SPK-8011 for Hemophilia A

"The number of cases of hemophilia A is increasing due to growing incidence and prevalence estimates, changing life-styles, and improving medical knowledge. Consequently, new forms of treatment to improve the quality of life of patients with haemophilia and to improve survival remain required. The availability of purified clotting factors for both prophylaxis and therapeutic purposes and the development of plasma fractionation techniques are two important innovations that have helped to improve survival rates." - Anonymous Online Contributor

"Hemophilia is a genetic disease that creates a severe form of blood clotting disorder. It manifests itself primarily through the skin and joints as bruises and sores. While there is still currently no cure, a vaccine is being researched for people with both sickle cell disease and hemophilia. Without the use of a vaccine, there is no way to prevent a hemophiliacs life from coming to an end.\n\n- http://www.nhs.gov.au/health/education/learn/healthprofessionals/medical_professions.html\n- https://web.archive.org/web/20070717025343/http://www.med." - Anonymous Online Contributor

"Although the cause of hemophilia is still not well understood, it is now known that a deficiency in the production of clotting factors leads to a hemorrhagic tendency. Patients with hemophilia type A are mostly affected by the most severe form of hemorrhagic disease of the newborn, and they develop hemophilia as a result of inheriting a faulty allele of the factor VIII gene. Those who do not inherit this mutation will have milder bleeding issues. Type B hemophilia, which occurs more frequently, is caused by a mutation in factor IX gene, leading to a deficiency in factor IX, a coagulation factor." - Anonymous Online Contributor

"The most common symptoms are painful or swollen joints, bone pain and joint instability. Other signs include bleeding into muscle or nose, easy bruising and bleeding from the joints. Bleeding into tendon is rare and is associated with an underlying problem rather than an underlying hemophilia. All patients with an underlying, active bleeding problem should have a bleeding history. In children, the condition can present differently. When a child presents with symptoms of bleeding after an injury, this may be a sign for the presence of hemophilia.\n" - Anonymous Online Contributor

"Around 45,000 American boys and girls have hemophilia A; 4 in 10 men and 2 in 10 women who meet the symptoms of hemophilia are asymptomatic. Hemophilia is a rare disorder that the CDC will need to add to its list of recommended childhood diseases." - Anonymous Online Contributor

"We suggest that hemophilia A be treated in a specialty center in the U.S. as soon as possible after contracting the disease since the risk of fatal hemorrhage of the joints is high in a newly diagnosed patient with hemophilia A. The optimal therapy that minimizes the risk of hemorrhage of the joints would be to treat the patient before a major joint is damaged so that surgery may be avoided if it can. These patients should have a life expectancy similar to that of the rest of the population." - Anonymous Online Contributor

"Hemophilia A is an almost non-existent disorder, and there have been no reported cases of fatal hemophilia A for many years because of the availability of anti-hemophilia A drugs and improved monitoring methods. These developments have led to an increased need for new treatments for this rare disorder. As well as the traditional treatments, the use of recombinant coagulation factors, as well as synthetic hemostatic drugs (hemostatic dressings), may be beneficial in some cases. Additionally, specific gene therapy procedures may allow for a cure for hemophilia A by halting the production of the protein the deficient (defective) hemoglobins." - Anonymous Online Contributor

"Significant progress has been made in the field, including the development of new recombinant products containing an FVIII peptide. The majority of people with hemophilia now use recombinant products in their treatment and are able to live normal lives. In addition, an effective therapeutic and prophylactic treatment for people with hemophilia B by somatic recombinant retroviral gene therapy is available." - Anonymous Online Contributor

"spk-8011 should be used as an alternative to conventional drugs used in HA for treatment of haemophilia during surgery and on prophylaxis. In this context, spk-8011, which has some of the advantages inherent in HA, should be used in the shortest time possible to avoid post-transfusion haemorrhaging, thereby achieving maximal benefit." - Anonymous Online Contributor

"Patients with hemophilia A typically get their diagnosis around age 3, but there is a wide range of age at diagnosis ranging between 1 and 25. There is a small percentage of patients with hemophilia A who do not develop immunothrombocytopenia. The median age of diagnosis was 11.5 years and the average age of development of bleeding symptoms was 15.1 years. The major bleeding episodes for patients with hemophilia were spontaneous. There were no reported bleeding episodes related to dental and orthopedic procedures or minor physical trauma. There are multiple risk factors associated with venous hemorrhage such as cigarette smoking, prior surgery, and previous viral infection, but no known risk factors for minor trauma induced hemorrhage." - Anonymous Online Contributor

"The rationale for clinical trials is that they are justified when a clinical benefit is demonstrated, when the trial compares well to the current standard of care, and when the chance of achieving benefit is high." - Anonymous Online Contributor

"Results from a recent paper shows that the common side effects of NVP-SPK-8011 are dose dependent. In addition, there is a clear trend that the lower the dose, the more the observed common side effects. The common side effects showed no gender preference. Some of these common pharmacodynamics side effects are: nausea, fatigue, headache/migraine/sinusitis, and constipation. Results from a recent paper are of great value in the development of new spk-8011 doses and clinical trials." - Anonymous Online Contributor