Blood Podcast: Season 1, Episode 21
In this week’s episode we’ll review data on the high frequency of germline RUNX1 mutations in patients with acute myeloid leukemia, learn more about the development of an international prognostic score that predicts time to first treatment in CLL patients with early, asympto Publish Changes matic disease, and explore the possibility of improving the activity of virus-directed immunotherapies against certain types of EBV tumors.
Epigenetic reprogramming sensitizes immunologically silent EBV1 lymphomas to virus-directed immunotherapy
Most Epstein-Barr virus (EBV)–associated lymphomas express viral proteins that are poorly immunogenic. Dalton and colleagues used a high-throughput screen to identify decitabine as a potent inducer of immunogenic EBV antigens and validate the drug’s ability to sensitize lymphomas to EBV-cytotoxic T lymphocytes in vitro and in vivo.
International prognostic score for asymptomatic early-stage chronic lymphocytic leukemia
A key question for many patients when first diagnosed with early-stage chronic lymphocytic leukemia is, “When will I need therapy?” Condoluci and an international consortium report a validated algorithm comprising clinical and laboratory measures to estimate the probability of requiring therapy within 5 years for individual patients.
High frequency of germline RUNX1 mutations in patients with RUNX1-mutated AML
POEMS syndrome is a rare plasma cell dyscrasia with variable manifestations. Bender and colleagues used a novel high-throughput sequencing technique to demonstrate that monoclonal l light-chain clones are detectable in the vast majority of patients with POEMS syndrome and that they show highly restricted VJ region usage that can help define and diagnose the disease.
Megakaryocyte migration defects due to nonmuscle myosin IIA mutations underlie thrombocytopenia in MYH9-related disease
Using a murine model, Pal and colleagues demonstrated that MYH9-RD mutations observed in patients impair megakaryocyte chemotaxis, disrupting migration towards the vasculature and interfering with proplatelet release, thereby causing macrothrombocytopenia.
Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum
Producing recombinant factor VIII in mammalian cells is inefficient. Poothong and colleagues provide novel insight into mechanisms that limit factor VIII secretion through endoplasmic reticulum stress, with implications for ongoing hemophilia A gene therapy clinical trials.
