Acquired Hemophilia A (AHA)
Acquired hemophilia A (AHA) is a rare autoimmune disease characterized by bleeding. With our pioneering efforts in AHA research, we are at the forefront of developing cutting-edge diagnostic tools and therapeutics to facilitate the effective management of AHA. As your reliable partner in AHA research, we offer unmatched support to fulfill your scientific research needs.
Introduction to AHA
Acquired hemophilia A (AHA) is a rare and potentially life-threatening bleeding disorder characterized by the presence of autoantibodies against coagulation factor VIII. Unlike congenital hemophilia, AHA is not a genetic disorder and may occur spontaneously in individuals with no previous history of bleeding disorders. AHA is extremely rare, with an incidence rate of approximately 1.5 cases/million/year.
Fig. 1 Different types of AHA and the mechanisms by which FVIII is affected. (Zimta, et al., 2021)Pathogenesis of AHA
The pathogenesis of AHA involves the development of autoantibodies against coagulation factor VIII, which is essential for normal blood clotting. The exact mechanisms leading to the loss of immune tolerance and the production of these autoantibodies are still not fully understood. However, several factors have been suggested to contribute to the pathogenesis of AHA.
- Genetic Predisposition
Research has indicated that certain gene polymorphisms, such as HLA and CTLA4, may be involved in the development of AHA. - Immune Dysregulation
Additionally, autoreactive CD4+ T lymphocytes have been implicated in the pathogenesis of this disorder.
Targets of AHA Therapy
Controlling Bleeding Episodes
Controlling bleeding episodes in AHA is an important therapeutic target. For example, by-pass agents, like activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa), promote hemostasis by bypassing the inhibitory effects of autoantibodies and activating coagulation factors involved in the coagulation cascade.
Eradicating Autoantibodies
Eradication of autoantibodies against factor VIII is a key goal in developing AHA therapies. For example, prednisolone is a corticosteroid frequently used as a first-line immunosuppressant. It helps suppress immune responses and reduce the production of autoantibodies. Prednisolone is often combined with other immunosuppressants, such as cyclophosphamide or rituximab, to enhance the therapeutic effect.
Types of AHA Therapy
- Gene therapy involves the introduction of a functional Factor VIII gene into the cells of an affected individual to restore normal production of Factor VIII and overcome the inhibitory effects of autoantibodies. Although still in the early stages of development, gene therapy shows immense potential for long-term remission in AHA individuals.
Our Services
With years of extensive involvement in rare disease research, our company boasts a highly skilled team and vast expertise. We harness state-of-the-art technology to drive the development of innovative diagnostic tools, enabling early detection of AHA. We advance therapeutic drug development by establishing animal models and conducting in-depth investigations into AHA pathogenesis and targets.
Our Research Platforms
Our Services
Animal Models of AHA
| Induced Models | |||
| Inducing the production of anti-FVIII antibodies and triggering autoimmune responses in animals are crucial methods for obtaining models of AHA. Our company utilizes FVIII-derived peptides or recombinant FVIII proteins as immunogens to stimulate immune responses against FVIII, effectively replicating the characteristics of AHA. | |||
| Genetically Engineered Models | |||
| Our company emphasizes the development of genetic engineering models for AHA. Our scientists employ cutting-edge gene editing technologies, such as CRISPR/Cas9, to specifically target AHA-associated gene deletions or introduce mutations that induce an autoimmune phenotype resembling AHA. | |||
| Optional Models |
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| Optional Species | Mice, Rats, Non-human Primates (Baboons), Others | ||
No matter what stage of research you are at, we can provide you with corresponding research services. If you are interested in our services, please contact us for more details and quotation information of related services.
References
- Zimta, Alina-Andreea, et al. "The possible non-mutational causes of FVIII deficiency: non-coding RNAs and acquired hemophilia A." Frontiers in Medicine 8 (2021): 654197.
- Tiede, Andreas, et al. "International recommendations on the diagnosis and treatment of acquired hemophilia A." haematologica 105.7 (2020): 1791.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
