Thalidomide therapy in adult patients with myelodysplastic syndrome: A North Central Cancer Treatment Group Phase II trial

Alvaro Moreno-Aspitia, Gerardo Colon-Otero, Antje Hoering, Ayalew Tefferi, Robert D. Niedringhaus, Allen Vukov, Chin Yang Li, David M. Menke, Susan M. Geyer, Steven R. Alberts

Research output: Contribution to journalArticlepeer-review

31 Scopus citations


BACKGROUND. Thalidomide has shown promise for the treatment of patients with myelodysplastic syndrome. The current prospective multicenter study examined the efficacy and toxicity of thalidomide in adult patients with myelodysplastic syndrome. METHODS. Using the International Prognostic Scoring System (IPSS), patients were stratified into 2 groups: favorable (IPSS score, 0-1.0) or unfavorable (IPSS score, 1.5-3.5). Seventy-two patients (42 of whom were favorable and 30 of whom were unfavorable) received a starting dose of oral thalidomide of 200 mg daily. The dose was increased by 50 mg per week to a targeted maximum daily dose of 1000 mg. RESULTS. According to the International Working Group response criteria for myelodysplastic syndrome, 1 patient in the unfavorable group achieved a partial remission with a complete cytogenetic response. Overall, 2 patients (5%) in the favorable group and 4 patients (14%) in the unfavorable group experienced either a hematologic improvement or a partial response. The most frequent Grade 3 or 4 (grading was based on the National Cancer Institute's Common Toxicity Criteria [version 2.0]) nonhematologic adverse events were fatigue (24%), infection (19%), neuropathy (13%), dyspnea (8%), and constipation (7%). CONCLUSIONS. Thalidomide alone, at the schedule and dose levels used in the current study, is not a safe and viable therapeutic option for patients with myelodysplastic syndrome. Limited efficacy and increased toxicity were observed in the current Phase II trial.

Original languageEnglish (US)
Pages (from-to)767-772
Number of pages6
Issue number4
StatePublished - Aug 15 2006


  • Clinical trials
  • Hematologic diseases
  • International Prognostic Scoring System
  • Leukemia
  • Myelodysplastic syndromes
  • Thalidomide

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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