P5-17-08: The Value of Progression-Free Survival from the Patient Perspective: An Online Survey of Women with Metastatic Breast Cancer in the United States.
Authors: SA Hurvitz, SD Mathias, JF Doan, RD Crosby
Journal: Cancer Research (2011)

Abstract Background: While PFS indicates the period of time a patient's’ disease does not progress, there is debate about the relative value of longer PFS in the absence of a meaningful difference in OS. However, there are no known published reports about the value of PFS from the patient's perspective. Material and Methods: This was a 2 -part project consisting of instrument development (via focus groups) and cross-sectional administration to 2 groups of women, those who had vs. those who had not experienced disease progression. Based on input from 19 women with MBC in the US, we developed an MBC-specific questionnaire (MBC-P). The questionnaire focuses on unique aspects relevant to MBC patients including concerns about the future, social isolation, fear, activity limitations, mental health, social functioning, and activity limitations. The MBC-P also presented descriptions of two hypothetical treatments: Treatment X, associated with 12 mos of PFS and Treatment Y with 16 mos of PFS, which had the same side effect profile. Respondents were asked to state which treatment was associated with better overall quality of life (O-QOL), physical functioning (PF), emotional well being (EWB), and which treatment was preferred. We compared mean scores at the item level for respondents based on progression status. One final aspect of the questionnaire asked respondents to rate (on a 0— 100 scale) O-QOL, PF, EWB at the time of initial breast cancer (BC) diagnosis, MBC diagnosis, and MBC progression (if applicable). Ethics approval was obtained. Results: 282 women completed the survey [mean age: 50 yrs (range: 21–80)]; 56% had experienced progressive disease. Women whose disease had progressed were more likely (p &amp;lt; 0.05) to report putting their life on hold, worrying about there not being a treatment that will work, feeling less confident about their treatment, and experiencing greater limitations in recreational or physical activities and accomplishing things/tasks. For the hypothetical descriptions, no differences emerged based on progression status. Despite being told that side effects and OS were the same for both scenarios, significantly more women responded that treatment Y (vs. treatment X) would result in better QOL, physical functioning, and emotional well being. Additionally, 88% of respondents preferred treatment Y over treatment X. Ratings for different stages of disease on O-QOL, PF and EWB were lowest the first time a woman was told her MBC had progressed and highest when told she had responded to treatment (O-QOL: 58 vs. 77; PF: 61 vs. 71; EWB: 51 vs. 77). Discussion: The MBC-P questionnaire was developed with input from women with MBC and is a useful tool to evaluate the value of PFS from the patient's perspective. Women with MBC whose cancer had progressed were more limited in areas pertaining to treatment and activity limitations compared to women who had not experienced progression. Surprisingly, even when told that side effects and OS were similar, women rated O-QOL, PF, and EWB as being better for a hypothetical drug that provided 4 additional months of PFS. Results should be confirmed with a larger, more diverse sample of MBC patients and eventually in a prospective study. Citation Information: Cancer Res 2011;71(24 Suppl):Abstract nr P5-17-08.