ABSTRACT
Five hundred and fifty-three metastatic breast cancer patients with no previous CT for metastatic disease were treated with 4-6 cycles of CAF. Responders (complete [CR] or partial [PR] response) were eligible for randomization to high-dose CT (HDCT) with stem cell transplant/support (SCT) or maintenance CMF. Of 310 responders, 199 were eligible and consented to randomization. Of these, 15 were found to be ineligible; 9 others with minor protocol violations were included in the analysis (total 184:101 HDCT, 83 CMF). The design allowed detection of a doubling in median OS using HDCT, with 85% power and an a error of 0.05. Stratification was for CR vs. PR, age, visceral disease, and estrogen receptor status. Final analysis, after 114 deaths and a median of 3 years follow-up, demonstrated no differences in median overall survival (24 m, 26 m, p 0.14), TTP (9.6 m, 9.0 m, p 0.3), 3-year progression-free survival (PFS) (6%, 12%, p 0.3) or 3-year OS (32%, 38%,) for the HDCT and CMF arms, respectively. The proportion of PRs that converted to CRs with subsequent treatment was the same in both arms. Toxicity was higher with HDCT, although this was generally short-lived; there was one toxic death.
