Blood Journals Portfolio/LinkedIn
Sep 9, 2025, 23:00
Blood Podcast Alert: LSD1 Inhibitors in SCD, Ruxolitinib+Dex in HLH, Low VWF Insights
Blood Journals Portfolio shared on LinkedIn:
”September is Sickle Cell Disease Awareness Month and together we are working to conquer SCD!
In this episode, “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” the methodology of three articles are discussed that feature sickle cell disease studies.
Key points are:
- Ruxolitinib plus Dexamethasone in Newly Diagnosed Adult Hemophagocytic Lymphohistiocytosis (HLH): A prospective study demonstrated that the combination of ruxolitinib and dexamethasone (Ru-D regimen) had a high response rate and led to favorable short-term survival outcomes in adult HLH patients. All patients responded to the treatment, and no significant adverse effects were observed.
- Lysine-Specific Demethylase-1 (LSD1) Inhibitors in Sickle Cell Disease: Novel, potent, and orally bioavailable LSD1 inhibitors have been shown to induce fetal hemoglobin synthesis in a sickle cell disease mouse model. These inhibitors effectively reduce reactive oxygen species and improve red blood cell lifespan, offering a promising new class of therapies for sickle cell disease and other beta-globinopathies.
- Clinical Characteristics of Patients with Low Von Willebrand Factor: Insights into the clinical phenotype and pathophysiological mechanisms underlying qualitative low von Willebrand factor (VWF) have been explored. Patients with plasma VWF levels between 30 to 50 IU/dL often present with mucocutaneous bleeding and may require tailored management strategies.
Listen to the full episode here.
What are your thoughts on these advancements in sickle cell disease research?”
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