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Archives for Pediatrics

Save the Date: ‘Care Across the Continuum’ BCI Conference

PITTSBURGH, Jan. 5, 2015 – The Brain Care Institute (BCI) of Children’s Hospital of Pittsburgh of UPMC will host a one-day conference on Aug. 1, 2015, in the Mary Jo Howard Dively Auditorium at the John G. Rangos Sr. Research Center at Children’s Hospital. Entitled “Care Across the Continuum,” the meeting is designed to provide local pediatricians and pediatric specialists with information regarding the multidisciplinary experts and services available to them via the BCI.

The topics will highlight the advantages of collaboration between the BCI and area pediatricians in the diagnosis and development of treatment plans for patients. The speakers are all BCI experts in their respective fields and include:

Please save the date, and check back soon for more information and registration details.

Radiation Dose Benchmarks During Cardiac Catheterization

Division of Pediatric Cardiology faculty member, Sara Trucco, MD, was part of a team that recently published results of a study that aimed to define age-stratified, procedure-specific benchmark radiation dose levels during interventional catheterization for congenital heart disease. The study found that radiation exposure was lowest in patent ductus arteriosus closure and highest in transcatheter pulmonary valve placement. The full article is available here.

Welcoming New Faculty to Children’s Hospital of Pittsburgh of UPMC

Children’s Hospital of Pittsburgh of UPMC is pleased to welcome three new faculty members.

Marcus Malek, MD, has joined the Division of Pediatric General and Thoracic Surgery as the director of pediatric surgical oncology. He has completed dual-fellowships in pediatric surgery (at Children’s Hospital of Pittsburgh) and pediatric surgical oncology at the Memorial Sloan-Kettering Cancer Center in New York City. Dr. Malek is one of the few dual-fellowship trained pediatric surgical oncologists in the Pennsylvania tri-state area.

Gary Mason, MD, has joined the division as a pediatric neuro-oncologist. He completed his fellowship in the Division of Neuro-Oncology at the Children’s National Medical Center in Washington DC. Dr. Mason will work closely with Ian Pollack, MD, in neurosurgery and will be leading several clinical trials in brain and CNS cancers.

Craig Byersdorfer, MD, PhD, is new to the Division of Pediatric Blood and Marrow Transplantation and Cellular Therapies. He completed his fellowship in pediatric hematology/oncology at the C.S. Mott Children’s Hospital in Ann Arbor, Michigan.

Leaders in Kidney Development Research

Pediatric Division of Nephrology faculty, Carlton Bates, MD, and Jacqueline Ho, MD, have recently been recognized for their work on kidney development research. One of the key missions of the Division of Pediatric Nephrology is to extend the knowledge of pathophysiology of renal disease through basic laboratory and clinical research.

Dr. Bates was elected to the American Society for Clinical Investigation; the medical honor society recognizes physicians who have accomplished meritorious original, creative, and independent investigations in the clinical or allied sciences of medicine. Dr. Bates studies genetic mouse models of kidney and lower urinary tract development. By suppressing or altering the activity of certain genes, he and his team are able to breed mice with structural kidney disease akin to what is seen in affected children, leading to new insights into the causes of congenital kidney and bladder diseases, which are leading causes of pediatric chronic kidney disease.

Dr. Ho was recently awarded a prestigious RO1 research grant by the NIH. She also has received numerous research awards, including the 2014 March of Dimes Basil O’Connor Starter Scholar Research Award and the 2013 International Pediatric Nephrology Association Renée Habib Young Investigator Award.

Intervention in School Health Centers is Effective in Counseling Teens About Abusive Relationships, Children’s Hospital of Pittsburgh of UPMC Study Finds

PITTSBURGH, Dec. 22, 2014 – A study by a Children’s Hospital of Pittsburgh of UPMC researcher provides the first evidence of the potential benefits of a brief, provider-delivered universal education and counseling intervention in school-based health centers to address and prevent the major public health problem of adolescent relationship abuse. The study appears online today in Pediatrics.

In collaboration with the California Adolescent Health Collaborative of the Public Health Institute, California School-Based Health Alliance and Futures Without Violence, the study was conducted during the 2012-2013 academic year at eight school-based health centers in California where students receive confidential clinical health services. Researchers surveyed 1,062 teens ages 14 to 19 for exposure to adolescent relationship abuse (including cyber dating abuse), sexual behavior, and care-seeking for sexual and reproductive health at their initial visit and again three months later.

Providers and staff in four school-based health centers received training on how to talk about healthy and unhealthy relationships; received palm-sized brochures about relationship abuse and available resources to hand out to patients; and learned how to refer youth to additional services and supports. No changes were implemented at the other four school-based health centers.

The researchers found students at the intervention sites were more likely than those at the other sites to recognize sexual coercion. Among students who reported relationship abuse at an initial visit  on a confidential survey, students at intervention schools were significantly less likely to report such abuse on the follow-up survey 3 months later.

“This study shows that a universal education and brief counseling approach in health care settings may be a useful way to address relationship abuse among adolescents,” said lead investigator Elizabeth Miller, M.D., Ph.D., chief, Division of Adolescent and Young Adult Medicine. “Clinicians talking about healthy and unhealthy relationships with all of their patients can make a difference.”

Among almost 400 youth who reported experiencing relationship abuse at an initial visit, 65 percent of students in intervention schools reported still experiencing such abuse about three months later, compared to 80 percent of students in the other schools. In addition, youth in the intervention clinics were much more likely to discuss being in an unhealthy relationship with their health care provider. “Embedding prevention messages and information about relevant resources within clinical settings for adolescents may be an effective way to reduce relationship abuse,” said Lisa James, the director of Health at Futures Without Violence and a co-investigator on the study.

“Youth seeking care in adolescent health settings appear to have more exposure to relationship abuse and associated poor health outcomes,” said Dr. Miller, also an associate professor of pediatrics, University of Pittsburgh School of Medicine. “Finding an intervention that may make a difference for these youth who are at higher risk for relationship abuse is encouraging.”

“This study effectively combined school-based clinical interventions with youth-led promotion of healthy adolescent relationships,” said co-investigator Samantha Blackburn, formerly with the California School-Based Health Alliance, and now an assistant professor of nursing at California State University Sacramento. “Not only did students receive needed services, they were also empowered to help their peers be healthy and safe.”

“Prevention of relationship abuse among adolescents requires a range of strategies from educating youth and adults about the extent of the problem; connecting youth to relevant supports and services; and engaging schools, parents, and other influential adults to talk about healthy relationships,” said co-investigator Alison Chopel from the Public Health Institute’s California Adolescent Health Collaborative.  “This intervention is a part of the prevention solution.”

Dr. Miller and her collaborators are hopeful that these findings will encourage schools and adolescent health care providers to implement this program.  For parents and educators, she adds, “This study suggests that creating spaces for young people to learn about healthy and unhealthy relationships and how to help their friends can really help to reduce adolescent relationship abuse.”

Collaborators with Dr. Miller on the study were: Heather L. McCauley, Sc.D., Kelley Jones, MPH, Rebecca Dick, MS, all with Children’s Hospital of Pittsburgh of UPMC; Sandi Goldstein, MPH, Johanna Jetton, California Adolescent Health Collaborative, Public Health Institute; Jay G. Silverman, Ph.D., Division of Global Public Health, University of California, San Diego School of Medicine; Samantha Blackburn, RN, MSN, PNP, California School-Based Health Alliance and California State University Sacramento School of Nursing; Erica Monasterio, RN, MN, FNP-BC, University of California San Francisco School of Nursing; Lisa James, Futures Without Violence; and Daniel J. Tancredi, Ph.D., University of California Davis School of Medicine.

The study was supported by Award No. 2011-MU-MU-0023 of the National Institute of Justice, Office of Justice Programs, U.S. Department of Justice.

For more information on Dr. Miller and the Division of Adolescent and Young Adult Medicine, visit www.chp.edu/CHP/am. To learn more about how to implement this intervention, visit http://www.futureswithoutviolence.org/hanging-out-or-hooking-up-clinical-guidelines-on-responding-to-adolescent-relationship-abuse-an-integrated-approach-to-prevention-and-intervention/.

Program Improves Diabetes Control in World’s Poorest Children, Pitt Public Health Finds

PITTSBURGH, Nov. 25 2014 – A nonprofit program that brings diabetes care and education to some of the world’s poorest children has successfully improved control of the disease, according to a University of Pittsburgh Graduate School of Public Health analysis published in the journal Diabetes Research and Clinical Practice. It is the first scientific evidence to show that improvement in long-term blood sugar control in type 1 diabetes is possible in sub-Saharan African youth.

The International Diabetes Federation Life for a Child (LFAC) program’s approach to providing care to children with type 1 diabetes in Rwanda led to major reductions in HbA1c, a long-term measure of blood sugar. The proportion of children who had an HbA1c level of more than 14 percent, a potentially lethal level, fell from 31 percent to 9 percent. The improvement was greatest in the children who had access to blood sugar testing supplies and regularly monitored their blood glucose levels.

“Type 1 diabetes can be a very difficult disease to manage, and, if not properly controlled, it is deadly,” said senior author Trevor Orchard, M.D., professor of epidemiology at Pitt Public Health. “When coupled with poverty, food insecurity and severely limited health care provision that many of the children in sub-Saharan Africa face, the need for proven programs to help these children control their diabetes becomes vital.”

Type 1 diabetes, usually diagnosed in children and young adults, happens when the body does not produce insulin, a hormone that is needed to convert sugar into energy.

LFAC supports the provision of insulin, glucose monitoring supplies, diabetes education, advice and training to children and youth with diabetes in developing countries. In Rwanda, the program provides assistance through the Association Rwandaise des Diabetiques (ARD) in the city of Kigali.

Dr. Orchard and his team followed and regularly measured the HbA1c levels in 214 people under age 25 who enrolled in the program between June 2009 and November 2010.

HbA1c develops when sugar binds to hemoglobin, a protein within red blood cells, in the blood. The higher the HbA1c, the greater the risk of developing diabetes-related complications, like heart disease, blindness and nerve damage. Those without diabetes typically have an HbA1c between 4 and 5.7 percent. For people with diabetes, an HbA1c level of less than 7 percent is considered good control.

In the Rwandan children that Dr. Orchard’s team followed, the average HbA1c initially was 11.2 percent. After two years in the program, the average fell to 9.8 percent.

Dr. Orchard became involved in the program in Rwanda in 2007 when it had only 25 children. LFAC has now enrolled more than 1,000 children and youth. Pitt Public Health sends at least one graduate student every year to assist with the program and the required annual assessments of the children enrolled. Dr. Orchard and a colleague from Northwestern University in Chicago, Deborah Edidin M.D., also visit Rwanda regularly to help develop and provide care and education for the children.

“It is very encouraging to see the Rwandan children get better control of their blood sugar levels thanks to this unique collaborative effort between the LFAC, ARD and Pitt,” said Dr. Orchard. “However, more work is needed. Only about 12 percent of the Rwandan children met American Diabetes Association glucose control goals, compared with 32 percent of U.S. children.”

A key part of controlling type 1 diabetes is regular blood sugar monitoring, which allows patients to adjust their insulin levels based on test results. Children who were better able to monitor their blood sugar had better HbA1c levels. In some cases, a lack of testing supplies prohibited children from testing their blood sugar as frequently as recommended.

Disturbingly, Dr. Orchard’s team also found that, as HbA1c levels improved, high blood pressure emerged as a major problem such that over 40 percent of the youth were hypertensive.

“This is troubling,” said Dr. Orchard. “Many of our participants were very young children during the Rwandan genocide of 1994 and grew up malnourished and underweight. It is possible to attribute some of the increasing blood pressure to weight gain and rehydration after enrollment in the program. Salt also often is used in food preparation and preservation in sub-Saharan Africa, so this may be a factor as well. Unfortunately, very few participants are able to take blood pressure medication due to limited supplies and prohibitive prices.”

Dr. Orchard noted that continued study is needed to better understand the causes of the high blood pressure among the Rwandan children receiving care for type 1 diabetes, as well as to determine how best to develop a sustainable program to maintain this improved care.

Additional researchers on this project are Sara L. Marshall, Ph.D., Vincent C. Arena, Ph.D., Dorothy J. Becker, M.B.B.Ch., Clareann H. Bunker, Ph.D., Ronald E. LaPorte, Ph.D., and Laurien Sibomana, M.S., all of Pitt; Graham Ogle, M.B.B.S., of the International Diabetes Federation Life for a Child Program and Australian Diabetes Council; and Crispin Gishoma, Francois Gishoma, and Veaste Kaberuka, all of the Association Rwandaise des Diabetiques.

This work was largely funded by donations to the Life for a Child –Pitt Initiative. Directions for donating to the initiative can be found at: http://www.upmc.com/lifeforachildprogram

Childhood Cancer Focus of St. Baldrick’s Foundation Research Grant to Children’s Hospital of Pittsburgh of UPMC

PITTSBURGH, Nov. 21, 2014 – To improve information flow about childhood cancers, Jean M. Tersak, M.D., an oncologist in the Division of Pediatric Hematology/Oncology at Children’s Hospital of Pittsburgh of UPMC, has been awarded a one-year, $50,000 grant from the St. Baldrick’s Foundation, a volunteer-driven charity dedicated to raising money for childhood cancer research.

Dr. Tersak’s is one of 40 infrastructure grants totaling more than $2.5 million awarded by St. Baldrick’s to support pediatric oncology research. These grants provide resources to institutions to conduct more research and enroll more children in ongoing clinical trials.

The funding will help to support efforts to enhance website communication of research activities to make information more easily accessible to potential patients, families, and referring physicians. Additionally, funding will support the transition to a new database to increase efficiency and refine ability to query the database as a more effective tool to conduct institutional research.

“This generous grant from St. Baldrick’s will help us to be sure that our medical treatments are more widely known and available to anyone who may need them to battle a diagnosis of cancer as a child or young adult,” said Dr. Tersak, who also directs the Survivorship Program at Children’s Hospital. “The grant will increase the efficiency of our program and increase awareness of novel treatments available to patients form our region and beyond.”

“These grants are critically important to saving children’s lives, and would not be possible without our dedicated volunteers and generous donors who believe kids deserve better than medicine is currently able to provide,” said Kathleen Ruddy, chief executive officer for the St. Baldrick’s Foundation.

“Each day in my work, I encounter patients who have been helped along the way through the tremendous efforts of St. Baldrick’s,” added Dr. Tersak, also principal investigator, Children’s Oncology Group.

Dr. Tersak has a special interest in caring for survivors of childhood cancer, including evaluation for medical late effects of treatment and the quality of life in these survivors. She is involved in national research to better understand late effects, ways to prevent them, and early intervention when they do occur.

The Division of Pediatric Hematology/Oncology at Children’s provides diagnosis, treatment and complete follow-up for children, adolescents, and young adults with cancer and blood disorders. The division is the largest, most comprehensive pediatric cancer and blood disease center in western Pennsylvania, eastern Ohio, and northern West Virginia and has been a member of the Children’s Oncology Group, a multi-institutional pediatric cancer research organization sponsored by the National Cancer Institute, since 1961. It’s one of the top-rated pediatric cancer programs in the United States as ranked by U.S. News & World Report.

For more information about Dr. Tersak, please visit www.chp.edu.

Early Diagnosis and Cord Blood Transplantation Prevents Permanent Brain Damage From Rare Inherited Disease, Children’s Hospital of Pittsburgh of UPMC Study Finds

PITTSBURGH, Nov. 19, 2014 – Umbilical cord blood from unrelated donors can halt the progression of the neurodegenerative disease Hurler syndrome if performed before the affected child is less than 9 months old, according to a Children’s Hospital of Pittsburgh of UPMC study that appears online in Annals of Neurology. The findings emphasize the need for early diagnosis of the condition, preferably through newborn screening programs.

Hurler syndrome is the most clinically severe form of an inherited disorder in which the patient lacks a key enzyme needed to break down complex sugars called glycosaminoglycans. The sugar buildup results in progressive organ deterioration and death in childhood. Affected children may not have symptoms until age 3, but the brain undergoes damage before symptoms present.

Umbilical cord blood transplantation from unrelated donors has previously been shown to improve neurological outcomes of children over 2 years of age and prolong life. Hematopoietic stem cells from the cord blood transplants provide a source for the normal enzyme that is donated to the deficient cells decreasing the accumulation of gycosaminoglycans.

Key findings of this study show that treatment of Hurler syndrome with umbilical cord blood transplantation before 9 months of age leads to normal cognitive development. The researchers found children transplanted at 12 and 25 months of age functioned cognitively at a level 2 to 5.3 years below that of those transplanted at 4 months. Early transplantation also predicted better outcomes for language skills and adaptive behaviors.

“The purpose of the study was to determine whether age at transplantation can predict cognitive outcomes,” said Maria Luisa Escolar, MD, MS, director, Program for the Study of Neurodevelopment in Rare Disorders, Children’s Hospital, and associate professor of pediatrics, University of Pittsburgh School of Medicine. “This study highlights the importance of early detection of brain diseases in babies and infants when brain growth is the most accelerated in life, placing them at increased vulnerability for permanent damage.”

Between June 1997 and February 2013, 31 children with Hurler syndrome underwent umbilical cord blood transplantation and were evaluated every six to 12 months thereafter for an average of seven years. Median age at transplantation was 13.8 months. The youngest babies in the study were diagnosed due to family history of the condition.

“Identification of asymptomatic children through statewide newborn screening programs is the only way to diagnose early and prevent brain damage to babies with no family history of Hurler syndrome,” Dr. Escolar said.

“Unfortunately, early diagnosis is often difficult as their initial symptoms may be common in the general population,” added Dr. Escolar. “Therefore, there is a need for newborn screening for Hurler syndrome and similar neurodegenerative diseases that can identify children before symptoms appear, giving the best opportunity for prompt intervention and optimal outcomes.”

Collaborators with Dr. Escolar on the study were Michele Poe, PhD, and Sarah Chagnon, MD, both with Children’s Hospital of Pittsburgh of UPMC.

Financial support for this work was received from the Caterina Marcus Foundation.

For more information on Dr. Escolar and the Program for the Study of Neurodevelopment in Rare Disorders, visit www.chp.edu/ndrd.

Cyber Dating Abuse Common Among Teens, Children’s Hospital of Pittsburgh of UPMC Study Finds

PITTSBURGH, Nov. 17, 2014 – Two in five teens surveyed experienced cyber dating abuse, which involves the use of technology to control, harass, threaten or stalk another person in the context of a dating relationship, in the previous three months, according to a Children’s Hospital of Pittsburgh of UPMC study that appears online today in Pediatrics.

The study is the first about cyber dating abuse among a group of young people who are using school-based health services, said senior investigator Elizabeth Miller, M.D., Ph.D., chief, Division of Adolescent and Young Adult Medicine at Children’s Hospital.

Done in collaboration with the California Adolescent Health Collaborative of the Public Health Institute, California School-Based Health Alliance and Futures Without Violence, the study was conducted at eight school-based health centers in California where students receive confidential clinical health services, including annual check-ups, sports physicals and birth control. The study, conducted during the 2012-2013 school year, assessed teens ages 14 to 19, for exposure to cyber dating abuse, adolescent relationship abuse, sexual behavior, and care-seeking for sexual and reproductive health.

Key findings showed 41 percent of teens reported experiencing this form of abuse within the last three months, with more females than males reporting such victimization. Most commonly, their partners used technology including mobile apps, social networks, texts, or other digital communication to repeatedly contact them to see where they were and who they were with.

“These findings underscore that cyber dating abuse is an emerging concern,” said Dr. Miller, also an associate professor of pediatrics, University of Pittsburgh School of Medicine. “We need to support prevention efforts that increase education about the many different forms of abuse in adolescent relationships, and to encourage parents, teachers, coaches and others to talk to young people about what healthy relationships look like.”

Like previous research examining this form of abuse, the researchers found that teens exposed to cyber dating abuse were more likely to also experience other forms of physical and sexual dating abuse, such as being slapped, choked or made to have sex by a dating partner, and also non-partner sexual assault. Additionally, greater exposure to cyber dating abuse was associated with less contraceptive use among adolescent girls.

“It is concerning to see such a large number of young people reporting these cyber dating abuse experiences, and to learn that experiencing this form of abuse is associated with other unhealthy behaviors such as not using any method of contraception for birth control,” said Rebecca Dick, MS, clinical research coordinator, Division of Adolescent and Young Adult Medicine at Children’s, and the first author of the study.

“Professionals should take cyber dating abuse seriously and actively ask teens if they are being monitored, threatened or sexually coerced by their partner using technology-based communication,” added Dr. Miller. “Given the prevalence of cyber dating abuse in this sample of adolescents, we recommend that relationship abuse prevention education include cyber dating abuse and that such education and counseling be integrated into health assessments in clinical settings.”

Collaborators with Dr. Miller on the study were: Rebecca Dick, MS, Heather L. McCauley, Sc.D., Kelley Jones, MPH, all with Children’s Hospital of Pittsburgh of UPMC; Daniel J. Tancredi, Ph.D., University of California Davis School of Medicine; Sandi Goldstein, MPH, Alison Chopel, DrPH, California Adolescent Health Collaborative, Public Health Institute; Samantha Blackburn, RN, MSN, PNP, California School-Based Health Alliance and California State University Sacramento School of Nursing; Erica Monasterio, RN, MN, FNP-BC, University of California San Francisco School of Nursing; Lisa James, MS, Futures Without Violence; and Jay G. Silverman, Ph.D., University of California, San Diego School of Medicine.

The study was supported by Award No. 2011-MU-MU-0023, awarded by the National Institute of Justice, Office of Justice Programs, U.S. Department of Justice.

For more information on Dr. Miller and the Division of Adolescent and Young Adult Medicine, visit www.chp.edu/CHP/am.

FDA Approves Blood Test Developed by UPMC Researchers to Predict Rejection in Organ Transplant Recipients

PITTSBURGH, Nov. 5, 2014 – A first-of-its-kind, personalized blood test to predict the likelihood of organ rejection in children with liver or intestine transplants has received U.S. Food and Drug Administration (FDA) approval. The test was developed by researchers at Children’s Hospital of Pittsburgh of UPMC to determine a personalized rejection-risk index with cell-based technology.

Cellular rejection affects half of all transplant recipients in their lifetime. If unchecked, rejection can lead to progressive loss of function of the transplanted organ. Therefore, predicting whether rejection will occur is an essential part of the recipient’s care, and has been an unmet need until recently. A biopsy is used to detect ongoing rejection, but this surgical procedure cannot predict rejection.

The research team led by Rakesh Sindhi, M.D., F.A.C.S., co-director, pediatric transplantation, at the Hillman Center for Pediatric Transplantation at Children’s, began work on this test system in late 2006. The technology was licensed by the University of Pittsburgh to Plexision, a Pittsburgh-based biotech company, for development.

Pleximmune, as the test is named, predicts acute cellular rejection with an accuracy approaching or exceeding 80 percent under a variety of conditions. This performance has been established in a study involving more than 200 children who received liver or intestine transplants at Children’s Hospital.

“A common theme in every encounter with our patients is an assessment of whether that child is at risk for rejection and whether this risk will be affected by the planned treatment,” said Dr. Sindhi, who is also director of pediatric transplant research. “It is hoped that the information from such personalized blood tests will improve clinical decision-making and benefit patients in the long run.”

“Our whole philosophy here at Children’s Hospital centers on improving outcomes after transplantation and achieving the goal of a normal life for all the children we transplant,” said George Mazariegos, M.D., F.A.C.S., chief, pediatric transplantation. “This test is a step toward achieving that goal. And it is doubly exciting that the research in Dr. Sindhi’s lab is affecting children at the bedside so directly.”

The Hillman Center for Pediatric Transplantation at Children’s Hospital maintains one of the busiest and most experienced pediatric transplant centers in the United States. The center was established in 1981 under renowned transplant pioneer Thomas E. Starzl, M.D., Ph.D., as the nation’s first pediatric transplant program. Children’s is one of the most active transplant centers for infants, children and adolescents with referrals from all over the world. The team specializes in heart, lung, liver, intestine, multivisceral, kidney, and blood and marrow transplants, and achieves patient outcomes that rank among the world’s best.

Dr. Sindhi is the inventor of technology licensed to Plexision, on which this test system is based. The University of Pittsburgh holds equity in the company; Dr. Sindhi has an unpaid consulting relationship with the company.

For more information on Dr. Sindhi and the Hillman Center for Pediatric Transplantation, visit www.chp.edu/transplant.

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