Platelet storage pool disorders (PSPD) is classified under a disorder type characterized by ineffectual platelet functioning. Based on our previous works in the PSPD area of study, Protheragen is leading the field in developing advanced therapeutic solutions to improve PSPD management. As your trusted collaborator for drug development research for PSPD, we are the foremost providers of unmatched research support to satisfy your requirements.
Platelet storage pool disorder (PSPD) includes a collection of congenital or acquired bleeding disorders which arise due to insufficient or malfunctioning platelet granules that result in altered secretion-dependent aggregation. The condition is divided into three principal subtypes based on the impacted granules:
| Subtypes | Defective Granules | Genetic Causes | Incidence |
|---|---|---|---|
| δ-Storage Pool Deficiency (δ-SPD) | δ-granules | HPS1-11, LYST | 1-2 per 1,000,000 |
| α-Storage Pool Deficiency (α-SPD) | α-granules | NBEAL2 | <1 per 1,000,000 |
| Combined αδ-SPD | Both δ-granules and α-granules | VPS33B/VPS16B | Extremely rare |
Pathogenesis of Platelet Storage Pool Disorder (PSPD)
The development of platelet storage pool disorders (PSPD) stems from a number of genetic mutations affecting the biogenesis and trafficking of organelles granules within megakaryocytes. The causative genes for the three PSPD subtypes are listed in the table above. Defects in either δ-granules or α-granules entail specific functional deficiencies such as the signal amplification failure in δ-SPD platelets and absence of adhesive proteins in α-SPD platelets which together explain diverse bleeding phenotypes.
Fig.1 Electron microscopic morphology of dense granules in platelet storage pool disease (PSPD). (Dupuis A, et al., 2020)
| Therapy | Types | Mechanism of Action | Development Phase |
|---|---|---|---|
| Tranexamic Acid | Antifibrinolytic | Inhibits plasminogen activation, reducing fibrin clot breakdown | Clinically used |
| DDAVP | Synthetic vasopressin | Increases endothelial release of vWF and FVIII, enhancing platelet adhesion | Clinically used |
| Activated FVIIa | Recombinant coagulation factor | Bypasses platelet defects by directly activating thrombin generation on platelet surfaces | Clinically used |
| Platelet Transfusion | Cellular therapy | Provides functional platelets containing normal granules to temporarily restore hemostasis | Clinically used |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
At Protheragen, the focus of our entrepreneurs is dedicated to the development of advanced diagnostics and therapeutics for the painstakingly complex bleeding disorders, such as platelet storage pool disorders (PSPD). Our focus lies on the creation of breakthrough therapies from various molecular classes which have been carefully analyzed in sophisticated disease models during the preclinical stage.
Protheragen takes great pride in providing integrated preclinical services for platelet storage pool disorders (PSPD). These services cover all aspects of drug research including pharmacodynamics (PD), pharmacokinetics (PK), and safety. We observe the highest quality and ethics in the execution of all our research services to make certain the outcome is dependable. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)