Krivit W, Peters C, Shapiro E G
University of Minnesota Hospitals and Clinics, Minniapolis 55455, USA.
Curr Opin Neurol. 1999 Apr;12(2):167-76. doi: 10.1097/00019052-199904000-00007.
Over 400 patients with lysosomal and peroxisomal storage diseases have received hematopoietic stem cell transplantation from normal donors. Without treatment, all of these diseases have an inexorable fate leading to central nervous system deterioration and early death. On the other hand, all of the engrafted hosts have had a remarkable positive clinical improvement in response to normalization of previously deficient enzymatic activity. Survival data for those engrafted indicates continued life-span as long as two decades beyond transplantation. The particular diseases treated in this way are included in this article. The specific indications and methods for transplantation are also included in this article.
400 多名患有溶酶体和过氧化物酶体贮积病的患者接受了来自正常供体的造血干细胞移植。如果不进行治疗,所有这些疾病都会不可避免地导致中枢神经系统恶化和过早死亡。另一方面,所有接受移植的宿主在先前缺乏的酶活性恢复正常后,临床症状都有了显著的积极改善。接受移植者的生存数据表明,移植后寿命可延长长达二十年。本文涵盖了以这种方式治疗的特定疾病。本文还包括移植的具体适应症和方法。