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Response-guided induction therapy in pediatric acute myeloid leukemia with excellent remission rate
Jonas Abrahamsson1, Erik Forestier, Jesper Heldrup
1Institution of Clinical Sciences, Department of Pediatrics, Sahlgrenska University Hospital, 41685 Gothenburg, Sweden. jonas.abrahamsson@vgregion.se
Insights
The NOPHO-AML 2004 protocol shows a high remission rate for childhood acute myeloid leukemia (AML) using a response-guided induction strategy. Intermediate responders had poorer outcomes, suggesting a need for intensified consolidation therapy.
Area of Science:
- Pediatric Hematology Oncology
- Acute Myeloid Leukemia (AML) Treatment
Background:
- Acute myeloid leukemia (AML) is a significant pediatric cancer.
- Optimizing induction strategies is crucial for improving treatment outcomes in children.
- Early response assessment can guide subsequent therapy.
Purpose of the Study:
- To evaluate the early treatment response in pediatric acute myeloid leukemia (AML).
- To assess a response-guided induction strategy incorporating idarubicin in the first course.
- To analyze outcomes based on early response to induction therapy.
Main Methods:
- A Nordic cohort of 151 children (<15 years) with AML were treated on the NOPHO AML 2004 protocol.
- Response-guided induction therapy included idarubicin, cytarabine, etoposide, and 6-thioguanine.
- Patients with poor/intermediate response proceeded immediately to the second course; good responders had hematologic recovery first. Consolidation varied based on response and remission status, including allogeneic stem-cell transplantation (SCT) for poor responders.
Main Results:
- After the first course, 74% showed good response, 17% intermediate, and 7% poor.
- Overall remission frequency was 97.4%, with 92% in remission after the second course.
- Event-free survival was significantly lower for intermediate responders (35%) compared to good (61%) and poor responders (82%). Induction death rate was 1.3%.
Conclusions:
- The NOPHO-AML 2004 response-guided induction strategy achieves excellent remission rates with low toxic mortality in children with AML.
- Intermediate responders exhibit worse outcomes, indicating a potential benefit from intensified consolidation, such as SCT.
- This strategy effectively stratifies patients for tailored consolidation therapy.
Purpose:
To evaluate the early treatment response in children with acute myeloid leukemia (AML) using a response-guided induction strategy that includes idarubicin in the first course.
Patients And Methods:
All Nordic children with AML younger than 15 years (n = 151) were treated on the Nordic Society for Pediatric Hematology and Oncology (NOPHO) AML 2004 protocol. After the first course of idarubicin, cytarabine, etoposide, and 6-thioguanin, patients with good response were allowed hematologic recovery before the second course, whereas patients with a poor (≥ 15% blasts) or intermediate (5% to 14.9% blasts) were recommended to proceed immediately with therapy. Patients not in remission after the second course received fludarabine, cytarabine, and granulocyte colony-stimulating factor. Poor responders received allogeneic stem-cell transplantation (SCT) as consolidation.
Results:
Seventy-four percent of patients had good response, 17% had intermediate response, and 7% had poor response after the first course. The overall remission frequency was 97.4%, with 92% in remission after the second course. The rate of induction death was 1.3%. Patients with an intermediate response had a lower event-free survival of 35% compared with good (61%) and poor responders (82%).
Conclusion:
The NOPHO-AML 2004 induction strategy gives an excellent remission rate with low toxic mortality in an unselected population. Outcome is worse in patients with intermediate response but may be improved by intensifying consolidation in this group using SCT.
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