What is Busilvex?
Busilvex is a concentrate for solution for infusion (drip into a vein) containing the active substance busulfan.
What is Busilvex used for?
Busilvex is indicated in the "conditioning" (preparatory) treatment prior to transplantation of hematopoietic progenitor cells (cells that generate blood cells) in adult and child patients. This type of transplant is performed in subjects suffering from blood changes (for example, a rare form of anemia) or blood cancers, in which it is therefore necessary to replace hematopoietic cells. Treatment with Busilvex is followed by therapy with another drug (cyclophosphamide in adults and cyclophosphamide or melphalan in children).
Since the number of patients undergoing this type of conditioning and transplantation is low in the European Union (EU), busulfan was designated an "orphan medicine" (a medicine used in rare diseases) on 29 December 2000.
The medicine can only be obtained with a prescription.
How is Busilvex used?
Administration of Busilvex should take place under the supervision of a physician experienced in pre-transplant treatments. The recommended dosage of Busilvex in adults is 0.8 mg per kilogram of body weight. In children and adolescents (up to 17 years), the recommended dosage of Busilvex depends on the patient's body weight and ranges from 0.8 to 1.2 mg / kg. Busilvex is given as a central intravenous infusion (drip into a central vein in the chest). Each infusion lasts two hours and is administered to the patient every six hours for four consecutive days before treatment with cyclophosphamide or melphalan and transplantation. Before receiving Busilvex, patients should be pretreated with anticonvulsant drugs (to prevent seizures) and anti-emetic drugs (to prevent vomiting).
How does Busilvex work?
The active substance in Busilvex, busulfan, belongs to a group of medicines called "alkylating agents". These substances are "cytotoxic", ie they kill cells, especially those that develop quickly, such as tumor or progenitor cells (or "stem cells") (ie cells that produce other types of cells). Busulfan is used before transplantation to neutralize abnormal cells and hematopoietic progenitor cells in the patient. This process is called "myeloablation". The treatment with cyclophosphamide or melphalan is then used to suppress the immune system, so that the body's natural defenses are lowered. This promotes the "grafting" of the transplanted cells (that is, the cells begin to grow and produce normal blood cells).
How has Busilvex been studied?
The effects of Busilvex were first tested in experimental models before being studied in humans. The pharmaceutical company presented data from experimental models derived from scientific literature.
Busilvex has been studied in patients, mostly suffering from blood cancer, who needed transplantation of haematopoietic progenitor cells. Two main studies were conducted, involving 103 adults, and one study involving 55 children. The main efficacy parameter was based on the number of patients with myeloablation (ie reduced number of white blood cells and platelets) and "graft" (the time required for white blood cells to return to higher levels).
What benefit has Busilvex shown during the studies?
Myeloablation has been observed in all patients, adults and children. The graft was obtained on average after ten days (in adults) or 11 days (in children) in cases of "autologous transplantation" (ie transplantation of the patient's own cells, collected and stored before transplantation). The graft was reached after 13 days (in adults) and 21 days (in children) in "allogeneic transplants" (donor cell transplantation).
What is the risk associated with Busilvex?
Apart from the reduction in the number of circulating blood cells, which is the goal of the medicine, infection, liver disorders, including obstruction of a hepatic vein, transplant disease towards transplant disease, are among the most serious side effects of treatment with Busilvex. the host (when the transplanted cells attack the patient's organism) and respiratory (pulmonary) disorders. For the full list of all side effects reported with Busilvex, see the Package Leaflet.
Busilvex should not be used in people who may be hypersensitive (allergic) to busulfan or any of the other components. It must not be given to pregnant women. Breastfeeding should be discontinued when starting treatment with Busilvex. Busilvex can impair fertility in both sexes. Female patients should therefore not become pregnant during and up to six months after treatment and male patients should not procreate during and up to six months after treatment with the medicine.
Particular care is recommended if concomitant drugs such as itraconazole (used for certain types of infections), chetobemidone (used to treat pain) and paracetamol are used. For more details, see the package leaflet.
Why has Busilvex been approved?
The Committee for Medicinal Products for Human Use (CHMP) concluded that the effectiveness of Busilvex has been demonstrated and that it is an alternative to busulfan tablets, which have disadvantages including the high number of tablets that need to be taken. The CHMP decided that the benefits of Busilvex outweigh its risks as a conditioning treatment prior to transplantation of haematopoietic progenitor cells. The Committee therefore recommended that Busilvex be given marketing authorization.
More information on Busilvex:
On 9 July 2003, the European Commission granted an EU-wide marketing authorization to Pierre Fabre Médicament for Busilvex. The marketing authorization was renewed on 9 July 2008.
To register as a Busilvex orphan medicinal product, click here .
For the full EPAR for Busilvex, click here.
Last update of this summary: 07-2008.
