Emerging trends in transplantation of inherited metabolic diseases

被引：54
作者：
Prasad, V. K. [1 ]
Kurtzberg, J. [1 ]
机构：
[1] Duke Univ, Med Ctr, Pediat Blood & Marrow Transplant Program, Durham, NC 27710 USA
来源：
BONE MARROW TRANSPLANTATION | 2008年 / 41卷 / 02期
关键词：
inherited metabolic diseases; lysosomal and peroxisomal storage disease; hematopoietic stem cell transplantation; umbilical cord blood transplantation;
D O I：
10.1038/sj.bmt.1705970
中图分类号：
Q6 [生物物理学];
学科分类号：
071011 ;
摘要：
Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell regeneration or repair. Unrelated cord blood is an exciting newer graft source for treatment of patients with these fatal disorders, providing increased access to donors and significant clinical efficacy, particularly when transplantation is performed in early stages. Pre-transplant performance status is highly predictive of overall survival.
引用
收藏
页码：99 / 108
页数：10
相关论文
共 50 条
  • [31] Allogeneic bone marrow stem cell transplantation following CD34+immunomagnetic enrichment in patients with inherited metabolic storage diseases
    Gaipa, G
    Dassi, M
    Perseghin, P
    Venturi, N
    Corti, P
    Bonanomi, S
    Balduzzi, A
    Longoni, D
    Uderzo, C
    Biondi, A
    Masera, G
    Parini, R
    Bertagnolio, B
    Uziel, G
    Peters, C
    Rovelli, A
    [J]. BONE MARROW TRANSPLANTATION, 2003, 31 (10) : 857 - 860
  • [32] Allogeneic bone marrow stem cell transplantation following CD34+ immunomagnetic enrichment in patients with inherited metabolic storage diseases
    G Gaipa
    M Dassi
    P Perseghin
    N Venturi
    P Corti
    S Bonanomi
    A Balduzzi
    D Longoni
    C Uderzo
    A Biondi
    G Masera
    R Parini
    B Bertagnolio
    G Uziel
    C Peters
    A Rovelli
    [J]. Bone Marrow Transplantation, 2003, 31 : 857 - 860
  • [33] Genetic testing experiences and genetics knowledge among families with inherited metabolic diseases
    Kenneson, Aileen
    Youngborg, Lauren
    Singh, Rani H.
    [J]. MOLECULAR GENETICS AND METABOLISM REPORTS, 2020, 24
  • [34] U-IMD: the first Unified European registry for inherited metabolic diseases
    Opladen, Thomas
    Gleich, Florian
    Kozich, Viktor
    Scarpa, Maurizio
    Martinelli, Diego
    Schaefer, Franz
    Jeltsch, Kathrin
    Julia-Palacios, Natalia
    Garcia-Cazorla, Angels
    Dionisi-Vici, Carlo
    Koelker, Stefan
    [J]. ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)
  • [35] U-IMD: the first Unified European registry for inherited metabolic diseases
    Thomas Opladen
    Florian Gleich
    Viktor Kozich
    Maurizio Scarpa
    Diego Martinelli
    Franz Schaefer
    Kathrin Jeltsch
    Natalia Juliá-Palacios
    Ángels García-Cazorla
    Carlo Dionisi-Vici
    Stefan Kölker
    [J]. Orphanet Journal of Rare Diseases, 16
  • [36] Guiametabolica.org: empowerment through internet tools in inherited metabolic diseases
    Armayones, Manuel
    Vilaseca, M. Antonia
    Cutillas, Julia
    Fabrega, Jordi
    Fernandez, Jorge Juan
    Garcia, Mei
    Egea, Natalia
    Pousada, Modesta
    Gomez-Zuniga, Beni
    Perez-Payarols, Jaume
    Artuch, Rafael
    Palau, Francesc
    Serrano, Mercedes
    [J]. ORPHANET JOURNAL OF RARE DISEASES, 2012, 7
  • [37] Guiametabolica.org: empowerment through internet tools in inherited metabolic diseases
    Manuel Armayones
    M Antònia Vilaseca
    Júlia Cutillas
    Jordi Fàbrega
    Jorge Juan Fernández
    Mei García
    Natàlia Egea
    Modesta Pousada
    Beni Gómez-Zuñiga
    Jaume Pérez-Payarols
    Rafael Artuch
    Francesc Palau
    Mercedes Serrano
    [J]. Orphanet Journal of Rare Diseases, 7
  • [38] Symptoms and Problems in Children with Inherited Metabolic Diseases and Factors Affecting the Caregiver Burden of Mothers
    Arpaci, Tuba
    Altay, Naime
    Toruner, Ebru Kilicarslan
    Gunduz, Mehmet
    [J]. COMPREHENSIVE CHILD AND ADOLESCENT NURSING-BUILDNG EVIDENCE FOR PRACTICE, 2020, 43 (04): : 363 - 377
  • [39] Clinical and biochemical footprints of inherited metabolic diseases. XIII. Respiratory manifestations
    Rossi, Alessandro
    Basilicata, Simona
    Borrelli, Melissa
    Ferreira, Carlos R.
    Blau, Nenad
    Santamaria, Francesca
    [J]. MOLECULAR GENETICS AND METABOLISM, 2023, 140 (03)
  • [40] Diagnostics of Inherited Metabolic Diseases in Newborns with the Hyperammonemia Syndrome at the Onset of Disease (Pilot Study)
    Kolchina, A. N.
    Yatsyshina, E. E.
    Malysheva, L., V
    Ledentsova, E. E.
    Lidyaeva, E. E.
    Khaletskaya, O., V
    [J]. SOVREMENNYE TEHNOLOGII V MEDICINE, 2021, 13 (01) : 59 - 64
12345