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Individually Tailored Prophylaxis in Patients With Severe Hemophilia A

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

Summary

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

Conditions

Hemophilia A

Intervention

FVIII, FVIII

Location

Hopital Edouard Herriot
Lyon
France
69437

Status

Recruiting

Source

Hospices Civils de Lyon

Results (where available)

View Results

Links

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

Clinical Trials [228 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

This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tole...

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

A novel immuno-assay is being evaluated for the measurement of Factor VIII. Current procedure measures a rate assay which is subject to much artefact. The hypothesis is that the new assay ...

Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies

This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives. For this p...

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

The purpose of this study is to assess the role of a FVIII/VWF complex concentrate (Emoclot) in successfully inducing immune tolerance (I.T.I.) in patients with Haemophilia A with inhibito...

Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII

The ability of a new recombinant porcine coagulation factor VIII, B-domain deleted (called "OBI-1"), to control the non-life- or limb-threatening bleeding episodes patients with hemophilia...

PubMed Articles [127 Associated PubMed Articles listed on BioPortfolio]

Inhibitor Formation in Congenital Hemophilia A: an Immunological Perspective.

The immunogenicity of therapeutic factor VIII (FVIII) in patients with hemophilia A has been puzzling scientific and clinical communities for more than 3 decades. Indeed, the development of inhibitory...

A Case of Acquired Hemophilia A: Usefulness of Various Methods for Judging Mixing Test Results for Monitoring the Effect of Immunosuppressive Therapy.

Measurement of FVIII inhibitor (FVIII INH) levels is important for determining the effect of immunosuppressive therapy on acquired hemophilia A (AHA). However, FVIII INH can only be measured at a limi...

Subcutaneous Administration of Lyso-Phosphatidylserine Nanoparticles Induces Immunological Tolerance Towards Factor VIII in a Hemophilia A Mouse Model.

A major complication with enzyme replacement therapy of Factor VIII (FVIII) in Hemophilia A (HA) is the development of anti-drug antibodies. Recently, we have shown that FVIII administration in the pr...

Optimization of pre-analytical heat treatment for inhibitor detection in haemophilia A.

Factor VIII (FVIII) antibody formation is the greatest clinical and laboratory challenge within the haemophilia centre. The Nijmegen-Bethesda assay (NBA) is the gold standard for inhibitor quantificat...

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 ...

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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