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SICKLE CELL ANEMIA & RELATED SYNDROMES
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Crizanlizumab-tmca, a monoclonal antibody, reduces vasoocclusive episodes by one-half in those with sickle cell anemia.
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A new therapeutic strategy for polycythemia vera is induction of apoptosis via the p53 pathway through pharmacologic inhibition of human double minute 2.
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PRIMARY MYELOFIBROSIS
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Fedratinib, a selective JAK2 inhibitor, can lead to sustained reduction in spleen size and improvement in disease-associated symptoms in advanced stage myelofibrosis.
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CHRONIC MYELOID LEUKEMIA
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In patients with chronic myeloid leukemia who have not responded to treatment with multiple tyrosine kinase inhibitors, the novel allosteric inhibitor asciminib can be tried. It has shown a 54% complete hematologic response rate and a 48% sustained major molecular response in heavily pretreated patients.
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Moxetumomab pasudotox is a recombinant CD22-targeting immunotoxin recently approved for patients with refractory hairy cell leukemia. It has shown a durable complete response rate of 31% in the pivotal trial.
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A fixed-dose combination of daratumumab (an anti-CD38 monoclonal antibody) plus hyaluronidase-fihj has now received FDA approval for treatment of patients with plasma cell myeloma, including newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients.