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Clinical features and current treatment status of essential thrombocythemia in older adults: a multicenter real-world study in China

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Abstract

Approximately half of patients diagnosed with essential thrombocythemia (ET) are older adults (aged ≥ 60 years), but to date, little is known about the clinical and molecular characteristics of older patients diagnosed according to the 2016 World Health Organization criteria. We retrospectively collected clinical and molecular data from 282 older (≥ 60 years) and 621 younger ET patients (18–59 years) in China from March 1, 2012 to November 1, 2021 and summarized the clinical characteristics and treatment of these older ET patients. Compared to younger patients, older patients had a higher incidence of the JAK2V617F mutation (P = 0.001), a lower incidence of CALR mutations (P = 0.033) and a higher rate of epigenetic mutations (P < 0.001), TP53 mutations (P = 0.005), and RNA splicing mutations (P < 0.001). Older patients had not only a higher incidence of thrombosis but also a higher incidence of bleeding events. Furthermore, older patients had a significantly higher mortality rate after disease progression (P = 0.050) or after thrombotic events (P = 0.013). Risk factors for thrombosis or prognosis were significantly different between older patients and the entire ET cohort. In older patients, non-driver mutations contributed significantly to thrombotic complications and a poor prognosis, while the JAK2V617F mutation was a risk factor for overall survival but not for thrombotic events. The application of interferon in older ET patients was not inferior to that of hydroxyurea in terms of efficacy and safety. Older patients presented unique characteristics different from those of younger patients, which could provide new information for formulating more appropriate treatment and follow-up strategies.

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Data availability

The data sets generated and analyzed during the current study are available from the corresponding authors upon reasonable request.

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Acknowledgements

This project was supported by grants from the CAMS Innovation Fund for Medical Sciences (CIFMS) (2021-I2M-1-073), the National Natural Science Foundation of China (81600099, 81970121, 82000136, 82270152, 82000127), National Key Research and Development Program of China (2019YFA0110802), Tianjin Municipal Science and Technology Commission Grant (18JCQNJC11900), CIFMS (2022-I2M-2-003, 2021-I2M-1-003), the Non-profit Central Research Institute Fund of the Chinese Academy of Medical Sciences (2020-PT310-011), and Haihe Laboratory of Cell Ecosystem Innovation Fund (HH22KYZX0022), and Clinical Research Fund of National Center for Clinical Medical Research of Hematology Diseases (2023NCRCA0109).

Funding

This project was supported by grants from the CAMS Innovation Fund for Medical Sciences (CIFMS) (2021-I2M-1–073), the National Natural Science Foundation of China (81600099, 81970121, 82000136, 82270152, 82000127), National Key Research and Development Program of China (2019YFA0110802), Tianjin Municipal Science and Technology Commission Grant (18JCQNJC11900), CIFMS (2022-I2M-2–003, 2021-I2M-1–003), the Non-profit Central Research Institute Fund of the Chinese Academy of Medical Sciences (2020-PT310-011), Haihe Laboratory of Cell Ecosystem Innovation Fund (22HHXBSS00022), and Clinical Research Fund of National Center for Clinical Medical Research of Hematology Diseases (2023NCRCA0109).

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Author notes

  1. Rongfeng Fu, Huan Dong and Donglei Zhang contributed equally to this work.

Authors and Affiliations

  1. State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, 300020, China

    Rongfeng Fu, Huan Dong, Xiaofan Liu, Feng Xue, Wei Liu, Yunfei Chen, Ting Sun, Mankai Ju, Xinyue Dai, Renchi Yang & Lei Zhang

  2. Tianjin Institutes of Health Science, Tianjin, 301600, China

    Rongfeng Fu, Huan Dong, Xiaofan Liu, Feng Xue, Wei Liu, Yunfei Chen, Ting Sun, Mankai Ju, Xinyue Dai, Renchi Yang & Lei Zhang

  3. Department of Hematology, Zhongnan Hospital of Wuhan University, Wuhan, 430071, Hubei, China

    Donglei Zhang & Xian Zhang

  4. Department of Hematology, The Affiliated Cancer Hospital of Zhengzhou University & Henan Cancer Hospital, Hemostasis and Thrombosis Diagnostic Engineering Research Center of Henan Province, Zhengzhou, 450008, Henan, China

    Hu Zhou

  5. Department of Hematology, The First Affiliated Hospital of Xiamen University, Xiamen, 361000, Fujian, China

    Yueting Huang

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  1. Rongfeng Fu
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Contributions

RFF, HD, and DLZ contributed equally to this study and collected data, analyzed data, and wrote the paper. LZ and RCY designed the study, analyzed data, and reviewed the manuscript. TS, MKJ, and XYD collected data and analyzed data. XFL, FX, YFC, WL, XZ, YTH and HZ helped with data collection.

Corresponding authors

Correspondence to Renchi Yang or Lei Zhang.

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Ethics approval

This study has been approved by the Institutional Ethics Committee of each participating center.

Patient consent

This study obtained the informed written consent of the patients.

Conflict of interest

The authors declare no competing financial interests.

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Rongfeng Fu, Huan Dong and Donglei Zhang should be regarded as cofirst authors.

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Fu, R., Dong, H., Zhang, D. et al. Clinical features and current treatment status of essential thrombocythemia in older adults: a multicenter real-world study in China. Ann Hematol 102, 2097–2107 (2023). https://doi.org/10.1007/s00277-023-05317-z

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