Paroxysmal Nocturnal Hemoglobinuria - Epidemiology Forecast - 2032

DelveInsight's 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview - Epidemiology Forecast-2032' report delivers an in-depth understanding of the PNH historical and forecasted epidemiology as well as the PNH epidemiology trends in the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan.

Paroxysmal Nocturnal Hemoglobinuria (PNH) Understanding

PNH Overview

PNH Understanding

PNH Overview

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of the pluripotent hematopoietic stem cell; therefore, it can affect erythrocytes, leukocytes, thrombocytes, and probably some endothelial cells. These hematopoietic stem cells have acquired a somatic mutation in an X-linked gene: the phosphatidylinositol glycan class A (PIG-A). This gene synthesizes the glycosylphosphatidylinositol (GPI) anchor, which is necessary to attach some proteins to the cell membrane.

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Clinical Forms of PNH

A clinical polymorphism of PNH has been described. Three forms are identified: classical PNH, PNH associated with aplastic anemia (AA), and subclinical PNH; however, it is important to understand that these entities are three clinical manifestations of a single disease.

Classical form

The classical form of PNH affects young people preferentially. They suffer from chronic intravascular hemolytic anemia due to a continuous state of complement activation, but brisk periods of hemolysis may result from complement activation due to infection, surgery, strenuous activity, and alcohol intake.in adults. The risk of bleeding correlates to the severity of thrombocytopenia. Patients may present without symptoms, minimal bleeding, or serious hemorrhage (e.g., mucosal, intracranial, gastrointestinal, genitourinary). Older patients, patients on antiplatelet therapy, and patients with comorbid conditions may have severe bleeding manifestations.

PNH associated with aplastic anemia

When PNH is associated with AA, most patients express only a small PNH clone (<10%). Physicians could find clinical and/or biological evidence of hemolysis associated with two or three cytopenias (Hb <10 g/dL, neutrophils <1,000/μL, platelets <80,000/μL). In general, bone marrow failure dominates the clinical picture.

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PNH Epidemiology

The epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Diagnosed Prevalence of PNH, Gender-specific Prevalence of PNH scenario in the 7MM covering the United States, the EU4 countries (Germany, France, Italy, and Spain), the United Kingdom and Japan from 2019 to 2032.

PNH Detailed Epidemiology Segmentation

Key Findings

• In 2021, the total cases of PNH in the 7MM countries were 12,358 and these cases are anticipated to increase during the study period. The US accounted for 6,043 cases of PNH in 2021.
• Among the EU4 countries, Germany had the highest number of cases of PNH in 2021, i.e., 1,355 cases, followed by the France which had 851 cases in 2021. On the other hand, Italy had the lowest number of cases of PNH, i.e. 250 cases in 2021.
• The United Kingdom accounted for 2,569 cases of PNH in 2021.
• In case of gender-specific prevalence of PNH, in 2021 females accounted for 3,148 cases as compared to 2,894 cases in males in the United States.

Scope of the Report:

• The report covers a descriptive overview of PNH, explaining its procedure, types, indications, and currently available therapies.
• The report provides insight into the 7MM historical and forecasted patient pool covering the United States, the EU4 countries (Germany, France, Italy, and Spain), the UK and Japan.
• The report assesses the PNH risk and burden.
• The report provides the segmentation of the epidemiology for 7MM by segmented by Total Diagnosed Prevalence of PNH, Gender-specific Prevalence of PNH

Report Highlights:

• 11-Year Forecast of PNH
• 7MM Coverage
• Total Cases of PNH Molecular Alteration- Total Diagnosed Prevalence of PNH
• Total Cases of PNH by Indication

Key Questions Answered

• What are the risk, burdens, and unmet needs of PNH?
• What is the historical PNH patient pool in the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan?
• What would be the forecasted patient pool of PNH at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to PNH?
• Out of the countries mentioned above, which country would have the highest cases of PNH during the forecast period (2022-2032)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2022-2032)?

Reasons to buy:

The PNH report will allow the user to -

• Develop business strategies by understanding the trends, shaping and driving the 7MM PNH epidemiology.
• The PNH epidemiology report and model were written and developed by Masters and PhD level epidemiologists.
• The PNH epidemiology model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based on transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over the 11-year forecast period using reputable sources.

Geographies Covered:

• The United States
• The EU4 (Germany, France, Italy, Spain) and the United Kingdom
• Japan

Study Period: 2019-2032.