In this open-label phase II trial, pelabresib was associated with clinical activity in patients with pretreated myelofibrosis who were refractory/resistant to, ineligible for, or intolerant to JAK inhibitors.
In this phase Ib study in previously untreated higher-risk MDS, venetoclax plus azacitidine was safe and appeared to induce rapid, durable responses and a high remission rate, with molecular responses observed across key mutational profiles.
Pevonedistat plus azacitidine did not improve EFS compared with azacitidine alone in patients with higher-risk MDS/CMML or low-blast AML.
Results from a phase II trial indicate that CPX-351 is as effective as first-line therapy in patients with higher-risk MDS, particularly to achieve blast clearance and as a bridge to allogeneic SCT.
In patients with high-risk or very high–risk MDS or newly diagnosed AML, sabatolimab in combination with HMA was generally well tolerated with durable efficacy.
In patients with MLNFGFR1, treatment with pemigatinib produced high and durable response rates despite previous treatment or HSCT.
In this phase II trial in patients with polycythemia vera, rusfertide was associated with rapid, sustained, durable hematocrit control; substantial reduction in phlebotomy; and improved patient-reported outcomes.
Patients with higher-risk MDS who received venetoclax and a hypomethylating agent as first-line therapy had higher response rates than those receiving HMA alone.
CPX-351 was associated with a tolerable safety profile and a high response rate in transplant-eligible patients with previously untreated HR-MDS.
The IPSS-M combines conventional parameters with mutations in 31 key genes to improve MDS risk stratification.
Treatment with lenalidomide and/or eltrombopag yielded favorable outcomes in patients with low-risk/intermediate-risk MDS and symptomatic anemia or thrombocytopenia, including hematologic improvement in 35% and a median duration of response of 1.5 years.
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