Current Standards and Future Directions of Duchenne Muscular Dystrophy Respiratory Care: The PPMD Italy Meeting Report.

Objective: This report summarizes key discussions from the meeting "Current Standards and Future Directions of Respiratory Assessment and Management of Duchenne Muscular Dystrophy (DMD)," organized by Parent Project Muscular Dystrophy (PPMD) Italy and the United States to address current challenges and opportunities in DMD respiratory care.

Methods: The meeting brought together researchers, clinicians, and patient advocates who shared experiences, discussed advancements in DMD respiratory management, and identified areas of debate that require further research.

Results: The speakers emphasized routine assessment of pulmonary function and of breathing during sleep to achieve timely diagnosis of respiratory complications.

Respiratory Management of the Patient With Duchenne Muscular Dystrophy.

In 2010, Care Considerations for Duchenne Muscular Dystrophy, sponsored by the Centers for Disease Control and Prevention, was published in , and in 2018, these guidelines were updated. Since the publication of the first set of guidelines, survival of individuals with Duchenne muscular dystrophy has increased. With contemporary medical management, survival often extends into the fourth decade of life and beyond.

An Official American Thoracic Society Clinical Practice Guideline: Pediatric Chronic Home Invasive Ventilation.

Background: Children with chronic invasive ventilator dependence living at home are a diverse group of children with special health care needs. Medical oversight, equipment management, and community resources vary widely. There are no clinical practice guidelines available to health care professionals for the safe hospital discharge and home management of these complex children.

Perioperative respiratory management of pediatric patients with neuromuscular disease.

Patients with neuromuscular disorders undergoing general anesthesia present a special set of respiratory problems for perioperative management. While there are disease-specific concerns, there are many common themes in the respiratory management of patients with neuromuscular disorders. These problems are discussed in this review.

The respiratory management of patients with duchenne muscular dystrophy: a DMD care considerations working group specialty article.

In 2001, the Muscular Dystrophy Community Assistance, Research and Education Amendments (MD-CARE Act) was enacted, which directed federal agencies to coordinate the development of treatments and cures for muscular dystrophy. As part of the mandate, the Centers for Disease Control and Prevention (CDC) initiated surveillance and educational activities, which included supporting development of care considerations for Duchenne muscular dystrophy (DMD) utilizing the RAND/UCLA Appropriateness Method (RAM). This document represents the consensus recommendations of the project's 10-member Respiratory Panel and includes advice on necessary equipment, procedures and diagnostics; and a structured approach to the assessment and management of the respiratory complications of DMD via assessment of symptoms of hypoventilation and identification of specific thresholds of forced vital capacity, peak cough flow and maximum expiratory pressure.

Airway clearance modalities in neuromuscular disease.

Airway clearance consists of two linked processes: mucociliary clearance and cough clearance. Patients with neuromuscular weakness are at risk for impaired cough clearance and therefore the development of pneumonia and atelectasis. Aiding airway clearance in the patient with neuromuscular weakness is critical to the maintenance of health and the prevention of significant respiratory morbidity.

A 2009 perspective on the 2004 American Thoracic Society statement, "respiratory care of the patient with Duchenne muscular dystrophy".

This is a summary of the presentation "A 2009 Perspective on the 2004 American Thoracic Society Statement, 'Respiratory Care of the Patient With Duchenne Muscular Dystrophy,'" presented as part of the program on pulmonary management of pediatric patients with neuromuscular disorders at the 30th annual Carrell-Krusen Neuromuscular Symposium on February 20, 2008.

Cystic fibrosis pulmonary guidelines: airway clearance therapies.

Cystic fibrosis (CF) is a genetic disease characterized by dehydration of airway surface liquid and impaired mucociliary clearance. As a result, there is difficulty clearing pathogens from the lung, and patients experience chronic pulmonary infections and inflammation. Clearance of airway secretions has been a primary therapy for those with CF, and a variety of airway clearance therapies (ACTs) have been developed.

American College of Chest Physicians consensus statement on the respiratory and related management of patients with Duchenne muscular dystrophy undergoing anesthesia or sedation.

This statement on the management of patients with Duchenne muscular dystrophy (DMD) undergoing procedural sedation or general anesthesia represents the consensus opinion of a multidisciplinary panel convened under the auspices of the American College of Chest Physicians. Expert recommendations on this subject are needed for several reasons. First, patients with DMD have an increased risk of complications when they undergo sedation or general anesthesia.

Role of IL-17A, IL-17F, and the IL-17 receptor in regulating growth-related oncogene-alpha and granulocyte colony-stimulating factor in bronchial epithelium: implications for airway inflammation in cystic fibrosis.

IL-17R signaling is critical for pulmonary neutrophil recruitment and host defense against Gram-negative bacteria through the coordinated release of G-CSF and CXC chemokine elaboration. In this study, we show that IL-17R is localized to basal airway cells in human lung tissue, and functional IL-17R signaling occurs on the basolateral surface of human bronchial epithelial (HBE) cells. IL-17A and IL-17F were potent inducers of growth-related oncogene-alpha and G-CSF in HBE cells, and significant synergism was observed with TNF-alpha largely due to signaling via TNFRI.

Liver transplantation in children with cystic fibrosis: a long-term longitudinal review of a single center's experience.

Background: Improved long-term survival in cystic fibrosis (CF) has led to an increased incidence of extrapulmonary complications of this disease. Of these, end-stage liver disease is a significant cause of morbidity and mortality with liver transplantation being the only effective therapy.

Methods: Records of all CF pediatric liver transplant recipients were reviewed.

Secretoglobins SCGB3A1 and SCGB3A2 define secretory cell subsets in mouse and human airways.

Clara cell secretory protein (CCSP) is expressed abundantly within the conducting airway epithelium and is thought to have immunoregulatory functions. Differences in the localization of CCSP between mouse and human airways led us to hypothesize that functional homologues of CCSP may compensate for the lack of CCSP expression in proximal airway locations. We previously identified an expressed sequence tag (W82219) whose expression is induced within Clara cells of CCSP knockout mice.