IS IT SYSTEMIC MASTOCYTOSIS?

Page 1

Best Practices for Diagnosis and Management


Tracy I. George, MD Professor of Pathology University of Utah School of Medicine President and Chief Medical Officer ARUP Laboratories Salt Lake City, Utah

Matthew J. Hamilton, MD Assistant Professor of Medicine Harvard Medical School Lead Gastroenterologist, Brigham and Women’s Hospital Mastocytosis Center Boston, Massachusetts

Anne Maitland, MD, PhD Assistant Clinical Professor Icahn School of Medicine, Department of Medicine, Allergy & Clinical Immunology Immunologist, Chiari EDS Mast Cell Activation Disorders Center Mount Sinai South Nassau Oceanside, New York

Dr. Tracy George is the President and Chief Medical Officer at ARUP Laboratories and a professor of pathology at the University of Utah School of Medicine. She completed her MD and residency in anatomic pathology and laboratory medicine at the University of California San Francisco, with fellowships in hematopathology and surgical pathology at Stanford University in Stanford, California. Dr. George is board certified in anatomic pathology, clinical pathology, and hematology by the American Board of Pathology. Her research interests include mast cell disease and laboratory hematology. Author of more than 100 publications, Dr. George has recently been elected President of the International Society for Laboratory Hematology. She received the College of American Pathologists Lifetime Achievement Award in 2014.

Dr. Matthew Hamilton is an assistant professor at Harvard Medical School and lead gastroenterologist in the division of Gastroenterology, Hepatology, and Endoscopy at Brigham and Women’s Hospital (BWH). Dr. Hamilton is a graduate of Haverford College in Haverford, Pennsylvania, and the University of Maryland School of Medicine in Baltimore. A leading member of the research team at the BWH Crohn’s and Colitis Center, he has garnered national recognition for his research into the underlying inflammatory processes of inflammatory bowel disease (IBD). His research on inflammation is focused on the mast cells of the immune system and the role they may play in IBD. Dr. Hamilton has helped identify a subset of patients with mast cell activation syndrome (MCAS), wherein activated mast cells are stuck in the “on” position releasing substances that fuel IBD. The National Institutes of Health and the Crohn’s and Colitis Foundation of America are funding his mast cell research.

Dr. Anne Maitland is an assistant clinical professor at Icahn School of Medicine and an immunologist at Chiari ES Mast Cell Activation Disorders Center. She earned her MD and PhD from the University of Pennsylvania and completed her residency in Internal Medicine and fellowship in Allergy and Immunology at Brigham and Women’s Hospital in Boston, Massachusetts, and at Mount Sinai Hospital in New York, New York. A Fellow of the American College of Allergy, Asthma, and Immunology and a member of the American Academy of Allergy, Asthma, and Immunology, Dr. Maitland is very active in local communities, increasing awareness of immune-mediated disorders. She is also involved in research to continually improve the treatments for allergies, asthma, and recurrent infections. Her clinical focus includes the diagnosis and treatment of allergic skin disorders, allergic rhinitis (hay fever), drug allergies, food allergies/sensitivities, asthma, and recurrent infections. She was named one of The New York Times’ 2011 Super Doctors and one of America’s Top 21 Women’s Doctors by Lifescript.com in 2009.


Lawrence B. Schwartz, MD, PhD Charles & Evelyn Thomas Professor of Medicine Chair, Division of Rheumatology, Allergy & Immunology Virginia Commonwealth University Richmond, Virginia

Dr. Lawrence B. Schwartz received his MD and PhD in medicine and biochemistry from Washington University in St. Louis, Missouri, before completing an internal medicine residency at its Barnes Hospital and fellowships in allergy, immunology, and rheumatology at Brigham and Women’s Hospital in Boston, Massachusetts, and in diagnostic laboratory immunology at VCU. Dr. Schwartz joined the faculty at Harvard in 1980 as an assistant professor and in 1983 moved to VCU, where he is now the Charles and Evelyn Thomas Professor of Medicine, chair of the Division of Rheumatology, Allergy and Immunology, and program director of allergy and immunology. A member of the American Society for Clinical Investigation and the Association of American Physicians, he has chaired sections for numerous organizations, including the American Academy of Allergy, Asthma, and Immunology, the National Institutes of Health, and the American Board of Allergy and Immunology, and has served as President of the Clinical Immunology Society. At VCU, he has received awards for research and for technical innovations that include the discovery, purification, cloning, and characterization of human α/β tryptases; development of immunoassays for tryptases as biomarkers of disorders involving mast cells; identification of in vitro conditions for the development of human mast cells from progenitors and for culturing tissue-derived human mast cells; and the identification and characterization of different types of human mast cells.


TARGET AUDIENCE

Allergists/clinical immunologists, dermatologists, gastroenterologists, hematologists/oncologists, and other specialist clinicians who treat patients with nonadvanced systemic mastocytosis.

PROGRAM OVERVIEW

This Interactive Exchange™ educational program focuses on the identification and management of patients with nonadvanced systemic mastocytosis (SM). The program will open with a brief video segment featuring a 3D animation detailing the pathophysiological drivers of SM and their implications for the development of targeted therapies. Dynamic discussions among a multidisciplinary panel of experts will address challenges in the diagnosis and classification of nonadvanced SM, highlight recent clinical trial data on emerging targeted therapies, and emphasize real-world strategies for the comprehensive management of nonadvanced SM. The program will conclude with an engaging case study discussion highlighting the various clinical presentations of nonadvanced SM along with expert advice for practical and effective disease management. Please join us for this informative and engaging session.

EDUCATIONAL OBJECTIVES

Upon completion of this activity, participants will be better able to: • Describe clinically relevant pathophysiology in the development of systemic mastocytosis, including the effects of driver mutations on mast cell hyperactivation • Identify patients with nonadvanced systemic mastocytosis based on symptomatology and appropriate ancillary testing • Discuss the clinical profiles and trial evidence for current and emerging targeted treatment options in nonadvanced systemic mastocytosis

are required to disclose all financial relationships with any ineligible company within the past 24 months to Integritas Communications. All financial relationships reported are identified as relevant and mitigated by Integritas Communications in accordance with the Standards for Integrity and Independence in Accredited CE in advance of delivery of the activity to learners. The content of this activity was vetted by Integritas Communications to assure objectivity and that the activity is free of commercial bias. All relevant financial relationships have been mitigated. The faculty have the following relevant financial relationships with ineligible companies: Tracy I. George, MD

Consulting fees: Blueprint Medicines Corporation, Incyte Corporation, Celgene Corporation, BluPrint Oncology Concepts, UBC (United BioSource LLC), Allakos Inc. Contracted Research: Blueprint Medicines Corporation, Incyte Corporation, Celgene Corporation, Deciphera Pharmaceuticals, Inc., Cogent Biosciences, Inc.; all received by ARUP Laboratories

Matthew J. Hamilton MD

Consulting fees: Allakos Inc., Blueprint Medicine, GlaxoSmithKline, InveniAI

Anne Maitland, MD, PhD Lawrence B. Schwartz, MD, PhD

Consulting fees: Allakos Inc., AstraZeneca, Blueprint Medicines Corporation, Celidex, CSL Behring, Genentech, GLG Pharma Corporation, GlaxoSmithKline, Inves, Takeda. Contracted Research: Blueprint Medicines Corporation, Deciphera. Royalty: BioLegent, Genentech, Hycult Biotech, Millipore, Santa Cruz, Thermo Fisher-Phadia

• Tailor therapeutic regimens for patients with nonadvanced systemic mastocytosis based on symptoms, risk stratification, treatment responses, and appropriate consultations with other specialist clinicians

The planners and managers have the following relevant financial relationships with ineligible companies: Rebecca Robinson, PhD

Nothing to disclose

DISCLOSURES OF CONFLICTS OF INTEREST

Rose O’Connor, PhD, CHCP

Nothing to disclose

Integritas Communications adheres to the policies and guidelines, including the Standards for Integrity and Independence in Accredited CE, set forth to providers by the Accreditation Council for Continuing Medical Education (ACCME) and all other professional organizations, as applicable, stating those activities where continuing education credits are awarded must be balanced, independent, objective, and scientifically rigorous. All persons in a position to control the content of an accredited continuing education program provided by Integritas Communications

DISCLOSURE OF UNLABELED USE

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the US Food and Drug Administration. Integritas Communications do not recommend the use of any agent outside of the labeled indications. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of any organization associated


with this activity. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

DISCLAIMER

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed in this activity should not be used by clinicians without evaluation of patient conditions and possible contraindications or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

This program is not sponsored or programmed by the AAAAI. This activity is provided by Integritas Communications. This activity is supported by an independent educational grant from Blueprint Medicines Corporation. This activity is endorsed by The Mast Cell Disease Society, Inc. This live activity will not offer continuing medical education (CME) credit.


PROGRAM AGENDA 6:45 am – 6:55 am

Introductions

6:55 am – 7:05 am

What Is the Latest on Systemic Mastocytosis Pathophysiology?

7:05 am – 8:05 am

Meet the Professors Discussion on Systemic Mastocytosis

8:05 am – 8:20 am

Choose-a-Case: Prerecorded Patient Examples of Systemic Mastocytosis

8:20 am – 8:45 am

Question & Answer Session












CLINICAL GUIDELINES

The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Arber DA, Orazi A, Hasserjian R, et al. Blood. 2016;127(20):2391-2405. https://www.sciencedirect.com/science/article/pii/S0006497120301567

NCCN Clinical Practice Guidelines in Oncology: Systemic Mastocytosis. National Comprehensive Cancer Network.

https://www.nccn.org/professionals/physician_gls/pdf/mastocytosis.pdf

PATIENT RESOURCES

The Mast Cell Disease Society, Inc. (TMS) TMS is dedicated to providing multifaceted support to patients, families, and medical professionals in the community and to leading the advancement of knowledge and research in mast cell diseases through education, advocacy, and collaboration. https://tmsforacure.org/

The European Competence Network on Mastocytosis (ECNM) The goal of the ECNM is to improve disease-recognition, diagnosis, and therapy in patients with mastocytosis in Europe. https://ecnm.meduniwien.ac.at/

Genetic and Rare Diseases Information Center (GARD) GARD helps patients find information, services, experts, financial aid, and support groups. https://beta.rarediseases.info.nih.gov/

National Organization for Rare Disorders (NORD) NORD is a patient advocacy organization committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. https://rarediseases.org/

SUGGESTED READING

Results from Pioneer: a randomized, double-blind, placebocontrolled, phase 2 study of avapritinib in patients with indolent systemic mastocytosis. Akin C, Elberink HO, Gotlib J, et al. American Society of Hematology Virtual Conference; December 5-8, 2020. Abstract 1248. https://ash.confex.com/ash/2020/webprogram/Paper139367.html

Update on mastocytosis (part 1): pathophysiology, clinical features, and diagnosis. Azaña JM, Torrelo A, Matito A. Actas Dermosifiliogr. 2016;107(1):5-14. https://www.actasdermo.org/en-update-on-mastocytosis-part-1-articulo-S1578219015003108

Patients with mast cell activation symptoms and elevated baseline serum tryptase level have unique bone marrow morphology. Giannetti MP, Akin C, Hufdhi R, et al. J Allergy Clin Immunol. 2021;147(4):1497-1501.e1491. https://linkinghub.elsevier.com/retrieve/pii/S009167492031633X

Patient perceptions in mast cell disorders. Jennings SV, Slee VM, Zack RM, et al. Immunol Allergy Clin North Am. 2018;38(3):505-525. https://www.sciencedirect.com/science/article/pii/S0889856118300328

Catching the clinical and biological diversity for an appropriate therapeutic approach in systemic mastocytosis. Mannelli F. Ann Hematol. 2021;100(2):337-344.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7646220/

Systemic mastocytosis in adults: 2021 update on diagnosis, risk stratification and management. Pardanani A. Am J Hematol. 2021;96(4):508-525. https://onlinelibrary.wiley.com/doi/10.1002/ajh.26118

How we diagnose and treat systemic mastocytosis in adults. Scherber RM, Borate U. Br J Haematol. 2018;180(1):11-23. https://onlinelibrary.wiley.com/doi/10.1111/bjh.14967


Mast cells, mastocytosis, and related disorders. Theoharides TC, Valent P, Akin C. N Engl J Med. 2015;373(2):163-172. https://www.nejm.org/doi/full/10.1056/NEJMra1409760

Mastocytosis: lessons learned from the 2019 Society for Hematopathology/European Association for Haematopathology Workshop. Tzankov A, Duncavage E, Craig FE, et al. Am J Clin Pathol. 2021;155(2):239-266. https://academic.oup.com/ajcp/article/155/2/239/6032728

Clinical features and survival of patients with indolent systemic mastocytosis defined by the updated WHO classification. Trizuljak J, Sperr WR, Nekvindova L, et al. Allergy. 2020;75(8):1923-1934. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7115854/pdf/EMS87367.pdf

Bone marrow mastocytosis: a diagnostic challenge. Zanotti R, Tanasi I, Bernardelli A, et al. J Clin Med. 2021;10(7):1420. https://www.mdpi.com/2077-0383/10/7/1420/htm


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