Immune Thrombocytopenic Purpura (ITP)
Immune thrombocytopenic purpura (ITP) is a hematologic condition marked by a reduction in platelet count, typically falling below 100 × 10^9/L. This disorder manifests with bleeding and hemorrhages on the mucosa or skin, primarily due to thrombocytopenia. Our company is well-equipped to address your requirements for drug and therapy development in ITP therapy with a strong foothold in biotechnology, operational expertise, and industry technologies.
Introduction to ITP
ITP is an autoimmune disorder specific to organs, wherein platelets and their precursors are marked as targets by an aberrant immune system. This interaction results in a reduction in platelet count, consequently causing a bleeding disorder that may manifest with skin hemorrhages, mucous membrane bleeding, or even intracranial hemorrhagic events. The incidence of ITP varies between adults (3.3 and 3.9 per 100,000 per year) and children (1.9 and 6.4 per 100,000 per year).
Pathogenesis of ITP
The pathogenesis of ITP involves aberrant recognition of platelet autoantigens by dendritic cells (DCs), leading to activation of proinflammatory CD4+ T helper cells and subsequent differentiation of B cells into autoantibody-secreting plasma cells. These autoantibodies mediate platelet destruction via macrophage Fcγ receptors and induce platelet desialylation for clearance via hepatocyte Ashwell–Morell receptors (AMRs). Additionally, CD8+ cytotoxic T lymphocytes (CTLs) contribute to platelet lysis and apoptosis while interfering with megakaryocyte maturation, further diminishing platelet production. This complex interplay between immune dysregulation and platelet dysfunction underlies the thrombocytopenia observed in ITP.
Fig.1 Pathophysiology of ITP. Thrombocytopenia in ITP is the result of both increased platelet destruction and suppressed platelet production. (Liu, X.G., et al., 2023)Diagnostics Development of ITP
ITP remains a diagnosis of exclusion due to the lack of a "gold standard" diagnostic test.
Tab.1 Diagnostics of ITP (Liu, X.G., et al., 2023)
| Initial diagnostic | Additional tests | Differential diagnoses |
|---|---|---|
| History taking; physical examination; complete blood count; peripheral blood film analysis | reticulocyte counts; immunoglobulin levels; viral screening | myelodysplastic syndrome; secondary thrombocytopenia |
Therapy Development of ITP
Small molecule drugs target specific pathways involved in ITP pathogenesis. For instance, thrombopoietin receptor agonists such as eltrombopag and romiplostim stimulate platelet production by mimicking the action of endogenous TPO.
Cell therapies aim to modulate immune responses or restore immune tolerance. Examples include the use of regulatory T cells (Tregs) to suppress autoimmunity or the infusion of mesenchymal stromal cells (MSCs) to modulate immune cell activity and promote tissue repair.
Monoclonal antibodies target immune cells or molecules involved in platelet destruction. Rituximab, for example, targets CD20 on B cells to deplete autoreactive B cells, while anti-CD61 monoclonal antibodies aim to block platelet destruction.
Gene therapy for ITP involves modifying the individual's own cells to enhance platelet production or regulate immune responses. For example, one approach is to introduce genes encoding thrombopoietin (TPO) into hematopoietic stem cells to increase platelet production.
Our Services
Our company prioritizes a partnership-driven approach, working hand-in-hand with our clients to develop innovative ITP therapies. Our team dedicates itself to crafting personalized strategies that align with your objectives, ensuring you are supported at every step.
Platforms of ITP Therapy Development
Animal Models of ITP
We have established expertise in developing and utilizing relevant animal models that closely mimic the disease characteristics and response to therapy. These models enable us to evaluate the safety and efficacy of potential therapies.
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| Our organization specializes in delivering top-notch services to create NON-GEMs. We provide diverse model choices customized to meet specific research needs related to ITP. | ||
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| Genetically Engineered Models | ||
| Our expertise in genetic engineering techniques, such as CRISPR/Cas9 technology, allows us to generate accurate and reliable models that recapitulate the genetic alterations observed in human ITP. | ||
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| Optional Species | Mice, Rats, Others | |
In addition to these models, our comprehensive services encompass other models that target specific signaling pathways and molecular targets.
If our services intrigue you, we encourage you to contact us immediately. Let's collaborate on customizing our solutions to perfectly align with your requirements and help you achieve your desired outcomes.
References
- Liu, X.G., et al., "How we treat primary immune thrombocytopenia in adults." J Hematol Oncol (2023). 16(1): p. 4.
- Desouza, S. and Angelini, D., "Updated guidelines for immune thrombocytopenic purpura: Expanded management options." Cleve Clin J Med (2021). 88(12): p. 664-668.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
