Dedicated to the treatment of patients with cystic fibrosis (CF) for almost a half century, the comprehensive, fully accredited Cystic Fibrosis Center at Washington University Medical Center and affiliated clinics are premier clinical and research programs. Its commitment to excellence in patient care, teaching, and research is regularly recognized by our division’s inclusion as one of America’s best pediatric pulmonary services.
During the past academic year, the Cystic Fibrosis Clinical Center saw more than 500 pediatric and adult patients with cystic fibrosis, most of whom live within the St. Louis region in Missouri and Illinois. Located in the St. Louis Children’s Hospital, the Pediatric Cystic Fibrosis Clinical Center follows over 200 children and adolescents regularly. Our Center is also involved in the care of patients with cystic fibrosis from across the United States who have been referred for or undergone lung or liver transplantation at our Center. The center clinical staff includes nurse coordinators, dieticians, social workers, physical therapists, respiratory therapists and other ancillary health personnel. A full complement of medical subspecialty consultants, including endocrinology, gastroenterology, and surgical consultants are available, many of whom are actively involved in the program.
The clinical research program at Washington University School of Medicine has grown substantially since our inclusion in the Cystic Fibrosis Foundation’s Therapeutics Development Network (TDN), and we have and are participating in network-supported clinical trials testing novel agents to modulate, correct or potentiate specific cystic fibrosis transmembrane conductance regulator (CFTR) defects; these agents may profoundly affect the clinical course of cystic fibrosis. The center is one of six primary sites of the National Institutes of Health (NIH)-supported Genetic Disorders of Mucociliary Clearance Consortium, a clinical research network to study rare diseases of the airways, focusing on variant forms of cystic fibrosis, as well as related diseases such as primary ciliary dyskinesia. We are participating in the NIH- and Cystic Fibrosis Foundation-supported Cystic Fibrosis Liver Disease Research Consortium, a multicenter, longitudinal study utilizing diagnostic, clinical and outcome data to understand the pathogenesis of cystic fibrosis-related liver disease. The Center also collaborates in investigator-initiated research on CF Related Diabetes, intestinal complications in CF, and hearing loss in CF due to aminoglycoside therapy. The Center continues its many quality improvement initiatives, including an Agency for Healthcare Research and Quality (AHRQ)-supported program to develop a computer-based system for the detection of adverse drug events in cystic fibrosis patients during care transitions.
The Cystic Fibrosis Center has maintained collaborative relationships with Washington University investigators in clinical and basic science departments, which has allowed us to consider questions fundamental to our understanding of the disease. Several NIH-funded investigators are collaborating on projects that deal with host-pathogen interactions, pulmonary inflammation and airway injury, as well as cell biology of the airway epithelia.
Pediatric Cystic Fibrosis Clinical Center
The Pediatric Cystic Fibrosis Clinical is a past recipient of the annual Cystic Fibrosis Foundation’s Quality Care Award: Recognizing Outstanding QI Processes and Accomplishments.
Patients with cystic fibrosis under 21 years of age receive their routine care at St. Louis Children’s Hospital, which are regularly scheduled in the Pediatric Ambulatory Clinics. Children colonized with Burkholderia cepacia complex are seen at alternate times according to CFF infection control guidelines. Individual physicians, who have primary responsibility for their patients, attend the clinic with the support of two cystic fibrosis nurse coordinators, dieticians, clinical social workers, and physical therapist. When necessary, children with cystic fibrosis are admitted to the St. Louis Children’s Hospital 7100 Respiratory Unit, a 16-bed, state-of-the-art pediatric ward, under the care of pediatric residents and pulmonary fellows with attending supervision from the Division of Pediatric Allergy and Pulmonary Medicine. Patients colonized with Burkholderia species are housed on a separate pediatric ward.
The Pediatric Cystic Fibrosis Clinical Center team meets regularly to review ambulatory and inpatient care, other patient-related issues, research projects and quality improvement initiatives. The adult and pediatric center directors meet frequently to critically review the programs and ongoing clinical research projects, and the entire adult and pediatric teams convene regularly. Cystic fibrosis patients admitted to St. Louis Children’s Hospital are also reviewed with the other members of the division (including physicians, nurses, respiratory therapists, dieticians and physical therapists) during a weekly patient care conference. The Pediatric Lung Transplantation team has regular meetings among their team to discuss patient care issues; as many of these patients have cystic fibrosis, the transplant and CF teams also meet regularly to review common patients.
As the child approaches 18 years of age, the patient and families begin the process of transitioning care to the Adult Cystic Fibrosis Center. With rare exception, all patients are transferred by age 21 years.
Laboratory and diagnostic services
The Pediatric Pulmonary Function Laboratory at St. Louis Children’s Hospital specializes in pediatric lung function testing from infancy through adolescence. It includes three rooms for standard pulmonary function testing (spirometry, lung volumes), an exercise testing area, and a dedicated room for infant pulmonary function measurements. The laboratory routinely performs approximately 6000 tests each year.
Microbiological analyses of specimens from the upper and lower respiratory tract are performed in the St. Louis Children’s Hospital Microbiology Laboratory.
Sweat chloride measurements for all patients seen at the Washington University Medical Center are performed in the Clinical Chemistry Laboratory at St. Louis Children’s Hospital. The Center Director receives and reviews all measured values. Nasal transepithelial potential difference (NPD) measurements have been validated, and the Cystic Fibrosis Foundation Therapeutics Development Network NPD Core has certified the Center.
Newborn screening for cystic fibrosis
Newborn screening for cystic fibrosis is ongoing in Missouri and Illinois. The Cystic Fibrosis Standing Committee of the Missouri Department of Health and Senior Services meet regularly with physicians and nurses representing the accredited cystic fibrosis centers. Educational resources and seminars for parents and caregivers have been developed by the center. Despite the different approaches that each state has adopted for cystic fibrosis newborn screening, we have been able to consistently perform diagnostic testing within a week of a positive screen, evaluate the child in the Pediatric Cystic Fibrosis Center and often begin treatment on the same day.
Lung transplantation for cystic fibrosis
The St. Louis Children’s Hospital is one of the most active pediatric solid organ transplant centers worldwide. Over 300 pediatric lung transplants have been performed here, and the majority of those were on children and adolescents with cystic fibrosis who have advanced lung disease. The Cystic Fibrosis Clinical Center remains committed to lung transplantation as a potential option to those eligible with severe pulmonary disease, and we will continue to care for all cystic fibrosis patients in need of lung transplantation in the U.S. and worldwide.
The Pediatric Cystic Fibrosis Centers supports several community activities, including a Cystic Fibrosis Parent Networking Group and Cystic Fibrosis Parents Night Out. The center has an active Parent Advisory Committee that continues to meet to provide input regarding quality improvement initiatives. We have created a social network for our cystic fibrosis families using Facebook, which brings patients and staff closer together. In addition, members of our Center care team collaborate with the local Gateway Chapter of the Cystic Fibrosis Foundation, as well as the CF Foundation’s national office and programs.
The Washington University Therapeutic Development Center is devoted to improving the health and well-being of patients with cystic fibrosis, whether it involves conventional treatments, novel therapies and pharmaceuticals or organ transplantation. Clinical research in cystic fibrosis at the Washington University School of Medicine has expanded, and includes TDN-supported clinical trials testing novel agents to correct or potentiate specific cystic fibrosis transmembrane conductance regulator (CFTR) defects, as well as novel approaches to treatment of pulmonary infections. Members of the Center have actively been involved in a number of TDN-based committees, including the Steering, Protocol Review and Patient Advocacy-Ethics Committees.
Our Center also supports clinical research initiated by Center members and collaborators. Topics addressed in these efforts include gastrointestinal complications of CF, CF related diabetes and hearing loss after aminoglycoside therapy in CF.
Please contact 314-454-2353 if you would like additional information regarding active clinical trials at our center.
Basic science and translational research
The Division of Pediatric Allergy and Pulmonary Medicine has a core group of physician-scientists and clinical investigators, many of whom have extramural funding, including NIH and Cystic Fibrosis Foundation-supported grants that focus on pathophysiology relevant to cystic fibrosis lung disease. The center collaborates with investigators in clinical and basic science departments, including as a key collaborator in the St. Louis Children’s Hospital Children’s Discovery Institute Center of Excellence in Pediatric Pulmonary Diseases & Asthma.
Affiliate cystic fibrosis programs
The Affiliate Cystic Fibrosis Clinical Centers at the Memorial Medical Center at Southern Illinois University School of Medicine in Springfield, Illinois, and the Carle Clinic in Urbana-Champaign, Illinois, are both active. The center staff reviews and visits the affiliate centers regularly. Each program cares for approximately 20 to 40 CF patients from central and southern Illinois, and has the facilities and complement of physician, nursing and ancillary staff to accommodate their patients.