Medicine and Health Sciences Commons^™

Principles Of Dormancy Evident In High-Grade Serous Ovarian Cancer, Trevor G. Shepherd, Frederick A. Dick

Paediatrics Publications

In cancer, dormancy refers to a clinical state in which microscopic residual disease becomes non-proliferative and is largely refractory to chemotherapy. Dormancy was first described in breast cancer where disease can remain undetected for decades, ultimately leading to relapse and clinical presentation of the original malignancy. A long latency period can be explained by withdrawal from cell proliferation (cellular dormancy), or a balance between proliferation and cell death that retains low levels of residual disease (tumor mass dormancy). Research into cellular dormancy has revealed features that define this state. They include arrest of cell proliferation, altered cellular metabolism, and unique …

The Ward Round: Friend Or Foe In Postgraduate Training? A Grounded Theory Study Of Residents’ Perspectives, Mariam Noorani

Paediatrics and Child Health, East Africa

The ward round has traditionally been a learning activity in medical education. Apart from education, ward rounds have multiple roles including patient care and communication. Some studies have described the ward round as an ideal place to learn patient management while others reported that little learning happens on rounds due to lack of time and patient volume. This study aimed to develop a deeper understanding of ward round learning from the perspec-tive of postgraduate trainees. A constructivist grounded theory approach was used and data was collected during focus group discussions. Data were analyzed by initial coding, then grouped into focused …

Ispad Clinical Practice Consensus Guidelines 2022: Assessment And Management Of Hypoglycemia In Children And Adolescents With Diabetes, Mary B. Abraham, Beate Karges, Klemen Dovc, Diana Naranjo, Ana Maria Arbelaez, Joyce Mbogo, Ganesh Javelikar, Timothy W. Jones, Farid H. Mahmud

Paediatrics and Child Health, East Africa

No abstract provided.

Iv Contrast For Mri And Ct In Patients With Kidney Disease, Children's Mercy Kansas City

Clinical Pathways (Formally Known As Care Process Models)

No abstract provided.

Management Of Multisystem Inflammatory Syndrome In Children (Mis-C) In Resource Limited Settings: The Kenyan Experience, Angela Migowa, Pauline Samia, Del-Rossi Sean, Oliver Ombeva Malande, Jasmit Shah, Chemutai Kenei, Joy Ayaya, Daisy Jeruto, Laura Oyiengo, Laura Lewandowski

Paediatrics and Child Health, East Africa

Background: Since the onset of the recent COVID-19 pandemic, there have been growing concerns regarding multisystem inflammatory syndrome in children (MIS-C). This study aims to describe the clinico-epidemiological profile and challenges in management of MIS-C in low-middle income countries by highlighting the Kenyan experience.

Methods: A retrospective study at the Aga Khan University Hospital Nairobi, Avenue Hospital Kisumu and Kapsabet County Referral Hospital was undertaken to identify cases of MIS-C. A detailed chart review using the World Health Organization (WHO) data collection tool was adapted to incorporate information on socio-demographic details and treatment regimens.

Findings: Twenty children with MIS-C were …

Blackwater Fever And Acute Kidney Injury In Children Hospitalized With An Acute Febrile Illness: Pathophysiology And Prognostic Significance, Andrea Conroy, Michael Hawkes, Aleksandra Leligdowicz, Ivan Mufumba, Michelle Starr, Kathleen Zhong, Sophie Namasopo, Robert Opoka, Chandy John, Kevin C. Kain

Paediatrics and Child Health, East Africa

Background: Acute kidney injury (AKI) and blackwater fever (BWF) are related but distinct renal complications of acute febrile illness in East Africa. The pathogenesis and prognostic signifcance of BWF and AKI are not well understood.

Methods: A prospective observational cohort study was conducted to evaluate the association between BWF and AKI in children hospitalized with an acute febrile illness. Secondary objectives were to examine the association of AKI and BWF with (i) host response biomarkers and (ii) mortality. AKI was defned using the Kidney Disease: Improving Global Outcomes criteria and BWF was based on parental report of tea-colored urine. Host …

Description Of Audiologic, Developmental, Ophthalmologic, And Neurologic Diagnoses At A Primary Care Clinic For Children In Foster Care, Jill Mcleigh, Lauren Malthaner, Melissa Reynoso, Kimberly Stone

Health Status, Outcomes, and Literacy and Healthcare Utilization

This study aimed to describe the prevalence of medically diagnosed audiologic, developmental, ophthalmologic, and neurologic conditions in a foster care primary care clinic and to identify any associations among these diagnoses and patient characteristics, placement type, and maltreatment type.

For questions about this study or others, please email [email protected]

childrens.com/fostercare

Facilitators And Inhibitors To First Adult Care Visits In The Transition From Paediatric To Adult Orientated Inflammatory Bowel Disease Healthcare, Joanna Rea

Theses

Background: Inflammatory bowel disease (IBD) can be a very debilitating disease and it is found to be more extensive in children with rapid early progression (Van Limbergen et al., 2008). As adolescents, these children transition from paediatric to adult oriented care. The first adult care visits are a pivotal phase of a successful transition process, where adolescents potentially begin to actively take control of their disease. The aim of this study is to explore the inhibitors and facilitators to first adult care visits in the transition process from paediatric to adult oriented IBD healthcare.

Methods: An exploratory, descriptive, qualitative approach …

Eosinophilic Myocarditis Secondary To Hypereosinophilic Syndrome Vs. Eosinophilic Granulomatosis With Polyangiitis: A Diagnostic Dilemma, Nathan Lavoy, Brian Birnbaum, Julia G. Harris, Aarti Pandya, Allison Taber

Posters

Introduction: Hypereosinophilic syndrome (HES) is a rare but detrimental diagnosis in the pediatric population. Cardiac involvement within HES can present as a diagnosis of Eosinophilic Myocarditis which can progress to irreversible damage and potentially death. This case report discusses a 16-year-old male treated for HES, although HES can have overlap with Eosinophilic Granulomatosis with Polyangiitis (EGPA) and requires a multi-disciplinary approach to patient management. Case Description: The patient was a 16-year-old male, with a history of asthma, presenting with acute onset of chest pain. He was found to have peripheral eosinophilia with an absolute eosinophil count of 11.71. He had …

Neonate, Infant, And Child Mortality In North Africa And Middle East By Cause: An Analysis For The Global Burden Of Disease Study 2019, Sadaf G. Sepanlou, Hossein Rezaei Aliabadi, Reza Malekzadeh, Ali. A. Asadi-Pooya

Global Health Articles

Background: During the past three decades, neonate, infant, and child mortality declined in North Africa and Middle East. However, there is substantial heterogeneity in mortality rates across countries.

Methods: This study is part of the Global Burden of Diseases study (GBD) 2019. We report the number as well as mortality rates for neonates, infants, and children by cause across 21 countries in the region since 1990.

Results: Between 1990 and 2019, the neonate mortality rate in the region declined from 31.9 (29.8, 34.0) to 12.2 (11.1, 13.3) per 1000 live births. Respective figures for under 5 mortality rates (U5MRs) were …

An Outbreak Of A Novel Recombinant Coxsackievirus A4 In A Kindergarten, Shandong Province, China, 2021, Juan Li, Nan Ni, Yanan Cui, Shuai Zong, Xue Yao, Tao Hu, Mengyuan Cao, Yong Zhang, Peiqiang Hou, Michael J Carr, Weijia Xing, Hong Zhou, Weifeng Shi

Faculty, Staff and Student Publications

In 2021, twenty children exhibiting influenza-like illnesses were reported from a kindergarten in Shandong Province, China. Eleven genomes of Coxsackievirus A4 (CV-A4) were obtained from the pediatric cases, sharing <93% genome sequence identities with known CV-A4 strains. Further analyses suggested potential genetic recombination in the P3 region of the novel strains.

Improving The Perioperative Experience Of Patients And Families In A Pediatric Setting, Anjanette Pong

Student Scholarly Projects

Practice Problem: The experience of surgery for pediatric patients and their families can be dependent on multiple factors including adequate preparation, English language proficiency and realistic expectations. Anxiety can contribute to a negative experience that may result in poor outcomes and a damaging reflection of the healthcare team and organization.

PICOT: The PICOT question that guided this project was: In preoperative pediatric patients and their families, how do therapeutic communication style and the provision of information about the perioperative experience affect their healthcare experience over eight weeks?

Evidence: A multi-modal approach to providing tailored preoperative education for the child and …

Clinical And Demographic Factors Contributing To Asparaginase-Associated Toxicities In Children With Acute Lymphoblastic Leukemia, Priyadarshani Dharia, Michael D Swartz, M Brooke Bernhardt, Han Chen, M Monica Gramatges, Philip J Lupo, Austin L Brown, Michael E Scheurer

Center for Medical Ethics and Health Policy Staff Publications

A total of 548 patients (age range: 1-22 years, 60.4% Hispanic, 55.8% male) diagnosed with acute lymphoblastic leukemia were reviewed for pegaspargase-associated hypersensitivity (14.8%), hyperbilirubinemia (9.7%), venous thromboembolism (VTE, 9.7%), and pancreatitis (5.3%). Odds ratios (OR) and 95% confidence intervals (CI) evaluated associations between clinical factors and each toxicity, cumulative number of toxicities, and toxicity clusters identified using k-mode analysis. Most (68.9%) did not experience any toxicity, 24.6% experienced one toxicity, and 6.3% two or more. Age >10 years was associated with hyperbilirubinemia (OR = 3.83; 95% CI: 1.64-8.95), pancreatitis (OR = 3.72; 95% CI: 1.29-10.68), VTE (OR = 4.65; …

Infrastructure Development For Personalized Risk Prediction To Reduce Cardiovascular Disease In Childhood Cancer Survivors, Suman Shrestha

Dissertations and Theses (Open Access)

Although childhood cancer survivors have lengthy life expectancies, they run the risk of experiencing long-term health issues as a result of their treatment. The most frequent non-cancerous cause of morbidity and mortality for these survivors is cardiac disease. Radiation therapy (RT) has been linked in numerous cohort studies to a higher chance of developing a late cardiac disease in these survivors, and this risk rises with higher mean heart doses and increased RT exposure to larger cardiac volumes. Since, the heart is a heterogeneous organ made up of several distinct substructures, RT dose received by the entire heart does not …

Design For A Cluster Randomized Controlled Trial To Evaluate The Effects Of The Catch Healthy Smiles School-Based Oral Health Promotion Intervention Among Elementary School Children, Ru-Jye Chuang, Courtney Byrd-Williams, Jose-Miguel Yamal, Kila Johnson, Steven Kelder, Suchitra Nelson, Dania Mofleh, Shreela V Sharma

Faculty, Staff and Student Publications

BACKGROUND: The top two oral diseases (tooth decay and gum disease) are preventable, yet dental caries is the most common childhood disease with 68% of children entering kindergarten having tooth decay. CATCH Healthy Smiles is a coordinated school health program to prevent cavities for students in kindergarten, 1st, and 2nd grade, and is based on the framework of Coordinated Approach to Child Health (CATCH), an evidence-based coordinated school health program. CATCH has undergone several cluster-randomized controlled trials (CRCT) demonstrating sustainable long-term effectiveness in incorporating the factors surrounding children, in improving eating and physical activity behaviors, and reductions in obesity prevalence …

Evaluation Of Murine Host Sex As A Biological Variable In Transplanted Human Intestinal Organoid Development, Eoin P Mcneill, Vikas S Gupta, David J Sequeira, Noah F Shroyer, Allison L Speer

Faculty, Staff and Student Publications

Background: Human intestinal organoids (HIOs), when transplanted into immunocompromised mice (tHIOs), demonstrate significant growth and maturation. While both male and female mice are reported to be viable hosts for these experiments, a direct comparison of sex-related differences in tHIO structure and development has not been performed.

Aims: We sought to identify host sex-related differences in tHIO engraftment, morphology, and epithelial and mesenchymal development.

Methods: HIOs were generated in vitro and transplanted beneath the kidney capsule of NSG male and female mice. tHIOs were harvested at 8-9 weeks. Anthropometric measurements were captured. tHIOs were divided in half and histology or RT-qPCR …

Which Children Are Frequently Victimized In Us Elementary Schools? Population-Based Estimates, Paul L Morgan, Adrienne D Woods, Yangyang Wang, George Farkas, Yoonkyung Oh, Marianne M Hillemeier, Cynthia Mitchell

Faculty, Staff and Student Publications

We analyzed a population-based cohort of 11,780 U.S. kindergarten children to identify risk and protective factors predictive of frequent verbal, social, reputational, and/or physical bullying victimization during the upper elementary grades. We also stratified the analyses by biological sex. Both girls and boys displaying kindergarten externalizing problem behaviors were at consistently higher risk of frequent victimization during 3rd-5th grade (for the combined sample of boys and girls, verbal odds ratio [OR] = 1.82, social OR = 1.60, reputational OR = 1.85, physical OR = 1.67, total OR = 1.93). Hispanic children relative to non-Hispanic White children and those from higher …

Delayed-Interval Delivery In Multiple Gestation Pregnancies: Neonatal Mortality, Morbidity, And Development, Nicolas J Bouey, Shampa Saha, Deanne E Wilson-Costello, Matthew A Rysavy, Michele Walsh, Myra H Wyckoff, Anna Maria Hibbs

Faculty, Staff and Student Publications

OBJECTIVE: Delayed-interval delivery (DID) is the delivery of the first fetus in a multiple gestation pregnancy without prompt delivery of the remaining fetus(es). We aimed to assess infant outcomes of DID.

STUDY DESIGN: We performed a retrospective cohort study of infants born 22-28 weeks' gestation or weighing 401-1500 g. DID was defined as a passage of >24 h between the birth of firstborn and retained infants. Rates of mortality, morbidity, and developmental outcomes were compared within DID multiples, to other multiples not born by DID, and all infants in the Generic Database and follow-up datasets (excluding DID-born).

RESULTS: DID-born multiples …

Accidental Organophosphate Poisoning: A Case Series Of 2 Pediatric Coumaphos Exposures, Justin A Seltzer, Sarah Friedland, Nathan A Friedman, Garret A Winkler, Emily Foreman, Yousef Al Mubarak, Brent Buccine, Branden Engorn, Allyson Kreshak, Alicia Minns, Christian A Tomaszewski, Daniel R Lasoff, Richard F Clark

Faculty, Staff and Student Publications

INTRODUCTION: Pediatric organophosphate insecticide poisonings are rare in the United States, and life-threatening toxicity is rarely seen. We report 2 accidental ingestions of the organophosphate insecticide coumaphos that resulted in life-threatening symptoms.

CASE REPORTS: A 7-year-old boy and 10-year-old girl both presented from home after accidental ingestion of 1 "spoonful" of coumaphos 20% liquid (Asuntol; Bayer de Mexico, S.A. de C.V., Mexico D.F., Mexico). There were no other known ingestions. Both became rapidly symptomatic, with the boy developing dyspnea, vomiting, and depressed mental status and the girl developing headache and nausea. Soon afterward, the boy had witnessed cardiopulmonary arrest and …

Children And Caregiver Proxy Quality Of Life From Peanut Oral Immunotherapy Trialschildren And Caregiver Proxy Quality Of Life From Peanut Oral Immunotherapy Trials, Audrey Dunn Galvin, Andrea Vereda, Pablo Rodríguez Del Río, Antonella Muraro, Carla Jones, Robert Ryan, David Norval, Jennifer Jobrack, Aikaterini Anagnostou, Julie Wang

Faculty, Staff and Students Publications

BACKGROUND: Health-related quality of life (HRQoL) is significantly and substantially reduced in individuals with peanut allergy due to many factors associated with unanticipated or potentially fatal reactions. Further insight on the impact of peanut oral immunotherapy in managing peanut allergy on HRQoL is needed. The aim of this analysis was to assess effects of peanut (Arachis hypogaea) allergen powder-dnfp (PTAH), a biologic drug for peanut oral immunotherapy, on HRQoL from three phase 3 and two follow-on trials of PTAH.

METHODS: HRQoL assessments from participants aged 4-17 in the PALISADE (ARC003), ARC004 (PALISADE follow-on), ARTEMIS (ARC010), RAMSES (ARC007), and ARC011 (RAMSES …

A Toddler With New Seizures, Progressive White Matter Lesions, And Multifocal Microhemorrhages, Dana Tlais, Stephanie Fetzko, Nitya Gulati, Huy Brandon D Tran, Sarah Risen, Yi-Chen Lai

Faculty, Staff and Students Publications

A 16-month-old, previously healthy male is hospitalized for new onset seizures. Initial investigation is significant for enterovirus/rhinovirus respiratory infection, abnormal T2 signal predominantly in the white matter and scattered microhemorrhages on brain MRI, transaminitis, and thrombocytopenia. His symptoms initially improve on steroid therapy and he is discharged from the hospital. During the ensuing month with the tapering of the steroids, he develops new motor deficits for which he is rehospitalized. His laboratory investigation on readmission is unremarkable. However, there is significant progression of white matter lesions and microhemorrhages on repeat MRI. While in the hospital, he becomes febrile and has …

Use Of Fenoldopam In Children With Congenital Heart Disease To Decrease Fluid Balance: A Retrospective, Descriptive Study And Insights Into Predictors Of Decreased Fluid Balance, Jacqueline Rausa, Rohit S Loomba, Vincent Dorsey, Priya Chandra, Jamie S Penk, Saul Flores, Enrique G Villarreal, Stuart L Goldstein

Faculty, Staff and Students Publications

This retrospective study aimed to determine if fenoldopam is associated with a decrease in fluid balance and to define the factors that may promote this in children with a history of congenital heart disease at the cardiac intensive care unit (CICU). Patients cared from January 2014 to December 2018 in the CICU were reviewed, and those on fenoldopam infusion were identified. Patient cohort data included demographics, clinical information, laboratory results, hemodynamic and urine output measurements, and information regarding fenoldopam infusion were compared between those with and without decrease in fluid balance. Forty-six patients were identified. Patients received a starting dose …

Growth Parameters In Children With Achondroplasia: A 7-Year, Prospective, Multinational, Observational Study, Ravi Savarirayan, Melita Irving, Paul Harmatz, Borja Delgado, William R Wilcox, John Philips, Natalie Owen, Carlos A Bacino, Louise Tofts, Joel Charrow, Lynda E Polgreen, Julie Hoover-Fong, Paul Arundel, Ignacio Ginebreda, Howard M Saal, Donald Basel, Rosendo Ullot Font, Keiichi Ozono, Michael B Bober, Valerie Cormier-Daire, Kim-Hanh Le Quan Sang, Genevieve Baujat, Yasemin Alanay, Frank Rutsch, Daniel Hoernschemeyer, Klaus Mohnike, Hiroshi Mochizuki, Asako Tajima, Yumiko Kotani, David D Weaver, Klane K White, Clare Army, Kevin Larrimore, Keith Gregg, George Jeha, Claire Milligan, Elena Fisheleva, Alice Huntsman-Labed, Jonathan Day

Faculty, Staff and Students Publications

Purpose: This study was undertaken to collect baseline growth parameters in children with achondroplasia who might enroll in interventional trials of vosoritide, and to establish a historical control.

Methods: In this prospective, observational study, participants (≤17 years) underwent a detailed medical history and physical examination and were followed every 3 months until they finished participating in the study by enrolling in an interventional trial or withdrawing.

Results: A total of 363 children were enrolled (28 centers, 8 countries). Mean (SD) follow up was 20.4 (15.0) months. In participants < 1 year, mean annualized growth velocity (AGV) was 11.6 cm/year for girls and 14.6 cm/year for boys. By age 1 year, mean AGV decreased to 7.4 cm/year in girls and 7.1 cm/year in boys. By age 10 years, mean AGV decreased to 3.6 cm/year for both sexes. Mean height z-score in participants < 1 year was -2.5 for girls and -3.2 for boys and decreased up to the age 5 years (-5.3 for girls; -4.6 for boys). Girls and boys had a disproportionate upper-to-lower body segment ratio. Mean ratio was highest in participants aged < 1 year (2.9 for girls; 2.8 for boys) and decreased gradually to approximately 2 in both sexes from 4 years of age onward.

Conclusion: This study represents one of the largest datasets of prospectively collected …

A New Chapter In An Evolving Pandemic: Successful Pediatric Liver Transplantation With Sars-Cov-2+ Donors, Matthew Benjamin Goss, Daniel Hao Bin Leung, Stephanie Marie Pouch, Flor M Munoz, Elizabeth Andrea Moulton, Tyler Malcolm Mccann Lambing, Sarah Koohmaraie, Nicolas Fernando Moreno, Christine A O'Mahony, John A Goss, Nhu Thao Nguyen Galván

Faculty, Staff and Students Publications

BACKGROUND: Amid a viral pandemic with poorly understood transmissibility and pathogenicity in the pediatric patient, we report the first pediatric liver transplants utilizing allografts from SARS-CoV-2+ donors.

METHODS: We describe the outcomes of two pediatric liver transplant recipients who received organs from SARS-CoV-2 nucleic acid test-positive (NAT+) donors. Data were obtained through the respective electronic medical record system and UNet DonorNet platform.

RESULTS: The first donor was a 3-year-old boy succumbing to head trauma. One of four nasopharyngeal (NP) swabs and 1 of 3 bronchoalveolar lavage (BAL) NAT tests demonstrated SARS-CoV-2 infection before organ procurement. The second donor was a …

Progress In Gastroparesis - A Narrative Review Of The Work Of The Gastroparesis Clinical Research Consortium, Pankaj J Pasricha, Madhusudan Grover, Katherine P Yates, Thomas L Abell, Kenneth L Koch, Richard W Mccallum, Irene Sarosiek, Cheryl E Bernard, Braden Kuo, Robert Bulat, Robert J Shulman, Bruno P Chumpitazi, James Tonascia, Laura A Miriel, Laura A Wilson, Mark L Van Natta, Emily Mitchell, Frank Hamilton, Gianrico Farrugia, Henry P Parkman, Niddk/Nih Gpcrc Consortium

Faculty, Staff and Students Publications

The Gastroparesis Clinical Research Consortium is a multicenter coalition created and funded by the National Institutes of Diabetes and Digestive and Kidney Disorders, with a mission to advance understanding of the pathophysiology of gastroparesis and develop an effective treatment for patients with symptomatic gastroparesis. In this review, we summarize the results of the published Gastroparesis Clinical Research Consortium studies as a ready and convenient resource for gastroenterologists and others to provide a clear understanding of the consortium's experience and perspective on gastroparesis and related disorders.

Implementing Molecular Tuberculosis Diagnostic Methods In Limited-Resource And High-Burden Countries, Anca Vasiliu, Antonia Morita Iswari Saktiawati, Raquel Duarte, Christoph Lange, Daniela Maria Cirillo

Faculty, Staff and Students Publications

Tuberculosis (TB) is one of the deadliest infectious diseases in the world with more than a million people dying of TB each year. Accurate and timely TB diagnosis has the potential to alleviate the global TB burden; therefore, one of the pillars of the End TB Strategy developed by the World Health Organization (WHO) is the early diagnosis of TB, including universal drug-susceptibility testing (DST). The WHO emphasises the importance of DST before treatment initiation, using molecular WHO-recommended rapid diagnostic tests (mWRDs). Currently available mWRDs are nucleic acid amplification tests, line probe assays, whole genome sequencing, and targeted next-generation sequencing. …

Risk Factors And Prognostic Significance Of Anemia In Children With Hiv Infection On Antiretroviral Therapy, Joseph Lubega, Amanda Grimes, Gladstone Airewele, Shaun Bulsara, Taylor Olmsted Kim, Heather Haq, Erin Peckham-Gregory, Sebastian R Wanless, Peter Elyanu, Philippa Musoke, Mwita Lumumba, Adeodata Kekitiinwa, Mogomotsi Matshaba, Jenny Despotovic, Michael Scheurer

Faculty, Staff and Students Publications

OBJECTIVES: To establish the incidence, risk factors and prognostic effect of anemia in children living with HIV (CLWH).

DESIGN: Retrospective nested case-control study of patients 0-18 years in five centers in sub-Saharan Africa, 2004-2014.

METHODS: Incident cases of anemia were identified from electronic records and matched with CLWH without anemia. We calculated the incidence density of anemia and used conditional logistic regression to evaluate its association with risk factors, stratified by severity and type of anemia. We used a Cox proportional hazards model to evaluate the impact of anemia on survival.

RESULTS: Two thousand, one hundred and thirty-seven children were …

Foxi3 Haploinsufficiency Contributes To Low T-Cell Receptor Excision Circles And T-Cell Lymphopenia, Rajarshi Ghosh, Marita Bosticardo, Sunita Singh, Morgan Similuk, Ottavia M Delmonte, Francesca Pala, Christine Peng, Colleen Jodarski, Michael D Keller, Ivan K Chinn, Andrew K Groves, Luigi D Notarangelo, Magdalena A Walkiewicz, Javier Chinen, Vanessa Bundy

Faculty, Staff and Students Publications

BACKGROUND: Newborn screening can identify neonatal T-cell lymphopenia through detection of a low number of copies of T-cell receptor excision circles in dried blood spots collected at birth. After a positive screening result, further diagnostic testing is required to determine whether the subject has severe combined immunodeficiency or other causes of T-cell lymphopenia. Even after thorough evaluation, approximately 15% of children with a positive result of newborn screening for T-cell receptor excision circles remain genetically undiagnosed. Identifying the underlying genetic etiology is necessary to guide subsequent clinical management and family planning.

OBJECTIVE: We sought to elucidate the genetic basis of …

A Phase I/Ii Trial Of Nivolumab Plus Ipilimumab In Children And Young Adults With Relapsed/Refractory Solid Tumors: A Children's Oncology Group Study Advl1412, Kara L Davis, Elizabeth Fox, Emasenyie Isikwei, Joel M Reid, Xiaowei Liu, Charles G Minard, Stephan Voss, Stacey L Berg, Brenda J Weigel, Crystal L Mackall

Faculty, Staff and Students Publications

PURPOSE: In many cancers, nivolumab in combination with ipilimumab improves response rates compared with either agent alone, but the combination has not been evaluated in childhood cancer. We conducted a phase I/II trial of nivolumab plus ipilimumab in children and young adults with recurrent/refractory solid tumors.

PATIENTS AND METHODS: ADVL1412, Part C assessed safety of nivolumab plus ipilimumab at two dose levels (DL): DL1 1 mg/kg of each drug and DL2 3 mg/kg nivolumab plus 1 mg/kg ipilimumab. Part D evaluated response at the recommended phase II dose (RP2D) in Ewing sarcoma, rhabdomyosarcoma, and osteosarcoma. Part E tested DL3 (1 …

Long-Term Relugolix Combination Therapy For Symptomatic Uterine Leiomyomas, Ayman Al-Hendy, Andrea S Lukes, Alfred N Poindexter, Roberta Venturella, Claudio Villarroel, Laura Mckain, Yulan Li, Rachel B Wagman, Elizabeth A Stewart

Faculty, Staff and Students Publications

OBJECTIVE: In the LIBERTY 1 and LIBERTY 2 placebo-controlled trials, once-daily relugolix combination therapy reduced menstrual blood loss volume and pain in women with heavy menstrual bleeding associated with uterine leiomyomas and was well tolerated, with preservation of bone mineral density (BMD) through 24 weeks. Here we report the long-term efficacy and safety of relugolix combination therapy treatment for up to 52 weeks.

METHODS: Women with uterine leiomyoma-associated heavy menstrual bleeding who completed any treatment arm in either the LIBERTY 1 or LIBERTY 2 trial were eligible to enroll in a 28-week long-term extension study. All participants received once-daily relugolix …