Speech difficulties and patient health communication mediating effects on worry and health-related quality of life in children, adolescents, and young adults with Neurofibromatosis Type 1.

The objective was to investigate the serial mediating effects of speech difficulties, patient health communication, and disease-specific worry in the relationship between neurofibromatosis (NF) symptoms (pain and skin symptoms) and total generic health-related quality of life (HRQOL) in children, adolescents, and young adults with NF Type 1 (NF1) from the patient perspective. The Speech, Communication, Worry, Pain, Skin Itch Bother, and Skin Sensations Scales from the Pediatric Quality of Life Inventory (PedsQL) NF1 Module and the PedsQL 4.0 Generic Core Scales were completed in a multi-site national study by 305 patients ages 5-25 years. A serial multiple mediator model analysis was conducted to test the hypothesized sequential mediating effects of speech difficulties, health communication, and worry as intervening variables in the association between NF1 symptoms and HRQOL. Symptoms predictive effects on total generic HRQOL were serially mediated by speech difficulties, patient health communication, and worry. In predictive analytics models utilizing hierarchical multiple regression analyses with age and gender demographic covariates, the pain, skin itch bother, and skin sensations multiple mediator models accounted for 61%, 59%, and 56% of the variance in generic HRQOL (p < .001), reflecting large effect sizes. Speech difficulties, patient health communication, and disease-specific worry explain in part the mechanism of symptoms predictive effects on total generic HRQOL in pediatric patients with NF1. Identifying NF1-specific predictors and serial mediators of total generic HRQOL in pediatric patients with NF1 from the patient perspective enables a patient-centered comprehensive care approach for children, adolescents, and young adults with NF1.

Pain, skin sensations symptoms, and cognitive functioning predictors of health-related quality of life in pediatric patients with Neurofibromatosis Type 1.

OBJECTIVES: The aim was to investigate pain, skin sensations symptoms and patient self-reported, and parent proxy-reported cognitive functioning as predictors of generic health-related quality of life (HRQOL) in pediatric patients with Neurofibromatosis Type 1 (NF1) from the perspectives of patients and parents. METHODS: The Pain, Skin Itch Bother, Skin Sensations, and Cognitive Functioning Scales from the PedsQL™ Neurofibromatosis Type 1 Module and the PedsQL™ Generic Core Scales were completed in a multi-site national study by 323 patients and 335 parents. Patients were 5-25 years of age. Pain and skin symptoms and cognitive functioning were tested for bivariate and multivariate linear associations with generic HRQOL. RESULTS: Pain, skin itch bother, skin sensations, and cognitive functioning were associated with decreased HRQOL in bivariate analyses (Ps < 0.001). In predictive analytics models, utilizing hierarchical multiple regression analyses controlling for demographic covariates, pain, skin itch bother, skin sensations, and cognitive functioning as a group accounted for 61 percent of the variance in patient-reported generic HRQOL (P < 0.001), reflecting a large effect size. For parent proxy-report, the predictor variables as a group accounted for 53% of the variance in generic HRQOL. CONCLUSIONS: Pain, skin symptoms, and patient self-reported and parent proxy-reported cognitive functioning are key predictors of generic HRQOL in pediatric patients with NF1. Delineating NF1-specific symptoms and cognitive functioning as high-priority predictors from the patient and parents perspective enhances a family-centered approach in clinical research, clinical trials, and clinical practice intended to improve the global generic HRQOL of pediatric patients with NF1.

PedsQL Neurofibromatosis Type 1 Module for children, adolescents and young adults: feasibility, reliability, and validity.

The objective of the present study was to report on the measurement properties of the Pediatric Quality of Life Inventory (PedsQL) Neurofibromatosis Type 1 Module for pediatric patients ages 5-25 from the perspectives of patients and parents. The 104-item PedsQL NF1 Module and 23-item PedsQL Generic Core Scales were completed in a multi-site national study by 323 patients and 335 parents (343 families). Patients were diagnosed with NF1 using the National Institutes of Health diagnostic criteria. In addition to a Total Scale Score, 18 unidimensional scales were derived measuring skin itch bother, skin sensations, pain, pain impact, pain management, cognitive functioning, speech, fine motor, balance, vision, perceived physical appearance, communication, worry, treatment anxiety, medicines, stomach discomfort, constipation, and diarrhea. The PedsQL NF1 Module Scales evidenced excellent feasibility, excellent reliability for the Total Scale Scores (patient self-report α = 0.98; parent proxy-report α = 0.98), and good to excellent reliability for the 18 individual scales (patient self-report α = 0.71-0.96; parent proxy-report α = 0.73-0.98). Intercorrelations with the Generic Core Scales supported construct validity. Factor analysis supported the unidimensionality of the 18 individual scales. The PedsQL NF1 Module Scales demonstrated acceptable to excellent measurement properties, and may be utilized as standardized metrics to assess NF1-specific symptoms and problems in clinical research and practice in children, adolescents, and young adults.

Development of the pediatric quality of life inventory neurofibromatosis type 1 module items for children, adolescents and young adults: qualitative methods.

Health-related quality of life (HRQOL) is arguably one of the most important measures in evaluating effectiveness of clinical treatments. At present, there is no disease-specific outcome measure to assess the HRQOL of children, adolescents and young adults with Neurofibromatosis Type 1 (NF1). This study aimed to develop the items and support the content validity for the Pediatric Quality of Life Inventory™ (PedsQL™) NF1 Module for children, adolescents and young adults. The iterative process included multiphase qualitative methods including a literature review, survey of expert opinions, semi-structured interviews, cognitive interviews and pilot testing. Fifteen domains were derived from the qualitative methods, with content saturation achieved, resulting in 115 items. The domains include skin, pain, pain impact, pain management, cognitive functioning, speech, fine motor, balance, vision, perceived physical appearance, communication, worry, treatment, medicines and gastrointestinal symptoms. This study is limited because all participants are recruited from a single-site. Qualitative methods support the content validity for the PedsQL™ NF1 Module for children, adolescents and young adults. The PedsQL™ NF1 Module is now undergoing national multisite field testing for the psychometric validation of the instrument development.

Weight gain and supplemental O2 : Risk factors during the hematopoietic cell transplant admission in pediatric patients.

BACKGROUND: Respiratory failure in the pediatric hematopoietic cell transplant (HCT) recipient is the leading cause for admission to the intensive care unit and carries a high mortality rate. The objective of this study is to investigate the association of clinical risk factors with the development of respiratory failure in the pediatric allogeneic HCT recipient. PROCEDURES: This is a single-center, retrospective review of allogeneic pediatric HCT from 2008 to 2014. Multiple variables were examined. The primary outcome was respiratory failure. Percent weight gain was investigated at multiple time points. Bivariate and multivariate regression was used. RESULTS: Of the 87 allogeneic HCT recipients, 22 (25%) developed respiratory failure. Mortality for entire cohort was 13.8%. All who died were intubated prior to death. The group with respiratory failure had significantly higher percent weight gain increase at multiple time points: peak weight prior to discharge or intubation (P = 0.008), weight at discharge or intubation (P = .001), and weight at day 43 (median day for intubation) (P = 0.02). Odds ratio (OR) for respiratory failure increased with increasing percentage peak weight gain: 10% increase (3.1 [1.1, 9.0]), 15% increase (4.1 [1.5, 11.2]), and 20% (8.3 [2.4. 28.9]). Fifty percent of all patients required supplemental O2 . OR for respiratory failure in patients requiring more than 1 l supplemental O2 is 25.3 (6.5, 98.7). CONCLUSION: Percent weight gain and need for supplemental oxygen is highly associated with the development of respiratory failure in pediatric HCT recipients, representing predictors of acute respiratory failure in pediatric HCT. These data could be incorporated into an algorithm that should be developed, implemented, and validated in a prospective, multicenter fashion.

Effectiveness of cross-cultural education for medical residents caring for burmese refugees.

BACKGROUND: Limited resources are available to educate health professionals on cultural considerations and specific healthcare needs of Burmese refugees. The objective of this study was to determine the effectiveness of a module focused on cross-cultural considerations when caring for Burmese refugees. METHODS: A brief educational module using anonymously tracked pre- and post-intervention, self-administered surveys was developed and studied. The surveys measured pediatric and family medicine residents' knowledge, attitudes, and comfort in caring for Burmese refugees. Paired t-tests for continuous variables and Fisher's exact tests for categorical variables were used to test pre- and post-intervention differences. We included open-ended questions for residents to describe their experiences with the Burmese population. RESULTS: The survey was available to 173 residents. Forty-four pre- and post-intervention surveys were completed (response rate of 25%). Resident comfort in caring for Burmese increased significantly after the module (P = 0.04). Resident knowledge of population-specific cultural information increased regarding ethnic groups (P = 0.004), appropriate laboratory use (P = 0.04), and history gathering (P = 0.001). Areas of improved resident attitudes included comprehension of information from families (P = 0.03) and length of time required with interpreter (P = 0.01). Thematic evaluation of qualitative data highlighted four themes: access to interpreter and resources, verbal communication, nonverbal communication, and relationship building with cultural considerations. DISCUSSION: A brief intervention for residents has the potential to improve knowledge, attitudes, and comfort in caring for Burmese patients. Interventions focused on cultural considerations in medical care may improve cultural competency when caring for vulnerable patient populations.

Development of the adult PedsQL™ neurofibromatosis type 1 module: initial feasibility, reliability and validity.

BACKGROUND: Neurofibromatosis type 1 (NF1) is a common autosomal dominant genetic disorder with significant impact on health-related quality of life (HRQOL). Research in understanding the pathogenetic mechanisms of neurofibroma development has led to the use of new clinical trials for the treatment of NF1. One of the most important outcomes of a trial is improvement in quality of life, however, no condition specific HRQOL instrument for NF1 exists. The objective of this study was to develop an NF1 HRQOL instrument as a module of PedsQL™ and to test for its initial feasibility, internal consistency reliability and validity in adults with NF1. METHODS: The NF1 specific HRQOL instrument was developed using a standard method of PedsQL™ module development - literature review, focus group/semi-structured interviews, cognitive interviews and experts' review of initial draft, pilot testing and field testing. Field testing involved 134 adults with NF1. Feasibility was measured by the percentage of missing responses, internal consistency reliability was measured with Cronbach's alpha and validity was measured by the known-groups method. RESULTS: Feasibility, measured by the percentage of missing responses was 4.8% for all subscales on the adult version of the NF1-specific instrument. Internal consistency reliability for the Total Score (alpha =0.97) and subscale reliabilities ranging from 0.72 to 0.96 were acceptable for group comparisons. The PedsQL™ NF1 module distinguished between NF1 adults with excellent to very good, good, and fair to poor health status. CONCLUSIONS: The results demonstrate the initial feasibility, reliability and validity of the PedsQL™ NF1 module in adult patients. The PedsQL™ NF1 Module can be used to understand the multidimensional nature of NF1 on the HRQOL patients with this disorder.

Evaluation of a Program to Reduce Infant Mortality Risk Factors in Central Indiana.

The purpose of the program WeCare was to provide a personalized approach to addressing the wide array of psycho-social-cultural-fiscal needs to reduce risk factors for infant mortality (IM) by supporting maternal and infant health through health coaching by community health workers (CHWs). A prospective cohort study of 1,513 women from highest risk ZIP codes for IM in central Indiana were followed over three years. The WeCare program focused on training and deploying CHWs to provide evidence-based, patient-centered social care and support to pregnant and postpartum women and tracked outcomes in health risk categories (mental health, nutrition, safe sleep, breastfeeding) targeted for behavioral change. The low birth weight rate among program participants was lower (8.9%) than Marion County's rate (10%) (p=0.23) with statistically significant improvement in many risk behaviors. Despite limitations, the personalized coaching provided by the CHWs provided an effective, practical approach to maternal and child health disparities.

Cognitive functioning and pain interference mediate pain predictive effects on health-related quality of life in pediatric patients with Neurofibromatosis Type 1.

OBJECTIVES: The objective was to investigate the serial mediating effects of perceived cognitive functioning and pain interference in daily living in the relationship between perceived pain and overall generic health-related quality of life (HRQOL) in children, adolescents, and young adults with Neurofibromatosis Type 1 (NF1). METHODS: The Pain, Cognitive Functioning, and Pain Impact Scales from the PedsQL Neurofibromatosis Type 1 Module and the PedsQL 4.0 Generic Core Scales were completed in a multi-site national study by 323 patients ages 5-25 and 335 parents. A serial multiple mediator model analysis was conducted to test the hypothesized sequential mediating effects of cognitive functioning and pain interference as intervening variables in the association between pain as a predictor variable and overall generic HRQOL. RESULTS: Pain predictive effects on overall generic HRQOL were serially mediated by cognitive functioning and pain interference. In predictive analytics models utilizing hierarchical multiple regression analyses with age and gender demographic covariates, pain, cognitive functioning and pain interference accounted for 66% of the variance in patient-reported generic HRQOL and 57% of the variance in parent proxy-reported generic HRQOL (P < 0.001), reflecting large effect sizes. CONCLUSIONS: Cognitive functioning and pain interference explain in part the mechanism of pain predictive effects on overall generic HRQOL in pediatric patients with NF1. Identifying NF1-specific pain, cognitive functioning, and pain interference as salient predictors of overall generic HRQOL from the patient and parent perspective facilitates a family-centered orientation to the comprehensive care of children, adolescents, and young adults with NF1.

Dental providers' attitudes regarding the application of fluoride varnish by pediatric health care providers.

OBJECTIVES: To ascertain what proportion of dental hygienists and dentists in Indiana, United States, support the application of fluoride varnish in medical offices, and to determine if support differed by dental provider characteristics, practice characteristics, a limited assessment of knowledge about fluoride, or use of fluoride. METHODS: Practicing dental hygienists and dentists in 2005 were asked to fill out a mail questionnaire. Logistic regression models tested the association of independent variables with support for medical providers applying varnish. RESULTS: Response rates were 36% (dental hygienists) and 37% (dentists); median year of graduation was 1988 and 1981. Sixty-six percent of respondents were in solo practices, 82% of dentists in general practice, 5% in dental pediatrics, and 13% were other specialists. While 51.2% of dental professionals agreed that medical practices could apply fluoride varnish, 29% responded "none" should be allowed, and 19% were undecided. In the multivariable logistic regression for support of medical practices applying fluoride versus not supporting it, three practice characteristics and two measures of fluoride use were significant. Provider characteristics and a limited assessment about knowledge about fluoride were not significant. CONCLUSIONS: Half of dental professionals felt that it was appropriate for medical providers to apply fluoride varnish; pediatric dental professionals were less supportive. A few dental practice characteristics were associated with acceptance of the use of fluoride varnish by medical care providers: targeting messages to dental hygienists and those with practices in mixed rural-urban areas may be a useful approach to garner greater support for this medical/dental partnership.

The health-related quality of life of children, adolescents, and young adults with neurofibromatosis type 1 and their families: Analysis of narratives.

PURPOSE: Provide an in-depth description of the health-related quality of life (HRQoL) in youth diagnosed with neurofibromatosis type 1 (NF1) and their families. DESIGN AND METHODS: Data were drawn from qualitative interviews conducted for a larger study aimed at developing the Pediatric Quality of Life Inventory™ (PedsQL™) NF1 module. RESULTS: Youth with NF1 and their families experience a wide range of concerns related to HRQoL due to the varied symptom expression and uncertain trajectory of the disorder. PRACTICE IMPLICATIONS: Pediatric nurses should routinely assess for HRQoL in this population and develop strategies tailored to those concerns that require intervention.

Implementation of continuous capnography is associated with a decreased utilization of blood gases.

BACKGROUND: Capnography provides a continuous, non-invasive monitoring of the CO2 to assess adequacy of ventilation and provide added safety features in mechanically ventilated patients by allowing for quick identification of unplanned extubation. These monitors may allow for decreased utilization of blood gases. The objective was to determine if implementation of continuous capnography monitoring decreases the utilization of blood gases resulting in decreased charges. METHODS: This is a retrospective review of a quality improvement project that compares the utilization of blood gases before and after the implementation of standard continuous capnography. The time period of April 2010 to September 2010 was compared to April 2011 to September 2011. Parameters collected included total number of blood gases analyzed, cost of blood gas analysis, ventilator and patient days. RESULTS: The total number of blood gases after the institution of end tidal CO2 monitoring decreased from 12,937 in 2009 and 13,171 in 2010 to 8,070 in 2011. The average number of blood gases per encounter decreased from 20.8 in 2009 and 21.6 in 2010 to 13.8 post intervention. The blood gases per ventilator day decreased from 4.94 in 2009 and 4.76 in 2010 to 3.30 post intervention. The total charge savings over a 6-month period was $880,496. CONCLUSIONS: Continuous capnography resulted in a significant savings over a 6-month period by decreasing the utilization of blood gas measurements.

Physiologic findings in children previously ventilator dependent at home due to bronchopulmonary dysplasia.

INTRODUCTION: Bronchopulmonary dysplasia (BPD) is the primary respiratory complication of premature birth. Some preterm newborns develop chronic respiratory failure, requiring home ventilator support. While physiologic measures have been described for prematurely born children, little is known about spirometric indices in patients with severe BPD who were previously ventilator dependent at home. METHODS: We retrospectively reviewed medical charts of patients with severe BPD who were ventilator dependent at home. We excluded patients with other comorbidities that could contribute to the severity of BPD. Spirometry was performed when the patient was able to follow commands. RESULTS: Between 1984 and 2012, within our severe BPD cohort who previously required home ventilator support, 19 patients were able to perform reproducible spirometry meeting ATS/ERS acceptability criteria. Ten (52.6%) were females, 13 (68.4%) were Caucasians. Mean age at liberation from ventilation was 2.4 years (C.I. 2.0, 2.9) and at decannulation was 3.5 years (C.I. 2.9, 4.0); median age at first reproducible spirometry measurement was 6.6 years (IQR: 4.9, 8.3). Spirometry results revealed significant airway obstruction, as demonstrated by Z-scores values of -1.5 (C.I. -2.5, -0.4) for FVC, -2.7 (C.I. -3.3, -1.9) for FEV1 , and -3.6 (C.I. -4.3, -2.9) for FEF25-75 . More so, serial spirometric measurements' slopes revealed that the airway obstruction remained static over time (FEV1 slope: -0.07, P-value: 0.2624; FVC slope: -0.01, P-value: 0.9064; and FEF25-75 : 0.0, P-value: 0.8532). CONCLUSIONS: Extreme prematurity associated with severe BPD requiring home ventilator support carries significant risks of morbidity. These patients had substantially diminished respiratory function reflecting airflow abnormalities that remained static over time.

Median Household Income: Association with Mortality in Children on Chronic Ventilation at Home Secondary to Bronchopulmonary Dysplasia.

Objective: The aim of this study was to determine if living in a lower income neighborhood is associated with mortality of patients with bronchopulmonary dysplasia (BPD) on home ventilation. Methods: Patients were divided into two groups by their ZIP code-based annual household income (Z-AHI), their year of birth, and the median state household income. Survival, liberation from ventilation, and decannulation rates were analyzed between the groups. Results: Over 27 years, 94 patients met our inclusion criteria: 58 (61.7%) were in the group with lower Z-AHI, and 36 (38.3%) were in the group with the Z-AHI above the median state household. Of the patients who died, 14/15 were in the lower Z-AHI group (p=0.003). Survival probability at 60 months of age showed no significant difference between the two groups: 81% [95% CI 70.9, 91.1] for the group with the Z-AHI below the median state household, and 100% [95% CI 100.0, 90.3] for the group with higher Z-AHI (p=0.31). Conclusions: The results of this study are descriptive, as the cause of the association between mortality rate and living in an area with lower household income is not yet understood. The difference in mortality rates between groups above and below the median state income suggests a serious health disparity, which warrants further study. Additional understanding of this effect requires more complete and direct measurement of socioeconomic status and individual characteristics, and better understanding of local environmental conditions.

A continuous quality improvement curriculum for residents: addressing core competency, improving systems.

PURPOSE: To describe the development, implementation, and evaluation of a residency continuous quality improvement (CQI) curriculum. METHOD: Forty-four medicine and pediatrics residents participated in a CQI curriculum. Resident-designed projects were scored for CQI construct skills using a grading tool. Pre- and post-tests evaluated knowledge, perceived knowledge, interest, and self-efficacy. RESULTS: Differences between pre- and post-test perceived knowledge and self-efficacy were highly significant (p <.001). The mean project score was 81.7% (SD 8.3%). Higher knowledge was associated with higher ratings of self-efficacy. There was no correlation of measured knowledge with project score or interest. CONCLUSIONS: Resident education and learning in CQI served to produce innovative and creative improvement projects that demonstrated individual residents' competency in practice-based learning and improvement.

Prevalence, attitudes, and behaviors related to the use of nonsteroidal anti-inflammatory drugs (NSAIDs) in student athletes.

PURPOSE: To describe NSAID use among football players and examine differences in attitudes and behaviors associated with reports of daily use. Data are from 681 self-administered questionnaires. Independent variables were race (white), insurance status (private), attitudes (whether NSAIDs helped performance), and behaviors (decided independently vs. asked an adult when to take an NSAID, what type, or dose; use of NSAIDs to treat pain vs. block pain). Among NSAID users, independent variables were entered into a logistic regression to determine their effects on the probability of daily use. Athletes were male, 90% white, mean age 15.8 years, and 88% had private insurance. Four-hundred-fifty-two (75%) used NSAIDs in the past 3 months, and 90 (15%) were daily users with no significant difference in age, race, or insurance between users and nonusers. Those who perceived better performance owing to NSAIDs (adjusted odds ratio [AOR] = 2.4), who decided on their own when to take NSAIDs (AOR = 2.2), and who used NSAIDs prophylactically were more likely to use daily NSAIDs (AOR = 2.5) after controlling for other variables in the model. NSAID use in high school football players is highly prevalent. Although package inserts caution against chronic use, nearly one of seven players uses NSAIDs daily. Daily users were more likely to decide independently and to use NSAID prophylactically, raising issues about the need for adult supervision and education of athletes regarding side effects and appropriate use.

Implementation and sustainability of statewide transition support services for youth with intellectual and physical disabilities.

PURPOSE: Significant gaps in care and limited existing models establish the need to innovate systems of care for youth with special health care needs in the transition between pediatric to adult health care settings. METHODS: Using implementation science, a statewide transition support program was created. University and community partners explored needs and adopted a strategic plan and funding sources. The existing consensus statement provided a framework. A team was hired, policies were piloted and the initial ambulatory consultative transition service for youth with special needs ages 11 to 22 was launched. Full program activities during year four were analyzed. RESULTS: During 2011, there were 139 consultations for youth with intellectual disability and/or physical disability (average 16.74 years, 46% female). Services include routine and focused co-morbidity screening and recommendations, care coordination of complex health and community service needs, and support for families. The evolving transdisciplinary team adapted their methods to collaborate with a growing population of youth and primary care providers. CONCLUSION: A statewide transition support program is a viable delivery model to provide needed resources for youth, families and primary care practices. Weekly improvement meetings continue to adapt services to sustain family satisfaction and community provider satisfaction.

Outcomes of children with severe bronchopulmonary dysplasia who were ventilator dependent at home.

OBJECTIVE: To describe the incidence and outcomes of children with chronic respiratory failure secondary to severe bronchopulmonary dysplasia (BPD) on chronic positive pressure ventilation (PPV) via tracheostomy at home. METHODS: We retrospectively reviewed medical charts of patients with severe BPD who were PPV dependent at home and who were enrolled in a university-affiliated home ventilator program between 1984 and 2010. We excluded patients with other comorbidities that could contribute to the development of chronic respiratory failure. We reported the incidence of these children in Indiana and cumulative incidences of survival, liberation from PPV, and decannulation. RESULTS: Over 27 years, 628 children were cared for in our home ventilator program. Of these, 102 patients met inclusion criteria: 83 (81.4%) were alive and 19 (18.6%) were deceased. Sixty-nine patients (67.6%) were liberated from PPV, and 97.1% of them were weaned before their fifth birthday, with a median age at liberation of 24 months (interquartile range, 19-33). Similarly, 60 patients (58.8%) were decannulated, of which 96.7% completed this process before their sixth birthday, with a median age at decannulation of 37.5 months (interquartile range, 31.5-45). The incidence of children with chronic respiratory failure secondary to BPD who were PPV-dependent at home in Indiana was 1.23 per 100 000 live births in 1984 and increased to 4.77 per 100 000 live births in 2010. CONCLUSIONS: Although extreme prematurity associated with severe BPD necessitating PPV at home carries significant risks of morbidity and mortality, successful liberation from mechanical ventilation and decannulation are likely to occur.