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Blood, Vol. 92 No. 10 (November 15), 1998:
pp. 3546-3556
Bone Marrow Transplantation for Children Less Than 2 Years of Age
With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
Ann E. Woolfrey,
Ted A. Gooley,
Eric L. Sievers,
Laurie A. Milner,
Robert G. Andrews,
Mark Walters,
Paul Hoffmeister,
John A. Hansen,
Claudio Anasetti,
Eileen Bryant,
Frederick R. Appelbaum, and
Jean
E. Sanders
From the Fred Hutchinson Cancer Research Center and University of
Washington Departments of Pediatrics and Medicine, Seattle, WA.
We analyzed results of 40 infants less than 2 years of age who
received bone marrow transplants (BMT) between May 1974 and January
1995 for treatment of acute myelogenous leukemia (AML; N = 34) or
myelodysplastic syndrome (MDS; N = 6) to determine outcome and
survival performance. Among the AML patients, 13 were in first
remission, 9 were in untreated first relapse or second remission, and
12 were in refractory relapse. Patients were conditioned with
cyclophosphamide in combination with either total body irradiation (TBI; N = 29) or busulfan (N = 11). Source of stem cells included 6 autologous donors, 15 HLA genotypically identical siblings, 14 haploidentical family members, and 5 unrelated donors.
Graft-versus-host disease (GVHD) prophylaxis was methotrexate (MTX) for
17, MTX plus cyclosporine (CSP) for 14, or CSP plus prednisone for 3. Incidence of severe (grade 3-4) regimen-related toxicity was 10% and
transplant-related mortality was 10%. Acute GVHD (grades II-III) occurred in 39% of allogeneic patients, and chronic GVHD developed in
40%. Relapse, the most significant problem for patients in this study,
occurred in 1 MDS patient and 23 AML patients and was the cause of
death for 19 patients. The 2-year probabilities of relapse are 46%,
67%, and 92%, respectively, for patients transplanted in first
remission, untreated first relapse or second remission, and relapse.
One MDS and 8 AML patients received second marrow transplants for
treatment of relapse, and 5 of these survive disease-free for more than
1.5 years. All 6 MDS patients and 11 of 34 AML patients survive more
than 1.5 years later. The 5-year probabilities of survival and
disease-free survival are 54% and 38% for patients transplanted in
first remission and 33% and 22% for untreated first relapse or second
remission. None of the patients transplanted with refractory relapse
survive disease-free. Outcome was significantly associated with phase
of disease at transplantation and pretransplant diagnosis of
extramedullary disease. Long-term sequelae included growth failure and
hormonal deficiencies. Survival performance was a median of 100% (80%
to 100%) and neurologic development for all survivors was appropriate
for age. This study indicates that infants with AML have similar
outcome after BMT compared with older children and that BMT should be
performed in first remission whenever possible. In addition, allogeneic
BMT provides effective therapy for the majority of infants with MDS.
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