Paroxysmal Nocturnal Hemoglobinuria - Epidemiology Forecast to 2030

Paroxysmal Nocturnal Hemoglobinuria - Epidemiology Forecast to 2030

  • February 2021 •
  • 75 pages •
  • Report ID: 6028051 •
  • Format: PDF
‘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.
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 underexpression 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

• 10-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 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

Geographies Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan
Study Period: 2017–2030
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening blood disease that can appear at any age, race, or gender but is diagnosed most often in people in their 30s and 40s. As it is a rare condition, its incidence and prevalence have been described only in a few small studies – studies in the past have estimated that there were between 1 and 10 cases per million people worldwide. Moreover, although the international PNH registry is a rich source of data on real-world PNH patients globally, it is not possible to estimate the incidence and prevalence of PNH directly from the registry. Approval of the pioneer drug, eculizumab, has paved the way in disease treatment, reducing the mortality rate, and improving quality of life. All of this further necessitates the need for a detailed study of epidemiology and patient characteristics to understand the responsiveness and monitor the efficacy and safety parameters.