Randomized Controlled Clinical Trial Of Treating Childhood AML With Homoharringtonine-based Regimens

1: Randomized controlled clinical trial of treating childhood AMLwith Homoharringtonine-based regimensObjectiveTo explore the possibility of replacing Anthracycline with homoharringtonine in treatingpediatric acute myelogenic leukemia (AML)(excluding APL) by Randomized clinical trial,comparing the efficacy and adverse effects of SCMC-AML-2009A with SCMC-AML-2009B.MethodsDe no vo AML cases diagnosed from May2009were randomly divided into groupA andgroup B(Homoharringtonine-based regimens). The efficacy and adverse effects of both groupswere analyzed.Results①Total of152children with AML were recruited，with the ratio of boys to girls,1.67:1.The complete remission (CR) rate were82.5%and80.6%in group A and B, respectively. Themedian follow-uptime were10months (range:14days～54months). The CR rate after the firstcourse of chemotherapy was80.3%，the total CR rate is90.1%. There was no statisticalsignificant relevant between efficacy and age, gender, the FAB classification (P>0.05).However，the disease reoccurrence was more common in children’s older than10years.②The total CR rate were90.4%and89.9%in group A and B, respectively. There was nosignificant difference between two groups. The4-year EFS rate were59%and57%in group Aand B, respectively.(P>0.05).However，the incidence of infection and sever infection washigher in group A than that was in B group (P <0.05).Conclusions①Comparing SCMC-AML-2009with AML-XH-99, the former improved the CR quality inpediatric AML，especially improved the first course CR rate and4-year EFS rate t②Though the outcomes of chemotherapy by protocol A and B were similar in treating deno vo acute myeloid leukemia.the side effects in B is lower. 2：The analysis of c-kit gene mutations in AML1-ETO-positive AMLObjectivesMonitor c-kit gene mutation in de no vo AML1-ETO+AML patients, to evaluate the clinicalcharacteristics and prognosis of mutated c-kit (mutKIT).Methods①cDNAs from26newly diagnosed patients with AML1-ETO+AMLwere screened formutKIT in exon8and17by PCR and sequencing.②Follow up the clinical and lab datas of these cases, then analyze c-kit gene mutation andprognosis.Results①Among26AML1-ETO+,12(46.2%) had mutKIT in exon17and exon8.Point mutationin exon17occurred mostly clustered in a hot spot D816spanning residues hated the C-KITkinase function domain (33.3%).②The peripheral WBC counts, age of at diagnosis and gender of patients with mutKIT orwtKIT were similar.The recurrence rate were41.6%and16.7%in mutKIT andwtKIT,respectively，the incidence of recurrence in mutKIT group was higher than wtKIT group,The4-year FFS rate (45%) was lower for patients with mutKIT than which for wtKIT patients(EFS rate54%).The4-year0S rate was45%and43.5%%in mutKIT and wtKIT group,respectively (P>0.05).③The recurrence rate were66.7%and17.6%in mutKIT8and others,respectively，therewas significant difference between them,P <0.05.Conclusion①There were various types of mutKIT in exon17in AML patients with AML1-ETO+andthe most frequently detected mutation of KIT exon17was D816and the most frequentlydetected mutation of exon8was insertion or deletion in419.②The AML1-ETO+patients accompanied by mutKIT had more trend to relapse, and theirEFS were short, predicting poor prognosis.