A Multidisciplinary Childrens Airway Center: Impact on the Care of Patients With Tracheostomy

BACKGROUND: Children with complex airway problems see multiple specialists. To improve outcomes and coordinate care, we developed a multidisciplinary Children's Airway Center. For children with tracheostomies, aspects of care targeted for improvement included optimizing initial hospital discharge, promoting effective communication between providers and caregivers, and avoiding tracheostomy complications. METHODS: The population includes children up to 21 years old with tracheostomies. The airway center team includes providers from pediatric pulmonology, pediatric otolaryngology/head and neck surgery, and pediatric gastroenterology. Improvement initiatives included enhanced educational strategies, weekly care conferences, institutional consensus guidelines and care plans, personalized clinic schedules, and standardized intervals between airway examinations. A patient database allowed for tracking outcomes over time. RESULTS: We initially identified 173 airway center patients including 123 with tracheostomies. The median number of new patients evaluated by the center team each year was 172. Median hospitalization after tracheostomy decreased from 37 days to 26 days for new tracheostomy patients <1 year old discharged from the hospital. A median of 24 care plans was evaluated at weekly conferences. Consensus protocol adherence increased likelihood of successful decannulation from 68% to 86% of attempts. The median interval of 8 months between airway examinations aligned with published recommendations. CONCLUSIONS: For children with tracheostomies, our Children's Airway Center met and sustained goals of optimizing hospitalization, promoting communication, and avoiding tracheostomy complications by initiating targeted improvements in a multidisciplinary team setting. A multidisciplinary approach to management of these patients can yield measurable improvements in important outcomes

Inhaled aztreonam for chronic Burkholderia infection in cystic fibrosis: A placebo-controlled trial

AbstractBackgroundIndividuals with Burkholderia spp. infection have historically been excluded from efficacy trials of inhaled antibiotics, including aztreonam for inhalation solution (AZLI).MethodsA double-blind, placebo-controlled, 24-week trial of continuous AZLI/placebo treatment was undertaken in individuals with cystic fibrosis (CF) and chronic Burkholderia spp. infection. All subjects also received usual medical care (determined by their physicians). Additional antibiotic use was not restricted.ResultsBaseline FEV1% predicted values ranged from 15.8% to 114.6%. No significant treatment differences (AZLI vs. placebo) were observed at week 24 for any endpoints, including FEV1% predicted, number of respiratory exacerbations requiring systemic/inhaled antibiotics, or hospitalizations. Continuous AZLI administration was well tolerated. Burkholderia spp. susceptibility to antibiotics commonly used in CF therapy showed little change.Conclusions24-weeks of continuous AZLI treatment did not significantly improve lung function in CF subjects with chronic Burkholderia spp. infection. Non-study antibiotic use may have confounded any potential AZLI effects

Impact of Sustained Eradication of New Pseudomonas aeruginosa Infection on Long-term Outcomes in Cystic Fibrosis

Background. Pseudomonas aeruginosa (Pa) is the most important pathogen infecting the airways in individuals with cystic fibrosis. A key question is whether children with newly acquired Pa infection who are able to achieve sustained eradication after early antipseudomonal therapy demonstrate improved long-term health outcomes compared with those who are unable to achieve a sustained microbiologic response

Highly Effective Cystic Fibrosis Clinical Research Teams: Critical Success Factors

BACKGROUND Bringing new therapies to patients with rare diseases depends in part on optimizing clinical trial conduct through efficient study start-up processes and rapid enrollment. Suboptimal execution of clinical trials in academic medical centers not only results in high cost to institutions and sponsors, but also delays the availability of new therapies. Addressing the factors that contribute to poor outcomes requires novel, systematic approaches tailored to the institution and disease under study. OBJECTIVE To use clinical trial performance metrics data analysis to select high-performing cystic fibrosis (CF) clinical research teams and then identify factors contributing to their success. DESIGN Mixed-methods research, including semi-structured qualitative interviews of high-performing research teams. PARTICIPANTS CF research teams at nine clinical centers from the CF Foundation Therapeutics Development Network. APPROACH Survey of site characteristics, direct observation of team meetings and facilities, and semi-structured interviews with clinical research team members and institutional program managers and leaders in clinical research. KEY RESULTS Critical success factors noted at all nine high-performing centers were: 1) strong leadership, 2) established and effective communication within the research team and with the clinical care team, and 3) adequate staff. Other frequent characteristics included a mature culture of research, customer service orientation in interactions with study participants, shared efficient processes, continuous process improvement activities, and a businesslike approach to clinical research. CONCLUSIONS Clinical research metrics allowed identification of high-performing clinical research teams. Site visits identified several critical factors leading to highly successful teams that may help other clinical research teams improve clinical trial performance

An 18-month study of the safety and efficacy of repeated courses of inhaled aztreonam lysine in cystic fibrosis

Chronic airway infection with Pseudomonas aeruginosa (PA) causes morbidity and mortality in patients with cystic fibrosis (CF). Additional anti-PA therapies are needed to improve health status and health-related quality of life. AIR-CF3 was an international 18-month, open-label study to evaluate the safety and efficacy of repeated courses of aztreonam for inhalation solution (AZLI, now marketed as Cayston®) in patients aged ≥6 years with CF and PA infection who previously participated in one of two Phase 3 studies: AIR-CF1 or AIR-CF2. Patients received up to nine courses (28 days on/28 days off) of 75 mg AZLI two (BID) or three times daily (TID) based on randomization in the previous trials. 274 patients, mean age 28.5 years (range: 8–74 years), participated. Mean treatment adherence was high (92.0% BID group, 88.0% TID group). Hospitalization rates were low and adverse events were consistent with CF With each course of AZLI, FEV1 and scores on the Cystic Fibrosis Questionnaire-Revised Respiratory Symptomscale improved and bacterial density in sputum was reduced. Benefits waned in the 28 days off therapy, but weight gain was sustained over the 18months. There were no sustained decreases in PA susceptibility. A dose response was observed; AZLI TID-treated patients demonstrated greater improvements in lung function and respiratory symptoms over 18 months. Repeated intermittent 28-day courses of AZLI treatment were well tolerated. Clinical benefits in pulmonary function, health-related quality of life, and weight were observed with each course of therapy. AZLI is a safe and effective new therapy in patients with CF and PA airway infection

Pseudomonas aeruginosa in vitro Phenotypes Distinguish Cystic Fibrosis Infection Stages and Outcomes

Rationale: Pseudomonas aeruginosa undergoes phenotypic changes during cystic fibrosis (CF) lung infection. Although mucoidy is traditionally associated with transition to chronic infection, we hypothesized that additional in vitro phenotypes correlate with this transition and contribute to disease

Inhaled Aztreonam Lysine for Chronic Airway Pseudomonas aeruginosa in Cystic Fibrosis

Rationale: The effectiveness and safety of aztreonam lysine for inhalation (AZLI) in patients with cystic fibrosis (CF) on maintenance treatment for Pseudomonas aeruginosa (PA) airway infection was evaluated in this randomized, double-blind, placebo-controlled study