Division of Critical Care Medicine, Nicklaus Children's Hospital, Miami, FL, USA.
Herbert Wertheim College of Medicine, Florida International University, Miami, FL, USA.
Pediatr Hematol Oncol. 2024 Oct;41(7):489-503. doi: 10.1080/08880018.2024.2378282. Epub 2024 Jul 15.
In patients with sickle cell disease (SCD) and beta-thalassemia major (TM), allogeneic hematopoietic stem cell transplantation (HSCT) was considered the only curative treatment option with a good survival rate. However, with the recent approval of gene therapies, more information is needed to understand the benefits and risks of these interventions. We performed a retrospective analysis of the Kids Inpatient Database to describe demographic features, short-term complications, and hospital charges of patients with SCD and TM treated with HSCT during 2006-2019 in the United States. The database was filtered using the , 9 and 10 edition codes to identify children under 20 years of age with SCD or TM who underwent HSCT. A total of 513 children with SCD or TM who received HSCT were analyzed. The prevalence of HSCT per 1000,000 U.S. population increased from 0.31 in 2006 to 1.99 in 2019 ( < 0.001). The median age of children with SCD who underwent HSCT was 10 (6-15) years, and that for TM was 6 (3-11.5) years ( < 0.001). The combined mortality rate was 4% (2.4%-6.6%) but higher in the TM group. The length-of-stay and total charges were higher in the TM population ( < 0.01). This study provides national data on HSCT among hospitalized children with SCD and TM in the United States, demonstrating an increasing use of HSCT between 2006 and 2019. Although hospital mortality of HSCT in these conditions is low, it still represents a challenge, especially in TM patients.
在镰状细胞病（SCD）和重型β地中海贫血（TM）患者中，异体造血干细胞移植（HSCT）被认为是唯一具有良好生存率的治愈性治疗选择。然而，随着基因治疗的最近批准，需要更多信息来了解这些干预措施的益处和风险。我们对美国儿童住院患者数据库进行了回顾性分析，以描述 2006 年至 2019 年期间接受 HSCT 治疗的 SCD 和 TM 患者的人口统计学特征、短期并发症和住院费用。使用 、 9 和 10 版代码对数据库进行过滤，以确定年龄在 20 岁以下的患有 SCD 或 TM 并接受 HSCT 的儿童。共分析了 513 例接受 HSCT 的 SCD 或 TM 儿童。每 100 万美国人口中的 HSCT 患病率从 2006 年的 0.31 增加到 2019 年的 1.99（<0.001）。接受 HSCT 的 SCD 患儿的中位年龄为 10 岁（6-15 岁），TM 患儿为 6 岁（3-11.5 岁）（<0.001）。合并死亡率为 4%（2.4%-6.6%），但 TM 组更高。TM 人群的住院时间和总费用更高（<0.01）。本研究提供了美国住院 SCD 和 TM 儿童 HSCT 的全国数据，表明 2006 年至 2019 年间 HSCT 的使用有所增加。尽管这些情况下 HSCT 的医院死亡率较低，但它仍然是一个挑战，尤其是在 TM 患者中。
