PURPOSE: This retrospective cohort study aimed to determine the predictive relevance of clinical characteristics, additional cytogenetic aberrations, and cKIT and RAS mutations, as well as to evaluate whether specific treatment elements were associated with outcomes in pediatric t(8;21)-positive patients with acute myeloid leukemia (AML). PATIENTS AND METHODS: Karyotypes of 916 pediatric patients with t(8;21)-AML were reviewed for the presence of additional cytogenetic aberrations, and 228 samples were screened for presence of cKIT and RAS mutations. Multivariable regression models were used to assess the relevance of anthracyclines, cytarabine, and etoposide during induction and overall treatment. End points were the probability of achieving complete remission, cumulative incidence of relapse (CIR), probability of event-free survival, and probability of overall survival. RESULTS: Of 838 patients included in final analyses, 92% achieved complete remission. The 5-year overall survival, event-free survival, and CIR were 74%, 58%, and 26%, respectively. cKIT mutations and RAS mutations were not significantly associated with outcome. Patients with deletions of chromosome arm 9q [del(9q); n = 104] had a lower probability of complete remission (P = .01). Gain of chromosome 4 (+4; n = 21) was associated with inferior CIR and survival (P < .01). Anthracycline doses greater than 150 mg/m(2) and etoposide doses greater than 500 mg/m(2) in the first induction course and high-dose cytarabine 3 g/m(2) during induction were associated with better outcomes on various end points. Cumulative doses of cytarabine greater than 30 g/m(2) and etoposide greater than 1,500 mg/m(2) were associated with lower CIR rates and better probability of event-free survival. CONCLUSION: Pediatric patients with t(8;21)-AML and additional del(9q) or additional +4 might not be considered at good risk. Patients with t(8;21)-AML likely benefit from protocols that have high doses of anthracyclines, etoposide, and cytarabine during induction, as well as from protocols comprising cumulative high doses of cytarabine and etoposide.

• MeSH
• akutní myeloidní leukemie farmakoterapie   genetika   mortalita   MeSH
• antitumorózní látky terapeutické užití   MeSH
• antracykliny aplikace a dávkování   škodlivé účinky   MeSH
• chromozomální aberace účinky léků   MeSH
• cílená molekulární terapie * metody   MeSH
• cytarabin aplikace a dávkování   MeSH
• dítě MeSH
• etoposid aplikace a dávkování   MeSH
• homologní transplantace MeSH
• incidence MeSH
• indukční chemoterapie MeSH
• Kaplanův-Meierův odhad MeSH
• karyotyp MeSH
• lidé MeSH
• lidské chromozomy, pár 21 MeSH
• lidské chromozomy, pár 8 MeSH
• mezinárodní spolupráce MeSH
• mladiství MeSH
• mutace účinky léků   MeSH
• prediktivní hodnota testů MeSH
• předškolní dítě MeSH
• přežití po terapii bez příznaků nemoci MeSH
• protoonkogenní proteiny c-kit účinky léků   genetika   MeSH
• ras proteiny účinky léků   genetika   MeSH
• retrospektivní studie MeSH
• translokace genetická účinky léků   MeSH
• transplantace hematopoetických kmenových buněk MeSH
• výsledek terapie MeSH
• Check Tag
• dítě MeSH
• lidé MeSH
• mladiství MeSH
• mužské pohlaví MeSH
• předškolní dítě MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• multicentrická studie MeSH
• práce podpořená grantem MeSH
• Geografické názvy
• Německo MeSH