Bone Marrow Transplantation (2018) 53:1188–1192 https://doi.org/10.1038/s41409-018-0135-3 CORRESPONDENCE Efficacy and safety of keratinocyte growth factor (palifermin) for prevention of oral mucositis in TBI-based allogeneic hematopoietic stem cell transplantation Volker Schmidt 1 ● Dietger Niederwieser 2 ● Thomas Schenk 1 ● Gerhard Behre 2 ● Anne Klink 1 ● Christian Pfrepper 2 ● Axel Hinke 3 ● Dietrich W. Beelen 4 ● Christian Junghanss 5 ● Lutz Uharek 6 ● William H. Krüger 7 ● Andreas Hochhaus 1 ● Herbert G. Sayer 1 for the East German Study Group for Hematology and Oncology (OSHO) Received: 23 August 2017 / Revised: 20 January 2018 / Accepted: 6 February 2018 / Published online: 15 March 2018 © Macmillan Publishers Limited, part of Springer Nature 2018 After myeloablative conditioning therapy and hematopoie- tic 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]. Observa- tional studies demonstrated a strong association between severity of oral mucositis and days with fever, significant infection, need for parenteral nutrition and injectable nar- cotic 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]. Here we report our results from a prospective, controlled multicenter trial designed to evaluate efficacy and safety of palifermin for prevention of oral mucositis in TBI-based allogeneic HSCT. Patients were randomized in a 2:1 ratio to receive either a mucositis prophylaxis by palifermin or standard supportive care. The trial was designed according to the Declaration of Helsinki, and was approved by the ethics committee of each participating center. The uniform conditioning regimen consisted of hyper- fractionated TBI (total dose 12 Gy) followed by cyclopho- sphamide (2 × 60 mg/kg). A cell count of 4.0 × 10 6 CD34- positive peripheral blood stem cells per kg bodyweight was requested, and no graft manipulation like cell sorting, T-cell depletion or cryopreservation was performed. Only HLA- identical family donors or unrelated donors with an HLA- match of at least 8/10 (HLA class I: A,B,C; HLA class II: DR, DQ) were accepted. All patients received cyclosporine (3–5 mg/kg per day) and low-dose methotrexate (MTX, 15 mg/m 2 on day 1 and 10 mg/m 2 on day 3, 6, 11) for pro- phylaxis of GvHD. Administration of antithymocyte glo- bulin (ATG) was allowed according to the standard institutional practice of each center. Palifermin was given as intravenous injections according to the approved dosing schedule of 60 μg/kg per day for 3 consecutive days prior to TBI and for 3 further days after HSCT (+6 h, day +2, and day +4). No palifermin was given within 24 h before and 48 h after conditioning ther- apy, as well as on days of MTX administration. Supportive * Herbert G. Sayer Herbert.Sayer@helios-gesundheit.de 1 Department of Haematology and Medical Oncology, Clinic of Internal Medicine II, University Hospital Jena, Jena, Germany 2 Department of Haematology, Oncology and Hemostaseology, University Clinical Center, University of Leipzig, Leipzig, Germany 3 WiSP Wissenschaftlicher Service Pharma GmbH, Langenfeld, Germany 4 Department of Bone Marrow Transplantation, West German Cancer Center, Faculty of Medicine, University Duisburg-Essen, Essen, Germany 5 Department of Haematology, Oncology, Palliative Care, University of Rostock, Rostock, Germany 6 Department of Hematology, Oncology and Tumorimmunology, Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum, Berlin, Germany 7 Internal Medicine C (Hematology and Oncology, Palliative Care, Transplant Centre), University Hospital Greifswald, Ernst-Moritz- Arndt-University, Greifswald, Germany 1234567890();,: