How I treat sickle cell disease with hematopoietic cell transplantation

被引:15
作者
Stenger, Elizabeth O. [1 ]
Shenoy, Shalini [2 ]
Krishnamurti, Lakshmanan [3 ]
机构
[1] UPMC Childrens Hosp Pittsburgh, Div Blood & Marrow Transplantat & Cellular Therap, Pittsburgh, PA USA
[2] Childrens Hosp St Louis, Div Hematol Oncol, St Louis, MO USA
[3] Childrens Healthcare Atlanta, Aflac Canc & Blood Disorders Ctr, Atlanta, GA USA
基金
美国国家卫生研究院;
关键词
BONE-MARROW-TRANSPLANTATION; CORD BLOOD TRANSPLANTATION; REVERSIBLE ENCEPHALOPATHY SYNDROME; MATCHED RELATED DONORS; QUALITY-OF-LIFE; REDUCED-INTENSITY; PERICARDIAL-EFFUSION; YOUNG-CHILDREN; ABO MISMATCH; GENE-THERAPY;
D O I
10.1182/blood.2019000821
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Sickle cell disease (SCD) leads to significant morbidity and early mortality, and hematopoietic cell transplantation (HCT) is the only widely available cure, with impacts seen on SCD-related organ dysfunction. Outcomes are excellent following matched-related donor (MRD) HCT, leading to significantly expanded application of this treatment over the past decade. The majority of SCD patients lack an MRD, but outcomes following alternative donor HCT continue to improve on clinical trials. Within this framework, we aim to provide our perspective on how to apply research findings to clinical practice, for an individual patient. We also emphasize that the preparation of SCD recipients for HCT and supporting them through HCT have special nuances that require awareness and close attention. Through the use of clinical vignettes, we provide our perpsective on the complex decision-making process in HCT for SCD as well as recommendations for the evaluation and support of these patients through HCT.
引用
收藏
页码:2249 / 2260
页数:12
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