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Clinical Benefits in Colorectal Cancer

Clinical Benefits in Colorectal Cancer. Richard M. Goldberg, MD The Mayo Clinic, Minnesota. Clinical Benefit and Pornography. “I can’t define it but I know it when I see it.”. -Supreme Court Justice, Potter Stewart Jacobellis v. Ohio. What is Clinical Benefit in Colorectal Cancer?.

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Clinical Benefits in Colorectal Cancer

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  1. Clinical Benefits in Colorectal Cancer Richard M. Goldberg, MD The Mayo Clinic, Minnesota

  2. Clinical Benefit and Pornography “I can’t define it but I know it when I see it.” -Supreme Court Justice, Potter Stewart Jacobellis v. Ohio

  3. What is Clinical Benefit in Colorectal Cancer? Symptom control or improvement • RUQ pain disappears when mets shrink Lack of decline related to toxicity • Chemo fatigue disappears between treatments • Cumulative toxicity is not severe Toxicity patients choose to tolerate • Sensory neuropathy bothersome, patient will trade it for response

  4. Is Clinical Benefit Quantifiable? • Spitzer Uniscale • Spitzer QOL index (QLI) • EORTC QLQ C 30 version 2 • EORTC QLQ CR38 • Symptom distress scale • Functional living index-cancer (FLIC) • Picture face scale • Q TWIST

  5. EORTC QLQ C-30 • 2: Do you have trouble taking a long walk? • 17: During the last week have you had diarrhea? (none, a little, quite a bit, very much) • 22: During the last week did you worry? • 30: How would you rate your QOL during the past week? Very poor to excellent (7 numeric choices)

  6. Calculating Quality-Adjusted Life Years (QALY) Survival • TWiST = Time Without Symptoms or Toxicity • TWiST = OS - TOX - REL. • Assign utility coefficients to days with toxicity and post disease progression (Ut, Ur). • QALY = Ut * TOX + TWiST + Ur * REL.

  7. Do we need this? FLIC-C

  8. Or is this sufficient?

  9. QOL Assessment in Recent Randomized Trials in CRC Saltz Trial: QLQ C-30 version 2 • % returned not specified Douillard Trial: QLQ 3-30 • 60% returned Rougier Trial: QLQ C-30 • 68% returned Cunningham Trial: QLQ C-30 • 80% declining to 50%

  10. QOL Assessment in Recent Randomized Trials in CRC De Gramont Trial: QLQ C-30 • 83% returned Levi Trial: • Not done

  11. A Clinical Trials Example • 66 patients entered into a first line trial of 5-EU + 5-FU for 7/5 days • EORTC QLQ C-30 version 2 • Uniscale • Response rate = 26%, median survival 12.6 months

  12. Table 4: Summary Statistics of the Distribution of QOL Scores (N=66) (a) The 1st reporting period during treatment. If fewer than 1 post-baseline report, the final report was used for this variable. (b) EORTC QLQ-C30. Data not shown for all Functioning or Symptomology Scales.

  13. Figure 1

  14. Figure 2

  15. Missing Values • Incomplete data a common problem • Data Not Missing at Random (NMAR) • Use only complete cases • Impute the last reported value • Impute the mean of the reported values • Impute the minimum of the reported values • Impute zero at time of death • Multiple imputation methods

  16. Can Clinical Benefit Be AssessedIn All or Some Patients? • All • Only if the subjects fill out the assessment tools • Uniform definitions are essential • Accurate reporting is essential

  17. Does Clinical Benefit Data Have Regulatory/Clinical Utility? • It has value to the patient • It has value to the MD and family • It is difficult to quantify and ODAC members may have difficulty interpreting it • Cancer Outcome Measures Working Group (COMWG), cosponsored by FDA and NCI • Joseph Lipscomb, Outcomes Research Branch

  18. When Will Clinical Benefit Be Ready for Prime Time? • When ODAC is ready to accept this data in support of NDAs • When quantitative measures are agreed upon and reported uniformly • When and if patients, physicians, and regulatory personnel agree that the data is useful

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