[9][10] It is used together with another medicine called fludarabine in adults and children from one month of age with blood cancers as well as in adults with other severe disorders requiring a bone marrow transplant.

[9] The most common side effects in adults and children are infections, nausea (feeling sick), stomatitis (inflammation of the lining of the mouth), vomiting, diarrhoea and abdominal pain (belly ache).

[9] Two main studies showed that treosulfan is at least as effective as busulfan, another medicine used to prepare patients for haematopoietic stem cell transplantation.

[9] In one of the studies, involving 570 adults with acute myeloid leukaemia (a blood cancer) or myelodysplastic syndromes (conditions in which large numbers of abnormal blood cells are produced), 64% of patients given treosulfan (with fludarabine) had a successful transplant and were alive and disease-free after 2 years, compared with 51% of patients given busulfan (with fludarabine).

[9] On 23 February 2004, orphan designation (EU/3/04/186) was granted by the European Commission to medac Gesellschaft fuer klinische Spezialpräparate mbH, Germany, for treosulfan for the conditioning treatment prior to haematopoietic progenitor cell transplantation.