SICKLE CELL ANEMIA & RELATED SYNDROMES
Crizanlizumab-tmca, a monoclonal antibody, reduces vasoocclusive episodes by one-half in those with sickle cell anemia.
A new therapeutic strategy for polycythemia vera is induction of apoptosis via the p53 pathway through pharmacologic inhibition of human double minute 2.
Fedratinib, a selective JAK2 inhibitor, can lead to sustained reduction in spleen size and improvement in disease-associated symptoms in advanced stage myelofibrosis.
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.
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.
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.