Reblozyl (luspatercept-aamt)

P2, N=70, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Suspended --> Recruiting

New therapeutic options to reduce the red blood cell (RBC) transfusion burden have emerged since 2020 and include luspatercept and imetelstat. Erythropoiesis-stimulating agents and lenalidomide also address anemia and are generally recommended to start at the time of transfusion dependency, although emerging data suggest that an earlier start of these interventions might offer clinical benefits...Targeted therapy directed to the presence of an IDH1 mutation is U.S. Food and Drug Administration (FDA) approved for the rare IDH1 mutated MDS (<10% of the time) and consideration to use an IDH2 inhibitor for IDH2 mutated MDS (<5% of the time) is reasonable. Interestingly, IDH mutations seem to appear with increased frequency in older patients and in patients with underlying autoimmune/rheumatological disorders.1.

P3, N=106, Active, not recruiting, Bristol-Myers Squibb | Trial primary completion date: Jan 2026 --> Oct 2025

Drugs designed to promote erythropoiesis in MDS patients include erythropoiesis-stimulating agents such as recombinant human erythropoietin and TGF-β inhibitors such as luspatercept, which is approved for the treatment of anemia associated with MDS or β-thalassemia...Our findings demonstrate that GATA1 binds to the UBE2O promoter, thereby regulating UBE2O transcription and expression. Although further studies are needed to explore the implications of UBE2O in MDS treatment, our work provides potential strategies for novel therapeutic approaches in MDS.

P4, N=60, Recruiting, Peking University Sixth Hospital; Peking University Sixth Hospital

Several recent targeted drug approvals including luspatercept and imetelstat have greatly expanded the treatment arsenal for lower-risk MDS. Standard of care therapy options for high-risk MDS, in particular TP53-mutated, remain limited beyond HMAs and transplant and are an active area of investigation.

P4, N=85, Active, not recruiting, Bristol-Myers Squibb | N=60 --> 85 | Trial completion date: Apr 2026 --> Dec 2028 | Trial primary completion date: Apr 2026 --> Dec 2028

P=N/A, N=200, Not yet recruiting, Bristol-Myers Squibb

P3, N=70, Active, not recruiting, GWT-TUD GmbH | Recruiting --> Active, not recruiting | Trial completion date: Jun 2025 --> Feb 2027 | Trial primary completion date: Dec 2024 --> Oct 2026

Erythropoiesis-stimulating agents (ESAs) and lenalidomide are the standard therapies; however, their effectiveness is limited by resistance and patient selection criteria. Additionally, the real-world use of these new agents remains limited in some regions owing to issues with local introduction and reimbursement. This review summarizes recent clinical data on luspatercept and imetelstat, highlights their current limitations, and discusses areas for future research based on recent trial outcomes and evolving clinical practices.

While luspatercept demonstrated short-term efficacy in this patient with non-clonal SA, caution is warranted regarding potential masking of the underlying etiology. Concurrent monitoring of iron overload and early initiation of iron chelation therapy are crucial in alcoholic SA to prevent hepatic damage.

P=N/A, N=250, Completed, Bristol-Myers Squibb | Active, not recruiting --> Completed