The clinical outcome of Hurler syndrome after stem cell transplantation

Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
Mieke AldenhovenTom J de Koning

Abstract

Hurler syndrome (HS) is a severe inborn error of metabolism causing progressive multi-system morbidity and death in early childhood. At present, stem cell transplantation (SCT) is the only available treatment that can prevent central nervous system disease progression in HS patients. Although SCT has been shown to be effective for several important clinical outcome parameters, the reported clinical outcome after successful SCT is variable among HS patients and there are still some major limitations. This review will focus on the clinical outcome of HS patients after successful SCT, with particular emphasis on the long-term outcome and complications. In addition, factors that are suggested to contribute to the variable outcome are outlined, as well as the limitations of SCT in HS patients.

References

Jun 1, 1992·The American Journal of Cardiology·E A BraunlinC B Whitley
Jun 1, 1992·The Annals of Otology, Rhinology, and Laryngology·J K BredenkampD M Crockett
Sep 1, 1990·Archives of Disease in Childhood·J E Wraith, S M Alani
Feb 1, 1987·The Journal of Clinical Investigation·R M ShullG Constantopoulos
Jul 1, 1988·Medicine·G L Semenza, R E Pyeritz
Sep 1, 1969·Proceedings of the National Academy of Sciences of the United States of America·J C FratantoniE F Neufeld
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
Mar 1, 1995·Acta Paediatrica·M A Cleary, J E Wraith
Apr 15, 1993·American Journal of Medical Genetics·C B WhitleyJ H Kersey
Jul 18, 1996·The New England Journal of Medicine·J KurtzbergP Rubinstein
Jul 1, 1997·The Journal of Bone and Joint Surgery. British Volume·F S HaddadM C Pitt
Jul 22, 1998·The Journal of Pediatrics·N GuffonP Guibaud
Jan 26, 1999·JAMA : the Journal of the American Medical Association·P J MeikleW F Carey
Feb 15, 2001·The New England Journal of Medicine·E D KakkisE F Neufeld
Mar 30, 2001·International Journal of Pediatric Otorhinolaryngology·S E LeightonR Lane
Oct 1, 2003·The American Journal of Cardiology·Elizabeth A BraunlinWilliam Krivit
Nov 20, 2003·JAMA : the Journal of the American Medical Association·Susan E WaisbrenHarvey L Levy
May 11, 2004·The Journal of Gene Medicine·N Matthew EllinwoodMark E Haskins
Jun 23, 2004·Seminars in Cell & Developmental Biology·Steven U Walkley
Jul 21, 2004·The Journal of Experimental Medicine·Gesine KöglerPeter Wernet
Nov 25, 2004·Hematology·Nelson J ChaoKenneth I Weinberg
Jan 27, 2005·Journal of Inherited Metabolic Disease·C J HendrikszJ E Wraith
Feb 3, 2005·British Journal of Haematology·Ashok Vellodi
Feb 17, 2005·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Satkiran S GrewalCharles Peters

❮ Previous
Next ❯

Citations

Jan 12, 2012·European Journal of Pediatrics·Kristin D'AcoPaige Kaplan
May 22, 2010·Journal of Inherited Metabolic Disease·Laura Bannach JardimCristina B Oliveira Netto
Nov 10, 2009·Bone Marrow Transplantation·P J OrchardJ Tolar
May 10, 2011·Bone Marrow Transplantation·M MynarekK-W Sykora
Dec 12, 2012·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Beth K PotterUNKNOWN Canadian Inherited Metabolic Diseases Research Network
Jan 28, 2012·Stem Cells and Development·Francesca GattoMarta Serafini
Jan 11, 2012·Rheumatology·Thomas J A LehmanJoan Keutzer
Jan 11, 2012·Rheumatology·Vassili Valayannopoulos, Frits A Wijburg
Jan 7, 2010·Pediatric Critical Care Medicine : a Journal of the Society of Critical Care Medicine and the World Federation of Pediatric Intensive and Critical Care Societies·Susan H MorrisJames D Fortenberry
Apr 25, 2012·Orphanet Journal of Rare Diseases·Minke H de RuFrits A Wijburg
Jun 4, 2011·Genetics and Molecular Biology·Roberto GiuglianiAna Maria Martins
Feb 11, 2014·Cardiovascular Pathology : the Official Journal of the Society for Cardiovascular Pathology·Elizabeth BraunlinJ Carlos Manivel
Mar 26, 2013·Clinical Biochemistry·Derbis CamposDaniurys de la Peña
May 14, 2014·Journal of Inherited Metabolic Disease·Carla E M Hollak, Frits A Wijburg
May 5, 2011·Journal of Inherited Metabolic Disease·María Verónica Muñoz-RojasAna Maria Martins
Feb 11, 2014·Journal of Inherited Metabolic Disease·Bas W M van BalkomMarianne C Verhaar
Apr 5, 2014·Journal of Inherited Metabolic Disease·Sandra D K KingmaNaomi van Vlies
Jul 17, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·Christian HindererJames M Wilson
Sep 27, 2012·Expert Opinion on Pharmacotherapy·Carin M van GelderArnold J J Reuser
Jun 8, 2012·Critical Reviews in Biochemistry and Molecular Biology·Kim M KeelingDavid M Bedwell
Dec 30, 2014·Molecular Genetics and Metabolism·Shunji TomatsuTadao Orii
Mar 24, 2010·Pediatric Clinics of North America·Jaap J BoelensCharles Peters
Jan 30, 2010·Seminars in Hematology·Vinod K Prasad, Joanne Kurtzberg
Aug 8, 2014·Pediatric Annals·Julian Raiman, Kristin D'Aco
Sep 1, 2015·Journal of Genetic Counseling·Emily C Lisi, Shawn E McCandless
Aug 12, 2014·Annals of Neurology·Michele D PoeMaria L Escolar
Jul 31, 2014·British Journal of Haematology·Jaap Jan BoelensRobert F Wynn
Feb 4, 2014·Pediatric Transplantation·Giovanna FerraraPaolo Tamaro
Mar 31, 2015·Blood·Michael A Pulsipher
Jun 23, 2015·Molecular Genetics and Metabolism·Elsa G ShapiroChester B Whitley
Jan 15, 2014·Molecular Genetics and Metabolism·Gwen GunnKim M Keeling
May 30, 2015·Molecular Therapy : the Journal of the American Society of Gene Therapy·Christian HindererJames M Wilson

❮ Previous
Next ❯

Related Concepts

Related Feeds

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.

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 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 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.