Myelodysplastic Syndromes

For patients with MDS who are relapsed/refractory to erythropoietin stimulating agents, low-dose deferasirox demonstrated promising efficacy and tolerability outcomes.

Smoking may contribute to genetic mutations and disease progression in myelodysplastic syndromes, a recent study found.

The guideline update follows the FDA’s approval of imetelstat in patients with lower-risk MDS based on data from the phase 3 IMerge trial.

The safety profile of oral azacitadine was shown to be similar across the 200-mg and 300-mg arms in patients with lower- to intermediate-risk MDS.

Data from the phase 3 COMMANDS trial support the expanded European approval of frontline luspatercept for transfusion-dependent anemia in lower-risk myelodysplastic syndromes.

Members of the committee reviewed findings from the phase 2/3 IMerge trial assessing imetelstat in patients with transfusion-dependent anemia in myelodysplastic syndromes.

Treatment with rusfertide may reduce the use of phlebotomy and limit debilitating disease-related symptoms in patients with polycythemia vera based on findings from the phase 2 REVIVE trial.

Treatment with imetelstat produces robust activity in patients with relapsed/refractory myelodysplastic syndrome regardless of ring sideroblasts in the phase 3 IMerge trial.

Danazol was reviewed as an effective treatment option for patients with myelodyspplastic syndromes, according to a recently published article by Sangam Shah, MBBS, et al.

The combination of pelabresib plus ruxolitinib shows improvement in hemoglobin response in the MANIFEST-2 trial, supporting a potential shift in the future treatment of patients with JAK inhibitor–naive myelofibrosis.

No patients with myelodysplastic syndrome have any grade 3 or higher acute graft-versus-host disease following treatment with Orca-T in a phase 1b study.

If momelotinib receives approval in the European Union, it will become the only agent for the treatment of myelofibrosis coupled with moderate to severe anemia.

Data from a retrospective study spotlight a need for more effective first-line and second-line clinical management strategies to improve outcomes in patients with lower-risk myelodysplastic syndrome.

Ivosidenib monotherapy is now approved by the FDA for patients with relapsed/refractory myelodysplastic syndrome with an IDH1 mutation.

A real-world study found most patients with lower-risk myelodysplastic syndrome do not appear to require dose escalations of luspatercept.

The marketing authorization application for imetelstat in lower-risk myelodysplastic syndrome is based on findings from the phase 3 IMerge trial.

Characteristics including Ogata score and phenotypic aberrancies may correlate with mutational patterns that are predictive of treatment response in patients with myelodysplastic syndrome.

Luspatercept may provide clinical benefit regardless of subgroup and baseline mutation burden in transfusion dependent patients with myelodysplastic syndrome with anemia, according to Guillermo Garcia-Manero, MD.

Patients with dyserythropoiesis appear to have higher PD-L1 expression than those without in a study population of those with myelodysplastic syndromes.

Luspatercept, the use of which is supported by results from the phase 3 COMMANDS trial presented at 2023 ASCO, is now available for patients with low-risk myelodysplastic syndrome and anemia.

Findings from the phase 2/3 IMerge trial support the new drug application for imetelstat in the treatment of patients with transfusion-dependent anemia in lower-risk myelodysplastic syndrome.

Findings from a phase 1 study highlight that ivosidenib tablets did not raise any new safety signals in the treatment of patients with IDH1-mutated relapsed/refractory myelodysplastic syndromes.

Results from the phase 3 MOMENTUM trial support the use of momelotinib as a treatment for patients with myelofibrosis, including those with anemia or thrombocytopenia.

Investigators note that although therapy-related myelodysplastic syndromes or acute myeloid leukemia are not common, efforts to reduce treatment-associated toxicity in survivors of lymphoid neoplasms are needed given the poor prognosis associated with the diagnosis.

Investigators will submit data from the phase 3 ENHANCE trial evaluating magrolimab plus azacitidine in higher-risk myelodysplastic syndromes for presentation at a future medical meeting.

Selinexor plus ruxolitinib is under investigation as a treatment for JAK inhibitor-naïve patients with myelofibrosis as part of the phase 3 XPORT-MF-034 trial.

Eltrombopag may be an effective therapy for the ‘challenging condition’ of severe thrombocytopenia in patients with low-risk myelodysplastic syndromes.

The new drug application for imetelstat is based on findings from the phase 3 IMerge trial, in which the telomerase inhibitor outperformed placebo in the treatment of transfusion-dependent anemia.

A recent analysis of patients with several different hematologic malignancies finds that vulnerable patients report having worse health-related quality of life across all measures.

Patients with highly transfusion dependent, relapsed/refractory myelodysplastic syndrome appear to have long-lasting transfusion independence and increased hemoglobin following treatment with imetelstat.