Background: The role of high-dose chemotherapy (HDCT) in primary and metastatic breast cancer remains controversial. Although results of several randomized trials have become available, the data is still inconclusive.
Adjuvant hdct: In the adjuvant setting, only one trial has shown an advantage in recurrence-free survival, whereas none of the randomized trials showed a significant improvement in overall survival.
Hdct for stage iv disease: In metastatic breast cancer, four of five randomized trials revealed a significant benefit in progression-free survival for patients treated with HDCT. However, differences in overall survival have not been observed.
Interpretation of trials: Interpretation of the trials is limited due to a lack of statistical power in some of the trials, a relatively short follow-up, a relatively intensive chemotherapy regimen in the control arm, and an inappropriate selection of cytotoxic agents with only moderate activity and/or unacceptable toxicity.
Conclusion: Several questions, especially the approach of early intensification or tandem HDCT, have to be answered yet. The results of ongoing studies are urgently needed.