title: Treatment with caspofungin in immunocompromised paediatric patients: a multicentre survey.
abstract: OBJECTIVE
  Although a paediatric dosage has not been established, caspofungin is occasionally used in paediatric patients. We conducted a multicentre retrospective survey to obtain data on immunocompromised paediatric patients considered to require caspofungin therapy.
  
  
  METHODS
  The survey identified 64 patients (median age: 11.5 years; 25 females, 39 males) with haematological malignancies (48), marrow failure (9), solid tumours (3), haematological disorders (2) and congenital immunodeficiency (2) who received caspofungin for proven (17), probable (14) and possible (17) invasive fungal infections or empirically (16). Caspofungin was administered until intolerance or maximum efficacy at dosages individually determined by the responsible physician for refractory infection (38), intolerance of other agents (10) or as best therapeutic option (16).
  
  
  RESULTS
  The 64 patients received caspofungin for a median of 37 days (range 3-218) as single agent (20) or in combination (44). The median daily maintenance dosage was 1.07 mg/kg (95% CI 1.09-1.35; range 0.40-2.92) or 34.3 mg/m2 (95% CI 32.3-37.3; range 16.3-57.5). In none of the patients was therapy discontinued due to adverse events (AEs). Clinical AEs were mild to moderate and observed in 34 patients (53.1%). While mean glutamate pyruvate transaminase and glutamate oxalate transaminase values were slightly (P < 0.005) higher at the end of treatment (EOT), serum bilirubin, alkaline phosphatase and creatinine values were not different from baseline. Complete responses, partial responses or stabilization were observed in 5/7/3 of 17 patients with proven, in 3/4/3 of 14 patients with probable and in 7/6/1 of 15 evaluable patients with possible invasive infections. Thirteen of 16 patients on empirical therapy completed without breakthrough infection. Overall survival was 75% at the EOT and 70% at 3 months post-EOT, respectively.
  
  
  CONCLUSIONS
  Caspofungin displayed favourable safety and tolerance and may have useful antifungal efficacy in severely immunocompromised paediatric patients.
authors.affiliation: Infectious Disease Research Program, Center for Bone Marrow Transplantation and Department of Paediatric Haematology/Oncology, Children's University Hospital, Muenster, Germany. grollan@ukmuenster.de
authors.forename: Andreas H
authors.initials: AH
authors.lastname: Groll
authors.forename: Andishe
authors.initials: A
authors.lastname: Attarbaschi
authors.forename: Friedhelm R
authors.initials: FR
authors.lastname: Schuster
authors.forename: Nadine
authors.initials: N
authors.lastname: Herzog
authors.forename: Lorenz
authors.initials: L
authors.lastname: Grigull
authors.forename: Michael N
authors.initials: MN
authors.lastname: Dworzak
authors.forename: Karin
authors.initials: K
authors.lastname: Beutel
authors.forename: Hans-Jürgen
authors.initials: HJ
authors.lastname: Laws
authors.forename: Thomas
authors.initials: T
authors.lastname: Lehrnbecher
mesh_terms: D000293:Adolescent; D000935:Antifungal Agents / Q000009:adverse effects / Q000627:therapeutic use*; D000077336:Caspofungin; D002648:Child; D002675:Child, Preschool; D054714:Echinocandins; D005260:Female; D006801:Humans; D016867:Immunocompromised Host; D007223:Infant; D055666:Lipopeptides; D008297:Male; D009181:Mycoses / Q000188:drug therapy; D010456:Peptides, Cyclic / Q000009:adverse effects / Q000627:therapeutic use*; D012189:Retrospective Studies
chemical_list: D000935:Antifungal Agents; D054714:Echinocandins; D055666:Lipopeptides; D010456:Peptides, Cyclic; D000077336:Caspofungin