Treosulfan in combination with Fludarabine for Malignant and Non-Malignant diseases - prior to stem cell transplantation

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Year: 2018

Acute myeloid leukaemia (AML) is a type of blood cancer that starts from young white blood cells in the bone marrow. Myelodysplastic syndrome (MDS) is a similar condition, where blood cells in the bone marrow do not mature and become healthy blood cells. Both AML and MDS usually occur in older people. If a patient with these conditions has been successfully treated, there may be a significant risk of the condition developing again. These patients may be offered stem cell transplantation (SCT), which will replace the stem cells in the bone marrow. Before a patient receives SCT they need to have a type of treatment called a “conditioning therapy” to prepare the body and get rid of the existing bone marrow.
Treosulfan given with fludarabine is being developed as a new conditioning therapy for people with conditions like AML or MDS prior to SCT. Both drugs are administered by injection and clinical studies have shown that, compared to current conditioning therapies, treosulfan might increase the success of the SCT with fewer complications, allowing people to live for longer.