Presentation on theme: "Amylou C. Dueck (Mayo Clinic) – Task 7 Lead Validation Study of the Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events."— Presentation transcript:
Amylou C. Dueck (Mayo Clinic) – Task 7 Lead Validation Study of the Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO- CTCAE)
Weekly “Task 7” teleconferences since March 2009 – Regular attendees All sites plus NCI and FDA Experts in validation studies Oncologists, psychometricians, statisticians Patient advocates – Adhoc experts Example: Arthur Stone (advice on design of recall substudy) Agenda and task documents available on the PRO- CTCAE Wiki – https://wiki.nci.nih.gov/x/cKul – Click on “Task 7” Process
Process (con’d 1) Literature search on validation of similar multi- symptom tools Developed a concept document Underwent review by a working group of the NCI’s Symptom/Quality of Life (SxQOL) Committee
Process (con’d 2) Developed interactive voice response (IVR) telephone platform with Perceptive Informatics (recall substudy) Developed protocol document Planned activation June 2010 Protocol modification underway to incorporate NCCCP sites
To assess measurement properties of newly developed PRO-CTCAE items: Validity Reliability Sensitivity Recall period N=900 to be accrued at 10 sites – Mayo, Memorial Sloan-Kettering, MD Anderson, Duke, Dana-Farber, 5 NCCCP Sites Objectives of Validation Study
Features of Validation Study Rigorous assessment of measurement properties on an item-by-item basis – Follows FDA guidance document even though PRO-CTCAE items are intended for adverse event monitoring instead of as primary endpoints Planned accrual of a diverse patient population – Six disease cohorts with a range of chemotherapies and/or radiation therapy – ECOG performance status 0-4 – Targeting minority patients through NCCCP participation
Eligibility (Inclusions) ≥18 years of age Disease and treatment matching 1 of the 6 following cohorts: – Adjuvant breast cancer on chemotherapy – Lymphoma/myeloma on chemotherapy – Metastatic prostate/bladder cancer on chemotherapy – Metastatic or locally advanced lung cancer on chemotherapy OR receiving daily radiation therapy for ≥21 more days (concurrent chemotherapy allowed) – Metastatic colorectal cancer on chemotherapy – Head/neck/gastroesophageal cancer receiving daily radiation therapy for ≥21 more days (concurrent chemotherapy allowed) Patient is expected to return to clinic in 1-6 weeks English speaking NOTE: CAN BE ANY ECOG PS – We are particularly interested in accruing patients with ECOG PS 2-4!
Eligibility (Exclusion) Clinically significant cognitive or memory impairment in the opinion of clinical or research staff
Schema Group A (N~100) – Patients receiving daily radiation for ≥21 more days in the Lung or Head/Neck/Gastroesophageal cohorts – Primary validation study plus recall and test-retest reliability substudies Group B (N~100) – Patients with 4-5 planned weekly clinic visits – Primary validation study plus recall substudy Group C (N~700) – All other eligible patients – Primary validation study (only requires two clinic visits 1-6 weeks apart) Rationale: Keep the study simple for most patients (Group C) but target accrual of patients with more frequent clinic visits (Groups A & B) for the recall and test-retest reliability substudies
* Clinical Anchors ECOG Performance Status Patient treatment information – RT yes/no – Surgery yes/no – Chemotherapy yes/no + regimen Patient medication information (yes/no for each) – Hormonal therapy – Opioid (narcotic) pain meds – Laxatives – Nausea meds – Sleep aids – Anti-diarrhea meds – Antacids – Anxiety meds – Inhalers – Depression meds.
Schema (Groups A & B) [ Patient compensation: $20 / visit ]
Statistics / Power Primary: Assess convergent validity of each PRO-CTCAE item – Compare each item between patients with ECOG PS 0-1 vs 2-4 at the Visit 1 time point using t-tests – Adjust for multiplicity using Hochberg’s step-up method – Power Core items: 99% for large effect size, 63-90% for moderate effect size Non-core items: 48-99% for large effect size, 9-65% for moderate effect size
Other Planned Analyses Assess the test-retest reliability of selected PRO-CTCAE items Assess the responsiveness (sensitivity to change) and clinical significance of each PRO- CTCAE item Investigate cut-points for notification of severe PRO-CTCAE symptoms
Other Planned Analyses (con’d) Compare clinician- and patient-reported CTCAE items Compare daily, 7-day, 2-week, 3-week, and 4- week recall periods Explore construction of an overall grade for symptoms with multiple items Assess measurement properties within ethnic/racial and education level subgroups
ASCO Poster Information Title: Validation study of the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE) Presenting author: Dueck AC Session: Trials in Progress Poster Session Date: Monday, June 07, 2010 Time: 8:00 AM - 12:00 PM Presentation Order: 175 Poster Board #: 47E Location: S Hall A2