造血干细胞移植治疗严重联合免疫缺陷病(SCID)

2014/8/8 15:17:11 本站原创 佚名 【字体:

主要内容:一项多中心的研究显示, 5年生存率为74

 

Hematopoietic-Cell Transplantation Provides Excellent Outcome in SCID

 

Severe combined immunodeficiency (SCID) is a heterogeneous primary condition that is often lethal in the absence of aggressive immune replacement therapy, curative hematopoietic-cell transplantation, or both. A multicenter collaboration reports on 240 children with SCID who were diagnosed at a median age of 139 days and underwent transplantation at a median of 180 days. The children had an overall 5-year survival of 74%, and 45 required a second transplant (56% survival).

 

The survival rate in infants transplanted at 3.5 months or younger was 94%, similar to that among older infants with no history of infection (90%) and among those with infections that were cured before transplantation (82%). Matched donor siblings offered the highest survival (97%). Older age and active infection at the time of transplantation were strongly associated with lower graft survival. Patients infected at the time of transplantation had better survival with reduced-intensity pretransplant conditioning. Among survivors, reduced conditioning was associated with improved CD3+ T-cell reconstitution, freedom from immunoglobulin infusions, and IgA recovery. The genetic subtype of SCID did not affect survival.

 

 

With newborn screening, SCID can now be diagnosed before the first infection. These results clearly show that early transplantation prior to any infection has a very high success rate. However, convincing families to approve transplantation for a perfectly normal-appearing newborn with SCID is a challenging, but important task.

 

 

 

Citation(s):

 

Pai S-Y et al. Transplantation outcomes for severe combined immunodeficiency, 2000–2009. N Engl J Med 2014 Jul 31; 371:434. (http://dx.doi.org/10.1056/NEJMoa1401177)

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