^
    1d
    Luspatercept in Preventing Poor Erythroid Engraftment for Hematological Malignancies With Moderate to Severe Myelofibrosis (clinicaltrials.gov)
    P2/3, N=196, Not yet recruiting, Nanfang Hospital, Southern Medical University
    New P2/3 trial
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    Reblozyl (luspatercept-aamt)
    2d
    ODYSSEY: Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis (clinicaltrials.gov)
    P2, N=68, Recruiting, GlaxoSmithKline | Trial completion date: Nov 2026 --> Mar 2028 | Trial primary completion date: Oct 2026 --> Mar 2027
    Trial completion date • Trial primary completion date
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    Reblozyl (luspatercept-aamt) • Ojjaara (momelotinib)
    7d
    ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions (clinicaltrials.gov)
    P3, N=360, Active, not recruiting, Bristol-Myers Squibb | Recruiting --> Active, not recruiting
    Enrollment closed
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    Reblozyl (luspatercept-aamt)
    21d
    A phase I open label study of fostamatinib, a SYK inhibitor, in patients with lower-risk myelodysplastic syndrome and chronic myelomonocytic leukemia. (PubMed, Leuk Lymphoma)
    Lower-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML) are commonly managed with erythropoiesis-stimulating agents, luspatercept, or hypomethylating agents. There were no dose-limiting toxicities. Although safe, fostamatinib demonstrated no clinical benefit, underscoring the need for alternative strategies to modulate inflammatory signaling in MDS/CMML.
    P1 data • Journal
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    SYK (Spleen tyrosine kinase)
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    Reblozyl (luspatercept-aamt) • Tavalisse (fostamatinib)
    24d
    QOL-ONE Phoenix: Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to Myelodysplastic Syndromes with del5q refractory/resistant/intolerant to Prior Treatments, Who Require Red Blood Cell Transfusion. (2024-519310-31-00)
    P1/2, N=22, Recruiting, Associazione Qol-One | Not yet recruiting --> Recruiting
    Enrollment open
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    Reblozyl (luspatercept-aamt)
    28d
    Overexpression of the TGF-β target CCN2 in megakaryocytes: a common feature of MDS with mutated SF3B1 : Uncovering novelinsights into the bone marrow microenvironment in MDS. (PubMed, Virchows Arch)
    In aggregate, these findings may contribute to a better understanding of disease pathophysiology and help elucidate the role of potentially clinically relevant TGF-β signaling. This is particularly significant given the clinical use of agents targeting TGF-β-signaling such as luspatercept, as well as the emergence of several CCN2-targeting therapies currently undergoing clinical or preclinical evaluation with promising results.
    Journal
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    RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TGFB1 (Transforming Growth Factor Beta 1) • CTGF (Connective tissue growth factor)
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    RUNX1 mutation • ASXL1 mutation • SF3B1 mutation
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    Reblozyl (luspatercept-aamt)
    1m
    A Trial Comparing Three Different Treatment Options for Adults With Low-Risk Myelodysplasia and Anemia (A MyeloMATCH Treatment Trial) (clinicaltrials.gov)
    P2, N=270, Not yet recruiting, National Cancer Institute (NCI)
    New P2 trial
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    emavusertib (CA-4948) • Reblozyl (luspatercept-aamt) • Retacrit (epoetin alfa-epbx)
    1m
    A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain (clinicaltrials.gov)
    P=N/A, N=1300, Active, not recruiting, Bristol-Myers Squibb
    New trial
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    lenalidomide • Reblozyl (luspatercept-aamt)
    2ms
    The Efficacy and Safety of Luspatercept in Improving Early Anemia After HSCT (clinicaltrials.gov)
    P2, N=20, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China
    New P2 trial
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    Reblozyl (luspatercept-aamt)
    2ms
    A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East (clinicaltrials.gov)
    P=N/A, N=200, Recruiting, Bristol-Myers Squibb | Not yet recruiting --> Recruiting | Initiation date: Oct 2025 --> Feb 2026
    Enrollment open • Trial initiation date • Real-world evidence
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    Reblozyl (luspatercept-aamt)
    2ms
    A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States (clinicaltrials.gov)
    P=N/A, N=418, Completed, Bristol-Myers Squibb | Active, not recruiting --> Completed | Trial completion date: Jun 2025 --> Mar 2025
    Trial completion • Trial completion date
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    Reblozyl (luspatercept-aamt)
    2ms
    TGFβ-Mediated Overexpression of Podoplanin Serves as a Potential Diagnostic Biomarker in Acute Promyelocytic Leukemia. (PubMed, Mol Carcinog)
    Early diagnosis of acute promyelocytic leukemia (APL), driven by PML-RARA oncoprotein, is vital for survival, as delays can cause fatal coagulopathy without prompt therapeutic intervention of all-trans retinoic acid and arsenic trioxide...Pharmacological inhibition of TGF-β1 ligand using luspatercept reduced SMAD phosphorylation and PDPN expression, indicating TGF-β/SMAD transcriptionally regulates PDPN. Additionally, ELISA-based serum profiling showed significantly elevated TGF-β1 levels in APL patients compared to non-APL AML (p < 0.0001). These findings identify PDPN overexpression as a downstream consequence of TGF-β/SMAD signaling and highlight its potential as a diagnostic biomarker for APL.
    Journal
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    PML (Promyelocytic Leukemia) • TGFB1 (Transforming Growth Factor Beta 1)
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    arsenic trioxide • Reblozyl (luspatercept-aamt)