Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market
Number of pages: 100 | Report Format: PDF | Published date: February 24, 2023
Historical Years – 2020 | Base Year – 2021 | Forecasted Years – 2022-2030
|
Report Attribute |
Details |
|
Market Size Value in 2021 |
US$ 28.35 million |
|
Revenue Forecast in 2030 |
US$ 37.31 million |
|
CAGR |
3.1% |
|
Base Year For Estimation |
2021 |
|
Forecast Period |
2022-2030 |
|
Historical Year |
2020 |
|
Segments Covered |
Treatment type, End user, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, and the Rest of the World (RoW) |
According to the deep-dive market assessment study by Growth Plus Reports, the global congenital thrombotic thrombocytopenic purpura (cTTP) market was valued at US$ 28.35 million in 2021 and is expected to register a revenue CAGR of 3.1% to reach US$ 37.31 million by 2030.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market Fundamentals
Congenital thrombotic thrombocytopenic purpura (cTTP) is a blood condition characterized by the formation of blood clots in small blood arteries throughout the body. Symptoms usually appear in infancy or early childhood. However, they might appear as late as adulthood in certain circumstances. Hemolytic anemia, low platelets (thrombocytopenia), and neurologic impairment are the most common symptoms of the condition. Anemia symptoms include fatigue, paleness, jaundice, shortness of breath, and a fast heart rate. Widespread thrombosis (abnormal clotting) can cause neurological system difficulties (such as personality changes, migraines, confusion, and seizures), improper renal function, cardiac problems, and gastrointestinal disorders.
Generally, cTTP is also caused by mutations in the ADAMTS13 gene, which generates a protein that aids in regulating blood clot formation. ADAMTS13 is located on chromosome 9q34 and encodes the 1427 amino acid ADAMTS13 protein. Since the discovery of ADAMTS13 causal mutations in 2001, over 200 distinct causative mutations have been documented globally. These mutations are passed down from parents to children in an autosomal recessive manner, and even one causal mutation decreases ADAMTS13 activity by 20%-50%. The following indicators are used to diagnose cTTP:
ADAMTS13 activity is primarily assessed using two separate assays: a FRET assay utilizing FRETS-VWF73 as a substrate and a chromogenic ADAMTS13 activity ELISA employing a murine monoclonal antibody that specifically identifies Y1605 of the VWF cleavage site. Even though all cTTP patients have drastically reduced ADAMTS13 activity over time, individuals may have normal platelet counts, lactate dehydrogenase (LDH), and other TTP symptoms in the absence of TTP triggers. Plasma exchange therapy (also known as plasmapheresis) is the backbone of treatment for cTTP. It includes extracting the patient’s plasma (the liquid part of the blood) and replacing it with donor plasma containing normal blood levels of ADAMTS13 protein. This can help to avoid blood clots and lower the possibility of catastrophic problems, such as stroke or organ damage.
Other treatments, in addition to plasma exchange therapy, may be used to manage symptoms and prevent problems. Corticosteroids and immunosuppressive medicines, for instance, can help lower inflammation and prevent the immune system from attacking the patient’s own blood cells. Antiplatelet medicines decrease platelet function and thus help avoid blood clot formation (small blood cells involved in the clotting process). Rituximab is a medicine that inhibits the formation of antibodies that attack the ADAMTS13 protein by targeting a specific type of immune cell (B cells). The treatment strategy for cTTP is devised in consultation with a certified healthcare practitioner based on the individual patient’s symptoms and medical history.
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Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market Dynamics
During the projected period, the global congenital thrombotic thrombocytopenic purpura (cTTP) market is expected to increase significantly. The factors driving this market’s revenue growth include emerging markets and massive expenditure in research and development. Additionally, as the incidence of cTTP is expected to be one in every million people, it is challenging to establish correlations between genotype and clinical presentation, effective treatment regimen, and long-term prognosis. Several countries, notably the United Kingdom, Switzerland (international hereditary TTP registry), France, and Japan, have discovered large-scale cTTP cohorts that have progressively provided compelling evidence over the last few decades, ultimately driving the market revenue growth.
The annual incidence of immunological thrombotic thrombocytopenic purpura (TTP) and cTTP is estimated to be 2 to 6 people per million: cTTP accounts for 2%-10% of all TTP cases in worldwide registries. Although the onset of symptoms is traditionally regarded as occurring in childhood, it can occur at any age, with a substantial rise in women during pregnancy. Additionally, several coagulation tests, including complete blood count, bleeding time, factor V assay, thrombin time, fibrinogen level, and platelet count, are now available to diagnose thrombophilia. These factors are creating significant growth opportunities in the market.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market Ecosystem
The global congenital thrombotic thrombocytopenic purpura (cTTP) market has been analyzed from three perspectives: treatment type, end user, and region.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market by Treatment Type
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Based on treatment type, the congenital thrombotic thrombocytopenic purpura (cTTP) market has been segmented into Infusion of ADAMTS13-rich blood products/plasma infusion, plasma exchange (plasmapheresis), immunotherapy, and others. The immunotherapy segment is further sub-segmented into corticosteroids, rituximab (Rituxan), and others.
The infusion of ADAMTS13-rich blood products/plasma infusion segment dominates the market. Acute episodes of congenital TTP can be treated with plasma infusion (10-15 ml/kg/day till remission), although newborns often require exchange transfusion. Prophylactic plasma therapy may be explored for patients with a persistent relapsing illness course. Regular plasma infusions are required during pregnancy to keep ADAMTS13 activity levels around 15% and close clinical and biological monitoring by a reference center. Therapeutic plasma exchanges may be required to prevent fluid excess during the third trimester. cTTP is a rapidly lethal condition without treatment (mortality rate > 90%). The introduction of therapeutic plasma exchange and plasma infusion has resulted in a 15% reduction in mortality. Plasma therapy also helps to avoid long-term organ damage caused by chronic relapses.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market by End User
Based on the end user, the congenital thrombotic thrombocytopenic purpura (cTTP) market has been segmented into hospitals & clinics, day care centers, and others.
The hospital & clinics segment dominates the market, as cTTP treatment is often prescribed only in hospital settings with trained medical professionals. Additionally, better and more advanced technological solutions are the key factors supporting the leading position of the hospitals and clinics segment. Perpetual growth in the rate of hospital admissions for cTTP is another segment driver.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market by Region
Based on region, the congenital thrombotic thrombocytopenic purpura (cTTP) market has been segmented into North America, Europe, Asia Pacific, and the Rest of the World.
North America dominates the market due to the increasing demand for disease-specific treatment, the rising use of modern technology, increased healthcare expenditure, greater patient awareness, and the high frequency of bleeding disorders.
The annual incidence of TTP is estimated at 0.25 to 1 per 250,000 individuals, of which immune-mediated TTP is much more common than congenital TTP, accounting for over 90%-95% of all TTP cases, and has a 3:1 female-to-male ratio globally. Disease onset is typically sudden and occurs in adulthood (median age 40 years). In the absence of treatment, TTP is a rapidly fatal disease (mortality rate > 90%). However, the introduction of TPE has led to a decrease in the rate of mortality to 10%-15%. With the recent addition of caplacizumab, mortality can further decrease to < 10%. In countries such as the United States (3–4 cases/million), the United Kingdom (6 cases/million), France (2–4 cases per million), and Germany (2.1 cases/million), the incidence is slightly higher than the global incidence for aTTP. The expected prevalence of cTTP is less than one case per million. Congenital TTP is much less common than the immune-mediated form of the disease (immune-mediated TTP), accounting for up to only 5-10% of all TTP cases.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market Competitive Landscape
The prominent players in the global congenital thrombotic thrombocytopenic purpura (cTTP) market include:
Strategic Development
North America is the key revenue growth region in the global congenital thrombotic thrombocytopenic purpura (cTTP) market.
The factors driving this market’s revenue growth include emerging markets and massive expenditure in research and development.
The global congenital thrombotic thrombocytopenic purpura (cTTP) market is expected to reach 37.31 million by 2030.
The global congenital thrombotic thrombocytopenic purpura (cTTP) market is expected to register a revenue CAGR of 3.1% during the forecast period.
Takeda Pharmaceutical Company Ltd., Sanofi, F. Hoffmann-La Roche Ltd., Novartis AG, and Mylan N.V. are among the top market players.
*Insights on financial performance are subject to availability of the information in the public domain.