Medical Specialties Commons^™

Characteristics And Treatment Of Acute Myeloid Neoplasms With Cutaneous Involvement In Infants Up To 6 Months Of Age: A Retrospective Study., Juliette Renaud, Bianca F. Goemans, Franco Locatelli, Martina Pigazzi, Shelagh Redmond, Claudia E. Kuehni, Alice Destaillats, Todd A. Alonzo, Robert B. Gerbing, Alan S. Gamis, Richard Aplenc, Raffaele Renella, Todd Cooper, Francesco Ceppi

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Myeloid neoplasms account for 50% of cases of pediatric leukemias in infants. Approximately 25%-50% of patients with newborn leukemia have cutaneous extramedullary disease (EMD). In less than 10% of patients, aleukemic leukemia cutis or isolated extramedullary disease with cutaneous involvement (cEMD) occurs when skin lesions appear prior to bone marrow involvement and systemic symptoms. Interestingly, in acute myeloid leukemia with cutaneous EMD (AML-cEMD) and cEMD, spontaneous remissions have been reported.

METHOD: This is a multicentric retrospective cohort study aiming to describe characteristics, treatment, and outcome of infants with either cEMD or presence of cutaneous disease with involvement of the …

Exploring The Role Of Pain On Physical Activity Among Youth With Acute Lymphoblastic Leukemia Using The Biopsychosocial Model, Anna E. Van Asselt, Renee Gilbert, Meghan Tokala, Jacee Weber, Meredith L. Dreyer Gillette, Robert C. Gibler, Carolyn R. Bates, Keith August

Research Days

The Role of Pain on Physical Activity in Pediatric ALL

Keywords: Biopsychosocial model, pediatric Acute Lymphoblastic Leukemia, pain, physical activity

Introduction: Engaging in physical activity (PA) during cancer treatment benefits a child’s physical and mental well-being (McLaughlin et al., 2021). However, engaging in PA may be challenging due to treatment and disease-related pain (Uhl et al., 2020). Pediatric cancer research has primarily focused on management of procedural and chronic pain, with fewer studies examining how pain may interfere with PA during treatment (Clews et al., 2022). The current study qualitatively investigated the impact of pain on PA using a …

Increasing Palliative Care Team Involvement In Pediatric Hematopoietic Stem Cell Transplant Patients, Meagan Vacek, Lisa Tarbell, Melissa List, Erin Hall, Jennifer Linebarger, Kelstan Ellis, Gina Jones, Joel Thompson

Research Days

Early implementation of palliative care in pediatric oncology is well established but its role in pediatric hematopoietic stem cell transplant (HSCT) patients is yet to be fully determined. Multiple organizations encourage palliative care involvement in HSCT patients. Our aim was to increase palliative care team consults for patients receiving HSCT for our targeted diagnosis. Though we did not achieve this aim, we were able to increase the palliative care team's involvement in the total cohort of patients undergoing HSCT.

A Genomics Driven Induced Pluripotent Stem Cell Model Of Infant Acute Lymphoblastic Leukemia - Early Results, Meagan Vacek, Jacqelyn Nemechek, Irina Pushel, Bradley Thornton, Molly Leyda, Priyanka Prem Kumar, Midhat Farooqi, Jay L. Vivian, Erin M. Guest, John M. Perry

Research Days

While the cure rates for pediatric ALL have improved over the decades, infants with ALL (iALL) have not benefitted from these advances and continue to have a devastating prognosis. Unfortunately progress in treatment has also been slowed by inadequate research models. With this project, we address this unmet need by investigating a novel model to understand the cellular and molecular changes that occur during iALL onset and progression.

Frailty In Children And Adolescents With Acute Lymphoblastic Leukemia Or Lymphoblastic Lymphoma Receiving Maintenance Chemotherapy – A Pilot Study, Thomas R. Cochran, Joy M. Fulbright, Samantha Butrous, Brian R. Lee, Keith J. August, David A. White

Research Days

The submitted study aims to identify children with acute lymphoblastic leukemia or lymphoblastic lymphoma that meet frailty criteria early to allow for prevention of chronic health conditions that lead to increased mortality as survivors age into adulthood. Prevention and/or reversal of frailty has the potential to improve quality of life, decrease burden of disease, and extend the lifespan of the growing population of childhood cancer survivors. Given that frailty has been accepted as a tool to identify elderly and chronically sick adults highly vulnerable to adverse health outcomes, better understanding of the relevance and applicability of frailty in pediatrics may …

Prognostic Impact Of Cooccurring Mutations In Flt3-Itd Pediatric Acute Myeloid Leukemia., Katherine Tarlock, Robert B. Gerbing, Rhonda E. Ries, Jenny L. Smith, Amanda Leonti, Benjamin J. Huang, Danielle Kirkey, Leila Robinson, Jack H. Peplinksi, Beverly Lange, Todd M. Cooper, Alan S. Gamis, E Anders Kolb, Richard Aplenc, Jessica A. Pollard, Todd A. Alonzo, Soheil Meshinchi

Manuscripts, Articles, Book Chapters and Other Papers

We sought to define the cooccurring mutational profile of FLT3-ITD-positive (ITDpos) acute myeloid leukemia (AML) in pediatric and young adult patients and to define the prognostic impact of cooperating mutations. We identified 464 patients with FLT3-ITD mutations treated on Children's Oncology Group trials with available sequencing and outcome data. Overall survival, event-free survival (EFS), and relapse risk were determined according to the presence of cooccurring risk stratifying mutations. Among the cohort, 79% of patients had cooccurring alterations across 239 different genes that were altered through mutations or fusions. Evaluation of the prognostic impact of the cooccurring mutations demonstrated that patients …

Structural Variants Involving Mllt10 Fusion Are Associated With Adverse Outcomes In Pediatric Acute Myeloid Leukemia., Oussama Abla, Rhonda E. Ries, Tim Triche, Robert B. Gerbing, Betsy Hirsch, Susana Raimondi, Todd Cooper, Jason E. Farrar, Nathaniel Buteyn, Lauren M. Harmon, Hong Wen, Aniruddha J. Deshpande, E Anders Kolb, Alan S. Gamis, Richard Aplenc, Todd Alonzo, Soheil Meshinchi

Manuscripts, Articles, Book Chapters and Other Papers

MLLT10 gene rearrangements with KMT2A occur in pediatric acute myeloid leukemia (AML) and confer poor prognosis, but the prognostic impact of MLLT10 in partnership with other genes is unknown. We conducted a retrospective study with 2080 children and young adults with AML registered on the Children's Oncology Group AAML0531 (NCT00372593) and AAML1031 trials (NCT01371981). Transcriptome profiling and/or karyotyping were performed to identify leukemia-associated fusions associated with prognosis. Collectively, 127 patients (6.1%) were identified with MLLT10 fusions: 104 (81.9%) with KMT2A::MLLT10, 13 (10.2%) with PICALM::MLLT10, and 10 (7.9%) X::MLLT10: (2 each of DDX3X and TEC), with 6 partners (DDX3Y, CEP164, SCN2B, …

Chromatin Profiles Are Prognostic Of Clinical Response To Bortezomib-Containing Chemotherapy In Pediatric Acute Myeloid Leukemia: Results From The Cog Aaml1031 Trial., Anneke D. Van Dijk, Fieke W. Hoff, Yihua Qiu, Stefan E. Hubner, Robin L. Go, Vivian R. Ruvolo, Amanda R. Leonti, Robert B. Gerbing, Alan S. Gamis, Richard Aplenc, Edward A. Kolb, Todd A. Alonzo, Soheil Meshinchi, Eveline S J M De Bont, Terzah M. Horton, Steven M. Kornblau

Manuscripts, Articles, Book Chapters and Other Papers

The addition of the proteasome inhibitor bortezomib to standard chemotherapy did not improve survival in pediatric acute myeloid leukemia (AML) when all patients were analyzed as a group in the Children's Oncology Group phase 3 trial AAML1031 (NCT01371981). Proteasome inhibition influences the chromatin landscape and proteostasis, and we hypothesized that baseline proteomic analysis of histone- and chromatin-modifying enzymes (HMEs) would identify AML subgroups that benefitted from bortezomib addition. A proteomic profile of 483 patients treated with AAML1031 chemotherapy was generated using a reverse-phase protein array. A relatively high expression of 16 HME was associated with lower EFS and higher 3-year …

Diagnosis Of Mosaic Rasopathy In A Child With Rhabdomyosarcoma, Meagan Vacek, Paige Johnson, Midhat S. Farooqi, Kristi M. Canty, Dihong Zhou, Brendan Lanpher, Wendy Allen-Rhoades, Erin M. Guest

Posters

Background: Phacomatosis pigmentokeratotica (PPK) is a subtype of epidermal nevus syndrome characterized by the co-existence of a sebaceous nevus and a speckled lentiginous nevus and described in approximately 30 cases in literature. PPK is now recognized as a mosaic RASopathy due a postzygotic mutation in the Ras-Raf-MEK-ERK pathway. RAS variants are also known to contribute to tumorigenesis, in some pediatric cancers, including rhabdomyosarcoma. Objective: Describe the presentation and evaluation of a child with pelvic rhabdomyosarcoma and evolving skin lesions found to have a rare mosaic-RASopathy. Design/Method: Case Report Results: The patient is a former 32-week premature female who presented to …

Increasing Palliative Care Team Involvement In Pediatric Hematopoietic Stem Cell Transplant Patients, Meagan Vacek, Lisa Tarbell, Melissa List, Erin Hall, Jennifer Linebarger, Kelstan Ellis, Gina Jones, Joel Thompson

Posters

Background: Palliative care facilitates communication, helps with physical and psychological symptom management, and assists in goals of care and advance care planning discussions.1 Multiple organizations, including the American Academy of Pediatrics and American Society of Clinical Oncology encourage palliative care engagement and their involvement with hematopoietic stem cell transplant (HSCT) patients has been shown to be beneficial as HSCT is associated with a high degree of morbidity and possible mortality.2-3 Objective: To increase the number of PaCT consults for patients receiving HSCT for our targeted diagnoses from 48% to 75% by December 2023. Methods: Chart review was conducted for patients …

Molecular-Guided Therapy For The Treatment Of Patients With Relapsed And Refractory Childhood Cancers: A Beat Childhood Cancer Research Consortium Trial., Giselle L Saulnier Sholler, Genevieve Bergendahl, Elizabeth C. Lewis, Jacqueline Kraveka, William Ferguson, Abhinav B. Nagulapally, Karl Dykema, Valerie I Brown, Michael S. Isakoff, Joseph Junewick, Deanna Mitchell, Jawhar Rawwas, William Roberts, Don Eslin, Javier Oesterheld, Randal K. Wada, Devang Pastakia, Virginia Harrod, Kevin Ginn, Raya Saab, Kevin Bielamowicz, Jason Glover, Eugenia Chang, Gina K. Hanna, Daniel Enriquez, Tyler Izatt, Rebecca F Halperin, Abigail Moore, Sara A. Byron, William P D Hendricks, Jeffrey M. Trent

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Children with relapsed central nervous system (CNS tumors), neuroblastoma, sarcomas, and other rare solid tumors face poor outcomes. This prospective clinical trial examined the feasibility of combining genomic and transcriptomic profiling of tumor samples with a molecular tumor board (MTB) approach to make real‑time treatment decisions for children with relapsed/refractory solid tumors.

METHODS: Subjects were divided into three strata: stratum 1-relapsed/refractory neuroblastoma; stratum 2-relapsed/refractory CNS tumors; and stratum 3-relapsed/refractory rare solid tumors. Tumor samples were sent for tumor/normal whole-exome (WES) and tumor whole-transcriptome (WTS) sequencing, and the genomic data were used in a multi-institutional MTB to make real‑time treatment …