Cystic Fibrosis Center

Introduction

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 continues to thrive.  The Cystic Fibrosis Clinical Center and affiliated clinics are premier clinical and research programs.  Its commitment to excellence in patient care, teaching, and research was recognized by our Division’s inclusion as one of America’s best pulmonary services, according to Parent magazine’s 2008 survey and the 2009 US & World Report Best Hospitals ranking.

During the past academic year, the Cystic Fibrosis Clinical Center saw more than 500 cystic fibrosis patients, most of whom live within a 50-mile radius of St. Louis.  Nevertheless, we continue to attract patients from other states, including Arkansas, Kansas, Kentucky, Oklahoma, Tennessee, and Texas.  Located in the St. Louis Children’s Hospital, the Pediatric Cystic Fibrosis Clinical Center now follows 262 children and adolescents regularly.  We are involved in the care of cystic fibrosis patients from across the United States who have been referred for or have survived lung or liver transplantation.  The Center clinical staff remains stable, supported by nurse coordinators, dieticians, social workers, physical therapists, respiratory therapists, and other ancillary health personnel.  A full complement of medical subspecialty and surgical consultants is available, many of whom are actively involved in the program.

The clinical research program at Washington University School of Medicine has grown since our inclusion in the Therapeutics Development Network (TDN), and we are participating in network-supported clinical trials testing novel agents to correct or potentiate specific cystic fibrosis transmembrane conductance regulator (CFTR) defects, which could 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.  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 continues its many quality improvement initiatives, we are participating in the 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 (including the co-directors of the pediatric center) are working together on projects that deal with host-pathogen interactions, pulmonary inflammation, and airway injury.


Pediatric Cystic Fibrosis Clinical Center

The Pediatric Cystic Fibrosis Clinical Center continues to flourish. Thomas Ferkol M.D. remains the director of the Cystic Fibrosis Center, and Albert Faro M.D. serves as the associate center director. The CF Center was selected as one of the 2009 recipients 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 on Thursday afternoons.  Children colonized with Burkholderia cepacia complex are only seen on Friday afternoons.  Patients may be seen at other times as clinically indicated.  Individual physicians, who have primary responsibility for their patients, attend the clinic with the support of two cystic fibrosis nurse coordinators, dietician, clinical social workers, and respiratory therapist.  When necessary, children with cystic fibrosis are admitted to the St. Louis Children’s Hospital 7 East Respiratory Unit, a new, 20-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, Immunology, and Pulmonary Medicine.  Patients colonized with Burkholderia species are housed on a separate pediatric ward.

The Pediatric Cystic Fibrosis Clinical Center team meets every Tuesday morning 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.  The entire adult and pediatric teams convene formally several times each year.  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 scheduled patient care conference that is held weekly.  The Pediatric Lung Transplantation team has regular meetings among their team to discuss patient care issues, many of whom have cystic fibrosis, and meets regularly with the Pediatric Cystic Fibrosis Center to review common patients.

As the child approaches 18 years of age, the patient and families begin to consider transition of care to the Adult Cystic Fibrosis Center.  With rare exception, all patients are transferred by age 21 years.

With respect to personnel, the Pediatric Cystic Fibrosis Clinical Center remains intact.  Karen Wilson R.N., M.S.N., and Beth Witzig R.N. are both highly skilled clinical nurse coordinators who have considerable experience in cystic fibrosis care.  Mary Boyle R.N., M.S.N., Patricia Burks R.N., Denise Rodgers R.P.F.T, and Jane Quante R.N. are the cystic fibrosis research coordinators for the Center, and participate in clinical trials for both adult and pediatric cystic fibrosis patients.  Their experience and dedication to our patients has allowed expansion our clinical and translational research programs. 

Andrea Clark R.D. remains the dietician for the Pediatric Cystic Fibrosis Center, and leads many of our quality improvement efforts.  Tracy Hartmann M.S.W. has been the cystic fibrosis clinical social worker now for over a decade. Sheila Hensler R.T. routinely instructs cystic fibrosis patients and their families proper techniques to perform effective airway clearance.  Jonica Huntman Pharm.D. serves as a clinical pharmacist for the new 7 East Respiratory Unit and provides pharmacological support for our patients.


Laboratory and diagnostic services

The Pediatric Pulmonary Function Laboratory at St. Louis Children's Hospital specializes in pediatric testing from infancy through adolescence.  It includes three rooms for standard pulmonary function testing, an exercise testing area, and a dedicated room for infant pulmonary function (IPL) 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, which has maintained high standards under the continued direction of Gregory Storch M.D. from the Division of Infectious Diseases.

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 under the supervision of Dennis Dietzen Ph.D.  Approximately 1300 tests are performed annually, and 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.  We are currently participating in clinical studies testing the efficacy of candidate drugs to correct or potentiate specific CFTR defects.


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

St. Louis Children's Hospital has performed more organ transplants than any other pediatric center in the world.  Of the solid organ transplants performed, the leading organ has been the lung.  Over 300 pediatric lung transplants have been done here, and the majority of those were performed 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 United States and world-wide.


Education and training

Washington University provides an Accreditation Council for Graduate Medical Education (ACGME)-accredited, three-year, training program in pediatric pulmonology. The pediatric pulmonology training program currently has three fellows who rotate through our clinical services and directly care for cystic fibrosis patients both in clinic and when they are hospitalized.

Several clinical conferences are regularly held for the pediatric pulmonology fellows, including a weekly lecture series that comprehensively reviews pulmonary physiology, pathophysiology, and clinical care where cystic fibrosis is extensively covered as part of the curriculum.  Cystic fibrosis-related topics are also presented at the divisional research conference and Journal Club.  Several research conferences are held weekly throughout the medical center, covering topics in pulmonary infection, inflammation, host defense, and lung injury that relate to cystic fibrosis. Several NIH Clinical Training (T32) Grants, administered through the Departments of Pediatrics and Internal Medicine, support our fellows during their research training.

The Department of Pediatrics at the Washington University School of Medicine consistently attracts talented house officers.  Residents are intimately involved in both the inpatient and ambulatory care of our cystic fibrosis patients. As residents rotate through the Division of Pediatric Allergy, Immunology and Pulmonary Medicine, which many do during their second- or third-year of training, they have the opportunity to participate in the care of cystic fibrosis patients in all stages of disease, particularly during rotations on the 7 East Respiratory Unit where a mixture of formal and informal teaching occurs regularly.  They receive more formal education in the scheduled departmental clinical conferences, and the Division of Pediatric Allergy, Immunology, and Pulmonary Medicine was recognized by the pediatric housestaff this year for our dedication and excellence in teaching with the presentation of the “Outstanding Division Teaching Award.”  Cystic fibrosis is often a topic for discussion in Department of Pediatrics Grand Rounds and Case Management Conference.

Washington University School of Medicine is distinguished as one of the most competitive in the nation, and has the largest Medical Scientist Training Program (MSTP) program in the country.  Medical students are exposed to cystic fibrosis in pulmonary pathophysiology courses, and several pre-clinical medical students and undergraduates have been attracted to both clinical and basic cystic fibrosis research, in some cases supported by Cystic Fibrosis Foundation Student Traineeship Awards.

During their third-year clinical training, medical students are involved in the care of hospitalized cystic fibrosis patients, and the division faculty review cases with students rotating through pediatrics.  These students also participate in the departmental lectures that deal with cystic fibrosis.  As fourth-year medical students, trainees from Washington University and other medical schools rotate through the cystic fibrosis clinics.

Cystic fibrosis has been the topic of the Markey Scholar Program, a progressive educational program that exposes Ph.D. students and post-doctoral fellows to important clinical problems.


Community outreach

The Pediatric Cystic Fibrosis Centers supports several community activities, including the monthly 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 (http://www.stlouischildrens.org), which brings patients and staff closer together.

Finally, the Center director is a medical advisor for the Gateway Chapter of the Cystic Fibrosis Foundation.


Clinical trials

Directed by Al Faro, M.D., and Dan Rosenbluth, M.D., 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 included the following studies this year:

  • Longitudinal assessment of risk factors for Pseudomonas aeruginosa and early anti-pseudomonal treatment in children with cystic fibrosis (EPIC-001) (TDN);
  • Effectiveness and safety of intermittent antimicrobial therapy for the treatment of new onset Pseudomonas aeruginosa airway infection in young patients with cystic fibrosis (EPIC-002) (TDN);
  • A randomized, open-label, multicenter, phase 3 trial to assess the safety of tobramycin inhalation powder (TIP) compared to TOBI in cystic fibrosis subjects. (Novartis);
  • A randomized, double-blind within dose, placebo controlled study to investigate safety, tolerability, and pharmacokinetics of increasing single- and multiple-dose (28-day dosing) of Tiotropium bromide administered once daily via the Respirmat device in cystic fibrosis patients (Boehringer-Ingelheim);
  • Antimicrobial resistance in cystic fibrosis (CFF);
  • Standard versus biofilm susceptibility testing in cystic fibrosis (CFF);
  • Pilot and feasibility study for the treatment of pre-diabetes in patients with cystic fibrosis (Washington University DRTC); Multidose safety and tolerability study of liposomal amikacin for inhalation (Arikase) in cystic fibrosis patients with chronic infections due to pseudomonas aeruginosa (Transave);
  • A phase I/II randomized, double-blind, placebo-controlled, single-dose, dose escalation study of KB001 in cystic fibrosis patients infected with Pseudomonas aeruginosa (Kalobios);
  • Infant study of inhaled saline (ISIS) (CFF); and
  • Multicenter, multinational, randomized, placebo-controlled trial of azithromycin in subjects with cystic fibrosis 6-18 years old, culture negative for Pseudomonas aeruginosa (CFF).

The Center is also participating in TDN-supported clinical trials testing novel agents (Vertex) to correct or potentiate specific cystic fibrosis transmembrane conductance regulator (CFTR) defects.  This past year, members of the Center have actively been involved in TDN-based committees, including the Steering, Protocol Review, and Patient Advocacy-Ethics Committees.

Please contact Mary Boyle or Patty Burks at (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, Immunology, and Pulmonary Medicine has established a core of committed, productive physician-scientists and clinical investigators, most 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, most notably with physicians and scientists from the Washington University Genome Sequencing Center and the Mallinckrodt Institute of Radiology, focusing on airway infection, host inflammation, liver disease, and nutrition.


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. 

Members of the Pediatric Cystic Fibrosis Center, including physicians, nurse coordinators, dietician, social worker, and respiratory therapist attend the Cystic Fibrosis Outreach Clinic, located at Cox South Medical Center in Springfield, Missouri.  Our team regularly follows 20 to 30 patients from southwest Missouri and Arkansas.

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