Blood Podcast Ruxolitinib plus dexamethasone in newly diagnosed adult hemophagocytic lymphohistiocytosis; lysine-specific demethylase-1 inhibitors in sickle cell disease; and clinical characteristics of patients with low von Willebrand factor
Jul 21, 2025
Discover the latest in hematological treatments, including the promising combination of ruxolitinib and dexamethasone for hemophagocytic lymphohistiocytosis. Delve into innovative lysine-specific demethylase-1 inhibitors that show potential for treating sickle cell disease. The discussion also covers the complexities of low von Willebrand factor levels, highlighting clinical implications and the necessity for personalized treatment approaches.
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Ruxolitinib's Promise in Adult HLH
- Ruxolitinib plus dexamethasone (RUD) shows 86% overall response rate in adult HLH with unknown triggers.
- RUD is safer and more effective than etoposide-based regimens, warranting phase 3 trials.
New LSD1 Inhibitors for Sickle Cell
- Novel reversible LSD1 inhibitors increase fetal hemoglobin and reduce sickling in sickle cell mice.
- These inhibitors improve safety and anemia compared to older irreversible LSD1 inhibitors.
Distinct Profiles in Low VWF Patients
- Patients with low VWF activity but antigen >50 (low VWFQL) differ from type 2 VWD genetically and functionally.
- Low VWFQL and low VWFQT may require management like mild type 1 VWD rather than type 2.