Highlights from the 2016 North American Cystic Fibrosis Conference

Zemanick, Edith T.; Daines, Cori; Dellon, Elisabeth P.; Esther, Charles R.; Kinghorn, BreAnna; Ong, Thida; Muhlebach, Marianne S.

doi:10.1002/ppul.23707

Cited by 10 publications

(7 citation statements)

References 20 publications

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“…More specifically, it has been shown that they consume less carbohydrates, proteins, different vitamins and omega 3 fatty acids than healthy subjects (40,41). In addition, those patients with CF also add malabsorption due to the associated impairment in exocrine pancreatic function (42). A relevant symptom that frequently appears in chronic respiratory diseases is anorexia (43)(44)(45), defined as a reduction or loss of appetite.…”

Section: Inappropriate Diet Anorexia and Malabsorptionmentioning

confidence: 99%

Nutritional status and muscle dysfunction in chronic respiratory diseases: stable phase versus acute exacerbations

Gea¹,

Sancho-Muñoz²,

Chalela³

2018

J. Thorac. Dis.

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Nutritional abnormalities are frequent in different chronic respiratory diseases such as chronic obstructive pulmonary disease (COPD), bronchiectasis, cystic fibrosis (CF), interstitial fibrosis and lung cancer, having important clinical consequences. However, nutritional abnormalities often remained underdiagnosed due to the relative lack of awareness of health professionals. Therefore, systematic anthropometry or even better, assessment of body composition, should be performed in all patients with chronic respiratory conditions, especially following exacerbation periods when malnutrition becomes more accentuated. Nutritional abnormalities very often include the loss of muscle mass, which is an important factor for the occurrence of muscle dysfunction. The latter can be easily detected with the specific assessment of muscle strength and endurance, and also negatively influences patients' quality of life and prognosis. Both nutritional abnormalities and muscle dysfunction result from the interaction of several factors, including tobacco smoking, low physical activity-sedentarism, systemic inflammation and the imbalance between energy supply and requirements, which essentially lead to a negative balance between protein breakdown and synthesis. Therapeutic approaches include improvements in lifestyle, nutritional supplementation and training. Anabolic drugs may be administered in some cases.

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Section: Inappropriate Diet Anorexia and Malabsorptionmentioning

confidence: 99%

Nutritional status and muscle dysfunction in chronic respiratory diseases: stable phase versus acute exacerbations

Gea¹,

Sancho-Muñoz²,

Chalela³

2018

J. Thorac. Dis.

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“…Most important is the coproduction with our parent partners in developing our relatable approaches to introduce MHS to families and revising implementation tools and processes 18 . Moreover, the use of electronic screening tools is an important strategy for engaging AYA, with evidence of enhancing disclosure of sensitive information (eg, psychological symptoms) 19 …”

Section: Discussionmentioning

confidence: 99%

Implementation strategies for depression and anxiety screening in a pediatric cystic fibrosis center: A quality improvement project

Liu

Lew

Andes

et al. 2020

Pediatric Pulmonology

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Background Guidelines to integrate mental health screening (MHS) into Cystic fibrosis (CF) care has increased adoption across the United States and European CF Centers, however, there has been limited discussion on specific strategies for implementation. Objective Share key strategies that led to successful MHS implementation in one pediatric CF center and report implementation and screening outcomes. Methods Parent partners, mental health experts from the Department of Psychiatry, and the CF clinic team codesigned interventions to support three major stakeholders: (a) patients and families (b) clinic team, and (c) hospital system. The mental health coordinator approached patients (12 and older) to introduce MHS and administered screening and reviewed results using an electronic tablet and digital measurement‐feedback system. We used strategies that promoted visibility of screening progress and sharing of data with hospital administration. Descriptive statistics were used to assess prevalence of clinically significant symptoms of anxiety and depression and symptom severity within our sample. Results Over the first 2 years of the project, we exceeded our goal of screening 80% of eligible patients per year (80%‐95% screened) and are on a similar trajectory within the first 7 months of year 3. We identified high prevalence of clinically significant symptoms of depression (16%) and anxiety (14%) similar to those found in other chronic illness populations. These data helped us advocate hospital leadership to support the development of new mental health services to address identified needs at our center. Conclusion Leveraging coproduction to address stakeholder needs led to successful implementation of a sustainable MHS process.

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“…The potentiator GLPG1837 was tested in adults with an FEV 1% predicted of !40% and at least one G551D mutation or one S1251N mutation. 42,43 QBW251 has shown promise as a new potentiator in both patients with chronic obstructive pulmonary disease (COPD) and patients with CF with residual function mutations. 44,45 However, phase 3 clinical trials were not done with these compounds as they are being tested in a trajectory toward triple-combination therapy for subjects with F508del mutations.…”

Section: Triple Combination Therapymentioning

confidence: 99%

“…48 Amplifier Plus Third-Generation Corrector Plus Potentiator CFTR amplifiers are mutation-agnostic compounds that increase the efficiency of translation from mRNA to protein by facilitating the docking of the nascent CFTR protein to the translocon through which the protein is released in the endoplasmic reticulum, thereby also decreasing mRNA decay. 43,47,49,50 More CFTR protein means more substrate for other CFTR modulators. Amplifier PTI-428 was tested in F508del-HBEs and together with corrector PTI-801 and potentiator PTI-808 it led to wild-type levels of CFTR chloride current.…”

Section: Triple Combination Therapymentioning

confidence: 99%

Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis

Cuyx

Boeck

2019

Semin Respir Crit Care Med

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Detailed knowledge of how mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene disturb the trafficking or function of the CFTR protein and the use of high-throughput drug screens have allowed novel therapeutic strategies for cystic fibrosis (CF). The main goal of treatment is slowly but surely shifting from symptomatic management to targeting the underlying CFTR defect to halt disease progression and even to prevent occurrence of CF complications. CFTR potentiators for patients with class III mutations, mutation R117H (and in United States also for patients with specific residual function mutations) and the combination of a CFTR modulator plus a potentiator for patients homozygous for F508del, are the two classes of modulators that are in use in the clinic. Approval of these therapeutics has progressively expanded to include both younger patients and a wider range of CFTR mutations. For a significant proportion of patients with CF, current treatment is however still insufficient or unavailable.This review provides an overview of the clinical trial results and the real-life efficacy data of approved CFTR modulators. In addition, we discuss the entire pipeline of CFTR modulators: novel potentiators and correctors, amplifiers, stabilizers, and read-through agents. Furthermore, we discuss other strategies to improve CFTR function like nonsense-mediated decay inhibitors, modified transfer ribonucleic acids, antisense oligonucleotides, and genetic therapies.CFTR modulators are already changing the face of CF and the pipeline of new therapies continues to be exciting.

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Highlights from the 2016 North American Cystic Fibrosis Conference

Cited by 10 publications

References 20 publications

Nutritional status and muscle dysfunction in chronic respiratory diseases: stable phase versus acute exacerbations

Nutritional status and muscle dysfunction in chronic respiratory diseases: stable phase versus acute exacerbations

Implementation strategies for depression and anxiety screening in a pediatric cystic fibrosis center: A quality improvement project

Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis

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