title: [Long-term follow-up of treatment outcome and prognosis on 46 children with acute promyelocytic leukemia].
abstract: OBJECTIVE
  Acute promyelocytic leukemia (APL) is a specific type of hematopoietic malignancy, accounting for 10% of the de novo acute myeloid leukemia (AML). The data on long-term outcome of APL in children are limited. The aim of this study was to investigate the clinical biological features, diagnosis, prognosis and long-term survival of childhood APL.
  
  
  METHODS
  A total of 46 children with newly diagnosed APL from April 1998 to October 2005 were enrolled into this study. Induction treatment containing all-trans retinoic acid (ATRA) plus daunorubicin (DNR) or pirarubicin (THP) was performed on these patients, followed by 6 courses of chemotherapy consolidation: DNR, homoharringtonine or etoposide plus Ara-C. A maintenance therapy was then administered once 3-6 months. The total period of treatment was 2.5 years.
  
  
  RESULTS
  Of the 39 patients who had completed the regular treatment, 36 (92.3%) achieved a complete remission. The 5-year cumulative incidence of relapse (CIR) was 28.6%. The estimated overall survival (OS) rates at 1, 3 and 5 years were (86.1 +/- 5.8)%, (76.1 +/- 7.5)% and (70.2 +/- 8.9)% respectively, while the event free survival (EFS) rates were (78.4 +/- 6.8)%, (63.6 +/- 8.7)% and (53.1 +/- 10.0)% respectively. The 5-year OS rate of patients with WBC less than or equal to 10.0 X 10(9)/L was (81.4 +/- 10.3)%, which was significantly higher than that with WBC greater than 10.0 X 10(9)/L[(51.6 +/- 14.7)%, P < 0.05]. Five patients with RT-PCR positive for PML/RARalpha S (short) subtype died eventually although all of them achieved CR, but none of the 13 patients with PML/RARalpha L (long) subtype died.
  
  
  CONCLUSIONS
  Remission induction therapy with ATRA + DNR or THP is effective and safe for newly diagnosed childhood APL. The remission induction therapy combined with chemotherapy containing high/intermediate dose Ara-C can improve the long-term survival rates of APL patients. High WBC count and S subtype of PML-RARa are two poor prognostic factors for children with APL.
authors.affiliation: Department of Hematology/Oncology, Children's Hospital of Zhejiang University School of Medicine, Hangzhou 310003, China.
authors.forename: Xiao-Jun
authors.initials: XJ
authors.lastname: Xu
authors.forename: Shu-Wen
authors.initials: SW
authors.lastname: Shi
authors.forename: Yong-Min
authors.initials: YM
authors.lastname: Tang
authors.forename: Hua
authors.initials: H
authors.lastname: Song
authors.forename: Shi-Long
authors.initials: SL
authors.lastname: Yang
authors.forename: Jian
authors.initials: J
authors.lastname: Wei
authors.forename: Wei-Qun
authors.initials: WQ
authors.lastname: Xu
authors.forename: Bin-Hua
authors.initials: BH
authors.lastname: Pan
authors.forename: Ying-Hu
authors.initials: YH
authors.lastname: Chen
authors.forename: Fen-Ying
authors.initials: FY
authors.lastname: Zhao
authors.forename: Hong-Qiang
authors.initials: HQ
authors.lastname: Shen
authors.forename: Bai-Qin
authors.initials: BQ
authors.lastname: Qian
authors.forename: Ling-Yan
authors.initials: LY
authors.lastname: Zhang
authors.forename: Bo-Tao
authors.initials: BT
authors.lastname: Ning
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D015473:Leukemia, Promyelocytic, Acute / Q000188:drug therapy* / Q000401:mortality; D008297:Male; D015514:Oncogene Proteins, Fusion / Q000235:genetics; D011379:Prognosis; D015996:Survival Rate; D016896:Treatment Outcome; D014212:Tretinoin / Q000008:administration & dosage
chemical_list: D015514:Oncogene Proteins, Fusion; C093476:promyelocytic leukemia-retinoic acid receptor alpha fusion oncoprotein; D014212:Tretinoin