Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast 2034

Key Highlights

• Paroxysmal nocturnal hemoglobinuria (PNH) is a rare non-malignant clonal hematological disorder that is characterized by a deficiency of the GPI-linked complement regulators on the membrane of hematopoietic cells, which renders them susceptible to complement-mediated damage. 
• Formerly, PNH diagnosis relied on Ham and sucrose tests, now replaced by flow cytometry (FCM) detecting GPI-anchored protein anomalies using monoclonal antibodies or FLAER. FLAER is suitable for nucleated cells but not red blood cells due to glycophorin interference. Also, the lack of international guidelines for proper screening and diagnosis leads to treatment delays.
• The main clinical conditions or diseases to consider in the differential diagnosis of PNH include Coombs-negative hemolytic anemia, venous thrombosis, anemia, and cytopenias.
• No universally accepted classification scheme is available; however, the International PNH Interest group classifies PNH into three categories: classical PNH (in which patients have clinical manifestations of hemolysis or thrombosis), PNH in the context of other primary bone marrow disorders (such as aplastic anemia or myelodysplastic syndromes); and subclinical PNH, in which patients have low proportions of PNH clones but no clinical or laboratory evidence of hemolysis or thrombosis.
• In 2024, the diagnosed prevalent cases of PNH were approximately 16,000 cases in the 7MM, which will increase by 2034. In the 7MM, the highest number of diagnosed prevalent cases of PNH were observed in the US.
• In the US, females were more affected by PNH than males, in 2024.
• Among EU4 and the UK, in 2024, the UK accounted for the highest number of PNH cases, followed by Germany, whereas Italy accounted for the lowest number of diagnosed prevalent cases.
• In 2024, the US recorded a higher prevalence of PNH cases in the 35-54 age group, with around 3,000 cases, followed by the 55-74 age group, which accounted for nearly 2,200 cases.

DelveInsight’s “Paroxysmal nocturnal hemoglobinuria (PNH)” report delivers an in-depth understanding of Paroxysmal nocturnal hemoglobinuria (PNH), historical and forecasted epidemiology in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The table given below further depicts the key segments provided in the report:

Paroxysmal nocturnal hemoglobinuria (PNH): Disease Understanding 

Paroxysmal nocturnal hemoglobinuria (PNH) Overview, and Diagnosis

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.

No universally accepted classification scheme is available; however, the International PNH Interest Group classifies PNH into three categories: classical PNH (in which patients have clinical manifestations of hemolysis or thrombosis), PNH in the context of other primary bone marrow disorders (such as aplastic anemia or myelodysplastic syndromes); and subclinical PNH, in which patients have low proportions of PNH clones but no clinical or laboratory evidence of hemolysis or thrombosis. 

PNH diagnosis was formerly comforted by in vitro complement activation by either acidity (Ham test) or osmolarity (sucrose test). These tests are obsolete as diagnosing PNH by flow cytometry (FCM) refers to detecting a pathognomonic anomaly. GPI-anchored proteins can be detected after labeling the cells with monoclonal antibodies (for example, anti-CD55 or anti-CD59) or a reagent known as fluorescein-tagged proaerolysin (FLAER), which binds to the glycan portion of the GPI anchor. FLAER is best used on nucleated cells; it does not stain red blood cells, as red blood cells express high glycophorin levels, a protein that binds to aerolysin and interferes with the assay.

Paroxysmal nocturnal hemoglobinuria (PNH) Epidemiology

The PNH epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total diagnosed prevalent cases, gender-specific cases, age-specific cases, and total treated cases in the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2021 to 2034.

• In 2024, the United States accounted for the highest number of diagnosed prevalent cases of PNH with around 8,000 cases among the 7MM.
• Among the EU4 and the UK, the UK accounted for the highest number of diagnosed prevalent cases of PNH which were around 2,600 cases in 2024.
• In the 7MM, Females accounted for more cases than males around 8,500 cases in 2024.
• In 2024, EU4 and the UK recorded a higher prevalence of PNH cases in the 35-54 age group, with 2,000 cases, followed by the 55-74 age group, which accounted for 1,500 cases. 
• In 2024, Japan accounted for approximately 2,500 diagnosed prevalent cases of PNH.

Stay ahead with in-depth analysis of the Paroxysmal Nocturnal Haemoglobinuria Market Foreacst discover emerging therapies, epidemiology data and growth opportunities.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of Paroxysmal nocturnal hemoglobinuria (PNH), explaining its causes, signs and symptoms, pathogenesis, and currently available diagnosis practices.
• Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression have been provided.
• A detailed review of current challenges in establishing diagnosis and diagnosis rate is provided.

Paroxysmal nocturnal hemoglobinuria (PNH) Report Insights

• Patient Population
• Country-wise Epidemiology Distribution

Paroxysmal nocturnal hemoglobinuria (PNH) Report Key Strengths

• Ten-year Forecast
• The 7MM Coverage 
• Paroxysmal nocturnal hemoglobinuria (PNH) Epidemiology Segmentation

Paroxysmal nocturnal hemoglobinuria (PNH) Report Assessment

• Epidemiology Segmentation
• Current Diagnostic Practices

FAQs

Epidemiology Insights

• What are the disease risks, burdens, and unmet needs of Paroxysmal nocturnal hemoglobinuria (PNH)? What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Paroxysmal nocturnal hemoglobinuria (PNH)?
• What is the historical and forecasted Paroxysmal nocturnal hemoglobinuria (PNH) patient pool in the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan?
• What is the diagnostic pattern of Paroxysmal nocturnal hemoglobinuria (PNH)?
• Which clinical factors will affect Paroxysmal nocturnal hemoglobinuria (PNH)?
• Which factors will affect the increase in the diagnosis of Paroxysmal nocturnal hemoglobinuria (PNH)?

Reasons to buy

• Insights on disease burden, details regarding diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the change in Paroxysmal nocturnal hemoglobinuria (PNH) cases in varying geographies over the coming years.
• A detailed overview of Diagnosed prevalent cases of Paroxysmal nocturnal hemoglobinuria (PNH), gender-specific cases, age-specific cases and total treated cases of Paroxysmal nocturnal hemoglobinuria (PNH) is included.
• To understand the perspective of key opinion leaders around the current challenges with establishing the diagnosis.
• Detailed insights on various factors hampering disease diagnosis and other existing diagnostic challenges.