Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience.
نویسندگان
چکیده
Department of Pediatric Neurology, Center for Childhood White Matter Disorders, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands; Department of Pediatrics, Blood and Marrow Transplantation Program, Laboratory of Translational Immunology and Department of Hematology, and Sylvia Toth Center for Multidisciplinary Follow up after Hematopoietic Cell Transplantation, University Medical Center Utrecht, Utrecht, The Netherlands; University of Groningen, Department of Neurology, University Medical Center Groningen, Groningen, The
منابع مشابه
Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report.
We report the case of a 23-year-old woman who presented with an adult form of metachromatic leukodystrophy (MLD) evolving over one year with a progressive neurological deterioration. A non-myeloablative matched related haematopoietic stem cell transplantation (HSCT) with concomitant mesenchymal stromal cells (MSCs) infusion was performed. Engraftment occurred rapidly with no significant toxicit...
متن کاملImprovement of white matter changes on neuroimaging modalities after stem cell transplant in metachromatic leukodystrophy.
IMPORTANCE We sought to illustrate improvement of cerebral white matter changes in metachromatic leukodystrophy after treatment with hematopoietic stem cell transplant (HSCT). OBSERVATIONS We conducted serial magnetic resonance imaging (MRI) and proton magnetic resonance spectroscopy (1H-MRS) as standard follow-up after HSCT with cord blood in 1 patient with juvenile metachromatic leukodystro...
متن کاملEfficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by a functional deficiency of arylsulfatase A (ASA). Previous studies in ASA-knockout mice suggested enzyme replacement therapy (ERT) to be a promising treatment option. The mild phenotype of ASA-knockout mice did, however, not allow to examine therapeutic responses of the severe neurological symptoms that dominate MLD. We...
متن کاملNeurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome.
Objective To describe survival and neurological outcomes after HSCT for these disorders. Methods Seven CALD, 2 MLD and 2 MPS-IH patients underwent HSCT between 2007 and 2014. Neurological examinations, magnetic resonance imaging, molecular and biochemical studies were obtained at baseline and repeated when appropriated. Results Favorable outcomes were obtained with 4/5 related and 3/6 unrel...
متن کاملCorrection of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.
Gene-based delivery can establish a sustained supply of therapeutic proteins within the nervous system. For diseases characterized by extensive CNS and peripheral nervous system (PNS) involvement, widespread distribution of the exogenous gene may be required, a challenge to in vivo gene transfer strategies. Here, using lentiviral vectors (LVs), we efficiently transduced hematopoietic stem cells...
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ورودعنوان ژورنال:
- Blood
دوره 127 24 شماره
صفحات -
تاریخ انتشار 2016