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- ||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Clinical, Real-world evidence, Real-world: Over 90% of patients with lower-risk MDS who had low transfusion burden before receiving luspatercept achieved transfusion independence within the first 24 weeks of treatment, according to a real-world study by @MukherzSudipto of @ClevelandClinic. https://t.co/uGczg09hT4 (Twitter) - Jan 6, 2023
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Isn't this the comparable Luspatercept study? https://t.co/t3uRrUnVQU Discontinuation is in the single digits. Weird (Twitter) - Jan 4, 2023
- |||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
New P2 trial: A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (?)-Thalassemia (clinicaltrials.gov) - Dec 27, 2022
P2, N=177, Recruiting, Sponsor: Bristol-Myers Squibb
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Review, Journal: Efficacy and Safety of Luspatercept in the Treatment of β-Thalassemia: A Systematic Review. (Pubmed Central) - Dec 22, 2022
Luspatercept might make patients less likely to need RBC transfusions, improve their clinical results, and improve their quality of life. Adverse events were hyperuricemia, arthralgia, dizziness, influenza hypertension, and bone pain, but they were manageable.
- |||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Trial completion date, Trial primary completion date: INDEPENDENCE: An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions (clinicaltrials.gov) - Dec 19, 2022
P3, N=309, Recruiting, Sponsor: Celgene
Adverse events were hyperuricemia, arthralgia, dizziness, influenza hypertension, and bone pain, but they were manageable. Trial completion date: Oct 2024 --> Aug 2025 | Trial primary completion date: Oct 2024 --> Mar 2025
- ||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Trial completion: BEYOND: A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (?)-Thalassemia (clinicaltrials.gov) - Dec 19, 2022
P2, N=145, Completed, Sponsor: Celgene
Trial completion date: Oct 2024 --> Aug 2025 | Trial primary completion date: Oct 2024 --> Mar 2025 Active, not recruiting --> Completed
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Luspatercept and reduced CrCl. Uptodate says 27%, but studies do not mention it. DOI: 10.1056/NEJMoa1908892. DOI: 10.1007/s12325-021-01663-4. Any one experienced worsening GFR? #ASKRENAL @kdjhaveri (Twitter) - Dec 16, 2022
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Long-term safety and erythroid response with luspatercept treatment in patients with β-thalassemia. (Pubmed Central) - Dec 13, 2022
P2
The most common side effects were headache (27 patients), bone pain (20 patients), and muscle pain (14 patients) with more than 90% of these patients experiencing these side effects as mild severity. The results of this study show that in patients with either transfusion-dependent or nontransfusion-dependent β-thalassemia, luspatercept provides lasting reduction in anemia with mostly mild and predictable side effects.
- ||||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Retrospective data: CONGRESS | #ASH22 @MukherzSudipto @ClevelandClinic presents results from a retrospective analysis where >90% of patients with LR-MDS and low transfusion at baseline burden achieved transfusion independence within the first 24 weeks of luspatercept treatment. #MDS #leusm (Twitter) - Dec 11, 2022
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Targeting inflammation in lower-risk MDS. (Pubmed Central) - Dec 10, 2022
Presently, various clinical trials are evaluating inhibitors of cytokines and their receptors in low-risk MDS. Taken together, an inflammatory microenvironment can support the pathogenesis of clonal hematopoiesis and low-risk MDS, and clinical trials are evaluating anti-inflammatory strategies in these diseases.
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Review, Journal: Lower-risk myelodysplastic syndromes: Current treatment options for anemia. (Pubmed Central) - Dec 6, 2022
Research into new treatment options, especially after ESA failure, is needed. In this review, we will focus on the current therapeutic options for MDS-related anemia.
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Great insight on the longer-term benefit of luspatercept in transfusion-dependent lower-risk #MDS with ring sideroblasts https://t.co/IqenqSKAtl @Dr_AmerZeidan @Ramikomrokji (Twitter) - Dec 6, 2022
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Benefit of Continuing Luspatercept Therapy in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Who Did Not Achieve Red Blood Cell Transfusion Independence (RBC5F5F5F5F5F5F5F52_TI) by Week 25 in the MEDALIST Study () - Nov 30, 2022 - Abstract #DKK2022DKK_1982;
P3
This abstract was presented at EHA 2021 (Germing et al. HemaSphere 2021;5[S2]:EP915).
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Luspatercept Reduces Red Blood Cell Transfusions (RBCT) in Patients with Lower-Risk Myelodysplastic Syndromes (LR5F5F5F5F5F5F5F52_MDS) Regardless of Baseline Transfusion Burden (TB) in the MEDALIST Study () - Nov 30, 2022 - Abstract #DKK2022DKK_1981;
P3
This abstract was presented at EHA 2021 (GarciaManero et al. HemaSphere 2021;5[S2]:EP920).
- Hemlibra (emicizumab-kxwh) / Roche, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Persistence of Enrollment Among Commercial Enrollee Populations with Rare Hematologic Diseases: Implications for Long-Term Outcome Assessment and Value-Based Agreements () - Nov 29, 2022 - Abstract #ASH2022ASH_8033;
Hemophilia was defined using ≥1 claim with a HA/HB diagnosis code and ≥2 claims, on different dates, for on-demand or prophylaxis treatment specific to HA/HB (factor VIII and IX concentrates and emicizumab-kxwh)...Providers, payers, and manufactures will need to assess current data assets, available follow-up, and innovative data-sharing solutions that allow for patient portability (e.g., multi-payer platforms) to measure long-term outcomes. These methodological considerations will provide the foundation for aligning risk-sharing interests so that patients may access and benefit from these innovative therapies.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Luspatercept in Combination with Recombinant-Erythropoietin in MDS RS Patients: Stimulating Early and Late Erythropoiesis () - Nov 29, 2022 - Abstract #ASH2022ASH_7580;
Notably, no treatment emerging adverse events were registered with the combination.In conclusion, despite the small number of cases, these data show for the first time that combination therapy of luspatercept plus rEPO is feasible and beneficial in about one third of subjects. Concomitant stimulation of early and late-stage erythropoiesis may be an interesting strategy that requires further investigation.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
First Single-Center Experience with Luspatercept Therapy in Low-Risk Myelodysplastic Syndrome (LR-MDS) Patients with Transfusion Dependence Refractory to Erythropoietin Therapy () - Nov 29, 2022 - Abstract #ASH2022ASH_7574;
We observed better responses in patients bearing single mutation in SF3B1, patients with LTB and lower initial baseline EPO levels. The higher response rate in our follow-up may be influenced by the combination with ESA and rapid dose escalation.This work was supported by the following grants: grant AZV 2021-2024 NU21-08-00312, Grant AZV 2019-2023 NV19-08-00144, UNCE/MED/016
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Supportive Care Strategies in Myelodysplastic Syndromes and Acute Myeloid Leukemia in Older Adults: A National Survey of Canadian Hematologists () - Nov 29, 2022 - Abstract #ASH2022ASH_7228;
Among survey respondents, there was variation in the use of supportive case strategies to address bleeding and infection risk in older adults with MDS/AML. Most hematologists who treat MDS/AML with less intensive therapy felt that clinical trials are needed to define the use of TXA and prophylactic antimicrobials in this patient population.
- ||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Clinical: Very nice. My only comment is that I wouldn't consider luspatercept a magic drug. As you know, These patients tend to do relatively well since they're LR-MDS & though the TI is nice, it was no different from placebo in changing progression to HR-MDS. https://t.co/4aLxBciO6C (Twitter) - Nov 29, 2022
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Longer-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts. (Pubmed Central) - Nov 22, 2022
Most hematologists who treat MDS/AML with less intensive therapy felt that clinical trials are needed to define the use of TXA and prophylactic antimicrobials in this patient population. No abstract available
- |||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Clinical: Long-Term Efficacy and Safety of Luspatercept for Anemia Treatment in Patients With Lower-Risk Myelodysplastic Syndromes: The Phase II PACE-MDS Study | Journal of Clinical Oncology https://t.co/KxLNNEzcHE (Twitter) - Nov 21, 2022
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
P2 data, Journal: Long-Term Efficacy and Safety of Luspatercept for Anemia Treatment in Patients With Lower-Risk Myelodysplastic Syndromes: The Phase II PACE-MDS Study. (Pubmed Central) - Nov 19, 2022
Lower baseline erythropoietin levels in non-RS patients (69.6 v 623.3 IU/L; P = .0077) and higher late to early erythroid progenitor cell ratio (10.44 v 4.48; P = .0106) in RS patients were associated with HI-E. This study highlights luspatercept's effects across LR-MDS subtypes, including untreated MDS-RS, serving as a platform for future trials.
- |||| Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Clinical: Long-Term Efficacy and Safety of Luspatercept for Anemia Treatment in Patients With Lower-Risk Myelodysplastic Syndromes: The Phase II PACE-MDS Study | Journal of Clinical Oncology https://t.co/oc4tm5FmsT (Twitter) - Nov 17, 2022
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Luspatercept, a two-edged sword in beta-thalassemia-associated paravertebral extramedullary hematopoietic masses (EHMs). (Pubmed Central) - Nov 15, 2022
Considering the rarity of paravertebral EHMs in BT, luspatercept might potentially represent a novel therapeutic option for these often-serious disease-associated complications. However, appropriate follow-up investigations are recommended to detect (early) treatment failures secondary to an undesired luspatercept-associated erythroid expansion.
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal, Tumor Mutational Burden: Luspatercept (RAP-536) modulates oxidative stress without affecting mutation burden in myelodysplastic syndromes. (Pubmed Central) - Nov 15, 2022
RAP-536 did not modify variant allele frequencies in vitro and did not have any effect against leukemic burden in our PDX model. These results suggest that RAP-536 promotes in vivo and in vitro erythroid cell differentiation by decreasing ROS level without any remarkable impact on iron homeostasis and on mutated allele burden.
- | Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Journal: Limited activity of luspatercept in myelofibrosis and myeloid neoplasms other than myelodysplastic syndromes with ring sideroblasts. (Pubmed Central) - Nov 15, 2022
These results suggest that RAP-536 promotes in vivo and in vitro erythroid cell differentiation by decreasing ROS level without any remarkable impact on iron homeostasis and on mutated allele burden. No abstract available
- || Aranesp (darbepoetin alfa) / Amgen, Kyowa Kirin, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
For MDS docs - any experience w combining luspatercept and aranesp? #mdssm (Twitter) - Nov 14, 2022
- || Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
@DrAhmedAlShehri السلام عليكم عندي بنت عندها ثلاسيما عمرها ١٨ تاخذ ابر جديده Luspatercept الحين جاها ألم برجلها وش الحل معاها بس على مسكن بورفين (Twitter) - Nov 9, 2022
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
A phase 3 efficacy and safety study of luspatercept versus placebo in patients with myelofibrosis on JAK2 inhibitor therapy who require red blood cell transfusions (INDEPENDENCE) () - Nov 7, 2022 - Abstract #IMPN2022IMPN_15;
- || Review, Journal: Anemia in myelofibrosis: current and emerging treatment options. (Pubmed Central) - Nov 5, 2022
This review summarizes current and emerging treatments for anemia in MF, including luspatercept and KER-050 (transforming growth factor-β ligand traps), momelotinib and pacritinib (JAK inhibitors), pelabresib (a bromodomain extra-terminal domain inhibitor), PRM-151 (an antifibrotic agent), imetelstat (a telomerase inhibitor), and navitoclax (a BCL-2/BCL-xL inhibitor). Therapeutic combinations with ruxolitinib may offer another treatment approach.
- Tavalisse (fostamatinib) / Rigel, Kissei
A Phase I Open Label Study of Fostamatinib, a SYK Inhibitor, in Patients with Lower-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_6233;
P1
Enrollment is ongoing to determine the potential efficacy of doses of up to 200mg bid. Additional correlative studies to determine predictors of response are underway.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Characterization of Patients with Lower-Risk Myelodysplastic Syndromes Experiencing Long-Term Responses with Luspatercept in the MEDALIST Study (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_6230;
Higher rates of RBC-TI and mHI-E response led to longer durations of treatment, and patients with longer durations of treatment were more likely to receive the maximum luspatercept dose level. There were no safety signals in terms of progression to AML/HR-MDS occurring according to the expected prognostic risk profile for these patients.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Single-Cell Transcriptome Analysis Reveals Pathways That Drive Dyserythropoiesis in Myelodysplastic Syndromes, Which Are Targeted By Luspatercept (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_6203;
Conclusion Single cell RNA sequencing revealed an altered erythroid differentiation landscape in patients with low risk myelodysplastic syndromes, delineating new dyserythropoietic subpopulations, driven by the dysregulation of key molecular pathways (Figure 1). Our data indicates that Luspatercept promotes the restoration of normal erythropoiesis by downregulating key pathways beyond the TGF beta pathway.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Effect of Luspatercept on Red Blood Cell (RBC) Transfusion Burden, Iron Chelation Therapy (ICT), and Iron Overload in Adults with Transfusion-Dependent ß-Thalassemia (TDT) from the BELIEVE Trial: A Long-Term Analysis (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_5458;
Pts with TDT receiving long-term luspatercept treatment continued to experience sustained and durable reductions in RBCT burden, ICT use, and SF level over time. Luspatercept also resulted in decreases or stabilization of LIC starting at wk 96.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Erythroid Response in Patients with Non-Transfusion-Dependent ß-Thalassemia Treated with Luspatercept: Long-Term Data from the BEYOND Trial (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_5456;
P2
The duration of erythroid response increased with an additional year of luspatercept treatment. Few patients required RBC transfusions and the cumulative incidence of RBC transfusions remained low and relatively stable through 144 wk.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Real-World Data on the Use of Luspatercept in Greek Patients with Transfusion Dependent Thalassemia (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_5454;
Real world data on the use of luspatercept in TDT parallel results from the registration trial, showing heterogeneous and lasting efficacy and acceptable toxicity, despite including pts with different characteristics. Longer follow up and increased number of pts are required to validate these observations.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Investigations into the Mechanisms and Clinical Implications of Modulation of Hepcidin Levels By Luspatercept in TD MDS and TD b-Thalassemia (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_5445;
P3
Our data provides a mechanistic rationale for downregulation of hepcidin levels with luspatercept treatment. Interestingly, cell-based assay data suggest that luspatercept directly modulates hepcidin transcription suggesting that in diseased conditions with abnormally higher levels of hepcidin and TGFß family ligands, luspatercept might also modulate hepcidin transcription by sequestering TGFß family ligands implicated in hepcidin synthesis.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
TMPRSS6 Inhibition with a Monoclonal Antibody Improves Red Blood Cell Health and Reduces Hepatic Iron Loading in Mouse Models of Iron Overload Diseases (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_5440;
Our data shows, that targeting TMPRSS6 has significant advantages over current treatments, such as Luspatercept, an ACVR2b receptor trap or iron chelation...After 8 weeks treatment, liver iron from HFE-/- mice was significantly reduced in these animals. Taken together, inhibition of TMPRSS6 is an advantageous strategy in different iron overload diseases.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Effect of Luspatercept in ß-Thalassemia Patients with ß0/ß0 Genotype: A Subgroup Analysis of the BELIEVE Study (ENMCC - 220-222) - Nov 4, 2022 - Abstract #ASH2022ASH_5183;
Furthermore, greater proportions of splenectomized pts with a ß0/ß0 genotype achieved reductions in RBC transfusion burden than ß0/ß0 pts who were not splenectomized. These longer-term data demonstrate the efficacy of luspatercept in a pt population with more severe disease.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Combination of a TGF-ß Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting ß-Thalassemia (ENMCC - 220-222) - Nov 4, 2022 - Abstract #ASH2022ASH_5182;
Luspatercept, a Transforming Growth Factor (TGF)-ß ligand-trap that is modeled after the extracellular domain of activin receptor (ACVR)-2B, gained FDA approval in 2019 to treat transfusion dependent BT patients (Cappellini and Taher Blood Adv, 2021)...Their combination maintains these positive endpoints, but also is also superior in improving RBC and Hb synthesis, with a significant improvement in the anemia. This study provides the first pre-clinical support for combining these two drugs to improve anemia as well as iron metabolism in BT patients.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
112. Thalassemia and Globin Gene Regulation I (ENMCC - 220-222) - Nov 4, 2022 - Abstract #ASH2022ASH_5177;
In this session, the first 4 presenters in this session describe the mechanism by which HIC2 expression is controlled during the developmental switch from fetal-to-adult hemoglobin production, locate the specific contacts driving the direct binding of ETO2 to NuRD, identify the obligate partner of BCL11A in silencing hemoglobin F and examined the role of individual Baf chromatin remodeling complex components in globin gene regulation. The two last abstracts summarize a subanalysis of Believe study evaluating the therapeutic effectiveness of luspatercept in patients with ß0/ß0 thalassemia and the effect of splenectomy on response to luspatercept and a combination therapy is explored in lab to increase response to luspatercept in beta thalassemia.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Multiple Episodes of Transfusion Independence with Luspatercept Treatment and the Impact of Dose Escalation in Patients with Lower-Risk Myelodysplastic Syndromes from the MEDALIST Study (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_4471;
LTB patients were more likely to respond to lower doses of luspatercept, whereas almost half of ITB and HTB patients required escalation to the maximum dose level to respond. ITB and HTB patients might be expected to wait longer for a second response to luspatercept than LTB patients.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Efficacy and Safety of Luspatercept in Adult Patients with Transfusion-Dependent Anemia Due to Very Low, Low and Intermediate Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts, Who Had an Unsatisfactory Response to or Are Ineligible for Erythropoietin-Based Therapy: A Retrospective Multicenter Study By Fondazione Italiana Sindromi Mielodisplastiche (FiSiM ETS) (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_4461;
The benefit extended beyond the achievement of TI and produced a significant reduction in the number of RBC transfusions. Higher baseline transfusion burden was associated with a reduced probability to achieve TI; in these patients, the reduction of transfused RBC units appears a more reliable treatment target.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Synergistic Activity of Luspatercept and Erythroid Stimulating Agents Combination for Treatment of Anemia in Lower-Risk Myelodysplastic Syndromes (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_4449;
Conclusions To our knowledge, this is the first proof of principle data confirming synergistic clinical activity combining luspatercept and ESA. Predictors of response included prior response to luspatercept monotherapy/frontline combination compared to primary luspatercept failure, endogenous serum epo levels < 500, SF3B1 mutation, and being HMA/Len treatment naïve.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
A Closer Look at Changes in Hemoglobin Levels in Patients with Non-Transfusion Dependent ß-Thalassemia Treated with Luspatercept: Post Hoc Analysis of the Phase 2 BEYOND Trial (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_3757;
P2
Clinically relevant Hb increases of = 1.0 g/dL were observed across all baseline Hb levels. These findings further support the ability of luspatercept to achieve clinically meaningful increases in Hb for the majority of patients with NTDT, irrespective of baseline severity of anemia.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Overall Survival and Progression-Free Survival of Patients Following Luspatercept Treatment in the MEDALIST Trial (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_2635;
Luspatercept was associated with increased 36-mo OS probability for pts with IPSS-R Very low-risk MDS and 36-mo PFS probability in pts with a BL serum EPO level of 100 to = 200 U/L. Therefore, pts with LR-MDS with these BL characteristics may derive greater survival benefit from luspatercept.
- AG-946 / Agios Pharma
A Phase 2a/2b Multicenter Study of AG-946 in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_2634;
Before receiving the first dose of AG-946, EPO or G-CSF must have been stopped for =28 days and luspatercept for =65 days. This ph2a/2b global study aims to provide proof-of-concept and evaluate efficacy and safety of AG-946, an investigational, once-daily, activator of PKR, vs PBO in adult pts with anemia due to LR-MDS.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Luspatercept for Treatment of Lower Risk Myelodysplastic Syndromes: Real World Data Replicates Medalist Study Results and Confirms Activity Among Hypomethylating Agents and Lenalidomide Treated Patients (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_2618;
Low baseline RBC-TB dependency and SF3B1 MT correlated with higher response rates. Luspatercept retained activity after HMA or lenalidomide failure however a trend of lower responses observed correlated with RBC-HTB among those patients.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Real-World Outcomes of Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Treated with Luspatercept: An Evaluation of US Clinical Practice Utilization and Treatment Patterns (ENMCC - 220-222) - Nov 4, 2022 - Abstract #ASH2022ASH_1813;
All pts with moderate TB prior to luspatercept had a decrease in level of TB within the first 24 weeks of starting luspatercept treatment. The MEDALIST trial showed 38% of pts treated with luspatercept- with IPSS-R Very Low to Intermediate risk reached TI within 24 weeks (Fenaux P, et al.
- Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Metabolic Dysfunctions Driven By PRPF8 Explain Cases with Ring Sideroblasts without SF3B1 Mutations: Similarity/ Differences of Genotype/ Phenotype Association (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_1148;
In analogy, given the proximal genomic location of PRPF8 and TP53 we do not exclude that this commonality might be involved in TP53MT with RS as well. These likely phenocopying mechanisms may enable therapeutic opportunities (luspatercept) in cases lacking SF3B1MT presenting with RS and transfusion-dependent anemia.