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
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.
Hematopoietic cell transplantation (HCT) is an intensive treatment for hematologic malignancies that has the potential to cure disease or prolong life, but also to impair quality of life for survivors. Earlier studies have suggested a variety of factors to be associated with physical and mental health after HCT. In this study we evaluated demographic and clinical factors before and after transplant and selected psychosocial factors after transplant, to explore their association with self-reported physical and mental health. We studied a cohort of 662 survivors at a median of 6.6 years after HCT. Pre-HCT demographic and clinical factors accounted for only a small amount of the variance in physical and mental health post-transplant (3% and 1%, respectively). Adding post-HCT clinical variables to the pre-transplant factors accounted for 32% and 7% of physical and mental outcomes, respectively. When both clinical and psychosocial factors were considered, better physical health after HCT was associated with younger age, race other than white, higher current family income, currently working or being a student, less severe transplant experience (not having GVHD), fewer current comorbidities, higher Karnofsky status, less social constraint, less social support, and less trait anxiety. This multivariate model accounted for 36% of the variance in physical health with the psychosocial variables contributing very little. When both clinical and psychosocial factors were considered, better mental health after HCT was associated with more severe transplant experience, less social constraint, greater spiritual well-being, and less trait anxiety. This multivariate model accounted for 56% of the variance in mental health, with the psychosocial factors accounting for most of the variance. These data suggest that clinical factors are explanatory for much of the post-HCT physical health reported by HCT survivors but for very little of self-perceived mental health. These observations provide insights into identification of factors that would allow recognition of at-risk patients as well as factors amenable to intervention.
To evaluate the Patient-Reported Outcomes Measurement Information System (PROMIS), which collected data from an Internet polling panel, and to compare PROMIS with national norms.
Study Design and Settings
We compared demographics and self-rated health of the PROMIS general Internet sample (N=11,796) and a sub-sample of it (n = 2,196) selected to approximate the joint distribution of demographics from the 2000 U.S. Census, with three national surveys and U.S. Census data. The comparisons were conducted using equivalence testing with weights created for PROMIS by raking.
The weighted PROMIS population and sub-sample had similar demographics compared to the 2000 U.S. Census except that the sub-sample had a higher percentage of people with more education than high school. Equivalence testing shows similarity between PROMIS general population and national norms with regard to body mass index, EQ-5D health index, and self-rating of general health.
Self-rated health of the PROMIS general population is similar to that of existing samples from the general U.S. population. The weighted PROMIS general population is more comparable to national norms than the unweighted population with regard to subject characteristics. The findings suggest that representativeness of the Internet data is comparable to those from probability-based general population samples.
PROMIS; patient-reported outcomes; re-sampling; weighting; raking; equivalence testing
The Patient-Reported Outcomes Measurement Information System (PROMIS) allows assessment of the impact of chronic conditions on health-related quality of life (HRQL) across diseases. We report on the HRQL impact of individual and comorbid conditions as well as conditions that are described as limiting activity.
Study Design and Setting
Data were collected through online and clinic recruitment as part of the PROMIS item calibration sample (n=21,133). Participants reported the presence or absence of 24 chronic health conditions and whether or not their activity was limited by each condition.
Across health status domains, the presence of a chronic condition was associated with poorer scores than those without a diagnosis, particularly for those individuals who reported their condition was disabling. The magnitude of detriment in HRQL was more pronounced for individuals with two or more chronic conditions and could not be explained by sociodemographic factors. Patterns of HRQL deficits varied across disease and comorbidity status.
The impact of chronic conditions, particularly when experienced with comorbid disease, is associated with detriments in HRQL. The negative impact on HRQL varies across symptoms and functional areas within a given condition.
Chronic disease; Comorbidity; Quality of life; Outcome measures
Patient-reported outcomes (PROs) are essential when evaluating many new treatments in health care, yet current measures have been limited by a lack of precision, standardization and comparability of scores across studies and diseases. The Patient-Reported Outcomes Measurement Information System (PROMIS™) provides item banks that offer the potential for PRO measurement that is efficient (minimizes item number without compromising reliability) flexible (enables optional use of interchangeable items), and precise (has minimal error in estimate) measurement of commonly-studied PROs. We report results from the first large-scale testing of PROMIS items.
Study Design and Setting
Fourteen item pools were tested in the U.S. general population and clinical groups using an online panel and clinic recruitment. A scale-setting sub-sample was created reflecting demographics proportional to the 2000 U.S. census.
Using item response theory (graded response model), 11 item banks were calibrated on a sample of 21,133, measuring components of self-reported physical, mental and social health, along with a 10-item global health scale. Short forms from each bank were developed and compared to the overall bank as well as with other well-validated and widely accepted (“legacy”) measures. All item banks demonstrated good reliability across the majority of the score distributions. Construct validity was supported by moderate to strong correlations with legacy measures.
PROMIS item banks and their short forms provide evidence they are reliable and precise measures of generic symptoms and functional reports comparable to legacy instruments. Further testing will continue to validate and test PROMIS items and banks in diverse clinical populations.
Outcome Measures; Quality of life; Chronic disease
The authors report on the development and calibration of item banks for depression, anxiety, and anger as part of the Patient-Reported Outcomes Measurement Information System (PROMIS®). Comprehensive literature searches yielded an initial bank of 1,404 items from 305 instruments. After qualitative item analysis (including focus groups and cognitive interviewing), 168 items (56 for each construct) were written in a first person, past tense format with a 7-day time frame and five response options reflecting frequency. The calibration sample included nearly 15,000 respondents. Final banks of 28, 29, and 29 items were calibrated for depression, anxiety, and anger, respectively, using item response theory. Test information curves showed that the PROMIS item banks provided more information than conventional measures in a range of severity from approximately −1 to +3 standard deviations (with higher scores indicating greater distress). Short forms consisting of seven to eight items provided information comparable to legacy measures containing more items.
depression; anxiety; anger; item response theory; measurement
To determine whether a telephone counseling program can improve psychosocial outcomes among breast cancer patients post-treatment.
A randomized trial was conducted involving 21 hospitals and medical centers, with assessments (self-administered questionnaires) at baseline, 12 and 18 months post-enrollment. Eligibility criteria included early stage diagnosis, enrollment during last treatment visit, and the ability to receive the intervention in English. Endpoints included distress (Impact of Event Scale), depression (Center for Epidemiologic Studies Depression Scale) and two study-specific measures: sexual dysfunction and personal growth. The control group (n = 152) received a resource directory for breast cancer; the intervention group (n = 152) also received a one year, 16 session telephone counseling program augmented with additional print materials.
Significant intervention effects were found for sexual dysfunction at 12 (p = 0.03) and 18 months (p = 0.04) and personal growth (12 months: p = 0.005; 18 months: p = 0.03). No differences by group were found in mean scores for distress and depression, with both groups showing significant improvement at 12 and 18 months (all p values for within-group change from baseline were ≤ .003). However, when dichotomized at cutpoints suggestive of the need for a clinical referral, the control group showed virtually no change at 18 months while the intervention group showed about a 50% reduction for both distress (p = 0.07) and depression (p = 0.06).
Telephone counseling may provide a viable method for extending psychosocial services to cancer survivors nationwide.
psychosocial oncology; psychosocial telephone counseling; breast cancer survivors
Dyspnea is a common symptom among patients with heart failure. Currently, there is no standardized, rapid, precise method to assess dyspnea.
Methods and Results
From a review of the literature, we pooled questions from various questionnaires assessing dyspnea. 201 patients with heart failure completed all questions in the preliminary item bank. Each item asks how much shortness of breath the patient had when doing an activity. Medical charts were reviewed for hospitalization within 1 or 3 months of completing the questions. We created a dyspnea item bank of 44 items. Computer Adaptive Tests (CAT) generated from this item bank can assess dyspnea by administering on average 10 questions. Simulation CAT scores were generated to compare with the item bank scores. The CAT scores had a correlation of 0.98 with item bank scores. Logistic regression models predicting the probability of being hospitalized from the dyspnea score were statistically significant (p<0.05). A 5-point score increase was associated with a 32% increased odds of hospitalization in1 month and a 20% increased odds of hospitalization in 3 months.
This computer based tool for dyspnea assessment obtains similar precision to that of answering the entire dyspnea item bank with less patient burden.
There has been some suggestion that the fatigue experienced by older cancer patients is more severe than that of younger cohorts; however, there is little empirical evidence to support this claim.
The goal of the present study was to determine the differential impact of age and cancer diagnosis on ratings of fatigue using a validated self-report instrument.
The Functional Assessment of Chronic Illness Therapy-Fatigue subscale (FACIT-F) consists of 13 items measuring fatigue experience and its impact on daily life, with scores ranging from 0 (severe fatigue) to 52 (no fatigue). Fatigue data were available from the U.S. general population (n = 1075; 51.3% female, 45.9 ± 16.5 yrs) and a sample of mixed-diagnosis cancer patients (n = 738; 64.3% female, 58.7 ± 13.6 yrs). General population participants were recruited using an internet-based survey panel; patients with cancer were recruited from Chicago-area oncology clinics.
On average, the cancer patient group reported more severe fatigue than the general population group (36.9 vs. 46.6; F[1,1797] = 271.95, P < 0.001). There was evidence for increased fatigue with age (F(6,719) = 2.56, p < 0.02) among patients with cancer, but not in the general population (P = 0.06). Furthermore, the group × age interaction was not significant (P = 0.44). Hemoglobin (Hgb) was treated as a covariate for 430 patients with available data; there was no main effect for age in this analysis.
Older adults, whether or not they had a cancer diagnosis, reported more fatigue than younger adults. These differences may be explained, in part, by Hgb level. Future research would be helpful to explore longitudinal changes in fatigue in the general population and to guide fatigue management for the older cancer patient.
Fatigue; cancer; assessment; patient-reported outcome; hemoglobin
To develop a social health measurement framework, to test items in diverse populations and to develop item response theory (IRT) item banks.
A literature review guided framework development of Social Function and Social Relationships sub-domains. Items were revised based on patient feedback, and Social Function items were field-tested. Analyses included exploratory factor analysis (EFA), confirmatory factor analysis (CFA), two-parameter IRT modeling and evaluation of differential item functioning (DIF).
The analytic sample included 956 general population respondents who answered 56 Ability to Participate and 56 Satisfaction with Participation items. EFA and CFA identified three Ability to Participate sub-domains. However, because of positive and negative wording, and content redundancy, many items did not fit the IRT model, so item banks do not yet exist. EFA, CFA and IRT identified two preliminary Satisfaction item banks. One item exhibited trivial age DIF.
After extensive item preparation and review, EFA-, CFA- and IRT-guided item banks help provide increased measurement precision and flexibility. Two Satisfaction short forms are available for use in research and clinical practice. This initial validation study resulted in revised item pools that are currently undergoing testing in new clinical samples and populations.
Patient-reported outcomes; Social health; Social function; Social relationships; Item banks
This paper describes the psychometric properties of the PROMIS Pain Interference (PROMIS-PI) bank. An initial candidate item pool (n=644) was developed and evaluated based on review of existing instruments, interviews with patients, and consultation with pain experts. From this pool, a candidate item bank of 56 items was selected and responses to the items were collected from large community and clinical samples. A total of 14,848 participants responded to all or a subset of candidate items. The responses were calibrated using an item response theory (IRT) model. A final 41-item bank was evaluated with respect to IRT assumptions, model fit, differential item function (DIF), precision, and construct and concurrent validity. Items of the revised bank had good fit to the IRT model (CFI and NNFI/TLI ranged from 0.974 to 0.997), and the data were strongly unidimensional (e.g., ratio of first and second eigenvalue = 35). Nine items exhibited statistically significant DIF. However, adjusting for DIF had little practical impact on score estimates and the items were retained without modifying scoring. Scores provided substantial information across levels of pain; for scores in the T-score range 50-80, the reliability was equivalent to 0.96 to 0.99. Patterns of correlations with other health outcomes supported the construct validity of the item bank. The scores discriminated among persons with different numbers of chronic conditions, disabling conditions, levels of self-reported health, and pain intensity (p< 0.0001). The results indicated that the PROMIS-PI items constitute a psychometrically sound bank. Computerized adaptive testing and short forms are available.
Quality-of-life outcomes; quality-of-life measurement; pain
The impact of neurological disorders on the lives of patients is often far more complex than what is measured in routine examination. Measurement of this impact can be challenging owing to a lack of brief, psychometrically sound and generally accepted instruments. Two NIH-funded initiatives are developing assessment tools, in English and Spanish, which address these issues, and should prove useful to the study and treatment of epilepsy and other neurological conditions. The first, Neuro-QOL, has created a set of health-related quality of life measures that are applicable for people with common neurological disorders. The second, the NIH Toolbox for the Assessment of Neurological and Behavioral Function, is assembling measures of cognitive, emotional, motor and sensory health and function that can be used across all ages, from 3 to 85 years. This article describes both the projects and their potential value to epilepsy treatment and research.
behavioral function; epilepsy; health-related quality of life; measurement; neurological function; patient reported outcomes; quality of life
The purpose of this study was to conduct a preliminary investigation of the number of response options for self-reports of pain interference. Responses to interference items of the 11-category Brief Pain Inventory (BPI) were obtained in a sample of 434 persons from two sites and modeled using the partial credit model. In successive calibrations, response categories were collapsed and new scores were generated. Scores based on two to three categories produced poor results. Four to five categories yielded better results. However, scoring using more than five categories did not appreciably improve the reliability, person separation, or validity of scores. These results suggest that fewer response categories—as few as five or six–may function as well as the 11 response categories that are conventionally used. The results are preliminary since the number of response categories actually presented was not manipulated in the study design. Future research should compare the reliability and validity of scores based on the BPI interference items when items are presented with the conventionally 11-response format, versus presentation with fewer response options.
psychometrics; outcomes; quality of life; measurement; pain
Responses following BRCA1/2 genetic testing are relevant for comprehension of risk status and may play a role in risk management decision making. The objective of this study was to evaluate a Psychosocial Telephone Counseling (PTC) intervention delivered to BRCA1/2 mutation carriers following standard genetic counseling (SGC). We examined the impact of the intervention on distress and concerns related to genetic testing.
Patients and Methods
This prospective randomized clinical trial included 90 BRCA1/2 mutation carriers. We measured anxiety, depression, and genetic testing distress outcomes at intervention baseline and 6- and 12-months following disclosure. We evaluated the effects of SGC versus SGC plus PTC on psychological outcomes using intention-to-treat analyses through Generalized Estimating Equations.
At 6 months, PTC reduced depressive symptoms (Z = −2.25, P = .02) and genetic testing distress (Z = 2.18, P = .02) compared to standard genetic counseling. Further, women in the intervention condition reported less clinically-significant anxiety at 6 months (χ21 = 4.11, P = .04) than women who received SGC. We found no differences in outcomes between the intervention groups at the 12-month follow-up.
As an adjunct to SGC, psychosocial telephone counseling delivered following disclosure of positive BRCA1/2 test results appears to offer modest benefits for distress and anxiety. These results build upon a growing literature of psychosocial interventions for BRCA1/2 carriers and, given the potential impact of affect on risk management decision making, suggest that some carriers may derive benefits from adjuncts to traditional genetic counseling.
Psychosocial intervention; BRCA1/2 mutation carriers; telephone counseling
The aim of this study was to evaluate the reliability and validity of the Pediatric Functional Assessment of Cancer Therapy Questionnaire Brain Tumor Survivor (version 2.0) Aged 13 years and older (Parent Form) (pedsFACT-BrS parent of adolescent).
The pedsFACT-BrS parent of adolescent was translated and cross-culturally adapted into Korean, following standard Functional Assessment of Chronic Illness Therapy (FACIT) methodology. The psychometric properties of the pedsFACT-BrS parent of adolescent were evaluated in 170 brain tumor patient's mothers (mean age=43.38 years). Pretesting was performed in 30 mothers, and the results indicated good symptom coverage and overall comprehensibility. The participants also completed the Child Health Questionnaire Parent Form 50 (CHQ-PF-50), Neuroticism in Eysenck Personality Questionnaire, and Karnofsky score.
In validating the pedsFACT-BrS parent of adolescent, we found high internal consistency, with Cronbach's α coefficients ranging from 0.76 to 0.94. The assessment of test-retest reliability using intraclass correlation coefficient revealed satisfactory values with ICCs ranging from 0.84 to 0.93. The pedsFACT-BrS for parent of adolescent also demonstrated good convergent and divergent validities when correlated with the Child Health Questionnaire Parent Form 50 (CHQ-PF-50) and the Neuroticism in Eysenck Personality Questionnaire. The pedsFACT-BrS parent of adolescent showed good clinical validity, and effectively differentiated between clinically distinct patient groups according to the type of treatment, tumor location, shunt, and Karnofsky score of parent proxy report.
We confirmed that this reliable and valid instrument can be used to properly evaluate the quality of life of Korean adolescent brain tumor patients by their parents' proxy report.
Quality of life; Adolescent brain tumor patient; pedsFACT-BrS parent of adolescent; Validity
This study evaluated the psychometric properties of the FCSI-9 administered to metastatic colorectal cancer patients in a phase III clinical trial. The results provide preliminary evidence to suggest that the FCSI-9, a brief, symptom-specific measure, is reliable, valid, and responsive in this population.
Patient-reported outcomes (PROs) are essential for evaluating treatment effects on health-related quality of life and symptoms from the patient's perspective. This study sought to evaluate the psychometric properties of the nine-item Functional Assessment of Cancer Therapy/National Comprehensive Cancer Network Colorectal Cancer Symptom Index (FCSI-9) in a metastatic colorectal cancer (mCRC) population.
The FCSI-9 and EQ-5D were administered every 2–4 weeks to mCRC subjects in a phase III clinical trial. Three hundred ninety-one mCRC subjects completed the questionnaires at baseline and at least one follow-up assessment. Internal consistency reliability, test–retest reliability, construct validity, known groups validity, responsiveness, and the minimum important difference (MID) of the FCSI-9 were evaluated.
The internal consistency and test–retest reliability of the FCSI-9 were acceptable (0.81 and 0.76, respectively). Construct validity was supported based on moderate correlations with the EQ-5D. Known groups validity was evaluated by examining the FCSI-9 scores of subjects categorized by their Eastern Cooperative Oncology Group performance status (PS) score. Subjects with better PS scores reported significantly higher FCSI-9 scores than those with lower PS scores at both baseline and week 8. Responsiveness, as measured by Guyatt's statistic, was 0.77 from baseline to week 8 and 0.60 from week 4 to week 12. Considering all data together, the MID of the FCSI-9 is estimated to be in the range of 1.5–3.0 points.
Results provide preliminary evidence of the reliability, validity, and responsiveness of the FCSI-9.
Quality of life; Reliability and validity; Questionnaire; Patient outcomes; Colorectal cancer