Tag Archive | "Department of Pediatrics"

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Miller School pediatric researchers publish study on heart disease in children

Professor of Pediatrics James Wilkinson, one of the study’s authors.

New findings from the Pediatric Cardiomyopathy Registry published online ahead of print in Circulation Heart Failure, a journal of the American Heart Association, provide information that could impact the diagnosis and natural progression of cardiomyopathy, a weakened or abnormal heart muscle.

The Pediatric Cardiomyopathy Registry, which is funded by the National Heart, Lung and Blood Institute of the National Institutes of Health, was set up to study the causes and natural history of the disease. A team of researchers from more than a dozen medical centers in the United States and Canada looked specifically at children in the registry who had cardiomyopathy caused by myocarditis, an inflammation of the heart. They looked at how those children were diagnosed and how their disease progressed over time.

“What we found is that children who had cardiomyopathy caused by myocarditis did much better than children who had dilated cardiomyopathy from an unknown cause,” said Steven E. Lipshultz, professor and chair of pediatrics at the University of Miami Miller School of Medicine, principal investigator, and senior author of the study. “Of those same children with myocarditis, those who had early remodeling of their left ventricle and with more heart wall thinning, did worse over time than if remodeling had not taken place.”

Another important finding from the study was that it didn’t matter how the problem was diagnosed in terms of clinical outcome. In older children and teenagers, if someone is suspected of having myocarditis, a biopsy is conducted by removing a piece of the heart muscle at some centers with expertise in this procedure. In younger children this can often be a risky procedure because a child must be sedated and their heart wall is much thinner, so making punctures to remove a sample is not without risk.

“Many people have thought a biopsy is essential to diagnose myocarditis in children, but our findings show that children diagnosed with myocarditis through a biopsy had the same future course as children who did not have heart muscle biopsies done, but whose pediatric cardiologists thought clinically they had myocarditis,” said James Wilkinson, professor of pediatrics and one of the study’s authors. “Something else we found is that the children with myocarditis who presented with the most evidence of cardiac abnormalities tended to be the same children who got better during the period of follow-up we used.”

Dilated cardiomyopathy affects 1 in 100,000 children, and nearly 40 percent with symptomatic disease receive a heart transplant or fail medical management and die within two years of diagnosis. The Pediatric Cardiomyopathy Registry has information on more than 4,000 children from 1990 through 2010.

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Miller School study bears hope for young cancer survivors

Steven E. Lipshultz

A new study published online last week ahead of print in Lancet Oncology on its fast track shows that using dexrazoxane—an iron chelator that reduces free-radical injury in some settings—to prevent heart damage in young cancer patients during chemotherapy provides long-term cardioprotection without compromising cancer treatment.

The study is a five-year follow-up to a 2004 landmark study published in The New England Journal of Medicine that showed it was possible to reduce and even prevent heart damage, a known side effect in children receiving doxorubicin (Adriamycin®), from occurring during cancer therapy. Doxorubicin is a very effective form of chemotherapy for acute lymphoblastic leukemia (ALL).

Although about 80 percent of children with ALL achieve a cancer cure, they face delayed toxic effects from ALL and its therapies years later. Cardiac toxicity is particularly a problem for survivors of childhood cancer, producing increased rates of heart failure and cardiac death 20 to 30 years after treatment. Children treated with doxorubicin have more than a three-fold increased risk of dying of heart disease than those who did not receive doxorubicin. Therefore, the prevention of this late cardiotoxicity in long-term survivors of childhood ALL was the primary end-point of this study and what Steven Lipshultz, professor and chair of pediatrics at the Miller School of Medicine and the study’s principal investigator, and his colleague report in Lancet Oncology.

More than 200 children newly diagnosed with high-risk ALL, the most common malignancy in children, were enrolled in this multicenter, randomized, controlled trial, conducted as part of the Dana-Farber Cancer Institute Childhood ALL Consortium platform in Boston under principal investigator Stephen Sallan. Half of the children were treated using the standard multi-agent protocol for ALL, which includes doxorubicin. The other half were treated with an infusion of dexrazoxane 30 minutes before receiving doxorubicin. Dexrazoxane had not been known to provide long-term protection of the heart in either children or adults receiving the same type of chemotherapy.

“When we were recently unblinded to the results of this study, we found that girls treated with dexrazoxane five years earlier had normal heart function and did not have any pathologic remodeling of the heart, in contrast to girls who did not receive dexrazoxane, where both heart function and remodeling showed progressive deterioration,” said Lipshultz. “For boys, dexrazoxane was not cardioprotective. We previously reported that young girls being treated with doxorubicin for childhood cancer at the same doses as boys were significantly more likely to have damaged hearts as long-term survivors, so the fact dexrazoxane can protect their hearts is very encouraging.”

“The findings of the present trial suggest that dexrazoxane use does not adversely affect oncological efficacy in children with high-risk ALL, and reduces cardiotoxicity in long-term survivors,” adds Lipshultz. “We encourage pediatric oncology protocols for children with high-risk ALL containing doxorubicin to use dexrazoxane before doxorubicin dosing, and to do so in the setting of clinical trials in which dexrazoxane’s use can be monitored.”

The implications in the years to come are enormous, considering the statistics. There are an estimated 300,000 survivors of childhood cancer currently alive in the United States. One study estimates that the childhood cancer survivors are eight times more likely to suffer cardiac death than the normal population, even 25 years after therapy, and this is significantly higher in childhood cancer survivors treated with doxorubicin. As such, having a new treatment that has been validated in a randomized clinical trial to protect the heart from the toxicity of doxorubicin chemotherapy may ultimately reduce the increased risk of heart failure and cardiac death these children face.

“This new paradigm for successfully treating childhood cancer is the balance over a child’s life of oncologic efficacy with toxicities and late effects, and how that balance is in terms of the patient’s quality of life over their lifetime,’‘ Lipshultz says. “Hopefully, use of dexrazoxane may improve this overall balance for girls and may lead to other ways to protect the hearts of boys.”

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Hyundai gift seeds Miller School cancer program

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Hyundai gift seeds Miller School cancer program

Attending the check presentation were, from left, Liz Davis; her daughter Kristen Davis, a cancer survivor; Steven E. Lipshultz; John Hyland, Hyundai regional sales manager; Daniel Armstrong; Daisy Healey, a cancer survivor; and her mother Julie Healey.

With grateful survivors of childhood cancer in the audience, officials of Hyundai Motor America presented the Department of Pediatrics with a check for $100,000 last week to establish a Children’s Cancer Survivorship Program to treat and minimize the late effects and complications from successful cancer therapies.

As Steven E. Lipshultz, chair of pediatrics, noted before accepting the grant, advances in treatment and research have dramatically improved survival rates from childhood cancer over the decades, but the improved therapies also have increased long-term complications, such as heart abnormalities and cognitive deficits, that require continued, multidisciplinary monitoring.

“Our feeling has always been that cancer is a lifelong diagnosis and it involves a lifetime commitment,” Lipshultz said at a recent ceremony. “There are so many issues that we’ve learned from this first generation of remarkable survivors.”

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Pediatric Grand Rounds: Children and Disasters

8:00 am

Annette M. La Greca, professor of psychology and pediatrics, will present “Children and Disasters” on Tuesday, September 14 from 8 to 9 a.m. in the Mailman Center for Child Development, eighth-floor auditorium. For more information, please contact Javier Salazar at 305-585-6042 or [email protected].

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Pediatric Grand Rounds: Viruses and Asthma: The Chicken or the Egg?

8:00 am

Tina V. Hartert, associate professor of medicine and director of the Center for Asthma and Environmental Health Research at Vanderbilt University School of Medicine, will present “Viruses and Asthma: The Chicken or the Egg?” on Tuesday, August 31 from 8 to 9 a.m. in the Mailman Center for Child Development, eighth-floor auditorium. For more information, please contact Javier Salazar at 305-585-6042 or [email protected].

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Debbie School gets dream playground

The Debbie School was a beehive of activity recently as 250-plus volunteers installed the kind of modern playground staff and students have been dreaming about for decades.

Here’s how to fulfill a dream: Take a mountain of mulch, a pile of sand, a few tons of concrete, 15 wheelbarrows, an army of volunteers, a blueprint designed by students, and a unique organization dedicated to bringing play to every child in America. Mix everything together on a sweltering July Saturday. Add some upbeat music, a lot of smiles and enough sweat to fill a pool, and voila!

Six hours later, you’ll have the kind of playground the staff, students, and alumni of the Department of PediatricsDebbie School, at the Mailman Center for Child Development, have been fantasizing about for decades.

“It’s awesome,” Angelica Stack, 10, a Debbie School grad and summer volunteer, said from the school’s rooftop patio as she watched some of the 250-plus volunteers wrangle a new, bright red triple slide into place. “The slides we had were babyish. One was all moldy.”

Now, thanks to KaBOOM!, a national non-profit born of tragedy, and its partnership with the John S. and James L. Knight Foundation, which sponsored the playground, nearly every student at the center, where young children with hearing impairments and other special needs attend classes with typically developing youngsters, will be able to play outside in the school’s tree-shaded courtyard.

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Pediatric Grand Rounds: Microarray-Based Comparative Genomic Hybridization for Neurodevelopmental Disorders

8:00 am

Yao-Shan Fan, research professor of pediatrics, will present “Microarray-Based Comparative Genomic Hybridization for Neurodevelopmental Disorders” on Tuesday, July 27 from 8 to 9 a.m. in the Mailman Center for Child Development, eighth-floor auditorium. For more information, please contact Javier Salazar at 305-585-6042 or [email protected].

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