Venetoclax in combination with azacitidine in Japanese patients with acute myeloid leukaemia: Phase 1 trial findings

Shuichi Taniguchi, Takahiro Yamauchi, Ilseung Choi, Noriko Fukuhara, Jalaja Potluri, Ahmed Hamed Salem, Wan Jen Hong, Hideyuki Honda, Yasuko Nishimura, Sumiko Okubo, Kensuke Usuki

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5 Citations (Scopus)


Background: Venetoclax plus azacitidine is indicated in the USA for the treatment of newly diagnosed acute myeloid leukaemia in older patients (≥75 years) or those ineligible for induction chemotherapy due to co-morbidities. Methods: In this phase 1/2 study (NCT02265731), Japanese patients (≥60 years) with untreated (ineligible for induction chemotherapy) or relapsed/refractory acute myeloid leukaemia received oral venetoclax 400 mg/day (3-day ramp up in cycle 1) plus subcutaneous or intravenous azacitidine 75 mg/m2on days 1-7 per 28-day cycle until disease progression or unacceptable toxicity. Results: As of 10 December 2019, six patients were enrolled (median age: 75 years; untreated: n = 5; relapsed/refractory: n = 1); median treatment duration: 10.3 months (range, 0.7-29.4). Most common grade ≥ 3 adverse events were lymphopaenia and febrile neutropaenia (n = 4 each). Four patients reported serious adverse events; only an event of grade 3 fungal pneumonia was considered possibly related to both study drugs, requiring dose interruption of venetoclax and delay of azacitidine. Five (83%) patients had responses (complete remission: n = 3). Median time to first response of complete remission/complete remission with incomplete count recovery was 1.0 month (range, 0.8-5.5); median overall survival: 15.7 months (95% confidence interval: 6.2, not reached). Conclusions: Venetoclax plus azacitidine was well tolerated and showed high response rates in Japanese patients with acute myeloid leukaemia.

Original languageEnglish
Pages (from-to)857-864
Number of pages8
JournalJapanese Journal of Clinical Oncology
Issue number6
Publication statusPublished - 2021 Jun 1


  • AML
  • Anti-neoplastic agents
  • apoptosis
  • BCL-2
  • Japan


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