The evolving role of genetic tests in reproductive medicine

Hematology: Heredity & Clotting Factors

TIME

December 27, 1963 12:00 AM GMT-5

All that most laymen know about hemophilia is that it is an odd and dangerous disease of the blood that strikes men only, although it is transmitted by women. Medical researchers have learned a great deal more than that, but when the nation's expert hematologists gathered in Washington, they realized that much more was still to be learned. They are baffled by at least eight bleeding disorders that are caused by the lack of any one of ten different clotting factors in the blood.

Genetic Lottery. Classical hemophilia, known since ancient times, is caused by a severe shortage of clotting Factor VIII. This disease, which afflicted a dozen descendants of Queen Victoria, results from a defect in a recessive gene carried on the x (female) chromosome. If a hemophilic man marries a normal woman, all their sons are normal but all their daughters are carriers. If a carrier woman marries a normal man (see diagram), each son has a fifty-fifty chance of being a victim and each daughter has a fifty-fifty chance of being a carrier. No one can predict whether a child will be affected, because a sort of genetic lottery decides which of the mother's chromosomes the child inherits. (How Victoria got the defective gene is a mystery. Medical sleuthing has failed to show that she inherited it, so she may have developed it herself—perhaps even from a direct hit on her ovaries by cosmic rays.†)

Classical hemophilia has now been renamed hemophilia A, because in 1952 a boy in England was found to be suffering from what had seemed to be the same disease, but his trouble was actually caused by lack of clotting Factor IX. This affliction is now called hemophilia B. It is transmitted the same way as hemophilia A, but the two diseases can be distinguished by the fact that blood from a hemophilia A victim, which contains Factor IX, will clot blood from a hemophilia B victim. A hemophilia B's blood, with its Factor VIII, will make an A's blood clot. Of an estimated 100,000 victims of clotting disorders in the U.S., 50,000 have hemophilia A and 15,000 have B.

Another bleeding disorder that is similar in its effects on patients, but totally different in inheritance and incidence is von Willebrand's disease. This is the most common of a group of clotting disorders that are transmitted by genes on some other chromosome than the x—which chromosome, remains a mystery. But it is a nonsex chromosome, so boys and girls are equally likely to inherit the defective gene.

No Direct Measure. "The trouble is," said the University of North Carolina's Dr. Kenneth M. Brinkhous, director of the U.S.'s leading hemophilia research program, "we can only measure the clotting factors indirectly by their effects—usually in the test tube."

But progress is advancing from the lab to the bedside. Factor VIII is now being extracted from human plasma and concentrated about 30 times. It is given by intravenous drip to victims of hemophilia A and von Willebrand's disease when they have crises of massive bleeding. Except in such emergencies, the usual treatment for all the clotting disorders remains a transfusion of fresh whole blood or plasma—not to replace blood that the patient has lost, but to supply the missing clotting factor and thus keep him from losing more.

Contrary to common belief, it is not outward bleeding from a wound that cripples and eventually kills most hemophiliacs; it is internal bleeding, especially into the joints, that does the damage. "This," said Manhattan's Dr. Henry H. Jordan, "is more crippling than either polio or arthritis. But it's incredible what rehabilitation can do. Many patients can discard a brace, for example, after five or even ten years." Today, some hemophiliacs work as longshoremen and loggers.

† She passed the gene to one son, two daughters: Leopold, who died at 31, was grandfather of Lord Trematon, who died at 21; Alice, who married Prince Louis of Hesse, had one hemophilic son who died at three, and two carrier daughters; Beatrice, who married Prince Henry of Battenberg, had two affected sons and a carrier daughter. Alice's elder carrier daughter Irene married Prince Henry of Prussia; one hemophilic son, Waldemar, lived to 56, but another died at four; Alice's younger carrier daughter Alexandra married Czar Nicholas II, was murdered with him and their hemophilic son, the Czarevitch Alexis. Beatrice's daughter Victoria Eugenie married Alfonso XIII of Spain, had two affected sons, Alfonso and Gonzalo, who bled to death after accidents.

Medications And Drugs For Treatment Of Von Willebrand Disease

von Willebrand's Disease (vWD) is an autosomal dominant inherited bleeding disorder that occurs from birth and affects both sexes.

Antihemophiliac Factor

Antihemophiliac Factor is an essential blood-clotting factor, prescribed for controlling and preventing bleeding episodes in people with low levels of factor VIII (hemophilia A) and AHG deficiency.

Caplacizumab-yhdp

Caplacizumab is prescribed for treating acquired thrombotic thrombocytopenic purpura (aTTP)

Bleeding Disorders Treatment Market Projected To Reach USD 35.17 Billion By 2032, Growing At A Robust CAGR Of 8.91%

(MENAFN- EIN Presswire)

Bleeding Disorders treatment Market

Driven by Biopharmaceutical Innovations and Gene Therapies, the market Booms Amidst Growing Incidence of Hemophilia and Von Willebrand Disease

AUSTIN, TX, UNITED STATES, January 23, 2025 /EINPresswire / -- According to SNS Insider, the global Bleeding Disorders Treatment Market , valued at USD 16.35 billion in 2023, is projected to grow to USD 35.17 billion by 2032 at a CAGR of 8.91% during the forecast period of 2024-2032.

Advancing Therapies Propel Growth in the Bleeding Disorders Treatment MarketThe bleeding disorders treatment market is undergoing significant growth due to the rising prevalence of hemophilia, Von Willebrand disease (vWD), and various other uncommon clotting disorders. Hemophilia particularly types A and B, impacts around 1 in 5,000 male births globally, driving the need for innovative treatments that enhance patient results. Recent advancements, including CSL's gene therapy etranacogene dezaparvovec-drlb and Takeda's recombinant-krhn for ADAMTS13, have established new standards in the treatment of hemophilia B and congenital thrombotic thrombocytopenic purpura (cTTP). Novo Nordisk has also made progress with Mim8 and concizumab to prevent hemophilia. The increase in biopharmaceutical advancements, such as recombinant coagulation factors, gene therapies, and continuing clinical trials, is anticipated to propel significant market growth, revolutionizing treatment for both common and rare bleeding disorders.

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Key Players in Bleeding Disorders Treatment Market.Lonza Group AG Suppliers.Thermo Fisher Scientific Inc. Suppliers.Merck KGaA (MilliporeSigma) Suppliers.Cytiva Suppliers.Bio-Rad Laboratories, Inc. Suppliers.Sartorius AG Suppliers.Charles River Laboratories International, Inc. Suppliers.WuXi Biologics Suppliers.Octapharma AG Suppliers.Catalent, Inc. Suppliers

Key Segments Analysis

By TypeIn 2023, the Hemophilia A segment dominated the bleeding disorders treatment market, holding a 55% market share. This prevalence is due to the reality that Hemophilia A makes up 80% of all hemophilia instances worldwide, impacting around 1 in 5,000 male births. The high occurrence of this condition creates a strong need for effective therapies. Progress in recombinant therapies and gene therapies aimed explicitly at Hemophilia A has strengthened its market dominance, providing enhanced efficacy and superior patient results.

The Von Willebrand Disease (vWD) segment has emerged as the fastest-growing segment in the market, expected to attain the highest CAGR throughout the forecast period. As the most prevalent hereditary bleeding condition, vWD influences around 1 in 1,000 people globally. The growth of the segment is driven by increasing diagnosis rates and access to advanced treatment choices, especially for critical cases. Growing awareness, improved diagnostic methods, and the introduction of new treatments specifically designed for vWD is anticipated to lead to substantial growth in this area, catering to a historically neglected patient group.

By Drug ClassIn 2023, the Recombinant Coagulation Factor Concentrates segment dominated the bleeding disorders treatment market, holding a 40% market share. These products have established themselves as the standard therapy for Hemophilia A and B because of their high effectiveness, improved safety profiles, and capability to efficiently prevent bleeding incidents. Recombinant therapies have predominantly supplanted plasma-derived treatments because they substantially lower the risk of transmitting blood-borne pathogens. Furthermore, their elevated purity levels and lasting advantages, like decreased injection frequency, render them a favored option among both healthcare professionals and patients.

The desmopressin segment is the fastest growing segment in the bleeding disorders market, fueled by its rising application in managing mild Hemophilia A and Von Willebrand Disease (vWD). Desmopressin is highly preferred due to its cost-effectiveness, simple administration, and demonstrated success in enhancing clot formation in individuals with less severe bleeding issues. Its increasing use, especially in areas with restricted availability of expensive treatments, is driving its growth. With growing recognition of its advantages, desmopressin is emerging as a crucial choice for managing mild bleeding disorders, facilitating its swift market growth.

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Bleeding Disorders Treatment Market SegmentationBy Type.Hemophilia A.Hemophilia B.Von Willebrand Disease (vWD).Others

By Drug Class.Plasma Derived Coagulation Factor Concentrates.Recombinant Coagulation Factor Concentrate.Desmopressin.Antifibrinolytics.Fibrin Sealants.Other bleeding Disorders Treatment

By End-Users.Hospital.Clinics.Research Institutes.Others

Regional OutlookIn 2023, North America dominated the bleeding disorders treatment market, accounting for a 40% market share due to the high prevalence of hemophilia and other bleeding disorders, advanced healthcare infrastructure, and the adoption of innovative treatments. Large markets, especially the United States, with its significant patient pool and tier pharmaceutical companies, are vigorously advancing next-generation innovative therapeutics such as gene therapies and recombinant factor concentrates. Bleeding disorder patients in the region have benefited from a comprehensive healthcare system, robust insurance coverage, and access to specialized medical centers. Additionally, the FDA's accelerated approvals and the ongoing research in the U.S. And Canada further assist in speeding up treatment innovation and market growth.

The Asia Pacific is poised to be the fastest-growing region of the bleeding disorders treatment market, due to rising awareness, improving healthcare infrastructure, and enhanced diagnostic capabilities. Emerging economies-particularly China and India-are experiencing rapid market growth as access to advanced therapies including recombinant coagulation factor concentrates and desmopressin is expanding to reach potentially large underserved populations. This growth is attributed to the increasing healthcare costs and the growing public health movements for the treatment and management of rare bleeding disorders.

Recent Developments.In October 2024, Novo Nordisk announced that its groundbreaking product, concizumab, received a positive recommendation from the EMA's Committee for Medicinal Products for Human Use (CHMP). Upon approval by the European Commission, concizumab would become the first once-daily, subcutaneous prophylactic treatment for hemophilia A or B with inhibitors in patients aged 12 and older..December 2023, Global biotechnology leader CSL unveiled positive three-year results from the pivotal HOPE-B study, highlighting the long-term durability and safety of its gene therapy, etranacogene dezaparvovec-drlb, in patients with hemophilia B.

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Table of Contents – Major Key Points1. Introduction2. Executive Summary3. Research Methodology4. Market Dynamics Impact Analysis5. Statistical Insights and Trends Reporting6. Competitive Landscape7. Bleeding Disorders Treatment Market by Type8. Bleeding Disorders Treatment Market by Drug Class9. Bleeding Disorders Treatment Market by End-Users10. Regional Analysis11. Company Profiles12. Use Cases and Best Practices13. Conclusion

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