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Cereblon expression is a prognostic marker in newly diagnosed POEMS syndrome treated with lenalidomide plus dexamethasone

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Abstract

POEMS syndrome is a rare plasma cell disorder. Lenalidomide has recently emerged as a therapeutic option for POEMS syndrome. Cereblon has been identified as the direct target of lenalidomide, and high cereblon expression is associated with better response and outcome to lenalidomide therapy in multiple myeloma patients. Here, we analyzed the predictive value of cereblon, IKZF1, and IKZF3 in CD138+ selected plasma cells from forty-one newly diagnosed POEMS syndrome patients treated with lenalidomide in combination with dexamethasone at both gene and protein levels. We found that patients with high cereblon expression tended to achieve better hematologic response compared to those with low expression (p = 0.024 for gene expression; p = 0.01 for protein expression). Multivariate Cox regression analysis revealed high cereblon mRNA expression as an independent prognostic marker for longer progression-free survival (hazard ratio 0.542; 95% CI 0.337–0.871; p = 0.011). In conclusion, our results emphasized the role of cereblon mRNA expression as a unique biomarker for predicting the clinical response and outcome of lenalidomide-based therapy in newly diagnosed POEMS syndrome patients.

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Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

Code availability

Statistical analysis was performed using the SPSS (version 23; Inc., Chicago, Illinois)

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Acknowledgements

The authors thank the patients that participated in this study.

Funding

This study was funded by Natural Science Foundation of Ningbo (Grant Number: 2019A610275).

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Authors and Affiliations

  1. Department of Hematology, Ningbo Medical Center Lihuili Hospital, Ningbo University, Ningbo, 315040, Zhejiang, China

    Qian-qian Cai & Jing Le

  2. Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China

    Xue-min Gao, Hao Zhao, Hao Cai, Xin-xin Cao & Jian Li

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  1. Qian-qian Cai
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  2. Xue-min Gao
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  3. Jing Le
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  4. Hao Zhao
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  5. Hao Cai
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  6. Xin-xin Cao
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  7. Jian Li
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Contributions

Q-Q.C was involved in study design, data collection, and analysis and performed the experiments and wrote the paper; X-M.G and H.Z collected the data and performed the experiments; Jing Le and X-X.C analyzed and interpreted the data; and H.C measured serum VEGF. Jian Li designed the study, recruited the patients, analyzed the data, wrote the manuscript, and revised the manuscript. All authors have approved the final manuscript.

Corresponding author

Correspondence to Jian Li.

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The study was approved by the local ethics committee (No. S-586) and was registered with ClinicalTrials.gov as NCT01816620.

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Informed consent was obtained from all individual participants included in the study.

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All of the authors have agreed with the publication.

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The authors declare no competing interests.

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Cai, Qq., Gao, Xm., Le, J. et al. Cereblon expression is a prognostic marker in newly diagnosed POEMS syndrome treated with lenalidomide plus dexamethasone. Ann Hematol 100, 1547–1552 (2021). https://doi.org/10.1007/s00277-021-04517-9

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  • DOI: https://doi.org/10.1007/s00277-021-04517-9

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