Rare Genetic Disorders Affecting the Periodontal Supporting Tissues in Adolescence
The Changing Market Dynamics Of Immune Thrombocytopenia, Aplastic Anemia, And Diamond-Blackfan AnemiaDelveInsight Insights
Blood disorders affect millions worldwide—an estimated 5% of the global population suffers from conditions like anemia, and others. Anemia is a silent epidemic, with over 2 billion people affected globally, often caused by iron deficiency or chronic diseases. With ongoing research, the treatment landscape for blood disorders continues to evolve, from targeted therapies to life-saving gene editing technologies.
LAS VEGAS, Dec. 9, 2024 /PRNewswire/ -- Blood disorders are a group of conditions that affect the production, function, or structure of blood cells, leading to various health complications. These disorders can involve issues with red blood cells, white blood cells, platelets, or the plasma components of blood. Common types of blood disorders include anemia, where there is a deficiency of red blood cells or hemoglobin, leading to fatigue and weakness, and thrombocytopenia, characterized by low platelet counts, which can cause excessive bleeding. The causes of blood disorders range from genetic factors and autoimmune conditions to infections and environmental factors. Treatment options depend on the specific disorder and may include medications, blood transfusions, or bone marrow transplants.
DelveInsight has recently released a series of epidemiology-based market reports focusing on blood disorders including Immune Thrombocytopenia, Aplastic Anemia, and Diamond-Blackfan Anemia. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034 segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Additionally, the reports feature an examination of prominent companies working with their lead candidates in different stages of clinical development. Let's deep dive into the assessment of these blood disorders markets individually.
Immune thrombocytopenic purpura (ITP) is an autoimmune condition marked by a low platelet count, resulting in bruising and bleeding. It manifests as acute in children and chronic in adults. The primary treatments are corticosteroids, intravenous immunoglobulin (IVIg), and rituximab.
In the 7MM, the United States reported the highest prevalence of ITP, with nearly 66,500 cases in 2023. Primary ITP, which accounts for 80% of cases, occurs without any underlying diseases, while secondary ITP, making up about 20% of cases, is linked to conditions such as systemic lupus erythematosus (SLE), medications, infections, and other autoimmune diseases.
In immune thrombocytopenia, the initial treatment generally consists of corticosteroids, with prednisone prescribed at 1 mg/kg/day for 21-28 days, followed by a gradual taper. High-dose dexamethasone (40 mg/day for 4 days) may lead to a more pronounced platelet response. Intravenous immunoglobulin (IVIG) or intravenous anti-D (Rho[D] immune globulin) may be used as first-line options, particularly in urgent situations requiring quick platelet increases.
However, many adults experience relapses or do not respond to first-line therapy, necessitating second-line treatments. IVIG is effective in 70-80% of cases, and anti-D works in 50-70% of RhD-positive patients. Splenectomy is a well-established second-line option, though it carries risks such as infection and bleeding.
Rituximab, a monoclonal antibody that targets CD20, has shown strong efficacy both before and after splenectomy. Thrombopoietin-receptor agonists, such as romiplostim and eltrombopag, are also effective second-line treatments as they promote platelet production by activating the thrombopoietin receptor.
In the US, the approved treatments for ITP are DOPTELET, TAVALISSE, PROMACTA, and NPLATE. The EU market for thrombocytopenia is primarily driven by both approved and off-label therapies. In the EU, approved treatments include DOPTELET (avatrombopag), TAVALISSE/TAVLESSE (fostamatinib), PROMACTA/REVOLADE (eltrombopag), and NPLATE/Romiplate (romiplostim). RITUXAN is only approved in Japan for the treatment of ITP.
Learn more about the FDA-approved drugs for ITP @ Drugs for Immune Thrombocytopenic Purpura Treatment
As per DelveInsight analysis, in 2023, the total ITP market size in the 7MM was USD 3.1 billion. As per the estimates, PROMACTA (eltrombopag) achieved the highest revenue among all medications in the 7MM in 2023. The market is expected to show positive growth, mainly attributed to the increasing cases and also, the launch of upcoming therapies during the forecast period (2024–2034).
Immune Thrombocytopenic Purpura Pipeline Therapies and Key Companies
Rilzabrutinib (PRN-1008): Sanofi/Principia Biopharma
Mezagitamab (TAK-079): Millennium Pharmaceuticals/Takeda
Efgartigimod (ARGX-113): argenx
BT-595: Biotest
Dive deeper for rich insights into the Immune Thrombocytopenic Purpura Clinical Trials
Aplastic Anemia Market
Aplastic anemia is a rare and serious non-cancerous condition marked by the autoimmune destruction of early blood-forming cells. The global incidence varies from 0.7 to 7.4 cases per million people annually, with higher rates observed in Asia compared to Europe and the United States.
As per DelveInsight estimates, the total Incident population of aplastic anemia in the 7MM were around 2,500 cases in 2023. These cases are projected to increase during the forecast period. Based on severity, severe and very severe aplastic anemia patients contributed roughly 65%-80% of the total aplastic anemia patient population.
HSCT remains the standard treatment for patients under 40 years old, while Immunosuppressive Therapies (IST) are the preferred approach for patients aged 40 and above. In terms of pharmacological treatments, Novartis' PROMACTA/REVOLADE is currently the leading revenue driver, followed by ISTs across the 7MM.
According to Novartis, the primary revenue drivers for PROMACTA/REVOLADE are its use in patients with chronic immune thrombocytopenic purpura (ITP) and severe aplastic anemia (SAA). PROMACTA was first approved in 2014 for use in relapsed/refractory SAA patients in the United States, with approvals following in Europe in 2015 and Japan in 2017. However, it was approved for use as a first-line treatment in combination with ISTs like ATG in the US in 2018, which expanded treatment options for first-line patients and contributed to increased revenue.
The total aplastic anemia market size was found to be ~USD 270 million in 2023 in the 7MM, which is further expected to increase by 2034 due to several factors such as an increase in disease incidence in Asian countries, high patient uptake of PROMACTA (until expected patent expiry in 2025), approval of Teva Pharma's ALVAIZ in the US, and Kyowa Kirin's ROMIPLATE in Japan along with the expected launch of potential emerging therapies.
Aplastic Anemia Pipeline Therapies and Key Companies
For a comprehensive view of the aplastic anemia market, check out the Aplastic Anemia Market Assessment
Diamond-Blackfan Anemia Market
Diamond-Blackfan anemia (DBA) is a rare genetic condition that primarily disrupts the bone marrow's ability to produce red blood cells, resulting in severe anemia. It is typically diagnosed during infancy or early childhood and presents with symptoms such as pale skin, fatigue, stunted growth, and physical abnormalities, which may include thumb deformities or heart defects in certain cases.
In 2023, there were around 7,870 reported cases of Diamond-Blackfan anemia across the 7MM, with the United States accounting for about 70% of these cases. Within the US, approximately 46% of cases in 2023 were linked to mutations in the RPS19 gene, which plays a crucial role in ribosomal protein synthesis, contributing to its high prevalence in DBA.
Diamond-Blackfan anemia is frequently associated with several congenital anomalies, with craniofacial and musculoskeletal defects being the most common. Craniofacial abnormalities were noted in over 30% of DBA patients across the 7MM in 2023.
Although DBA has a significant impact on patient health, there are currently no FDA-approved treatments available for the condition. Treatment primarily involves corticosteroid combinations, chronic blood transfusions, iron chelation, and potentially curative hematopoietic stem cell transplantation (HSCT).
In 2023, the total market size for Diamond-Blackfan anemia was USD 1.56 million in the 7MM and is projected to increase by 2034. The United States represents the largest share of the Diamond Blackfan Anemia market, accounting for a significant proportion compared to the EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
In 2023, corticosteroids were the leading therapy for DBA, generating USD 1.1 million in revenue, making them the preferred first-line treatment option. Since there are no FDA-approved therapies available and no emerging companies are working to fill this gap, there is a significant opportunity for innovative treatments, like gene therapy, to make a meaningful difference and address the needs of DBA patients.
For a deeper understanding of the Diamond-Blackfan anemia market landscape, explore the Diamond-Blackfan Anemia Market Outlook
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Effects Of Lymphocyte Counts On Diagnosis, Staging, And Treatment Of CLL
Lymphocyte counts help doctors diagnose, stage, and treat chronic lymphocytic leukemia (CLL). Other factors, such as the risk level of the type of CLL you have, also play a role.
CLL is a slowly progressing type of leukemia that affects stem cells, which eventually develop into white blood cells called lymphocytes. It's the most common type of leukemia in adults.
Because CLL affects cells that become lymphocytes, the number of lymphocytes in your blood is important for many aspects of CLL. But the rate at which the count doubles is more important.
In this article, we cover how doctors use lymphocyte counts to diagnose, stage, and treat CLL.
Lymphocytes are a type of white blood cell. All white blood cells, including lymphocytes, are made in the bone marrow. As a part of your immune system, lymphocytes play an important role in protecting you from infections.
Doctors determine lymphocyte counts using a complete blood count (CBC). A CBC is a blood test that calculates the number of different types of blood cells, including lymphocytes, in a blood sample drawn from a vein in your arm. A lymphocyte count is the number of lymphocytes you have per microliter (uL) of blood.
A peripheral blood smear can also assess the number of lymphocytes in your blood. In this test, a healthcare professional spreads a blood sample on a slide and analyzes it under a microscope. They can also review the size and shape of different blood cells using this test.
In CLL, lymphocytic cells begin to grow and divide uncontrollably. This leads to high levels of lymphocytes in the blood, a condition called lymphocytosis.
When your lymphocyte count is above the upper part of this range, you have lymphocytosis. According to a 2017 review, many people with CLL present to their doctor with lymphocyte counts of over 10,000 lymphocytes per uL of blood.
If your lymphocyte count is high, your doctor will likely order a blood flow cytometry test. A flow cytometry test can identify whether your cancer is CLL and not another form of leukemia.
In addition to diagnosing CLL, a flow cytometry test can help doctors stage your cancer, look for markers that determine how aggressive your cancer might be, and choose your initial treatment.
Many people with CLL may never require treatment. In these cases, a doctor routinely monitors them and checks their blood counts every few months.
The need for treatment depends on whether a person:
Some research shows that the trajectory of lymphocyte levels before diagnosis can predict the time to treatment for CLL. People whose lymphocyte levels were growing faster before diagnosis had a higher risk of needing treatment sooner.
Experts consider a lymphocyte count of 15,000 per uL a risk factor for needing treatment within 5 years of diagnosis.
If you need treatment for CLL, your doctor will monitor your lymphocyte levels throughout your treatment. Dropping lymphocyte levels can indicate a particular treatment is effective against your CLL.
Generally speaking, having a high lymphocyte count and a rapidly progressive lymphocyte count are both factors associated with a less favorable outlook for someone with CLL. A 2017 review notes this is typically greater than 50,000 lymphocytes per uL.
However, lymphocyte counts are only one piece of the puzzle. Other factors important for your CLL outlook are:
Your care team will consider all these factors to help predict your outlook and whether treatment is needed.
According to the National Cancer Institute's SEER database, the average 5-year survival rate for CLL is 88.5%. However, your care team can give you a better idea of your outlook based on your specific situation.
In CLL, cells that will eventually become lymphocytes begin to grow and divide out of control. Because of this, people with CLL have high lymphocyte counts.
Lymphocyte counts play an important role in many aspects of CLL. These include diagnosis, staging, and treatment.
While higher lymphocyte counts are associated with a less favorable CLL outlook, many other factors play vital roles as well. As such, be sure to have an open conversation with your care team for a better idea of your individual outlook.
Mother's Urgent Plea To Find Stem Cell Donor For Nine-year-old Daughter
A woman is urgently seeking a stem cell donor for her daughter after she was diagnosed with a rare and potentially fatal blood disorder.
Amilah Hussain, nine, from Horsham in West Sussex, was taken to her GP to investigate repeated bruising on her shins earlier this year.
Blood tests showed low platelet counts and she was diagnosed with aplastic anaemia at Great Ormond Street Hospital in October.
Aplastic anaemia occurs when the bone marrow cannot make enough new blood cells for the body to work normally, making it harder to fight infection, stop bleeding or carry oxygen.
Doctors told Amilah's mother, Mobeen Hussain, 42, that a stem cell transplant was the only hope for a cure but there is currently no match on the stem cell register.
"When we were told Amilah's diagnosis, they told us the only cure was a stem cell transplant," Mrs Hussain said.
"It needed to be treated soon and would be better when Amilah is well because she'd make a really good recovery."
Mrs Hussain is now working with Anthony Nolan on the Amilah Means Hope campaign to encourage people between the ages of 16 and 30 – particularly those from minority ethnic backgrounds – to sign the stem cell register.
As Amilah has Pakistani heritage, she is more likely to have a unique tissue type and less likely to find a donor on UK registers compared to those from white, northern European backgrounds.
Unfortunately, Amilah's older brother Naa'il is not a match.
Mrs Hussain added: "When we looked into it, it seemed there's a lack of donors suitable for patients from ethnic minority communities. The general awareness is just not there.
"Knowing it will be difficult for Amilah to find a donor makes me want to do something."
A transplant will introduce healthy stem cells into the little girl's bloodstream to help her body grow and produce new blood cells.
It is hoped the procedure will allow Amilah – known as Milly to friends and family – to get back to her hobbies of horse riding and karate.
Mrs Hussain said: "Until this happened, I didn't realise that this (stem cell donation) is something people can do. It's why I'm doing this.
"Stem cell donation is giving a gift of life; it's such a precious gift to give anyone."
There are only about 100 to 150 people diagnosed with aplastic anaemia every year in the UK, according to the charity Anthony Nolan.
It is most common in people aged between 10 and 20, as well as people over the age of 60.
Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: "Finding a donor for Amilah would mean giving her a second chance at life.
"We need more people aged 16-30 in good general health to sign up to the Anthony Nolan register.
"Amilah's donor will most likely share her background, which is why we're particularly calling on people with Pakistani heritage to join the register and give someone like Amilah hope for the future."
Mrs Hussain explained that Amilah means "hope" in Arabic, which she has embraced for the campaign name.
"Hope not just for herself but for all those who need a hero right now and for all those who will need one in the future," she added.
"Most heroes are ordinary people who do extraordinary things. Signing up to the Anthony Nolan register is a chance to change a life and gift someone hope for the future.
"She means a lot to a lot of people."
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