Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome

Staba, Susan; Escolar, Maria L.; Poe, Michele D.; Kim, Young; Martin, Paul L.; Szabolcs, Paul; Allison-Thacker, June; Wood, Susan; Wenger, David A.; Rubinstein, Pablo; Hopwood, John J.; Krivit, William; Kurtzberg, Joanne

doi:10.1056/nejmoa032613

Cited by 455 publications

(351 citation statements)

References 28 publications

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“…The safety and efficacy of UCBT from unrelated donors was similar to that reported by previous studies (Staba et al 2004;Martin et al 2006;Prasad et al 2008). However, this is the only comprehensive prospective longitudinal study on a large cohort of patients with Hurler syndrome who were evaluated before and after transplanta- Fig.…”

Section: Discussionsupporting

confidence: 82%

“…Prophylaxis against graft-versus-host disease (GvHD) was given using cyclosporine for 9 months and methylprednisolone for 2-3 months. Supportive care was provided as previously described (Staba et al 2004;Thomas et al 2006;Prasad et al 2008). Most patients underwent tonsillectomy, adenoidectomy, and pressure-equalizing tube placement before transplant.…”

Section: Transplantation Proceduresmentioning

confidence: 99%

“…There is evidence of improved neurocognitive outcomes after umbilical cord blood transplantation (UCBT) in children with Hurler syndrome, adrenoleukodystrophy, and Krabbe disease, particularly when performed early in the disease course (Staba et al 2004;Escolar et al 2005;Beam et al 2007;Prasad et al 2008). Various studies of UCBT in Hurler syndrome patients report high rates of sustained engraftment with full donor chimerism and normal IDUA activity (Staba et al 2004;Martin et al 2006;Prasad et al 2008). Furthermore, umbilical cord blood is readily available, reducing the time between diagnosis and transplantation, which is important for optimal prognosis (Staba et al 2004;Escolar et al 2005).…”

Section: Introductionmentioning

confidence: 99%

“…Various studies of UCBT in Hurler syndrome patients report high rates of sustained engraftment with full donor chimerism and normal IDUA activity (Staba et al 2004;Martin et al 2006;Prasad et al 2008). Furthermore, umbilical cord blood is readily available, reducing the time between diagnosis and transplantation, which is important for optimal prognosis (Staba et al 2004;Escolar et al 2005).…”

Section: Introductionmentioning

confidence: 99%

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Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

et al. 2014

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Objectives: Hurler syndrome is characterized by progressive multisystem deterioration leading to early death in childhood. This prospective study evaluated the longterm outcomes of patients with Hurler syndrome who underwent umbilical cord blood transplantation from unrelated donors.Study design: Only patients with Hurler syndrome who underwent umbilical cord blood transplantation between December 1995 and March 2006 (n ¼ 25) and who were followed for at least 5 years (n ¼ 19) were included in the analysis. The patients were longitudinally evaluated by a multidisciplinary team of specialists following a standardized protocol.Results: Median age at transplantation was 15.9 months (range 2.1-35), and patients were followed up until a median age of 10

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Section: Discussionsupporting

confidence: 82%

Section: Transplantation Proceduresmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

et al. 2014

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“…While HSC therapies have shown therapeutic effects on visceral organs of MPS1 mice, they do not adequately correct 8, or show only partial benefit to the neurological disease 9. Clinical HSC transplantation also alleviated many of the disease manifestations of MPS1‐H patients with the exception of neurological symptoms and bone morphology 4, 10, 11, 12, 13. Additionally, the morbidity and mortality associated with HSC transplantation are notable, chiefly due to rejection and graft versus host disease.…”

Section: Introductionmentioning

confidence: 99%

Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model

Rodríguez¹,

Yanuaria²,

Parducho³

et al. 2017

Stem Cells Translational Medicine

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Mucopolysaccharidosis type 1 (MPS1) is an inherited lysosomal storage disorder caused by a deficiency in the glycosaminoglycan (GAG)‐degrading enzyme α‐l‐iduronidase (IDUA). In affected patients, the systemic accumulation of GAGs results in skeletal dysplasia, neurological degeneration, multiple organ dysfunction, and early death. Current therapies, including enzyme replacement and bone marrow transplant, improve life expectancy but the benefits to skeletal and neurological phenotypes are limited. In this study, we tested the therapeutic efficacy of liver‐directed transplantation of a placental stem cell, which possesses multilineage differentiation potential, low immunogenicity, and high lysosomal enzyme activity. Unfractionated human amniotic epithelial cells (hAECs) were transplanted directly into the liver of immunodeficient Idua knockout mouse neonates. The hAECs engraftment was immunohistochemically confirmed with anti‐human mitochondria staining. Enzyme activity assays indicated that hAECs transplantation restored IDUA function in the liver and significantly decreased urinary GAG excretion. Histochemical and micro‐computed tomography analyses revealed reduced GAG deposition in the phalanges joints and composition/morphology improvement of cranial and facial bones. Neurological assessment in the hAEC treated mice showed significant improvement of sensorimotor coordination in the hAEC treated mice compared to untreated mice. Results confirm that partial liver cell replacement with placental stem cells can provide long‐term (>20 weeks) and systemic restoration of enzyme function, and lead to significant phenotypic improvement in the MPS1 mouse model. This preclinical data indicate that liver‐directed placental stem cell transplantation may improve skeletal and neurological phenotypes of MPS1 patients. Stem Cells Translational Medicine 2017;6:1583–1594

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Cord Blood Transplants: Perinatal Stem Cells in Clinical Practice

Haspel

Ballen

2009

Perinatal Stem Cells

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Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome

Cited by 455 publications

References 28 publications

Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model

Cord Blood Transplants: Perinatal Stem Cells in Clinical Practice

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