Aiming Targeted Medical Therapies at Neurofibromatosis Type 1: An Interactive, Case-Based Guide for the Pediatric Hematologist/Oncologist

April 21, 2021

This educational initiative is targeted to pediatric hematologists/oncologists, general hematologists/oncologists, hematologists, oncology researchers, and hem/onc fellows attending the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) Annual Meeting, as well as other members of the multidisciplinary hematology/oncology NF1 care team. Designed and developed to provide an interactive and exhaustive overview of novel and emerging data, as well as establish a foundational context of NF1 disease state complexity, this activity will begin by identifying established diagnostic signs, symptoms, and hallmark aspects of NF1 natural history and clinical presentation in pediatric patients, and describing the genetic etiologies and multi-system pathophysiologies that have historically made NF1-associated plexiform neurofibromas so difficult to treat. Galvanized by a dynamic case-based approach, attendees will then gain insights from key opinion leaders regarding the pivotal shortcomings of traditional NF1 management modalities—surgery, radiation, and surveillance—and the new horizons that have accompanied the burgeoning emergence of targeted medical therapies, most notably MEK inhibitors. Finally, top-level clinical experts will guide attendees through a case-driven exploration of the role of the pediatric hematologist/oncologist and researcher in the evolving NF1 treatment paradigm, and provide activity participants an opportunity to design multidisciplinary, evidence-based targeted therapy regimens for symptomatic, inoperable NF1-related tumors, with emphasis on plexiform neurofibromas, low-grade gliomas, and malignant peripheral nerve sheath tumors (MPNSTs).


Presented by Creative Educational Concepts, Inc (CEC Oncology).

Supported through an independent educational grant from AstraZeneca.

Target Audience

This educational initiative is targeted to pediatric hematologists/oncologists, general hematologists/oncologists, hematologists, oncology researchers, hem/onc fellows, and other members of the multidisciplinary hematology/oncology NF1 care team attending the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) Annual Meeting.

Learning Objectives

  • Recognize established diagnostic signs and symptoms of neurofibromatosis type 1 (NF1), and identify key aspects of NF1 clinical presentation in pediatric patients.
  • Describe the genetic genesis and the multi-system pathophysiology of NF1, with a focus on the clinical gravity and historical intractability of plexiform neurofibromas.
  • Review traditional management modalities for treating NF1-associated plexiform neurofibromas, including surgery, radiation, and surveillance, and emphasize the crucial clinical chasms that remain.
  • Evaluate the pharmacologic rationale and emerging clinical trial data for MEK inhibitors and other novel targeted therapies in NF1-associated tumors (plexiform neurofibromas, low-grade gliomas, and malignant peripheral nerve sheath tumors). 
  • Discuss the critical role of the pediatric hematologist/oncologist in delivering targeted medical therapies in NF1, and design evidence-based, multidisciplinary treatment strategies for symptomatic, inoperable NF1-related plexiform neurofibromas.

Additional Information

AttachmentSize
PDF icon ASPHO 4.21.21 CME Sympo Handout.pdf6.14 MB
Course summary
Available credit: 
  • 1.00 AMA PRA Category 1 Credit™
  • 1.00 Participation
Course opens: 
04/21/2021
Course expires: 
05/21/2021
Event starts: 
04/21/2021 - 11:30am
Event ends: 
04/21/2021 - 12:30pm
Rating: 
0
11:30 AM-11:35 AM CDTWelcome and Introductions/Pre-test
 AeRang Kim, MD, PhD (Activity Chair)
11:35 AM-11:45 AM CDTLaying the Groundwork: NF1 Etiology, Pathophysiology, & Clinical Hallmarks
 Michael J. Fisher, MD
11:45 AM-12:05 PM CDTBringing MEK Inhibition into Focus: A Practical Exploration of the Evolving Plexiform Neurofibroma Treatment Paradigm
 Andrea M. Gross, MD
12:05 PM-12:25 PM CDTReal-World NF1 Perspectives: Elucidating the Role of the Pediatric Hematologist/Oncologist in the New Landscape
 AeRang Kim, MD, PhD
12:25 PM-12:30 PM CDTConversation with the Experts: Audience Q&A/Post-test
 Faculty Panel Moderated by Dr. Kim

AeRang Kim, MD, PhD 
Associate Professor of Pediatrics
Director of Clinical Research
Division of Oncology
Children’s National Hospital
Washington, DC

 

Dr. AeRang Kim is an Associate Professor of Pediatrics and Director of Clinical Research in the Division of Oncology at Children’s National Hospital in Washington, DC. Her research has focused on the development of novel therapeutics for pediatric solid cancer, sarcomas, and NF1 associated tumors. Dr. Kim has developed and lead multiple early phase trials of targeted therapies for NF1 associated plexiform neurofibroma and malignant peripheral nerve sheath tumors. She serves as the MPNST disease committee chair for the Department of Defense Neurofibromatosis Clinical Trials Consortium.

 

Andrea M. Gross, MD
Assistant Research Physician
Pediatric Oncology Branch
Center for Cancer Research
National Institutes of Health 
Bethesda, MD


Dr. Andrea Gross is a board-certified pediatrician and pediatric oncologist who earned her medical degree at the University of Connecticut and completed pediatric residency and a chief resident year at Cincinnati Children’s Hospital Medical Center. She completed a pediatric hematology/oncology fellowship at Children’s National Medical Center and is currently an Assistant Research Physician working in the Pediatric Oncology Branch at the National Cancer Institute in the lab of Dr. Brigitte Widemann. Dr. Gross has been the lead associate investigator on the phase 2 trial of selumetinib for patients with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas since 2015, which led to the first FDA approved medication for NF1 in 2020. Her research focuses on clinical trials for tumor predisposition syndromes.  Her areas of interest include developing and utilizing functional outcome measures for tumor predisposition syndromes, working with rare disease patient advocates to increase patient engagement in clinical trial design and dealing with the challenge of medication adherence in the NF1 population.

 

Michael J. Fisher, MD
Chief of the Neuro-Oncology Section
Division of Oncology
Director of the Neurofibromatosis (NF) Program
Hubert J.P. and Anne Faulkner Schoemaker Endowed Chair in Pediatric Neuro-Oncology
The Children's Hospital of Philadelphia
Philadelphia, PA

Dr. Michael Fisher is Professor of Pediatrics (Perelman School of Medicine at the University of Pennsylvania), Chief of the Neuro-Oncology Section (Division of Oncology), Director of the Neurofibromatosis (NF) Program, and the Hubert J.P. and Anne Faulkner Schoemaker Endowed Chair in Pediatric Neuro-Oncology at The Children's Hospital of Philadelphia. His research focuses on identifying new treatments and novel biomarkers (particularly using new imaging modalities) and exploring functional outcomes for children with tumors associated with NF1. Dr. Fisher is Chair of the Steering Committee, and previously was Chair of the Plexiform Neurofibroma Committee, of the Department of Defense NF Clinical Trials Consortium. He is a member of the Steering Committee and former Chair of the Visual Outcomes Committee for REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis), an international effort to develop standardized outcome measures for clinical trials.


In accordance with the Food and Drug Administration, the speakers have disclosed that there is the potential for discussions concerning off-label uses of a commercial product/device during this educational activity.

Any person who may contribute to the content of this continuing education activity must disclose relevant relationships (and any known relationships of their spouse/partner) with commercial interests whose products or services are discussed in educational presentations. A commercial interest is defined as any entity producing, marketing, re-selling, or distributing health care goods or services consumed by, or used on, patients. Relevant relationships include receiving from a commercial interest research grants, consultant fees, travel, other benefits, or having a self-managed equity interest in a company.

Disclosure of a relationship is not intended to suggest or condone any bias in any presentation but is made to provide participants with information that might be of potential importance to their evaluation of a presentation.

Planner:
AeRang Kim, MD, PhD–has no relevant financial relationships to disclose in relation to the content of this activity.
Bryan C. Taylor, PharmD–has no relevant financial relationships to disclose in relation to the content of this activity.

Authors/Presenters:
AeRang Kim, MD, PhD–has no relevant financial relationships to disclose in relation to the content of this activity.
Andrea M. Gross, MD–has no relevant financial relationships to disclose in relation to the content of this activity.
Michael J. Fisher, MD–has disclosed that he is a consultant for the AstraZeneca and SpringWorks. He also receives grant/research support from AstraZeneca, Array BioPharma Inc., a wholly owned subsidiary of Pfizer Inc., and Exelixis.

Reviewer:
Miriam Bornhorst, MD—has disclosed that she is a consultant for AstraZeneca.

In support of improving patient care, Creative Educational Concepts is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

 


Medicine (ACCME)
CEC designates this live educational activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Upon completion of a post-test and evaluation, statements of credit for physicians will be issued within 30 business days.

Available Credit

  • 1.00 AMA PRA Category 1 Credit™
  • 1.00 Participation
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