2021 Grant Finalists
The six finalists listed here will present their project proposals to Leadership Circle members in April 2021. If you’re a current member, you’ll receive further information about the presentation in early 2021.
Not a current member? Renew your membership or join now for the opportunity to invest in these and other innovative, impactful projects that have the potential to transform pediatric medicine.
Post-Traumatic Stress Disorder in Bone Marrow Transplant Patients and Their Families
Principal Investigator: Cady Berkel, PhD, Data Analyst
Department/Division: Palliative Care/Bone Marrow Transplant
Amount Requested: $39,766
Bone marrow transplant (BMT) is a critical lifesaving intervention used to treat cancer and other life-threatening diseases at Phoenix Children’s. However, children undergoing this treatment—and members of their families—are at significant risk for post-traumatic stress disorder (PTSD). PTSD is a serious mental health diagnosis that does not go away without intervention. However, no pediatric hospitals systematically screen for or treat PTSD symptoms for BMT patients or their families.
We propose to develop a system that would allow us to use validated and freely available screeners to monitor BMT patients and families for PTSD symptoms prior to each regularly scheduled visit for a year following transplant. We will link families who report symptoms to mental health support. We will also use the information as part of a research project that would allow us to be more proactive in providing support for families who are likely to develop symptoms. Once the system is built, it can be used on an ongoing basis for all BMT families. It can also be used as a model for other programs at Phoenix Children’s.
Home Clinical Exercise Monitoring: Unraveling the Exercise Potential
Principal Investigator: Rajeev Bhatia, MD, Pediatric Pulmonologist; Pulmonology Division Chief
Amount Requested: $127,509
Cardiopulmonary exercise testing (CPET) provides a global assessment of lung, heart, blood and skeletal muscle systems under stress. CPET is becoming increasingly crucial as exercise capacity is a significant predictor of health outcomes (e.g., the mortality rate in cystic fibrosis) in nearly every chronic disease state, particularly in patients with heart or lung disease. These indicators are not evident in regular testing done at rest with the routine clinic or hospital visits. Though the exercise response may change rapidly over weeks to months, the burden of equipment and resources to perform CPET in the clinic or hospital setting makes frequent testing impractical. For example, a typical hospital-based lab may only perform five or six clinical studies per day.
This project seeks to develop new equipment and software that are miniaturized and portable so that CPET may be performed more frequently and in different environments such as the patient’s home—something extremely relevant in our current environment. This project would supplement existing hospital testing to allow more frequent home assessment without overloading the hospital resources.
A portable system would also allow us to evaluate a patient in unique environments, which may be relevant for athletes or for those who live at elevation, which is among the several environments we cannot replicate in the more sterile laboratory setting. The benefits of home exercise testing could significantly impact children with pulmonary and congenital heart disease and many other children with chronic disease states (e.g., muscle disorders, chest deformity and sickle cell disease) or concerning symptoms during exercise.
Dyslexia Screening Questionnaire (DySQ)
Principal Investigator: Jodi P. Carter, MD, Chief Clinical Integration Officer, Phoenix Children’s; Chief Medical Officer, Phoenix Children’s Care Network; Section Chief, Phoenix Children’s Pediatrics
Amount Requested: $149,711
Dyslexia is a common learning disorder that involves difficulty reading due to problems identifying speech sounds and learning how they relate to letters and words (decoding). Dyslexia affects areas of the brain that process language. People with dyslexia have normal intelligence and usually have normal vision. Without timely diagnosis and treatment, children with dyslexia are much more likely to have poor academic achievement, anxiety, low self-esteem and depression. They are also more likely to drop out of school, which in turn results in limited employment opportunities, poor health outcomes and lower life expectancy. Although there is no cure for dyslexia, early assessment and intervention result in the best outcome.
This project addresses the critical challenge that many children diagnosed with dyslexia are diagnosed too late to optimally benefit from intervention. Although pediatricians routinely screen for many developmental milestones, they do not routinely screen for dyslexia because there is no tool with which to do so. Instead, the general pediatric community relies on the public education system to identify children with dyslexia. Ironically, often the public education system relies on the pediatric community to facilitate the formal diagnosis of dyslexia. In order to solve this problem, we developed a dyslexia screening questionnaire (DySQ) for pediatricians to use in order to screen for reading disorders in children finishing kindergarten or enrolled in first grade at the time of their 6-year well-check visit.
The primary outcome of this project is to prove that the DySQ works (i.e., it accurately screens positive for children with dyslexia and accurately screens negative for children who do not have dyslexia). In order to accomplish this, we plan to have the parents of 600 children complete the DySQ in a pediatrician’s office while their respective children meet with the ASU team and complete the current “gold standard” tests necessary to diagnose dyslexia. The ultimate goal of this project is to have a screening questionnaire that accurately screens for dyslexia and may be used nationally by all pediatricians.
A Complete Solution Suite for Fetal Cardiac MRI: Accurate Cardiac Gating, Motion Correction and 3D Reconstruction of the Fetal Heart
Principal Investigator: Luis F. Goncalves, MD, Pediatric Radiologist; Director of Fetal Imaging
Amount Requested: $129,380
In Arizona, which recorded 80,393 births in 2018, congenital heart disease (CHD) is expected to affect approximately 8,000 children per year. CHD is responsible for 28 percent of neonatal deaths due to birth defects and 50 percent of deaths occurring during the first two to 12 months. A large number of these children seek advanced clinical and surgical treatment at Phoenix Children’s Hospital, often after being diagnosed while still in the mother’s womb. These fetuses require advanced fetal imaging to achieve a precise prenatal diagnosis that can lead to an optimal transition from fetal to postnatal life and improved outcome.
Although fetal MRI already plays a large role in the prenatal diagnosis of the more challenging cases of CHD, there is tremendous room for improvement. Current technology provides superb evaluation of larger anatomical structures and pathologies and thus helps us identify additional anomalies (for example the brain) that may also be present in these fetuses. However, the inherent motion of the fetus coupled with the small size of the fetal heart and the extremely fast fetal heart rate make it challenging to image the fetal heart in order to provide clinically useful interpretations.
To solve this problem, computational pipelines have been recently developed to compensate for sources of motion (namely the beating fetal heart, gross fetal body motion and maternal breathing), synchronize the fetal heart rate with the images, and increase the original resolution. These computational pipelines, although promising, currently suffer from two major problems: (1) the lack of a user-friendly interface, which requires 24/7 availability of a professional with advanced skills in computer engineering for implementation, and (2) a substantial amount of processing time of approximately 24–36 hours before images are available for interpretation.
Our proposal is to solve the two problems described above by producing a fast, user-friendly software for post processing of fetal cardiac MRI that can be used clinically by any technologist or radiologist to generate diagnostic-quality 2D and 3D images of the fetal heart. The entire cost for this project is the initial cost of creating the software. Once the software is incorporated into clinical workflow, the technologists that regularly process our images will be able to process fetal cardiac scans. This project will be completed and result in a workflow with zero additional cost to the Radiology Department after the first year.
Female Low Iron Prevalence and Prediction (FLIPP) Study
Principal Investigator: Ajay Perumbeti, MD, Transfusion Medicine/Medical Director of Lab Informatics/Clinical Integration/Decision Support
Department/Division: Pathology/Transfusion Medicine and Lab Informatics
Amount Requested: $105,000
Iron deficiency secondary to low body iron has a negative impact on health and quality of life in adolescent girls and young adult women (AYA females). This has been well described in the medical literature and media, including the journals Blood and Pediatrics as well as the New York Times, the Washington Post and Oprah.
Iron deficiency can first present with subtle or substantial symptoms including poor concentration, fatigue, inability to exercise and restless leg syndrome. More moderate to severe iron deficiency causes anemia. Approximately 8 million AYA females in the United States are estimated to have iron deficiency anemia, and double this number have low body iron. This equates to up to one in five AYA females having iron deficiency. The gold standard test for detecting low body iron is the ferritin blood test, which requires big, expensive machines and longer times to receive results.
What if we could leverage a simpler, faster and cheaper complete blood count (CBC) test for early detection of low body iron? This could allow less missed opportunities and faster risk-based treatment for low iron. We have preliminary data showing that we can use the CBC blood test with combinatorial analysis to predict greater than 50 percent of iron deficiency in its early stages in AYA females. We have additional data that shows machine learning algorithms may further improve the predictive value.
Funding is requested for a prospective trial, Female Low Iron Prevalence and Prediction (FLIPP) Study, to evaluate predictive value of CBC and electronic medical record data using combinatorial analysis and machine learning technology for low body iron. The funding would support patient recruitment, laboratory testing, study coordination, institutional review board fees and hospital information technology support. FLIPP will facilitate systematic evaluation and potential treatment of low iron body iron in 2,000 adolescent and young adult females, determine low iron prevalence in adolescent girls cared for at Phoenix Children’s, and evaluate promising combinatorial and machine learning approaches for predicting low iron state. FLIPP clinical decision-support tools may further contribute to the worldwide effort to reduce iron deficiency in adolescent girls and young adult women.
Men are from Mars, Women are from Venus: Equipment and Supplies to Evaluate How Sex Hormones Influence Brain Function
Principal Investigator: Theresa Currier Thomas, PhD
Department/Division: Barrow Neurological Institute at Phoenix Children’s Hospital, NeuroTrauma Lab
Amount Requested: $99,924
This research will impact adolescent and postpubescent pediatric patients with known endocrine or neurological deficits. Identification of sex differences in brain function will further drive personalized medicine, improving quality of care for female pediatric patients with translatability to adult female patients.
Historically, females are underrepresented in preclinical and clinical research trials across all disciplines, especially in neurosciences. Therefore, preclinical research data from males is often generalized to females for clinical translation, making dangerous assumptions that the neurobiology is the same and putting female patients at higher risk for adverse effects.
Depression, anxiety and other mood disorders are more prevalent in females and often have a basis in dysregulation of the neuroendocrine axis. While receptors for hormones are located on all cells in the body, the impact of circulating sex hormones (estrogen, progesterone, etc.) on neurotransmission in both sexes is poorly understood.
The proposed project is aligned with Phoenix Children’s mission, vision and strategic foci as it seeks to improve female patient care through innovative research that capitalizes on the unique skill sets and knowledge of current Phoenix Children’s employees and the use of state-of-the-art equipment in order to lead the nation in the evaluation of sex differences in basic neurotransmission, after neurological insult and during recovery. Publication of results will highlight the growth and expansion of Phoenix Children’s research programs, which is associated with training residents, graduate students, etc. Publications are available worldwide, enhancing the geographic presence of Phoenix Children’s.