A Staging System for Infantile Krabbe Disease to Predict Outcome After Unrelated Umbilical Cord Blood Transplantation

Escolar, Maria L.; Poe, Michele D.; Martin, Holly; Kurtzberg, Joanne

doi:10.1542/peds.2006-0747

Cited by 78 publications

(71 citation statements)

References 6 publications

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“…Previous reports of MLD and other leukodystrophies have made recommendations to transplant patients in an early age and disease to prevent serious neurological damage. 25,[35][36][37] In patient D, MRI showed progression of white matter lesions in the period of lower enzyme levels but remained stable during further follow-up. DLI was necessary to convert mixed chimerism into complete donor chimerism, resulting in higher levels of ARSA and stabilization of cerebral progression.…”

Section: Discussionmentioning

confidence: 99%

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

Hosson

Warrenburg

Preijers

et al. 2010

Bone Marrow Transplant

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Five patients with adult-onset metachromatic leukodystrophy (MLD) underwent allo-SCT. Conditioning was reduced in intensity and grafts were obtained from voluntary unrelated donors. All but one graft were depleted of T-lymphocytes. Patient age at transplantation varied from 18 to 29 (median, 27) years. Two patients rejected their graft and MLD progressed. The recipient of the unmanipulated graft converted to complete donor chimerism with normalization of arylsulphatase A (ARSA) levels. Despite ARSA normalization, he deteriorated. Another patient was a mixed chimera. Following escalated doses of donor lymphocyte infusions he converted to complete donor chimerism. His levels of ARSA correlated positively with the percentage of donor cells and MLD was not progressive. The fifth patient died after 35 days from complications associated with GVHD. We conclude that results of allo-SCT in symptomatic MLD patients are poor. However, allo-SCT may stop progression of MLD in selected patients.

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Section: Discussionmentioning

confidence: 99%

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

Hosson

Warrenburg

Preijers

et al. 2010

Bone Marrow Transplant

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“…16 Although hematopoietic stem cell transplantation performed during the first month of life may provide benefit to some infants with infantile-onset KD, 23,24,38 it is challenging for Specialty Care Centers to diagnose, evaluate, and refer infants to qualified transplant centers within the first few weeks of life. Even when transplantation is performed early, delays in development of gross motor skills have developed in most patients, including our two surviving patients.…”

Section: Original Research Articlementioning

confidence: 99%

“…21 Early studies showed that a small cohort of infantile KD patients who received umbilical cord blood transplantation from unrelated donors prior to symptom onset had preserved neurological function. [22][23][24] However, even when transplantation is performed early, delays in development of gross motor skills have developed in most patients. 22 It is critical that families be fully educated about benefits, risks, and limitations of treatment following a positive screen.…”

mentioning

confidence: 99%

Newborn screening for Krabbe disease in New York State: the first eight years’ experience

Orsini

Kay

Saavedra-Matiz

et al. 2016

Genetics in Medicine

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125

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“…Transplantation of umbilical cord blood in babies with infantile Krabbe's disease that favorably altered their natural history could be the first, promising example from human clinic. Infants who underwent HUCB cell graft before the development of symptoms showed progressively increasing central myelination and continued gains in developmental skills and cognitive function (Escolar et al, 2005;Escolar et al, 2007). These therapeutic benefits of cord blood infusion may hold hope for slowing the progression of other chronic neural diseases.…”

Section: The Cord Blood Challenge For Brain Repairmentioning

confidence: 99%

A novel, neural potential of non-hematopoietic human umbilical cord blood stem cells

Domanska-Janik¹,

Bużañska²,

Łukomska³

2008

Int. J. Dev. Biol.

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From the time of discovery that among the cord blood mononuclear cell population there are cells capable of changing their fate towards the neural lineage and producing functional neurons and macroglial cells, our attempts have been focused on the understanding of the underlying mechanism of this transition. We have deciphered the first steps of neural stem/ progenitor gene induction in aggregating culture of cord blood mononuclear cells, their rapid phenotypic conversion under the influence of neuromorphogenic signals due to mitogen activation and their ability to expand and develop a prototypic, long-living line with neural stem cell properties. Evidence has accumulated that human umbilical cord-derived and neurally committed cells, due to their capacity for self-renewal, multilineage differentiation, plasticity and ability for long-lasting growth in vitro, provide unique material for the cell therapy of a wide spectrum of neurological diseases. The putative regenerating potential of these cord blood-derived neural stem/progenitor cells was evaluated after transplantation in experimental models of brain injury. In spite of initial promising data, the results indicate an urgent need to improve available animal model protocols in order to increase immuno-tolerance toward transplanted human cells.

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A Staging System for Infantile Krabbe Disease to Predict Outcome After Unrelated Umbilical Cord Blood Transplantation

Cited by 78 publications

References 6 publications

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

Newborn screening for Krabbe disease in New York State: the first eight years’ experience

A novel, neural potential of non-hematopoietic human umbilical cord blood stem cells

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