Abstract - 2018 - Research and Practice in Thrombosis and Haemostasis
What Is Hemophilia? Symptoms, Causes, Diagnosis, And Treatment
It's common for people who have or have had family members with hemophilia to get their baby boys tested for the condition shortly after they're born. About one-third of babies who have hemophilia have no relatives with the disorder, so infants may be evaluated if they show symptoms.[9]
Ideally, testing is planned before the baby's birth so that a blood sample can be drawn from the umbilical cord. Umbilical cord blood testing is better at discovering low levels of factor 8 than at finding low levels of factor 9, because factor 9 levels are not at a normal level until a baby is at least 6 months old.
Two types of tests are used for diagnosis: screening tests and clotting factor tests, which are also called factor assays.
Screening TestsScreening tests are blood tests that reveal whether the blood is clotting properly. There are several types of screening tests for hemophilia:
Complete Blood Count (CBC) This test measures the amount of hemoglobin (the red pigment inside red blood cells that carries oxygen), the percentage of blood volume occupied by red blood cells (called the hematocrit), the size and amount of red blood cells, and the amounts of white blood cells and platelets in the blood. The CBC is normal in people with hemophilia, but if you have hemophilia and you have unusually heavy bleeding or bleed for a long period of time, the hemoglobin and the hematocrit can be low.
Activated Partial Thromboplastin Time (APTT) Test This test reveals how much time it takes for blood to clot; specifically, it measures the clotting ability of factors 8, 9, 11, and 12. If any of these clotting factors are diminished, it takes longer than normal for the blood to clot. The results of the APTT test will show a longer clotting time if you have hemophilia A or B.
Prothrombin Time (PT) Test This test also reveals how long it takes for blood to clot. It focuses on the clotting ability of factors 1, 2, 5, 7, and 10. If any of these factors are in short supply, it takes longer for the blood to clot. Most people with hemophilia A and B will have normal results on this test.
Fibrinogen Test This test also reveals how well a person can form a blood clot. Fibrinogen is also known as clotting factor 1.
Clotting Factor TestsClotting factor tests, which are required to diagnose a bleeding disorder, reveal the type of hemophilia and how severe it is. They check the levels of factor 8 or factor 9 in the blood, and show whether you have mild, moderate, or severe hemophilia.
How Hemophilia C Is DiagnosedHemophilia C can be diagnosed via genetic testing.
Prognosis of Hemophilia People with hemophilia are likely to have a relatively normal life expectancy and quality of life if they receive the treatment that they need and are knowledgeable about their condition.[10]
Some people with hemophilia develop inhibitors, which are antibodies that the immune system creates to attack the clotting factors in hemophilia treatment.[11] This happens when the body mistakes the clotting factors in the treatment for foreign intruders. Inhibitors can negatively affect the course of hemophilia, but new medications are currently being developed.
[10]
Around 60 to 70 percent of people with hemophilia A have severe hemophilia, and about 15 percent have moderate hemophilia.[10] The rest have a mild form of the condition.
Acquired hemophilia often goes away with treatment.[11]
New Hemophilia Treatment Approved By FDA
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Today, the U.S. Food and Drug Administration approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies).
"Today's approval of Hympavzi provides patients with hemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process," said Ann Farrell, M.D., Director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research. "This new type of treatment underscores the FDA's commitment to advance the development of innovative, safe and effective therapies."
Hemophilia A and hemophilia B are genetic bleeding disorders caused by a dysfunction or deficiency of coagulation factor VIII (FVIII) or IX (FIX), respectively. Patients with these hemophilias are unable to clot properly and may bleed for a longer time than normal after injury or surgery. They may also have spontaneous bleeding in muscles, joints and organs, which can be life-threatening. These bleeding episodes are typically managed by either on-demand, episodic treatment or prophylaxis using products containing FVIII or FIX, or a product that mimics a factor.
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Subscribe for FREEHympavzi is a new type of drug that, rather than replacing a clotting factor, works by reducing the amount, and therefore, the activity of, the naturally occurring anticoagulation protein called tissue factor pathway inhibitor. This increases the amount of thrombin, an enzyme that is critical in blood clotting, that is generated. This is expected to reduce or prevent the frequency of bleeding episodes.
Hympavzi's approval is based on an open-label, multi-center study in 116 adult and pediatric male patients with either severe hemophilia A or severe hemophilia B, both without inhibitors. For the first six months of this study, patients received treatment with replacement factor either on-demand (33 patients) or prophylactically (83 patients). These patients then received Hympavzi prophylaxis for 12 months. The primary measure of efficacy of Hympavzi was the annualized bleeding rates of treated bleeds. In the patients receiving on-demand factor replacement during the first six months of the study, the estimated annualized bleeding rate was 38 compared to the estimated annualized bleeding rate during treatment with Hympavzi of 3.2, showing that Hympavzi was superior to on-demand factor replacement. In the initial six-month period during which patients received prophylactic factor replacement, the estimated annualized bleeding rate was 7.85 and was 5.08 during the subsequent 12 months on Hympavzi prophylaxis, showing that Hympavzi provided similar bleeding rates.
Hympavzi comes with warnings and precautions about circulating blood clots (thromboembolic events), hypersensitivity and embryofetal toxicity.
The most common side effects of Hympavzi are injection site reactions, headache and itching (pruritis).
The FDA granted Hympavzi Orphan Drug designation for this application.
The FDA granted the approval of Hympavzi to Pfizer Inc.
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Hemophilia B Treatment Market Surges With 7.6% CAGR, Set To Hit $8.97 Billion By 2034Fact.MR Report
FACT.MRGlobal Hemophilia B Treatment Market Expected to Taste Huge Growth with Emerging Innovations into the New Treatment Regiment
Rockville, MD, Nov. 25, 2024 (GLOBE NEWSWIRE) -- According to Fact.MR, a market research and competitive intelligence provider, the global Hemophilia B treatment market is estimated to reach a valuation of US$ 4,291.8 million in 2024 and is expected to grow at a CAGR of 7.6% during the forecast period of (2024 to 2034).
The global hemophilia B treatment market is growing rapidly,
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While several strategic partnerships between pharmaceutical firms and research institutions have been announced, the acceleration of treatment landscapes has been furthered. The collaboration between UniQure and CSL Behring on gene therapy production and distribution for hemophilia B led to the start of the launch of Hemgenix in 2022 as the first gene therapy for Christmas disease and is considered a historic milestone in medical science. It is a development that presents one treatment for several years, promising long-term benefits, hence gradually shifting the paradigm from regular infusions to potentially curative therapies. Increased regulatory approvals of gene therapies instill confidence in their wider adoption across the globe.
In addition, the transition to homecare settings and self-administration of factor IX concentrates has significantly empowered patients, engendering a greater level of adherence to prophylactic regimens and quality of life. Pharmaceutical firms are hugely investing in patient-friendly solutions, including extended half-life therapies that reduce infusion frequency from various times a week to once every two weeks for added convenience and greater effectiveness in treatment.
Story Continues
Hemophilia B, though a very rare disorder, is increasing at a gradual rate. According to recent accounts from the World Federation of Hemophilia, approximately 25,000 people worldwide suffer from hemophilia B. This ever-growing patient base with superior diagnosis capabilities and improved healthcare in developing countries will provide an upward thrust to seek treatment options.
In the emerging world, gene therapy will be a life-changing treatment for hemophilia B with the approval of Hemgenix, this may mean a single infusion could potentially provide a long-lasting solution without having to be on lifelong regular replacement therapy for factor IX. Ongoing research and clinical trials continue to refine the gene therapies, promising improved efficacy, reduced side effects, and lower treatment costs in the future.
Therefore, awareness campaigns and the development of patient advocacy organizations have provided better diagnosis rates, improvement in adherence to treatment, and accessing more care. Programs by the National Hemophilia Foundation and other international organizations are increasing support systems for patients suffering from hemophilia B, leading to better outcomes in treatment and creating demand in the market.
Key Takeaways from Market Study
The global hemophilia B treatment market is projected to grow at 6% CAGR and reach US$ 8,965.8 million by 2034
The market created an opportunity of US$ 1,086.4 million growing at a CAGR of 3% between 2019 to 2023
North America is a prominent region that is estimated to hold a market share of 6% in 2034
Predominating market players include uniQure (CSL Behring), Pfizer among others
Hospitals under distribution channel type are estimated to grow at a CAGR of 7% between 2024 and 2034
"Hemophilia B treatment if on the verge of transformative growth with wide treatment modalities and huge investment" says a Fact.MR analyst.
Leading Players Driving Innovation in the Hemophilia B Treatment Market:
Key industry participants like uniQure (CSL Behring); Pfizer; Hoffmann-La Roche Ltd; Octapharma; CSL Behring; Sanofi; Bayer AG; Baxter; Swedish Orphan Biovitrum AB; Grifols International S.A; Kedrion Biopharma, Inc.; Biomarin Pharmaceutical, Inc.; Chugai Pharmaceutical; Biogen, Inc. Etc. Are driving the hemophilia B treatment industry.
Market Development
The companies are also extending their activities to developing regions like the Asia-Pacific, Latin America, and the Middle East, where few state-of-the-art hemophilia treatments have been available until recently, but new opportunities are opening up due to increasing healthcare investments. Expansion in these areas could result in the market share growing 20% by 2028. With the successful introduction of Hemgenix, among other gene therapy candidates under the pipeline, the manufacturing fraternity forecasts that the adoption of gene therapies will grow 10% in the next five years. This will cut overdependence on conventional factor IX replacement therapies and present long-term solutions to patients.
Companies will introduce new recombinant clotting factors with extended half-lives, increasing convenience and thus reducing treatment burden. This factor is expected to give a boost of 15% to the market share for this segment by 2030.
Enhanced partnerships with research bodies would mean several strategic collaborations with various research institutions and universities in the process of discoveries regarding the treatment of hemophilia B. This will ensure that approval processes by regulators are expedited and accelerate innovative therapies to market faster.
In March 2023, the FDA granted Breakthrough Therapy Designation to Novo Nordisk for its novel extended half-life recombinant factor IX currently in development, which is expected to decrease the frequency of infusion to once every three weeks.
June 2023- Pfizer announces the expansion in clinical trials for a new gene therapy candidate for hemophilia B, to be designed to deliver steady-state Factor IX expression levels with further improvement of long-term patient outcomes.
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More Valuable Insights on Offer
Fact.MR, in its new offering, presents an unbiased analysis of the global Hemophilia B treatment market, presenting historical data for 2019 to 2023 and forecast statistics for 2024 to 2034.
The study reveals essential insights on the basis of the by treatment type (recombinant factor IX concentrates, plasma-derived factor IX concentrates, gene therapy, and extended half-life Factor IX) by patient demographics (pediatrics, adults) by severity of hemophilia B (mild hemophilia B, moderate hemophilia B, and severe hemophilia B) by distribution channel (hospitals, specialty clinics, and online pharmacies) across major regions of the world (North America, Latin America, Western Europe, Eastern Europe, East Asia, South Asia, and Pacific, and Middle East & Africa).
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