Orphan Lung Diseases

Published on May 1, 2019in Medical Clinics of North America2.716
· DOI :10.1016/j.mcna.2018.12.009
Muhammad Ali7
Estimated H-index: 7
(MCW: Medical College of Wisconsin),
Uzair Ghori2
Estimated H-index: 2
(MCW: Medical College of Wisconsin),
Ali I. Musani22
Estimated H-index: 22
(University of Colorado Denver)
  • References (60)
  • Citations (0)
#1Aasim MohammedH-Index: 1
#2Uzair GhoriH-Index: 2
Last. Stephen DolanH-Index: 1
view all 5 authors...
1 CitationsSource
#1Gaohong Sheng (HUST: Huazhong University of Science and Technology)H-Index: 1
#2Peng Chen (HUST: Huazhong University of Science and Technology)H-Index: 1
Last. Huilan Zhang (HUST: Huazhong University of Science and Technology)H-Index: 5
view all 6 authors...
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare pulmonary disease caused by functional deficiency of granulocyte-macrophage colony-stimulating factor (GM-CSF). GM-CSF therapy in aPAP has been reported effective in some studies. This meta-analyses aimed to evaluate whether GM-CSF therapy, including inhaled and subcutaneous GM-CSF have therapeutic effect in aPAP patients. We analyzed 10 studies searched from PubMed, EmBase, Web of Science, Wiley Online Library and Cochrane Collaboration...
5 CitationsSource
#1Cormac McCarthy (Boston Children's Hospital)H-Index: 10
#2Ruzan AvetisyanH-Index: 2
Last. Bruce C. TrapnellH-Index: 65
view all 5 authors...
Pulmonary alveolar proteinosis (PAP) is a rare syndrome of alveolar surfactant accumulation, resulting hypoxemic respiratory failure, and increased infection risk. Despite advances in our understanding of disease pathogenesis and the availability of improved diagnostics, the epidemiology and healthcare burden of PAP remain poorly defined. To determine the prevalence, and healthcare utilization and costs associated with PAP, we interrogated a large health insurance claims database containing comp...
4 CitationsSource
#1Arda Kiani (Shahid Beheshti University of Medical Sciences and Health Services)H-Index: 6
#2T Parsa (Shahid Beheshti University of Medical Sciences and Health Services)H-Index: 3
Last. Atefeh Abedini (Shahid Beheshti University of Medical Sciences and Health Services)H-Index: 6
view all 8 authors...
Introduction: Pulmonary alveolar proteinosis (PAP) is a rare disease in the field of pulmonary medicine. The efficacy of whole-lung lavage (WLL) as the treatment of PAP had never been evaluated in the Iranian population. Therefore, there is a real need to investigate the characteristics of PAP and also to evaluate the efficacy of WLL in this rare disease. The study aimed to investigate demographic features, clinical presentation and treatment outcomes of the disease in Iranian PAP patients. Mate...
1 CitationsSource
#1Olga TorreH-Index: 5
#2Davide EliaH-Index: 5
Last. Sergio HarariH-Index: 27
view all 4 authors...
Lymphangioleiomyomatosis (LAM) and pulmonary Langerhans cell histiocytosis (PLCH) are rare diseases that lead to progressive cystic destruction of the lungs. Despite their distinctive characteristics, these diseases share several features. Patients affected by LAM or PLCH have similar radiological cystic patterns, a similar age of onset, and the possibility of extrapulmonary involvement. In this review, the recent advances in the understanding of the molecular pathogenesis, as well as the curren...
5 CitationsSource
#1Mouhamad NasserH-Index: 1
#2Nader ChebibH-Index: 2
Last. Vincent CottinH-Index: 49
view all 7 authors...
Introduction: Pleuroparenchymal fibroelastosis (PPFE) is a progressive fibrotic lung disease characterized by pleural thickening, pleural and parenchymal fibrosis predominately involving upper parts of the lungs. Survival is dismal and no drug has been shown to modify disease course. Aim and objectives Since parenchymal fibrosis is an important histological feature, we hypothesized that anti-fibrotic treatments might be effective in reducing disease progression. Here, we report our experience us...
2 CitationsSource
#1Hui HuangH-Index: 5
#2Ruie FengH-Index: 6
Last. Jingyu ChenH-Index: 1
view all 7 authors...
3 CitationsSource
#1Martina Bonifazi (Marche Polytechnic University)H-Index: 16
#2M Angeles Montero (Imperial College London)H-Index: 4
Last. Elisabetta A. Renzoni (Imperial College London)H-Index: 35
view all 3 authors...
Purpose of the review Idiopathic pleuroparenchymal fibroelastosis (IPPFE) is a rare fibrosing lung disease, affecting the visceral pleura and the subpleural parenchyma with an upper lobe predilection, included as a distinct clinicopathologic entity in the latest international multidisciplinary classification of the idiopathic interstitial pneumonias (IIP). We aim to summarize the current evidence on IPPFE, in terms of clinical features and potential treatments.
16 CitationsSource
Background: Lymphangioleiomyomatosis (LAM) is a rare cystic lung disease that primarily affects women. The purpose of these guidelines is to provide recommendations for the diagnosis and treatment of LAM.Methods: Systematic reviews were performed to summarize evidence pertinent to our questions. The evidence was summarized and discussed by a multidisciplinary panel. Evidence-based recommendations were then formulated, written, and graded using the Grading of Recommendations, Assessment, Developm...
72 CitationsSource
#1Takuji Suzuki (Cincinnati Children's Hospital Medical Center)H-Index: 30
#2Bruce C. Trapnell (Cincinnati Children's Hospital Medical Center)H-Index: 65
Abstract Pulmonary alveolar proteinosis (PAP) is a rare syndrome characterized by the accumulation of surfactant in alveoli and terminal airways resulting in respiratory failure. PAP comprises part of a spectrum of disorders of surfactant homeostasis (clearance and production). The surfactant production disorders are caused by mutations in genes required for normal surfactant production. The PAP syndrome is identified based on history, radiologic, and bronchoalveolar lavage and/or histopathologi...
35 CitationsSource
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