Evrenzo (roxadustat) / Astellas, AstraZeneca, FibroGen - LARVOL DELTA

The efficacy and safety of luspatercept in patients with low-blasts myelodysplastic neoplasms in real world (ASH 2025) - "The ESA group were received rhEPO ± roxadustat treatments. It need to optimize the treatment of luspatercept in further. Key words : MDS-LB, luspatercept, EPO refractory, anemia, immune state"

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • CRP • IFNG • IL2 • IL6 • SF3B1 • TNFA

Hypoxia-driven complement activation expands circulating endothelial cells in transplant-associated thrombotic microangiopathy: A multi-omics single-cell and functional study (ASH 2025) - "Both siHIF1A and eculizumab halved C3b loading and restored barrier integrity; roxadustat (HIF-PHD inhibitor) phenocopied hypoxia-induced complement activation. Conclusions Hypoxia activates complement and fuels CEC expansion in TA-TMA, linking two key pathogenic drivers.CEC percentage and a hypoxia transcriptomic score emerge as non-invasive early biomarkers, whilecombined hypoxia- and complement-targeted interventions warrant clinical testing to improve allograftoutcomes."

Transplantation • CD31 • HIF1A • KDR • MCAM • PECAM1 • PTPRC

FDA approval of imetelstat: a new era in the treatment of lower-risk myelodysplastic syndrome. (PubMed, Ann Med Surg (Lond)) - "Previously available treatments-including erythropoiesis-stimulating agents, lenalidomide, luspatercept, roxadustat, and azacitidine-have demonstrated variable efficacy and tolerability, with limited impact on long-term disease progression. Its approval underscores the importance of targeting fundamental disease mechanisms rather than merely alleviating symptoms. As additional data emerge from real-world settings and ongoing trials, Imetelstat may further reshape therapeutic algorithms and improve both quality of life and survival outcomes in this patient population."

FDA event • Journal • Review • CNS Disorders • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Psychiatry

Effect of Roxadustat on Heart Failure Patients With Anaemia and Moderate-to-Severe Chronic Kidney Disease (clinicaltrials.gov) - P=N/A | N=200 | Not yet recruiting | Sponsor: Zhongda Hospital

New trial • Anemia • Cardiovascular • Chronic Kidney Disease • Congestive Heart Failure • Heart Failure • Hematological Disorders • Nephrology • Renal Disease

The effect of SGLT2 inhibitor and HIF-PHI on the podocyte-specific molecules and cytoskeleton of diabetic podocytes. (PubMed, BMC Nephrol) - "SGLT2i Dapagliflozin and HIF-PHI Roxadustat partly restore the podocyte morphology and intracellular mRNA and protein levels of podocyte-associated molecule in a diabetic milieu."

Journal • Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Hematological Disorders • Metabolic Disorders • Nephrology • Renal Disease • NPHS1 • PODXL

Study of Roxadustat for Reducing the Incidence of Acute Kidney Injury After Coronary Artery Bypass Grafting (clinicaltrials.gov) - P2 | N=98 | Terminated | Sponsor: Peking Union Medical College Hospital | N=318 ➔ 98 | Trial completion date: Oct 2023 ➔ May 2025 | Recruiting ➔ Terminated | Trial primary completion date: Aug 2023 ➔ May 2025; slow enrollment and inability to reach the planned sample size within the expected timeframe

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Acute Kidney Injury • Nephrology

Comparative Effectiveness and Safety of Roxadustat versus Erythropoiesis-Stimulating Agents in Patients Receiving Maintenance Hemodialysis: A Real-World Cohort Study. (PubMed, Kidney Dis (Basel)) - "Roxadustat demonstrated superior efficacy in elevating and sustaining Hb levels compared to ESA over a longer observation period, with comparable cardiovascular safety in hemodialysis-dependent CKD patients. Roxadustat represents a viable alternative to ESA for renal anemia management, though long-term multicenter studies are needed to validate safety and optimize clinical use."

HEOR • Journal • Real-world evidence • Cardiovascular • Chronic Kidney Disease • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hypertension • Nephrology • Renal Disease

Lactic acid promotes an MDSC-like phenotype via HIF1α stabilization with impact on prognosis in renal cell carcinoma. (PubMed, Cancer Lett) - "In line, the HIF1α-stabilizing drug Roxadustat increased the number of CD14+ HLA-DR low cells...Our data dissect the intratumoral network of RCC and show that tumor-derived lactate promotes a pro-tumorigenic myeloid phenotype with low MHC-II but high immune-checkpoint, LOX-1 and S100A8/9 expression. Blocking MCT disrupts this interplay, offering a promising strategy to re-educate tumor-associated myeloid cells and enhance tumor immune surveillance."

Journal • Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • CD14 • HIF1A • IL6 • S100A8

Procyanidin displayed a synergistic effect with roxadustat on renal anemia in mice. (PubMed, Front Pharmacol) - "These findings suggest that procyanidin may enhance the safety and efficacy of roxadustat in CKD-related renal anemia. Further studies are needed to optimize treatment regimens for clinical application."

Journal • Preclinical • Anemia • Cardiovascular • Chronic Kidney Disease • Fibrosis • Hematological Disorders • Immunology • Inflammation • Nephrology • Renal Disease • Thrombosis

Concerns from bench and insights from bedside: the puzzle of Roxadustat in cancer patients with chemotherapy-induced anemia. (PubMed, Front Pharmacol) - "Consequently, Roxadustat poses a clinical dilemma: its efficacy in CIA offers a promising ESAs alternative, but its HIF-driven oncogenic potential necessitates long-term safety assessment in cancer patients. Future studies must prioritize longitudinal monitoring to define the benefit-risk profile."

Journal • Review • Anemia • Chemotherapy-Induced Anemia • Hematological Disorders • Oncology • Solid Tumor • HIF1A

A Study of Roxadustat to Treat Anemia in Children and Teenagers With Chronic Kidney Disease (clinicaltrials.gov) - P3 | N=100 | Recruiting | Sponsor: Astellas Pharma Global Development, Inc. | Trial completion date: Sep 2026 ➔ Oct 2027 | Trial primary completion date: Nov 2025 ➔ Oct 2027

Trial completion date • Trial primary completion date • Anemia • Chronic Kidney Disease • Hematological Disorders • Nephrology • Pediatrics • Renal Disease

Comparative effectiveness of darbepoetin vs other agents in chronic kidney disease-related anemia: a systematic review and network meta-analysis. (PubMed, BMC Nephrol) - "Daprodustat and roxadustat indicated equivalent or higher efficacy when compared to darbepoetin in elevating hemoglobin levels in CKD-related anemia, with daprodustat demonstrating a good safety profile. While conventional ESAs remain beneficial, HIF-PHIs offer promising oral alternatives with possible benefits in cardiovascular safety and decreased hypertension risk. Further head-to-head trials are necessary to confirm these findings and guide individualized treatment strategies."

Clinical • HEOR • Journal • Retrospective data • Review • Anemia • Cardiovascular • Chronic Kidney Disease • Diabetes • Hematological Disorders • Hypertension • Metabolic Disorders • Nephrology • Renal Disease

HIF-1α-Dependent Autophagy Deficiency in Bone Marrow Endothelial Progenitor Cells Contributes to Acute Graft-Versus-Host Disease after Allotransplant (ASH 2023) - "However, intervention with HIF-1α activators Roxadustat, which served to upregulate HIF-1α levels in ECs, activated autophagy, and improved cellular function, also enhanced the hematopoiesis-supporting ability... The results revealed that the autophagy deficiency was mediated by reduced expression of HIF-1α in EPCs of patients with aGVHD. The deficiency of autophagy also led to abnormal activation of SIRT1 and PARP1, which in turn affected the levels of NAD+ and mitochondrial membrane potential, ultimately impairing cellular function and hematopoiesis-supporting ability. These molecular changes may be identified as potential contributors to the pathogenesis of aGVHD after allo-HSCT."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Immunology • Metabolic Disorders • Transplantation • CD133 • CD34 • HIF1A • KDR • PARP1 • SIRT1

The Efficacy of Roxadustat in Multiple Myeloma Patients with Renal Insufficiency and Anemia: A Real-World Study (ASH 2023) - "The choice anti-tumor therapy was commonly consisted of combination therapy with a bortezomib, cyclophosphamide/thalidomide and dexamethasone or an equivalent regimen. This is the first study exploring the efficacy of Roxadustat in multiple myeloma patients with renal insufficiency. According to this study, Roxadustat is well tolerated by MM patients with renal insufficiency and effectively improves their anemia conditions. Metabolic acidosis and hyperkalemia occurred more frequently in the Roxadustat group than in control group."

Clinical • Real-world • Real-world evidence • Anemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Metabolic Disorders • Multiple Myeloma • Nephrology • Oncology • Renal Disease • Thrombosis

Efficacy and Safety of Roxadustat for Treatment of Anemia in Patients with Lower-Risk Myelodysplastic Syndrome (LR-MDS) with Low Red Blood Cell (RBC) Transfusion Burden: Results of Phase III Matterhorn Study (ASH 2023) - P3 | "Despite not meeting the primary endpoint, roxadustat plus BSC was well-tolerated, and a high percentage of pts with LR-MDS and LTB were TI responders. The high TI response rate in the PBO arm, historically poor outcomes in pts with ESA-refractory disease, and the inclusion of pts who were not transfusion-dependent (1 pRBC unit Q8W) may have contributed to the lack of a statistically significant difference in TI response rates between arms. MATTERHORN outcomes highlight the continued unmet need for effective and safe therapies that reduce RBC transfusion burden in LR-MDS."

Clinical • P3 data • Acute Myelogenous Leukemia • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Hematological Malignancies • Infectious Disease • Ischemic stroke • Leukemia • Myelodysplastic Syndrome • Myocardial Infarction • Nephrology • Oncology • Pneumonia • Renal Disease • Respiratory Diseases

Direct Bilirubin Affects Roxadustat Efficacy for Anemia in Patients on Dialysis (KIDNEY WEEK 2025) - "At the 12-week mark, direct bilirubin, but not neutrophil counts, significantly impacted roxadustat efficacy. Patients with a favorable initial response demonstrated higher hemoglobin change levels during follow-up, irrespective of the roxadustat dosage, compared to those with a poor initial response."

Clinical • Anemia • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Erythropoietin-Mimetic Peptide Pegmolesatide Therapy for Pure Red Cell Aplasia in a Patient with Nondialysis-Dependent Type 1 Diabetic Nephropathy: A Case Report (KIDNEY WEEK 2025) - "Enarodustat and roxadustat were administered successively after discontinuation of EPO, but anemia did not improve, and the patient was maintained with weekly blood transfusions...Discussion The new EPO-mimetic peptide pegmolesatide, which consists of 44 amino acids and has a completely different peptide chain structure from peginesatide and EPO (Fig 4), was independently developed in China...Thus, there is no cross-immune response between pegmolesatide and EPO. In addition, pegmolesatide rarely produces drug antibodies, attributed to the low immunogenicity.Based on these findings, we speculate that pegmolesatide can provide a safe, effective, and convenient therapeutic strategy for PRCA in Chinese patients with chronic kidney disease."

Case report • Clinical • Anemia • Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Hematological Disorders • Nephrology • Renal Disease • Type 1 Diabetes Mellitus

A Rare Case of VEXAS (Vacuoles, E1 Enzyme, X-Linked, Autoinflammatory, Somatic)-Relapsing Polychondritis (RP) Syndrome with AKI (KIDNEY WEEK 2025) - "The patient was diagnosed with VEXAS-RP-TIN, and received glucocorticoids and cyclophosphamide. Roxadustat was prescribed for anemia...Discussion For AKI patients with fever, hyper-inflammatory status, and refractory hematological abnormalities, bone marrow studies and gene tests should be employed to estimate VEXAS syndrome. Bone marrow smear showed vacuoles in myeloid precursor cells Kidney biopsy showed infiltration of lymphocytes and monocytes in renal interstitium（HE stain, ×200）"

Clinical • Anemia • Cardiovascular • Diabetes • Hematological Disorders • Hypertension • Infectious Disease • Metabolic Disorders • Musculoskeletal Pain • Renal Disease • CRP

Improvement of Blood Pressure, Anemia, and CKD-MBD Management in Patients on Peritoneal Dialysis: Insights from the Smart Peritoneal Dialysis Care Program (SPDC) at a Single Center (KIDNEY WEEK 2025) - "Hemoglobin (Hb ≥110 g/L) improved from 38% to 53% ( P < 0.05), linked to expanded roxadustat use and reduced erythropoiesis-stimulating agent reliance...Conclusion The SPDC program improved, anemia management, and metabolic outcomes in PD patients through structured remote monitoring and multidisciplinary care. Its success highlights the potential of digital health integration to enhance home-based dialysis management, particularly in resource-limited settings."

Clinical • Anemia • Chronic Kidney Disease • Hematological Disorders

Effect of SGLT2 Inhibitors and Hypoxia-Inducible Factor-Prolyl Hydroxylase Inhibitors (HIF-PHIs) on Gene Expression and Metabolomic Profile of Podocytes (KIDNEY WEEK 2025) - "Conclusion Dapagliflozin and roxadustat restore podocyte carbohydrate metabolism, amino acids profile and arginine biosynthesis alterations under high glucose. SGLT2 inhibition and HIF stabilization are potential interventions for the dysregulated podocyte energy metabolism in DKD."

Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Nephrology • Renal Disease • STAT3

Roxadustat Response by Inflammation/Iron Status Subgroups in Patients on Peritoneal Dialysis and CKD-Related Anemia: Secondary Analysis of the NANLING Study (KIDNEY WEEK 2025) - P4 | "The changes in iron parameters suggest roxadustat suppresses hepcidin and mediates iron utilization. Hb was corrected and maintained without dose escalation, supporting roxadustat as a viable alternative therapy for PD patients."

Clinical • Anemia • Chronic Kidney Disease • Hematological Disorders • Inflammation • Nephrology • Renal Disease • CRP

Peridialytic Erythropoietin vs. Roxadustat in Hemodialysis-Dependent Patients with CKD (KIDNEY WEEK 2025) - "Conclusion Peridialytic erythropoietin was associated with a more favorable cardiovascular safety profile versus roxadustat in hemodialysis-dependent CKD patients. Proportions of patients with hospitalization for complications and length of hospital stay"

Clinical • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Darbepoetin vs. Other Agents in CKD Anemia: A Meta-Analysis (KIDNEY WEEK 2025) - "Results Molidustat showed the best cardiovascular and thrombotic safety despite lower efficacy, while methyl polyethylene glycol-epoetin beta showed the largest Hb increase in this network meta-analysis of 12 randomized trials involving over 4,000 CKD patients with anemia...Conclusion Daprodustat and roxadustat indicated equivalent or higher efficacy when compared to darbepoetin in elevating hemoglobin levels in CKD-related anemia, with daprodustat demonstrating a good safety profile...Comparitive efficacy of ESAs + Lower rank number = greater hgb-raising efficacy. * Comparison only, not an exact quantitative ranking."

Retrospective data • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Hypertension • Nephrology • Renal Disease

Association Between Erythrocyte Indices and Roxadustat Response in Patients with CKD and Anemia: ROXSTAR Registry Secondary Analysis (KIDNEY WEEK 2025) - P4 | "Conclusion MCV and MCH increased over 52-week roxadustat, with ND-CKD patients showing a marginal Hb-MCV association. Baseline MCV and iron status may not influence Hb response to roxadustat."

Clinical • Anemia • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Risk prediction for cardiovascular events and all-cause mortality in maintenance hemodialysis patients. (PubMed, Front Med (Lausanne)) - "Significant predictors included dialysis vintage, post-dialysis β2-microglobulin levels, B-Carboxy-Terminal Peptide of Type I Collagen, total bilirubin, lymphocyte count, lactate dehydrogenase, mean corpuscular hemoglobin concentration, and the use of roxadustat. Across all endpoints, the ML models demonstrated better discrimination than Cox regression models. Overall, ML models provided a more reliable prognostic assessment than Cox regression models for predicting CVE and all-cause mortality in MHD patients over the observation period."

Journal • Cardiovascular • Renal Disease • B2M