No abstract

No abstract

for the Macrolide Study Group C YSTIC FIBROSIS (CF) IS CHARacterized by a recurrent cycle of pulmonary infection and inflammation. Pseudomonas aeruginosa is the most common pathogen in patients with CF and by age 18 years, 80% of patients are chronically infected. Neutrophils are the dominant inflammatory cells and much of the airway destruction characteristic of CF is secondary to neutrophil-derived proteases and oxidants. 1 Treatment strategies for CF lung disease have included antibiotics, mucolytics, and antiinflammatory therapies. 2 There is evidence suggesting that macrolide antibiotics may be beneficial for patients with CF. Macrolide antibiotics substantially reduced morbidity and mortality in patients in Japan with diffuse panbronchiolitis. 3,4 Diffuse panbronchiolitis shares many clinical features with CF; patients are often infected with mucoid strains of P aeruginosa and mortality is secondary to chronic pro

It is recommended that diagnoses associated with CFTR mutations in all individuals, from newborn to adult, be established by evaluation of CFTR function with a sweat chloride test. The latest mutation classifications annotated in the Clinical and Functional Translation of CFTR project (http://www.cftr2.org/index.php) should be used to aid in diagnosis. Newborns with a high immunoreactive trypsinogen level and inconclusive CFTR functional and genetic testing may be designated CFTR-related metabolic syndrome or CF screen positive, inconclusive diagnosis; these terms are now merged and equivalent, and CFTR-related metabolic syndrome/CF screen positive, inconclusive diagnosis may be used. International Statistical Classification of Diseases and Related Health Problems, 10th Revision codes for use in diagnoses associated with CFTR mutations are included.

The objective of this study was to create a 5-year survivorship model to identify key clinical features of cystic fibrosis. Such a model could help researchers and clinicians to evaluate therapies, improve the design of prospective studies, monitor practice patterns, counsel individual patients, and determine the best candidates for lung transplantation. The authors used information from the Cystic Fibrosis Foundation Patient Registry (CFFPR), which has collected longitudinal data on approximately 90% of cystic fibrosis patients diagnosed in the United States since 1986. They developed multivariate logistic regression models by using data on 5,820 patients randomly selected from 11,630 in the CFFPR in 1993. Models were tested for goodness of fit and were validated for the remaining 5,810 patients for 1993. The validated 5-year survivorship model included age, forced expiratory volume in 1 second as a percentage of predicted normal, gender, weight-for-age z score, pancreatic sufficiency, diabetes mellitus, Staphylococcus aureus infection, Burkerholderia cepacia infection, and annual number of acute pulmonary exacerbations. The model provides insights into the complex nature of cystic fibrosis and supplies a rigorous tool for clinical practice and research.Keywords cystic fibrosis; logistic models; models, theoretical; multivariate analysis; proportional hazards models; survival analysis Cystic fibrosis is an autosomal recessive, multisystem disease leading to significant morbidity and early death. Since the disease was described in 1938 (1,2), treatments for its pancreatic and pulmonary manifestations have improved median survival in the United States from less than 6 months to about 32 years in 1998 (3). Severe pulmonary disease is the primary cause of cystic-fibrosis-related mortality, constituting 76.4 percent of such deaths in 1998 (3). Chronic inflammation and infection of the airways characterize cystic-fibrosis-related (4,5). Malnutrition, in part due to pancreatic insufficiency, was the major feature of the disease according to early reports, and it continues to be a substantial problem (1-3). With improved survival, additional manifestations such as diabetes mellitus have been recognized (6,7).Many studies have considered the survival effect of a variety of clinical and physiologic features of cystic fibrosis such as forced expiratory volume in 1 second as a percentage of predicted normal (FEV 1 %), gender, age, pregnancy, or particular therapies (4-26). We developed a single survivorship model that integrated many characteristics of cystic fibrosis and quantified the relative contribution of each.The current most commonly used survival model of cystic fibrosis was developed in 1992 and is based on FEV 1 % alone or on age, gender, and FEV 1 % (15). Clinicians often use this model to select patients to refer for lung transplantation. The model is relatively simple to use for estimating cystic fibrosis survival, but it has not been validated and does not incorporate clinical features of cystic fib...

Non-tuberculous mycobacteria (NTM) are ubiquitous environmental organisms that can cause chronic pulmonary infection, particularly in individuals with pre-existing inflammatory lung disease such as cystic fibrosis (CF). Pulmonary disease caused by NTM has emerged as a major threat to the health of individuals with CF but remains difficult to diagnose and problematic to treat. In response to this challenge, the US Cystic Fibrosis Foundation (CFF) and the European Cystic Fibrosis Society (ECFS) convened an expert panel of specialists to develop consensus recommendations for the screening, investigation, diagnosis and management of NTM pulmonary disease in individuals with CF. Nineteen experts were invited to participate in the recommendation development process. Population, Intervention, Comparison, Outcome (PICO) methodology and systematic literature reviews were employed to inform draft recommendations. An anonymous voting process was used by the committee to reach consensus. All committee members were asked to rate each statement on a scale of: 0, completely disagree, to 9, completely agree; with 80% or more of scores between 7 and 9 being considered ‘good’ agreement. Additionally, the committee solicited feedback from the CF communities in the USA and Europe and considered the feedback in the development of the final recommendation statements. Three rounds of voting were conducted to achieve 80% consensus for each recommendation statement. Through this process, we have generated a series of pragmatic, evidence-based recommendations for the screening, investigation, diagnosis and treatment of NTM infection in individuals with CF as an initial step in optimising management for this challenging condition.

Newborn screening for cystic fibrosis (CF) offers the opportunity for early medical and nutritional intervention that can lead to improved outcomes. Management of the asymptomatic infant diagnosed with CF through newborn screening, prenatal diagnosis, or sibling screening is different from treatment of the symptomatically diagnosed individual. The focus of management is on maintaining health by preventing nutritional and respiratory complications. The CF Foundation convened a committee to develop recommendations based on a systematic review of the evidence and expert opinion. These guidelines encompass monitoring and treatment recommendations for infants diagnosed with CF and are intended to help guide families, primary care providers, and specialty care centers in the care of infants with CF.

The 2013 Infection Prevention and Control (IP&C) Guideline for Cystic Fibrosis (CF) was commissioned by the CF Foundation as an update of the 2003 Infection Control Guideline for CF. During the past decade, new knowledge and new challenges provided the following rationale to develop updated IP&C strategies for this unique population:1. The need to integrate relevant recommendations from evidence-based guidelines published since 2003 into IP&C practices for CF. These included guidelines from the Centers for Disease Control and Prevention (CDC)/Healthcare Infection Control Practices Advisory Committee (HICPAC), the World Health Organization (WHO), and key professional societies, including the Infectious Diseases Society of America (IDSA) and the Society for Healthcare Epidemiology of America (SHEA). During the past decade, new evidence has led to a renewed emphasis on source containment of potential pathogens and the role played by the contaminated healthcare environment in the transmission of infectious agents. Furthermore, an increased understanding of the importance of the application of implementation science, monitoring adherence, and feedback principles has been shown to increase the effectiveness of IP&C guideline recommendations.2. Experience with emerging pathogens in the non-CF population has expanded our understanding of droplet transmission of respiratory pathogens and can inform IP&C strategies for CF. These pathogens include severe acute respiratory syndrome coronavirus and the 2009 influenza A H1N1. Lessons learned about preventing transmission of methicillin-resistant Staphylococcus aureus (MRSA) and multidrug-resistant gram-negative pathogens in non-CF patient populations also can inform IP&C strategies for CF.