Thalassemia, a chronic blood disease, necessitates life-long adherence to blood transfusions and chelation therapy to reduce iron overload. We examine stability of Health-Related Quality of Life (HRQOL) in thalassemia and adherence to chelation therapy over time, especially after changes in chelator choice.
Thalassemia Longitudinal Cohort participants in the US, UK, and Canada completed the SF-36v2 (ages 14+), and the PF-28 CHQ (parents of children<14 years). Chelation adherence was defined as self-reported percent of doses administered in the last 4 weeks.
258 adults/adolescents (mean 29.7 years) and 133 children (mean 8.5 years) completed a mean of 2.8 years follow-up. Children made few chelator changes, whereas a mean of 2.2 changes was observed among the 37% of adults/adolescents who made chelator changes, mainly, due to patient preference or medical necessity. Physical HRQOL improved among those with lower iron burden (better health status) at baseline who made a single change in chelator, but declined among participants with multiple changes and/or high iron burden (worse health status). Mental health improved among participants with lower iron burden, but iron overload was negatively associated with social functioning. Adherence did not significantly change over follow-up except for an increase after a change from DFO infusion to oral deferasirox (p=0.03). Predictors of lower adherence for adults/adolescents at follow-up included side effects, smoking, younger age, problems preparing DFO, increased number of days per week DFO prescribed, and lower physical QOL.
Strategies to balance medical needs with family, work, and personal life may assist in adherence.
iron chelation; adherence; quality of life; thalassemia
Estimate quality-adjusted life expectancy (QALE) loss due to smoking and examine trends and state differences in smoking-related QALE loss in the U.S.
Population health-related quality of life (HRQOL) scores were estimated from the Behavioral Risk Factor Surveillance System. This study constructed life tables based on U.S. mortality files and the mortality linked National Health Interview Survey and calculated QALE for smokers, non-smokers, and the total population.
In 2009, an 18-year-old smoker was expected to have 43.5 (SE = 0.2) more years of QALE, and a non-smoker of the same age was expected to have 54.6 (SE = 0.2) more years of QALE. Therefore, smoking contributed 11.0 (SE = 0.2) years of QALE loss for smokers and 4.1 years (37%) of this loss resulted from reductions in HRQOL alone. At the population level, smoking was associated with 1.9 fewer years of QALE for U.S. adults throughout their lifetime, starting at age 18.
This study demonstrates an application of a recently developed QALE estimation methodology. The analyses show good precision and relatively small bias in estimating QALE—especially at the individual level. Although smokers may live longer today than before, they still have a high disease burden due to morbidities associated with poor HRQOL.
Quality of life; Life expectancy; Quality-adjusted life year; Smoking; Mortality; Morbidity
Patient-reported outcomes (PROs) is an umbrella term covering a range of outcomes, including symptoms, functioning, health-related quality-of-life, and supportive care needs. Research regarding the appropriate PRO questionnaires to use is informative. A previously published latent class analysis (LCA) examined patterns of function, symptoms, and supportive care needs in a sample of U.S. cancer patients. The current analysis investigated whether the findings from the original study were replicated in new samples from different countries and whether a larger sample combining all the data would affect the classes identified.
This secondary analysis of data from 408 Japanese and 189 Canadian cancer patients replicated the methods used in the original LCA using data from 117 U.S. cancer patients. In all samples, subjects completed the EORTC-QLQ-C30 and Supportive Care Needs Survey-Short Form-34 (SCNS-SF34). We first dichotomized individual function, symptom, and need domain scores. We then performed LCA to investigate the patterns of domains for each of the outcomes, both in the individual country samples and then combining the data from all three samples.
Across all analyses, class assignment was made by level of function, symptoms, or needs. In individual samples, only two-class models (“high” vs. “low”) were generally identifiable while in the combined sample, three-class models (“high” vs. “moderate” vs. “low”) best fit the data for all outcomes.
In this analysis, the level of burden experienced by patients was the key factor in defining classes.
Patient-reported outcomes; quality of life; cancer; supportive care needs; latent class analysis
The objective of this study was to examine health-related quality of life among adults with work-related asthma.
We analyzed 2006–2009 Behavioral Risk Factor Surveillance System Asthma Call-back Survey data for ever-employed adults with current asthma from 38 states and District of Columbia. Individuals with work-related asthma had been told by a doctor or other health professional that their asthma was related to any job they ever had. Health-related quality of life indicators included poor self-rated health, impaired physical health, impaired mental health, and activity limitation. We calculated prevalence ratios (PRs) adjusted for age, sex, race/ethnicity, education, income, employment, and health insurance.
Of ever-employed adults with current asthma, an estimated 9.0 % had work-related asthma, 26.9 % had poor self-rated health, 20.6 % had impaired physical health, 18.2 % had impaired mental health, and 10.2 % had activity limitation. Individuals with work-related asthma were significantly more likely than those with non-work-related asthma to have poor self-rated health [PR, 1.45; 95 % confidence interval (CI), 1.31–1.60], impaired physical health (PR, 1.60; 95 % CI, 1.42–1.80), impaired mental health (PR, 1.55; 95 % CI, 1.34–1.80), and activity limitation (PR, 2.16; 95 % CI, 1.81–2.56).
Future research should examine opportunities to improve health-related quality of life among individuals with work-related asthma.
Occupational asthma; Quality of life; Occupational health; Behavioral Risk Factor Surveillance System; Asthma
Most breast cancer survivorship research focuses on the general population of survivors. Scant research investigates the potentially unique experiences of minorities, especially during and after the difficult transition from primary treatment to post-treatment. This qualitative study explored African American breast cancer survivors’ and caregivers’ quality-of-life in the post-treatment period with a focus on social and spiritual well-being.
Participants included a convenience sample of African American women with stage I-III breast cancer (N=23) who completed treatment 6–24 months before enrollment. Primary caregivers (N=22) included friends, spouses and other family members (21 complete dyads). Participants completed separate semi-structured telephone interviews. Template analysis was used to evaluate themes related to religiousness and spirituality, both across and within dyads.
After treatment, religiousness and spirituality played a major role in both survivors’ and caregivers’ lives by: 1) providing global guidance, 2) guiding illness management efforts and 3) facilitating recovery. Participants described a spiritual connectedness with God and others in their social networks. Dyad members shared the goal of keeping a positive attitude and described positive growth from cancer. Few future concerns were expressed due to the belief that survivors were healed and “done” with cancer. Beyond practical and emotional support, provision of spiritual assistance was common.
Results highlight the principal, positive role of religiousness and spirituality for African American breast cancer survivors and caregivers after treatment. Findings emphasize the need to assess the importance of religious and spiritual beliefs and practices, and if appropriate, to provide resources that promote spiritual well-being.
breast cancer; African American; spirituality; religiousness
Given that mothers often—but do not always—report children's health status in surveys, it is essential to gain an understanding of whether the relationship between children's general health status and relevant covariates depends on who reports children's general health status.
Using data from the first wave of the National Longitudinal Study of Youth 1997 cohort (N=6,466), a nationally representative sample of adolescents in the United States aged 12 to 17 in 1997,the study examines the concordance between self and maternal reports of adolescents' general health status. Then, self and maternal reports of adolescents' general health status are each regressed on health-relevant covariates and tests of differences across coefficients are estimated.
Self and maternal reports of adolescents' general health status are moderately concordant. Furthermore, the association of adolescents' general health status with adolescent BMI and the adolescent being female significantly differs across reporters such that the negative relationships are even more negative with self compared to maternal reports of adolescents' general health status. The association of adolescents' general health status with measures of adolescents' health limitations, maternal self-rated health, and sociodemographic covariates such as adolescent age, race, ethnicity, and household net worth differ across reporters in that each have greater relationships with maternal compared to self reports of adolescents' general health status.
The results are important for interpreting research on the causes and consequences of child and adolescent health, as results across studies may not be comparable if the reporter is not the same.
self-rated health; general health status; adolescent health; proxy reports; surveys
Cancer registry survival analyses have shown that adolescent and young adult patients with low socioeconomic status (SES) have reduced survival compared to those with higher SES. The objective of this study was to determine whether neighborhood- (nSES) and/or individual-level SES (iSES) also predicted current quality of life in adolescent and young adult survivors.
The Socioeconomics and Quality of Life study surveyed adolescent and young adult survivors of leukemia and lymphoma at least one year post-diagnosis using population-based ascertainment. Factor analysis was used to create a multidimensional age-relevant iSES score and compared with a preexisting census-block-group derived nSES score. Four quality of life domains were assessed: physical health, psychological and emotional well-being, social relationships, and life skills. Nested multivariable linear regression models were run to test the associations between both SES measures and quality of life and to compare the explanatory power of nSES and iSES.
Data from 110 individuals aged 16–40 were included in the final analysis. After adjustment for sociodemographic confounders, low nSES was associated only with poorer physical health, whereas low iSES was related to poorer quality of life in all four domains with iSES accounting for an additional 14, 12, 25, and 10 % of the variance, respectively.
Measures of SES at the individual as compared to the neighborhood level may be stronger indicators of outcomes in adolescents and young adults, which has important implications for SES measurement in the context of cancer surveillance.
Adolescents and young adults; Quality of life; Socioeconomic status; Health disparities; Leukemia; Lymphoma; Cancer; Cancer survivorship
The Radiation Therapy Oncology Group (RTOG) conducted a randomized, placebo-controlled, trial evaluating the efficacy of GM-CSF in reducing mucosal injury and symptom burden from curative radiotherapy for head-and-neck (H&N) cancer.
Eligible patients with H&N cancer receiving radiation encompassing ≥ 50% of the oral cavity or oropharynx received subcutaneous GM-CSF or placebo. Quality of life (QoL) was assessed using the RTOG modified University of Washington H&N symptom questionnaire at baseline, 4, 13, 26 and 48 weeks from radiation initiation.
Of 125 eligible patients, 114 were evaluable for QoL (58 GM-CSF, 56 placebo). Patient demographics, clinical characteristics, and baseline symptom scores were well balanced between the treatment arms. At the end of the acute period (13 weeks) patients in both arms reported negative change in total symptom score indicating increase in symptom burden relative to baseline (mean −18.4 GM-CSF, −20.8 placebo). There was no difference in change in total symptom score (p>0.05) or change in mucous, pain, eating, or activity domain scores (p>0.01) between patients in the GM-CSF and placebo arms. Analysis limited to patients treated per protocol or with an acceptable protocol deviation also found no difference in change in total symptom score (p>0.05) or change in domain scores (p>0.01) between treatment arms. Provider assessment of acute mucositis during treatment did not correlate with patient-reported mucous domain and total symptom scores (p>0.05)
GM-CSF administered concurrently during head-and-neck radiation does not appear to significantly improve patient-reported QoL symptom burden.
Measuring constructs such as mobility with patient-reported outcomes (PROs) can enhance clinical and scientific understanding of how health conditions, like lower limb amputation, impact patients’ lives. When developing PRO questionnaires, cognitive interviews (CIs) are used to examine if survey items are understandable, clear, and meaningful. The aim of this study was to use CIs to inform item development for the Prosthetic Limb Users Survey of Mobility (PLUS-M), a PRO that measures mobility in prosthetic limb users.
Thirty-six CIs were conducted with 30 prosthetic limb users. Each participant responded to up to 30 items from the PLUS-M candidate item set. Each item was reviewed by a minimum of five participants who differed in self-reported mobility, literacy, level of amputation, and time since amputation. Items were revised based on participant feedback and substantially revised items were re-evaluated through additional CIs.
Feedback from CIs identified substantial issues in 76 of the total 156 items. These items were subsequently modified or eliminated.
CIs were an essential qualitative step in the development of the PLUS-M item bank and resulted in better functioning items.
cognitive interviews; artificial limb; qualitative research; mobility; patient-reported outcome measure
Quality of life (QoL) has been increasingly emphasized in National Cancer Institute (NCI) sponsored multi-site clinical trials. Little is known about the outcomes of these trials in pediatric cancer. Objectives were to describe the proportion of Children’s Oncology Group (COG) QoL studies that successfully accrued subjects and were analyzed, presented or published.
We conducted a survey to describe outcomes of COG QoL studies. We included studies that contained at least one QoL assessment and were closed to patient accrual at the time of survey dissemination. Respondents were the investigators most responsible for the QoL aim.
Sixteen studies were included; response rate was 100%. Nine (56%) studies were embedded into a cancer treatment trial. Only 3 (19%) studies accrued their intended sample size. Seven (44%) studies were analyzed, 9 (56%) were presented and 6 (38%) were published.
NCI-sponsored pediatric QoL studies have high rates of failure to accrue. Many were not analyzed or disseminated. Using this data, strategies have been implemented to improve conduct in future trials. Monitoring of QoL studies is important to maximize the chances of study success.
Health state valuations, used to evaluate the effectiveness of healthcare interventions, can be obtained either by the patients or by the general population. The general population seems to value somatic conditions more negatively than patients, but little is known about valuations of psychological conditions. This study examined whether individuals with and without depression differ in their valuations of depression and whether perceptions regarding depression (empathy, perceived susceptibility, stigma, illness perceptions) and individual characteristics (mastery, self-compassion, dysfunctional attitudes) bias valuations of either individuals with or without depression.
In an online study, a general population sample used a time-trade-off task to value 30 vignettes describing depression states (four per participant) and completed questionnaires on perceptions regarding depression and individual characteristics. Participants were assigned to depression groups (with or without depression), based on the PHQ-9. A generalized linear mixed model was used to assess discrepancies in valuations and identify their determinants.
The sample (N = 1268) was representative of the Dutch population on age, gender, education and residence. We found that for mild depression states, individuals with depression (N = 200) valued depression more negatively than individuals without depression (N = 1068) (p = .007). Variables related to perceptions of depression and individual characteristics were not found to affect valuations of either individuals with or individuals without depression.
Since the general population values depression less negatively, using their perspective might result in less effectiveness for interventions for mild depression. Perceptions of depression or to individual characteristics did not seem to differentially affect valuations made by either individuals with or without depression.
Depression; Discrepancies; Valuations; Health-related quality of life; Time-trade-off
The ASCOT-Carer is a self-report instrument designed to measure social care-related quality of life (SCRQoL). This article presents the psychometric testing and validation of the ASCOT-Carer four response-level interview (INT4) in a sample of unpaid carers of adults who receive publicly funded social care services in England.
Unpaid carers were identified through a survey of users of publicly funded social care services in England. Three hundred and eighty-seven carers completed a face-to-face or telephone interview. Data on variables hypothesised to be related to SCRQoL (e.g. characteristics of the carer, cared-for person and care situation) and measures of carer experience, strain, health-related quality of life and overall QoL were collected. Relationships between these variables and overall SCRQoL score were evaluated through correlation, ANOVA and regression analysis to test the construct validity of the scale. Internal reliability was assessed using Cronbach’s alpha and feasibility by the number of missing responses.
The construct validity was supported by statistically significant relationships between SCRQoL and scores on instruments of related constructs, as well as with characteristics of the carer and care recipient in univariate and multivariate analyses. A Cronbach’s alpha of 0.87 (seven items) indicates that the internal reliability of the instrument is satisfactory and a low number of missing responses (<1 %) indicates a high level of acceptance.
The results provide evidence to support the construct validity, factor structure, internal reliability and feasibility of the ASCOT-Carer INT4 as an instrument for measuring social care-related quality of life of unpaid carers who care for adults with a variety of long-term conditions, disability or problems related to old age.
Informal care; Caregiving; Quality of life; Social care; Outcomes; ASCOT; Construct validity
VesT was a three-armed, placebo-controlled, randomized clinical trial designed to study the effects of 30mg or 60mg/day vesnarinone. Certain contradictory results involving patient health-related quality-of-life (HRQOL) and overall survival (OS) have made a definitive and unified conclusion difficult. To reconcile these findings, we have focused on the HRQOL-adjusted OS, commonly known as QALYs (quality-adjusted life years). Currently, analyses of QALYs incorporate a single HRQOL dimension or subscale. However, the VesT HRQOL instrument had two subscales, physical (PHYS) and emotional (EMOT). We sought to simultaneously analyze PHYS- and EMOT-adjusted OS in order to gain additional insight and to reach an unequivocal conclusion. Therefore, we have developed new ways to visualize and compare EMOT and PHYS-adjusted OS in the VesT by introducing a simple QALY trade-off function. In each VesT arm, there was an increased probability of superior EMOT-adjusted OS, compared to PHYS-adjusted OS. The magnitude of these findings was comparable across trial arms.
Cardiac Disease; Data Visualization; HRQOL; Overall Survival; Trade-off Function; Vesnarinone Trial
Informal caregivers play a critical role in the care of individuals who are aging or have disabilities, and are at increased risk for poor health outcomes. This study sought to determine if and to what extent: 1) global stress and health-related quality of life (HRQoL) differed between caregivers and non-caregivers; 2) global stress mediated the relationship between caregiving status and HRQoL; and 3) caregiver strain (i.e., stress attributable to caregiving) was associated with worse HRQoL after accounting for global stress.
Cross-sectional data were from the 2008–2010 Survey of the Health of Wisconsin (SHOW), a representative sample of adults aged 21–74 years. Participants (n=1,364) completed questionnaires about caregiving status, socio-demographics, global stress, and HRQoL. Staged generalized additive models assessed the impact of caregiving on HRQoL and the role of caregiver strain and global stress in this relationship.
17.2% of the sample reported caregiving in the last 12 months. Caregivers reported worse mental HRQoL than non-caregivers (Beta: −1.88, p=0.02); global stress mediated this relationship (p<0.01). Caregivers with the highest levels of strain reported worse mental and physical HRQoL (Beta: −7.12, p<0.01) and caregivers with the lowest levels of strain reported better mental HRQoL (Beta: 2.06, p=0.01) than non-caregivers; these associations were attenuated by global stress (p<0.01).
Global stress, rather than caregiving per se, contributes to poor HRQoL among caregivers, above and beyond the effect of caregiving strain. Screening, monitoring, and reducing stress in multiple life domains presents an opportunity to improve HRQoL outcomes for caregivers.
Caregivers; Stress; Psychological; Quality of Life; Population-Based; Survey of the Health of Wisconsin
To investigate the level of agreement between child self-reports and parent proxy-reports of the health-related quality of life (HRQoL) in boys with Duchenne muscular dystrophy (DMD) using both classical test theory (CTT) and Rasch analysis.
A total of 63 boys with DMD and their parents completed the pediatric quality of life inventory version 4.0 child self-report and parent proxy-report of HRQoL, respectively. The data were analyzed using both the CTT (scale-score level) and Rasch analysis (item-level).
The intraclass correlation coefficient (ICC, scale-score level) between children and parents showed good to moderate agreement, although parents consistently underestimated their child HRQoL. In Rasch analysis (item-level), 1 out of 8 items was significantly different between children and parents in the physical health scale. Also, 3 out of 15 items were significantly different between those two groups in the psychosocial health scale.
By applying both scale-score and item-level analyses, our study seeks to broaden the understanding of the discrepancy of the ratings between child self-reports and parent proxy-reports. The findings could provide further information about the decision-making process when selecting therapy and care programs.
Duchenne muscular dystrophy; DMD; Health-related quality of life; HRQoL; Item difficulty; Person ability; Rasch analysis
To examine the relationship between child self-report and parent proxy report of health-related quality of life (HRQL) and how parents’ mental health status relates to the HRQL ratings 6 years after minor to severe injury of the child.
Materials and methods
This cross-sectional cohort study was performed at a regional pediatric trauma center in Stockholm, Sweden. The PedsQL 4.0 versions for ages 5–7, 8–12, and 13–18 years were completed by 177 child–parent dyads 6 years after injury to the child. The parents also rated their own mental health through the mental health domain (MH) in the SF-36 Health Survey.
The children’s median age was 13 years (IQR 10–16 years), 54 % were males, and the median ISS was 5 (IQR 2–9). Most of the parents were female (77 %), born in Sweden (79 %), and half had university degrees. There was no statistically significant difference between child self-report and parent proxy report in any of the PedsQL 4.0 scales or summary scales. The levels of agreement between child self-report and parent proxy reports were excellent (ICC ≥ 0.80) for all scales with the exception of emotional functioning (ICC 0.53) which also was the scale with the lowest internal consistency in child self-report (α 0.60). Multiple regression analyses showed that worse parental mental health status correlated with worse child self-report and parent proxy report of children’s HRQL.
Children and their parents’ reports on child’s HRQL were in agreement. Decreased mental health in parents was associated with lower scores on parent proxy reports and child self-reports of HRQL after injury. The current investigation highlights the possible relationship between parent’s mental health status and children’s HRQL long after an injury, which should be considered in future investigations and in clinical care.
Injury; Trauma; Pediatric; Parents; PedsQL; Mental health; Depression; Health-related quality of life
This paper reports on the psychometric properties of a computerized adaptive test (CAT) version of the Mobility Assessment Tool (MAT) for older adults (MAT-CAT).
An item pool of 78 video-animation-based items for mobility was developed, and response data were collected from a sample of 234 participants aged 65–90 years. The video-animation-based instrument was designed to minimize ambiguity in the presentation of task demands. In addition to evaluating traditional psychometric properties including dimensionality, differential item functioning (DIF), and local dependence, we extensively tested the performance of several MAT-CAT measures and compared their performances with a fixed format.
Operationally, the MAT-CAT was sufficiently unidimensional and had acceptable levels of local independence. One DIF item was removed. Most importantly, the CAT measures showed that even starting with a single fixed item at the mean ability, the adaptive version delivered better performance than the fixed format in terms of several criteria including the standard error of estimate.
The MAT-CAT demonstrated satisfactory psychometric properties and superior performance to a fixed format. The video-animation-based adaptive instrument can be used for assessing mobility with specificity and precision.
Mobility Assessment Tool; Item response theory; Health-related quality of life; Mobility disability; Animation
This article presents data on the psycho-metric properties of a new measure, the Adolescent Quality of Life Mental Health Scale (AQOL-MHS), designed to measure quality of life in clinical samples of Latino adolescents aged 12–18 years. Participants were recruited in Puerto Rico to have one of five prevalent mental health disorders. The initial instrument development was achieved through a grounded theory approach with the use of focus groups and in-depth interviews.
We conducted two stages of exploratory factor analyses (EFA) on 60 candidate items. The first stage was to establish the number of factors to extract, and the second was to improve the model by selecting the best items. A final EFA model retained 31 items and 3 factors labeled Emotional Regulation (11 items), Self-Concept (10 items) and Social Context (10 items).
The instrument showed good internal consistency, test–retest reliability, and construct validity. The hypotheses-driven validity tests were all supportive of the AQOL-MHS. There was evidence for convergent validity and discriminant validity, and results for known-groups’ validity were overwhelmingly supportive of the ability of the instrument to identify differences between groups.
These preliminary findings support our conceptual model and the use of the AQOL-MHS domain and overall scores. We believe that this instrument will provide clinicians additional insight into the different aspects of quality of life that are important to adolescents with mental health problems. Therefore, we consider the AQOL-MHS a vital patient-centered outcome measure for assessment strategies in the prevention and treatment of this population.
Quality of life; Adolescents; Mental health; Patient-centered outcome measure
To develop a shorter version of the Wisconsin Upper Respiratory Symptom Survey (WURSS-21), -a self-report questionnaire for evaluating daily symptoms and functional impairments during acute respiratory illness (ARI).
WURSS-21 data were retrieved from 4 studies (n=1167) spanning the years 2002–2010. Similar methodologies were employed among these studies. Degree of missingness, ceiling/floor effects, and exploratory (EFA) and confirmatory (CFA) factor analyses were investigated and used to guide item retention. Stability of the reduced WURSS was evaluated across the 1st 3 days of ARI-illness.
Degree of missingness was < 1% and appeared to be completely at random. Seven WURSS items with >30% of ratings of zero (floor effects) were eliminated. Cross-loading items (Head-congestion, Sleep-well and Breathe-easily) were excluded following EFA on subset-1. Subsequent CFA using subset-2 showed satisfactory indices of fit. The reduced WURSS-11 instrument demonstrated 3 dimensions of 3 items each, and was stable across 3 days of illness. The indicated dimensions (items) include; Nasal (Runny-nose, Plugged-nose, Sneezing), Throat (Cough, Sore-throat, Scratchy-throat) and Quality of life (Feeling-tired, Think-clearly, Accomplish-daily-activities).
The WURSS-11 has similar dimensional structure as the WURSS-21. This shorter version may reduce the time and burden required for completing the survey.
Common cold; quality of life; factor analysis; acute respiratory infection; symptom assessment
To develop a practical, efficient, and valid pediatric global health measure that would be useful for clinical, quality improvement, and research applications.
Using the PROMIS mixed-methods approach for item bank development, we identified an item pool that was well understood by children as young as age 8 years, and tested its psychometric properties in an internet panel sample of 3,635 children 8–17 years-old and 1,807 parents of children 5–17 years-old.
The final version of the pediatric global health measure included 7 items assessing general, physical, mental, and social health. Four of these items had the same wording as the PROMIS adult global health measure. Internal consistency was 0.88 for the child-report form and 0.84 for the parent form; both had excellent test-retest reliability. The measures showed factor invariance across age categories. There was no differential item functioning by age, gender, race, or ethnicity. Because the measure includes the general health rating question, it is possible to estimate the pediatric global health scale using this widely used single item.
The PROMIS Pediatric Global Health measure is a brief and reliable 7-item summary assessment of a child’s self-reported health. Future work will attempt to statistically link this pediatric form with the PROMIS adult global health measures to create a single global health metric that can be used across the life course.
patient-reported outcome; quality of life; health status; child; global health
To assess the impact of weight loss on health-related quality-of-life (HRQL), to describe the factors associated with improvements in HRQL after weight loss, and to assess the relationship between obesity as assessed by body mass index (BMI) and HRQL before and after weight loss.
We studied 188 obese patients with BMI ≥32 kg/m2 with one or more comorbidities or ≥35 kg/m2. All patients had baseline and follow-up assessments of BMI and HRQL using the EuroQol (EQ-5D) and its visual analog scale (VAS) before and after 6 months of medical weight loss that employed very low calorie diets, physical activity, and intensive behavioral counseling.
At baseline, age was 50 ± 8 years (mean ± SD), BMI was 40. 0 ± 5.0 kg/m2, EQ-5D-derived health utility score was 0.85 ± 0.13, and VAS-reported quality-of-life was 0.67 ± 0.18. At 6 months follow-up, BMI decreased by 7.0 ± 3.2 kg/m2, EQ-5D increased by 0.06 (interquartile range (IQR) 0.06 – 0.17), and VAS increased by 0.14 (IQR 0.04 – 0.23). In multivariate analyses, improvement in EQ-5D and VAS were associated with lower baseline BMI, greater reduction in BMI at follow-up, fewer baseline comorbidities, and lower baseline HRQL. For any given BMI category, EQ-5D and VAS tended to be higher at follow-up than at baseline.
Measured improvements in HRQL between baseline and follow-up were greater than predicted by the reduction in BMI at follow-up. If investigators use cross-sectional data to estimate changes in HRQL as a function of BMI, they will underestimate the improvement in HRQL associated with weight loss and underestimate the cost-utility of interventions for obesity treatment.
Pharmacotherapy is an effective treatment for anxiety disorders, but
its effects on quality of life have not been examined systematically. Our
objective was to conduct an effect size analysis of pharmacological
interventions on quality of life outcomes in patients with DSM-IV anxiety
Manual and electronic searches using PubMed, PsycINFO, and the
Cochrane Library were conducted for records from the first available date
through May 1st, 2013 for trials of pharmacological interventions
in patients with anxiety disorders, which had measures of quality of life
before and after treatment. Of 1,865 entries, 93 studies were identified as
potentially relevant and 32 met inclusion criteria, of which results were
examined from 22 studies reporting 27 distinct pharmacological trials,
representing data from 4,344 anxiety disorder patients. Data were extracted
independently by multiple observers to estimate within-group and
placebo-controlled random effects of the treatment changes on quality of
life. We hypothesized that pharmacotherapy improves quality of life, which
is associated with improvement in anxiety symptoms.
Pharmacological interventions effectively improved quality of life
from before to after treatment (Hedges' g = .59), although
the controlled effect size is smaller among those trials with placebo
interventions (Hedges' g = .32). These effect sizes were
robust, increased with publication year, and increased with reductions in
Pharmacological therapy is effective for improving quality of life in
anxiety disorders, and larger symptom reductions are associated with greater
improvement in quality of life.
meta-analysis; anxiety; pharmacotherapy; quality of life; psychopharmacology; SSRI
Cognitive dysfunction is a common concern for children with brain tumors (BTs) or those receiving central nervous system (CNS) toxic cancer treatments. Perceived cognitive function (PCF) is an economical screening that may be used to trigger full, formal cognitive testing. We assessed the potential clinical utility of PCF by comparing parent-reported scores for children with cancer with scores from the general US population.
Children (n = 515; mean age =13.5 years; 57.0 % male) and one of their parents were recruited from pediatric oncology clinics. Most children (53.3 %) had a diagnosis of CNS tumor with an average time since diagnosis of 5.6 years. PCF was evaluated using the pediatric PCF item bank (pedsPCF), which was developed and normed on a sample drawn from the US general pediatric population. Children also completed computer-based neuropsychological tests. We tested relationships between PCF and clinical variables. Differential item functioning (DIF) was used to evaluate measurement bias between the samples.
No item showed DIF, supporting the use of pedsPCF in the cancer sample. PedsPCF differentiated children with (vs. without) a BT, p < 0.01, and groups defined by years since diagnosis, p < 0.01. It significantly (p < 0.05) correlated with computerized neuropsychological tests in 40 of 60 comparisons. Children with BTs were rated as having worse pedsPCF scores than the norm, regardless of years since diagnosis.
PCF significantly differentiated cancer survivors with various clinical characteristics. It is brief and easy to implement. PCF should be considered for routine care of pediatric cancer survivors.
Perceived cognitive function; Item bank; Pediatric cancer; Brain tumor; Item response theory; Quality of life
The objectives of the present study are to investigate the precision of static (fixed-length) short forms versus computerized adaptive testing (CAT) administration, response pattern scoring versus summed score conversion, and test-retest reliability (stability) of the Patient Reported Outcomes Measurement Information System (PROMIS®) pediatric self-report scales measuring the latent constructs of depressive symptoms, anxiety, anger, pain interference, peer relationships, fatigue, mobility, upper extremity functioning and asthma impact with polytomous items.
Participants (N = 331) between the ages of 8 and 17 were recruited from outpatient general pediatrics and subspecialty clinics. Of the 331 participants, 137 were diagnosed with asthma. Three scores based on item response theory (IRT) were computed for each respondent: CAT response pattern expected a posteriori estimates, short form response pattern expected a posteriori estimates, and short form summed score expected a posteriori estimates. Scores were also compared between participants with and without asthma. To examine test-retest reliability, 54 children were selected for retesting approximately two weeks after the first assessment.
A short CAT (maximum 12 items with a standard error of 0.4) was found, on average, to be less precise than the static short forms. The CAT appears to have limited usefulness over and above what can be accomplished with existing static short forms (8–10 items). Stability of the scale scores over a two week period was generally supported.
The study provides further information on the psychometric properties of the PROMIS pediatric scales and extends the previous IRT analyses to include precision estimates of dynamic versus static administration, test-retest reliability, and validity of administration across groups. Both the positive and negative aspects of using CAT vs. short forms are highlighted.
PROMIS; pediatrics; self-report; patient reported outcomes; item response theory; computerized adaptive testing
To test whether or not adults assign the same values to hypothetical health states that describe health in adults as when those same descriptions refer to the health of a child.
A two-part self-completion questionnaire was designed in which respondents were asked firstly to rate a fixed set of EQ-5D-Y health states on a 0–100 visual analogue scale as if they themselves were in these states. Two versions of the questionnaire were produced each with a different second part. One version instructed respondents to value the same states but to imagine them describing another adult. The second version required respondents to value these states as if they applied to a 10-year-old child. Questionnaires were distributed to adults recruited in three countries (Germany, Spain and England) using convenience sampling methods.
A total of 1085 questionnaires were completed. Despite some significant differences in the characteristics of the achieved samples in the three countries involved, the rank order of health states was largely consistent across each adult/child reference perspective. In all countries, the mean values were lower when health states described children rather than adults. Significant differences were found for 16/24 states when values for those states applied to adult respondent themselves were compared with the values for those states applied to a 10-year-old child. A near-uniform pattern was found across all three countries in which health state values for children were found to be lower than for adults.
Values for health states when ascribed to adults are higher than when those same states are associated with children. Were EQ-5D-3L values for adults applied to EQ-5D-Y health states, then this would effectively lead to an misrepresentation of the value assigned to a health status in children.
EQ-5D; EQ-5D-Y; Valuation of health; Children