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Factor VIII (Factor VIII, Ipsen / Hyate C / Hyate)

✓ Approved

Sanofi S.A · F8 · 细胞治疗

什么是 Factor VIII？

Factor VIII 是一种细胞治疗，由Sanofi S.A研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)。

药物档案

商品名Factor VIII, Ipsen, Hyate C, Hyate
公司Sanofi S.A
药物类别细胞治疗
分子靶点F8, VWF
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, Sanofi S.A

作用机制

分子靶点

Factor VIII 作用于 2 个分子靶点：

F8coagulation factor VIII (AHF, FVIII)
VWFvon Willebrand factor (VWD, F8VWF)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

Factor VIII 针对 1 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersFactor VIII deficiency✓ Approved

相关研究文献

PubMedJournal of clinical medicine2026-10-27

Managing Pediatric Acquired Hemophilia A When Guideline-Recommended Bypassing Agents Are Unavailable: A Case Report.

Zhang Li L, Guo Xiao-Li XL, Xiao Jian-Wen JW, Guo Yu-Xia YX

Background/Objectives: Acquired hemophilia A (AHA) is caused by neutralizing factor VIII (FVIII) autoantibodies and is exceptionally rare in children. Pediatric treatment evidence is limited, and recommended hemostatic agents may be inaccessible. Case: An 11-year-old previously healthy girl presented with soft-tissue, mucosal, and genitourinary bleeding, an isolated markedly prolonged activated partial thromboplastin time, FVIII activity of approximately 1.4-2%, and an FVIII inhibitor. Recombinant activated factor VII was financially inaccessible to the family, and activated prothrombin complex concentrate (APCC) was unavailable. After a multidisciplinary discussion with the family, non-activated four-factor prothrombin complex concentrate (4F-PCC) was administered off-label; corticosteroid-based treatment was initiated to eradicate the inhibitor. Results: Gross hematuria resolved during the treatment period, and microscopic hematuria cleared subsequently. By July 2025, FVIII activity was 125%, the inhibitor was undetectable, immunosuppression had been withdrawn, and no recurrent major AHA-related bleeding had been reported. Conclusions: This case illustrates individualized decision-making in pediatric AHA when recommended hemostatic agents cannot be implemented. The temporal association between 4F-PCC administration and bleeding resolution does not establish efficacy, safety, or equivalence to APCC.

PMID 42941373
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PubMedJournal of clinical medicine2026-10-27

Viscoelastic Testing for Hemostatic Assessment in Liver Transplantation.

Bashir Asad H AH, Giordano Chris C, Brennan Meghan M

Background/Objectives: End-stage liver disease leads to a unique coagulation profile. This rebalanced coagulation status is characterized by the propensity to bleed easily and form clots in blood vessels. Managing these patients requires close monitoring and an understanding of the underlying pathophysiology. Decreased production of coagulation factors by the liver and splenic sequestration of platelets lead to conditions favoring a hypocoagulable picture. Increased levels of factor VIII and von Willebrand factor along with decreased production of anticlotting factors lead to an environment favoring thrombosis. Viscoelastic testing allows clinicians to navigate this challenging coagulation scenario and preserve the delicate balance. Methods: We review recent technological advancements in coagulation monitoring during liver transplantation. Viscoelastic testing has been used and validated in liver transplantation initially using mechanical methods for assessing clot formation and strength, but newer viscoelastic testing platforms are transitioning toward resonance-based technologies and cartridge-based point-of-care devices that allow for faster assessment of coagulopathies. Development of faster point-of-care devices is beneficial for liver transplantation given acute coagulopathies that can occur throughout phases of the case. Results/Conclusions: This paper will discuss briefly transfusion thresholds; how the SEER-based Quantra, resonance-based TEG 6s, and automated cartridge-based ROTEM SIGMA work; gaps using conventional coagulation testing; and the integration of these systems into perioperative decision making to support goal-directed transfusion strategies.

PMID 42941387
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PubMedJournal of veterinary diagnostic investigation : official publication of the American Association of Veterinary Laboratory Diagnosticians, Inc2026-10-27

Presumed myofibroblastic hepatic sarcoma associated with a retained surgical swab in a 3.5-year-old dog.

Horváth Dávid G DG, Tráj Patrik P, Virág Zsolt Z, Márialigeti Márton M et al.

A 3.5-y-old spayed female Labrador Retriever was presented with inappetence, weakness, vomiting, and a tense abdomen. Via abdominal ultrasonography, radiography, and diagnostic laparotomy, we identified a hepatic mass. Thereafter, we performed a lobectomy and sent the resected specimen for histologic examination. The dog was euthanized given the lack of clinical improvement and the formation of an intrathoracic soft-tissue mass identified with radiography. Histologically, the thoracic mass was an infiltrative, highly cellular mesenchymal neoplasm. Its spindle cells were arranged in interwoven fascicles and bundles with marked anisocytosis, anisokaryosis, and nuclear atypia, and a high mitotic count. The tumor tissue surrounded large necrotic areas with numerous cotton fibers originating from a retained surgical swab, which had elicited a marked granulomatous inflammatory response. The neoplastic cells uniformly expressed strong cytoplasmic vimentin immunolabeling. Approximately 50% of the neoplastic cells had strong cytoplasmic "tram-track" α-SMA and moderate cytoplasmic calponin immunolabeling. Tumor cells did not have desmin, S100, CD31, factor VIII-related antigen (von Willebrand factor), HepPar1, or IBA1 immunolabeling. The microscopic and immunohistochemical features were consistent with a soft-tissue sarcoma, most likely a myofibroblastic sarcoma. Hepatic myofibroblastic sarcoma related to retained surgical swab has not been reported previously in dogs, to our knowledge, nor has the use of HepPar1 immunohistochemistry to investigate the possible hepatocellular origin of the tumor.

PMID 42934880
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PubMedBlood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis2026-10-27

Clinical burden of haemophilia in Abu Dhabi, United Arab Emirates: real-world insights from the Malaffi platform.

Alsafi Muna M, Aldhaheri Alia A, Ganesan Subhashini S, Cantarutti Flavia Martinez FM et al.

This study aims to understand the clinical burden of haemophilia by assessing the real-world patient profile, treatment, and healthcare utilization of people with haemophilia (PwH) in Abu Dhabi. A retrospective observational study was conducted using electronic health records from the Malaffi database (patient health information platform). Patients with a diagnosis of haemophilia A (ICD-10 D66) or haemophilia B (ICD-10 D67) between 1 January 2021, and 14 April 2023, and with documented haemophilia-related treatment, were included. Demographic characteristics, clinical presentation, comorbidities, treatment patterns, and healthcare encounters were analysed using descriptive statistics. A total of 146 PwH were identified, 83% (n = 121) presented with haemophilia A. Paediatric patients comprised 65% (n = 95) with mean age 6.3 ± 5.6 years and adults 35% (n = 51) with average age of 30.4 ± 10.1 years. Pain and swelling were the most common symptoms documented in both groups (77% paediatric; 88% adult). The most frequently identified comorbid conditions include joint/arthritic conditions among adults (75%) and anaemia among the paediatric patients (61%). PwH recorded 6052 healthcare encounters during the study period with a median of 10 visits per PwH per year. The ordered medications included 1422 prescriptions, and factor VIII (862 prescriptions) was the most prescribed medication. The study provides robust evidence on the clinical burden and intense utilization of healthcare resource of PwH in Abu Dhabi and offer foundation for future research and policy initiatives aimed at optimizing care delivery and resource allocation for PwH.

PMID 42937007
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PubMedBiomolecules2026-10-27

Stromal Vascular Cell-Mediated Inflammatory and Remodeling Pathways in Alcohol-Induced Visceral Adipose Tissue Dysfunction: A Pilot Study.

Ai Weilun W, John Kathiravan Arul Daniel AD, Casey Carol A CA, Mahalingam Sundararajan S et al.

Chronic alcohol use induces adipocyte lipolysis and production of inflammatory mediators from adipose tissue (AT). Stromal vascular cells (SVCs) are a heterogeneous group of AT cells and contribute to AT inflammation and remodeling in metabolic syndrome. However, the role of SVCs in promoting AT dysfunction upon alcohol exposure remains unclear. Male rats were fed a Lieber-DeCarli diet containing 6.7% (v/v) ethanol (ET) or an isocaloric control diet (CON) for six weeks. Primary rat SVCs were isolated from visceral AT (VAT) of CON and ET rats. RNA sequencing analysis was performed to determine the overall changes in the gene-expression profile. Real-time PCR, Western blotting, and histological analyses were performed to validate the transcriptomics results. Chronic alcohol feeding decreased the adipocyte size and increased the formation of crown-like structures in the rat VAT. RNA-seq analysis revealed that signaling pathways related to cellular response to interleukin 1 (IL1) and collagen-containing extracellular matrix were significantly altered upon ethanol exposure in SVCs. Along these lines, ethanol exposure increased the protein levels of purinergic receptor P2Y2 (P2RY2) and IL1β in the SVCs of rat VAT. Furthermore, ethanol increased collagen deposition in both interstitial and perivascular regions, which was associated with an increase in the level of phosphorylated SMAD2/3 and collagen type VIII alpha 1 chain expression. These findings suggest that chronic ethanol exposure is associated with inflammatory and remodeling-related alterations in VAT and the total VAT-derived SVC fraction. This exploratory pilot study identifies P2RY2-associated inflammatory markers and TGFβ-related remodeling markers as candidates for future mechanistic investigation.

PMID 42939694
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PubMedJournal of clinical medicine2026-10-27

Examining the Associations Between Menstrual Disorders and SARS-CoV-2 Exposure in Greek Adolescents: A Retrospective Cohort Study.

Svigkou Asimenia A, Stefanaki Charikleia C, Kokkou Eleni E, Kalogirou Dimitrios D et al.

Background: Exposure to severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), whether via infection or vaccination, seems to influence the character of the menstrual cycle by disrupting hormonal and immune function. Methods: The main aim of this cohort study was to explore the association between exposure to SARS-CoV-2-through either vaccination or infection-and menstrual disorders in adolescent females. We also aimed at identifying the most prevalent menstrual disorders observed and assessing potential differences between the exposure groups that may contribute to these conditions. Results: This study included data from adolescent females who visited the Center for Adolescent Medicine and UNESCO Chair in Adolescent Health Care due to various menstrual disorders. Data from 92 adolescents, 77 participants exposed to SARS-CoV-2 and 15 controls, were retrieved. Significant associations were found between exposure to SARS-CoV-2 and abnormally frequent uterine bleeding (p = 0.032) and abnormal uterine bleeding (p = 0.025). Adolescents with a past confirmed case of coronavirus disease 2019 (COVID-19) demonstrated significantly lower serum concentrations of fibrinogen (p = 0.023) and iron (p = 0.03), as well as higher γ-GT concentrations (p = 0.028), than those who were non-infected, whereas vaccinated adolescents presented with significantly lower concentrations of coagulation factors VIII (p = 0.002), vWFAg (p = 0.023), and Rcof (p = 0.007) than those of their unvaccinated peers. Overall, adolescents exposed [infection (or/and) vaccination] to SARS-CoV-2 demonstrated increased estradiol (E2) concentrations compared to those of the controls. In conclusion, adolescent exposure to SARS-CoV-2 was strongly linked to abnormal uterine bleeding and polymenorrhea, potentially due to decreased coagulation factors or increased E2 concentrations. Conclusions: Further research involving larger samples and a more sophisticated design is needed to validate these findings.

PMID 42941546
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