One of the first centers of its kind in the United States, the Cystic Fibrosis Center at Oishei Children's Hospital has always been at the vanguard of patient care, research, and education. Composed of a multidisciplinary team of medical professionals, the Center strives to provide exemplary family-centered care to patients, to participate in research for the improvement of care and treatment, and to educate patients, families, the medical community, and the public about the disease. The CF Center is presently one of just 118 nationally accredited CF centers in the country and one of 77 accredited therapeutic development network centers.
Working to control the disease
Cystic Fibrosis (CF) is a genetic disease that affects approximately 30,000 people in the United States. With CF, a defective gene causes the body to produce abnormally thick and sticky mucus. The mucus clogs the lungs and obstructs the pancreas, as well as impacting other organs, making breathing and food absorption difficult. According to the Cystic Fibrosis Foundation’s National Patient Registry, the median age of survival for a person with the disease is approximately 36 years.
Early Diagnosis; Improved Intervention
Early and correct detection make a tremendous and positive impact on patient care. Before 2002, when New York State began newborn screening (NBS), children were diagnosed through symptoms. Today, individuals identified by NBS are diagnosed with CF if they have an elevated sweat chloride level or if they have inherited disease-causing mutations in the CF transmembrane conductance regulator (CFTR) gene. Detection is, however, not as straightforward as it might seem. Mutations in the CFTR gene can cause CF, but not all mutations are disease-causing. Infants identified by the NBS program in which CF cannot be clearly diagnosed or ruled out are given the designation CFTR-related metabolic syndrome (CRMS). The Buffalo Center led the way in helping to define standards for care of infants with CF and with CRMS, a definition now used by the CF Foundation and other facilities involved in NBS. As of 2011, all 50 states had implemented NBS.
Quality care all along the way
The Center serves the eight counties of Western New York and has both a pediatric and adult program providing continuity of care throughout a patient’s lifetime. Dr. Danielle Goetz became the new pediatric program director in 2014; Dr. Carla Frederick is the adult program director. A team of physicians, coordinators, a dietitian, respiratory therapists, a social worker, a genetic counselor, and a psychiatrist make up the pediatric and adult teams. Finding ways to improve patient care is always at the forefront of the CF Center’s work. A Parent-Patient Advisory Council was founded to help in the redesign of patient care. The Center uses a microsystems-based approach to quality improvement (QI). In 2007-2008, it received a national award recognizing its outstanding QI processes and accomplishments.
Looking for a Breakthrough
Bringing new treatments into the clinical realm through research directly betters the lives of those affected by CF. Since the early 1990s, the CF Center has been involved in over 30 clinical trials, led by Nadine Caci, RNC, CCRC, senior research coordinator, and assisted by Christine Roach, RN. Investigations are underway to find a molecule to change the CFTR protein; one study is currently in Phase 3, the last phase before seeking FDA approval. Also showing early promise is a novel technique in which a small device called the SmartPill is used to measure the acidity of the gastrointestinal (GI) tract; CF affects the pH in the GI tract. This groundbreaking work could represent a big step forward in care.
The work carried out at the center has not gone unnoticed. In 2008, The Stephen P. Holesko Fund was established by Stephen and Joan Holesko in memory of their son to support the Cystic Fibrosis Center at Oishei Children's Hospital. The Fund helps working families that are struggling with the expenses associated with caring for a child with CF and, starting in 2011, it will support the Center’s annual Family Education Night.
The success of multidisciplinary care at the CF Center is reflected in its clinical outcomes, which are among the best in the country, according to data compiled by the National Cystic Fibrosis Foundation.
Measures of pulmonary and nutritional status are the strongest predictors of future health in people with CF. For the measure of lung function (FEV1), the percentage predicted for patients 18 to 29 in 2009 (the most recent data available) at Women & Children’s Hospital of Buffalo was 82; the national median was 70. The measure of body mass index (a nutritional indicator) for patients age 20 or older treated by the Buffalo Center in 2009 was 23.3; the national median for BMI was 21.9. For both measurements, the higher number value demonstrates better outcomes.