Proof of Concept Study: The Process to New Device Development

Affecting approximately 75,000 individuals across North America, Europe, and Australia, cystic fibrosis (CF) has been described as the most common inherited lethal disease in Caucasians. Over the past few decades, there have been significant advances in screening, early diagnosis, and treatment options. Children born today are expected to have improved life expectancy with projected life spans beyond 50 years of age. Intense interest in the clinical and research communities to identify strategies to correct the underlying defect in CF has led to new research into small molecule CFTR modulator therapy. In December 2015, Pediatric Allergy, Immunology, and Pulmonology dedicated a special issue to Advances and Controversies in Cystic Fibrosis. Drs. Trimble and Donaldson reviewed current and upcoming CFTR modulators. ¹ I would recommend this special issue to those who may not have had the opportunity to review it in its entirety.

Despite these advances, children with CF are still challenged by chronic bacterial pulmonary infections and the ongoing need for proactive respiratory management. Chronic colonization with Pseudomonas aeruginosa, a common pathogen in CF, has been associated with pulmonary morbidity and accelerated decline in lung function. This has led to the Cystic Fibrosis Foundation's recommendation for the chronic use of aerosolized tobramycin in children with CF (>6 years)—not only in children with moderate to severe disease and positive P. aeruginosa sputum cultures but also in those with mild pulmonary disease to decrease exacerbation risk. A noninvasive, easy-to-use technique to monitor infection, mucoid status, and response to treatment would be a valuable adjunct to management and clinical care. In this issue, Dr. Raissy et al. explore the hypothesis that ¹³C-urea can be safely inhaled and used to detect urease-producing bacteria from a breath exhalate of patients with CF. ² Their study was an U.S. Food and Drug Administration (FDA)-approved, prospective, two-part, open-label, single-center, single-arm, single-administration, dose-escalation investigational device exemption trial. It is hoped that the results of this study will help to establish a basis for future studies to use inhaled ¹³C-urea as a diagnostic tool to monitor bacterial load in children with CF.

Chronic rhinosinusitis (CRS) in children has also been a topic of great interest to physicians caring for children and one that Dr. Hopp et al. from the Children's Hospital and Medical Center in Omaha, Nebraska, have addressed in this issue in their review of CRS in children. ³ In this review, the authors explore areas of ongoing speculation—including the microbiome of pediatric CRS, the best use of standard imaging, alternatives in antibiotic treatment, and treatment of refractory CRS.

The prevalence of food allergies has increased significantly. They impact not only on the affected children but also on those who are charged with their care. Are they adequately prepared? Who is responsible for their training should anaphylaxis occur? What can be done to support parents? White et al. report on the EPIPEN4SCHOOLS pilot survey looking at anaphylaxis triggers and treatments by grade level and staff training. ⁴ In a series of 757 events of anaphylaxis experienced by students, almost half occurred in the high school population. Their results showed that majority of schools (54.2%) restricted emergency administration of epinephrine to the school nurse and select staff. Within this same survey, only 36% of schools reported providing training in the recognition of anaphylaxis. Based on these findings, the authors concluded that an increased anaphylaxis risk for teens and the possibility of unrecognized anaphylaxis in the school setting were a distinct concern, requiring further research. Ongoing and expanded recognition and management training for students, staff, and visitors was recommended.

Williams and Hankey's article in this issue focuses on sociobehavioral issues associated with food allergy. ⁵ Their study evaluated the impact of social support and parenting self-efficacy on food allergy-related parenting behaviors of parents with food-allergic children of ages 5–11 years. The authors recommend that professionals working with families of children with food allergies evaluate parent perceptions of available social support and guide those with low levels of support to build a positive social support network.

In this issue as well as in previous issues, we have chosen to include articles on strategies to improve health education, health literacy, and patient–provider communication. We know that reading literacy and health literacy are distinct entities and are both important factors in developing patient education materials. Effective communication of medications, doses, and what to do in an emergency are key elements in an asthma action plan. In our last issue, Dr. John Kelso asked the question “Do written asthma action plans improve outcomes?” ⁶ Are written asthma action plans a substitute for ongoing discussion and education? This topic is further explored in this issue by Mitchell and colleagues. ⁷ The authors acknowledge that a barrier to the use of asthma action plans is based on a discrepancy between the readability of asthma action plans and parental level of health literacy. In their study, they evaluated the feasibility of using picture-based asthma medication plans in urban pediatric outpatient clinics to facilitate and reinforce both the discussion and education.

Pediatric Allergy, Immunology, and Pulmonology strives to provide a blend of translational and clinical research to engage readers and provide practical strategies and information to support them in their care of children. We welcome feedback on topics readers found useful and suggestions for future reviews.

I am pleased to announce that our special issue this year will focus on atopic dermatitis. Dr. Norito Katoh, MD, PhD, from the Kyoto Prefectual University of Medicine in Japan has kindly agreed to serve as guest editor for this issue.