Predictive factors of survival after thalidomide therapy in advanced multiple myeloma: long-term follow-up of a prospective multicenter nonrandomized phase II study in 120 patients

Clin Lymphoma Myeloma Leuk. 2012 Dec;12(6):418-22. doi: 10.1016/j.clml.2012.06.003. Epub 2012 Sep 29.

Abstract

Thalidomide monotherapy has demonstrated consistent results in the treatment of advanced multiple myeloma. We report a 9-year follow-up of a French multicenter nonrandomized phase II study that evaluated the effect of oral thalidomide in 120 patients with advanced multiple myeloma. Independent predictors of survival were response to last therapy, performance status, serum β(2)-microglobulin level, platelet count, and response at day 60 of treatment.

Background: Thalidomide monotherapy has demonstrated consistent results in the treatment of advanced multiple myeloma.

Patients and methods: We report the 9-year follow-up of a French multicenter, nonrandomized, phase II study that evaluated the effect of oral thalidomide in advanced multiple myeloma. Thalidomide was started at 200 mg/d and increased to 400 mg/d at day 15.

Results: One hundred twenty patients were enrolled in 2 months at 33 centers. The overall response rate was 31.7% (38/120) on day 60. Overall survival rates were 47.5% (95% confidence interval [CI], 38.6-56.4), 25.0% (95% CI, 17.3-32.7), 11.7% (95% CI, 5.9-17.4), and 7.5% (95% CI, 2.8-12.2) at 1, 3, 6, and 9 years, respectively. Independent predictors of short survival at 1, 3, 6, and 9 years were multiple myeloma refractory to last therapy, performance status ≥ 2, serum β(2)-microglobulin level ≥ 3.5 mg/L, platelet count < 152 × 10(9)/L, and nonresponse at day 60 (Cox proportional hazards regression model).

Conclusion: Our study identified 5 independent unfavorable prognostic factors associated with short survival. These prognostic factors were very robust, allowing the prediction of patient survival not only during the first year but also during 3, 6, and even 9 years after the beginning of treatment.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Disease-Free Survival
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Multiple Myeloma / drug therapy*
  • Prognosis
  • Prospective Studies
  • Survival Analysis
  • Thalidomide / therapeutic use*
  • Treatment Outcome

Substances

  • Thalidomide