Early initiation of riluzole may improve absolute survival in amyotrophic lateral sclerosis.

INTRODUCTION/AIMS: Riluzole improves survival in amyotrophic lateral sclerosis (ALS), but optimal time and duration of treatment are unknown. The aim of this study was to examine if timing of riluzole initiation and duration of treatment modified its effect on survival. METHODS: Patients from the PRO-ACT dataset with information on ALS Functional Rating Scale, time from onset to enrollment (TFOE), and riluzole use were selected for analysis. Survival from enrollment was the outcome. Multivariable Cox proportional hazard models were examined for interactions between riluzole and TFOE. Inverse probability of treatment weighting (IPTW) was used to assess average treatment effect. RESULTS: Of 4778 patients, 3446 (72.1%) had received riluzole. In unadjusted analyses, riluzole improved median survival significantly (22.6 vs. 20.2 months, log-rank p < 0.001). In multivariable analyses, no significant interaction between TFOE and riluzole was found. Riluzole effect was uniform during follow-up. By IPTW, estimated riluzole hazard ratio was 0.798 (95% confidence interval 0.686-0.927). Delaying riluzole initiation by 1 y (6 to 18 months from onset) may translate to reducing median survival from onset by 1.9 months (40.1 to 38.2 months). DISCUSSION: Riluzole appears to reduce risk of death uniformly, regardless of time from onset to treatment, and duration of treatment. Earlier treatment with riluzole may be associated with greater absolute survival gain from onset. Early diagnosis of ALS will facilitate early treatment and is expected to improve survival.

Mapping PROMIS physical function and pain interference to the modified low back pain disability questionnaire.

PURPOSE: The Modified Low Back Pain Disability Questionnaire (MDQ) is a commonly used tool to assess functioning of patients with low back pain (LBP). Recently, the Patient-Reported Outcomes Measurement Information System (PROMIS) was suggested as an alternative platform to assess LBP patient-reported health. We sought to map between the MDQ and PROMIS Physical Function (PROMIS-PF) and Pain Interference (PROMIS-PI) scales using multiple methods. METHODS: In a retrospective analysis of LBP patients seen at Cleveland Clinic 11/14/18-12/11/19, T-scores from each PROMIS scale were mapped to MDQ total score individually and together. MDQ item and total scores were mapped to each PROMIS scale. Linear regression as well as linear and equipercentile equating were used. Split sample internal validation using root mean squared error (RMSE), mean absolute error (MAE), and correlations were used to assess accuracy of mapping equations. RESULTS: 13585 patients completed the three scales. In the derivation cohort, average age was 59.0 (SD = 15.8); 53.3% female and 82.9% white. Average MDQ total, PROMIS-PF, and PROMIS-PI T-scores were 40.3 (SD = 19.0), 37.2 (SD = 7.6), and 62.9 (SD = 7.2), respectively. For estimating MDQ total scores, methods that used both PROMIS-PF and PROMIS-PI had closest estimated means, lowest RMSE and MAE, and highest correlations. For estimating each of PROMIS-PF and PROMIS-PI T-scores, the best performing method was equipercentile equating using the MDQ items. CONCLUSIONS: We created and internally validated maps between MDQ and PROMIS-PF and PROMIS-PI using linear regression, linear and equipercentile equating. Our equations can be used by researchers wishing to translate scores between these scales.

Evidence of Stability in Patient-Reported Global Health During the COVID-19 Pandemic.

OBJECTIVES: Measures of health-related quality of life (HRQOL) are collected throughout healthcare systems and used in clinical, economic, and outcomes studies to direct patient-centered care and inform health policy. Studies have demonstrated increases in stressors unique to the COVID-19 pandemic, however, their effect on HRQOL is unknown. Our study aimed to assess the change in self-reported global health during the pandemic for patients receiving care in a large healthcare system compared with 1 year earlier. METHODS: An observational cross-sectional study of 2 periods was conducted including adult patients who had a healthcare appointment and completed the Patient-Reported Outcomes Measurement Information System Global Health (PROMIS GH) as standard care during the COVID-19 pandemic and a year earlier. The effect of time on PROMIS global mental health (GMH) and global physical health (GPH) was evaluated through multiple statistical methods. RESULTS: There were 38 037 patients (mean age 56.1 ± 16.6 years; 61% female; 87% white) who completed the PROMIS GH during the pandemic (August 2020) and 33 080 (age 56.7 ± 16.5 years; 61% female; 86% white) who had completed it 1 year earlier (August 2019). GMH was significantly worse, whereas GPH was similar during the pandemic compared with a year earlier (adjusted estimate [standard error]: -1.21 (0.08) and 0.11 (0.08) T-score points, respectively). CONCLUSIONS: Our study found modest, nonclinically meaningful decreases in GMH and similar GPH during the COVID-19 pandemic compared with a year earlier in patients cared for in a large healthcare system. Nevertheless, healthcare systems are likely seeing a biased sample of patients during these times. Findings from our study have implications for the interpretation of HRQOL during this pandemic.

The validity of proxy responses on patient-reported outcome measures: Are proxies a reliable alternative to stroke patients' self-report?

PURPOSE: Caregivers, or proxies, often complete patient-reported outcome measures (PROMs) on behalf of patients with stroke. The objective of our study was to assess the validity and responsiveness of proxy-responses compared to patient-responses across multiple domains of health. METHODS: Stroke patients and their proxies were recruited to complete PROMs between 7/2018-11/2019. PROMs included Neuro-QoL cognitive function, PROMIS physical function, satisfaction with social roles, anxiety, fatigue, pain interference, sleep disturbance, Global Health, and PHQ-9. Internal consistency and convergent validity were compared between patient- and proxy-reported measures. Known-groups validity was assessed across levels of stroke disability. Internal responsiveness was evaluated using paired t-tests for a subset of patients who attended rehabilitation following stroke. Analyses were stratified by patients ≤ 3 vs > 3 months from stroke. RESULTS: This cross-sectional study included 200 stroke patients (age 62.2 ± 13.3, 41.5% female) and their proxies (age 56.5 ± 13.9, 70% female, 72% spouses). PROMs had high internal consistency and were significantly correlated for patients and proxies. Patient- and proxy-reported measures worsened with increasing stroke disability. For 34 (17%) patients who attended rehabilitation, patients self-reported improvement on 5 domains whereas proxies reported no improvement. Compared to patient self-reports, validity was worse for proxy-reports on patients ≤ 3 months but better > 3 months from stroke. CONCLUSIONS: Both patient- and proxy-reported PROMs demonstrated strong validity. Only patient-reported PROMs were responsive to change, and proxies had worse validity for patients ≤ 3 months from stroke but better validity for patients > 3 months from stroke. These findings justify the utilization of proxy responses in stroke patients > 3 months from stroke.

Patient-reported experience with patient-reported outcome measures in adult patients seen in rheumatology clinics.

OBJECTIVES: Patient-reported outcome measures (PROMs) are increasingly utilized in the evaluation of patients with rheumatic diseases. The aim of our study was to assess the patient experience with completing PROMs within rheumatology clinics, and identify patient characteristics associated with a more positive experience. METHODS: We conducted a retrospective cross-sectional study of adult patients seen in rheumatology clinics between 1/1/2017 and 6/30/2017. Patients were included in the study if they completed at least one patient-reported experience question following completion of PROMs. Patient characteristics associated with more positive experiences were identified through multivariable proportional odds models. RESULTS: 12,597 adult patients (mean age 59 ± 15; 76% female; 84% white) completed PROMs, as well as questions on their experience completing PROMs. Patients agreed/strongly agreed that PROM questions were easy to understand (97%), useful (84%), helped their physician understand their health (78%), improved communication with their provider (78%) and improved control over their own care (70%). Predictors of better experience with PROMs included being younger, non-white, having lower income, and being a new patient. Worse self-reported health also predicted better experience with PROMs. CONCLUSION: Our study found a positive patient experience with PROMs, which is a crucial component of their successful implementation and utilization. Findings from this study suggest PROMs may be particularly beneficial in new patients, minorities, those with lower income, and worse self-reported quality of life. Collecting PROMs could provide opportunities to improve patient-provider communication and enhance control over care for rheumatology patients who could most benefit.

Magnitude and Variability of Stroke Patient-Proxy Disagreement Across Multiple Health Domains.

OBJECTIVES: To quantify the extent and variability of bias introduced when caregivers, or proxies, complete patient-reported outcome measures (PROM) on behalf of stroke patients. DESIGN: Cross-sectional survey study conducted between July 2018 and November 2019. SETTING: Ambulatory clinic of a cerebrovascular center or rehabilitation unit. PARTICIPANTS: A consecutive sample of stroke patients (N=200) and their proxies who were able and willing to complete PROMs. Proxies completed PROMs as they believed the patient would answer. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: PROMs included Neuro-QoL cognitive function, PROMIS physical function, social role satisfaction, anxiety, fatigue, pain interference, sleep disturbance, Patient Health Questionnaire-9 translated to PROMIS Depression, and PROMIS Global Health. RESULTS: The study included 200 stroke patients (age, 62.2±13.3; 41.5% women) and their proxies (age 56.5±13.9; 70% women, 72% spouses). Proxies reported worse functioning and more symptoms across all PROM domains compared with patients (average difference, 0.3-3.0 T score points). Reliability between dyad responses was moderate across all domains (intraclass correlation coefficients (2,1), 0.49-0.76) and effect sizes were small (d=0.04-0.35). Cognitive function, anxiety, and depression had the lowest agreement, whereas physical function, pain, and sleep had the highest agreement based on the Bland-Altman method. At the individual level, a large proportion of dyads had meaningfully different scores across domains (range, 40%-57%; dyads differed >5 T score points). Few predictors of disagreement were identified through multinomial regression models. CONCLUSIONS: At the aggregate level, small differences were detected between stroke patient-proxy pairs, with lower agreement on more subjective domains. At the individual level, a large proportion of dyads reported meaningfully different scores on all domains, affecting the interpretability of proxy responses on PROMs in a clinical setting.

A Cost-Effectiveness Framework for Amyotrophic Lateral Sclerosis, Applied to Riluzole.

OBJECTIVES: Reexamine cost-effectiveness of riluzole in the treatment of amyotrophic lateral sclerosis (ALS) in light of recent advances in disease staging and understanding of stage-specific drug effect. METHODS: ALS was staged according to the "fine'til 9" (FT9) staging method. Stage-specific health utilities (EQ-5D, US valuation) were estimated from an institutional cohort, whereas literature informed costs and transition probabilities. Costs at 2018 prices were disaggregated into recurring costs (RCs) and "one-off" transition/"tollgate" costs (TCs). Five- and 10-year horizons starting in stage 1 disease were examined from healthcare sector and societal perspectives using Markov models to evaluate riluzole use, at a threshold of $100 000/quality-adjusted life year (QALY). Probabilistic and deterministic sensitivity analyses were conducted. RESULTS: Mean EQ-5D utilities for stages 0 to 4 were 0.79, 0.74, 0.63, 0.54, and 0.46, respectively. From the healthcare sector perspective at the 5-year horizon, riluzole use contributed to 0.182 QALY gained at the cost difference of $12 348 ($5403 riluzole cost, $8870 RC and -$1925 TC differences), translating to an incremental cost-effectiveness ratio (ICER) of $67 658/QALY. Transition probability variation contributed considerably to ICER uncertainty (-30.2% to +90.0%). ICER was sensitive to drug price and RCs, whereas higher TCs modestly reduced ICER due to delayed tollgates. CONCLUSION: This study provides a framework for health economic studies of ALS treatments using FT9 staging. Prospective stage-specific and disaggregated cost measurement is warranted for accurate future cost-effectiveness analyses. Appropriate separation of TCs from RCs substantially mitigates the high burden of background cost of care on the ICER.

PROMIS Global Health item nonresponse: is it better to impute missing item responses before computing T-scores?

PURPOSE: Item response theory (IRT) scoring provides T-scores for physical and mental health subscales on the Patient-Reported Outcomes Measurement Information System Global Health questionnaire (PROMIS-GH) even when relevant items are skipped. We compared different item- and score-level imputation methods for estimating T-scores to the current scoring method. METHODS: Missing PROMIS-GH items were simulated using a dataset of complete PROMIS-GH scales collected at a single tertiary care center. Four methods were used to estimate T-scores with missing item scores: (1) IRT-based scoring of available items (IRTavail), (2) item-level imputation using predictive mean matching (PMM), (3) item-level imputation using proportional odds logistic regression (POLR), and (4) T-score-level imputation (IMPdirect). Performance was assessed using root mean squared error (RMSE) and mean absolute error (MAE) of T-scores and comparing estimated regression coefficients from the four methods to the complete data model. Different proportions of missingness and sample sizes were examined. RESULTS: IRTavail had lowest RMSE and MAE for mental health T-scores while PMM had lowest RMSE and MAE for physical health T-scores. For both physical and mental health T-scores, regression coefficients estimated from imputation methods were closer to those of the complete data model. CONCLUSIONS: The available item scoring method produced more accurate PROMIS-GH mental but less accurate physical T-scores, compared to imputation methods. Using item-level imputation strategies may result in regression coefficient estimates closer to those of the complete data model when nonresponse rate is high. The choice of method may depend on the application, sample size, and amount of missingness.

Association Between Patient Experience With Patient-Reported Outcome Measurements and Overall Satisfaction With Care in Neurology.

BACKGROUND: There has been increasing focus on both patient-reported outcome measurement (PROM) collection and patient satisfaction ratings; nevertheless, little is known about their relationship. OBJECTIVES: To determine the association between patient experience with PROM collection and visit satisfaction and to identify characteristics of better ratings for each. METHODS: This cross-sectional observational study included all patients seen in 15 neurological clinics who completed PROMs as well as 6 questions on the patient experience with PROMs at least once from October 1, 2015 to December 31, 2016. Visit satisfaction was evaluated using a composite measure of physician communication, overall physician rating, and the likelihood of recommending that physician as indicated on the Clinician and Group Consumer Assessment of Healthcare Providers and Systems survey. Predictors of PROM experience and satisfaction were identified using proportional odds and logistic regression models, respectively. RESULTS: There were 6454 patients (average age 58 ± 15 years, 59% women) who completed PROMs and responded to the Clinician and Group Consumer Assessment of Healthcare Providers and Systems survey. There were significant positive associations between each PROM experience question and visit satisfaction (r = 0.11-0.19; P<.010), although factors predicting visit satisfaction differed from those predicting PROM experience. A differential effect of PROMs on visit satisfaction was identified for patients who were nonwhite, had lower income, and had more comorbidities. CONCLUSIONS: Although there was a significant association between better PROM experience and higher visit satisfaction, relationships with clinical characteristics differed, providing insights into how PROMs may be associated with patients' visit satisfaction. Further research is necessary to confirm whether PROMs can be used to improve visit satisfaction, particularly in patients who historically have reported lower quality of care.

Patient versus proxy response on global health scales: no meaningful DIFference.

PURPOSE: Assessment of outcomes from a proxy is often substituted for the patient's self-report when the patient is unable or unwilling to report their status. Research has indicated that proxies over-report symptoms on the patient's behalf. This study aimed to quantify the extent of proxy-introduced bias on the Patient-Reported Outcomes Measurement Information System Global Health (PROMIS GH) scale for mental (GMH) and physical (GPH) scores. METHODS: This retrospective cohort study included incident stroke patients seen in a cerebrovascular clinic who completed PROMIS GH between 10/12/15 and 6/6/18. Differential item functioning (DIF) evaluated measurement invariance of patient versus proxy responses. DIF impact was assessed by comparing the initial score to the DIF-adjusted score. Subgroup analyses evaluated DIF within strata of stroke severity, measured by modified Rankin Scale (≤ 1, 2, 3+), and time since stroke (≤ 30, 31-90, > 90 days). RESULTS: Of 1351 stroke patients (age 60.5 ± 14.9, 45.1% female), proxy help completing PROMIS GH was required by 406 patients (30.1%). Proxies indicated significantly worse response to all items. No items for GMH or GPH were identified as having meaningful DIF. In subgroup analyses, no DIF was found by severity or 31-90 days post-stroke. In patients within 30 and > 90 days of stroke, DIF was detected for 2 items. Accounting for DIF had negligible effects on scores. CONCLUSIONS: Our findings revealed the overestimation of symptoms by proxies is a real difference and not the result of measurement non-invariance. PROMIS GH items do not perform differently or have spuriously inflated severity estimates when administered to proxies instead of patients.

Trajectories of impairment in amyotrophic lateral sclerosis: Insights from the Pooled Resource Open-Access ALS Clinical Trials cohort.

INTRODUCTION: Rate of decline of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score is a common outcome measure and a powerful predictor of mortality in ALS. METHODS: Observed rate of decline (postslope) of ALSFRS-R, its linearity, and its relationship to decline at first visit (preslope) were examined in the Pooled Resource Open-Access ALS Clinical Trials cohort by using longitudinal mixed effects models. RESULTS: Mean ALSFRS-R postslope in 3,367 patients was -0.99 points/month. Preslope and postslope were correlated and had powerful effects on survival. ALSFRS-R trajectories were slightly accelerated overall, but slope and direction/degree of curvature varied. Subscore decline was sequential by site of onset. Respiratory subscore decline was the least steep. DISCUSSION: Variable curvilinearity of ALSFRS-R trajectories confounds interpretation in clinical studies that assume linear decline. Subscore trajectories recapitulate phenotypic diversity and topographical progression of ALS. ALSFRS-R is better used as a multidimensional measure. Muscle Nerve 57: 937-945, 2018.

Accuracy of Linking VR-12 and PROMIS Global Health Scores in Clinical Practice.

OBJECTIVES: To examine the accuracy of general health cross-walk tables in a clinical sample of patients with spine disorders. Published tables (Schalet BD, Rothrock NE, Hays RD, et al. Linking physical and mental health summary scores from the Veterans RAND 12-Item Health Survey (VR-12) to the PROMIS(®) Global Health Scale. J Gen Intern Med 2015;30:1524-30) link scores from the Veterans RAND 12-Item Health Survey (VR-12) to the 10-Item Patient-Reported Outcome Measurement Information System (PROMIS), a global health scale metric for both mental (GMH) and physical (GPH) summary scores. METHODS: We assessed the accuracy of administered PROMIS and VR-12 scores with scores predicted by cross-walks in 4606 adult patients seen in a spine clinic from October 2015 to 2016. Accuracy of linking scores was evaluated using Pearson correlation, intraclass correlation coefficients, and mean and SD of score differences. Bland-Altman plots were used to graphically assess the levels of agreement. The consistency in scores' discrimination across levels of pain severity, depression, and other patient characteristics was assessed. Bootstrap methods estimated linking precision across varying sample sizes. RESULTS: Actual and cross-walked PROMIS scores showed moderate correlation (ICC(3,1): GMH 0.73; GPH 0.81), with Bland-Altman plots suggesting smaller differences between scores in patients with lower and higher general health. Significant discrimination between patient subgroups was demonstrated reliably by both actual and estimated scores. Bootstrapped resamples indicated adequate precision for 200 patients (95% confidence interval for mean difference: GMH -1.38 to 0.60; GPH 0.39 to 1.93). CONCLUSIONS: VR-12 and PROMIS global health scores can be accurately linked within a sample of patients with spine disorders; nevertheless, bias is high and precision is low for linking on the patient level. Linked scores at the group level for more than 200 patients can be used in comparative effectiveness research and for comparing results across studies.

Screening Colonoscopy Withdrawal Time Threshold for Adequate Proximal Serrated Polyp Detection Rate.

INTRODUCTION: For adequate adenoma detection rate (ADR), guidelines recommend a mean withdrawal time (MWT) of ≥ 6 min. ADR has been shown to correlate strongly with proximal serrated polyp detection rate (PSP-DR), which is another suggested quality measure for screening colonoscopy. However, the impact of directly measured withdrawal time on PSP-DR has not been rigorously studied. We examined the relationship between MWT to ADR and PSP-DR, with the aim of identifying a functional threshold withdrawal time associated with both increased ADR and PSP-DR. METHODS: This was a retrospective study of endoscopy and pathology data from average-risk screening colonoscopy examinations performed at a large system with six endoscopy laboratories. A natural language processing tool was used to determine polyp location and histology. ADR and PSP-DR were calculated for each endoscopist. MWT was calculated from colonoscopy examinations in which no polyps were resected. RESULTS: In total, 31,558 colonoscopy examinations were performed, of which 10,196 were average-risk screening colonoscopy examinations with cecal intubation and adequate prep by 24 gastroenterologists. When assessing the statistical significance of increasing MWT by minute, the first significant time mark for PSP-DR was at 11 min at a rate of 14.2% (p = 0.01). There was a significant difference comparing aggregated MWT < 11 min compared to ≥ 11 min looking at the rates of adenomas [OR 1.65 (1.09-2.51)] and proximal serrated polyps [OR 1.81 (1.06-3.08)]. While ADR linearly correlated well with MWT (R = 0.76, p < 0.001), the linear relationship with PSP-DR was less robust (R = 0.42, p = 0.043). CONCLUSION: In this large cohort of average-risk screening colonoscopy, a MWT of 11 min resulted in a statistically significant increase in both ADR and PSP-DR. Our data suggest that a longer withdrawal time may be required to meet both quality metrics.

Life after prostate cancer treatment: a mixed methods study of the experiences of men with sexual dysfunction and their partners.

BACKGROUND: Prostate cancer is the most common non-skin cancer in men and sexual dysfunction is the most frequently reported long-term side effect of prostate cancer surgery or radiation. The aim of this study was to examine the experiences of men with sexual dysfunction and their partners following prostate cancer treatment. METHODS: Men with sexual dysfunction from either surgical removal or radiation therapy 1-5 years after treatment were interviewed, as well as their partners. A mixed method design was used to determine the lived experience of men with sexual dysfunction. Open-ended questions guided the interviews. RESULTS: Twenty seven men completed the study with a mean age of 61 years (SD = 8.0; range = 44-77 years). Nine partners also participated. The majority of men (92.6%) had surgery. The average time from treatment to the interview was 23.5 months (SD = 11.7). Themes were frustration with sexual dysfunction, importance of support and understanding from others, depression and anxiety related to sexual dysfunction, importance of intimacy with partner, factors that impact treatment satisfaction, and education and comprehensive information about sex. CONCLUSIONS: Prostate cancer survivors and partners need accurate information about sexual side effects before during and after treatment. Men and partners required individualized help and guidance to manage sexual dysfunction. Support and understanding from partners, family, and others was also identified as an important aspect of healing and adjustment after prostate cancer treatment. Prostate cancer education/support groups played a key role in helping men and partners gain advocacy, education, and support. Psychological problems such as depression and anxiety need to be identified and addressed in men after prostate cancer treatment. Men and partners need assistance in understanding and navigating their way through intimacy to move forward with connectedness in their relationship. Satisfaction with treatment and with providers is dependent on patient education and understanding of all aspects of prostate cancer treatment including sexual side effects and incontinence.

Trends and variation in the use of nipple-sparing mastectomy for breast cancer in the United States.

PURPOSE: For many women, nipple-sparing mastectomy (NSM) provides aesthetic and quality-of-life outcomes superior to skin-sparing mastectomy. Accumulating data suggest that NSM provides similar oncologic outcomes in select breast cancer patients. This study sought to determine national trends in NSM use. METHODS: Using the National Cancer Data Base, 6254 women with breast cancer who underwent NSM between 2010 and 2013 were identified. NSM rates were determined relative to the number of patients who received a mastectomy with reconstruction (n = 114,849). Associations between patient, tumor, and facility characteristics and NSM were assessed using logistic regression. RESULTS: The rate of NSM increased from 2.9 to 8.0 % between 2010 and 2013. NSM was most commonly performed in academic (adjusted odds ratio [OR] 1.43, p < 0.001) and high-volume (OR 1.59, p < 0.001) breast centers. There was up to a 5.8-fold variation in its delivery between geographic census regions (p < 0.001). Of 1231 hospitals, only 491 (39.9 %) reported performing at least one NSM during the study period. Half of all NSMs were performed by the top 6 % (n = 30) of NSM-performing centers. NSM was associated with small tumor size (p < 0.001), lower tumor grades (p < 0.05), and negative nodal status (p < 0.001). However, half of NSM patients had at least one tumor characteristic that diverged from current (2016) NCCN recommendations for the procedure. CONCLUSIONS: The use of therapeutic NSM is increasing dramatically in the United States, despite recommendations that the procedure be used with caution. As NSM becomes increasingly common, efforts are needed to monitor its long-term oncologic outcomes and to ensure equitable access to it.

Utilities Estimated from PROMIS Scales for Cost-Effectiveness Analyses in Stroke.

BACKGROUND: The EQ-5D and Health Utilities Index Mark 3 (HUI-3) are preference-based measures used in cost-effectiveness studies. The Patient Reported Outcomes Measurement Information System (PROMIS) Preference scoring system (PROPr) is a new preference-based measure. In addition, algorithms were previously developed to map PROMIS Global Health (PROMIS-GH) items to HUI-3 using linear equating (HUILE) and 3-level EQ-5D using linear (EQ5DLE). We sought to evaluate and compare estimated utilities based on PROPr and PROMIS-GH in adult stroke survivors. METHODS: We performed a retrospective cohort study of adults diagnosed with 1 of ischemic stroke, intracerebral hemorrhage, or subarachnoid hemorrhage seen in an outpatient clinic between 2015 and 2019. Patients completed PROMIS scales and other measures. We computed a modified version of PROPr (mPROPr) and compared the distributional characteristics and correlations with stroke outcomes for mPROPr, HUILE, and EQ5DLE. RESULTS: T toal of 4,159 stroke survivors (average age 62.7 ± 14.7 y, 48.4% female, 77.6% ischemic stroke) were included. Mean utility estimates for mPROPr, EQ5DLE, and HUILE were 0.333 ± 0.244, 0.739 ± 0.201, and 0.544 ± 0.301, respectively. Correlations between the modified Rankin Scale and each of mPROPr and HUILE were both -0.48 and -0.43 for EQ5DLE. Regression analyses indicated that mPROPr scores may be too low for stroke patients in good health and that EQ5DLE scores may be too high for stroke patients in poor health. CONCLUSIONS: All 3 PROMIS-based utilities were associated with measures of stroke disability and severity, but the distributions of utilities were very different. Our study highlights the problem cost-effectiveness researchers face of valuing health states with certainty. For researchers using utilities estimated from PROMIS scales, our study indicates that mapping PROMIS-GH item scores to HUI-3 via linear equating may be most appropriate in stroke patients. HIGHLIGHTS: A new preference-based measure has been developed from the Patient Reported Outcomes Measurement Information System (PROMIS), known as the PROMIS-Preference (PROPr) scoring system, and published equations mapping PROMIS Global Health (PROMIS-GH) items to the Health Utilities Index Mark 3 (HUI-3) and EQ-5D-3L are available for use in cost-effectiveness studies.Our study provides distributional characteristics and comparisons of utilities estimated using a modified version of PROPr and equations mapping PROMIS-GH items to EQ-5D-3L and HUI-3 in a sample of stroke survivors.The results of our study show large differences in the distributions of utilities estimated using the different health state measures, and these differences highlight the ongoing difficulty researchers face in valuing health states with certainty.

Patient-proxy agreement on change in acute stroke patient-reported outcome measures: a prospective study.

OBJECTIVES: Research has indicated proxies overestimate symptoms on patients' behalves, however it is unclear whether patients and proxies agree on meaningful change across domains over time. The objective of this study is to assess patient-proxy agreement over time, as well as agreement on identification of meaningful change, across 10 health domains in patients who underwent acute rehabilitation following stroke. METHODS: Stroke patients were recruited from an ambulatory clinic or inpatient rehabilitation unit, and were included in the study if they were undergoing rehabilitation. At baseline and again after 30 days, patients and their proxies completed PROMIS Global Health and eight domain-specific PROMIS short forms. Reliability of patient-proxy assessments at baseline, follow-up, and the change in T-score was evaluated for each domain using intra-class correlation coefficients (ICC(2,1)). Agreement on meaningful improvement or worsening, defined as 5+ T-score points, was compared using percent exact agreement. RESULTS: Forty-one patient-proxy dyads were included in the study. Proxies generally reported worse symptoms and functioning compared to patients at both baseline and follow-up, and reported less change than patients. ICCs for baseline and change were primarily poor to moderate (range: 0.06 (for depression change) to 0.67 (for physical function baseline)), and were better at follow-up (range: 0.42 (for anxiety) to 0.84 (for physical function)). Percent exact agreement between indicating meaningful improvement versus no improvement ranged from 58.5-75.6%. Only a small proportion indicated meaningful worsening. CONCLUSIONS: Patient-proxy agreement across 10 domains of health was better following completion of rehabilitation compared to baseline or change. Overall change was minimal but the majority of patient-proxy dyads agreed on meaningful change. Our study provides important insight for clinicians and researchers when interpreting change scores over time for questionnaires completed by both patients and proxies.

Group-level analyses involving scores linked from legacy scales to PROMIS scales: A novel alternative using imputation.

As the implementation of Patient-Reported Outcomes Measurement Information System (PROMIS) scales has increased, so has the number of studies linking legacy scale scores to PROMIS scale scores. Variability in linked scores for a given PROMIS score can be considerable, leading to potential bias. An alternative method is imputation using a bridge study. We sought to compare linking to this alternative novel method in group-level analyses using linked legacy scores. Adult patients who completed PROMIS Depression and Patient Health Questionnaire (PHQ-9) were included. We randomly allocated data samples to be missing either PROMIS Depression or PHQ-9. We estimated PROMIS T scores using six methods: Linking methods by Choi et al. (2014), linking in our internal data, imputation using bridge study data from external data and from our internal data, each with and without patient demographics. Estimated mean PROMIS T scores using the linking and imputation methods were compared to actual PROMIS T scores across varying proportions of missingness and sample size. We also compared regression coefficients for the six estimation methods to a model using actual PROMIS T scores. Mean estimated versus actual PROMIS T scores varied between 1 and 4 points for the linking methods and within 0.4 points for the imputation method using internal data with patient demographics. The imputation methods had estimated regression coefficients closer to that of the model using actual scores as compared to the linking methods. For group-level analyses, imputation using a bridge study may be a feasible alternative to using linked scores or can be used as a sensitivity analysis. (PsycInfo Database Record (c) 2021 APA, all rights reserved).

Progressive arm muscle weakness in ALS follows the same sequence regardless of onset site: use of TOMS, a novel analytic method to track limb strength.

Objective: Examine sequence of weakness in arm muscles from longitudinal hand-held dynamometry (HHD) data in ALS for congruence with contiguous spread of neurodegeneration along spinal cord segments. Methods: Longitudinal HHD data from the Ceftriaxone clinical trial were examined using nonlinear mixed models, assuming a logistic trajectory from normal to zero strength. Unobserved baseline normal strength of weak muscles was assumed using strength of the best-preserved muscle. A novel metric called "time from onset to midway strength" (TOMS) was estimated for each muscle group, and TOMS ratios were examined to identify sequence of weakness, overall and by onset site. Results: Shoulder flexion (SF), elbow flexion (EF), elbow extension (EE), wrist extension (WE), and first dorsal interosseous (FDI) were measured on each side. Over a median of 36 weeks, 513 subjects provided 2589 sets of HHD measures. TOMS increased sequentially in the following order: FDI, WE, SF, EF, and EE. TOMS ratios estimates with 95% CIs (adjusted for multiple comparisons) were: WE/FDI 1.32 (1.24-1.41), SF/WE 1.06 (1.01-1.10), EF/SF 1.06 (1.02-1.10), and EE/EF 1.18 (1.12-1.23). Elbow and shoulder flexors weakened sooner than did elbow extensors. The sequence of arm muscle weakness progression was similar regardless of onset site. Conclusion: Nonsegmental progression of arm muscle weakness that is similar for different onset sites favors cortical influence/network spread over contiguous spread of neurodegeneration in the spinal cord. Furthermore, this study confirms the "split elbow" pattern. TOMS and other proposed methods may have value as outcome measures in clinical research.

Considerations for Reporting and Reviewing Studies Including Health-Related Quality of Life.

Self-reported measures of health-related quality of life (HRQOL) are increasingly used in clinical management and evaluation of patient outcomes. HRQOL measures are used to monitor patient progress and treatment response, investigate effects of medical interventions, and provide patient-based data for quality improvement initiatives and policy decisions. Given the importance of HRQOL, it is imperative that the instruments used to assess HRQOL are precise, valid, reliable, and responsive, and that the HRQOL data are appropriately collected, analyzed, and presented. This article reviews the key attributes of studies involving HRQOL data, discusses best practices for selecting appropriate instruments, and provides guidelines for the assessment, analysis, and presentation of these data. A checklist and a reviewer guide are included to serve as templates for authors and reviewers when submitting and reviewing studies involving HRQOL.