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Comprehensive Molecular And Clinical Characterization Of Nup98 Fusions In Pediatric Acute Myeloid Leukemia, Eline J M Bertrums, Jenny L. Smith, Lauren Harmon, Rhonda E. Ries, Yi-Cheng J. Wang, Todd A. Alonzo, Andrew J. Menssen, Karen M. Chisholm, Amanda R. Leonti, Katherine Tarlock, Fabiana Ostronoff, Era L. Pogosova-Agadjanyan, Gertjan J L Kaspers, Henrik Hasle, Michael Dworzak, Christiane Walter, Nora Muhlegger, Cristina Morerio, Laura Pardo, Betsy Hirsch, Susana Raimondi, Todd M. Cooper, Richard Aplenc, Alan S. Gamis, Edward A. Kolb, Jason E. Farrar, Derek Stirewalt, Xiaotu Ma, Tim I. Shaw, Scott N. Furlan, Lisa Eidenschink Brodersen, Michael R. Loken, Marry M. Van Den Heuvel-Eibrink, C Michel Zwaan, Timothy J. Triche, Bianca F. Goemans, Soheil Meshinchi Aug 2023

Comprehensive Molecular And Clinical Characterization Of Nup98 Fusions In Pediatric Acute Myeloid Leukemia, Eline J M Bertrums, Jenny L. Smith, Lauren Harmon, Rhonda E. Ries, Yi-Cheng J. Wang, Todd A. Alonzo, Andrew J. Menssen, Karen M. Chisholm, Amanda R. Leonti, Katherine Tarlock, Fabiana Ostronoff, Era L. Pogosova-Agadjanyan, Gertjan J L Kaspers, Henrik Hasle, Michael Dworzak, Christiane Walter, Nora Muhlegger, Cristina Morerio, Laura Pardo, Betsy Hirsch, Susana Raimondi, Todd M. Cooper, Richard Aplenc, Alan S. Gamis, Edward A. Kolb, Jason E. Farrar, Derek Stirewalt, Xiaotu Ma, Tim I. Shaw, Scott N. Furlan, Lisa Eidenschink Brodersen, Michael R. Loken, Marry M. Van Den Heuvel-Eibrink, C Michel Zwaan, Timothy J. Triche, Bianca F. Goemans, Soheil Meshinchi

Manuscripts, Articles, Book Chapters and Other Papers

NUP98 fusions comprise a family of rare recurrent alterations in AML, associated with adverse outcomes. In order to define the underlying biology and clinical implications of this family of fusions, we performed comprehensive transcriptome, epigenome, and immunophenotypic profiling of 2,235 children and young adults with AML and identified 160 NUP98 rearrangements (7.2%), including 108 NUP98-NSD1 (4.8%), 32 NUP98-KDM5A (1.4%) and 20 NUP98-X cases (0.9%) with 13 different fusion partners. Fusion partners defined disease characteristics and biology; patients with NUP98-NSD1 or NUP98-KDM5A had distinct immunophenotypic, transcriptomic, and epigenomic profiles. Unlike the two most prevalent NUP98 fusions, NUP98-X variants are typically not …


Prevalence, Management And Efficacy Of Treatment In Portal Vein Obstruction After Paediatric Liver Transplantation: Protocol Of The Retrospective International Multicentre Portal Registry., Bader A. Alfares, Hubert P J Van Der Doef, Barbara E. Wildhaber, Thomas Casswall, Greg Nowak, Martin Delle, Denise Aldrian, Valeria Berchtold, Georg F. Vogel, Piotr Kaliciński, Malgorzata Markiewicz-Kijewska, Adam Kolesnik, Jesús Q. Bernabeu, María Mercadal Hally, Mauricio Larrarte K, Paolo Marra, Michela Bravi, Domenico Pinelli, Mureo Kasahara, Seisuke Sakamoto, Hajime Uchida, Vidyadhar Mali, Marion Aw, Stéphanie Franchi-Abella, Emmanuel Gonzales, Florent Guérin, Guillermo Cervio, Julia Minetto, Sergio Sierre, Martin De Santibañes, Victoria Ardiles, Jimmy Walker Uno, Helen Evans, David Duncan, John Mccall, Steffen Hartleif, Ekkehard Sturm, Jai Patel, Marumbo Mtegha, Raj Prasad, Cristina T. Ferreira, Luiza S. Nader, Marco Farina, Catalina Jaramillo, Manuel I. Rodriguez-Davalos, Peter Feola, Amit A. Shah, Phoebe M. Wood, Michael R. Acord, Ryan T. Fischer, Bhargava Mullapudi, Richard J. Hendrickson, Rajeev Khanna, Viniyendra Pamecha, Amar Mukund, Khalid Sharif, Girish Gupte, Simon Mcguirk, Gilda Porta, Marco Spada, Tommaso Alterio, Giuseppe Maggiore, Winita Hardikar, Marisa Beretta, Rudi Dierckx, Ruben H J De Kleine, Reinoud P H Bokkers Jul 2023

Prevalence, Management And Efficacy Of Treatment In Portal Vein Obstruction After Paediatric Liver Transplantation: Protocol Of The Retrospective International Multicentre Portal Registry., Bader A. Alfares, Hubert P J Van Der Doef, Barbara E. Wildhaber, Thomas Casswall, Greg Nowak, Martin Delle, Denise Aldrian, Valeria Berchtold, Georg F. Vogel, Piotr Kaliciński, Malgorzata Markiewicz-Kijewska, Adam Kolesnik, Jesús Q. Bernabeu, María Mercadal Hally, Mauricio Larrarte K, Paolo Marra, Michela Bravi, Domenico Pinelli, Mureo Kasahara, Seisuke Sakamoto, Hajime Uchida, Vidyadhar Mali, Marion Aw, Stéphanie Franchi-Abella, Emmanuel Gonzales, Florent Guérin, Guillermo Cervio, Julia Minetto, Sergio Sierre, Martin De Santibañes, Victoria Ardiles, Jimmy Walker Uno, Helen Evans, David Duncan, John Mccall, Steffen Hartleif, Ekkehard Sturm, Jai Patel, Marumbo Mtegha, Raj Prasad, Cristina T. Ferreira, Luiza S. Nader, Marco Farina, Catalina Jaramillo, Manuel I. Rodriguez-Davalos, Peter Feola, Amit A. Shah, Phoebe M. Wood, Michael R. Acord, Ryan T. Fischer, Bhargava Mullapudi, Richard J. Hendrickson, Rajeev Khanna, Viniyendra Pamecha, Amar Mukund, Khalid Sharif, Girish Gupte, Simon Mcguirk, Gilda Porta, Marco Spada, Tommaso Alterio, Giuseppe Maggiore, Winita Hardikar, Marisa Beretta, Rudi Dierckx, Ruben H J De Kleine, Reinoud P H Bokkers

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: Portal vein obstruction (PVO) consists of anastomotic stenosis and thrombosis, which occurs due to a progression of the former. The aim of this large-scale international study is to assess the prevalence, current management practices and efficacy of treatment in patients with PVO.

METHODS AND ANALYSIS: The Portal vein Obstruction Revascularisation Therapy After Liver transplantation registry will facilitate an international, retrospective, multicentre, observational study, with 25 centres around the world already actively involved. Paediatric patients (agedyears) with a diagnosed PVO between 1 January 2001 and 1 January 2021 after liver transplantation will be eligible for inclusion. The primary endpoints are …


Hlh-Like Toxicities Predict Poor Survival After The Use Of Tisagenlecleucel In Children And Young Adults With B-All., Kevin O. Mcnerney, Stephanie J. Si Lim, Kyle Ishikawa, Alexandra Dreyzin, Anant Vatsayan, John J. Chen, Christina Baggott, Snehit Prabhu, Holly L. Pacenta, Christine Philips, Jenna Rossoff, Heather E. Stefanski, Julie-An Talano, Amy Moskop, Michael Verneris, Douglas Myers, Nicole A. Karras, Patrick Brown, Challice L. Bonifant, Muna Qayed, Michelle Hermiston, Prakash Satwani, Christa Krupski, Amy K. Keating, Susanne H C Baumeister, Vanessa A. Fabrizio, Vasant Chinnabhandar, Emily Egeler, Sharon Mavroukakis, Kevin J. Curran, Crystal L. Mackall, Theodore W. Laetsch, Liora M. Schultz Jun 2023

Hlh-Like Toxicities Predict Poor Survival After The Use Of Tisagenlecleucel In Children And Young Adults With B-All., Kevin O. Mcnerney, Stephanie J. Si Lim, Kyle Ishikawa, Alexandra Dreyzin, Anant Vatsayan, John J. Chen, Christina Baggott, Snehit Prabhu, Holly L. Pacenta, Christine Philips, Jenna Rossoff, Heather E. Stefanski, Julie-An Talano, Amy Moskop, Michael Verneris, Douglas Myers, Nicole A. Karras, Patrick Brown, Challice L. Bonifant, Muna Qayed, Michelle Hermiston, Prakash Satwani, Christa Krupski, Amy K. Keating, Susanne H C Baumeister, Vanessa A. Fabrizio, Vasant Chinnabhandar, Emily Egeler, Sharon Mavroukakis, Kevin J. Curran, Crystal L. Mackall, Theodore W. Laetsch, Liora M. Schultz

Manuscripts, Articles, Book Chapters and Other Papers

Chimeric antigen receptor-associated hemophagocytic lymphohistiocytosis (HLH)-like toxicities (LTs) involving hyperferritinemia, multiorgan dysfunction, coagulopathy, and/or hemophagocytosis are described as occurring in a subset of patients with cytokine release syndrome (CRS). Case series report poor outcomes for those with B-cell acute lymphoblastic leukemia (B-ALL) who develop HLH-LTs, although larger outcomes analyses of children and young adults (CAYAs) with B-ALL who develop these toxicities after the administration of commercially available tisagenlecleucel are not described. Using a multi-institutional database of 185 CAYAs with B-ALL, we conducted a retrospective cohort study including groups that developed HLH-LTs, high-grade (HG) CRS without HLH-LTs, or no to low-grade …


Recruitment And Reach In A School-Based Pediatric Obesity Intervention Trial In Rural Areas., Bethany Forseth, Brittany Lancaster, Megan Olalde, Christie A. Befort, Rebecca E. Swinburne Romine, Meredith Dreyer, Kelsey Dean, Eve-Lynn Nelson, Ann M. Davis Jun 2023

Recruitment And Reach In A School-Based Pediatric Obesity Intervention Trial In Rural Areas., Bethany Forseth, Brittany Lancaster, Megan Olalde, Christie A. Befort, Rebecca E. Swinburne Romine, Meredith Dreyer, Kelsey Dean, Eve-Lynn Nelson, Ann M. Davis

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: The purpose of this study is to evaluate two recruitment strategies on schools and participant participation rates and representativeness (reach) within a pediatric obesity treatment trial tailored for families who live in rural areas.

METHODS: Recruitment of schools was evaluated based on their progress toward enrolling participants. Recruitment and reach of participants were evaluated using (1) participation rates and (2) representativeness of demographics and weight status of participants compared to eligible participants (who did not consent and enroll) and all students (regardless of eligibility). School recruitment, as well as participant recruitment and reach, were evaluated across recruitment methods comparing …


Climate Change Imperils Pediatric Health: Child Advocacy Through Fossil Fuel Divestment., Sandra H. Jee, Elizabeth Friedman, Ruth A. Etzel, Vi T. Nguyen, Todd L. Sack, Kathi J. Kemper Jun 2023

Climate Change Imperils Pediatric Health: Child Advocacy Through Fossil Fuel Divestment., Sandra H. Jee, Elizabeth Friedman, Ruth A. Etzel, Vi T. Nguyen, Todd L. Sack, Kathi J. Kemper

Manuscripts, Articles, Book Chapters and Other Papers

Climate change poses an existential threat to children's health. Divestment of ownership stakes in fossil fuel companies is one tool available to pediatricians to address climate change. Pediatricians are trusted messengers regarding children's health and therefore bear a unique responsibility to advocate for climate and health policies that affect children. Among the impacts of climate change on pediatric patients are allergic rhinitis and asthma; heat-related illnesses; premature birth; injuries from severe storms and fires; vector-borne diseases; and mental illnesses. Children are disproportionately affected as well by climate-related displacement of populations, drought, water shortages, and famine. The human-generated burning of fossil …


Measurable Residual Disease And Fusion Partner Independently Predict Survival And Relapse Risk In Childhood Kmt2a-Rearranged Acute Myeloid Leukemia: A Study By The International Berlin-Frankfurt-Münster Study Group, Romy E. Van Weelderen, Kim Klein, Christine J. Harrison, Yilin Jiang, Jonas Abrahamsson, Nira Arad-Cohen, Emmanuelle Bart-Delabesse, Barbara Buldini, Barbara De Moerloose, Michael N. Dworzak, Sarah Elitzur, José M. Fernández Navarro, Robert B. Gerbing, Bianca F. Goemans, Hester A. De Groot-Kruseman, Erin M. Guest, Shau-Yin Ha, Henrik Hasle, Charikleia Kelaidi, Hélène Lapillonne, Guy Leverger, Franco Locatelli, Riccardo Masetti, Takako Miyamura, Ulrika Norén-Nyström, Sophia Polychronopoulou, Mareike Rasche, Jeffrey E. Rubnitz, Jan Stary, Anne Tierens, Daisuke Tomizawa, C Michel Zwaan, Gertjan J L Kaspers Jun 2023

Measurable Residual Disease And Fusion Partner Independently Predict Survival And Relapse Risk In Childhood Kmt2a-Rearranged Acute Myeloid Leukemia: A Study By The International Berlin-Frankfurt-Münster Study Group, Romy E. Van Weelderen, Kim Klein, Christine J. Harrison, Yilin Jiang, Jonas Abrahamsson, Nira Arad-Cohen, Emmanuelle Bart-Delabesse, Barbara Buldini, Barbara De Moerloose, Michael N. Dworzak, Sarah Elitzur, José M. Fernández Navarro, Robert B. Gerbing, Bianca F. Goemans, Hester A. De Groot-Kruseman, Erin M. Guest, Shau-Yin Ha, Henrik Hasle, Charikleia Kelaidi, Hélène Lapillonne, Guy Leverger, Franco Locatelli, Riccardo Masetti, Takako Miyamura, Ulrika Norén-Nyström, Sophia Polychronopoulou, Mareike Rasche, Jeffrey E. Rubnitz, Jan Stary, Anne Tierens, Daisuke Tomizawa, C Michel Zwaan, Gertjan J L Kaspers

Manuscripts, Articles, Book Chapters and Other Papers

Purpose: A previous study by the International Berlin-Frankfurt-Münster Study Group (I-BFM-SG) on childhood KMT2A-rearranged (KMT2A-r) AML demonstrated the prognostic value of the fusion partner. This I-BFM-SG study investigated the value of flow cytometry-based measurable residual disease (flow-MRD) and evaluated the benefit of allogeneic stem-cell transplantation (allo-SCT) in first complete remission (CR1) in this disease.

Methods: A total of 1,130 children with KMT2A-r AML, diagnosed between January 2005 and December 2016, were assigned to high-risk (n = 402; 35.6%) or non-high-risk (n = 728; 64.4%) fusion partner-based groups. Flow-MRD levels at both end of induction 1 (EOI1) …


The Child Healthcare At Mater Pediatric Study (Champs): A 2-Arm Cluster Randomized Control Trial Of Group Well Child Care For Mothers In Treatment For Opioid Use Disorder And Their Children, Vanessa L. Short, Diane J. Abatemarco, Erica Sood, Dennis J. Hand, Meghan Gannon, Jobayer Hossain, Neera K. Goyal May 2023

The Child Healthcare At Mater Pediatric Study (Champs): A 2-Arm Cluster Randomized Control Trial Of Group Well Child Care For Mothers In Treatment For Opioid Use Disorder And Their Children, Vanessa L. Short, Diane J. Abatemarco, Erica Sood, Dennis J. Hand, Meghan Gannon, Jobayer Hossain, Neera K. Goyal

Department of Pediatrics Faculty Papers

BACKGROUND: Studies suggest that group-based well child care-a shared medical appointment where families come together as a group to receive pediatric primary care-increases patient-reported satisfaction and adherence to recommended care. Evidence supporting the use of group well child care for mothers with opioid use disorder, however, is lacking. The overall objective of the Child Healthcare at MATER Pediatric Study (CHAMPS) trial is to evaluate a group model of well child care for mothers with opioid use disorder and their children.

METHODS: CHAMPS is a single-site 2-arm cluster randomized controlled trial. A total of 108 mother-child dyads will be enrolled into …


A Lifestyle Modification Intervention In Hispanic Adolescents With Nonalcoholic Fatty Liver Disease, Brianne Miller May 2023

A Lifestyle Modification Intervention In Hispanic Adolescents With Nonalcoholic Fatty Liver Disease, Brianne Miller

Evidence-Based Practice Project Reports

U.S. population studies have confirmed that the Hispanic population has the highest prevalence and the worst prognosis for non-alcoholic fatty liver disease (NAFLD) (Katz et al., 2021). The purpose of this evidence-based practice project was to implement a multicomponent, lifestyle modification intervention in Hispanic pediatric primary care patients to combat the disease process and sequela of NAFLD. The PICOT question for this project was: In Hispanic adolescents aged 10-21 with obesity and NAFLD (P), what is the effect of a multicomponent, lifestyle modification intervention (I), on alanine aminotransferase (ALT) (O), over a 12-week time period (T)? A comprehensive literature search …


Use, Durability, And Risks For Discontinuation Of Initial And Subsequent Biologics In A Large Pediatric-Onset Ibd Cohort., Jess L. Kaplan, Chunyan Liu, Eileen C. King, Julie A. Bass, Ashish S. Patel, Jeanne Tung, Shiran Chen, Trevor Lissoos, Ninfa Candela, Shehzad Saeed, Richard B. Colletti, Improvecarenow Network May 2023

Use, Durability, And Risks For Discontinuation Of Initial And Subsequent Biologics In A Large Pediatric-Onset Ibd Cohort., Jess L. Kaplan, Chunyan Liu, Eileen C. King, Julie A. Bass, Ashish S. Patel, Jeanne Tung, Shiran Chen, Trevor Lissoos, Ninfa Candela, Shehzad Saeed, Richard B. Colletti, Improvecarenow Network

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Biologic medications are recommended for treatment of moderately-to-severely active Crohn disease (CD) or ulcerative colitis (UC) in children. However, many patients require sequential biologic treatment because of nonresponse or loss of response to the initial biologic.

METHODS: We analyzed pediatric inflammatory bowel disease (IBD) data from the ImproveCareNow Network registry between May 2006 and September 2016, including time to biologic initiation, choice of first subsequent biologics, biologic durability, and reasons for discontinuation.

RESULTS: Of 17,649 patients with IBD [CD: 12,410 (70%); UC: 5239 (30%)], 7585 (43%) were treated with a biologic agent before age 18 (CD: 50%; UC: 25%). …


Real-World Experience Of Pediatric Patients Treated With Peanut (Arachis Hypogaea) Allergen Powder-Dnfp., Jay M. Portnoy, Jodi Shroba, Stephen Tilles, Hela Romdhani, Sarah M. Donelson, Dominick Latremouille-Viau, Rebecca Bungay, Kathleen Chen, William Mccann May 2023

Real-World Experience Of Pediatric Patients Treated With Peanut (Arachis Hypogaea) Allergen Powder-Dnfp., Jay M. Portnoy, Jodi Shroba, Stephen Tilles, Hela Romdhani, Sarah M. Donelson, Dominick Latremouille-Viau, Rebecca Bungay, Kathleen Chen, William Mccann

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Peanut (Arachis hypogaea) allergen powder-dnfp (PTAH) is the first oral immunotherapy indicated for children aged 4 to 17 years with peanut allergy. There are limited real-world data on patients treated with PTAH.

OBJECTIVE: To characterize pediatric patients treated with PTAH and associated treatment patterns in US clinical practice.

METHODS: US-based physicians with allergy and immunology training treating patients with peanut allergy aged 4 to 17 years with PTAH were recruited from an existing physician panel and completed an online case report form (October to December 2021) with data abstracted from patient medical charts. Physician practice circumstances, patient characteristics, and …


What To Look For In Relationships: Development, Inter-Rater Reliability, And Initial Validity Estimates For A Young Child-Caregiver Relationship Assessment, Joy D. Osofsky, Margaret A. Fields-Olivieri, Andrew L. Frazer, Rebecca A. Graham, Bethany H. Mccurdy, Carl F. Weems Mar 2023

What To Look For In Relationships: Development, Inter-Rater Reliability, And Initial Validity Estimates For A Young Child-Caregiver Relationship Assessment, Joy D. Osofsky, Margaret A. Fields-Olivieri, Andrew L. Frazer, Rebecca A. Graham, Bethany H. Mccurdy, Carl F. Weems

School of Medicine Faculty Publications

Introduction: Observational assessments are important for understanding a range of behaviors and emotions in the young child-caregiver relationship. This paper provides initial data on a multidimensional assessment for professionals who work with young children and their caregivers, the What to Look for in Relationships (WLR). The WLR was designed to assist providers in evaluating strengths and areas for improvement in five areas of young child-caregiver relationship dimensions. This paper reports on the development, interrater reliability, initial convergent and discriminant validity, and incremental utility of the scales. Methods: Data were collected from caregiver-child dyads, who participated in a semi-structured observational caregiver-child …


Protocol And Biomarker Strategy For A Multi-Site Randomized Controlled Trial Examining Biological Mechanisms And Dosing Of Active Music Engagement In Children With Acute Lymphoblastic Leukemia And Lymphoma And Parents., Sheri L. Robb, Kristen A. Russ, Steven J. Holochwost, Kristin Stegenga, Susan M. Perkins, Seethal A. Jacob, Amanda K. Henley, Jessica A. Maclean Mar 2023

Protocol And Biomarker Strategy For A Multi-Site Randomized Controlled Trial Examining Biological Mechanisms And Dosing Of Active Music Engagement In Children With Acute Lymphoblastic Leukemia And Lymphoma And Parents., Sheri L. Robb, Kristen A. Russ, Steven J. Holochwost, Kristin Stegenga, Susan M. Perkins, Seethal A. Jacob, Amanda K. Henley, Jessica A. Maclean

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Music therapy is a standard palliative care service in many pediatric and adult hospitals; however, most research has focused on the use of music to improve psychosocial dimensions of health, without considering biological dimensions. This study builds on prior work examining psychosocial mechanisms of action underlying an Active Music Engagement (AME) intervention, designed to help manage emotional distress and improve positive health outcomes in young children with cancer and parents (caregivers), by examining its effects on biomarkers of stress and immune function.

METHODS: This two-group randomized controlled trial (R01NR019190) is designed to examine biological mechanisms of effect and dose-response …


Three-Year Update Of Tisagenlecleucel In Pediatric And Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia In The Eliana Trial., Theodore W. Laetsch, Shannon L. Maude, Susana Rives, Hidefumi Hiramatsu, Henrique Bittencourt, Peter Bader, André Baruchel, Michael Boyer, Barbara De Moerloose, Muna Qayed, Jochen Buechner, Michael A. Pulsipher, Douglas Myers, Heather E. Stefanski, Paul L. Martin, Eneida Nemecek, Christina Peters, Gregory Yanik, Seong Lin Khaw, Kara L. Davis, Joerg Krueger, Adriana Balduzzi, Nicolas Boissel, Ranjan Tiwari, Darragh O'Donovan, Stephan A. Grupp Mar 2023

Three-Year Update Of Tisagenlecleucel In Pediatric And Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia In The Eliana Trial., Theodore W. Laetsch, Shannon L. Maude, Susana Rives, Hidefumi Hiramatsu, Henrique Bittencourt, Peter Bader, André Baruchel, Michael Boyer, Barbara De Moerloose, Muna Qayed, Jochen Buechner, Michael A. Pulsipher, Douglas Myers, Heather E. Stefanski, Paul L. Martin, Eneida Nemecek, Christina Peters, Gregory Yanik, Seong Lin Khaw, Kara L. Davis, Joerg Krueger, Adriana Balduzzi, Nicolas Boissel, Ranjan Tiwari, Darragh O'Donovan, Stephan A. Grupp

Manuscripts, Articles, Book Chapters and Other Papers

In the primary analysis of the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849), tisagenlecleucel provided an overall remission rate of 81% in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), with 59% of responders remaining relapse-free at 12 months. Here, we report an update on efficacy, safety, and patient-reported quality of life in 79 pediatric and young adult patients with R/R B-ALL following a median follow-up of 38.8 months. The overall remission rate was 82%. The median event-free survival was 24 months, and the median overall survival was not reached. Event-free …


Risk And Resilient Functioning Of Families Of Children With Cancer During The Covid-19 Pandemic., Renee Gilbert, Carolyn R Bates, Devanshi Khetawat, Meredith Dreyer Gillette, Rachel Moore Mar 2023

Risk And Resilient Functioning Of Families Of Children With Cancer During The Covid-19 Pandemic., Renee Gilbert, Carolyn R Bates, Devanshi Khetawat, Meredith Dreyer Gillette, Rachel Moore

Manuscripts, Articles, Book Chapters and Other Papers

Previous literature highlights the impact of COVID-19 on family functioning. Less is known about the impact of the pandemic on families of pediatric cancer patients. In order to determine universal and unique risk and resilience factors of these families during the pandemic, a qualitative analysis was conducted on families currently receiving cancer treatment at a Midwestern hospital. Results of the data analysis depict ways in which these families have been impacted by and have adapted to COVID-19. These findings suggest that families of pediatric cancer patients have unique experiences in the context of COVID-19, in addition to universal experiences outlined …


Higher Doses Of Tisagenlecleucel Are Associated With Improved Outcomes: A Report From The Pediatric Real-World Car Consortium., Heather E. Stefanski, Anne Eaton, Christina Baggott, Jenna Rossoff, Michael R. Verneris, Snehit Prabhu, Holly L. Pacenta, Christine L. Phillips, Julie-An Talano, Amy Moskop, Steven P. Margossian, Douglas Myers, Nicole A. Karras, Patrick A. Brown, Muna Qayed, Michelle Hermiston, Prakash Satwani, M Christa Krupski, Amy K. Keating, Rachel Wilcox, Cara A. Rabik, Vanessa A. Fabrizio, Vasant Chinnabhandar, A Yasemin Goksenin, Kevin J. Curran, Crystal L. Mackall, Theodore W. Laetsch, Liora M. Schultz Feb 2023

Higher Doses Of Tisagenlecleucel Are Associated With Improved Outcomes: A Report From The Pediatric Real-World Car Consortium., Heather E. Stefanski, Anne Eaton, Christina Baggott, Jenna Rossoff, Michael R. Verneris, Snehit Prabhu, Holly L. Pacenta, Christine L. Phillips, Julie-An Talano, Amy Moskop, Steven P. Margossian, Douglas Myers, Nicole A. Karras, Patrick A. Brown, Muna Qayed, Michelle Hermiston, Prakash Satwani, M Christa Krupski, Amy K. Keating, Rachel Wilcox, Cara A. Rabik, Vanessa A. Fabrizio, Vasant Chinnabhandar, A Yasemin Goksenin, Kevin J. Curran, Crystal L. Mackall, Theodore W. Laetsch, Liora M. Schultz

Manuscripts, Articles, Book Chapters and Other Papers

Remarkable complete response rates have been shown with tisagenlecleucel, a chimeric antigen receptor (CAR) T-cell therapy targeting CD19, in patients up to age 26 years with refractory/relapsed B-cell acute lymphoblastic leukemia; it is US Food and Drug Administration approved for this indication. Currently, patients receive a single dose of tisagenlecleucel across a wide dose range of 0.2 to 5.0 × 106 and 0.1 to 2.5 × 108 CAR T cells per kg for patients ≤50 and >50 kg, respectively. The effect of cell dose on survival and remission is not yet well established. Our primary goal was to determine if …


Tobacco Exposure In Adults And Children With Proteinuric Glomerulopathies: A Neptune Cohort Study., Linda Wang, Bayle Smith-Salzberg, Kevin Ec Meyers, Dorey A. Glenn, Katherine R. Tuttle, Vimal K. Derebail, Tammy M. Brady, Keisha Gibson, Abigail R. Smith, Michelle M. O'Shaughnessy, Tarak Srivastava, Gentzon Hall, Jarcy Zee, Markus Bitzer, Christine B. Sethna Feb 2023

Tobacco Exposure In Adults And Children With Proteinuric Glomerulopathies: A Neptune Cohort Study., Linda Wang, Bayle Smith-Salzberg, Kevin Ec Meyers, Dorey A. Glenn, Katherine R. Tuttle, Vimal K. Derebail, Tammy M. Brady, Keisha Gibson, Abigail R. Smith, Michelle M. O'Shaughnessy, Tarak Srivastava, Gentzon Hall, Jarcy Zee, Markus Bitzer, Christine B. Sethna

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Tobacco exposure has been recognized as a risk factor for cardiovascular disease (CVD) and progression of kidney disease. Patients with proteinuric glomerulopathies are at increased risk for cardiovascular morbidity and mortality. Multiple studies have linked tobacco exposure to CVD and chronic kidney disease, but the relationships between smoking and proteinuric glomerulopathies in adults and children have not been previously explored.

METHODS: Data from the Nephrotic Syndrome Study Network (NEPTUNE), a multi-center prospective observational study of participants with proteinuric glomerulopathies, was analyzed. 371 adults and 192 children enrolled in NEPTUNE were included in the analysis. Self-reported tobacco exposure was classified …


Bnt162b2 Mrna Vaccination Against Coronavirus Disease 2019 Is Associated With A Decreased Likelihood Of Multisystem Inflammatory Syndrome In Children Aged 5-18 Years-United States, July 2021 - April 2022., Laura D. Zambrano, Margaret M. Newhams, Samantha M. Olson, Natasha B. Halasa, Ashley M. Price, Amber O. Orzel, Cameron C. Young, Julie A. Boom, Leila C. Sahni, Aline B. Maddux, Katherine E. Bline, Satoshi Kamidani, Keiko M. Tarquinio, Kathleen Chiotos, Jennifer E. Schuster, Melissa L. Cullimore, Sabrina M. Heidemann, Charlotte V. Hobbs, Ryan A. Nofziger, Pia S. Pannaraj, Melissa A. Cameron, Tracie C. Walker, Stephanie P. Schwartz, Kelly N. Michelson, Bria M. Coates, Heidi R. Flori, Elizabeth H. Mack, Laura Smallcomb, Shira J. Gertz, Samina S. Bhumbra, Tamara T. Bradford, Emily R. Levy, Michele Kong, Katherine Irby, Natalie Z. Cvijanovich, Matt S. Zinter, Cindy Bowens, Hillary Crandall, Janet R. Hume, Manish M. Patel, Angela P. Campbell, Adrienne G. Randolph, Overcoming Covid-19 Investigators Feb 2023

Bnt162b2 Mrna Vaccination Against Coronavirus Disease 2019 Is Associated With A Decreased Likelihood Of Multisystem Inflammatory Syndrome In Children Aged 5-18 Years-United States, July 2021 - April 2022., Laura D. Zambrano, Margaret M. Newhams, Samantha M. Olson, Natasha B. Halasa, Ashley M. Price, Amber O. Orzel, Cameron C. Young, Julie A. Boom, Leila C. Sahni, Aline B. Maddux, Katherine E. Bline, Satoshi Kamidani, Keiko M. Tarquinio, Kathleen Chiotos, Jennifer E. Schuster, Melissa L. Cullimore, Sabrina M. Heidemann, Charlotte V. Hobbs, Ryan A. Nofziger, Pia S. Pannaraj, Melissa A. Cameron, Tracie C. Walker, Stephanie P. Schwartz, Kelly N. Michelson, Bria M. Coates, Heidi R. Flori, Elizabeth H. Mack, Laura Smallcomb, Shira J. Gertz, Samina S. Bhumbra, Tamara T. Bradford, Emily R. Levy, Michele Kong, Katherine Irby, Natalie Z. Cvijanovich, Matt S. Zinter, Cindy Bowens, Hillary Crandall, Janet R. Hume, Manish M. Patel, Angela P. Campbell, Adrienne G. Randolph, Overcoming Covid-19 Investigators

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Multisystem inflammatory syndrome in children (MIS-C), linked to antecedent severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, is associated with considerable morbidity. Prevention of SARS-CoV-2 infection or coronavirus disease 2019 (COVID-19) by vaccination might also decrease MIS-C likelihood.

METHODS: In a multicenter, case-control, public health investigation of children ages 5-18 years hospitalized from 1 July 2021 to 7 April 2022, we compared the odds of being fully vaccinated (2 doses of BNT162b2 vaccine ≥28 days before hospital admission) between MIS-C case-patients and hospital-based controls who tested negative for SARS-CoV-2. These associations were examined by age group, timing of vaccination, …


Circulation Of Rhinoviruses And/Or Enteroviruses In Pediatric Patients With Acute Respiratory Illness Before And During The Covid-19 Pandemic In The Us., Danielle A. Rankin, Andrew J. Spieker, Ariana Perez, Anna L. Stahl, Herdi K. Rahman, Laura S. Stewart, Jennifer E. Schuster, Joana Y. Lively, Zaid Haddadin, Varvara Probst, Marian G. Michaels, John V. Williams, Julie A. Boom, Leila C. Sahni, Mary A. Staat, Elizabeth P. Schlaudecker, Monica M. Mcneal, Christopher J. Harrison, Geoffrey A. Weinberg, Peter G. Szilagyi, Janet A. Englund, Eileen J. Klein, Susan I. Gerber, Meredith Mcmorrow, Brian Rha, James D. Chappell, Rangaraj Selvarangan, Claire M. Midgley, Natasha B. Halasa, Nvsn Network Investigators Feb 2023

Circulation Of Rhinoviruses And/Or Enteroviruses In Pediatric Patients With Acute Respiratory Illness Before And During The Covid-19 Pandemic In The Us., Danielle A. Rankin, Andrew J. Spieker, Ariana Perez, Anna L. Stahl, Herdi K. Rahman, Laura S. Stewart, Jennifer E. Schuster, Joana Y. Lively, Zaid Haddadin, Varvara Probst, Marian G. Michaels, John V. Williams, Julie A. Boom, Leila C. Sahni, Mary A. Staat, Elizabeth P. Schlaudecker, Monica M. Mcneal, Christopher J. Harrison, Geoffrey A. Weinberg, Peter G. Szilagyi, Janet A. Englund, Eileen J. Klein, Susan I. Gerber, Meredith Mcmorrow, Brian Rha, James D. Chappell, Rangaraj Selvarangan, Claire M. Midgley, Natasha B. Halasa, Nvsn Network Investigators

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Rhinoviruses and/or enteroviruses, which continued to circulate during the COVID-19 pandemic, are commonly detected in pediatric patients with acute respiratory illness (ARI). Yet detailed characterization of rhinovirus and/or enterovirus detection over time is limited, especially by age group and health care setting.

OBJECTIVE: To quantify and characterize rhinovirus and/or enterovirus detection before and during the COVID-19 pandemic among children and adolescents seeking medical care for ARI at emergency departments (EDs) or hospitals.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study used data from the New Vaccine Surveillance Network (NVSN), a multicenter, active, prospective surveillance platform, for pediatric patients who sought …


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 …


Prospective Evaluation Of The Fungitell® (1→3) Beta-D-Glucan Assay As A Diagnostic Tool For Invasive Fungal Disease In Pediatric Allogeneic Hematopoietic Cell Transplantation: A Report From The Children's Oncology Group, William R Otto, Christopher C Dvorak, Craig L K Boge, Luis Ostrosky-Zeichner, Adam J Esbenshade, Michael L Nieder, Sarah Alexander, William J Steinbach, Ha Dang, Doojduen Villaluna, Lu Chen, Micah Skeens, Theoklis E Zaoutis, Lillian Sung, Brian T Fisher Feb 2023

Prospective Evaluation Of The Fungitell® (1→3) Beta-D-Glucan Assay As A Diagnostic Tool For Invasive Fungal Disease In Pediatric Allogeneic Hematopoietic Cell Transplantation: A Report From The Children's Oncology Group, William R Otto, Christopher C Dvorak, Craig L K Boge, Luis Ostrosky-Zeichner, Adam J Esbenshade, Michael L Nieder, Sarah Alexander, William J Steinbach, Ha Dang, Doojduen Villaluna, Lu Chen, Micah Skeens, Theoklis E Zaoutis, Lillian Sung, Brian T Fisher

Journal Articles

BACKGROUND: Invasive fungal disease (IFD) is a major source of morbidity and mortality for hematopoietic cell transplant (HCT) recipients. Non-invasive biomarkers, such as the beta-D-glucan assay, may improve the diagnosis of IFD. The objective was to define the utility of surveillance testing using Fungitell® beta-D-glucan (BDG) assay in children receiving antifungal prophylaxis in the immediate post-HCT period.

METHODS: Weekly surveillance blood testing with the Fungitell® BDG assay was performed during the early post-HCT period in the context of a randomized trial of children, adolescents, and young adults undergoing allogeneic HCT allocated to triazole or caspofungin prophylaxis. Positivity was defined at …


A Critical Evaluation Of Oral Baclofen In Pediatric Patients With Cerebral Palsy., Matthew J. Mclaughlin, Mark T. Fisher Jan 2023

A Critical Evaluation Of Oral Baclofen In Pediatric Patients With Cerebral Palsy., Matthew J. Mclaughlin, Mark T. Fisher

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.


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 …