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Favorable outcome in infants with AML after intensive first- and second-line treatment: an AML-BFM study group report
U Creutzig1, M Zimmermann, J-P Bourquin
1Klinik und Poliklinik für Kinderheilkunde, Pediatric Hematology and Oncology, University Hospital Münster, Münster, Germany.
Insights
Intensive treatment for infant acute myeloid leukemia (AML) is feasible and manageable. Survival rates are favorable, even after relapse, with manageable toxicities comparable to older children.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Research
Background:
- Infants under one year old have a high incidence of poor-prognosis leukemias.
- Infants are presumed to be more susceptible to treatment-related toxicities.
Purpose of the Study:
- To evaluate the feasibility and outcomes of intensive frontline and relapse treatment for acute myeloid leukemia (AML) in infants.
- To compare treatment outcomes and toxicities in infants versus older children with AML.
Main Methods:
- A total of 125 infants with AML were treated across two studies (AML-BFM-98 and -2004).
- Treatment involved intensive induction followed by four courses of chemotherapy, with optional allogeneic-hematopoietic stem-cell-transplantation (allo-HSCT) for high-risk patients.
- Patients were stratified as high-risk (HR) based on morphological, cytogenetic/molecular genetic, and response criteria.
Main Results:
- Most infants (96%) were classified as high-risk (HR).
- Five-year overall survival was 66%, showing improvement from 61% in study-98 to 75% in study-2004.
- Event-free survival rates were 44% and 51% for study-98 and -2004, respectively.
- Survival in HR infants was comparable to older HR children.
- Survival after allo-HSCT in first remission, initial partial response, and relapse was high (13/14, 2/8, and 20/30 patients, respectively).
- Five-year survival after relapse was 50%.
- Treatment-related death rate was 3%, identical to older children, despite more severe infections and pulmonary toxicities in infants.
Conclusions:
- Intensive frontline and relapse treatment for infant AML is feasible.
- Toxicities associated with intensive AML treatment in infants are manageable.
- Outcomes for infants with AML are favorable, with survival rates comparable to older children.
Abstract:
Infants <1 year of age have a high prevalence of prognostically unfavorable leukemias and a presumed susceptibility to treatment-related toxicities. A total of 125 infants with acute myeloid leukemia (AML) were treated in studies AML-BFM-98 (n = 59) and -2004 (n = 66). Treatment regimens of both studies were comparable, consisting of intensive induction followed by four courses (mainly high-dose cytarabine and anthracyclines). Allogeneic-hematopoietic stem-cell-transplantation (allo-HSCT) in 1st remission was optional for high-risk (HR) patients. Most infants (120/125=96%) were HR patients according to morphological, cytogenetic/molecular genetic and response criteria. Five-year overall survival was 66 ± 4%, and improved from 61 ± 6% in study-98 to 75 ± 6% in study-2004 (P(logrank) 0.14) and event-free survival rates were 44 ± 6% and 51 ± 6% (P(logrank) 0.66), respectively. Results in HR infants were similar to those of older HR children (1-<2- or 2-<10-year olds, P(logrank) 0.90 for survival). Survival rates of HSCT in 1st remission, initial partial response and after relapse were high (13/14, 2/8 and 20/30 patients, respectively). The latter contributes to excellent 5-year survival after relapse (50±8%). Despite more severe infections and pulmonary toxicities in infants, treatment-related death rate was identical to that of older children (3%). Our data indicate that intensive frontline and relapse AML treatment is feasible in infants, toxicities are manageable, and outcome is favorable.