Hemophilia Treatment at The National Hemophilia Center
At the National Hemophilia Center in Israel, we offer comprehensive services for patients with hemophilia and bleeding disorders. Our highly qualified, experienced doctors evaluate, treat and provide follow-up care for patients with coagulation diseases. We also function as a tertiary care center, treating patients with hemophilia referred from throughout Israel.
Hemophilia is an inherited bleeding disorder, and people with this disease lack the ability to stop bleeding. It is caused by low or nonexistent levels of coagulation “factors,” which are specific proteins necessary for blood clotting. Hemophilia treatment aims primarily to prevent and control bleeding, as well as to manage any associated health complications. The specific type of treatment depends on the type and severity of the condition.
As a world-renowned Hemophilia Training Center (HTC), our team stays up to date with the latest patient therapies to improve the quality of life for each individual. Our physicians and experienced study coordinators (rated as excellent by FDA evaluators) actively lead many cutting-edge clinical studies, including revolutionary gene therapy as a treatment for hemophilia. We are proud to be recognized as international leaders in promoting novel therapies in all areas of clinical hemostasis management.
Sheba’s medical approach is multidisciplinary, and doctors from a range of departments collaborate with nurses, psychosocial teams, experienced laboratory workers, physiotherapist, genetic counselor, and study coordinators to provide premier, custom-tailored care for each patient with hemophilia. In addition, you will be assigned a personal coordinator from the International Medical Tourism Division to assist with all aspects of your treatment at Sheba.
Risk Factors for Hemophilia
Hemophilia is an X linked recessive genetic disease (see below), usually passed down from parents to children. However, in approximately one-third of all children with hemophilia, there is no family history of the disease. In these sporadic cases, it is believed that the disorder results from a new gene mutation.
Hemophilia stems from malfunction of the gene that controls the production of clotting factor 8 or 9. These particular genes are located on the X chromosome, and hemophilia is therefore much more common in males and rarely occurs in females.
The genetics of hemophilia are as follows:
- A female carrier of hemophilia has the abnormal gene on one of her X chromosomes. When she is pregnant, there is a 50/50 chance of her passing on the hemophilia gene. If the gene is passed on to a son, he will have hemophilia. If the gene is passed on to a daughter, she will be a carrier.
- If a father has hemophilia but the mother is not a carrier, then none of the sons will have hemophilia. However, all of the daughters will be carriers.
Types of Hemophilia
There are two main forms of hemophilia:
- Hemophilia A – this is the most common type of the disease, accounting for about 85% of all cases. It is caused by a deficiency of blood clotting factor 8.
- Hemophilia B – caused by low levels of blood clotting factor 9.
Symptoms of Hemophilia
As a bleeding disorder, the primary symptom of hemophilia may be uncontrolled bleeding. In Israel Hemophilia may be diagnosed due to post circumcision bleeding, in the 8th day of life. The severity of hemophilia symptoms is dependent largely on the amount of clotting factors in the individual’s blood. In mild cases, people may not be aware that they have hemophilia until a surgery or major injury complicated by bleeding. In contrast, someone with severe hemophilia may bleed spontaneously, with no known reason. When this occurs in a joint, such as the elbow, knee, hip, shoulder or ankle, it can be very painful. Repeated joint bleeds lead to chronic synovitis and orthopedic damages, requiring jpoint replacement surgeries, at young age.
The most common symptoms of hemophilia include:
- Bleeding that does not stop after circumcision
- Bruising, from even minor accidents, that may lead to skin hematoma
- Bleeding into a joint
- Bleeding into muscles
- Bleeding internally- usually following trauma. Brain bleeds may occur following delivery and may be fatal
- Bleeding in the urine or stool
- May occur yet is much less frequent as compared to joint and muscle bleeds
Special Treatments for Hemophilia
At the National Hemophilia Center at Sheba, we offer integrative, individualized treatments for hemophilia. We monitor each patient closely to recommend the most appropriate medical therapies. Presently, the standard treatment involves receiving regular intravenous infusions of coagulation concentrates to promote blood clotting and reduce bleeding. Novel non-replacement therapies are also offered at the center, wither as a registered treatment or within large international multicenter trials. However, research into the use of gene therapy to eradicate hemophilia may offer a new, revolutionary treatment.
Hemophilia gene therapy, based on genetic engineering, is breaking new ground as a way to treat the disease for life with just a single dose. This experimental treatment uses the common adenovirus to deliver a healthy copy of a gene to compensate for the defective gene that is responsible for this coagulation disorder. Given by infusion, the virus travels to the liver, where the healthy gene is “unloaded” from the virus. This gene then permeates the liver cells, divides, and multiplies – thereby curing hemophilia through the subsequent production of clotting factors.
Ultimately, the goal is to administer just one dosage of this innovative treatment to eradicate hemophilia. So far, the results are dramatic and promising – however, not everyone is a suitable candidate for gene therapy. For example, children cannot receive the treatment because their liver tissue is still developing, and patients with liver illnesses or antibodies directed against blood clotting factors, or patients with antibodies towards the specific viral carrier, are also not suitable candidates.
Meet Our Department Manager
Prof. Gili Kenet, MD
Prof. Gili Kenet, MD
Director, National Hemophilia Center and Institute of Thrombosis & Hemostasis
Prof. Kenet is a pediatric hematologist, director of the Israeli National Hemophilia Center and the Amalia Biron Thrombosis Research Institute of Tel Aviv University. Prof Kenet is currently leading Sheba’s clinical research studies on treating hemophilia with gene therapy. She is also involved in advanced research projects focused on neonatal hemostasis, pediatricthrombosis and novel non replacement therapies for patients with severe bleeding disorders. She has chaired the scientific subcommittee (SSC) of ISTH for pediatric and perinatal hemostasis and is now co-chair of the SSC for FVIII/ FIX and rare bleeding disorders. She has been awarded many international grants for her varied research and authored more than 200 manuscripts published in high impact medical journals, as well as several chapters in books. Professor Kenet received her medical degree from the Hebrew University in Jerusalem.