title: Safety, tolerance and outcome of treatment with liposomal amphotericin B in paediatric patients with cancer or undergoing haematopoietic stem cell transplantation.
abstract: OBJECTIVE
  To assess safety, tolerance and efficacy of liposomal amphotericin B (LAMB) in a large unselected series of paediatric cancer/haematopoietic stem cell transplantation (HSCT) patients requiring LAMB therapy.
  
  
  METHODS
  The study included 84 children and adolescents (median age: 11 years) who received 141 consecutive courses of LAMB for prophylaxis (32), empirical therapy (83), possible (19) or probable/proven (7) invasive infections. LAMB was administered until intolerance or maximum efficacy at dosages individually determined by the responsible physician.
  
  
  RESULTS
  Fifty-nine courses were post-HSCT (42%, 49 allogeneic), and 92 courses were started during granulocytopenia (65%). The median duration of LAMB therapy was 13 days (range 1-79), and the median maximum dosage was 2.8 mg/kg (range 0.93-5.10). Mild-to-moderate adverse events were noted during 109 courses (77%; hepatic, 58.8%; electrolyte wasting, 52.5%; renal, 31.9%; infusion-related reactions, 8.5%); adverse events necessitating discontinuation of LAMB occurred in 6 courses (4.3%; renal, 3; anaphylaxis, 2; hepatic, 1). While median hepatic transaminase, alkaline phosphatase and blood urea nitrogen values were slightly (P < 0.01) higher at end of treatment (EOT), bilirubin and creatinine values were not different from baseline. Complete or partial responses were observed in 16/19 and 2/7 courses for possible and probable/proven invasive infections. Thirty-two of 33 courses of prophylaxis and 74 of 83 courses of empirical therapy were completed with success. Overall survival was 90.8% at 3 months post-EOT.
  
  
  CONCLUSIONS
  LAMB had acceptable safety and tolerance and was useful in prevention and treatment in unselected, mostly granulocytopenic paediatric patients undergoing treatment for cancer or HSCT.
authors.affiliation: Infectious Disease Research Program, Center for Bone Marrow Transplantation and Department of Pediatric Hematology/Oncology, University Childrens Hospital Münster, Albert-Schweitzer-Strasse 33, 48149 Münster, Germany.
authors.forename: Hedwig
authors.initials: H
authors.lastname: Kolve
authors.forename: Elvira
authors.initials: E
authors.lastname: Ahlke
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Fegeler
authors.forename: Jörg
authors.initials: J
authors.lastname: Ritter
authors.forename: Heribert
authors.initials: H
authors.lastname: Jürgens
authors.forename: Andreas H
authors.initials: AH
authors.lastname: Groll
mesh_terms: D000293:Adolescent; D000666:Amphotericin B / Q000009:adverse effects* / Q000627:therapeutic use*; D000935:Antifungal Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D016867:Immunocompromised Host; D007223:Infant; D008297:Male; D009181:Mycoses / Q000188:drug therapy* / Q000517:prevention & control*; D009369:Neoplasms / Q000150:complications; D033581:Stem Cell Transplantation; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000935:Antifungal Agents; C068538:liposomal amphotericin B; D000666:Amphotericin B