Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy,Journal of Inherited Metabolic Disease

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Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy
Journal of Inherited Metabolic Disease ( IF 4.2 ) Pub Date : 2021-09-09 , DOI: 10.1002/jimd.12435
Ashish O Gupta ₁ , David R Nascene ₂ , Ryan Shanley ₃ , Daniel L Kenney-Jung ₄ , Julie B Eisengart ₁ , Troy C Lund ₁ , Paul J Orchard ₁ , Elizabeth I Pierpont ₁

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In the most common variant of childhood cerebral adrenoleukodystrophy (cALD), demyelinating brain lesions are distributed predominately in parieto-occipital white matter. Less frequently, lesions first develop in frontal white matter. This matched cohort study examined whether outcomes after standard treatment with hematopoietic cell transplantation (HCT) differ in patients with early stage frontal lesions as compared to parieto-occipital lesions. Retrospective chart review identified seven pediatric patients with frontal cALD lesions and MRI severity score < 10 who underwent a single HCT at our center between 1990 and 2019. Concurrent MRI, neurocognitive and psychiatric outcomes at last comprehensive follow-up (mean 1.2 years; range 0.5-2.1 years) were compared with a group of seven boys with the parieto-occipital variant matched on pre-HCT MRI severity score. Both groups showed similar rates of transplant complications and radiographic disease advancement. Neurocognitive outcomes were broadly similar, with more frequent working memory deficits among individuals with frontal lesions. Psychiatric problems (hyperactivity, aggression, and atypical behavior) were considerably more common and severe among patients with frontal lesions. Aligned with the critical role of the frontal lobes in emotional and behavioral regulation, functional disruption of self-regulation skills is widely observed among patients with frontal lesions. Comprehensive care for cALD should address needs for psychiatric care and management.

中文翻译：

儿童脑肾上腺脑白质营养不良的额叶脱髓鞘与后脱髓鞘的不同结果

在儿童期脑肾上腺脑白质营养不良 (cALD) 的最常见变型中，脱髓鞘性脑损伤主要分布在顶枕白质中。不太常见的是，病变首先在额叶白质中发展。这项匹配的队列研究检查了早期额叶病变患者与顶枕叶病变患者标准造血细胞移植 (HCT) 治疗后的结果是否不同。回顾性图表审查确定了 1990 年至 2019 年间在我们中心接受了单次 HCT 的 7 名额叶 cALD 病变且 MRI 严重程度评分 < 10 的儿科患者。最后一次全面随访时的并发 MRI、神经认知和精神病学结果（平均 1.2 年；范围 0.5 -2。1 年）与一组 7 名男孩进行比较，这些男孩的顶枕变异在 HCT 前 MRI 严重程度评分上匹配。两组的移植并发症和影像学疾病进展率相似。神经认知结果大致相似，额叶病变个体的工作记忆缺陷更为常见。精神问题（多动、攻击性和非典型行为）在额叶病变患者中更为常见和严重。与额叶在情绪和行为调节中的关键作用相一致，在额叶病变患者中广泛观察到自我调节技能的功能破坏。cALD 的综合护理应满足精神科护理和管理的需求。两组的移植并发症和影像学疾病进展率相似。神经认知结果大致相似，额叶病变个体的工作记忆缺陷更为常见。精神问题（多动、攻击性和非典型行为）在额叶病变患者中更为常见和严重。与额叶在情绪和行为调节中的关键作用相一致，在额叶病变患者中广泛观察到自我调节技能的功能破坏。cALD 的综合护理应满足精神科护理和管理的需求。两组的移植并发症和影像学疾病进展率相似。神经认知结果大致相似，额叶病变个体的工作记忆缺陷更为常见。精神问题（多动、攻击性和非典型行为）在额叶病变患者中更为常见和严重。与额叶在情绪和行为调节中的关键作用相一致，在额叶病变患者中广泛观察到自我调节技能的功能破坏。cALD 的综合护理应满足精神科护理和管理的需求。精神问题（多动、攻击性和非典型行为）在额叶病变患者中更为常见和严重。与额叶在情绪和行为调节中的关键作用相一致，在额叶病变患者中广泛观察到自我调节技能的功能破坏。cALD 的综合护理应满足精神科护理和管理的需求。精神问题（多动、攻击性和非典型行为）在额叶病变患者中更为常见和严重。与额叶在情绪和行为调节中的关键作用相一致，在额叶病变患者中广泛观察到自我调节技能的功能破坏。cALD 的综合护理应满足精神科护理和管理的需求。

更新日期：2021-11-09

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