Palifermin Does Not Influence the Incidence and Severity of GvHD Nor Long-Term Survival of Patients with Hematological Diseases Undergoing HSCT
Authors: Nasilowska-Adamska Barbara , Tomaszewska Agnieszka , Szydlo Richard M. , Rzepecki Piotr , Czyz Anna , Manko Joanna , Markiewicz Miroslaw , Torosian Tigran , Bieniaszewska Maria , Piatkowska-Jakubas Beata , Wierzbowska Agnieszka , Duda Dorota , Szczepinski Andrzej , Lange Andrzej , Robak Tadeusz , Skotnicki Aleksander , Hellman Andrzej , Jedrzejczak Wieslaw W. , Holowiecki Jerzy , Dmoszynska Anna , Komarnicki Mieczyslaw , Warzocha Krzysztof , Marianska Bozena 
Published: April 2020

Abstract
AIMS: Oral mucositis (OM) is a frequent complication of myeloablative therapy and hematopoietic stem cell transplantation (HSCT). Palifermin was found to reduce the incidence, duration and severity of OM induced by high-dose chemotherapy with HSCT (Bone Marrow Transplant.2007;40:983–8). However, additional data on the long-term safety of palifermin and its potential influence on graft versus host disease (GvHD) were missing. In this multi-center, non-randomized, matched-control study we assessed overall survival (OS), incidence and severity of acute/chronicGvHD (a/cGvHD) and incidence of secondary malignancies in patients with hematological diseases treated with HSCT who received palifermin as a prophylaxis for OM.
METHODS AND RESULTS: One hundred and twenty patients with hematological diseases transplanted between December 2001 and December 2007 were enrolled to this study. Sixty patients (50%) received palifermin (60μg/kg/day) for three consecutive days before and after conditioning therapy (palifermin group). Median age of patients was 37.5 years (range, 19 to 63) in the palifermin group and 35.5 years (range, 18 to 64) in the control group. There were no statistical differences between studied groups in terms of other clinical characteristics, including gender, diagnosis, type of transplant, conditioning regimens or GvHD prophylaxis. Kaplan-Meier curves for OS were calculated and compared using the log-rank test. Fisher’s exact and the χ2 trend tests were applied for the analysis of the GvHD data. Twenty one (35%) autologous and thirty nine (65%) allogeneic HSCT (HLA-matched related and unrelated) were performed in each group. Following allogeneic HSCT, the incidence of aGvHD grade 1–4 was 28.2% and 38.4% (p=0.34) and cGvHD was 41% and 53.8% (p=0.70) in the palifermin and control groups, respectively. The incidence of aGvHD severity grade 0,I,II,III,IV was 69.2%, 5.1%, 17.9%, 2.5%, 2.5% in the palifermin and 61.5%, 12.8%, 12.8%, 10.2%, 5.2% in the control group, respectively (p&gt;0.4 for each). The incidence of limited and extensive cGvHD was 17.9% and 23% in the palifermin group versus 25.6% and 28.2% in the control group (p=0.32 and p=0.50, respectively). The estimated 2-years OS (72.8% and 79.8%, respectively) also didn’t differ significantly between studied groups (p=0.13). We didn’t observe any secondary malignancies in patients enrolled into the study.
CONCLUSIONS: Administration of palifermin doesn’t seem to influence the incidence and severity of a/cGvHD, secondary malignancies occurrence and OS in patients with hematological diseases undergoing HSCT.