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We compared patient-reported outcomes (PROs) with once-weekly carfilzomib 70 mg/m2 (Kd70 mg/m2) vs. twice-weekly carfilzomib 27 mg/m2 (Kd27 mg/m2) plus dexamethasone in relapsed or refractory multiple myeloma (RRMM). Patient-reported convenience/satisfaction collected at Cycle 2, Day 1 was compared between groups using logistic regression. European Organization for Research and Treatment of Cancer QOL Questionnaire (QLQ-C30), MM-module (QLQ-MY20), and EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) questionnaires were administered at baseline, then every other cycle. PROs were compared between groups using mixed models for repeated measures. Times from randomization to first deterioration (TTD) in scores were analyzed using Cox regression. PRO analyses included 469 patients. Once-weekly Kd70 mg/m2 patients reported greater convenience (odds ratio [OR], 4.98; p < 0.001) and satisfaction (OR, 2.41; p = 0.059) vs. twice-weekly Kd27 mg/m2. The mixed models for repeated measures demonstrated no clinically meaningful differences in scores between treatment arms. Clinically meaningful deterioration in QLQ-C30 Global Health Status/QOL rates were 34.2% (once-weekly Kd70 mg/m2) vs. 40.3% (twice-weekly Kd27 mg/m2). TTD was longer for once-weekly Kd70 mg/m2 vs. twice-weekly Kd27 mg/m2 for QLQ-C30 fatigue (HR, 0.79; p = 0.035), QLQ-MY20 disease symptoms (HR, 0.67; p = 0.008), EQ-5D-5L index score (HR, 0.58; p = 0.002), and EQ-5D-5L Visual Analog Scale (HR, 0.75, p = 0.031). Once-weekly Kd70 mg/m2 improved convenience/satisfaction, and reduced HRQOL deterioration vs. twice-weekly Kd27 mg/m2, supporting convenient, once-weekly Kd70 mg/m2 dosing in RRMM. © 2019, The Author(s).

Background: Health-related quality of life (HRQOL) is a key aspect for chronic myeloid leukemia (CML) patients. The aim of this study was to develop a disease-specific HRQOL questionnaire for patients with CML to supplement the European Organization for Research and Treatment of Cancer (EORTC)-QLQ C30. Patients and methods: The process followed a predefined and systematic stepwise iterative process as defined by the EORTC guidelines for questionnaire development. The process was divided into 3 phases: (1) generation of relevant HRQOL issues, (2) operationalization of the HRQOL issues into a set of items, and (3) pretesting the questionnaire for relevance and acceptability. Descriptive statistics and psychometric analyses were also performed. Results: Overall, 655 CML patients were enrolled in 10 countries including the USA and countries in Europe and Asia. Interviews with health-care professionals experienced in CML (n = 59) were also conducted. Results from the interviews, clinical experiences, and statistical analyses were used to develop the EORTC QLQ-CML24. The final module consists of 24 items assessing the following aspects: symptom burden, impact on daily life and on worry/mood, body image problems, and satisfaction with care and with social life. Internal consistency, assessed with Cronbach's alpha coefficients, ranged from 0.73 to 0.83 for the proposed scales. Conclusion: The EORTC QLQ-CML24 is an internationally developed HRQOL questionnaire for CML patients, and its implementation in clinical research and practice can provide important information to facilitate clinical decision-making. © 2013 Springer Science+Business Media Dordrecht.

Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) might be crucial in comparing effectiveness of treatments as they could provide invaluable information to better inform clinical decision-making. This is particularly true in the era of targeted therapies (TT). A systematic review was undertaken on all studies with CML patients published from 1980 to 2010 and including a PRO evaluation. Out of 619 articles scrutinized, 15 met eligibility criteria and no study was published before 1995. Six dealt mainly with interferon-based therapies, 7 with bone marrow transplantation and only 2 evaluated PROs in the context of TT. No disease-specific, validated PRO instrument for these patients was found. The main evidence being that Imatinib provides clear advantage in terms of HRQOL over interferon-based treatments. There is lack of data concerning PROs in patients treated with current TT. Documenting HRQOL and side effects of CML treatments, from the patients' perspective is needed to evaluate overall treatment effectiveness and net clinical benefit of newer therapeutic strategies. (C) 2011 Elsevier Ireland Ltd. All rights reserved

Purpose: To use published literature to estimate large, medium, and small differences in quality of life (QOL) data from the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30). Methods: An innovative method combining systematic review of published studies, expert opinions, and meta-analysis was used to estimate large, medium, and small differences for QLQ-C30 scores. Published mean data were identified from the literature. Differences (contrasts) between groups (eg, between treatment groups, age groups, and performance status groups) were reviewed by 34 experts in QOL measurement and cancer treatment. The experts, blinded to actual QOL results, were asked to predict these differences. A large difference was defined as one representing unequivocal clinical relevance. A medium difference was defined as likely to be clinically relevant but to a lesser extent. A small difference was one believed to be subtle but nevertheless clinically relevant. A trivial difference was used to describe circumstances unlikely to have any clinical relevance. Actual QOL results were combined using meta-analytic techniques to estimate differences corresponding to small, medium, or large effects. Results: Nine hundred eleven articles were identified, leading to 152 relevant articles (2,217 contrasts) being reviewed by at least two experts. Resulting estimates from the meta-analysis varied depending on the subscale. Thus, the recommended minimum to detect medium differences ranges from 9 (cognitive functioning) to 19 points (role functioning). Conclusion: Guidelines for the size of effects are provided for the QLQ-C30 subscales. These guidelines can be used for sample size calculations for clinical trials and can also be used to aid interpretation of differences in QLQ-C30 scores. © 2010 by American Society of Clinical Oncology.

AIM: To review reporting standard, presentation and interpretation for quality of life (QOL) outcomes in randomised controlled trials (RCTs) using the European Organisation for Research and Treatment of Cancer quality of life questionnaire core 30 (EORTC QLQ-C30). METHODS: Cancer RCTs reporting EORTC QLQ-C30 data were identified and reviewed against a reporting quality checklist. Interpretation/presentation methods for QOL data were also recorded. RESULTS: Eighty-two papers were reviewed. Seventy percent met criteria for high quality reporting; 94% reported mean scores; 84% presented results in tables/graphs; 80% reported p-values or statistical significance. Clinical significance was addressed in 38%. Where clinical significance was not addressed, reliance was usually on statistical significance to interpret the results. DISCUSSION: EORTC QLQ-C30 results are generally reported well, although it was common to rely on statistical significance alone for interpreting results. Whilst interpretation in terms of clinical significance has improved in recent years, there is still a lack of robust clinical interpretation of QOL results even in papers reported to a high standard.