1.1 Treosulfan with fludarabine is recommended as an option for conditioning treatment before allogeneic haematopoietic stem cell transplant (allo-HSCT) for people with malignant diseases for whom a reduced intensity regimen, such as low-dose busulfan with fludarabine, would be suitable.
Why the committee made these recommendations
People with malignant diseases having an allo-HSCT need to have conditioning treatment first to prepare their bone marrow. If they cannot tolerate high-intensity myeloablative conditioning, they can have reduced-intensity conditioning such as low-dose busulfan with fludarabine.
The clinical evidence compares treosulfan and fludarabine with low-dose busulfan and fludarabine. Not enough evidence was presented for children or for people who could tolerate a high-intensity myeloablative regimen, so it is not possible to make recommendations for these groups.
The evidence in people for whom reduced-intensity is the most appropriate conditioning regimen shows that people are less likely to die from the transplant or associated complications if they have treosulfan and fludarabine instead of busulfan and fludarabine. The risk of disease recurrence was similar after either treatment.
Treosulfan with fludarabine is more effective and costs less than low-dose busulfan with fludarabine in most analyses. Therefore, treosulfan with fludarabine is recommended as an option in the NHS for conditioning treatment for people who would normally have a reduced intensity regimen.