Bronchiolitis therapies and misadventures.

Viral bronchiolitis, which is most commonly caused by an infection with the respiratory syncytial virus (RSV), can lead to respiratory difficulties in young children which may require hospitalization. Despite years of research and medical trials, the mainstay of bronchiolitis treatment remains supportive only. This review provides an overview of the history of different treatments for bronchiolitis, including those that failed, as well as new therapies that are under study. Future studies for the treatment of bronchiolitis should consider different age-groups, important subgroups (i.e., those with a prior history of wheezing, those with a family history of asthma and those with non-RSV viral etiologies) whose response to treatment may differ from that of the composite group.

Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis.

The advent of CFTR modulators, a genomic specific medication, revolutionized the treatment of CF for many patients. However, given that these therapeutics were only developed for specific CFTR mutations, not all people with CF have access to such disease-modifying drugs. Racial and ethnic minority groups are less likely to have CFTR mutations that are approved for CFTR modulators. This exclusion has the potential to widen existing health disparities.

School Nurses' Perspectives on Components of Asthma Programs to Address Pediatric Disparities.

OBJECTIVE: To use a community engaged, collaborative approach with school nurses working within an urban community, to develop recommendations for future school-based interventions targeting pediatric asthma disparities. METHODS: Open-ended survey data were collected from 33 nurses (77% of nurses in the school district) during a face-to-face monthly health services meeting. Questions asked nurses to estimate the proportion of students with asthma with the necessary forms and medications at school and to describe perceived barriers to having such forms and medications, and potential initiatives that could be implemented. A 30-min asthma education class was also piloted with school nurses, who then rated its acceptability and feasibility. Open-ended survey data were analyzed using thematic analysis. RESULTS: Nurses estimated that 12% of students with asthma had an asthma action plan, 19% had a medication release form, and 15% had medications at school (i.e. inhalers). Four themes emerged regarding barriers to asthma management in schools and strategies for promoting asthma management in schools: coordination of care, asthma education, access to care, and medication adherence. Nurses noted the need for education focused specifically on teaching inhaler technique, and better communication between schools, providers, and families. CONCLUSIONS: School nurses provided valuable information regarding specific barriers, as well as approaches to addressing these barriers in a future intervention. Findings suggest that a school-based intervention needs to address coordination among schools, parents, and medical providers, and will be optimally effective if it also addresses structural barriers.

Engaging with the Richmond Community to Reduce Pediatric Asthma Disparities: Findings from a Community-engaged Needs Assessment.

Childhood asthma disparities persist, with children living in low-income areas experiencing worse morbidity. We partnered with a community-academic research team and stakeholders to conduct a needs assessment to understand barriers and supports to asthma treatment. We convened a community advisory board, comprised of parents of children with asthma, youth with asthma, and members of key community organizations. Two focus groups with parents of children with asthma and four focus groups with youth with asthma were conducted, and a survey was administered to 100 parents. A visual mapping process was used to gather qualitative data about barriers, strategies, and outcomes, and allowed advisory board members to interpret focus group and survey data within the lived experiences of families. Focus group themes included parent stress/anxiety, concerns about school nurses, and lack of trust in providers. Findings from focus groups and surveys suggested that emergency department visits were not perceived negatively by families, although health providers and researchers generally view them as such. Public health implications include systemic changes that allow the healthcare system to address families' acute needs and worry. A community program focused on education and coordination among families, schools, and medical homes might improve asthma outcomes at the population level.

Readiness for transition and health-care satisfaction in adolescents with complex medical conditions.

BACKGROUND: The purpose of this study is to examine contributions to patient perceptions of transition readiness and satisfaction with care amongst adolescents and young adults (AYAs) with complex health conditions engaging in paediatric care. METHODS: Participants included 94 patients aged 14-20 years (M = 16.41, SD = 1.56) with cystic fibrosis (n = 31), sickle cell disease (n = 27), and solid organ transplants (n = 36). Participants completed self-report questionnaires and medical providers completed measures of their medication regimen complexity. One-way analysis of variance compared differences between disease groups on study variables. Pearson product-moment correlation coefficients and linear regression models evaluated factors associated with AYA reported transition readiness and satisfaction with health care. RESULTS: There were no significant differences between disease groups on patient-reported transition readiness, barriers to medication adherence, health care self-management, or satisfaction. Patient age, self-reported health-care responsibility, medication barriers, and academic performance predicted a large portion of the variance in AYA perceptions of transition readiness (R2  = 0.27, F (4, 83) = 7.74, p < 0.001, Cohen's f2 = 0.37). Patient gender, self-reported health-care responsibility, and medication barriers predicted a medium portion of the variance in AYA satisfaction with health care (R2  = 0.23, F (3, 88) = 8.56, p < 0.001, Cohen's f2 = 0.30). CONCLUSIONS: Patient perceptions of health care self-management and barriers to medication adherence are important predictors of readiness for transition and satisfaction with care. Considering a holistic approach that includes these factors allows for improved understanding of individual needs for transition interventions that can improve adult outcomes for individuals with complex health conditions.

Identifying and Integrating Parent Priorities for Psychosocial Support Services in a Pediatric Cystic Fibrosis Clinic.

Engaging parents early in the development of psychosocial support programs in cystic fibrosis (CF) clinics may enable services and care team recommendations to be tailored appropriately. This pilot study identified psychosocial priorities of parents of children with CF related to treatment adherence, parent/child mental health, and CF-related communication. Forty parents of children with CF (2 months to 17 years) completed an anonymous 17-item survey during routine clinic visits that assessed priorities related to psychosocial services. Elements of a quality improvement framework were used to develop the survey and determine recommendations based on findings. Parents reported the most interest in support related to improving adherence to respiratory therapies and helping children complete treatments independently. Other priority areas included services that helped children cope with feelings of isolation or abnormality due to CF and strategies to improve communication with the care team. Additionally, the majority of families indicated that they preferred receiving psychosocial services during routine clinic visits, followed by periodic parent workshops. Based on survey results, the psychosocial team at our center developed a survey/response model (e.g., roundtables, workshops) that may serve useful for other CF care teams as they identify the priorities of parents and adapt to their needs.

Bladder Reconstruction Rates Differ among Centers Participating in National Spina Bifida Patient Registry.

PURPOSE: We performed an exploratory analysis of data from the NSBPR (National Spina Bifida Patient Registry) to assess variation in the frequency of bladder reconstruction surgeries among NSBPR centers. MATERIALS AND METHODS: We queried the 2009-2014 NSBPR to identify patients who had ever undergone bladder reconstruction surgeries. We evaluated demographic characteristics, spina bifida type, functional level, mobility and NSBPR center to determine whether any of these factors were associated with reconstructive surgery rates. Multivariable logistic regression was used to simultaneously adjust for the impact of these factors. RESULTS: We identified 5,528 patients with spina bifida enrolled in the NSBPR. Of these patients 1,129 (20.4%) underwent bladder reconstruction (703 augmentation, 382 continent catheterizable channel, 189 bladder outlet procedure). Surgical patients were more likely older, female, nonHispanic white, with a higher lesion level, myelomeningocele diagnosis, nonambulators (all p <0.001) and nonprivately insured (p=0.018). Bladder reconstruction surgery rates varied among NSBPR centers (range 12.1% to 37.9%, p <0.001). After correcting for known confounders NSBPR center, spina bifida type, mobility, gender and age (all p <0.001) were significant predictors of surgical intervention. Race (p=0.19) and insurance status (p=0.11) were not associated with surgical intervention. CONCLUSIONS: There is significant variation in rates of bladder reconstruction surgery among NSBPR centers. In addition to clinical factors such as mobility status, lesion type and lesion level, nonclinical factors such as patient age, gender and treating center are also associated with the likelihood of an individual undergoing bladder reconstruction.

Relationship of Initial Pancreatic Enzyme Replacement Therapy Dose With Weight Gain in Infants With Cystic Fibrosis.

OBJECTIVE: The aim of the study is to test the hypothesis of a positive relationship between initial dose of pancreatic enzyme replacement therapy (PERT) in infants with cystic fibrosis (CF) and optimal weight gain over the first 2 years of life. METHODS: Using the CF Foundation Patient Registry, we identified 502 children born in 2010 and used multivariable models to compare as our primary analysis their 2-year changes in weight-for-age z score (WAZ) and as our secondary analysis weight-for-length percentile (W/L%) by initial PERT dose. We focused on initial dose without reference to subsequent changes in treatment to avoid confounding by indication (severity). RESULTS: Initial PERT dose demonstrated a linear relationship to change in WAZ and W/L% at age 2 years. An initial dose of >1500 lipase units/kg/largest meal resulted in a higher likelihood of attaining WAZ at 2 years at or above the birth WAZ (adjusted odds ratio [aOR] 1.87, 95% confidence interval [CI] 1.22-2.86) and at the top quartile for improvement over 2 years in WAZ (aOR 1.90, 95% CI 1.19-3.05). There was no correlation between initial PERT dose and weight at initial PERT encounter (P = 0.35). Findings were similar for W/L% and when the cohort was restricted to infants who began PERT in the first 3 months of life. CONCLUSIONS: Infants receiving higher initial PERT dose demonstrate better weight-related outcomes, as reflected by attainment of favorable changes in WAZ and W/L%, at age 2 years.

Comparing effectiveness and outcomes in asthma and cystic fibrosis.

As technology yields new treatments, pediatric pulmonologists need determine how best to use them and how to decide which ones are best for any specific group or individual patient. Physicians have always customized therapies based upon patient response, but the new concept of "Personalized (or precision) medicine" focuses attention to a greater degree on the individual needs of patients based on their genetic, biomarker, phenotypic, or psychosocial characteristics. The newly developed biologics for treatment of asthma and CFTR modulators for treatment of cystic fibrosis (CF) highlight this newer approach. As we have more treatments available, new approaches to testing efficacy and effectiveness of these new therapies is necessary in order to efficiently bring them to market and compare their benefits in real world practice. While comparative effectiveness can be tested in pragmatic clinic trials, the most common approaches make use of observational data such as administrative databases and patient registries but their use for this is fraught with pitfalls that may or may not be methodologically surmountable. Once new therapies have been shown to be efficacious and effective, it is important to be cognizant of methods for ensuring that all patients actually receive the treatments that will be best for them. Comparisons of the effectiveness of clinical practice in the form of benchmarking is helpful for this, and consideration of costs and cost-effectiveness is essential to judging the best treatment for patients in a real-world setting.

Design and Methodological Considerations of the Centers for Disease Control and Prevention Urologic and Renal Protocol for the Newborn and Young Child with Spina Bifida.

PURPOSE: Care of children with spina bifida has significantly advanced in the last half century, resulting in gains in longevity and quality of life for affected children and caregivers. Bladder dysfunction is the norm in patients with spina bifida and may result in infection, renal scarring and chronic kidney disease. However, the optimal urological management for spina bifida related bladder dysfunction is unknown. MATERIALS AND METHODS: In 2012 the Centers for Disease Control and Prevention convened a working group composed of pediatric urologists, nephrologists, epidemiologists, methodologists, community advocates and Centers for Disease Control and Prevention personnel to develop a protocol to optimize urological care of children with spina bifida from the newborn period through age 5 years. RESULTS: An iterative quality improvement protocol was selected. In this model participating institutions agree to prospectively treat all newborns with spina bifida using a single consensus based protocol. During the 5-year study period outcomes will be routinely assessed and the protocol adjusted as needed to optimize patient and process outcomes. Primary study outcomes include urinary tract infections, renal scarring, renal function and bladder characteristics. The protocol specifies the timing and use of testing (eg ultrasonography, urodynamics) and interventions (eg intermittent catheterization, prophylactic antibiotics, antimuscarinic medications). Starting in 2014 the Centers for Disease Control and Prevention began funding 9 study sites to implement and evaluate the protocol. CONCLUSIONS: The Centers for Disease Control and Prevention Urologic and Renal Protocol for the Newborn and Young Child with Spina Bifida began accruing patients in 2015. Assessment in the first 5 years will focus on urinary tract infections, renal function, renal scarring and clinical process improvements.

Official American Thoracic Society technical standards: flexible airway endoscopy in children.

BACKGROUND: Flexible airway endoscopy (FAE) is an accepted and frequently performed procedure in the evaluation of children with known or suspected airway and lung parenchymal disorders. However, published technical standards on how to perform FAE in children are lacking. METHODS: The American Thoracic Society (ATS) approved the formation of a multidisciplinary committee to delineate technical standards for performing FAE in children. The committee completed a pragmatic synthesis of the evidence and used the evidence synthesis to answer clinically relevant questions. RESULTS: There is a paucity of randomized controlled trials in pediatric FAE. The committee developed recommendations based predominantly on the collective clinical experience of our committee members highlighting the importance of FAE-specific airway management techniques and anesthesia, establishing suggested competencies for the bronchoscopist in training, and defining areas deserving further investigation. CONCLUSIONS: These ATS-sponsored technical standards describe the equipment, personnel, competencies, and special procedures associated with FAE in children.

The National Spina Bifida Patient Registry: profile of a large cohort of participants from the first 10 clinics.

OBJECTIVE: To use data from the US National Spina Bifida Patient Registry (NSBPR) to describe variations in Contexts of Care, Processes of Care, and Health Outcomes among individuals with spina bifida (SB) receiving care in 10 clinics. STUDY DESIGN: Reported here are baseline cross-sectional data representing the first visit of 2172 participants from 10 specialized, multidisciplinary SB clinics participating in the NSBPR. We used descriptive statistics, the Fisher exact test, χ(2) test, and Wilcoxon rank-sum test to examine the data. RESULTS: The mean age was 10.1 (SD 8.1) years with slightly more female subjects (52.5%). The majority was white (63.4%) and relied upon public insurance (53.5%). One-third had sacral lesions, 44.8% had mid-low lumbar lesions, and 24.9% had high lumbar and thoracic lesions. The most common surgery was ventricular shunt placement (65.7%). The most common bladder-management technique among those with bladder impairment was intermittent catheterization (69.0%). Almost 14% experienced a pressure ulcer in the last year. Of those ages 5 years or older with bowel or bladder impairments, almost 30% were continent of stool; a similar percentage was continent of urine. Most variables were associated with type of SB diagnosis. CONCLUSION: The NSBPR provides a cross section of a predominantly pediatric population of patients followed in specialized SB programs. There were wide variations in the variables studied and major differences in Context of Care, Processes of Care, and Health Outcomes by type of SB. Such wide variation and the differences by type of SB should be considered in future analyses of outcomes.

Early Life Growth Trajectories in Cystic Fibrosis are Associated with Pulmonary Function at Age 6 Years.

OBJECTIVE: To determine whether severity of lung disease at age 6 years is associated with changes in nutritional status before age 6 within individual children with cystic fibrosis (CF). STUDY DESIGN: Children with CF born between 1994 and 2005 and followed in the CF Foundation Patient Registry from age ≤2 through 7 years were assessed according to changes in annualized weight-for-length (WFL) percentiles between ages 0 and 2 years and body mass index (BMI) percentiles between ages 2 and 6 years. The association between growth trajectories before age 6 and forced expiratory volume in 1 second (FEV1)% predicted at age 6-7 years was evaluated using multivariable linear regression. RESULTS: A total of 6805 subjects met inclusion criteria. Children with annualized WFL-BMI always >50th percentile (N = 1323 [19%]) had the highest adjusted mean (95% CI) FEV1 at 6-7 years (101.8 [100.1, 103.5]). FEV1 at 6-7 years for children whose WFL-BMI increased >10 percentile points by age 6 years was 98.3 (96.6, 100.0). This was statistically significantly higher than FEV1 for children whose WFL-BMI was stable (94.4 [92.6, 96.2]) or decreased >10 percentile points (92.9 [91.1, 94.8]). Among children whose WFL-BMI increased >10 percentile points, achieving and maintaining WFL-BMI >50th percentile at younger ages was associated with significantly higher FEV1 at 6-7 years. CONCLUSIONS: Within-patient changes in nutritional status in the first 6 years of life are significantly associated with FEV1 at age 6-7 years. The establishment of a clear relationship between early childhood growth measurements and later lung function suggests that early nutritional interventions may impact on eventual lung health.

Pancreatic enzyme replacement therapy dosing and nutritional outcomes in children with cystic fibrosis.

OBJECTIVES: To utilize the Cystic Fibrosis Foundation Patient Registry to evaluate whether pancreatic enzyme dose is associated with better nutritional status as measured by average body mass index (BMI) percentile. STUDY DESIGN: A retrospective analysis of the Cystic Fibrosis Foundation Patient Registry from 2005-2008 was performed. The final analysis included 42 561 patient visits from 14 482 patients 2-20 years of age taking pancreatic enzyme replacement therapy from 179 programs. Cystic fibrosis care programs were assigned to quartiles based on adjusted mean patient BMI percentiles. Differences in median lipase dose between programs in the highest and lowest BMI quartiles were examined using a mixed effects model that adjusted for individual patient BMI, age, race, ethnicity, forced expiratory volume in 1 second percent, acid-blocker use, presence of Pseudomonas aeruginosa, nutritional supplement use, growth hormone use, and diagnosis of cystic fibrosis-related diabetes. RESULTS: A significant difference in median enzyme dose existed between the highest and lowest BMI quartiles. Multivariable analysis demonstrated the effect persisted after adjustment for covariates. Highest quartile programs had a median enzyme dose of 1755 lipase units/kg/meal compared with 1628 lipase units/kg/meal for lowest quartile programs. CONCLUSION: Patients attending US cystic fibrosis programs achieving highest nutritional outcomes, measured by mean BMI percentile, have higher enzyme dosing than those attending programs at lower performance levels. Further randomized clinical trials are necessary to determine the role of enzyme dose in improving nutritional outcomes.

The impact of switching to race-neutral reference equations on FEV1 percent predicted among people with cystic fibrosis.

RATIONALE: The American Thoracic Society recommended switching to race-neutral spirometry reference equations, as race is a social construct and to avoid normalizing disparities in lung function due to structural racism. Understanding the impact of the race-neutral equations on percent predicted forced expiratory volume in one second (ppFEV1) in people with cystic fibrosis (PwCF) will help prepare patients and providers to interpret pulmonary function test results. OBJECTIVE(S): To quantify the impact of switching from Global Lung Initiative (GLI) 2012 race-specific to GLI 2022 Global race-neutral reference equations on the distribution of ppFEV1 among PwCF of different races. METHODS: Cross-sectional analysis of FEV1 among PwCF ages ≥6 years in the 2021 U.S. Cystic Fibrosis Foundation Patient Registry. We describe the absolute difference in ppFEV1 between the two reference equations by reported race and the effect of age and height on this difference. RESULTS: With the switch to GLI Global, ppFEV1 will increase for White (median increase 4.7, (IQR: 3.1; 6.4)) and Asian (2.6 (IQR: 1.6; 3.7)) individuals and decrease for Black individuals (-7.7, (IQR: -10.9; -5.2)). Other race categories will see minimal changes in median ppFEV1. Individuals with higher baseline ppFEV1 and younger age will see a greater change in ppFEV1 (i.e., a greater improvement among White and Asian individuals and a greater decline among Black individuals). CONCLUSIONS: Switching from GLI 2012 race-specific reference equations to GLI 2022 Global race-neutral equations will result in larger reductions in ppFEV1 among Black individuals with CF than increases among White and Asian people with CF.

An emotional journey: caregiver experiences with gastrostomy tube decision-making for children with cystic fibrosis.

BACKGROUND: Nutritional challenges are common in early CF care and stressful for caregivers of children with CF (cwCF) to navigate. Gastrostomy tube (G-tube) placement can improve weight gain, however the decision to proceed with placement is personalized and preference-sensitive. Little is known about the experiences of caregivers of cwCF and the G-tube decision-making process. OBJECTIVES: The present study used a qualitative approach to explore the perceptions and experiences of caregivers of cwCF with G-tube introductions and recommendations, as well as factors influencing G-tube decision-making. METHODS: Caregivers of cwCF aged ≤ 10 years completed audio-taped, semi-structured interviews describing their experiences with G-tube placement discussions. Interviews were transcribed and two independent researchers coded the transcripts and conducted content and thematic analysis using an inductive approach. RESULTS: Participants included 43 caregivers, 84 % were mothers (36/43). CwCF had a mean age of 4 years (SD=2.6), 84 % were White (36/43), and 60 % reported weights below <50th percentile (26/43). All caregivers knew about G-tubes, 44 % (19/43) were recommended a G-tube and 35 % (15/43) had a G-tube placed. Major findings included descriptions of the stages of G-tube decision-making from a heads up, to the game plan, to making a first difficult decision and finally living with the decision to pursue G-tube placement. CONCLUSION: G-tube decision-making is an emotional and personalized journey for caregivers of cwCF. Efforts to explore the values and priorities of caregivers is imperative to supporting families making difficult decisions in CF care.

Standards for the care of people with cystic fibrosis (CF); Planning for a longer life.

This is the final of four papers updating standards for the care of people with CF. That this paper "Planning a longer life" was considered necessary, highlights how much CF care has progressed over the past decade. Several factors underpin this progress, notably increased numbers of people with CF with access to CFTR modulator therapy. As the landscape for CF changes, so do the hopes and aspirations of people with CF and their families. This paper reflects the need to consider people with CF not as a "problem" to be solved, but as a success, a potential and a voice to be heard. People with CF and the wider CF community have driven this approach, reflecting many of the topics in this paper. This exercise involved wide stakeholder engagement. People with CF are keen to contribute to research priorities and be involved in all stages of research. People with CF want healthcare professionals to respect them as individuals and consider the impact of our actions on the world around us. Navigating life presents challenges to all, but for people with CF these challenges are heightened and complex. In this paper we highlight the concerns and life moments that impact people with CF, and events that the CF team should aim to support, including the challenges around having a family. People with CF and their care teams must embrace the updated standards outlined in these four papers to enjoy the full potential for a healthier life.