Inside the Science of Pediatric Lung Disease

A Special Section
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Speeding Up Science With Patient-Derived Xenografts

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Meeting the Challenge of Clinical Trial Recruitment and Retention

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Featured Researcher: Sarah A. Keim, PhD, MA, MS
 Featured Researcher: Sarah A. Keim, PhD, MA, MS 150 150 Gina Vitale, PTA
By: Gina Vitale, PTA

Epidemiology studies and analyzes the who, what, where, when and why of disease. Applying this approach to studying children’s health is critically important, particularly given all the health challenges today’s children face. Children’s health needs are unique because they are still growing and developing.…

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colorful water color style illustration of lungs
Inside the Science of Pediatric Lung Disease: A Special Section
 Inside the Science of Pediatric Lung Disease: A Special Section 1024 624 Abbie Miller
By: Abbie Miller

A NOTE FROM THE EDITOR Our breath connects us with the moment, grounds us and has the power to calm our minds and bodies. But for millions of children, an easy breath is not guaranteed. Lung disease accounts for about a third of admissions…

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Two smiling young children sit side by side indoors, facing the camera, with a colorful blurred artwork in the background.
Beyond Modulators: Ensuring All Patients With Cystic Fibrosis Benefit From the Next Wave of Therapy
 Beyond Modulators: Ensuring All Patients With Cystic Fibrosis Benefit From the Next Wave of Therapy 1024 618 Lauren Dembeck
By: Lauren Dembeck

Before CFTR modulators transformed care, cystic fibrosis (CF) was defined by relentless daily treatment and progressive lung disease. Children grew up with thick airway secretions, chronic cough, recurrent pulmonary infections, and frequent hospitalizations. Maintaining weight was a constant struggle, and lung function typically declined…

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Blue-toned illustration showing lungs beside silhouettes of people across the lifespan, from a crawling baby and toddler to a child, adult, and older adult with a cane.
From Bronchopulmonary Dysplasia to Chronic Obstructive Pulmonary Disease: A Developmental Continuum
 From Bronchopulmonary Dysplasia to Chronic Obstructive Pulmonary Disease: A Developmental Continuum 1024 774 Yan Hu, PhD
By: Yan Hu, PhD

A Prolonged Window of Vulnerability Lung development is a highly orchestrated process that begins early in gestation and continues well into postnatal life. Following airway branching during the embryonic and pseudoglandular stages, distal lung maturation, including small airway and alveolar formation, extends through infancy…

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A close-up medical illustration of blood vessels showing normal round red blood cells flowing through the vessel, with a cluster of rigid, crescent-shaped sickle cells causing a blockage. White blood cells are also visible against the dark background.
Sickle Cell Lung Disease: Opportunities to Advance Care and Research
 Sickle Cell Lung Disease: Opportunities to Advance Care and Research 1024 575 Pam Georgiana
By: Pam Georgiana

A series of studies aims to close gaps by integrating immunology, microbiology and environmental health into clinical research.  For children with sickle cell disease, lung complications remain among the most serious and least predictable drivers of morbidity and mortality. Despite advances in supportive care…

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Newborn with bronchopulmonary dysplasia sleeping in a NICU crib, wrapped in a hospital blanket and receiving oxygen through a nasal cannula.
Bronchopulmonary Dysplasia Research: Innovating on All Fronts
 Bronchopulmonary Dysplasia Research: Innovating on All Fronts 1024 509 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

From understanding the disease at a molecular level to improving strategies for oxygen support after discharge, researchers are tackling bronchopulmonary dysplasia from all angles. The doctors in the Neonatal Intensive Care Unit at Nationwide Children’s Hospital care for more babies with bronchopulmonary dysplasia (BPD)…

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Microscopy image of a mouse heart sample colored with red and green fluorescent dye, and marked for single-cell sampling.
How Diabetes Rewires the Heart’s Smallest Vessels
 How Diabetes Rewires the Heart’s Smallest Vessels 1024 597 Lauren Dembeck
By: Lauren Dembeck

A landmark study uncovers how diabetes alters the heart at the cellular level. Cardiovascular disease is the leading cause of death in type 2 diabetes. While its effects on large arteries are well recognized, growing evidence suggests the earliest cardiovascular injury occurs in the…

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New Health Challenges in the Post-CFTR Modulator Era
 New Health Challenges in the Post-CFTR Modulator Era 1024 576 Lauren Dembeck
By: Lauren Dembeck

The remarkable progress in CF care has also revealed new challenges. As individuals with CF live longer, clinicians are seeing higher frequencies of complications and other down- stream health concerns that rarely emerged in the past and are now focused on aging-related diseases and…

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Newborn baby in an NICU bed with a nasogastric tube.
Precision Medicine Brings Clarity to Preterm Infant Reflux
 Precision Medicine Brings Clarity to Preterm Infant Reflux 1024 492 Alaina Doklovic
By: Alaina Doklovic

Sudarshan Jadcherla, MD, and team aim to distinguish normal reflux from GERD in preterm infants. Gastroesophageal reflux (GER) is common in preterm infants regardless of whether they are being fed formula or breast milk, often presenting as irritability, coughing or feeding difficulties. These symptoms…

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simple cartoon illustration of lungs
Decoding Pediatric Asthma: From Cells to Care
 Decoding Pediatric Asthma: From Cells to Care 1024 448 Madison Storm
By: Madison Storm

How clinician-scientist teams are mapping the molecular drivers of wheezing and asthma to guide smarter, faster and more personalized treatment. Asthma is one of the most common reasons children visit the Emergency Department (ED) at Nationwide Children’s Hospital. While frontline clinicians work quickly to…

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Upset confused Black woman holding cellphone having problem with mobile phone, frustrated angry mixed race girl reading bad news in message looking at smartphone annoyed by spam or missed call
Effectiveness and Adoption of a Mental Health Crisis App
 Effectiveness and Adoption of a Mental Health Crisis App 1024 683 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

Youth who used a statewide mental health crisis app in Utah reported significant declines in the intensity of their presenting concerns and were generally satisfied with the app. Youth who used SafeUT, a statewide crisis app in Utah, reported experiencing significant decreases in the…

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microscopy image of podocytes, a type of cell found in the kidney, stained with blue and fluorescent yellow. small red dots mark the cell
Lowering Thrombin Levels Offers a Double Benefit in Glomerular Disease Model
 Lowering Thrombin Levels Offers a Double Benefit in Glomerular Disease Model 1024 410 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Preclinical studies suggest that reducing levels of the clotting enzyme may simultaneously reduce kidney damage and lower risk of blood clots. Preclinical research in an in vivo model now confirms what Bryce Kerlin, MD, and his team first proposed in a publication 8 years…

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Illustration of research and healthcare symbols, including gears, a clipboard with a checklist and medical cross, a magnifying glass, a lab flask, and green icons with a check mark, an X, and a question mark.
Meeting the Challenge of Clinical Trial Recruitment and Retention
 Meeting the Challenge of Clinical Trial Recruitment and Retention 1024 483 Lynn Dosky
By: Lynn Dosky

Participant recruitment and retention strategies are crucial features of any successful clinical study design. In fact, an article published in the Journal of Clinical and Translational Science noted, “up to 85% of clinical trials fail to recruit or retain a sufficient sample size, leading…

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cropped microscopy image of lungs tissue with tumors
Speeding Up Science With Patient-Derived Xenografts
 Speeding Up Science With Patient-Derived Xenografts 1024 483 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

A perfect storm of advanced technologies and scientific collaboration opens doors to rapid progress in pediatric oncology research. The world of pediatric cancer research faces the challenges of small patient numbers, increased ethical considerations, limited funding and poorly classified diseases. In some ways, these…

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Ashley Fernandes, MD, PhD, wearing a white coat with a Nationwide Children's logo, standing in front of a blurred hospital interior background
Professionalism in Pediatric Medicine: Why We Must Get It Right
 Professionalism in Pediatric Medicine: Why We Must Get It Right 1024 580 Ashley Fernandes, MD, PhD
By: Ashley Fernandes, MD, PhD

A talented junior attending physician on a high acuity service is impatient on rounds and visibly irritated by nursing concerns, making trainees and team members uncomfortable and anxious about speaking up. A senior medical researcher pushes the limits on deadlines for important grants and…

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abstract art of magnifying glass over DNA strand
Genetic Evaluation Practices for Neonates With Congenital Heart Disease
 Genetic Evaluation Practices for Neonates With Congenital Heart Disease 1024 614 Pam Georgiana
By: Pam Georgiana

Multi-institutional survey reveals wide variation in cardiac testing and care. Advances in genomic medicine have significantly improved the understanding of congenital heart disease etiology. However, the integration of genetic evaluation into neonatal cardiac critical care remains inconsistent. A recent multi-institutional study, led by Amee…

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Neurosurgeon Albert Isaacs posed in the operating room, looking at the camera.
Early Temporization Linked to Better Early Development in Premature Infants With Posthemorrhagic Hydrocephalus
 Early Temporization Linked to Better Early Development in Premature Infants With Posthemorrhagic Hydrocephalus 864 576 Nationwide Children's
By: Nationwide Children's

A new HCRN study led by Nationwide Children’s links early temporary cerebral spinal fluid diversion and timely shunt conversion to better early development in preterm infants with posthemorrhagic hydrocephalus. A new Hydrocephalus Clinical Research Network (HCRN) study, published in Journal of Neurosurgery: Pediatrics, suggests…

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Finding the Unexpected: Pulmonary Sclerosing Pneumocytoma in a Pediatric Patient
 Finding the Unexpected: Pulmonary Sclerosing Pneumocytoma in a Pediatric Patient 1024 575 Pam Georgiana
By: Pam Georgiana

A case study demonstrates the need for a structured evaluation and consideration of genetic risk factors in rare pediatric tumors. Primary lung tumors in children are rare and present diagnostic uncertainty. A recent case study published in Pediatric Pulmonology highlights the evaluation and management…

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Genome Database Analysis Suggests Cystic Fibrosis is More Globally Distributed Than Previously Believed
 Genome Database Analysis Suggests Cystic Fibrosis is More Globally Distributed Than Previously Believed 1024 768 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Despite lower CF incidence in non-European populations, total CF births in Asia, Africa and South America likely equal or surpass those in North America and Europe. Cystic fibrosis (CF) has long been viewed as a disease that primarily affects people of European descent. Even…

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In Sight: Targeted Muscle Reinnervation
 In Sight: Targeted Muscle Reinnervation 150 150 Abbie Miller
By: Abbie Miller

Amputation in pediatrics is often a result of traumatic injury or cancers, such as osteosarcoma. At Nationwide Children’s, Thomas Scharschmidt, MD, director of the Orthopedic Oncology Program, is a leader in limb salvage surgery and targeted muscle reinnervation.  Targeted muscle reinnervation (TMR) is a…

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