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Multiple Myeloma, Gammopathies

Impact of pretransplant induction therapy on autologous stem cell transplantation for patients with newly diagnosed POEMS syndrome

Leukemia volume 31, pages 1375–1381 (2017)Cite this article

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Abstract

POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes) is a rare plasma cell dyscrasia sometimes treated with a haematopoietic cell autotransplant. We analyzed data from 138 subjects with newly diagnosed POEMS syndrome receiving a autotransplant at our center. Thirty-two subjects with severe end-organ dysfunction ineligible for immediate autotransplant received pretransplant therapy, which made a subsequent autotransplant feasible. Pretransplant therapy resulted in vascular endothelial growth factor (VEGF) remissions in 15 (47%). Thirty-three transplant recipients (24%) had early posttransplant complications. Risk factors for these complications identified through multivariate analysis included age >50 years (odds ratio (OR) 2.79, 95% confidence interval (CI) 1.09–7.14; P=0.033), time from symptom onset to transplant >5 years (OR 4.71, 95% CI 1.10–20.18; P=0.037) and pleural effusion (OR 3.39, 95% CI 1.26–9.12; P=0.016). Subjects receiving pretransplant therapy had fewer early complications than those who did not (OR 0.17, 95% CI 0.04–0.71; P=0.015), especially in subjects with a VEGF remission (OR 0.05, 95% CI 0.01–0.49; P=0.010). Autotransplants resulted in hematological remission in 60 (50%), VEGF remissions in 76 (72%) and improvements in other organ functions (65–90%). The 5-year progression-free survival (PFS) and overall survival were 76% (95% CI 64–84%) and 94% (95% CI 87-97%), respectively. Hematological (5-year PFS 83 vs 66%, P=0.008), VEGF (5-year PFS 79 vs 57%, P=0.021) remissions and especially both (5-year PFS 95 vs 61%, P=0.004) were associated with better PFS.

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Acknowledgements

We thank all the patients who participated in this study. The National Natural Science Foundation of China (Grant No. 81570195), the Beijing Natural Science Foundation (Grant No. 7142130), the Specialized Research Fund for the Doctoral Program of Higher Education (Grant No. 2013110611000), the Peking Union Medical College New Star (2011, to LJ) and the CAMS Innovation Fund for Medical Sciences (Grant No. 2016-12M-1-002) supported this research.

Author contributions

JL designed the study, recruited the patients, analyzed the data, wrote the manuscript and critically revised the manuscript. M-HD and CW were involved in study design, data collection, results’ interpretation and wrote the manuscript. X-FH, WZ, X-XC, T-NZ, J-LZ, BH and H-CC participated in patient recruitment. HC measured the serum VEGF. D-BZ supervised the study, interpreted the data and critically revised the manuscript. All authors have approved the final manuscript.

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  1. These authors contributed equally to this work.

Authors and Affiliations

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

    J Li, M-H Duan, C Wang, X-F Huang, W Zhang, X-X Cao, T-N Zhu, J-L Zhuang, B Han, H Cai, H-C Cai & D-B Zhou

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  1. J Li
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Correspondence to D-B Zhou.

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Li, J., Duan, MH., Wang, C. et al. Impact of pretransplant induction therapy on autologous stem cell transplantation for patients with newly diagnosed POEMS syndrome. Leukemia 31, 1375–1381 (2017). https://doi.org/10.1038/leu.2017.27

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