National U.S. Patient and Transplant Data for Krabbe Disease.
National U.S. Patient and Transplant Data for Krabbe Disease.
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DOI：
10.3389/fped.2021.764626
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发表时间：
2021
影响因子：
2.6
通讯作者：
Bonkowsky JL
中科院分区：
文献类型：
--
作者：
Ghabash G;Wilkes J;Bonkowsky JL
克拉伯病（Krabbe disease， KD）是一种由半乳糖神经酰胺酶基因突变引起的脑白质营养不良。症状前造血干细胞移植（HSCT）与改善预后相关，但大多数数据来自单中心研究。我们的目的是描述KD的国家HSCT模式，包括HSCT的使用和结果是否存在差异。我们使用美国儿童医院协会的儿科健康信息系统数据库，对2015年11月1日至2019年12月31日≤18岁的KD患者进行了回顾性研究。我们通过比较年龄、性别、种族/民族、农村/城市位置和家庭收入中位数，评估了HSCT、重症监护病房天数和死亡率的结果。我们确定了91例KD患者。32%的患者接受HSCT后死亡率降低，31%对68%未接受HSCT的患者（p < 0.003）。趋势包括接受HSCT的男性多于女性（39%对23%）；与黑人或西班牙裔患者相比，更多的亚裔和白人患者接受了HSCT（分别为75%、33%、25%和17%）；来自最低收入四分之一家庭（< 25,000美元）的患者比高收入四分之一家庭的患者有更多的HSCT（44%对33%，30%和0%）。总的来说，接受造血干细胞移植与死亡率降低有关。我们注意到接受造血干细胞移植的患者群体的趋势。我们的研究结果表明，接受HSCT的差异可能会影响KD患者的预后。
Krabbe disease (KD) is a leukodystrophy caused by mutations in the galactosylceramidase gene. Presymptomatic hematopoietic stem cell transplantation (HSCT) is associated with improved outcomes, but most data are from single-center studies. Our objective was to characterize national patterns of HSCT for KD including whether there were disparities in HSCT utilization and outcomes. We conducted a retrospective study of KD patients ≤ age 18 years from November 1, 2015, through December 31, 2019, using the U.S. Children's Hospital Association's Pediatric Health Information System database. We evaluated outcomes for HSCT, intensive care unit days, and mortality, comparing age, sex, race/ethnicity, rural/urban location, and median household income. We identified 91 KD patients. HSCT, performed in 32% of patients, was associated with reduced mortality, 31 vs. 68% without HSCT (p < 0.003). Trends included the fact that more males than females had HSCT (39 vs. 23%); more Asian and White patients had HSCT compared to Black or Hispanic patients (75, 33, 25, and 17%, respectively); and patients from households with the lowest-income quartile (< $25,000) had more HSCT compared to higher-income quartiles (44 vs. 33, 30, and 0%). Overall, receiving HSCT was associated with reduced mortality. We noted trends in patient groups who received HSCT. Our findings suggest that disparities in receiving HSCT could affect outcomes for KD patients.
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