Impact of Palifermin on Transplant Outcomes in Children and Adolescents Undergoing Allogeneic Hematopoietic Cell Transplantation

Abstract

Background: Oral mucositis (OM) is considered to be one of the worst and most debilitating complications of conditioning for hematopoietic cell transplantation (HCT). Prevention and treatment of this complication is one of the utmost priorities of supportive therapy during transplant procedure. The objective of this study was the analysis of the influence of palifermin, keratinocyte growth factor (KGF), on transplant outcomes in patients undergoing allo-HCT. Patients and Methods: A total of 253 allo-HCTs performed between 2003-2018 in patients aged 0-19 years at a single center were analyzed. KGF was administered in 161 HCTs. Uni-and multivariate risk factor analyses were performed. Results: In spite of reducing the duration and grade of mucositis, no prognostic impact of KGF was shown for overall survival, event-free survival, relapse incidence, acute and chronic graftversus-host disease (GVHD), nor GVHD-free relapse-free survival. Conclusion: Palifermin had no impact on transplant outcomes in children and adolescents undergoing allo-HCT.Oral mucositis (OM) is one of the most common and distressful (debilitating) complications in cancer chemotherapy, radiotherapy, conditioning and transplantation (1-5). It is often accompanied by pain, dysgeusia, odynophagia and occurs more frequent in children and adolescents than that in adult patients (2). OM is reported in 75% to 100% patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) (6-10).Numerous methods for the prevention and treatment of OM have been developed including palifermin [recombinant human keratinocyte growth factor (KGF)], granulocytecolony stimulating factor, amifostine, aloe vera, honey and polymixin/tobramycin/amphotericin, antibiotic pastille/paste, intravenous glutamine, polaprezinc sodium alginate suspension and sucralfate, cryotherapy, and laser (2, 11). Current recommendations of Multinational Association of Supportive Care in Cancer and International Society of Oral Oncology for the prevention of OM include: KGF, benzydamine, morphine, cryotherapy and low-level laser light therapy (12,13).The use of palifermin in prophylaxis can significantly reduce the incidence and severity of mucositis in patients after total body irradiation (TBI)/high-dose chemotherapy. We previously showed that patients receiving KGF after autologous HCT had better overall survival (OS) (5). We have also shown that patients with allo-HCT who received palifermin prophylactically, had shorter duration of mucositis (median: 9 vs. 13 days), lower OM grade (median: III vs. IV), shorter total parenteral nutrition (median: 19 vs. 22 days) and lower incidence of episodes of febrile neutropenia (median: 39% vs. 83%) ( 14). Since data on the effect of palifermin on outcomes of allo-HCT in children are scarce, we analyzed the impact of prophylactic use of palifermin on transplant outcomes in a large cohort of children undergoing allo-HCT. Patients and MethodsStudy design. We carried out analysis of the impact of palifermin administered under compassionate use on...

Factors related to oral mucositis or dysgeusia in breast cancer patients undergoing fluorouracil, epirubicin, and cyclophosphamide therapy: A single-center, retrospective study

“…In the rst step, univariate ordered logistic regression analysis between the outcomes and each potential independent variable was conducted. Subsequently, a multivariate ordered logistic regression model was constructed by including those candidate variables showing values of P < 0.2 on univariate regression analysis or selected based on previous studies [7][8][9][10][11][12][13][14][15][16]. The degree of OM or dysgeusia was evaluated using a graded scale, since multiple factors could potentially contribute to the development of OM or dysgeusia, thereby necessitating simultaneous evaluation of the factors.…”

“…Previous studies have explored preventive measures for these conditions, investigating the potential bene ts of dexamethasone [7,8], statins [9], renin-angiotensin system (RAS) inhibitors [10], and others [11][12][13][14][15]. Factors related to OM or dysgeusia have also been statistically analyzed in previous studies [1,6,7,16].…”

Acute Lymphoblastic Leukemia in Children: Better Transplant Outcomes After Total Body Irradiation-based Conditioning

“…Busulfan was administered four times a day with age and body weight dose adjustment. Other transplant procedures and supportive care were described elsewhere (11)(12)(13)(14). Neutrophil engraftment was defined as the first of three consecutive days of absolute neutrophil counts exceeding 0.5×10 9 /l.…”