Update of hematopoietic cell transplantation for sickle cell disease

被引：27

作者：

Walters, Mark C. ^{[1
]}

机构：

[1] UCSF Benioff Childrens Hosp, Blood & Marrow Transplant Program, Div Hematol Oncol BMT, Oakland, CA 94609 USA

来源：

CURRENT OPINION IN HEMATOLOGY | 2015年 / 22卷 / 03期

关键词：

alternate donor; hematopoietic cell transplantation; sickle cell disease; BONE-MARROW-TRANSPLANTATION; CORD BLOOD TRANSPLANTATION; PEDIATRIC-PATIENTS; REDUCED-TOXICITY; SIBLING DONORS; CHILDREN; ANEMIA; THALASSEMIA; FLUDARABINE; PULMONARY;

D O I：

10.1097/MOH.0000000000000136

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Purpose of review Hematopoietic cell transplantation (HCT) is a curative therapy for sickle cell disease (SCD) that is utilized very rarely because of limited allogeneic donor availability, limited healthcare resources needed to expand the treatment to regions in the world where most affected individuals reside, and by a view among SCD experts that HCT lacks the evidential rigor with short and long-term toxicity profiles that together might support its broader application. Recent findings In this update, recent advances focused on donor selection, reduced toxicity preparation for HCT, and treatment of young adults will be presented. The current status of conventional bone marrow transplantation with a human leukocyte antigen-identical sibling donor is summarized. Summary HCT for SCD is curative in almost all children who have a human leukocyte antigen-matched sibling donor. The future of this therapy will hinge on expanding the number of individuals who might be treated.

引用

页码：227 / 233

页数：7

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