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Abnormal Uterine Bleeding May Be Caused By Clotting Factor Deficiencies

A new review article illuminates why physicians should keep coagulation disorders in mind when evaluating patients with heavy abnormal uterine bleeding.

Physicians treating patients with heavy menstrual bleeding (HMB) should consider the possibility that rare bleeding disorders and hemophilia carrier states are at play if other, more common causes of bleeding have been excluded, according to a new report.

The report, a literature review published in Life (Basel),1 outlines several coagulation disorders and offers physicians guidelines on how to spot and treat hemostatic disorders.

Authors explain that current literature suggests 10% to 35% of women experience HMB at some point during their reproductive years. There are several potential causes of such bleeding, and physicians can use the PALM-COEIN system to help identify the underlying cause.

This system of abnormal uterine bleeding classification, from the International Federation of Gynaecology and Obstetrics,2 includes structural causes like polyps, adenomyosis, leiomyoma, malignancy, and hyperplasia, and functional causes, including coagulopathy, ovulatory disorder, endometrial disorders, iatrogenic conditions, or not-yet-classified causes. The existing literature suggests that an underlying inherited bleeding disorder is at play for 10% to 62% of adolescents with HMB. The most common such disorder is von Willebrand disease, but the authors said several other, less-common conditions should also be considered. Those less-common conditions are the focus of the review article.

The authors first reviewed several rare coagulation factor disorders. For instance, they said factor I, or fibrinogen, is a soluble plasma glycoprotein that in some patients is absent (congenital afibrinogenemia) or reduced (hypofibrinogenemia). In the former, patients can have a wide range of clinical manifestations, from minimal bleeding to serious hemorrhages, the authors said. Such patients may also experience arterial or venous thrombosis. In coagulation tests, partial thromboplastin time (PTT), prothrombin time (PT), thrombin time (TT) are all "infinitely prolonged," the authors said, since all of the parameters require the formation of fibrin.

Patients with hypofibrinogenemia are often asymptomatic if their fibrinogen levels are above 1 g/L, but the authors said these patients sometimes experience bleeding or thrombotic complications.

"In affected individuals, PT, PTT, and TT are variably prolonged, with TT being the most sensitive assay," they said.

Factor VII deficiency is believed to make up about one-third of rare coagulation disorders. The factor is vitamin K dependent and, like factor I, is synthesized in the liver and secreted in the plasma, the authors said. Patients with a deficiency of factor VII can have a wide range of manifestations, the authors said, "from asymptomatic or mildly symptomatic cases—for example, easy bruising, gum bleeding, and epistaxis—to severe cerebral and gastrointestinal hemorrhage."

Unfortunately, there are no clear treatment guidelines for factor VII deficiency, the authors said, although the published literature suggests a number of potential treatments, including surgery and blood transfusions.

Factor XIII deficiency is one of the rarest coagulation disorders, with only 500 cases reported globally since 1960, the authors said.

"Factor XIII is a transglutaminase that cross-links fibrin fibers between amino acid residues, hence stabilizing a fibrin clot," they wrote.

Patients with the deficiency can present with abnormal uterine bleeding, umbilical bleeding, or severe ovulation bleeding, among other clinical manifestations. However, the authors said factor XIII deficiency can be difficult to diagnose because clotting factor tests and platelet counts are normal in patients with the deficiency.

"The diagnosis can be made by measuring FXIII antigen levels using enzyme-linked immunosorbent assays and/or by measuring its activity with functional methods," the authors said. "Genetic testing of the genes encoding FXIII A or FXIII B is also an option for more specific assessment."

The authors also described several other disorders, including deficiencies of factors V, VIII, X, and XI, and hemophilia.

The investigators said they hope their report serves as a "useful guide" for physicians investigating abnormal uterine bleeding.

"We hope that this review can serve as a roadmap for busy clinicians to familiarize themselves with coagulation factor disorders as a cause of heavy menstrual bleeding and guide them towards more targeted research," they said.

They added, however, that theirs was not a systematic review, and therefore it should not be considered an exhaustive guide to diagnostic decisions.

References

1. Livanou ME, Matsas A, Valsami S, Papadimitriou DT, Kontogiannis A, Christopoulos P. Clotting factor deficiencies as an underlying cause of abnormal uterine bleeding in women of reproductive age: a literature review. Life (Basel). Published online June 5, 2023. Doi:10.3390/life13061321

2. Munro MG, Critchley HOD, Broder MS, Fraser IS; FIGO Working Group on Menstrual Disorders. FIGO classification system (PALM-COEIN) for causes of abnormal uterine bleeding in nongravid women of reproductive age. Int J Gynaecol Obstet. 2011;113(1):3-13. Doi:10.1016/j.Ijgo.2010.11.011

Recombinant Coagulation Factors Market To Be Worth $24.21 Billion By 2030 - Exclusive Report By Meticulous Research®

Meticulous Market Research Pvt. Ltd.

Redding, California, July 05, 2023 (GLOBE NEWSWIRE) -- According to a new market research report titled, 'Recombinant Coagulation Factors Market by Type (Factor VIII, Factor IX), Source (CHO, HEK), Application (Hemophilia (Type A, Type B), Others), and End User (Hospitals, Clinical Research Laboratories) - Global Forecast to 2030,' published by Meticulous Research®, the recombinant coagulation factors market is projected to reach $24.21 billion by 2030, at a CAGR of 8.6% from 2023 to 2030.

Recombinant coagulation factors are glycoproteins produced by recombinant DNA technology. They are majorly used for the treatment of various coagulation disorders. They are gaining popularity due to their benefits such as reduced risk of bloodborne disease transmission and effective treatment.

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The increasing prevalence of hemophilia and other bleeding disorders, growing R&D for coagulation factors, growing awareness about the benefits of the recombinant coagulation factors and increasing prophylactic treatment for hemophilia across the globe are driving the growth of the recombinant coagulation factors market. Furthermore, the potential emerging economies and rising healthcare expenditure are expected to offer significant growth opportunities for the recombinant coagulation factors market.

However, the high cost of recombinant factors compared to plasma-derived ones and the limited accessibility and availability of recombinant clotting factors in developing & underdeveloped countries are expected to restrain the growth of this market. Additionally, manufacturing challenges in the production of recombinant clotting factors, stringent regulatory requirements, lack of reimbursement in some countries, and lack of awareness among patients are some of the challenges to the growth of this market.

Increased Prevalence of Infectious Diseases and Rising Need to Reduce Transmission of Bloodborne Disease to Drive the Growth of this Market

There is a rising prevalence of infectious diseases globally. Infectious diseases such as hepatitis B virus, hepatitis C virus and human immunodeficiency virus are highly infectious and can be transmitted through blood. Utilizing blood-derived coagulation factors carries a high risk of transmitting infectious diseases. This has created a need for safer treatment options for the treatment of various coagulation disorders.  In this regard, recombinant factor VIII was first introduced in the 1980s. Recombinant coagulation factors are not derived from blood and are manufactured in the laboratory using recombinant DNA technology. This avoids the risk of transmission of bloodborne disease and facilitates safer treatment for various coagulation disorders.

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The key players profiled in this market study are Baxter International Inc. (U.S.), Grifols, S.A. (Spain), CSL Limited (Australia), Octapharma AG (Switzerland), Novo Nordisk A/S (Denmark), Biogen Inc. (U.S.), Bayer AG (Germany), Kedrion S.P.A. (Italy), Emergent BioSolutions (U.S.), and Pfizer Inc. (U.S.).

The recombinant coagulation factors market is segmented based on Type (Recombinant Factors VIII, Recombinant Factor IX, and Other Types), Source (Chinese Hamster Ovary (CHO) Cell Line, Human Embryonic Kidney (HEK) Cell Line, and Other Sources), Application (Hemophilia A, Hemophilia B, and Other Applications), End User (Hospitals & Clinics and Clinical Research Laboratories), and Geography. The study also evaluates industry competitors and analyzes their market shares at the country and regional levels.   The study also provides valuable insights into the key growth strategies adopted by major market players in the last three to four years.

Based on type, in 2023, the recombinant factor VIII segment is expected to account for the largest share of the recombinant coagulation factors market. The large market share of this segment is attributed to the high prevalence of haemophilia A, the high efficacy of recombinant factors VIII, and an established market presence of recombinant factors VIII products.

Based on source, in 2023, the Chinese hamster ovary (CHO) cell line segment is expected to account for the largest share of the recombinant coagulation factors market. The large market share of this segment is attributed to the high popularity of the CHO cell lines for the production of biologics, the high similarity of the CHO cells to the human cell system, and high reproducibility.

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Based on application, the hemophilia A segment is estimated to account for the largest share of the recombinant coagulation factors market. The large market share of this segment is attributed to factors such as the increased number of patients with hemophilia A, increased awareness about advanced treatment options, and the high efficacy of recombinant coagulation factors for the treatment of hemophilia A.

Based on end user, in 2023, the hospitals & clinics segment is estimated to account for the largest share of the recombinant coagulation factors market. The large market share of this segment can be attributed to the comparatively higher adoption of recombinant coagulation factors by hospitals & clinics, rising healthcare expenditure, and growing awareness among healthcare professionals.

Based on geography, in 2023, North America is expected to account for the largest share of the recombinant coagulation factor market, followed by Europe and Asia-Pacific. The large share of this market is primarily attributed to the robust healthcare infrastructure in the region, high healthcare expenditure, technological advancements, favorable reimbursement policies, high awareness, and the presence of key market players in the region.

However, Asia-Pacific is expected to witness rapid growth during the forecast period. The rapid growth of this market is driven by the increasing hereditary disorders, the increasing investments in healthcare in developing countries such as China and India, and the growing preference for recombinant technology owing to advantages compared to traditional are driving the growth of the recombinant coagulation factors market in Asia-Pacific.

The report also includes an extensive assessment of the key growth strategies adopted by leading market players in the past three to four years. In the last couple of years, the recombinant coagulation factors market has witnessed various developments.

To gain more insights into the market with a detailed table of content and figures, click here: https://www.Meticulousresearch.Com/product/recombinant-coagulation-factors-market-1254

Scope of the Report:

Recombinant Coagulation Factors Market Assessment - by Type

Recombinant Factor VIII

Recombinant Factor IX

Other Types

Recombinant Coagulation Factors Market Assessment - by Source

Recombinant Coagulation Factors Market Assessment - by Application

Hemophilia A

Hemophilia B

Other Applications

Recombinant Coagulation Factors Market Assessment - by End User

Recombinant Coagulation Factors Assessment - by Geography

North America

Europe

Germany

France

Italy

U.K.

Spain

Rest of Europe (RoE)

Asia-Pacific

China

Japan

India

Rest of Asia-Pacific

Latin America

Middle East & Africa

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$2.9 Million Gene Therapy For Severe Hemophilia Is Approved By FDA

WASHINGTON -- U.S. Officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, a $2.9 million infused treatment that can significantly reduce dangerous bleeding problems.

The Food and Drug Administration approved Roctavian for adult patients with severe cases of hemophilia A, the inherited blood-clotting disorder that can lead to bleeding after minor injuries or scrapes. It's the first gene therapy for those patients.

The IV therapy is a long-awaited alternative to current treatments, including weekly doses of a protein needed to help blood clot. Some patients take a newer, longer-acting biotech drug that replaces the protein.

BioMarin said in a statement that the FDA approval was based on a three-year study showing a 50% reduction in annual bleeding incidents among 134 patients who received the treatment. Most patients continued to respond to the treatment beyond three years, without needing regular IV infusions, the company said.

BioMarin said Roctavian's $2.9 million price tag reflects "the possibility of freedom from years" of infusions, which cost about $800,000 annually for a typical patient. The price is less than the $3.5 million announced last year for a similar gene therapy for hemophilia B, a less common form of the disease.

Like most medicines in the U.S., the new treatment will mainly be paid for by insurers, not patients.

BioMarin said it estimates about 2,500 U.S. Patients will be eligible to receive the therapy under the terms of the FDA's approval. Patients with certain underlying health problems and conditions are excluded under the prescribing information.

Hemophilia is caused by mutations that prevent the production of proteins needed for blood clotting. Hemophilia A is the most severe variant of the condition, and some patients can experience spontaneous bleeding even without any injury. Left untreated, the condition can cause bleeding that seeps into joints and organs, including the brain.

Roctavian uses an inactivated virus, created in a lab, to deliver a replacement gene to the liver cells that produce the clotting protein. When the therapy is successful, patients can then produce the protein themselves. The label warns that rare, severe allergic reactions can occur.

Dr. Margaret Ragni called Roctavian "a major improvement in terms of reducing the burden of disease." But she notes that many patients are comfortable with their current treatments and may be hesitant to try a new gene therapy.

"I think there's a group that will want to do this, but patients need to hear what the risks and benefits are," said Ragni, who treats patients at the Hemophilia Center of Western of Pennsylvania in Pittsburgh.

BioMarin was among the first companies to begin testing an experimental gene therapy in patients more than six years ago.

The San Rafael, California-based company excluded patients with certain potentially complicating conditions, including liver disorders and resistance to the standard blood clotting protein, which often develops in some hemophilia patients. BioMarin's president for research and development, Dr. Henry Fuchs, said the company is conducting studies in some of those excluded groups to see if they can safely receive the therapy.

Another key question is how long the therapy's benefits last. BioMarin has followed the patients for more than three years and they continue to experience reduced bleeding. But levels of the clotting protein in the bloodstream fall over time, suggesting additional treatments may eventually be needed.

"Let's make sure the expectation isn't that this is taken one time, forever and it will work perfectly for the rest of your life," Fuchs said, adding "at some point we'll know a lot more about durability."

Roctavian was approved in Europe last August, but the therapy has faced pushback from government health programs over its cost.

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The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education. The AP is solely responsible for all content.

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