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Sinusoidal obstruction syndrome/veno-occlusive disease after high-dose intravenous busulfan/melphalan conditioning therapy in high-risk Ewing Sarcoma

Bone Marrow Transplantation volume 53, pages 591–599 (2018)Cite this article

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Abstract

This mono-institutional observational study was conducted to determine incidence, severity, risk factors, and outcome of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in high-risk Ewing sarcoma (ES) patients treated with intravenous busulfan and melphalan (BU-MEL) followed by autologous stem cell transplantation (ASCT). During the past 10 years, 75 consecutive ES patients resulted evaluable for the analysis. After diagnosis of SOS/VOD, defibrotide therapy was started as soon as the medication was available. The variables analyzed as potential risk factors were: gender, patient's age at diagnosis, primary tumor site, disease stage, and prior radiation therapy (RT) given, focusing on RT liver exposure. The median age at diagnosis was 18.8 years. Five patients developed moderate to severe SOS/VOD (cumulative incidence, 6.67%). None of 32 pediatric patients (≤17 years) developed SOS/VOD (p = 0.0674). In univariate analysis, prior RT liver exposure resulted statistically significant (p = 0.0496). There was one death due to severe SOS/VOD. This study reports the largest series of high-risk ES patients treated with intravenous BU-MEL before ASCT. The incidence of SOS/VOD was lower when compared with other studies that used oral busulfan. Any prior RT liver exposure should be avoided. Earlier defibrotide treatment confirms to be effective.

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Acknowledgements

We thank the medical and nursing staffs who cared for these patients.

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Author notes

  1. Paioli Anna, Cammelli Silvia, Cesari Marilena, Longhi Alessandra, Palmerini Emanuela, Ferrari Stefano, Carretta Elisa, Picci Piero, and Piscaglia Fabio contributed equally to this work.

Authors and Affiliations

  1. Chemotherapy Unit, Istituto Ortopedico Rizzoli, Bologna, Italy

    Massimo Eraldo Abate, Anna Paioli, Marilena Cesari, Alessandra Longhi, Emanuela Palmerini & Stefano Ferrari

  2. Radiation Oncology Center, Department of Experimental, Diagnostic and Specialty Medicine-DIMES, University of Bologna, Bologna, Italy

    Sivlia Cammelli

  3. PhD, Istituto Ortopedico Rizzoli, Bologna, Italy

    Elisa Carretta

  4. Laboratory of Experimental Oncology, Istituto Ortopedico Rizzoli, Bologna, Italy

    Piero Picci

  5. Unit of Internal Medicine, Department of Medical and Surgical Sciences, University of Bologna, Bologna, Italy

    Fabio Piscaglia

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  1. Massimo Eraldo Abate
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Abate, M.E., Paioli, A., Cammelli, S. et al. Sinusoidal obstruction syndrome/veno-occlusive disease after high-dose intravenous busulfan/melphalan conditioning therapy in high-risk Ewing Sarcoma. Bone Marrow Transplant 53, 591–599 (2018). https://doi.org/10.1038/s41409-017-0066-4

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