Medicine and Health Sciences Commons^™

Consensus Parameter: Research Methodologies To Evaluate Neurodevelopmental Effects Of Pubertal Suppression In Transgender Youth., Diane Chen, John F Strang, Victoria D Kolbuck, Stephen M Rosenthal, Kim Wallen, Deborah P Waber, Laurence Steinberg, Cheryl L Sisk, Judith Ross, Tomas Paus, Sven C Mueller, Margaret M Mccarthy, Paul E Micevych, Carol L Martin, Baudewijntje P C Kreukels, Lauren Kenworthy, Megan M Herting, Agneta Herlitz, Ira R J Hebold Haraldsen, Ronald Dahl, Eveline A Crone, Gordon J Chelune, Sarah M Burke, Sheri A Berenbaum, Adriene M Beltz, Julie Bakker, Lise Eliot, Eric Vilain, Gregory L Wallace, Eric E Nelson, Robert Garofalo

Department of Pediatrics Faculty Papers

Purpose: Pubertal suppression is standard of care for early pubertal transgender youth to prevent the development of undesired and distressing secondary sex characteristics incongruent with gender identity. Preliminary evidence suggests pubertal suppression improves mental health functioning. Given the widespread changes in brain and cognition that occur during puberty, a critical question is whether this treatment impacts neurodevelopment.

Methods: A Delphi consensus procedure engaged 24 international experts in neurodevelopment, gender development, puberty/adolescence, neuroendocrinology, and statistics/psychometrics to identify priority research methodologies to address the empirical question: is pubertal suppression treatment associated with real-world neurocognitive sequelae? Recommended study approaches reaching 80% consensus were …

A Novel Noninvasive Approach For Evaluating Work Of Breathing Indices In A Developmental Rat Model Using Respiratory Inductance Plethysmography., Yan Zhu, Zubair H. Aghai, Suhita G. Betal, Michael Favara, Gina Fong, Tariq Rahman, Thomas H. Shaffer

Department of Pediatrics Faculty Papers

Pulmonary function testing (PFT) is an important component for evaluating the outcome of experimental rodent models of respiratory diseases. Respiratory inductance plethysmography (RIP) provides a noninvasive method of PFT requiring minimal cooperation. RIP measures work of breathing (WOB) indices including phase angle (Ф), percent rib cage (RC %), breaths per minute (BPM), and labored breathing index (LBI) on an iPad. The aim of this study was to evaluate the utility of a recently developed research instrument, pneuRIP, for evaluation of WOB indices in a developmental rat model. Sprague Dawley rats (2 months old) were commercially acquired and anaesthetised with isoflurane. …

Variation In Early Number Skills And Mathematics Achievement: Implications From Cognitive Profiles Of Children With Or Without Turner Syndrome, Sarah L Lukowski, Emily R Padrutt, Kyriakie Sarafoglou, Judith L. Ross, Jennifer R Law, Rachel E Olson, Michèle M M Mazzocco

Department of Pediatrics Faculty Papers

Individuals with Mathematics Learning Disabilities have persistent mathematics underperformance but vary with respect to their cognitive profiles. The present study examined mathematics ability and achievement, and associated mathematics-specific numerical skills and domain-general cognitive abilities, in young children with Turner syndrome compared to their matched peers. We utilized two independent peer groups so that group comparisons would account for verbal skills, a hypothesized strength of girls with Turner syndrome, and nonsymbolic magnitude comparison skills, a hypothesized difference of girls with Turner syndrome. This individual matching approach afforded characterization of mathematics profiles of girls with Turner syndrome and girls without Turner syndrome …

Peripubertal Serum Concentrations Of Organochlorine Pesticides And Semen Parameters In Russian Young Men., Ramy Abou Ghayda, Oleg Sergeyev, Jane S Burns, Paige L Williams, Mary M Lee, Susan A Korrick, Luidmila Smigulina, Yury Dikov, Russ Hauser, Lidia Mínguez-Alarcón

Department of Pediatrics Faculty Papers

BACKGROUND: Epidemiologic literature on the relation of organochlorine pesticides (OCPs) with semen quality among adult men has been inconclusive, and no studies have prospectively explored the association between peripubertal serum OCPs and semen parameters in young men.

OBJECTIVE: To evaluate prospective associations of peripubertal serum concentrations of hexachlorobenzene (HCB), β-hexachlorocylohexane (β-HCH), and p,p'-dichlorodiphenyldichloroethylene (p,p'-DDE) with semen parameters among young Russian men.

METHODS: This prospective cohort study included 152 young men who enrolled in the Russian Children's Study (2003-2005) at age 8-9 years and were followed annually until young adulthood. HCB, β-HCH, and p,p'-DDE concentrations were measured at the CDC by …

Fatal Case Of Newborn Lassa Fever Virus Infection Mimicking Late Onset Neonatal Sepsis: A Case Report From Northern Nigeria, Taofik Oluwaseun Ogunkunle, Surajudeen Oyeleke Bello, Chinwe Immaculata Anderson, Rashida Musa, Rasaq Olaosebikan, Abdulazeez Imam

Department of Pharmacology and Experimental Therapeutics Faculty Papers

BACKGROUND: Lassa fever is a zoonotic viral infection endemic to the West Africa countries. It is highly fatal during pregnancy and as such reports of neonatal onset Lassa fever infections are rare in scientific literature. We report a fatal case of Lassa fever in a 26-day-old neonate mimicking the diagnosis of late-onset neonatal sepsis.

CASE PRESENTATION: The patient is a 26-day-old neonate who was admitted with a day history of fever, poor feeding, pre-auricular lymphadenopathy and sudden parental death. He was initially evaluated for late onset neonatal sepsis. He later developed abnormal bleeding and multiple convulsions while on admission, prompting …

Hearing Loss In Mucopolysaccharidoses: Current Knowledge And Future Directions., Jeremy Wolfberg, Keerthana Chintalapati, Shunji Tomatsu, Kyoko Nagao

Department of Pediatrics Faculty Papers

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a deficiency of one of the enzymes involved in the degradation of glycosaminoglycans. Hearing loss is a common clinical presentation in MPS. This paper reviews the literature on hearing loss for each of the seven recognized subtypes of MPS. Hearing loss was found to be common in MPS I, II, III, IVA, VI, and VII, and absent from MPS IVB and MPS IX. MPS VI presents primarily with conductive hearing loss, while the other subtypes (MPS I, MPS II, MPS III, MPS IVA, and MPS VII) can present with …

Implementation Of Family Psychosocial Risk Assessment In Pediatric Cancer With The Psychosocial Assessment Tool (Pat): Study Protocol For A Cluster-Randomized Comparative Effectiveness Trial, Anne E Kazak, Janet A Deatrick, Michele A Scialla, Eric Sandler, Rebecca E Madden, Lamia P Barakat

Department of Pediatrics Faculty Papers

BACKGROUND: Childhood cancer affects and is affected by multiple levels of the social ecology, including social and relational determinants of health (e.g., economic stability, housing, childcare, healthcare access, child and family problems). The 2015 Standards of Psychosocial Care in Pediatric Cancer outline optimal psychosocial care sensitive to these ecological factors, starting with assessment of psychosocial healthcare needs to promote medical and psychosocial outcomes across all children with cancer. To address the first standard of family psychosocial assessment, the Psychosocial Assessment Tool (PAT) is a validated screener ready for broad implementation.

METHOD: The PAT will be implemented across a national sample …

Dosing Common Medications In Hospitalized Pediatric Patients With Obesity: A Review, Barbara Ameer, Michael Weintraub

Department of Medicine Faculty Papers

Medication management in children and adolescents with obesity is challenging since both developmental and pathophysiologic changes may impact drug disposition and response. Evidence to date indicates an effect of obesity on drug disposition for certain drugs used in this population. This work identified published studies evaluating drug dosing, pharmacokinetics, and effect in pediatric patients with obesity, focusing on 70 common medications used in a pediatric network of 42 U.S. medical centers. A PubMed search revealed 33 studies providing pharmacokinetic and/or effectiveness data for 23% (16/70) of medications, 44% of which have just one study and can be considered exploratory. This …

Vaccines, Children, And The Public Health Trust, Christopher Chambers, Md

Department of Family & Community Medicine Presentations and Grand Rounds

Objectives

1. Review the impact of immunizations on the public health over the past 50 years.
2. Childhood Vaccines Now
3. Describe the challenges associated with trying to eliminate “immunizable” diseases. Examples: Invasive Pneumoccal Disease, Neisseria meningitis
4. The Future

Ultra-Low-Dose Estrogen Therapy For Female Hypogonadism., Yukihiro Hasegawa, Tomoyo Itonaga, Kento Ikegawa, Satsuki Nishigaki, Masanobu Kawai, Eri Koga, Hideya Sakakibara, Judith L Ross

Department of Pediatrics Faculty Papers

In females, endogenous estrogen secretion increases gradually before pubertal development. The benefits of low-dose estrogen therapy in patients with Turner syndrome were originally discussed by Ross et al. and Quigley et al. These seminal studies used ethinyl estradiol (EE2), starting at a dose of 25 ng/kg/d. We hypothesized that the initial dosage of estrogen could be titrated to more closely mimic physiological increments of endogenous estrogen. Therefore, our recent study initiated EE2 treatment at a dosage of 1-2 ng/kg/d, an ultra-low-dose estrogen therapy in pediatric patients with Turner syndrome. The ultra-low-dose estrogen therapy in this syndrome produced a good …

Pediatric Resident Knowledge, Experience, Comfort, And Perceived Competency In Providing Sibling Psychosocial Support., David Buchbinder, Sonam Sidhu, Melissa A. Alderfer, Anne Lown, Russ C. Kolarik, Tommy Wang

Department of Pediatrics Faculty Papers

This article is the author’s final published version in International Journal of Medical Education, Volume 11, March 2020, Pages 73-75.

The published version is available at https://doi.org/10.5116/ijme.5e63.6a46. Copyright © Buchbinder et al.

Using The Medical Expenditure Panel Survey To Explore The Association Between High Healthcare Utilization And Behavioral Health Diagnoses In A Pediatric Population With Chronic Conditions, Marlon D. Satchell, Phd, Mph

Population Health Science Program Doctoral Dissertations

The 2010 Affordable Care Act (ACA) introduced an era of changing healthcare delivery and utilization, which is still evolving and changing to this day. Healthcare providers and healthcare systems need to identify strategies to provide better care to patients, that results in better patient outcomes, in a cost effective way. One strategy to accomplish this goal is to focus on managing the care of the most vulnerable patients, particularly including pediatric patients with one or more chronic conditions. The objective of this study was to assess the extent to which a behavioral health diagnosis in addition to another chronic condition …

Mucopolysaccharidosis Iva: Diagnosis, Treatment, And Management., Kazuki Sawamoto, José Víctor Álvarez González, Matthew Piechnik, Francisco J. Otero, Maria L. Couce, Yasuyuki Suzuki, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis type IVA (MPS IVA, or Morquio syndrome type A) is an inherited metabolic lysosomal disease caused by the deficiency of the N-acetylglucosamine-6-sulfate sulfatase enzyme. The deficiency of this enzyme accumulates the specific glycosaminoglycans (GAG), keratan sulfate, and chondroitin-6-sulfate mainly in bone, cartilage, and its extracellular matrix. GAG accumulation in these lesions leads to unique skeletal dysplasia in MPS IVA patients. Clinical, radiographic, and biochemical tests are needed to complete the diagnosis of MPS IVA since some clinical characteristics in MPS IVA are overlapped with other disorders. Early and accurate diagnosis is vital to optimizing patient management, which provides a …

Long-Term Follow-Up Posthematopoietic Stem Cell Transplantation In A Japanese Patient With Type-Vii Mucopolysaccharidosis, Kenji Orii, Yasuyuki Suzuki, Shunji Tomatsu, Tadao Orii, Toshiyuki Fukao

Department of Pediatrics Faculty Papers

The effectiveness of hematopoietic stem cell transplantation (HSCT) for type-VII mucopolysaccharidosis (MPS VII, Sly syndrome) remains controversial, although recent studies have shown that it has a clinical impact. In 1998, Yamada et al. reported the first patient with MPS VII, who underwent HSCT at 12 years of age. Here, we report the results of a 22-year follow-up of that patient post-HSCT, who harbored the p.Ala619Val mutation associated with an attenuated phenotype. The purpose of this study was to evaluate changes in physical symptoms, the activity of daily living (ADL), and the intellectual status in the 34-year-old female MPS VII patient …

Assessment Of Activity Of Daily Life In Mucopolysaccharidosis Type Ii Patients With Hematopoietic Stem Cell Transplantation., Yasuyuki Suzuki, Madeleine Taylor, Kenji Orii, Toshiyuki Fukao, Tadao Orii, Shunji Tomatsu

Department of Pediatrics Faculty Papers

The effectiveness of hematopoietic stem cell transplantation (HSCT) for mucopolysaccharidosis type II (MPS II, Hunter disease) remains controversial although recent studies have shown HSCT provides more clinical impact. This study aims to evaluate the long-term effectiveness of HSCT using the activity of daily living (ADL) scores in patients with MPS II. Sixty-nine severely affected MPS II patients (19 patients who received HSCT and 50 untreated patients) and 40 attenuated affected patients (five with HSCT and 35 untreated) were investigated by a simplified ADL questionnaire. The frequency of clinical findings and the scores of ADL (verbal, gross motor, and the level …

Quality Improvement Study Of Behavioral Health Resources Access In 19121 And 19132, Courtney Kaye, Rickie Brawer, Mph, Phd, Mches, Jeanne Felter, Phd, Lpc

Phase 1

Introduction: Children who have experienced four or more ACES (adverse childhood events) are shown to have negative mental health outcomes. Adolescents living in the Philadelphia zip codes 19121 and 19132 have higher ACE scores. This makes it necessary for adolescent providers to screen for behavioral health issues in their patients and refer out to appropriate resources.

Objective: The purpose of this quality improvement study is to see what current barriers exist relating to physician referral and access to behavioral health resources for their patients.

Methods: A guide of behavioral health resources available to adolescents in 19121 and 19132 was …

Obesity And Cardiovascular Health Differences Between Urban And Suburban Philadelphia High School Athletes, Peri Levey, Kyle Prochno, Prashant Rao, Md, David Shipon, Md, Facc, Facp, Jeremy Close, Md

Phase 1

Introduction: The strongest risk factor for primary hypertension in children is obesity, and concern for pediatric hypertension rises with the rising prevalence of obesity in American children. Specifically, in urban Philadelphia schools from 2009 to 2012, preparticipation physical evaluations (PPEs) demonstrated a positive correlation between blood pressure measurements demonstrating stage 1 or 2 hypertension and elevated body mass index (P < 0.00001).

Objective: The purpose was to identify potential differences in cardiovascular health and prevalence of obesity between adolescents in the urban and suburban Philadelphia areas.

Methods: Data from PPEs administered to urban Philadelphia high school athletes in 2018 by the Athlete …

Effect Of Sustained Attention On Early Start Denver Model Outcomes, Katherine Mcleod, Giacomo Vivanti, Phd

Phase 1

Introduction: There is very little research on the use of sustained attention as an outcome predictor for the Early Start Denver Model (ESDM). We hypothesized that a child’s level of sustained attention prior to therapy will correlate with positive ESDM outcomes.

Methods: 10 pre-preschool age children with a diagnosis of autism spectrum disorder received one year of ESDM therapy. A novel coding scheme was developed to evaluate each child’s pre-treatment level of sustained attention. Mullen Scales of Early Learning were evaluated prior to and after one year of ESDM. Pre-treatment levels of sustained attention were compared to pre-and post-treatment Mullen …

Advocating For Protective Measures Against Childhood Lead-Poisoning In Philadelphia Rental Homes, Claire Miller, Colleen Mccauley

Phase 1

Introduction: Lead exposure has been linked to delays in development and behavioral problems in children. Over 1,500 children are poisoned by lead each year in Philadelphia; the majority (62%) in rental homes.

Objective: The purpose of this project was to understand the impact of lead poisoning on children in Philadelphia, the current policies in place to protect children and the ways in which various stakeholders can advocate for policy changes that will better protect children.

Methods: A mixed-methods approach was utilized including meetings with councilmembers, stakeholder interviews and data analysis, visualization and presentation. This information was used to gain councilmembers’ …

Obesity And Cardiovascular Health Differences Between Urban And Suburban Philadelphia High School Athletes, Kyle W. Prochno, Peri Levey, Prashant Rao, Md, David M. Shipon, Md, Facc, Facp, Jeremy D. Close, Md

Phase 1

Introduction: Limited data exist regarding hypertension and obesity levels of Philadelphia-area high school student-athletes. We aim to assess their prevalence in an urban population, investigate whether they are correlated with demographic factors, and determine whether urban student-athletes differ in these outcomes compared to their suburban/non-urban counterparts.

Methods: Demographic and physical data were extracted from Athlete Health Organization (AHO) 2018 pre-participation physical exams. Adiposity classifications were made from body mass index (BMI) calculations applied to appropriate Centers for Disease Control and Prevention (CDC) growth charts, while systolic/diastolic measurements were used to classify students’ blood pressure according to current American Academy of …

Sound Dampening Headband For Infants, Alexandra A. Mccullough, Mohammad Rasool, Alison Romisher, Robert S. Pugliese, Pharmd, Bcps, Bon Ku, Md, Mpp

Phase 1

Background: Noise in the Intensive Care Nursery (ICN) has been linked to sleep disruption, vital sign destabilization, abnormal development, and stress response induction in infants. Specifically, a sound level ≥60 decibels (dB) was linked to sleep disruption in infants, and the American Academy of Pediatrics (AAP) set a maximum recommended sound level of 45dB in ICNs. The present work was conducted to confirm that the Jefferson ICN exceeds the 60dB and 45dB levels, like most hospitals do, and to conduct preliminary testing on materials for a wearable intervention to reduce infants’ exposure to noise.

Methods: A group of …

Creating A Noise-Reducing, Wearable Intervention For Newborns In The Nicu, Mohammad Rasool, Alexandra A. Mccullough, Alison Romisher, Robert S. Pugliese, Pharmd, Bcps, Bon Ku, Md, Mpp

Phase 1

Background: Excessive auditory stimulation can have negative effects on the growth and development of newborn babies. The American Academy of Pediatrics states that newborns should not be exposed to sounds in excess of 45dB while they are in the hospital, however noise levels in NICUs across the country are often between 50-100dB. A design project was conducted to develop an intervention that could reduce infant exposure to excessive noise.

Methods: Neonatologists, nurses, audiologists, music therapists, sound designers, soft materials experts, and medical device designers were interviewed and consulted throughout the design process. A 24-hour sound recording using a REED-SD-4023 meter …

Assessing The Temporality Of Adverse Effects Of Vaccines In The Nicu, Julia Tonnessen, Pedro Urday, Md

Phase 1

Introduction: Premature infants have an increased risk of contracting vaccine-preventable diseases and experience worse outcomes. Evidence supports keeping them on a regular vaccination schedule, but it is often delayed, partly because preterm infants have an increased incidence of cardiorespiratory events compared to term infants. The aim of our study was to determine if immunizations in the NICU contribute to an increased number of adverse events.

Methods: This was a retrospective, single-center, observational study with the target population infants born at less than 32 weeks gestation at Thomas Jefferson University Hospital who received the two-month vaccination series in the NICU. The …

Vaccination Exemptions For School-Aged Children In Delaware, Anna Melnick, Mph, Meghan Gannon, Phd

Phase 1

Background: Current awareness around childhood vaccinations and associated requirements for school entry has been heightened both nationally and internationally as governments work to find a balance between protecting their residents’ health and personal freedoms. Scant research exists examining the characteristics of these vaccine exemptors, but as the percentage of students seeking these exemptions increases yearly, this yields an opportunity to identify trends and inform future policy.

Question: What are the associations between reason for exemption from mandated vaccinations and the demographics of race and gender for school-aged children in the state of Delaware?

Results: Drawing from school nurse vaccine exemption …

Impact Of Smartphone Usage On Children’S Horizontal Fusional Amplitudes, Jordan Hamburger, Judith Lavrich, Md, Kammi Gunton, Md

Phase 1

Introduction: Cell phone usage has increased in pediatric patients and little research has been done on its effects on patients’ fusional vergence amplitudes. We aim to study the impact of smartphone usage on healthy pediatric patients’ fusional vergence amplitudes and report findings that may lead to future eye problems.

Methods: This is a prospective randomized study. We are currently recruiting healthy patients between the ages of 8-17 years old with no ocular problems. Data collected include refractive error, ocular alignment in the distance and at near, divergence amplitudes at near and in distance, convergence amplitudes at near and in distance, …

Effects Of Neonatal Abstinence Syndrome On Long-Term Growth Parameters, Ayoung Kim, Anastasiya Latushko, Md, Zubair H. Aghai, Md

Phase 1

Introduction: Past studies have shown that babies with Neonatal Abstinence Syndrome (NAS) have lower weight, head circumference, and height at birth. This study aims to compare their growth at 9 and 18 months of age, and to compare growth parameter of NAS infants below 10th percentile to those above 10th percentile at birth.

Methods: In this retrospective review, 260 infants admitted to Jefferson Neonatal Intensive Care Unit between 2006 and 2018 were included. The weight, height, and head circumference at birth, 9 months, and 18 months were collected and correlated by Pearson correlation. The growth parameters of infants below …

Development Of Vaccine Preferences In Parents Of Newborns, Ramon Ruiz, Neera Goyal

Phase 1

Introduction: The resurgence in cases of vaccine-preventable childhood diseases in the U.S. may be associated with increasing rates of vaccine hesitancy. Although previous research has sought to identify attributes of non-vaccinating parents, the timing in which parents make such vaccination decisions has yet to be explored. Our goal is to identify when first-time parents develop their vaccination preferences: before, after, or during pregnancy.

Methods: A cross sectional survey was conducted using a convenience sample of 73 first time parents at Thomas Jefferson University Hospital. Survey questions addressed timing of vaccination decisions and projected compliance with vaccine schedules. Eligibility for …

Comorbidities And Treatments In United States Youth With Chronic Musculoskeletal Pain, Matthew T. Taylor, Sabrina Gmuca, Alexis Boneparth, L. Nandini Moorthy, Daniel B. Horton

Phase 1

Introduction: Chronic musculoskeletal (MSK) pain has been associated with chronic illnesses and high rates of pain medication use, often in referral centers, European populations, or studies focused on single drug classes. We aimed to characterize patterns of comorbidities and treatments associated with chronic MSK pain in a nationally-representative sample of US youth.

Methods: We used the National Ambulatory Medical Care Survey (2002-2015) and National Hospital Ambulatory Medical Care Survey (2002-2011), which contain cross-sectional data for US outpatient visits. The study included all visits for youth age 8-24, excluding those with malignancy or sickle cell disease. We compared comorbidities and drugs …

The Role Of Rassf5 On Cell Growth And Hippo Signaling In Rhabdomyosarcoma, Megan Garver, Lee Helman, Md, Katrina Slemmons, Phd

Phase 1

Introduction: Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood. Dysregulation of the Hippo pathway, a signaling cascade that regulates many biological processes, is associated with many human cancers. The goal of my project was to delete RASSF5, a regulator of the Hippo pathway, in RMS cells utilizing CRISPR/Cas9 and then to evaluate how the absence of RASSF5 affects RMS cell growth and Hippo signaling, with and without DNMTi Tx.

Methods: A lipofectamine transfection was performed in two different RMS cell lines, Rh30 & RD, in which two different CRIPSR/Cas9 vectors with RASSF5 guide RNA were introduced. …