Onozawa,
  • Kiyoshi Ando,
  • Yoshiaki Ogawa,
  • Kensuke Usuki,
  • Takahiro Yamauchi,
  • Shuichi Ota,
  • Satoru Takada,
  • Yasuyoshi Morita,
  • Takayuki Ishikawa,
  • Katsuto Takenaka,
  • Junya Kuroda,
  • Naohiro Sekiguchi,
  • Toshiro Kawakita,
  • Yasushi Miyazaki

摘要

This phase 1/2 study investigated the association between genetic characteristics and outcomes for NS-87/CPX-351 in Japanese patients with high-risk acute myeloid leukemia. Blood samples collected from 29 patients were analyzed using a 70-gene next-generation sequencing panel. The most frequently mutated genes were TP53 (44.8%), TET2 (24.1%), DNMT3A (13.8%), and NRAS (13.8%). The rates of composite complete remission (CRc; complete remission [CR] or CR with incomplete hematologic recovery [CRi]) were comparable between patients with and without mutations in TP53, TET2, DNMT3A, and NRAS (P = 0.571 for all). Notably, patients with TP53 mutations had a similar CRc rate (69.2% vs. 56.3%), but shorter overall survival (OS), event-free survival (EFS), and relapse-free survival (RFS) compared to patients with wild-type TP53 (median OS: 7.43 vs. 18.18 months; P = 0.108, median EFS: 2.43 vs. 6.28 months; P = 0.012, median RFS: 1.48 vs. 10.19 months; P = 0.012). In conclusion, no gene mutations directly associated with the efficacy of NS-87/CPX-351 were found. While NS-87/CPX-351 achieved remission even in patients with TP53 mutations, relapse risk was higher in these patients. Therefore, it is advisable to consider treatment strategies such as early transplantation after achieving remission with NS-87/CPX-351, especially in patients with TP53 mutations.