Pure red cell aplasia (PRCA) is a rare disorder characterized by a reduction or absence of red blood cells in the bone marrow which leads to anemia. Protheragen has a capable team of researchers and scientists with profound knowledge in PRCA. They are focused on developing new therapies that disrupt the current inadequacies in the integrated treatment frameworks in PRCA.
Pure red cell aplasia (PRCA) is an uncommon hematological condition defined by the marked inhibition of the bone marrow's erythroid precursor cell lines, resulting in severe normocytic anemia (Hb<7 g/dL) alongside normal levels of leukocytes and platelets. There are two distributions observed in this condition: congenital forms which appear in infancy and acquired forms which appear in adulthood.
Fig.1 The step-by-step laboratory tests in normocytic anemia. (Halfon P, et al., 2024)
The pathogenesis of pure red cell aplasia (PRCA) involves intrinsic defects in erythroid cells, for example, ribosomal protein mutations in Diamond-Blackfan anemia trigger p53-mediated apoptosis. PRCA also involves extrinsic inhibition of erythropoiesis, for example, anti-EPO antibodies, parvovirus B19 cytotoxicity, or thymoma-derived inhibitory cytokines. All of these factors lead to the selective depletion of erythroid precursors with the survival of other hematopoietic lineages.
Fig.2 Schematic diagram of the pathogenesis of pure red cell aplasia (PRCA). (Gurnari C, Maciejewski J P., 2021)
| Drug Names | Mechanism of Action | Targets | NCT Number | Phase |
|---|---|---|---|---|
| Tacrolimus | Inhibits calcineurin-mediated T-cell activation, reducing cytotoxic T-cell attack on erythroid progenitors. | Calcineurin/NFAT pathway | NCT03540472 | Phase II |
| Sirolimus | Blocks mTOR signaling to suppress aberrant cytotoxic T-cell and NK-cell proliferation. | mTORC1 complex | NCT03364764 | Phase II |
| Bortezomib + Dexamethasone | Proteasome inhibition depletes plasma cells (anti-EPO antibody producers); dexamethasone enhances erythroblast survival. | Proteasome/glucocorticoid receptor | NCT04423367 | Phase II |
| Isatuximab | Anti-CD38 monoclonal antibody depletes pathogenic plasma cells and autoantibody-producing B-cell clones. | CD38 | NCT05559827 | Phase I/II |
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.
Recognizing the complexity of diagnosing and treating pure red cell aplasia (PRCA), Protheragen is committed to building a team of experts to provide cutting-edge diagnostic and therapeutic development solutions. Our commitment lies in providing a variety of customized therapy development services to meet the diverse research needs of our customers. We also excel in generating precise disease models that are carefully engineered to replicate the unique features of PRCA. These models are valuable tools for validating the safety and efficacy of potential therapeutics.
| Types | Description |
|---|---|
| PRCA Serum Induced Model | After normal mice were injected with PRCA serum IgG, erythropoiesis was continuously inhibited, hematocrit gradually decreased, and serum erythropoietin (Ep) levels increased inversely. This model simulates the pathophysiological pattern of type I PRCA. |
| Erythropoietin Induced Model | A rabbit injected with enhanced Ep develops an immune response leading to a decrease in bone marrow Gct values and disappearance of erythroid precursor cells. Subsequent immune attenuation is associated with increased reticulocytes and resolution of anemia, recapitulating the pathophysiological pattern of type II PRCA. |
Protheragen is steadfastly dedicated to meticulously validating and optimizing therapies for pure red cell aplasia (PRCA) through a thorough series of pharmacodynamics (PD), pharmacokinetics (PK) and toxicology studies. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)