A "Special" Kind of Care
Shortage of Pediatric Subspecialists Makes Training Next Generation a Priority

BY MITZI BAKER

WINTER 2005 -- At many hospitals, the same specialist will treat a tiny one-day-old baby born with a congenital kidney defect and a 70-year-old diabetic woman on kidney dialysis. Yet children tend to experience different medical problems from adults, and specialists at community hospitals may have little training and experience dealing with specific pediatric conditions.

"What is needed for optimal care is someone who really understands not only kidney problems, but kids with kidney problems," says Harvey Cohen, MD, PhD, the Adalyn Jay Chief of Staff and the Arline and Pete Harman Chair of the Department of Pediatrics.

"Even if they had a similar kidney problem, it's really different what you do for a one-day-old compared to a 70-year-old. The margin of error is much less for a newborn. You have to do things much more rapidly, and in ways that relate to the child’s developmental stage," explains Cohen, who has long been an advocate of specialization within pediatrics. "A stay in the hospital itself can affect how a child develops later in life," he adds.

Today, a wide range of pediatric subspecialties exist, ranging from immunology and oncology, to orthopedics and cardiology.

"As the complexity of care and our understanding of children's diseases improve, we have more and more need for pediatric subspecialists," says Packard pediatric immunologist Alan Krensky, MD, the Shelagh Galligan Professor in the School of Medicine.

Problem is, there aren't enough of them to go around. Currently, pediatrics attracts only 10 percent of all medical students, with a meager 2 percent going on to train in a pediatric subspecialty.

Lucile Packard Children's Hospital aims to help fill in this gap by focusing on training the next wave of pediatric subspecialists. Thanks to the $500 plus million investment from the successful Campaign for Lucile Packard Children's Hospital, Packard has been able to add more than 44 new pediatric subspecialists to its existing faculty during the last five years, strengthening leadership in both clinical and research programs.

"You build a house from the bottom up, starting with the foundation, the first floor, etc," says Cohen. "Academic programs, however, are built from the top down, bringing in the leaders first, then the junior faculty, and then the trainees."

But in addition to training a pediatric endocrinologist, for example, Hospital leaders hope to inspire innovative problem solvers who can develop novel approaches to curing diabetes by exchanging ideas and knowledge with engineers, physicists, chemists, stem cell biologists, and other Stanford scientists.

"By pushing forward, kids with diabetes will be cured. Kids with heart disease and cancer will be cured. All of the chronic care issues will be things of the past," Krensky says. "But that is only going to happen through subspecialty care and by connecting young doctors interested in pediatrics with people on this campus who are on the cutting edge of technology."

At Packard, pediatric immunologists, otolaryngologists, neonatologists, and many other subspecialists are joining forces with basic and clinical scientists to attack the illnesses that devastate children's lives.

Kari Nadeau, MD, PhD
Pediatric Immunologist

Five years ago, Kari Nadeau, MD, PhD, was the vice-president of a biotech company, far from the colorful exam rooms in Packard's new outpatient clinic. Then one day in 2001, her one-month-old daughter became seriously ill with respiratory syncytial virus, or RSV. She was in the Hospital for nearly three months, including a two week stay in Packard's intensive care unit. "I was totally amazed at the care she got here," says Nadeau.

Pediatric immunologist Kari Nadeau, M.D., Ph.D., monitors 3-year-old Lesley Martinez's immunogloblin levels after the little girl's second liver transplant.

Her experience with her daughter's illness prompted her to go back into the hands-on practice of medicine. Nadeau finished a fellowship in pediatric allergy, asthma and immunology at Packard, gaining experience treating children with severe immune system deficiencies, as well as those with allergies and asthma.

"Asthma is a horrible disease that affects up to 20 percent of kids today. Yet we still treat it with medicines that have been around for decades," says Nadeau. By comparing immune cells in blood and bronchial fluid samples of patients with asthma to those without, she hopes to find new therapies aimed at these immune cells to counteract some of the problems she sees in her patients. Her research focuses on finding out how to tame an overly active immune response that is at the root of allergies, asthma, autoimmune diseases, and the rejection of transplanted organs.

One of her patients is a 3-year-old named Lesly Martinez, who came to Nadeau after her second liver transplant. Lesly's liver turned cancerous soon after she was born and her body rejected the first attempt at transplantation with a healthy liver. The second try appears to be successful except for Lesly's extremely low levels of immunoglobulins, proteins in the blood critical for a strong immune system. Nadeau is treating her using medications and infusions of immunoglobulins to help Lesly fight infections.

Nadeau also has helped initiate a pediatric clinical trial at Packard to see if an experimental asthma therapy called anti-IgE could work for treating children with food allergies and eczema. Early intervention of therapies to treat allergies, she says, can stop the all-too-common progression to asthma. Nadeau says conducting clinical trials in children is critical to understanding how a child's immune system develops. "You can't do developmental research in adults," she says. "It has to be done in pediatrics."

Peter Koltai, MD
Pediatric Otolaryngologist

In medical school, Peter Koltai, MD, thought that he wanted to be an orthopedic surgeon, but he became intrigued one day while observing an ear surgery. Instead Koltai decided to train in general otolaryngology, a subspecialty that concentrates on disorders and diseases of the ear, nose, and throat. "Then it became obvious to me that I loved working with kids and that I had a gift for dealing with the fears of parents," he says, "so I gravitated more and more to the pediatric subspecialty."

One-year-old Oliver VonFeldt at a follow-up appointment with pediatric otolaryngologist Peter Koltai, M.D., almost a year after Koltai repaired the boy's laryngeal cleft.

Koltai soothes anxious parents by conveying to them that he is shouldering full responsibility for their child. "I want parents to know I have a higher level of commitment than just meticulous care," he explains. "When I take your kid into the OR, it's as if I'm taking my own child."

Parents also can be assured by Koltai's experience, which spans more than 25 years, including six as head of pediatric otolaryngology at Cleveland Clinic Children's Hospital. Just over a year ago, Koltai was recruited to Stanford as Packard's chief of pediatric otolaryngology to further develop a program in airway reconstruction for children.

Pediatric otolaryngology problems, says Koltai, are often quite different from those seen in adults. For example, pediatric head and neck surgical cases generally involve congenital abnormalities, such as lymphatic malformations or developmental cysts, while diseases such as head and neck cancer are more common in grown ups.

This year, little Christian Myers presented Koltai with an especially interesting and rewarding case. The boy was born with a laryngeal cleft—a congenital abnormality in which the partition between the voice box and the esophagus is not completely formed, causing food to go down the trachea instead of the esophagus. Christian's cleft extended below the level of the vocal cords, which usually meant the repair would need to be done externally.

Instead, Koltai wanted to make the procedure less invasive and speed recovery time by doing the surgery endoscopically, using a method he had helped develop for clefts that were not as deep as Christian's. "The procedure was technically challenging," says Koltai, "somewhat like building a ship in a bottle using chopsticks." But the surgery worked beautifully. Christian was able to begin eating normally after the surgery and just returned after almost a year for a follow-up visit.

Always in a search for better surgical options for his young patients, Koltai is looking forward to setting up an airway research lab to explore new methods of robotic laryngeal surgery and developing different types of synthetic materials that can be used for airway reconstruction.

Susan Hintz, MD
Neonatologist

Neonatologist Susan Hintz, MD, knew she wanted to specialize in the problems of newborns from the time she was an undergraduate, working in the lab of David Stevenson, MD. "It seemed to me that I could potentially help an infant in need and make a difference that would last for his or her whole, hopefully long, life," she says.

Neonatologist Susan Hintz, M.D., has guided the care of Erick Sanchez-Zamudio during his lengthy stay in Packard's neonatal intensive care unit (NICU).

Almost 20 years later, Hintz is still working with Stevenson, who is now director of the Johnson Center for Pregnancy and Newborn Services at Packard. Hintz did all of her medical training at Stanford, and applies her initial interest in newborns daily. The need for clinical innovations in the field has spurred Hintz to pursue a range of research interests studying the outcomes of extremely ill infants.

Recently, she cared for a young patient who needed a whole team of specialists, and the latest technologies.

Erick Sanchez-Zamudio was born on June 21 with a congenital diaphragmatic hernia. That, Hintz explains, means that early in gestation, the baby's diaphragm didn't close completely, so the bowel and other abdominal organs went into the chest. Having the organs all in the wrong places can mean that the lungs don't have a chance to form properly. This is what happened in Erick's case.

Fortunately, Erick's condition was identified before his birth, so Hintz had an opportunity to prepare the family for the events that would occur. At birth, Erick couldn't breathe for himself and needed the assistance of a heart-lung bypass device called extra-corporeal membrane oxygenation, or ECMO, for a few weeks. He needed surgery to push his abdominal organs back down where they belonged and to create a new diaphragm out of Gore-Tex. Erick's condition was so unstable that the surgery had to take place while he was still on ECMO, right in the neonatal intensive care unit.

With a calmness that belies the troubles he has had in his short life, Erick was able to breathe on his own. "We used all the technologies available to allow that to happen," says Hintz. Advances such as ECMO and nitric oxide, an inhaled gas that opens up the vessels of the lungs to allow more blood flow, were an essential part of getting Erick this far.

"Erick's case is also an example of the level of collaboration needed in such complex cases," she says. "Pediatric surgeons and cardiologists, especially the pulmonary hypertension specialists, have been very involved in his care." But, Hintz says, despite the great strides Erick has made, he still has many difficult challenges to overcome before doctors can determine if he will be able to survive.

According to Hintz, it's critical to train young people as they launch their medical careers not just how to be good clinicians but also to understand the research needed to advance their specialized areas. "What we need to know about disease processes is becoming increasingly complex," she says. "We really want to train young people to work on the next generation of problems facing sick children."