Psychometric Evaluation of Clinical Outcomes Assessments in A Phase II Trial

Abstract

A 3 4 7 -A 7 6 6 of the DCEs for QLU-C10D utility elicitation in cancer patients. Methods: We aimed at 40 "think-aloud" cognitive interviews on feasibility and appropriateness in a heterogeneous sample of cancer patients (exclusion criteria: cognitive impairments, age < 18 and > 85, expected survival time < 6 months). Exactly the same survey material and design as in general population valuations were used, i.e. web-based presentation, 16 binary choice sets per respondent, hypothetical health states including 10 quality of life attributes and a survival time (1, 2, 5, 10 years). Number and reasons for non-participation were recorded. Results: 120 patients were approached and 42 interviews could be completed (43% female, mean age 59.3+-13.5 years, diagnoses: thyroid, breast, lung, pancreatic, haematological, and gastrointestinal cancers; stages: UICC 1 to 4; active treatment 64%, chemotherapy 48%, radiation therapy 45%, surgery only: 7%). Reasons for non-participation were largely organisational and required time resources. Most patients appreciated being asked about their perspective on quality of life and survival time and in principle would be willing to do the DCEs web-based. The trading-off was perceived very burdensome by 11% through evoking thoughts about their own death. Other points of criticism raised by more than 10% of patients were: too many choice sets; explanation was insufficient. For 26% of patients no changes were necessary. ConClusions: Apparently web-based DCEs for the valuation of the QLU-C10D are feasible for cancer patients. Survey adaptations to cancer patients' needs need to be discussed carefully against the background of comparability with general population utilities.objeCtives: This study utilized in-trial data to evaluate the validity and reliability of scores of 3 patient reported outcomes (PRO): Gastrointestinal (GI) Symptom Diary, Modified Satisfaction with Iron Chelation Therapy (MSICT) Questionnaire and Palatability Questionnaire. Methods: This was a randomized, open-label, multi-center, 2-arm, phase 2 study to evaluate the safety of Deferasirox film-coated tablet and Deferasirox dispersible tablet formulations in patients with transfusiondependent thalassemia or myelodysplastic syndrome treated for 24 weeks (wks). PROs were administered at wks 2, 3, 13 and end of treatment (EOT). Upon completion of data collection, psychometric analyses were conducted and included reliability (i.e. test-retest, internal consistency using Cronbach's α ), construct-related validity (including convergent and discriminant validity correlation estimates and conducting a set of known-groups analyses), and distribution-based testing. Results: Data were available for 154 patients for the MSICT and Palatability Questionnaires and for 149 patients for GI Symptom Diary (at either wks 2, 3, 13 or EOT). 51% of the patient population was females with a mean age of 35 years. All PRO scores demonstrated adequate test-retest reliability (intra-class correlations ≥ 0.69). Internal consistency reliability estimate...

Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias

“…Patients completed all PRO questions via a hand-held electronic device either at home (daily diaries) or during scheduled site visits. The instruments underwent full qualitative evaluation [ 22 ]; linguistic and psychometric evaluation of the PRO questionnaires was conducted during the trial, supporting the reliability and construct-related validity of the scores [ 23 ].…”

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia

“…Ten were multicentre trials (Calvaruso 2015; Elalfy 2015; Galanello 2006; Maggio 2009; Olivieri 1997; Pennell 2006; Pennell 2014; Taher 2017; Tanner 2007; Vichinsky 2007) and ranged from two centres in one country (Calvaruso 2015; Elalfy 2015; Olivieri 1997) to 44 centres in multiple countries (Vichinsky 2007). Six were single-centre trials (Aydinok 2007; Bahnasawy 2017; Badawy 2010; El Beshlawy 2008; Hassan 2016, Mourad 2003).…”

“…Follow-up ranged from six months in two trials (Bahnasawy 2017; Taher 2017) to five years in two trials (Calvaruso 2015; Maggio 2009). The remainder of the trials were of 12 months duration, except in the Badawy trial, which did not report follow-up time (Badawy 2010); and the Olivieri trial, which had 24 months follow-up (Olivieri 1997).…”

“…Trials were published between 1997 and 2017. Five were conducted in Egypt (Badawy 2010; Bahnasawy 2017; Elalfy 2015; El Beshlawy 2008, Hassan 2016); five in Italy (Calvaruso 2015; Galanello 2006; Maggio 2009; Pennell 2006; Tanner 2007); and three were international multicentre trials conducted in several countries (Pennell 2014; Taher 2017; Vichinsky 2007). One trial was conducted in each of the following countries: Turkey (Aydinok 2007); Lebanon (Mourad 2003); and Canada (Olivieri 1997).…”

“…Fourteen trials included only participants with β -thalassaemia major (Aydinok 2007; Badawy 2010; Bahnasawy 2017; Elalfy 2015; El Beshlawy 2008; Galanello 2006; Hassan 2016; Maggio 2009; Mourad 2003; Olivieri 1997; Pennell 2006; Pennell 2014; Taher 2017; Tanner 2007). One trial included only participants with SCD (Vichinsky 2007); and one trial included only participants with thalassaemia intermedia (Calvaruso 2015).…”