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Development of treatment and clinical results in childhood AML in Austria (1993-2013)
Heidrun Boztug1, Nora Mühlegger1, Evgenia Glogova1
11St. Anna Kinderspital and Children's Cancer Research Institute, Department of Pediatrics, Medical University of Vienna, Kinderspitalgasse 6, 1090 Vienna, Austria.
Insights
Austrian pediatric acute myeloid leukemia (AML) trials show survival rates comparable to international standards. Continued international collaboration is essential for improving cure rates and reducing treatment events in pediatric AML.
Area of Science:
- Pediatric Hematology Oncology
- Cancer Research
- Clinical Trials
Background:
- Three consecutive pediatric acute myeloid leukemia (AML) trials (AML-BFM 93, 98, 04) were conducted in Austria from 1993 to 2013.
- These trials were performed in close collaboration with the international Berlin-Frankfurt-Münster (BFM) study center.
Purpose of the Study:
- To review patient characteristics, therapy, and outcome data from Austrian pediatric AML trials.
- To assess treatment effectiveness and survival rates in pediatric AML patients over two decades.
Main Methods:
- Analysis of data from 249 children and adolescents diagnosed with AML enrolled in three Austrian BFM studies (1993-2013).
- Patients were treated in five major Austrian pediatric hematology/oncology centers.
- Stratification into standard-risk (SR) and high-risk (HR) groups, with analysis of genetic aberrations like MLL rearrangements.
Main Results:
- Complete remission (CR) rates ranged from 84-95%.
- Leukemic relapse was the primary event (5-year cumulative incidence 40-21%, with a trend towards higher rates in SR patients in the 2004 study).
- Overall survival (pSU) showed improvement across studies (57% to 75%), particularly in the HR group (40% to 66%).
Conclusions:
- Pediatric AML treatment in Austria achieves survival rates aligning with international best practices.
- Small patient numbers and recruitment variations limit definitive comparisons between treatment periods.
- International collaboration is crucial for advancing pediatric AML treatment and improving cure rates.
Background:
Since the early 1990s, three consecutive pediatric acute myeloid leukemia (AML) trials have been performed in Austria (AML-Berlin-Frankfurt-Münster (BFM) 93, AML-BFM 98, and AML-BFM 2004) in close cooperation with the international BFM study center. Herein, we review the pertinent patient characteristics, therapy, and outcome data.
Patients And Methods:
From January 1993 to April 2013, 249 children and adolescents (193 protocol patients) diagnosed with AML were enrolled in the three BFM studies. Patients were mainly treated in one of five pediatric hematology/oncology centers distributed over Austria.
Results:
Many characteristics and outcome parameters were not statistically different between the three trials. Almost similar proportions of patients were stratified into two risk groups: standard risk (SR) (approximately 37 % overall) and high-risk (HR) (61 %). MLL rearrangements were found in 23 % of patients overall as the most frequent genetic aberration subtype. Complete remission (CR) was achieved by 84-95 % of patients. The most important type of event was leukemic relapse (5-year cumulative incidence 40 ± 8 %, 21 ± 5 %, and 39 ± 6 %; p = 0.058), with a trend to a higher rate specifically in SR patients of study AML-BFM 2004 compared with AML-BFM 98. Importantly, the frequency of death from causes other than relapse sequelae declined over the years (AML-BFM 93: 5/42 12 %, AML-BFM 98: 5/57 9 %, and AML-BFM 2004: 5/94 5 %). Altogether, event-free survival at 5 years varied insignificantly (48 ± 8 %, 61 ± 7 %, and 50 ± 6 %; p = 0.406). Nevertheless, survival (pSU) apparently improved from BFM 93 to subsequent studies, both overall (57 ± 8 %, 75 ± 6 %, and 62 ± 6 %; p = 0.046) and regarding the HR group (5-year-probability of survival (pSU) 40 ± 10 %, 66 ± 8 %, and 52 ± 8 %; p = 0.039).
Conclusion:
Treatment of pediatric AML in Austria renders survival rates in the range of international best practice. However, unambiguous statistical comparison of treatment periods is eventually hampered by small numbers and inequalities of recruitment. Hence, only internationally collaborative trials will allow developing treatment further to achieve higher cure rates with fewer events.
