Participating Trials

1. A quality of life assessment of patients participating in phase I clinical trials confirms a decrease during treatment (MC0115) Pamela J. Atherton1, Daniel W. Szydlo1, Charles Erlichman2, Jeff A. Sloan1 1Division of Biostatistics, Mayo Clinic, Rochester, MN 2Division of Medical Oncology, Department of Oncology, Mayo Clinic, Rochester, MN Results Control Preferences Scale Abstract Results Results (Degner L, Sloan JA, Venkatesh P. The control preferences scale. Canadian Journal of Nursing Research 29:21-43, 1997.) Participating Trials Change from Baseline of LASA Scores Trial Satisfaction with Maximum Severity of AEs Background:There are psychological challenges associated with participation in Phase I clinical trials due to many consenting patients hoping for a cure despite assertions to dissuade such expectations. This study explored the impact of Phase I trial participation on patient quality of life (QOL). Methods:Patients enrolled onto Phase I studies between 9/10/2002 and 3/6/2007 were asked to complete the Control Preferences Scale (CPS) at baseline, the Linear Analogue Self Assessment (LASA) weekly during cycle 1, and the Trial Satisfaction (TS) survey at the end of cycle 1. All QOL was measured on a 0-10 point scale, 10 being best. The primary endpoint was the change in overall QOL at the end of cycle 1. Summary statistics were calculated for baseline covariates, QOL responses, treatment response and adverse events (AEs). Student’s t-test, Kruskall-Wallis and Pearson/Spearman correlation methodology were utilized. Results:30 patients from 12 different Phase I trials participated. Overall QOL change from baseline was not significant (mean of -0.5, 95% CI [-0.92, 0.05], p=0.08). Four patients experienced a clinically meaningful deficiency by having an overall QOL decrease of at least 2 points. Significant decreases were seen in Physical Well-Being (WB), Emotional WB, and Mood (p=0.04, 0.04, 0.03, respectively). Correlations comparing QOL with the maximum AE grade were weak, the highest being for Emotional WB and Hopefulness (r=-0.41, -0.35). Gender and performance status were related to QOL domains of Fatigue, Anxiety and Hopefulness. CPS indicates concordance in preferred compared to played roles. CPS results and treatment response were unrelated to QOL scores. According to TS, patients seemed to feel participation in the treatment trial was worthwhile, they would do it again, and they would recommend the treatment trial to others (means of 8.7, 8.7 and 9.2, respectively), but they did not feel that their QOL improved as a result of the trial (mean of 4.0). Conclusions:Despite being satisfied with their participation in Phase I clinical trials, deficits in QOL appeared, independent of AEs or tumor response. It hence would seem advisable to include assessments in Phase I trials in order to identify and improve patient QOL. Supported in part by CA 69912. • Circle the letter next to the phrase below that best describes the role you have actually been playing in dealing with your cancer diagnosis. AE grade did not affect patient determination of whether it was worthwhile participating in the clinical trial. Also asked which role the patient would have preferred. Linear Analogue Self Assessment Pain Frequency was the only QOL domain that improved. Change from Baseline of Overall QOL Other Results Comparison of QOL score means and tumor response indicated no differences between best treatment responses (1 Partial Response, 15 Stable Disease, 12 Progressive Disease). Correlation of QOL scores with Treatment Satisfaction yielded moderate associations at best. The largest correlation occurred when comparing the decrease in pain frequency to the worthwhileness of participating on a clinical trial (Pearson r=0.66). There were no differences in changes in baseline in any QOL scores as compared between patients rating the parent trial as worthwhile (as indicated by a score of 5 or higher in the Trial Satisfaction Survey) vs those who did not. No trial enrolled more than 5 patients. Patient Demographics (N=30) Background Also asked other questions regarding overall emotional well being, social activity, spiritual well being, level of hopefulness, average fatigue, level of anxiety, mood and level of social support. • Phase I patients have hopes for a cure • Phase I trials are not designed to be curative • The disparity results in psychological challenges for the patient • This study explored the impact of Phase I trial participation on patient quality of life (QOL) Trial Satisfaction Survey Overall QOL decreased independent of select demographics. Directions: Please circle the number (0-10) best reflecting your response to the following statements: 1. It was worthwhile to go on this clinical trial. 0 1 2 3 4 5 6 7 8 9 10 Not worthwhile at all Very Worthwhile 2. Your quality of life has improved on this trial. 0 1 2 3 4 5 6 7 8 9 10 No improvement at all Very much improved 3. If you had to do it over, would you participate in this trial again. 0 1 2 3 4 5 6 7 8 9 10 Would never participate Would definitely participate 4. You would recommend going on this trial to others. 0 1 2 3 4 5 6 7 8 9 10 Would never recommend Would definitely recommend Conclusion Summary of Trial Satisfaction Methods Despite being satisfied with their participation in Phase I clinical trials, deficits in QOL appeared independently of AEs or tumor response. It hence would seem advisable to include assessments in Phase I trials in order to identify and improve patient QOL. • Patients enrolled onto Phase I studies between 9/10/2002 and 3/6/2007 were asked to complete: • the Control Preferences Scale (CPS) at baseline • the Linear Analogue Self Assessment (LASA) weekly during cycle 1 • the Trial Satisfaction (TS) survey at the end of cycle 1 • All QOL was measured on a 0-10 point scale, 10 being best. • The primary endpoint was the change in overall QOL at the end of cycle 1. Summary statistics were calculated for baseline covariates, QOL responses, treatment response and adverse events (AEs). Student’s t-test, Kruskall-Wallis and Pearson/Spearman correlation methodology were utilized. Contact Information: atherton@mayo.edu *All patients reported that they were actually playing the role they preferred. Patients had high trial satisfaction in spite of little QOL improvement.