title: Thalidomide for the treatment of oral aphthous ulcers in patients with human immunodeficiency virus infection. National Institute of Allergy and Infectious Diseases AIDS Clinical Trials Group.
abstract: BACKGROUND
  In patients with advanced human immunodeficiency virus (HIV) infection, aphthous ulceration of the mouth and oropharynx can become extensive and debilitating. Preliminary reports suggest that thalidomide may promote the healing of oral aphthous ulcers.
  
  
  METHODS
  We performed a double-blind, randomized, placebo-controlled study of thalidomide as therapy for oral aphthous ulcers in HIV-infected patients. The patients received a four-week course of either 200 mg of thalidomide or placebo orally once per day. They were evaluated weekly for the condition of the ulcers, their quality of life, and evidence of toxicity. Assays were performed for plasma tumor necrosis factor alpha (TNF-alpha), soluble TNF-alpha receptors, and HIV RNA.
  
  
  RESULTS
  Sixteen of 29 patients in the thalidomide group (55 percent) had complete healing of their aphthous ulcers after four weeks, as compared with only 2 of 28 patients in the placebo group (7 percent; odds ratio, 15; 95 percent confidence interval after adjustment for group sequential testing, 1.8 to 499; unadjusted P<0.001). Pain diminished and ability to eat improved with thalidomide treatment. The adverse effects noted with thalidomide included somnolence and rash (7 patients each), and 6 of the 29 patients discontinued treatment because of toxicity. Thalidomide treatment increased HIV RNA levels (median increase, 0.42 log10 copies per milliliter; increase with placebo, 0.05; P=0.04). With thalidomide treatment there were unexpected increases in the plasma concentrations of TNF-alpha and soluble TNF-alpha receptors.
  
  
  CONCLUSIONS
  Thalidomide is an effective treatment for aphthous ulceration of the mouth and oropharynx in patients with HIV infection.
authors.affiliation: Department of Medicine, Bronx Veterans Affairs Medical Center and Mount Sinai School of Medicine, NY 10468, USA.
authors.forename: J M
authors.initials: JM
authors.lastname: Jacobson
authors.forename: J S
authors.initials: JS
authors.lastname: Greenspan
authors.forename: J
authors.initials: J
authors.lastname: Spritzler
authors.forename: N
authors.initials: N
authors.lastname: Ketter
authors.forename: J L
authors.initials: JL
authors.lastname: Fahey
authors.forename: J B
authors.initials: JB
authors.lastname: Jackson
authors.forename: L
authors.initials: L
authors.lastname: Fox
authors.forename: M
authors.initials: M
authors.lastname: Chernoff
authors.forename: A W
authors.initials: AW
authors.lastname: Wu
authors.forename: L A
authors.initials: LA
authors.lastname: MacPhail
authors.forename: G J
authors.initials: GJ
authors.lastname: Vasquez
authors.forename: D A
authors.initials: DA
authors.lastname: Wohl
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000150:complications*; D000328:Adult; D004311:Double-Blind Method; D005260:Female; D015497:HIV-1 / Q000187:drug effects / Q000302:isolation & purification; D006801:Humans; D008297:Male; D011788:Quality of Life; D018124:Receptors, Tumor Necrosis Factor / Q000097:blood; D013281:Stomatitis, Aphthous / Q000188:drug therapy* / Q000209:etiology; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D014409:Tumor Necrosis Factor-alpha / Q000032:analysis
chemical_list: D018124:Receptors, Tumor Necrosis Factor; D014409:Tumor Necrosis Factor-alpha; D013792:Thalidomide