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A Better To-Marrow

December 10, 2022

Imagine a better tomorrow … or, for the hematologists out there, should I say, a better to-marrow? With groundbreaking acceleration in bone marrow research and discovery, we are on the cusp of great advances in the diagnosis and treatment of primary bone marrow disorders. As underscored by the Education Spotlight Sessions at this year’s meeting, the future looks especially bright for those with a range of hematologic disorders, including bone marrow failure syndromes, histiocytic disorders, and myeloid neoplasms.

To many, there is nothing more frustrating than peering into an empty marrow. The lack of cellularity can be frightening. When stem cells go awry and bone marrow failure ensues, making the correct diagnosis is half the remedy. However, the process of arriving at a final diagnosis and the familial implications involved in diagnosing a potentially heritable disorder are not always clear-cut. As was highlighted in the Education Spotlight Session Bone Marrow Failure Syndrome: Diagnostic Principles in 2022, the key is making an accurate diagnosis by combining key pathologic findings and molecular genetics, along with the understanding and foresight to incorporate genetic counselors to better clarify potential ethical and familial challenges that may arise.

In contrast to the desolation of an empty marrow, for hematopathologists, there is no greater sight than histiocytes. In fact, what better way to strike up a conversation with a fellow hematologist than to discuss histiocytes? These cells leave the bone marrow as monocytes and then home in on target tissues, where they morph into dendritic cells and macrophages called histiocytes. Though disorders of these cells are rare, the implications of common alterations in the MAP kinase pathway make them especially vulnerable to targeted therapies. Excellent talks by Drs. Carl Allen and Eli Diamond on both the genetic drivers of histiocytic disorders and the efficacy of targeted treatments in their management shed light on new strategies to better control these rare debilitating disorders. Hence the session’s title “New Horizons for Histiocyte Disorders,” which aptly describes the bright future for targeted therapeutics in these diseases.

The discussion of histiocytes illuminates a broader theme in the field. While there are advances in targeting genomic alterations in these rare myeloid-derived disorders, has anyone else wondered why, for the most part, myeloid disorders have had less immune-based therapeutic options than lymphoid disorders? For the typical lymphoid disorder, there is an arsenal of potential therapies including uniquely designed chimeric antigen receptor T cells (CAR-Ts), bispecific T-cell engagers (BiTEs), and creative antibody drug conjugates; yet, in the myeloid space, this is unfortunately not the case. Yes, the CD33 antibody-drug conjugate gemtuzumab ozogamicin (which has had its own wild adventure on and off the market) is approved for acute myeloid leukemia, and the CD123 drug conjugate tagraxofusp is approved for blastic plasmacytoid dendritic cell neoplasm. But there is still a gaping lag in utilization of immunotherapy in the myeloid versus lymphoid space. The Monday Spotlight Session Immunotherapies for Myeloid Neoplasms: Are We Ready for Primetime? promises a terrific introduction to the state of immunotherapeutics in myeloid malignancies, with a focus on the future application of CAR-Ts, BiTEs and immunotherapy in the myeloid space.

Just as equity between myeloid and lymphoid neoplasms is desired, so, too, is making sure that clinical trials are inclusive of diverse populations and reflective of the underlying patient population. The Spotlight Session Underrepresented Minorities in Clinical Trials for Hematologic Malignancies: What’s the Data on the Data? will provide a meaningful discussion and assessment of the critical work still needed to improve underrepresented minority registration in clinical trials for hematologic malignancies. Finding solutions and removing barriers to enrollment so that clinical trials can match real-world patient populations is necessary for improving the health of everyone in our society.

Overall, the themes of the Education Spotlight Sessions reflect a nascent merging of the past and the hopeful future, suggesting that a better to-marrow is on the horizon.

Dr. Hermel indicated no relevant conflicts of interest


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