Pediatrics Commons^™

Hla-Dr Expression At Diagnosis Is Not Predictive Of Relapse Risk Post Allogeneic Hematopoietic Stem Cell Transplantation For Pediatric Acute Myeloid Leukemia: A Report From The Children's Oncology Group., Vasant Chinnabhandar, Michael R. Verneris, Kirk R. Schultz, Alan S. Gamis, Richard Aplenc, Chad A. Hudson, Todd A. Alonzo, Robert B. Gerbing, John T. Horan, Joseph H. Chewning

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.

Pre-Implementation Factors For Active Music Engagement (Ame): Barriers And Facilitators To Integrating An Evidence-Based Dyadic Intervention Into Pediatric Oncology Care., Kristin M. Story, Elizabeth Harman, Kristin Stegenga, Jelena Golubovic, Sheri L. Robb

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Pediatric cancer treatment is a difficult and distressing experience that often results in sustained, interrelated distress in young children and parents, and traumatic stress symptoms for some parents. Few evidence-based interventions address this dyadic distress. Active Music Engagement (AME) is a manualized, evidence-based music play intervention that significantly reduces distress in child/parent dyads and mitigates traumatic stress and improves well-being among highly distressed parents and those experiencing sociodemographic risk. Successfully integrating AME into standard care requires a systematic understanding of implementation factors. This study aimed to identify site-specific barriers and facilitators that influenced successful AME delivery during a recently …

Current Practices Of Index Hospitalization Anticoagulant Use In Pediatric Patients With Acute Myeloid Leukemia., Abbey Elsbernd, Natalie Grills, James W. Antoon, Kathyrn Kyler, Lucas E. Orth, Timothy J. Savage, Matt Hall, Jennifer Goldman, Shannon L. Carpenter

Manuscripts, Articles, Book Chapters and Other Papers

Management and prevention of thromboembolism (TE) in pediatric acute myeloid leukemia (AML) are challenging because of prolonged periods of thrombocytopenia and associated bleeding. There are no guidelines for prophylactic or therapeutic anticoagulation for children with AML. This study aimed to understand the use of anticoagulation and its association with bleeding events (BEs) during index hospitalization in children with AML, both with and without thromboses. A retrospective cohort study was performed using the Pediatric Health Information System administrative database of index hospitalizations for patients aged 1 to 21 years from 2016 to 2024 with the diagnosis of AML across 49 children's …

Gene Expression And Apoptotic Responses To Panobinostat Treatment And Yap Knockdown In Ewing Sarcoma Cells, Luna Collazo-Garcia, Alejandra Favela Santos, Madeline Torres-Salazar, Isabella Toscano

Posters - 2026

Ewing sarcoma is an aggressive pediatric cancer driven by abnormal gene expression caused by the EWS-FLI1 fusion protein

Usually treated with intensive treatments such as chemotherapy and radiation

Outcomes remain poor for metastatic disease, highlighting the need for new therapeutic strategies

The Hippo signaling pathway regulates cell proliferation and survival through the transcriptional co-activator YAP1

Clostridioides Difficile Colonization And Infection In Pediatric Oncology And Stem Cell Transplant Patients, Daniel N. Willis, Erik R. Dubberke, Robert J. Hayashi, Phillip I. Tarr, David B. Haslam, Tiffany Hink, Jingqin Luo, Yu Tao, Amruta Padhye, Erin Hall, Gregory A. Storch

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Pediatric oncology and hematopoietic stem cell transplant (HSCT) patients have elevated risk for Clostridioides difficile infection (CDI), which can prolong hospitalization and delay chemotherapy. Colonization is an important prelude to symptomatic CDI. We sought to characterize colonization status in these patients.

METHODS: We retrospectively studied 276 stools longitudinally collected over 34 months from 32 HSCT and 12 oncology patients treated at a single tertiary center. Specimens were cultured for C difficile and compared by whole genome sequencing. The fecal microbiome was characterized by 16S rRNA gene sequencing.

RESULTS: Baseline cultures were positive in 16 (50%) HSCT patients and 2 …

Integrated Multi-Omic Analysis Reveals Novel Subtype-Specific Regulatory Interactions In Pediatric B-Cell Acute Lymphoblastic Leukemia., Irina Pushel, Zachary S. Clark, Lisa A. Lansdon, Byunggil Yoo, Michaella J. Rekowski, Nicole Wood, Michael P. Washburn, Midhat S. Farooqi

Manuscripts, Articles, Book Chapters and Other Papers

Background/Objectives: Molecular subtyping of pediatric B-cell acute lymphoblastic leukemia (B-ALL) has improved patient outcomes through stratification and selection of targeted therapies. Despite extensive genomic and transcriptomic profiling of this cancer, few studies to date have characterized the proteomic landscape, although proteins are the direct targets of many therapeutic agents. Methods: In this study, we demonstrate the utility of multi-omic integration of global transcriptomic, proteomic, and phosphoproteomic profiles of samples from patients diagnosed with either of two B-ALL subtypes-Ph-like (BCR::ABL1-like) and ETV6::RUNX1. Through individual and multi-omic analysis, we recapitulate known transcriptomic findings and identify novel subtype-specific …

Clinical Validation Of Targeted Somatic Structural Variant Detection In Pediatric Cancer Via Long-Read Sequencing, Aravindh Nagarajan, Byunggil Yoo, Emily Farrow, John Herriges, Lei Zhang, Elena Repnikova, Tomi Pastinen, Carol J. Saunders, Midhat Farooqi, Lisa Lansdon

Posters

Introduction: Structural variants (SVs) leading to gene fusions are one of the most common classes of driver mutations in pediatric cancer. Clinical SV detection relies on a combination of techniques including karyotyping, FISH, microarray and RNA fusion testing, and, more recently, optical genome mapping. SV detection via long-read genome sequencing (lr-GS) offers the advantage of breakpoint and complex event resolution; however, its implementation has been limited by the lack of an optimized somatic SV caller, among other factors. Here we describe the clinical validation of lr-GS for targeted somatic SV detection in pediatric cancer using Severus, a newly available somatic …

Cancer Treatment Induced Skeletal Muscle Loss As A Predictor Of Late Cardiotoxicity In Aya: A Pilot Study, Savannah V. Rauschendorfer, Efstratios Koutroumpakis, Behrang Amini, Juhee Song, Michelle Hildebrandt, Anita Deswal Md Mph, J. Andrew Livingston, Michael Roth, Eugenie S. Kleinerman Md

Cancer Survivorship Research Retreat

No abstract provided.

Liberalizing Hospital Proximity Requirements For Children/Young Adults With Low-Burden B-All Receiving Tisagenlecleucel., Lauren E. Appell, Christina Baggott, Khanh Nguyen, Snehit Prabhu, Holly Pacenta, Samuel John, Vanessa A. Fabrizio, Christine L. Phillips, Jenna Rossoff, Julie-An Talano, Amy Moskop, Rachel Phelan, Susanne H C Baumeister, Michael R. Verneris, Erin Hall, Gary Douglas Myers, Nicole A. Karras, Stacy L. Cooper, Muna Qayed, Sunil S. Raikar, Lena Winestone, Michelle Hermiston, Prakash Satwani, Christa Krupski, Amy K Keating, Danielle N. Friedman, Kevin J. Curran, Alex Hoover, Margaret L. Macmillan, Christen L. Ebens, Archana Ramgopal, April L. Rahrig, Crystal L. Mackall, Theodore W. Laetsch, Kevin Mcnerney, Liora M. Schultz

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.

Leukemia, Children's Health School Services

School Guides

Educator guide about how to support students with leukemia.

Lymphoma, Children's Health School Services

School Guides

Educator guide about how to support students with lymphoma.

Brain Tumors, Children's Health School Services

School Guides

Educator guide about how to support students with brain tumors.

Osteosarcoma, Children's Health School Services

School Guides

Educator guide about how to support students with osteosarcoma.

Investigation Of A Global Mouse Methylome Atlas Reveals Subtype-Specific Copy Number Alterations In Pediatric Cancer Models., Melanie Schoof, Tuyu Zheng, Martin Sill, Roland Imle, Alessia Cais, Lea Altendorf, Alicia Fürst, Nina Hofmann, Kati Ernst, Dominik Vonficht, Kenneth Chun-Ho Chan, Tim Holland-Letz, Andreas Postlmayr, Ryo Shiraishi, Wanchen Wang, Alaide Morcavallo, Michael Spohn, Carolin Göbel, Judith Niesen, Levke-Sophie Peter, Franck Bourdeaut, Zhi-Yan Han, Yanxin Pei, Najiba Murad, Fredrik J. Swartling, Jessica Taylor, Monika Yadav, Garrett R. Gibson, Richard J. Gilbertson, Matthias Dottermusch, Rajanya Roy, Kornelius Kerl, Rainer Glass, Jiying Cheng, Martin A. Horstmann, Gerrit Wolters-Eisfeld, Haotian Zhao, Dominik Sturm, Viveka Nand Yadav, Louis Chesler, Simon Haas, William A. Weiss, Paul A. Northcott, Lena M. Kutscher, Ana Guerreiro Stucklin, Olivier Ayrault, Julia E. Neumann, Daisuke Kawauchi, David T W Jones, Kristian Pajtler, Ana Banito, Stefan M. Pfister, Ulrich Schüller, Marc Zuckermann

Manuscripts, Articles, Book Chapters and Other Papers

Copy number alterations (CNAs) are hallmarks of cancer, yet investigation of their oncogenic role has been hindered by technical limitations and missing model systems. Here we generated a genome-wide DNA methylation and CNA atlas of 106 genetic mouse models across 31 pediatric tumor types, including 18 new models for pediatric glioma. We demonstrated their epigenetic resemblance to human disease counterparts and identified entity-specific patterns of immune infiltration. We discovered that mouse tumors harbor highly recurrent CNA signatures that occur distinctly based on the tumor subgroup and driving oncogene and showed that these CNAs share syntenic regions with the matching human …

Using A Heuristic Tool To Improve Symptom Self-Management In Adolescents And Young Adults With Cancer: Protocol For A Randomized Controlled Trial., Suzanne Ameringer, Grace Hodges, R K Elswick, Lauri Linder, Catherine Fiona Macpherson, Kristin Stegenga

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Adolescents and young adults (AYAs) with cancer experience multiple distressing symptoms during treatment, yet few developmentally relevant resources have been developed to help them self-manage their symptoms. Empowering patients to have a more active role in self-management during cancer treatment may lessen their symptom severity and distress.

OBJECTIVE: The aim of this study is to test an intervention designed to improve symptom self-management, the Computerized Symptom Capture Tool (C-SCAT), by helping AYAs understand their unique symptom experience and discuss it with their health care providers.

METHODS: We are conducting a multisite, 2-group randomized controlled trial to evaluate the effects …

Beyond Precision: Ambiomic Survivorship In Childhood And Aya Cancer., Juan Antonio Ortega-García, Omar Shakeel, Nicole Wood, Antonio Pérez-Martínez, Jose Luís Fuster-Soler, Mark D. Miller

Manuscripts, Articles, Book Chapters and Other Papers

Background: Survival among children and adolescents and young adults (AYA) with cancer has improved substantially over recent decades; however, dominant survivorship models remain reactive-activated post-treatment and anchored to static exposure- and organ-based screening. This design underuses the anticipatory window at diagnosis and overlooks environmental and social determinants that modulate outcomes across the life course. Methods: We narratively reviewed international frameworks including the Children's Oncology Group (COG), the International Late Effects of Childhood Cancer Guideline Harmonization Group (IGHG), the Pan-European Network for Care of Survivors after Childhood and Adolescent Cancer (PanCare) and the National Comprehensive Cancer Network (NCCN), and synthesized evidence …

Microrna Profiling Identifies Diagnostic And Prognostic Markers In Pediatric Sarcoma., Terrie Flatt, Leonid M. Yermakov, Shreeram Akilesh, Eleanor Y. Chen, Elizabeth Gonzalez, Alejandro Parrales, Marta Zapata-Tarres, Rocio Cardenas-Cardos, Liliana Velasco-Hidalgo, Celso Corcuera-Delgado, Rodolfo Rodriguez-Jurado, Lillia García-Rodríguez, Midhat S. Farooqi, Atif Ali Ahmed

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: MicroRNAs (miRNAs) function as post-transcriptional gene expression regulators and influence the development and progression of several cancers, yet their roles in pediatric sarcomas remain poorly defined.

METHODS: RNA extracted from formalin-fixed paraffin-embedded tumor tissue scrolls of 108 pediatric tumors, including 32 osteosarcoma (OS), 26 Ewing's sarcoma (EWS), and 50 rhabdomyosarcoma (RMS) cases, were analyzed for microRNA expression using the NanoString multiplex nCounter platform that yielded information on 827 human miRNAs. The expression of candidate miRNAs was validated with in situ hybridization (miRNA-ISH) and QuPath quantification on tissue microarray slides comprising an independent set of 48 OS, 17 EWS, and …

Salvage Therapies For First Relapse Of Shh Medulloblastoma In Early Childhood., Craig Erker, Martin Mynarek, Marie Simbozel, Brandon T. Craig, Virginia L. Harrod, Andrea M. Cappellano, Kenneth J. Cohen, Vicente Santa-Maria Lopez, Andres Morales La Madrid, Chantel Cacciotti, Lorena Baroni, Ralph Salloum, Ashley S. Margol, George Michaiel, Dolly Aguilera, Claire M. Mazewski, Cassie N. Kline, Jonathan L. Finlay, Mohamed S. Abdelbaki, Jeffrey C. Murray, Kathleen Dorris, Bruce Crooks, Kevin Ginn, Nisreen Amayiri, Stephan Tippelt, Gudrun Fleischhack, Svenja Tonn, Nicolas U. Gerber, Alvaro Lassaletta, Jordan R. Hansford, Sara Khan, Stephen W. Gilheeney, Lindsey M. Hoffman, Michal Zapotocky, Valérie Larouche, Shafqat Shah, Vijay Ramaswamy, Amar Gajjar, Sébastien Perreault, Sabine Mueller, Juliette Hukin, Sylvia Cheng, Zhihong J. Wang, Kara Matheson, Simon Bailey, Eric Bouffet, Steven C. Clifford, Giles Robinson, Christelle Dufour, Stefan Rutkowski, Lucie Lafay-Cousin

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Sonic hedgehog (SHH) medulloblastoma is the most common molecular group of infant and early childhood medulloblastoma (iMB) and has no standard of care at relapse. This work aimed to evaluate the post-relapse survival (PRS) and explore prognostic factors of patients with nodular desmoplastic (ND) and/or SHH iMB.

METHODS: This international retrospective study included 147 subjects diagnosed with relapsed ND/SHH iMB between 1995 and 2017, < 6 years old at original diagnosis, and treated without initial craniospinal irradiation (CSI). Univariable and multivariable Cox models with propensity score analyses were used to assess PRS for those in the curative intent cohort.

RESULTS: The 3-year PRS was 61.6% (95% confidence interval [CI], 52.2-69.6). The median age at relapse was 3.4 years (interquartile range [IQR], 2.6-4.1). Those with local relapse (40.8%) more often received salvage treatment with surgery …

Severe Thiopurine-Induced Myelosuppression In A Pediatric All Patient With The Nudt15 *1/*6 Genotype, Jillian Fry, Keith August, Terrie Flatt Do, Laura B. Ramsey

Posters

Introduction: Genetic variants in TPMT and NUDT15 that affect the metabolism of mercaptopurine (6MP) are routinely tested for to guide 6MP dosing. Nudix hydroxylase 15 (NUDT15) deficiency can cause myelosuppression when affected patients are treated with 6MP. The NUDT15 *6 allele is classified as having uncertain function by the Clinical Pharmacogenetics Implementation Consortium Guidelines given insufficient data to determine its phenotype. Here, we present the case of a pediatric patient with the NUDT15 *1/*6 genotype experiencing significant mercaptopurine-induced myelosuppression. Case Description: We present a 4-year-old female with standard risk-average precursor B-cell acute lymphoblastic leukemia treated per AALL1731. Exome sequencing reported …

Contemporary Characteristics And Outcomes Of Pediatric Oncology Patients Participating In Early Phase Clinical Trials., Avina Rami, Kira Bona, Suzanne Shusterman, Karen Wright, Andrew E. Place, Puja J. Umaretiya, Ketki Bhushan, Steven G. Dubois, Kevin Campbell

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Phase 1 or phase 1/2 trials are a first step in pediatric cancer drug development. Currently, there is a paucity of information regarding contemporary outcomes for pediatric patients enrolled in these trials. We describe characteristics and outcomes of patients enrolled in pediatric phase 1 clinical trials over a 9-year period at a single institution.

METHODS: We queried our clinical trials management system to generate a list of patients enrolled and treated on pediatric phase 1 or phase 1/2 trials from 2011 to 2019. We collected baseline demographics, clinical data, efficacy, and safety endpoints post-enrollment including: time to death, objective …

Provider Attitudes And Perspectives On Rehabilitation For Pediatric Cancer Patients, Maria C Swartz, Eduardo Gonzalez Villarreal, Keri Schadler, Donna Kelly, Alakh P Rajan, Clark Andersen, Shiming Zhang, Stephanie J Wells, Amy Heaton, Karen M Moody

Faculty, Staff and Student Publications

PurposeTwenty percent of childhood cancer survivors experience physical function impairments, and ∼75% develop a chronic health condition. Physical and occupational therapists (PT/OTs) can mitigate these late effects, yet few children receive cancer rehabilitation (CR). This research aimed to identify provider attitudes and perspectives towards CR services for children across inpatient and outpatient settings at a cancer center.MethodsThree cardiac rehabilitation instruments were adapted to evaluate knowledge, attitudes, and perceptions regarding CR delivery. Descriptive statistics were used to summarize participant survey results.ResultsTwenty administrators, 20 physicians/advanced practice providers (APPs), and 20 PT/OTs completed surveys. All disciplines strongly agreed on the value of CR …

Who Benefits From Active Music Engagement During Cancer Treatment? Associations Of Sociodemographic Characteristics And Risk Factors With Moderators Of Intervention Effects., Steven J. Holochwost, Elizabeth Harman, Kristin Stegenga, Seethal A. Jacob, Sheri L. Robb

Manuscripts, Articles, Book Chapters and Other Papers

Young children and their parents experience frequent and repeated exposure to potentially traumatic events during treatment for cancer. Active Music Engagement (AME) is a dyadic music therapy intervention that has been found to mitigate traumatic stress symptoms among parents who screened high for traumatic stress symptoms (TSS) and reported higher levels of child distress with cancer-related hospitalizations/treatment (child distress). The current study examined sociodemographic characteristics and risk factors that were associated with higher levels of parent TSS and child distress as a means to identify families that may benefit most from AME in the future. Data were collected from the …

Factors Associated With Self-Report Symptom Screening Adherence In Pediatric Cancer Patients, L. Lee Dupuis, Emily Vettese, Catherine Aftandilian, Vibhuti Agarwal, Christina Baggott, Scott M. Bradfield, Nicole Crellin-Parsons, David R. Freyer, Kara M. Kelly, Allison A. King, Wade Kyono, Ramamoorthy Nagasubramanian, Etan Orgel, Michael E. Roth, Farha Sherani, Lolie Yu, Allison C. Grimes, Melissa P. Beauchemin, Lisa M. Klesges, George A. Tomlinson, Lillian Sung

School of Medicine Faculty Publications

Introduction: Objective was to describe the association between baseline characteristics and the number of Symptom Screening in Pediatrics Tool (SSPedi) assessments completed over an 8-week period. Methods: This was a sub-analysis of a cluster randomized controlled trial among 10 sites that were randomized to the intervention group. Participants were English- or Spanish-speaking pediatric patients 8–18 years of age newly diagnosed with cancer. Participants were prompted to complete SSPedi three times weekly for 8 weeks. The outcome was the number of SSPedi assessments completed during the 8-week period. Factors associated with the number of assessments were determined using mixed effects Poisson …

Patient Information Needs During Ambulatory Medication Changes: A Qualitative Study, Elaine Giletta, Vanessa B. Hurley, Yushi Yang, Jessica L. Schwartz, Rabia Jalalzai, Nicole L. Mollenkopf, Raquel Mayne, Samantha I. Pitts

Journal of Patient-Centered Research and Reviews

Purpose: We performed interviews with patients and caregivers to explore whole-person, whole-regimen information needs at times of routine outpatient medication changes. We categorized information needs in this relatively unstudied context and identified which needs were commonly recognized as insufficiently met.

Methods: We explored the content of the semi-structured interviews, which involved nine patients and two caregivers of pediatric patients, all of whom experienced a recent medication change in the outpatient setting of a large urban academic healthcare system. We used inductive thematic analysis to organize information needs into categories to identify key information gaps.

Results: We identified five categories of …

Environmental Exposures Increase Health Risks In Childhood Cancer Survivors., Omar Shakeel, Nicole Wood, Hannah M. Thompson, Michael E. Scheurer, Mark D. Miller

Manuscripts, Articles, Book Chapters and Other Papers

Childhood cancer survivors (CCSs) are at increased risk for chronic health issues due to late effects of cancer and its treatment. We address the impact of environmental exposures, such as air pollution, tobacco smoke, extreme weather events, and pesticides, on the health and survival of CCSs. These environmental hazards have been associated with worsening health outcomes and decreased survival among CCSs on a global scale. We also highlight that providers at a major pediatric cancer center in the United States have limited knowledge and practical skills about environmental risk factors and how to reduce exposures. Our survey results show that …

Current Landscape Of Preclinical Models For Pediatric Gliomas: Clinical Implications And Future Directions., Syed M. Faisal, Monika Yadav, Garrett R. Gibson, Adora T. Klinestiver, Ryan M. Sorenson, Evan Cantor, Maria Ghishan, John R. Prensner, Andrea T. Franson, Kevin Ginn, Carl Koschmann, Viveka Nand Yadav

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric high-grade gliomas (pHGGs), particularly diffuse midline gliomas (DMGs), are among the most lethal brain tumors due to poor survival and resistance to therapies. DMGs possess a distinct genetic profile, primarily driven by hallmark mutations such as H3K27M, ACVR1, and PDGFRA mutations/amplifications and TP53 inactivation, all of which contribute to tumor biology and therapeutic resistance. Developing physiologically relevant preclinical models that replicate both tumor biology and the tumor microenvironment (TME) is critical for advancing effective treatments. This review highlights recent progress in in vitro, ex vivo, and in vivo models, including patient-derived brain organoids, genetically engineered mouse models (GEMMs), and …

Splenic Function Post Radiation Therapy In Childhood Cancer Survivors, Joy M. Fulbright, Kyla Alsman, Ashley K. Sherman, Kris Laurence, Becky Lowry

Manuscripts, Articles, Book Chapters and Other Papers

Study evaluated splenic function in childhood cancer survivors (CCS) who had received radiation to the spleen utilizing pRBC PIT (vesiculated erythrocytes) count compared to a non-radiated control group. Those exposed to < 20 Gy of radiation had PIT counts not significantly different from the control group. There was trend for higher pRBC PIT count in those exposed to higher doses of radiation.

Hematopoietic Stem Cell Transplantation Outcomes For High-Risk Aml: A Report From The Children's Oncology Group., Benjamin J. Huang, Lauren K. Meyer, Todd A. Alonzo, Yi-Cheng Wang, Adam J. Lamble, Rhonda E. Ries, Weijie Wang, Betsy Hirsch, Gordana Raca, Xiaotu Ma, Alan S. Gamis, Richard Aplenc, E Anders Kolb, Todd M. Cooper, Katherine Tarlock, Michael R. Loken, Soheil Meshinchi, Joseph H. Chewning, William G. Woods, John T. Horan

Manuscripts, Articles, Book Chapters and Other Papers

PURPOSE: Hematopoietic stem cell transplantation (HSCT) is used as consolidation for pediatric patients with high-risk AML in first complete remission (CR1). The definition of high-risk AML has evolved considerably over the past two decades with the successive identification of new unfavorable risk factors. We conducted a cross-study analysis to determine whether HSCT improves the outcomes of patients with contemporarily defined high-risk AML.

METHODS: We combined data from AAML0531 and AAML1031, the last two phase III clinical trials completed by the Children's Oncology Group (COG). These two trials established the prognostic importance of measurable residual disease (MRD) and several high-risk cryptic …

Real-World Outcomes For Young Adult Patients Receiving Cd19 Car T-Cell Therapy., Hannah Lust, Liora M. Schultz, Soyang Kwon, Gregory W. Roloff, Ibrahim Aldoss, Christina Baggott, Samuel John, Jenna E. Rossoff, Kevin O. Mcnerney, Vanessa A. Fabrizio, Julie-An Talano, Amy Moskop, Kevin J. Curran, Christine L. Phillips, Nicole Karras, Susanne H C Baumeister, Stacy L. Cooper, Michelle Hermiston, Prakash Satwani, Muna Qayed, Sunil S. Raikar, Margaret Macmillan, Erin Hall, Khanh Nguyen, Ryan D. Cassaday, Noam E. Kopmar, Vamsi K. Kota, John Mathews, Paul Shaughnessy, Marc S. Schwartz, Abdullah Ladha, George Yaghmour, Muthu V. Kumaran, Veronika Bachanova, Sean Tracy, Tamer Othman, Marlise R. Luskin, Evan C. Chen, Anjani S. Advani, Nikeshan Jeyakumar, Katharine Miller, Amy Zhang, Katherine C. Sutherland, Bijal D. Shah, Lori Muffly, Rawan Faramand

Manuscripts, Articles, Book Chapters and Other Papers

Tisagenlecleucel (tisa-cel) and brexucabtagene autoleucel (brexu-cel) are approved CD19 chimeric antigen receptor T-cell therapy (CAR T) products for young adults (YA) with relapsed/refractory B-cell acute lymphoblastic leukemia. A distinct analysis of YAs receiving commercial CD19 CAR T has not been reported. Using retrospective data from the Pediatric Real-World CAR T Consortium and the Real-World Outcomes of CAR T in Adult ALL collaboration, we describe the efficacy and safety of tisa-cel and brexu-cel in 70 YAs (18-26 years; tisa-cel, n = 50; brexu-cel, n = 20). Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were observed more frequently …

Cytarabine Pharmacogenomics And Outcomes Among Children And Young Adults With Acute Myeloid Leukemia., Richard J. Marrero, Vivek M. Shastri, Deedra Nicolet, Krzysztof Mrózek, Christopher J. Walker, William G. Blum, Bayard L. Powell, Jonathan E. Kolitz, Joseph O. Moore, Geoffrey L. Uy, Wendy Stock, Andrew J. Carroll, John C. Byrd, Richard Aplenc, Todd M. Cooper, Alan S. Gamis, Huiyun Wu, Stanley Pounds, Yi-Cheng Wang, Todd A. Alonzo, Soheil Meshinchi, Ann-Kathrin Eisfeld, Edward A. Kolb, Jatinder K. Lamba

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Therapeutic responses in acute myeloid leukemia (AML) demonstrate considerable variability both across and within established risk stratifications and age groups. Moreover, significant racial disparities persist, with Black patients experiencing inferior survival outcomes compared with their White counterparts.

OBJECTIVE: To validate the association of the previously reported 10 single nucleotide variant (SNV)-based ara-C pharmacogenomics score (ACS10) with survival outcomes in a large cohort of pediatric AML patients; to evaluate whether ACS10 remains relevant in an adolescent and young adult (AYA) population of patients with AML treated with similar intensive induction chemotherapy protocols; and to assess the association of ACS10 with …