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In the News: Inspirational Patients, Pioneering Fetal Surgery, Trauma Recovery, ADHD Overdiagnosed in ASD, AAP Annual Meeting
Although October is just ending, we’re already looking ahead to the New Year in this week’s installment of In the News. Read about two inspirational patients, a mother and daughter who fought neuroblastoma, who will join their physician Yael Mossé, MD, a Children’s Hospital of Philadelphia childhood cancer researcher, on a Rose Parade® float to celebrate their perseverance and dedication to research.
Other stories not to miss: CHOP fetal medicine experts report the first successful case of fetal surgery to treat a rare heart-threatening tumor. Researchers want to better understand how parents talk with their children after a traumatic injury. A study team raises questions about a screening tool to assess attention-deficit hyperactivity disorder (ADHD) in children with autism spectrum disorder (ASD). And CHOP experts share their insights at the annual meeting of the American Academy of Pediatrics (AAP).
Rose Parade® Float to Honor Family and Childhood Cancer Researcher
One of the magical moments of the 2017 Rose Parade®, a time-honored New Year’s celebration to be held Jan. 2 in Pasadena, Calif., will feature Yael Mossé, MD, a CHOP physician and childhood cancer researcher, who treated a daughter and mother who fought neuroblastoma together. The Gilger family of South Carolina will join Dr. Mossé on Northwestern Mutual’s parade float, highlighting the company’s commitment to fighting childhood cancer.
Seven-year-old Edie Gilger was diagnosed with an aggressive form of neuroblastoma at 6 months old. Neuroblastoma is a tumor of nerve tissue that most commonly begins in the abdomen in the adrenal gland. Researchers at CHOP have discovered many of the reasons why neuroblastomas occur, and in some cases, there appears to be an inherited genetic predisposition. Genetic testing confirmed that Edie’s mother, Emily, also had the same form of neuroblastoma.
A press release and video distributed by Northwestern Mutual this week shared the Gilgers’ incredible story and described how a breakthrough treatment for neuroblastoma discovered by Dr. Mossé saved their lives.
“Success for this family has been the power of the knowledge that we now have, being able to harness that for more effective treatment options,” Dr. Mossé said in the video. “That’s what Edie has done for us. She has brought the science to life. She’s validated it.”
Northwestern Mutual’s Childhood Cancer Program, with the help of its employees and financial representatives nationwide, has raised more than $12 million. The company continues to support Dr. Mossé’s research in partnership with Alex’s Lemonade Stand Foundation.
Read more about Dr. Mossé’s search for new therapies that target the gene mutations that drive high-risk forms of neuroblastoma.
In-utero Surgery for Heart-Threatening Tumor Successful for First Time
CHOP fetal medicine experts reported the first successful case of a pioneering fetal surgery to remove a life-threatening tumor from a baby’s heart. Intrapericardial teratoma is a rapidly growing tumor that arises in the sac surrounding the heart that can be detected during the fetal period. An ideal treatment is to perform surgery prenatally, before cardiac dysfunction occurs. The patient, who underwent the operation at 24 weeks of gestation while in his mother’s womb, is now a healthy 3-year-old.
“We have shown that we can accurately diagnose and provide a prognosis for this rare condition in utero, as well as perform fetal surgery,” said co-study leader Jack Rychik, MD, director of the Fetal Heart Program at CHOP, in a press release.
The research team, from CHOP and the Perelman School of Medicine at the University of Pennsylvania, reported a case series of eight fetuses with suspected intrapericardial teratoma seen at CHOP between 2009 and 2015. Two fetuses survived, both following fetal surgery. One underwent open fetal surgery at 24 weeks gestation, while the other underwent a successful EXIT (ex utero intrapartum treatment) procedure at 31 weeks gestation. Four fetuses with intrapericardial teratoma did not undergo fetal surgery, in some cases because referral was late and the fetuses were too sick to benefit from intervention. They did not survive. Two fetuses were found to have other types of heart-related tumors.
“Managing this type of teratoma presenting before birth is a challenge, but we show that survival with good outcome may be possible in some patients,” Dr. Rychik said.
Research Tool Aims to Help Prevent Pediatric Post-traumatic Stress
Researchers need to better understand how to help guide parents’ interaction with their children after a traumatic event to encourage their physical and mental recovery, according to a new Research in Action blog post.
“To date, much research has focused on what children and parents tell us, but we have yet to observe how parents support their children in the early aftermath of trauma,” wrote Meghan Marsac, PhD, a pediatric psychologist, behavioral researcher, and associate fellow of the Center for Injury Research and Prevention at CHOP. “To facilitate this learning, we created the Trauma Ambiguous Situations Tool (TAST) to observe how parents talk with their children about appraisals of situations and how to cope with challenges.”
Results from a pilot study of 25 child-parent pairs that completed the TAST within two weeks of an injury, while the child was in the hospital for treatment, appeared in the European Journal of Psycho-Traumatology. The findings suggest children and parents engaged well during the TAST and that parents influenced children’s reported appraisals and coping solutions.
“Future research with this tool may be able to help us better understand these early interactions after a traumatic event,” Dr. Marsac wrote. “We also hope to learn how to best partner with parents to support our pediatric patients.”
Read more about other research by Dr. Marsac and her colleagues who are championing trauma-informed care in pediatrics.
Measuring Tool May Overestimate ADHD in Children With Autism
A well-validated screening tool for ADHD, called the ADHD Rating Scale Fourth Edition (ADHD-RS-IV), may overestimate ADHD in children with ASD, according to a CHOP study team.
They suggest that the tool needs to be refined to better identify the correct disorder, and that clinicians should supplement the screening tool with careful clinical interviews. This is important for understanding what are the right services and treatments for a child, but sorting those out can be complicated because an estimated 30 percent or more of children with ASD also have ADHD.
Study co-author Thomas J. Power, PhD, director of CHOP's Center for Management of ADHD, developed ADHD-RS-IV in the 1990s (An updated version, the fifth edition, was published earlier this year, but not used in the current study). "I'm excited to be involved in this study, and in efforts to refine our screening tools," he said, "especially since few researchers have previously investigated using this scale in children with ASD. Our research raises questions not only about this rating tool, but all such measures that rely on parent and teacher ratings to assess ADHD in children with ASD."
The scale asks parents and teachers to provide numerical ratings in reply to 18 items about a child's behavior: nine items on inattention and nine on hyperactivity and impulsivity. The study team recommends modifying the rating scale to better minimize the influence of ASD on ratings of target ADHD behaviors. For example, some questions focus on how well a child stays on task with play activity. ADHD may cause a child to be easily distracted from an activity, but another child may instead stop playing because of ASD-related difficulties with social play.
The study, from researchers at CHOP, Penn, and Baylor University, appeared online in the Journal of Autism and Developmental Disorders.
CHOP Experts Participate in AAP Annual Meeting
More than 50 experts from CHOP addressed colleagues at this year’s AAP annual meeting on current issues in vaccine education, autism, sports injuries, sleep medicine, emergency medicine, otolaryngology, telemedicine, and neonatology, among others. Specific presentation highlights included:
- Kathy Abel, DNP, RN APNC, and John Chuo, MD: "Can Telemedicine Replace the First Post-op Visit for Knee Arthroscopy in Adolescents?"
- Susan E. Levy, MD, MPH: "Update on Screening from Diagnosis to Treatment: the Critical First Transitions (Autism)"
- Christina L. Master, MD, FAAP, CAQSM: "Vestibular and Visual Dysfunction as Predictors of Prolonged Recovery Following Pediatric Concussion"
- Jodi Mindell, PhD: "Helping Babies Sleep: Research and Evidence Behind Behavioral Interventions"
- Paul Offit, MD: "Talking to Parents: Strategies for Addressing Vaccine Hesitancy"
- Steven Sobol, MD: "Noisy Breathing: Benign or a Cause for Concern? (ENT)"
- Nicholas Tsarouhas, MD : "Course on Neonatal and Pediatric Critical Care Transport Medicine"
- Huayan Zhang, MD, PhD: "Caring for Infants with Severe Chronic Lung Disease — Five-year Experience of a Multidisciplinary Care Program"
In case you missed it earlier this week on Cornerstone, we sat down with Elaine H. Zackai, MD, director of clinical genetics at CHOP, who was recently selected as the first-ever recipient of the mentorship award from the American Society of Human Genetics. In our Q&A, get to know Dr. Zackai and her perspectives on working with trainees and helping families.
Last week’s In the News post featured two data-sharing initiatives that will help to propel the national Cancer Moonshot, a surprising finding by CHOP hemophilia researchers, and the exciting announcement that Douglas Wallace, PhD, founder and director of the Center for Mitochondrial and Epigenomic Medicine, received the 2017 Benjamin Franklin Medal in Life Science award given by the Franklin Institute.
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