title: In a high-dose melphalan setting, palifermin compared with placebo had no effect on oral mucositis or related patient's burden.
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.
authors.affiliation: Department of Haematology, Radboud University Nijmegen Medical Centre, Nijmegen, Nijmegen, The Netherlands. n.blijlevens@hemat.umcn.nl
authors.forename: N
authors.initials: N
authors.lastname: Blijlevens
authors.forename: M
authors.initials: M
authors.lastname: de Château
authors.forename: G
authors.initials: G
authors.lastname: Krivan
authors.forename: W
authors.initials: W
authors.lastname: Rabitsch
authors.forename: A
authors.initials: A
authors.lastname: Szomor
authors.forename: R
authors.initials: R
authors.lastname: Pytlik
authors.forename: A
authors.initials: A
authors.lastname: Lissmats
authors.forename: H E
authors.initials: HE
authors.lastname: Johnsen
authors.forename: T
authors.initials: T
authors.lastname: de Witte
authors.forename: H
authors.initials: H
authors.lastname: Einsele
authors.forename: T
authors.initials: T
authors.lastname: Ruutu
authors.forename: D
authors.initials: D
authors.lastname: Niederwieser
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D064592:Autografts; D005260:Female; D051523:Fibroblast Growth Factor 7 / Q000008:administration & dosage* / Q000009:adverse effects; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D009101:Multiple Myeloma / Q000453:epidemiology / Q000628:therapy*; D019653:Myeloablative Agonists; D033581:Stem Cell Transplantation; D013280:Stomatitis / Q000453:epidemiology* / Q000209:etiology / Q000517:prevention & control; D019172:Transplantation Conditioning
chemical_list: D019653:Myeloablative Agonists; D051523:Fibroblast Growth Factor 7; D008558:Melphalan