Reblozyl (luspatercept-aamt) / BMS, Merck (MSD) 
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 2 Diseases   40 Trials   40 Trials   1659 News 


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  • ||||||||||  Journal:  Advancing Drug Development in Myelodysplastic Syndromes. (Pubmed Central) -  Jan 12, 2025   
    Challenges to advancing drug development in MDS are multifold but may be grouped into specific categories including criteria for risk stratification and eligibility, response definitions, time-to-event endpoints, transfusion endpoints, functional assessments, and biomarker development. Strategies to address these challenges and optimize future clinical trial design for patients with MDS are presented here.
  • ||||||||||  Journal:  Beyond HMAs: Novel Targets and Therapeutic Approaches. (Pubmed Central) -  Dec 14, 2024   
    Furthermore, imetelstat has recently been added to the FDA-approved therapeutic armamentarium for lower-risk MDS...While several novel hypomethylating agent combinations have and are being studied in large randomized phase 3 clinical trials, including the combination of azacitidine and venetoclax, no combination to date have improved overall survival to azacitidine monotherapy...Despite recent advancements, the lack of therapeutic agents, particularly after the failure of first line therapy in higher risk MDS, continues to be a major hurdle in the management of MDS. In this review, we discuss the current treatment landscape of MDS and provide an overview of novel agents currently in clinical development that have the potential to alter our current treatment paradigms.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Trial primary completion date:  Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms (clinicaltrials.gov) -  Dec 9, 2024   
    P2,  N=70, Recruiting, 
    In this review, we discuss the current treatment landscape of MDS and provide an overview of novel agents currently in clinical development that have the potential to alter our current treatment paradigms. Trial primary completion date: Sep 2024 --> Jun 2025
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Long-Term Real-World Follow-up Study of Luspatercept for Lower-Risk Myelodysplastic Neoplasms () -  Dec 7, 2024 - Abstract #ASH2024ASH_8767;    
    It is noteworthy that relapsed or refractory to prior therapy and IPSS-M higher-risk was not a predictive factor of luspatercept treatment, either in univariate or multivariate analysis.Conclusion Luspatercept was effective for refractory LR-MDS patients in the long-term study, with mild side effects. Serum EPO < 500 IU/L was the only independent predictor for higher HI-E rate.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Efficacy of Luspatercept on Transfusion Burden and Cytopenias in Intermediate, High, and Very High Risk Myelodysplastic Syndromes () -  Dec 7, 2024 - Abstract #ASH2024ASH_8752;    
    Analysis of available NGS data did not reveal any genetic markers associated with response.Conclusions : These findings demonstrate clinical activity with luspatercept in patients with intermediate, high, and very high risk MDS, characterized by improvement in RBC transfusion needs across NTD, LTB, and HTB patients, as well as evidence of platelet and neutrophil responses among thrombocytopenic and neutropenic patients, respectively. Given the high burden of cytopenias in patients with higher-risk MDS, these data support further evaluation of luspatercept in this population, including better understanding of mechanisms of response and combination agents.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Safety and Efficacy of Luspatercept in Low-Risk Myelodysplastic Neoplasms with Anaemia () -  Dec 7, 2024 - Abstract #ASH2024ASH_8751;    
    Most adverse events were low-grade and mangeable. Low transfusion frequency and low serum erythropoietin concentration but ring sideroblasts nor SF3B1 mutation correlated with efficay.PROSPERO CRD registration number : CRD42024513235 Key Points : 1.Luspatercept was safe and effectiveive in people with low-risk MDNswith anaemia.2.Low transfusion frequency and low serum erythropoietin concentration but ring sideroblasts nor SF3B1mutation correlated with efficay.
  • ||||||||||  tebapivat (AG-946) / Agios Pharma
    A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes () -  Dec 7, 2024 - Abstract #ASH2024ASH_8746;    
    P2
    Low transfusion frequency and low serum erythropoietin concentration but ring sideroblasts nor SF3B1 mutation correlated with efficay.PROSPERO CRD registration number : CRD42024513235 Key Points : 1.Luspatercept was safe and effectiveive in people with low-risk MDNswith anaemia.2.Low transfusion frequency and low serum erythropoietin concentration but ring sideroblasts nor SF3B1mutation correlated with efficay. Key exclusion criteria include : history of acute myeloid leukemia; secondary MDS; prior exposure to a PK activator, including tebapivat in the phase 2a portion of this trial; prior exposure to disease-modifying agents including imetelstat, lenalidomide, hypomethylating agents, and immunosuppressive therapies; treatment with ESAs
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Registry of Transfusion-Dependent Beta-Thalassemia (TDT) Cases in Spain Molecularly Characterized over the Past 20 Months () -  Dec 7, 2024 - Abstract #ASH2024ASH_7337;    
    Fourteen were treated with luspatercept...The diversity of genotypes and variability in transfusion needs highlight the complexity of managing this condition and the necessity for individualized treatment strategies. This work underlines the importance of molecular characterization in the management of ?-thalassemia TDT and the need for a personalized approach based on phenotype and molecular data, which can optimize treatment, adjust transfusion frequency, iron chelator dosage, and monitor ferritin levels, thus improving patients' quality of life.
  • ||||||||||  Rytelo (imetelstat) / Geron, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Journal:  Treatment of high risk myelodysplastic syndrome. (Pubmed Central) -  Dec 5, 2024   
    Current challenges encompass to decrease the relapse risk, the main cause of HSCT failure. This review will summarize current knowledge of options of transplant- and non-transplant treatment approaches for these patients and demonstrate the unmet clinical need for more effective therapies.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Review, Journal:  Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing. (Pubmed Central) -  Nov 27, 2024   
    Among adults, the only available drug is luspatercept, which is currently undergoing clinical trials in pediatric populations. However, in many countries around the world, the new therapeutic options remain a mirage, and even transfusion therapy itself is not guaranteed for most patients, while the choice of iron chelation therapy depends on drug availability and affordability.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Pregnancy and Childbirth in Women with Thalassemia: Past and Present (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_6760;    
    Exclusive or mixed breastfeeding was chosen for 66 children, with an increasing trend over time (Rp = 0.40, p = 0.07). This analysis confirms that many aspects of pregnancy in women with TDT, as well as neonatal characteristics, have changed over time along with the underlying disease.
  • ||||||||||  Association between Targeted Therapy and Survival in Patients with MDS (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_5741;    
    In addition, our study confirms that a significant number of biomarker-positive MDS patients do not receive targeted therapies, highlighting challenges such as disparities in access to care and provider awareness. Addressing these issues is essential to ensure all eligible patients benefit from advancements in targeted therapies, ultimately improving survival and quality of life.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Luspatercept Reduces Clonal Hematopoiesis-Associated Cardiac Stress Via Modulation of mTORC1 and Inflammatory Signaling Pathways (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_4072;    
    P3
    Conclusions : Our reverse and forward translational approach identified novel mechanisms of luspatercept that may have potential implications in patients with CH-related mutations and associated consequences (hematologic and cardiovascular). Moreover, these findings offer insights into how luspatercept treatment can modify cellular and tissue microenvironments, which could be leveraged in tailored therapeutic approaches, either alone or in conjunction.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Rap-536, a Murine Luspatercept Analog Ameliorates Anemia and Vaso-Occlusion in Experimental Model of Sickle Cell Disease (Room 31 (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_2417;    
    The observed synergy with HbF inducers like HU, TZM and FTX6058 suggests potential combination strategies to enhance therapeutic outcomes. These findings warrant clinical evaluation of luspatercept in SCD patients, aiming to address both anemia and vaso-occlusive complications in this patient population with high unmet medical need.
  • ||||||||||  Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
    Combining ESA and Luspatercept in Non-RS MDS Patients Having Failed ESA - Results of the Phase 1-2 Part a of the GFM Combola Study (Ballroom 20AB (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_2200;    
    Based on a TITE-BOIN-ET design, Epoetin alfa was administered weekly at dose concentrations ranging from 30 000UI to 60 000 UI...All patients had resistance to ESA and 7 had also received Revlimid (n=3), IDH inhibitors (n=2), Thalidomide (n=1), AZA (n=1)...Based on the results of this Phase 1 study, the dosing schedule Luspatercept 1.75 mg/kg/21d and EPO 60 000 UI/w, that balanced clinical efficacy and safety profile was selected as the RP2D. This regimen is being compared to Luspatercept 1.75 mg/kg/21d in the ongoing randomized Phase 2 study.