ells. This is called ‘myeloablation’. Cyclophosphamide or melphalan is then used to suppress the immune system, so that the body’s natural defences are decreased. This helps the transplanted cells to ‘engraft’ (when they start to grow and produce normal blood cells).
How has it been studied?
The effects of Busilvex were first tested in experimental models before being studied in humans. The applicant also presented data from the scientific literature.
Busilvex has been studied in patients, mainly with blood cancer, who needed transplantation of haematopoietic progenitor cells. There were two main studies involving 103 adults and one involving 55 children. The main measures of effectiveness were the number of patients with myeloablation (with reduced counts of white blood cells and platelets) and ‘engraftment’ (the time for the white blood cells to return to higher levels).
What benefits has it shown during the studies?
All adults and children achieved myeloablation. On average, engraftment was reached in 10 days in adults and 11 days in children with ‘autotransplantation’ (when the patient receives their own cells, harvested and stored before the transplant). Engraftment was obtained in 13 days in adults and 21 days in children with ‘allotransplantation’ (when the patient receives cells from a donor).
What is the risk associated?
Apart from the decrease in blood cell counts, which this is the intended effect of the medicine, the most serious side effects of Busilvex are infection, liver disorders including blocking of a liver vein, graft versus host disease (when the transplanted cells attack the body) and respiratory (lung) 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 ingredients. It must not be used in women who are pregnant. Breast-feeding should be stopped when treatment with Busilvex is started. Busilvex can affect fertility in both sexes. Because of this, female patients should not become pregnant during treatment and for up to six months afterwards, and male patients are advised not to father a child during and for up to six months after treatment with Busilvex.
Caution is needed when Busilvex is used at the same time as other medicines, such as itraconazole (used to treat some infections), ketobemidone (used to treat pain) and paracetamol. See the Package Leaflet for full details.
Why has it been approved?
The Committee for Medicinal Products for Human Use (CHMP) concluded that the effectiveness of Busilvex had been shown, and that it provides an alternative to busulfan tablets, which have disadvantages such as the large number of tablets that need to be taken. The Committee decided that Busilvex’s benefits are greater than its risks as conditioning treatment prior to haematopoietic progenitor cell transplantation. The Committee recommended that Busilvex be given marketing authorisation.
Further information
The European Commission granted a marketing authorisation valid throughout the EU for Busilvex to Pierre Fabre Médicament on 9 July 2003. The marketing authorisation was renewed on 9 July 2008.
The record of Busilvex’s designation as an orphan medicine is avai
