Myelodysplastic Syndromes

“Transfusion independence is the key to my excitement [with] momelotinib as a drug. I will likely use it in most patients in the second-line setting,” says an expert from the University of Texas MD Anderson Cancer Center.

Investigators say that a phase 1/2 trial evaluating MGTA-117 in 2 hematologic malignancies has been paused following a serious adverse effect that may be related to the agent.

Imetelstat demonstrated clinically meaningful and statistically significant improvement in transfusion independence at 24 weeks among patients with myelodysplastic syndrome in the phase 3 IMerge trial.

Findings from the final analysis phase 3 SINTRA-REV trial indicated that lenalidomide lowered the risk of transfusion dependency when administered at a low dose in patients with myelodysplastic syndrome.

Among adult patients with low- or intermediate-risk myelodysplastic syndromes requiring red blood cell transfusions, luspatercept-aamt improved red blood cell transfusion independence with concurrent hemoglobin increase compared with epoetin alfa.

Results from a phase 1/2 trial highlighted improved clinical outcomes for patients receiving eltanexor monotherapy for relapsed/refractory myelodysplastic syndrome.

The highly selective JAK inhibitor momelotinib is being considered for the treatment of patients with myelofibrosis by the FDA following submission of a new drug application for this indication.

Combining magrolimab with azacitidine led to manageable anemia in patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia.

Early discontinuation of a hypomethylating agent was associated with an increase in economic burden for those with refractory anemia with excess blasts, a diagnosis that overlaps with high-risk myelodysplastic syndrome.

JSP191 plus fludarabine and low-dose total body radiation to target CD117 was a safe strategy to induce facilitation of full donor myeloid chimerism and clear minimal residual disease in older patients with myelodysplastic syndrome and acute myeloid leukemia receiving non-myeloablative allogenic hematopoietic cell transplantation.

Patients who underwent allogeneic hematopoietic cell transplantation using younger matched unrelated donors had a better disease-free survival compared who those who used older matched sibling donors.

CD123- and CD3-engaging bispecific antibody, APVO436, caused cytokine release syndrome that could be managed through the use of steroids in patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

Patients with low-risk myelodysplastic syndrome saw more durable responses and higher tolerability when taking luspatercept-aamt compared with the placebo.

Patients with myelodysplastic syndromes showed favorable event-free survival with allogeneic stem cell transplant compared with those being treated with continuous 5-azacytidine.

The combination of venetoclax and azacitidine will be considered as a treatment for patients with newly diagnosed, higher-risk myelodysplastic syndrome.

Data presented from the phase 3 SIMPLIFY 1 and SIMPLIFY 2 trials at 2021 EHA indicate success of momelotinib in extending overall survival in patients with myelofibrosis who achieve transfusion independence.

A study published in The Lancet Hematology found an increased risk of developing myelodysplastic syndrome and acute myeloid leukemia when patients with cancer were treated with PARP inhibitors compared with placebo.

Data from initial dose cohorts of a phase 1/2 trial indicated the agent was found to safely drive natural killer cell proliferation in patients with high-risk myelodysplastic syndromes and acute myeloid leukemia.

The FDA granted breakthrough therapy designation to magrolimab for the treatment of newly diagnosed myelodysplastic syndrome.

The study evaluated the efficacy and safety of IV rigosertib in patients with high-risk MDS who had progressed on, failed to respond to, or relapsed after previous treatment with an HMA within 9 cycles over the course of 1 year after initiation of HMA treatment.

Takeda Pharmaceutical Company Limited announced the FDA granted pevonedistat, its investigation NEDD8-activating enzyme inhibitor, breakthrough therapy designation to treat patients with higher-risk myelodysplastic syndrome.

The FDA has approved an oral combination of decitabine and cedazuridine for adult patients with myelodysplastic syndromes.

The FDA approved the first and only erythroid maturation agent for the treatment of anemia in patients with lower-risk myelodysplastic syndromes.

The study, published in JAMA Oncology, examined whether treatment of 22 solid tumor types was associated with two therapy-related conditions.

In this first part of our two-part review, we introduce mutation profiling as a relevant clinical tool for hematologists treating patients with myeloid malignancies.