Humate-P (human plasma derived von Willebrand factor/FVIII complex) / CSL Behring - LARVOL DELTA

Blood tells a hidden story: Von willebrand disease in the puzzle of refractory postpartum hemorrhage (ASH 2025) - "Over the subsequent four weeks, she was rehospitalized four additional times due to life-threatening hemorrhaging that necessitated several massive transfusion protocols, antifibrinolytics, iron repletion, and replacement of factor VIII with a VWF product (Humate-P)... This case highlights the diagnostic pitfalls of vWD in the postpartum period, where the biochemical derangements can be obscured physiologically and iatrogenically. Clinical history remains the most important factor. If a patient has unexplained delayed PPH and evidence of bleeding diathesis, we would recommend repeating the baseline tests for vWD."

Cardiovascular • Gynecology • Hemophilia • Obstetrics • Postpartum Hemorrhage

Purified VWF Corrects Dysfunctional Primary Hemostasis in Patients with Extracorporeal Life Support. in Vitro Studies Against Cryoprecipitate (ASH 2023) - "Platelet function was evaluated by PFA-200 ( Siemens Healthineers®) with collagen-epinephrine and collagen-ADP cartridges, and platelet aggregation with ristocetin 1 mg/mL ( Helena BioSciences APACT4 ®). The addition of Haemate-P ® in vitro significantly improved the hemostatic efficiency compared to Cp only when ECMO was discontinued. More studies are needed to evaluate the role of VWF-containing concentrates in the treatment of these patients, being Total-TAS ® a potentially useful mechanism to investigate it."

Preclinical • Cardiovascular • Hemophilia • Pulmonary Embolism • Respiratory Diseases

The Variable Heavy Chain Antibody Syn-VWFA1 Inhibits the Interaction of Platelets to Von Willebrand Factor in Solution but Not to Immobilized Von Willebrand Factor Thereby Inhibiting 3-Dimensional but Not 2-Dimensional Thrombus Formation (ASH 2023) - "Syn-VWFA1 (1.5 µM) abolished platelet binding to ristocetin-activated VWF (native and Haemate® P VWF) and R1306W VWF (VWF type 2B) in a flow-cytometric platelet function assay (Figure 1)...This implies that immobilization of VWF to collagen affects VWF conformation in such a way (1) that access of Syn-VWFA1 to its epitope in the A1 domain is hindered or (2) that conformational changes following Syn-VWFA1 binding are blocked, while allowing platelet binding. This implies that Syn-VWFA1 has potential treatment opportunities for patients with increased circulating active VWF, to prevent 3-dimensional thrombus formation (pathological platelet-VWF aggregate formation) but not 2-dimensional thrombus formation (leaving physiological hemostasis at sites of vascular injury intact)."

Hematological Disorders • Hemophilia • Thrombocytopenic Purpura • Thrombosis

Successful desensitization with FVIII/Von Willebrand Factor concentrate in Type III Von Willebrand Disease. (PubMed, Allergol Immunopathol (Madr)) - "A 40-year-old female patient with VWD Type 3 presented to our clinic with symptoms including jaw numbness and tightness, chills, fatigue, nausea, and dyspnea following administration of a FVIII/VWF concentrate (Haemate® P)...This case highlights the importance of desensitization and multidisciplinary approach in the case of drug hypersensitivity in patients with VWD Type 3. Our desensitization protocol with FVIII/VWF concentrate is highly effective and safe."

Journal • Allergy • Fatigue • Hematological Disorders • Hemophilia • Immunology • Pulmonary Disease

Ability of Coagulation Tests to Assess Hemotherapeutic Agent Response Testing (ASH 2024) - "HA agents were Humate-P, CSL Behring (CSL), King of Prussia, PA for hemophilia and VWD; HemosIL normal assayed plasma, Instrumentation Laboratory, Bedford, MA for dilution; RiaSTAP, CSL for hypofibrinogenemia; reagent grade F : VIIa, Enzyme Research Laboratories, South Bend, IN for F : VII deficiency...Optimizing doses of HA may improve MCTS performance. If MCTS can perform automated HART, treatment truly targeted to each patient's unique coagulopathy may become a reality."

Hematological Disorders • Hemophilia • Rare Diseases • Thrombosis

FVIII and VWF PK: Key Aspects in Safety and Efficiency of VWF Concentrates. Review and Reflection (ASH 2024) - P3 | "These VWFCs are of plasma origin, with one recombinant origin, rVWFC (Vonvendi®), containing only VWF. Among the plasma-derived ones, we have plasma derived Von Willebrand Factor Concentrate with FVIII (pd-VWFC) (Wilate®) with a VWF/FVIII ratio of 1 : 1, and other pd-VWFC with a low FVIII content (Wilfact®)(FVIII : VWF ratio <0.2 : 1).Methods : For this analysis, data for rVWFC comes from post hoc analysis of prospective clinical trial (CT) NCT02973087, 2 and for pd-VWFC (with FVIII), from prospective CT NCT04052698...4Regarding this risk, it could be assumed that VWFCs without FVIII would be exempt from it, but TEs were reported in scheduled surgeries with rVWFC 5 and with other VWFCs/FVIII with a lower proportion of FVIII than the pd-VWFC with FVIII analized (for example, pd-VWFC rich in VWF, Haemate P®)...5,13 With pd-VWFC (ratio VWF/FVIII 1 : 1), the obtained FVIII peak is higher, coinciding with the VWF peak, generating a higher thrombin..."

Clinical • Review • Cardiovascular • Hematological Disorders • Hemophilia • Thrombosis

Rondaptivon Pegol (BT200) to Correct Thrombocytopenia in a Peri-Interventional Setting in Type 2B Von Willebrand Disease (VWD) (ASH 2024) - "The second patient had a femoral neck fracture that required hip surgery under prophylactic substitution wirh Haemate P, which increased VWF activity to 101%. Blood. 2024 Jul 12 : blood.2024024055"

Cardiovascular • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Orthopedics • Thrombocytopenia • LRP1

Disparities in Real-World Treatment Patterns Among Patients with Von Willebrand Disease in a Large US Population-Based Dataset (ASH 2024) - "On-demand medications included antifibrinolytics and desmopressin, which were used 46% and 31% of the time, respectively, while 23% used VWF replacement therapies. Among prophylaxis users, 49% of patients were treated with VWF replacement therapies, most commonly Humate-P (77%), followed by wilate (16%), Vonvendi (11%), and Alphanate (8%)...Hemlibra was used in 2% of patients, mainly to treat those with joint bleeds...While heavy menstrual bleeding and nosebleeds were the most common bleeding phenotypes among VWD patients with bleeding comorbidities (and likely underrepresented in claims data), only a minority of patients received VWF replacement therapies. These findings suggest a high unmet need for better treatment options for VWD patients who may benefit from prophylactic therapy."

Clinical • HEOR • Real-world • Real-world evidence • Anemia • Gastrointestinal Disorder • Hematological Disorders • Hemophilia

Ulcerative Colitis in a Patient with Type II Von Willebrand Disease: A Rare Coexistence of Monogenic and Polygenic Disorders (ACG 2025) - "To our knowledge, this is the first documented case.Case Description/ We present a 28-year-old male with Type II VWD and ulcerative pancolitis, currently in deep clinical and endoscopic remission on infliximab and methotrexate...He did not respond to DDAVP and required Humate-P infusions perioperatively...Colonoscopy showed pancolitis; initial treatment with mesalamine and adalimumab provided brief relief...Prompt endoscopic evaluation and coordinated hemostatic and anti-inflammatory therapy are essential. This case highlights the complexity of managing concurrent monogenic and polygenic disorders, and the need for coordinated, multidisciplinary care in such patients."

Clinical • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Immunology • Inflammation • Inflammatory Bowel Disease • Mucositis • Ulcerative Colitis

Recurrent Gastrointestinal Bleeding in Type 2M Von Willebrand Disease: A Diagnostic and Therapeutic Challenge (ACG 2025) - "Medical therapies—Desmopressin (DDAVP), tranexamic acid, and atorvastatin—were ineffective. Between ages 69 and 71, she required 13 PRBC transfusions, 19 iron infusions, multiple doses of DDVAP, and Humate P. Ultimately, she was started on weekly rVWF which resolved her GI bleeding, her hemoglobin stabilized, and she has remained completely transfusion-independent for over 12 months...Accurate VWD subtyping is essential for optimizing treatment. To our knowledge, this is the first reported case of successful long-term rVWF use in type 2M VWD with recurrent GI bleeding, resulting in complete transfusion independence."

Anemia • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Gynecology • Hematological Disorders • Hemophilia

Glioblastoma Multiforme Resection in the Setting of Von Willebrand Disease (ASA 2025) - "He was pretreated with tranexamic acid and Antihemophilic Factor/von Willebrand Factor Complex (Humate-P®) and administered recombinant von Willebrand factor (Vonvendi®) which was continued for two weeks postoperatively. The intraoperative course was uneventful with 500mL blood loss and hemostasis was achieved. Postoperative hematological and coagulation panels were stable, and he was discharged home."

Brain Cancer • Glioblastoma • Hemophilia • Solid Tumor

Epidural Anesthetic Management of a Parturient with Von Willebrand Disease Type 1C (ASA 2025) - "Postpartum management included tranexamic acid and continued Humate-P. This case highlights the importance of meticulous hemostatic optimization and interdisciplinary collaboration in safely administering neuraxial anesthesia in vWD type 1c patients."

Anesthesia • Cardiovascular • Hemophilia • Hypertension • Postpartum Hemorrhage

Budget impact analysis of Haemate-P as long-term prophylaxis and on-demand therapy for von Willebrand disease in Spain. (PubMed, J Med Econ) - "In univariate and multiway deterministic sensitivity analyses, the alternative scenario remained cost-saving. Haemate-P was found to be a cost-saving strategy, and increased use of Haemate-P over Fanhdi, and Wilate is expected to reduce overall costs to the healthcare system in Spain."

HEOR • Journal • Hemophilia

MECHANICAL ASSISTANCE SYSTEMS: T-TAS MONITORING OF PRIMARY HEMOSTASIS AND IN VITRO CORRECTION BY VWF (ISTH 2025) - "Blood counts, ADAMTS13 activity (FRETS assay) and platelet function (PFA-200 -Siemens Healthineers®- collagen-epinephrine and collagen-ADP cartridges and ristocetin-induced aggregation -Helena BioSciences®-) was evaluated. After explantation, Haemate-P® improved OST (p=0.04) and AUC values (p=0.04) (Table 1). Table or Figure Upload"

Preclinical • Respiratory Diseases

Cost-effectiveness and cost-utility analysis of haemate-P versus other von willebrand disease treatments in Spain. (PubMed, J Med Econ) - "Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Results were generally robust to sensitivity analyses. In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies."

HEOR • Journal • Hematological Disorders • Hemophilia

Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Haemate P) in von Willebrand Disease: A Systematic Review and Pharmacovigilance Update. (PubMed, Haemophilia) - "This systematic literature review and analysis of pharmacovigilance data summarize evidence of over 40 years of clinical use of pdVWF/FVIII, supporting its safety and efficacy in VWD."

Adverse events • Journal • Hematological Disorders • Hemophilia

Thoracic Radiofrequency Ablation in a Patient With Von Willebrand's Disease: A Case Report. (PubMed, Cureus) - "An antihemophilic factor (vWF, Humate-P) injection was administered by a hematologist just before thoracic medial branch blocks...Careful planning with hematologists can enable safe and effective interventional pain procedures in patients with congenital hematologic disorders. This appears to be the first case report in world literature of a patient with Von Willebrand's disease successfully receiving thoracic radiofrequency ablations."

Journal • Hematological Disorders • Immunology • Pain • Rheumatology

Anesthetic Management of a Patient with von Willebrand Disease Type 2A Undergoing Craniotomy for Resection of Cavernous Malformation (ASA 2024) - "Preoperative optimization involved Humate-P administration, correcting her clotting factor deficiency...Despite the high bleeding risk associated with her condition, the surgery was completed successfully without significant complications. This case demonstrates the importance of a combined tailored preoperative and perioperative approach in managing patients with complex coagulopathies undergoing neurosurgery."

Clinical • Anesthesia • Hemophilia

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

Successful Management of Recurrent Ankle Hemarthrosis in a Patient with Von Willebrand Disease Type 3 using Emicizumab: A Case Report (ISTH 2024) - "Prophylactic treatments with Humate-P and Wilate had limited efficacy, leading to a high treatment burden and arthropathy. The patient's history involves a misdiagnosis of severe hemophilia A, intracranial hemorrhage, and the development of factor VIII inhibitors treated with immune tolerance therapy. Recurrent mucocutaneous bleeds, hemarthrosis, and a right ankle target joint significantly impacted the patient's quality of life. Genetic analysis confirmed a homozygous insertion mutation in the VWF gene."

Case report • Clinical • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Osteoarthritis • Rare Diseases

Personalized treatment by pharmacokinetic-guided dosing in von Willebrand disease; A prospective multicenter study, OPTI-CLOT/To WiN study (ISTH 2024) - "Aims: We aimed to investigate the reliability of PK-guided dosing of VWF/FVIII concentrates (Haemate P®) in Von Willebrand disease (VWD)... Twenty-nine VWD patients were included: 11 type 1 and 18 type 2 (Table 1). Overall, predefined targets were achieved (Figure 1,boxplot A/B). On day of medical intervention, one patient had a VWF:Act peak level below the predefined target (0.42 instead of >0.5 IU/mL)."

Clinical • PK/PD data • Hemophilia

The novel variable heavy chain antibody Syn-VWFA1 inhibits platelet von Willebrand factor interaction in solution (ISTH 2024) - "Cleavage of Haemate® P VWF (HVWF) by recombinant ADAMTS13 was measured using semi-automated electrophoresis (Hydrasys 2 instrument). Syn-VWFA1 abolished platelet binding to ristocetin-activated VWF (native and HVWF) and R1306W (VWF type 2B) (Figure 1) and inhibited ristocetin-activated VWF and R1306W VWF platelet agglutination. Syn-VWFA1 did not alter adhesion of platelets to collagen under flow or binding of GPIbα to immobilized VWF. Syn-VWFA1 reduced platelet string length and number on histamine stimulated endothelial cells and increased the cleavage of VWF by ADAMTS13."

Hematological Disorders • Hemophilia • Thrombocytopenic Purpura • Thrombosis

RECURRENT PREGNANCY LOSS IN TYPE 3 VON WILLEBRAND DISEASE WITH A UNIQUE MUTATION PROFILE (THSNA 2024) - "Due to recurrent gastrointestinal bleeding, patient was started on Humate-P prophylaxis three times per week...This has further implications for future offspring, including the likelihood of inheriting both variants and predicting associated bleeding phenotype. This highlights the utility but also potential limitations of genetic testing for potential risk assessment with family planning and during pregnancy."

Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Obstetrics • Postpartum Hemorrhage

ACQUIRED VON WILLEBRAND SYNDROME IN MONOCLONAL GAMMOPATHY OF UNDETERMINED SIGNIFICANCE: A FOCUS ON RESPONSE TO TREATMENT WITH IVIG (THSNA 2024) - "In this large single-center cohort study assessing IVIG response in AvWS-MGUS, IVIG was typically used for bleeding refractory to VWF concentrate and was associated with a good initial clinical and laboratory response."

Amyloidosis • Anesthesia • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Lymphoma • Lymphoplasmacytic Lymphoma • Monoclonal Gammopathy • Multiple Myeloma • Thrombosis • Waldenstrom Macroglobulinemia

Successful rescue immune tolerance induction with haemate-P and emicizumab in hemophilia A patient with recurrence inhibitor post Covid-19 infection (WFH 2024) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Infectious Disease • Novel Coronavirus Disease • Rare Diseases