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
  • [21] Inherited metabolic diseases: benefits of metabolomics
    Ricquier, D
    M S-MEDECINE SCIENCES, 2005, 21 (05): : 512 - 516
  • [22] HEPATIC MORPHOLOGY IN THE INHERITED METABOLIC DISEASES
    ISHAK, KG
    SEMINARS IN LIVER DISEASE, 1986, 6 (03) : 246 - 258
  • [23] When to suspect inherited metabolic diseases
    Shribman, Samuel
    Lachmann, Robin H.
    PRACTICAL NEUROLOGY, 2024, 24 (05) : 354 - 356
  • [24] Outcomes After Living Donor Liver Transplantation in Pediatric Patients with Inherited Metabolic Diseases
    Sanada, Yukihiro
    Sakuma, Yasunaru
    Onishi, Yasuharu
    Okada, Noriki
    Yamada, Naoya
    Hirata, Yuta
    Miyahara, Go
    Katano, Takumi
    Horiuchi, Toshio
    Omameuda, Takahiko
    Lefor, Alan Kawarai
    Sata, Naohiro
    ANNALS OF TRANSPLANTATION, 2021, 26
  • [25] LIVER TRANSPLANTATION FOR THE TREATMENT OF CHILDREN WITH INHERITED METABOLIC LIVER DISEASES: SINGLE CENTER EXPERIENCE
    Pei, Jiahao
    Wang, Zhengxin
    Shen, Conghuan
    Zhang, Quanbao
    Li, Jianhua
    TRANSPLANTATION, 2020, 104 (09) : S549 - S549
  • [26] Transplantation for Inherited Metabolic Disorders of the Liver
    Schilsky, M. L.
    TRANSPLANTATION PROCEEDINGS, 2013, 45 (02) : 455 - 462
  • [27] Kidney Transplantation in Inherited Kidney Diseases
    Caliskan, Y.
    Yazici, H.
    Ouseph, R.
    Ozluk, Y.
    Randall, H.
    Demir, E.
    Oguz, F. Savran
    Turkmen, A.
    Sever, M. S.
    Lentine, K.
    AMERICAN JOURNAL OF TRANSPLANTATION, 2020, 20 : 688 - 688
  • [28] Emerging Trends in Foodborne Diseases
    Braden, Christopher R.
    Tauxe, Robert V.
    INFECTIOUS DISEASE CLINICS OF NORTH AMERICA, 2013, 27 (03) : 517 - +
  • [29] Hematopoietic cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions
    Rovelli, AM
    Steward, CG
    BONE MARROW TRANSPLANTATION, 2005, 35 (Suppl 1) : S23 - S26
  • [30] Predictors of gastrostomy placement in children with inherited metabolic diseases treated by umbilical cord blood transplantation
    Gulack, Brian C.
    Schweitzer, Michelle
    Englum, Brian R.
    Kuchibhatla, Maragatha
    Prasad, Vinod K.
    Adibe, Obinna O.
    JOURNAL OF PEDIATRIC SURGERY, 2015, 50 (07) : 1109 - 1111
12345