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Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast

DelveInsight’s ‘Paroxysmal nocturnal hemoglobinuria (PNH) – Epidemiology Forecast—2030’ report delivers an in-depth understanding of the historical and forecasted epidemiology of PNH in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Geographies Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2017–2030

Paroxysmal nocturnal hemoglobinuria (PNH): Disease 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 is required to synthesize the glycosylphosphatidylinositol (GPI) anchor, which is necessary to attach some proteins to the cell membrane.


The lack of synthesis of the GPI anchor leads to the under expression of various proteins on the hematopoietic stem cell surface and on all cell lines generated by it. By this mechanism, a lack of two important complement regulatory proteins is observed on the cell surface: ‘decay‐accelerating factor’ (DAF), also called ‘CD55’ and ‘membrane inhibitor of reactive lysis’ (MIRL), also called ‘CD59’. Thus, red blood cells are more vulnerable to the action of complement. This leads to complement‐mediated intravascular hemolysis. As a result, a high concentration of free hemoglobin is found in the plasma, responsible for nitric oxide (NO) scavenging. NO depletion causes the majority of symptoms experienced by paroxysmal nocturnal hemoglobinuria (PNH) patients. Smooth muscle dystonia is responsible for dysphagia and abdominal pain. Erectile dysfunction is frequent. NO depletion may also contribute to the development of arterial constriction, leading to reduced blood flow to the kidneys (with renal failure), arterial hypertension, and pulmonary hypertension (associated with frequent but underdiagnosed pulmonary embolism).


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 the diagnosis of PNH by flow cytometry (FCM) refers to the detection of the 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, therefore, interferes with the assay.


Speaking of treatment, the only disease-modifying therapeutic strategies for PNH are complement inhibition therapy (eculizumab and ravulizumab) and bone marrow transplantation. Eculizumab and ravulizumab are the only licensed therapy for PNH, and their efficacy has relegated bone marrow transplantation to second-line therapy for hemolytic PNH in countries where the drug is available. Bone marrow transplantation might be an option if eculizumab is unavailable, and is a reasonable therapeutic strategy in patients with PNH and severe bone marrow failure. Adjunctive therapies (for example, immunosuppression) could be prescribed to patients with PNH who also have bone marrow failure to ameliorate the latter. However, these adjunctive treatments are not specific for PNH, nor do they have consistent effects on expanding or reducing PNH clones. The only curative strategy for PNH is allogeneic stem cell transplantation, but this procedure continues to carry a considerable risk of mortality.

Paroxysmal nocturnal hemoglobinuria: Epidemiology

The PNH epidemiology division provides insights about the historical and current patient pool, along with the forecasted trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends along with assumptions undertaken.


Key Findings

The disease epidemiology covered in the report provides historical and forecasted PNH epidemiology segmented as the Total diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria and Gender-specific Cases of PNH. The report includes the prevalent scenario of PNH in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.

Country-wise PNH Epidemiology

The epidemiology segment also provides the PNH epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.


The total prevalent population of PNH in the 7MM countries was estimated to be 8,385 cases in 2020.


As per the estimates, the United States had the highest prevalent population of Paroxysmal nocturnal hemoglobinuria in 2020. Among the EU5 countries, Germany had the highest prevalent population of PNH with 1,280 cases, followed by the United Kingdom in 2020. On the other hand, Spain had the lowest prevalent population of 199 cases in 2020.

Scope of the Report

  • PNH report covers a detailed overview explaining its causes, symptoms, classification, pathophysiology, diagnosis, and treatment patterns.
  • PNH Epidemiology Report and Model provide an overview of the risk factors and global trends of PNHin the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan).
  • The report provides insight into the historical and forecasted patient pool of PNH in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan
  • The report helps recognize the growth opportunities in the 7MM concerning the patient population.
  • The report assesses the disease risk and burden and highlights the unmet needs of Paroxysmal nocturnal hemoglobinuria.
  • The report provides the segmentation of the PNH epidemiology by total diagnosed prevalence cases of PNH in the 7MM
  • The report provides the segmentation of the PNH epidemiology by gender-specific cases of PNH in the 7MM.

Report Highlights

  • 11-year Forecast of PNH epidemiology
  • 7MM Coverage
  • Total Diagnosed Prevalent Cases of Paroxysmal nocturnal hemoglobinuria
  • Gender-specific Cases of Paroxysmal nocturnal hemoglobinuria

KOL Views

We interview KOLs and obtain SME’s opinion through primary research to fill the data gaps and validate our secondary research. The opinion helps understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.

Key Questions Answered

  • What will be the growth opportunities in the 7MM for the patient population pertaining to PNH?
  • What are the key findings pertaining to the Paroxysmal nocturnal hemoglobinuria epidemiology across 7MM, and which country will have the highest number of patients during the forecast period (2017–2030)?
  • What would be the total number of patients with Paroxysmal nocturnal hemoglobinuria across the 7MM during the forecast period (2017–2030)?
  • Among the EU5 countries, which country will have the highest number of patients during the forecast period (2017–2030)?
  • At what CAGR the patient population is expected to grow by in the 7MM during the forecast period (2017–2030)?
  • What are the disease risk, burdens, and unmet needs of Paroxysmal nocturnal hemoglobinuria?
  • What are the currently available treatments for Paroxysmal nocturnal hemoglobinuria?

Reasons to buy

PNH Epidemiology report will allow the user to:

  • Develop business strategies by understanding the trends shaping and driving the global PNH market
  • Quantify patient populations in the global Paroxysmal nocturnal hemoglobinuria market to improve product design, pricing, and launch plans
  • Organize sales and marketing efforts by identifying the age groups and sex that present the best opportunities for Paroxysmal nocturnal hemoglobinuria therapeutics in each of the markets covered
  • Understand the magnitude of the Paroxysmal nocturnal hemoglobinuria population by its prevalent cases.
  • Understand the magnitude of the Paroxysmal nocturnal hemoglobinuria population by its gender-specific cases.
  • The PNH epidemiology report and model were written and developed by Masters and Ph.D. 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 an 11-year forecast period using reputable sources

Key Assessments

  • Patient Segmentation
  • Disease Risk and Burden
  • Risk of disease by the segmentation
  • Factors driving growth in a specific patient population

1. Key Insights

2. Paroxysmal Nocturnal Hemoglobinuria (PNH): Market Overview at a Glance

2.1. Market Share (%) Distribution of PNH in 2017

2.2. Market Share (%) Distribution of PNH in 2030

3. Organizations

4. Executive Summary

5. Disease Overview: Paroxysmal Nocturnal Hemoglobinuria (PNH)

5.1. Clinical forms of PNH

5.2. Clinical manifestations

5.3. Pathophysiology of PNH

5.4. Diagnosis

5.4.1. Differential diagnosis

6. Epidemiology and Patient Population

6.1. Key Findings

6.2. Total Diagnosed Prevalence of PNH in 7MM

6.3. Epidemiology of PNH in 7MM

6.3.1. Assumptions and Rationale

6.4. The United States

6.4.1. Total Diagnosed Prevalence of PNH in the United States

6.4.2. Gender-specific Cases of PNH in the United States

6.5. Germany

6.5.1. Total Diagnosed Prevalence of PNH in Germany

6.5.2. Gender-specific Cases of PNH in Germany

6.6. Italy

6.6.1. Total Diagnosed Prevalence of PNH in Italy

6.6.2. Gender-specific Cases of PNH in Italy

6.7. France

6.7.1. Total Diagnosed Prevalence of PNH in France

6.7.2. Gender-specific Cases of PNH in France

6.8. Spain

6.8.1. Total Diagnosed Prevalence of PNH in Spain

6.8.2. Gender-specific Cases of PNH in Spain

6.9. The United Kingdom

6.9.1. Total Diagnosed Prevalence of PNH in the United Kingdom

6.9.2. Gender-specific Cases of PNH in the United Kingdom

6.10. Japan

6.10.1. Total Diagnosed Prevalence of PNH in Japan

6.10.2. Gender-specific Cases of PNH in Japan

7. Treatment and Management

8. SWOT Analysis

9. Case Studies

9.1. Paroxysmal Nocturnal Hemoglobinuria: Diagnostic Challenges in Pediatric Patient

9.2. Two Roads Diverge: Treatment Choice in Coexisting Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

10. KOL Views

11. Bibliography

12. Appendix

12.1. Report Methodology

13. DelveInsight Capabilities

14. Disclaimer

15. About DelveInsight

List of Table

Table 1: Clinical manifestations associated with PNH

Table 2: Indications for PNH screening

Table 3: Laboratory tests for the diagnosis of paroxysmal nocturnal hemoglobinuria (PNH)

Table 4: Laboratory/imaging analyses

Table 5: Differential Diagnosis of PNH

Table 6: Total Diagnosed prevalence of PNH in the 7MM (2017–2030)

Table 7: Total Diagnosed prevalence of PNH in the US (2017–2030)

Table 8: Gender-specific Cases of PNH in the US (2017–2030)

Table 9: Total Diagnosed prevalence of PNH in Germany (2017–2030)

Table 10: Gender-specific Cases of PNH in Germany (2017–2030)

Table 11: Total diagnosed prevalence of PNH in Italy (2017–2030)

Table 12: Gender-specific cases of PNH in Italy (2017–2030)

Table 13: Total Diagnosed prevalence of PNH in France (2017–2030)

Table 14: Gender-specific Cases of PNH in France (2017–2030)

Table 15: Total Diagnosed prevalence of PNH in Spain (2017–2030)

Table 16: Gender-specific Cases of PNH in Spain (2017–2030)

Table 17: Total Diagnosed prevalence of PNH in the UK (2017–2030)

Table 18: Gender-specific Cases of PNH in the UK (2017–2030)

Table 19: Total diagnosed prevalence of PNH in Japan (2017–2030)

Table 20: Gender-specific Cases of PNH in Japan (2017–2030)

List of Figures

Figure 1: Types of PNH

Figure 2: Clinical manifestations of PNH

Figure 3: Paroxysmal nocturnal hemoglobinuria (PNH) pathophysiology

Figure 4: Mechanism of hemolysis in paroxysmal nocturnal hemoglobinuria red blood cells

Figure 5: Effect of GPI-AP deficiency on blood cell populations

Figure 6: Clonal expansion in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Figure 7: Total Diagnosed prevalence of PNH in the 7MM (2017–2030)

Figure 8: Total Diagnosed prevalence of PNH in the US (2017–2030)

Figure 9: Gender-specific Cases of PNH in the US (2017–2030)

Figure 10: Total Diagnosed prevalence of PNH in Germany (2017–2030)

Figure 11: Gender-specific Cases of PNH in Germany (2017–2030)

Figure 12: Total diagnosed prevalence of PNH in Italy (2017–2030)

Figure 13: Gender-specific cases of PNH in Italy (2017–2030)

Figure 14: Total Diagnosed prevalence of PNH in France (2017–2030)

Figure 15: Gender-specific Cases of PNH in France (2017–2030)

Figure 16: Total Diagnosed prevalence of PNH in Spain (2017–2030)

Figure 17: Gender-specific Cases of PNH in Spain (2017–2030)

Figure 18: Total Diagnosed prevalence of PNH in the UK (2017–2030)

Figure 19: Gender-specific Cases of PNH in the UK (2017–2030)

Figure 20: Total diagnosed prevalence of PNH in Japan (2017–2030)

Figure 21: Gender-specific Cases of PNH in Japan (2017–2030)

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