Corporate Satellite Symposia and Education Theater Schedule

Learning Objectives:

1. Summarize the unmet needs of children with VWD, discuss the potential benefits of prophylaxis in this population, when to initiate prophylaxis, and explore why it is underused.
2. Discuss recent data from clinical trials in children with VWD, demonstrating the benefits of prophylaxis for bleed control.

Faculty:

• Veronica Flood, MD, Versiti Blood Research Institute, Milwaukee, WI
• Fernando Corrales-Medina, MD, University of Miami, Miami, Florida
• Robert F. Sidonio, MD, Emory University School of Medicine, Atlanta, Georgia

This non-CME symposium is supported by Octapharma USA.

Asparaginase therapies are a cornerstone treatment component of chemotherapy regimens for pediatric and adolescent and young adult (AYA) patients with acute lymphoblastic leukemia (ALL). The clinical benefits derived from inclusion of asparaginase therapies is well established, and recent data and FDA approvals for asparaginase products with differing formulations and safety profiles are expanding treatment options for patients with ALL. Notably, the recent the FDA approval and updated safety labeling for asparaginase erwinia chrysanthemi offers clinical benefits of novel dosing regimens, and provides a reliable treatment option for affected patients faced not only with the barrier of hypersensitivity but also with the manufacturing issues that have plagued native asparaginase E chrysanthemi (Erwinaze/Erwinase) and led to global shortages.

However, the optimal use of asparaginase therapy in pediatric and AYA patients with ALL is not without its challenges. The complexity of ALL requires a nuanced understanding of disease progression, and approach to treatment selection after hypersensitivity reactions to Escherichia coli–derived L-asparaginases. Furthermore, HCPs must be aware of strategies to mitigate treatment interruptions while mitigating serious toxicities associated with asparaginase therapy - which is critical to optimize patient outcomes. Lastly, in an evolving treatment landscape of ALL, updated therapeutic regimens and treatment guidelines including the emergence of immune-targeted therapies, pose challenges in treatment sequencing and understanding the place in therapy for asparaginase therapies.

Learning Objectives:

1. Discuss current efficacy evidence and unmet needs related to the optimized use of asparaginase in pediatric ALL, including the role of novel Erwinia asparaginase compounds
2. Devise strategies to address therapeutically relevant considerations related to asparaginase use for pediatric ALL such as appropriate dosing, pre-empting truncation/discontinuation, and drug-related toxicity
3. Implement monitoring protocols to identify hypersensitivity, silent inactivation, or toxicity in pediatric ALL and increase completion rates of asparaginase treatment
4. Evaluate current clinical evidence and expert recommendations for implementing pediatric-inspired therapeutic regimens for ALL into treatment plans for adolescents and young adults, understanding the place in therapy of asparaginase regimens in an evolving clinical landscape

Faculty:

• Keith J. August, MD, MS, Professor of Pediatrics, University of Missouri-Kansas City; Oncology Section Chief; Director, Leukemia and Lymphoma Program; Associate Director, Experimental Therapeutics in Pediatric Cancer; Division of Hematology/Oncology/BMT, Children’s Mercy Hospital, Kansas City, Missouri
• Emily Curran, MD, Assistant Professor, Division of Hematology/Oncology; Medical Director, UCC Clinical Trials Office, University of Cincinnati Cancer Center, Cincinnati, Ohio
• Van Huynh, MD, Assistant Division Chief of Oncology; Director, Leukemia Program; Section Director, CAR T-Cell Program; CHOC Children's Hospital, Orange, California; Associate Clinical Professor, UC Irvine, Irvine, California

This CME symposium is provided by Clinical Care Options, LLC. Supported by an educational grant from Jazz Pharmaceuticals, Inc.

This symposium will explore clinical trial data on targeted therapies for NF1-associated tumors, focusing on toxicity profiles and AE management. Real-world cases will highlight interprofessional strategies for patient assessment, education, emotional support, toxicity mitigation, and care transitions.

Learning Objectives:

1. Identify key characteristics of neurofibromatosis type 1 (NF1) clinical presentation and disease advancement across the lifespan, including genetic etiology and multisystem pathophysiology of NF1, that impact early and accurate diagnosis.
2. Integrate MEK inhibitors in the management of NF1-associated plexiform neurofibromas (PNs) in pediatric patients.
3. Develop comprehensive care plans that include disease monitoring, managing toxicities, and personalized treatment for patients with NF1 across the lifespan.

Faculty:

• Angela Hirbe, MD, PhD
  Associate Professor of Medicine and Pediatrics
  Director, Adult Neurofibromatosis Clinical Program
  Division of Oncology, Sarcoma Section
  Washington University School of Medicine
  St. Louis, MO

• Christopher Moertel, MD
  Kenneth and Betty Jayne Dahlberg Professor of Pediatrics
  University of Minnesota School of Medicine
  Minneapolis, MN

This CME symposium is provided by Creative Educational Concepts LLC and supported by educational grants from Alexion Pharmaceuticals and SpringWorks Therapeutics, Inc.

Presentation will focus on Aspasrlas as a component of a multi-agent chemotherapy regimen for the treatment of acute lymphoblastic leukemia in pediatric and young adult patients aged 1 month to 21 years.

Learning Objectives:

1. Review the clinical efficacy profile of Asparlas.
2. Explore patient profiles to help identify patients who may be appropriate for treatment with Asparlas.
3. Discuss the clinical safety profile, adverse reaction management, and important safety information for Asparlas.

Faculty:

• Natalie Bezler, MD, Connecticut Children's Medical Center

This non-CME symposium is supported by Servier Pharmaceuticals.

Platelet transfusions are the second most commonly prescribed blood product for pediatric patients.¹ The risks of platelet transfusions include acute transfusion reactions, transfusion-transmitted infection (TTI), and transfusion-associated graft-versus-host disease (TA-GVHD). Furthermore, pediatric patients have been demonstrated to be more susceptible to acute transfusion reactions compared to adults.² Pathogen reduction (PR) of platelet components (PC) with amotosalen-UVA is designed to mitigate the risks of TTI and TA-GVHD,³ but clinical data on transfusion reactions for pediatric patients are limited. Dr. Bryce Pasko and Dr. Samantha Phou of Phoenix Children’s Hospital will present findings from a recent retrospective study that analyzed transfusion reactions over two 30-month periods before and after the implementation of PR-platelets. This study provides insight into the safety profile of PR-platelets in pediatric patients and their impact on transfusion-related adverse events.

1. Delaney M, et al. Pediatr Crit Care Med. 2022 Jan 1;23(13 Suppl 1 1S):e1-e13.
2. Oakley FD, et al. Transfusion. 2015 Mar;55(3):563-70.3.
3. The INTERCEPT Blood System for Platelets Package Insert, Cerus Corporation; December 19, 2023.

Faculty:

• Bryce Pasko, MD, Phoenix Children’s Hospital
• Samantha Phou, MD, Phoenix Children’s Hospital

This educational theater is supported by Cerus Corporation

Provide an overview of the different cell & gene therapy (CGT) options for pediatric patients with hemoglobinopathies and the long-term clinical impact of CGT according to age for these patients.

Faculty:

• Beth Stenger, MD, Associate Professor of Pediatrics, Emory University/Children’s Healthcare of Atlanta

This educational theater is supported by Vertex Parmaceuticals

This is a resource that takes a Problem Based Learning approach by including a hypothetical pediatric patient case diagnosed with Ph(-) B-cell precursor ALL, and is designed to encourage clinical discussions among prescribers on the latest innovations in B cell precursor ALL.

Learning Objectives:

1. To increase awareness among prescribers of the AALL1731 data presented at 2024 ASH
2. To discuss how BLINCYTO® can be integrated into 1L consolidation across patient types (excl. SR-fav) based on superior survival (AALL1731)

Faculty:

• Mitchell S. Cairo, MD, Chief, Pediatric Hematology, Oncology, and Stem Cell Transplantation Director, Children and Adolescent Cancer and Blood Diseases Center, Maria Fareri Children’s Hospital Westchester Medical Center

This non-CME Satellite Dinner Symposium is supported by Amgen, Inc.

Join Y-mAbs Therapeutics, Inc. for an education theater featuring Jacqueline M Kraveka, DO, Professor of Pediatrics and Pediatric Hematologist-Oncologist at the Medical University of South Carolina. She will focus on an FDA-approved cytotoxic immunotherapy for high-risk neuroblastoma, including the importance of Curie scores and updated, multi-center efficacy and safety data.

Faculty:

• Jacqueline M Kraveka, DO, Professor of Pediatrics & Pediatric Hematologist-OncologistMedical University of South Carolina

This educational theater is supported by Y-mAbs Therapeutics, Inc

Alexion invites you to join a dynamic physician-led case-based discussion with a specialist in neurofibromatosis type 1 (NF1). Dr. Zsila Sadighi a nationally recognized specialist in NF1, will discuss NF1 PN patient diagnosis, treatment, and the importance of timely intervention to optimize outcomes for patients. The presentation includes cases of pediatric NF1 patients with symptomatic, inoperable plexiform neurofibromas (PN).

Zsila Sadighi, MD, currently serves as the Chief Medical Officer at Adjuvant Behavioral Health, focusing on mental health in patients affected with neurological diseases, including those with neurofibromatosis type 1 (NF1). As a brain tumor survivor herself, she is passionate about improving mental and emotional health. Previously, she was an associate professor in the Division of Pediatrics at The University of Texas MD Anderson Cancer Center and director of their NF1 Center for five years. Dr Sadighi attended the University of Texas at Houston for medical school. She completed her residency in pediatrics and fellowship in child and adolescent neurology at the University of Texas Health Science Center and a fellowship in adult and pediatric neuro-oncology at the University of Texas MD Anderson Cancer Center. Dr Sadighi also served as a pediatric neurologist and pediatric neuro-oncologist at St. Jude Children's Research Hospital, where she saw patients with neurological complaints or complications from brain and spinal Tumors.

Faculty:

• Zsila Sadighi, MD, Chief Medical Officer at Adjuvant Behavioral Health

This educational theater is supported by Alexion Pharmaceuticals

During this education theatre, join us to learn about WHIM syndrome (warts, hypogammaglobulinemia, infections, myelokathexis), a rare combined primary immunodeficiency and chronic neutropenic disorder. This presentation will enhance your awareness and understanding of WHIM syndrome through a discussion of its pathophysiology, clinical manifestations, complications, diagnosis, and management strategies.

Faculty:

• Dr. Niraj Patel, MD, MS

This educational theater is supported by X4 Pharmaceuticals