Skip to main content

Results summary of St. Jude clinical trial: 



AML08: A Phase III Randomized Trial of Clofarabine Plus Cytarabine Versus Conventional Induction Therapy and A Phase II Study of Natural Killer Cell Transplantation in Patients with Newly Diagnosed Acute Myeloid Leukemia

Why was this study done?

Nearly 500 children are found to have acute myeloid leukemia (AML) each year in the U.S. About half of these children are cured with standard therapy, which means they show no signs of leukemia for five years.

This study’s main goal was to improve the cure rate of children and adolescents with AML.

The study’s other goals were to:

  • Compare the response rates after one course of chemotherapy (chemo) in patients treated with the standard drug combination of cytarabine, daunorubicin and etoposide versus the experimental combination of clofarabine and cytarabine
  • Find out if natural killer (NK) cell therapy will be useful for patients with standard-risk AML
  • Learn more about the biology and genetic makeup of AML by studying blood and bone marrow samples
  • Learn more about how AML treatment drugs work in the body of patients with AML
  • Learn how the drugs affect the body and how patients’ own genetic makeup may predict who will have more side effects and who will respond to treatment
  • Find out if we can prevent major infections by giving antibiotics and antifungal drugs when blood counts are very low

When was this study done?

The study opened in August 2008 and closed in March 2017.

What did the study consist of?

  • All patients were randomly assigned to receive clofarabine and cytarabine or cytarabine, daunorubicin, and etoposide as their first treatment course
  • Low-risk patients received four courses of chemotherapy
  • Standard-risk patients who had an appropriate NK cell donor received four courses of chemotherapy followed by an infusion of NK cells. Other standard-risk patients received four courses of chemotherapy without NK cell therapy.
  • High-risk patients received two or three courses of chemotherapy followed by hematopoietic cell transplantation.

What did we learn from this study?

After two courses of therapy, 92.3% of the 285 patients were in complete remission. Minimal residual disease was present on Day 22 of therapy in 47% of patients who received clofarabine and cytarabine and in 35% of patients who received cytarabine, daunorubicin and etoposide. Despite this result, the 3-year event-free survival rate did not differ significantly between the two treatment arms. The 3-year overall survival rate was 75% for patients who received clofarabine and cytarabine and 65% for those who received cytarabine, daunorubicin and etoposide (result is not statistically different).

Patients who received NK cells tolerated the treatment well. But, the therapy with NK cells did not improve survival rates.  

What are the next research steps as a result of this study?

The findings suggest that the use of clofarabine with cytarabine during remission induction might reduce the need for anthracycline and etoposide in pediatric patients with AML and may reduce rates of cardiomyopathy and treatment-related cancer.

How does this study affect my child?

Every survivor of AML should receive long-term follow-up care. Your child will receive information and guidance for care. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

For more information

Please talk with your child’s St. Jude doctor about questions or concerns you have as a result of this study.

Publications generated from this study:

Clofarabine Can Replace Antracyclines and Etoposide in Remission Induction Therapy for Childhood Acute Myeloid Leukemia: The AML08 Multicenter, Randomized Trial. Rubnitz JE, Lacayo NJ, Inaba H, Heym K, Ribeiro RC, Taub J, McNeer J, Degar B, Schiff D, Yeoh AE, Coustan-Smith E, Wang L, Triplett B, Raimondi SC, Klco J, Choi J, Pounds S, Pui CH. J Clin Oncol. 2019 Aug 10;37(23):2072-2081.

A Phase II Clinical Trial of Adoptive Transfer of Haploidentical Natural Killer Cells for Consolidation Therapy of Pediatric Acute Myeloid Leukemia. Nguyen R, Wu H, Pounds S, Inaba H, Ribeiro RC, Cullins D, Rooney B, Bell T, Lacayo NJ, Heym K, Degar B, Schiff D, Janssen WE, Triplett B, Pui CH, Leung W, Rubnitz JE. J Immunother Cancer. 2019 Mar 20; 7: 81.