CFTR modulators: transformative therapies for cystic fibrosis

Dwight, Mary; Marshall, Bruce C.

doi:10.18553/jmcp.2021.27.2.281

Cited by 7 publications

(7 citation statements)

References 4 publications

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“…Many PwCF started this treatment during 2020 at the start of the iLab. This treatment led to significant decline in exacerbation rates and improved lung function 20. The pandemic further influenced exacerbation rates, with multiple PwCF experiencing less exacerbations due to reduced social interactions and decreased exposure to all respiratory viruses, including COVID-19 21.…”

Section: Discussionmentioning

confidence: 99%

Improved recognition of lung function decline as signal of cystic fibrosis pulmonary exacerbation: a Cystic Fibrosis Learning Network Innovation Laboratory quality improvement initiative

List,

Solomon,

Bichl

et al. 2023

BMJ Open Qual

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IntroductionCystic fibrosis (CF) is a systemic autosomal recessive condition characterised by progressive lung disease. CF pulmonary exacerbations (PEx) are episodes of worsening respiratory status, and frequent PEx are a risk factor for accelerated lung function decline, yet many people with CF (PwCF) go untreated at the time of decline. The goal of this quality improvement (QI) initiative was to improve recognition, treatment and follow-up of PEx in PwCF.MethodsUsing the Model for Improvement, the Cystic Fibrosis Learning Network (CFLN) initiated a QI innovation laboratory (iLab) with a global aim to decrease the rate of lung function decline in PwCF. The iLab standardised definitions for signals of PEx using a threshold for decline in forced expiratory volume in one second (FEV1) and/or changes in symptoms. The FEV1decline signal was termed FIES (FEV1-indicated exacerbation signal). Processes for screening and recognition of FIES and/or symptom changes, a treatment algorithm and follow-up in the presence of a signal were tested concurrently in multiple settings.Specific aimsThe specific aim is to increase the per cent of PwCF assessed for a PEx signal at ambulatory encounters and to increase the per cent of recommendations to follow-up within 6 weeks for PwCF experiencing a PEx signal.ResultsFIES recognition increased from 18.6% to 73.4% across all teams during the iLab, and every team showed an improvement. Of PwCF assessed, 15.8% experienced an FIES event (>10% decline in FEV1per cent predicted (FEV1pp)). Follow-up within 6 weeks was recommended for an average of 70.5% of those assessed for FIES and had an FEV1pp decline greater than 5%.ConclusionThe CFLN iLab successfully defined and implemented a process to recognise and follow-up PEx signals. This process has the potential to be spread to the larger CF community. Further studies are needed to assess the impact of these processes on PwCF outcomes.

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Section: Discussionmentioning

confidence: 99%

Improved recognition of lung function decline as signal of cystic fibrosis pulmonary exacerbation: a Cystic Fibrosis Learning Network Innovation Laboratory quality improvement initiative

List,

Solomon,

Bichl

et al. 2023

BMJ Open Qual

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“…The expansion of telehealth in 2020 also came soon after the more widespread introduction of CFTR modulator therapies in the US in late 2019. This resulted in a rapid decrease in the number of hospitalizations reported in the US CFF Patient Registry, a decline that preceded the onset of the COVID-19 pandemic [13] . As inpatient care is one of the largest cost drivers in CF care [5] , a reduction in hospitalizations at the population level also could lead to financial changes for an institution to support a CF care program.…”

Section: Discussionmentioning

confidence: 99%

Financial impacts of the COVID-19 pandemic on cystic fibrosis care: lessons for the future

Sawicki

Citters

Dieni

et al. 2021

Journal of Cystic Fibrosis

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Background Chronic care delivery models faced unprecedented financial pressures, with a reduction of in-person visits and adoption of telehealth during the COVID-19 pandemic. We sought to understand the reported financial impact of pandemic-related changes to the cystic fibrosis (CF) care model. Methods The U.S. CF Foundation State of Care surveys fielded in Summer 2020 (SoC1) and Spring 2021 (SoC2) included questions for CF programs on the impact of pandemic-related restrictions on overall finances, staffing, licensure, and reimbursement of telehealth services. Descriptive analyses were conducted based on program type. Results Among the 286 respondents (128 pediatric, 118 adult, 40 affiliate), the majority (62%) reported a detrimental financial impact to their CF care program in SoC1, though fewer (42%) reported detrimental impacts in SoC2. The most common reported impacts in SoC1 were redeployment of clinical staff (68%), furloughs (52%), hiring freezes (51%), decreases in salaries (34%), or layoffs (10%). Reports of lower reimbursement for telehealth increased from 30% to 40% from SoC1 to SoC2. Projecting towards the future, only a minority (17%) of program directors in SoC2 felt that financial support would remain below pre-pandemic levels. Conclusions The COVID-19 pandemic resulted in financial strain on the CF care model, including challenges with reimbursement for telehealth services and reductions in staffing due to institutional changes. Planning for the future of CF care model needs to address these short-term impacts, particularly to ensure a lack of interruption in high-quality multi-disciplinary care.

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“…Das Nebenwirkungsprofil von Elexacaftor-Tezacaftor-Ivacaftor erwies sich als insgesamt akzeptabel, und als häufigste Nebenwirkungen wurden Blutdruckschwankungen, Anstieg der Transaminasen und der Kreatinkinase (CK) sowie Hautausschläge, insbesondere bei gleichzeitiger Einnahme hormoneller Kontrazeptiva, beschrieben [ 29 ]. In einer Arbeit von Dwight und Marshall aus den USA konnte eindrücklich gezeigt werden, dass die Therapie der Grunderkrankung den besten ABS-Ansatz bei der CF darstellt [ 31 ]. Durch den Einsatz der Medikamente bei Patienten mit CF ab 12 Jahren konnte die Exazerbationsrate seit 2019 deutlich reduziert werden (Abb.…”

Section: Antibiotic Stewardship Bei Zystischer Fibroseunclassified

Antibiotic Stewardship und chronische Lungenerkrankungen

2022

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Hintergrund Die Therapie akuter Exazerbationen von Patienten mit chronischen Lungenerkrankungen ist häufig mit der Gabe von Antibiotika vergesellschaftet. Wiederholte stationäre Aufenthalte, hohe Antibiotikaverbräuche sowie die Gefahr des Auftretens multiresistenter Keime stellen eine Belastung für das Gesundheitssystem dar. Ziel dieser Arbeit Ziel dieser Arbeit ist es, einen Überblick über Antibiotic-Stewardship-Strategien für das Management von Patienten mit chronischen Lungenerkrankungen mit besonderem Fokus auf COPD („chronic obstructive pulmonary disease“) sowie Bronchiektasien und zystischer Fibrose (CF) zu geben. Material und Methoden Die Übersichtsarbeit basiert auf einer Literaturrecherche in PubMed. Ergebnisse und Diskussion Point-of-Care-Tests (POCT) zum schnelleren Erregernachweis ergaben uneinheitliche Ergebnisse in Bezug auf die Reduktion antibiotischer Therapien. Als relativ einfaches Verfahren konnte mittels POCT C‑reaktives Protein (CRP) („cut off“ < 20 bzw. ≥ 40 mg/l) die Anwendung von Antibiotika bei Patienten mit exazerbierter COPD reduzieren, ohne die Sicherheit zu gefährden. Die Rolle von Procalcitonin bleibt unklar. Bei nichtpurulentem Sputum und einer leichten bis mittelschweren COPD-Exazerbation kann Leitliniengerecht auf eine Antibiotikatherapie verzichtet werden. Der Einsatz von Makroliden führt zu einer zunehmenden Resistenzentwicklung und birgt die Gefahr v. a. kardiovaskulärer Nebenwirkungen, sodass eine Dauertherapie bei Patienten trotz häufiger COPD-Exazerbationen kritisch gesehen werden muss. Bei der CF hat sich durch den Einsatz von CFTR(„cystic fibrosis transmembrane conductance regulator“)-Modulatoren das Management der Grunderkrankung so verbessert, dass weniger Antibiotikatherapien gebraucht wurden. Auch bei den Bronchiektasen gibt es Anlass zur Hoffnung, dass durch neue Therapien, wie z. B. Inhibitoren der neutrophilen Elastase, Antibiotika eingespart werden können. Jeder Einsatz von Antibiotika bei chronischen Lungenerkrankungen sollte leitliniengerecht erfolgen.

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CFTR modulators: transformative therapies for cystic fibrosis

Cited by 7 publications

References 4 publications

Improved recognition of lung function decline as signal of cystic fibrosis pulmonary exacerbation: a Cystic Fibrosis Learning Network Innovation Laboratory quality improvement initiative

Improved recognition of lung function decline as signal of cystic fibrosis pulmonary exacerbation: a Cystic Fibrosis Learning Network Innovation Laboratory quality improvement initiative

Financial impacts of the COVID-19 pandemic on cystic fibrosis care: lessons for the future

Antibiotic Stewardship und chronische Lungenerkrankungen

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