Epogen (epoetin alfa) / Amgen - LARVOL DELTA

Luspatercept for the treatment of myelodysplastic syndrome: A retrospective cohort study utilizing trinetx (ASH 2025) - "Secondary outcomes included overall mortality, transition to hypomethylating agent therapy (decitabine or azacitidine), and the need for allogeneic stem cell transplant...The COMMANDS trial further confirmed the superiority of luspatercept over epoetin alfa in ESA-naïve, transfusion-dependent lower-risk MDS, with a higher rate of RBC-TI ≥12 weeks and a concurrent mean hemoglobin increase ≥1.5 g/dL during weeks 1–24 (60% vs. 35%, p<0.0001)...Conclusion Our findings suggest that luspatercept can provide meaningful and durable benefits for a substantial subset of patients and may serve as an important component of therapy in appropriately selected individuals with very low to intermediate-risk MDS. Future studies are necessary to evaluate which patients would most benefit from treatment with luspatercept."

Retrospective data • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • SF3B1 • TGFB1

Predictive biomarkers of luspatercept and erythropoiesis-stimulating agent (ESA) hematological response and overall survival (OS) in patients with lower-risk myelodysplastic syndromes (LR-MDS) in the commands trial (ASH 2025) - P3 | "In thephase 3 COMMANDS trial (NCT03682536), luspatercept demonstrated superiority for the primaryendpoint (PE) of transfusion independence (TI) vs ESA epoetin alfa, including a trend for improved OS. Key baseline variables including IPSS-M, number of mutations, mutational burden, soluble proteins, andpatient characteristics were balanced between tx arms (luspatercept, n=182; ESA, n=181). Luspaterceptdemonstrated superiority vs ESA for RBC-TI (PE; 60% vs 35%) with a trend for improved OS (median, notestimable vs 46 mo).Univariate analysis identified significant explanatory variables across both tx arms (P<0.05) for the PE(n=35) and OS (n=40). For the luspatercept arm, significant PE predictors were lower IPSS-M anderythropoietin (EPO) levels, and higher alpha-2 macroglobulin (P<0.001)."

Biomarker • Clinical • Tumor mutational burden • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • A2M • ASXL1 • GDF15 • MB • SF3B1 • TMB

Efficacy of erythropoietin and intravenous iron for treatment of critical anemia in patients who decline blood transfusions for religious reasons (ASH 2025) - "Previous studies have focused on preoperative optimization in this patient population, but dataon emergent management remain limited.Objective:Evaluate clinical outcomes following administration of epoetin alfa (EPO) and intravenous (IV) iron inpatients with critical anemia who decline blood transfusion for religious reasons.We performed a retrospective single-arm observational study using data from Montefiore MedicalCenter's electronic health records from 2016 to 2025...This study represents the first evaluation of EPO and IV iron treatment outcomes in an emergent settingfor patients who decline blood transfusions. Our data demonstrate that this approach is a feasiblealternative to transfusion, as evidenced by a 74% hemoglobin recovery rate with a median increase in Hbof 4.4 g/dL. However, approximately 1 in 4 patients did not recover, and overall mortality remainedsignificant at 14%, underscoring EPO and IV iron therapy as a second-line option after..."

Clinical • Anemia • Chronic Kidney Disease

Clinical benefit of luspatercept in erythropoiesis-stimulating agent (ESA)-naive patients (pts) with early disease characteristics and very low-, low-, or intermediate-risk Myelodysplastic Syndromes (LR-MDS): A post hoc analysis from the commands trial (ASH 2025) - P3 | "Background :In the phase 3 COMMANDS trial (NCT03682536) evaluating pts with ESA-naive transfusion-dependent(TD) LR-MDS, luspatercept was superior to epoetin alfa (EA) in achieving red blood cell-transfusionindependence (RBC-TI) ≥12 weeks (wks) and demonstrated a more durable clinical benefit. Pts with less severe disease characteristics, reflective of an earlier disease stage, derive greater clinicalbenefit from treatment than those with more advanced characteristics. Pts on luspatercept vs EAdemonstrated higher response rates and longer duration of response regardless of disease stage, furthersupporting its role as a preferred first-line therapy in LR-MDS."

Clinical • Retrospective data • Hematological Malignancies • Myelodysplastic Syndrome

Real-world comparison of thrombotic events in patients with Myelodysplastic Syndromes treated with luspatercept versus erythropoietin-stimulating agents using trinetx database (ASH 2025) - "Patients were stratified based on treatment with luspatercept or ESAs(epoetin alfa or darbepoetin alfa). In this large, multicenter real-world analysis, we observed a numerically lower incidence of thromboticevents with luspatercept compared to erythropoietin-based agents in patients with MDS, thoughdifferences did not reach statistical significance. These findings support the thrombotic safety profile ofluspatercept observed in clinical trials and warrant further prospective validation."

Clinical • Real-world • Real-world evidence • Anemia • Beta-Thalassemia • Cardiovascular • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Respiratory Diseases • Venous Thromboembolism

Durable Transfusion Independence in Lower-Risk Myelodysplastic Syndrome (LR-MDS) Is Associated with Better Survival: A Population Level Analysis Based on a Large US Health Insurance Claims Database (ASH 2023) - "Lines of treatment were determined based on claims for MDS treatments contained in the database: erythropoietin stimulating agents (ESAs; darbepoetin, epoetin alfa), hypomethylating agents (HMAs; azacitidine, decitabine, decitabine-cedazuridine), lenalidomide, luspatercept, eltrombopag, and cyclosporine. Among patients with LR-MDS, 35% and 49% were RBC-TD before 1L and 2L of therapy, respectively. Achievement of TI was associated with improved survival, suggesting that RBC-TD is a modifiable predictor of clinical outcomes in LR-MDS. Despite the currently available therapies, RBC-TD after any line of therapy is associated with poorer outcomes."

Claims database • Clinical • Reimbursement • US reimbursement • Acute Myelogenous Leukemia • Anemia • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Myelodysplastic Syndrome • Oncology

EVEREST: EVR and EPO for Liver Transplant Tolerance (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | Initiation date: Sep 2025 ➔ Dec 2025 | Not yet recruiting ➔ Recruiting

Enrollment open • Trial initiation date • Transplantation

Treatment of an Anemic Patient with CKD with Oral Vadadustat (KIDNEY WEEK 2025) - "Patient's extra-renal systemic lupus has stablized with use of hydroxychloroquine and low dose mycophenolate...In January of 2025 she was swithced from epogen to vadadustat 300 mg oral daily...This case demonstrates that some patients may respond quicker, reducing requirement of frequent high dose of synthetic erythropoietin. Anemia Management Report"

Clinical • Anemia • Chronic Kidney Disease • Glomerulonephritis • Hematological Disorders • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Renal Disease • Systemic Lupus Erythematosus • STAT3

Longitudinal Safety of Luspatercept in the Treatment of Anemia in Patients with Myelofibrosis: Results from the ACE-536-MF-001 Study (ASH 2023) - P2 | "Methods Pts were enrolled into 4 cohorts based on transfusion dependence (TD) and stable ruxolitinib (RUX) treatment – cohort 1: NTD, no RUX; cohort 2: TD, no RUX; cohort 3A: NTD, RUX; cohort 3B: TD, RUX...Results In the safety population (N = 95; cohort 1, n = 22; cohort 2, n = 14; cohort 3A, n = 21; cohort 3B, n = 38), the most frequently used prior medications for anemia treatment were erythropoiesis-stimulating agents (23.2%, most frequently epoetin alfa [14.7%]), followed by danazol (11.6%), lenalidomide (2.1%), and thalidomide (1.1%)...In addition to benefits in anemia, the safety profile of LUSPA allowed most pts receiving RUX to maintain or increase their RUX dose, supporting maintenance of Janus kinase inhibitor therapy for adequate disease control of MF. Study support: Bristol Myers Squibb."

Clinical • Anemia • Beta-Thalassemia • Cardiovascular • Cerebral Hemorrhage • CNS Disorders • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Hypertension • Infectious Disease • Inflammation • Ischemic stroke • Musculoskeletal Pain • Myelodysplastic Syndrome • Myelofibrosis • Oncology • Pain • Pneumonia • Renal Disease • Respiratory Diseases • Septic Shock

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Lower-Risk Myelodysplastic Syndromes Who Have Been Treated with Luspatercept (ASH 2023) - "The two most used ESAs were darbepoetin alfa (54. 5%) and epoetin alfa (44...7% of patients had previously received a non-ESA treatment, most commonly azacitidine (62. 9%) or lenalidomide (31... This real-world study described treatment patterns and outcomes in patients with LR-MDS receiving luspatercept. An improvement in hemoglobin levels was observed in more than half of patients. In a quarter of patients who had a ≥1."

Clinical • Real-world • Real-world evidence • Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

The ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes (ASH 2023) - P3 | "The study is registered at ClinicalTrials. gov (NCT05949684) and EudraCT (2022-500430-29-00)."

Clinical • P3 data • Acute Myelogenous Leukemia • Anemia • Cardiovascular • Fatigue • Hematological Disorders • Hematological Malignancies • Hypertension • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology

Patient-Reported Outcomes (PRO) of Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent (ESA)-Naïve, Transfusion-Dependent (TD), Lower-Risk Myelodysplastic Syndromes (LR-MDS): Results from the Phase 3 COMMANDS Study (ASH 2023) - "The findings from these interim PRO analyses indicate that treatment with luspatercept increased sustained improvement across quality-of-life domains when compared to epoetin alfa. Luspatercept was also found to be well-tolerated by the majority of patients. All of these PRO findings further support the benefits of luspatercept in ESA-naïve and TD patients with LR-MDS."

Clinical • P3 data • Patient reported outcomes • Anemia • Anorexia • CNS Disorders • Constipation • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Insomnia • Myelodysplastic Syndrome • Oncology • Pulmonary Disease • Sleep Disorder

A Phase 1b/2 Study of the Efficacy and Safety of Combination Hydroxyurea and Dose-Escalated Erythropoietin for Treatment of Anemia in Sickle Cell Disease (ACHiEvE-SCD) (ASH 2024) - P1/2 | "Here, we present the preliminary results of a Phase 1b/2 multicenter, open-label, dose escalation study of epoetin alfa (EPO) for the treatment of anemia in patients (pts) with SCD on HU...Inclusion criteria included age ≥ 18 years, diagnosis of SCD (HbSS or HbS/β0-thalassemia), screening Hb ≤ 9.0 g/dL, on stable-dose HU, and no recent transfusions, voxelotor or ESA treatment...Nearly all participants experienced a Hb response with a relatively low-to-moderate dose of EPO. Subsequent analyses will evaluate the effect of EPO therapy on transfusion burden and other clinical outcomes."

Clinical • P1/2 data • Anemia • Cardiovascular • Chronic Kidney Disease • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Hypertension • Myelodysplastic Syndrome • Nephrology • Oncology • Renal Disease • Sickle Cell Disease • Thrombosis

Luspatercept Modulates Inflammation in the Bone Marrow, Restores Effective Erythropoiesis/Hematopoiesis, and Provides Sustained Clinical Benefit Versus Epoetin Alfa (EA): Biomarker Analysis from the Phase 3 COMMANDS Study (ASH 2023) - P3 | "Multiplatform analysis of primary BM samples from a phase 3 trial of luspatercept revealed novel response-associated biomarkers. Compared with EA, luspatercept led to sustained increase of EPs and retics over 48 wk with a concomitant Hb increase. RS− pts showed Hb accumulation peaked at wk 24 in the EA arm."

Biomarker • Clinical • P3 data • Anemia • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hematological Malignancies • Inflammation • Myelodysplastic Syndrome • Oncology • IFNA1 • IL10 • IL1R1 • IL6 • TGFB1

Efficacy and Safety of Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent (ESA)-Naive Patients (Pts) with Transfusion-Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS): Full Analysis of the COMMANDS Trial (ASH 2023) - P3 | "Results of this full analysis confirm the findings from the interim analysis; RBC-TI duration and erythroid responses achieved with luspatercept are superior compared with epoetin alfa. Luspatercept safety results were consistent with previous MDS studies. These data show that luspatercept could represent a new standard of care for pts with TD LR-MDS."

Clinical • Acute Myelogenous Leukemia • Anemia • Cardiovascular • Fatigue • Hematological Disorders • Hematological Malignancies • Hypertension • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Novel Coronavirus Disease • Oncology • SF3B1

Transfusion-Dependent Angiodysplasia-Related Anemia in a Patient with ESRD Treated with Thalidomide (KIDNEY WEEK 2025) - "Despite optimal dose of epogen analogues, iron infusions and octreotide infusions he needed weekly blood transfusions. This unique case with florid GI AVM, pancytopenia required a tailored approach of yearlong thalidomide. Patient did not have side effects like somnolence or constipation despite the duration."

Clinical • Anemia • Cardiovascular • Chronic Kidney Disease • CNS Disorders • Constipation • Diabetes • Diabetic Nephropathy • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hypertension • Metabolic Disorders • Nephrology • Peripheral Arterial Disease • Renal Disease

Model-Based Vadadustat Starting Dose Recommendations in Patients with Dialysis-Dependent CKD (KIDNEY WEEK 2025) - "Simulations were performed in virtual dialysis-dependent patients with bl characteristics of interest (ESA experienced/naïve, bl Hb ≥ or <10 g/dL, and bl standardized ESA dose ≥ or < 90 IU/kg/wk Epogen equivalents). Patients with higher bl Hb or lower prior ESA dose were more likely to achieve satisfactory Hb response at a lower VADA starting dose. Conclusion A VADA starting dose of 450 mg QD or 900 mg TIW should ameliorate the transitory decline in Hb following a switch from ESA in most patients."

Clinical • Late-breaking abstract • Anemia • Chronic Kidney Disease • Hematological Disorders

Vadadustat Outcomes In-Center Experience (VOICE) Study: Pragmatic Randomized Controlled Trial to Test the Safety of Three Times Weekly Vadadustat (KIDNEY WEEK 2025) - P3 | "Methods This randomized controlled trial is testing whether TIW vadadustat is non-inferior to epoetin alfa on the primary hierarchical outcome of all-cause mortality and all-cause hospitalization analyzed using the win ratio ( NCT06520826) . Conclusion The VOICE trial design showcases a pragmatic approach to embed individual-level randomized clinical trials in routine dialysis care. The trial will generate critical data on the safety of a new anemia management strategy while leveraging a composite hierarchical outcome to optimize statistical efficiency and clinical relevance."

Clinical • Anemia • Hematological Disorders

Efficacy and Cardiovascular Safety of Pegmolesatide in Renal Anemia: Post Hoc Analysis of Two Phase 3 Trials (KIDNEY WEEK 2025) - P3 | "The incidences of 5P MACE (DD-CKD: 2.0% vs. 5.6%, HR 0.35, 95%CI 0.11-1.10; NDD-CKD: 0.9% vs. 6.9%, HR 0.14, 95%CI 0.02-1.28; Table; Figure) and expanded CV events (DD-CKD: 6.9% vs. 10.5%, HR 0.64, 95%CI 0.31-1.32; NDD-CKD: 5.2% vs. 10.3%, HR 0.57, 95%CI 0.18-1.75) were consistently lower in the pegmolesatide group across both patient populations. Conclusion Pegmolesatide demonstrates effectiveness in Hb management for both NDD-CKD and DD-CKD populations, with potential benefits in reducing the risk of CV events compared to epoetin alfa."

Clinical • P3 data • Retrospective data • Anemia • Cardiovascular • Chronic Kidney Disease • Congestive Heart Failure • Heart Failure • Hematological Disorders • Myocardial Infarction • Nephrology • Renal Disease

Navigating Therapeutic Challenges: Parvovirus-Induced Pure Red Cell Aplasia in a Kidney Transplant (KT) Recipient Who Declines Blood Transfusions (KIDNEY WEEK 2025) - "Mycophenolate Mofetil (MMF) was stopped, and the due dose of Belatacept was held. She received IVIG 2 g/kg in 2 doses, IV Iron Dextran 1,000 mg, and Epoetin Alfa 40,000 units daily for 2 days, then 10,000 units daily...Compassionate use of hemoglobin-based oxygen carriers should be considered in cases of critical anemia (Hb<4 g/dL). In the absence of antivirals, reducing immunosuppression and providing passive immunity with IVIG can help as seen in this case by a dramatic increase in the R count."

Acute Kidney Injury • Anemia • Antibody-mediated Rejection • Cardiovascular • Chronic Kidney Disease • Gastroenterology • Hematological Disorders • Hypotension • Infectious Disease • Myocardial Ischemia • Nephrology • Renal Disease • Transplantation

Health-Related Quality of Life of Luspatercept Versus Epoetin Alfa in Red Blood Cell Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes: Results from the Final Datacut of the Phase 3 COMMANDS Study (ASH 2024) - P3 | "Conclusions : The analyses indicate superior treatment effects of luspatercept over epoetin alfa on shortening time to achieving a meaningful HRQoL improvement or delaying time to experiencing a meaningful HRQoL deterioration or RBC transfusion. These findings provide further evidence to support the use of luspatercept as a front-line treatment for patients with RBC transfusion-dependent LR-MDS."

Clinical • HEOR • P3 data • Anemia • Fatigue • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Long-Term Response Analysis of Transfusion Independence in Erythropoiesis Stimulating Agent–Naive Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes Treated with Luspatercept Vs Epoetin Alfa in the COMMANDS Trial (ASH 2024) - P3 | "Progression rates to high-risk MDS and AML were low in both treatment arms. These data support luspatercept as the treatment of choice in TD ESA-naive pts with lower-risk MDS."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • SF3B1

Changes in Red Blood Cell Transfusion Burden with Luspatercept Versus Epoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes in the Phase 3, Open-Label, Randomized, Controlled COMMANDS Trial (ASH 2024) - P3 | "Conclusions : The findings from the COMMANDS trial demonstrated that luspatercept treatment led to a decrease in RBCT burden compared with epoetin alfa in ESA-naive pts with LR-MDS, evident by the number of RBC units transfused and RBCT visits over a 1.5-year period. When analyzed by RBCT burden category, transfusion burden was also substantially lower for luspatercept compared with epoetin alfa."

Clinical • P3 data • Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Overallsurvival and duration of transfusion independence for first-line ESA-naive patients with lower-risk Myelodysplastic Syndromes treated with Luspatercept versus Epoetin alfa in the COMMANDS Trial (DGHO 2025) - P3 | "Luspatercept treatment resulted in a clinically meaningful and statistically significant longer duration of response compared with epoetin alfa. There was a positive OS trend compared with epoetin alfa, and no new safety concerns."

Clinical • Hematological Malignancies • Myelodysplastic Syndrome

Clinical Benefits of Achieving Hemoglobin (Hb) Levels ≥ 10 g/dL in Transfusion-Dependent (TD) Erythropoiesis-Stimulating Agent (ESA)-Naive Patients (Pts) with Lower-Risk (LR) Myelodysplastic Syndromes (MDS) Treated with Luspatercept in the COMMANDS Trial (ASH 2024) - P3 | "Luspatercept demonstrated superior response vs epoetin alfa in RBC TI with concurrent mean Hb increase ≥1.5 g/dL in weeks 1-24 (60.4% vs 34.8%, P < 0.0001; Della Porta MG, et al. Luspatercept dose escalation maintained target Hb levels without significantly increasing incidence or severity of TEAEs. Pts with lower Hb levels at baseline showed a trend requiring higher dose, supporting the clinical benefit of luspatercept dose escalation in pts with LR-MDS."

Clinical • Hematological Malignancies • Myelodysplastic Syndrome • Oncology