title: The safety and efficacy of pimecrolimus, 1%, cream for the treatment of Netherton syndrome: results from an exploratory study.
abstract: BACKGROUND
  Impaired skin integrity in patients with Netherton syndrome (NS) results in significant systemic absorption of topically applied medications. Some have advocated the administration of pimecrolimus, 1%, topical cream for the treatment of patients with NS. Insufficient data exist with regard to its safety, systemic absorption, and efficacy.
  
  
  METHODS
  An exploratory study was conducted involving 3 children with NS who received twice-daily application of pimecrolimus, 1%, cream over 18 months. There were no notable abnormalities in hematologic or chemistry profiles. Blood levels of pimecrolimus ranged from 0.625 to 7.08 ng/mL, with peak levels reached during the first month in all 3 patients. Dramatic reductions were observed in the Netherton Area and Severity Assessment, Eczema Area and Severity Index, Investigator Global Evaluation of Disease, and pruritus scores compared with baseline levels.
  
  
  CONCLUSIONS
  Use of pimecrolimus, 1%, cream was well tolerated and demonstrated marked improvements in nearly all of the parameters evaluated. Patients treated with pimecrolimus responded rapidly, within the first month of treatment, and improvement persisted throughout the study period. In adult patients receiving oral pimecrolimus, blood levels as high as 54 ng/mL for 3 months have not shown clinically significant immunosuppression. Absorption of pimecrolimus, 1%, cream was detectable, but levels were much lower than expected even when applied to 50% of total body surface area. Larger studies are warranted to determine the safety and efficacy of pimecrolimus, 1%, cream in the treatment of NS.
  
  
  BACKGROUND
  clinicaltrials.gov Identifier: NCT00208026.
authors.affiliation: Section of Dermatology, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, 3550 Market St, Second Floor, Philadelphia, PA 19104, USA. yana@email.chop.edu
authors.forename: Albert C
authors.initials: AC
authors.lastname: Yan
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Honig
authors.forename: Michael E
authors.initials: ME
authors.lastname: Ming
authors.forename: Janice
authors.initials: J
authors.lastname: Weber
authors.forename: Kara N
authors.initials: KN
authors.lastname: Shah
mesh_terms: D000287:Administration, Topical; D065095:Calcineurin Inhibitors; D002648:Child; D003879:Dermatologic Agents / Q000008:administration & dosage* / Q000493:pharmacokinetics; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004485:Eczema / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D056770:Netherton Syndrome / Q000097:blood / Q000150:complications / Q000188:drug therapy*; D009824:Ointments; D011788:Quality of Life; D012867:Skin / Q000187:drug effects* / Q000473:pathology; D012869:Skin Absorption; D016559:Tacrolimus / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000493:pharmacokinetics; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D065095:Calcineurin Inhibitors; D003879:Dermatologic Agents; D009824:Ointments; C117268:pimecrolimus; D016559:Tacrolimus