Aug 27 2015

Research, Clinical Care Combine to Improve Childhood Arthritis

When children with multiple swollen joints come to the Center for Childhood Arthritis and Rheumatic Disease for treatment, and then a few weeks later they are back on the field playing sports, their energy is contagious for pediatric rheumatologist Pamela Weiss, MD MSCE.

A clinician, researcher, and mentor at The Children’s Hospital of Philadelphia, Dr. Weiss received the Spondylitis Association of America Bruckel Young Investigator Award in honor of her contributions to the understanding of juvenile spondyloarthritis (JSpA).

Children with JSpA experience inflammation and stiffness of joints that can result in growth disturbances and loss of range of motion if not controlled properly. They tend to develop arthritis of lower extremities and are as at risk of developing arthritis of the lower back or spine. JSpA is associated with more frequent and higher intensity pain and poorer quality of life than other categories of juvenile arthritis.

“One of the questions we always get is, ‘Do a lot of kids actually have arthritis?’” said Dr. Weiss, clinical research director for CHOP’s division of rheumatology. “Actually, our clinic is full of these children. Our treatments are so effective that unless our patients chose to share that they have a chronic condition, most of the time no one knows there’s anything wrong with them.”

Juvenile arthritis is the most common rheumatologic disease among children, ranging from one to four cases per 1,000 children, which is about the same prevalence as Type 1 diabetes mellitus and cystic fibrosis in children. JSpA accounts for about 20 percent of childhood arthritis.

Dr. Weiss’ research concentrates on characterizing the disease features of children with JSpA, with a focus on enthesitis (inflammation of the tendon insertions) and arthritis of the lower back. She has an ongoing project evaluating the uses and effects of drugs for this condition. Recently she led an international effort to develop and validate a disease activity score for children with JSpA based on input from more than 100 physicians.

“This disease activity score is a really important metric because we need to gauge whether our patients are getting better on treatment or not,” Dr. Weiss said. “Prior to the development of this score, no metrics existed for these kids.”

Dr. Weiss also conducted a study that determined using ultrasound is more reliable than physical exams for accurate diagnosis of enthesitis. This is crucial because detection of enthesitis influences disease classification and subsequent treatment decisions.

In July, Dr. Weiss published results from another study that focused on determining how many children with newly diagnosed JSpA develop early lower back arthritis. Her team found that 20 percent of children had imaging evidence of lower back arthritis within six months of diagnosis, and that the majority of these children did not have the traditional symptoms used to screen for this condition, including back pain.

“Looking at kids at diagnosis to see if they have early arthritis of the lower back could impact the rapidity with which we start medications and could potentially alter their entire disease course,” Dr. Weiss said.

Currently, Dr. Weiss is analyzing data from a large database she created that includes children with enthesitis-related arthritis from five pediatric rheumatology practices, including one in Italy. She is exploring the effectiveness of treatment strategies for newly diagnosed patients.

When she is not improving children’s outcomes through research, Dr. Weiss has leadership roles within several national organizations dedicated to finding treatments and improving care for pediatric rheumatic diseases. One of her major projects was leading an update of the American College of Rheumatology’s juvenile arthritis treatment guidelines published in 2013. She is a member of the literature review team for the American College of Rheumatology’s spondyloarthritis treatment guidelines that will be published later in 2015.

Dr. Weiss also is the principal investigator at CHOP for a new national registry launching this summer by the Childhood Arthritis & Rheumatology Research Alliance (CARRA). About 60 sites are participating, and the data collection will be geared toward monitoring the long-term safety of medications prescribed for pediatric patients.

“I want to make kids better, but with the least amount of side effects,” said Dr. Weiss, who is co-chair of the Juvenile Idiopathic Arthritis Research Committee for CARRA. “In rheumatology, there are so many new drugs coming on the market, yet pediatrics is always behind adult medicine in terms of approval for the drugs. So there is a lot of work to be done to make sure the new medications are both effective and safe for our kids.”

Dr. Weiss shares her passion for rheumatology by acting as a mentor for fellows who are interested in pursuing research in this area, and she presents lectures to CHOP residents on pediatric rheumatology topics.

“By doing a combination of research and clinical care, I feel like I’m doing something different and meaningful every single day, and I love all aspects of it,” Dr. Weiss said.

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Aug 25 2015

Researchers Using EEG Find Seizures in Newborns After Cardiac Surgery

seizureIt is imperative for clinicians to detect and treat seizures that may occur in newborns with congenital heart disease (CHD) who have cardiac surgery. Seizures are associated with underlying brain injury, greater illness severity, and worse neurodevelopmental outcomes. The problem is that seizures can be difficult to identify during a routine bedside clinical assessment.

Continuous postoperative encephalographic (EEG) monitoring can show physicians when these medically fragile patients are experiencing a seizure by measuring the electrical activity in their brains. Yet, many centers do not follow professional guidelines issued in 2011 by The American Clinical Neurophysiology Society (ACNS) that call for continuous EEG monitoring of neonates undergoing surgery for CHD.

Experts at The Children’s Hospital of Philadelphia acted on the ACNS recommendations and began routine postoperative EEG monitoring for these patients in 2012. In a study published in the July issue of The Journal of Thoracic and Cardiovascular Surgery, CHOP cardiac critical care physicians and colleagues at the Perelman School of Medicine at the University of Pennsylvania reported results from implementation of the EEG monitoring clinical pathway.

“We found that continuous EEG monitoring identified newborns who had seizures, which indicated underlying brain injury, and are a risk factor for worse neurodevelopmental outcomes and mortality,” said study leader Maryam Y. Naim, MD, an attending physician in CHOP’s Pediatric Cardiac Intensive Care Unit. The majority of the seizures found using EEG would not have been visible to clinical caregivers, she added.

Over an 18-month period, the researchers studied 161 newborns with CHD who underwent open heart surgery at CHOP and received continuous EEG monitoring as part of routine postoperative monitoring. The study team found seizures in 13 of the infants (8 percent). Of those 13 infants, 11 (85 percent) had subclinical seizures, detectable only by EEG. Eight of the 13 infants had status epilepticus seizures, which are particularly dangerous. Five of the 13 infants with seizures died, which is a mortality rate of 38 percent, compared to four deaths among the 148 infants who did not have seizures (3 percent).

“Recognizing these seizures early is crucial for implementing treatments to prevent their recurrence,” Dr. Naim said, although she acknowledged that investigators have not yet demonstrated that treating seizures will improve long-term outcomes in high-risk pediatric heart patients.

An editorial commentary that accompanied the study calls for a larger, multicenter study to analyze continuous EEG monitoring following infant heart surgery and to investigate seizure prevention and treatment. The commenters also advocate for a less expensive and less labor-intensive screening tool than continuous EEG monitoring.

Funds from the National Institutes of Health and the June and Steve Wolfson Family Foundation supported this research. For additional information, view the press release.

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Aug 20 2015

Vismodegib’s Activity Tested in Medulloblastoma Subgroups

medulloblastomaResearchers’ understanding of the molecular pathways involved in cancer has enabled the development of targeted drugs for diseases such as pediatric and adult recurrent medulloblastoma, a type of rare brain cancer.

Two studies reported in the Journal of Clinical Oncology assessed the efficacy of a drug called vismodegib (Erivedge) in a subgroup of patients with recurrent medulloblastoma. Tom Curran, PhD, FRS, deputy scientific director of The Children’s Hospital of Philadelphia Research Institute, contributed to the article.

“It is terrific to see this exciting application of precision medicine to brain tumors that arise in children and adults,” Dr. Curran said. “While there is a long way still to go, and drug resistance is an issue, this is an extremely promising result.”

Dr. Curran, who has a background in brain development and cancer research, has a longstanding interest in finding drugs to treat medulloblastoma with fewer long-term side effects than current surgery, chemotherapy, and radiation approaches. New therapies especially are crucial for patients with high-risk or recurrent/relapsed disease who have poor chances of survival.

Medulloblastoma is comprised of four molecular subgroups. The studies focused on the SHH-MB subgroup, which is associated with activation of the sonic hedgehog (SHH) pathway. The SHH signaling pathway is crucial for the normal development of the cerebellum, a part of the brain that helps to coordinate movements such as posture, balance, and speech. Researchers suspect that overactive SHH signaling can cause tumors to proliferate.

Previous research suggests that SHH pathway inhibitors, such as vismodegib, could be useful to treat patients in the SHH-MB subgroup. Dr. Curran identified the SHH pathway as an ideal target for medullobastoma therapies after noting that Gorlin syndrome, a disorder caused by a mutation in the gene associated with the SHH pathway, is associated with a predisposition to these brain tumors.

In the current studies, participants’ tumors were prescreened for SHH-pathway activation. Out of 20 participants with recurrent SHH-MB who received oral vismodegib daily, the researchers observed a reduction in tumor area in three adults and a child. While this disease response was seen in only a few patients, prolonged disease stabilization occurred in 41 percent of patient cases with SHH-MB, which indicates that vismodegib has favorable activity. They observed no responses in patients with recurrent non-SHH-MB.

“This work represents the culmination of efforts of a large collaborative team starting in 1995 to develop a molecular-targeted treatment for medulloblastoma,” Dr. Curran said. “The clinical results are very consistent with preclinical studies indicating that the HH pathway inhibitor, vismodegib, is efficacious in a particular molecular subtype of tumor.”

The variable response to vismodegib in patients with recurrent SHH-MB suggests the presence of additional tumor mutations that may occur downstream of the transmembrane protein called Smoothened that the drug helps to block. The investigators concluded that while SHH inhibitors “should be advanced in SHH-MB studies, molecular and genomic work remains imperative to identify target populations that will truly benefit.”

The SHH-MB subgroup appears to be less common in the pediatric population than in the adult population, so the researchers do not have enough data yet to make any conclusions about using vismodegib for children. In future research studies, they emphasized that children exposed to vismodegib should continue to be monitored for safety and efficacy.

The studies were performed as part of the Pediatric Brain Tumor Consortium, a multidisciplinary cooperative research organization devoted to the study of correlative tumor biology and new therapies for primary central nervous system tumors of childhood. The co-investigators hold positions at St. Jude Children’s Research Hospital, Memphis; Preston Robert Tisch Brain Tumor Center, Duke University, Durham, N.C.; Center for Neuroscience Research, Children’s National Medical Center, Washington, D.C.; Ann & Robert Lurie Children’s Hospital of Chicago; Helen Diller Cancer Center, University of California, San Francisco; Texas Children’s Cancer Center, Houston; National Cancer Institute, Bethesda, MD; and Cincinnati Children’s Hospital.

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Aug 18 2015

Researchers Testing Video to Increase Teens’ Acceptance of HPV Vaccine

HPVAbout 79 million Americans are currently infected with human papillomavirus (HPV), and 14 million people become newly infected each year. Yet, despite these very real numbers, misconceptions about HPV seem to overshadow attempts to increase vaccination rates to prevent infection.

In 2014, coverage estimates for HPV continued to lag behind other routine immunizations recommended for adolescents’ ages 11 and 12, according to the Centers for Disease Control’s Morbidity and Mortality Weekly Report released July 25. Three vaccines that protect against HPV infection are currently available in the U.S. The quadrivalent (HPV4) and bivalent (HPV2) vaccines protect against HPV types 16 and 18, which cause 70 percent of cervical cancers; HPV4 also protects against HPV types 6 and 11, which cause 90 percent of genital warts. The newly introduced nonavalent vaccine (HPV9) includes an additional 5 serotypes that increases overall coverage to more than 85% of cervical cancers.

HPV is a sexually transmitted infection that can be spread by having oral, vaginal, or anal sex with someone who has the virus. It is so common that nearly all sexually active men and women get or are exposed to it at some point in their lives.

These are some of the important facts that will be conveyed in an educational video being tested at The Children’s Hospital of Philadelphia. It is geared toward 11 to 17 year olds and aims to improve parents’ and teens’ acceptance of HPV vaccination. Kristen Feemster MD, MPH, MSHP, principal investigator for the study, helped to develop the content in collaboration with a medical education company that is producing the video.

“I think a video-based education tool could be helpful,” said Dr. Feemster, a faculty member at PolicyLab at CHOP and an assistant professor of Pediatrics in the division of infectious diseases at the Perelman School of Medicine at the University of Pennsylvania. “It’s interactive, and it’s coming from a different voice. The video includes images of teens, and cartoon images to illustrate the HPV virus. I think all of those methods help get the message across.”

The study will include about 300 participants who will be randomized to either receive routine care, which is receipt of the HPV Vaccine Information Statement (VIS), or to receive the VIS in addition to viewing the educational video. The study team expects to begin data analysis by the end of this year.

“We are hoping to see an impact,” Dr. Feemster said. “The video is shown before the provider comes in to make a recommendation, so this is not the only thing that will drive their decision making.”

If the study shows that the education video is effective, it eventually could be disseminated to clinicians as a helpful cancer prevention resource to addresses parents’ and teens’ questions regarding HPV vaccine safety and efficacy in a format that teens are comfortable using.

For example, boys may think that they do not need to be vaccinated against HPV because they cannot develop cervical cancer. Actually, the vaccine protects against HPV strains that also can help prevent penile, anal, and oropharyngeal cancers. Also, since sexual partners can be exposed to the disease, vaccinating males and females helps to increase the protection of others against cancers associated with HPV.

“You receive great individual protection from the vaccine, but to block transmission it helps to have as many people vaccinated as possible,” Dr. Feemster said. “This vaccine has a potential to make a significant public health impact globally and understanding ways to implement recommendations will be really important.”

According to the Center for Disease Control and Prevention, about 360,000 people in the U.S. get genital warts each year and more than 11,000 women in the U.S. get cervical cancer each year. These numbers seem unnecessarily high when a safe and effective vaccination to prevent HPV transmission has been available since 2006.

A compilation of common questions and answers about HPV and the vaccine is available on The Children’s Hospital of Philadelphia’s website.

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Aug 13 2015

Hospitals Missed Screening Opportunities for Child Abuse

child abuseMore than 1,500 children die from child abuse in the U.S each year, and many more are injured. Hospitals, however, may be missing opportunities to protect these young victims of abuse.

A study published in the August issue Pediatrics showed a wide variation in how hospitals screen for hidden injuries, also called “occult injuries,” in young children with a diagnosis of physical abuse. These are injuries, such as cracked ribs or brain injuries, which may not be seen immediately during a physical exam and are only discovered via screening imaging. A series of X-rays, called a skeletal survey, are recommended by American Academy of Pediatrics guidelines for children younger than 2 years with fractures suspicious for child abuse.

The study results revealed that out of 2,500 children under 2 years old with an abuse diagnosis, only half (48 percent) were evaluated for occult fractures using skeletal surveys. The data showed a wide variation in evaluation practices among the 366 U.S. hospitals included in the study. None of the infants underwent a skeletal survey at some hospitals, while all of the infants underwent a skeletal survey at other hospitals.

“We need to work to improve and standardize care for this vulnerable population,” wrote Joanne Wood, MD, MSHP, in a blog about the study that she conducted with colleagues from The Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania. “Some hospitals have begun implementing clinical guidelines and other supports to aid medical providers in identifying which young injured children should undergo an evaluation for physical abuse. Research suggests that implementation of such guidelines can decrease disparities in evaluation practices and improve the detection of abuse.

A 2012 study also led by Dr. Wood revealed variation in rates of skeletal surveys performed at 40 large pediatric hospitals — 55 percent to 93 percent — but overall, 83 percent of the children received a skeletal survey. In the current study, the investigators looked at primarily non-pediatric focused hospitals. They noted that the majority of injured children receive care at these general hospitals, which are less likely to have specialized child abuse consultants than pediatric centers.

Taking into account previous research estimates of the percentage of children expected to have occult fractures revealed on skeletal surveys, Dr. Wood and her co-authors suggest that one of every seven or eight children who participated in the study sample may have had undiagnosed occult fractures.

At CHOP, Dr. Wood is a faculty member of PolicyLab, an attending physician in the division of general pediatrics, and director of research for Safe Place: Center for Child Protection and Health. She also is an assistant professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. The study team included Benjamin French, PhD, of biostatistics and epidemiology at Upenn; Lihai Song of healthcare analytics for PolicyLab; and Chris Feudtner, MD, MPH, PhD, an attending physician in CHOP’s division of general pediatrics and director of CHOP’s department of medical ethics.

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Aug 11 2015

PEDSnet Awarded $8.6 Million to Support Clinical Data Research Network

PEDSnetPEDSnet, a patient-centered clinical data research network led by The Children’s Hospital of Philadelphia, received $8.6 million in funding to continue its discovery and implementation of new ways to provide the best care and outcomes for children.

The three-year grant from the Patient-Centered Outcomes Research Institute (PCORI), an independent nonprofit organization based in Washington, D.C., allows the 1-year-old PEDSnet to embark on phase two of a project to create a sustainable pediatric clinical research infrastructure. It aims to integrate data available from eight of the nation’s largest children’s hospital systems with three condition-specific networks and two national data partners.

Most pediatric disorders are rare diseases, so no single pediatric institution usually has enough patients to generate large numbers of study participants. Providing access to diverse, nationally representative health information from millions of children will facilitate a range of study designs and reduce the time and effort needed to launch new studies and accelerate scientific knowledge.

“With our first phase of funding, PEDSnet developed infrastructure for rapid learning with observational studies and clinical trials,” said CHOP’s Christopher Forrest, MD, PhD, who is the principal investigator of PEDSnet and a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “This second phase of funding will enable PEDSnet to reach a high level of operational excellence, ultimately moving into a sustainable research network that advances children’s health through faster and cheaper clinical research.”

Some of the goals for phase two include adding natural language processing expertise for the data coordinating center to use in developing computable phenotypes and outcome measures. The PEDSnet investigators also will strengthen the engagement of parents and clinicians throughout the network to ensure that concerns important to families are research priorities.

Linking data from PEDSnet’s national data partners and regional health plans will allow for the creation of a cohort of patients with complete data available through PEDSnet’s electronic health record data resource. PEDSnet is comprised of The Children’s Hospital of Philadelphia, Cincinnati Children’s Hospital Medical Center, Children’s Hospital Colorado, Nemour’s Children’s Health System, Nationwide Children’s Hospital, St. Louis Children’s Hospital, Seattle Children’s Hospital, and Boston Children’s Hospital. The specialty networks include the National Pediatric Cardiology Improvement Collaborative (hypoplastic left heart syndrome), ImproveCareNow (inflammatory bowel disease), and Healthy Weight Network (childhood obesity). The data partners are Express Scripts and the health analytics company IMS Health.

PEDSnet is one of 10 other clinical data research networks under the umbrella of PCORnet: the National Patient-Centered National Clinical Research Network. During the phase one development stage, the individual systems worked to standardize their data and develop policies governing data sharing, security, and collaboration.

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Aug 06 2015

Home Plate Study Hits the Ground Running on Healthy Food Prep

healthy foodNearly one in three children are considered to be overweight or obese in the U.S. What these children have on their plates at home today can affect their health for tomorrow.

Senbagam Virudachalam MD, MSHP, faculty member at The Children’s Hospital of Philadelphia’s PolicyLab, is leading a three-year study to test a community-based intervention that helps parents of toddlers practice healthy food preparation.

The “Home Plate” study will include parents and mentors who will participate in weekly cooking classes for six weeks. The trained study staff will collect 24-hour dietary recalls from adult caregivers, and the results will then be used to calculate the energy density of the children’s diets.

A unique aspect of the study is that investigators are working closely with several respected West Philadelphia community organizations including WIC, Early Head Start, and The Enterprise Center, who have been instrumental in recruiting and engaging interested families in the study.

“Twenty parents and five mentors will participate in each six-week series of classes, where they will come together weekly to cook and eat a meal, with the goal of learning by doing, rather than through didactic instruction alone,” Dr. Virudachalam said. “We hope that parents who participate in the classes will gain both the confidence and skills to regularly prepare healthy food at home, resulting in improved diets for the entire family.”

In previous research, Dr. Virudachalam examined data from the National Health and Nutrition Examination Survey 2007-2008 to determine the prevalence and patterns of cooking dinner at home in the U.S. The analysis defined the scope of just how large of an issue home dinner preparation habits are for the daily lives of Americans.

“We found that while half of all Americans reported that they always cook dinner at home, the remainder obtain many or most dinners away from home,” Dr. Virudachalam noted. “Preparing healthy food at home is potentially one of the most significant points for effective interventions to curb the obesity epidemic, both at the individual and population level.”

Dr. Virudachalam and her co-authors concluded that dinner preparation habits vary with socio-economic status and race/ethnicity. Poorer, less educated households were more likely to either always or never cook dinner at home, and wealthier, more educated households were more likely to sometimes cook dinner at home.

“Half of all African American and Latino children born in the year 2000 will develop diabetes or heart disease [due to obesity],” Dr. Virudachalam stated in her analysis.

She suggested that tailored interventions could help families limit fast food and promote healthy eating from home, which are the strategies that the Home Plate study is serving up. It has the potential to help parents learn how to make nutritious food choices that will become part of an overall healthier lifestyle for their families.

“If successful, the Home Plate intervention would provide a low-cost model for changing eating behaviors that could be replicated by community-based organizations throughout the country,” said Dr. Virudachalam, who also is an assistant professor of Pediatrics at CHOP and the Perelman School of Medicine at the University of Pennsylvania. “Such interventions are crucial for successfully addressing the obesity epidemic.”

A $750,000 investment from The Aramark Charitable Fund to the Healthy Weight Program at CHOP is funding the Home Plate study, which is expected to be completed in October 2017.

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Aug 04 2015

New Ways Needed to Predict, Treat Relapsed Leukemia

leukemiaBench-to-bedside research at The Children’s Hospital of Philadelphia involves talented scientists who make discoveries in the lab that translate into investigations of clinical applications. CHOP experts studying T-cell acute lymphoblastic leukemia (T-ALL) are taking this model one step further — bench to bedside and back again — by turning around their findings from clinical studies to better inform new series of basic science experiments.

T-ALL is a cancer of a type of white blood cells called T-lymphocytes. Normally, T-lymphocytes help fight infections. When the bone marrow and thymus gland produce too many immature T-lymphocytes, the body’s ability to fight infections becomes compromised.

Despite advances in T-ALL treatment, between 15 and 20 percent of children and young adults who achieve an initial complete remission will have the disease return (relapse). They may need more intensive therapy or alternative approaches, but physicians do not yet have a reliable way of predicting which patients are at high-risk of relapse.

David Teachey, MD, an attending physician and researcher at CHOP with expertise in both oncology and hematology, is leading a study involving researchers from across the country who aim to develop new insights to improve risk stratification of T-ALL by increasing their understanding of its biochemical underpinnings. This work is being funded by the National Cancer Institute through an R01.

“This new grant is trying to tackle two problems,” Dr. Teachey said. “One is we don’t know how to identify the kids who relapse when they walk in the door — you don’t know until it’s too late. And two, we need to develop new therapies for the ones who do relapse.”

The study team will analyze patient samples collected from an international, phase 3 clinical trial called AALL1231 that Dr. Teachey chairs for the Children’s Oncology Group. AALL1231 includes approximately 1200 children and young adults from more than 200 hospitals, and the goal is to determine if standard chemotherapy with or without the drug bortezomib is more effective in treating T-ALL.

In the new study, the investigators will take a protein level approach to help identify subsets of patients with T-ALL who are likely to relapse. They will study participants’ leukemia cells and the proteins that they make, looking for patterns that clinicians could use as a high-risk molecular signature. Next, they will see if there is any correlation between these protein profiles and results from sophisticated next-generation sequencing that allows researchers to detect minute levels of cancer cells that remain after intensive, high-dose, multi-agent chemotherapy.

If the study team can figure out which proteins are abnormal in the patients who do poorly, then they could eventually test drugs that target those altered pathways. They will accomplish this by generating mouse models using the participants’ leukemia cell samples. Since the patients already are involved in the AALL1231 trial, the study team will know who did well and who went on to relapse, so they will be able to see how different therapies affect their prognosis.

For instance, the JAK/STAT signaling pathway is implicated in a high-risk subtype of T-ALL, according to a paper published in the journal Blood earlier this year by Dr. Teachey and colleagues. If drugs are available that can turn off that pathway, then they may be beneficial in combination with existing chemotherapy agents. The study team intends to identify other signaling pathways that are abnormally activated in T-ALL, which may give them clues to better predict chemotherapy response and resistance.

“In the big picture, the hope is the correlative biology performed in the lab will let us learn how to better diagnosis and treat children with T-ALL. Then, we can open a future trial using new drugs to treat children who we think aren’t going to do well and improve their chance of being cured,” Dr. Teachey said.

The project will involve researchers from Seattle Children’s Hospital; UCSF Medical Center, California; The University of Texas MD Anderson Cancer Center, and the University of Texas Southwestern Medical School, in addition to researchers from The Children’s Hospital of Philadelphia.

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Jul 30 2015

Researchers Launch New Investigations With CPCE Pilot Grants

CPCEIf scientific research were like building a house, pilot studies would be the foundation. Their purpose is to establish solid evidence that will attract external support for large-scale studies. The Center for Pediatric Clinical Effectiveness (CPCE) at The Children’s Hospital of Philadelphia is giving three investigators the tools that they need to get to start digging.

Through the CPCE Pilot Grant Program, fellows, junior faculty, and other Children’s Hospital investigators interested in clinical effectiveness research have the opportunity twice a year to receive funding to support their pilot studies designed to produce evidence of what works best for treating, diagnosing, and preventing disease. The three recipients of the 2015 Spring Pilot Grant Award are Sandra Amaral, MD, MHS, Sabrina Gmuca, MD, and Laura Figueroa -Phillips, MD.

Find out more about the awardees and highlights of their projects in this Q&A:

Sandra Amaral, MD, MHS

Tell us a little bit about your background:

I am an assistant professor and attending physician in CHOP’s Division of Nephrology and the Kidney Transplant and Dialysis Program. I received my MD from the MCP-Hahnemann University School of Medicine and a Master of Health Science degree in clinical epidemiology from Johns Hopkins University, Rollins School of Public Health.

What is unique about your pilot research study?

My pilot study, “Using a Mobile Game Application to Improve Healthcare Self-management in Adolescents With Solid Organ Transplants” aims to develop a novel educational mobile health (mHealth) game for adolescents with solid organ transplants (SOT) and test its feasibility and acceptability among adolescent SOT recipients and their caregivers. Adolescent SOT recipients have poor long-term graft survival rates, and are at particularly high risk for unnecessary graft loss due to non-adherence in the management of their complex chronic health conditions. This study will employ novel mHealth gaming technology to augment current care practices and to change how adolescents think about healthcare management, with the ultimate goal of reducing graft loss due to non-adherence.

How do you anticipate your study results will lead to future research?

The findings from this study will provide pilot data in support of future research to test the generalizability and efficacy of the mHealth game in a broader adolescent patient population. If successful, this study will transform positive health behavior and medication adherence research, making patient-centered interventions available in an enjoyable and user-friendly platform. This line of research will be broadly relevant to improving outcomes for all pediatric patients managing complex treatment regimens for chronic health conditions.

What was your reaction to receiving the CPCE award?

I was tremendously grateful and excited. The seeds for this project were planted for me about two years ago when I took a class at Penn on Healthcare Innovations. I have been working steadily on refining the project since then but progress has been slow — mostly due to lack of funding. The CPCE pilot funding will provide me with the opportunity to work closely with a game developer and programmer so I can move this idea into a real, testable product for patients and modified by patients and their caregivers.

Sabrina Gmuca, MD

Tell us a little bit about your background:

I am a first-year Rheumatology fellow in CHOP’s Department of Pediatric Rheumatology and the Center for Childhood Arthritis and Rheumatic Diseases. I received my MD from State University of New York Downstate Medical Center College of Medicine and completed my residency in pediatrics at NYU Langone Medical Center. My research interests include the evaluation of treatment and outcomes in autoimmune conditions affecting the central nervous system.

What is unique about your pilot research study?

Pediatric Neuromyelitis Optica (NMO) is a chronic inflammatory disease of the central nervous system that preferentially targets the optic nerves and spinal cord. By analyzing NMO patient outcomes data over a 13-year period, my retrospective cohort pilot study, “Treatment and Outcomes in Neuromyelitis Optica,” will attend to the current lack of standardized care in the therapeutic management of pediatric NMO.

How do you anticipate your study results will lead to future research?

The results of this study will provide an increased understanding of disease outcomes in pediatric and adult NMO, and its findings will be clinically useful for both rheumatologists and neurologists. Further, if successful, the study’s findings will facilitate future comparative effectiveness trials of treatment with biologics, and it will ultimately make possible the establishment of the first standardized therapeutic protocol for pediatric NMO.

What was your reaction to receiving the CPCE award?

Upon receiving word that I had been awarded funding through the CPCE pilot grant, I felt immediately honored and overjoyed. The Center for Pediatric Clinical Effectiveness allows fellow physicians such as myself to jumpstart our research interests and put ideas into action by providing us both valuable mentorship and important financial support. I am excited and eager to start my research and help improve patient outcomes in autoimmune diseases.

Laura Figueroa-Phillips, MD

Tell us a little bit about your background:

I am a first-year Pediatric Hospital Epidemiology and Outcomes Training fellow at CHOP. I received my MD from the University of Maryland School of Medicine and completed my residency in pediatrics at CHOP. My research and clinical interests revolve around improving patient-centered outcomes for children and their families in an inpatient setting, as well as the epidemiology, diagnosis, and treatment of healthcare related infections.

What is unique about your pilot research study?

Long-term, outpatient central venous access is necessary for the treatment of many pediatric conditions, but can result in serious complications, including central line-associated bloodstream infections (CLABSIs). My nested case-control pilot study, “Development of a Clinical Prediction Model for Pediatric Outpatients at Risk for Central Line-Associated Bloodstream Infections” aims to identify risk factors and derive a preliminary clinical prediction model for CLABSI among ambulatory pediatric patients with central venous lines presenting with bacteremia-associated symptoms. This study will be the first to determine the overall rate of CLABSI among the ambulatory pediatric population with central venous lines, to evaluate the entire population of patients at risk for CLABSI, and to derive a clinical prediction model for CLABSI.

How do you anticipate your study results will lead to future research?

The results of this study will serve as a first step in the development of a clinical prediction tool to guide clinical decision-making when patients present to the Emergency Room, and it could ultimately decrease unnecessary hospital admissions, lower the number of adverse effects associated with exposure to broad spectrum antibiotics, improve patient and family quality of life, and decrease healthcare spending.

What was your reaction to receiving the CPCE award?

I was thrilled and honored to be chosen as a CPCE Pilot Grant awardee. The grant will give me a great launching pad to successfully complete my project and will help me set the groundwork to receive funding for related projects in the future.

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Jul 28 2015

Young Adults Find Selecting Health Insurance Plans Confusing

health insuranceWhile young adults are essential to the implementation of the Affordable Care Act (ACA), the process of applying for health insurance using the federal insurance marketplace can be confusing to them, according to a small, observational study.

Historically, a significant part of the young adult population in the U.S. has been uninsured. Under the ACA’s goal to expand access to health insurance for everyone, they have new choices when shopping for affordable insurance plans. They can compare insurance options on with plans outside the marketplace such as those offered by employers, schools, or their parents’ health insurance.

“Most of the young people in our study were buying health insurance for the first time since many had previously been on their parents’ plans, and they found the process of selecting from 30 plan options in Philadelphia challenging and overwhelming,” said Charlene Wong, MD, principal investigator of the study published in the Journal of Adolescent Health. “The young adults’ thoughts on health insurance and, for example what they are looking for and the challenges they identified, can inform more effective outreach efforts and improvements to the website.”

In the study conducted from January to March 2014, Dr. Wong and colleagues described young adults’ experiences using and their attitudes toward health insurance, health insurance literacy, and benefit and plan preferences. Thirty-three study participants’ ages 19 to 30 who lived in Philadelphia County and were highly educated spent up to 30 minutes exploring During the sessions, the study team recorded study participants’ “think aloud” reactions in real-time.

For example, one participant said, “I just wasn’t able to comprehend all of the things on — I got confused. I’m not a person to give up, not at all — but with the system, I just wanted to quit.”

Study participants struggled with many of the basic insurance terms that appeared on the website. Of the 33 participants, 48 percent incorrectly defined “deductible” and 78 percent incorrectly defined “coinsurance.”

The investigators conducted follow-up interviews with the participants to discuss their perspectives in more detail and also to identify factors important to their enrollment decisions. The results showed that deductible and premium amounts and preventive care coverage were most important to the participants’ plan selection.

“Based on this study, we developed recommendations for improvements to that have been disseminated to the Office of Health Reform. For example, the young adults suggested that pop-up explanations that appear when your cursor is over an important term like ‘deductible’ would have been helpful. We also have ongoing work on the tools available to consumers on and other state-based insurance marketplaces to help simplify or support them in making informed health insurance choices,” said Dr. Wong, who is a fellow with the Craig-Dalsimer Division of Adolescent Medicine program at The Children’s Hospital of Philadelphia.

The next open enrollment period for and other state-run health insurance marketplaces is November 2015.

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