Results of unrelated cord blood transplantation (UCBT) in childhood acute myeloid leukemia (AML) have not been previously reported. We analyzed 95 children receiving UCB transplants for AML (20 in first complete remission [CR1], 47 in CR2, and 28 in more advanced stage). Poor prognosis cytogenetic abnormalities were identified in 29 cases. Most patients received a 1 or 2 HLA antigens-mismatched UCB transplants. The median number of collected nucleated cells (NCs) was 5.2 ؋ 10 7 / kg. Cumulative incidence (CI) of neutrophil recovery was 78% ؎ 4%, acute graftversus-host disease (GVHD) was 35% ؎ 5%, and 100-day transplantation-related mortality (TRM) was 20% ؎ 4%. In multivariable analysis, a collected NC dose higher than 5.2 ؋ 10 7 /kg was associated with a lower 100-day TRM. The 2-year CI of relapse was 29% ؎ 5% and was associated with disease status. The 2-year leukemia-free survival (LFS) was 42% ؎ 5% (59% ؎ 11% in CR1, 50% ؎ 8% in CR2, and 21% ؎ 9% for children not in CR). Children with poor prognosis cytogenetic features had similar LFS compared with other patients (44% ؎ 11% vs 40% ؎ 8%). In CR2, LFS was not influenced by the length of CR1 (53% ؎ 11% in CR1 < 9.5 months compared with 50% ؎ 12% in later relapses). We conclude that UCBT is a therapeutic option for children with very poorprognosis AML and who lack an HLAidentical sibling.
IntroductionBone marrow transplantation (BMT) from an HLA-matched sibling or unrelated donor plays a major role in the treatment of children with relapsed acute myeloid leukemia (AML). [1][2][3][4][5][6] However, although there are currently more than 8 million donors registered in marrow donor registries around the world, a substantial proportion of children who lack a sibling donor will never undergo BMT from an HLA-matched unrelated donor either because such a donor cannot be found or because the time to identify a donor is too long. Moreover, for those children who received unrelated bone marrow transplants, increased HLA disparity adversely affects survival because of high risk of graft-versus-host disease (GVHD) and opportunistic infections. [7][8][9] The use of haploidentical family donors provides a potential source of hematopoietic stem cells for children who lack both a sibling and an unrelated donor. [10][11] T-cell depletion of the graft can in part overcome the risk of severe GVHD, but it substantially increases the risk of severe and prolonged posttransplantation immunodeficiency.Hematopoietic stem cells from an unrelated cord blood (UCB) transplant can restore hematopoiesis and immune function after a myeloablative conditioning regimen, even if the graft is not perfectly HLA identical to the recipient. [12][13][14][15] This important medical advance led to the establishment of large cord blood banks that made possible the use of UCB to provide transplants for patients who lack a conventional related or unrelated donor. In addition, UCB offers the advantage of significantly faster availability of banked cryopreserved UCB units compared with the availability of u...
A high incidence of hyperleukocytosis in children with APL was confirmed. Besides low toxicity and a high degree of compliance, a risk-adapted therapy combining ATRA and anthracycline monochemotherapy showed an antileukemic efficacy comparable to those previously reported with other chemotherapy combinations in children.
This prospective study focused on risk factors and clinical outcome of pulmonary and cardiac late effects after allogeneic hematopoietic stem cell transplantation (allo-HSCT). We prospectively evaluated 162 children by pulmonary function tests (PFTs) and cardiac shortening fraction (SF) before allo-HSCT and yearly up to the 5th year of follow-up. The 5-year cumulative incidence of lung and cardiac impairment was 35 (hazard rate=0.03) and 26% (hazard rate=0.06), respectively. Patients presenting abnormal PFTs and SF at last follow-up were 19 and 13%, respectively, with a median Lansky performance status of 90% (70-100). Chronic graft-versus-host disease (c-GVHD) was the major risk factor for reduced lung function in univariate (P=0.02) and multivariate analysis (P=0.02). Total body irradiation (TBI) alone and TBI together with pre-transplant anthracycline administration were significant risk factors for reduced cardiac function in univariate analysis, only (P=0.04 and 0.004, respectively). In conclusion, our prospective study demonstrates an asymptomatic post-allo-HSCT deterioration of pulmonary and cardiac function in some long-term survivors, who had been transplanted in childhood, and thus emphasizes the need for lifelong cardiopulmonary monitoring and the development of new strategies both to reduce pre-transplant cardiotoxic regimens and to treat more efficiently c-GVHD.
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