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Leave No Patient Behind: Rare Malignancies and Unique Populations

December 9, 2023
Diego Adrianzen Herrera, MD, MSc, (@diegoah66)
Division of Hematology & Oncology, Larner College of Medicine, University of Vermont, Burlington

The “War on Cancer” was launched with President Nixon’s National Cancer Act of 1971, and bellicose metaphors for cancer treatment have been ubiquitous in mass media since. Conceptualizing an illness status as the “enemy” is problematic and encourages a disease-centered view of medicine that undermines the importance of individual patients’ characteristics. However, even in the era of personalized medicine, we can rescue one important notion from the war analogy: Leave no person (patient) behind. Some conditions and populations can be particularly problematic for the practicing hematologist, but we strive to provide the highest quality of care for all our patients and should be ready to take on the challenge. The #ASH23 Education Program has organized special sessions to help us in this endeavor.  

Anita D’Souza, MD, will chair the session How Do We Enhance Results in Rare Hematologic Malignancies? (Sunday, 9:30 a.m. – 10:45 a.m., Convention Center, Room 29). The session addresses rare hematologic cancers which can be difficult to diagnose and treat, as practicing clinicians only encounter them sporadically and lack established treatment guidelines. Oussama Abla, MD, will provide an update on Langerhans cell histiocytosis, addressing emerging targeted therapies and treatment modalities, particularly focusing on patients with high-risk disease and central nervous system involvement, who often endure long term sequelae. Deepti Radia, MD, will present a comprehensive breakdown of the diagnostic procedures, risk stratification, and therapeutic options in systemic mastocytosis, a highly heterogeneous hematologic neoplasm with ominous consequences in cases of advanced disease. This talk is timely, as a new generation of agents targeting the KIT D816V mutation (some, in fact, highlighted at this year’s meeting) make their foray into the therapeutic landscape. Finally, Dr. D’Souza’s session will specifically address the symptoms and clinical features that should trigger suspicion for amyloidosis, along with a systematic approach for its diagnosis, which is critical to prevent permanent organ damage resulting from delayed diagnosis. We caught up with Dr. D’Souza, who described this session overall as “illuminating,” taking us on a “journey covering a roadmap to early detection of amyloidosis, personalized therapies for systemic mastocytosis, and cutting-edge treatments for high-risk Langerhans cell histiocytosis,” cementing this session as a must-see. 

Beyond rare diseases, treatment of common hematologic malignancies in certain patient populations can also be challenging for the practicing hematologist. Adolescents and young adults (AYAs) are an important example, comprising a unique group, affected by significant physical and psychological change, and social circumstances resulting in risk of diagnosis delay, suboptimal access to care, and sometimes limited engagement with health professionals. Tamara Miller, MD, will chair the session Not Kids Anymore and Not Yet Adults: How Do We Treat Adolescent and Young Adults (AYA) Patients with ALL? (Monday, 2:45 p.m. – 4:00 p.m., Manchester Grand Hyatt, Grand Hall B). The session will focus on: (1) current outcomes associated with upfront therapy of ALL with “adult” or “pediatric” chemotherapy regimens; (2) the particular psychosocial and genetic differences between AYAs and pediatric patients affecting survival, risk stratification, and frequency of adverse events; and (3) existing strategies to overcome poor AYA outcomes through the introduction of immunotherapy, targeted therapy, minimal residual disease assessment, and stem cell transplantation. To further drive the point home, the Special-Interest Session ASH Guidelines on ALL in Adolescents and Young Adults (Monday, 10:30 a.m. – 12:00 pm., Manchester Grand Hyatt, Grand Hall C) will provide a comprehensive overview of what everybody really needs to know about ALL in AYAs. You don’t want to miss these sessions. 

Less common but equally important hematologic diseases affecting AYAs include bone marrow failure syndromes. Emma M. Groarke, MD, will chair the session Inherited Bone Marrow Failure Syndromes – From Pediatrics to Adult (Saturday, 2:00 p.m. – 3:15 p.m., Convention Center, Room 24). The session will address Fanconi anemia and telomere biology disorders, inherited bone marrow failure syndromes that are increasingly recognized in AYAs. Presenters will focus on the suggestive clinical features, specialized laboratory testing required to make a diagnosis, and existing evidence-based recommendations for managing these conditions. Dr. Groarke dove deeper into the rationale for these discussions: “I think the main point of this session is that germline disease is increasingly recognized in patients presenting with marrow failure and is something we should think about in adults and not just children and adolescents.” While we can’t all become experts in telomeropathies, we can surely benefit from a deeper appreciation of the many faces of these disorders and the complexity of diagnosing and managing them in AYAs. 

Collectively, these Education Program sessions are an exciting opportunity to learn about conditions and patient populations that may be challenging in practice. These are groups for whom timely diagnosis and appropriate treatment stand to make the greatest impact. We therefore invite you to explore the unique and often unmet needs of these patients, as we each respond to that compelling call: Leave no patient behind. 


Dr. Adrianzen Herrera indicated no relevant conflicts of interest. 


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