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Post-transplant Events

In a high-dose melphalan setting, palifermin compared with placebo had no effect on oral mucositis or related patient’s burden

Bone Marrow Transplantation volume 48, pages 966–971 (2013)Cite this article

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Abstract

This randomized-controlled trial studied the efficacy of palifermin in a chemotherapy-only, high-dose Melphalan (HDM) transplant setting, to reduce oral mucositis (OM) and its sequelae measured by patient-reported outcomes (PRO) and medical resource use. Palifermin, relative to placebo was given either pre-/post-HDM or pre-HDM in patients with multiple myeloma (MM) undergoing auto-SCT at 39 European centers. Oral cavity assessment (WHO) and PRO questionnaires (oral mucositis daily questionnaire (OMDQ) and EQ 5D) were used in 281 patients (mean age 56, ±s.d.=8 years). 57 patients received placebo. One hundred and fifteen subjects were randomized to pre-/post-HDM receiving palifermin on 3 consecutive days before HDM and after auto-SCT and 109 patients were randomized to pre-HDM, receiving palifermin (60 μg/kg/day) i.v. for 3 consecutive days before HDM. There was no statistically significant difference in maximum OM severity. Severe OM occurred in 37% (placebo), 38% (pre-/post-HDM) and 24% (pre-HDM) of patients. No significant difference was observed with respect to PRO assessments or medical resource use, but more infections and fever during neutropenia were reported in pre-/post-HDM vs placebo (for example, 51 and 26%). To conclude, palifermin was unable to reduce OM or OM-related patient’s burden in MM transplant patients.

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Acknowledgements

Study investigators were as follows (listed alphabetically): Emanuele Angelucci, Cagliari Italy; Lotfi Benboubker, Tours France; Nicole Blijlevens, Nijmegen the Netherlands; Paul Browne, Dublin Ireland; Jean-Henri Bourhis, Villejuif France; Donald Bunjes, Ulm Germany; Angelo Michele Carella, San Martino Italy; Charles Crawley, Cambridge UK; Ilse Christiansen, Aalborg Denmark; Johannes Clausen, Innsbruck Austria; Peter Dreger, Heidelberg Germany; Hermann Einsele, Wurzburg Germany; Gordon Cook, Leeds UK; Mark Cook, Birmingham UK; Thierry Facon, Lille France; Michael Fillitz, Vienna Austria; Laurent Garderet, Villejuif France; Alois Gratwohl, Basel Switzerland; Jean Luc Harrousseau, Nantes France; Pasquale Iacopino, AO Melacrino Morelli Italy; Ladislav Jebavy, Hradec Kralove Czech Republic; Jan-Erik Johansson, Gothenburg Sweden; Hedvig Kasparu, Linz Austria; Nicolas Ketterer, Lausanne Switzerland; Joachim Kienast, Muenster Germany; Guido Kobbe, Dusseldorf Germany; Gergely Krivan, Budapest Hungary; Giorgio Lambertenghi Deliliers, Milan Italy; Roberto M. Lemoli, Bologna Italy; Werner Linkesch, Graz Austria; Hajna Losonczy, Pecs Hungary; Johan Maertens, Leuven Belgium; Dieter Lutz, Linz Austria; Giovanna Meloni Roma Italy; Tamas Masszi, Budapest Hungary; Gareth Morgan, London UK; Dietger Niederwieser, Leipzig Germany; Robert Pytlik, Prague Check Republic; Werner Rabitsch, Wien Austria; Amin Rahemtulla, London UK; Jean-Francois Rossi, Montpellier France; Nigel Russell, Nottingham UK; Tapani Ruutu, Helsinki Finland; Giuseppe Saglio, Turin Italy; Ann Sonet, Yvoir Belgium; Arpad Szomor, Pécsi Hungary Jan Van Droogenbroeck, Brugge Belgium; Pierre Zachee, Antwerpen Belgium. This study was supported by Swedish Orphan Biovitrum AB. Author contribution: Nicole Blijlevens, Maarten de Château, Dietger Niederwieser designed research, performed research, analyzed data and wrote the paper. Gergely Krivan, Werner Rabitsch, Arpad Szomor, Robert Pytik, Hans E Johnsen, Hermann Einsele, Tapani Ruutu performed research and wrote the paper. Theo de Witte wrote the paper. Agneta Lissmats designed research, analyzed data and wrote the paper.

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Authors and Affiliations

  1. Department of Haematology, Radboud University Nijmegen Medical Centre, Nijmegen, Nijmegen, The Netherlands

    N Blijlevens

  2. Clinical Development, Swedish Orphan Biovitrum AB, Stockholm, Sweden

    M de Château & A Lissmats

  3. St Laszlo Hospital, Budapest, Hungary

    G Krivan

  4. Department of Internal Medicine I, Bone Marrow Transplantation Unit, Medical University of Vienna, Vienna, Austria

    W Rabitsch

  5. First Department of Medicine, University Hospital Pecs, Pecs, Hungary

    A Szomor

  6. University Hospital, Praha, Czech Republic

    R Pytlik

  7. Department of Haematology, Aalborg Hospital Science and Innovation Centre, Aalborg Hospital, Aalborg, Denmark

    H E Johnsen

  8. Department of Tumorimmunology, Radboud University Nijmegen Medical Centre, Nijmegen, Nijmegen, The Netherlands

    T de Witte

  9. Medizinische Klinik II, Wurzburg, Germany

    H Einsele

  10. University Hospital, Helsinki, Finland

    T Ruutu

  11. Department of Hematology/Oncology, University of Leipzig, Leipzig, Germany

    D Niederwieser

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Corresponding author

Correspondence to N Blijlevens.

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Competing interests

Maarten de Château employment by Swedish Orphan Biovitrum AB, medical program director. Agneta Lissmats employment by Swedish Orphan Biovitrum AB, biostatistician. The remaing authors declare no competing financial interests.

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Blijlevens, N., de Château, M., Krivan, G. et al. In a high-dose melphalan setting, palifermin compared with placebo had no effect on oral mucositis or related patient’s burden. Bone Marrow Transplant 48, 966–971 (2013). https://doi.org/10.1038/bmt.2012.257

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  • DOI: https://doi.org/10.1038/bmt.2012.257

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