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Defibrotide-treated patients with anicteric or icteric veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic cell transplantation: an EBMT study

Bone Marrow Transplantation volume 57, pages 664–667 (2022)Cite this article

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Severe hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of hematopoietic cell transplantation (HCT) [1]. It is estimated that 5–14% of patients who undergo HCT develop VOD/SOS [2]. Without therapeutic intervention, a high mortality rate (>80%) is observed in patients with VOD/SOS and multi-organ dysfunction/failure (MOD/MOF) [1].

Hyperbilirubinemia (bilirubin >2 mg/dL) is a key criterion for VOD/SOS diagnosis; however, a considerable number of patients with VOD/SOS may not develop hyperbilirubinemia. In an expanded-access study, 23% of patients had bilirubin <2 mg/dL at VOD/SOS diagnosis, with anicteric VOD/SOS occurring in 15% of adult and 29% of pediatric patients [3]. Recent diagnostic guidelines acknowledge, but do not require, elevated bilirubin for VOD/SOS diagnosis [4,5,6].

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Fig. 1: Outcomes by bilirubin levels at diagnosis.

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All relevant data are provided within the manuscript and supporting files.

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Acknowledgements

This study was supported by Jazz Pharmaceuticals. Medical writing and editorial assistance were provided by Erica Chevalier-Larsen, PhD, CMPP™, of Cello Health Communications/SciFluent Communications, Inc., and were financially supported by Jazz Pharmaceuticals.

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Authors and Affiliations

  1. Department of Clinical Haematology and Cellular Therapy, Hôpital St Antoine, Sorbonne University, INSERM UMRs 938, Paris, France

    Mohamad Mohty

  2. Hôpital de L’Archet, Nice, France

    Thomas Cluzeau

  3. CHU Bordeaux Groupe Hospitalier Pellegrin-Enfants, Bordeaux, France

    Charlotte Jubert

  4. Department of Haematology, Birmingham Children’s Hospital, Birmingham, UK

    Sarah Lawson

  5. Biostatistics, Jazz Pharmaceuticals, Philadelphia, PA, USA

    Robert J. Ryan

  6. Research and Development, Jazz Pharmaceuticals, Oxford, UK

    Raj Hanvesakul

  7. Mother and Child Health, Santa Maria della Misericordia Hospital, Perugia, Italy

    Katia Perruccio

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  1. Mohamad Mohty
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Contributions

MM, RJR, and RH designed the research study. MM, TC, CJ, SL, and KP performed the research. MM, RJR, and RH analyzed the data. All authors wrote and critically revised the manuscript and gave final approval for it to be published.

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Correspondence to Mohamad Mohty.

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Competing interests

MM has received honoraria and research funding from Jazz Pharmaceuticals. TC and SL have received honoraria from Jazz Pharmaceuticals. CJ has received research funding from Jazz Pharmaceuticals. RJR is a former employee of and holds stock in Jazz Pharmaceuticals. RH is an employee of and holds stock and/or stock options in Jazz Pharmaceuticals. KP has no conflicts to disclose.

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Mohty, M., Cluzeau, T., Jubert, C. et al. Defibrotide-treated patients with anicteric or icteric veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic cell transplantation: an EBMT study. Bone Marrow Transplant 57, 664–667 (2022). https://doi.org/10.1038/s41409-022-01588-8

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