Purpose An essential aspect of patient-centered outcomes research (PCOR) and comparative effectiveness research (CER) is the integration of patient perspectives and experiences with clinical data to ...evaluate interventions. Thus, PCOR and CER require capturing patient-reported outcome (PRO) data appropriately to inform research, healthcare delivery, and policy. This initiative’s goal was to identify minimum standards for the design and selection of a PRO measure for use in PCOR and CER. Methods We performed a literature review to find existing guidelines for the selection of PRO measures. We also conducted an online survey of the International Society for Quality of Life Research (ISOQOL) membership to solicit input on PRO standards. A standard was designated as “recommended” when >50 % respondents endorsed it as “required as a minimum standard.” Results The literature review identified 387 articles. Survey response rate was 120 of 506 ISOQOL members. The respondents had an average of 15 years experience in PRO research, and 89 % felt competent or very competent providing feedback. Final recommendations for PRO measure standards included: documentation of the conceptual and measurement model; evidence for reliability, validity (content validity, construct validity, responsiveness); interpretability of scores; quality translation, and acceptable patient and investigator burden. Conclusion The development of these minimum measurement standards is intended to promote the appropriate use of PRO measures to inform PCOR and CER, which in turn can improve the effectiveness and efficiency of healthcare delivery. A next step is to expand these minimum standards to identify best practices for selecting decision-relevant PRO measures.
Objective To assess minimally important differences (MIDs) for several pediatrie 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.
Abstract
Background
Patient-reported outcome (PRO) measurements linked to Common Terminology Criteria for Adverse Events (CTCAE) grading may improve symptom adverse event (AE) reporting in pediatric ...oncology trials. We evaluated construct validity, responsiveness, and test-retest reliability of the Ped-PRO–CTCAE measurement system for children and adolescents undergoing cancer care.
Methods
A total of 482 children and adolescents (7–18 years, 41.5% not non-Hispanic white) newly diagnosed with cancer and their caregivers participated from nine pediatric oncology hospitals. Surveys were completed at 72 hours preceding treatment initiation (T1) and at follow-up (T2) approximately 7–17 days later for chemotherapy, and 4+ weeks for radiation. Psychometric analyses examined the relationship of Ped-PRO-CTCAE items (assessing 62 symptom AEs) with Patient-Reported Outcomes Measurement Information System, Memorial Symptom Assessment Scale, Lansky Play-Performance Scale, and medication use. A separate test-retest study included 46 children.
Results
Ped-PRO-CTCAE and Memorial Symptom Assessment Scale were strongly correlated across age groups at T2: 7–12 years (r = 0.62–0.80), 13–15 years (r = 0.44–0.94), and 16–18 years (r = 0.65–0.98); and over time. The Ped-PRO-CTCAE was strongly correlated with Patient-Reported Outcomes Measurement Information System Pediatric measures at T2; for example, pain interference (r = 0.70, 95% confidence interval CI = 0.64 to 0.76), fatigue severity (r = 0.63, 95% CI = 0.56 to 0.69), and depression severity (r = 0.76, 95% CI = 0.71 to 0.81). Ped-PRO-CTCAE items differentiated children by Lansky Play-Performance Scale and by medication use. Test-retest agreement ranged from 54.3% to 93.5%.
Conclusions
This longitudinal study provided evidence for the construct validity and reliability of the core Ped-PRO-CTCAE symptom AE items relative to several established measures. Additional responsiveness data with clinical anchors are recommended. Incorporation of Ped-PRO-CTCAE in trials may lead to a better understanding of the cancer treatment experience.
Most pediatric clinicians aspire to promote the physical, emotional, and developmental well-being of children, hoping to bestow a long and healthy life. Yet, some infants, children, and adolescents ...confront life-threatening illnesses and life-shortening conditions. Over the past 70 years, the clinician's response to the suffering of these children has evolved from veritable neglect to the development of pediatric palliative care as a subspecialty devoted to their care. In this article, we review the history of how clinicians have understood and responded to the suffering of children with serious illnesses, highlighting how an initially narrow focus on anxiety eventually transformed into a holistic, multidimensional awareness of suffering. Through this transition, and influenced by the adult hospice movement, pediatric palliative care emerged as a new discipline. Becoming a discipline, however, has not been a panacea. We conclude by highlighting challenges remaining for the next generation of pediatric palliative care professionals to address.
Children in treatment for cancer experience multiple, troubling, and interrelated symptoms. Analyzing the interrelatedness of symptoms and how that changes during treatment could yield clinically ...relevant patient profiles to guide patient care.
The aims were to identify pediatric profiles with respect to Patient-Reported Outcomes Measurement Information System® (PROMIS®; U.S. Department of Health and Human Services, National Institutes of Health) symptom measures, changes in profile status throughout a chemotherapy cycle, and if a baseline single-item fatigue measure could significantly predict symptom profile status and its changes.
In a longitudinal, single-site, three data point (T1, T2, and T3) design, children between 8 and 18 years completed the PROMIS Pediatric short form measures for fatigue, depression, anxiety, and pain and one fatigue item from the Symptom Distress Scale. Latent profile analysis and latent transition analysis were conducted.
About 96 children participated; 58.3% were between 13 and 18 years, and 54.2% were males. Two latent profiles (less severe symptoms and severe symptoms) were identified. The prevalence of the severe symptom profile remained relatively unchanged from T1 to T2 but significantly declined at T3. The baseline single-item fatigue score significantly predicted the child's profile membership and its changes.
Children experiencing troubling symptoms during cancer treatment are heterogeneous. With respect to the PROMIS symptom measures, two a priori unknown distinct latent profiles of patients were identified in a course of chemotherapy, and the transitions in the profile status were significantly predicted by a baseline single-item fatigue measure.
Adult patients are considered the best reporters of their own health-related quality of life (HRQOL). Self-report in pediatrics has been challenged by a limited array of valid measures. Caregiver ...report is therefore often used as a proxy for child report.
To examine the degree of alignment between child and caregiver proxy report for Patient-Reported Outcomes Measurement Information System (PROMIS) HRQOL domains among children with cancer and to identify factors associated with better child and caregiver-proxy congruence.
In this multicenter cohort study, children with a first cancer diagnosis and their caregivers completed surveys at 2 time points: within 72 hours preceding treatment initiation (T1) and during follow-up (T2), when symptom burden was expected to be higher (eg, 7-17 days later for chemotherapy). Data were collected from October 26, 2016, to October 5, 2018, at 9 pediatric oncology hospitals. Five hundred eighty children (aged 7-18 years) and their caregivers were approached; 482 child-caregiver dyads completed surveys at T1 (response rate 83%), and 403 completed surveys at T2 (84% of T1 participants). Data were analyzed from July 1, 2019, to April 22, 2020.
Participants received up-front cancer treatment, including chemotherapy and radiotherapy.
Congruence between child self-report and caregiver-proxy report of PROMIS pediatric domains of mobility (physical functioning), pain interference, fatigue, depressive symptoms, anxiety, and psychological stress.
Of the 482 dyads included in the analysis, 262 children (54%) were male (mean SD age, 12.9 3.4 years), 80 (17%) were Black, and 71 (15%) were Hispanic. Intraclass correlations between child self-report and caregiver proxy report showed moderate agreement for mobility (0.57 95% CI, 0.50-0.63) and poor agreement for symptoms (range, 0.32 95% CI, 0.24-0.41 for fatigue to 0.42 95% CI, 0.34-0.50 for psychological stress). Children reported lower symptom burden and higher mobility than caregivers reported. In a multivariable model adjusted for child and parent sociodemographic factors and the caregiver's own self-reported HRQOL, caregivers reported the child's mobility score 6.00 points worse than the child's self-report at T2 (95% CI, -7.45 to -4.51), exceeding the PROMIS minimally important difference of 3 points. Caregivers overestimated the child's self-reported symptom levels, ranging from 5.79 (95% CI, 3.99-7.60) points for psychological stress to 13.69 (95% CI, 11.60-15.78) points for fatigue. The caregiver's own self-reported HRQOL was associated with the magnitude of difference between child and caregiver scores for all domains except mobility; for example, for fatigue, the magnitude of difference between child and caregiver-proxy scores increased by 0.21 (95% CI, 0.13-0.30) points for each 1-point increase in the caregiver's own fatigue score.
This study found that caregivers consistently overestimated symptoms and underestimated mobility relative to the children themselves. These results suggest that elicitation of the child's own report should be pursued whenever possible.
To identify factors important to parents making decisions for their critically ill child.
Prospective cross-sectional study.
Single center, tertiary care PICU.
Parents making critical treatment ...decisions for their child.
One-on-one interviews that used the Good Parent Tool-2 open-ended question that asks parents to describe factors important for parenting their ill child and how clinicians could help them achieve their definition of "being a good parent" to their child. Parent responses were analyzed thematically. Parents also ranked themes in order of importance to them using the Good Parent Ranking Exercise.
Of 53 eligible parents, 43 (81%) participated. We identified nine themes through content analysis of the parent's narrative statements from the Good Parent Tool. Most commonly (60% of quotes) components of being a good parent described by parents included focusing on their child's quality of life, advocating for their child with the medical team, and putting their child's needs above their own. Themes key to parental decision making were similar regardless of parent race and socioeconomic status or child's clinical status. We identified nine clinician strategies identified by parents as helping them fulfill their parenting role, most commonly, parents wanted to be kept informed (32% of quotes). Using the Good Parent Ranking Exercise, fathers ranked making informed medical decisions as most important, whereas mothers ranked focusing on the child's health and putting their child's needs above their own as most important. However, mothers who were not part of a couple ranked making informed medical decisions as most important.
These findings suggest a range of themes important for parents to "be a good parent" to their child while making critical decisions. Further studies need to explore whether clinician's knowledge of the parent's most valued factor can improve family-centered care.
Adolescents with cardiac diagnoses face unique challenges that can cause psychosocial distress. This study compares a Mindfulness-Based Stress Reduction (MBSR) program to a video online support group ...for adolescents with cardiac diagnoses. MBSR is a structured psycho-educational program which includes yoga, meditation, cognitive restructuring, and group support. A published feasibility study by our group showed significant reduction in anxiety following this intervention. Participants were randomized to MBSR or video online support group, and completed measures of anxiety, depression, illness-related stress, and coping pre- and post-6-session interventions. Qualitative data were obtained from post-intervention interviews. A total of 46 teens participated (mean 14.8 years; 63% female). Participants had congenital heart disease and/or cardiac device (52%), or postural orthostatic tachycardia syndrome (48%). Illness-related stress significantly decreased in both groups. Greater use of coping skills predicted lower levels of depression in both groups post-study completion. Higher baseline anxiety/depression scores predicted improved anxiety/depression scores in both groups. Each group reported the benefits of social support. The MBSR group further expressed benefits of learning specific techniques, strategies, and skills that they applied in real-life situations to relieve distress. Both the MBSR intervention and video support group were effective in reducing distress in this sample. Qualitative data elucidated the added benefits of using MBSR techniques to manage stress and symptoms. The video group format is useful for teens that cannot meet in person but can benefit from group support. Psychosocial interventions with stress management techniques and/or group support can reduce distress in adolescents with cardiac diagnoses.
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