title: Favorable outcome in infants with AML after intensive first- and second-line treatment: an AML-BFM study group report.
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.
authors.affiliation: Klinik und Poliklinik für Kinderheilkunde, Pediatric Hematology and Oncology, University Hospital Münster, Münster, Germany.
authors.forename: U
authors.initials: U
authors.lastname: Creutzig
authors.forename: M
authors.initials: M
authors.lastname: Zimmermann
authors.forename: J-P
authors.initials: JP
authors.lastname: Bourquin
authors.forename: M N
authors.initials: MN
authors.lastname: Dworzak
authors.forename: B
authors.initials: B
authors.lastname: Kremens
authors.forename: T
authors.initials: T
authors.lastname: Lehrnbecher
authors.forename: C
authors.initials: C
authors.lastname: von Neuhoff
authors.forename: A
authors.initials: A
authors.lastname: Sander
authors.forename: A
authors.initials: A
authors.lastname: von Stackelberg
authors.forename: I
authors.initials: I
authors.lastname: Schmid
authors.forename: J
authors.initials: J
authors.lastname: Starý
authors.forename: D
authors.initials: D
authors.lastname: Steinbach
authors.forename: J
authors.initials: J
authors.lastname: Vormoor
authors.forename: D
authors.initials: D
authors.lastname: Reinhardt
mesh_terms: D018943:Anthracyclines / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D011495:Histone-Lysine N-Methyltransferase; D006801:Humans; D007223:Infant; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality / Q000628:therapy*; D008297:Male; D015999:Multivariate Analysis; D051788:Myeloid-Lymphoid Leukemia Protein / Q000235:genetics; D016879:Salvage Therapy; D016896:Treatment Outcome
chemical_list: D018943:Anthracyclines; C497422:KMT2A protein, human; D003561:Cytarabine; D051788:Myeloid-Lymphoid Leukemia Protein; D011495:Histone-Lysine N-Methyltransferase