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Driving and Autism, Epilepsy Learning System, Youth-onset Diabetes, Mitochondrial Research Award, MicroRNA and Obesity, Well-being Measures

Published on June 28, 2019 in Cornerstone Blog · Last updated 2 weeks 6 days ago
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Even as the lazy days of summer approach, our researchers are busy making news with innovations in their respective fields. In this edition of In The News, a multidisciplinary study lauds parental support as key for autistic adolescents who want to drive, Children’s Hospital of Philadelphia announces a pioneering partnership in the Epilepsy Learning Healthcare System, and the TODAY study shows youth-onset type 2 diabetes leads to later complications. 

For more summer reading, Dr. Douglas Wallace is honored for mitochondrial research; scientists find a correlation among gut microbiota, microRNA 181 (miR-181), and obesity; and a validated pediatric tool for self-reported outcomes becomes available for clinical use.

Parental Support Crucial for Autistic Adolescents to Drive 

A study of specialized driving instructors showed autistic adolescents need the support of their parents or guardians to prioritize independence so they are prepared for learning to drive. The findings, published in Autism in Adulthood, also highlighted the need to define best practices to guide assessment and delivery of highly individualized instruction for autistic adolescents.

Researchers from Children’s Hospital of Philadelphia’s Center for Injury Research and Prevention (CIRP), Center for Autism Research (CAR), and Division of Emergency Medicine, as well as the University of Pennsylvania School of Nursing and the Virginia Tech Transportation Institute, conducted the study as part of a five-year project aimed at understanding mobility issues for autistic adolescents.

The study revealed common themes underscoring the importance of parents of autistic adolescents in helping their children to prepare for the learning-to-drive process. Specialized driving instructors who participated in study interviews said parents can support and prioritize independence by encouraging their autistic adolescents to develop other life skills before learning to drive, such as mowing the lawn, cooking, and taking public transportation.

“Some parents may not let their autistic adolescents use a stovetop oven, but are asking if their teens are ready to drive,” said Benjamin E. Yerys, PhD, study author and psychologist at CAR. “Whether or not their children decide to drive, parents should encourage greater independence by encouraging them to get around on their own. Traveling independently by driving or other modes of transportation is key to continuing their education, working, and staying connected with friends and family.”

Previous CHOP research led by Allison Curry, PhD, MPH, senior scientist and director of Epidemiology and Biostatistics at CIRP, reported nearly one-third of autistic adolescents obtain a driver’s license by the time they are 21-years-old. 

Epilepsy Foundation Announces Epilepsy Learning Healthcare System 

The Epilepsy Foundation will launch the Epilepsy Learning Healthcare System (ELHS), a multi-stakeholder collaboration to improve outcomes by implementing best practices and continual learning. CHOP is among six pioneer hospitals accredited by the National Association of Epilepsy Centers (NAEC) that have committed to the ELHS.

A learning health system is structured for patients, families, community service providers, clinicians, researchers, and health system leaders to design, implement, and disseminate collaborative research and quality improvement. The ELHS will gather data about people with epilepsy from U.S. clinics and analyze it to identify best practices. Data collected include diagnosis, evaluation, and care from healthcare providers, as well as information about seizure frequency, treatment side effects, comorbidities, self-management, and quality of life.

In addition to CHOP, Children's Hospital of Atlanta, Partners Healthcare (including the Massachusetts General Hospital and Brigham and Women's Hospital), Beth Israel Deaconess Medical Center, and Barrow Neurological Institute, have committed to the initiative. 

The ELHS grew out of the Rare Epilepsy Network, a research collaboration of more than 30 rare epilepsy organizations that is hosted by the Epilepsy Foundation and was partially funded through the Patient-Centered Outcomes Research Institute (PCORI). The ELHS is made possible by a Learning Health System Network Pilot Collaborative grant awarded earlier this year by PCORI and the James M. Anderson Center for Health Systems Excellence, with additional contributions from the NAEC, participating ELHS centers, industry sponsors, and the Epilepsy Foundation. 

TODAY Study Links Youth-onset Type 2 Diabetes and Later Complications

Pediatric endocrinologist Lorraine Levitt Katz, MD, and other leaders of the TODAY (Treatment Options for Type 2 Diabetes in Adolescents and Youth) study of youth-onset type 2 diabetes presented longitudinal outcomes of the study at the American Diabetes Association 79th Scientific Sessions.

TODAY2 found that young people diagnosed with type 2 diabetes (T2D) in their early teens had a high rate of diabetes-associated complications affecting the heart, kidneys, eyes, nerves, and pregnancy by the time they were in their mid-20s. Dr. Katz, the CHOP principal investigator in the multicenter national study, reported on the high rates of hypertension, hyperlipidemia, and echocardiographic abnormalities in this cohort. 

“These outcomes of the longest running study on youth with T2D indicate that youth-onset type 2 diabetes is more aggressive than adult-onset T2D,” Dr. Katz said. “We now know the importance of underscoring the fact that younger patients require even more intensive management than many adults with type 2 diabetes. These implications have the potential to change the course of treatment and improve care overall.”

Screening Tools Determine Pediatric Patients’ Well-being 

Clinicians and researchers have a new way to assess psychological well-being in children and adolescents. A team led by Christopher Forrest, MD, PhD, tested several tools for collecting children’s self-reported outcomes previously developed under the pediatric Patient-Reported Outcome Measurement Information System (PROMIS) initiative. In their article published in the Journal of Pediatric Psychology, they demonstrate that the PROMIS Pediatric Meaning and Purpose item banks and their short forms are ready for use in clinical research and practice to monitor patients’ care. These measures of children's eudaimonic well-being are indicative of children's hopefulness, optimism, goal-directedness, and feelings that life is worth living.

Under PROMIS, Dr. Forrest led the development of survey tools for collecting and scoring a variety of patient-reported outcome measures in pediatric populations. Data collected from two samples – one comprising 1,895 children 8 to 17-years-old and 927 parents of children 5 to 17-years-old recruited from an Internet panel, medical clinics, and schools, and the second comprising a nationally representative sample of 990 children 8 to 17-years-old and 1,292 parents of children 5 to 17-years-old recruited from a different Internet panel – were combined for item response theory calibration of the item bank, and both samples were used in validation studies.

The final versions of the scales showed reliability of  > 0.90, and short form scales (i.e., 4 or 8 items) had a high degree of precision across over 4 standard deviation units of the latent variable. The item bank positively correlated with extant measures of positive psychological functioning, and negatively correlated with measures of emotional distress, pessimism, and pain. Adolescence and presence of a special healthcare need corresponded to lower meaning and purpose scores.

Douglas Wallace Honored for Outstanding Contributions in Mitochondrial Research

Douglas Wallace, PhD, director of the Center for Mitochondrial and Epigenomic Medicine at CHOP, is the 2019 recipient of the Charles L. Hoppel Prize for Outstanding Contributions in Mitochondrial Research.

Dr. Wallace, who also holds the Michael and Charles Barnett Endowed Chair in Pediatric Mitochondrial Medicine and Metabolic Diseases, was selected for the international award based on his extraordinary career achievement in mitochondrial biology. The award announcement highlighted “his commitment to students and colleagues, his ability to advance discoveries from laboratories to patients, and his continued service to the broader community reflect the values that Charles Hoppel has displayed over his 50-year career as an academic scientist.”

Dr. Wallace will be honored when he presents the third annual Charles L. Hoppel Lecture this fall at Case Western Reserve University School of Medicine in Cleveland.

Gut Microbiota Regulate Obesity Through MicroRNA 181

Results from a new study published in Science Translational Medicine  indicate that host metabolism is tuned in response to dietary and environmental changes via a central mechanism – the regulation of miR-181 in white adipose tissue (WAT) by gut microbiota-derived metabolites.

CHOP researcher Sarah Henrickson, MD, PhD, in the Division of Allergy and Immunology, and Jorge Henao-Mejia, MD, PhD, a scientist in the Division of Transplant Immunology at CHOP, and colleagues from the Perelman School of Medicine at the University of Pennsylvania linked the increased expression of this microRNA to a reduction in circulating microbiota-derived metabolites produced by tryptophan metabolism in the gut. A high-fat diet led to the activation of miR-181 in WAT and subsequent obesity, insulin resistance, and WAT inflammation.

Viewed in conjunction with their finding that miR-181 expression in WAT and the plasma abundance of tryptophan-derived metabolites were dysregulated in a cohort of obese children, the miR-181 family may represent a potential therapeutic target to modulate WAT function in the complex, prevalent disease of obesity.

ICYMI

Did you hear the great news? Children’s Hospital of Philadelphia once again ranked as one of the nation’s Best Children’s Hospitals by U.S.News & World Report, marking the 15th consecutive year that our divisions have received top-notch distinctions. CHOP secured second place in the 2019-2020 Honor Roll of Best Children’s Hospitals, a list that seeks to provide an answer to the question that crosses almost every family’s mind: “Which hospitals are best prepared to care for the sickest kids?”

Seven of our programs at CHOP ranked first, second, or third in an evaluation of 10 specialty areas. Read all the details about our accomplishment in our recent Cornerstone post.

Thank you and congratulations to all of our faculty and staff for the work they do to help patients today as well as the groundbreaking research they conduct to change outcomes for the children of tomorrow.

Catch up on our headlines from our last edition of In the News:

  • Eagles Autism Challenge Raises More Than $3.5 Million
  • Dr. Allison Barz Leahy Receives ASCO Young Investigator Award
  • Dr. Sarah Tasian Awarded SU2C Innovation in Collaboration Grant
  • LiBi Study Examines Trauma and Development
  • Study Assesses Depression and PTSD Following Injury
  • Diane Spatz, PhD, Honored for Nursing Research Excellence

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