☆ 4.4 Article

Emerging Treatments for Childhood Interstitial Lung Disease

PEDIATRIC DRUGS (2023)

相关参考文献

注意：仅列出部分参考文献，下载原文获取全部文献信息。

Editorial Material Respiratory System

Nintedanib in chILD: a small step, yes ... but at least a step forward in a marathon! ...

David Gozal et al.

EUROPEAN RESPIRATORY JOURNAL (2023)

添加到收藏夹

Article Medicine, General & Internal

Interstitial Lung Disease in Immunocompromised Children

Xianfei Gao et al.

Summary: This study evaluated the range of pulmonary complications in pediatric immunocompromised patients and found that interstitial lung disease (ILD) was the most common. It also found that immunological disorders not yet associated with ILD can also cause ILD. Therefore, specialized pneumological expertise is necessary for managing the full spectrum of respiratory complications in immunocompromised children.

DIAGNOSTICS (2023)

添加到收藏夹

Article Respiratory System

Nintedanib in children and adolescents with fibrosing interstitial lung diseases

Robin Deterding et al.

Summary: The study aimed to determine the dose-exposure and safety of nintedanib in children and adolescents with fibrosing ILD. The results showed that a weight-based dosing regimen of nintedanib in children and adolescents with fibrosing ILD resulted in exposure similar to adults and an acceptable safety profile, providing a scientific basis for its use in this patient population.

EUROPEAN RESPIRATORY JOURNAL (2023)

添加到收藏夹

Article Biochemistry & Molecular Biology

ABCA3 Deficiency-Variant-Specific Response to Hydroxychloroquine

Xiaohua Yang et al.

Summary: Biallelic variants in ABCA3 can cause interstitial lung diseases, and the sensitivity to hydroxychloroquine varies depending on the genetic mutation.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2023)

添加到收藏夹

Article Pediatrics

Efficacy and safety of baricitinib in Japanese patients with autoinflammatory type I interferonopathies (NNS/CANDLE, SAVI, And AGS)

Nobuo Kanazawa et al.

Summary: This study evaluated the efficacy and safety of baricitinib in Japanese patients with NNS/CANDLE, SAVI, and AGS. The results suggest that baricitinib may offer a potential therapeutic option for these diseases and has a positive benefit/risk profile in a vulnerable patient population.

PEDIATRIC RHEUMATOLOGY (2023)

添加到收藏夹

Article Respiratory System

ABCA3-related interstitial lung disease beyond infancy

Yang Li et al.

Summary: This register-based cohort study reviews the long-term clinical course of patients with ABCA3 lung disease who survived beyond the age of 1 year. The results show that ABCA3-related interstitial lung disease progresses during childhood and adolescence, emphasizing the need for disease-modifying treatments to delay the disease course.

THORAX (2023)

添加到收藏夹

Review Respiratory System

Diagnostic workup of childhood interstitial lung disease

Nadia Nathan et al.

Summary: Childhood interstitial lung diseases (chILDs) are rare and heterogeneous diseases with significant morbidity and mortality. An accurate and rapid diagnosis is crucial for effective management and personalized treatment. This review outlines the roles of general pediatricians, pediatric pulmonologists, and expert centers in the complex diagnostic process.

EUROPEAN RESPIRATORY REVIEW (2023)

添加到收藏夹

Article Critical Care Medicine

Progressive Disease With Low Survival in Adult Patients With Pulmonary Fibrosis Carrying Surfactant-Related Gene Mutations An Observational Study

Dymph Klay et al.

Summary: This study found that ILD patients with an SRG mutation experience progressive loss of lung function and reduced survival, despite potential beneficial effects of treatment.

CHEST (2023)

添加到收藏夹

Article Respiratory System

Diffuse alveolar haemorrhage in children: an international multicentre study

Astrid Madsen Ring et al.

ERJ Open Research (2023)

添加到收藏夹

Article Biochemistry & Molecular Biology

High-Content Screening Identifies Cyclosporin A as a Novel ABCA3-Specific Molecular Corrector

Maria Forstner et al.

Summary: ABCA3 gene mutations are a common cause of respiratory distress syndrome and interstitial lung disease. There is currently no cure for ABCA3 deficiency. Researchers developed a cell-based assay and used machine learning algorithms to identify and study mutant cells, with the aim of finding therapeutic options.

AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY (2022)

添加到收藏夹

Article Cardiac & Cardiovascular Systems

Indications and outcome after lung transplantation in children under 12 years of age: A 16-year single center experience

Pavel Iablonskii et al.

Summary: Pediatric lung transplantation poses unique challenges, but the outcomes are comparable to those in older children.

JOURNAL OF HEART AND LUNG TRANSPLANTATION (2022)

添加到收藏夹

Article Pediatrics

Pulmonary alveolar proteinosis due to heterozygous mutation in OAS1: Whole lung lavages for long-term bridging to hematopoietic stem cell transplantation

Elias Seidl et al.

Summary: Congenital cerebellar hypoplasia is a rare neurodevelopmental disorder that often results in intellectual disability, motor impairments, and speech difficulties, placing a significant burden on patients and their families.

PEDIATRIC PULMONOLOGY (2022)

添加到收藏夹

Article Respiratory System

Healthcare resource utilisation and medical costs for children with interstitial lung diseases (chILD) in Europe

Elias Seidl et al.

Summary: This prospective longitudinal study provides the first analysis of healthcare resource utilization and costs for children's interstitial lung diseases (chILD) across different European countries. The study results indicate that chILD is associated with high utilization of healthcare services, placing a substantial economic burden on health systems.

THORAX (2022)

添加到收藏夹

Article Respiratory System

Acute exacerbations in children's interstitial lung disease

Elias Seidl et al.

Summary: The characteristics and impact of acute exacerbations (AEs) on children's interstitial lung disease (chILD) were comprehensively analyzed in this study. The results showed that AEs have a significant and deleterious effect on the clinical course and health-related quality of life in chILD.

THORAX (2022)

添加到收藏夹

Review Nursing

Cardiovascular, Brain, and Lung Involvement in a Newborn With a Novel FLNA Mutation A Case Report and Literature Review

Giovanni Meliota et al.

Summary: This study presents a case of a female infant with aortic valve stenosis, brain periventricular nodular heterotopia, interstitial lung disease, and severe pulmonary hypertension. A novel FLNA gene mutation was detected, indicating a loss of function of FLNA.

ADVANCES IN NEONATAL CARE (2022)

添加到收藏夹

Article Respiratory System

Incidence and Prevalence of Children's Diffuse Lung Disease in Spain

Alba Torrent-Vernetta et al.

Summary: This study analyzed the incidence and prevalence of children's diffuse lung disease (chILD) in Spain. The results showed a higher incidence and prevalence of chILD than previously described, probably due to greater understanding and increased clinician awareness of these rare diseases.

ARCHIVOS DE BRONCONEUMOLOGIA (2022)

添加到收藏夹

Article Respiratory System

Methionine supplementation for multi-organ dysfunction in MetRS-related pulmonary alveolar proteinosis

Alice Hadchouel et al.

Summary: Methionine supplementation in children with pulmonary alveolar proteinosis related to mutations in the MARS1 gene led to significant improvements in respiratory condition, liver function, growth delay, and inflammation. It also normalized the production of reactive oxygen species by peripheral monocytes, suggesting potential benefits for other tRNA synthetase deficiencies.

EUROPEAN RESPIRATORY JOURNAL (2022)

添加到收藏夹

Editorial Material Respiratory System

Targeted therapy for pulmonary alveolar proteinosis: the time is now

Francesco Bonella et al.

EUROPEAN RESPIRATORY JOURNAL (2022)

添加到收藏夹

Article Medicine, General & Internal

Etiologic Classification of Diffuse Parenchymal (Interstitial) Lung Diseases

Matthias Griese

Summary: Interstitial lung diseases (ILD) or diffuse parenchymal lung diseases (DPLD) encompass a wide range of disorders. Disease definition and classification are important for clinical decision-making, genetic/environmental risk assessment, and precision medicine treatments. This article provides a brief history of ILD classification systems and focuses on an etiologic classification, highlighting the importance of molecularly defined entities and the potential for precision medicine.

JOURNAL OF CLINICAL MEDICINE (2022)

添加到收藏夹

Article Immunology

Living Donor Lobar Lung Transplant for a Patient With Lung Disease Caused by ABCA3 Gene Mutations: A Case Report

Sakiko Kumata et al.

Summary: This article reports a case of a 20-month-old boy with interstitial lung disease who was treated with hydroxychloroquine and later underwent living-donor lobar lung transplantation. The boy's parents served as donors and the boy has had good lung function since the surgery.

TRANSPLANTATION PROCEEDINGS (2022)

添加到收藏夹

Article Genetics & Heredity

Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease

Matthias Griese et al.

Summary: Background: No controlled trials are available for treatments in children with chILD, making it difficult to evaluate their efficacy. This study evaluated the use of HCQ in a phase 2 trial and found that it did not show significant treatment effects compared to placebo. Although the study had limitations, the small effect size of HCQ treatment observed suggests a need for careful reevaluation of its prescription in everyday practice.

ORPHANET JOURNAL OF RARE DISEASES (2022)

添加到收藏夹

Review Pediatrics

Interstitial Lung Disease in Children: Specific Conditions of Undefined Etiology Becoming Clearer

Santiago Presti et al.

Summary: NEHI and PIG are specific infant groups of chILD characterized by dysmaturity of airway development and consequent respiratory distress.

CHILDREN-BASEL (2022)

添加到收藏夹

Article Medicine, Research & Experimental

Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases

Marta del Alamo et al.

Summary: Academic-sponsored trials for rare diseases face numerous challenges, including administrative burden, lack of harmonization for trial-site agreements, complexity and restriction on the use of public funding, drug supply issues, and lack of harmonization on regulatory requirements. Better understanding of the non-commercial clinical research landscape and providing resources are needed to address the challenges associated with the set-up of academic-sponsored multinational trials.

TRIALS (2022)

添加到收藏夹

Article Pediatrics

Long-term evolution of neuroendocrine cell hyperplasia of infancy: the FRENCHI findings

Morgane Dervaux et al.

Summary: This study reports the long-term evolution of patients with neuroendocrine cell hyperplasia of infancy (NEHI). The majority of patients showed improvement in respiratory and nutritional aspects during follow-up, but the progress varied among individuals.

EUROPEAN JOURNAL OF PEDIATRICS (2022)

添加到收藏夹

Article Multidisciplinary Sciences

Organoids

Zixuan Zhao et al.

Summary: Organoids are simple tissue-engineered cell-based models that mimic the complex structure and function of in vivo tissues. They have a wide range of applications in developmental studies, disease modeling, and personalized medicine. Various engineering strategies have been developed to support their culture, growth, differentiation, and maturation.

NATURE REVIEWS METHODS PRIMERS (2022)

添加到收藏夹

Article Respiratory System

Human umbilical cord mesenchymal stem cells combined with pirfenidone upregulates the expression of RGS2 in the pulmonary fibrosis in mice

Xian Wu et al.

Summary: The combined therapy of umbilical cord-derived mesenchymal stem cells (hUC-MSCs) and low-dose pirfenidone (PFD) has a better therapeutic effect on bleomycin-induced pulmonary fibrosis in mice than each treatment alone. This combination therapy attenuates fibrosis through increasing the expression of RGS2.

RESPIRATORY RESEARCH (2022)

添加到收藏夹

Article Respiratory System

Autoimmune pulmonary alveolar proteinosis in children

Matthias Griese et al.

Summary: This article discusses the cases of four children with autoimmune PAP and describes the presentation and management issues. By considering various treatment options, including inhaled GM-CSF, the most effective and least invasive treatment method can be selected for patients. Establishing an appropriate registry enables the collection of patient cohorts with rare conditions. To expedite the authorization of novel treatments, adolescents should be included in trials in adults.

ERJ OPEN RESEARCH (2022)

添加到收藏夹

Review Biotechnology & Applied Microbiology

Gene Therapy Potential for Genetic Disorders of Surfactant Dysfunction

Ashley L. Cooney et al.

Summary: Pulmonary surfactant is important for preventing atelectasis, and genetic mutations in key genes can cause severe respiratory distress syndrome or childhood interstitial lung disease. Gene therapy using viral vectors such as adeno-associated virus, lentivirus, and adenovirus shows promise in treating these lung diseases.

FRONTIERS IN GENOME EDITING (2022)

添加到收藏夹

Review Rheumatology

COPA syndrome, 5 years after: Where are we?

Marie-Louise Fremond et al.

Summary: Heterozygous missense mutations in COPA cause an interferonopathy involving lung, joint, and kidney, with high clinical non-penetrance. Patients mainly exhibit lung features such as chronic DAH, and may benefit from JAK inhibitors as a promising treatment. Further targeted therapies for COPA syndrome are eagerly anticipated.

JOINT BONE SPINE (2021)

添加到收藏夹

Article Respiratory System

Genetic disorders of the surfactant system: focus on adult disease

Coline H. M. van Moorsel et al.

Summary: Mutations in surfactant-related genes can cause a spectrum of lung diseases, with patients of all ages affected. Genetic analysis has potential diagnostic value, but limited genotype-phenotype correlations hinder development of mutation-specific treatment options.

EUROPEAN RESPIRATORY REVIEW (2021)

添加到收藏夹

Review Pediatrics

Surfactant protein disorders in childhood interstitial lung disease

Jagdev Singh et al.

Summary: Surfactant plays a crucial role in the respiratory system and is associated with various diseases such as childhood interstitial lung disease. Surfactant protein disorders are rare conditions caused by genetic mutations, with ongoing research and evolving diagnostic and treatment options.

EUROPEAN JOURNAL OF PEDIATRICS (2021)

添加到收藏夹

Article Immunology

Case Report: Successful Treatment of Refractory Interstitial Lung Disease With Cyclosporine A and Pirfenidone in a Child With SLE

Linxia Deng et al.

Summary: Interstitial lung disease (ILD) as an initial manifestation of lupus is rare in young children. Prompt work-up for connective tissue disease (CTD) should be considered in young children with ILD. Pirfenidone might be a useful add-on therapy with immunosuppressive agents for refractory CTD-ILD in pediatric patients. Further clinical trials are needed to assess the efficiency and safety of this combination therapy for refractory CTD-ILD.

FRONTIERS IN IMMUNOLOGY (2021)

添加到收藏夹

Review Cell Biology

Mesenchymal Stromal Cells for the Treatment of Interstitial Lung Disease in Children: A Look from Pediatric and Pediatric Surgeon Viewpoints

Gloria Pelizzo et al.

Summary: MSCs have been proposed as a potential treatment for chILD, which is characterized by inflammatory and fibrotic changes in the lungs leading to respiratory failure. Current therapeutic strategies involve corticosteroids, hydroxychloroquine, azithromycin, and supportive care, but long-term use is associated with toxicity and varying efficacy.

CELLS (2021)

添加到收藏夹

Article Cell & Tissue Engineering

Type 2 Alveolar Epithelial Cells Differentiated from Human Umbilical Cord Mesenchymal Stem Cells Alleviate Mouse Pulmonary Fibrosis Through β-Catenin-Regulated Cell Apoptosis

Jiang Liu et al.

Summary: This study demonstrated that intratracheal transplantation of hUC-MSC-derived AEC2s could improve mortality and alleviate fibrosis in bleomycin-induced PF mice through regulating apoptosis mediated by beta-catenin, providing a viable strategy for the treatment of PF.

STEM CELLS AND DEVELOPMENT (2021)

添加到收藏夹

Article Respiratory System

TELO-SCOPE study: a randomised, double-blind, placebo-controlled, phase 2 trial of danazol for short telomere related pulmonary fibrosis

John A. Mackintosh et al.

Summary: Recent discoveries have found shortened telomeres and related mutations in people with pulmonary fibrosis. Androgens, including danazol, are believed to be effective in lengthening telomeres in blood cells. This study aims to assess the safety and efficacy of danazol in both adults and children with PF associated with telomere shortening.

BMJ OPEN RESPIRATORY RESEARCH (2021)

添加到收藏夹

Article Cell & Tissue Engineering

Stem cells for bronchopulmonary dysplasia in preterm infants: A randomized controlled phase II trial

So Yoon Ahn et al.

Summary: The study demonstrated the safety and feasibility of MSC transplantation for BPD in preterm infants through a phase I clinical trial. The efficacy of MSCs for BPD treatment in premature infants was investigated in a phase II trial, where although inflammatory cytokines were significantly reduced, the overall primary outcome was not significantly improved. Subgroup analysis showed a significant improvement with MSC transplantation in the 23-24 GW group.

STEM CELLS TRANSLATIONAL MEDICINE (2021)

添加到收藏夹

Review Biochemistry & Molecular Biology

Cellular Therapy for the Treatment of Paediatric Respiratory Disease

Laura C. Brennan et al.

Summary: Respiratory diseases are the leading cause of death in children under 5 years old, yet there is currently no cure. Cellular therapy offers potential as a treatment option, but further research and clinical trials are needed, with a focus on understanding the differences in pediatric disease pathology and physiology.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

添加到收藏夹

Article Respiratory System

Transition of patients with interstitial lung disease from paediatric to adult care

Vaclav Koucky et al.

Summary: The study found that there is a highly variable and inadequate current system of transitions of children with interstitial lung diseases to adult care in most centers. Therefore, the Working Group prepared a model procedure for transition to ensure a smooth transfer of patients to a new specialist and team.

ERJ OPEN RESEARCH (2021)

添加到收藏夹

Article Rheumatology

Serum KL-6 level as a biomarker of interstitial lung disease in childhood connective tissue diseases: a pilot study

Ayse Ayzit Kilinc et al.

RHEUMATOLOGY INTERNATIONAL (2020)

添加到收藏夹

Article Respiratory System

Use of ruxolitinib in COPA syndrome manifesting as life-threatening alveolar haemorrhage

Marie-Louise Fremond et al.

THORAX (2020)

添加到收藏夹

Article Medicine, Research & Experimental

Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial

Matthias Griese et al.

TRIALS (2020)

添加到收藏夹

Review Pediatrics

Childhood rare lung disease in the 21st century: -omics technology advances accelerating discovery

Timothy J. Vece et al.

PEDIATRIC PULMONOLOGY (2020)

添加到收藏夹

Article Pediatrics

Persistent tachypnea of infancy: Follow up at school age

Elias Seidl et al.

PEDIATRIC PULMONOLOGY (2020)

添加到收藏夹

Article Multidisciplinary Sciences

A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency

Martin H. Kang et al.

NATURE COMMUNICATIONS (2020)

添加到收藏夹

Article Medicine, General & Internal

Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis

Bruce C. Trapnell et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

添加到收藏夹

Article Pediatrics

Rescue of respiratory failure in pulmonary alveolar proteinosis due to pathogenic MARS1 variants

Dominic Lenz et al.

PEDIATRIC PULMONOLOGY (2020)

添加到收藏夹

Review Respiratory System

Pulmonary alveolar proteinosis in children

Andrew Bush et al.

BREATHE (2020)

添加到收藏夹

Article Respiratory System

Neuroendocrine Cell Hyperplasia of Infancy Clinical Score and Comorbidities

Deborah R. Liptzin et al.

ANNALS OF THE AMERICAN THORACIC SOCIETY (2020)

添加到收藏夹

Review Rheumatology

Baricitinib in therapy of COPA syndrome in a 15-year-old girl

Sophia Krutzke et al.

EUROPEAN JOURNAL OF RHEUMATOLOGY (2020)

添加到收藏夹

Article Medicine, General & Internal

Pulmonary alveolar proteinosis

Bruce C. Trapnell et al.

NATURE REVIEWS DISEASE PRIMERS (2019)

添加到收藏夹

Review Critical Care Medicine

Approaching Clinical Trials in Childhood Interstitial Lung Disease and Pediatric Pulmonary Fibrosis

Robin R. Deterding et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2019)

添加到收藏夹

Article Respiratory System

Phenotype characterisation of TBX4 mutation and deletion carriers with neonatal and paediatric pulmonary hypertension

Csaba Galambos et al.

EUROPEAN RESPIRATORY JOURNAL (2019)

添加到收藏夹

Article Cell Biology

Potentiation of ABCA3 lipid transport function by ivacaftor and genistein

Susanna Kinting et al.

JOURNAL OF CELLULAR AND MOLECULAR MEDICINE (2019)

添加到收藏夹

Article Critical Care Medicine

Pulmonary Aptamer Signatures in Children's Interstitial and Diffuse Lung Disease

Robin R. Deterding et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2019)

添加到收藏夹

Article Medicine, General & Internal

Nintedanib in Progressive Fibrosing Interstitial Lung Diseases

K. R. Flaherty et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

添加到收藏夹

Article Medicine, General & Internal

Inhaled GM-CSF for Pulmonary Alveolar Proteinosis

Ryushi Tazawa et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

添加到收藏夹

Letter Respiratory System

Bi-allelic missense ABCA3 mutations in a patient with childhood ILD who reached adulthood

Effrosyni D. Manali et al.

ERJ OPEN RESEARCH (2019)

添加到收藏夹

Review Genetics & Heredity

Genetics of COPA syndrome

Rajni Kumrah et al.

APPLICATION OF CLINICAL GENETICS (2019)

添加到收藏夹

Review Pediatrics

Children's interstitial and diffuse lung disease

Steve Cunningham et al.

LANCET CHILD & ADOLESCENT HEALTH (2019)

添加到收藏夹

Article Respiratory System

International management platform for children's interstitial lung disease (chILD-EU)

Matthias Griese et al.

THORAX (2018)

添加到收藏夹

Article Genetics & Heredity

MARS variant associated with both recessive interstitial lung and liver disease and dominant Charcot-Marie-Tooth disease

Jonathan Rips et al.

EUROPEAN JOURNAL OF MEDICAL GENETICS (2018)

添加到收藏夹

Article Biochemistry & Molecular Biology

Functional rescue of misfolding ABCA3 mutations by small molecular correctors

Susanna Kinting et al.

HUMAN MOLECULAR GENETICS (2018)

添加到收藏夹

Article Pediatrics

Systemic Lupus Erythematosus, Sjogren Syndrome, and Mixed Connective Tissue Disease in Children and Adolescents

Stacey E. Tarvin et al.

PEDIATRIC CLINICS OF NORTH AMERICA (2018)

添加到收藏夹

Article Pediatrics

Development and validation of a health-related quality of life questionnaire for pediatric patients with interstitial lung disease

Mandy Niemitz et al.

PEDIATRIC PULMONOLOGY (2018)

添加到收藏夹

Article Cell & Tissue Engineering

iPSC-Derived Macrophages Effectively Treat Pulmonary Alveolar Proteinosis in Csf2rb-Deficient Mice

Adele Mucci et al.

STEM CELL REPORTS (2018)

添加到收藏夹

Meeting Abstract Respiratory System

A national registry for childhood interstitial and diffuse lung diseases in the United States.

Lisa Young et al.

EUROPEAN RESPIRATORY JOURNAL (2018)

添加到收藏夹

Review Respiratory System

Chronic interstitial lung disease in children

Matthias Griese

EUROPEAN RESPIRATORY REVIEW (2018)

添加到收藏夹

Article Respiratory System

Lung disease caused by ABCA3 mutations

Carolin Kroener et al.

THORAX (2017)

添加到收藏夹

Article Biotechnology & Applied Microbiology

Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency

Miriam Hetzel et al.

HUMAN GENE THERAPY METHODS (2017)

添加到收藏夹

Article Critical Care Medicine

Persistent Tachypnea of Infancy Usual and Aberrant

Daniela Rauch et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2016)

添加到收藏夹

Review Pediatrics

Interstitial lung disease in infancy: A general approach

Erica J. Hines et al.

JOURNAL OF PAEDIATRICS AND CHILD HEALTH (2016)

添加到收藏夹

Article Medicine, General & Internal

Danazol Treatment for Telomere Diseases

Danielle M. Townsley et al.

NEW ENGLAND JOURNAL OF MEDICINE (2016)

添加到收藏夹

Article Genetics & Heredity

Assessing the feasibility of a web-based registry for multiple orphan lung diseases: the Australasian Registry Network for Orphan Lung Disease (ARNOLD) experience

K. Casamento et al.

ORPHANET JOURNAL OF RARE DISEASES (2016)

添加到收藏夹

Article Genetics & Heredity

Biallelic Mutations of Methionyl-tRNA Synthetase Cause a Specific Type of Pulmonary Alveolar Proteinosis Prevalent on Reunion Island

Alice Hadchouel et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2015)

添加到收藏夹

Article Respiratory System

Genotype alone does not predict the clinical course of SFTPC deficiency in paediatric patients

Carolin Kroener et al.

EUROPEAN RESPIRATORY JOURNAL (2015)

添加到收藏夹

Letter Biotechnology & Applied Microbiology

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

Azita J. Mahiny et al.

NATURE BIOTECHNOLOGY (2015)

添加到收藏夹

Review Anesthesiology

Anesthetic techniques to facilitate lung lavage for pulmonary alveolar proteinosis in children-new airway techniques and a review of the literature

Caroline A. Wilson et al.

PEDIATRIC ANESTHESIA (2015)

添加到收藏夹

Review Pediatrics

Childhood Interstitial Lung Disease: A Systematic Review

Neil J. Hime et al.

PEDIATRIC PULMONOLOGY (2015)

添加到收藏夹

Article Physiology

Work of breathing in children with diffuse parenchymal lung disease

Sonia Khirani et al.

RESPIRATORY PHYSIOLOGY & NEUROBIOLOGY (2015)

添加到收藏夹

Review Respiratory System

European protocols for the diagnosis and initial treatment of interstitial lung disease in children

Andrew Bush et al.

THORAX (2015)

添加到收藏夹

Article Respiratory System

GATA2 deficiency in children and adults with severe pulmonary alveolar proteinosis and hematologic disorders

Matthias Griese et al.

BMC PULMONARY MEDICINE (2015)

添加到收藏夹

Review Immunology

Therapy and pharmacological properties of hydroxychloroquine and chloroquine in treatment of systemic lupus erythematosus, rheumatoid arthritis and related diseases

K. D. Rainsford et al.

INFLAMMOPHARMACOLOGY (2015)

添加到收藏夹

Review Pediatrics

Hydroxychloroquine in Children With Interstitial (diffuse parenchymal) Lung Diseases

Sarah Braun et al.

PEDIATRIC PULMONOLOGY (2015)

添加到收藏夹

Article Medicine, General & Internal

A Phase 3 Trial of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis

Talmadge E. King et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

添加到收藏夹

Article Medicine, General & Internal

Efficacy and Safety of Nintedanib in Idiopathic Pulmonary Fibrosis

Luca Richeldi et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

添加到收藏夹

Article Medicine, General & Internal

Activated STING in a Vascular and Pulmonary Syndrome

Y. Liu et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

添加到收藏夹

Article Genetics & Heredity

Characterization of CSF2RA mutation related juvenile pulmonary alveolar proteinosis

Jenna Hildebrandt et al.

ORPHANET JOURNAL OF RARE DISEASES (2014)

添加到收藏夹

Article Critical Care Medicine

An Official American Thoracic Society Clinical Practice Guideline: Classification, Evaluation, and Management of Childhood Interstitial Lung Disease in Infancy

Geoffrey Kurland et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2013)

添加到收藏夹

Editorial Material Ophthalmology

Screening for hydroxychloroquine toxicity in children

Bobeck S. Modjtahedi et al.

CUTANEOUS AND OCULAR TOXICOLOGY (2013)

添加到收藏夹

Review Critical Care Medicine

Corticosteroids in Respiratory Diseases in Children

Fernando M. de Benedictis et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2012)

添加到收藏夹

Review Genetics & Heredity

The telomere syndromes

Mary Armanios et al.

NATURE REVIEWS GENETICS (2012)

添加到收藏夹

Letter Respiratory System

Hereditary pulmonary alveolar proteinosis caused by recessive CSF2RB mutations

T. Suzuki et al.

EUROPEAN RESPIRATORY JOURNAL (2011)

添加到收藏夹

Review Medicine, Research & Experimental

Alveolar Surfactant Homeostasis and the Pathogenesis of Pulmonary Disease

Jeffrey A. Whitsett et al.

ANNUAL REVIEW OF MEDICINE (2010)

添加到收藏夹

Review Genetics & Heredity

Interstitial lung diseases in children

Annick Clement et al.

ORPHANET JOURNAL OF RARE DISEASES (2010)

添加到收藏夹

Article Anesthesiology

Whole-lung lavage in infants and children with pulmonary alveolar proteinosis

Karl Reiter et al.

PEDIATRIC ANESTHESIA (2010)

添加到收藏夹

Article Allergy

Genetic Basis of Children's Interstitial Lung Disease

Lawrence M. Nogee

PEDIATRIC ALLERGY IMMUNOLOGY AND PULMONOLOGY (2010)

添加到收藏夹

Article Oncology

Dyskeratosis Congenital

Sharon A. Savage et al.

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA (2009)

添加到收藏夹

Article Respiratory System

Clinical, radiological and pathological features of ABCA3 mutations in children

M. L. Doan et al.

THORAX (2008)

添加到收藏夹

Article Critical Care Medicine

Diffuse lung disease in young children - Application of a novel classification scheme

Gail H. Deutsch et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2007)

添加到收藏夹

Review Obstetrics & Gynecology

Inherited surfactant protein-B deficiency and surfactant protein-C associated disease: Clinical features and evaluation

Aaron Hamvas

SEMINARS IN PERINATOLOGY (2006)

添加到收藏夹

Article Pediatrics

Validation of the National Institutes of Health consensus definition of bronchopulmonary dysplasia

RA Ehrenkranz et al.

PEDIATRICS (2005)

添加到收藏夹

Article Respiratory System

Therapeutic lung lavages in children and adults

C Paschen et al.

RESPIRATORY RESEARCH (2005)

添加到收藏夹

Review Respiratory System

Task force on chronic interstitial lung disease in immunocompetent children

A Clement et al.

EUROPEAN RESPIRATORY JOURNAL (2004)

添加到收藏夹

Article Pediatrics

Idiopathic interstitial pneumonitis in children: A national survey in the United Kingdom and Ireland

R Dinwiddle et al.

PEDIATRIC PULMONOLOGY (2002)

添加到收藏夹

© Peeref 2019-2024. All rights reserved.