Acquired Aplastic Anemia

Acquired Aplastic Anemia

Acquired aplastic anemia (AAA) is a rare and life-threatening hematological disorder characterized by bone marrow failure, resulting in pancytopenia. Our company, a leading provider of autoimmune diseases and inflammation research services, is dedicated to advancing the understanding and therapy development of acquired aplastic anemia.

Introduction to Acquired Aplastic Anemia

Acquired aplastic anemia is a rare hematological disorder characterized by a reduction in the production of all three blood cell types - red blood cells, white blood cells, and platelets. This bone marrow failure leads to pancytopenia, resulting in symptoms such as fatigue, increased susceptibility to infections, and bleeding tendencies. The pathogenesis of acquired aplastic anemia involves a complex interplay between immune dysregulation, genetic factors, and environmental triggers. Immune dysregulation plays a crucial role in the destruction of hematopoietic stem cells, mediated by activated T cells and pro-inflammatory cytokines. This immune attack leads to the impairment of hematopoiesis and the subsequent development of pancytopenia.

Current therapeutic strategies for children with acquired aplastic anemia.Fig.1 Therapeutic strategies for acquired aplastic anemia in children. (Yoshida, N., 2024)

Drug Discovery and Development for Acquired Aplastic Anemia

The therapeutics of acquired aplastic anemia aim to restore normal hematopoiesis and improve blood cell counts. The initial approach involves immunosuppressive therapy (IST), which typically includes a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). In recent years, alternative therapies have also shown promise in the therapeutics of acquired aplastic anemia. A monoclonal antibody targeting CD52 has demonstrated efficacy as a monotherapy, particularly for older patients or those who experienced significant toxicities with CsA. Additionally, eltrombopag, a thrombopoietin mimetic, has shown potential in stimulating platelet production and improving blood cell counts.

With a diverse therapy development platform, our company can meet all of your acquired aplastic anemia therapy research needs. If you want to know more, please click on the link below.

Our Services

At our company, we are committed to advancing the field of acquired aplastic anemia through our comprehensive diagnostics and therapy development services. We utilize advanced cell culture techniques, animal models, and cutting-edge imaging technologies to generate robust data that informs decision-making in the drug development process.

  • Cyclophosphamide and Cyclosporine-Induced Model Development
    One of the key animal models developed by our company for acquired aplastic anemia research involves the intraperitoneal injection of cyclophosphamide and cyclosporine in mice. Mice are injected with cyclophosphamide and cyclosporine intraperitoneally to induce bone marrow failure, pancytopenia, and immune dysregulation, which are characteristic features of acquired aplastic anemia.
  • Xenograft Model Development
    Our company excels in developing and utilizing xenograft models for acquired aplastic anemia research. By transplanting patient-derived hematopoietic stem cells into immunodeficient mice, researchers can assess the engraftment and differentiation capacity of these cells and evaluate the efficacy of various therapeutic approaches.
  • Acquired Aplastic Anemia iPSC Model Development
    We employ state-of-the-art reprogramming techniques to derive iPSCs from unaffected individuals as well as patients diagnosed with acquired aplastic anemia.

Telomere length maintenance is crucial for the function and stability of hematopoietic stem cells. In Acquired Aplastic Anemia, abnormalities in telomere length dynamics have been observed. Our iPSC models accurately recapitulate this feature, as iPSCs derived from AAA patients consistently show telomere shortening during reprogramming and hematopoietic differentiation.

Our Advantages

Time-saving services with high efficiency

Fast and cost-efficient workflow

Timely project reporting and after-sales service

Professional and experienced multidisciplinary experts

Careful design and transparent operation process

Superior data quality and fast turnaround

Our team of experienced scientists and researchers conduct in vitro and in vivo studies to assess the pharmacokinetics, pharmacodynamics, and toxicology profiles of candidate compounds. In addition to the services and models listed above, we also provide customized services and disease model development services to meet your specific needs. If you are interested in our services, please don't hesitate to contact us.

References

  1. Yoshida, N. "Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia." International Journal of Hematology 119.3 (2024): 240-247.
  2. Giudice V., and Selleri C. "Aplastic anemia: pathophysiology." Seminars in Hematology. Vol. 59. No. 1. WB Saunders, 2022.
Please note that we are a research service provider, not a pharmacy or clinic, so we are unable to see patients and do not offer diagnostic and treatment services for individuals.