Plastic bronchitis or cast bronchitis is a rare disease of unclear etiology characterized by formation of airway casts that can lead to life-threatening airway obstruction. There is currently limited data regarding optimal treatment of plastic bronchitis. Several therapies have been suggested, but recurrences are common and mortality remains high. We report the case of a 6-year-old boy with refractory eosinophilic bronchial casts, unresponsive to low-dose systemic corticosteroids, inhaled corticosteroids, azithromycin, and dornase alfa, who was treated successfully and safely with direct instillation of tissue-type plasminogen activator (tPA) to the obstructing casts during flexible bronchoscopy and inhaled tPA. Our case illustrates that the current therapy for plastic bronchitis remains inadequate. To our knowledge, this case is the first to show that direct instillation of tPA can be used safely for treatment of this disease. The use of tPA via direct administration into the airways during bronchoscopy and via a nebulizer appeared to be a safe and effective therapy for plastic bronchitis and should be considered early in the course of the disease to prevent complications of severe airway obstruction.

No abstract

Functional profiling of CFTR-directed therapeutics offers the potential to provide significant benefits to young people with cystic fibrosis (CF). However, the development of 2D airway epithelial cell models for individual response tests in CF children remains a central task. The objective of this study was to determine the utility of EpiX TM technology for expansion of nasal epithelial cells for use in electrophysiological CFTR function measurements. An initial harvest of as few as 20,000 cells was sufficient to expand up to 50 million cells that were used to generate air-liquid interface (ALI) cultures for ion transport studies with the Ussing assay. CFTR function was assessed by measuring responses to forskolin and the CFTR potentiator VX-770 (ivacaftor) in ALI cultures generated from passage 3 and 4 cells. Short-circuit current (Isc) measurements of blocked CFTR currents (I CFTRinh) discriminated CFTR function between healthy control (wild type, WT) and patients with intermediate (F508del/R117H-7T: 56% WT) and severe (F508del/F508del: 12% WT) CF disease. For the mixed genotypes, CFTR activity for F508del/c.850dupA was 12% WT, R334W/406-1G>A was 24% WT, and CFTRdele2,3(21 kb)/CFTRdele2,3(21 kb) was 9% WT. The CFTR correctors VX-809 (lumacaftor) and VX-661 (tezacaftor) significantly increased CFTR currents for F508del/R117H to 73 and 67% WT, respectively. Cultures with the large deletion mutation CFTRdele2,3(21 kb) unexpectedly responded to VX-661 treatment (20% WT). Amiloride-sensitive sodium currents were robust and ranged between 20-80 µA/cm 2 depending on the subject. In addition to characterizing the electrophysiological profile of mutant CFTR activity in cultures for five genotypes, our study exemplifies the promising paradigm of bed-to-bench side cooperation and personalized medicine.

In response to the novel coronavirus (COVID-19) pandemic, all in-person cystic fibrosis (CF) appointments were converted to telemedicine visits at UCSF Benioff Children's Hospital. The purpose of our study was to learn about the experiences that patients, families, and providers had with telemedicine visits and to assess their interest in using telemedicine in the future. Our hypothesis was that most patients, families, and providers want to continue telemedicine visits in the future. An anonymous 11-question survey was distributed to patients, families, and providers in November and December 2020. The survey was completed by 46 of 72 families (64% response rate) and 24 of 25 providers (96% response rate). Thirty-seven families (80%) and 21 providers (88%) were satisfied with their telemedicine experience. Thirty-three families (72%) want to have telemedicine visits in the future. Thirty-five families (76%) and 22 providers (92%) were satisfied with their experience using Zoom. Forty families (87%) and 19 providers (90%) want 2 or more visits each year to be via telemedicine. Our study showed that most families and providers were satisfied with telemedicine, would like to continue using telemedicine, and prefer to have at least 2 of the 4 recommended annual CF visits via telemedicine. Our survey identified the following benefits to telemedicine: decreased travel time, decreased cost, and avoiding exposure to COVID. However, we need to ensure that we do not exacerbate existing health disparities for families that do not speak English and/or do not have the internet capabilities to support telemedicine technology.

Background: The COVID-19 pandemic impacted many households due to shelter-in-place orders and economic hardship. People with cystic fibrosis (CF) experienced increased food insecurity compared to the general population before the pandemic, even though adequate food access is needed to maintain nutrition goals associated with improved health-related outcomes. Little is known about the impact the pandemic had on the food insecurity of people with CF and their families.Objective: To investigate how the COVID-19 pandemic impacted food insecurity, mental health, and self-care in people with CF.Methods: Adults with CF and parents/guardians of children with CF were recruited via social media to complete online questionnaires from May 2020 to February 2021. Questionnaires in English and Spanish included USDA 2-question food insecurity screening, Patient Health Questionnaire-4 for mental health screening, and directed questions on the impact of the pandemic.Results: Of 372 respondents, 21.8% of the households experienced food insecurity during the pandemic compared to 18.8% prepandemic (p < .001). More food insecure patients with CF reported weight loss (32.1% vs. 13.1%, p < .001), worse airway clearance adherence (13.6% vs. 5.8%, p < .01), and worse medication adherence (12.4% vs. 1.7%, p < .01) compared to food secure patients.Food insecure subjects were more likely to have an abnormal mental health screen compared to food secure subjects (53.1% vs. 16.2%, p < .001). Conclusion:Food insecurity increased in the CF population during the COVID-19 pandemic. Food insecure subjects reported worse mental health and self-care during the pandemic compared to food secure subjects.

No abstract

No abstract

No abstract