Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy
Child; Humans; Male; Survival Rate; Treatment Outcome; Cognition; Predictive Value of Tests; Motor Activity; Preschool; Outcomes; Adrenoleukodystrophy/complications/mortality/physiopathology/therapy; Cord Blood Stem Cell Transplantation/methods; Graft Survival; Graft vs Host Disease; Histocompatibility Testing; Language Development; Myeloablative Agonists/therapeutic use; Neurophysiology; Transplantation Conditioning/methods
Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents in young boys and adolescent boys. Allogeneic bone marrow transplantation has been used to halt progression of the disease. However, many patients lack suitable HLA- matched related donors and must rely on unmatched donors for a source of stem cells. The purpose of this study was to evaluate outcomes of unrelated donor umbilical cord blood transplantation after chemotherapy-based myeloablative conditioning and retrospectively determine if baseline studies correlate and help predict outcome. Between November 22, 1996, and November 3, 2005, 12 boys with X-linked ALD who lacked HL- matched related donors were referred to Duke University Medical Center for transplantation. These children were conditioned with myeloablative therapy including busulfan, cyclophosphamide, and antithymocyte globulin before receiving umbilical cord-blood transplants from unrelated donors. Baseline studies of neurophysiologic, neuroimaging, and neurodevelopmental status were performed and patients were subsequently evaluated for survival, engraftment, graft-versus-host disease, and neurodevelopmental outcomes. A substudy evaluated whether baseline neuroimaging and neurophysiologic studies correlated with cognitive and motor function and if these studies were predictive of posttransplantation outcomes. The umbilical cord blood grafts had normal levels of very long chain fatty acids. They delivered a median of 6.98 x 10(7) nucleated cells per kilogram of recipient body weight and were discordant for up to 4 of 6 HLA markers. Neutrophil engraftment occurred at a median of 22.9 days after transplantation. Three patients had grade II-IV acute graft-versus-host disease; 2 had extensive chronic graft-versus-host disease. Cumulative incidence of overall survival of the group at 6 months is 66.7% (95% confidence interval 39.9-93.3%). Median follow-up was 3.3 years (range 12 days to 6.3 years). As previously reported with bone marrow transplantation, symptomatic patients faired poorly with lower survival and rapid deterioration of neurologic function. This study included 3 patients transplanted at a very young age (2.6-3.5 years) before the onset of clinical symptoms who continue to develop at a normal rate for 3-5 years posttransplant. Although baseline Loes scores correlated with cognitive and motor outcome, neurophysiologic studies failed to show statistically significant differences. Transplantation of boys with X-linked ALD using partial HLA-matched umbilical cord blood yields similar results to those previously reported after bone marrow transplantation. Superior outcomes were seen in neurologically asymptomatic boys less than 3.5 years of age at the time of transplantation. Baseline Loes scores were a strong predictor of cognitive and motor outcome.
2007
Beam D; Poe MD; Provenzale JM; Szabolcs P; Martin PL; Prasad V; Parikh S; Driscoll T; Mukundan S; Kurtzberg J; Escolar ML
Biology Of Blood And Marrow Transplantation : Journal Of The American Society For Blood And Marrow Transplantation
2007
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1016/j.bbmt.2007.01.082" target="_blank" rel="noreferrer">10.1016/j.bbmt.2007.01.082</a>
Prevalence and Impact of Financial Hardship Among New England Pediatric Stem Cell Transplant Families
PURPOSE: Poverty is correlated with negative health outcomes in pediatric primary care and subspecialties; its association with childhood HSCT patterns of care and clinical outcomes is not known. We describe family-reported financial hardship at a primary referral center in New England, and explore the relationship between measures of poverty and child patterns of care and clinical outcomes. PATIENTS AND METHODS: Forty-five English-speaking parents of children status-post allogeneic HSCT in the prior twelve months completed a one-time survey (response rate 88%). RESULTS: Low-income families defined as ≤200% federal poverty level (FPL) were compared to all others. Eighteen (40%) families reported pre-HSCT incomes ≤200% FPL. Material hardship including food, housing, or energy insecurity was reported by 17 (38%) families in the cohort. Low-income families reported disproportionate transplant-related income losses with 7 (39%) reporting annual income losses of >40% compared to 2 (18%) wealthier families (p=0.02). In univariate analyses, 11 (61%) low-income children experienced Graft Versus Host Disease (GVHD) of any grade in the first 180 days post-HSCT compared to 2 (7%) wealthier children (p=0.004). CONCLUSION: We conclude that low income and in particular, material hardship, are prevalent in a New England pediatric HSCT population and represent targets for improvement in quality of life. The role of poverty in mediating GVHD deserves further investigation in larger studies which can control for known risk factors, and may provide a targetable source of transplant-associated morbidity.
2014-10
Bona K; London WB; Guo D; Abel G; Lehmann L; Wolfe J
Biology Of Blood And Marrow Transplantation : Journal Of The American Society For Blood And Marrow Transplantation
2014
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1016/j.bbmt.2014.10.016" target="_blank" rel="noreferrer">10.1016/j.bbmt.2014.10.016</a>