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Hemophilia A
What is hemophilia ?
Hemophilia is an hereditary bleeding disorder that prevents blood from clotting, causing abnormally long bleeding in the event of injury and sometimes in the absence of injury.
Hemophilia
Prevalence
Around 148200
people with hemophilia A worldwide
Unmet medical needs
Around % 1
of people with hemophilia worldwide still receive inadequate treatment or have no access to treatment
Cost
More than $ 199900
of treatment costs per patient per year, including indirect costs
What are the causes of hemophilia ?
Hemophilia is caused by an inherited genetic mutation that affects clotting factors. Coagulation is a complex process that involves several proteins and clotting factors that activate in chain. The coagulation factors VIII for hemophilia A and IX for hemophilia B are suppressed or made dysfunctional by the hemophilia gene mutation.
Hemophilia mainly affects men because it is inherited on the X chromosome. Since men have only one copy of the X chromosome, they are systematically affected if they inherit a mutated gene. Women, on the other hand, have two X chromosomes and only become ill if both X chromosomes are affected, which makes it a very rare case.
Let's focus on hemophilia A:
Most common form of hemophilia, accounting for 80% of patients
Absence or presence in a dysfunctional form of the factor VIII responsible for coagulation
Major complications: spontaneous bleeding and haemarthrosis
Am-I hemophiliac ?
The diagnosis of hemophilia is based on blood tests measuring both the clotting time and the levels of clotting factors VIII and IX. If there is a family history, a prenatal diagnosis may be considered.
Which treatments for hemophilia ?
There is no cure for hemophilia but it is possible to control the disease with prophylactic (preventive) treatments.
These treatments consist of multi-weekly injections (2 to 3 times a week) of functional clotting factor VIII, for moderate and severe forms of the disease. These injections are regular as they are intended to maintain a sufficient concentration of clotting factor in the blood to allow near-normal coagulation. This treatment is restrictive and has its limits. The more concentrated the treatment and the longer it is spread out, the more the body develops antibodies against the coagulation factor injected, which leads to its inactivation.
In parallel with this treatment, there are certain precautions that allow the patient to live better with the disease:
- Physiotherapy
- The sparing use of aspirin
- Limiting the practice of risky sports
- Monitoring the disease
Our team is involved in a European consortium which aims to develop a new treatment solution for haemophilia.
Encapsulation of FVIII in the MailPan® device
Principle of MailPan ®
- A medical device for the encapsulation of factor VIII secreting cells, currently under development for hemophilia.
- Prolonged and continuous delivery of factor VIII avoiding the development of antibodies directed against factor VIII.
- Possibility of renewing the cells without surgery, thanks to an input and output connected to the device.
- Suitable for cell therapies other than haemophilia that require protection from the immune system.
- Autonomous and physiological regulation of the concentration of coagulant agent in the blood.