Individually Tailored Prophylaxis in Patients With Severe Hemophilia A

2014-07-24 14:11:14 | BioPortfolio


Patients with severe haemophilia A lack clotting factor FVIII and suffer from spontaneous and traumatic bleeds. In the absence of treatment, frequent bleeds in joints lead to severe joint destruction. In 1960s, prophylactic therapy was developed involving the infusion of clotting factor on a regular schedule in order to keep clotting levels sufficiently high to prevent spontaneous bleeding episodes. Prophylaxis is started at an early age before the age of 2 years or after the first joint bleed. The Malmö experience indicates that treatment is most effective when administered in large doses at least 3 times weekly. However, such an intensive treatment in young boys may be very difficult to carry out for home treatment. Currently, there is no international recommendation on prophylactic therapy regimens. Because of the high cost and limited availability of factor concentrates, dosing is an important issue in prophylaxis therapy. It was recently shown that 24 hours after FVIII concentrate administration, in patients presenting similar FVIIII levels, thrombin generation capacity may be significantly different. In addition, independently of the FVIII level, a correlation was found between severe clinical bleeding phenotype and thrombin generating capacity. The aim of the present clinical study is to assess the thrombin generation test as the main surrogate marker to evaluate the coagulating capacity of haemophiliacs on prophylaxis regimen. Optimizing prophylactic therapy to patient's phenotype with no loss of clinical effectiveness can significantly improve patients' quality of life, protect haemophilic children against arthropathy and possibly limit the cost of the prophylaxis therapy.

Study Design

Allocation: Non-Randomized, Control: Dose Comparison, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label


Hemophilia A




Hopital Edouard Herriot




Hospices Civils de Lyon

Results (where available)

View Results


Published on BioPortfolio: 2014-07-24T14:11:14-0400

Clinical Trials [209 Associated Clinical Trials listed on BioPortfolio]

Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive

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Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay

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Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients

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PubMed Articles [128 Associated PubMed Articles listed on BioPortfolio]

Pharmacokinetic drug evaluation of recombinant Factor VIII for the treatment of hemophilia A.

The prevention of bleeding by prophylactic factor replacement is the recommended approach for the treatment of severe hemophilia. Prophylaxis should be individualized to provide the best clinical bene...

Factor XIII Co-Treatment with Hemostatic Agents in Hemophilia A Increases Fibrin α-Chain Crosslinking.

Hemophilia A results from the absence, deficiency, or inhibition of factor VIII (FVIII). Bleeding is treated with hemostatic agents (FVIII, recombinant activated factor VIIa [rFVIIa], anti-inhibitor c...

Inhibitors in Nonsevere Hemophilia A: What Is Known and Searching for the Unknown.

Nonsevere hemophilia A (NSHA) is an inherited X-linked bleeding disorder, caused by mutations of thegene, leading to decreases of clotting factor VIII (FVIII) levels to 1 to 40 IU/dL. Desmopressin is ...

Human Dendritic Cells Synergistically Activated by FVIII Plus LPS Induce Activation of Autologous CD4+ T Cells.

The most severe side effect in haemophilia A (HA) treatment is the development of anti-factor VIII antibodies, also called inhibitors. Why inhibitors develop in a proportion of treated HA patients and...

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The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is the most severe complication in the early phases of treatment of severe haemophilia A. Recently a randomized tria...

Medical and Biotech [MESH] Definitions

A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)

The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.

A hereditary deficiency of blood coagulation factor XI (also known as plasma thromboplastin antecedent or PTA or antihemophilic factor C) resulting in a systemic blood-clotting defect called hemophilia C or Rosenthal's syndrome, that may resemble classical hemophilia.

Bleeding into the joints. It may arise from trauma or spontaneously in patients with hemophilia.

Stable blood coagulation factor involved in the intrinsic pathway. The activated form XIa activates factor IX to IXa. Deficiency of factor XI is often called hemophilia C.

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