title: Phase II trial of GM-CSF + thalidomide in patients with androgen-independent metastatic prostate cancer.
abstract: BACKGROUND
  Androgen-independent metastatic prostate cancer is a major therapeutic dilemma. Granulocyte-macrophage colony stimulating factor (GM-CSF) and thalidomide have some biologic activity as single agents in this disease subset. We performed a Phase II trial of this combination to assess its toxicity and potential utility as an immunomodulatory approach to management of advanced prostate cancer.
  
  
  METHODS
  Twenty-two patients were treated with GM-CSF 250 microg administered SC on Monday, Wednesday and Friday of each week. Thalidomide was escalated to reach the study dose of 200 mg/day. Patients were assessed every 4 weeks with therapy continuing to a maximum of 6 months.
  
  
  RESULTS
  All 22 patients had a decrement in PSA at 2 weeks postinitiation of therapy. Five patients had a > or =50% decline (56, 64,66,66, and 94%, respectively) from baseline verified at 4 weeks post best response. This corresponds to an observed response rate of 23% (95% confidence interval 8-45%). Therapy was well tolerated with the majority of patients experiencing only one event.
  
  
  CONCLUSIONS
  The combination of GM-CSF + thalidomide is relatively well tolerated and has the potential to produce antitumor activity in a population of patients with metastatic, androgen independent prostate cancer. This nonchemotherapy combination should be explored in a subset of patients with less advanced disease.
authors.affiliation: Department of Hematology/Oncology, Cleveland Clinic Foundation, Cleveland, OH, USA. dreicer@cc.ccf.org
authors.forename: Robert
authors.initials: R
authors.lastname: Dreicer
authors.forename: Eric A
authors.initials: EA
authors.lastname: Klein
authors.forename: Paul
authors.initials: P
authors.lastname: Elson
authors.forename: David
authors.initials: D
authors.lastname: Peereboom
authors.forename: Tatiana
authors.initials: T
authors.lastname: Byzova
authors.forename: Edward F
authors.initials: EF
authors.lastname: Plow
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D007167:Immunotherapy; D007279:Injections, Subcutaneous; D008297:Male; D008875:Middle Aged; D017430:Prostate-Specific Antigen / Q000097:blood; D011471:Prostatic Neoplasms / Q000188:drug therapy*; D013792:Thalidomide / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D020533:Angiogenesis Inhibitors; D013792:Thalidomide; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D017430:Prostate-Specific Antigen