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Cardiovascular Medicine, In the News, Pediatrics, Surgery

Marathon surgery at Stanford gives 6-year-old boy a chance to thrive

Marathon surgery at Stanford gives 6-year-old boy a chance to thrive

image.img.320.highA rare chromosomal disorder called Williams syndrome left 6-year-old Jordan Ervin with a host of medical problems, including severe heart defects. But it also gave him a gregarious personality and an infectious smile, one that made the multiple medical appointments and hospitalizations much easier to handle, according to his mother, Seville Spearman.

“Jordan is such a champ,’’  Spearman said in a recent Inside Stanford Medicine article. “He’s always been just a really happy kid.”

And in December, he became a much healthier one thanks to the skillful work of Stanford cardiothoracic surgeon Frank Hanley, MD. More from the piece:

It was a complicated case. The stenotic arteries caused severe pulmonary hypertension. In less-severe cases, in which there is only one area of stenosis near or at the pulmonary valve, doctors can perform a fairly simple surgical catheter procedure that uses a tiny balloon to expand the artery. But Jordan had multiple narrowings: 12 in his left lung and 14 in the right lung. The balloon technique is much less effective in this scenario, and no other surgical techniques have been developed to treat these stenoses. So Jordan would need a different approach.

That approach was developed by Hanley, who receives referrals from all over the world. He’s the pioneer of a one-stage, fix-all-the-defects surgery called unifocalization.

“We’re definitely on the leading edge of this kind of surgery,’’ said Hanley, who holds the Lawrence Crowley, MD, Endowed Professorship in Child Health. “Jordan is going to have perfectly normal life expectancy.”

Ervin is back in school in Illinois, where his parents are delighted with the outcome. His mother said in the story, “Everything is back to normal, but I will never take anything for granted again.”

Previously: How better understanding Williams syndrome could advance autism research, Pediatric surgeon fixes “heart that can’t be fixed” and Patient is “living to live instead of living to survive,” thanks to heart repair surgery
Photo by Norbert von der Groeben

Events, Pediatrics, Public Health, Research, Stanford News

Countdown to Childx: Q&A with pediatric health expert Alan Guttmacher

Countdown to Childx: Q&A with pediatric health expert Alan Guttmacher

jumpforjoyIt’s just a few weeks until the inaugural Childx conference, a TED-style meeting at Stanford that will highlight innovations in health problems of pregnancy, infancy and childhood. (Conference registration for the April 2-3 event is still open, with details available on the conference website.) Childx is attracting nationally and internationally prominent speakers: keynotes will be given by Alan Guttmacher, MD, head of the Eunice Kennedy Shriver National Institute of Child Health and Human Development, and by Rajiv Shah, MD, former head of USAID.

I spoke recently with Guttmacher about the upcoming conference. Because I spend most of my time working with scientists who focus their attention on specific research niches within obstetric and pediatric medicine, I was interested in getting his take on the “big picture” of these fields. An edited version of our conversation is below.

What are you planning to say in your keynote address at the Childx conference?

Children’s lives are about more than just health. While biomedical research is crucial to improving kids’ lives, we should put it in the larger context of kids’ lives and do not just research that has an impact on health, but also on children’s overall well-being.

Within the health sphere, I’ll talk about several areas where we need more research. We need to study how to do a better job of preventing prematurity, both to gain a better understanding of biological and environmental causes of preterm birth, and also of how to do a better job of employing the knowledge we already have.

Another topic I’ll address is vaccination: How do we both pursue the science of vaccination to figure out how to make more vaccines more effective, and also, how do we work with parents so they make decisions about kids’ lives that are in the best interests of the kids and are evidence based, rather than based on, say, something they recently read on the web?

I’ll also discuss the developmental origins of health and disease. Pediatricians have always been very invested in anticipatory guidance, telling families about the kinds of things to do to prevent future disease for their children. But this goes farther; this is the idea that health factors, not only in childhood but even in utero, have lifelong impact on health. For instance, what happens in pregnancy potentially has large impact on whether someone develops hypertension in their 60s or 70s. We’re beginning to do science that will tell us the connections between early factors and later health, that will actually influence health along the entire age span. It’s an area of very important research.

And I’ll address intellectual and developmental disabilities. We need research to figure out how to more effectively prevent intellectual and developmental disabilities, research to understand how to allow kids who have these disabilities to function more effectively in society, and also research to figure out how to have society function better in the lives of kids with intellectual and developmental disabilities.

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Pediatrics, Pregnancy, Stanford News, Surgery

A difficult decision that saved three young lives

A difficult decision that saved three young lives

Estrada-Triplets_013I first met Lily Estrada and her identical triplets almost a year ago. The three babies, who were nearly ready to go home from Lucile Packard Children’s Hospital Stanford, looked pretty ordinary. In fact, that’s why I love the photo at the right, which was taken at the time. Baby Pedro, in blue, was gazing at his mom; Ayden, in orange, was wiggling; and William, in grey, was sucking contentedly on his pacifier.

But they had survived an extraordinarily complicated and rare prenatal disorder. The single placenta that connected all three boys to their mother during pregnancy developed a vascular problem called twin-to-twin transfusion syndrome. Blood flowing through the placenta was not being shared equally between the fetuses, straining their hearts and putting all of them at risk of dying before birth.

When Estrada was diagnosed in late 2013, she and her husband, Guillermo Luevanos, faced a difficult decision. A surgery on the placenta might help save the babies, but it was by no means a sure bet. And, at the time, no one at Stanford performed the procedure, although a new partnership between our maternal-fetal medicine experts and their counterparts at Texas Children’s Hospital, in Houston, provided an opportunity for Estrada to be treated there. In the Stanford Medicine magazine story I wrote about the case, Estrada described how her family felt:

“We were saddened and sort of confused,” Estrada says, recalling the first reactions that she and her husband had to the news. “It was: We could wait and see what happened, but the likelihood was that we were going to have no baby, or we could terminate one and see what happened with the other two, or take the risk, go to Houston, have the surgery and hope it worked for all three. But they didn’t guarantee anything.”

One piece of background that helped inform the couple’s decision was the fact that when the surgery worked, research had shown it helped moms stay pregnant about four weeks longer, allowing their babies more time to develop before birth. (Because the uterus gets so crowded, twins and other multiples are almost always born early, but a less premature delivery makes a huge difference for the babies’ health.) Sealing the connecting blood vessels also seemed to protect surviving fetuses in the event that one died. “We’re separating, or attempting to separate, their fates,” [Estrada’s obstetrician] Yair Blumenfeld, MD, says.

After a lot of counseling and discussion with the Stanford team, “we decided to go for it and do surgery,” Estrada says.

Once they had made the choice, they had no second thoughts. “My husband was a little bit stronger,” Estrada recalls. “He just wanted me to go for it, and see what happened.”

triplets-medresThe surgery, performed at Texas Children’s by Michael Belfort, MD, PhD, was a success. And, as my story describes, the collaboration between the two institutions is going well, too. Stanford researcher Christopher Contag, PhD, and colleagues are studying how to make better and safer surgical tools for future maternal-fetal surgeries, while surgeons here have advanced their capabilities and now offer the surgery for twin-to-twin transfusion here in Palo Alto.

Meanwhile, William, Ayden and Pedro are doing well. My favorite moment in preparing the story was when I got to see our new photo of them, above. As their mom told me, “They’re really happy babies.”

Previously: NIH puts focus on the placenta, the “fascinating” and “least understood” organStanford Medicine magazine reports on time’s intersection with healthPlacenta: the video game and Program focuses on the treatment of placental disorders
Photo of triplets as infants by Norbert von der Groeben; photo of triplets as toddlers by Gregg Segal

otolaryngology, Patient Care, Pediatrics, Research

A serendipitous save that changed treatment of the most common tumor of infancy

A serendipitous save that changed treatment of the most common tumor of infancy

IsabellaManley1stgrade-cropAt research institutions like Stanford, we often talk about the value of evidence-based medical care, the kind based on careful scientific comparisons of which treatments work best.

But sometimes, even the best-studied treatments fail. That’s what happened in 2008, when a baby named Isabella Manley was brought by her parents from their Sacramento, Calif., home to Lucile Packard Children’s Hospital Stanford because of a tumor in her trachea that threatened to block her breathing. Her case illustrates that serendipity sometimes plays a key role in medical success.

Isabella had a hemangioma, the most common tumor of infancy. Most hemangiomas, which consist of extra blood vessels, create harmless red marks on a baby’s skin that fade with time. Isabella’s was much more serious. Although her doctors tried all the hemangioma treatments then reported in the medical literature, including high-powered steroid drugs and two types of surgery, her breathing problems persisted. Pediatric otolaryngologist Kay Chang, MD, who oversaw her care at Stanford, ordered an MRI to find out why. A story I wrote about her case explains what happened next:

“We found, to our horror, that this hemangioma was massive, surrounding her entire windpipe and also her heart,” Chang said.

“It was becoming tangled into every structure in her neck and crawling down into her chest,” said Mai Thy Truong, MD, a pediatric otolaryngologist now with the hospital’s vascular malformations clinic.

… The tumor was too extensive for surgery and was still growing. Truong and Chang feared that it would soon block Isabella’s airway. They were not sure they could save her.

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Genetics, Pediatrics, Podcasts, Research, Stem Cells

Countdown to Childx: Stanford expert highlights future of stem cell and gene therapies

Countdown to Childx: Stanford expert highlights future of stem cell and gene therapies

RoncaroloNext month’s inaugural Childx conference will bring a diverse group of experts to Stanford to discuss big challenges in infant, child and maternal health. Today, in a new 1:2:1 podcast interview, stem cell and gene therapy expert Maria Grazia Roncarolo, MD, provides an interesting preview of a once-controversial area of research that will be featured at the conference.

Roncarolo talks about the history and future of stem cell and gene therapy treatments, which have recovered from tragic setbacks such as the 1999 death of 18-year-old Jesse Gelsinger in an early gene therapy trial. The early problems forced researchers to reevaluate what they were doing, with the result that the entire field has reemerged stronger, she explains:

I would say that there were major problems, that we underestimated the complexity that it takes to manipulate the genome, and to introduce a healthy gene to fix a genetic disease. However, from these mistakes and from these tragedies, we learned a lot. We were really forced as doctors, and more importantly, as scientists, to go back to the bench and develop better technologies and to understand more of what was required. … [Today] we use better vectors — which are the carriers to introduce the healthy gene — we know much more about what we have to do to prepare the patient to receive the gene therapy, and we also learned that we need to do a very careful monitoring of the patients to really understand where the gene lands in the genome.

At the Childx conference, Roncarolo will moderate a panel on “Definitive Stem Cell and Gene Therapy for Child Health,” hosting such guests as GlaxoSmithKline’s senior vice president of rare diseases, Martin Andrews, and Nadia Rosenthal, PhD, founding director of the Australian Regenerative Medicine Institute.

Information about registration for Childx, being held here April 2–3, is available on the conference website.

Previously: Stanford hosts inaugural Childx conference this spring and Stanford researchers receive $40 million from state stem cell agency
Photo by Norbert von der Groeben

In the News, Infectious Disease, Nutrition, Pediatrics

Raw milk still a health hazard, says Stanford doctor

Raw milk still a health hazard, says Stanford doctor

MoooooooIn spite of looser regulations around the sale of unpasteurized milk, it’s still unsafe to drink. That’s the message from Stanford pediatric infectious disease expert Yvonne Maldonado, MD, who is quoted in a new story on Today.com about the relaxation of raw-milk regulations in West Virginia and Maine.

In the United States, each state writes its own rules for in-state sales of raw milk, and they vary — a lot. Until last week, West Virginia required all dairy products sold in the state to be pasteurized, or heated briefly to kill germs. The state’s new laws allow for “cow shares,” in which individuals can pay to share ownership of a cow in exchange for some of the cow’s unpasteurized milk. Maine, meanwhile, is considering relaxing its license regulations on farmers who sell milk directly to consumers. (Other states take different approaches, ranging from entirely banning raw milk sales to allowing it in retail stores.)

Raw-milk aficionados claim that unpasteurized dairy products are safe and have health benefits.

Not so fast, says Maldonado, who was the lead author of the American Academy of Pediatrics’ 2013 policy statement discouraging the consumption of raw milk. In the Today.com story, she explains:

“People want to be more responsible for their sustainable environment and what they are putting into their bodies but they conflate the two issues because natural doesn’t always equal healthy,” says [Maldonado].

… “Our recommendations are evidence-based and there is no scientific evidence that drinking raw milk is better than drinking pasteurized milk and milk products,” says Maldonado, an infectious disease expert and pediatrician at Lucile Packard Children’s Hospital. “But we do see a very large number of diseases and illnesses from raw milk and raw milk products and the infections can be just horrible,” causing diarrhea, fever, cramps, nausea and vomiting, and some may even become systemic.

Previously: Stanford pediatrician and others urge people to shun raw milk products and Stanford study spoils hopes that raw milk can aid those who are lactose-intolerant
Photo by Steven Zolneczko

Addiction, FDA, Health Policy, Pediatrics, Public Health

Raising the age for tobacco access would benefit health, says new Institute of Medicine report

Raising the age for tobacco access would benefit health, says new Institute of Medicine report

cigarette packToday, the Institute of Medicine released a new report evaluating the public health effects of reducing teenagers’ access to cigarettes and other tobacco products. Right now, in most places in the United States, you must be 18 years old to buy cigarettes and other tobacco products. But a few states and cities have higher minimums, and in 2013, the IOM convened a committee, at the request of the U.S. Food and Drug Administration, to examine the potential effects of a higher minimum legal age for tobacco access across the country.

The committee, which was led by Richard Bonnie of the University of Virginia and included Stanford adolescent medicine expert Bonnie Halpern-Felsher, PhD, reviewed the existing scientific literature on tobacco use in teens. They also devised mathematical models to predict what would happen if the federal minimum legal age were 19, 21 or 25.

The report brief (.pdf) says, in part:

Based on its review of the literature, the committee concludes that overall, increasing the MLA [minimum legal age] for tobacco products will likely prevent or delay  initiation of tobacco use by adolescents and young adults. The age group most impacted will be those age 15 to 17 years. The committee also concludes that the impact of raising the MLA to 21 will likely be substantially higher than raising it to 19. However, the added effect of raising the MLA from 21  to 25 will likely be considerably less.

The parts of the brain most responsible for decision making, impulse control, sensation seeking, and susceptibility to peer pressure  continue to develop and change through young adulthood, and adolescent brains are uniquely vulnerable to the effects of nicotine. In  addition, the majority of underage users rely on social sources—like family and friends—to get tobacco. Raising the MLA to 19 will therefore not have much of an effect on reducing the social sources of those in high school. Raising the MLA to 21 will mean that those who can legally obtain tobacco are less likely to be in the same social networks as high school students.

Although it can take time to fully realize the benefits of reduced smoking, since heart disease, lung cancer and other diseases linked to smoking take decades to develop, the payoff would ultimately be significant, the report adds:

…if the MLA were raised now to 21 nationwide, there would be approximately 223,000 fewer premature deaths, 50,000 fewer deaths from lung  cancer, and 4.2 million fewer years of life lost for those born between 2000 and 2019.

Previously: How e-cigarettes are sparking a new wave of tobacco marketing, To protect teens’ health, marijuana should not be legalized, says American Academy of Pediatrics and UN’s top health official: Anti-tobacco efforts can lead to better health “in every corner of the world”
Photo by Thomas Lieser

Mental Health, Nutrition, Pediatrics

Incorporating the family in helping teens overcome eating disorders

Incorporating the family in helping teens overcome eating disorders

Apple on plateLearning that your teen has an eating disorder is baffling and deeply troubling news for parents. Our instincts are to protect and try to help our children out of the morass, but for decades, families were kept out of the treatment loop for teens with conditions like anorexia and bulimia.

The team at the Comprehensive Eating Disorders Program at Lucile Packard Children’s Hospital Stanford, led by psychiatrist James Lock, MD, PhD, is integrating the family into helping teens overcome their eating disorders. Lock recently sat down with the Stanford Medicine Newsletter for an informative Q&A about teen eating disorders.  He spoke about the historic reasoning for cutting parents out of treatment plans:

For most of the early 20th century, parents were erroneously blamed for mental illnesses in their offspring: So-called refrigerator mothers (those lacking warmth) caused autism, and overcontrolling parents caused anorexia nervosa, experts claimed. These ideas about causation are without foundation.

Research at Stanford and elsewhere has shown that parents can play a big role in helping their teens recover from eating disorders. For example, we have demonstrated that a specific family-based therapy is twice as effective as individual psychotherapy for treating anorexia nervosa.

And what to watch for in teens:

Warning signs include changes in eating patterns, skipping meals, increased driven exercise or discussion about weight, avoidance of desirable but calorically dense foods, refusing to eat with the family, vomiting, large amounts of food missing from the refrigerator and increased irritability and emotionality. If a parent sees these signs, it would be a good idea to make an appointment for an evaluation and consultation.

The full Q&A is worth a read.

Previously: Families can help their teens recover from anorexia, new study showsA growing consensus for revamping anorexia nervosa treatmentPossible predictors of longer-term recovery from eating disordersWhat a teenager wishes her parents knew about eating disorders and Research links bulimia to disordered impulse control
Photo by daniellehelm

Cardiovascular Medicine, Pediatrics, Pregnancy, Surgery

Baby with rare heart defect saved by innovative surgery

Baby with rare heart defect saved by innovative surgery

jackson-lane-stanford-childrens560

Elyse Lane was 20-weeks pregnant when she learned that her unborn son had a rare and severe heart defect. Her baby was missing his pulmonary valve and his pulmonary artery was 10 times the normal size.

The outlook was bleak. The baby’s enlarged artery hampered his blood and oxygen flow, a condition called tetralogy of Fallot, and his missing pulmonary valve made the defect worse.

Fortunately, Lane and her husband, Andy Lane, a former Major League Baseball coach with the Chicago Cubs, were referred to Frank Hanley, MD, a cardiothoracic surgeon at Stanford Children’s Health. Hanley had experience with this kind of heart defect and knew how to perform the delicate surgery needed to repair their baby’s heart.

The Lanes recount the story of their son’s lifesaving surgery on the Lucile Packard Children’s Hospital blog:

When he was just five days old, Jackson underwent a 13-hour operation that would save his life. Hanley and his team did a complex overhaul of Jackson’s heart: they inserted a pulmonary valve, reduced the size of Jackson’s right pulmonary artery, and enlarged his small, disconnected, left pulmonary artery. Hanley also used an innovative and intricate procedure known as the LeCompte maneuver, which altered the pathway of Jackson’s right and left pulmonary arteries from the back of the heart and aorta to the front. This gave his severely compromised bronchial tubes room to grow and remodel after surgery was over.

As the story explains, Jackson’s heart will need some maintenance in the future, but he should live a normal and long life.

“He can now do anything he wants in life,” said Elyse Lane in in the blog piece. “He’s already made it through the biggest challenge.”

Previously: Patient is “living to live instead of living to survive,” thanks to heart repair surgery, A very special small package: Three-pound baby receives pacemaker, Advancing heart surgery for the most fragile babies, and Little hearts, big tools
Photo courtesy of Lucile Packard Children’s Hospital

Immunology, In the News, Nutrition, Pediatrics, Research

Peanut products and babies: Now okay?

Peanut products and babies: Now okay?

peanut butter2 - big

Updated 2-25-15: Allergy expert Sharon Chinthrajah, MD, discussed the study and its implications on KQED’s Forum today:

***

2-24-15: Any parent of young children is likely familiar with the warnings: It’s not okay to give your baby peanut butter, or any other peanut product, before he or she turns one. Don’t do it! These instructions are so imprinted on my brain that I practically did a double-take when I came across headlines about new research suggesting that infants should, indeed, be fed peanut products – in order to prevent the development of peanut allergies.

This isn’t the first time that the benefits of giving allergenic foods to babies have been outlined, but the language surrounding this study has been particularly strong. As the writer of a New York Times blog entry explained, the authors of the study and accompanying editorial “called the results ‘so compelling’ and the rise of peanut allergies ‘so alarming’ that guidelines for how to feed infants at risk of peanut allergies should be revised soon.” He went on to outline the study findings:

In the study, conducted in London, infants 4 to 11 months old who were deemed at high risk of developing a peanut allergy were randomly assigned either to be regularly fed food that contained peanuts or to be denied such food. These feeding patterns continued until the children were 5 years old. Those who consumed the foods that had peanuts in them were far less likely to be allergic to peanuts when they turned 5.

After hearing the news, I reached out to the folks at the Sean N. Parker Center for Allergy Research at Stanford to get their take on the findings. Sharon Chinthrajah, MD, a clinical assistant professor of medicine, explained that this work is the first randomized controlled study to look at how to prevent peanut allergies. She told me:

We’ve all been waiting for the results of this landmark study to confirm the shift in the paradigm of when to introduce foods into the diet. Early introduction of peanut in the right infants can prevent peanut allergy. Dr. [Gideon Lack, the leader of the study] and colleagues were able to show an 80 percent reduction in peanut allergy in children who started eating peanut early and incorporated it into their regular diet.

Chinthrajah believes the guidelines on babies and peanut products should be revised, “because peanut allergies affect 2 percent of our population in the U.S. and most people do not outgrow this allergy.” But, as other experts have done, she cautions that not everyone should introduce peanuts and other foods into their diet early. “Those who are ‘high-risk’ – who have other allergic conditions such as eczema or other food allergies – should consult with their allergist to see if it would be safe to introduce peanut into their child’s diet,” she advised.

Previously: Taking a bite out of food allergies: Stanford doctors exploring new way to help sufferers, Simultaneous treatment for several food allergies passes safety hurdle, Stanford team shows, Researchers show how DNA-based test could keep peanut allergy at bay, A mom’s perspective on a food allergy trial and Searching for a cure for pediatric food allergies
Photo by Anna

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