U.S. News & World Report 2011-12 edition of “Best Children’s Hospitals” ranks pediatric pulmonology care at Nemours/Alfred I. duPont Hospital for Children among the best in the country.
Nemours Pediatric Pulmonology Ranks Among the Best
Cystic Fibrosis
Cystic fibrosis (CF) is an inherited disease that affects the lungs and digestive system. It makes kids who have it more vulnerable to repeated lung infections. This is because it causes the lungs to produce thick, sticky mucus that clogs the airways. The intestines are also affected in a way that interferes with the body’s ability to digest and absorb food.
Our CF programs in Jacksonville, Orlando, and Pensacola, Florida, as well as at the Alfred I. duPont Hospital for Children in Wilmington, Delaware, are nationally accredited as Cystic Fibrosis Care Centers by the Cystic Fibrosis Foundation. Membership in this network helps us to ensure that children living with CF get the expert care they need to live longer, healthier lives. It also means our caregivers participate in the Cystic Fibrosis Foundation Registry, which contains a wealth of information on topics like diet and nutrition, which are essential in managing CF.
The CF Registry also serves as a database for researchers studying new drugs and therapies.
Helping Children with CF Live Healthier Lives
Our team of health care providers works together to support children and their families, teaching them about CF and how to manage the condition on a daily basis. We focus on meeting the needs of each of our patients from birth to age 18. We offer newborn screening; diagnostic testing (the gold standard of which is a sweat test performed in one of our state-of-the-art laboratories); treatment for newly diagnosed infants and children; long-term care plans; and assistance with transitioning our teenage patients to adult CF programs.
We offer comprehensive care in a family-oriented and convenient environment, with all care provided at one location. Our CF team includes:
- pulmonologists
- cystic fibrosis clinical nurse specialists
- registered dietitians
- respiratory therapists
- physical therapists
- social workers
An integrated electronic medical record allows all of your child’s doctors and caregivers to communicate in “real time” and keeps all team members up to date on your child’s care and progress.
Respiratory Care
Because monitoring and maintaining good respiratory function is essential for all children with CF, several tests are standard parts of the routine care we provide:
- chest X-rays
- bacterial studies of sputum (mucus from the chest) to look for the growth of bacteria in the lungs
- pulmonary function tests (PFTs) to measure lung function
Lung function is one of the primary ways we track the overall health of our CF patients. Our caregivers follow all the national guidelines for care, and because of our participation in quality improvement efforts, we have seen a consistent upward climb in our patients’ overall lung function.
We also provide routine pulmonary therapy (treatments to maintain lung function). Our respiratory therapists are skilled in airway clearance techniques and can educate children and families on the most effective ways to maintain clear airways. Our nurses are also available to provide ongoing education and to answer any questions you may have.
Nutritional Support
Another part of our routine care includes blood tests to evaluate your child’s nutritional status. CF puts children at risk for slow growth, difficulty gaining weight, and a decreased ability to fight infection. They are also likely to have a condition called pancreatic insufficiency, which means that the digestive enzymes created in the pancreas aren't properly passed into the intestines.
By providing good overall nutrition, along with extra fat and calories and prescribed vitamin and enzyme supplements, you can help your child with CF grow healthy and strong. A CF registered dietitian can design a medical nutrition therapy plan for your child, as well as track his or her growth and weight gain over time.
Education and Emotional Support
Education is a key component in our goal to help children and families manage CF. We teach families what to do in emergencies, provide education and the latest information about the disease, and host support groups with speakers and topics ranging from insurance issues to community and school resources. Social workers are available to help individual families with issues like funding care, insurance coverage, and transitioning teenagers into adult CF programs.
We also take into account the emotional toll of living with CF. Children with CF often have periods where they cannot interact with other children because of the risk of developing infections. Nemours CF experts understand how lonely this can be, so they’ve developed resources to help children cope with these feelings. They offer newsletters and support groups, and often pair children of similar ages so they can develop friendships among their peers. In addition, Nemours is developing an online chat service for CF patients.
Similar resources are available for families, so that they are able to become advocates for their children’s needs.
Nemours CF teams are also active participants in quality improvement projects and research. We’re involved in a number of studies looking at new drugs, and we also have a grant to examine how using our electronic medical record, NemoursOne, helps us to determine best practices and improve care.
We do all of this because we are committed to improving the quality of life for children with CF, so they can remain active and physically able to participate in the activities they enjoy, and achieve their best possible health.
The Nemours Aerosol Research Laboratory in Orlando was founded in 1999 to evaluate delivery of inhaled therapies. The delivery of drugs directly to the lungs by the inhaled route improves efficacy and reduces side effects for treatment of lung diseases such as cystic fibrosis (CF).
Many of the new drugs are for treatment of CF lung disease. The CF Foundation estimates that there are more than two-dozen new drugs in development for the disease, and many require inhalation. Some of the new drug entities include inhaled antibiotics, airway wetting and clearance agents, long-acting bronchodilators, antioxidants, gene-modifying agents, and gene replacement therapy.
With so many treatments for CF that require inhalation, the time-burden on the patient is so great that non-adherence with therapy becomes a real problem. Therefore, this laboratory explores efficient new technologies to shorten treatment times and participates in clinical research projects designed to improve treatment adherence and study new therapies.
Nemours is in a unique position to advance the progress in inhaled drug development. Researchers have the capability of testing various drugs and delivery devices in the lab and have the clinical population and experience to carry that research forward into phase 1 through phase 3 testing. This laboratory also has close ties to industry both on the device side and the drug-development side and often acts as an intermediary for companies seeking to find the most efficient way to aerosolize their new drugs, thus achieving the maximum benefit for children (and adults) with chronic lung diseases. With expertise in aerosol science as well as clinical experience, researchers can offer the pharmaceutical and biotech industries significant value-added services that 1) reduce drug-development time, 2) offer new therapies to Nemours patients, and 3) reduce the overwhelming burden of treatment on patients with chronic lung diseases.
Examples of the Aerosol Lab partnering with industry for drug development include the in vitro assessment of delivery devices for PulmozymeÒ, tobramycin, levofloxacin and a-1 antitrypsin for CF, and nitrite for pulmonary hypertension. On the clinical side, the lab was the leading enrolling site for phase 1 and 3 studies of inhaled tobramycin powder and phase 1 and 2 studies of inhaled levofloxacin.
The unique strategy employed by the Aerosol Lab for clinical trials has led to success in enrollment and has allowed investigators to participate in the data analysis and present the data from multicenter trials both at meetings and in publications. It opens up new avenues for patients to experience cutting-edge therapies and give them hope for a better tomorrow. It also has allowed Nemours to participate on a national and international level to affect the care of children with CF and respiratory diseases.
Nemours Aerosol Research Lab and Clinical Studies include:
- Long-term administration of inhaled mannitol in cystic fibrosis: a safety and efficacy study, sponsored by Pharmaxis Ltd.
- MPEX-204: A phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and efficacy of three dosage regimens of MP-376 solution for inhalation given for 28 days to stable cystic fibrosis patients, sponsored by Mpex Pharmaceuticals.
- Phase 3 study of inhaled aztreonam lysinate in patients with cystic fibrosis and mild lung disease, sponsored by Gilead.
- Randomized, placebo-controlled, multicenter study to evaluate the safety and efficacy of ciprofloxacin (inhaled) compared to placebo in patients with cystic fibrosis (Bay q3939), sponsored by Bayer.
- Mpex 206: a Phase 1B, multicenter, open-label study to evaluate the safety, tolerability and pharmacokinetics of MP-376 inhalation dolution given daily for 14 days to stable pediatric cystic fibrosis patients, sponsored by Mpex.
- A phase 2, double-blind, multicenter, randomized, placebo-controlled trial evaluating fosfomycin/tobramycin for inhalation in patients with cystic fibrosis and Pseudomonas aeruginosa, sponsored Gilead.