Luspatercept significantly reduced transfusion burden and improved hemoglobin levels compared with placebo for transfusion-dependent anemia in patients with lower-risk myelodysplastic syndrome with ring sideroblasts.
Transfusion-dependent adults with beta-thalassemia who received luspatercept were more likely to achieve clinically meaningful reductions in transfusion burden compared with placebo.
Randomized study observed EFS benefit associated with deferasirox-based iron chelation therapy in lower-risk MDS.
Combination therapy achieved objective clinical improvements in disease status, including durable hematologic responses.
Immune checkpoint inhibitors ipilimumab and nivolumab were active in MDS, both individually in the setting of HMA failure and combined with 5-azacitidine in the frontline setting, producing promising survival and response outcomes with acceptable toxicity profiles.
Rapid and durable responses to the selective JAK1/JAK2 inhibitor ruxolitinib plus corticosteroids were observed across all patient subgroups defined by baseline steroid-refractory criteria, organ involvement, and disease stage.
In an intraindividual comparison, emicizumab prophylaxis was associated with a greatly reduced risk of treated bleeds compared with prior treatment with bypassing agents.
In MF patients with poor prognosis following failure of JAK inhibition, a high dose of imetelstat, an investigational telomerase inhibitor, was associated with markedly longer median OS compared with historical data.
In this early study, the addition of pulsed dexamethasone to eltrombopag as frontline therapy for ITP appeared safe and effective with prolonged responses.
Early initiation of defibrotide in adults with post-HSCT VOD/SOS improves Day +100 survival regardless of renal/pulmonary dysfunction status.
In this analysis of gastric biomarkers from 2 phase III trials, no significant gastric toxicity was associated with the use of avatrombopag to treat thrombocytopenia in patients with chronic ITP.
In ambulatory patients with cancer, rivaroxaban significantly reduced VTE with a low incidence of major bleeding, and with the use of the Khorana score cutoff of 2 or higher identifying patients at high risk of thrombotic events.