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13min chapter
The treatment landscape and challenges in rare diseases: BPDCN, CMML, MPN-AP/BP, and VEXAS
VJHemOnc Podcast
Understanding VEXAS and its Therapeutic Challenges
This chapter explores VEXAS, a newly recognized disease due to mutations in the UBA1 gene, alongside its connection to inflammatory and hematologic issues. It examines treatment strategies, including steroids and JAK inhibitors, and discusses the complexities involved in determining patient eligibility for allogeneic stem cell transplants. Additionally, the chapter emphasizes the significance of risk stratification in treatment decisions and the development of targeted therapies for rare diseases like VEXAS and Chronic Myelomonocytic Leukemia (CMML).
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