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Expert Review of Respiratory Medicine 2023Bronchopulmonary dysplasia (BPD) is a chronic respiratory disease in neonates and infants, which often presents with multisystem organ involvement, co-morbidities, and... (Review)
Review
INTRODUCTION
Bronchopulmonary dysplasia (BPD) is a chronic respiratory disease in neonates and infants, which often presents with multisystem organ involvement, co-morbidities, and prolonged hospital stays. Therefore, a multidisciplinary chronic care approach is needed in the severest forms of BPD to optimize outcomes. However, this approach can be challenging to implement. The objective of this article is to review and synthesize the available literature regarding multidisciplinary care in infants and children with established BPD, and to provide a framework that can guide clinical practice and future research.
AREAS COVERED
A literature search was conducted using Ovid MEDLINE, CINAHL, and Embase and several components of multidisciplinary management of BPD were identified and reviewed, including chronic care, team development, team members, discharge planning, and outpatient care.
EXPERT OPINION
Establishing a core multidisciplinary group familiar with the chronicity of established BPD is recommended as best practice for this population. Acknowledging this is not feasible for all individual centers, it is important for clinical practice and future research to focus on the development and incorporation of national consulting services, telemedicine, and educational resources.
Topics: Infant, Newborn; Infant; Child; Humans; Infant, Premature; Respiration, Artificial; Bronchopulmonary Dysplasia
PubMed: 37982177
DOI: 10.1080/17476348.2023.2283120 -
Gastroenterology Clinics of North... Mar 2024Early detection of dysplasia and effective management are critical steps in halting neoplastic progression in patients with Barrett's esophagus (BE). This review... (Review)
Review
Early detection of dysplasia and effective management are critical steps in halting neoplastic progression in patients with Barrett's esophagus (BE). This review provides a contemporary overview of the BE-related dysplasia, its role in guiding surveillance and management, and discusses emerging diagnostic and therapeutic approaches that might further enhance patient management. Novel, noninvasive techniques for sampling and surveillance, adjunct biomarkers for risk assessment, and their limitations are also discussed.
Topics: Humans; Barrett Esophagus; Esophageal Neoplasms; Hyperplasia
PubMed: 38280743
DOI: 10.1016/j.gtc.2023.11.002 -
NeoReviews Nov 2023Bronchopulmonary dysplasia (BPD) is a common, severe chronic respiratory disease that affects very preterm infants. In utero and postnatal exposure to proinflammatory... (Review)
Review
Bronchopulmonary dysplasia (BPD) is a common, severe chronic respiratory disease that affects very preterm infants. In utero and postnatal exposure to proinflammatory stimuli contribute to the pathophysiology of BPD. Corticosteroids, because of their potent anti-inflammatory properties, may decrease respiratory morbidity and reduce the risk of BPD in very preterm infants. However, these medications can have adverse effects on the developing brain and other organ systems. This review examines current evidence on the risks and benefits of postnatal corticosteroids used to prevent BPD in preterm infants.
Topics: Humans; Infant; Infant, Newborn; Adrenal Cortex Hormones; Bronchopulmonary Dysplasia; Dexamethasone; Glucocorticoids; Infant, Premature; Infant, Premature, Diseases
PubMed: 37907402
DOI: 10.1542/neo.24-11-e691 -
Frontiers in Cell and Developmental... 2023Bronchopulmonary dysplasia (BPD) is a common complication in preterm infants, leading to chronic respiratory disease. There has been an improvement in perinatal care,... (Review)
Review
Bronchopulmonary dysplasia (BPD) is a common complication in preterm infants, leading to chronic respiratory disease. There has been an improvement in perinatal care, but many infants still suffer from impaired branching morphogenesis, alveolarization, and pulmonary capillary formation, causing lung function impairments and BPD. There is an increased risk of respiratory infections, pulmonary hypertension, and neurodevelopmental delays in infants with BPD, all of which can lead to long-term morbidity and mortality. Unfortunately, treatment options for Bronchopulmonary dysplasia are limited. A growing body of evidence indicates that mesenchymal stromal/stem cells (MSCs) can treat various lung diseases in regenerative medicine. MSCs are multipotent cells that can differentiate into multiple cell types, including lung cells, and possess immunomodulatory, anti-inflammatory, antioxidative stress, and regenerative properties. MSCs are regulated by mitochondrial function, as well as oxidant stress responses. Maintaining mitochondrial homeostasis will likely be key for MSCs to stimulate proper lung development and regeneration in Bronchopulmonary dysplasia. In recent years, MSCs have demonstrated promising results in treating and preventing bronchopulmonary dysplasia. Studies have shown that MSC therapy can reduce inflammation, mitochondrial impairment, lung injury, and fibrosis. In light of this, MSCs have emerged as a potential therapeutic option for treating Bronchopulmonary dysplasia. The article explores the role of MSCs in lung development and disease, summarizes MSC therapy's effectiveness in treating Bronchopulmonary dysplasia, and delves into the mechanisms behind this treatment.
PubMed: 37965579
DOI: 10.3389/fcell.2023.1247339 -
Experimental Cell Research Oct 2023Primary cilia are non-motile, microtubule-based sensory organelle present in most vertebrate cells with a fundamental role in the modulation of organismal development,... (Review)
Review
Primary cilia are non-motile, microtubule-based sensory organelle present in most vertebrate cells with a fundamental role in the modulation of organismal development, morphogenesis, and repair. Here we focus on the role of primary cilia in embryonic and postnatal skeletal development. We examine evidence supporting its involvement in physiochemical and developmental signaling that regulates proliferation, patterning, differentiation and homeostasis of osteoblasts, chondrocytes, and their progenitor cells in the skeleton. We discuss how signaling effectors in mechanotransduction and bone development, such as Hedgehog, Wnt, Fibroblast growth factor and second messenger pathways operate at least in part at the primary cilium. The relevance of primary cilia in bone formation and maintenance is underscored by a growing list of rare genetic skeletal ciliopathies. We collate these findings and summarize the current understanding of molecular factors and mechanisms governing primary ciliogenesis and ciliary function in skeletal development and disease.
Topics: Cilia; Humans; Animals; Bone and Bones; Skeleton; Organogenesis; Osteogenesis; Signal Transduction; Mechanotransduction, Cellular
PubMed: 37574037
DOI: 10.1016/j.yexcr.2023.113751 -
European Journal of Pediatrics Mar 2024As we continue to understand more about the complex mechanism of growth, a plethora of novel therapies have recently been developed that aim to address barriers and... (Review)
Review
As we continue to understand more about the complex mechanism of growth, a plethora of novel therapies have recently been developed that aim to address barriers and optimize efficacy. This review aims to explore these novel therapies and provide a succinct review based on the latest clinical studies in order to introduce clinicians to therapies that will soon constitute the future in the field of short stature. Conclusion: The review focuses on long-acting growth hormone formulations, a novel growth hormone oral secretagogue, novel treatments for children with achondroplasia, and targeted therapies for rare forms of skeletal dysplasias. What is Known: • Recombinant human growth hormone has been the mainstay of treatment for children with short stature for years. • Such therapy is not always effective based on the underlying diagnosis (e.g achondroplasia, Turner syndrome). Compliance with daily injections is challenging and can directly affect efficacy. What is New: • Recent development of long-acting growth hormone regimens and oral secretagogues can overcome some of these barriers, however several limitations need to be taken into consideration. • Newer therapies for achondroplasia, and other rare forms of skeletal dysplasias introduce us to a new era of targeted therapies for children with short stature. Clinicians ought to be aware of pitfalls and caveats before introducing these novel therapies to every day practice.
Topics: Child; Humans; Growth Disorders; Human Growth Hormone; Growth Hormone; Turner Syndrome; Achondroplasia
PubMed: 37831302
DOI: 10.1007/s00431-023-05239-y -
Hand Surgery & Rehabilitation Apr 2024Congenital nail disorders are an uncommon presenting symptom which can be difficult to diagnose and manage. Nail diseases in the pediatric population differ from those... (Review)
Review
Congenital nail disorders are an uncommon presenting symptom which can be difficult to diagnose and manage. Nail diseases in the pediatric population differ from those in adults in terms of diagnosis, approach and management. In most cases, they do not require treatment and resolve with growth. Physicians need to be able to recognize them, to reassure the parents. The most frequently encountered pathologies associated with nail disorder are syndactyly, acrosyndactyly, symbrachydactyly, macrodactyly, Wassel I thumb duplication, Kirner's deformity and congenital onychodysplasia of the index finger. Treatment usually consists in surgical correction of the deformity. Nail malformation can also be an aspect of a systemic disease. It may provide a clue for screening, and should not be overlooked. Nail conditions can be the first sign of nail-patella syndrome, ectodermal dysplasia, dyskeratosis congenita, epidermolysis bullosa, pachyonychia congenita or lung disease. Medical treatment is therefore discussed on a case-by-case basis.
Topics: Humans; Nails, Malformed; Nail Diseases; Ectodermal Dysplasia
PubMed: 38879228
DOI: 10.1016/j.hansur.2023.01.011 -
Current Opinion in Endocrinology,... Feb 2024The purpose of this review is to provide a comprehensive overview into the diagnosis and management of fibrous dysplasia (FD) in children. (Review)
Review
PURPOSE OF REVIEW
The purpose of this review is to provide a comprehensive overview into the diagnosis and management of fibrous dysplasia (FD) in children.
RECENT FINDINGS
FD is a mosaic disorder arising from somatic Gα s variants, leading to impaired osteogenic cell differentiation. Fibro-osseous lesions expand during childhood and reach final disease burden in early adulthood. The mainstay of treatment focuses on surgical correction of skeletal deformities, physiatric care, and medical management of associated hyperfunctioning endocrinopathies. Bisphosphonates may be helpful to treat bone pain, but do not alter lesion quality or progression. Emerging evidence suggests that the RANKL inhibitor denosumab may be effective in improving lesion activity and mineralization, however further studies are needed to determine the potential utility of this and other novel therapies, particularly in children with FD.
SUMMARY
Management of children with FD has unique challenges related to skeletal growth and age-related lesion progression. Inclusion of children in clinical research is critical to develop effective treatment strategies to treat FD lesions and prevent their development.
Topics: Child; Humans; Adult; Fibrous Dysplasia of Bone; Bone and Bones; Cell Differentiation; Diphosphonates; Bone Diseases
PubMed: 38010041
DOI: 10.1097/MED.0000000000000847 -
The British Journal of Radiology Jul 2023Bone dysplasias are individually rare but collectively common. The prenatal diagnosis of bone dysplasias, especially perinatally lethal dysplasias, is of major interest... (Review)
Review
Bone dysplasias are individually rare but collectively common. The prenatal diagnosis of bone dysplasias, especially perinatally lethal dysplasias, is of major interest to obstetric services. The current nosology of genetic skeletal disorders addresses over 400 disorders. However, in clinical practice, we encounter only a limited number of disorders, such as . The recent development of non-invasive prenatal genetic testing using cell-free fetal DNA in maternal blood samples has had a major impact on the prenatal diagnosis of genetic diseases. However, imaging examinations remain critical for the final diagnosis of bone dysplasias because molecular testing only shows genetic variants, and not their pathogenicity - most variants are clinically insignificant. Bone dysplasias are typically suspected when limb shortening is identified by screening ultrasound. Further assessment can be followed by more detailed ultrasound, magnetic resonance imaging (MRI), and CT. Based on these data, rational decision-making is feasible, even when the definitive prenatal diagnosis is not feasible. Here, we highlight key images of common bone dysplasias obtained by currently available modalities.
Topics: Pregnancy; Female; Humans; Prenatal Diagnosis; Bone Diseases, Developmental; Ultrasonography; Fetus; Receptor, Fibroblast Growth Factor, Type 3; Ultrasonography, Prenatal
PubMed: 37351952
DOI: 10.1259/bjr.20221025