A combination of distribution- and anchor-based approaches determined minimally important differences (MIDs) for four endpoints in a breast cancer scale

Objective: To determine distribution- and anchor-based minimal important difference (MID) estimates for four scores from the Functional Assessment of Cancer Therapy-Breast (FACT-B): the breast cancer subscale (BCS), Trial Outcome Index (TOI), FACT-G (the general version), and FACT-B. Study Design and Setting: We used data from a Phase III clinical trial in metastatic breast cancer (ECOG study 1193; n = 739) and a prospective observational study of pain in metastatic breast cancer (n = 129). One third and one half of the standard deviation and I standard error of measurement were used as distribution-based criteria. Clinical indicators used to determine anchor-based differences included ECOG performance status, current pain, and response to treatment. Results: FACT-B scores were responsive to performance status and pain anchors, but not to treatment response. By combining the results of distribution- and anchor-based methods, MID estimates were obtained: BCS = 2-3 points, TOI = 5-6 points, FACT-G = 5-6 points, and FACT-B = 7-8 points. Conclusion: Distribution- and anchor-based estimates of the MID do show convergence. These estimates can be used in combination with other measures of efficacy to determine meaningful benefit and provide a basis for sample size estimation in clinical trials. (C) 2004 Elsevier Inc. All rights reserved.