Woodard P, Helton K, McDaniel H, Khan R B, Thompson S, Hale G, Benaim E, Kasow K, Leung W, Horwitz E, Srivastava D K, Tong X, Yusuf U, Cunningham J M, Handgretinger R
Division of Stem Cell Transplantation, Department of Hematology-Oncology, St Jude Children's Research Hospital, Memphis, TN 38105-2794, USA.
Bone Marrow Transplant. 2004 Jun;33(11):1151-7. doi: 10.1038/sj.bmt.1704480.
Encephalopathy is a poorly characterized complication of hematopoietic stem cell transplantation (HSCT). No comprehensive report of encephalopathy exists for children, and the literature contains only a few for adults. We analyzed a large cohort of 405 pediatric patients who underwent allogeneic HSCT during a 10-year period and identified 26 patients (6.4%) who experienced encephalopathy. Identifiable causes of encephalopathy included infection (n=5), single or multiorgan failure (n=4), medication-related complications (n=3), nonconvulsive seizures (n=4), acute disseminated encephalomyelitis (n=2), thrombotic thrombocytopenic purpura (n=2), and stroke (n=1). We were unable to identify the etiology of encephalopathy in five (19%) patients. The prognosis for pediatric patients with encephalopathy was poor: only four (15%) experienced complete neurologic recovery, and 10 (38%) patients experienced partial recovery with residual neurologic deficits. Nine (35%) patients with complete or partial recovery survive long term. A total of 17 patients died; one died of progressive encephalopathy, and 16 died of either relapse of primary disease or toxicity. MRI, CSF analysis including molecular testing for infectious pathogens, and brain biopsy were helpful in obtaining a diagnosis in most of our patients. However, a standardized approach to accurate and timely diagnosis and treatment is needed to improve outcome in these patients.
脑病是造血干细胞移植（HSCT）的一种特征不明的并发症。目前尚无关于儿童脑病的综合报告，而针对成人的文献也仅有少数几篇。我们分析了405例在10年期间接受异基因HSCT的儿科患者的大样本队列，确定了26例（6.4%）发生脑病的患者。脑病的可识别病因包括感染（n = 5）、单器官或多器官功能衰竭（n = 4）、药物相关并发症（n = 3）、非惊厥性癫痫发作（n = 4）、急性播散性脑脊髓炎（n = 2）、血栓性血小板减少性紫癜（n = 2）和中风（n = 1）。我们无法确定5例（19%）患者脑病的病因。患有脑病的儿科患者预后较差：只有4例（15%）实现了完全神经功能恢复，10例（38%）患者部分恢复但仍有神经功能缺损。9例（35%）完全或部分恢复的患者长期存活。共有17例患者死亡；1例死于进行性脑病，16例死于原发性疾病复发或毒性反应。MRI、包括对感染病原体进行分子检测的脑脊液分析以及脑活检对我们大多数患者的诊断有帮助。然而，需要一种标准化的方法来进行准确及时的诊断和治疗，以改善这些患者的预后。
