title: Long-term outcomes of thalidomide in refractory Crohn's disease.
abstract: BACKGROUND
  Several open-label and retrospective studies have indicated that thalidomide may be beneficial in patients with refractory Crohn's disease (CD).
  
  
  OBJECTIVE
  To report our long-term experience with the use of thalidomide for adults with refractory Crohn's disease.
  
  
  METHODS
  We conducted a retrospective study of long-term clinical and safety outcomes among adults treated with thalidomide for refractory Crohn's disease. Response was defined as a clinician's assessment of improvement after at least 7 days treatment of one or more of the following: bowel movement frequency, fistula output, rectal bleeding, abdominal pain, extraintestinal manifestations, or well-being. Remission required all of the following: <3 stools/day, no bleeding, abdominal pain or extraintestinal manifestations and increased well-being.
  
  
  RESULTS
  Thirty-seven adults with refractory Crohn's disease were treated with thalidomide for a median of 4.4 months and followed up for a median of 58 months. Clinical response and remission rates were 54% and 19%, respectively. About 40% of patients were able to stop steroids. Response rates were higher for those treated with more than 50 mg/day (85%) than for those treated with a maximum of 50 mg/day (40%; P = 0.01). An adverse event occurred in 68% of patients. Approximately one-third of patients (38%) experienced neuropathy.
  
  
  CONCLUSIONS
  Thalidomide appears to be safe and effective in some patients with refractory Crohn's disease. Although side effects may limit long-term use, thalidomide has potential to induce significant clinical responses.
authors.affiliation: Cedars-Sinai Medical Center, Los Angeles, CA, USA.
authors.forename: M E
authors.initials: ME
authors.lastname: Gerich
authors.forename: J L
authors.initials: JL
authors.lastname: Yoon
authors.forename: S R
authors.initials: SR
authors.lastname: Targan
authors.forename: A F
authors.initials: AF
authors.lastname: Ippoliti
authors.forename: E A
authors.initials: EA
authors.lastname: Vasiliauskas
mesh_terms: D000328:Adult; D003424:Crohn Disease / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D013256:Steroids / Q000627:therapeutic use; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D007166:Immunosuppressive Agents; D013256:Steroids; D013792:Thalidomide