NovoSeven (eptacog alfa) / Novo Nordisk - LARVOL DELTA

Glanzmann's thrombasthenia in a twin pregnancy: A case report. (PubMed, Case Rep Womens Health) - "This case report details the contemporary, multidisciplinary management of a 35-year-old primigravid woman with a dichorionic, diamniotic twin pregnancy and GT which included an elective caesarean section under general anaesthesia performed at 36 + 5 weeks of gestation, with recombinant factor VIIa (NovoSeven) and tranexamic acid (TXA) treatment preoperatively and pre-emptive uterotonic administration intraoperatively to minimise haemorrhage. Postpartum management included a seven-week course of TXA. This case highlights the importance of a proactive, multidisciplinary care plan to optimize pregnancy outcomes in women with GT, with a focus on minimizing the risk of severe haemorrhage."

Journal • Anesthesia • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Comparative Efficacy of Recombinant FVIII and Recombinant FVII Biosimilars in Severe Hemophilia A. (PubMed, Clin Appl Thromb Hemost) - "The only possible treatment for this bleeding condition is factor concentrate.AimThe aim of this study is to compare the effect of recombinant factor VIII (rFVIII) and recombinant factor VII (rFVII) on prothrombin time (PT), activated partial thromboplastin time (aPTT), FVIII and FVII in severe HA.MethodologyA mixing study was conducted on 30 samples of severe HA patients to assess the correction of PT, aPTT, FVIII, and FVII values using biosimilars of rFVIII (NovoEight and Kogenate FS) and rFVII (NovoSeven and AryoSeven) using a fully automated coagulation analyser 'Ceveron alpha'.ResultsAll the four drugs demonstrated a significant alteration for both PT (P < .0001) and aPTT (P < .0001) values. Two different groups of biosimilars were found to have a high potential to alter the PT and aPTT values. The FVIII biosimilars are efficient in increasing the FVIII levels."

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

Inhibitor Eradication in Postpartum Acquired Haemophilia A: Real-Life Case Series and Literature Review. (PubMed, Haemophilia) - "Notwithstanding that postpartum AHA has been reported to have a high rate of spontaneous remission, nearly half of this series experienced inhibitor eradication more than 1 month after disease onset and using immunosuppressive treatment for more than 2 months, with additional drugs being used in more than 40% of them, thus showing difficulties in disease remission in this postpartum AHA subpopulation."

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

Recombinant factor VIIa: new insights into the mechanism of action through product innovation. (PubMed, Res Pract Thromb Haemost) - "Eptacog alfa and eptacog beta share the same amino acid sequence but differ in posttranslational modifications. Further research is required to assess the clinical relevance of these differences. A better understanding of the mechanisms by which rFVIIa promotes hemostasis in patients will provide insights when evaluating clinical outcomes of safety and efficacy for innovative bypassing therapies."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases • PROCR

Novo Nordisk's sales increased by 25% in Danish kroner and by 26% at constant exchange rates to DKK 290.4 billion in 2024 (GlobeNewswire) - "Sales of rare blood disorder products increased by 7% in both Danish kroner and at CER, mainly driven by increased haemophilia B sales and NovoSeven...EMEA...Sales of rare blood disorder products decreased by 2% in both Danish kroner and CER, driven by lower NovoSeven and haemophilia A sales, partially countered by increased haemophilia B sales."

Sales • Hemophilia A • Hemophilia B

The Influence of Coagulation Factor Biosimilars in Shortening the Activated Partial Thromboplastin Time in Patients with Hemophilia a (ASH 2024) - "Negative correlation was observed between PT and FVII values after adding Kogenate FS (r=-0.103, p<0.001), aPTT and FVIII values after adding Kogenate FS (r=-0.898, p<0.001), aPTT and FVIII values after adding NovoEight (r=-0.865, p<0.001), as well as aPTT and FVIII values after adding AryoSeven (r=-0.647, p<0.001).Conclusion : All the investigated drugs significantly shorten aPTT values and increase values of FVIII and FVII. In terms of efficacy, no difference exists between AryoSeven and NovoSeven, as well as between Kogenate FS and NovoEight only in altering PT."

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

Labor Epidural in Patient with Von Willebrand Disease and Factor VII Deficiency (ASA 2024) - "Postpartum, von Willebrand factor activity (targeting >50% with Humate-P) and Factor VII activity (targeting >20% with NovoSeven) were trended, and she received peripartum tranexamic acid. This case highlights obstetric, anesthetic, and ethical challenges that inherited bleeding disorders carry."

Clinical • Anesthesia • CNS Disorders • Hematological Disorders • Hemophilia • Obstetrics • Pain • Postpartum Hemorrhage

Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series. (PubMed, Haemophilia) - "In this case series, eptacog beta was safe, effective, and economical as first-line therapy, treatment of refractory BEs, management of perioperative bleeding, or prophylaxis in haemophilia patients with inhibitors."

Journal • Real-world • Real-world effectiveness • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Osteoarthritis • Rare Diseases

Acquired haemophilia: Update in 2024 (PubMed, Rev Med Interne) - "Eradication of inhibitor should be established as soon as the diagnosis is confirmed with steroid alone often associated with cytotoxic agents or rituximab, depending on FVIII activity and inhibitor titer. The purpose of this review is to summarize the epidemiology, etiopathogenesis, diagnosis, treatment of AHA and discuss current recommendations."

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

Severe acquired hemophilia A associated with COVID-19 vaccination: A case report and literature review. (PubMed, Medicine (Baltimore)) - "This rare case underscores the importance of monitoring for AHA following COVID-19 vaccination. Although the benefits outweigh the risks of vaccination, AHA should be considered in the differential diagnosis of unusual bleeding following the vaccinations. Early diagnosis and management before severe bleeding are critical for successfully controlling life-threatening bleeding."

Journal • Review • Cardiovascular • Cerebral Hemorrhage • CNS Disorders • Critical care • Dermatology • Diabetes • Dyslipidemia • Hematological Disorders • Hemophilia • Hypertension • Immunology • Infectious Disease • Metabolic Disorders • Novel Coronavirus Disease • Oncology • Rare Diseases • Septic Shock

SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment (clinicaltrials.gov) - P1/2 | N=24 | Completed | Sponsor: Jiangsu Gensciences lnc. | Recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Rare Diseases

THE ROLE OF IMMUNOSUPPRESSIVE THERAPY FOR INHIBITORS ERADICATION IN POSTPARTUM ACQUIRED HEMOPHILIA A. ARE STEROIDS ALONE ENOUGH? A REAL-LIFE RETROSPECTIVE EXPERIENCE. (EHA 2024) - "All patients required anti-hemorrhagic therapy with a medium duration of 8 days (range 1-28days): 60% (9/15) received eptacog alfa, one was treated with activated prothrombin complex concentrate,while both drugs were administered in combination in three of them (20%). The immunosuppressive treatmentwas steroids alone in nine patients, 27% (4/15) received azathioprine or cyclophosphamide in combination withprednisone, while in two patients rituximab immunotherapy was used following immunosuppressivecombination therapy... Major bleeding was present in two-thirds of postpartum AHA patients and was successfully treated withbypassing agents. Postpartum AHA has been reported to have a high rate of spontaneous remission. However,nearly half of the population had inhibitor eradication more than one month after disease onset, usingimmunosuppressive treatment for more than 2 months, and additional drugs were used in 40% of them,evidencing difficulties in treatment remission in..."

Retrospective data • Gastroenterology • Gastrointestinal Disorder • Gynecology • Hematological Disorders • Hemophilia • Immunology • Rare Diseases

MANAGEMENT OF ACQUIRED SEVERE HEMOPHILIA ACCORDING TO THE LATEST INTERNATIONAL GUIDELINES AND THE INFECTIOUS AND INFLAMMATORY COMPLICATIONS DERIVED. (EHA 2024) - "It isstarted at this time with the diagnosis of Severe Acquired Hemophilia A, eptacog alfa 5 mg/8 hours,methylprednisolone 100 mg/24 hours and, on October 3, 2023, cyclophosphamide 2 mg/Kg is started. Early diagnosis of acquired hemophilia is of vital importance to provide timely treatment in order to avoidbleeding with serious complications. The availability of the new guidelines that support the first-line use of corticosteroids and immunosuppressantstogether with Rituximab when it is a severe case, represents an important advance in terms of the resolution ofthe condition, but may condition the appearance of a greater number of infectious complications. Infectious complications are a cause of important morbidity and mortality in patients with acquired HemophiliaA under immunosuppressive treatment."

Cytomegalovirus Infection • Dermatology • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Immunology • Infectious Disease • Mood Disorders • Musculoskeletal Pain • Pain • Psoriasis • Rare Diseases • Rheumatology

A survey of clinical practice of laboratory monitoring and management of bleeding in children on emicizumab prophylaxis in the PedNet centers (ISTH 2024) - "In children with previous inhibitors on emicizumab and major bleed 12 treat with Novoseven, 11 with FVIII concentrate and 6 based on clinical situation. 30/32 centers responded, 25 had used emicizumab > 4 , 2 centers 3-4 ,3 centers 2-3 years. Emicizumab concentrations are monitored by19/30 (63%) centers: 9 every 3-6 months, 4 annually, 1 “sometimes”, 3 more in the beginning, 8 -if clinical concerns. FVIII inhibitor testing is available in all centers."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

An Open-label,Dose-Escalation,Multicenter Phase I Study to Evaluate the Safety,Immunogenicity,and Pharmacokinetics/pharmacodynamics（PK/PD）of Single Dose SS109 in Hemophilia A/B patients with Inhibitor (ISTH 2024) - "Nonclinical studies showed that the hemostasis of SS109 is better than that of NovoSeven® in same dose, and half-life is 2.5 times longer than that of NovoSeven® in cynomolgus monkeys... Single dose of SS109 at all 5 doses was well-tolerated. Two adverse events occurred in 2 patients (7.4%) were possibly related to SS109. No hypersensitivity or allergic reactions occurred."

Clinical • P1 data • PK/PD data • Hematological Disorders • Hemophilia • Immunology • Rare Diseases

Evaluation of Coagulation Factor VII Biosimilars (AryoSeven Vs NovoSeven) in Modulating Prothrombin Time, activated Partial Thromboplastin Time, and levels of Factors VII and VIII in Hemophilia A (ISTH 2024) - "In a comparison of both of the investigational drugs to the original values of PT, aPTT, FVII, and FVIII, among-group comparison with post-hoc pairwise comparisons showed a high statistically significant difference in values of all four parameters, compared to pre-treatment values (Table 1). No difference was observed between the values of all four investigated parameters after adding AryoSeven and NovoSeven. A negative correlation was observed between aPTT and FVIII values after adding AryoSeven (r=-0.647, p< 0.001)."

Hematological Disorders • Hemophilia • Rare Diseases

Acquired Hemophilia A: A Rare Cause of Uncontrolled Hemorrhage in the Intensive Care Unit (ATS 2024) - "Prednisone was initiated...Treatment of active bleeding involves therapies that bypass FVIII in the coagulation cascade, such as recombinant factor VIIa (NovoSeven), activated prothrombin complex concentrate (FEIBA), or emicizumab, a recombinant monoclonal antibody that mimics FVIII activity...First-line therapy includes corticosteroids, and in non-steroid-responsive patients, cyclophosphamide can be added. Second-line treatment includes rituximab, plasmapheresis, or mycophenolate mofetil. Although rare, a high index of suspicion for AHA should remain for actively bleeding patients with a prolonged PTT, especially in elderly patients without family history of bleeding diathesis. Early recognition of AHA allows for consultation with a hematologist and prompt initiation of treatment to restore hemostasis and prevent the morbid consequences of this condition."

Alzheimer's Disease • CNS Disorders • Critical care • Dementia • Hematological Disorders • Hemophilia • Mood Disorders • Musculoskeletal Pain • Pain • Rare Diseases

Plastic Surgery Gone Wrong: Catastrophic Post-operative Hemorrhagic Shock Due to Newly Acquired Factor VIII Inhibitor (ATS 2024) - "The patient received over 35 units of packed red blood cells along with fresh frozen plasma and cryoprecipitate and was started on high dose prednisone to counteract the inhibitor. The bleeding persisted through several trials of immunosuppressive and immunomodulatory therapies including intravenous immunoglobulin, rituximab, and cyclophosphamide. She also received factor VII product (NovoSeven) repletion to bypass the inhibited coagulation pathway to attempt to synthesize adequate coagulation factors...Her hospital course was further complicated by the development of vancomycin-resistant enterococcus (VRE) bacteremia and Acinetobacter pneumonia...In our case, the factor inhibitor level responded only minimally despite all immune therapies. Early detection of this phenomenon in instances of unexplained hemorrhagic shock and ideally prior to surgery is necessary to rapidly suppress inhibitor levels before the development of catastrophic sequelae, as seen in our patient."

Surgery • Aesthetic Medicine • Cardiovascular • Diabetes • Endocrine Disorders • Hematological Disorders • Hemophilia • Hypertension • Immunology • Infectious Disease • Metabolic Disorders • Pneumonia • Rare Diseases • Respiratory Diseases • Type 2 Diabetes Mellitus

SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Jiangsu Gensciences lnc. | Trial primary completion date: Jan 2024 ➔ May 2024

Trial primary completion date • Hematological Disorders • Hemophilia • Rare Diseases

Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective. (PubMed, Haemophilia) - "In this review, we summarise current data regarding the mode of action, clinical efficacy and safety of eptacog beta for the management of haemophilia A and B in patients with inhibitors from a European perspective. In addition to providing a valuable summary of the analyses of the clinical data for eptacog beta conducted for the EMA, our review summarises the potential differentiators for eptacog beta compared with other current bypassing agents."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

Inpatient NovoSeven Utilization and Cost Review at an Academic Medical Center (SCCM 2024) - "NovoSeven is a high-cost medication that may be necessary to achieve hemostasis in urgent bleeding situations in patients with clotting disorders. Overall utilization is low, but there are prolonged times to first dose administration and there is significant cost from medication. Future involvement of pharmacists at the bedside upon order entry has potential to reduce waste and decrease time to therapy."

Clinical • Review • Critical care • Hematological Disorders • Hemophilia • Rare Diseases

COMPARING THE EFFECTIVENESS OF CONCENTRATE OF ALL PROTHROMBIN COMPLEX FACTORS WITH RECOMBINANT HUMAN COAGULATION FACTOR VIIa IN THE TREATMENT OF BLEEDING AFTER CARDIAC SURGERY. (PubMed, Pol Merkur Lekarski) - " Treatment with recombinant human coagulation factor VIIa and concentrate of all prothrombin complex factors is effective and safe for cardiac surgery patients."

Journal • Surgery • Anesthesia • Cardiovascular • Hematological Disorders

Transplacental Transfer of Emicizumab: Experience with Emicizumab in a Pregnant Female with Severe Hemophilia Α and an Inhibitor (ASH 2023) - "She was not treated with immune tolerance induction and had remained on factor eight inhibitor bypassing activity (FEIBA) as prophylaxis until transferring to our center. She was transitioned to monthly emi prophylaxis and recombinant FVIIa (rFVIIa, NovoSeven) for breakthrough bleeding at age 26...Her labor was induced with oxytocin while on rFVIIa and tranexamic acid (TXA) support... In this highly unusual case, we demonstrate that emi crossed the placenta and is excreted in breast milk. We also demonstrate that the maternal FVIII inhibitor did not cross the placenta into the newborn. Drawing conclusions on the safety of emi despite placental and breast milk transmission and transmissibility of inhibitor based on a single case would be premature."

Hematological Disorders • Hemophilia • Postpartum Hemorrhage • Rare Diseases

A Single Center Experience of 13 Episodes of Acquired Hemophilia A (2019 - 2023) (ASH 2023) - "The use of aPCC (FEIBA) instead of rFVIIa (NovoSeven) as the first-line BPA led to total estimated cost avoidance of $13. We describe 13 episodes of AHA successfully treated at a single academic medical center between 2019 and 2023. aPCC was the first-line BPA administered in all patients, with significant cost savings relative to rFVIIa. Immunosuppression, including prednisone and either cyclophosphamide or rituximab, was used to successfully eliminate autoantibodies in all patients."

Clinical • Hematological Disorders • Hemophilia • Hepatology • Immunology • Infectious Disease • Novel Coronavirus Disease • Oncology • Pneumonia • Rare Diseases • Respiratory Diseases

Analyzing the safety and efficacy of high versus low dose recombinant activated Factor VIIa (NovoSeven®RT) for the treatment of bleeding in non-hemophiliac cardiac surgery patients. (ASHP 2023) - No abstract available

Clinical • Surgery • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases