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Patient-Reported Outcomes Measurement Information System (PROMIS) enables the use of computer adaptive testing (CAT). The aim of this prospective cohort study was to compare the most commonly used disease-specific instruments with PROMIS CAT questionnaires in patients with trauma. All patients with trauma (ages 18-75) who underwent an operative intervention for an extremity fracture between June 1, 2018, and June 30, 2019, were included. The disease-specific instruments were the Quick Disabilities of the Arm, Shoulder, and Hand for upper extremity fractures and the Lower Extremity Functional Scale (LEFS) for lower extremity fractures. Pearson's correlation (r) between the disease-specific instruments and the PROMIS CAT questionnaires (PROMIS Physical Function, PROMIS Pain Interference, and PROMIS Ability to Participate in Social Roles and Activities) was calculated at week 2, week 6, month 3, and month 6. Construct validity and responsiveness were calculated. A total of 151 patients with an upper extremity fracture and 109 patients with a lower extremity fracture were included. At month 3 and month 6, the correlation was strong between the LEFS and PROMIS Physical Function (r = 0.88 and r = 0.90, respectively), and at month 3, the correlation was strong between the LEFS and PROMIS Social Roles and Activities (r = 0.72). At week 6, month 3, and month 6, there was a strong correlation between the Quick Disabilities of the Arm, Shoulder, and Hand and PROMIS Physical Function (r = 0.74, r = 0.70, and r = 0.76, respectively). The PROMIS CAT measures are acceptably related to existing non-CAT instruments and may be a useful tool during follow-up after operative interventions for extremity fractures. • Patient-Reported Outcomes Measurement Information System computer adaptive testing (CAT) questionnaires could be used as a supplement to outpatient visits to provide insight into perceived health and the domain of experienced limitations. Knowledge about the results and the clinical consequences has yet to grow, but using these questionnaires could enhance communication between clinicians and patients. • Patient-Reported Outcomes Measurement Information System CAT measures are acceptably related to existing non-CAT instruments, resulting in a lower response burden than that with injury-specific instruments. [ABSTRACT FROM AUTHOR]

Cystic fibrosis (CF) limits survival and negatively affects health-related quality of life (HRQOL). Cost-effectiveness analysis (CEA) may be used to make reimbursement decisions for new CF treatments; nevertheless, generic utility measures used in CEA, such as EQ-5D, are insensitive to meaningful changes in lung function and HRQOL in CF. Here we develop a new, CF disease–specific, preference-based utility measure based on the adolescent/adult version of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely used, CF-specific, patient-reported measure of HRQOL. Blinded CFQ-R data from 4 clinical trials (NCT02347657, NCT02392234, NCT01807923, and NCT01807949) were used to identify discriminating items for a classification system using psychometric (eg, factor and Rasch) analyses. Thirty-two health states were selected for a time trade-off (TTO) exercise with a representative sample of the UK general population. TTO utilities were used to estimate a preference-based scoring algorithm by regression analysis (tobit models with robust standard errors clustered on participants with censoring at −1). A classification system with 8 dimensions (CFQ-R-8 dimensions; physical functioning, vitality, emotion, role functioning, breathing difficulty, cough, abdominal pain, and body image) was generated. TTO was completed by 400 participants (mean age, 47.3 years; 49.8% female). Among the regression models evaluated, the tobit heteroscedastic–ordered model was preferred, with a predicted utility range from 0.236 to 1, no logical inconsistencies, and a mean absolute error of 0.032. The CFQ-R-8 dimensions is the first disease-specific, preference-based scoring algorithm for CF, enabling estimation of disease-specific utilities for CEA based on the well-validated and widely used CFQ-R. • Cost-effectiveness analysis (CEA) may guide reimbursement decisions for cystic fibrosis (CF) treatments; nevertheless, standard measures of utility used in CEA are insensitive to changes in lung function and health-related quality of life in CF. • We used blinded Cystic Fibrosis Questionnaire-Revised (CFQ-R) data from 4 clinical trials to develop the CFQ-R-8 dimensions (CFQ-R-8D), a new, CF disease–specific, preference-based utility measure with 8 dimensions (physical functioning, vitality, emotion, role functioning, breathing difficulty, cough, abdominal pain, and body image). • The CFQ-R-8D enables disease-specific utilities for use in CEA to be generated from the CFQ-R, a measure that is widely used in CF trials. [ABSTRACT FROM AUTHOR]

A frequently used patient-reported outcome measure for assessing physical functioning in patients with hip problems is the 5-item Hip Disability and Osteoarthritis Outcome Score-Physical Function Shortform (HOOS-PS). Nevertheless, its content validity (whether this instrument adequately reflects the construct of physical functioning) is unknown. This study aimed to assess the content validity of the HOOS-PS. A quantitative and qualitative research approach was used. Physical functioning was defined as the ability to perform activities that require physical actions, ranging from self-care to more complex activities that require a combination of skills, often within a social context. Patients (n = 51) and experts (n = 25) completed questionnaires regarding the relevance, comprehensiveness, and comprehensibility of the HOOS-PS. Semistructured interviews (n = 5) explored issues identified in the quantitative data in more depth. Thematic content analysis was conducted using a coding frame. One of the 5 items was considered relevant to measure physical functioning. Comprehensiveness was considered insufficient by both patients and experts. Furthermore, comprehensibility was considered inadequate. Several items were found ambiguous or double barreled. Regarding interpretability, floor or ceiling effects were not found. This study showed concerns about the content validity of the HOOS-PS: most items are considered not relevant, the HOOS-PS is considered not comprehensive, and several items are considered not comprehensible. These findings challenge the applicability of the HOOS-PS in clinical practice, research, value-based healthcare, and benchmarking. • This study showed that the content validity of the Hip Disability and Osteoarthritis Outcome Score-Physical Function Shortform (HOOS-PS) in patients undergoing hip arthroplasty is problematic in 3 areas: (1) lack of relevance, (2) insufficient comprehensiveness, and (3) insufficient comprehensibility of the items. • Given that the HOOS-PS does not reflect physical functioning in total hip arthroplasty patients, this will challenge the applicability of the HOOS-PS in clinical practice, research, and benchmarking. • The lack of content validity in the observed data can subsequently lead to drawing incorrect conclusions. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to determine the relationship between frequently used patient-reported outcome (PRO) measures and a multitask performance outcome (PerfO) measure of general physical function (PF) and to examine the association of these measures with depressive mood, pain, and age.Methods: Frequently used PRO measures of general PF (Patient-Reported Outcomes Measurement Information System [PROMIS] PF item bank, PROMIS PF Short Form 20a, Short Form 36 Physical Function Scale) and a PerfO test battery, namely, the Physical Performance Test (PPT), were administered to 78 adult patients from 3 inpatient clinics (cardiology and angiology, rheumatology and clinical immunology, and psychosomatic medicine) at Charité - Universitätsmedizin Berlin. Pearson correlations were used to investigate the associations between PRO measures and the PPT. To explore the predictive value of age, depressive symptoms, and pain intensity, we conducted multiple linear regression analysis for each PF measure.Results: We found strong linear relationships between PRO measures and PPT sum scores. Correlations between PPT sum scores and PROMIS PF T-scores were r > 0.75. For all PRO and PerfO measures, age was a predictor of general PF whereas depressive mood was not found to be a relevant predictor. Moreover, pain intensity was found to be a significant predictor of PRO measures but not for PPT sum scores.Conclusions: Our findings suggest that frequently used PRO measures and a multitask PerfO measure of general PF can be used to measure a common PF construct. Nevertheless, PF scores based on PRO measures should ideally be controlled for self-rated pain intensity. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to compare the treatment preference among oncologists, patients with lung cancer, and their family members in China.Methods: A face-to-face discrete choice experiment survey was conducted among oncologists, patients, and their family members. Participants completed 13 choice sets describing 6 key attributes, namely, overall survival time, risk of severe adverse effect, severity of pain, appetite, physical functioning status, and monthly cost. Mixed logit model and latent class analysis were used to estimate attribute level preference weights and the relative importance (RI) for attributes. The willingness to pay (WTP) and maximum acceptable risk (MAR) were also estimated. The RI, WTP, and MAR of oncologists, patients, and family members were compared.Results: A total of 121 oncologists and 161 dyads of patients and family members completed the survey. Overall survival time, physical functioning status, and pain were the 3 most important attributes across all 3 groups. Oncologists and family members had higher RI on overall survival time than patients (48% and 51% vs 38%). Patients had higher RI on physical functioning status and pain (23% and 14%) than oncologists (13% and 12%) and family members (16% and 11%). For extending survival, patients had the least WTP, and family members had the highest MAR. The latent class analysis identified 2 classes in the patient group and 3 classes in oncologist and family member groups.Conclusions: There were differences in preferences for survival, risk, quality of life, and costs associated with cancer treatments among patients, oncologists, and family members. This finding highlights the need of involving patients in treatment decision making in China. [ABSTRACT FROM AUTHOR]

Objectives: Many trials conclude "no clinically meaningful detriment" to health-related quality of life (HRQL) or function between arms, even when notable differential toxicity is observed. Mean change from baseline analyses of function or HRQL can possibly obscure important change in subgroups experiencing symptomatic toxicity. We evaluate the impact of diarrhea, a key treatment arm toxicity, on patient-reported HRQL and functioning in clinical trials submitted to US Food and Drug Administration.Methods: This study used 4 randomized, breast cancer trials (adjuvant to late-line metastatic) as case examples. Diarrhea, physical functioning (PF), and global health status and quality of life (GHS/QoL) from the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-C30 were analyzed at baseline and approximately 3 and 6 months.Results: Generally, patients reporting very much diarrhea at months 3 and 6 had worse PF (9-19 points lower) and GHS/QoL (16-19 points lower) than patients reporting no diarrhea regardless of treatment arm. In the change from baseline analysis, patients reporting very much diarrhea also experienced a greater decrease in PF (6-13 points) and GHS/QoL (6-16 points) versus patients reporting no diarrhea in both arms.Conclusions: In trials with moderate to large differences in symptomatic toxicity by arm, reporting "no meaningful difference in functioning and HRQL between arms" based on mean change from baseline analysis is insufficient and may obscure important impacts on subgroups experiencing symptomatic adverse events. Additional exploratory analyses with simple data visualizations evaluating functioning or HRQL in patient subgroups experiencing expected symptomatic toxicities can further inform the safety and tolerability of an investigational agent. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to determine whether responses to Patient-Reported Outcomes Measurement Information System Short Form v2.0 - Physical Function 8c (PROMIS PF8c) items differed when the use of a 7-day recall period was compared with no specified recall period.Methods: Using a within-subject design, we surveyed 1810 individuals from the US general population, administering PROMIS PF8c at survey beginning and end. The order of measure presentation was randomly assigned. We calculated recall difference scores (RDSs) as no recall score minus 7-day recall score using both item response theory-based T scores and raw summed scores. We examined the distribution and created Bland-Altman plots for both RDSTscore and RDSRaw. We also calculated correlations between no recall versus 7-day recall T score and raw scores. Finally, we determined whether differences in no recall versus 7-day recall scores were associated with patient-reported PF.Results: RDSTscore and RDSRaw had means (root mean square differences) of 0.00 (5.43) and -0.04 (3.79), respectively. The vast majority (%) of RDSTscore and RDSRaw values fell between the Bland-Altman limits of agreement (-10.65 to 10.66 and -7.46 to 7.38, respectively). Pearson's correlations between no recall and 7-day recall for T scores and raw scores were 0.88 and 0.87, respectively. Effect sizes for mean RDSTscore and RDSRaw compared across level of Eastern Oncology Cooperative Group performance status, patient global impression of PF severity, and single PF items were near 0.Conclusions: We did not find any significant recall period effect on PF8c responses. Therefore, we recommend the use of the PROMIS physical function standard, with no specified recall time period. [ABSTRACT FROM AUTHOR]

Objectives: Assessment of patient-reported outcomes (PROs) in oncology is of critical importance because it provides unique information that may also predict clinical outcomes.Methods: We conducted a systematic review of prognostic factor studies to examine the prognostic value of PROs for survival in cancer. A systematic literature search was performed in PubMed for studies published between 2013 and 2018. We considered any study, regardless of the research design, that included at least 1 PRO domain in the final multivariable prognostic model. The protocol (EPIPHANY) was published and registered in the International Prospective Register of Systematic Reviews (CRD42018099160).Results: Eligibility criteria selected 138 studies including 158 127 patients, of which 43 studies were randomized, controlled trials. Overall, 120 (87%) studies reported at least 1 PRO to be statistically significantly prognostic for overall survival. Lung (n = 41, 29.7%) and genitourinary (n = 27, 19.6%) cancers were most commonly investigated. The prognostic value of PROs was investigated in secondary data analyses in 101 (73.2%) studies. The EORTC QLQ-C30 questionnaire was the most frequently used measure, and its physical functioning scale (range 0-100) the most frequent independent prognostic PRO, with a pooled hazard ratio estimate of 0.88 per 10-point increase (95% CI 0.84-0.92).Conclusions: There is convincing evidence that PROs provide independent prognostic information for overall survival across cancer populations and disease stages. Further research is needed to translate current evidence-based data into prognostic tools to aid in clinical decision making. [ABSTRACT FROM AUTHOR]

XLH is a rare, life-long, often debilitating genetic disorder which causes rickets, lower extremity deformity, diminished growth, leading to physical function limitations, pain and fatigue. This study sought to evaluate the item/scale properties, reliability, validity, sensitivity to change of three PROMIS® scales measuring Physical Function Mobility (Phy), Pain Interference (Pain), Fatigue (Ftg) in a small cohort paediatric XLH patients. [Extracted from the article]