Treosulfan in combination with fludarabine for paediatric non-malignant disease before allogeneic stem cell transplant

Treosulfan in addition to fludarabine is in clinical development for paediatric non-malignant disease prior to allogeneic stem cell transplant. Treosulfan is a medicine given to patients before they have a bone marrow transplant from a donor known as ‘allogeneic haematopoietic stem cell transplantation’. It is used as a ‘conditioning’ treatment to clear the patient’s bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells. Treosulfan is used together with another medicine called fludarabine for the treatment of a variety of disorders that require a bone marrow transplant.
In the body, treosulfan is converted into compounds called epoxides which kill cells, especially those that develop rapidly such as bone marrow cells, by attaching to their DNA while they are dividing. If licensed, treosulfan in addition to fludarabine will offer an additional option for conditioning therapy prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases. This combination has the potential to reduce toxicity and improve treatment-related outcomes compared to other conditioning regimens.