Hemlibra (emicizumab-kxwh) / Roche, JW Pharma - LARVOL DELTA

When A Fall Isn't Just a Fall: Delayed Diagnosis of Acquired Hemophilia A in a Nonagenarian Patient. (PubMed, Am J Case Rep) - "During current admission, prolonged aPTT prompted further workup, revealing low factor VIII activity of 5% and a high factor VIII inhibitor titer of 15.6 Bethesda Units, establishing the diagnosis of acquired hemophilia A. Bleeding control required sequential bypassing agents, corticosteroids, rituximab, cyclophosphamide, and eventually emicizumab. Basic coagulation testing including prothrombin time and aPTT can help screen patients who warrant additional coagulopathy evaluation. Timely diagnosis is critical to avoid life-threatening bleeding complications."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Mood Disorders • Pain • Rare Diseases

Patient Perspectives in Haemophilia A: Unmet Needs of Individuals Receiving Factor and Non-Factor Treatments. (PubMed, Haemophilia) - "This study examined treatment patterns and unmet needs in adults with severe haemophilia A receiving emicizumab or EHL-FVIII. Persistent symptoms (bleeds, pain, and joint damage) imposed limitations on PwSHA's daily life, and both subgroups reported continued use of additional factor therapy, as well as reduced satisfaction with treatment protection and administration frequency."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Pain • Pain • Rare Diseases

Development of Inno8, an orally administered factor VIIIa-mimetic antibody fragment for treatment of hemophilia A. (PubMed, Blood) - P1 | "Inno8 demonstrated, on average, 90‑fold greater in vitro potency than a sequence‑identical emicizumab analogue. Inno8 is currently in clinical evaluation, with first-in-human dosing completed (VOYAGER1, NCT06649630). Pending further clinical evaluation, Inno8 may represent an advance in biologics development and a potential non-invasive prophylactic therapy for individuals with HA."

First-in-human • Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Emicizumab enables rapid tolerance, reducing bleeding during low-dose ITI in pediatric hemophilia A. (PubMed, Blood Vessel Thromb Hemost) - "In this pediatric cohort, LD-ITI combined with emicizumab was associated with faster tolerance achievement, bleeding reduction, and no increase in direct costs. These findings suggest that ITI associated with emicizumab may provide a more efficient and clinically manageable approach to inhibitor eradication in pediatric patients."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Inflammation • Pediatrics • Rare Diseases

An evaluation of denecimig for bleeding prophylaxis in hemophilia A. (PubMed, Expert Opin Biol Ther) - "Despite advances in factor VIII (FVIII) replacement and emicizumab prophylaxis, people with hemophilia A may experience breakthrough bleeding, progressive joint disease, treatment burden, and inhibitor-related limitations. Denecimig (Mim8) is a fully human, next-generation FVIIIa-mimetic bispecific antibody engineered to provide subcutaneous prophylaxis irrespective of FVIII inhibitor status...However, it has not demonstrated superiority over established prophylactic therapies. Its clinical value will ultimately depend on mature thrombotic and immunogenic safety, perioperative evidence, head-to-head or real-world comparative effectiveness, treatment persistence, cost-effectiveness, and equitable global access rather than apparently favorable early pivotal-trial annualized bleeding rates alone."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Rheumatology

Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A. (PubMed, Res Pract Thromb Haemost) - "The lack of correlation of bleed outcomes with plasma emicizumab concentration suggests that minimal effective drug concentrations should be reconsidered. Studies to rationalize and personalize therapy should be explored."

Journal • Observational data • Hematological Disorders • Hemophilia • Hemophilia A • Pediatrics • Rare Diseases

FRONTIER5: Safety and patient experience of a direct switch from emicizumab to denecimig (Mim8) in patients with haemophilia A with or without inhibitors. (PubMed, J Thromb Haemost) - P3 | "The direct switch from emicizumab to denecimig without a washout period was well tolerated in adolescents and adults with HA with or without inhibitors. No new safety concerns were observed."

Journal • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Rare Diseases

Cost-effectiveness analysis of emicizumab use in hospitalized older individuals with acquired hemophilia A. (PubMed, Blood Vessel Thromb Hemost) - "The addition of up-front emicizumab to SOC is less costly and more effective, even at current emicizumab pricing. Our results provide strong economic and clinical justification to consider up-front emicizumab use in AHA management."

HEOR • Journal • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases

Discovery and development of a bispecific antibody for the treatment of hemophilia. (PubMed, Proc Natl Acad Sci U S A) - "Importantly, they identified the biophysical requirements for the bispecific to mimic FVIII function effectively and safely, and their breakthrough protein engineering design made possible favorable pharmacokinetics and physicochemical properties with efficient manufacturability. Their collective research efforts demonstrate how imagination, determination, and multidisciplinary scientific research come together to improve patients' lives."

Journal • Review • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

Plasma-Derived Factor VIIa/Factor X Improves Thrombin Generation Potential in a Plasma Model of Haemophilia A Supplemented With Direct Oral Anticoagulants. (PubMed, Haemophilia) - "Pd-FVIIa/FX improved coagulation potential in the in vitro model plasma of PwHA-inh spiked with DOACs, irrespective of concomitant emicizumab. The clinical efficacy and safety of pd-FVIIa/FX require further evaluation."

Journal • Atrial Fibrillation • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

Emicizumab and shared medical decision in patients living with hemophilia A. (PubMed, Hematol Transfus Cell Ther) - "This study highlights the importance of shared decision-making in hemophilia treatment, encouraging open communication between patients and physicians for more personalized care."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Multidisciplinary management of fractures in patients with hemophilia: Advances in perioperative hemostasis and functional restoration in the era of novel therapies. (PubMed, Injury) - "Particular attention is given to the role of multidisciplinary collaboration in optimizing surgical outcomes and minimizing perioperative complications. By integrating available evidence with practical clinical considerations, this review aims to highlight critical elements of perioperative care that contribute to safer surgical management and improved functional outcomes in patients with hemophilic arthropathy."

Journal • Review • Anesthesia • Gene Therapies • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Orthopedics • Rare Diseases • Rheumatology

Serum Sclerostin Levels in Children with Hemophilia A on Emicizumab (ESPE 2026) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Physical Activity, Bleeding Incidence and Health-Related Quality of Life, in Patients With Haemophilia A Without Inhibitors, Treated With Emicizumab: A Post-Hoc Descriptive Analysis of the Power Study Data. (PubMed, Haemophilia) - No abstract available

HEOR • Journal • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Real-World Experience With Emicizumab for Haemophilia A From the Physician Perspective in a Real-World Clinical Setting. (PubMed, Haemophilia) - "Among PwHA receiving emicizumab, rates and severity of bleeds decreased after emicizumab initiation, regardless of the clinical setting."

Journal • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Oncology • Rare Diseases

Bridging the Gap: A Systematic Review of Modern Hemophilia Therapies and Global Inequities in Clinical Trial Participation. (PubMed, Haemophilia) - "Modern therapies demonstrate strong efficacy and acceptable safety, with several approved agents. However, persistent global inequities in trial participation and access highlight the need for more inclusive research and equitable implementation strategies."

Journal • Review • Gene Therapies • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

Coagulation potential of concomitant factor VIII administration in people with hemophilia A receiving emicizumab prophylaxis (CAGUYAMA study): a multicenter, open-label, nonrandomized clinical trial. (PubMed, Res Pract Thromb Haemost) - "No thromboembolic events, thrombotic microangiopathy, or hypersensitivity reactions were reported. FVIII administered at a target dose of 30 IU/kg (allowable range, 25-35 IU/kg) improved global coagulation parameters and achieved effective hemostasis without thrombotic complications, warranting evaluation as part of emicizumab prophylaxis."

Clinical • Journal • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases

Successful immune tolerance induction using efanesoctocog alfa in severe hemophilia A: A case series. (PubMed, J Thromb Haemost) - "Transition to once-weekly individualized prophylaxis maintained functional FVIII activity and therapeutic FVIII trough levels (4%-6%), allowing discontinuation of emicizumab. Sustained bleed-free outcomes were achieved with individualized prophylactic dosing without inhibitor recurrence. These cases support efanesoctocog alfa-based ITI as a promising strategy for rescue and retreatment patients with complex inhibitor histories."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Heat treatment of emicizumab samples - a way forward? (PubMed, Res Pract Thromb Haemost) - No abstract available

Journal

Acquired hemophilia A: an illustrated review based on the French National Guidelines. (PubMed, Res Pract Thromb Haemost) - "More recently, prophylaxis with emicizumab has emerged as a promising strategy to reduce recurrent bleeding, with early clinical data suggesting favorable efficacy and safety. Second, inhibitor eradication relies on immunosuppressive therapy, typically corticosteroids alone or in combination with agents such as cyclophosphamide or rituximab, guided by baseline FVIII activity and inhibitor titer. Close and prolonged follow-up is mandatory, combining clinical assessment of bleeding control, recurrence risk, and treatment toxicity, with laboratory monitoring of hemoglobin, factor VIII activity, von Willebrand factor levels, and inhibitor titers. Surveillance should be maintained for at least 2 years after complete remission."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases

Changes in Hypertrophic Synovium Status in Adult Patients With Severe Hemophilia A Receiving Emicizumab Prophylaxis: The RESOLVE Study. (PubMed, Haemophilia) - "In this real-world setting, emicizumab provides bleeding protection and is associated with reduction/resolution of HS in more than 50% of affected joints. In case of persistent HS, a tailored approach to joint health management may be needed to fully prevent or reverse HS."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Rare Diseases • Rheumatology

Remission-driven discontinuation shapes the observed duration of emicizumab in acquired hemophilia A. Comment on: "Short-term emicizumab in patients with acquired hemophilia A". (PubMed, Haematologica) - "Not available."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Acquired haemophilia A associated with prostate cancer managed with recombinant activated factor VII and emicizumab. (PubMed, BMJ Case Rep) - "Emicizumab was used for haemostatic prophylaxis while outpatient once-weekly rituximab was planned for inhibitor eradication. This case highlights early recognition, evaluation for associated malignancy and use of emicizumab as prophylaxis that may allow individualised immunosuppression in selected older patients."

Journal • Genito-urinary Cancer • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Oncology • Prostate Cancer • Rare Diseases • Solid Tumor

Severe Spontaneous Hematoma in an Elderly Female: A Case of Acquired Hemophilia A. (PubMed, J Community Hosp Intern Med Perspect) - "Management included recombinant activated factor VII (rFVIIa), porcine FVIII (Obizur), corticosteroids, and rituximab, leading to clinical stabilization. We also review diagnostic criteria, the role of inhibitor levels in monitoring, and current treatment guidelines, including the emerging role of emicizumab."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases

Erector Spinae Block in Hemophila A Patient on Prophylactic Emicizumab (ASA 2026) - "Emicizumab interferes with standard clot-based assays, rendering procedural risk assessment challenging. Our case illustrates the clinical reasoning and multidisciplinary coordination required for regional anesthesia decision-making in the emicizumab era and demonstrates that continuous erector spinae plane catheterization can provide effective analgesia for traumatic rib fractures when neuraxial techniques are avoided."

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Orthopedics • Rare Diseases