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Published results

AML02: Treatment of Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplasia

Why was this study done?

About half of children and adolescents with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) become long-term survivors. When these diseases do not go into remission or return after treatment, they are even harder to treat. This study’s main goal was to improve the cure rate of children and adolescents with these disorders.

The study’s other goals were to:

  • Compare the remission rates of those who receive two different doses of cytarabine during induction therapy
  • Learn more about the effects of gemtuzumab ozogamicin (GO), when used either alone or with other chemotherapy drugs
  • Study how leukemia cells react to chemotherapy during treatment
  • Compare the amounts of leukemia cells in the blood and bone marrow to study minimal residual disease (MRD)
  • Try to improve how fungal infections are diagnosed in patients with AML and MDS
  • Compare the results of magnetic resonance imaging (MRI) with lab tests of the bone marrow

When was this study done?

The study opened in 2002 and closed in 2008.

What did the study consist of?

  • In the Induction 1 phase of the study, patients received cytarabine along with daunorubicin and etoposide.
  • MRD levels were used to decide who would receive GO and to determine the timing of induction 2.
  • During Induction 2, patients received the drugs used in Induction 1 with or without GO.
  • Consolidation therapy included more courses of chemotherapy or a blood stem-cell transplant. Low-risk patients got five courses of chemo, while high-risk and standard-risk patients with matched sibling donors were eligible for transplant.

What did we learn from this study?

Eighty percent of children in this study had a complete remission after Induction 1 and 94% after Induction 2. These findings show we can improve outcomes for children with AML by using a risk-related strategy, genetic features, targeted chemo, MRD findings and blood stem-cell transplants.

The main reason some children with AML relapse is that they have leukemic stem cells (LSCs) that have survived treatment. LSCs can develop into leukemia cells and are also linked to resistance to chemotherapy drugs. As part of this study we identified six stem cell genes that predict how well patients respond to therapy. This discovery may help clinicians identify patients who have high-risk disease and who would benefit from novel treatments.

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

More studies are needed to learn more about the role each of the six stem cell genes play in AML.

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:

A Six-gene Leukemic Stem Cell Score Identifies High Risk Pediatric Acute Myeloid Leukemia. Elsayed AH, Rafiee R, Cao X, Raimondi S, Downing JR, Ribeiro R, Fan Y, Gruber TA, Baker S, Klco J, Rubnitz JE, Pounds S, Lamba JK. Leukemia. 2020 Mar;34(3):735-745.

Comprehensive Genetic Analysis of Cytarabine Sensitivity in a Cell-based Model Identifies Polymorphisms Associated with Outcome in AML Patients. Gamazon ER, Lamba JK, Pounds S, Stark AL, Wheeler HE, Cao X, Im HK, Mitra AK, Rubnitz JE, Ribeiro RC, Raimondi S, Campana D, Crews KR, Wong SS, Welsh M, Hulur I, Gorsic L, Hartford CM, Zhang W, Cox NJ, Dolan ME. Blood. 2013 May 23;121(21):4366-76.

Minimal Residual Disease-directed Therapy for Childhood Acute Myeloid Leukaemia: Results of the AML02 Multicentre Trial. Rubnitz JE, Inaba H, Dahl G, Ribeiro RC, Bowman WP, Taub J, Pounds S, Razzouk BI, Lacayo NJ, Cao X, Meshinchi S, Degar B, Airewele G, Raimondi SC, Onciu M, Coustan-Smith E, Downing JR, Leung W, Pui CH, Campana D. Lancet Oncol. 2010 Jun;11(6):543-52.

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.


Full title:

A Collaborative Trial for the Treatment of Patients with Newly Diagnosed Acute Myeloid Leukemia or Myelodysplasia

Study goal:

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

For physicians and researchers

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