Gordon, J.N., Trebble, T.M., Ellis, R.D., Duncan, H.D., Johns, T., & Goggin, P.M. (2005). Thalidomide in the treatment of cancer cachexia: A randomised placebo controlled trial. Gut, 54, 540–545.

DOI Link

Study Purpose

To evaluate the safety and efficacy of thalidomide in attenuating weight loss in patients with cachexia secondary to advanced pancreatic cancer

Intervention Characteristics/Basic Study Process

Fifty patients were randomized to receive 200 mg of thalidomide by mouth daily or placebo for 24 weeks. At four weeks, 33 patients were evaluated; at eight weeks, 20 patients were evaluated.

Sample Characteristics

Patients were included in the study if they

  • Had inoperable pancreatic cancer
  • Experienced 10% weight loss over the preceding six months
  • Had a life expectancy of at least six weeks.

Patients were excluded if they

  • Had received any form of antineoplastic treatment in the preceding six weeks
  • Weighed less than 40 kg
  • Concurrently used steroids, anabolic drugs, hormonal agents, or appetite stimulants
  • Were younger than 18 years of age
  • Had peripheral neuropathy
  • Had severe constipation.

Study Design

The study was a randomized, placebo-controlled trial.

Measurement Instruments/Methods

  • Primary outcome: Change in weight at four weeks
  • Secondary outcomes:
    • Change in bone and free muscle mass
    • Grip strength
    • Quality of life (physical performance and global health status)
    • Survival

Results

At four weeks, 33 patients were evaluable. The thalidomide arm gained 0.37 kg in weight and 1 cm3 of arm circumference muscle mass. The placebo arm lost 2.21 kg in weight and 4.46 cm3 of arm circumference muscle mass.

At eight weeks, 20 patients were evaluable. The thalidomide arm lost 0.06 kg in weight and 0.5 cm3 of arm circumference muscle mass. The placebo arm lost 3.62 kg in weight and 8.4 cm3 of arm circumference muscle mass.

Conclusions

Thalidomide was well tolerated and effective at attenuating weight loss and loss of lean body mass. Findings were unable to demonstrate that attenuation in weight loss led to improvement in quality of life.

Limitations

  • The study had a high attrition rate: 70% were evaluable at four weeks, and 43% were evaluable at eight weeks.
  • Baseline weight in the control group was 4 kg lighter.
  • Change in appetite was not measured as a primary outcome.