Transplant outcomes in mucopolysaccharidoses

Seminars in Hematology
Vinod K Prasad, J Kurtzberg

Abstract

The mucopolysaccharidoses (MPSs) are inherited metabolic disorders (IMDs) caused by single-gene defects leading to progressive cellular accumulation of glycosaminoglycans (GAGs) and damage to multiple organs, including the central nervous, musculoskeletal, cardiorespiratory, and other systems. Hurler syndrome (MPS IH), the most severe form, is the prototypical model. Enzyme replacement therapy (ERT), available for MPS I, II, and VI, is beneficial in some patients. However, ERT does not improve neurocognitive function because of its inability to cross the blood-brain barrier. In contrast, allogeneic hematopoietic stem cell transplantation (HSCT) allows donor-derived, enzyme-producing cells to migrate to the brain and other organs to provide permanent enzyme therapy and thus help somatic organs, improve neurocognitive function and quality of life, and prolong survival, particularly when performed early in the course of the disease. Bone marrow has been the graft source in the past. However, in the last 5 years many patients have been treated with unrelated donor (URD) umbilical cord blood transplant (UCBT), allowing rapid and increased access to transplantation with favorable outcomes. This review describes published and our inst...Continue Reading

References

Jan 1, 1992·Journal of Inherited Metabolic Disease·A VellodiK Hugh-Jones
Sep 1, 1995·Archives of Disease in Childhood·M A GatzoulisA N Redington
Jan 1, 1995·Journal of Inherited Metabolic Disease·E G ShapiroW Krivit
Jan 1, 1995·Journal of Inherited Metabolic Disease·G V CoppaP Di Natale
Apr 15, 1993·American Journal of Medical Genetics·C B WhitleyJ H Kersey
Jul 1, 1996·The Journal of Pediatrics·E J McKinnisC R Scott
Jul 22, 1998·The Journal of Pediatrics·C PetersW Krivit
Jul 22, 1998·The Journal of Pediatrics·N GuffonP Guibaud
Jun 1, 2000·Bone Marrow Transplantation·C Peters, W Krivit
Mar 7, 2003·Bone Marrow Transplantation·C PetersUNKNOWN Working Party on Inborn Errors, European Bone Marrow Transplant Group
May 7, 2004·The New England Journal of Medicine·Joseph Muenzer, Amy Fisher
Jun 11, 2004·Journal of Inherited Metabolic Disease·R J Desnick
Jun 23, 2004·Seminars in Cell & Developmental Biology·Steven U Walkley
Jul 21, 2004·The Journal of Experimental Medicine·Gesine KöglerPeter Wernet
Feb 3, 2005·British Journal of Haematology·Ashok Vellodi
Dec 17, 2005·Pediatric Physical Therapy : the Official Publication of the Section on Pediatrics of the American Physical Therapy Association·Stacey C DusingMaria Escolar
Jan 31, 2006·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Paul L MartinJoanne Kurtzberg
Jun 10, 2006·Journal of Inherited Metabolic Disease·Jaap Jan Boelens
Aug 15, 2006·Journal of Developmental and Behavioral Pediatrics : JDBP·Kendra J BjorakerElsa G Shapiro
Apr 28, 2007·Documenta Ophthalmologica. Advances in Ophthalmology·D TzetziG N Dutton
Sep 25, 2007·The Journal of Pediatrics·Paul J OrchardJakub Tolar
Jan 8, 2008·Bone Marrow Transplantation·V K Prasad, J Kurtzberg
Apr 16, 2008·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Mieke AldenhovenTom J de Koning
Dec 23, 2008·Biochemical and Biophysical Research Communications·Shawn J ChuaRobert F Casper

❮ Previous
Next ❯

Citations

Sep 6, 2012·Archivum Immunologiae Et Therapiae Experimentalis·Zyta Banecka-MajkutewiczGrzegorz Węgrzyn
Nov 1, 2011·European Journal of Pediatrics·Joseph MuenzerPaul Harmatz
Jan 12, 2012·European Journal of Pediatrics·Kristin D'AcoPaige Kaplan
Jul 12, 2011·Journal of Inherited Metabolic Disease·Elizabeth A BraunlinRoberto Giugliani
Jul 7, 2011·Journal of Inherited Metabolic Disease·Lorne Andrew ClarkeJohn Mitchell
Nov 16, 2012·Journal of Inherited Metabolic Disease·Kenneth I BergerDavid M Rapoport
Nov 3, 2010·Bone Marrow Transplantation·L D de HossonA V M B Schattenberg
Sep 4, 2013·Nature Reviews. Genetics·Kym M BoycottAlex E MacKenzie
Jan 27, 2012·Pediatric Research·Jos DomenJignesh Dalal
Jan 11, 2012·Rheumatology·Joseph Muenzer
Jan 11, 2012·Rheumatology·Vassili Valayannopoulos, Frits A Wijburg
Aug 1, 2010·Therapeutic Advances in Endocrinology and Metabolism·Gregory M Pastores
Feb 16, 2013·Genetics and Molecular Biology·Roberto Giugliani
Feb 16, 2013·Therapeutics and Clinical Risk Management·Mohamed F Algahim, G Hossein Almassi
Mar 23, 2011·Annual Review of Biomedical Engineering·Stephen F BadylakKorkut Uygun
Nov 23, 2013·Current Rheumatology Reports·Rolando Cimaz, Francesco La Torre
Aug 31, 2014·Atlas of the Oral and Maxillofacial Surgery Clinics of North America·Victor Nannini
Aug 5, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·Salim S El-AmouriDao Pan
Aug 15, 2014·Metabolites·Shunji TomatsuTadao Orii
Dec 17, 2014·Expert Opinion on Drug Delivery·Daniel A WolfWalter C Low
Jul 26, 2014·Molecular Genetics and Metabolism Reports·Pravin PatelShunji Tomatsu
Nov 2, 2013·Neurologic Clinics·Gregory M Pastores, Gustavo H B Maegawa
Oct 7, 2014·Molecular Genetics and Metabolism·Christian J HendrikszChristine Haller
Dec 30, 2014·Molecular Genetics and Metabolism·Shunji TomatsuTadao Orii
Mar 23, 2011·Clinica Chimica Acta; International Journal of Clinical Chemistry·Carole S ElbinX Kate Zhang
Oct 29, 2010·Molecular Genetics and Metabolism·Sean TurbevilleAmal Al-Seraihy
Apr 2, 2010·Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis·Paolo Rebulla
Jul 13, 2014·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Troy C LundPaul J Orchard
Mar 13, 2014·Journal of Clinical Pharmacy and Therapeutics·H Noh, J I Lee
May 28, 2014·La Presse médicale·Pierre Kaminsky, Olivier Lidove
Aug 15, 2014·Pediatric Hematology and Oncology·Wolfgang SchwingerChristian Urban
May 20, 2015·Best Practice & Research. Clinical Endocrinology & Metabolism·Gregory M Pastores, Derralynn A Hughes
Mar 21, 2012·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Mary EapenK Scott Baker
Jan 4, 2012·Molecular Genetics and Metabolism·Clara D M van Karnebeek, Sylvia Stockler
Jun 15, 2016·European Journal of Internal Medicine·John MitchellKlane White
Dec 3, 2016·Scientific Reports·Gerald V RaymondTroy C Lund

❮ Previous
Next ❯

Related Concepts

Related Feeds

Blood Brain Barrier Regulation in Health & Disease

The blood brain barrier is essential in regulating the movement of molecules and substances in and out of the brain. Disruption to the blood brain barrier and changes in permeability allow pathogens and inflammatory molecules to cross the barrier and may play a part in the pathogenesis of neurodegenerative disorders. Here is the latest research in this field.

Adult Stem Cells

Adult stem cells reside in unique niches that provide vital cues for their survival, self-renewal, and differentiation. They hold great promise for use in tissue repair and regeneration as a novel therapeutic strategies. Here is the latest research.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Allogenic & Autologous Therapies

Allogenic therapies are generated in large batches from unrelated donor tissues such as bone marrow. In contrast, autologous therapies are manufactures as a single lot from the patient being treated. Here is the latest research on allogenic and autologous therapies.

Blood Brain Barrier Chips

The blood brain barrier (BBB) is comprised of endothelial cells that regulate the influx and outflux of plasma concentrations. Lab-on-a-chip devices allow scientists to model diseases and mechanisms such as the passage of therapeutic antibodies across the BBB. Discover the latest research on BBB chips here.

Blood Brain Barrier

The blood brain barrier is a border that separates blood from cerebrospinal fluid. Discover the latest search on this highly selective semipermeable membrane here.

Related Papers

Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
Jaap Jan BoelensEUROCORD, Inborn error Working Party of EBMT and Duke University
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
Mieke AldenhovenTom J de Koning
Rheumatology
Vassili Valayannopoulos, Frits A Wijburg
© 2021 Meta ULC. All rights reserved