BONE-MARROW TRANSPLANTATION FOR CHILDREN LESS-THAN 2 YEARS OF AGE WITH ACUTE MYELOGENOUS LEUKEMIA OR MYELODYSPLASTIC SYNDROME

We analyzed results of 40 infants less than 2 years of age who receive d bone marrow transplants (BMT) between May 1974 and January 1995 for treatment of acute myelogenous leukemia (AML; N = 34) or myelodysplast ic syndrome (MDS; N = 6) to determine outcome and survival performance . Among the AML patients, 13 were in first remission, 9 were in untrea ted first relapse or second remission, and 12 were in refractory relap se. Patients were conditioned with cyclophosphamide in combination wit h either total body irradiation (TBI; N = 29) or busulfan (N = 11). So urce 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 predniso ne for 3. Incidence of severe (grade 3-4) regimen-related toxicity was 10% and transplant-related mortality was 10%. Acute GVHD (grades II-I II) occurred in 39% of allogeneic patients, and chronic GVHD developed in 40%. Relapse, the most significant problem for patients in this st udy, occurred in 1 MDS patient and 23 AML patients and was the cause o f death for 19 patients. The 5-year probabilities of relapse are 46%, 67%, and 92%, respectively, for patients transplanted in first remissi on, 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 ye ars later. The 5-year probabilities of survival and disease-free survi val are 54% and 38% for patients transplanted in first remission and 3 3% and 22% for untreated first relapse or second remission. None of th e patients transplanted with refractory relapse survive disease-free. Outcome was significantly associated with phase of disease at transpla ntation and pretransplant diagnosis of extramedullary disease. Longter m sequelae included growth failure and hormonal deficiencies. Survival performance was a median of 100% (80% to 100%) and neurologic develop ment for all survivors was appropriate for age. This study indicates t hat infants with AML have similar outcome after BMT compared with olde r children and that BMT should be performed in first remission wheneve r possible. In addition, allogeneic BMT provides effective therapy for the majority of infants with MDS. (C) 1998 by The American Society of Hematology.