In a nutshell
This study aimed to investigate the combination of clofarabine (Clolar)-fludarabine (Fludara)-busulfan (Busulfex) as a conditioning regime in hematopoietic stem cell transplant (HSCT) for children with acute leukemia.
This study concluded that this combination is safe and effective in these patients.
A conditioning regime is one that is used to kill cancer cells and make room in the patient's bone marrow for new blood stem cells to grow. It also prevents the patient's body from rejecting transplanted cells and helps kill any cancer cells in the body. It is used with hematopoietic stem cell transplant (HSCT).
Standard conditioning regimes for children involve total body irradiation (TBI) and chemotherapy. The optimal dosages are important to prevent relapse and also late side effects in growing children. TBI is very effective but has important later-life side effects. The combination of clofarabine–fludarabine–busulfan (CloFluBu) has shown promising effectiveness. However, it is not known if CloFluBu would be suitable as a conditioning regime before HSCT in children with acute leukemia.
Methods & findings
This study involved 155 children with acute leukemia. Of these, 60 had acute lymphoblastic leukemia (ALL), 69 had acute myeloid leukemia (AML), and 26 had other cancers. The average age was 9.7 years. All patients received the CloFluBu conditioning treatment followed by HSCT. Patients were followed for an average of 2 years.
The estimated 2-year event free survival (EFS; survival without complications from leukemia) was 72% in patients with ALL and 62.4% in patients with AML. Patients who had positive minimal residual disease (MRD; a few cancer cells left behind after treatment) were more likely to relapse.
8.3% of the patients with ALL developed acute graft versus host disease (aGVHD) at 6 months. GVHD is a side effect of HSCT where the transplanted cells attack the patient. 5.5% of those with ALL developed chronic GVHD after 2 years. In patients with AML, 14.6% developed aGVHD and 8.1% developed chronic GVHD.
The bottom line
This study concluded that the CloFluBu combination is safe and effective as a conditioning regime before HSCT in children with ALL or AML. The authors suggested that it is a potentially less toxic alternative to conventional conditioning regimes.
The fine print
This study had a small number of participants and did not have a comparison group.
Published By :
Nov 15, 2021