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Cystic Fibrosis

Featured Researcher – Sriram Vaidyanathan, PhD
 Featured Researcher – Sriram Vaidyanathan, PhD 150 150 Gina Vitale, PTA
By: Gina Vitale, PTA

Sriram Vaidyanathan, PhD, is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and an assistant professor of Pediatrics at The Ohio State University College of Medicine. Dr. Vaidyanathan’s path pivoted during his education, as it does for many researchers. His biomedical…

Real-Life Impact of ETI Therapy in Cystic Fibrosis: Insights From a Large Treatment Center
 Real-Life Impact of ETI Therapy in Cystic Fibrosis: Insights From a Large Treatment Center 706 470 Pam Georgiana
By: Pam Georgiana

Greater clinical gains observed in patients with severe lung disease, regardless of genotype. A recent prospective observational study led by Shahid I. Sheikh, MD, pediatric pulmonologist at Nationwide Children’s Hospital and professor of Pediatrics at The Ohio State University College of Medicine, offers insights into the effectiveness of a new medication for adolescents and adults…

Pediatric Restless Legs Syndrome: More Common (and Impactful) Than You Might Think
 Pediatric Restless Legs Syndrome: More Common (and Impactful) Than You Might Think 1024 683 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

This underdiagnosed condition is more than just a bedtime annoyance — it may reflect nutrient deficiencies and have major impacts on daytime behavior. Restless legs syndrome (RLS) affects 2-4% of school-aged children, with symptoms that can significantly impact sleep quality, family relationships and daytime behavior. The condition causes discomfort in the arms or legs during…

Cystic Fibrosis: Screening, Diagnosis and Therapy
 Cystic Fibrosis: Screening, Diagnosis and Therapy 150 150 Pam Georgiana
By: Pam Georgiana

Since the implementation of universal newborn screening for cystic fibrosis in all states by 2010, the expectation has been that every child with cystic fibrosis would be identified early and benefit from life-extending treatments. The reality, however, is more complex. “Because of the complexities of the genetic causes of cystic fibrosis and significant variations in…

Evaluating the Mental Health Impact of a Promising New Treatment for Cystic Fibrosis
 Evaluating the Mental Health Impact of a Promising New Treatment for Cystic Fibrosis 706 470 Pam Georgiana
By: Pam Georgiana

Insights from Nationwide Children’s study on how Trikafta affects children with cystic fibrosis. In response to the promising clinical benefits of elexacaftor/tezacaftor/ivacaftor (Trikafta) in adults with cystic fibrosis, a team in Nationwide Children’s Hospital Cystic Fibrosis Clinic recently investigated the effects of the drug in pediatric patients. Kimberly Pasley, PsyD, mental health coordinator in the…

Establishing Meaningful Clinical Outcome Measures for Gastrointestinal Symptoms in Cystic Fibrosis
 Establishing Meaningful Clinical Outcome Measures for Gastrointestinal Symptoms in Cystic Fibrosis 1024 683 Lauren Dembeck
By: Lauren Dembeck

Understanding the minimal clinically important differences of PROMs is crucial in interpreting results in clinical trials. Patient-reported outcome measures (PROMs) are a set of standardized questions that directly assess the patient’s perspective of their symptoms and quality of life to better understand their experience with a disease or treatment. PROMs support patient-centered care and are…

Modulator Therapy Improves Chronic Sinus Disease in Individuals with Cystic Fibrosis
 Modulator Therapy Improves Chronic Sinus Disease in Individuals with Cystic Fibrosis 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

Following 1 year of therapy, patients showed improved sinus CT metrics and clinical outcomes. Over the last decade, the development of cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs has revolutionized the treatment of cystic fibrosis (CF). The most widely used modulator is the triple combination elexacaftor-tezacaftor-ivacaftor (ETI), which is approved for people with CF…

Modulator Therapy Improves Lives of Patients With Cystic Fibrosis With Severe Lung Disease
 Modulator Therapy Improves Lives of Patients With Cystic Fibrosis With Severe Lung Disease 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Patients receiving ETI on a compassionate use basis experienced substantial clinical improvement. The development of cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs, most recently elexacaftor/tezacaftor/ivacaftor (ETI), has transformed the treatment of patients with cystic fibrosis. A new study from researchers at Nationwide Children’s Hospital shows that ETI therapy has the potential to change even…

Expanding Use of Trikafta for Cystic Fibrosis to Patients as Young as 2 Years Old
 Expanding Use of Trikafta for Cystic Fibrosis to Patients as Young as 2 Years Old 1024 683 Karen McCoy, MD
By: Karen McCoy, MD
Smiling boy outside

When I first started my pulmonary training, the average life expectancy in the United States was about 70 years, and the average life expectancy for someone with cystic fibrosis (CF) was 14 years! Intense study and pursuit of consistent care across the country yielded improvements. By 2010, with standards of treatment very consistent and implementation…

Addressing the Clinical Challenges of Caring for Children with Cystic Fibrosis and Autism Spectrum Disorder
 Addressing the Clinical Challenges of Caring for Children with Cystic Fibrosis and Autism Spectrum Disorder 150 150 JoAnna Pendergrass, DVM
By: JoAnna Pendergrass, DVM

A recent study sheds light on the unique clinical challenges faced by children with cystic fibrosis and autism spectrum disorder and offers potential solutions to these challenges.   Cystic fibrosis (CF) and autism spectrum disorder (ASD) pose significant clinical and emotional burdens to patients and their families. A concurrent CF-ASD diagnosis presents even greater challenges, but…

Re-evaluating GI Medication Regimens in People With Cystic Fibrosis
 Re-evaluating GI Medication Regimens in People With Cystic Fibrosis 1024 681 Emily Siebenmorgen
By: Emily Siebenmorgen

After widespread adoption of highly effective modulator therapy (HEMT), a new study evaluated the GI medication regimens of people with cystic fibrosis (CF). The study, published in Pediatric Pulmonolgy, found patient-initiated decreases in dosing and withdrawal from common GI medications is already occurring, yet there is no evidence to support this practice. Highly effective modulator…

CFTR Modulation Therapy Improves Markers of Inflammation and Lung Function in Cystic Fibrosis
 CFTR Modulation Therapy Improves Markers of Inflammation and Lung Function in Cystic Fibrosis 1024 575 Jessica Nye, PhD
By: Jessica Nye, PhD
Illustration of lungs on blue silhouette of upper chest on black background

Six months of elexacaftor-tezacaftor-ivacaftor (ETI) therapy significantly reduced proinflammatory cytokines, increased immune cell composition, and improved clinical outcomes in cystic fibrosis (CF).   “Cystic fibrosis is a progressive lung disease that carries significant morbidity,” says Shahid Sheikh, MD, of the Pulmonary Medicine and Allergy/Immunology section at Nationwide Children’s Hospital and a professor at The Ohio…

The Importance of Body Composition in Cystic Fibrosis
 The Importance of Body Composition in Cystic Fibrosis 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Lean mass deficits are common in pediatric patients with cystic fibrosis and associated with impaired lung and bone health. Now, experts are calling for increased use of body composition measurements in clinical care.  In cystic fibrosis — a multisystem disorder characterized by progressive lung disease, pancreatic insufficiency, malabsorption and malnutrition — nutrition status is correlated with…

After Lung Transplantation for Cystic Fibrosis, Migration of Stem Cells May Lead to Chimeric Phenotype
 After Lung Transplantation for Cystic Fibrosis, Migration of Stem Cells May Lead to Chimeric Phenotype 1024 575 Jessica Nye, PhD
By: Jessica Nye, PhD
Illustration of lungs on blue silhouette of upper chest on black background

Lung transplantation (LTx) for cystic fibrosis (CF) may trigger bidirectional, long-distance migration of tissue specific stem cells (TSC), causing a chimeric phenotype which could have implications for host defense capabilities. CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene and is characterized by progressive chronic obstructive lung disease which can lead…

Measuring the Progression of CF in School-Aged Children
 Measuring the Progression of CF in School-Aged Children 1024 575 Abbie Miller
By: Abbie Miller
Illustration of lungs on blue silhouette of upper chest on black background

A multicenter study utilized chest computed tomography and lung function tests taken repeatedly over two years in school-aged children with mild cystic fibrosis. Understanding disease progression in chronic illnesses is important to managing patients and improving outcomes. For children with cystic fibrosis, it can be difficult to predict how long it will take for negative…

Exploring the Link Between Cystic Fibrosis and Celiac Disease
 Exploring the Link Between Cystic Fibrosis and Celiac Disease 1024 575 Lauren Dembeck
By: Lauren Dembeck

Overlapping symptoms can cause delayed diagnosis of celiac disease in patients with cystic fibrosis. Physician scientists at Nationwide Children’s Hospital have recently identified three cases of patients with cystic fibrosis, all of whom had the same genotype, were treated with the CFTR modulator, ivacaftor, and were diagnosed with celiac disease. This adds to a novel body…

A Major Milestone in Cystic Fibrosis Treatment
 A Major Milestone in Cystic Fibrosis Treatment 1024 575 Mary Bates, PhD
By: Mary Bates, PhD
Illustration of lungs on blue silhouette of upper chest on black background

A triple medication combination could be life-changing for the majority of patients with cystic fibrosis. A Phase III clinical trial shows that elexacaftor added to ivacaftor and tezacaftor improves lung function and quality of life in cystic fibrosis patients with the most common genetic mutation, F508del. The triple therapy, known as Trikafta, could effectively treat 90%…

Gene Expression Changes With CFTR Modulator Treatment
 Gene Expression Changes With CFTR Modulator Treatment 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Patients with cystic fibrosis show transcriptomic changes after starting treatment with lumacaftor/ivacaftor. In a new study, researchers from Nationwide Children’s analyzed the gene expression profiles of cystic fibrosis patients before and after treatment with the drug lumacaftor/ivacaftor. The findings have implications for the evaluation of existing medications as well as the development of new treatments. Care…

Do All Cystic Fibrosis Modulators Improve Killing of Bacteria?
 Do All Cystic Fibrosis Modulators Improve Killing of Bacteria? 150 150 Lauren Dembeck
By: Lauren Dembeck

Could Earlier Spirometry Get CF Inpatients Home Sooner?
 Could Earlier Spirometry Get CF Inpatients Home Sooner? 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

A retrospective chart review suggests early spirometry is associated with shorter length of stay. As multicenter studies examine whether length of hospital stay and duration of treatment can be safely shortened for cystic fibrosis-related pulmonary exacerbations, physician-researchers at Nationwide Children’s Hospital have been exploring another unresolved angle related to these admissions: when to do pulmonary…

Sweat Chloride Should Be the First-Line CF Test, but Often Isn’t
 Sweat Chloride Should Be the First-Line CF Test, but Often Isn’t 150 150 Jeb Phillips
By: Jeb Phillips

A study finds that CFTR mutation analysis is being ordered before (or without) sweat chloride in some cases, potentially leading to missed diagnoses, increased patient anxiety and unnecessary costs. The Cystic Fibrosis Foundation is clear in its diagnostic guidelines: sweat chloride needs to be the first test ordered after a positive newborn screening for cystic…

Boosting Research With a Down Syndrome Biobank
 Boosting Research With a Down Syndrome Biobank 150 150 Abbie Miller
By: Abbie Miller

A new biobank for Down syndrome blood samples will enable clinical and translational researchers everywhere to shed light on conditions related to Down syndrome, including Alzheimer’s disease. Certain conditions such as congenital heart disease, childhood leukemia and epilepsy are more common in patients with Down syndrome than in the general population. Other conditions, including solid…

New Drug Advances Cystic Fibrosis Care
 New Drug Advances Cystic Fibrosis Care 150 150 Kevin Mayhood
By: Kevin Mayhood

Orkambi helps reduce infections and slows loss of lung function in some patients. Matt Hennessey knew he was on the experimental medicine and not a placebo just days after he began participating in a drug trial. He felt better. The addition of Orkambi pills to his daily routine was just another part of his maintenance…