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Treatment of refractory or relapsed myelodysplastic neoplasms with luspatercept: a multicenter Chinese study

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Abstract

Few effective therapies are available to treat patients with relapsed/refractory myelodysplastic neoplasms (MDS). Luspatercept was shown to display good efficacy in a phase 3 clinical trial for lower-risk MDS (LR-MDS) patients, yet real-world data are limited, especially in China. Therefore, data from patients diagnosed as having MDS with low blasts and SF3B1 mutation (MDS-SF3B1) and MDS with SF3B1 mutation and thrombocytosis were retrospectively analyzed. Of the 23 enrolled patients, 17 (73.9%) were males (median age 67 years: range 29 to 80 years). Previously, a total of 22 (95.7%) patients had received recombinant human erythropoietin (rhEPO), 9 (39.1%) roxadustat, 7 (30.4%) lenalidomide and 3 (13.0%) hypomethylating agents (HMA). The median treatment time was 22.9 weeks (9.0–32.4). At week 12, 60.9% (14/23) of the patients achieved a hematologic improvement–erythroid (HI-E) response. Red blood cell transfusion independence (RBC-TI) for ≥ 8 weeks was found in 57.1% (8/14) of transfusion-dependent patients. The median hemoglobin concentration was 84 g/L, and patients had significantly higher hemoglobin concentrations after 12 weeks of treatment (P < 0.001). It is noteworthy that responders had a greater reduction in serum ferritin (P = 0.021). Those with serum EPO < 500 IU/L at baseline tended to have a higher HI-E rate (P = 0.081), but only patients in non-transfusion and low transfusion burden (LTB) subgroups had statistical differences (P = 0.024). The most commonly occurring adverse events were blood bilirubin increase (17.4%), fatigue (13.0%) and dizziness (13.0%). Luspatercept was effective and tolerated well in refractory LR-MDS-SF3B1 patients. In particular, baseline non-transfusion and LTB patients exhibited a greater response rate to treatment.

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Data availability

The datasets are available from the corresponding author upon reasonable request.

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Acknowledgements

We appreciate every patient who allowed us to access their medical records and responded to our review telephone calls. We appreciate all the centers for providing cases and assisting with our follow-up.

Funding

The National High Level Hospital Clinical Research Funding (2022-PUMCH-C-026, 2022-PUMCH-D-002, 2022-PUMCH-B-046) CAMS, Innovation Fund for Medical Sciences (CIFMS 2021-I2M-1–003) and Beijing Natural Science Foundation (7232109).

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Author notes

  1. Zhuxin Zhang and Qinglin Hu contributed equally.

Authors and Affiliations

  1. Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China

    Zhuxin Zhang, Qinglin Hu, Chen Yang, Miao Chen & Bing Han

  2. Department of Hematology, Chinese Academy of Chinese Medical Science, Xiyuan Hospital, Beijing, China

    Xudong Tang

  3. Department of Hematology, Union Hospital Tongji Medical College Huazhong University of Science and Technology, Wuhan, China

    Min Zhang

  4. Peking University Institute of Hematology, Peking University People’s Hospital, Beijing, China

    Jinsong Jia & Hongxia Shi

  5. Department of Hematology, Dongfang Hospital Beijing University of Chinese Medicine, Beijing, China

    Xiaoqing Ding

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  1. Zhuxin Zhang
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Contributions

Conceptualization: Zhuxin Zhang, Bing Han; Formal analysis and investigation: Zhuxin Zhang; Qinglin Hu; Writing – preparation of the first draft of the manuscript: Zhuxin Zhang; Writing, reviewing and editing: Zhuxin Zhang, Bing Han; Funding acquisition: Bing Han; Resources: Xudong Tang, Min Zhang, Jinsong Jia, Hongxia Shi, Xiaoqing Ding, Chen Yang, Miao Chen, Bing Han; Supervision: Bing Han.

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Correspondence to Bing Han.

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All procedures followed were in accordance with guidelines of the ethics committee of Peking Union Medical College Hospital (project number: K3531) and the revised Helsinki Declaration of 2008 (5).

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Consent was provided by all patients who took part in the study.

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Zhang, Z., Hu, Q., Tang, X. et al. Treatment of refractory or relapsed myelodysplastic neoplasms with luspatercept: a multicenter Chinese study. Ann Hematol 102, 3039–3047 (2023). https://doi.org/10.1007/s00277-023-05334-y

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