- Acquired CFTR Dysfunction and Radiographic Bronchiectasis in Current and Former Smokers: A Cross-Sectional Study. [Journal Article]
- AAAnn Am Thorac Soc 2018 Sep 19
- Induced Pluripotent Stem Cells for Primary Ciliary Dyskinesia Modeling and Personalized Medicine. [Journal Article]
- AJAm J Respir Cell Mol Biol 2018 Sep 19
- Primary ciliary dyskinesia (PCD) is a rare and heterogeneous genetic disorder that affects the structure and function of motile cilia. In the airway epithelium, impaired ciliary motion results in red...
Primary ciliary dyskinesia (PCD) is a rare and heterogeneous genetic disorder that affects the structure and function of motile cilia. In the airway epithelium, impaired ciliary motion results in reduced or absent mucociliary clearance that leads to the appearance of chronic airway infection, sinusitis and bronchiectasis. Currently, there is no effective treatment for PCD, and research is limited by the lack of convenient models to study this disease and investigate innovative therapies. Furthermore, the high heterogeneity of PCD genotypes is likely to hinder the development of a single therapy for all patients. The generation of patient-derived induced pluripotent stem cells (iPSC) and their differentiation into airway epithelium as well as genome editing technologies could represent major tools for in vitro PCD modelling and for developing personalized therapies. Here, we review PCD pathogenesis, and then discuss how human iPSC could be used to model this disease for the development of innovative patient-specific biotherapies.
- Coexisting cystic lung disease as a rare extra-renal manifestation of autosomal dominant polycystic kidney disease. [Journal Article]
- RCRadiol Case Rep 2018; 13(5):1048-1052
- Autosomal dominant polycystic kidney disease (ADPKD) classically presents with multiple bilateral renal cysts and ultimately progresses to end stage renal disease. While many of the extra-renal manif...
Autosomal dominant polycystic kidney disease (ADPKD) classically presents with multiple bilateral renal cysts and ultimately progresses to end stage renal disease. While many of the extra-renal manifestations of ADPKD are well-documented, associated pulmonary findings are particularly rare, having only been recently been reported in a handful of studies to date. A 69-year-old female with ADPKD presented to our hospital with respiratory complaints. High resolution computed tomography revealed bronchiectasis, cystic lung disease, and interstitial fibrosis. The patient did not have concurrent risk factors or coexisting disease processes to explain the etiology of her airway and cystic lung disease, which we suggest are manifestations of ADPKD. We have not found a previous report of interstitial lung disease in this setting.
- Case of Missing Plastic: Foreign Body Bronchiectasis. [Journal Article]
- CCureus 2018 Jul 12; 10(7):e2974
- Bronchiectasis is a well-known entity where the airways abnormally dilate losing their natural function. Most common causes of non-cytic fibrosis bronchiectasis in the middle age group include second...
Bronchiectasis is a well-known entity where the airways abnormally dilate losing their natural function. Most common causes of non-cytic fibrosis bronchiectasis in the middle age group include secondary immunodeficiency, aspiration, and allergic bronchopulmonary aspergillosis (ABPA). Obstructive foreign body is an uncommon cause of bronchiectasis and is often a missed diagnosis in a localized disease. Foreign bodies can be missed making the diagnosis and treatment more challenging and hence foreign body bronchiectasis should be considered in patients presenting with focal disease. Here we describe a patient with a retained foreign body that was discovered post lobectomy during gross pathological examination of the specimen with no significant aspiration history, non-diagnostic imaging of the chest and negative bronchoscopy.
- Clinical & laboratory features of seventy-eight UK patients with Good's syndrome (thymoma & hypogammaglobulinemia). [Journal Article]
- CEClin Exp Immunol 2018 Sep 14
- Good's syndrome (thymoma and hypogammaglobulinemia) is a rare secondary immunodeficiency disease, previously reported in the published literature as mainly individual cases or small case series. We u...
Good's syndrome (thymoma and hypogammaglobulinemia) is a rare secondary immunodeficiency disease, previously reported in the published literature as mainly individual cases or small case series. We use the national UK-Primary Immune Deficiency (UKPID) registry to identify a large cohort of patients in the UK with this PID to review its clinical course, natural history and prognosis. Clinical information, laboratory data, treatment and outcome were collated and analysed. Seventy-eight patients with a median age of 64 years, 59% of whom were females were reviewed. Median age of presentation was 54 years. Absolute B cell numbers and serum immunoglobulins were very low in all patients and all received immunoglobulin replacement therapy. All patients had undergone thymectomy and 9 (12%) had thymic carcinoma (four locally invasive and five had disseminated disease) requiring adjuvant radiotherapy and/or chemotherapy. CD4 T-cells were significantly lower in these patients with malignant thymoma. Seventy-four (95%) presented with infections, 35 (45%) had bronchiectasis, seven (9%) chronic sinusitis, but only eight (10%) had serious invasive fungal or viral infections. Patients with AB-type thymomas were more likely to have bronchiectasis. Twenty (26%) suffered from autoimmune diseases (pure red cell aplasia, hypothyroidism, arthritis, myasthenia gravis, SLE, Sjogren's syndrome). There was no association between thymoma type and autoimmunity. Seven (9%) patients had died. Good's syndrome is associated with significant morbidity relating to infectious and autoimmune complications. Prospective studies are required to understand why some patients with thymoma develop persistent hypogammaglobulinemia. This article is protected by copyright. All rights reserved.
- Amikacin Liposome Inhalation Suspension for Treatment-Refractory Lung Disease Caused by Mycobacterium avium Complex (CONVERT): A Prospective, Open-Label, Randomized Study. [Journal Article]
- AJAm J Respir Crit Care Med 2018 Sep 14
- Rationale Improved therapeutic options are needed for patients with treatment-refractory nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex (MAC). Objectives To evaluate ...
Rationale Improved therapeutic options are needed for patients with treatment-refractory nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex (MAC). Objectives To evaluate the efficacy and safety of daily amikacin liposome inhalation suspension (ALIS) added to standard guideline-based therapy (GBT) in patients with refractory MAC lung disease. Methods Adults with amikacin-susceptible MAC lung disease and MAC-positive sputum cultures despite ≥6 months of stable GBT were randomly assigned (2:1) to receive ALIS with GBT (ALIS+GBT) or GBT alone. Once-daily ALIS was supplied in single-use vials delivering 590 mg amikacin to the nebulizer. The primary endpoint was culture conversion, defined as 3 consecutive monthly MAC-negative sputum cultures by month 6. Measurements and Main Results Enrolled patients (ALIS+GBT, n=224; GBT-alone, n=112) were a mean 64.7 years old and 69.3% female. Most had underlying bronchiectasis (62.5%), chronic obstructive pulmonary disease (14.3%), or both (11.9%). Culture conversion was achieved by 65 of 224 patients (29.0%) with ALIS+GBT and 10 of 112 (8.9%) with GBT alone (OR, 4.22; 95% CI [2.08, 8.57]; P<0.001). Patients in the ALIS+GBT arm vs GBT alone were more likely to achieve conversion (hazard ratio, 3.90; 95% CI, [2.00, 7.60]). Respiratory adverse events (primarily dysphonia, cough, and dyspnea) were reported in 87.4% of patients receiving ALIS+GBT and 50.0% receiving GBT alone; serious treatment-emergent adverse events occurred in 20.2% and 17.9% of patients, respectively. Conclusions Addition of ALIS to GBT for treatment-refractory MAC lung disease achieved significantly greater culture conversion by month 6 than GBT alone, with comparable rates of serious adverse events. Clinical trial registration available at www.clinicaltrials.gov, ID NCT02344004.
- Advances in bronchiectasis: endotyping, genetics, microbiome, and disease heterogeneity. [Review]
- LctLancet 2018 Sep 08; 392(10150):880-890
- Bronchiectasis is characterised by pathological dilation of the airways. More specifically, the radiographic demonstration of airway enlargement is the common feature of a heterogeneous set of condit...
Bronchiectasis is characterised by pathological dilation of the airways. More specifically, the radiographic demonstration of airway enlargement is the common feature of a heterogeneous set of conditions and clinical presentations. No approved therapies exist for the condition other than for bronchiectasis caused by cystic fibrosis. The heterogeneity of bronchiectasis is a major challenge in clinical practice and the main reason for difficulty in achieving endpoints in clinical trials. Recent observations of the past 2 years have improved the understanding of physicians regarding bronchiectasis, and have indicated that it might be more effective to classify patients in a different way. Patients could be categorised according to a heterogeneous group of endotypes (defined by a distinct functional or pathobiological mechanism) or by clinical phenotypes (defined by relevant and common features of the disease). In doing so, more specific therapies needed to effectively treat patients might finally be developed. Here, we describe some of the recent advances in endotyping, genetics, and disease heterogeneity of bronchiectasis including observations related to the microbiome.
- Bronchiectasis in children: diagnosis and treatment. [Review]
- LctLancet 2018 Sep 08; 392(10150):866-879
- Bronchiectasis is conventionally defined as irreversible dilatation of the bronchial tree. Bronchiectasis unrelated to cystic fibrosis is an increasingly appreciated cause of chronic respiratory-rela...
Bronchiectasis is conventionally defined as irreversible dilatation of the bronchial tree. Bronchiectasis unrelated to cystic fibrosis is an increasingly appreciated cause of chronic respiratory-related morbidity worldwide. Few randomised controlled trials provide high-level evidence for management strategies to treat the children affected by bronchiectasis. However, both decades-old and more recent studies using technological advances support the notion that prompt diagnosis and optimal management of paediatric bronchiectasis is particularly important in early childhood. Although considered to be of a non-reversible nature, mild bronchiectasis determined by radiography might be reversible at any age if treated early, and the lung function decline associated with disease progression could then be halted. Although some management strategies are extrapolated from cystic fibrosis or adult-based studies, or both, non-cystic fibrosis paediatric-specific data to help diagnose and manage these children still need to be generated. We present current knowledge and an updated definition of bronchiectasis, and review controversies relating to the management of children with bronchiectasis, including applying the concept of so-called treatable traits.
- Lung volume reduction with endobronchial coils for patients with emphysema. [Review]
- JTJ Thorac Dis 2018; 10(Suppl 23):S2797-S2805
- The lung volume reduction coil treatment is a minimally invasive bronchoscopic treatment option for emphysema patients who suffer from severe hyperinflation. The treatment is aimed at a large group o...
The lung volume reduction coil treatment is a minimally invasive bronchoscopic treatment option for emphysema patients who suffer from severe hyperinflation. The treatment is aimed at a large group of patients where lung volume reduction surgery and bronchoscopic lung volume reduction using endobronchial valves are no option, or alternatively, can be offered as a bridge to lung transplantation. The nitinol coil exhibits a shape memory effect and is biologically inert. The lung volume reduction coil procedure is performed in two separate treatment sessions, targeting one lobe per session, with the contralateral lobe being treated 4 to 8 weeks after the first session. In one treatment session, around 10 to 14 coils, thereby treating an entire lobe, are being placed. Selecting optimally treated, symptomatic chronic obstructive pulmonary disease (COPD) patients with emphysema and severe hyperinflation, while avoiding significant airway disease such as asthma, chronic bronchitis and bronchiectasis, is key to achieve treatment success. Three randomized clinical trials investigating lung volume reduction coil treatment have been published until now, reporting the results of 452 treated patients up to 12 months after coil treatment. Lung volume reduction coil treatment results in significant improvement of pulmonary function outcomes and quality of life in patients with severe hyperinflation. The most common complications of lung volume reduction coil treatment are: COPD exacerbations, pneumonia, Coil Associated Opacity and an increased risk of pneumothorax. The purpose of this article is to describe the coil technique and review the available literature regarding effect, safety and future perspectives of lung volume reduction with coils for emphysema patients.
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- High-intensity inspiratory muscle training in bronchiectasis: A randomized controlled trial. [Journal Article]
- RRespirology 2018 Sep 12
- CONCLUSIONS: The H-IMT increased exercise capacity in patients with non-cystic fibrosis bronchiectasis. It has also positive effects on respiratory muscle strength and endurance, and social aspects of QOL.