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Efficacy and safety of keratinocyte growth factor (palifermin) for prevention of oral mucositis in TBI-based allogeneic hematopoietic stem cell transplantation

Bone Marrow Transplantation volume 53, pages 1188–1192 (2018)Cite this article

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After myeloablative conditioning therapy and hematopoietic stem cell transplantation (HSCT), patients judged oral mucositis as the most debilitating side effect of their therapy [1]. For most myeloablative regimens the incidence of severe mucositis ranges between 30 to 70 % [2], and may exceed even 95 % for highly toxic regimen [3]. Observational studies demonstrated a strong association between severity of oral mucositis and days with fever, significant infection, need for parenteral nutrition and injectable narcotic therapy, as well as therapy-related mortality [4].

Palifermin (Kepivance®, Swedish Orphan Biovitrum), a truncated form of recombinant human keratinocyte growth factor 1, demonstrated its ability to reduce mucositis after chemo- / irradiation therapy in preclinical models [5] and human studies [6], and became FDA approved in 2004. The updated Clinical Practice Guidelines by the MASCC/ISOO recommended the use of palifermin only for total body irradiation (TBI)-based autologous HSCT, while no recommendation could be given in the allogeneic setting due to conflicting results and insufficient evidence [7].

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Acknowledgements

The study was conducted in cooperation with the Eastern German Study Group of Haematology and Oncology (OSHO, protocol code OSHO #76). Palifermin (Kepivance®) was provided by Swedish Orphan Biovitrum AB (Sobi), SE-112 76 Stockholm, Sweden. We further thank GMIHO (Gesellschaft für medizinische Innovation/Hämatologie und Onkologie mbH, Berlin, Germany) for the technical support of this trial, and Dr. Andreas Engelen (WiSP—Wissenschaftlicher Service Pharma GmbH, Langenfeld, Germany) for data monitoring and biostatistics.

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Authors and Affiliations

  1. Department of Haematology and Medical Oncology, Clinic of Internal Medicine II, University Hospital Jena, Jena, Germany

    Volker Schmidt, Thomas Schenk, Anne Klink, Andreas Hochhaus & Herbert G. Sayer

  2. Department of Haematology, Oncology and Hemostaseology, University Clinical Center, University of Leipzig, Leipzig, Germany

    Dietger Niederwieser, Gerhard Behre & Christian Pfrepper

  3. WiSP Wissenschaftlicher Service Pharma GmbH, Langenfeld, Germany

    Axel Hinke

  4. Department of Bone Marrow Transplantation, West German Cancer Center, Faculty of Medicine, University Duisburg-Essen, Essen, Germany

    Dietrich W. Beelen

  5. Department of Haematology, Oncology, Palliative Care, University of Rostock, Rostock, Germany

    Christian Junghanss

  6. Department of Hematology, Oncology and Tumorimmunology, Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum, Berlin, Germany

    Lutz Uharek

  7. Internal Medicine C (Hematology and Oncology, Palliative Care, Transplant Centre), University Hospital Greifswald, Ernst-Moritz-Arndt-University, Greifswald, Germany

    William H. Krüger

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  1. Volker Schmidt
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for the East German Study Group for Hematology and Oncology (OSHO)

Corresponding author

Correspondence to Herbert G. Sayer.

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The authors declare that they have no conflict of interest.

Additional information

Support: Palifermin was provided by Swedish Orphan Biovitrum

EudraCT database: 2006-003683-54.

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Schmidt, V., Niederwieser, D., Schenk, T. et al. Efficacy and safety of keratinocyte growth factor (palifermin) for prevention of oral mucositis in TBI-based allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant 53, 1188–1192 (2018). https://doi.org/10.1038/s41409-018-0135-3

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  • DOI: https://doi.org/10.1038/s41409-018-0135-3

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