Chemotherapy alternatives for children with acute myeloid leukemia

This study aimed to identify an effective and safe chemotherapy for children with acute myeloid leukemia (AML).   

This study concluded that clofarabine during remission induction may reduce the need for other treatments which cause side effects in these patients.  

Clofarabine (Clolar) is a chemotherapy and is used to treat children and young adults with acute lymphoblastic leukemia (ALL) or older patients with AML. Remission induction is the treatment used to produce a complete remission (no sign of cancer left). Current induction chemotherapy for children with AML includes the use of anthracycline-based drugs, such as daunorubicin (Cerubidine). This type of chemotherapy is known to cause heart side effects. Daunorubicin is used together with etoposide (Etopophos). This is a chemotherapy that can be used to treat a variety of cancers. 

It was unknown if clofarabine would have fewer side effects than standard treatments for children with AML. 

This study involved 262 patients with untreated AML aged 22 years or younger. 129 patients received clofarabine and cytarabine (Cytosar-U). 133 patients received high-dose cytarabine, daunorubicin, and etoposide (HD-ADE). This was induction I. For induction II patients received low-dose ADE (LD-ADE) alone or in combination with sorafenib (Nexavar) or vorinostat (Zolinza). Sorafenib is a targeted therapy used for AML. Vorinostat is a chemotherapy. Patients then received two or three additional courses of chemotherapy or stem cell transplant for consolidation therapy. Consolidation therapy is used to kill any cancer cells that may be left in the body after initial treatment. The main outcome measured was minimal residual disease (MRD – small number of cancer cells after treatment that remain in the blood) at day 22. 

Complete remission (all signs of cancer gone) was induced after two courses of therapy in 92.3% of the patients. Induction failures were four early deaths and 15 cases of resistant leukemia (treatment did not work). Day 22 MRD was positive in 47% of patients who received clofarabine and cytarabine (Clo+AraC) and in 35% of patients who received HD-ADE.  

The 3-year event-free survival rate was 52.9% for Clo+AraC group compared to 52.4% for the HD-ADE group. The 3-year overall survival rate was 74.8% for the Clo+AraC group compared to 64.6% for the HD-ADE group.   

This study concluded that clofarabine with cytarabine during remission induction may reduce the need for anthracycline and etoposide in pediatric patients with AML. It was also concluded that clofarabine may reduce rates of heart disease and treatment-related cancer.