机构：[1]Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, Peking University, 100044, Beijing.[2]Shanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital affiliated to Shanghai Jiao Tong University School of Medicine, 200025, Shanghai.[3]Department of Hematology, Union hospital, Tongji Medical College, Huazhong University of Science and Technology, 430022, Wuhan.华中科技大学同济医学院附属协和医院[4]Department of Hematology, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, 519041, Guangzhou.广东省人民医院[5]Department of Hematology, Second Hospital of Shanxi Medical University, Taiyuan.[6]National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 030001, Tianjing.[7]Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, 530021, Guangxi.[8]Medical Center of Hematology, Xinqiao Hospital, State Key Laboratory of Trauma, Burn and Combined Injury, Army Medical University, 400030, Chongqing.[9]Peking University People's Hospital, 266111, Qingdao.[10]Department of Hematology, Henan Cancer Hospital, The Affiliated Cancer Hospital of Zhengzhou University, 450008, Henan.河南省肿瘤医院[11]Department of Hematology, Xijing Hospital, 710000, Shanxi.[12]Department of Hematology, The Affiliated Hospital of Qingdao University, 266000, Qingdao.[13]Department of Hematology, The Affiliated Hospital of Inner Mongolia Medical University, 010030, Inner Mongolia.[14]Department of Hematology, The First Hospital of Jilin University, 130031, Jilin.[15]Department of Hematology, Tongji Hospital of Tongji Medical College, Tongji Medical College of Huazhong University of Science and Technology, 430030, Wuhan.华中科技大学同济医学院附属同济医院[16]Department of Hematology, Weifang People's Hospital, 261041, Weifang.[17]Department of Hematology, Sichuan Academy of Medical Sciences Sichuan Provincial People's Hospital, 610072, Sichuan.四川省人民医院[18]Department of Hematology, Jining No.1 People's Hospital, 272011, Jining.[19]Department of Hematology, First Affiliated Hospital of Kunming Medical University, Hematology Research Center of Yunnan Province, 650032, Kunming.内科科室血液内科昆明医科大学附属第一医院[20]Anshan Central Hospital, 114001, Anshan.[21]Centre for Haematology, Department of Immunology and Inflammation, Imperial College London, SW72az, London, United Kingdom.[22]Peking-Tsinghua Center for Life Sciences, Academy for Advanced Interdisciplinary Studies, 100871, Peking University, Beijing.
We studied 130 consecutive subjects who presented with (n = 29) or transformed to (n = 101) accelerated phase chronic myeloid leukemia (CML) and who received olverembatinib. 62 were in 2nd chronic phase. All failed ≥ 1 tyrosine kinase-inhibitors (TKIs) and 91 had BCR::ABL1T315I. Median follow-up was 28 months (InterQuartile Range, 10-74 months). The 6-year cumulative incidences of major cytogenetic response (MCyR), complete cytogenetic response (CCyR), major molecular response (MMR) and molecular response 4.0 (MR4.0) were 59% (95% Confidence Interval [CI] (49, 69%), 53% (42, 62%), 52% (41, 62%) and 42% (31, 53%), respectively. The 6-year probabilities of transformation-free survival (TFS), CML-related survival and survival were 81% (72, 90%), 76% (67, 87%) and 71% (61, 82%), respectively. In multi-variable analyses, an interval from diagnosis of CML to olverembatinib start < 29 months, failure to achieve complete hematologic response (CHR) on prior TKI therapy, hemoglobin concentration < 98 g/L, blood and/or bone marrow blasts ≥ 8%, and/or high-risk additional chromosome abnormalities at the start of olverembatinib therapy, as well as not achieving early MCyR on olverembatinib correlated with worse outcomes. RUNX1 and STAT5A variants were significantly associated with worse TFS in the 82 subjects with targeted DNA sequencing data. There were acceptable treatment-related adverse events. We conclude olverembatinib is effective and tolerable in subjects in accelerated phase CML failing prior TKI therapy.
基金：
Funded, in part, by the National Natural Science Foundation of China
(No. 81970140 and No. 82370161) to QJ.
语种：
外文
PubmedID：
中科院(CAS)分区：
出版当年[2026]版：
无
最新[2025]版：
大类|1 区医学
小类|2 区血液学
第一作者：
第一作者机构：[1]Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, Peking University, 100044, Beijing.
共同第一作者：
通讯作者：
通讯机构：[1]Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, Peking University, 100044, Beijing.[9]Peking University People's Hospital, 266111, Qingdao.
推荐引用方式(GB/T 7714)：
Yuan Mengyao,Zhou Li,Li Weiming,et al.Olverembatinib in accelerated-phase chronic myeloid leukemia: efficacy and safety evaluation[J].Haematologica.2025,doi:10.3324/haematol.2025.288249.
APA：
Yuan Mengyao,Zhou Li,Li Weiming,Du Xin,Weng Jianyu...&Jiang Qian.(2025).Olverembatinib in accelerated-phase chronic myeloid leukemia: efficacy and safety evaluation.Haematologica,,
MLA：
Yuan Mengyao,et al."Olverembatinib in accelerated-phase chronic myeloid leukemia: efficacy and safety evaluation".Haematologica .(2025)
医学