Paroxysmal Nocturnal Hemoglobinuria (PNH) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033


 Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal stem cell disorder that develops later in life and is characterized by complement-triggered hemolysis, heightened clotting tendencies, and bone marrow dysfunction, potentially stemming from an autoimmune origin. PNH patients have a heightened risk of thrombosis within their abdominal veins, a risk rarely paralleled by other conditions. The increased propensity for blood clot formation can be attributed to several factors:

1.       Altered Fibrinolysis: Abnormal post-translational modification of the GPI-linked uPAR receptor leads to reduced fibrinolysis.

2.       Platelet Activation: There is an upsurge in complement-mediated activation of CD59-negative platelets.

3.       Thrombin Generation: Platelet-derived microparticles contribute to heightened thrombin generation.

4.       Tissue Factor Inhibition: Abnormalities in the GPI-linked tissue factor pathway inhibitor also contribute to hypercoagulability.

5.       Endothelial Aberrations: The presence of a large PNH clone can lead to endothelial cell abnormalities.

The clinical manifestations of PNH primarily result from intravascular hemolysis, unusual venous clot formation, and bone marrow underactivity. Intravascular hemolysis occurs because the abnormal red blood cells lack proteins that normally regulate complement activation, such as the accelerating factor (DAF, CD55) and CD59 (membrane inhibitor of reactive lysis). Around 70% of PNH patients experience granulocytopenia and thrombocytopenia, primarily due to reduced production of these cells in the bone marrow. This occurs even in the absence of spleen enlargement. Notably, 5 to 30% of individuals recovering from aplastic anemia show blood cell characteristics indicative of PNH.

·       PNH is a rare disorder with a minimum prevalence of 1-1.5 cases per 1000,000 population.

 

Thelansis’s “Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Paroxysmal Nocturnal Hemoglobinuria (PNH) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

 

KOLs insights of Paroxysmal Nocturnal Hemoglobinuria (PNH) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.

Read more: Paroxysmal Nocturnal Hemoglobinuria (PNH) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Tags: Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Nocturnal Hemoglobinuria (PNH) market outlook, Paroxysmal Nocturnal Hemoglobinuria (PNH) competitive landscape, Paroxysmal Nocturnal Hemoglobinuria (PNH) market forecast, Thelansis, Primary market research, KOL insights, Competitive Intelligence (CI)    

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