Presentation on theme: "Validation Study of the Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) Amylou C. Dueck (Mayo Clinic)"— Presentation transcript:
1 Validation Study of the Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)Amylou C. Dueck (Mayo Clinic) – Task 7 Lead
2 Process Weekly “Task 7” teleconferences since March 2009 Regular attendeesAll sites plus NCI and FDAExperts in validation studiesOncologists, psychometricians, statisticiansPatient advocatesAdhoc expertsExample: Arthur Stone (advice on design of recall substudy)Agenda and task documents available on the PRO-CTCAE Wikihttps://wiki.nci.nih.gov/x/cKulClick on “Task 7”
3 Process (con’d 1)Literature search on validation of similar multi-symptom toolsDeveloped a concept documentUnderwent review by a working group of the NCI’s Symptom/Quality of Life (SxQOL) CommitteeLiterature search on validation of similar multi-symptom toolsMemorial Symptom Assessment Scale (MSAS)MD Anderson Symptom Inventory (MDASI)EORTC QLQ-C30 + modulesPROMISDeveloped a concept documentEstablished the overall design of the studyDefined disease cohortsIdentified core items (based on review of data from multiple sources)Assigned items to each cohort (included discussion of patient burden)Selected clinical anchorsDeveloped the recall substudy (based on literature review and expert input)Developed a comprehensive statistical analysis planUnderwent review by a working group of the NCI’s Symptom/Quality of Life (SxQOL) CommitteeProvided written responses to all inquiries3
4 Process (con’d 2)Developed interactive voice response (IVR) telephone platform with Perceptive Informatics (recall substudy)Developed protocol documentPlanned activation June 2010Protocol modification underway to incorporate NCCCP sitesParticipated in the development of an interactive voice response (IVR) telephone platform with Perceptive Informatics for the recall substudySelected itemsReviewed scripts & patient materialsTested platform within the testing and live environmentsProvided feedback on training materials for site staff4
5 Objectives of Validation Study To assess measurement properties of newly developed PRO-CTCAE items:ValidityReliabilitySensitivityRecall periodN=900 to be accrued at 10 sitesMayo, Memorial Sloan-Kettering, MD Anderson, Duke, Dana-Farber, 5 NCCCP Sites
6 Features of Validation Study Rigorous assessment of measurement properties on an item-by-item basisFollows FDA guidance document even though PRO-CTCAE items are intended for adverse event monitoring instead of as primary endpointsPlanned accrual of a diverse patient populationSix disease cohorts with a range of chemotherapies and/or radiation therapyECOG performance status 0-4Targeting minority patients through NCCCP participation
7 Eligibility (Inclusions) ≥18 years of ageDisease and treatment matching 1 of the 6 following cohorts:Adjuvant breast cancer on chemotherapyLymphoma/myeloma on chemotherapyMetastatic prostate/bladder cancer on chemotherapyMetastatic or locally advanced lung cancer on chemotherapy OR receiving daily radiation therapy for ≥21 more days (concurrent chemotherapy allowed)Metastatic colorectal cancer on chemotherapyHead/neck/gastroesophageal cancer receiving daily radiation therapy for ≥21 more days (concurrent chemotherapy allowed)Patient is expected to return to clinic in 1-6 weeksEnglish speakingNOTE: CAN BE ANY ECOG PSWe are particularly interested in accruing patients with ECOG PS 2-4!
8 Eligibility (Exclusion) Clinically significant cognitive or memory impairment in the opinion of clinical or research staff
9 Schema Group A (N~100) Group B (N~100) Group C (N~700) Patients receiving daily radiation for ≥21 more days in the Lung or Head/Neck/Gastroesophageal cohortsPrimary validation study plus recall and test-retest reliability substudiesGroup B (N~100)Patients with 4-5 planned weekly clinic visitsPrimary validation study plus recall substudyGroup C (N~700)All other eligible patientsPrimary 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
11 * Clinical Anchors ECOG Performance Status Patient treatment informationRT yes/noSurgery yes/noChemotherapy yes/no + regimenPatient medication information (yes/no for each)Hormonal therapyOpioid (narcotic) pain medsLaxativesNausea medsSleep aidsAnti-diarrhea medsAntacidsAnxiety medsInhalersDepression meds.
12 Schema (Groups A & B) [ Patient compensation: $20 / visit ]
13 Statistics / PowerPrimary: Assess convergent validity of each PRO-CTCAE itemCompare each item between patients with ECOG PS 0-1 vs 2-4 at the Visit 1 time point using t-testsAdjust for multiplicity using Hochberg’s step-up methodPowerCore items: 99% for large effect size, 63-90% for moderate effect sizeNon-core items: 48-99% for large effect size, 9-65% for moderate effect size
14 Other Planned Analyses Assess the test-retest reliability of selected PRO-CTCAE itemsAssess the responsiveness (sensitivity to change) and clinical significance of each PRO-CTCAE itemInvestigate cut-points for notification of severe PRO-CTCAE symptoms
15 Other Planned Analyses (con’d) Compare clinician- and patient-reported CTCAE itemsCompare daily, 7-day, 2-week, 3-week, and 4-week recall periodsExplore construction of an overall grade for symptoms with multiple itemsAssess measurement properties within ethnic/racial and education level subgroups
16 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 ACSession: Trials in Progress Poster SessionDate: Monday, June 07, 2010Time: 8:00 AM - 12:00 PMPresentation Order: 175Poster Board #: 47ELocation: S Hall A2