Quiz and Survey for Remaining Barriers to Normalcy in Cystic Fibrosis, Volume 6 In each question there is only one correct answer. 1. Which one of the following statements is true regarding the bacteriophage treatment of multi-drug resistant infections in cystic fibrosis (CF)? a) These treatments are clearly life-saving b) In some, but not all patients, there are reports of decreases in the density of sputum bacteria and apparent improvement in patient health c) There are consistent reports of improvements in pulmonary infiltrates on chest radiographs of phage-treated CF patients d) The good thing about phage therapy for CF lung infections is that the current phages consistently work against all the multi-drug resistant organisms e) The efficacy of phage therapy in CF has been confirmed in a rigorous, double-blinded, randomized controlled clinical trial 2. Which one of the following statements regarding clinical trials for drug development in CF is true? a) A proof of concept trial is an example of a study that bridges a Phase I trial with a Phase II trial into a single study b) Phase IIb trials are typically shorter in duration than phase IIa trials c) Phase III trials are focused on safety and adverse reactions and not so much on efficacy d) Phase III trials, to be considered valid, must always be done in a randomized, blinded fashion and compared to a placebo, even if there are ethical concerns regarding the withholding of standard therapy e) The duration of survival of CF patients receiving chronic drug therapy in a controlled trial is a standard endpoint, because survival is the most important endpoint and the survival of CF patients is still very short 3. Which one of the following statements regarding sleep disordered breathing in cystic fibrosis children is true? a) Only 10% of children affected by CF report daytime sleepiness b) There is no evidence that obstructive sleep apnea in children with CF can lead to deteriorating school performance, failure to thrive or worsening lung function c) The prevalence of obstructive sleep apnea and associated nocturnal hypoxia in children with CF may be as high as 30% d) The level of reduction of FEV1 in CF children does not correlate with the number of nocturnal awakenings e) Overnight oximetry by itself has been validated for diagnosing sleep disordered breathing and for the diagnosis of hypoventilation during sleep in children with CF 4. Which one of the following statements regarding emerging technologies for the restoration of CF transmembrane conductance regulator CFTR is true? a) With the advent of triple therapy (such as elexacaftor/tezacaftor/ivacaftor) for abnormal CFTR, essentially 100% of the known mutations can be corrected b) Small molecule therapies have been proven to generate and increase CFTR protein production in CF patients with nonsense mutations c) In theory, mRNA therapies, like transfer RNA therapies, will likely benefit only patients with nonsense mutations d) Transfer RNAs have been utilized in vitro to bypass stop codons in CF airway epithelial cells so that full length CFTR proteins can be produced 5. Which one of the following statements regarding novel imaging technologies for CF patients is true? a) There are not health concerns about the cumulative exposure to ionizing radiation in CF patients who receive frequent regular dose computed tomographic examinations, because they have a shortened life expectancy b) The use of routine frequent chest radiographs in CF patients has been documented to improve outcomes c) Hyperpolarizing magnetic resonance imaging (MRI) holds the promise to develop into a useful mode of analysis of CF lung disease, because it is developing rapidly and can demonstrate both structural and functional features and it does not use ionizing radiation d) Proton magnetic resonance imaging may yet demonstrate greater resolution than a plain chest radiograph, but the level of radiation is still problematic 6. Which one of the following statements regarding non tuberculous mycobacterial pulmonary disease (NTM-PD) in CF is true? a) Over 90% of CF patients treated for NTM-PD achieve negative cultures with current therapies b) Current Cystic Fibrosis Foundation/European Cystic Fibrosis Society guidelines call for treatment of NTM-PD (that is sensitive to a macrolide antibiotic) with a macrolide (preferably azithromycin), rifampin and ethambutol c) NTM-PD caused by M Abcessus in CF persons is relatively responsive to therapy because the bacteria are slow growing and do not tend to acquire new mechanisms of drug resistance d) The continuation of therapy for M Abscessus is usually limited to 3 months e) Clarithromycin (as compared to azithromycin) is usually the preferred macrolide of choice in treating NTM-PD, because of its once daily dosing and its demonstration of fewer drug interactions 7. Which one of the following statements is true regarding enhancing care for individuals with advanced CF lung disease ACFLD? a) Because of improvements in outcomes only 2% of CF persons over the age of 30 years have an FEV1 less than 40% predicted b) CF children with FEV1 in the range of 40-50% are not thought to have more aggressive lung disease than CF adults with a similar FEV1 c) Nocturnal non-invasive ventilation to relieve chronic hypercarbia is recommended for all CF patients expected to receive lung transplantation d) The development of hypoxemia, hypercarbia or pulmonary hypertension are associated with poor outcomes in individuals with ACFLD e) As of 2019 the survival of CF individuals post lung transplantation, whether they were children or adults, was 10 years 8. Which one of the following statements is true regarding the topic of entering the era of highly effective modulator therapy for CF? a) Of the 2000 known variants of the CFTR gene, over 200 have an allele prevalence greater than 1% b) The currently available CFTR modulators are effective therapies for individuals with CF whose CFTR genes have premature stop codons c) Therapies directed to premature stop codons are more effective than therapies for mutations at the cell surface that have reduced function or are expressed in smaller amounts d) The currently available corrector and potentiator agents and their combinations for abnormal CFTR proteins are believed to be effective for 85% of CF individuals 9. Which one of the following statements regarding advances in the assessment of CF lung disease is true? a) Computed tomography can well assess CF lung disease in infants and because of advances, exposure to ionizing radiation is no longer a concern b) Body plethysmography done in CF infants diagnosed by newborn screening has failed to detect elevations in function residual capacity c) Studies of respiratory impedance impulse oscillometry in CF children ages 2-8 years have consistently shown elevations of impedance in these children compared with healthy controls d) Lung clearance index studies have not demonstrated treatment effects in pre-school and school-aged CF subjects e) Although the raised volume thoraco-abdominal compression technology replicates adult-type spirometry in infants, its use is limited by the requirement of sedation with chloral hydrate and the procedure is very time- and labor-intensive 10. Which one of the following statements regarding the promotion of emotional wellness in children with CF- child and family resilience- is true? a. The European Best Practice Guidelines do not recommend annual screening for anxiety and depressive symptoms for CF caregivers b. CF parental depressive symptoms are not known to be linked to CF children’s medical adherence, including the administration of pancreatic enzymes c. The witnessing of CF children by their parents being upset with treatments has not been associated with parental feelings of guilt and discomfort d. An example of healthy developmental stimulation to offset the increase of tasks by parents associated with CF care of infants/toddlers or an increase in the number of diaper changes or increased time with nebulizer treatments is to increase bonding through conversations and increased attention (bonding) with praise for positive behavior e. Children with CF are not usually aware of decreased life expectancy or an expectation of exacerbations and illness until adolescence 11. Which one of the following statements regarding the promotion of emotional wellness in children with CF- mental health assessment and intervention- is true? a) If a pre-adolescent with CF persistently complains of boredom, this is very likely not to be related to depression b) Female children with CF are just as likely as males with CF to be diagnosed with attention deficit with hyperactivity disorder ADHD c) The absorption of amphetamines given to treat ADHD can be increased by the co-administration of proton pump inhibitors to increase the activity of pancreatic enzymes d) Only 10% of CF children affected by ADHD have behavioral feeding problems such as food selectivity and feeding rituals How much do you agree with the following statements: (1 = Strongly Disagree, 5= Strongly Agree)The supplement enhanced my knowledge on the subject. 1) Strongly disagree 2) Somewhat disagree 3) Neither agree or disagree 4) Somewhat agree 5) Strongly agree The supplement was relevant to my practice. 1) Strongly disagree 2) Somewhat disagree 3) Neither agree or disagree 4) Somewhat agree 5) Strongly agree The overall quality of the articles in the supplement were satisfactory. 1) Strongly disagree 2) Somewhat disagree 3) Neither agree or disagree 4) Somewhat agree 5) Strongly agree Do you have any general comments or recommendations for future topics to be covered in future editions of this supplement series?What behavioral changes do you expect to make in your own practice based on reading the articles in this supplement?Pediatric Pulmonology would like to determine how readers changed their behavior and adopted into practice what they learned by reading the articles in this supplement.Pediatric Pulmonology would like to determine how readers changed their behavior and adopted into practice what they learned by reading the articles in this supplement. 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