Assessing responsiveness over time of the PROMIS® pediatric symptom and function measures in cancer, nephrotic syndrome, and sickle cell disease.

PURPOSE: Previous studies provided evidence for the validity of the PROMIS Pediatric measures in cross-sectional studies. This study evaluated the ability of the PROMIS Pediatric measures to detect change over time in children and adolescents with cancer, nephrotic syndrome (NS), or sickle cell disease (SCD). METHODS: Participants (8-17 years) completed measures of fatigue, pain interference, anger, anxiety, depressive symptoms, mobility, upper extremity, and peer relationships at three or four time points (T1-T4). Between T1 and T2, children with cancer received chemotherapy and children with SCD experienced a pain exacerbation. Children with NS were first assessed during active disease (T2), with T3 and T4 conducted at disease remission. For the primary analysis of responsiveness, we expected better scores at T3 (recovery) compared to T2 (event) for all diseases. T1 and T4 are also expected to have better scores than T2. Linear mixed models were used and adjusted for time, gender, age, race/ethnicity, education, comorbid conditions, and disease. RESULTS: Enrolled were 96 children with cancer, 121 children with SCD, and 127 children with NS. Fatigue, pain interference, mobility, and upper extremity scores worsened from T1 (baseline) to T2 (event) (p < 0.01), and significantly improved from T2 to T3 and T4 (p < 0.01). Similarly, anxiety and depressive symptoms significantly improved from T2 to T3 and T4 (p < 0.01). CONCLUSIONS: This study provides evidence for the responsiveness of seven PROMIS Pediatric measures to clinical disease state in three chronic illnesses. The findings support use of PROMIS Pediatric measures in clinical research.

Voices of children and adolescents on phase 1 or phase 2 cancer trials: A new trial endpoint?

BACKGROUND: Pediatric participants on phase 1 or phase 2 clinical trials for incurable cancer are at risk of experiencing toxicities (adverse events [AEs]) related to trial participation. Multiple AEs are subjective; thus, the real impact of trial treatment cannot be known unless patient subjective reports are solicited. METHODS: The authors assessed the feasibility and acceptability of soliciting symptom, function, and quality of life (QOL) reports from participants aged 8 to 18 years who were enrolled on phase 1/2 clinical trials at 4 cancer centers during the first course of chemotherapy. The authors also assessed the reliability and validity of 6 self-report Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric measures and 4 open-ended interview questions at 2 time points (at the time of trial enrollment [T1] and 3 to 4 weeks later [T2]). RESULTS: The enrollment rate of 75.9% (20 participants) exceeded the feasibility criterion, and missingness of measures by person, measure, and items at T1 and T2 were lower than the acceptability criteria. New QOL themes were limited to the impact of treatment on families and being away from home, family, and friends for treatment. All but one measure at T1 met the reliability criterion and all measures did so at T2. Validity support was limited however because as theorized, mobility decreased and fatigue increased as AEs increased. CONCLUSIONS: Soliciting and documenting symptom, function, and QOL reports from patients aged 8 to 18 years who are enrolled on a phase 1/2 clinical trial is feasible and acceptable to participants, particularly when embedded in trials. Reliable and valid findings can result, making patient self-reported outcomes a possible new trial endpoint. Cancer 2017;123:3799-3806. © 2017 American Cancer Society.

Responsiveness of the PROMIS® measures to changes in disease status among pediatric nephrotic syndrome patients: a Midwest pediatric nephrology consortium study.

BACKGROUND: Nephrotic syndrome represents a condition in pediatric nephrology typified by a relapsing and remitting course, proteinuria and the presence of edema. The PROMIS measures have previously been studied and validated in cross-sectional studies of children with nephrotic syndrome. This study was designed to longitudinally validate the PROMIS measures in pediatric nephrotic syndrome. METHODS: One hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17 years participated in this prospective cohort study. Patients completed a baseline assessment while their nephrotic syndrome was active, a follow-up assessment at the time of their first complete proteinuria remission or study month 3 if no remission occurred, and a final assessment at study month 12. Participants completed six PROMIS measures (Mobility, Fatigue, Pain Interference, Depressive Symptoms, Anxiety, and Peer Relationships), the PedsQL version 4.0, and two global assessment of change items. RESULTS: Disease status was classified at each assessment: nephrotic syndrome active in 100% at baseline, 33% at month 3, and 46% at month 12. The PROMIS domains of Mobility, Fatigue, Pain Interference, Depressive Symptoms, and Anxiety each showed a significant overall improvement over time (p < 0.001). When the PROMIS measures were compared to the patients' global assessment of change, the domains of Mobility, Fatigue, Pain Interference, and Anxiety consistently changed in an expected fashion. With the exception of Pain Interference, change in PROMIS domain scores did not correlate with changes in disease activity. PROMIS domain scores were moderately correlated with analogous PedsQL domain scores. CONCLUSION: This study demonstrates that the PROMIS Mobility, Fatigue, Pain Interference, and Anxiety domains are sensitive to self-reported changes in disease and overall health status over time in children with nephrotic syndrome. The lack of significant anchoring to clinically defined nephrotic syndrome disease active and remission status may highlight an opportunity to improve the measurement of HRQOL in children with nephrotic syndrome through the development of a nephrotic syndrome disease-specific HRQOL measure.

Responsiveness to Change in PROMIS(®) Measures among Children with Asthma: A Report from the PROMIS(®) Pediatric Asthma Study.

OBJECTIVE: To compare the responsiveness to change of the Patient-Reported Outcomes Measurement Information System (PROMIS(®)) asthma impact, pain interference, fatigue, depressive symptoms, mobility, and peer relationship scales to a legacy scale, the Paediatric Asthma Quality of Life Questionnaire (PAQLQ). METHODS: Two hundred and twenty-nine child-parent dyads from public insurance programs were enrolled. PROMIS pediatric short forms (SFs) and the PAQLQ were used to measure health-related quality of life across four time points (T1-T4) over 2 years. The Asthma Control and Communication Instrument was used to measure the change in asthma control, and the Global Rating of Change (GRC) Index for breathing problems and overall health was used to measure the change in health status. Responsiveness was tested by comparing the changes in health-related quality of life with the changes in asthma control and health status over time using t tests, generalized estimating equations, and relative validity approaches. Magnitudes of the responsiveness between the Pediatric PROMIS and PAQLQ were assessed through statistical significance, Cohen's d effect size (ES), and standardized response mean (SRM). RESULTS: The PROMIS asthma impact scale and all PAQLQ scales exhibited significant responsiveness (p's<0.05) and small to medium ES/SRM when anchored to asthma control, GRC breathing problems, and overall health. Relative validity, especially related to change in asthma control status and GRC breathing problems, was equivalent. PROMIS pain interference, fatigue, and mobility SFs also indicated adequate responsiveness. CONCLUSIONS: The PROMIS asthma impact SF indicated similar responsiveness to the PAQLQ scales. Due to its brevity and responsiveness, the PROMIS asthma impact SF is useful for clinical practice or research.

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

BACKGROUND: 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). PROCEDURE: A convenience sample of SCD children, aged 8-17 years, from two sickle cell programs was recruited at routine clinic visits, including some for hydroxyurea monitoring or monthly transfusions. Children completed PROMIS pediatric items using an online data collection platform, the PROMIS Assessment Center Web site. RESULTS: A total of 235 participants (mean age 12.5 ± 2.8 years, 49.8% female) participated in the study. Adolescents (ages 12-17 years) reported significantly higher pain interference and depressive symptoms, and worse lower extremity physical functioning domain scores compared to younger children (ages 8-11 years). Female participants reported significantly higher pain interference, fatigue, and depressive symptoms, and worse lower extremity physical functioning domain scores compared with their male counterparts. Participants with hip or joint problems that limited usual activities reported significantly higher pain, fatigue, and depressive symptoms scores, and worse upper/lower extremity physical functioning scores as did participants who had experienced sickle pain in the previous 7 days. CONCLUSIONS: PROMIS pediatric measures are feasible in a research setting and identify expected differences in known group comparisons in a sample of SCD children. The large domain score differences between those with or without SCD-related complications suggest the potential usefulness of these measures in clinical research, but further validation studies are needed, particularly in clinical practice settings.

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

BACKGROUND: 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). PROCEDURE: A convenience cohort of symptomatic SCD children, aged 8-17 years, was asked to complete PROMIS pediatric scales at an initial clinic visit, at the end of a subsequent hospitalization for sickle pain, at a subsequent clinic visit or at home 2-3 weeks after hospitalization, and at a clinic visit 1-2 years after their initial assessment. RESULTS: A total of 121 participants (mean age 12.5 ± 3.1 years, 56.2% female) participated in the study. Pain interference and fatigue domain scores were elevated at baseline, increased substantially during hospitalization, and largely returned to baseline by the recovery period, whereas the depressive symptoms, anger, and anxiety domain scores displayed a less pronounced elevation during hospitalizations and a slower return to baseline levels. The two physical functioning scales showed a substantial decline in response to hospitalization, but only modest improvements at the recovery assessment, likely representing incomplete recovery. CONCLUSIONS: Several PROMIS pediatric measures were responsive to changes in health status associated with occurrence and resolution of acute vaso-occlusive pain requiring hospitalization. The substantial differences in these domains during SCD-related pain exacerbations support their potential usefulness in clinical research or in clinical practice. Further studies to characterize variations in symptom patterns over time may provide insights into strategies for more effective management of sickle pain.

An item-level response shift study on the change of health state with the rating of asthma-specific quality of life: a report from the PROMIS(®) Pediatric Asthma Study.

PURPOSE: To examine item-level response shift associated with the change in asthma-related health state (i.e., change in asthma control status and global rating of change (GRC) in breathing problems). METHODS: Study sample comprised 238 asthmatic children who were between 8 and 17.9 years and completed the Pediatric Asthma Quality of Life Questionnaire (PAQLQ) symptoms, emotion function, and activity limitation domains at baseline and a follow-up assessment. Structural equation modeling was implemented to assess item-level response shift associated with the change in asthma-related health state with the adjustment for the influence of confounding variables. The magnitude of item-level response shift and its influence on the change of domain scores was estimated using Cohen's effect sizes. RESULTS: We found no instances of item-level response shift. However, two items were identified with measurement bias related to GRC due to breathing problems. Specifically, asthmatic children with better/about the same GRC due to breathing problems reported lower scores for one item in the emotional domain at follow-up compared to those with deteriorated GRC due to breathing problems. In addition, asthmatic children with better/about the same GRC due to breathing problems reported better scores for another item in the symptom domain at baseline compared to those with deteriorated GRC due to breathing problems. The impact of measurement bias was small and did not bias the change of domain scores over time. CONCLUSIONS: No item-level response shift, but two instances of measurement bias, appears in asthmatic children. However, the impact of these measurement issues is negligible.

Known-groups validity of the Patient-Reported Outcomes Measurement Information System (PROMIS(®)) in adolescents and young adults with special healthcare needs.

PURPOSE: To examine known-groups validity of the Patient-Reported Outcomes Measurement Information System (PROMIS(®)) Short Forms (SFs) for adolescents and young adults with special healthcare needs (SHCN) using data collected from the PROMIS Linking Study. METHODS: In total, 292 adolescents aged 14-17.9 years and 300 young adults aged 18-20 years with SHCN from the PROMIS Linking Study were used for analyses. Presence of SHCN was classified into three categories (medication use, service use, and functional limitations). HRQoL was measured with the PROMIS Pediatric and Adult SFs. Differences in health-related quality of life (HRQoL) domain scores between SHCN sample and the norms of the PROMIS pediatric and adult calibration samples, respectively, were compared using t tests. Associations of HRQoL scores with the presence of individual SHCN categories were tested using linear regression. RESULTS: All HRQoL domain scores for the SHCN samples were significantly worse than the respective calibration samples. Adolescents and young adults with SHCN-related service use and functional limitations reported worse HRQoL than those without service use and functional limitations (p's < 0.01). CONCLUSIONS: PROMIS Pediatric and Adult SFs possess satisfactory known-groups validity related to SHCN status.

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

OBJECTIVE: To 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. METHODS: Respondents 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. RESULTS: Correlations 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. CONCLUSIONS: Scores 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.

Linkage between the PROMIS® pediatric and adult emotional distress measures.

PURPOSE: Research studies that measure health-related quality of life (HRQOL) in both children and adults and longitudinal studies that follow children into adulthood need measures that can be compared across these age groups. This study links the PROMIS pediatric and adult emotional distress measures using data from participants with diverse health conditions and disabilities. METHODS: Analyses were conducted and compared in two separate samples to confirm the stability of results. One sample (n = 874) included individuals aged 14-20 years with special health care needs and who require health services. The other sample (n = 641) included individuals aged 14-25 years who have a physical or cognitive disability. Participants completed both PROMIS pediatric and adult measures. Item response theory-based scores were linked using the linear approximation to calibrated projection. RESULTS: The estimated latent-variable correlation between pediatric and adult PROMIS measures ranged from 0.87 to 0.94. Regression coefficients ß 0 (intercept) and ß 1 (slope), and mean squared error are provided to transform scores from the pediatric to the adult measures, and vice versa. CONCLUSIONS: This study used a relatively new linking method, calibrated projection, to link PROMIS pediatric and adult measure scores, thus expanding the use of PROMIS measures to research that includes both populations.

Estimating minimally important difference (MID) in PROMIS pediatric measures using the scale-judgment method.

OBJECTIVE: To assess minimally important differences (MIDs) for several pediatric self-report item banks from the National Institutes of Health Patient-Reported Outcomes Measurement Information System(®) (PROMIS(®)). METHODS: We presented vignettes comprising sets of two completed PROMIS questionnaires and asked judges to declare whether the individual completing those questionnaires had an important change or not. We enrolled judges (including adolescents, parents, and clinicians) who responded to 24 vignettes (six for each domain of depression, pain interference, fatigue, and mobility). We used item response theory to model responses to the vignettes across different judges and estimated MID as the point at which 50 % of the judges would declare an important change. RESULTS: We enrolled 246 judges (78 adolescents, 85 parents, and 83 clinicians). The MID estimated with clinician data was about 2 points on the PROMIS T-score scale, and the MID estimated with adolescent and parent data was about 3 points on that same scale. CONCLUSIONS: The MIDs enhance the value of PROMIS pediatric measures in clinical research studies to identify meaningful changes in health status over time.

The impact of disease duration on quality of life in children with nephrotic syndrome: a Midwest Pediatric Nephrology Consortium study.

BACKGROUND: The Patient Reported Outcomes Measurement Information System (PROMIS) II is a prospective study that evaluates patient reported outcomes in pediatric chronic diseases as a measure of health-related quality of life (HRQOL). We have evaluated the influence of disease duration on HRQOL and, for the first time, compared the findings of the PROMIS measures to those of the PedsQL™ 4.0 Generic Scales (PedsQL) from the PROMIS II nephrotic syndrome (NS) longitudinal cohort. METHODS: This was a prospective study in which 127 children (age range 8-17 years) with active NS from 14 centers were enrolled. Children with active NS defined as the presence of nephrotic range proteinuria (>2+ urinalysis and edema or urine protein/creatinine ratio >2 g/g) were eligible. Comparisons were made between children with prevalent (N = 67) and incident (N = 60) disease at the study enrollment visit. RESULTS: The PROMIS scores were worse in prevalent patients in the domains of peer relationship (p = 0.01) and pain interference (p < 0.01). The PedsQL showed worse scores in prevalent patients for social functioning (p < 0.01) and school functioning (p = 0.03). Multivariable analyses showed that prevalent patients had worse scores in PROMIS pain interference (p = 0.02) and PedsQL social functioning (p < 0.01). CONCLUSION: The PROMIS measures detected a significant impact of disease duration on HRQOL in children, such that peer relationships were worse and pain interfered with daily life to a greater degree among those with longer disease duration. These findings were in agreement with those for similar domains in the PedsQL legacy instrument.

PROMIS(®) pediatric self-report scales distinguish subgroups of children within and across six common pediatric chronic health conditions.

PURPOSE: To conduct a comparative analysis of eight pediatric self-report scales for ages 8-17 years from the National Institutes of Health (NIH) Patient Reported Outcomes Measurement Information System (PROMIS(®)) in six pediatric chronic health conditions, using indicators of disease severity. METHODS: Pediatric patients (N = 1454) with asthma, cancer, chronic kidney disease, obesity, rheumatic disease, and sickle cell disease completed items from the PROMIS pediatric mobility, upper extremity functioning, depressive symptoms, anxiety, anger, peer relationships, pain interference, and fatigue self-report scales. Comparisons within the six pediatric chronic health conditions were conducted by examining differences in groups based on the disease severity using markers of severity that were specific to characteristics of each disease. A comparison was also made across diseases between children who had been recently hospitalized and those who had not. RESULTS: In general, there were differences in self-reported health outcomes within each chronic health condition, with patients who had higher disease severity showing worse outcomes. Across health conditions, when children with recent hospitalizations were compared with those who had not been hospitalized in the past 6 months, we found significant differences in the expected directions for all PROMIS domains, except anger. CONCLUSIONS: PROMIS measures discriminate between different clinically meaningful subgroups within several chronic illnesses. Further research is needed to determine the responsiveness of the PROMIS pediatric scales to change over time.

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

BACKGROUND: The 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. METHODS: Data 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. RESULTS: Twenty-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. CONCLUSIONS: The 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.

Gaining the Patient Reported Outcomes Measurement Information System (PROMIS) perspective in chronic kidney disease: a Midwest Pediatric Nephrology Consortium study.

BACKGROUND AND OBJECTIVES: Chronic kidney disease is a persistent chronic health condition commonly seen in pediatric nephrology programs. Our study aims to evaluate the sensitivity of the Patient Reported Outcomes Measurement Information System (PROMIS) pediatric instrument to indicators of disease severity and activity in pediatric chronic kidney disease. METHODS: This cross sectional study included 233 children 8-17 years old, with chronic kidney disease from 16 participating institutions in North America. Disease activity indicators, including hospitalization in the previous 6 months, edema, and number of medications consumed daily, as well as disease severity indicators of kidney function and coexisting medical conditions were captured. PROMIS domains, including depression, anxiety, social-peer relationships, pain interference, fatigue, mobility, and upper extremity function, were administered via web-based questionnaires. Absolute effect sizes (AES) were generated to demonstrate the impact of disease on domain scores. Four children were excluded because of missing glomerular filtration rate (GFR) estimations. RESULTS: Of the 229 children included in the final analysis, 221 completed the entire PROMIS questionnaire. Unadjusted PROMIS domains were responsive to chronic kidney disease activity indicators and number of coexisting conditions. PROMIS domain scores were worse in the presence of recent hospitalizations (depression AES 0.33, anxiety AES 0.42, pain interference AES 0.46, fatigue AES 0.50, mobility AES 0.49), edema (depression AES 0.50, anxiety AES 0.60, pain interference AES 0.77, mobility AES 0.54) and coexisting medical conditions (social peer-relationships AES 0.66, fatigue AES 0.83, mobility AES 0.60, upper extremity function AES 0.48). CONCLUSIONS: The PROMIS pediatric domains of depression, anxiety, social-peer relationships, pain interference, and mobility were sensitive to the clinical status of children with chronic kidney disease in this multi-center cross sectional study. We demonstrated that a number of important clinical characteristics including recent history of hospitalization and edema, affected patient perceptions of depression, anxiety, pain interference, fatigue and mobility. The PROMIS instruments provide a potentially valuable tool to study the impact of chronic kidney disease. Additional studies will be required to assess responsiveness in PROMIS score with changes in disease status over time.

Psychometric properties of the PROMIS ® pediatric scales: precision, stability, and comparison of different scoring and administration options.

OBJECTIVES: 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. METHODS: 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 2 weeks after the first assessment. RESULTS: 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 the existing static short forms (8-10 items). Stability of the scale scores over a 2-week period was generally supported. CONCLUSION: 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 versus short forms are highlighted.

PROMIS® Parent Proxy Report Scales for children ages 5-7 years: an item response theory analysis of differential item functioning across age groups.

OBJECTIVE: The objective of the present study is to describe the extension of the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS(®)) pediatric parent proxy-report item banks for parents of children ages 5-7 years, and to investigate differential item functioning (DIF) between the data obtained from parents of 5-7-year-old children with the data obtained from parents of 8-17 year-old children in the original construction of the scales. METHODS: Item response theory (IRT) analyses of DIF were conducted comparing data from the 5-7 age group with data from the established scales for ages 8-17 across 5 generic health domains (physical functioning, pain, fatigue, emotional health, and social health) and asthma. RESULTS: IRT DIF analyses revealed that the majority of the items functioned similarly with responses from parents of younger and older children. A small number of items were removed from the item bank for younger children, and a few items that exhibited statistical DIF were retained in the pools with the caveat that they should not be used in studies that involve comparisons of younger children with older children. CONCLUSIONS: The study confirms that most of the items in the PROMIS parent proxy-report item banks can be used with parents of children ages 5-7. It is anticipated that these new scales will have application for younger pediatric populations when pediatric self-report is not feasible.

PROMIS pediatric measures in pediatric oncology: valid and clinically feasible indicators of patient-reported outcomes.

BACKGROUND: 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. PROCEDURES: A total of 203 8- to 17-year olds were administered eight PROMIS pediatric measures in a cross-sectional study design to establish known-group validity. Of the 200 who completed all or most of the items, a slight majority were male (55.5%) and white (54%). Patients were either undergoing treatment for cancer (n = 93) or in survivorship following treatment for cancer (n = 107). Measures were completed using computer interface during an in-person interaction with researchers. RESULTS: Only 3 of 203 participants did not complete the PROMIS pediatric measures. As hypothesized, participants in treatment were significantly different (worse) on parent-reported clinical indicators (blood counts, fatigue, and appetite) and on seven self-reported measures (depression, anxiety, peer relationships, pain interference, fatigue, upper extremity function, and mobility) from participants in survivorship. Females reported worse fatigue, anger, and pain interference than males. Worse patient-reported outcomes for patients in active treatment persisted after adjusting for potential confounding variables. CONCLUSIONS: Children and adolescents in treatment for cancer or in survivorship and ranging from 8 to 17 years of age can complete multiple PROMIS pediatric measures using a computer interface during an outpatient clinic visit or inpatient admission. Findings establish known-group validity for PROMIS pediatric measures in pediatric oncology.

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

BACKGROUND: Childhood 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. METHODS: The 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. RESULTS: 136 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). CONCLUSIONS: Children 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.

Gaining the PROMIS perspective from children with nephrotic syndrome: a Midwest pediatric nephrology consortium study.

BACKGROUND AND OBJECTIVES: Nephrotic syndrome (NS) represents a common disease in pediatric nephrology typified by a relapsing and remitting course and characterized by the presence of edema that can significantly affect the health-related quality of life in children and adolescents. The PROMIS pediatric measures were constructed to be publically available, efficient, precise, and valid across a variety of diseases to assess patient reports of symptoms and quality of life. This study was designed to evaluate the ability of children and adolescents with NS to complete the PROMIS assessment via computer and to initiate validity assessments of the short forms and full item banks in pediatric NS. Successful measurement of patient reported outcomes will contribute to our understanding of the impact of NS on children and adolescents. DESIGN: This cross-sectional study included 151 children and adolescents 8-17 years old with NS from 16 participating institutions in North America. The children completed the PROMIS pediatric depression, anxiety, social-peer relationships, pain interference, fatigue, mobility and upper extremity functioning measures using a web-based interface. Responses were compared between patients experiencing active NS (n = 53) defined by the presence of edema and patients with inactive NS (n = 96) defined by the absence of edema. RESULTS: All 151 children and adolescents were successfully able to complete the PROMIS assessment via computer. As hypothesized, the children and adolescents with active NS were significantly different on 4 self-reported measures (anxiety, pain interference, fatigue, and mobility). Depression, peer relationships, and upper extremity functioning were not different between children with active vs. inactive NS. Multivariate analysis showed that the PROMIS instruments remained sensitive to NS disease activity after adjusting for demographic characteristics. CONCLUSIONS: Children and adolescents with NS were able to successfully complete the PROMIS instrument using a web-based interface. The computer based pediatric PROMIS measurement effectively discriminated between children and adolescents with active and inactive NS. The domain scores found in this study are consistent with previous reports investigating the health-related quality of life in children and adolescents with NS. This study establishes known-group validity and feasibility for PROMIS pediatric measures in children and adolescents with NS.