Quality of life among boys with adrenoleukodystrophy following hematopoietic stem cell transplant
Quality of life among boys with adrenoleukodystrophy following hematopoietic stem cell transplant
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DOI：
10.1080/09297049.2017.1380176
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发表时间：
2018-01-01
影响因子：
2.2
通讯作者：
Orchard, Paul J.
中科院分区：
文献类型：
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作者：
Beckmann, Nicole B.;Miller, Weston P.;Orchard, Paul J.
造血干细胞移植(HSCT)是唯一能够阻止X连锁脑肾上腺脑白质营养不良(CALD)进展的公认治疗方法。虽然存活和神经结局已经被描述，但关于CALD移植患者的生活质量(QOL)的信息很少。这项分析是对16名在单一机构接受HSCT的CALD男性患者的生活质量的横断面研究。每个儿童或家长代表完成神经生活质量量表和PROMIS儿科档案工具中代表生理、心理和社会健康领域的子量表。描述性统计总结了人口学特征和QOL子量表T分数，Spearman Rho相关性确定了变量之间的关系，Mann-Whitney检验检查了HSCT前LOES评分为10的组之间的差异。受访者接受HSCT时的中位年龄为8岁(5-14岁)，HSCT后的中位数为5年(0.5-11岁)。选择的QOL子量表的得分与健康同龄人相似，尽管HSCT前LOES得分>；=10的人行动不便、上肢功能、同伴交往和焦虑得分较高。尽管HSCT有阻止CALD进展的能力，但HSCT前LOES评分<gt；=10的患者存在生活质量差的风险。有必要进行纵向监测，以进一步了解影响HSCT后CALD男孩生活质量的因素，以及如何改善这一影响因素。
Hematopoietic stem cell transplant (HSCT) is the only accepted treatment capable of halting the progression of X-linked cerebral adrenoleukodystrophy (CALD). While survival and neurological outcomes have been described, there is little information regarding the quality of life (QoL) of transplanted patients with CALD. This analysis is a cross-sectional study of QoL in 16 males diagnosed with CALD who underwent HSCT at a single institution. Each child or parent proxy completed subscales from the Neuro-QoL and the PROMIS Pediatric Profile Instrument representing physical, mental, and social health domains. Descriptive statistics summarized the demographic characteristics and QoL subscale T-scores, Spearman Rho correlations identified the relationships among the variables, and Mann-Whitney tests examined group differences between those with pre-HSCT Loes scores = 10. The median age of respondents at the time of transplant was 8 years at HSCT (5-14) with a median of 5 years since HSCT (0.5-11). Scores from the selected QoL subscales were similar to healthy peers, though those with pre-HSCT Loes scores >= 10 had lower mobility, upper extremity function, peer interaction, and higher scores for anxiety. Although HSCT has the capability of halting progression of CALD, those with pre-HSCT Loes scores >= 10 after HSCT are at-risk for poor QoL. Longitudinal monitoring is necessary to further appreciate the factors affecting QoL among boys with CALD after HSCT, and how this may be improved.