Y. Ravindranath et al., AUTOLOGOUS BONE-MARROW TRANSPLANTATION VERSUS INTENSIVE CONSOLIDATIONCHEMOTHERAPY FOR ACUTE MYELOID-LEUKEMIA IN CHILDHOOD, The New England journal of medicine, 334(22), 1996, pp. 1428-1434
Background. The value of autologous bone marrow transplantation in the
treatment of children with acute myeloid leukemia (AML) is unknown. W
e compared autologous bone marrow transplantation with intensive conso
lidation chemotherapy as treatments for children with AML in first rem
ission. Methods. We induced remission with one course of daunorubicin,
cytarabine, and thioguanine, followed by one course of high-dose cyta
rabine (3 g per square meter of body-surface area for six doses), Pati
ents in remission after the second course of induction therapy were el
igible for randomization, Between June 1988 and March 1993, 552 of 649
enrolled patients who could be evaluated (85 percent) entered remissi
on, A total of 209 patients were not eligible for randomization; of th
e remaining 343 patients, 232 were randomly assigned to receive six co
urses of intensive chemotherapy (117 patients) or autologous transplan
tation (115 patients), Of the original 649 patients, 189, including 21
with Down's syndrome, were nonrandomly assigned to receive intensive
chemotherapy. Results, The mean (+/-SE) rates of event-free survival a
nd overall survival for the entire group at three years were 34+/-2.5
percent and 42+/-2.6 percent, respectively, For patients who were rand
omly assigned to one of the two treatment groups, the rates of event-f
ree survival three years after randomization were not significantly di
fferent in the two groups when examined by intention-to-treat analysis
: 36+/-5.8 percent for the intensive-chemotherapy group as compared wi
th 38+/-6.4 percent for the autologous-transplantation group; and the
relative risk of treatment failure for the chemotherapy group as compa
red with the autologous-transplantation group was 0.81 (P=0.20 by the
log-rank test; 95 percent confidence interval, 0.58 to 1.12), Overall
survival at three years followed a similar pattern. There was a lower
relapse rate (31 percent vs, 58 percent, P<0.001) but a higher rate of
treatment-related mortality (15 percent vs, 2.7 percent, P=0.005) in
the group treated with autologous transplantation than in the intensiv
e-chemotherapy group. The event-free survival at three years for the n
onrandomized intensive-chemotherapy group was 39+/-5.1 percent, and fo
r a contemporaneous group of patients each of whom received a histocom
patible bone marrow transplant from a sibling, it was 52+/-8.0 percent
. Conclusions. Treatment of children with AML in first remission with
either autologous bone marrow transplantation or intensive chemotherap
y prolongs event-free survival equally. (C) 1996, Massachusetts Medica
l Society.