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非清髓性造血干细胞移植对老年青少年和成年镰状细胞病患者的纵向神经认知影响:描述及与同胞供者的比较。

Longitudinal neurocognitive effects of nonmyeloablative hematopoietic stem cell transplant among older adolescents and adults with sickle cell disease: A description and comparison with sibling donors.

机构信息

Department of Psychology, American University, Washington, United States.

Pediatric Oncology Branch, National Cancer Institute, National Institutes of Health, Bethesda, United States.

出版信息

Neuropsychol Rehabil. 2024 Aug;34(7):899-918. doi: 10.1080/09602011.2023.2238948. Epub 2023 Aug 4.

DOI:10.1080/09602011.2023.2238948
PMID:37540620
Abstract

Sickle cell disease (SCD) is associated with increased risk of neurocognitive deficits. However, whether functioning changes following nonmyeloablative hematopoietic stem cell transplant (HSCT) remains unclear. This study aimed to examine changes in neuropsychological functioning pre- to post-transplant among patients with SCD and compare patients and siblings. Adults with SCD ( = 47; M = 31.8 ± 8.9) and their sibling stem cell donors ( = 22; M = 30.5± 9.2) enrolled on a nonmyeloablative HCST protocol completed cognitive and patient-reported outcome assessments at baseline and 12 months post-transplant. Path analyses were used to assess associations between pre-transplant variables and sibling/patient group status and post-transplant function. Mean patient cognitive scores were average at both timepoints. Patient processing speed and somatic complaints improved from baseline to follow-up. Baseline performance predicted follow-up performance across cognitive variables; patient/sibling status predicted follow-up performance on some processing speed measures. Results suggest that patients with SCD demonstrate slower processing speed than siblings. Processing speed increased pre- to post-HSCT among patients and siblings, and on some measures patients demonstrated greater improvement. Thus, HSCT may improve processing speed in patients, although further confirmation is needed. Findings provide promising evidence that neurocognitive functioning remains stable without detrimental effects from pre- to 12-months post nonmyeloablative HSCT in individuals with SCD.

摘要

镰状细胞病(SCD)与神经认知功能缺陷的风险增加有关。然而,非清髓性造血干细胞移植(HSCT)后功能是否发生变化尚不清楚。本研究旨在检查 SCD 患者移植前后神经心理功能的变化,并比较患者和兄弟姐妹。接受非清髓性 HSCT 方案的 SCD 成人患者(n=47;M=31.8±8.9)及其兄弟姐妹干细胞供者(n=22;M=30.5±9.2)在基线和移植后 12 个月完成认知和患者报告的结果评估。路径分析用于评估移植前变量与兄弟姐妹/患者组状态和移植后功能之间的关联。在两个时间点,患者的平均认知评分都处于中等水平。患者的处理速度和躯体抱怨从基线到随访都有所改善。基线表现预测了认知变量的随访表现;患者/兄弟姐妹的状态预测了某些处理速度测量的随访表现。结果表明,SCD 患者的处理速度比兄弟姐妹慢。患者和兄弟姐妹在 HSCT 前到后处理速度都有所提高,而在某些方面,患者的改善更大。因此,HSCT 可能会改善患者的处理速度,尽管需要进一步证实。研究结果提供了有希望的证据,表明在 SCD 患者中,非清髓性 HSCT 前后 12 个月内,神经认知功能保持稳定,没有不良影响。

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