Considerable research has demonstrated the negative psychosocial impact of cancer. Recent work has highlighted positive psychosocial outcomes. Research is now needed to evaluate the relationship between negative and positive impacts. This paper reports the development and validation of a measurement model capturing positive and negative psychosocial illness impacts.
The sample included 754 cancer patients on- or post-treatment. Item development was informed by literature review, expert input patient interviews and the results of a pilot study of 205 cancer patients, resulting in 43 positive and 46 negative items. Factor analyses were used to evaluate the dimensionality of the item pools. Analysis of variance (ANOVA) was used to examine relationships between psychosocial illness impact and other variables.
Unidimensionality was demonstrated within but not across negative and positive impact items. ANOVA results showed differential relationships between negative and positive impacts, respectively, and patient sociodemographic and clinical variables.
Positive and negative psychosocial illness impacts are best conceptualized and measured as two independent factors. Computerized adaptive tests and short-form measures developed from this comprehensive psychosocial illness impact item bank may benefit future research and clinical applications.
Psychosocial sequelae; Cancer; Cancer survivors; Bi-factor analysis
Cancers of the head and neck are associated with detriments in health-related quality of life (HRQOL), however little is known about different experiences between African Americans and non-Hispanic whites.
HRQOL was measured by the Functional Assessment of Cancer Therapy – Head and Neck approximately five months post diagnosis among 222 cancer patients from North Carolina. Higher scores represent better HRQOL. Regression models included sociodemographic characteristics and clinical factors.
African Americans reported higher Physical Well-Being than Caucasians (adjusted means 23.1 vs 20.9). African Americans with incomes <$20,000 reported higher Emotional Well-Being (21.4) and fewer head and neck symptoms (22.0). Non-Hispanic whites making <$20,000 reported the poorest Emotional Well-Being (17.3) while African Americans making >$20,000 reported the most head and neck symptoms (18.7).
Further investigation is needed to explore variation in HRQOL experiences among different race and socio-economic groups that may inform resource allocation to improve cancer care.
health-related quality of life; head and neck cancer; African Americans
Purpose of review
In this review, we briefly summarize three fruitful, emerging areas in liver transplantation research: quality of life; risk assessment; and patient safety. Our goal is to highlight recent findings in these areas, with a call for increased integration of social scientists and transplant clinicians to address how best to shape policy and improve outcomes.
After liver transplantation, recipients generally experience clinically significant, sustained improvement in their physical, social and emotional well-being. However, a sizeable minority of patients do experience excess morbidity that may benefit from ongoing surveillance and/or intervention. There is growing body of research that describes risks associated with liver transplantation, which can be useful aids to better inform decision making by patients, clinicians, payers, and policy makers. In contrast, there has been a relative lack of empirical data on transplant patient safety vulnerabilities, placing the field of surgery in stark contrast to other high risk industries, where such assessments inform continuous process improvement.
Health services and outcomes research has grown in importance in the liver transplantation literature, but several important questions remain unanswered that merit programmatic, interdisciplinary research.
liver transplantation; recipients; quality of life; risk assessment; patient safety; outcomes
Women diagnosed with ovarian cancer are at risk for reduced quality of life (QOL). It is imperative to further define these declines to interpret treatment outcomes and design appropriate clinical interventions.
The primary objective of this study was to compare data obtained from ovarian cancer patients with normative data to assess the degree to which QOL differs from the norm. Secondary objectives were to examine demographic variables and determine if there was a correlation between physical/functional and social/emotional scores during chemotherapy.
Patients with Stage III/IV ovarian cancer on Gynecologic Oncology Group Protocols 152 and 172 who underwent surgery followed by intravenous paclitaxel and cisplatin completed the Functional Assessment of Cancer Therapy-Ovarian. The Functional Assessment of Cancer Therapy scale includes the four domains of physical, functional, social, and emotional well-being (PWB, FWB, SWB, and EWB, respectively).
Ovarian cancer patients had a total QOL (Functional Assessment of Cancer Therapy-General) score similar to the U.S. female adult population. However, the reported subscale scores were 2.0 points (95% confidence interval [CI] 1.4–2.5, P < 0.001, effect size = 0.37) lower in PWB, 0.9 points (95% CI 0.3–1.5, P = 0.005, effect size = 0.13) lower in FWB, 5.0 points (95% CI 4.6–5.3, P < 0.001, effect size = 0.74) higher in SWB, and 0.8 points (95% CI 0.3–1.2, P < 0.001, effect size = 0.16) lower in EWB. Correlation between the sum of PWB and FWB and the sum of SWB and EWB was r = 0.53 (P < 0.001). Age was positively correlated with EWB (r = 0.193; 95% CI 0.09–0.29).
Ovarian cancer patients have decreased QOL in physical, functional, and emotional domains; however, they may compensate with increased social support. At the time of diagnosis and treatment, patients’ QOL is affected by inherent characteristics. Assessment of treatment outcomes should take into account the effect of these independent variables.
Ovarian cancer; chemotherapy; quality of life
A comprehensive, reliable, and valid measurement system is needed to monitor changes in children with neurological conditions who experience lifelong functional limitations.
This article describes the development and psychometric properties of the pediatric version of the Quality of Life in Neurological Disorders (Neuro-QOL) measurement system.
The pediatric Neuro-QOL consists of generic and targeted measures. Literature review, focus groups, individual interviews, cognitive interviews of children and consensus meetings were used to identify and finalize relevant domains and item content. Testing was conducted on 1018 children aged 10 to 17 years drawn from the US general population for generic measures and 171 similarly aged children with muscular dystrophy or epilepsy for targeted measures. Dimensionality was evaluated using factor analytic methods. For unidimensional domains, item parameters were estimated using item response theory models. Measures with acceptable fit indices were calibrated as item banks; those without acceptable fit indices were treated as summary scales.
Ten measures were developed: 8 generic or targeted banks (anxiety, depression, anger, interaction with peers, fatigue, pain, applied cognition, and stigma) and 2 generic scales (upper and lower extremity function). The banks reliably (r > 0.90) measured 63.2% to 100% of the children tested.
The pediatric Neuro-QOL is a comprehensive measurement system with acceptable psychometric properties that could be used in computerized adaptive testing. The next step is to validate these measures in various clinical populations.
health-related quality of life; children; neurological disorders; item bank; item response theory
The Patient-Reported Outcomes Measurement Information System (PROMIS) is a National Institutes of Health initiative to develop item banks measuring patient-reported outcomes (PROs) and to create and make available a computerized adaptive testing system (CAT) that allows for efficient and precise assessment of PROs in clinical research and practice.
Aims of the Study
Based on the presentation from a symposium on “Evidence-based Outcomes in Psychiatry: Updates on Measurement Using Patient-Reported Outcomes (PRO)” at the 2011 American Psychiatry Association Convention, this paper provides an overview of PROMIS and its application to mental health research.
The PROMIS methodology for item bank development and testing is described, with a focus on the implications of this work for mental health research.
Utilizing qualitative item review and state-of-the-art applications of item response theory (IRT), PROMIS investigators have developed, tested, and released item banks measuring physical, mental, and social health components. Ongoing efforts continue to add new item banks and further validate existing banks.
PROMIS provides item banks measuring several domains of interest to mental health researchers including emotional distress, social function, and sleep. PROMIS methodology also provides a rigorous standard for the development of new mental health measures.
Implications for Health Care Provision
Web-based CAT or administration of short forms derived from PROMIS item banks provide efficient and precise dimensional estimates of clinical outcomes that can be utilized to monitor patient progress and assess quality improvement.
Implications for Future Research
Use of the dimensional PROMIS metrics (and co-calibration of the PROMIS item banks with existing PROs) will allow comparisons of mental health and related health outcomes across disorders and studies.
To illustrate how measurement practices can be advanced using as an example the fatigue item bank (FIB) and its applications (short-forms and computerized adaptive test) that were developed via the NIH Patient Reported Outcomes Measurement Information System (PROMIS) Cooperative Group.
Psychometric analysis of data collected by an internet survey company using Item Response Theory (IRT) related techniques.
A United States general population representative sample collected via internet.
803 respondents used for dimensionality evaluation of the PROMIS FIB and 14,931 respondents used for item calibrations
Main Outcome Measures
112 fatigue items developed by the PROMIS fatigue domain working group, 13-item Functional Assessment of Chronic Illness Therapy-Fatigue, and 4-item SF-36 Vitality scale.
The PROMIS FIB version 1 which consists of 95 items demonstrated acceptable psychometric properties. Computerized Adaptive Testing (CAT) showed consistently better precision than short-forms. However, all three short-forms showed good precision for the majority of participants, in that more than 95% of sample could be precisely measured with a reliability greater than 0.9.
Measurement practice can be advanced by using a psychometrically sound measurement tool and its applications. This example shows that CAT and short-forms derived from the PROMIS FIB can reliably estimate fatigue reported by the US general population. Evaluation in clinical populations is warranted before the item bank can be used for clinical trials.
PROMIS; fatigue; CAT; short-form
The Patient-Reported Outcomes Measurement Information System (PROMIS) was developed as one of the first projects funded by the NIH Roadmap for Medical Research Initiative to re-engineer the clinical research enterprise. The primary goal of PROMIS is to build item banks and short forms that measure key health outcome domains that are manifested in a variety of chronic diseases which could be used as a “common currency” across research projects. To date, item banks, short forms and computerized adaptive tests (CAT) have been developed for 13 domains with relevance to pediatric and adult subjects. To enable easy delivery of these new instruments, PROMIS built a web-based resource (Assessment Center) for administering CATs and other self-report data, tracking item and instrument development, monitoring accrual, managing data, and storing statistical analysis results. Assessment Center can also be used to deliver custom researcher developed content, and has numerous features that support both simple and complicated accrual designs (branching, multiple arms, multiple time points, etc.). This paper provides an overview of the development of the PROMIS item banks and details Assessment Center functionality.
The purpose of this study was to examine health-related quality of life (HRQL) among women with metastatic breast cancer treated on E2100 with paclitaxel or paclitaxel plus bevacizumab. Trial participants (N = 670) completed the Functional Assessment of Cancer Therapy-Breast (FACT-B) pre-treatment and following 4 and 8 cycles of treatment to assess HRQL and breast cancer-specific concerns. A significantly higher proportion of missing FACT-B assessments was observed among patients receiving paclitaxel only, due to faster time to death. To account for this non-ignorable pattern of missing data, we conducted a survival-adjusted HRQL analysis by jointly modeling the longitudinal HRQL outcome and time to non-ignorable dropout using a two-stage model. FACT scores assessing HRQL did not differ following 4 and 8 cycles of treatment; however mean scores on the 9-item Breast Cancer Scale were significantly higher after 4 and 8 cycles of treatment among patients receiving paclitaxel plus bevacizumab. No differences were observed between treatment arms on FACT-B total scores. The addition of bevacizumab was not associated with additional side effect burden from the patient perspective and was associated with a greater reduction in breast cancer-specific concerns. No other differences were noted.
Health-related quality of life; Metastatic breast cancer; Survival-adjusted quality of life; Patient-reported outcomes
To describe the lessons learned in the initial development of PROMIS social function item banks.
Development and testing of two item pools within a general population to create item banks that measure ability-to-participate and satisfaction-with-participation in social activities.
Administration via the Internet.
General population members (N=956) of a national polling organization registry participated; data for 768 and 778 participants used in the analysis.
Main Outcome Measures
Measures of ability-to-participate and satisfaction-with-participation in social activities.
Fifty six items measuring the ability-to-participate were essentially unidimensional but did not fit an IRT model. As a result, item banks were not developed for these items. Of the 56 items measuring satisfaction-with-participation, 14 items measuring social roles and 12 items measuring discretionary activities were unidimensional and met IRT model assumptions. Two 7-item short forms were also developed.
Four lessons, mostly concerning item content, were learned in the development of banks measuring social function. These lessons led to item revisions that are being tested in subsequent studies.
Quality of life; Rehabilitation
Content validity of patient-reported outcomes (PROs) is evaluated primarily during item development, but subsequent psychometric analyses, particularly for item-response theory (IRT)-derived scales, often result in considerable item pruning and potential loss of content. After selecting items for the PROMIS banks based on psychometric and content considerations, we invited external content expert reviews of the degree to which the initial domain names and definitions represented the calibrated item bank content.
A minimum of four content experts reviewed each item bank and recommended a domain name and definition based on item content. Domain names and definitions then were revealed to the experts who rated how well these names and definitions fit the bank content and provided recommendations for definition revisions.
These reviews indicated that the PROMIS domain names and definitions remained generally representative of bank content following item pruning, but modifications to two domain names and minor to moderate revisions of all domain definitions were needed to optimize fit with the item bank content.
This reevaluation of domain names and definitions following psychometric item pruning, although not previously documented in the literature, appears to be an important procedure for refining conceptual frameworks and further supporting content validity.
content validity; conceptual framework; domain definition; item-response theory
Pain is prevalent among patients with cancer, yet pain management patterns in outpatient oncology are poorly understood.
Patients and Methods
A total of 3,123 ambulatory patients with invasive cancer of the breast, prostate, colon/rectum, or lung were enrolled onto this prospective study regardless of phase of care or stage of disease. At initial assessment and 4 to 5 weeks later, patients completed a 25-item measure of pain, functional interference, and other symptoms. Providers recorded analgesic prescribing. The pain management index was calculated to assess treatment adequacy.
Of the 3,023 patients we identified to be at risk for pain, 2,026 (67%) reported having pain or requiring analgesics at initial assessment; of these 2,026 patients, 670 (33%) were receiving inadequate analgesic prescribing. We found no difference in treatment adequacy between the initial and follow-up visits. Multivariable analysis revealed that the odds of a non-Hispanic white patient having inadequate pain treatment were approximately half those of a minority patient after adjusting for other explanatory variables (odds ratio, 0.51; 95% CI, 0.37 to 0.70; P = .002). Other significant predictors of inadequate pain treatment were having a good performance status, being treated at a minority treatment site, and having nonadvanced disease without concurrent treatment.
Most outpatients with common solid tumors must confront issues related to pain and the use of analgesics. There is significant disparity in pain treatment adequacy, with the odds of undertreatment twice as high for minority patients. These findings persist over 1 month of follow-up, highlighting the complexity of these problems.
Multiple myeloma (MM) is a common hematologic malignancy and is associated with symptom burden and impairments in health-related quality of life (HRQL).
To develop a disease-specific, patient-reported outcomes (PRO) measure for the assessment of HRQL among patients with MM as part of the Functional Assessment of Cancer Therapy (FACT) measurement system.
HRQL concerns and symptoms associated with MM were tabulated based on a literature review, and 52 candidate PRO items were identified. Expert clinicians (n=13) rated 52 items on relevance to HRQL for MM patients (0-3 scale). Experts added 11 items for comprehensive PRO assessment in MM. A list of 63 candidate items was rated (0-3 scale) by 13 MM patients enrolled through the International Myeloma Foundation website. Qualitative data and quantitative item ratings were reviewed to select FACT-MM scale items.
Expert clinicians provided the highest HRQL relevance ratings for bone pain, bodily pain, difficulty walking (2.9), tiring easily (2.6), feeling discouraged (2.5), interference with activities and difficulty with self-care as a result of bone pain (2.5), and fatigue (2.5). Mean age of patients was 57 years; Eastern Cooperative Oncology Group performance status was 0 (38%), 1 (31%) or 2 (31%). Quantitative ratings by patients identified sexual function (1.3), uncertainty about health (1.2), fatigue (1.0), weight gain (1.0), and emotional concerns such as worry about new symptoms and difficulty planning for the future (1.0) as most relevant to HRQL.
The 14-item FACT-MM PRO measure was developed based on expert clinician and patient data, ensuring relevance to HRQL for MM patients.
Multiple myeloma; health-related quality of life; patient-reported outcomes; patient self-report
In order to fully capture the impact of a disease or condition on the lives of individuals, patient-reported outcomes (PROs) are considered a necessary component of health measurement in rehabilitation. This article provides an overview of the involvement of rehabilitation stakeholders in the development of sound measurement tools for the Patient-Reported Outcomes Measurement Information System (PROMIS), a National Institutes of Health-funded initiative. PROMIS is a multi-site study that included many different populations. Here we focus on the involvement of people with several chronic conditions, including multiple sclerosis, spinal cord injury, and arthritis in the development of PROMIS measures. We describe both qualitative and quantitative methods used, including expert panels, focus groups, cognitive interviews and item response theory modeling, which resulted in enhanced utility of PROMIS measures in rehabilitation. The measures include a set of global health items and twelve item banks representing six domains. Scores are reported in the T-score metric (Mean = 50, SD=10) and centered on means from the U.S. general population. The PROMIS item banks measure quality of life and symptoms of people with chronic conditions and have the potential to enhance research and clinical practice by facilitating comparisons of scores across domains and populations.
Outcome Assessment (Health Care); rehabilitation; Disabled Persons
The NIH Patient-Reported Outcomes Measurement Information System (PROMIS) Roadmap initiative is a cooperative group program of research designed to develop, evaluate, and standardize item banks to measure patient-reported outcomes relevant across medical conditions. For adults, 11 domains have been developed in physical, mental, and social health.
The objective of the current study was to assess feasibility and construct validity of PROMIS item banks versus legacy measures in a observational study in systemic sclerosis (SSc).
Patients with SSc in a single academic center completed computerized adaptive technology (CAT) administered PROMIS item banks during the clinic visit and legacy domains (using paper-and-pencil). The construct validity of PROMIS items was evaluated by examining correlations with corresponding legacy measures using multitrait-multimethod analysis.
Participants consisted of 143 SSc patients with an average age of 51.5 years; 71% were female and 68% were Caucasian. The average number of items completed for each CAT-administered item bank ranged from 5 to 8 (69 CAT items per patient), and the average time to complete each CAT-administered item bank ranged from 48 seconds to 1.9 minutes per patient (average time= 11.9 minutes/per patient for 11 banks). All correlations between PROMIS domains and respective legacy measures were large and in the hypothesized direction (ranged from .61 to .82).
Our study supports the construct validity of the CAT-administered PROMIS item banks and shows that they can be administered successfully in a clinic with support staff. Future studies should assess the feasibility of PROMIS item banks in a busy clinical practice
Systemic sclerosis; PROMIS; health-related quality of life; construct validity
The field of solid organ transplantation has historically concentrated research efforts on basic science and translational studies. However, there has been increasing interest in health services and outcomes research. The aim was to build an effective and sustainable, inter- and transdisciplinary health services and outcomes research team (NUTORC), that leveraged institutional strengths in social science, engineering, and management disciplines, coupled with an international recognized transplant program. In 2008, leading methodological experts across the university were identified and intramural funding was obtained for the NUTORC initiative. Inter- and transdisciplinary collaborative teams were created across departments and schools within the university. Within 3 years, NUTORC became fiscally sustainable, yielding more than tenfold return of the initial investment. Academic productivity included funding for 39 grants, publication of 60 manuscripts, and 166 national presentations. Sustainable educational opportunities for students were created. Inter- and transdisciplinary health services and outcomes research in transplant can be innovative and sustainable.
Transdisciplinary research teams; Health Services and Outcomes Research; Educational opportunities; Academic productivity; Sustainable research efforts
Many patient-reported outcome (PRO) instruments used in systemic sclerosis (SSc) trials are limited by lack of validation, licensing fees, and complicated scoring systems. We assessed the construct validity for discriminative purposes of two new PRO instruments-the Patient Reported Outcomes Measurement Information System 29-item Health Profile (PROMIS-29) and the Functional Assessment of Chronic Illness Therapy-Dyspnea short form (FACIT-Dyspnea), measuring health status and dyspnea in SSc patients.
Seventy-three patients participated in a cross-sectional study at a tertiary SSc program. PROMIS-29, FACIT-Dyspnea, and legacy PRO instruments used in clinical trials (Medical Research Council [MRC] Dyspnea Score, St. George’s Respiratory Questionnaire [SGRQ], Health Assessment Questionnaire-Disability Index [HAQ-DI] and Short-Form 36 [SF-36]) were administered. Composite severity scores using an adaptation of the Medsger Disease Severity Index were generated using clinical, diagnostic and laboratory information. PROMIS-29 and FACIT-Dyspnea scores were compared with legacy PRO measures and composite severity scores.
The mean (range) patient age (84% women) was 51 years (22–72). The mean (range) SSc disease duration from the onset of the first non-Raynaud symptom was 7 years (0–45). Spearman correlation coefficients across FACIT-Dyspnea and PROMIS Physical Functioning scores with legacy PRO instruments were generally high (range=0.64–0.86); those between PROMIS and FACIT-Dyspnea with composite disease severity scores were more modest, but statistically significant (range=0.33–0.48, p<0.01).
PROMIS-29 and FACIT-Dyspnea are valid instruments to measure the health status of SSc patients. PROMIS-29 and FACIT-Dyspnea may be preferable to legacy instruments because they are freely available in multiple languages, and simple to administer, score and interpret.
The purpose of this trial was to evaluate the role of radiation therapy with concurrent gemcitabine (GEM) compared with GEM alone in patients with localized unresectable pancreatic cancer.
Patients and Methods
Patients with localized unresectable adenocarcinoma of the pancreas were randomly assigned to receive GEM alone (at 1,000 mg/m2/wk for weeks 1 to 6, followed by 1 week rest, then for 3 of 4 weeks) or GEM (600 mg/m2/wk for weeks 1 to 5, then 4 weeks later 1,000 mg/m2 for 3 of 4 weeks) plus radiotherapy (starting on day 1, 1.8 Gy/Fx for total of 50.4 Gy). Measurement of quality of life using the Functional Assessment of Cancer Therapy–Hepatobiliary questionnaire was also performed.
Of 74 patients entered on trial and randomly assigned to receive GEM alone (arm A; n = 37) or GEM plus radiation (arm B; n = 34), patients in arm B had greater incidence of grades 4 and 5 toxicities (41% v 9%), but grades 3 and 4 toxicities combined were similar (77% in A v 79% in B). No statistical differences were seen in quality of life measurements at 6, 15 to 16, and 36 weeks. The primary end point was survival, which was 9.2 months (95% CI, 7.9 to 11.4 months) and 11.1 months (95% CI, 7.6 to 15.5 months) for arms A and B, respectively (one-sided P = .017 by stratified log-rank test).
This trial demonstrates improved overall survival with the addition of radiation therapy to GEM in patients with localized unresectable pancreatic cancer, with acceptable toxicity.
The National Institute of Neurological Disorders and Stroke (NINDS) commissioned the Neurology Quality of Life (Neuro-QOL) project to develop a bilingual (English/Spanish), clinically relevant and psychometrically robust HRQL assessment tool. This paper describes the development and calibration of these banks and scales.
Classical and modern test construction methodologies were used, including input from essential stakeholder groups.
An online patient panel testing service and eleven academic medical centers and clinics from across the United States and Puerto Rico that treat major neurological disorders.
Adult and pediatric patients representing different neurological disorders specified in this study, proxy respondents for select conditions (stroke and pediatric conditions), and English and Spanish speaking participants from the general population.
Main Outcome Measures
Multiple generic and condition specific measures used to provide construct validity evidence to new Neuro-QOL tool.
Neuro-QOL has developed 14 generic item banks and 8 targeted scales to assess HRQL in five adult (stroke, multiple sclerosis, Parkinson’s disease, epilepsy, and amyotrophic lateral sclerosis) and two pediatric conditions (epilepsy and muscular dystrophies).
The Neuro-QOL system will continue to evolve, with validation efforts in clinical populations, and new bank development in health domains not currently included. The potential for Neuro-QOL measures in rehabilitation research and clinical settings is discussed.
Neurology; Clinical Research; Health-Related Quality of Life; Quality of Life; Patient Reported Outcomes
In 2002, the NIH launched the ‘Roadmap for Medical Research’. The Patient-Reported Outcomes Measurement Information System (PROMIS®) is one of the Roadmap’s key aspects. To create the next generation of patient-reported outcome measures, PROMIS utilizes item response theory (IRT) and computerized adaptive testing. In 2009, the NIH funded the second wave of PROMIS studies (PROMIS II). PROMIS II studies continue PROMIS’s agenda, but also include new features, including longitudinal analyses and more sociodemographically diverse samples. PROMIS II also includes increased emphasis on pediatric populations and evaluation of PROMIS item banks for clinical research and population science. These aspects bring new psychometric challenges. To address this, investigators associated with PROMIS gathered at the Third Psychometric Summit in September 2010 to identify, describe and discuss pressing psychometric issues and new developments in the field, as well as make analytic recommendations for PROMIS. The summit addressed five general themes: linking, differential item functioning, dimensionality, IRT models for longitudinal applications and new IRT software. In this article, we review the discussions and presentations that occurred at the Third PROMIS Psychometric Summit.
computerized adaptive testing; dimensionality; factor analysis; item response theory; patient-reported outcomes; PROMIS; psychometrics; structural equation modeling
To use item response theory (IRT) methods to link physical functioning items in the Activity Measure for Post-acute Care (AM-PAC) and the Quality of Life Outcomes in Neurological Disorders (Neuro-QOL)
Secondary data analysis of the physical functioning items of AM-PAC and Neuro-QOL. We used a non-equivalent group design with 36 core items common to both instruments. We used a test characteristic curve transformation method to for linking AM-PAC and Neuro-QOL scores. Linking was conducted so that both raw scores and scaled AM-PAC and Neuro-QOL scores (converted-logit scores with mean = 50 and SD = 10) could be compared.
AM-PAC items were administered to rehabilitation patients in post-acute care settings. Neuro-QOL items were administered to a community sample of adults via the Internet.
The AM-PAC sample consisted of 1,041 post acute care patients; the Neuro-QOL sample was 549 community-dwelling adults.
Main Outcome Measures
25 Mobility items and 11 ADL items common to both instruments were included in the analysis.
Neuro-QOL items were linked to the AM-PAC scale using the Generalized Partial Credit Model. Mobility and ADL subscale scores from the two instruments were calibrated to the AM-PAC metric.
An IRT-based linking method placed AM-PAC and NeuroQOL Mobility and ADL scores on a common metric. This linking allowed estimation of AM-PAC Mobility and ADL subscale scores based on Neuro-QOL Mobility and ADL subscale scores, and vice versa. The accuracy of these results should be validated in a future sample in which participants respond to both instruments.
outcome assessment (Health Care); linking; neurological diseases; rehabilitation
Objective The purpose of this study is to report the reliability, validity, and clinical utility of a parent-report perceived cognitive function (pedsPCF) item bank. Methods From the U.S. general population, 1,409 parents of children aged 7–17 years completed 45 pedsPCF items. Their psychometric properties were evaluated using Item Response Theory (IRT) approaches. Receiver operating characteristic (ROC) curves and discriminant function analysis were used to predict clinical problems on child behavior checklist (CBCL) scales. A computerized adaptive testing (CAT) simulation was used to evaluate clinical utility. Results The final 43-item pedsPCF item bank demonstrates no item bias, has acceptable IRT parameters, and provides good prediction of related clinical problems. CAT simulation resulted in correlations of 0.98 between CAT and the full-length pedsPCF. Conclusions The pedsPCF has sound psychometric properties, U.S. general population norms, and a brief-yet-precise CAT version is available. Future work will evaluate pedsPCF in other clinical populations in which cognitive function is important.
assessment; cancer and oncology; cognitive assessment; computer applications/eHealth; neuropsychology; quality of life
We combined anchor- and distribution-based methods to establish minimally important differences (MIDs) for six PROMIS-Cancer scales in advanced-stage cancer patients.
Study Design and Setting
Participants completed six PROMIS-Cancer scales and 23 anchor measures at an initial (n=101) and follow-up (n=88) assessment 6 to 12 weeks later. Three a priori criteria were used to identify useable cross-sectional and longitudinal anchor-based MID estimates. The mean standard error of measurement was also computed for each scale. The focus of the analysis was on IRT-based MIDs estimated on a T-score scale. Raw score MIDs were estimated for comparison purposes.
Many cross-sectional (64%) and longitudinal (73%) T-score anchor-based MID estimates were excluded because they did not meet a priori criteria. The following are recommended T-score MID ranges: 17-item Fatigue (2.5–4.5), 7-item Fatigue (3.0–5.0), 10-item Pain Interference (4.0–6.0), 10-item Physical Functioning (4.0–6.0), 9-item Emotional Distress-Anxiety (3.0–4.5), and 10-item Emotional Distress-Depression (3.0–4.5). Effect sizes corresponding to these MIDs averaged between 0.40 and 0.63.
This study is the first to address MIDs for PROMIS measures. Studies are currently being conducted to confirm these MIDs in other patient populations and to determine whether these MIDs vary by patients’ level of functioning.
A phase III, randomized, double-blind, placebo-controlled trial was conducted in patients with metastatic renal cell carcinoma. There was no evidence of a difference between everolimus and placebo in longitudinal patterns of disease-related symptoms, and little difference between the arms in physical functioning or global quality of life trends. This supports the conclusion that delay in tumor progression demonstrated by everolimus is associated with minimal impact on symptoms, physical functioning, or quality of life, as reported by patients.
A phase III, randomized, double-blind, placebo-controlled trial was conducted in patients with metastatic renal cell carcinoma. The focus of this paper is to evaluate the patient-reported outcomes.
Patients were randomly assigned (2:1) to receive oral everolimus 10 mg once daily or placebo. The Functional Assessment of Cancer Therapy Kidney Symptom Index—Disease-Related Symptoms (FKSI-DRS) and European Organization for the Research and Treatment of Cancer (EORTC) QLQ-C30 were administered before randomization and on day 1 of each cycle. The FKSI-DRS and the EORTC QLQ-C30 Physical Functioning and Global Quality of Life scores were the primary endpoints examined. Longitudinal models were used to compare treatment arms. Sensitivity analyses were conducted to explore the impact of missing data assumptions.
Longitudinal trends for FKSI-DRS scores did not differ by treatment arm. Taking nonignorable missing data into account, there were significant differences between treatment arms in the trend over time for physical functioning and global quality of life, with the everolimus arm exhibiting greater decreases. All three of these measures of health-related quality of life were significantly related to progression-free survival.
There was no evidence of a difference between everolimus and placebo in longitudinal patterns of disease-related symptoms, and little difference between the arms in physical functioning or global quality of life trends. This supports the conclusion that delay in tumor progression demonstrated by everolimus is associated with minimal impact on symptoms, physical functioning, or quality of life, as reported by patients.
Everolimus; Renal cell carcinoma; Quality of life; Metastatic
Human Papillomavirus (HPV) infection is the most common sexually transmitted infection in the United States, and has major physical and psychological consequences including fear, anxiety, and stigma. To date, there is no measure of health-related quality of life specifically designed to assess symptoms and functioning for people with HPV infection. In the present study, we set out to develop an HPV-specific measure of health-related quality of life. We conducted literature reviews, open-ended interviews with patients, clinican surveys, and cognitive interviews which guided item development. The result is a 36-item measure, the Functional Assessment of Chronic Illness Therapy – HPV, which assesses the physical and psychological health-related quality of life aspects of HPV infection.