Effectiveness and Utility of a Second-Line Treatment in Metastatic Breast Cancer

Abstract
Background: It is commonly accepted either to stop or to change an anticancer treatment in patients who do not or do not any longer respond to a first-line treatment, although the advantage of a salvage therapy over the continued first-line treatment has not been demonstrated yet. To describe the advantage of a treatment, several dimensions have been suggested but no formal distinction of effectiveness and utility of treatments has been proposed. In this study a proposal is made to distinguish between effectiveness and utility of a treatment. This distinction is applied to compare a salvage therapy with a continued first-line therapy in metastatic breast cancer patients with progression of their disease. Material and Methods: In 233 patients with metastatic breast cancer (no previous chemotherapy except adjuvant) the response to the treatment was assessed after 2 cycles of a first-line treatment. Patients with stable disease (SD) or progressive disease (PD) were each randomized into either continuation of the first-line treatment or salvage therapy. The effectiveness of these treatments was described by response rates and side effects. The utility was described by survival and quality of life (EORTC QLQ C-30). Results: Different response rates and toxicities indicate differences in the effectiveness of continued first-line treatments and salvage therapies. More important, these differences in effectiveness do not correlate with the differences in the utility of treatments. Conclusions: The effectiveness of a treatment can be described by the response rate and by side effects. To describe the utility of the treatment, survival and quality of life have to be measured. Using these definitions it can be shown in the special setting of this randomized trial that neither the effectiveness nor the utility of our salvage therapy is superior to the continued first-line treatment. The demonstrated significant differences in survival of the randomized groups have still to be interpreted with caution, because not all data of this study which might have prognostic significance could be evaluated so far.

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