title: A prospective, multicenter study of caspofungin for the treatment of documented Candida or Aspergillus infections in pediatric patients.
abstract: We evaluated the safety, tolerability, and efficacy of caspofungin in pediatric patients with invasive aspergillosis, invasive candidiasis, or esophageal candidiasis.
  
  
  
  This was a multicenter, prospective, open-label study in children 3 months to 17 years of age with proven or probable invasive aspergillosis, proven invasive candidiasis, or proven esophageal candidiasis. All of the patients received caspofungin 70 mg/m(2) on day 1, followed by 50 mg/m(2) per day (maximum: 70 mg/day), as primary or salvage monotherapy. Favorable response was defined as complete resolution of clinical findings and microbiologic (or radiographic/endoscopic) eradication (complete response) or significant improvement in these parameters (partial response). Efficacy was assessed at the end of caspofungin therapy in patients with a confirmed diagnosis who received >/=1 dose of caspofungin. The primary safety evaluation was the proportion of patients with clinical or laboratory drug-related adverse events.
  
  
  
  Of the 49 patients enrolled, 3 were <2 years of age, 30 were 2 to 11 years of age, and 16 were 12 to 17 years of age. Forty-eight patients had confirmed disease: invasive aspergillosis (10), invasive candidiasis (37), and esophageal candidiasis (1). Eight of 10 patients with invasive aspergillosis had pulmonary involvement; 34 of 37 patients with invasive candidiasis had candidemia. Caspofungin was given for 2 to 87 days. Success at end of therapy was achieved in 5 of 10 patients with invasive aspergillosis, 30 of 37 with invasive candidiasis, and 1 of 1 with esophageal candidiasis. One patient (invasive candidiasis) relapsed during the 28-day follow-up period. Drug-related clinical or laboratory adverse events occurred in 27% and 35% of patients, respectively. There were no serious drug-related adverse events or discontinuations of caspofungin because of toxicity.
  
  
  
  Caspofungin was generally well tolerated in pediatric patients aged 6 months through 17 years. Efficacy outcomes in patients with invasive aspergillosis or invasive candidiasis were consistent with previous adult studies in these indications.
authors.affiliation: Children's Hospital of Philadelphia, Division of Infectious Diseases, 34th Street and Civic Center Boulevard, CHOP North, Suite 1527, Philadelphia, PA 19104, USA. zaoutis@email.chop.edu
authors.forename: Theoklis E
authors.initials: TE
authors.lastname: Zaoutis
authors.forename: Hasan S
authors.initials: HS
authors.lastname: Jafri
authors.forename: Li-Min
authors.initials: LM
authors.lastname: Huang
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Asher
authors.initials: A
authors.lastname: Barzilai
authors.forename: Wolfram
authors.initials: W
authors.lastname: Ebell
authors.forename: William J
authors.initials: WJ
authors.lastname: Steinbach
authors.forename: John
authors.initials: J
authors.lastname: Bradley
authors.forename: Jay M
authors.initials: JM
authors.lastname: Lieberman
authors.forename: Chih-Cheng
authors.initials: CC
authors.lastname: Hsiao
authors.forename: Nita
authors.initials: N
authors.lastname: Seibel
authors.forename: Hans-Juergen
authors.initials: HJ
authors.lastname: Laws
authors.forename: Melinda
authors.initials: M
authors.lastname: Gamba
authors.forename: Maria
authors.initials: M
authors.lastname: Petrecz
authors.forename: Arlene F
authors.initials: AF
authors.lastname: Taylor
authors.forename: Kim M
authors.initials: KM
authors.lastname: Strohmaier
authors.forename: Joseph W
authors.initials: JW
authors.lastname: Chow
authors.forename: Nicholas A
authors.initials: NA
authors.lastname: Kartsonis
authors.forename: Angela L
authors.initials: AL
authors.lastname: Ngai
mesh_terms: D000293:Adolescent; D000935:Antifungal Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001228:Aspergillosis / Q000188:drug therapy*; D002177:Candidiasis / Q000188:drug therapy*; D000077336:Caspofungin; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D064420:Drug-Related Side Effects and Adverse Reactions; D054714:Echinocandins / Q000009:adverse effects / Q000627:therapeutic use*; D004935:Esophageal Diseases / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D055666:Lipopeptides; D008111:Liver Function Tests; D008297:Male; D009894:Opportunistic Infections / Q000188:drug therapy*; D011446:Prospective Studies; D055732:Pulmonary Aspergillosis / Q000188:drug therapy*; D012008:Recurrence; D016896:Treatment Outcome
chemical_list: D000935:Antifungal Agents; D054714:Echinocandins; D055666:Lipopeptides; D000077336:Caspofungin