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1.
Cystic fibrosis foundation consensus statements for the care of cystic fibrosis lung transplant recipients.
Shah, P, Lowery, E, Chaparro, C, Visner, G, Hempstead, SE, Abraham, J, Bhakta, Z, Carroll, M, Christon, L, Danziger-Isakov, L, et al
The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation. 2021;(7):539-556
Abstract
Cystic fibrosis (CF) is the indication for transplantation in approximately 15% of recipients worldwide, and Cystic Fibrosis Lung Transplant Recipients (CFLTRs) have excellent long-term outcomes. Yet, CFLTRs have unique comorbidities that require specialized care. The objective of this document is to provide recommendations to CF and lung transplant clinicians for the management of perioperative and underlying comorbidities of CFLTRs and the impact of transplantation on these comorbidities. The Cystic Fibrosis Foundation (CFF) organized a multidisciplinary committee to develop CF Lung Transplant Clinical Care Recommendations. Three workgroups were formed to develop focused questions. Following a literature search, consensus recommendations were developed by the committee members based on literature review, committee experience and iterative revisions, and in response to public comment. The committee formulated 32 recommendation statements in the topics related to infectious disease, endocrine, gastroenterology, pharmacology, mental health and family planning. Broadly, the committee recommends close coordination of care between the lung transplant team, the cystic fibrosis care center, and specialists in other disciplines with experience in the care of CF and lung transplant recipients. These consensus statements will help lung transplant providers care for CFLTRs in order to improve post-transplant outcomes in this population.
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2.
Novel imaging techniques for cystic fibrosis lung disease.
Goralski, JL, Stewart, NJ, Woods, JC
Pediatric pulmonology. 2021;(Suppl 1):S40-S54
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Abstract
With an increasing number of patients with cystic fibrosis (CF) receiving highly effective CFTR (cystic fibrosis transmembrane regulator protein) modulator therapy, particularly at a young age, there is an increasing need to identify imaging tools that can detect and regionally visualize mild CF lung disease and subtle changes in disease state. In this review, we discuss the latest developments in imaging modalities for both structural and functional imaging of the lung available to CF clinicians and researchers, from the widely available, clinically utilized imaging methods for assessing CF lung disease-chest radiography and computed tomography-to newer techniques poised to become the next phase of clinical tools-structural/functional proton and hyperpolarized gas magnetic resonance imaging (MRI). Finally, we provide a brief discussion of several newer lung imaging techniques that are currently available only in selected research settings, including chest tomosynthesis, and fluorinated gas MRI. We provide an update on the clinical and/or research status of each technique, with a focus on sensitivity, early disease detection, and possibilities for monitoring treatment efficacy.
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3.
CFTR targeted therapies: recent advances in cystic fibrosis and possibilities in other diseases of the airways.
Patel, SD, Bono, TR, Rowe, SM, Solomon, GM
European respiratory review : an official journal of the European Respiratory Society. 2020;(156)
Abstract
Cystic fibrosis transmembrane conductance regulator (CFTR) is an ion transporter that regulates mucus hydration, viscosity and acidity of the airway epithelial surface. Genetic defects in CFTR impair regulation of mucus homeostasis, causing severe defects of mucociliary clearance as seen in cystic fibrosis. Recent work has established that CFTR dysfunction can be acquired in chronic obstructive pulmonary disease (COPD) and may also contribute to other diseases that share clinical features of cystic fibrosis, such as asthma, allergic bronchopulmonary aspergillosis and bronchiectasis. Protean causes of CFTR dysfunction have been identified including cigarette smoke exposure, toxic metals and downstream effects of neutrophil activation pathways. Recently, CFTR modulators, small molecule agents that potentiate CFTR or restore diminished protein levels at the cell surface, have been successfully developed for various CFTR gene defects, prompting interest in their use to treat diseases of acquired dysfunction. The spectrum of CFTR dysfunction, strategies for CFTR modulation, and candidate diseases for CFTR modulation beyond cystic fibrosis will be reviewed in this manuscript.
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Rational use of mucoactive medications to treat pediatric airway disease.
Linssen, RSN, Ma, J, Bem, RA, Rubin, BK
Paediatric respiratory reviews. 2020;:8-14
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Abstract
Many airway diseases in children, notably bronchiolitis, cystic fibrosis (CF), non-CF bronchiectasis including primary ciliary dyskinesia, pneumonia, and severe asthma are associated with retention of airway secretions. Medications to improve secretions clearance, the mucoactive medications, are employed to treat these diseases with varying degrees of success. This manuscript reviews evidence for the use of these medications and future directions of study.
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Iron and Sphingolipids as Common Players of (Mal)Adaptation to Hypoxia in Pulmonary Diseases.
Ottolenghi, S, Zulueta, A, Caretti, A
International journal of molecular sciences. 2020;(1)
Abstract
Hypoxia, or lack of oxygen, can occur in both physiological (high altitude) and pathological conditions (respiratory diseases). In this narrative review, we introduce high altitude pulmonary edema (HAPE), acute respiratory distress syndrome (ARDS), Chronic Obstructive Pulmonary Disease (COPD), and Cystic Fibrosis (CF) as examples of maladaptation to hypoxia, and highlight some of the potential mechanisms influencing the prognosis of the affected patients. Among the specific pathways modulated in response to hypoxia, iron metabolism has been widely explored in recent years. Recent evidence emphasizes hepcidin as highly involved in the compensatory response to hypoxia in healthy subjects. A less investigated field in the adaptation to hypoxia is the sphingolipid (SPL) metabolism, especially through Ceramide and sphingosine 1 phosphate. Both individually and in concert, iron and SPL are active players of the (mal)adaptation to physiological hypoxia, which can result in the pathological HAPE. Our aim is to identify some pathways and/or markers involved in the physiological adaptation to low atmospheric pressures (high altitudes) that could be involved in pathological adaptation to hypoxia as it occurs in pulmonary inflammatory diseases. Hepcidin, Cer, S1P, and their interplay in hypoxia are raising growing interest both as prognostic factors and therapeutical targets.
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Weight Gain and Growth After Fundoplication in the Pediatric Patient: A Case Report in the Pediatric Patient With Cystic Fibrosis and Literature Review.
Gomes, A, Lapin, C, Ziegler, J
Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition. 2020;(4):745-754
Abstract
Gastroesophageal reflux (GER) is common among infants, but when symptoms become troublesome, that is defined as gastroesophageal reflux disease (GERD). Making a diagnosis of GERD is difficult because there is no gold standard. GERD can be especially problematic for infants with cystic fibrosis (CF). There are signs and symptoms (ie, malnutrition, recurring aspiration pneumonias, refusal to eat, wheezing, coughing, and asthma) in addition to invasive and noninvasive methods used to determine a diagnosis. The treatments for GERD span from nonpharmacological to surgical, with a laparoscopic Nissen fundoplication being the gold standard of surgical intervention. Although surgical interventions have been demonstrated to reduce symptoms associated with GERD, there is little known about the weight/growth-related outcomes. This case report discusses an infant with CF and GERD requiring multiple interventions and ultimately a laparoscopic Nissen fundoplication and the weight changes from presurgery to postsurgery. The case report is expanded upon with a structured literature review of fundoplication and growth studies. There were 4 available studies that assessed changes in weight/growth before and after fundoplication among children with GERD. Because of the heterogeneity of the nutrition outcomes used, the weight/growth benefits after fundoplication are unclear at this time. Further research in needed to assess the nutrition outcomes among patients with GERD requiring fundoplication.
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Cystic fibrosis bone disease treatment: Current knowledge and future directions.
Putman, MS, Anabtawi, A, Le, T, Tangpricha, V, Sermet-Gaudelus, I
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2019;:S56-S65
Abstract
Bone disease is a frequent complication in adolescents and adults with cystic fibrosis (CF). Early detection and monitoring of bone mineral density and multidisciplinary preventive care are necessary from childhood through adolescence to minimize CF-related bone disease (CFBD) in adult CF patients. Approaches to optimizing bone health include ensuring adequate nutrition, particularly intake of calcium and vitamins D and K, addressing other secondary causes of low bone density such as hypogonadism, encouraging weight bearing exercise, and avoiding bone toxic medications. Of the currently available anti-resorptive or anabolic osteoporosis medications, only bisphosphonates have been studied in individuals with CF. Future studies are needed to better understand the optimal approach for managing CFBD.
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[Diagnostic possibilities of modern biochemical study of sputum from patients with cystic fibrosis (literature review).].
Kozlov, AV, Gusyakova, OA, Ereshchenko, AA, Khaliulin, AV
Klinicheskaia laboratornaia diagnostika. 2019;(1):24-28
Abstract
The review presents the pathobiochemical and molecular mechanisms of sputum formation in patients with cystic fibrosis associated with the pathophysiological features of the disease. Statistical data on the prevalence of this pathology in the world and in the Russian Federation are presented. The mechanisms of sputum formation and disorders of the mucociliary apparatus, leading to the accumulation of viscous bronchopulmonary secret in cystic fibrosis, are considered. The principles of the relationship between the rheological properties of sputum and the formation of inflammation in the lungs with the addition of a concomitant specific microflora in the bronchopulmonary system in patients with cystic fibrosis are presented. Describes the opportunities for biochemical studies of sputum of patients with this pathology: determining the activity of enzymes (myeloperoxidase), the content of proteinase inhibitors (α2-macroglobulin and α1-antitrypsin) and proinflammatory cytokines (IL-8 and TNFa), concentrations of iron and ferriferous proteins (lactoferrin and ferritin), which makes biochemical studies of sputum available, non-invasive, quick and cost-effective method of diagnosis, which can be widely used as an auxiliary laboratory method and makes it possible to use these metabolites as diagnostic markers to assess the severity of inflammation and infection of the lower respiratory tract and predict the development of respiratory complications in patients with cystic fibrosis.
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Hypoglycemia in cystic fibrosis: Prevalence, impact and treatment.
Moheet, A, Chan, CL, Granados, A, Ode, KL, Moran, A, Battezzati, A
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2019;:S19-S24
Abstract
Hypoglycemia is a common and feared complication of insulin therapy. As in type 1 and type 2 diabetes, people with cystic fibrosis related diabetes are also at risk for hypoglycemia related to insulin therapy. Spontaneous hypoglycemia is also common in patients with CF without diabetes, who are not on glucose lowering medications. Spontaneous hypoglycemia in CF may also occur during or after an oral glucose tolerance test. In this review, we will discuss the definition, epidemiology, pathophysiology and impact of hypoglycemia, with a focus on people with cystic fibrosis. We will also review strategies to manage and prevent hypoglycemia.
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10.
Vitamin D deficiency and its treatment in cystic fibrosis.
Daley, T, Hughan, K, Rayas, M, Kelly, A, Tangpricha, V
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2019;:S66-S73
Abstract
Vitamin D deficiency is a common finding in individuals with cystic fibrosis (CF), despite routine supplementation. Hypovitaminosis D is often the result of fat malabsorption, but other contributors include increased latitude, poor nutritional intake, decreased sun exposure, impaired hydroxylation of vitamin D, and non-adherence to the prescribed vitamin D regimen. Vitamin D is critical for calcium homeostasis and optimal skeletal health, and vitamin D deficiency in CF can lead to skeletal complications of osteopenia and osteoporosis. Over time, our understanding of treatment regimens for vitamin D deficiency in CF has evolved, leading to recommendations for higher doses of vitamin D to achieve target levels of circulating 25-hydroxyvitamin D. There is also some evidence that vitamin D deficiency may have non-skeletal consequences such as an increase in pulmonary exacerbations. The exact mechanisms involved in the non-skeletal complications of vitamin D deficiency are not clearly understood, but may involve the innate immune system. Future clinical studies are needed to help address whether vitamin D has a role in CF beyond skeletal health.