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Thrombocytopenia- Market Insight, Epidemiology and Market Forecast -2030

Thrombocytopenia- Market Insight, Epidemiology and Market Forecast -2030

  • January 2021
  • 200 pages
  • ID: 6025276
  • Format: PDF
  • Delve Insight

Summary

Table of Contents

‘Thrombocytopenia —Market Insights, Epidemiology, and Market Forecast–2030’ report deliver an in-depth understanding of the Thrombocytopenia, historical and forecasted epidemiology as well as the Thrombocytopenia market trends in the United States, EU5(Germany, France, Italy, Spain, and the United Kingdom) and Japan.
The Thrombocytopenia market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Thrombocytopenia market size from 2018 to 2030. The report also covers current Thrombocytopenia treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Geography Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2018–2030
Thrombocytopenia Disease Understanding and Treatment Algorithm

Overview
Thrombocytopenia is a condition in which the blood has a lower than a normal number of blood cell fragments called platelets. Platelets are made in the bone marrow along with other kinds of blood cells. They travel through the blood vessels and stick together (clot) to stop any bleeding that may happen if a blood vessel is damaged. Platelets also are called thrombocytes because a clot also is called a thrombus. When a patient’s blood has too few platelets, mild to serious bleeding can occur. Bleeding can occur inside the body (internal bleeding) or underneath the skin, or from the skin’s surface (external bleeding). A normal platelet count in adults ranges from 150,000 to 450,000 platelets/?L of blood. A platelet count of fewer than 150,000 platelets/?L is lower than normal. If the blood platelet count falls below normal, the patient has thrombocytopenia. However, the risk for serious bleeding does not occur until the count becomes very low—less than 10,000 or 20,000 platelets/?L. Mild bleeding sometimes occurs when the count is less than 50,000 platelets per microliter.
Many factors can cause thrombocytopenia (a low platelet count). The condition can be inherited or acquired. “Inherited” means parents pass the gene for the condition to the patient. “Acquired” means the patients are not born with the condition, but they develop it. Sometimes the cause of thrombocytopenia is not known.

Thrombocytopenia Diagnosis
The diagnostic test that may be recommended include complete blood count (CBC) with platelet count and mean platelet volume (MPV) and peripheral blood smear (PBS). Thrombocytopenia can result from decreased platelet production, increased platelet consumption, or sequestration. A systematic approach should be used to evaluate incidental thrombocytopenia. Physicians should inquire about easy bruising or petechiae, melena, rashes, fevers, and bleeding during the patient’s history. They also should inquire about medication use, immunizations, recent travel, transfusion history, family history, and medical history. A history of acute and chronic alcohol use should be obtained.

Thrombocytopenia Treatment
Treatment for thrombocytopenia depends on its cause and severity. The main goal of treatment is to prevent death and disability caused by bleeding. If the condition is mild, the patient may not need treatment. A fully normal platelet count is not necessary to prevent bleeding, even with severe cuts or accidents.
If the cause of thrombocytopenia is unknown, and there are no contraindications, such as infections, corticosteroids may be used to increase the platelet count. More specific treatment plans usually depend on the underlying etiology of thrombocytopenia.
Patients with inherited thrombocytopenia have been treated with platelet transfusions (reserved for bleeding cases or its prevention, e.g., before surgery). While the fear of sensitization has dominated the restriction of platelet transfusion, the availability of leukoreduction has greatly decreased this risk. In this case, one of the two thrombopoietin-receptor agonists, eltrombopag, was investigated ted as a possible treatment option in MYH9-related disorders. The option of allogeneic stem cell transplantation is reserved for inherited thrombocytopenias with a high risk of marrow failure or a high risk of acute leukemia.
Many cases of ITP can be left untreated, and spontaneous remission in children is common. If therapy is required, the first-line treatment option is generally corticosteroids, with the recommended prednisone dose 1 mg/kg/day orally for up to 21–28 days depending upon the response, followed by slow tapering. Also, intravenous immunoglobulin or intravenous anti-D (Rho[D] immune globulin) can be used as an initial treatment with or without steroids. The most effective second-line treatment option is splenectomy. Additional second-line treatment options with documented evidence of efficacy (allowing postponement of splenectomy) include many agents: azathioprine, cyclosporine, cyclophosphamide, danazol, dexamethasone, vinca alkaloids, mycophenolate mofetil, rituximab, and thrombopoietin-receptor agonists.
The use of thrombopoietin-receptor agonists is an effective and safe second-line treatment strategy. Two thrombopoietin-receptor agonists were approved for the treatment of chronic ITP in adults by the US Food and Drug Administration (FDA) in 2008 and have as of now been used extensively for treatment of chronic ITP (ITP duration ?1 year) in relapsed and refractory patients. These were romiplostim, a thrombopoietin mimetic formed from peptides (peptibody), and eltrombopag (a small molecule, non-peptide).
The mainstay of treatment in TTP is plasmapheresis with plasma replacement. Other treatment modalities in non-responders to plasmapheresis include immune-suppression, not limited to high dose steroids and B-cell depleting agents (e.g., rituximab).
In the event of drug-induced thrombocytopenia/DITP, it is a universal practice to stop the suspected drug immediately. Platelet transfusions may be required to treat patients with severe thrombocytopenia and bleeding. Other supportive measures include high dose intravenous immunoglobulin, a brief course of corticosteroids, or even plasmapheresis.

Thrombocytopenia Epidemiology
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalent Cases of Thrombotic Thrombocytopenic Purpura (TTP), Prevalent Cases of Immune Thrombocytopenia (ITP), Prevalent Cases of Thrombocytopenia in Chronic Liver Disease, Incident Cases of Chemotherapy-induced Thrombocytopenia, Number of Cases of Heparin-induced Thrombocytopenia and Total Cases of Thrombocytopenia scenario of Thrombocytopenia in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2018 to 2030.

Key Findings
This section provides glimpses of the Thrombocytopenia epidemiology in the 7MM.
• As per the analysis, the total number of cases of Thrombocytopenia in the 7MM was 1,653,934 in 2020. These cases of Thrombocytopenia in the 7MM are expected to increase throughout the study period i.e. 2018–2030, with CAGR of 0.66% for the study period 2018–2030.
• As per the analysis, the United States accounted for the highest number of Thrombocytopenia cases among the 7MM. The total number of Thrombocytopenia cases in the US was estimated to be 467,078 in 2020.
• In 2020, the prevalent population of Thrombotic Thrombocytopenia in the United States was 6,002 which are expected to rise with the CAGR of 0.79% for the study period 2018–2030.
• In 2020, there were 66,708 prevalent cases of Immune thrombocytopenia (ITP) in the United States.
• Germany ranked second-highest for Thrombocytopenia cases among 7MM. In 2020, Germany had 325,202 Thrombocytopenia cases.
• Among the European 5 countries, Germany had the highest cases of Thrombocytopenia with 325,202 cases, followed by the United Kingdom and Spain, with 230,597 cases and 182,796 cases, respectively. On the other hand, Italy had the lowest cases, i.e., 102,933 in 2020.
• As per the analysis, Japan accounted for the fourth-highest number of Thrombocytopenia cases among the 7MM. The total number of Thrombocytopenia cases in Japan was estimated to be 219,017 in 2020.

Country Wise-Thrombocytopenia Epidemiology
The epidemiology segment also provides the Thrombocytopenia epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Thrombocytopenia Drug Chapters
This segment of the Thrombocytopenia report encloses the detailed analysis of the marketed and pipeline drugs/devices. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval, and patent details of each included drug and the latest news and press releases.
Thrombocytopenia Marketed Products
Cablivi (Caplacizumab/ALX-0081): Sanofi (Ablynx)
Cablivi is a von Willebrand factor (vWF)-directed antibody fragment indicated for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. It is approved in the US and EU for the treatment of aTTP. A Phase II/III trial is ongoing in Japan for the same indication. Cablivi was approved in the European Union in August 2018. The US FDA approved the drug in February 2019 for the treatment of aTTP. The drug is also marketed in Germany, Denmark, Italy, and Finland and is available in France under a temporary user license.
Products detail in the report…
Tavalisse (Fostamatinib): Rigel Pharmaceuticals/Kissei Pharmaceutical
Fostamatinib disodium (also known as Tavalisse; R-985788) is an orally-bioavailable investigational agent being developed by Rigel pharmaceuticals and approved for the treatment of patients suffering from persistent/chronic adult idiopathic thrombocytopenic purpura. The therapeutic candidate inhibits FcR-triggered, Syk-dependent cytoskeletal rearrangement during phagocytosis. As stated by Rigel Pharmaceuticals, fostamatinib has a unique mechanism of action, blocking IgG receptor signaling in both macrophages and B cells via SYK kinase. In April 2018, the US FDA approved Tavalisse (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. In July 2020, Rigel Pharmaceuticals announced that Grifols, its collaborative partner in Europe, had launched Tavalisse in Germany and the United Kingdom.
Products detail in the report…
List to be continued in the report…
Thrombocytopenia Emerging Therapies
Rozanolixizumab (UCB7665): UCB Biopharma
Rozanolixizumab (also known as UCB7665) is UCB Biopharma’s intravenously and subcutaneously administered, investigational humanized monoclonal IgG antibody being developed by UCB for the treatment of idiopathic thrombocytopenic purpura and myasthenia gravis (MG). The body’s immune system produces antibodies that normally help fight off infections and threats. In MG patients, pathogenic IgG autoantibodies mistakenly attack healthy cells and tissues, causing weakness and voluntary muscle fatigue. High concentrations of pathogenic IgG autoantibodies in the circulation occur due to the action of the neonatal Fc receptor (FcRn) that bind to IgG, inhibiting its degradation. Rozanolixizumab works by binding with high affinity to human neonatal Fc receptor (FcRn), selectively inhibiting IgG rescue and recycling. The company is currently evaluating this molecule in the phase III stage of development in subjects with primary immune thrombocytopenia (ITP). The company expects topline the phase III trial results by the second half of 2022.
Products detail in the report…
Rilzabrutinib (PRN-1008): Principia Biopharma
Rilzabrutinib (PRN-1008) is an orally administered reversible covalent inhibitor of Bruton Tyrosine Kinase (BTK). BTK is an essential signaling element downstream of the B cell receptor (BCR), Fc-gamma receptor, and Fc-epsilon receptor pathways. BTK activation is critical for B cell activation and maturation. BTK also regulates antibody-mediated activation of other immune cells, such as macrophages, neutrophils, and mast cells through Fc receptor signaling. The drug candidate is a fast-acting, orally available therapy that could effectively and safely modulate B-cell function without depleting the B-cells and is expected to be a major advancement in treating autoimmune and inflammatory diseases. PNR-1008 is under development by Principia Biopharma and is currently in the phase III stage of development to treat Immune Thrombocytopenia in adults and adolescents with persistent or chronic immune thrombocytopenia (ITP).
Products detail in the report…
List to be continued in the report…

Thrombocytopenia Market Outlook
Treatment for thrombocytopenia depends on its cause and severity. The main goal of treatment is to prevent death and disability caused by bleeding. If the condition is mild, the patient may not need treatment. A fully normal platelet count is not necessary to prevent bleeding, even with severe cuts or accidents.
If the cause of thrombocytopenia is unknown, and there are no contraindications, such as infections, corticosteroids may be used to increase the platelet count. More specific treatment plans usually depend on the underlying etiology of thrombocytopenia.
Patients with inherited thrombocytopenia have been treated with platelet transfusions (reserved for bleeding cases or its prevention, e.g., before surgery). While the fear of sensitization has dominated the restriction of platelet transfusion, the availability of leukoreduction has greatly decreased this risk. In this case, one of the two thrombopoietin-receptor agonists, eltrombopag, was investigated ted as a possible treatment option in MYH9-related disorders. The option of allogeneic stem cell transplantation is reserved for inherited thrombocytopenias with a high risk of marrow failure or a high risk of acute leukemia.
Many cases of ITP can be left untreated, and spontaneous remission in children is common. If therapy is required, the first-line treatment option is generally corticosteroids, with the recommended prednisone dose 1 mg/kg/day orally for up to 21–28 days depending upon the response, followed by slow tapering. Also, intravenous immunoglobulin or intravenous anti-D (Rho[D] immune globulin) can be used as an initial treatment with or without steroids. The most effective second-line treatment option is splenectomy. Additional second-line treatment options with documented evidence of efficacy (allowing postponement of splenectomy) include many agents: azathioprine, cyclosporine, cyclophosphamide, danazol, dexamethasone, vinca alkaloids, mycophenolate mofetil, rituximab, and thrombopoietin-receptor agonists.
The use of thrombopoietin-receptor agonists is an effective and safe second-line treatment strategy. Two thrombopoietin-receptor agonists were approved for the treatment of chronic ITP in adults by the US Food and Drug Administration (FDA) in 2008 and have as of now been used extensively for treatment of chronic ITP (ITP duration ?1 year) in relapsed and refractory patients. These were romiplostim, a thrombopoietin mimetic formed from peptides (peptibody) and eltrombopag (a small molecule, non-peptide).
According to the report, the Thrombocytopenia market is expected to change in the study period 2018–2030.

Key Findings
This section includes a glimpse of the Thrombocytopenia 7MM market.
• The market size of Thrombocytopenia in the seven major markets was USD 4,142.40 million in 2020 which is expected to grow at a CAGR of 4.69%, for the study period of 2018–2030.
• The market size of Thrombocytopenia in the US was USD 2,327.88 million in 2020.
• Among the EU5 countries, the UK had the highest market size with USD 391.79 million in 2020, while France had the lowest market size of Thrombocytopenia with USD 215.47 million in 2020.
• The United States accounts for the highest market size of Thrombocytopenia, in comparison to the other major markets i.e., EU5 countries (the United Kingdom, Germany, Italy, France, and Spain), and Japan.
• With the expected launch of upcoming therapies, Rozanolixizumab (UCB7665); Rilzabrutinib (PRN-1008); BAX930; Efgartigimod (ARGX-113); Cablivi (caplacizumab); Tavalisse/Tavlesse (Fostamatinib); Doptelet (Avatrombopag); Nplate (Romiplostim)-for CIT; and Mulpleta (lusutrombopag), the market might experience a significant growth.
• According to the analysis, for ITP, the market of current treatment includes TPO-RAs (Nplate (Romiplostim); Promacta (eltrombopag), which generated the revenue of nearly USD 642.98 million and USD 751.44 million in 2020.

Market Outlook for Seven Major Markets
This section provides the total Thrombocytopenia market size and market size by therapies in the United States, Germany, France, Italy, Spain, the United Kingdom, and Japan.

Thrombocytopenia Drugs Uptake
This section focuses on the rate of uptake of the potential drugs that are expected to get launched in the market during the study period 2018–2030. The analysis covers Thrombocytopenia market uptake by drugs; patient uptake by therapies; and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Thrombocytopenia Development Activities
The report provides insights into different therapeutic candidates in the clinical and premarket clinical studies. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Thrombocytopenia emerging therapies.
Competitive Intelligence Analysis
We perform competitive and market intelligence analysis of the Thrombocytopenia market by using various competitive intelligence tools that include–SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report
• The report covers the descriptive overview of Thrombocytopenia, explaining its causes, symptoms, diagnosis, and currently available therapies.
• Comprehensive insight has been provided into the Thrombocytopenia epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for Thrombocytopenia is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of the Thrombocytopenia market; historical and forecasted is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM Thrombocytopenia market.

Report Highlights
• The thrombocytopenia market is anticipated to increase for the study period 2018–2030.
• In the coming years, the Thrombocytopenia market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Thrombocytopenia R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Thrombocytopenia. The expected launch of emerging therapies will significantly impact the Thrombocytopenia market.
• Our in-depth analysis of the pipeline assets across different stages of development, different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key competitors, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Thrombocytopenia Report Insights
• Patient Population
• Therapeutic Approaches
• Thrombocytopenia Pipeline Analysis
• Thrombocytopenia Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Thrombocytopenia Report Key Strengths
• 10-Years Forecast
• 7MMCoverage
• Thrombocytopenia Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market

• Drugs Uptake

Thrombocytopenia Report Assessment
• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
• SWOT Analysis

Key Questions

Market Insights:
• What was the Thrombocytopenia market share (%) distribution in 2018 and how it would look like in 2030?
• What would be the Thrombocytopenia total market size as well as market size by therapies across the 7MM during the forecast period (2021–2030)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest Thrombocytopenia market size during the forecast period (2021–2030)?
• At what CAGR, the Thrombocytopenia market is expected to grow at the 7MM level during the forecast period (2021–2030)?
• What would be the Thrombocytopenia market outlook across the 7MM during the forecast period (2021–2030)?
• What would be the Thrombocytopenia market growth until 2030, and what will be the resultant market size in the year 2030?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:
• What is the disease risk, burden, and unmet needs of Thrombocytopenia?
• What is the historical Thrombocytopenia patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan?
• What would be the forecasted patient pool of Thrombocytopenia at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Thrombocytopenia?
• Out of the countries mentioned above, which country would have the highest number of Thrombocytopenia cases during the forecast period (2021–2030)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:
• What are the current options for the treatment of Thrombocytopenia along with the approved therapy?
• What are the Thrombocytopenia marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of Thrombocytopenia?
• How many therapies are developed by each company for the treatment of Thrombocytopenia?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Thrombocytopenia?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Thrombocytopenia therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Thrombocytopenia and their status?
• What are the key designations that have been granted for the emerging therapies for Thrombocytopenia?
• What are the 7MM historical and forecasted market of Thrombocytopenia?

Reasons to buy
• The report will help in developing business strategies by understanding trends shaping and driving the Thrombocytopenia.
• To understand the future market competition in the Thrombocytopenia market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Thrombocytopenia in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the Thrombocytopenia market.
• To understand the future market competition in the Thrombocytopenia market.

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