Patient-Reported Factors in Treatment Satisfaction in Patients with Relapsed/Refractory Multiple Myeloma (RRMM).

BACKGROUND: Therapy choices in relapsed/refractory multiple myeloma (RRMM) should consider patient satisfaction with treatment, because it is associated with adherence to therapy, health outcomes, and medical safety. The primary objective of this pilot cross-sectional observational study was to ascertain factors associated with patient-reported treatment satisfaction in RRMM. PATIENTS AND METHODS: Patients with a self-reported diagnosis of RRMM recruited from PatientsLikeMe, MyelomaCrowd, and Facebook were administered an electronic survey that included questions on demographics and clinical history, treatment experience, economic burden, and standardized patient-reported outcome measures, including the Treatment Satisfaction Questionnaire for Medication, Eastern Cooperative Oncology Group performance status (ECOG PS) measure, and Work Productivity and Activity Impairment Questionnaire: Specific Health Problem V2.0. Univariable and multivariable analyses were used to identify predictors of patient-perceived treatment satisfaction. RESULTS: One hundred sixty patients with RRMM participated in the study, with a median of two prior relapses and 66.3% reporting the most recent relapse within the last 12 months. ECOG PS ≥2 was associated with lower patient-reported global satisfaction and perceived effectiveness of current treatment. In addition to shorter time spent receiving therapy, orally administered treatment was the strongest predictor of higher satisfaction with treatment convenience. For patients receiving an injectable drug-containing regimen versus an all-oral regimen, respectively, time spent receiving multiple myeloma-directed therapy was higher (12.6 vs. 4.0 hours per month), and total monthly indirect costs were $1,033 and $241. CONCLUSION: Poor ECOG PS was linked to reduced treatment satisfaction and perceived effectiveness of current therapy, whereas an all-oral regimen was associated with increased treatment convenience satisfaction. IMPLICATIONS FOR PRACTICE: This study suggests that attributes including better Eastern Cooperative Oncology Group performance status, less time spent receiving treatment, and oral route of treatment administration lead to higher patient-perceived satisfaction with relapsed/refractory multiple myeloma (RRMM) treatment. Oral route of administration was also associated with less time spent receiving treatment and reduced economic burden for patients. Increased attention to these factors in shared treatment decision making is warranted to help identify individual patient needs, preferences, and expectations for RRMM treatments, to resolve dissatisfaction issues, and to improve the experience of patients with RRMM.

A Modular Health-Related Quality of Life Instrument for Electronic Assessment and Treatment Monitoring: Web-Based Development and Psychometric Validation of Core Thrive Items.

BACKGROUND: Patient-reported outcome (PRO) measures describe natural history, manage disease, and measure the effects of interventions in trials. Patients themselves increasingly use Web-based PRO tools to track their progress, share their data, and even self-experiment. However, existing PROs have limitations such as being: designed for paper (not screens), long and burdensome, negatively framed, under onerous licensing restrictions, either too generic or too specific. OBJECTIVE: This study aimed to develop and validate the core items of a modular, patient-centric, PRO system (Thrive) that could measure health status across a range of chronic conditions with minimal burden. METHODS: Thrive was developed in 4 phases, largely consistent with Food and Drug Administration guidance regarding PRO development. First, preliminary core items (common across multiple conditions: core Thrive items) were developed through literature review, analysis of approximately 20 existing PROs on PatientsLikeMe, and feedback from psychometric and content experts. Second, 2 rounds of cognitive interviews were iteratively conducted with patients (N=14) to obtain feedback on the preliminary items. Third, core Thrive items were administered electronically along with comparator measures, including 20-item Short-Form General Health Survey (SF)-20 and Patient Health Questionnaire (PHQ)-9, to a large sample (N=2002) of adults with chronic diseases through the PatientsLikeMe platform. On the basis of theoretical and empirical rationale, items were revised or removed. Fourth, the revised core Thrive items were administered to another sample of patients (N=704) with generic and condition-specific comparator measures. A psychometric evaluation, which included both modern and classical test theory approaches, was conducted on these items, and several more items were removed. RESULTS: Cognitive interviews helped to remove confusing or redundant items. Empirical testing of subscales revealed good internal consistency (Cronbach alpha=.712-.879), test-retest reliability (absolute intraclass correlations=.749-.912), and convergent validity with legacy PRO scales (eg, Pearson r=.5-.75 between Thrive subscales and PHQ-9 total). The finalized instrument consists of a 19-item core including 5 multi-item subscales: Core symptoms, Abilities, Mobility, Sleep, and Thriving. Results provide evidence of construct (content, convergent) validity, high levels of test-retest and internal consistency reliability, and the ability to detect change over time. The items did not exhibit bias based on gender or age, and the items generally functioned similarly across conditions. These results support the use of Thrive Core items across diverse chronic patient populations. CONCLUSIONS: Thrive appears to be a useful approach for capturing important domains for patients with chronic conditions. This core set serves as a foundation to begin developing modular condition-specific versions in the near future. Cross-walking against traditional PROs from the PatientsLikeMe platform is underway, in addition to clinical validation and comparison with biomarkers. Thrive is licensed under Creative Commons Attribution ShareAlike 4.0.

Treatment Satisfaction and Burden of Illness in Patients with Newly Diagnosed Multiple Myeloma.

OBJECTIVES: This observational study identified attributes of patient-reported satisfaction with therapy for multiple myeloma (MM), described the treatment-related time burden and indirect costs, and investigated the effect of administration route (oral vs. injectable) on these outcomes among patients with newly diagnosed MM (NDMM) and among caregivers. METHODS: Patients residing in the USA with a self-reported diagnosis of NDMM were recruited from PatientsLikeMe, MyelomaCrowd, and Facebook (16 December 2016 and 6 July 2017) to complete an electronic survey including questions on treatment experience, economic burden, and standardized patient-reported outcome measures, including the Treatment Satisfaction Questionnaire for Medication with three domains (global satisfaction, effectiveness, and convenience) and the Work Productivity and Activity Impairment Questionnaire. Univariate and multivariate analyses identified predictors of patient-perceived treatment satisfaction. RESULTS: Among 188 patients, worse Eastern Cooperative Oncology Group performance status (ECOG PS) was correlated with lower patient-perceived effectiveness and convenience of their current treatment. White race and oral administration route were independently correlated with higher patient-perceived convenience of treatment. Injectable therapy use was associated with a trend towards increased activity impairment (43 vs. 34%; p = 0.05) and significantly higher time burden of treatment administration, with threefold higher adjusted indirect costs of MM therapy compared with solely orally administered therapies (monthly mean $US482 vs. 153; 2016 values; p < 0.0001). CONCLUSIONS: Factors associated with patient-perceived satisfaction with NDMM treatment-ECOG PS, race, administration route-warrant increased attention in shared treatment decision making to help identify patient needs and improve the patient's treatment experience. The use of orally administered therapies could improve patients' activity impairment and reduce the time burden associated with therapy.

Understanding 'Good Health care' from the Patient's Perspective: Development of a Conceptual Model Using Group Concept Mapping.

BACKGROUND: There is an increasing focus on measuring performance indicators of health care providers, but there is a lack of patient input into what defines 'good care.' OBJECTIVE: The primary objective was to develop a conceptual model of 'good health care' from the patient's perspective. Exploratory analyses were also conducted to investigate (1) differences in patient priorities based on demographic and clinical factors, and (2) differences between patients and health stakeholders (e.g., clinicians, researchers) with respect to patient health care priorities. METHOD: These objectives were accomplished using group concept mapping. Following statement generation, PatientsLikeMe members, Baltimore community members recruited through a university-affiliated clinic, and stakeholders individually sorted the statements into meaningful categories and rated the statements with respect to importance. Qualitative and quantitative analyses generated a final conceptual model. RESULTS: One hundred and fifty-seven patients and 17 stakeholders provided input during statement generation. The 1779-statement pool was reduced to 79 statements for the structuring (sorting and rating) activities. In total, 221 patients and 16 stakeholders completed structuring activities through group concept mapping software. Results yielded a 10-cluster solution, and patient priorities were found to be relatively invariant across demographic/clinical groups. Results were also similar between patients and stakeholders. CONCLUSIONS: This comprehensive qualitative and quantitative investigation is an important first step in developing patient-reported outcome performance measures that capture the aspects of health care that are most important and relevant for patients. Limitations and future directions are discussed.