Medical Specialties Commons^™

Position Statement:Emerging Genetic Therapies For Rare Disorders, Pauline Samia, Adam Kirton, Russell Dale, Silvia Tenembaum, Chahnez Charfi Triki, Anaita Hegde, Richard Idro, Jo M. Wilmshurst, Ingrid Tein, Haluk Topaloğlu

Paediatrics and Child Health, East Africa

Emerging genetic therapies for rare disorders at high cost, cannot realistically address the global burden of disease. Stakeholders must develop new pathways to ensure safe, fair and sustainable provision of such therapies.

Initiating The Ketogenic Diet In Infants With Treatment Refractory Epilepsy While Maintaining A Breast Milk Diet, Jean-Baptist Lepichon, Lindsey M. Thompson, Megan Gustafson, Ahmed Abdelmoity

Manuscripts, Articles, Book Chapters and Other Papers

© 2019 The Authors

Purpose: The ketogenic diet has been found to be safe and effective in the treatment of drug resistant epilepsy in childhood. The age range of children undergoing this treatment has steadily been going down. There is strong evidence that it is a safe alternative in infants with drug resistant seizures. The American Academy of Pediatrics strongly supports continuing a breast milk diet until infants are at least six months of age. The purpose of this study is to evaluate the safety and efficacy of the ketogenic diet in infants while maintaining a breast milk diet.

Method: …

Biochemical And Clinical Response After Umbilical Cord Blood Transplant In A Boy With Early Childhood-Onset Beta-Mannosidosis., Troy C. Lund, Weston P. Miller, Julie B. Eisengart, Katrina Simmons, Laura Pollard, Deborah L. Renaud, David A. Wenger, Marc C. Patterson, Paul J. Orchard

Department of Neurology Faculty Papers

BACKGROUND: Deficiency in the enzyme β-mannosidase was described over three decades ago. Although rare in occurrence, the presentation of childhood-onset β-mannosidase deficiency consists of hypotonia in the newborn period followed by global development delay, behavior problems, and intellectual disability. No effective pharmacologic treatments have been available.

METHODS: We report 2-year outcomes following the first umbilical cord blood transplant in a 4-year-old boy with early childhood-onset disease.

RESULTS: We show restoration of leukocyte β-mannosidase activity which remained normal at 2 years posttransplant, and a simultaneous increase in plasma β-mannosidase activity and dramatic decrease in urine-free oligosaccharides were also observed. MRI of …

Using Physician Champions To Extend The Reach Of The Let’S Go! 5-2-1-0 Obesity Prevention Program In Clinical Practice, Michael Dedekian, Jackie Vine, Jonathan Fanburg, Naomi Anderson Schucker, Victoria Rogers

Journal of Maine Medical Center

ABSTRACT

Introduction: There is limited evidence for large scale interventions to address childhood obesity. Let’s Go! is a Maine-based obesity prevention program that deploys a consistent message across multiple community settings to encourage children and families to make healthy choices. This report describes implementation of Let's Go! in primary care offices.

Methods: Consideration for physician champion recruitment included previous involvement with Let’s Go! and ensuring statewide geographic coverage. Champions received standardized training and became local leaders in obesity prevention and treatment. 13 champions engaged 99 practices at baseline; 71 practices were engaged all 4 years (2012-2015). Data were analyzed on …

Pediatric Interfacility Transfers – Association Of Pre-Transfer Vital Signs With Length Of Stay At A Tertiary Care Center, Sarah Bunting, Leah Mallory, Logan Murray

Journal of Maine Medical Center

Objective: Determine which abnormal pre-transfer vital signs predict longer length of stay (LOS) for pediatric patients transferred to a tertiary care center.

Patients and Methods: A retrospective study of all patients transferred between Aug 2016 and Jan 2017 to Maine Medical Center’s pediatric inpatient units, PICU, and Emergency Department with pediatric consult. Charts were examined for pre-transfer vital signs and diagnosis. The primary outcome of interest was LOS. Vital signs were determined to be normal or abnormal using the 2015 PALS Guidelines.

Results: Two hundred thirty-six pediatric patients were included. Median LOS was 42.5 hours; median age was 68.0 months. …

Epilepsy Diagnosis And Management Of Children In Kenya: Review Of Current Literature, Pauline Samia, Jane Hassell, Jessica-Anne Hudson, Symon M. Kariuki, Charles R. Newton, Jo M. Wilmshurst

Paediatrics and Child Health, East Africa

Introduction: The growing impact of non-communicable diseases in low- to middle-income countries makes epilepsy a key research priority. We evaluated peer-reviewed published literature on childhood epilepsy specific to Kenya to identify knowledge gaps and inform future priorities.

Methodology: A literature search utilizing the terms “epilepsy” OR “seizure” as exploded subject headings AND “Kenya” was conducted. Relevant databases were searched, generating 908 articles. After initial screening to remove duplications, irrelevant articles, and publications older than 15 years, 154 papers remained for full-article review, which identified 35 publications containing relevant information. Data were extracted from these reports on epidemiology, etiology, clinical features, …

Evaluation Of Clinical Outcomes In An Interdisciplinary Abdominal Pain Clinic: A Retrospective, Exploratory Review., Amanda D. Deacy, Craig A. Friesen, Vincent S. Staggs, Jennifer Verrill Schurman

Manuscripts, Articles, Book Chapters and Other Papers

Background: Pediatric functional gastrointestinal disorders (FGIDs) are common and well-accepted to be etiologically complex in terms of the contribution of biological, psychological, and social factors to symptom presentations. Nonetheless, despite its documented benefits, interdisciplinary treatment, designed to address all of these factors, for pediatric FGIDs remains rare. The current study hypothesized that the majority of pediatric patients seen in an interdisciplinary abdominal pain clinic (APC) would demonstrate clinical resolution of symptoms during the study period and that specific psychosocial variables would be significantly predictive of GI symptom improvement.

Aim: To evaluate outcomes with interdisciplinary treatment in pediatric patients with pain-related …

Placental Micrornas In Pregnancies With Early Onset Intrauterine Growth Restriction And Preeclampsia: Potential Impact On Gene Expression And Pathophysiology., Zain Awamleh, Gregory B Gloor, Victor K M Han

Paediatrics Publications

BACKGROUND:
A normally developed placenta is integral to a successful pregnancy. Preeclampsia (PE) and intrauterine growth restriction (IUGR) are two common pregnancy related complications that maybe a result of abnormal placental development. Placental microRNAs (miRNAs) have been investigated as potential biomarkers for these complications, as they may play a role in placental development and pathophysiology by influencing gene expression. The purpose of this study is to utilize next-generation sequencing to determine miRNA and gene expression in human placental (chorionic villous) samples from three distinct patient groups with early-onset (EO) PE, IUGR, or PE + IUGR.

METHODS:
Placental tissues were collected …

The Stability Of Retrospective Pre-Injury Symptom Ratings Following Pediatric Concussion., Elizabeth F Teel, Roger L Zemek, Kenneth Tang, Gerard Gioia, Christopher Vaughan, Maegan Sady, Isabelle J Gagnon, Gurinder Sangha

Paediatrics Publications

Objective:
To determine the stability of children's retrospective ratings of pre-injury levels of symptoms over time following concussion.

Methods:
Children and adolescents (n = 3,063) between the ages of 5–17 diagnosed with a concussion by their treating pediatric emergency department (PED) physician within 48 h of injury completed the Post-Concussion Symptom Inventory (PCSI) at the PED and at 1, 2, 4, 8, and 12-weeks post-injury. At each time point, participants retrospectively recalled their pre-injury levels of post-injury symptoms. The PCSI has three age-appropriate versions for children aged 5–7 (PCSI-SR5), 8–12 (PCSI-SR8), and 13–18 (PCSI-SR13). Total scale, subscales (physical, cognitive, …

Association Of Early Interventions With Birth Outcomes And Child Linear Growth In Low-Income And Middle-Income Countries: Bayesian Network Meta-Analyses Of Randomized Clinical Trials, Jay J. Park H., Mei Lan Fang, Ofir Harari, Louis Dron, Ellie G. Siden, Reham Majzoub, Virginia Jeziorska, Kristian Thorlund, Edward J. Mills, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Importance:
The first 1000 days of life represent a critical window for child development. Pregnancy, exclusive breastfeeding (EBF) period (0-6 months), and complementary feeding (CF) period (6-24 months) have different growth requirements, so separate considerations for intervention strategies are needed. No synthesis to date has attempted to quantify the associations of interventions under multiple domains of micronutrient and balanced energy protein and food supplements, deworming, maternal education, water sanitation, and hygiene across these 3 life periods with birth and growth outcomes.
Objective:
To determine the magnitude of association of interventions with birth and growth outcomes based on randomized clinical trials …

Cost Effectiveness Of School-Located Influenza Vaccination Programs For Elementary And Secondary School Children., Byung-Kwang Yoo, Stanley J. Schaffer, Sharon Humiston, Cynthia M. Rand, Nicolas P N Goldstein, Christina S. Albertin, Cathleen Concannon, Peter G. Szilagyi

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Studies have noted variations in the cost-effectiveness of school-located influenza vaccination (SLIV), but little is known about how SLIV's cost-effectiveness may vary by targeted age group (e.g., elementary or secondary school students), or vaccine consent process (paper-based or web-based). Further, SLIV's cost-effectiveness may be impacted by its spillover effect on practice-based vaccination; prior studies have not addressed this issue.

METHODS: We performed a cost-effectiveness analysis on two SLIV programs in upstate New York in 2015-2016: (a) elementary school SLIV using a stepped wedge design with schools as clusters (24 suburban and 18 urban schools) and (b) secondary school SLIV …

Armed Conflicts And National Trends In Reproductive, Maternal, Newborn And Child Health In Sub-Saharan Africa: What Can National Health Surveys Tell Us?, Ties Boerma, Hannah Tappis, Ghada Saad-Haddad, Jai K. Das, Dessalegn Y. Melesse, Jocelyn Dejong, Paul Spiegel, Robert Black, Cesar Victora, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Armed conflicts are widespread in sub-Saharan Africa and considered to be an important factor in slowing down national progress in reproductive, maternal, newborn and child health (RMNCH). The measurement of the impact of conflicts on national levels and trends in RMNCH is difficult. National surveys conducted before and sometimes during and after conflicts are a major source of information on the national and local effects of conflicts on RMNCH. We examined data from national surveys in 13 countries in sub-Saharan Africa with major conflicts during 1990–2016 to assess the levels and trends in RMNCH intervention coverage, nutritional status and mortality …

Advancing Measurement And Monitoring Of Reproductive, Maternal, Newborn And Child Health And Nutrition: Global And Country Perspectives, Tanya Marchant, Zulfiqar Ahmed Bhutta, Robert Black, John Grove, Catherine Kyobutungi, Stefan Peterson

Department of Paediatrics and Child Health

No abstract provided.

Postoperative Pain: Factors And Tools To Improve Pain Management In Children, Mai M. Makhlouf, Eric Robles Garibay, Brooke N. Jenkins, Zeev N. Kain, Michelle Fortier

Psychology Faculty Articles and Research

Outpatient surgery has made it increasingly common for parents to manage pain in the home setting. Studies have shown that parents often under treat pain, leaving children vulnerable to the negative side effects of suboptimal pain management. Multiple factors affect pain management like child’s age and developmental stage, language, cultural values like stoicism, parental beliefs about medication, biological differences among groups, etc. Understanding all the factors involved can help healthcare providers and parents better understand pain and contribute to optimal pain management. Multiple tools and technological interventions have been created to help create a better understanding of pain and a …

Nash-Wo-Numa (Childhood Growth & Development) Study Protocol: Factors That Impact Linear Growth In Children 9 To 15 Years Of Age In Matiari, Pakistan, Susan C. Campisi, Yaqub Wasan, Sajid Bashir Soofi, Suneeta Monga, Daphne J. Korczak, Wendy Lou, Olle Soder, Ashley Vandermorris, Khadija N Humayun, Ayesha Mian, Peter Szatmari, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Introduction: Adolescence is a time of significant physical and emotional change, and there is emerging concern that adolescents living in low- and middle-income countries (LMIC) may face substantial challenges in relation to linear growth and mental health. Data on the global burden of stunting after 5 years of age are limited, but estimates suggest up to 50 per cent of all adolescents in some LMIC are stunted. Additionally, many LMIC lack robust mental health care delivery systems. Pakistan has one of the world's largest populations of adolescents (10 to 19 years) at approximately 40 million. The Nash-wo-Numa study's primary objective …

A National Survey Of Resources To Address Sepsis In Children In Tertiary Care Centers In Nigeria., Odiraa C Nwankwor, Brianna Mckelvie, Meg Frizzola, Krystal Hunter, Halima S Kabara, Abiola Oduwole, Tagbo Oguonu, Niranjan Kissoon

Paediatrics Publications

Background:
Infections leading to sepsis are major contributors to mortality and morbidity in children world-wide. Determining the capacity of pediatric hospitals in Nigeria to manage sepsis establishes an important baseline for quality-improvement interventions and resource allocations.

Objectives:
To assess the availability and functionality of resources and manpower for early detection and prompt management of sepsis in children at tertiary pediatric centers in Nigeria.

Methods:
This was an online survey of tertiary pediatric hospitals in Nigeria using a modified survey tool designed by the World Federation of Pediatric Intensive and Critical Care Societies (WFPICCS). The survey addressed all aspects of pediatric …

The Impact Of Pediatric Emergency Department Crowding On Patient And Health Care System Outcomes: A Multicentre Cohort Study., Quynh Doan, Hubert Wong, Garth Meckler, David Johnson, Antonia Stang, Andrew Dixon, Scott Sawyer, Tania Principi, April J Kam, Gary Joubert, Jocelyn Gravel, Mona Jabbour, Astrid Guttmann

Paediatrics Publications

BACKGROUND:
Emergency department overcrowding has been associated with increased odds of hospital admission and mortality after discharge from the emergency department in predominantly adult cohorts. The objective of this study was to evaluate the association between crowding and the odds of several adverse outcomes among children seen at a pediatric emergency department.

METHODS:
We conducted a retrospective cohort study involving all children visiting 8 Canadian pediatric emergency departments across 4 provinces between 2010 and 2014. We analyzed the association between mean departmental length of stay for each index visit and hospital admission within 7 days or death within 14 days …

Exploring Parental Wishes And Personhood In The Grey Zones Of Neonatal Resuscitation, Alison Lindsay

Honors Projects

The intense societal debate churning around the moral status of fetuses includes topics such as qualifications for personhood, the role of the autonomous decisions of a fetus’ mother, and the obligations of society to protect fetuses. This paper analyzes extending this discussion to newborns in five sections. The first section presents a literature review of responses to a philosophical paper about the respective interests of parents and fetuses and newborns, elaborating on aspects of personhood and parental decision-making. The second section presents a literature review of medical and nursing discussion around resuscitation for extremely premature newborns, focusing on similar evaluations …

Using Entrustable Professional Activities To Improve Pediatric Residency Evaluations, Kelli N. Harajda Md, Kris Rooney Md

Department of Pediatrics

No abstract provided.

Quality Improvement: Enhancing Provider Screening Of Adolescent Substance Use With Crafft Questionnaire, Julia C. Durante Md, Emily Pollack Md, Richard J. Mazzaccaro Md, Phd, Rosa Gomez-Dejesus Md

Department of Pediatrics

No abstract provided.

Twelve Tips For Enhancing Student Engagement., Harm Peters, Marko Zdravkovic, Manuel João Costa, Antonio Celenza, Kulsoom Ghias, Debra Klamen, Liz Mossop, Michael Rieder, Vishna Devi Nadarajah, Danai Wangsaturaka, Martin Wohlin, Margot Weggemans

Paediatrics Publications

Student engagement refers to a broad range of activities where students participate in management, education, research, and community activities within their institutions. It is a mutually beneficial collaborative approach between students and their institutions. This article provides practical advice for the implementation or further development of student engagement at medical, dental, and veterinary schools. The tips provided are based on the experiences of a group of universities recently recognized for best practice in student engagement, and are supported by evidence from the literature. The tips cover overarching themes which include the creation of an institutional culture and formal framework for …

External Cold And Vibration For Pain Management Of Children Undergoing Needle-Related Procedures In The Emergency Department: A Randomised Controlled Non-Inferiority Trial Protocol., Ariane Ballard, Christelle Khadra, Samara Adler, Evelyne D Trottier, Benoit Bailey, Naveen Poonai, Jean Théroux, Sylvie Le May

Paediatrics Publications

INTRODUCTION: Needle-related procedures are considered as the most important source of pain and distress in children in hospital settings. Considering the physiological and psychological consequences that could result from these procedures, management of pain and distress through pharmacological and non-pharmacological methods is essential. Therefore, it is important to have interventions that are rapid, easy-to-use and likely to be translated into clinical practice for routine use. The aim of this study will be to determine whether a device combining cold and vibration (Buzzy) is non-inferior to a topical anaesthetic (liposomal lidocaine 4% cream) for pain management of children undergoing needle-related procedures …

Unanticipated Admissions To Paediatric Cardiac Critical Care After Cardiac Catheterisations., Erin Peebles, Michael R Miller, Lee N Benson, Tilman Humpl

Paediatrics Publications

OBJECTIVES: Cardiac catheterisation is commonly used for diagnosis and therapeutic interventions in paediatric cardiology. The inherent risk of the procedure can result in unanticipated admissions to critical care. Our goals were to provide a qualitative description of characteristics and evaluation of children admitted unexpectedly to the cardiac critical care unit (CCCU).

METHODS: A retrospective single centre review of cardiac catheterisation procedures was done between 1 January, 2003 and 30 April, 2013.

RESULTS: Of 9336 cardiac catheterisations performed, 146 (1.6%) were admitted from the catheterisation laboratory to the CCCU and met inclusion criteria. Of these 146 patients, 117 (1.3%) met criteria …

Use Of A Radial Artery ‘Slender’ Sheath For Facilitating Transfemoral Arterial Access For Neuroendovascular Embolization In A Very Young Infant, Timothy L. Miao, Enriqueta Lucar Figueroa, Khalid Bajunaid, Michael Mayich, Sandrine De Ribaupierre, Sachin K. Pandey

Paediatrics Publications

Pediatric neuroendovascular procedures are increasingly performed as several studies have shown the safety of these interventions. In the literature, an upper limit of 4 French catheter size is conventionally used for femoral access in neuroangiography of very young infants. However, this constraint in luminal size may not allow for more complex procedures. We present the previously unreported use of a radial 5 French slender catheter with ultrathin walls for femoral access for aneurysm embolization in a 3-month-old boy presenting with left M2 aneurysm rupture.

Promoting Adherence To Influenza Vaccination Recommendations In Pediatric Practice., Lloyd N. Werk, Maria Carmen Diaz, Adriana Cadilla, James P. Franciosi, Jobayer Hossain

Jefferson Health - Northeast Papers (formerly Aria Health Papers)

OBJECTIVES: In the United States, nonadherence to seasonal influenza vaccination guidelines for children and adolescents is common and results in unnecessary morbidity and mortality. We conducted a quality improvement project to improve vaccination rates and test effects of 2 interventions on vaccination guidelines adherence.

METHODS: We conducted a cluster randomized control trial with 11 primary care practices (PRACTICE) that provided care for 11 293 individual children and adolescents in a children's health care system from September 2015 through April 2016. Practice sites (with their clinicians) were randomly assigned to 4 arms (no intervention [Control], computerized clinical decision support system [CCDSS], …

Quantitative Sensory Testing In Children With Sickle Cell Disease: Additional Insights And Future Possibilities., Robin E. Miller, Dawn S. Brown, Scott W. Keith, Sarah E. Hegarty, Yamaja Setty, Claudia M. Campbell, Suzanne M. Mccahan, Suhita Gayen-Betal, Hal Byck, Marie Stuart

Department of Pharmacology and Experimental Therapeutics Faculty Papers

Quantitative sensory testing (QST) is used in a variety of pain disorders to characterize pain and predict prognosis and response to specific therapies. In this study, we aimed to confirm results in the literature documenting altered QST thresholds in sickle cell disease (SCD) and assess the test-retest reliability of results over time. Fifty-seven SCD and 60 control subjects aged 8-20 years underwent heat and cold detection and pain threshold testing using a Medoc TSAII. Participants were tested at baseline and 3 months; SCD subjects were additionally tested at 6 months. An important facet of our study was the development and …

Association Of Nutritional Status Measures With Self-Efficacy And Experiencing Depressed Mood Among Pakistani Young Women (P10-090-19), Jo-Anna Baxter, Yaqub Wasan, Sajid Bashir Soofi, Imran Ahmed, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Objectives:
We aimed to assess self-efficacy and the experience of depression-related emotions among non-pregnant adolescent and young women (15–23 years) living in rural Pakistan, and determine their association with measures of nutritional status.
Methods:
Outcomes were assessed from the Matiari emPowerment and Preconception Supplementation (MaPPS) Trial baseline data (n = 25,279). Self-efficacy and depression-related emotions were determined and categorized using the general self-efficacy scale (low, moderate, and high) and DASS-21 tool (normal, mild, moderate, severe, and extremely severe), respectively. Nutritional status was evaluated using hemoglobin concentration (HemoCue Hb 301 System) and body mass index (BMI). Associations were assessed using ordinal …

Effect Of Lipid-Based Nutrient Supplement - Medium Quantity On Reduction Of Stunting In Children 6-23 Months Of Age In Sindh, Pakistan: A Cluster Randomized Controlled Trial (Or25-06-19), Sajid Bashir Soofi, Gul Nawaz Khan Nawaz Khan, Cecilia Garzon

Department of Paediatrics and Child Health

Objectives:
We evaluated the effectiveness of a locally produced lipid-based nutrient supplement - medium-quantity (LNS-MQ) known as Wawamum to prevent stunting in children 6–23 months of age in Thatta and Sujawal districts of Sindh province, Pakistan. Methods: A cluster randomized controlled trial was conducted and a total of 870 children between 6–18 months old were enrolled. Children in the intervention group received 50 grams/day of Wawamum for 6 months, while children in the control group received standard government health services, not including Wawamum. The primary outcome was stunting risk reduction among children 6–23 months of age.
Results:
Children who received …

Clinical Screening For Congenital Heart Disease In Newborns At A Tertiary Care Hospital Of A Developing Country, Muhammad Mohsin, Khadija N Humayun, Mehnaz Atiq

Department of Paediatrics and Child Health

Objective:
To screen all newborns admitted to a tertiary care hospital to rule out congenital heart disease before discharge and to find out the utility of pulse oximetry to detect congenital heart disease.
Methodology:
This prospective study was done at Aga Khan University Hospital from January 2014 to December 2014 in 1,650 newborns over a period of 12 months. Pulse oximetry and clinical examination were done. Persistent oxygen saturation less than 95% was considered as positive pulse oximetry. Newborns who had positive pulse oximetry or abnormal clinical examinations findings were subjected to echocardiography.
Result:
Pulse oximetry was performed on 1,650 …

Recommendations For The Design Of Therapeutic Trials For Neonatal Seizures., Janet S. Soul, Ronit Pressler, Marilee Allen, Geraldine Boylan, Heike Rabe, Ron Portman, Pollyanna Hardy, Sarah Zohar, Klaus Romero, Brian Tseng, Varsha Bhatt-Mehta, Cecil Hahn, Scott Denne, Stephane Auvin, Alexander Vinks, John Lantos, Neil Marlow, Jonathan M. Davis, International Neonatal Consortium

Manuscripts, Articles, Book Chapters and Other Papers

Although seizures have a higher incidence in neonates than any other age group and are associated with significant mortality and neurodevelopmental disability, treatment is largely guided by physician preference and tradition, due to a lack of data from well-designed clinical trials. There is increasing interest in conducting trials of novel drugs to treat neonatal seizures, but the unique characteristics of this disorder and patient population require special consideration with regard to trial design. The Critical Path Institute formed a global working group of experts and key stakeholders from academia, the pharmaceutical industry, regulatory agencies, neonatal nurse associations, and patient advocacy …