Impact of the COVID-19 pandemic: How our response is shaping the future of cystic fibrosis care.

The findings of this body of work are presented in the eight articles included in this supplement. The impact and perspectives of adult and pediatric care teams and patient/families are covered with special attention to mental health care, the financial and personnel impacts within care programs, the experiences of vulnerable and underrepresented patient populations, and implementation of remoting monitoring. Commentaries from colleagues provide a broader perspective, offering reflections on the findings and their implications regarding the future CF care model.

Survival and Lung Transplant Outcomes for Individuals With Advanced Cystic Fibrosis Lung Disease Living in the United States and Canada: An Analysis of National Registries.

BACKGROUND: Understanding how health outcomes differ for patients with advanced cystic fibrosis (CF) lung disease living in the United States compared with Canada has health policy implications. RESEARCH QUESTION: What are rates of lung transplant (LTx) and rates of death without LTx in the United States and Canada among individuals with FEV1 < 40% predicted? STUDY DESIGN AND METHODS: This was a retrospective population-based cohort study, 2005 to 2016, using the US CF Foundation, United Network for Organ Sharing, and Canadian CF registries. Individuals with CF and at least two FEV1 measurements < 40% predicted within a 5-year period, age ≥ 6 years, without prior LTx were included. Multivariable competing risk regression for time to death without LTx (LTx as a competing risk) and time to LTx (death as a competing risk) was performed. RESULTS: There were 5,899 patients (53% male) and 905 patients (54% male) with CF with FEV1 < 40% predicted living in the United States and Canada, respectively. Multivariable competing risk regression models identified an increased risk of death without LTx (hazard ratio [HR], 1.79; 95% CI, 1.52-2.1) and decreased LTx (HR, 0.66; 95% CI, 0.58-0.74) among individuals in the United States compared with Canada. More pronounced differences were seen in the patients in the United States with Medicaid/Medicare insurance compared with Canadians (multivariable HR for death without LTx, 2.24 [95% CI, 1.89-2.64]; multivariable HR for LTx, 0.54 [95% CI, 0.47-0.61]). Patients of nonwhite race were also disadvantaged (multivariable HR for death without LTx, 1.56 [95% CI, 1.32-1.84]; multivariable HR for LTx, 0.47 [95% CI, 0.36-0.62]). INTERPRETATION: There are lower rates of LTx and an increased risk of death without LTx for US patients with CF with FEV1 < 40% predicted compared with Canadian patients. Findings are more striking among US patients with CF with Medicaid/Medicare health insurance, and nonwhite patients in both countries, raising concerns about underuse of LTx among vulnerable populations.

CFTR modulators: transformative therapies for cystic fibrosis.

DISCLOSURES: No funding contributed to the writing of this commentary. Both authors are employed by the Cystic Fibrosis Foundation. The Cystic Fibrosis Foundation has entered into therapeutic development award agreements and licensing agreements to assist with the development of CFTR modulators that may result in intellectual property rights, royalties, and other forms of consideration provided to CFF. Some of these agreements are subject to confidentiality restrictions and, thus, CFF cannot comment on them.

Ambulatory quality, special health care needs, and emergency department or hospital use for US children.

OBJECTIVE: This study examined family-reported ambulatory care quality and its association with emergency department and hospital utilization, and how these relationships differed across levels of medical complexity. DATA SOURCES: The 2006-2013 Medical Expenditure Panel Survey (MEPS). STUDY DESIGN: Secondary analysis of MEPS data. Variables fitting the National Quality Measures Clearinghouse clinical quality measures domain framework were selected. Exploratory factor analysis grouped ambulatory quality into 12 access, experience, or process measures. Weighted negative binomial regression stratified by health status identified associations between ambulatory quality and ED visits or hospitalizations. DATA COLLECTION: 41,497 children ≤18 years were included. The 5-item special health care needs (SHCN) screener categorized health status as complex, less complex, or no SHCN. PRINCIPAL FINDINGS: Weighted SHCN proportions were 1.6 Percent complex, 18.2 Percent less complex, and 80.0 Percent no SHCN. Mean ED visits were 130 and 335 visits/1000 children/year for no/ complex SHCN, respectively. Mean hospitalizations were 20 and 175 hospitalizations/1000 children/year for no/complex SHCN, respectively. ED visits were associated with 8 of 12 quality measures for no/less complex SHCN. For example, usually/always receiving needed care right away was associated with 22 Percent lower ED visit rate (95% CI 0.64-0.96). Hospitalizations were associated with 4 of 12 quality measures for less complex SHCN. In complex SHCN, associations between ambulatory quality and ED/hospital use were weak and inconsistent. CONCLUSIONS: Ambulatory quality may best predict ED and hospital use for children with no or less complex SHCN. Whether and how ambulatory care predicts emergency and hospital care in complex SHCN remains an important question.

Survival Comparison of Patients With Cystic Fibrosis in Canada and the United States: A Population-Based Cohort Study.

BACKGROUND: In 2011, the median age of survival of patients with cystic fibrosis reported in the United States was 36.8 years, compared with 48.5 years in Canada. Direct comparison of survival estimates between national registries is challenging because of inherent differences in methodologies used, data processing techniques, and ascertainment bias. OBJECTIVE: To use a standardized approach to calculate cystic fibrosis survival estimates and to explore differences between Canada and the United States. DESIGN: Population-based study. SETTING: 42 Canadian cystic fibrosis clinics and 110 U.S. cystic fibrosis care centers. PATIENTS: Patients followed in the Canadian Cystic Fibrosis Registry (CCFR) and U.S. Cystic Fibrosis Foundation Patient Registry (CFFPR) between 1990 and 2013. MEASUREMENTS: Cox proportional hazards models were used to compare survival between patients followed in the CCFR (n = 5941) and those in the CFFPR (n = 45 448). Multivariable models were used to adjust for factors known to be associated with survival. RESULTS: Median age of survival in patients with cystic fibrosis increased in both countries between 1990 and 2013; however, in 1995 and 2005, survival in Canada increased at a faster rate than in the United States (P < 0.001). On the basis of contemporary data from 2009 to 2013, the median age of survival in Canada was 10 years greater than in the United States (50.9 vs. 40.6 years, respectively). The adjusted risk for death was 34% lower in Canada than the United States (hazard ratio, 0.66 [95% CI, 0.54 to 0.81]). A greater proportion of patients in Canada received transplants (10.3% vs. 6.5%, respectively [standardized difference, 13.7]). Differences in survival between U.S. and Canadian patients varied according to U.S. patients' insurance status. LIMITATION: Ascertainment bias due to missing data or nonrandom loss to follow-up might affect the results. CONCLUSION: Differences in cystic fibrosis survival between Canada and the United States persisted after adjustment for risk factors associated with survival, except for private-insurance status among U.S. patients. Differential access to transplantation, increased posttransplant survival, and differences in health care systems may, in part, explain the Canadian survival advantage. PRIMARY FUNDING SOURCE: U.S. Cystic Fibrosis Foundation.

The Cystic Fibrosis Foundation Patient Registry. Design and Methods of a National Observational Disease Registry.

RATIONALE: The Cystic Fibrosis Foundation Patient Registry (CFFPR) is an ongoing patient registry study that collects longitudinal demographic, clinical, and treatment information about persons with cystic fibrosis (CF) in the United States. CF is a life-shortening genetic disorder that occurs in approximately 1 in 3,500 births in the United States. High-quality observational data is important for clinical research, quality improvement, and clinical management. OBJECTIVES: To describe the data collection, patient population, and key limitations of the CFFPR. METHODS: Inclusion criteria for the CFFPR include diagnosis with CF or a CFTR-associated disorder, care at an accredited care center program, and provision of informed consent. Data from clinic visits and hospitalizations are collected through a secure website. Loss to follow-up and generalizability were examined using several methods. The accuracy of CFFPR data was evaluated with an audit of 2012 CFFPR data compared to the medical record. MEASUREMENTS AND MAIN RESULTS: Since 1986, the CFFPR contains the records of 48,463 individuals with CF. Participation among individuals seen at accredited care centers is high, and loss to follow-up is low. An audit of 2012 CFFPR data suggests that the CFFPR contains 95% of clinic visits and 90% of hospitalizations found in the medical record for these patients, and nearly all of the audited fields were highly accurate. CONCLUSIONS: Registries such as the CFFPR are important tools for research, clinical care, and tracking incidence, mortality and population trends.

Report of the European Respiratory Society/European Cystic Fibrosis Society task force on the care of adults with cystic fibrosis.

The improved survival in people with cystic fibrosis has led to an increasing number of patients reaching adulthood. This trend is likely to be maintained over the next decades, suggesting a need to increase the number of centres with expertise in the management of adult patients with cystic fibrosis. These centres should be capable of delivering multidisciplinary care addressing the complexity of the disease, in addition to addressing the psychological burden on patients and their families. Further issues that require attention are organ transplantation and end of life management.Lung disease in adults with cystic fibrosis drives most of the clinical care requirements, and major life-threatening complications, such as respiratory infection, respiratory failure, pneumothorax and haemoptysis, and the management of lung transplantation require expertise from trained respiratory physicians. The taskforce therefore strongly reccommends that medical leadership in multidisciplinary adult teams should be attributed to a respiratory physician adequately trained in cystic fibrosis management.The task force suggests the implementation of a core curriculum for trainees in adult respiratory medicine and the selection and accreditation of training centres that deliver postgraduate training to the standards of the HERMES programme.

Key findings of the US Cystic Fibrosis Foundation's clinical practice benchmarking project.

Benchmarking is the process of using outcome data to identify high-performing centres and determine practices associated with their outstanding performance. The US Cystic Fibrosis Foundation (CFF) Patient Registry contains centre-specific outcomes data for all CFF-certified paediatric and adult cystic fibrosis (CF) care programmes in the USA. The CFF benchmarking project analysed these registry data, adjusting for differences in patient case mix known to influence outcomes, and identified the top-performing US paediatric and adult CF care programmes for pulmonary and nutritional outcomes. Separate multidisciplinary paediatric and adult benchmarking teams each visited 10 CF care programmes, five in the top quintile for pulmonary outcomes and five in the top quintile for nutritional outcomes. Key practice patterns and approaches present in both paediatric and adult programmes with outstanding clinical outcomes were identified and could be summarised as systems, attitudes, practices, patient/family empowerment and projects. These included: (1) the presence of strong leadership and a well-functioning care team working with a systematic approach to providing consistent care; (2) high expectations for outcomes among providers and families; (3) early and aggressive management of clinical declines, avoiding reliance on 'rescues'; and (4) patients/families that were engaged, empowered and well informed on disease management and its rationale. In summary, assessment of practice patterns at CF care centres with top-quintile pulmonary and nutritional outcomes provides insight into characteristic practices that may aid in optimising patient outcomes.

Accelerating implementation of biomedical research advances: critical elements of a successful 10 year Cystic Fibrosis Foundation healthcare delivery improvement initiative.

CONTEXT SCIENTIFIC AND THERAPEUTIC ADVANCES: Remarkable biomedical research advances have led to innovative and increasingly effective therapies. We highlight several scientific milestones in elucidating the pathophysiology of cystic fibrosis (CF) and review the therapies that have become available over the past 20 years. IMPACT OF THE QUALITY IMPROVEMENT INITIATIVE: In 2002, the CF Foundation launched a multifaceted quality improvement initiative to accelerate improvement in CF care. We present evidence of substantial improvement in process measures, such as more consistent outpatient follow-up, and key medical outcomes, including survival, pulmonary function and nutritional status. CRITICAL SUCCESS FACTORS: We offer our perspective on factors critical to the success of the quality improvement initiative, including a compelling strategic plan and the commitment of the CF Foundation to its implementation; the investment in building improvement capacity at CF care centres; the engagement of people with CF and their families as partners; and the integration of quality improvement into the existing CF care framework. DIRECTIONS FOR THE NEXT DECADE: In addition to a continued investment in building and sustaining improvement capacity at CF care centres, and deeper patient engagement, we will address the oppressive treatment burden. We will also complement the measurement of clinical outcomes with patient reported outcomes and healthcare costs for a balanced assessment of the quality and value of care. CONCLUSIONS: Major advances in basic science and therapeutic development coupled with improvements in healthcare delivery have resulted in striking gains in medical outcomes for people with CF.

A preliminary evaluation of the effectiveness of the Cystic Fibrosis Foundation Mentoring Program for Respiratory Care.

BACKGROUND: In 2008 the Cystic Fibrosis (CF) Foundation launched the Respiratory Therapy Mentoring Program, which pairs a respiratory therapist (RT) relatively new to CF (apprentice) with a highly experienced RT (mentor) from a similar CF care center. We wished to determine if we had achieved our short-term goal of increasing CF-specific knowledge among the apprentices who participated in the program. METHODS: Selected apprentices were each matched with a mentor, based on characteristics of CF population, clinical setting, center size, and geographic location of their care centers. Apprentices completed a CF-specific RT knowledge self assessment tool prior to and after a site visit to their mentor's center. Mentors also completed a post site visit knowledge self assessment tool regarding their apprentice. RESULTS: Thirty-seven apprentices completed a pre and post site visit knowledge self assessment tool. The median pre and post site visit scores were 12 and 31 (P < .001) respectively. The mentors' post site visit scores of their apprentices (median 29, P = .07) did not significantly differ from the apprentices' post site visit scores. CONCLUSIONS: The results of this preliminary evaluation suggest that the RT mentoring program has achieved its short-term goal of increasing CF-specific knowledge among RTs relatively new to CF care.

Cystic fibrosis foundation: achieving the mission.

The Cystic Fibrosis Foundation is a voluntary, nonprofit, health organization whose mission is "to assure the development of the means to cure and control cystic fibrosis and to improve the quality of life for those with the disease." While substantial progress has been made, as evidenced by a marked increase in the median predicted age of survival, much work remains to be done. Ongoing medical programs and activities of the Cystic Fibrosis Foundation, which span basic science, drug discovery, drug development, clinical care, patient education, and advocacy, will be described in this article. The key role of respiratory therapists in the cystic fibrosis community will be highlighted.