The US national registry for childhood interstitial and diffuse lung disease: Report of study design and initial enrollment cohort.

INTRODUCTION: Childhood interstitial and diffuse lung disease (chILD) encompasses a broad spectrum of rare disorders. The Children's Interstitial and Diffuse Lung Disease Research Network (chILDRN) established a prospective registry to advance knowledge regarding etiology, phenotype, natural history, and management of these disorders. METHODS: This longitudinal, observational, multicenter registry utilizes single-IRB reliance agreements, with participation from 25 chILDRN centers across the U.S. Clinical data are collected and managed using the Research Electronic Data Capture (REDCap) electronic data platform. RESULTS: We report the study design and selected elements of the initial Registry enrollment cohort, which includes 683 subjects with a broad range of chILD diagnoses. The most common diagnosis reported was neuroendocrine cell hyperplasia of infancy, with 155 (23%) subjects. Components of underlying disease biology were identified by enrolling sites, with cohorts of interstitial fibrosis, immune dysregulation, and airway disease being most commonly reported. Prominent morbidities affecting enrolled children included home supplemental oxygen use (63%) and failure to thrive (46%). CONCLUSION: This Registry is the largest longitudinal chILD cohort in the United States to date, providing a powerful framework for collaborating centers committed to improving the understanding and treatment of these rare disorders.

Retrospective analysis of inpatient polysomnogram characteristics and discharge outcomes in infants with bronchopulmonary dysplasia requiring home oxygen therapy.

RATIONALE: Little is known about the polysomnogram (PSG) characteristics in infants with bronchopulmonary dysplasia (BPD), especially severe BPD, who do not need home ventilatory support but are at increased risk for chronic hypoxia and are vulnerable to its effects. OBJECTIVE: This study aims to assess PSG characteristics and change in discharge outcomes in premature infants with BPD who required oxygen therapy at discharge. METHODS: This is a retrospective chart review of premature infants with BPD who were admitted to a quaternary newborn and infant intensive care unit from January 1, 2012 to December 31, 2015 and who underwent polysomnography before discharge. MEASUREMENTS AND MAIN RESULTS: Data from 127 patients were analyzed. The median gestational age of our patients was 26 weeks and 1 day (interquartile range [IQR]: 24.71, 28.86). The majority of the patients had moderate-to-severe BPD. The median obstructive apnea-hypopnea index was 5.3 events/h (IQR: 2.2, 10.1). The median oxygen desaturation index was 15.7 events/h (IQR: 4.7, 35). Nadir oxygen saturation measured by pulse oximeter was 81% (IQR: 76-86) and the arousal/awakening index was 21.9 (IQR: 13.3-30.9). No statistically significant difference was noted between severe and nonsevere BPD groups for PSG characteristics. However, average end-tidal CO2 was significantly higher in the severe BPD group (p = .0438). Infants in the severe BPD group were intubated longer than infants with nonsevere BPD (p = .0082). The corrected gestational age (CGA) at the time of discharge (CGA-PSG) and PSG (CGA-DC) was higher in severe BPD patients but not statistically different. The majority of premature infants who underwent a PSG were discharged home with oxygen, and 69% required a titration of their level of support based on results from the PSG. CONCLUSION: Our results highlight the presence of abnormal PSG characteristics in BPD patients, as early as 43 weeks CGA. These findings have not been previously described in this patient population prior to initial discharge from the hospital. A severe BPD phenotype tends to be associated with higher respiratory morbidity compared with a nonsevere BPD phenotype for the comparable CGA. PSG, when available, may be helpful for individualizing and streamlining treatment in preparation for discharge home and mitigating the effects of intermittent hypoxic episodes.

Actions Speak Louder Than Words: The U.N. Convention on the Rights of the Child and U.S. Pediatric Bioethicists.

In exploring the relationship between "child rights" and "pediatric bioethics" and how these disciplines might provide mutual support in advancing the health and wellness of children around the world, our article responds to the questions of whether the U.N. Convention on the Rights of the Child (CRC) could be of any benefit in the United States, the only country that has not yet ratified this international treaty, and whether the CRC has any value for addressing clinical pediatric bioethics' questions. We describe the considerable influence that the United States had in developing significant components of the CRC, and we argue that the CRC may be useful for U.S. pediatric bioethicists as a tool to advance children's health policy. We note that ratification of the CRC does not equate with compliance or success. Lastly, we identify a distinction between the use of the term "best interests" by child rights advocates and its use by clinical pediatric bioethicists.

When Rights Just Won't Do: Ethical Considerations When Making Decisions for Severely Disabled Newborns.

Children born with severe handicapping conditions, where survival and quality of survival is indeterminate, present special challenges for families and health-care professionals tasked with deciding the best courses of treatment and care. The case of Baby G presents an opportunity to compare the relative effectiveness of ethical versus rights theories in providing guidance about what obligations are owed to such children at bedside and how those obligations pertain to broader societal duties in a rights framework. We review common theories of determining the "best interests standard" of newborn decision-making and the priority of families to decide on behalf of their children. We then discuss what support the rights framework of the U.N. Convention on the Rights of the Child (CRC) might lend to the best implementation of clinical ethics decision-making. Finally, we conclude that the universal nature of rights theory does not provide the particular, specific guidance needed at the bedside of the critically ill infant.

The Potential Value of the U.N. Convention on the Rights of the Child in Pediatric Bioethics Settings.

This article provides support for the use of a particular international human rights law document, the U.N. Convention on the Rights of the Child (CRC), in contemporary pediatric bioethics practice without relying on the legally binding force of the document. It first demonstrates that the CRC's core commitments and values substantially overlap with the core commitments and values of mainstream bioethics and with the laws of many domestic jurisdictions where mainstream bioethics are currently practiced. It then explores some implications of this overlap. For instance, the substantial international human rights law scholarship on how to understand these commitments and values can be helpful in suggesting ways to operationalize them in domestic bioethics practice and can offer insightful, internationally generated ethical perspectives that may not have been considered. The article also argues that the CRC can help health-care organizations develop policies consistent with the best interests of children and that the CRC can serve as a common language of values for transnational health-care collaborations. However, as a final case discussion demonstrates, whatever the merits of the CRC, one may face practical difficulties in trying to use it.

Her own decision: impairment and authenticity in adolescence.

This case describes an adolescent in a crisis of a chronic medical condition whose situation is complicated by substance abuse and mental illness. D. Micah Hester provides an analytic approach, teasing apart the multiple layers of medical, developmental, and moral issues at hand and describing possible responses and outcomes. Amy T. Campbell examines existing legal guidelines for adolescent decision making, arguing that greater space exists for clinical discretion in these matters than commonly thought. Cheryl D. Lew discusses the development of agency in adolescent patients, the ideal of autonomous decision making in the context of impairment and chronic illness, and the obligation of healthcare teams to examine an adolescent patient's decisions in relation to her identity.