In a nutshell
This study looked at the effect of treosulfan combined with fludarabine (Fludara) and low-dose radiotherapy in the treatment of children with acute myeloid leukemia (AML). The researchers found that treosulfan combined with these therapies improved successful graft transplantation in the treatment of AML.
Acute myeloid leukemia is a cancer of the bone marrow that produces abnormal white blood cells. It is often treated by first destroying the abnormal cells (myeloablation conditioning), and then replacing them with normal donor cells (stem cell transplantation). Conditioning is generally done with chemotherapy and/or total body irradiation. There may be side effects, however, such as infection and transplant rejection, particularly with the chemotherapy busulfan.
Treosulfan is a type of chemotherapy used in other types of cancer. It has been shown to be safe and effective, with fewer side effects, in adults. It is not clear whether it is safe and effective in children with AML.
Methods & findings
40 patients with AML aged from 1 to 19 were included in the study (20% of these patients had myelodysplastic syndrome rather than AML). All patients received treosulfan, fludarabine, and a low-dose of total body radiotherapy. Patients also received tacrolimus and methotrexate (Trexall) to prevent graft versus host disease (GVHD – a type of severe transplant rejection when the transplanted cells attack healthy tissue).
The 1-year overall survival rate (time from treatment until death from any cause) was 80%. 63% of AML patients survived without disease relapse after 1 year. 38% of AML patients had a relapse (return of the disease).
Acute (early) GVHD occurred in 22% of patients and chronic (later) GVHD occurred in 40%.
The bottom line
The study concluded that treosulfan was a safe and effective drug as part of transplant treatment for young patients with acute myeloid leukemia.
The fine print
The study had a small sample size and treosulfan is not currently widely available on the market.
Published By :
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
May 09, 2018