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Hemophilia News

2 New Findings Offer Hope for Those With Severe Hemophilia

Posted 26 May 2016 by Drugs.com

WEDNESDAY, May 25, 2016 – Two new studies could pave the way to major changes in how doctors treat severe cases of hemophilia – a rare genetic disorder that can cause uncontrolled bleeding. Both studies tackle a key challenge: Up to one-third of children with severe hemophilia develop antibodies against the standard therapy. But one study highlights the value of an old therapy, while the other shows promising early results with an experimental drug. Experts said both should stir discussion among doctors, patients and parents who deal with hemophilia. But they were especially hopeful about the new drug, known as emicizumab. In the United States, about 20,000 people – mostly boys and men – are living with hemophilia, according to the U.S. Centers for Disease Control and Prevention. The disorder is caused by a defect in one of the genes that controls proteins needed for normal blood ... Read more

Related support groups: Blood Disorders, Bleeding Disorder, Anemia, Blood Transfusion, Hemophilia A, Hemophilia, Coagulation Defects and Disorders, Hemophilia B, Hemophilia A with Inhibitors, Blood Cell Transplantation

Review Finds Mixed Success With Hemophilia Treatment

Posted 16 Mar 2016 by Drugs.com

WEDNESDAY, March 16, 2016 – Though the past 50 years have brought major treatment advances, men with severe hemophilia are still at high risk for bleeding and physical disability, experts say. Hemophilia is a genetic disease that prevents blood from clotting normally, leading to an increased risk of serious bleeding. More common in men than in women, it affects about one out of every 5,000 men, according to the U.S. Centers for Disease Control and Prevention. Researchers analyzed data from nearly 7,500 male hemophilia patients in the United States between 1998 and 2011. Their findings were published online March 16 in the journal Blood. "Our analysis provides a snapshot of how improvements in care have translated into outcomes across different generations of men with hemophilia," study author Dr. Paul Monahan said in a journal news release. Monahan is a former professor of hematology ... Read more

Related support groups: Blood Disorders, Bleeding Disorder, Hemophilia A, Hemophilia, Coagulation Defects and Disorders, Hemophilia B, Hemophilia A with Inhibitors

FDA Approves Nuwiq (Antihemophilic Factor [Recombinant]) for Hemophilia A

Posted 17 Sep 2015 by Drugs.com

HOBOKEN, N.J. September 15th, 2015: Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved Nuwiq, Antihemophilic Factor (Recombinant), an intravenous therapy for adults and children living with Hemophilia A. The Nuwiq approval includes on-demand treatment and control of bleeding episodes; routine prophylaxis to reduce the frequency of bleeding episodes; and perioperative management of bleeding. Nuwiq is the first B-domain deleted recombinant Factor VIII (FVIII) derived from a human cell-line, not chemically modified or fused with another protein, designed for the treatment of patients with Hemophilia A, congenital FVIII deficiency. Hemophilia A impacts the lives of up to 16,000 individuals in the U.S. and their caregivers. Although present therapies for Hemophilia A treatment exist in the U.S., significant challenges still remain, including development ... Read more

Related support groups: Hemophilia A, Hemophilia, Antihemophilic Factor, Nuwiq

Kids' Hemophilia Drugs a Big Part of State Medicaid Spending

Posted 31 Jul 2015 by Drugs.com

FRIDAY, July 31, 2015 – Treatment costs for one childhood illness, hemophilia, may use up a big chunk of a state's Medicaid budget, a new study out of California shows. The researchers found that treatments for hemophilia – a rare, inherited disorder in which blood does not clot normally – accounted for the largest share of spending on outpatient drugs among publicly insured children in California with serious chronic illnesses. The study "underscores the potential effect of new, expensive but [effective] pharmaceuticals on public insurance programs for children with chronic illness," wrote a group led by Sonja Swenson of Stanford University. Her team published the findings July 28 in the Journal of the American Medical Association. The researchers tracked 2010-2012 data from more than 34,300 publicly insured children and young adults under the age of 21 in California with serious ... Read more

Related support groups: Hemophilia A, Hemophilia B, Hemophilia, Antihemophilic Factor, Advate, ReFacto, Fanhdi, Monarc-M, NovoEight, Koate-DVI, Haemate P, Monoclate-P, Obizur, Alphanate, Kogenate, Helixate FS, Recombinate, Bioclate, Kogenate FS, Antihemophilic Factor/Von Willebrand Factor

FDA Approves Obizur [Antihemophilic Factor (Recombinant)] for Acquired Hemophilia A

Posted 27 Oct 2014 by Drugs.com

DEERFIELD, Ill., October 24, 2014 - Baxter International Inc. today announced that the United States Food and Drug Administration (FDA) has approved Obizur [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired hemophilia A (AHA), a very rare and potentially life-threatening acute bleeding disorder. Obizur was granted orphan-drug status by the FDA and its review was prioritized based on AHA's classification as a rare disease and the potential for the treatment to address an important unmet medical need. Obizur is the first recombinant porcine FVIII treatment approved for AHA that allows physicians to manage the treatment's efficacy and safety by measuring factor VIII activity levels in addition to clinical assessments. Obizur replaces the inhibited human factor VIII with a recombinant porcine sequence factor VIII based on ... Read more

Related support groups: Hemophilia A, Hemophilia, Antihemophilic Factor

New Treatment Obizur Approved for Rare Form of Hemophilia

Posted 24 Oct 2014 by Drugs.com

FRIDAY, Oct. 24, 2014 – Obizur (antihemophilic factor recombinant) has been approved to treat a rare, non-inherited form of hemophilia in adults. Unlike the more common form of hemophilia that's inherited and affects males, acquired hemophilia affects both males and females. The rarer form of the blood disorder occurs when the body's immune system attacks a protein that's necessary for normal blood clotting. About half the cases of acquired hemophilia are related to other medical conditions, such as pregnancy, cancer or use of certain medications. But about half of cases have no known cause, the FDA said Friday in a news release. Obizur was evaluated in clinical studies involving 29 adults with acquired hemophilia, who were given Obizur to treat a serious bleeding episode. The trial didn't identify any safety concerns, the FDA said. Obizur is manufactured by Baxter Healthcare, based in ... Read more

Related support groups: Hemophilia

'Female' X Chromosome May Play Part in Sperm Production

Posted 22 Jul 2013 by Drugs.com

SUNDAY, July 21 – Researchers have made the surprising finding that the "female" X chromosome may have an important function in sperm production. Women have two X chromosomes and men have an X and Y chromosome. This study found that large portions of the X chromosome have evolved to play a specialized role in producing sperm. And despite its reputation as the most stable chromosome of the genome, the X chromosome has actually been undergoing relatively rapid changes, according to the study published online July 21 in the journal Nature Genetics. Taken together, these two findings suggest that it's time to reexamine the biological and medical importance of the X chromosome, said the researchers at the Whitehead Institute for Biomedical Research in Cambridge, Mass. "We view this as the double life of the X chromosome," Whitehead director David Page said in an institute news release. "The ... Read more

Related support groups: Oligospermia, Hemophilia, Color Vision Defect (Acquired)

FDA Approves Rixubis - First Recombinant Coagulation Factor IX For Use in Preventing Bleeding Episodes

Posted 27 Jun 2013 by Drugs.com

June 27, 2013 – The U.S. Food and Drug Administration yesterday approved Rixubis [Coagulation Factor IX (Recombinant)] for use in people with hemophilia B who are 16 years of age and older. Rixubis is indicated for the control and prevention of bleeding episodes, perioperative (period extending from the time of hospitalization for surgery to the time of discharge) management, and routine use to prevent or reduce the frequency of bleeding episodes (prophylaxis). An inherited blood clotting disorder mainly affecting males, Hemophilia B is caused by mutations in the Factor IX gene and leads to deficiency of Factor IX. Hemophilia B affects about 3,300 people in the United States. Individuals with Hemophilia B can experience potentially serious bleeding, mainly into the joints, which can be destroyed by such bleeding. “As the first recombinant coagulation factor IX indicated s ... Read more

Related support groups: Coagulation Defects and Disorders, Hemophilia B, Hemophilia, Coagulation Factor Ix

Kids With Hemophilia Should Be Active, But Avoid Risky Sports: Study

Posted 9 Oct 2012 by Drugs.com

TUESDAY, Oct. 9 – Children and adolescents with the bleeding disorder hemophilia are at increased risk for bleeds when they participate in some contact sports, but the overall risk is low and varies by sport, a new study shows. People with hemophilia lack one of the blood proteins necessary for clotting. Because clotting helps stop bleeding, people with hemophilia may bleed longer than people without this disorder. For a long time, they were told that they could not and should not exercise due to risks of bleeding. Times have changed, however, and thanks to better treatments, many are now encouraged to play some sports. The new study, published in the Oct. 10 issue of the Journal of the American Medical Association, puts real numbers on risks associated with specific sports for children and adolescents with hemophilia. Researchers looked at the increased risk for bleeding associated ... Read more

Related support groups: Hemophilia

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Hemophilia A, Hemophilia B, Coagulation Defects and Disorders