Bioethics and Medical Ethics Commons^™

Phase Ii Study Of Ulixertinib In Children And Young Adults With Tumors Harboring Activating Mitogen-Activated Protein Kinase Pathway Alterations: Apec1621j Of The National Cancer Institute-Children's Oncology Group Pediatric Match Trial, Kieuhoa T Vo, Amit J Sabnis, P Mickey Williams, Sinchita Roy-Chowdhuri, David R Patton, Brent Coffey, Joel M Reid, Jin Piao, Lauren Saguilig, Todd A Alonzo, Stacey L Berg, Alok Jaju, Elizabeth Fox, Brenda J Weigel, Douglas S Hawkins, Margaret M Mooney, Naoko Takebe, James V Tricoli, Katherine A Janeway, Nita L Seibel, D Williams Parsons

Center for Medical Ethics and Health Policy Staff Publications

Purpose: The National Cancer Institute-Children's Oncology Group (NCI-COG) Pediatric MATCH trial assigns patients age 1-21 years with refractory malignancies to phase II treatment arms of molecularly targeted therapies on the basis of genetic alterations detected in their tumor. Patients with activating alterations in the mitogen-activated protein kinase pathway were treated with ulixertinib, an extracellular signal-regulated kinase (ERK)1/2 inhibitor.

Methods: As there were no previous pediatric data, ulixertinib was initially tested in a dose escalation cohort to establish the recommended phase II dose (RP2D) before proceeding to the phase II cohort. Ulixertinib was administered at 260 mg/m2/dose orally twice a day …

Parent-Led Cognitive Behavioral Teletherapy For Anxiety In Autistic Youth: A Randomized Trial Comparing Two Levels Of Therapist Support, Andrew G Guzick, Sophie C Schneider, Minjee Kook, Julia Rose Iacono, Saira A Weinzimmer, Troy Quast, Sean M Olsen, Killian Rainey Hughes, Emily Jellinek-Russo, Amanda Perozo Garcia, Abigail Candelari, Leandra N Berry, Robin P Goin-Kochel, Wayne K Goodman, Eric A Storch

Center for Medical Ethics and Health Policy Staff Publications

Parent-led cognitive behavioral therapy (CBT) is an efficient, promising form of therapy that may be well suited for autistic youth with anxiety disorders, though to date it has been minimally tested. In this study, 87 autistic youth (7 to 13 years old) with anxiety disorders and their parents were randomized to two forms of parent-led CBT in which parents led their child through a guided CBT workbook across 12 weeks: one with low therapist contact (four 30-minute telehealth calls), and one with standard therapist contact (ten 60-minute telehealth calls). Anxiety, functional impairment, and autism features significantly declined across therapy, without …

Family Accommodation In Children And Adolescents With Misophonia, Eric A Storch, Andrew G Guzick, Johann D'Souza, Jane Clinger, Daphne Ayton, Minjee Kook, Conor Rork, Eleanor E Smith, Isabel A Draper, Nasim Khalfe, Catherine E Rast, Nicholas Murphy, Marijn Lijfijjt, Wayne K Goodman, Matti Cervin

Center for Medical Ethics and Health Policy Staff Publications

Family accommodation (e.g., reassurance, modifying routines, assisting avoidance) has not been explored among youth with misophonia but may have important clinical and intervention implications. We examined family accommodation in 102 children and adolescents with interview-confirmed misophonia and compared its frequency and content to family accommodation in 95 children and adolescents with anxiety disorders. Findings showed that family accommodation was ubiquitous in pediatric misophonia and may be even more frequent than in youth with anxiety disorders. Assisting the child, participating in misophonia-related behaviors, and modifying family routines were endorsed by more than 70% of parents of children with misophonia. Further, compared …

A Phase 1 Trial Utilizing A Pharmacokinetic Endpoint To Determine The Optimal Dose Of Ramucirumab In Children And Adolescents With Relapsed Or Refractory Solid Tumors, Including Central Nervous System Tumors, Kristy L Pilbeam, Kamnesh Pradhan, James Croop, Charles G Minard, Xiaowei Liu, Stephan D Voss, Emasenyie Isikwei, Stacey L Berg, Joel M Reid, Elizabeth Fox, Brenda J Weigel

Center for Medical Ethics and Health Policy Staff Publications

Background: Ramucirumab is a monoclonal antibody that binds the extracellular domain of vascular endothelial growth factor receptor (VEGFR-2) and prevents binding of VEGF ligands. Based on population pharmacokinetic (PK) analysis and correlation with efficacy in adults, a target steady state trough concentration (Css,min ) ≥ 50 µg/mL was established.

Procedures: This phase 1 trial (ADVL1416) used a rolling six design and a PK primary endpoint to define the recommended phase 2 dose (RP2D) of ramucirumab in children with recurrent/refractory solid tumors. Two dose levels (DL) were planned (DL1: 8 mg/kg, DL2: 12 mg/kg administered intravenously [IV] every 2 weeks). Toxicity …

Risk Factors For Childhood Brain Tumours: A Systematic Review And Meta-Analysis Of Observational Studies From 1976 To 2022, Felix M Onyije, Roya Dolatkhah, Ann Olsson, Liacine Bouaoun, Isabelle Deltour, Friederike Erdmann, Audrey Bonaventure, Michael E Scheurer, Jacqueline Clavel, Joachim Schüz

Center for Medical Ethics and Health Policy Staff Publications

BACKGROUND: Childhood brain tumours (CBTs) are the leading cause of cancer death in children under the age of 20 years globally. Though the aetiology of CBT remains poorly understood, it is thought to be multifactorial. We aimed to synthesize potential risk factors for CBT to inform primary prevention.

METHODS: We conducted a systematic review and meta-analysis of epidemiological studies indexed in the PubMed, Web of Science, and Embase databases from the start of those resources through 27 July 2023. We included data from case-control or cohort studies that reported effect estimates for each risk factor around the time of conception, …

Should Secondary Pharmacogenomic Variants Be Actively Screened And Reported When Diagnostic Genome-Wide Sequencing Is Performed In A Child?, Jan M Friedman, Yvonne Bombard, Bruce Carleton, Amalia M Issa, Bartha Knoppers, Sharon E Plon, Vasiliki Rahimzadeh, Mary V Relling, Marc S Williams, Clara Van Karnebeek, Danya Vears, Martina C Cornel

Center for Medical Ethics and Health Policy Staff Publications

This white paper was prepared by the Global Alliance for Genomics and Health Regulatory and Ethics Work Stream's Pediatric Task Team to review and provide perspective with respect to ethical, legal, and social issues regarding the return of secondary pharmacogenomic variants in children who have a serious disease or developmental disorder and are undergoing exome or genome sequencing to identify a genetic cause of their condition. We discuss actively searching for and reporting pharmacogenetic/genomic variants in pediatric patients, different methods of returning secondary pharmacogenomic findings to the patient/parents and/or treating clinicians, maintaining these data in the patient's health record over …

Racial And Ethnic Disparities In Acuity Of Presentation Among Children With Newly Diagnosed Acute Leukemia, Lena E Winestone, Kelly D Getz, Yimei Li, Evanette Burrows, Michael E Scheurer, Vicky Tam, M Monica Gramatges, Jennifer J Wilkes, Tamara P Miller, Alix E Seif, Karen R Rabin, Brian T Fisher, Richard Aplenc

Center for Medical Ethics and Health Policy Staff Publications

We evaluated disparities in disease burden, organ dysfunction, vital signs, and timing of therapy in children newly presenting with acute leukemia. Among 899 patients with acute leukemia diagnosed at two large children's hospitals, a priori lab-based definitions of high disease burden, infection risk, renal dysfunction, and coagulopathy were applied to electronic health record data. Black patients with acute myeloid leukemia had increased prevalence of elevated white blood cell count and uric acid; Black patients with acute lymphoblastic leukemia demonstrated increased prevalence of coagulopathy. Black patients' presentation more frequently included multiple lab abnormalities consistent with advanced physiologic dysfunction. No differences were …

Interrogating The Value Of Return Of Results For Diverse Populations: Perspectives From Precision Medicine Researchers, Caitlin E Mcmahon, Nicole Foti, Melanie Jeske, William R Britton, Stephanie M Fullerton, Janet K Shim, Sandra Soo-Jin Lee

Center for Medical Ethics and Health Policy Staff Publications

Background: Over the last decade, the return of results (ROR) in precision medicine research (PMR) has become increasingly routine. Calls for individual rights to research results have extended the "duty to report" from clinically useful genetic information to traits and ancestry results. ROR has thus been reframed as inherently beneficial to research participants, without a needed focus on who benefits and how. This paper addresses this gap, particularly in the context of PMR aimed at increasing participant diversity, by providing investigator and researcher perspectives on and questions about the assumed value of ROR in PMR.

Methods: Semi-structured interviews with a …

Depressive Symptoms In Autistic Youth With Anxiety Disorders, Rebecca L Greenberg, Andrew G Guzick, Sophie C Schneider, Saira A Weinzimmer, Minjee Kook, Amanda B Perozo Garcia, Eric A Storch

Center for Medical Ethics and Health Policy Staff Publications

Objective: Anxiety and depression often coexist in youth and share overlapping symptomatology; however, little is known about the comorbidity of anxiety and depression in autistic youth. This study explores (1) the frequency of depressive symptoms among autistic children with clinically significant anxiety, (2) clinical variables that may be associated with elevated depressive symptoms, and (3) whether pretreatment depressive symptoms predict cognitive behavioral therapy (CBT) outcomes for anxiety.

Method: Children aged 7 to 13 years (N = 87) and their parents participated in a randomized controlled trial comparing 2 versions of a parent-led, telehealth-delivered CBT program. Parents and children completed a …

Acute And Chronic Kidney Injury During Therapy For Pediatric Acute Leukemia: A Report From The Leukemia Electronic Abstraction Of Records Network (Learn), Wendy Hsiao, Yimei Li, Kelly Getz, Lusha Cao, Edward Krause, Mark Ramos, Judy Lee, Maria Monica Gramatges, Karen R Rabin, Michael E Scheurer, Richard Aplenc, Michelle Denburg, Tamara P Miller

Center for Medical Ethics and Health Policy Staff Publications

Children with acute leukemia are at increased risk of kidney injury. Using electronic health record data from three centers between 2010 and 2018, this study retrospectively described acute kidney injury (AKI) and chronic kidney disease (CKD) prevalence in children with acute lymphoblastic or myeloid leukemia (ALL, AML) using Common Terminology Criteria for Adverse Events (CTCAE) and Kidney Disease Improving Global Outcomes (KDIGO) definitions. AKI during therapy was 25% (ALL) and 32% (AML) using CTCAE, versus 84% (ALL) and 74% (AML) using KDIGO. CKD prevalence was low and Grade 1/Stage 2. Further investigation is needed to optimally define kidney injury in …

Idiographic Coping Outcomes In Youth With Autism Spectrum Disorder And Co-Occurring Anxiety: Results From The Taasd Study, Lesley A Norris, Jonathan C Rabner, Eric A Storch, Jeffrey J Wood, Connor Kerns, Adam B Lewin, Brent J Small, Philip C Kendall

Center for Medical Ethics and Health Policy Staff Publications

Versions of cognitive behavioral therapy (Coping Cat, CC; Behavioral Interventions for Anxiety in Children with Autism, BIACA) have shown efficacy in treating anxiety among youth with autism spectrum disorder. Measures of efficacy have been primarily nomothetic symptom severity assessments. The current study examined idiographic coping outcomes in the Treatment of Anxiety in Autism Spectrum Disorder study (N = 167). Longitudinal changes in coping with situations individualized to youth fears (Coping Questionnaire) were examined across CC, BIACA and treatment as usual (TAU) in a series of multilevel models. CC and BIACA produced significantly greater improvements than TAU in caregiver-reported coping. Youth …

Pediatric Hiv+ Kaposi Sarcoma Exhibits Clinical, Virological, And Molecular Features Different From The Adult Disease, Carolina Caro-Vegas, Alice Peng, Angelica Juarez, Allison Silverstein, William Kamiyango, Jimmy Villiera, Casey L Mcatee, Rizine Mzikamanda, Tamiwe Tomoka, Erin C Peckham-Gregory, Razia Moorad, Carrie L Kovarik, Liane R Campbell, Parth S Mehta, Peter N Kazembe, Carl E Allen, Michael E Scheurer, Nmazuo W Ozuah, Dirk P Dittmer, Nader Kim El-Mallawany

Center for Medical Ethics and Health Policy Staff Publications

BACKGROUND

Kaposi sarcoma (KS) is among the most common childhood cancers in Eastern and Central Africa. Pediatric KS has a distinctive clinical presentation compared with adult KS, which includes a tendency for primary lymph node involvement, a considerable proportion of patients lacking cutaneous lesions, and a potential for fulminant disease. The molecular mechanisms or correlates for these disease features are unknown.

METHODS

This was a cross-sectional study. All cases were confirmed by IHC for KS-associated herpesvirus (KSHV) LANA protein. Baseline blood samples were profiled for HIV and KSHV genome copy numbers by qPCR and secreted cytokines by ELISA. Biopsies were …

Safety And Immunogenicity Of Etvax®, An Oral Inactivated Vaccine Against Enterotoxigenic Escherichia Coli Diarrhoea: A Double-Blinded, Randomized, Placebo-Controlled Trial Amongst Finnish Travellers To Benin, West Africa, Anu Kantele, Marianna Riekkinen, T Sakari Jokiranta, Sari H Pakkanen, Jukka-Pekka Pietilä, Anu Patjas, Mari Eriksson, Tamim Khawaja, Peter Klemets, Kati Marttinen, Heli Siikamäki, Anna Lundgren, Jan Holmgren, Agneta Lissmats, Nils Carlin, Ann-Mari Svennerholm

Faculty and Staff Publications

Background: No licensed human vaccines are available against enterotoxigenic Escherichia coli (ETEC), a major diarrhoeal pathogen affecting children in low- and middle-income countries and foreign travellers alike. ETVAX®, a multivalent oral whole-cell vaccine containing four inactivated ETEC strains and the heat-labile enterotoxin B subunit (LTB), has proved promising in Phase 1 and Phase 1/ 2 studies.

Methods: We conducted a Phase 2b double-blinded, randomized, placebo-controlled trial amongst Finnish travellers to Benin, West Africa. This report presents study design and safety and immunogenicity data. Volunteers aged 18-65 years were randomized 1:1 to receive ETVAX® or placebo. They visited Benin for 12 …

Tazemetostat For Tumors Harboring Smarcb1/Smarca4 Or Ezh2 Alterations: Results From Nci-Cog Pediatric Match Apec1621c, Susan N Chi, Joanna S Yi, P Mickey Williams, Sinchita Roy-Chowdhuri, David R Patton, Brent D Coffey, Joel M Reid, Jin Piao, Lauren Saguilig, Todd A Alonzo, Stacey L Berg, Nilsa C Ramirez, Alok Jaju, Joyce C Mhlanga, Elizabeth Fox, Douglas S Hawkins, Margaret M Mooney, Naoko Takebe, James V Tricoli, Katherine A Janeway, Nita L Seibel, D Williams Parsons

Center for Medical Ethics and Health Policy Staff Publications

Background: National Cancer Institute-Children's Oncology Group Pediatric Molecular Analysis for Therapy Choice assigns patients aged 1-21 years with refractory solid tumors, brain tumors, lymphomas, and histiocytic disorders to phase II trials of molecularly targeted therapies based on detection of predefined genetic alterations. Patients whose tumors harbored EZH2 mutations or loss of SMARCB1 or SMARCA4 by immunohistochemistry were treated with EZH2 inhibitor tazemetostat.

Methods: Patients received tazemetostat for 28-day cycles until disease progression or intolerable toxicity (max 26 cycles). The primary endpoint was objective response rate; secondary endpoints included progression-free survival and tolerability of tazemetostat.

Results: Twenty patients (median age = …

Clinically Indicated Genomic Sequencing Of Children In Foster Care: Legal And Ethical Issues, Hadley Stevens Smith, Emily S Bonkowski, Madison R Hickingbotham, Stacey Pereira, Thomas May, Christi J Guerrini

Center for Medical Ethics and Health Policy Staff Publications

There are approximately 400 000 children in foster care in the US, approximately one-half of whom have chronic health problems and approximately 10% of whom have complex healthcare needs. Given the increasing relevance of genomic sequencing to guide clinical care for children with rare, chronic, and undiagnosed conditions, it may be an important component of diagnostic evaluation for children in foster care. Clinically indicated genomic sequencing may provide information that has health implications for children in foster care, as well as for their biological parents and other relatives. Whether and how genomic sequencing results impact legal decision making and family …

Closing The Gap For Children With Ocd: A Staged-Care Model Of Cognitive Behavioural Therapy With Exposure And Response Prevention, Lara J Farrell, Allison M Waters, Eric A Storch, Gabrielle Simcock, Iain E Perkes, Jessica R Grisham, Katelyn M Dyason, Thomas H Ollendick

Center for Medical Ethics and Health Policy Staff Publications

Childhood obsessive-compulsive disorder (OCD) is among the most prevalent and disabling mental health conditions affecting children and adolescents. Although the distress and burden associated with childhood OCD are well documented and empirically supported treatments are available, there remains an unacceptable "treatment gap" and "quality gap" in the provision of services for youth suffering from OCD. The treatment gap represents the large number of children who never receive mental health services for OCD, while the quality gap refers to the children and young people who do access services, but do not receive evidence-based, cognitive behavioural therapy with exposure and response prevention …

Childhood Exposure To Parental Threatening Behaviors And Anxiety In Emerging Adulthood: Indirect Effects Of Perceived Stress, Erika S Trent, Andres G Viana, Elizabeth M Raines, Haley E Conroy Busch, Karina Silva, Eric A Storch, Michael J Zvolensky

Center for Medical Ethics and Health Policy Staff Publications

Background: Although childhood exposure to parental threatening behaviors is associated with elevated anxiety in emerging adulthood, the underlying mechanisms remain unexplored. Perceived stress-a subjective experience comprised of feelings of helplessness (being unable to cope or exert control) and poor self-efficacy (confidence in one's ability to manage stressors)-is one candidate mechanism. The present investigation examined the underlying role of perceived stress in the association between childhood exposure to parental threatening behaviors and anxiety symptom severity in a sample of emerging adults.

Methods: Participants (N = 855; Mage = 18.75 years, SD = 1.05, range 18-24; 70.8% female) were recruited from a …

Child And Adolescent Psychiatrists’ Use, Attitudes, And Understanding Of Genetic Testing And Pharmacogenetics In Clinical Practice, Takahiro Soda, Amanda R Merner, Brent J Small, Laura N Torgerson, Katrina Muñoz, Jehannine Austin, Eric A Storch, Stacey Pereira, Gabriel Lázaro-Muñoz

Center for Medical Ethics and Health Policy Staff Publications

The purpose of this study was to report current practices and attitudes of child and adolescent psychiatrists (CAP) regarding diagnostic genetic and pharmacogenetic (PGx) testing. We surveyed 958 US-based practicing CAP. 54.9% of respondents indicated that they had ordered/referred for a genetic test in the past 12 months. 87% of respondents agreed that it is their role to discuss genetic information regarding psychiatric conditions with their patients; however, 45% rated their knowledge of genetic testing practice guidelines as poor/very poor. The most ordered test was PGx (32.2%), followed by chromosomal microarray (23.0%). 73.4% reported that PGx is at least slightly …

Cognitive Behavioral Therapy For Children And Adolescents With Anxiety Disorders, Jordan T Stiede, Erika S Trent, Andres G Viana, Andrew G Guzick, Eric A Storch, Jonathan Hershfield

Center for Medical Ethics and Health Policy Staff Publications

Anxiety disorders are the most common class of psychiatric conditions among children and adolescents. The cognitive behavioral model of childhood anxiety has a strong theoretic and empirical foundation that provides the basis for effective treatment. Cognitive behavioral therapy (CBT), with an emphasis on exposure therapy, is the gold standard treatment for childhood anxiety disorders, with strong empirical support. A case vignette demonstrating CBT for childhood anxiety disorders in practice, as well as recommendations for clinicians, are also provided.

Call For Moral Recognition As Part Of Paediatric Assent, Jared Smith, Jennifer Blumenthal-Barby

Center for Medical Ethics and Health Policy Staff Publications

No abstract provided.

Child And Adolescent Psychiatrists’ Use, Attitudes, And Understanding Of Genetic Testing And Pharmacogenetics In Clinical Practice, Takahiro Soda, Amanda R Merner, Brent J Small, Laura N Torgerson, Katrina Muñoz, Jehannine Austin, Eric A Storch, Stacey Pereira, Gabriel Lázaro-Muñoz

Center for Medical Ethics and Health Policy Staff Publications

The purpose of this study was to report current practices and attitudes of child and adolescent psychiatrists (CAP) regarding diagnostic genetic and pharmacogenetic (PGx) testing. We surveyed 958 US-based practicing CAP. 54.9% of respondents indicated that they had ordered/referred for a genetic test in the past 12 months. 87% of respondents agreed that it is their role to discuss genetic information regarding psychiatric conditions with their patients; however, 45% rated their knowledge of genetic testing practice guidelines as poor/very poor. The most ordered test was PGx (32.2%), followed by chromosomal microarray (23.0%). 73.4% reported that PGx is at least slightly …

Divergent Clinical Presentations And Outcomes Among Children And Adolescents With Kaposi Sarcoma In Malawi And Tanzania, Liane R Campbell, Allison Silverstein, Erin Peckham-Gregory, William Kamiyango, Jimmy Villiera, Casey L Mcatee, Jason M Bacha, Carrie L Kovarik, Parth S Mehta, Toni Chanroo, Asulwisye Kapesa, Beatrice Malingoti, Rizine Mzikamanda, Nmazuo W Ozuah, Carl E Allen, Michael E Scheurer, Nader K El-Mallawany

Center for Medical Ethics and Health Policy Staff Publications

Objectives: The Kaposi sarcoma (KS) T0 versus T1 staging classification does not address the unique clinical features of paediatric KS in human gammaherpesvirus 8 (HHV-8) endemic regions of Africa. This study seeks to define patterns of childhood KS using a paediatric-specific approach.

Methods: The Lilongwe paediatric KS staging classification categorizes disease based on clinical phenotype: stage 1 = mild/moderate KS limited to cutaneous/oral involvement, stage 2 = primarily lymphadenopathic disease, stage 3 = woody edema KS, stage 4 = visceral and/or severe/disseminated mucocutaneous disease. Characteristics and outcomes were evaluated from paediatric referral centres in Lilongwe, Malawi, and Mbeya, Tanzania.

Results: …

Key Drivers Of Family-Level Utility Of Pediatric Genomic Sequencing: A Qualitative Analysis To Support Preference Research, Hadley Stevens Smith, Emily S Bonkowski, Raymond Belanger Deloge, Amanda M Gutierrez, Alva M Recinos, Tara A Lavelle, David L Veenstra, Amy L Mcguire, Stacey Pereira

Center for Medical Ethics and Health Policy Staff Publications

Given that pediatric genomic sequencing (GS) may have implications for the health and well-being of both the child and family, a clearer understanding of the key drivers of the utility of GS from the family perspective is needed. The purpose of this study is to explore what is important to caregivers of pediatric patients regarding clinical GS, with a focus on family-level considerations. We conducted semi-structured interviews with caregivers (n = 41) of pediatric patients who had been recommended for or completed GS that explored the scope of factors caregivers considered when deciding whether to pursue GS for their child. …

Parents’ Decision-Making Regarding Whether To Receive Adult-Onset Only Genetic Findings For Their Children: Findings From The Babyseq Project, Stacey Pereira, Amanda M Gutierrez, Jill Oliver Robinson, Kurt D Christensen, Casie A Genetti, Carrie L Blout Zawatsky, Rebecca L Hsu, Bethany Zettler, Melissa Kurtz Uveges, Richard B Parad, Alan H Beggs, Ingrid A Holm, Robert C Green, Amy L Mcguire

Center for Medical Ethics and Health Policy Staff Publications

Purpose: Most professional guidelines recommend against genetic screening for adult-onset only (AO) conditions until adulthood, yet others argue that there may be benefit to disclosing such results. We explored parents' decision-making on this issue in the BabySeq Project, a clinical trial of newborn genomic sequencing.

Methods: We conducted interviews with parents (N = 24) who were given the option to receive actionable AO results for their children. Interviews explored parents' motivations to receive and reasons to decline AO genetic disease risk information, their decision-making process, and their suggestions for supporting parents in making this decision.

Results: Parents noted several motivations …

Effortful Control Moderates The Relation Between Negative Emotionality And Child Anxiety And Depressive Symptom Severity In Children With Anxiety Disorders, Elizabeth M Raines, Andres G Viana, Erika S Trent, Haley E Conroy, Karina Silva, Michael J Zvolensky, Eric A Storch

Center for Medical Ethics and Health Policy Staff Publications

The present study investigated the interactive effect of reactive (negative emotionality) and regulatory (effortful control) aspects of temperament in the prediction of child anxiety and depressive symptoms. Clinically anxious children and their mothers completed a battery of questionnaires that included self- and mother-ratings of child effortful control, negative emotionality, anxiety, and depressive symptoms. Multiple regression analyses were conducted to examine the moderating effect of effortful control on the relation between negative emotionality and child anxiety and depressive symptom severity. The interaction between negative emotionality and effortful control was statistically significant and simple slopes revealed that as effortful control increased, the …

Deep Brain Stimulation For Pediatric Dystonia: Clinicians' Perspectives On The Most Pressing Ethical Challenges, Kristin M Kostick-Quenet, Lavina Kalwani, Laura N Torgerson, Katrina Munoz, Clarissa Sanchez, Eric A Storch, J S Blumenthal-Barby, Gabriel Lazáro-Muñoz

Center for Medical Ethics and Health Policy Staff Publications

Introduction: Pediatric deep brain stimulation (pDBS) is commonly used to manage treatment-resistant primary dystonias with favorable results and more frequently used for secondary dystonia to improve quality of life. There has been little systematic empirical neuroethics research to identify ethical challenges and potential solutions to ensure responsible use of DBS in pediatric populations.

Methods: Clinicians (n = 29) who care for minors with treatment-resistant dystonia were interviewed for their perspectives on the most pressing ethical issues in pDBS.

Results: Using thematic content analysis to explore salient themes, clinicians identified four pressing concerns: (1) uncertainty about risks and benefits of pDBS …

Clinician Perceptions Of Passport For Care, A Web-Based Clinical Decision Support Tool For Survivorship Care Plan Delivery, Jason E King, Michael C O'Connor, Ellen Shohet, Susan M Krause, Michael E Scheurer, Marc E Horowitz, David G Poplack, C Michael Fordis, Maria M Gramatges

Center for Medical Ethics and Health Policy Staff Publications

Background: The Children's Oncology Group Long-Term Follow-Up Guidelines provide exposure-based risks and recommendations for late effects screening of survivors of childhood cancer. Passport for Care (PFC) is a web-based clinical decision support tool for generating a personalized survivorship care plan (SCP) derived from the Guidelines and user-entered exposures. We assessed PFC clinician user practices and perceptions of PFC impact on clinic workflow, guidelines application, and survivor shared decision-making.

Procedure: A 35-item REDCap survey was emailed to all PFC users (n = 936) in 146 current and former PFC user clinics. Anonymous responses were permitted. Results were summarized and compared with …

Validation Of A Process For Shared Decision-Making In Pediatrics, Douglas J Opel, Holly Hoa Vo, Nicolas Dundas, Heather Spielvogle, Amanda Mercer, Benjamin S Wilfond, Jonna Clark, Carrie L Heike, Elliott M Weiss, Mersine A Bryan, Seema K Shah, Carolyn A Mccarty, Jeffrey D Robinson, Jennifer Blumenthal-Barby, Jon Tilburt

Center for Medical Ethics and Health Policy Staff Publications

OBJECTIVE: We sought to confirm, refute, or modify a 4-step process for implementing shared decision-making (SDM) in pediatrics that involves determining 1) if the decision includes >1 medically reasonable option; 2) if one option has a favorable medical benefit-burden ratio compared to other options; and 3) parents' preferences regarding the options; then 4) calibrating the SDM approach based on other relevant decision characteristics.

METHODS: We videotaped a purposive sample of pediatric inpatient and outpatient encounters at a single US children's hospital. Clinicians from 7 clinical services (craniofacial, neonatology, oncology, pulmonary, pediatric intensive care, hospital medicine, and sports medicine) were eligible. …

Medical Ethics Principles Underscore Advocating For Human Papillomavirus Vaccine, C Mary Healy, Lara S Savas, Ross Shegog, Rebecca Lunstroth, Sally W Vernon

Faculty, Staff and Student Publications

Studies have consistently shown that vaccination rates against human papillomavirus (HPV) lag far behind other adolescent vaccinations recommended at the same age, resulting in exposing adolescents to unnecessary future risk of infection, and genital and head and neck cancers. Studies also have demonstrated that a major barrier to vaccination is lack of a strong provider recommendation. Factors that providers offer for failing to give a strong recommendation range from perception that the child is not at risk or the need to explain that the vaccine is not mandated (lack of equity and justice) or respect for parental autonomy. We look …

Incidence Of An Insulin-Requiring Hyperglycemic Syndrome In Sars-Cov-2-Infected Young Individuals: Is It Type 1 Diabetes?, Massimo Pietropaolo, Peter Hotez, Nick Giannoukakis

Center for Medical Ethics and Health Policy Staff Publications

Pancreatic ACE2 receptor expression, together with increased prevalence of insulin-requiring hyperglycemia in patients with coronavirus disease 2019 (COVID-19), suggested that severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pancreatic infection might trigger a β-cell-selective inflammation precipitating autoimmune type 1 diabetes (T1D). We examined T1D incidence in patients with COVID-19 inside a large, global population using a "big data" approach. The incidence in 0-30-year-old patients with confirmed COVID-19 over an ∼15-month period from the beginning of the COVID-19 pandemic was compared with an age-matched population without COVID-19 inside the TriNetX COVID-19 Research Network (>80 million deidentified patient electronic medical records globally). …