Assuring the patient centeredness of patient-reported outcomes: Content validity in medical product development and comparative effectiveness research

Not all patient-reported outcomes are patient-centered, and not all patient-centered outcomes are patient-reported. The essential characteristic of a patient-centered approach to outcome measurement is that it assesses concepts (i.e., health-related phenomena) that are considered most important by members of a given target population, based on direct input from representatives of that population. Concepts for measurement should not be selected based solely on convenience or interest to investigators. Patient-centered patient-reported outcome measures must meet this criterion and also be meaningful and comprehensible to members of a population when administered, including among those with diverse racial/cultural backgrounds and lower educational/literacy levels

Investigating associations between health-related quality of life and endocrine therapy underuse in women with early-stage breast cancer

Purpose Endocrine therapy (ET) underuse puts women at increased risk for breast cancer (BC) recurrence. Our objective was to determine if health-related quality of life (HRQOL) subgroups were associated with underuse. Methods Data came from the third phase of the Carolina Breast Cancer Study. We included 1,599 women with hormone receptor-positiveBCage 20 to 74 years.HRQOLwas measured, on average,5months postdiagnosis. Subgroups were derived using latent profile (LP) analysis. Underuse was defined as not initiating or adhering to ET by 36 months postdiagnosis. Multivariable logistic regression models estimated adjusted odds ratios (ORs) between HRQOL LPs and underuse. The best HRQOL LP was the reference. Chemotherapy- and race-stratified models were estimated, separately. Results Initiation analyses included 953womenwhohad not begun ET by their 5-month survey. Of these, 154 never initiated ET. Adherence analyses included 1,114 ET initiators, of whom 211 were nonadherent.HRQOLwas not significantly associated with noninitiation, except among nonchemotherapy users, with membership in the poorest LP associated with increased odds of noninitiation (adjusted OR, 5.5; 95% CI, 1.7 to 17.4). Membership in the poorest LPs was associated with nonadherence (LP1: adjusted OR, 2.2; 95% CI, 1.2 to 4.0 and LP2: adjusted OR,1.9;95%CI, 1.1 to 3.6). Membershipin the poorestLPwasassociated with nonadherence among nonchemotherapy users (adjusted OR, 2.1; 95% CI, 1.2 to 5.1). Conclusion Ourresults suggestwomenwith poorHRQOLduring active treatmentmaybe at increased risk for ET underuse. Focusing on HRQOL, a modifiable factor, may improve targeting of future interventions early in theBCcontinuum to improve ET initiation and adherence and prevent BC recurrence

Racial differences in user experiences and perceived value of electronic symptom monitoring in a cohort of black and white bladder and prostate cancer patients

Purpose: Electronic patient-reported outcomes (ePROs) are increasingly being used for symptom monitoring during routine cancer care, but have rarely been evaluated in diverse patient populations. We assessed ePRO user experiences and perceived value among Black and White cancer patients. Methods: We recruited 30 Black and 49 White bladder and prostate cancer patients from a single institution. Participants reported symptoms using either a web-based or automated telephone interface over 3 months and completed satisfaction surveys and qualitative interviews focused on user experiences and value. Using a narrative mixed methods approach, we evaluated overall and race-specific differences in ePRO user experiences and perceived value. Results: Most participants selected the web-based system, but Blacks were more likely to use the automated telephone-based system than Whites. In satisfaction surveys, Whites more commonly reported ease in understanding and reporting symptoms compared with Blacks. Blacks more often reported that the ePRO system was helpful in facilitating symptom-related discussions with clinicians. During interviews, Blacks described how the ePRO helped them recognize symptoms, while Whites found value in better understanding and tracking symptoms longitudinally. Blacks also expressed preferences for paper-based ePRO options due to perceived ease in better understanding of symptom items. Conclusion: Electronic patient-reported outcomes are perceived as valuable for variable reasons by Black and White cancer populations, with greater perceived value for communicating with clinicians reported among Blacks. To optimize equitable uptake of ePROs, oncology practices should offer several ePRO options (e.g., web-based, phone-based), as well as paper-based options, and consider the e-health literacy needs of patients during implementation

Activities, function, and health-related quality of life (HRQOL) of older adults with cancer

Objective This study aims to (1) describe the activities, function, and health-related quality of life (HRQOL) of a large sample of older adults (age ≥ 65) with cancer, (2) identify the associations with demographics, cancer type, comorbid conditions, and ability to participate in activities and functional status. Materials and Methods The Health Registry/Cancer Survivorship Cohort is an institutional database designed to aid cancer survivorship research. The registry includes three measures of patient-reported HRQOL: FACT-G and PROMIS® Global measures for physical and mental health. Other measures included in the registry are cancer type, date from diagnosis, number of comorbid conditions and specific conditions and their limitations in daily activity, and self-reported daily activity/function. Results Our sample consists of 768 older adults with cancer, mean age 72 years, 60% female, and 90% White. Mean scores for HRQOL: FACT-G (85, range: 25–108), PROMIS-physical (48, range: 16–67) and, PROMIS-mental (51, range: 21–67). In multivariable models, Black race, one or more comorbid conditions, and Gastrointestinal cancer (p < .05), and patient- reported decreased levels of activities/function were all independently associated with poor HRQOL (p < .0001). Conclusions Older Black adults with cancer, those that have high comorbidity burden, with gastrointestinal cancers and those that report decreased ability to participate in daily activities/function reported poorer HRQOL. As geriatric oncology moves towards trying to identify who may need supportive services, this study demonstrated that a one question patient-reported level of activities and functional ability were independently associated with physical, mental, and cancer-specific HRQOL

Electronic symptom monitoring in pediatric patients hospitalized for chemotherapy

Background: Using patient-reported outcomes for symptom monitoring in oncology has resulted in significant benefits for adult patients with cancer. The feasibility of this approach has not been established in the routine care of children with cancer. Methods: The Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE) is an item library that enables children and caregivers to self-report symptoms. Ten symptom items from the Ped-PRO-CTCAE were uploaded to an online platform. Patients at least 7 years old and their caregivers were prompted by text/email message to electronically self-report daily during a planned hospitalization for chemotherapy administration. Symptom reports were emailed to the clinical team caring for the patient, but no instructions were given regarding the use of this information. Rates of patient participation and clinician responses to reports were systematically tracked. Results: The median age of the participating patients (n = 52) was 11 years (range, 7-18 years). All patients and caregivers completed an initial login, with 92% of dyads completing at least 1 additional symptom assessment during hospitalization (median, 3 assessments; range, 0-40). Eighty-one percent of participating dyads submitted symptom reports on at least half of hospital days, and 54% submitted reports on all hospital days. Clinical actions were taken in response to symptom reports 21% of the time. Most patients felt that the system was easy (73%) and important (79%). Most clinicians found symptom reports easy to understand and useful (97%). Conclusions: Symptom monitoring using patient-reported outcome measures for hospitalized pediatric oncology patients is feasible and generates data valued by clinicians and patients

Examining health-related quality of life patterns in women with breast cancer

Purpose: We aimed to identify subgroups of women with breast cancer who experience different health-related quality of life (HRQOL) patterns during active treatment and survivorship and determine characteristics associated with subgroup membership. Methods: We used data from the third phase of the population-based Carolina Breast Cancer Study and included 2142 women diagnosed with breast cancer from 2008 to 2013. HRQOL was measured, on average, 5 and 25 months post diagnosis. Latent profile analysis was used to identify HRQOL latent profiles (LPs) at each time point. Latent transition analysis was used to determine probabilities of women transitioning profiles from 5 to 25 months. Multinomial logit models estimated adjusted odds ratios (aORs) and 95% confidence intervals for associations between patient characteristics and LP membership at each time point. Results: We identified four HRQOL LPs at 5 and 25 months. LP1 had the poorest HRQOL and LP4 the best. Membership in the poorest profile at 5 months was associated with younger age aOR 0.95; 0.93–0.96, White race aOR 1.48; 1.25–1.65, being unmarried aOR 1.50; 1.28–1.65 and having public aOR 3.09; 1.96–4.83 or no insurance aOR 6.51; 2.12–20.10. At 25 months, Black race aOR 1.75; 1.18–1.82 was associated with the poorest profile membership. Black race and smoking were predictors of deteriorating to a worse profile from 5 to 25 months. Conclusions: Our results suggest patient-level characteristics including age at diagnosis and race may identify women at risk for experiencing poor HRQOL patterns. If women are identified and offered targeted HRQOL support, we may see improvements in long-term HRQOL and better breast cancer outcomes

Geriatric Assessment as a Predictor of Tolerance, Quality of Life, and Outcomes in Older Patients With Head and Neck Cancers and Lung Cancers Receiving Radiation Therapy

Purpose To evaluate the association between functional status based on a geriatric assessment (GA) and outcomes of tolerance to treatment in patients with lung or head and neck cancer receiving radiation therapy (RT) or chemoradiation (CRT). Methods and Materials A prospective cohort study was conducted in patients aged ≥65 years with head and neck cancer or lung cancer undergoing curative intent RT or CRT. Pretreatment GA, health-related quality of life (HRQoL), and patient-reported outcomes (PRO) were obtained. Questionnaires were repeated biweekly during RT and at 6 weeks after treatment. Dysfunction was defined as scores 3-day treatment delay, change in RT or CRT regimen, or death. Associations of dysfunction with tolerance to radiation therapy, HRQoL changes, and PRO ratings were evaluated. Results Of the 50 patients accrued, 46 had evaluable data. Mean age was 72.5 years (range, 65-92 years). At baseline, 37% had dysfunction. Poor tolerance to RT or CRT occurred in 39%. There was no association between dysfunction and tolerance. Patients with dysfunction had lower baseline HRQoL scores. From baseline to end of RT, those with baseline dysfunction had less of a decline in Role Functioning (P=.01) and Global Health Score (P=.04) domains. However, from end of RT to 6-week follow-up, those with dysfunction were more likely to continue to drop in the Physical, Role Functioning, and Social domains (all P<.01). Dysfunction at baseline was also associated with higher severity of certain PROs. Conclusions Pretreatment dysfunction was associated with continued decline and lack of recovery of HRQoL in this patient population. Larger studies could further elucidate the GA's predictive value

Frailty and health-related quality of life in older women with breast cancer

Purpose: In older women, breast cancer and its treatment can have profound impact on their physical, mental, and social health, especially in frail patients. This study evaluated the association between frailty and long-term health-related quality of life (HRQOL) in older women undergoing breast cancer treatment. Methods: Using the Carolina Senior Registry (CSR), participants with breast cancer were contacted to complete a follow-up HRQOL questionnaire (median 4 years). Baseline Geriatric Assessment (GA) variables were used to calculate the Carolina Frailty Index (CFI) and categorize participants as robust, pre-frail, or frail. Outcomes included HRQOL domains of physical function, social roles, fatigue, depression, anxiety, pain, and sleep disturbance assessed using PROMIS® instruments. Regression modeling compared outcomes between frailty groups using adjusted mean differences (AMD). Results: Of 190 eligible patients, 63 completed follow-up HRQOL survey. Mean age was 70 years (range 65–86) and 91% were white. Based on the CFI, 49 (78%) patients were robust, 11 (18%) pre-frail, and 3 (5%) frail. After controlling for age and cancer stage, patients identified as pre-frail/frail reported worse physical function (AMD − 9.2, p < 0.001) and social roles (AMD − 7.2, p = 0.002) and more fatigue (AMD 7.6, p = 0.008), depression (AMD 5.6, p = 0.004), and sleep disturbance (AMD 6.9, p = 0.008) compared to robust patients at follow-up. Conclusions: Frailty in older women with breast cancer was associated with worse long-term HRQOL outcomes. Further research is needed to develop interventions for frail patients at-risk for reduced HRQOL

Composite grading algorithm for the National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

Background: The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events is an item library designed for eliciting patient-reported adverse events in oncology. For each adverse event, up to three individual items are scored for frequency, severity, and interference with daily activities. To align the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events with other standardized tools for adverse event assessment including the Common Terminology Criteria for Adverse Events, an algorithm for mapping individual items for any given adverse event to a single composite numerical grade was developed and tested. Methods: A five-step process was used: (1) All 179 possible Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events score combinations were presented to 20 clinical investigators to subjectively map combinations to single numerical grades ranging from 0 to 3. (2) Combinations with <75% agreement were presented to investigator committees at a National Clinical Trials Network cooperative group meeting to gain majority consensus via anonymous voting. (3) The resulting algorithm was refined via graphical and tabular approaches to assure directional consistency. (4) Validity, reliability, and sensitivity were assessed in a national study dataset. (5) Accuracy for delineating adverse events between study arms was measured in two Phase III clinical trials (NCT02066181 and NCT01522443). Results: In Step 1, 12/179 score combinations had <75% initial agreement. In Step 2, majority consensus was reached for all combinations. In Step 3, five grades were adjusted to assure directional consistency. In Steps 4 and 5, composite grades performed well and comparably to individual item scores on validity, reliability, sensitivity, and between-arm delineation. Conclusion: A composite grading algorithm has been developed and yields single numerical grades for adverse events assessed via the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events, and can be useful in analyses and reporting