title: Reliability and efficacy of palifermin in prevention and management of oral mucositis in patients with acute lymphoblastic leukemia: a randomized, double-blind controlled clinical trial.
abstract: BACKGROUND
  Myeloablative and hematopoietic stem cells transplantation therapy (HSCT) often acts as side-effect to oral mucositis (OM) with no effective treatment. This randomized-controlled trial analyzed the efficacy of palifermin, administered as a dose during HSCT therapy, as primary prophylaxis on pediatric patients with acute lymphoblastic leukemia (ALL).
  
  
  METHODS
  In this study forty-six patients (9-15 years) with B-cell acute lymphoblastic leukemia (B-ALL) were analyzed. The patients underwent allogenic HSCT conditioned by myeloablative regimen. Subsequently to randomization, patients in the palifermin group were assigned to receive palifermin, 60 mg/kg, intravenously as a single dose 3 days before and after transplant conditioning regimen cycle. The patients in the Control group received only a placebo treatment. Maximum severity of OM, incidence and duration of ulcerative OM, incidence and duration of severe OM limitations were evaluated.
  
  
  RESULTS
  A statistically significant reduction in the incidence of OM up to grade 3 in the palifermin group compared to the control group was discovered. There was also a reduction, confirmed at 60 days, in the degree of severity of mucositis in the palifermin group, with an average of 1.54 grade in the palifermin group, and of 2.16 in the Control group and in the use of opioid analgesics.
  
  
  CONCLUSIONS
  This study indicates that a single dose of palifermin used as primary prophylaxis during HSTC therapy can prevent severe OM in pediatric patients with ALL and used as secondary prophylaxis can prevent the recurrence of severe OM in high-risk patients with previous mucosal injury and improves the quality of life in pediatric patients with ALL.
authors: Researcher in Dentofacial Orthopedics, Orthodontics and Pediatric Dentistry, Oral Pathology and Implantology, IRCCS, San Raffaele Hospital, Milan, Italy - lucchese.alessandra@hsr.it.
authors: Alessandra
authors: A
authors: Lucchese
authors: Giovanni
authors: G
authors: Matarese
authors: Maurizio
authors: M
authors: Manuelli
authors: Claudio
authors: C
authors: Ciuffreda
authors: Luca
authors: L
authors: Bassani
authors: Gaetano
authors: G
authors: Isola
authors: Giancarlo
authors: G
authors: Cordasco
authors: Enrico
authors: E
authors: Gherlone
mesh_terms: D000293:Adolescent; D064591:Allografts; D000970:Antineoplastic Agents / Q000009:adverse effects; D002648:Child; D003131:Combined Modality Therapy; D004311:Double-Blind Method; D005260:Female; D051523:Fibroblast Growth Factor 7 / Q000009:adverse effects / Q000627:therapeutic use*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists / Q000009:adverse effects; D019226:Oral Ulcer / Q000209:etiology / Q000517:prevention & control; D015452:Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / Q000150:complications / Q000628:therapy*; D011788:Quality of Life; D011832:Radiation Injuries / Q000209:etiology / Q000517:prevention & control; D011878:Radiotherapy / Q000009:adverse effects; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D013280:Stomatitis / Q000188:drug therapy / Q000209:etiology / Q000517:prevention & control*; D019172:Transplantation Conditioning / Q000009:adverse effects
chemical_list: D000970:Antineoplastic Agents; D019653:Myeloablative Agonists; D011994:Recombinant Proteins; D051523:Fibroblast Growth Factor 7