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Full-Text Articles in Medicine and Health Sciences

Using Exterior Housing Conditions To Predict Elevated Pediatric Blood Lead Levels., Neal J Wilson, Elizabeth Friedman, Kevin Kennedy, Panayiotis T. Manolakos, Lori Reierson, Amy Roberts, Steve Simon Feb 2023

Using Exterior Housing Conditions To Predict Elevated Pediatric Blood Lead Levels., Neal J Wilson, Elizabeth Friedman, Kevin Kennedy, Panayiotis T. Manolakos, Lori Reierson, Amy Roberts, Steve Simon

Manuscripts, Articles, Book Chapters and Other Papers

Housing-based lead paint dust is the most common source of lead exposure for US-born children. Although year of housing construction is a critical indicator of the lead hazard to US children, not all housing of the same age poses the same risk to children. Additional information about housing condition is required to differentiate the housing-based lead risk at the parcel level. This study aimed to identify and assess a method for gathering and using observations of exterior housing conditions to identify active housing-based lead hazards at the parcel level. We used a dataset of pediatric blood lead observations (sample years …


Nutritional Management Of The Infant With Chronic Kidney Disease Stages 2-5 And On Dialysis., Vanessa Shaw, Caroline Anderson, An Desloovere, Larry A. Greenbaum, Dieter Haffner, Christina L. Nelms, Fabio Paglialonga, Nonnie Polderman, Leila Qizalbash, José Renken-Terhaerdt, Stella Stabouli, Jetta Tuokkola, Johan Vande Walle, Bradley A. Warady, Rukshana Shroff Jan 2023

Nutritional Management Of The Infant With Chronic Kidney Disease Stages 2-5 And On Dialysis., Vanessa Shaw, Caroline Anderson, An Desloovere, Larry A. Greenbaum, Dieter Haffner, Christina L. Nelms, Fabio Paglialonga, Nonnie Polderman, Leila Qizalbash, José Renken-Terhaerdt, Stella Stabouli, Jetta Tuokkola, Johan Vande Walle, Bradley A. Warady, Rukshana Shroff

Manuscripts, Articles, Book Chapters and Other Papers

The nutritional management of children with chronic kidney disease (CKD) is of prime importance in meeting the challenge of maintaining normal growth and development in this population. The objective of this review is to integrate the Pediatric Renal Nutrition Taskforce clinical practice recommendations for children with CKD stages 2-5 and on dialysis, as they relate to the infant from full term birth up to 1 year of age, for healthcare professionals, including dietitians, physicians, and nurses. It addresses nutritional assessment, energy and protein requirements, delivery of the nutritional prescription, and necessary dietary modifications in the case of abnormal serum levels …


Registry-Based Cohort Study Of Alpha-1 Antitrypsin Deficiency Prevalence, Incidence And Mortality In Denmark 2000-2018, John Acquavella, Emese Vágó, Henrik Toft Sorensen, Erzsébet Horváth-Puhó, Gregory P. Hess Dec 2022

Registry-Based Cohort Study Of Alpha-1 Antitrypsin Deficiency Prevalence, Incidence And Mortality In Denmark 2000-2018, John Acquavella, Emese Vágó, Henrik Toft Sorensen, Erzsébet Horváth-Puhó, Gregory P. Hess

College of Population Health Faculty Papers

OBJECTIVE: To estimate the prevalence of diagnosed alpha-1 antitrypsin deficiency (dAATD) in Denmark as of 31 December 2018, and dAATD incidence and mortality from 1 January 2000 to 31 December 2018.

STUDY DESIGN AND SETTING: We used the Danish National Patient Registry to identify patients with dAATD based on the International Classification of Diseases, 10th Revision (ICD-10) code E88.0A and the Danish Civil Registration System (CRS) for population counts and vital status. We estimated dAATD prevalence, incidence and mortality. We compared mortality among patients with dAATD and an age-matched and sex-matched cohort extracted from the Danish CRS. We conducted a …


The Mentored Experience To Enhance Opportunities In Research (Meteor) Program., Lisa Schwartz, Naomi Luban, Alison Hall, Diane Mcquail, Yolanda Haywood Dec 2022

The Mentored Experience To Enhance Opportunities In Research (Meteor) Program., Lisa Schwartz, Naomi Luban, Alison Hall, Diane Mcquail, Yolanda Haywood

Health Sciences Education Research

PROBLEM: Medical students from groups that are underrepresented in medicine are less likely to pursue careers that incorporate research as compared to their white peers. Clinical and Translational Science Award (CTSA)-funded institutions encouraged centers to establish short-term, mentored summer research opportunities to motivate students underrepresented in medicine to enroll in medical school and ideally choose a career that incorporates research into their clinical practice.

APPROACH: The Mentored Experience To Enhance Opportunities in Research (METEOR) Program was established in 2012 in partnership with the Clinical and Translational Science Institute at Children's National (CTSI-CN) and The George Washington University (GW) School of …


Wellness And Coping Of Physicians Who Worked In Icus During The Pandemic: A Multicenter Cross-Sectional North American Survey, Karen E. A. Burns, Rachel K. Mcdonald, Et Al. Dec 2022

Wellness And Coping Of Physicians Who Worked In Icus During The Pandemic: A Multicenter Cross-Sectional North American Survey, Karen E. A. Burns, Rachel K. Mcdonald, Et Al.

2020-Current year OA Pubs

OBJECTIVES: Few surveys have focused on physician moral distress, burnout, and professional fulfilment. We assessed physician wellness and coping during the COVID-19 pandemic.

DESIGN: Cross-sectional survey using four validated instruments.

SETTING: Sixty-two sites in Canada and the United States.

SUBJECTS: Attending physicians (adult, pediatric; intensivist, nonintensivist) who worked in North American ICUs.

INTERVENTION: None.

MEASUREMENTS AND MAIN RESULTS: We analysed 431 questionnaires (43.3% response rate) from 25 states and eight provinces. Respondents were predominantly male (229 [55.6%]) and in practice for 11.8 ± 9.8 years. Compared with prepandemic, respondents reported significant intrapandemic increases in days worked/mo, ICU bed occupancy, and …


Follow-Up Household Assessment For Child Unintentional Injuries Two Years After The Intervention: A Community-Based Study From Karachi, Pakistan, Uzma Rahim Khan, Asrar Ali, Umerdad Khudadad, Ahmed Raheem Buksh, Nukhba Zia, Iqbal Azam Syed, Aruna Chandran, Junaid Abdul Razzak, Adnan Ali Hyder Nov 2022

Follow-Up Household Assessment For Child Unintentional Injuries Two Years After The Intervention: A Community-Based Study From Karachi, Pakistan, Uzma Rahim Khan, Asrar Ali, Umerdad Khudadad, Ahmed Raheem Buksh, Nukhba Zia, Iqbal Azam Syed, Aruna Chandran, Junaid Abdul Razzak, Adnan Ali Hyder

Department of Emergency Medicine

Background: Unintentional childhood injuries are a growing public health concern, and the home is the most common location for non-fatal injuries in children less than 5 years of age. This study describes the long-term effects of two injury prevention educational interventions for caregivers-an educational pamphlet and an in-home tutorial guide-by comparing the change in the prevalence of home injury hazards before and after the interventions.
Methods: This was a pre- (June and July 2010) and post-study with short-term follow-up (November-December 2010) and long-term follow-up (November 2012- January 2013). Neighborhood one included households that received only educational pamphlets after completing a …


Tonsillectomy In Children With 22q11.2 Deletion Syndrome., Jill M. Arganbright, Paul Bryan Hankey, Meghan Tracy, Srivats Narayanan, Janelle R. Noel-Macdonnell Phd, David G. Ingram Md Nov 2022

Tonsillectomy In Children With 22q11.2 Deletion Syndrome., Jill M. Arganbright, Paul Bryan Hankey, Meghan Tracy, Srivats Narayanan, Janelle R. Noel-Macdonnell Phd, David G. Ingram Md

Manuscripts, Articles, Book Chapters and Other Papers

Tonsillectomy is one of the most common procedures performed in children, however there are currently no published studies evaluating tonsillectomy in children with 22q11.2 deletion syndrome (22q11DS). With this study, our goal was to investigate the indications, efficacy, and complications of tonsillectomy in a pediatric cohort of patients with 22q11DS. This is a retrospective chart review of patients in our 22q Center's repository. Inclusion criteria were a diagnosis of 22q11DS and a history of tonsillectomy or adenotonsillectomy. Data collected included: indications for tonsillectomy, preoperative and postoperative polysomnography (PSG) results, and surgical complications. In total, 33 patients were included. Most common …


Language Development And Disorders, Sudhin Karuppali Dr. Nov 2022

Language Development And Disorders, Sudhin Karuppali Dr.

Health collection

The field of language development and its disorders is a widely researched area in the field of speech-language pathology. However, language development in the Indian population is vaguely studied, probably because of the diversity of languages and cultures in this country. This has time and again resulted in difficulties in estimating the language levels of children with language impairments, consequently becoming challenging to prepare customized interventional plans. The lack of standardized language assessment tools has always been a concern in clinical practice. This lacuna in establishing accurate language levels has been observed in the adolescent population as well, which is …


A Review Of Ferric Citrate Clinical Studies, And The Rationale And Design Of The Ferric Citrate And Chronic Kidney Disease In Children (Fit4kid) Trial., Mark R. Hanudel, Marciana L. Laster, Anthony A. Portale, Aditi Dokras, Raymond P. Quigley, German A Lozano Guzman, Joshua J. Zaritsky, Nicole A. Hayde, Frederick J. Kaskel, Mark M. Mitsnefes, Jorge A. Ramirez, Peace D. Imani, Poyyapakkam R. Srivaths, Amy J. Kogon, Michelle R. Denburg, Tom D. Blydt-Hansen, Loretta Z. Reyes, Larry A. Greenbaum, Darcy K. Weidemann, Bradley A. Warady, David A. Elashoff, Susan R. Mendley, Tamara Isakova, Isidro B. Salusky Nov 2022

A Review Of Ferric Citrate Clinical Studies, And The Rationale And Design Of The Ferric Citrate And Chronic Kidney Disease In Children (Fit4kid) Trial., Mark R. Hanudel, Marciana L. Laster, Anthony A. Portale, Aditi Dokras, Raymond P. Quigley, German A Lozano Guzman, Joshua J. Zaritsky, Nicole A. Hayde, Frederick J. Kaskel, Mark M. Mitsnefes, Jorge A. Ramirez, Peace D. Imani, Poyyapakkam R. Srivaths, Amy J. Kogon, Michelle R. Denburg, Tom D. Blydt-Hansen, Loretta Z. Reyes, Larry A. Greenbaum, Darcy K. Weidemann, Bradley A. Warady, David A. Elashoff, Susan R. Mendley, Tamara Isakova, Isidro B. Salusky

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric chronic kidney disease (CKD) is characterized by many co-morbidities, including impaired growth and development, CKD-mineral and bone disorder, anemia, dysregulated iron metabolism, and cardiovascular disease. In pediatric CKD cohorts, higher circulating concentrations of fibroblast growth factor 23 (FGF23) are associated with some of these adverse clinical outcomes, including CKD progression and left ventricular hypertrophy. It is hypothesized that lowering FGF23 levels will reduce the risk of these events and improve clinical outcomes. Reducing FGF23 levels in CKD may be accomplished by targeting two key stimuli of FGF23 production-dietary phosphate absorption and iron deficiency. Ferric citrate is approved for use …


Active Vs Traditional Methods Of Recruiting Children For A Clinical Trial In Rural Primary Care Clinics: A Cluster-Randomized Clinical Trial, Paul M Darden, Ann M Davis, Jeannette Y Lee, Milan Bimali, Alan E Simon, Andrew M Atz, Crystal S Lim, Thao-Ly Phan, James R Roberts, Russell J Mcculloh, Lee Pyles, Michelle Shaffer, Jessica N Snowden Nov 2022

Active Vs Traditional Methods Of Recruiting Children For A Clinical Trial In Rural Primary Care Clinics: A Cluster-Randomized Clinical Trial, Paul M Darden, Ann M Davis, Jeannette Y Lee, Milan Bimali, Alan E Simon, Andrew M Atz, Crystal S Lim, Thao-Ly Phan, James R Roberts, Russell J Mcculloh, Lee Pyles, Michelle Shaffer, Jessica N Snowden

Department of Pediatrics Faculty Papers

Importance: To our knowledge, there are no published randomized clinical trials of recruitment strategies. Rigorously evaluated successful recruitment strategies for children are needed.

Objective: To evaluate the feasibility of 2 recruitment methods for enrolling rural children through primary care clinics to assess whether either or both methods are sufficiently effective for enrolling participants into a clinical trial of a behavioral telehealth intervention for children with overweight or obesity.

Design, setting, and participants: This cluster-randomized clinical trial of 2 recruitment methods was conducted at 4 primary care clinics in 4 separate states. Each clinic used both recruitment methods in random order. …


Real-World Evidence Supporting Tandem Control-Iq Hybrid Closed-Loop Success In The Medicare And Medicaid Type 1 And Type 2 Diabetes Populations, Gregory P Forlenza, Anders L Carlson, Rodolfo J Galindo, Davida F Kruger, Carol J Levy, Janet B Mcgill, Guillermo Umpierrez, Grazia Aleppo Nov 2022

Real-World Evidence Supporting Tandem Control-Iq Hybrid Closed-Loop Success In The Medicare And Medicaid Type 1 And Type 2 Diabetes Populations, Gregory P Forlenza, Anders L Carlson, Rodolfo J Galindo, Davida F Kruger, Carol J Levy, Janet B Mcgill, Guillermo Umpierrez, Grazia Aleppo

2020-Current year OA Pubs

No abstract provided.


Genetic Testing To Inform Epilepsy Treatment Management From An International Study Of Clinical Practice, Dianalee Mcknight, Ana Morales, Kathryn E. Hatchell, Sara L. Bristow, Joshua L. Bonkowsky, Michael Scott Perry, Anne T. Berg, Felippe Borlot, Edward D. Esplin, Chad Moretz, Katie Angione, Loreto Ríos-Pohl, Robert L. Nussbaum, Swaroop Aradhya, Chad R. Haldeman-Englert, Rebecca J. Levy, Venu G. Parachuri, Guillermo Lay-Son, David J. Dávila-Ortiz De Montellano, Miguel Angel Ramirez-Garcia, Edmar O. Benítez Alonso, Julie Ziobro, Adela Chirita-Emandi, Temis M. Felix, Dianne Kulasa-Luke, Andre Megarbane, Shefali Karkare, Sarah L. Chagnon, Jennifer B. Humberson, Melissa J. Assaf, Sebastian Silva, Katherine Zarroli, Oksana Boyarchuk, Gary R. Nelson, Rachel Palmquist, Katherine C. Hammond, Sean T. Hwang, Susan B. Boutlier, Melinda Nolan, Kaitlin Y. Batley, Devraj Chavda, Carlos Alberto Reyes-Silva, Oleksandr Miroshnikov, Britton Zuccarelli, Louise Amlie-Wolf, James W. Wheless, Syndi Seinfeld, Manoj Kanhangad, Jeremy L. Freeman, Susana Monroy-Santoyo, Natalia Rodriguez-Vazquez, Monique M. Ryan, Michelle Machie, Patricio Guerra, Muhammad Jawad Hassan, Meghan S. Candee, Caleb P. Bupp, Kristen L. Park, Eric Muller, Pamela Lupo, Robert C. Pedersen, Amir M. Arain, Andrea Murphy, Krista Schatz, Weiyi Mu, Paige M. Kalika, Lautaro Plaza, Marissa A. Kellogg, Evelyn G. Lora, Robert P. Carson, Victoria Svystilnyk, Viviana Venegas, Rebecca R. Luke, Huiyuan Jiang, Tetiana Stetsenko, Milagros M. Dueñas-Roque, Joseph Trasmonte, Rebecca J. Burke, Anna C. E. Hurst, Douglas M. Smith, Lauren J. Massingham, Laura Pisani, Carrie E. Costin, Betsy Ostrander, Francis M. Filloux, Amitha L. Ananth, Ismail S. Mohamed, Alla Nechai, Jasmin M. Dao, Michael C. Fahey, Ermal Aliu, Stephen Falchek, Craig A. Press, Lauren Treat, Krista Eschbach, Angela Starks, Ryan Kammeyer, Joshua J. Bear, Mona Jacobson, Veronika Chernuha, Bailey Meibos, Kristen Wong, Matthew T. Sweney, A. Chris Espinoza, Colin B. Van Orman, Arie Weinstock, Ashutosh Kumar, Claudia Soler-Alfonso, Danielle A. Nolan, Muhammad Raza, Miguel David Rojas Carrion, Geetha Chari, Eric D. Marsh, Yael Shiloh-Malawsky, Sumit Parikh, Ernesto Gonzalez-Giraldo, Stephen Fulton, Yoshimi Sogawa, Kaitlyn Burns, Myroslava Malets, Johnny David Montiel Blanco, Christa W. Habela, Carey A. Wilson, Guillermo G. Guzmán, Mariia Pavliuk Oct 2022

Genetic Testing To Inform Epilepsy Treatment Management From An International Study Of Clinical Practice, Dianalee Mcknight, Ana Morales, Kathryn E. Hatchell, Sara L. Bristow, Joshua L. Bonkowsky, Michael Scott Perry, Anne T. Berg, Felippe Borlot, Edward D. Esplin, Chad Moretz, Katie Angione, Loreto Ríos-Pohl, Robert L. Nussbaum, Swaroop Aradhya, Chad R. Haldeman-Englert, Rebecca J. Levy, Venu G. Parachuri, Guillermo Lay-Son, David J. Dávila-Ortiz De Montellano, Miguel Angel Ramirez-Garcia, Edmar O. Benítez Alonso, Julie Ziobro, Adela Chirita-Emandi, Temis M. Felix, Dianne Kulasa-Luke, Andre Megarbane, Shefali Karkare, Sarah L. Chagnon, Jennifer B. Humberson, Melissa J. Assaf, Sebastian Silva, Katherine Zarroli, Oksana Boyarchuk, Gary R. Nelson, Rachel Palmquist, Katherine C. Hammond, Sean T. Hwang, Susan B. Boutlier, Melinda Nolan, Kaitlin Y. Batley, Devraj Chavda, Carlos Alberto Reyes-Silva, Oleksandr Miroshnikov, Britton Zuccarelli, Louise Amlie-Wolf, James W. Wheless, Syndi Seinfeld, Manoj Kanhangad, Jeremy L. Freeman, Susana Monroy-Santoyo, Natalia Rodriguez-Vazquez, Monique M. Ryan, Michelle Machie, Patricio Guerra, Muhammad Jawad Hassan, Meghan S. Candee, Caleb P. Bupp, Kristen L. Park, Eric Muller, Pamela Lupo, Robert C. Pedersen, Amir M. Arain, Andrea Murphy, Krista Schatz, Weiyi Mu, Paige M. Kalika, Lautaro Plaza, Marissa A. Kellogg, Evelyn G. Lora, Robert P. Carson, Victoria Svystilnyk, Viviana Venegas, Rebecca R. Luke, Huiyuan Jiang, Tetiana Stetsenko, Milagros M. Dueñas-Roque, Joseph Trasmonte, Rebecca J. Burke, Anna C. E. Hurst, Douglas M. Smith, Lauren J. Massingham, Laura Pisani, Carrie E. Costin, Betsy Ostrander, Francis M. Filloux, Amitha L. Ananth, Ismail S. Mohamed, Alla Nechai, Jasmin M. Dao, Michael C. Fahey, Ermal Aliu, Stephen Falchek, Craig A. Press, Lauren Treat, Krista Eschbach, Angela Starks, Ryan Kammeyer, Joshua J. Bear, Mona Jacobson, Veronika Chernuha, Bailey Meibos, Kristen Wong, Matthew T. Sweney, A. Chris Espinoza, Colin B. Van Orman, Arie Weinstock, Ashutosh Kumar, Claudia Soler-Alfonso, Danielle A. Nolan, Muhammad Raza, Miguel David Rojas Carrion, Geetha Chari, Eric D. Marsh, Yael Shiloh-Malawsky, Sumit Parikh, Ernesto Gonzalez-Giraldo, Stephen Fulton, Yoshimi Sogawa, Kaitlyn Burns, Myroslava Malets, Johnny David Montiel Blanco, Christa W. Habela, Carey A. Wilson, Guillermo G. Guzmán, Mariia Pavliuk

Department of Pediatrics Faculty Papers

IMPORTANCE: It is currently unknown how often and in which ways a genetic diagnosis given to a patient with epilepsy is associated with clinical management and outcomes.

OBJECTIVE: To evaluate how genetic diagnoses in patients with epilepsy are associated with clinical management and outcomes.

DESIGN, SETTING, AND PARTICIPANTS: This was a retrospective cross-sectional study of patients referred for multigene panel testing between March 18, 2016, and August 3, 2020, with outcomes reported between May and November 2020. The study setting included a commercial genetic testing laboratory and multicenter clinical practices. Patients with epilepsy, regardless of sociodemographic features, who received a …


Enteric Pathogen Testing Importance For Children With Acute Gastroenteritis: A Modified Delphi Study, Gillian A M Tarr, Drew J Persson, Phillip I Tarr, Stephen B Freedman Oct 2022

Enteric Pathogen Testing Importance For Children With Acute Gastroenteritis: A Modified Delphi Study, Gillian A M Tarr, Drew J Persson, Phillip I Tarr, Stephen B Freedman

2020-Current year OA Pubs

The application of clinical diagnostics for gastroenteritis in children has implications for a broad collection of stakeholders, impacting clinical care, communicable disease control, and laboratory utilization. To support diagnostic stewardship as gastroenteritis testing options continue to advance, it is critical to understand which enteropathogens constitute priorities for testing across stakeholder groups. Using a modified Delphi technique, we elicited opinions of subject matter experts to determine clinical and public health testing priorities. There was a high level of overall agreement (≥80%) among stakeholders (final round


Genetic Conditions Of Short Stature: A Review Of Three Classic Examples, Merlin G Butler, Bradley S Miller, Alicia Romano, Judith L. Ross, M Jennifer Abuzzahab, Philippe Backeljauw, Vaneeta Bamba, Amrit Bhangoo, Nelly Mauras, Mitchell Geffner Oct 2022

Genetic Conditions Of Short Stature: A Review Of Three Classic Examples, Merlin G Butler, Bradley S Miller, Alicia Romano, Judith L. Ross, M Jennifer Abuzzahab, Philippe Backeljauw, Vaneeta Bamba, Amrit Bhangoo, Nelly Mauras, Mitchell Geffner

Department of Pediatrics Faculty Papers

Noonan, Turner, and Prader-Willi syndromes are classical genetic disorders that are marked by short stature. Each disorder has been recognized for several decades and is backed by extensive published literature describing its features, genetic origins, and optimal treatment strategies. These disorders are accompanied by a multitude of comorbidities, including cardiovascular issues, endocrinopathies, and infertility. Diagnostic delays, syndrome-associated comorbidities, and inefficient communication among the members of a patient's health care team can affect a patient's well-being from birth through adulthood. Insufficient information is available to help patients and their multidisciplinary team of providers transition from pediatric to adult health care systems. …


Parent And Child Anxiety Evaluated During An Early Period Of The Covid-19 Pandemic: A Mixed-Methods Study, Karla M. Abela, Darlene Acorda, Stanley Cron, Geri Lobiondo-Wood Oct 2022

Parent And Child Anxiety Evaluated During An Early Period Of The Covid-19 Pandemic: A Mixed-Methods Study, Karla M. Abela, Darlene Acorda, Stanley Cron, Geri Lobiondo-Wood

Journal of Patient-Centered Research and Reviews

Purpose: The objective of this study was to explore parent and child anxiety during the pandemic. Unlike previous pandemics, measures implemented to prevent the transmission of the SARS-CoV-2 virus have been much more limiting.

Methods: An explanatory convergent mixed-methods design was used to describe anxiety of children 9–17 years of age and their parents during August–October 2020. Adult and child versions of State-Trait Anxiety Inventory (STAI) were used to examine levels as measured on STAI’s state-anxiety subscale. Web-based interviews with a subset of patients were conducted qualitatively to analyze anxiety-related themes.

Results: A total of 188 parents …


Cross-Species Efficacy Of Enzyme Replacement Therapy For Cln1 Disease In Mice And Sheep, Hemanth R. Nelvagal, Sophie H. Wang, Elizabeth M. Eultgen, Keigo Takashash, Steven Q. Le, Rachel Nesbitt, Joshua T. Dearborn, Patricia I. Dickson, Mark S. Sands, Jonathan D. Cooper, Et Al. Oct 2022

Cross-Species Efficacy Of Enzyme Replacement Therapy For Cln1 Disease In Mice And Sheep, Hemanth R. Nelvagal, Sophie H. Wang, Elizabeth M. Eultgen, Keigo Takashash, Steven Q. Le, Rachel Nesbitt, Joshua T. Dearborn, Patricia I. Dickson, Mark S. Sands, Jonathan D. Cooper, Et Al.

2020-Current year OA Pubs

CLN1 disease, also called infantile neuronal ceroid lipofuscinosis (NCL) or infantile Batten disease, is a fatal neurodegenerative lysosomal storage disorder resulting from mutations in the CLN1 gene encoding the soluble lysosomal enzyme palmitoyl-protein thioesterase 1 (PPT1). Therapies for CLN1 disease have proven challenging because of the aggressive disease course and the need to treat widespread areas of the brain and spinal cord. Indeed, gene therapy has proven less effective for CLN1 disease than for other similar lysosomal enzyme deficiencies. We therefore tested the efficacy of enzyme replacement therapy (ERT) by administering monthly infusions of recombinant human PPT1 (rhPPT1) to PPT1-deficient …


Respiratory Virus Surveillance Among Children With Acute Respiratory Illnesses - New Vaccine Surveillance Network, United States, 2016-2021., Ariana Perez, Joana Y. Lively, Aaron Curns, Geoffrey A. Weinberg, Natasha B. Halasa, Mary Allen Staat, Peter G. Szilagyi, Laura S. Stewart, Monica M. Mcneal, Benjamin Clopper, Yingtao Zhou, Brett L. Whitaker, Elizabeth Lemasters, Elizabeth Harker, Janet A. Englund, Eileen J. Klein, Rangaraj Selvarangan, Christopher J. Harrison, Julie A. Boom, Leila C. Sahni, Marian G. Michaels, John V. Williams, Gayle E. Langley, Susan I. Gerber, Angela Campbell, Aron J. Hall, Brian Rha, Meredith Mcmorrow, New Vaccine Surveillance Network Collaborators Oct 2022

Respiratory Virus Surveillance Among Children With Acute Respiratory Illnesses - New Vaccine Surveillance Network, United States, 2016-2021., Ariana Perez, Joana Y. Lively, Aaron Curns, Geoffrey A. Weinberg, Natasha B. Halasa, Mary Allen Staat, Peter G. Szilagyi, Laura S. Stewart, Monica M. Mcneal, Benjamin Clopper, Yingtao Zhou, Brett L. Whitaker, Elizabeth Lemasters, Elizabeth Harker, Janet A. Englund, Eileen J. Klein, Rangaraj Selvarangan, Christopher J. Harrison, Julie A. Boom, Leila C. Sahni, Marian G. Michaels, John V. Williams, Gayle E. Langley, Susan I. Gerber, Angela Campbell, Aron J. Hall, Brian Rha, Meredith Mcmorrow, New Vaccine Surveillance Network Collaborators

Manuscripts, Articles, Book Chapters and Other Papers

The New Vaccine Surveillance Network (NVSN) is a prospective, active, population-based surveillance platform that enrolls children with acute respiratory illnesses (ARIs) at seven pediatric medical centers. ARIs are caused by respiratory viruses including influenza virus, respiratory syncytial virus (RSV), human metapneumovirus (HMPV), human parainfluenza viruses (HPIVs), and most recently SARS-CoV-2 (the virus that causes COVID-19), which result in morbidity among infants and young children (1-6). NVSN estimates the incidence of pathogen-specific pediatric ARIs and collects clinical data (e.g., underlying medical conditions and vaccination status) to assess risk factors for severe disease and calculate influenza and COVID-19 vaccine effectiveness. Current NVSN …


Entrectinib In Children And Young Adults With Solid Or Primary Cns Tumors Harboring Ntrk, Ros1, Or Alk Aberrations (Startrk-Ng), Ami V Desai, Karen Gauvain, Amy E Armstrong, Et Al. Oct 2022

Entrectinib In Children And Young Adults With Solid Or Primary Cns Tumors Harboring Ntrk, Ros1, Or Alk Aberrations (Startrk-Ng), Ami V Desai, Karen Gauvain, Amy E Armstrong, Et Al.

2020-Current year OA Pubs

BACKGROUND: Entrectinib is a TRKA/B/C, ROS1, ALK tyrosine kinase inhibitor approved for the treatment of adults and children aged ≥12 years with NTRK fusion-positive solid tumors and adults with ROS1 fusion-positive non-small-cell lung cancer. We report an analysis of the STARTRK-NG trial, investigating the recommended phase 2 dose (RP2D) and activity of entrectinib in pediatric patients with solid tumors including primary central nervous system tumors.

METHODS: STARTRK-NG (NCT02650401) is a phase 1/2 trial. Phase 1, dose-escalation of oral, once-daily entrectinib, enrolled patients aged <22 years with solid tumors with/without target NTRK1/2/3, ROS1, or ALK fusions. Phase 2, basket trial at the RP2D, enrolled patients with intracranial or extracranial solid tumors harboring target fusions or neuroblastoma. Primary endpoints: phase 1, RP2D based on toxicity; phase 2, objective response rate (ORR) in patients harboring target fusions. Safety-evaluable patients: ≥1 dose of entrectinib; response-evaluable patients: measurable/evaluable baseline disease and ≥1 dose at RP2D.

RESULTS: At data cutoff, 43 patients, median age of 7 years, were response-evaluable. In phase 1, 4 …


Outcomes Of Children With Low-Grade Gliomas In Low- And Middle-Income Countries: A Systematic Review, Richard Ward, Hannah M Jones, Davis Witt, Frederick Boop, Eric Bouffet, Carlos Rodriguez-Galindo, Ibrahim Qaddoumi, Daniel C Moreira Oct 2022

Outcomes Of Children With Low-Grade Gliomas In Low- And Middle-Income Countries: A Systematic Review, Richard Ward, Hannah M Jones, Davis Witt, Frederick Boop, Eric Bouffet, Carlos Rodriguez-Galindo, Ibrahim Qaddoumi, Daniel C Moreira

Department of Medical Oncology Faculty Papers

Purpose: Pediatric CNS tumors are increasingly a priority, particularly with the WHO designation of low-grade glioma (LGG) as one of six index childhood cancers. There are currently limited data on outcomes of pediatric patients with LGGs in low- and middle-income countries (LMICs).

Methods: To better understand the outcomes of LGGs in LMICs, this systematic review interrogated nine literature databases.

Results: The search identified 14,977 publications. Sixteen studies from 19 countries met the selection criteria and were included for data abstraction and analysis. Eleven studies (69%) were retrospective reviews from single institutions, and one (6%) captured institutional data prospectively. The studies …


Paediatrician's Guide To Post-Operative Care For Functionally Univentricular Chd: A Review, Christina Ronai, Sarosh P Batlivala, Quang-Tuyen Nguyen, Markus S Renno, Lindsay Arthur, Julie Glickstein, Jonathan N Johnson, Jacob R Miller, Carolyn A Altman, Elif Seda Selamet Tierney Oct 2022

Paediatrician's Guide To Post-Operative Care For Functionally Univentricular Chd: A Review, Christina Ronai, Sarosh P Batlivala, Quang-Tuyen Nguyen, Markus S Renno, Lindsay Arthur, Julie Glickstein, Jonathan N Johnson, Jacob R Miller, Carolyn A Altman, Elif Seda Selamet Tierney

2020-Current year OA Pubs

IMPORTANCE: Single ventricle CHD affects about 5 out of 100,000 newborns, resulting in complex anatomy often requiring multiple, staged palliative surgeries. Paediatricians are an essential part of the team that cares for children with single ventricle CHD. These patients often encounter their paediatrician first when a complication arises, so it is critical to ensure the paediatrician is knowledgeable of these issues to provide optimal care.

OBSERVATIONS: We reviewed the subtypes of single ventricle heart disease and the various palliative surgeries these patients undergo. We then searched the literature to detail the general paediatrician's approach to single ventricle patients at different …


Consensus-Based Recommendations On Priority Activities To Address Acute Kidney Injury In Children: A Modified Delphi Consensus Statement, Stuart L Goldstein, Tara M Neumayr, Et Al. Sep 2022

Consensus-Based Recommendations On Priority Activities To Address Acute Kidney Injury In Children: A Modified Delphi Consensus Statement, Stuart L Goldstein, Tara M Neumayr, Et Al.

2020-Current year OA Pubs

Importance: Increasing evidence indicates that acute kidney injury (AKI) occurs frequently in children and young adults and is associated with poor short-term and long-term outcomes. Guidance is required to focus efforts related to expansion of pediatric AKI knowledge.

Objective: To develop expert-driven pediatric specific recommendations on needed AKI research, education, practice, and advocacy.

Evidence Review: At the 26th Acute Disease Quality Initiative meeting conducted in November 2021 by 47 multiprofessional international experts in general pediatrics, nephrology, and critical care, the panel focused on 6 areas: (1) epidemiology; (2) diagnostics; (3) fluid overload; (4) kidney support therapies; (5) biology, pharmacology, and …


Multimodal Magnetic Resonance Neuroimaging Measures Characteristic Of Early Cart-Treated Pediatric Hiv: A Feature Selection Approach, Isaac L Khobo, Marcin Jankiewicz, Martha J Holmes, Francesca Little, Mark F Cotton, Barbara Laughton, Andre J W Van Der Kouwe, Allison Moreau, Emmanuel Nwosu, Ernesta M Meintjes, Frances C Robertson Sep 2022

Multimodal Magnetic Resonance Neuroimaging Measures Characteristic Of Early Cart-Treated Pediatric Hiv: A Feature Selection Approach, Isaac L Khobo, Marcin Jankiewicz, Martha J Holmes, Francesca Little, Mark F Cotton, Barbara Laughton, Andre J W Van Der Kouwe, Allison Moreau, Emmanuel Nwosu, Ernesta M Meintjes, Frances C Robertson

2020-Current year OA Pubs

Children with perinatally acquired HIV (CPHIV) have poor cognitive outcomes despite early combination antiretroviral therapy (cART). While CPHIV-related brain alterations can be investigated separately using proton magnetic resonance spectroscopy (


Does Social Intolerance Vary According To Cognitive Styles, Genetic Cognitive Capacity, Or Education?, Aino Saarinen, Liisa Keltikangas-Järvinen, Henrik Dobewall, C Robert Cloninger, Ari Ahola-Olli, Terho Lehtimäki, Nina Hutri-Kähönen, Olli Raitakari, Suvi Rovio, Niklas Ravaja Sep 2022

Does Social Intolerance Vary According To Cognitive Styles, Genetic Cognitive Capacity, Or Education?, Aino Saarinen, Liisa Keltikangas-Järvinen, Henrik Dobewall, C Robert Cloninger, Ari Ahola-Olli, Terho Lehtimäki, Nina Hutri-Kähönen, Olli Raitakari, Suvi Rovio, Niklas Ravaja

2020-Current year OA Pubs

BACKGROUND: Low education, low cognitive abilities, and certain cognitive styles are suggested to predispose to social intolerance and prejudices. Evidence is, however, restricted by comparatively small samples, neglect of confounding variables and genetic factors, and a narrow focus on a single sort of prejudice. We investigated the relationships of education, polygenic cognitive potential, cognitive performance, and cognitive styles with social intolerance in adulthood over a 15-year follow-up.

METHODS: We used data from the prospective population-based Young Finns Study (n = 960-1679). Social intolerance was evaluated with the Social Intolerance Scale in 1997, 2001, and 2011; cognitive performance with the Cambridge …


Association Of Blood-Based Brain Injury Biomarker Concentrations With Outcomes After Pediatric Cardiac Arrest, Ericka L Fink, Stuart Friess, Et Al. Sep 2022

Association Of Blood-Based Brain Injury Biomarker Concentrations With Outcomes After Pediatric Cardiac Arrest, Ericka L Fink, Stuart Friess, Et Al.

2020-Current year OA Pubs

Importance: Families and clinicians have limited validated tools available to assist in estimating long-term outcomes early after pediatric cardiac arrest. Blood-based brain-specific biomarkers may be helpful tools to aid in outcome assessment.

Objective: To analyze the association of blood-based brain injury biomarker concentrations with outcomes 1 year after pediatric cardiac arrest.

Design, Setting, and Participants: The Personalizing Outcomes After Child Cardiac Arrest multicenter prospective cohort study was conducted in pediatric intensive care units at 14 academic referral centers in the US between May 16, 2017, and August 19, 2020, with the primary investigators blinded to 1-year outcomes. The study included …


A Pilot Randomized Trial To Examine Effects Of A Hybrid Closed-Loop Insulin Delivery System On Neurodevelopmental And Cognitive Outcomes In Adolescents With Type 1 Diabetes, Allan L Reiss, Ana Maria Arbelaez, Et Al Aug 2022

A Pilot Randomized Trial To Examine Effects Of A Hybrid Closed-Loop Insulin Delivery System On Neurodevelopmental And Cognitive Outcomes In Adolescents With Type 1 Diabetes, Allan L Reiss, Ana Maria Arbelaez, Et Al

2020-Current year OA Pubs

Type 1 diabetes (T1D) is associated with lower scores on tests of cognitive and neuropsychological function and alterations in brain structure and function in children. This proof-of-concept pilot study (ClinicalTrials.gov Identifier NCT03428932) examined whether MRI-derived indices of brain development and function and standardized IQ scores in adolescents with T1D could be improved with better diabetes control using a hybrid closed-loop insulin delivery system. Eligibility criteria for participation in the study included age between 14 and 17 years and a diagnosis of T1D before 8 years of age. Randomization to either a hybrid closed-loop or standard diabetes care group was performed …


Notes From An Epicenter: Navigating Behavioral Clinical Trials On Autism Spectrum Disorder Amid The Covid-19 Pandemic In The Bronx, Alaina S Berruti, Roseann C Schaaf, Emily A Jones, Elizabeth Ridgway, Rachel L. Dumont, Benjamin E Leiby, Catherine Sancimino, Misung Yi, Sophie Molholm Aug 2022

Notes From An Epicenter: Navigating Behavioral Clinical Trials On Autism Spectrum Disorder Amid The Covid-19 Pandemic In The Bronx, Alaina S Berruti, Roseann C Schaaf, Emily A Jones, Elizabeth Ridgway, Rachel L. Dumont, Benjamin E Leiby, Catherine Sancimino, Misung Yi, Sophie Molholm

Department of Occupational Therapy Faculty Papers

Background: The COVID-19 pandemic impacted nearly all facets of our daily lives, and clinical research was no exception. Here, we discuss the impact of the pandemic on our ongoing, three-arm randomized controlled trial (RCT) Sensory Integration Therapy (SIT) in Autism: Mechanisms and Effectiveness (NCT02536365), which investigates the immediate and sustained utility of SIT to strengthen functional daily-living skills and minimize the presence of maladaptive sensory behaviors in autistic children.

Main text: In this text, we detail how we navigated the unique challenges that the pandemic brought forth between the years 2020 and 2021, including the need to rapidly …


The Global Network Socioeconomic Status Index As A Predictor Of Stillbirths, Perinatal Mortality, And Neonatal Mortality In Rural Communities In Low And Lower Middle Income Country Sites Of The Global Network For Women's And Children's Health Research, Archana B Patel, Carla M Bann, Cherryl S Kolhe, Adrien Lokangaka, Antoinette Tshefu, Melissa Bauserman, Lester Figueroa, Nancy F Krebs, Fabian Esamai, Sherri Bucher, Sarah Saleem, Robert L Goldenberg, Elwyn Chomba, Waldemar A Carlo, Shivaprasad Goudar, Richard J Derman, Marion Koso-Thomas, Elizabeth M Mcclure, Patricia L Hibberd Aug 2022

The Global Network Socioeconomic Status Index As A Predictor Of Stillbirths, Perinatal Mortality, And Neonatal Mortality In Rural Communities In Low And Lower Middle Income Country Sites Of The Global Network For Women's And Children's Health Research, Archana B Patel, Carla M Bann, Cherryl S Kolhe, Adrien Lokangaka, Antoinette Tshefu, Melissa Bauserman, Lester Figueroa, Nancy F Krebs, Fabian Esamai, Sherri Bucher, Sarah Saleem, Robert L Goldenberg, Elwyn Chomba, Waldemar A Carlo, Shivaprasad Goudar, Richard J Derman, Marion Koso-Thomas, Elizabeth M Mcclure, Patricia L Hibberd

Global Health Articles

BACKGROUND: Globally, socioeconomic status (SES) is an important health determinant across a range of health conditions and diseases. However, measuring SES within low- and middle-income countries (LMICs) can be particularly challenging given the variation and diversity of LMIC populations.

OBJECTIVE: The current study investigates whether maternal SES as assessed by the newly developed Global Network-SES Index is associated with pregnancy outcomes (stillbirths, perinatal mortality, and neonatal mortality) in six LMICs: Democratic Republic of the Congo, Guatemala, India, Kenya, Pakistan, and Zambia.

METHODS: The analysis included data from 87,923 women enrolled in the Maternal and Newborn Health Registry of the NICHD-funded …


Safety And Efficacy Of Voxelotor In Pediatric Patients With Sickle Cell Disease Aged 4 To 11 Years., Jeremie H. Estepp, Ram Kalpatthi, Gerald Woods, Sara Trompeter, Robert I. Liem, Kacie Sims, Adlette Inati, Baba P D Inusa, Andrew Campbell, Connie Piccone, Miguel R. Abboud, Kim Smith-Whitley, Sandra Dixon, Margaret Tonda, Carla Washington, Noelle M. Griffin, Clark Brown Aug 2022

Safety And Efficacy Of Voxelotor In Pediatric Patients With Sickle Cell Disease Aged 4 To 11 Years., Jeremie H. Estepp, Ram Kalpatthi, Gerald Woods, Sara Trompeter, Robert I. Liem, Kacie Sims, Adlette Inati, Baba P D Inusa, Andrew Campbell, Connie Piccone, Miguel R. Abboud, Kim Smith-Whitley, Sandra Dixon, Margaret Tonda, Carla Washington, Noelle M. Griffin, Clark Brown

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as young as 6 months of age, and pediatric patients are at risk for acute and life-threatening complications. Early intervention with treatments that target the underlying pathophysiological mechanism of SCD, sickle hemoglobin (HbS) polymerization, are expected to slow disease progression and circumvent disease-associated morbidity and mortality.

PROCEDURE: The HOPE-KIDS 1 trial (NCT02850406) is an ongoing four-part, phase 2a, open-label, single- and multiple-dose study to evaluate the pharmacokinetics, efficacy, and safety of voxelotor-a first-in-class HbS polymerization …


Experiences With Menses In Transgender And Gender Nonbinary Adolescents., Beth I. Schwartz, Arielle Effron, Benjamin Bear, Vanessa L Short, Julia Eisenberg, Sarah Felleman, Anne E Kazak Aug 2022

Experiences With Menses In Transgender And Gender Nonbinary Adolescents., Beth I. Schwartz, Arielle Effron, Benjamin Bear, Vanessa L Short, Julia Eisenberg, Sarah Felleman, Anne E Kazak

Department of Obstetrics and Gynecology Faculty Papers

STUDY OBJECTIVE: To describe menstrual history, associated dysphoria, and desire for menstrual management in transgender male and gender diverse adolescents who were assigned female at birth

DESIGN: Retrospective chart review

SETTING: Tertiary care children's hospital

PARTICIPANTS: All patients seen in a multidisciplinary pediatric gender program from March 2015 through December 2020 who were assigned female at birth, identified as transgender male or gender nonbinary, and had achieved menarche

INTERVENTION: None

MAIN OUTCOME MEASURES: Patient demographics, menstrual history, interest in and prior experiences with menstrual management, parental support, and concerns about menstrual management

RESULTS: Of the 129 included patients, 116 (90%) …


Firearm-Related Pediatric Head Trauma: A Scoping Review., Ron Gadot, Melissa A Lopresti, David N Smith, Lara Ouellette, Sandi Lam Aug 2022

Firearm-Related Pediatric Head Trauma: A Scoping Review., Ron Gadot, Melissa A Lopresti, David N Smith, Lara Ouellette, Sandi Lam

Library Staff Publications

BACKGROUND: Firearm-related injury is a significant cause of morbidity and mortality in pediatric populations. Despite a disproportionate role in the most morbid outcomes in both traumatic brain injury and firearm-related injury populations, firearm-related traumatic brain injury (frTBI) is an understudied epidemiological entity. There is need to increase understanding and promote interventions that reduce this burden of disease.

OBJECTIVE: To assess the evidence characterizing pediatric frTBI to highlight trends and gaps regarding burden of disease and interventions to reduce frTBI.

METHODS: We conducted a scoping review under Preferred Reporting Items for Systematic Reviews and Meta-Analyses-Scoping Review (PRISMA-ScR) guidelines on peer-reviewed studies …