Vol 142 Dec 2023
1. Familial platelet disorder with associated myeloid malignancies
-RUNX1 mutation
- associated GI symptoms and allergy symptoms in a high percentage
-AML, MDS, CMML, ALL, Smoldering myeloma
-plt study may show findings similar to aspirin effect
-3/4 pt had bone marrow dysmegakaryopoiesis
2. Gene therapy for Sickle Cell ( March 2024)
Exa-cel uses gene editing to infuse patient's own CD34positive HSC after pt has received myeloablative chemo. This is a non viral cell therapy.
3. Arterial occlusive disease is increased with ponatinib, nilotinib and dasatinib. Not with imatinib, and probably not with bosutinib. These include MI, TIA, PAD. ( March 2024)
4. Transfusion threshold in adults with thalassemia ( March 2024)- maintain Hb > 10.5 gm. There is a 9% improvement in survival compared to those with Hb less than 10 gm.
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