Heather E. Gross

PROMIS Pediatric Measures in Pediatric Oncology: Valid and Clinically Feasible Indicators of Patient-Reported Outcomes

Establishing the ability of children and adolescents with cancer to complete the NIH‐sponsored PROMIS pediatric measures electronically and the preliminary validity estimates of the measures (both full item banks and short forms) in pediatric oncology will contribute to our knowledge of the impact of cancer treatment on these young patients.

Development of six PROMIS pediatrics proxy-report item banks

BackgroundPediatric self-report should be considered the standard for measuring patient reported outcomes (PRO) among children. However, circumstances exist when the child is too young, cognitively impaired, or too ill to complete a PRO instrument and a proxy-report is needed. This paper describes the development process including the proxy cognitive interviews and large-field-test survey methods and sample characteristics employed to produce item parameters for the Patient Reported Outcomes Measurement Information System (PROMIS) pediatric proxy-report item banks.MethodsThe PROMIS pediatric self-report items were converted into proxy-report items before undergoing cognitive interviews. These items covered six domains (physical function, emotional distress, social peer relationships, fatigue, pain interference, and asthma impact). Caregivers (n = 25) of children ages of 5 and 17 years provided qualitative feedback on proxy-report items to assess any major issues with these items. From May 2008 to March 2009, the large-scale survey enrolled children ages 8-17 years to complete the self-report version and caregivers to complete the proxy-report version of the survey (n = 1548 dyads). Caregivers of children ages 5 to 7 years completed the proxy report survey (n = 432). In addition, caregivers completed other proxy instruments, PedsQL™ 4.0 Generic Core Scales Parent Proxy-Report version, PedsQL™ Asthma Module Parent Proxy-Report version, and KIDSCREEN Parent-Proxy-52.ResultsItem content was well understood by proxies and did not require item revisions but some proxies clearly noted that determining an answer on behalf of their child was difficult for some items. Dyads and caregivers of children ages 5-17 years old were enrolled in the large-scale testing. The majority were female (85%), married (70%), Caucasian (64%) and had at least a high school education (94%). Approximately 50% had children with a chronic health condition, primarily asthma, which was diagnosed or treated within 6 months prior to theinterview. The PROMIS proxy sample scored similar or better on the other proxy instruments compared to normative samples.ConclusionsThe initial calibration data was provided by a diverse set of caregivers of children with a variety of common chronic illnesses and racial/ethnic backgrounds. The PROMIS pediatric proxy-report item banks include physical function (mobility n = 23; upper extremity n = 29), emotional distress (anxiety n = 15; depressive symptoms n = 14; anger n = 5), social peer relationships (n = 15), fatigue (n = 34), pain interference (n = 13), and asthma impact (n = 17).

PROMIS Pediatric Peer Relationships Scale: Development of a Peer Relationships Item Bank as Part of Social Health Measurement

OBJECTIVE This studys objective was to develop a measure of social health using item response theory as part of the Patient Reported Outcomes Measurement Information System (PROMIS). METHODS After candidate items were generated from review of prior literature, focus groups, expert input, and cognitive interviews, items were administered to youth aged 8-17 as part of the PROMIS pediatric large scale testing. Exploratory and confirmatory factor analyses were used to assess dimensionality and to identify instances of local dependence. Items that met the unidimensionality criteria were subsequently calibrated using Samejimas Graded Response Model. Differential item functioning was examined by gender and age. RESULTS The sample included 3,048 youth who completed the questionnaire (51.8% female, 60% white, and 22.7% with chronic illness). The initial conceptualization of social function and sociability did not yield unidimensional item banks. Rather, factor analysis revealed dimensions contrasting peer relationships and adult relationships. The analysis also identified dimensions formed by responses to positively versus negatively worded items. The resulting 15-item bank measures quality of peer relationships and has strong psychometric characteristics as a full bank or an 8-item short form. CONCLUSIONS The PROMIS pediatric peer relationships scale demonstrates good psychometric characteristics and addresses an important aspect of child health.

Promising insights into the health related quality of life for children with severe obesity

BackgroundChildhood obesity is a growing health concern known to adversely affect quality of life in children and adolescents. The Patient Reported Outcomes Measurement Information System (PROMIS) pediatric measures were developed to capture child self-reports across a variety of health conditions experienced by children and adolescents. The purpose of this study is to begin the process of validation of the PROMIS pediatric measures in children and adolescents affected by obesity.MethodsThe pediatric PROMIS instruments were administered to 138 children and adolescents in a cross-sectional study of patient reported outcomes in children aged 8–17 years with age-adjusted body mass index (BMI) greater than the 85th percentile in a design to establish known-group validity. The children completed the depressive symptoms, anxiety, anger, peer relationships, pain interference, fatigue, upper extremity, and mobility PROMIS domains utilizing a computer interface. PROMIS domains and individual items were administered in random order and included a total of 95 items. Patient responses were compared between patients with BMI 85 to < 99th percentile versus ≥ 99th percentile.Results136 participants were recruited and had all necessary clinical data for analysis. Of the 136 participants, 5% ended the survey early resulting in missing domain scores at the end of survey administration. In multivariate analysis, patients with BMI ≥ 99th percentile had worse scores for depressive symptoms, anger, fatigue, and mobility (p < 0.05). Parent-reported exercise was associated with better scores for depressive symptoms, anxiety, and fatigue (p < 0.05).ConclusionsChildren and adolescents ranging from overweight to severely obese can complete multiple PROMIS pediatric measures using a computer interface in the outpatient setting. In the 5% with missing domain scores, the missing scores were consistently found in the domains administered last, suggesting the length of the assessment is important. The differences in domain scores found in this study are consistent with previous reports investigating the quality of life in children and adolescents with obesity. We show that the PROMIS instrument represents a feasible and potentially valuable instrument for the future study of the effect of pediatric obesity on quality of life.

Feasibility and acceptability of the patient-reported outcomes measurement information system measures in children and adolescents in active cancer treatment and survivorship

Background: Patient-reported outcomes related to symptoms, function, and quality of life during and following cancer treatment can guide care for pediatric cancer patients. To advance the science of patient-reported outcomes, the National Institutes of Health funded the Patient-Reported Outcomes Measurement Information System (PROMIS). Objective: The objective of this study was to assess feasibility and acceptability of the PROMIS pediatric measures, as defined by enrollment and attrition rates as well as missingness by measure, item, participant, and assessment time point. Methods: Eight- to18-year-olds participated in 2 studies: PROMIS I, a cross-sectional study of children in active cancer treatment or survivorship, and PROMIS II, a longitudinal study with 3 assessment time points for children receiving curative treatment. Results: PROMIS I (n = 200) and PROMIS II (n = 94) had enrollment rates of 92.5% and 89.7%, respectively. For PROMIS I, measure missingness was acceptable (8% missed any measures) and was not related to other study variables. For PROMIS II, measure missingness was minimal (0.8%), and item-level missingness was relatively low. In general, items that were skipped asked about experiences that participants had not encountered in the past 7 days. Conclusions: In both studies, the PROMIS instruments demonstrated good feasibility and acceptability among pediatric cancer patients. Overall, we had high enrollment, low attrition, and acceptable rates of measure and item missingness. Implications for Practice: Our results demonstrate that PROMIS measures are acceptable to 8- to 18-year-olds in different points of cancer care and feasible for use in pediatric cancer inpatient and outpatient settings.

Responsiveness of PROMIS® Pediatric Measures to Hospitalizations for Sickle Pain and Subsequent Recovery.

The Patient‐Reported Outcomes Measurement Information System® (PROMIS®) created pediatric self‐report scales measuring a variety of health attributes (domains), but their responsiveness to changes in health status has not yet been determined in children with sickle cell disease (SCD).

Initial Evaluation of the Pediatric PROMIS® Health Domains in Children and Adolescents With Sickle Cell Disease.

The Patient Reported Outcomes Measurement Information System (PROMIS®) has developed pediatric self‐report scales measuring several unidimensional health attributes (domains) suitable for use in clinical research, but these measures have not yet been validated in sickle cell disease (SCD).

Using item response theory to enrich and expand the PROMIS® pediatric self report banks.

BackgroundThe primary objective was to enhance the content coverage of some of the pediatric self-report item banks for ages 8-17 years from the National Institutes of Health (NIH) Patient Reported Outcomes Measurement Information System (PROMIS®), and extend the range of precise measurement to higher levels of physical functioning.MethodsData from 1,419 pediatric patients with cancer, chronic kidney disease, obesity, rehabilitation needs, rheumatic disease, and sickle cell disease were combined with item responses from the original standardization sample of 3,048 children to calibrate new items for the pediatric PROMIS Anger, Anxiety, Depressive Symptoms, Pain Interference, Fatigue, and physical functioning Upper Extremity and Mobility scales. Simultaneous or concurrent calibration using the graded item response theory model placed all of the items on the same scale.ResultsTwenty-two of 28 potential new items were added across the seven scales. A recommended short form was proposed for the Anger scale, and the recommended short forms for the Anxiety and Depressive Symptoms scales were revised. Unfortunately, we were not particularly successful at extending the range of measurement for the physical functioning banks.ConclusionsThe present study expanded PROMIS pediatric item banks to add new content and to increase the range of measurement. Using item response theory, the banks were revised and expanded without changing the underlying scale of measurement. For Anger, Anxiety, and Depressive Symptoms, we successfully added new content that may render those banks more robust and flexible.

Longitudinal associations among asthma control, sleep problems, and health-related quality of life in children with asthma: a report from the PROMIS(®) Pediatric Asthma Study.

Objectives Few studies have investigated the complex relationship among asthma control, sleep problems, and health-related quality of life (HRQOL) among children with asthma. This study aimed to test the longitudinal effect of asthma control status on asthma-specific HRQOL through the mechanism of nighttime sleep quality and daytime sleepiness. Methods 229 dyads of asthmatic children and their parents engaged in the Patient-Reported Outcomes Measurement Information System (PROMIS®) Pediatric Asthma Study with 2 years of follow-up to assess the change of asthma control, sleep problems, and asthma-specific HRQOL. The Asthma Control and Communication Instrument was used to measure asthma control status. Nighttime sleep quality assessment was based on difficulty falling asleep and getting up, and sleep disturbance. The Iowa Pediatric Daytime Sleeping Scale was used to assess daytime sleepiness. The PROMIS Asthma Impact Scale was used to measure asthma-specific HRQOL. Multilevel structural equation modeling was performed to quantify the direct and indirect effects of asthma control status on asthma-specific HRQOL through nighttime sleep quality and daytime sleepiness. Results Poorer asthma control status was directly associated with lower asthma-specific HRQOL at within-subject and between-subject levels (p’s<0.05); however, effects of asthma control on asthma-specific HRQOL were indirectly influenced through daytime sleepiness at within-subject level (p<0.05), and through nighttime sleep quality and daytime sleepiness at between-subject level (p<0.05). Conclusions Asthma control status is associated with asthma-specific HRQOL, and this association is mediated by nighttime sleep quality and daytime sleepiness. Clinicians should address sleep problems related to asthma control to improve HRQOL for asthmatic children.

Mode effects between computer self-administration and telephone interviewer-administration of the PROMIS® pediatric measures, self- and proxy report

ObjectiveTo test equivalence of scores obtained with the PROMIS® pediatric Depressive Symptoms, Fatigue, and Mobility measures across two modes of administration: computer self-administration and telephone interviewer-administration. If mode effects are found, to estimate the magnitude and direction of the mode effects.MethodsRespondents from an internet survey panel completed the child self-report and parent proxy-report versions of the PROMIS® pediatric Depressive Symptoms, Fatigue, and Mobility measures using both computer self-administration and telephone interviewer-administration in a crossed counterbalanced design. Pearson correlations and multivariate analysis of variance were used to examine the effects of mode of administration as well as order and form effects.ResultsCorrelations between scores obtained with the two modes of administration were high. Scores were generally comparable across modes of administration, but there were some small significant effects involving mode of administration; significant differences in scores between the two modes ranged from 1.24 to 4.36 points.ConclusionsScores for these pediatric PROMIS measures are generally comparable across modes of administration. Studies planning to use multiple modes (e.g., self-administration and interviewer-administration) should exercise good study design principles to minimize possible confounding effects from mixed modes.