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Blood, 15 March 2002, Vol. 99, No. 6, pp. 2002-2008
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Factors associated with outcome after unrelated marrow
transplantation for treatment of acute lymphoblastic leukemia
in children
Ann E. Woolfrey,
Claudio Anasetti,
Barry Storer,
Kristine Doney,
Laurie A. Milner,
Eric L. Sievers,
Paul Carpenter,
Paul Martin,
Effie Petersdorf,
Frederick R. Appelbaum,
John A. Hansen, and
Jean E. Sanders
From the Fred Hutchinson Cancer Research Center and
University of Washington Departments of Pediatrics and Medicine,
Seattle, WA.
Acute lymphoblastic leukemia (ALL) is the most common indication
for transplantation of marrow from unrelated donors in children. We
analyzed results of this procedure in children with ALL treated according to a standard protocol to determine risk factors for outcome.
From January 1987 to 1999, 88 consecutively seen patients with ALL who
were younger than 18 years received a marrow transplant from an
HLA-matched (n = 56) or partly matched (n = 32) unrelated donor
during first complete remission (CR1; n = 10), second remission (CR2;
n = 34), third remission (CR3; n = 10), or relapse (n = 34).
Patients received cyclophosphamide and fractionated total-body irradiation as conditioning treatment and were given methotrexate and
cyclosporine for graft-versus-host disease (GVHD) prophylaxis. Three-year rates of leukemia-free survival (LFS) according to phase of
disease were 70% for CR1, 46% for CR2, 20% for CR3, and 9% for
relapse (P < .0001). Three-year cumulative relapse rates were 10%, 33%, 20%, and 50%, respectively, and 3-year cumulative rates of death not due to relapse were 20%, 22%, 60%, and 41%, respectively, for patients with CR1, CR2, CR3, and relapse. Grades III
to IV acute GVHD occurred in 43% of patients given HLA-matched transplants and in 59% given partly matched transplants
(P = .10); clinical extensive chronic GVHD occurred in
32% and 38%, respectively (P = .23). LFS rates were
lower in patients with advanced disease (P < .0001), age
10 years or older (P = .002), or short duration of CR1
(P = .007). Thus, in addition to phase of disease, age and duration of CR1 were predictors of outcome after unrelated-donor transplantation for treatment of ALL in children. Outcome was particularly favorable in younger patients with early phases of the disease.

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