Valcyte (valganciclovir) / Roche, Mitsubishi Tanabe - LARVOL DELTA

Epidemiology, risk factors, and outcomes of cytomegalovirus (CMV) reactivation in allogeneic hematopoietic stem cell transplantation: A retrospective observational single-centre study from western India (ASH 2025) - "While pre-emptive therapy has reduced CMV disease, reactivation remains common, particularly in alternate donor transplants and resource-limited settings where access to newer antivirals like letermovir is limited...Serotherapy type did not impact overall reactivation, but early reactivation was significantly higher with Grafalon vs. Thymoglobulin (90% vs. 33.6%, p<0.001)...Adverse effects included neutropenia (ganciclovir/valganciclovir) and nephrotoxicity (foscarnet/cidofovir)... This single-centre study demonstrates a high CMV reactivation rate (89.7%) in a high-seroprevalence Indian cohort, especially in alternate donor and PTCy-treated patients. While CMV disease was rare due to effective surveillance and pre-emptive therapy, recurrence and antiviral toxicity were common. The type of serotherapy influenced early reactivation risk."

Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Immunology • Neutropenia • Transplantation

Paroxysmal nocturnal hemoglobinuria: Transplant experience using post-transplant cyclophosphamide in a resource-limited setting (ASH 2025) - "On the other hand, complement inhibitors (eculizumab, ravulizumab) have emerged as effective treatments with lower toxicity, though they are not curative...MRD transplant patients received Fludarabine/Melphalan (Flu/Mel) and haploidentical transplant received standard Baltimore protocol...Among them, two patients developed CMV reactivation, which was well controlled with valganciclovir... Allogeneic HSCT remains a potentially curative but high risk option for PNH patients, particularly in resource limited settings. Our experience highlights the significant transplant related morbidity and mortality, even with modern GVHD prophylaxis such as PTCy.Given the availability of safer, though non-curative, therapeutic alternatives like complement inhibitors, urgent advocacy is needed. Patient groups, healthcare providers, policy-makers, and international aid organizations must collaborate to improve access to life-saving treatments in low-income countries."

Post-transplantation • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Inflammation • Mucositis • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Pneumonia • Rare Diseases • Respiratory Diseases • Thrombosis • Transplantation

Concurrent culprits: Deciphering CMV co-infection in persistent COVID-19 scenarios. (PubMed, Przegl Epidemiol) - "This case series highlights the complex relationship between SARS-CoV-2 and CMV, emphasizing the need for a comprehensive virological evaluation of persistent COVID-19 cases. As the global medical landscape has grappled with the evolving challenges of the pandemic, incorporating such multifaceted clinical insights remains paramount."

Journal • Cytomegalovirus Infection • Immunology • Infectious Disease • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases

Baseline cytomegalovirus IgG levels predict reactivation in patients with multiple myeloma receiving elranatamab (ASH 2025) - "Preemptive valganciclovir was initiated upon reactivation...No significant differences were observed between the groups in monitoring frequency or duration, baselinecharacteristics, or the use of tocilizumab and corticosteroids for cytokine release syndrome (CRS)... High baseline CMV IgG titers were associated with an increased risk of CMV reactivation following elranatamabtherapy, consistent with findings in allogeneic transplantation and CAR-T cell therapy settings. Prophylactic immunoglobulin mayhelp reduce reactivation risk in high-risk patients. Although limited by its single-center, retrospective design and small samplesize, this study suggests that baseline CMV IgG titers may serve as a useful biomarker to guide risk-adapted CMV monitoring andprevention strategies during elranatamab treatment."

Clinical • IO biomarker • Cytomegalovirus Infection • Genetic Disorders • Hematological Malignancies • Inflammation • Multiple Myeloma • Ocular Inflammation • Ophthalmology • Retinal Disorders

Clinical characteristics of human herpesvirus 7 (HHV-7) reactivation following umbilical cord blood transplantation: A retrospective study (ASH 2025) - "HHV-7 was detected during ganciclovir (GCV)or valganciclovir (VGCV) treatment in 9 blood samples (47.4%) and 3 BAL samples (30.0%). A total of 1,625 CBT was performed during the study period. Among them, 1,325 patients (81.5%)underwent at least one multiplex PCR test, with a median of 4 tests per patient (range, 1–43), totaling6,744 samples (blood n=5,328; CSF n=1,069; BAL n=140; ascites n=114; pleural effusion n=91; lymph noden=2). HHV-7 DNA was detected in 33 of 1,325 patients (2.5%) who underwent post-transplant viralsurveillance."

Retrospective data • Bone Marrow Transplantation • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Herpes Simplex • Herpes Zoster • Immunology • Infectious Disease • Respiratory Diseases • Transplantation • Varicella Zoster

Human hepatocyte engraftment in F8KO TK-NOG mice: Creating a novel Hemophilia A mouse model with functional human coagulation cascade (ASH 2025) - "Thesemice received 0.1 mg/mL valganciclovir hydrochloride in drinking water for 48 hours...This phenomenon was supported by ECL measurements, which confirmed hFIX andhFX expression with positive correlation to chimerism rate, indicating that humanized TK-NOG F8KO micewith higher chimerism rates express greater quantities of human coagulation factors.Finally, activated partial thromboplastin time (APTT) and clot waveform analysis were performed in micewith sufficient plasma volume to evaluate the effects of rhFVIII and emicizumab, that has no crossreactivity to mouse FIX and FX...We successfully developed a novel humanized mouse model expressing human coagulation factorsderived from transplanted human hepatocytes. This model demonstrates that human-derivedcoagulation factors increase proportionally with chimerism rate, enabling effective evaluation of humanfactor-specific therapeutics. With stable production of high-chimerism mice, applications to variousdisease research..."

Preclinical • Hematological Disorders • Hemophilia • Hemophilia A • Hepatology • Liver Failure • Rare Diseases • PRKDC

A phase I/IIa, open-label, multicentre study evaluating the safety, tolerability, and efficacy of RZ-001 in combination with valganciclovir (VGCV) in patients (pts) with human telomerase reverse transcriptase (hTERT)-positive glioblastoma multiforme (GBM) (ESMO Asia 2025) - "RZ-001 was well tolerated with an acceptable preliminary safety profile and encouraging efficacy results."

Clinical • Combination therapy • P1/2 data • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • TERT

Prospective real-world evaluation of branded and bioequivalent letermovir prophylaxis for cytomegalovirus reactivation in allogeneic HSCT: First report from an LMIC (ESMO Asia 2025) - "Conventional pre-emptive antivirals (ganciclovir, valganciclovir, foscarnet) are limited by myelosuppression and nephrotoxicity...All transplants were from haploidentical donors, and 78% used post-transplant cyclophosphamide–based GVHD prophylaxis... This first prospective LMIC report shows both innovator and bioequivalent letermovir are feasible, safe, and well tolerated for CMV prophylaxis in adult HSCT recipients. Larger multicenter trials are warranted to confirm efficacy and cost-effectiveness in resource-limited settings."

Clinical • HEOR • Real-world • Real-world evidence • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Intracellular Ganciclovir Tri-Phosphate Concentrations in Children with Congenital Cytomegalovirus Infection. (PubMed, J Infect Dis) - "These findings underscore the need to define the kinetics of GCV-TP in cell matrices relevant to its activity to determine appropriate VGCV dosing strategies in this population and establish safe and define effective therapeutic concentration targets."

Clinical • Journal • Cytomegalovirus Infection • Hematological Disorders • Infectious Disease • Neutropenia • Otorhinolaryngology • Pediatrics

Low-Dose Valganciclovir for Primary Cytomegalovirus Prophylaxis After Heart Transplant: A 10-Year Experience. (PubMed, Clin Transplant) - "This is the largest report of LD VGCV use in HTR. Despite reduced VGCV exposure, HTR remain at high risk for cytopenias. Regardless of potential risk factors, including a high prevalence of overweight BMI and adequate renal function, LD VGCV was associated with expected rates of BT CMV and CMV resistance, suggesting LD VGCV could be considered in HTR. Identification of CMV end-organ disease and GCV resistance suggests that LD VGCV could be considered cautiously in CMV D+/R- HTR."

Journal • Retrospective data • Cytomegalovirus Infection • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders • Immunology • Leukopenia • Neutropenia • Obesity • Ocular Inflammation • Ophthalmology • Retinal Disorders • Transplantation

A Matched Case-Control Study to Evaluate Predicted Drug Exposures and Neutropenia during Valganciclovir Prophylaxis in Pediatric Solid Organ Transplant Recipients. (PubMed, Transpl Infect Dis) - "Predicted GCV exposures were similar among pSOT recipients with and without neutropenia, suggesting that differences in dosing and pharmacokinetics covariates did not drive toxicity in our population. Measurement of GCV concentrations may discern whether toxicity relates to exposures/concentrations or intrinsic factors (i.e., genetics) in the pSOT population."

Journal • Cytomegalovirus Infection • Hematological Disorders • Infectious Disease • Neutropenia • Pediatrics • Solid Organ Transplantation • Transplantation

Generation of CD68+ macrophage-depleted NOG mice for in vivo engraftment of human red blood cells. (PubMed, Int Immunol) - "To counteract this, we created thymidine kinase (TK) transgenic (Tg) mice under the human CD68 promoter (NOG-pCD68-TK Tg), which, upon administration of valganciclovir (VGCV), led to the successful depletion of macrophages. Consequently, hRBCs showed significantly prolonged engraftment in NOG-pCD68-TK Tg mice compared to non-Tg mice following macrophage depletion, maintaining engraftment for up to 14 days post-transplantation. This study elucidates the erythrophagocytosis mechanisms of hRBCs in mice and establishes NOG-pCD68-TK Tg mice as a valuable model for the long-term engraftment of hRBCs, potentially advancing in vivo hRBC research."

Journal • Preclinical • Hematological Disorders • Infectious Disease • Malaria • Transplantation • CD68

Cytomegalovirus and Crohn's disease as competing causes of small bowel inflammation after double-lung transplantation. (PubMed, Eur Clin Respir J) - "CMV infection was identified in intestinal biopsies and initially managed with two courses of valganciclovir. Due to persistent symptoms, genotypic analysis was performed, revealing UL97 mutations conferring ganciclovir resistance. Sequential therapy with foscarnet, maribavir, and letermovir achieved virological clearance, but diarrhea persisted...The patient was commenced on standard biological therapy for Crohn's disease, resulting in marked clinical improvement and recovery. This case illustrates the diagnostic challenge of distinguishing CMV enteritis from de novo Crohn's disease and determining the primary driver of the clinical presentation."

Journal • Crohn's disease • Cytomegalovirus Infection • Gastroenterology • Gastrointestinal Disorder • Immunology • Infectious Disease • Inflammation • Inflammatory Bowel Disease • Mucositis • Respiratory Diseases • Solid Organ Transplantation • Transplantation

Does treatment with antiviral medication affect mortality among immunocompetent individuals with cytomegalovirus reactivation? A systematic review and meta-analysis. (PubMed, BMC Infect Dis) - No abstract available

Journal • Retrospective data • Review • Cytomegalovirus Infection

Protein losing enteropathy due to congenital disorder of glycosylation: A case report. (PubMed, SAGE Open Med Case Rep) - "Urine CMV PCR was positive, and he was treated with valganciclovir for suspected Menetrier's disease, with no improvement in symptoms...Treatment is well-tolerated and highly effective. In the case presented, the patient began showing symptomatic and biochemical improvement within a week of initiating therapy."

Journal • Gastrointestinal Disorder • Hematological Disorders • Hypoglycemia • Immunology

GVO-1102: Phase 1 Study of GEN2 in Patients With Advanced Solid Tumors (clinicaltrials.gov) - P1 | N=37 | Active, not recruiting | Sponsor: GenVivo, Inc. | Recruiting ➔ Active, not recruiting | N=124 ➔ 37

Enrollment change • Enrollment closed • Oncology • Solid Tumor

Replication-competent adenovirus-mediated double suicide gene therapy with fractionated stereotactic radiosurgery in progressive glioblastoma: A phase 1 trial (SNO 2025) - "Patients received prodrug therapy consisting of 4 days of 5-fluorocytosine (150 mg/kg/day, orally) and valganciclovir (1800 mg/daily) for 13 days...Patients on Ad5-yCD/mutTKSR39rep-ADP gene therapy with combined fSRS had an mOS of 12.3 months. Complete study results will be reported."

Gene therapy • P1 data • Surgery • Brain Cancer • Cardiovascular • CNS Disorders • Epilepsy • Gene Therapies • Glioblastoma • Heart Failure • Solid Tumor

Replication-competent adenovirus-mediated double suicide gene therapy with fractionated stereotactic radiosurgery in progressive glioblastoma: A phase 1 trial (SNO 2025) - "Patients received prodrug therapy consisting of 4 days of 5-fluorocytosine (150 mg/kg/day, orally) and valganciclovir (1800 mg/daily) for 13 days...Patients on Ad5-yCD/mutTKSR39rep-ADP gene therapy with combined fSRS had an mOS of 12.3 months. Complete study results will be reported."

Gene therapy • P1 data • Surgery • Brain Cancer • Cardiovascular • CNS Disorders • Epilepsy • Gene Therapies • Glioblastoma • Heart Failure • Solid Tumor

Cytomegalovirus-specific cell-mediated immunity for prediction of post-prophylaxis CMV disease in a phase 3 trial of letermovir vs valganciclovir prophylaxis in donor CMV-seropositive recipient CMV-seronegative kidney transplant recipients. (PubMed, Clin Infect Dis) - "Among adult CMV high-risk D+R- KTRs who received prophylaxis, CMV-CMI by QFT-CMV increased over time. The result at the end of prophylaxis had limited clinical utility for identifying the risk for subsequent CMV disease."

Journal • P3 data • Cytomegalovirus Infection • Transplantation

CLEAR-CMV: Combination Letermovir and Standard of Care Antiviral for Enhanced Antiviral Response in Cytomegalovirus Infection in Lung Transplant Recipients (clinicaltrials.gov) - P4 | N=40 | Not yet recruiting | Sponsor: University Health Network, Toronto

New P4 trial • Cytomegalovirus Infection • Infectious Disease • Respiratory Diseases • Transplantation

Replication-competent adenovirus-mediated double suicide gene therapy with fractionated stereotactic radiosurgery in progressive glioblastoma: A phase 1 trial (WFNOS 2025) - "Patients received prodrug therapy consisting of 4 days of 5-fluorocytosine (150 mg/kg/day, orally) and valganciclovir (1800 mg/daily) for 13 days...Patients on Ad5-yCD/mutTKSR39rep-ADP gene therapy with combined fSRS had an mOS of 12.3 months. Complete study results will be reported."

Gene therapy • P1 data • Surgery • Brain Cancer • Cardiovascular • CNS Disorders • Epilepsy • Gene Therapies • Glioblastoma • Glioma • Heart Failure • High Grade Glioma • Oncology • Solid Tumor

Replication-competent adenovirus-mediated double suicide gene therapy with fractionated stereotactic radiosurgery in progressive glioblastoma: A phase 1 trial (WFNOS 2025) - "Patients received prodrug therapy consisting of 4 days of 5-fluorocytosine (150 mg/kg/day, orally) and valganciclovir (1800 mg/daily) for 13 days...Patients on Ad5-yCD/mutTKSR39rep-ADP gene therapy with combined fSRS had an mOS of 12.3 months. Complete study results will be reported."

Gene therapy • P1 data • Surgery • Brain Cancer • Cardiovascular • CNS Disorders • Epilepsy • Gene Therapies • Glioblastoma • Glioma • Heart Failure • Solid Tumor

Disseminated Kaposi sarcoma patients exhibit an expanded population of CD8+CD57+ T cells and an immunosenescence profile. (PubMed, Front Immunol) - "Valganciclovir (VGC), as an add-on therapy in patients with disseminated Kaposi Sarcoma/human immunodeficiency virus (DKS/HIV), modulates the activation of T-cell subsets; however, its effect on the T-cell immunosenescence profile is unclear...The microenvironment generated in DKS/HIV patients increases the frequency of T-cells exhibiting an immunosenescence phenotype, and this effect is independent of the treatment schedule used, suggesting that during coinfection, a chronic immunosuppressive microenvironment may develop, impairing immune surveillance and resilience. These results could be explored to identify novel therapeutic approaches."

IO biomarker • Journal • Epstein-Barr Virus Infections • Human Immunodeficiency Virus • Infectious Disease • Kaposi Sarcoma • Metabolic Disorders • Oncology • Sarcoma • Solid Tumor • B3GAT1 • CD27 • CD4 • CD8 • CDH1 • HAVCR2 • KLRG1 • PD-1 • SLC2A1

Impact of Early Anti-Cytomegalovirus Therapy on the Incidence of Chronic Graft-Versus-Host Disease (ASH 2023) - "Ganciclovir (GCV) and foscarnet (FCN) are essential intravenous anti-CMV agents...We excluded patients who received FCN, GCV, valganciclovir, or letermovir as prophylactic therapy; those who began anti-CMV therapy before the day of transplantation; those who received both FCN and GCV; those who underwent T cell-depleting treatment... In this study, patients requiring early anti-CMV therapy were at a high risk of chronic GVHD, primary due to their increased incidence of acute GVHD. Remarkably, FCN could potentially mitigate the incidence of chronic GVHD for them. Especially, FCN would be beneficial for male recipients of female donor transplants who are typically at high risk for chronic GVHD."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation

Haploidentical Donor Transplants with Cytokine Release Syndrome Have Increased Risk of Clinically Significant Early CMV Reactivation Compared with Matched Donor Transplants Receiving Post-Transplant Cyclophosphamide (ASH 2023) - "Matched-donor alloSCTs also received cyclosporin from Day +5 while haploidentical alloSCTs also received tacrolimus and mycophenolate from Day +5...Prophylactic valganciclovir (900 mg daily) was planned upon engraftment for seropositive recipients and those receiving stem cells from a seropositive donor... This single-center retrospective comparison of PTCy-based transplants shows that haploidentical transplants have increased risk of early and clinically significant reactivation of CMV. Potential explanations include increased immunosuppression with mycophenolate and the presence of CRS, where inflammatory cytokines may de-repress the CMV major immediate early promoter. Selecting this high-risk group (haploidentical transplants with CRS) may be a cost-effective use of letermovir, which can be initiated prior to engraftment."

Clinical • Cytokine release syndrome • Post-transplantation • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Graft versus Host Disease • Immunology • Inflammation • Transplantation