The experience of patients with acute myeloid leukemia in remission post-transplant.

The symptoms of acute myeloid leukemia (AML) and its treatment can negatively impact patient functioning and quality of life. Through concept elicitation interviews, we sought to evaluate the experience of patients with AML in remission following hematopoietic stem cell transplant (HSCT). Thirty patients with AML in remission post-HSCT, and eight clinicians with experience treating such patients, were asked to identify symptoms and impacts associated with AML and/or its treatment. The findings were used to develop an AML conceptual disease model to reflect the experience of these patients. We identified five symptoms and six impacts that were salient to patients with AML in remission post-HSCT. Although clinician and patient perspectives largely aligned, emotional and cognitive impacts were most important to patients, whereas clinicians focused on physical impacts. This model could be used to ensure patient-reported outcome measures included in clinical trials are reflective of the post-HSCT AML patient experience.

Development and Validation of the Pediatric Sleep Practices Questionnaire: A Self-Report Measure for Youth Ages 8-17 Years.

Objective: To develop and evaluate the validity of a self-report measure of sleep practices for youth 8-17 years. Methods: Following recommended guidelines for the development of patient reported outcomes (PROs), sleep practice concepts were identified through expert (n = 8) and child (n = 28) concept elicitation interviews and a systematic literature review. Items were developed based on these concepts and tested in cognitive interviews with children (n = 32). Psychometric analyses were applied to item response data collected from a diverse sample of youth 8-17 years (n = 307). Construct validity was evaluated through tests of associations between sleep practices and sleep disturbance and sleep-related impairment. Finally, clinical validity of the tool was assessed by comparing scores of youth with and without a parent-identified sleep problem. Results: The final Pediatric Sleep Practices Questionnaire (PSPQ) included 15 items that were used to identify 5 sleep practices: sleep timing, sleep routines and consistency, technology use before bedtime, sleep environment, and the need for parental presence to fall asleep. A confirmatory factor analysis supported the hypothesized structure (all factor loadings ≥ 0.72) and PSPQ indices were significantly associated with self-reported sleep disturbances and sleep-related impairment. Finally, children with parent-reported sleep problems had shorter sleep opportunity, later bedtimes, greater need for parental presence, poorer bedtime routines, and more technology use than children without parent-reported sleep problems. Conclusions: The PSPQ was developed using best-practice PRO development methodology. The PSPQ can be used in clinical settings and for research assessment to capture modifiable sleep practices that may promote or interfere with healthy sleep.

Establishing the content validity of PROMIS Pediatric pain interference, fatigue, sleep disturbance, and sleep-related impairment measures in children with chronic kidney disease and Crohn's disease.

BACKGROUND: PROMIS Pediatric patient-reported outcome measures were developed with children from the general population, and their content validity has not been established in children with chronic disease. This study was done to evaluate the content validity of the PROMIS Pediatric Pain Interference and Fatigue measures in children 8-17 years-old with Crohn's disease and the PROMIS Pediatric Fatigue, Sleep Disturbance, and Sleep-related Impairment measures for children 8-17 years-old with chronic kidney disease. METHODS: We conducted semi-structured interviews with individuals affected by Crohn's disease and chronic kidney disease. The interviews were done to elicit children's lived experiences of the PROMIS outcomes of interest. We used deductive content analysis to contrast the participants' reports of their symptoms and impacts on daily life with existing conceptual frameworks for the PROMIS measures, each of which was developed with input from children in the general population. RESULTS: On average, we elicited an average of 7 pain interference and 7 fatigue concepts from Crohn's disease participants (n = 37), while chronic kidney disease participants (n = 26) provided 9 concepts for fatigue, 4 for sleep disturbance, and 7 for sleep-related impairment. Concept saturation was achieved after 16-19 interviews across the four PROMIS measures. Children with these two chronic health conditions reported the same breadth and types of lived experiences as children from the development samples drawn from the general population. CONCLUSION: The study supports the content validity of several PROMIS Pediatric measures for children with Crohn's disease and chronic kidney disease. These findings provide evidence that PROMIS Pediatric measures, developed as universally relevant patient-reported outcomes, may be more broadly applicable to children with chronic disease.

Clinical Validity of the PROMIS Pediatric Sleep Measures across Populations of Children with Chronic Illnesses and Neurodevelopment Disorders.

OBJECTIVE: To examine the clinical validity of the Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Sleep Disturbance (SD) and Sleep-Related Impairment (SRI) short forms. METHODS: Youth (8-17 years) from clinical populations with known SDs (sleep clinic n = 126, autism n = 276, asthma n = 82, asthma + eczema n = 68) and the general population (n = 902) completed the PROMIS Pediatric SD and SRI 8-item short forms, along with established measures of sleep (Children's Report of Sleep Patterns, Sleep Habits Survey), PROMIS Pediatric Fatigue, and parent-reported clinical indicators (does child have sleep problem, use melatonin, use prescription sleep medication). RESULTS: Confirmatory factor analyses demonstrated factorial invariance for all clinical groups. Significant differences between the general population and clinical groups were found for SD and SRI (medium to large effect sizes). Convergent validity was demonstrated through separate hierarchical regression models that showed significant associations between parent-reported clinical indicators and SD and SRI, above and beyond clinical group, as well as moderate to strong correlations between the PROMIS sleep measures and both established measures of sleep and fatigue. CONCLUSIONS: The PROMIS Pediatric SD and SRI short forms provide clinicians and researchers a brief, accurate, and valid way to measure patient-reported sleep outcomes in pediatric populations.

Qualitative Development and Content Validation of the PROMIS Pediatric Sleep Health Items.

Objective: To develop the Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Sleep Health item pool and evaluate its content validity. Participants: Participants included 8 expert sleep clinician-researchers, 64 children ages 8-17 years, and 54 parents of children ages 5-17 years. Methods: We started with item concepts and expressions from the PROMIS Sleep Disturbance and Sleep Related Impairment adult measures. Additional pediatric sleep health concepts were generated by expert (n = 8), child (n = 28), and parent (n = 33) concept elicitation interviews and a systematic review of existing pediatric sleep health questionnaires. Content validity of the item pool was evaluated with item translatability review, readability analysis, and child (n = 36) and parent (n = 21) cognitive interviews. Results: The final pediatric Sleep Health item pool includes 43 items that assess sleep disturbance (children's capacity to fall and stay asleep, sleep quality, dreams, and parasomnias) and sleep-related impairments (daytime sleepiness, low energy, difficulty waking up, and the impact of sleep and sleepiness on cognition, affect, behavior, and daily activities). Items are translatable and relevant and well understood by children ages 8-17 and parents of children ages 5-17. Conclusions: Rigorous qualitative procedures were used to develop and evaluate the content validity of the PROMIS Pediatric Sleep Health item pool. Once the item pool's psychometric properties are established, the scales will be useful for measuring children's subjective experiences of sleep.

Development and validation of the PROMIS Pediatric Sleep Disturbance and Sleep-Related Impairment item banks.

Study Objectives: To develop and evaluate the measurement properties of child-report and parent-proxy versions of the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Sleep Disturbance and Sleep-Related Impairment item banks. Methods: A national sample of 1104 children (8-17 years old) and 1477 parents of children 5-17 years old was recruited from an internet panel to evaluate the psychometric properties of 43 sleep health items. A convenience sample of children and parents recruited from a pediatric sleep clinic was obtained to provide evidence of the measures' validity; polysomnography data were collected from a subgroup of these children. Results: Factor analyses suggested two dimensions: sleep disturbance and daytime sleep-related impairment. The final item banks included 15 items for Sleep Disturbance and 13 for Sleep-Related Impairment. Items were calibrated using the graded response model from item-response theory. Of the 28 items, 16 are included in the parallel PROMIS adult sleep health measures. Reliability of the measures exceeded 0.90. Validity was supported by correlations with existing measures of pediatric sleep health and higher sleep disturbance and sleep-related impairment scores for children with sleep problems and those with chronic and neurodevelopmental disorders. The sleep health measures were not correlated with results from polysomnography. Conclusions: The PROMIS Pediatric Sleep Disturbance and Sleep-Related Impairment item banks provide subjective assessments of child's difficulty falling and staying asleep as well as daytime sleepiness and its impact on functioning. They may prove useful in the future for clinical research and practice. Future research should evaluate their responsiveness to clinical change in diverse patient populations.