Bredinin (mizoribine) / Asahi Kasei, Lee's Pharm - LARVOL DELTA

LONG-TERM EFFICACY AND SAFETY OF PERIODIC REPEATED RITUXIMAB ADMINISTRATIONS FOR PEDIATRIC REFRACTORY NEPHROTIC SYNDROME: A MULTICENTER RETROSPECTIVE OBSERVATIONAL STUDY (ISN-WCN 2026) - "RTX was administered four times at 6-month intervals, followed by mizoribine pulse therapy and early discontinuation of calcineurin inhibitors. However, hypogammaglobulinemia occurred frequently. Low serum IgG levels and B-cell depletion at the time of RTX administration were identified as independent risk factors for its development."

Observational data • Retrospective data • Agranulocytosis • Glomerulonephritis • Granulocytopenia • Infectious Disease • Nephrology • Pediatrics • Renal Disease

A CASE OF A MAINTENANCE DIALYSIS PATIENT WITH COMBINED PULMONARY-LIMITED AL AMYLOIDOSIS AND MALT LYMPHOMA REVEALED AT AUTOPSY (ISN-WCN 2026) - "She was treated with prednisolone, mizoribine, and azathioprine but developed end-stage renal disease requiring hemodialysis ten years later.Seven years before death, she exhibited elevated immunoglobulins and an IgA-κ M-protein, diagnosed as monoclonal gammopathy of undetermined significance (MGUS)...Melphalan and dexamethasone therapy were initiated for localized pulmonary amyloidosis, but respiratory failure gradually progressed, requiring home oxygen therapy.She later suffered a fatal intracerebral hemorrhage unrelated to coagulation disorders or hypoglycemia...MALT lymphoma occurs in approximately 4–8% of SS-related lymphomas and most frequently arises in the stomach, though endoscopic findings can be subtle and nonspecific.In this patient, MALT lymphoma was diagnosed only postmortem, indicating that earlier endoscopic surveillance might have enabled detection and management. The case highlights the need for continuous vigilance for lymphoproliferative complications..."

Clinical • Amyloidosis • Cerebral Hemorrhage • Conjunctivitis • Dental Disorders • Dry Eye Disease • Extranodal Marginal Zone Lymphoma • Hematological Malignancies • Hypoglycemia • Immunology • Indolent Lymphoma • Infectious Disease • Interstitial Lung Disease • Lymphoma • Marginal Zone Lymphoma • Monoclonal Gammopathy • Nephrology • Ocular Infections • Ocular Inflammation • Ophthalmology • Pneumonia • Pulmonary Disease • Renal Disease • Respiratory Diseases • Sjogren's Syndrome • Xerostomia

EXPERIENCE WITH AVACOPAN FOR THE TREATMENT OF ANTI-NEUTROPHIL CYTOPLASMIC ANTIBODY (ANCA)-ASSOCIATED VASCULITIS (AAV) IN THE NEPHROLOGY DEPARTMENT OF NISHI-KOBE MEDICAL CENTER. (ISN-WCN 2026) - "Concomitant immunosuppressive agents included cyclophosphamide in 6 patients, rituximab in 2, azathioprine in 5, and mizoribine in 6; 2 patients underwent plasma exchange.Clinical remission was achieved in 85% of patients at 26 weeks...Avacopan was restarted at a lower dose in all cases—one with concomitant ursodeoxycholic acid and another after alcohol abstinence. No infections requiring hospitalization were observed during the observation periodConclusion Avacopan appeared to be a safe and effective glucocorticoid-sparing therapeutic option for AAV. However, regular monitoring of liver function is warranted.I have potential conflict of interest to disclose.I have received lecture fees from KISSEI PHARMACEUTICAL CO.,LTD.I did not use generative AI and AI-assisted technologies in the writing process."

ANCA Vasculitis • Atherosclerosis • Cardiovascular • Infectious Disease • Nephrology • Rare Diseases • Vasculitis

A STEROID-SPARING APPROACH FOR ADOLESCENT IGA NEPHROPATHY: TONSILLECTOMY, STEROID PULSE THERAPY, AND SUBSEQUENT MIZORIBINE (ISN-WCN 2026) - "Compared with conventional long-term oral steroid regimens, this approach may reduce adverse effects, particularly growth impairment, which is a critical concern in adolescents. Nonetheless, the small sample size and retrospective design limit the generalizability of these findings, and larger prospective studies are warranted to confirm efficacy and safety.In conclusion, our findings suggest that tonsillectomy combined with steroid pulse therapy and subsequent mizoribine administration may represent a promising therapeutic strategy for adolescent patients with IgA nephropathy, particularly those approaching adulthood, in whom growth and quality-of-life considerations are paramount."

Glomerulonephritis • IgA Nephropathy • Renal Disease

RISK FACTORS FOR REFRACTORY CYTOMEGALOVIRUS INFECTION IN HEMATOPOIETIC STEM CELL AND SOLID ORGAN TRANSPLANT RECIPIENTS (EBMT 2026) - "Hot map of risk factors for refractory or resistance CMV infection.Notes: The numbers in the hot map indicate the number of articles corresponding to the factors.Abbreviations: aGVHD, acute graft-versus-host disease; ATG, anti-thymocyte globulin; CMV: cytomegalovirus; BM: bone marrow; BU: busulfan; CB: cord blood; Cy: cyclophosphamide; EB, Epstein-Bar; MMF: mycophenolate mofetil; MP: methylprednisolone; MSD: matched sibling donor; MUD: matched unrelated donor; MZR: mizoribine; PB: peripheral blood; TBI: total body irradiation; VGCV: valganciclovir; WBC: white blood cell. The HSCT-specific risk factors for refractory CMV infection included host immune status, transplantation regimens and viral load, genotypes and blood parameters, while, CMV serological status of donor and recipient, anti-CMV drug exposure, and persistent disease were SOT-specific risk factors for refractory and resistance CMV. Early identification of patients at risk of refractory CMV infection can help..."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Immunology • Infectious Disease • Solid Organ Transplantation • Transplantation • CD8

RISK FACTORS FOR REFRACTORY CYTOMEGALOVIRUS INFECTION IN HEMATOPOIETIC STEM CELL AND SOLID ORGAN TRANSPLANT RECIPIENTS (EBMT 2026) - "Hot map of risk factors for refractory or resistance CMV infection.Notes: The numbers in the hot map indicate the number of articles corresponding to the factors.Abbreviations: aGVHD, acute graft-versus-host disease; ATG, anti-thymocyte globulin; CMV: cytomegalovirus; BM: bone marrow; BU: busulfan; CB: cord blood; Cy: cyclophosphamide; EB, Epstein-Bar; MMF: mycophenolate mofetil; MP: methylprednisolone; MSD: matched sibling donor; MUD: matched unrelated donor; MZR: mizoribine; PB: peripheral blood; TBI: total body irradiation; VGCV: valganciclovir; WBC: white blood cell. The HSCT-specific risk factors for refractory CMV infection included host immune status, transplantation regimens and viral load, genotypes and blood parameters, while, CMV serological status of donor and recipient, anti-CMV drug exposure, and persistent disease were SOT-specific risk factors for refractory and resistance CMV. Early identification of patients at risk of refractory CMV infection can help..."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Immunology • Infectious Disease • Solid Organ Transplantation • Transplantation • CD8

Efficacy of mizoribine in renal transplant recipients on calcineurin inhibitor-based immunosuppression: a network meta-analysis. (PubMed, Front Med (Lausanne)) - "To systematically evaluate the efficacy of mizoribine (MZR) in renal transplant recipients on a calcineurin inhibitor (CNI)-based maintenance regimen and to compare it with other immunosuppressants [mycophenolic acid (MPA), mycophenolate mofetil (MMF), cyclophosphamide (CTX)] utilizing network meta-analysis (NMA). Clinical selection should involve weighing the risks of infection (particularly BK virus) against gastrointestinal tolerability based on individual patient characteristics. MZR represents an effective and well-tolerated important alternative to traditional MMF/MPA."

Journal • Retrospective data • Review • Hematological Disorders • Infectious Disease • Leukopenia • Nephrology • Transplant Rejection • Transplantation

MYC-driven Medulloblastoma is Sensitive to the Pyrimidine Synthesis Inhibitor H3B-120 (AAN 2026) - "Both MYC tumors were more sensitive than healthy NES cells to inhibition of pyrimidine synthesis (H3B-120), but not purine (Mizoribine) or pyrimidine/purine (5-FU). Pyrimidine synthesis represents a key vulnerability for MYC-driven MB and offers a potential therapeutic alternative to Complex I inhibition with reduced systemic toxicity."

Brain Cancer • Medulloblastoma • Solid Tumor • MYC • MYCN

Initial treatment patterns of primary membranoproliferative glomerulonephritis in Japan (2017-2021): an updated analysis based on nationwide personal clinical records. (PubMed, Clin Exp Nephrol) - "Compared with the earlier report (Report 1), patients with newly registered primary MPGN presented with nephrotic syndrome more often, highlighting the continued risk of poor prognosis and the need for more refined therapeutic approaches."

Journal • Chronic Kidney Disease • Glomerulonephritis • Lupus Nephritis • Nephrology • Pediatrics • Renal Disease

HSP60 Mediates NLRP3 Inflammasome-Dependent Microglial Pyroptosis Via the TLR4/MyD88/NF-κB Signaling Axis After Subarachnoid Hemorrhage. (PubMed, Inflammation) - "In vitro, 80 µmol/L Mizoribine markedly attenuated microglial activation and pyroptosis, downregulated pro-inflammatory cytokines, and mitigated neuroinflammation. The upregulation of HSP60 after SAH promotes NLRP3 inflammasome assembly by activating the TLR4/MyD88/NF-κB signaling pathway, thereby inducing microglial pyroptosis and exacerbating the progression of early brain injury. Inhibition of HSP60 represents a potential therapeutic strategy for ameliorating EBI after SAH."

Journal • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Hematological Disorders • Inflammation • Subarachnoid Hemorrhage • Vascular Neurology • HSPD1 • MYD88 • NLRP3 • TLR4

A Retrospective Study on the Efficacy and Safety of Mizoribine in Anti-Rejection Therapy for Renal Transplant Recipients (ChiCTR) - P=N/A | N=200 | Not yet recruiting | Sponsor: West China Hospital, Sichuan University; West China Hospital, Sichuan University

New trial • Transplantation

Efficacy and safety of initial administration of mizoribine in deceased donor kidney transplant recipients: a Chinese single-center, prospective, randomized controlled study (ChiCTR) - P4 | N=202 | Not yet recruiting | Sponsor: The Second Affiliated Hospital of Guangxi Medical University; The Second Affiliated Hospital of Guangxi Medical University

New P4 trial • Nephrology • Transplantation

Efficacy and Safety of Conversion from Mycophenolate Mofetil to Mizoribine in Kidney Transplant Recipients with BK Polyomavirus Nephropathy (WTC 2025) - "Conversion from MMF to MZR can achieve rapid viral clearance and histological remission. This strategy maintains the stability of serum creatinine and uric acid levels with a favorable safety profile, representing a viable therapeutic option for BKPyVN."

Clinical • Renal Disease • Transplantation

Formononetin and mizoribine inhibit Porcine Reproductive and Respiratory Syndrome Virus replication in vitro. (PubMed, Front Nutr) - "Noteworthy was the observation of their synergistic effects when combined with Ribavirin. These results position FMN and MZR as promising antiviral agents against PRRSV, underscoring their low cytotoxicity and efficacy in early-stage viral inhibition. Such findings pave the way for their potential inclusion in future PRRSV management strategies."

Journal • Preclinical • Infectious Disease • Respiratory Diseases

Clinicopathological features of familial fibronectin glomerulopathy caused by a splice site variant in the Fibronectin 1 gene: a case report. (PubMed, CEN Case Rep) - "Treatment with glucocorticoids, combined with the immunosuppressant mizoribine and an angiotensin II receptor blocker (ARB), resulted in an incomplete remission of nephrotic syndrome; however, renal function has been preserved...Due to the administration of azathioprine, aspirin, and ARB, renal function and proteinuria have been stable over 10 years. The kidney biopsy revealed MPGN-like histological features in both the mother and the daughter; however, the mesangial area exhibited a milder expansion in the mother than in the daughter. Accumulating genotype-phenotype correlation data will be essential for managing FNG."

Journal • Genetic Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology • Rare Diseases • Renal Disease • FN1 • NECTIN1

Mizoribine or Cyclophosphamide for Lupus Nephritis: A Randomized Clinical Trial. (PubMed, JAMA Netw Open) - P3 | "Mizoribine can be used as an alternative to intravenous cyclophosphamide as induction therapy for lupus nephritis. ClinicalTrials.gov Identifier: NCT02256150."

Clinical • Journal • Glomerulonephritis • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Respiratory Diseases • Systemic Lupus Erythematosus

Efficacy and safety of new combination therapy with prednisolone, mizoribine, and lisinopril for severe childhood IgA Nephropathy (IPNA 2025) - "In our previous randomized control trial, combination treatment including prednisolone (PSL) and mizoribine (MZB) with warfarin and dipyridamole may be better for severe cIgAN than the combination treatment including PSL and MZB without warfarin nor dipyridamole from the point of proteinuria remission. Conclusions We confirmed the usefulness of the new combination treatment with PSL, MZB, and lisinopril for severe cIgAN in achieving early proteinuria remission and shortening PSL use without changing the proteinuria recurrence rate. Long-term outcomes will be needed for further investigation."

Clinical • Combination therapy • Glomerulonephritis • IgA Nephropathy • Renal Disease

Systemic inhibition of de novo purine biosynthesis prevents weight gain and improves metabolic health by increasing thermogenesis and decreasing food intake. (PubMed, bioRxiv) - "Crucially, given that many purine inhibitors have been FDA-approved for use in treating various conditions, our results indicate that they may benefit overweight or obese patients. A purine biosynthesis inhibitor, mizoribine, protects against diet-induced weight gainMizoribine prevents fat mass gain in high-fat diet-fed male miceMizoribine reduces food intake and increases thermogenesisMizoribine induces expression of sarcolipin, a regulator of thermogenesisMizoribine treatment reduces ectopic lipids and increases glucose tolerance."

Journal • Cardiovascular • Diabetes • Genetic Disorders • Metabolic Disorders • Obesity • Oncology • Type 2 Diabetes Mellitus

An Alternatively Spliced Gain-of-Function NT5C2 Isoform Contributes to Chemoresistance in Acute Lymphoblastic Leukemia. (PubMed, Cancer Res) - "Incorporation of the 8-amino acid sequence SQVAVQKR into this enzyme created a putative phosphorylation site and resulted in elevated nucleosidase activity, which is a known consequence of gain-of-function mutations in NT5C2 and a common determinant of 6-mercaptopurine (6-MP) resistance. Furthermore, both the NT5C2ex6a and R238W variants induced collateral sensitivity to the inosine monophosphate dehydrogenase (IMPDH) inhibitor mizoribine. These results ascribe an important role for splicing perturbations in chemotherapy resistance in relapsed B-ALL and suggest that IMPDH inhibitors, including the commonly used immunosuppressive agent mycophenolate mofetil, could be a valuable therapeutic option for treating thiopurine-resistant leukemias."

Journal • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • NT5C2

An Alternatively Spliced Gain-of-Function NT5C2 Isoform Contributes to Chemoresistance in Acute Lymphoblastic Leukemia. (PubMed, bioRxiv) - "Incorporation of the 8-amino acid sequence SQVAVQKR into this enzyme created a putative phosphorylation site and resulted in elevated nucleosidase activity, which is a known consequence of gain-of-function mutations in NT5C2 and a common determinant of 6-mercaptopurine (6-MP) resistance. Furthermore, both the NT5C2ex6a and R238W variants induced collateral sensitivity to the inosine monophosphate dehydrogenase (IMPDH) inhibitor mizoribine. These results ascribe an important role for splicing perturbations in chemotherapy resistance in relapsed B-ALL and suggest that IMPDH inhibitors, including the commonly used immunosuppressive agent mycophenolate mofetil, could be a valuable therapeutic option for treating thiopurine-resistant leukemias."

Journal • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • NT5C2

A case of membranous nephropathy complicated by Cronkhite-Canada syndrome successfully treated with mizoribine. (PubMed, CEN Case Rep) - "Nephrotic-range proteinuria persisted despite treatment with prednisolone and cyclosporine. Additional treatment with mizoribine resulted in incomplete remission type 1 of nephrotic syndrome, suggesting that mizoribine may be a treatment option for patients with CCS with steroid-resistant MN. Considering a high prevalence of hypoproteinemia due to chronic diarrhea and protein-losing enteropathy in patients with CCS, proteinuria might be overlooked; thus, follow-up urinalysis would be recommended in patients with CCS."

Journal • Alopecia • Gastrointestinal Disorder • Genetic Disorders • Glomerulonephritis • Nephrology • Oncology • Renal Disease • Solid Tumor

Mizoribine Promotes Molecular Chaperone HSP60/HSP10 Complex Formation. (PubMed, Int J Mol Sci) - "On the other hand, high concentrations of Mizoribine promoted HSP60-HSP10 complex formation and consequently suppressed IL-6 expression. Here, we propose a novel mechanism of immunosuppressive action of Mizoribine."

Journal • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Renal Disease • Rheumatoid Arthritis • Rheumatology • Transplantation • HSPD1 • IL6

POWER DOPPLER AND GREYSCALE CHANGES ON SALIVARY GLAND ULTRASONOGRAPHY IN SJOGREN'S DISEASE: AN OBSERVATIONAL STUDY (EULAR 2024) - "Ninety patients (61%) received immunosuppressants (including mizoribine [42 patients], glucocorticoids [42 patients], hydroxychloroquine [28 patients], abatacept [14 patients], etc.) for over four months, 33 received Japanese-Kampo medicine, five received saliva secretion stimulants, and 35 received no medication. GS scores worsened, but SMG PD scores decreased over time in SjD patients. PDs correlated with ESSDAI, SSFR, SWE, and SWV. PDs presence suggests the possibility of future salivary gland structural changes."

Clinical • Observational data • Immunology • Inflammation • Rheumatology • Sjogren's Syndrome

Safety and Efficacy of Mycophenolate Mofetil Associated With Tacrolimus for Kidney-pancreas and Kidney Transplantation: A Systematic Review and Meta-Analysis of Randomized Studies. (PubMed, Transplant Proc) - "Gastrointestinal and hematological adverse events and infections are the most common with TAC+MMF for kidney-pancreas and kidney. TAC+MMF effectively prevents acute cellular rejection, and alternatives with AZA, CyA, SRL, ECMPS, EVL, MAN, and MSR have similar efficacy and safety profiles. TAC monotherapy and MZR may be associated with a lower risk of infections."

Clinical • Journal • Retrospective data • Review • Cytomegalovirus Infection • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Leukopenia • Transplant Rejection • Transplantation

Immunosuppressive therapy for IgA nephropathy in children. (PubMed, Cochrane Database Syst Rev) - "There is a lack of high-quality evidence to guide the management of IgAN in children. There is no evidence to indicate that steroids, other immunosuppressive therapies, or tonsillectomy, when added to optimal supportive care, prevent a decline in eGFR or proteinuria in children with IgAN. Available studies were few, with small numbers, low-quality evidence, high or uncertain risk of bias, did not systematically assess harms associated with treatment, or report net benefits or harms. Severe cases and atypical presentations of IgAN were not included in the reviewed studies, and our findings cannot be generalised to these situations."

Clinical • Journal • Review • Glomerulonephritis • IgA Nephropathy • Lupus Nephritis • Nephrology • Renal Disease • Transplantation