Greetings:
The National Interns and Residents Match is complete and we are very pleased to have a terrific group of new residents, seven Pediatrics and three Med/Peds.
Our Annual Spring Symposium in conjunction with the Kansas Chapter of the American Academy of Pediatrics was held on Friday, May 7, 2004 in Wahl Hall East. The hot topics were “Preventing a Lost Generation: Recognizing Major Children’s Health Issues in 2004”. The workshop on Thursday, May 6, 2004 included presentations related to the main topics. The KAAP dinner and talk featuring Stephen Edwards, MD, FAAP, AAP, immediate past president of the American Academy of Pediatrics, was held on Thursday, May 6, 2004 at the McCormick and Schmicks Seafood Restaurant.
Pediatric Grand Rounds held their last program for the year on June 4th and will resume on September 10, 2004 in Lied Auditorium.
Professor and Chair
Cystic fibrosis (CF) is the most common lethal genetic disease in the United States. It affects approximately 30,000 people, and 1 in 25 individuals of northern European extraction are asymptomatic carriers of abnormal CF genes. The disorder is caused by mutations of the CF gene, resulting in abnormal function of cystic fibrosis transmembrane regulator. The diagnosis of CF is established through (1) two CF mutations or two quantitative pilocarpine iontophoresis sweat chloride values of 60 mEq/L or higher, (2) an abnormal nasal transepithelial potential difference, and (3) one or more of the following clinical features:
The goals of management of CF are to promote good nutrition and normal growth, to delay or prevent lung disease, and to recognize and treat complications of the disease. Good nutrition and growth are promoted by pancreatic enzyme replacement, using microsphere-coated capsules to protect the enzyme from gastric acid.
Reports from United Kingdom and the United States have reported strictures of the ascending colon — meconium ileus equivalent — associated with high-concentration pancreatic enzyme capsules. Reducing capsule size and limiting the amylase has drastically reduced these complications.
Adequate treatment includes life-long supplementation with vitamins, particularly fat-soluble vitamins. The recommended doses of A and D are double the usual doses for infants and children.
In a European study by Campana, et al., treatment with inhaled colistin and oral ciprofloxacin achieved eradication of PA in 82.4% of patients. Tests of antimicrobial resistance patterns demonstrated that eradication of PA prevents the development of antibiotic resistant strains.
At KU’s CF Center, our approach is similar. Patients are given oral ciprofloxacin for three weeks plus inhaled preservative-free tobramycin (TOBI) for three months. In stubborn cases, patients receive outpatient IV antibiotics and TOBI twice daily for one month. TOBI is then given chronically in alternate months. Newer therapies include tri-weekly azithromycin, which has anti-inflammatory properties and affects Pseudomonas binding.
Recent recommendations by the National Cystic Fibrosis Foundation include:
Current research is aimed at replacing the defective CF gene, with clinical trials under way.
Due to improved diets, adequate vitamin supplementation and aggressive therapy for pulmonary infection, both length and quality of life have increased, with median survival now in the 30s. Although both genders benefited from this trend, female patients have had consistently poorer survival rates.
The first successful heart/lung transplant operations in CF patients were performed in 1983 and 1987. For the terminally-ill patient with severe lung disease, lung transplantation has now become a reality, and the waiting period has been somewhat less than before. New criteria have been developed; precedence and preference will be given to those who are most in need of immediate transplantation. Since the mid-80s, nearly 450 CF patients in the U.S. alone have undergone thoracic transplantation. International registry documents over 5,000 living transplants, for CF patients nearly 800 or 14% of all lung transplants. There are 113 accredited CF care centers in the U.S., and over 60 perform lung transplantation. Age of recipients ranges from 5 to 59, with a mean of 26; 80% are 18 years and older. The availability of liver transplantation also has made a big difference in CF patient survival.
Although much research still is needed, we can offer more than ever to your patients with CF. If we can serve you in any way, please give us a call.
Director, Cystic Fibrosis Center
913-588-6377
Do you find your child failing asleep at unusual times? Are there indications of poor school performance because of low levels of alertness? Has you child shown episodes of perceived clumsiness such as dropping cups or spilling drinks when surprised? Does your child have terrifying dreams or has he ever mentioned not being able to move as he falls asleep? If so, then your child might have narcolepsy.
First symptoms often develop near puberty with peak age between fifteen and twenty-five years of age. Some patients have been reported to have symptoms as early as 3-6 years old.
There is no gender predominance. A special circumstance such as an abrupt change of sleep-wake schedule or a severe psychological stress — e.g., death of a relative, divorce — precedes the occurrence of the first symptom in half of the cases.
The most common symptoms are excessive daytime sleepiness and irresistible sleep episodes, especially at idle times. Attacks of cataplexy often appear at the same time but can occur as late as 20 years later. Nocturnal hallucinations and sleep paralysis are not universal and often transient.
Diagnosis requires a minimum of two major symptoms of excessive daytime sleepiness (EDS) and sleep attacks or cataplexy, associated with objectively-documented sleep-onset REM episodes confirmed by a Multiple Sleep Latency Test (MSLT). An MSLT measures physiologic sleep tendencies polygraphically during five naps. The mean sleep onset (sleep latency) and REM onset are determined for each nap. Normal MSLT scores vary with age but, in general, a mean sleep latency under eight minutes is considered pathologic. Two REM onsets within any of the five twenty-minute naps is diagnostic of narcolepsy if other causes of EDS (such as sedating medications or inappropriate sleep-wake cycle) have been eliminated. A negative nocturnal polysomnogram the night prior to the scheduled MSLT is necessary to rule out other reasons for EDS.
Treatment for prepubertal children should include close communication with the child’s teachers and care providers. Scheduling naps at lunchtime and between 4 and 5 pm will help with the daytime sleepiness. For older children, emphasis on a regular nocturnal sleep schedule with the goal of nine hours of sleep is beneficial. Naps at noon and around 4 to 5 pm should continue. In the past, medications have focused on stimulants such as methylphenidate. Milder cases have been treated with pemoline which is not a schedule II drug and often is tolerated better than other stimulants. However, this is no longer a first-line drug because of associated liver toxicity. More recently, Modafinil or Provigil have been used in narcolepsy. These are given only once daily, have low abuse potential and are not schedule II drugs. In general, they are felt to have fewer side effects than methylphenidate and other stimulants. However, this has not been proven nor has efficacy been proven to be better than traditional treatments in randomized controlled studies.
If you suspect your child may have narcolepsy you should discuss this with your pediatrician. He/she would most likely recommend your child be evaluated by an individual who is familiar with sleep disorders in children such as a pediatric pulmonologist, neurologist, or sleep medicine specialist.
Division Chief, Pediatric Pulmonology
913-588-6377
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The University of Kansas Medical Center