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Clinical outcomes of interferon therapy for polycythemia vera and essential thrombocythemia: a systematic review and meta-analysis

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Abstract

Interferon therapy has been used in clinical practice for more than three decades to treat polycythemia vera (PV) and essential thrombocythemia (ET). However, there has been no systematic investigation of its expected outcomes and potential risks. We performed a systematic review and single-arm meta-analysis to assess the clinical outcomes (hematological response, molecular response, vascular events, hematological transformation, and adverse events) after interferon therapy for patients with PV and ET. A systematic search identified 37 reports, including data from 1794 patients that were published before March 2021. The pooled overall hematological response (OHR) rate was 86%, with better OHR rates observed in studies using long-acting interferon (p < 0.001) and studies with younger patients (p = 0.038). The pooled overall molecular response rate was 48%, and inter-study heterogeneity was also related to patient age (p = 0.009). The overall incidence was 0.42/100 person-years for thrombosis, 0.01/100 person-years for hemorrhage, 0.21/100 person-years for myelofibrotic transformation, and 0.08/100 person-years for leukemic transformation. Compared with hydroxyurea, interferon produced a non-inferior hematological response and a superior molecular response. In conclusion, interferon therapy provided high rates of hematological and molecular response for patients with PV and ET and was associated with a favorable prognosis.

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Acknowledgements

This work was supported by the National Key Research and Development Program of China (2019YFA0110802); the National Natural Science Foundation of China (81970121, 81900126, 82000136); the Beijing–Tianjin–Hebei basic research project (18JCZDJC44600, H2018206423); the Tianjin Municipal Science and Technology Commission Grant (19JCZDJC33000, 18JCQNJC11900); the Non-profit Central Research Institute Fund of Chinese Academy of Medical Sciences (2019PT310022, 2018PT31038); and the CAMS Innovation Fund for Medical Sciences (CIFMS) (2019–I2M–1–006).

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Authors and Affiliations

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

    Wenjing Gu, Renchi Yang, Zhijian Xiao & Lei Zhang

  2. Center for Stem Cell Medicine, Chinese Academy of Medical Sciences, Tianjin, 300020, China

    Wenjing Gu, Renchi Yang, Zhijian Xiao & Lei Zhang

  3. Department of Stem Cell & Regenerative Medicine, Peking Union Medical College, Tianjin, 300020, China

    Wenjing Gu, Renchi Yang, Zhijian Xiao & Lei Zhang

  4. CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China

    Wenjing Gu, Renchi Yang & Lei Zhang

  5. Tianjin Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China

    Wenjing Gu, Renchi Yang & Lei Zhang

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  1. Wenjing Gu
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  3. Zhijian Xiao
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  4. Lei Zhang
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Individual contributions were as follows: study concept and design, all authors; acquisition, selection, and interpretation of data, WG and RY; statistical analysis and drafting of the manuscript, WG; critical revision of the manuscript for important intellectual content, RY, LZ and ZX.

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Correspondence to Lei Zhang.

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Gu, W., Yang, R., Xiao, Z. et al. Clinical outcomes of interferon therapy for polycythemia vera and essential thrombocythemia: a systematic review and meta-analysis. Int J Hematol 114, 342–354 (2021). https://doi.org/10.1007/s12185-021-03171-1

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