title: Efficacy and safety of lenalidomide for refractory cutaneous lupus erythematosus.
abstract: BACKGROUND
  Cutaneous lupus erythematosus (CLE) is a chronic disease characterized by disfigurement and a relapsing course. Thalidomide has proven its efficacy in refractory cutaneous lupus disease, although it is not exempt from significant side effects and frequent relapses after withdrawal. New thalidomide analogues have been developed but lack clinical experience. The aim of this preliminary phase II study was to evaluate the efficacy and safety of lenalidomide in patients with refractory CLE.
  
  
  METHODS
  Fifteen patients with refractory cutaneous lupus disease were enrolled in this single-center, open-label, non-comparative pilot trial between January 2009 and December 2010. Oral lenalidomide (5 to 10 mg/day) was administered and tapered according to clinical response. Patients were followed up for a mean of 15 months (range: 7 to 30). Primary efficacy endpoint was the proportion of patients achieving complete response, defined by a Cutaneous Lupus Erythematosus Disease Area and Severity index (CLASI) activity score of 0. Other secondary endpoints included development of side effects, evaluation of cutaneous and systemic flares, and impact on the immunological parameters.
  
  
  RESULTS
  One patient discontinued treatment due to side effects. All remaining patients saw clinical improvement and this was already noticeable after 2 weeks of treatment. Twelve of those patients (86%) achieved complete response but clinical relapse was frequent (75%), usually occurring 2 to 8 weeks after lenalidomide's withdrawal. No influence on systemic disease, immunological parameters or CLASI damage score was observed. Side effects including insomnia, grade 2 neutropenia and gastrointestinal symptoms, were minor (13%). These resolved after withdrawing medication. Neither polyneuropathy nor thrombosis was observed.
  
  
  CONCLUSIONS
  Lenalidomide appears to be efficacious and safe in patients with refractory CLE, but clinical relapse is frequent after its withdrawal.
  
  
  BACKGROUND
  ClinicalTrials.gov: NCT01408199.
authors.forename: Josefina
authors.initials: J
authors.lastname: Cortés-Hernández
authors.forename: Gabriela
authors.initials: G
authors.lastname: Ávila
authors.forename: Miquel
authors.initials: M
authors.lastname: Vilardell-Tarrés
authors.forename: Josep
authors.initials: J
authors.lastname: Ordi-Ros
mesh_terms: D000284:Administration, Oral; D000328:Adult; D003967:Diarrhea / Q000139:chemically induced; D004351:Drug Resistance; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D000077269:Lenalidomide; D008178:Lupus Erythematosus, Cutaneous / Q000188:drug therapy* / Q000473:pathology; D008875:Middle Aged; D009503:Neutropenia / Q000139:chemically induced; D010865:Pilot Projects; D012008:Recurrence; D012074:Remission Induction; D012720:Severity of Illness Index; D007319:Sleep Initiation and Maintenance Disorders / Q000139:chemically induced; D013792:Thalidomide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome; D014839:Vomiting / Q000139:chemically induced
chemical_list: D007155:Immunologic Factors; D013792:Thalidomide; D000077269:Lenalidomide