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儿童神经母细胞瘤患者自体外周血造血干细胞移植后移植相关血栓性微血管病的早期临床指标。

Early clinical indicators of transplant-associated thrombotic microangiopathy in pediatric neuroblastoma patients undergoing auto-SCT.

机构信息

Division of Nephrology and Hypertension, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH 45229, USA.

出版信息

Bone Marrow Transplant. 2011 May;46(5):682-9. doi: 10.1038/bmt.2010.182. Epub 2010 Aug 9.

Abstract

Patients undergoing auto-SCT for neuroblastoma present a unique population to study transplant-associated thrombotic microangiopathy (TA-TMA), due to standardized chemotherapy and later exposure to radiation and cis-retinoic acid (cis-RA). We retrospectively analyzed 20 patients after auto-SCT to evaluate early clinical indicators of TA-TMA. A total of 6 patients developing TA-TMA (30% prevalence) were compared with 14 controls. Four of six patients were diagnosed with TA-TMA by 25 days after auto-SCT. Compared with controls, TA-TMA patients had higher average systolic and diastolic blood pressure levels during high-dose chemotherapy and developed hypertension by day 13 after auto-SCT. Proteinuria was a significant marker for TA-TMA, whereas blood and platelet transfusion requirements were not. Serum creatinine did not differ between groups post transplant. However, patients with TA-TMA had a 60% decrease in renal function from baseline by nuclear glomerular filtration rate, compared with a 25% decrease in those without TA-TMA (P=0.001). There was no TA-TMA-related mortality. Significant complications included end-stage renal disease (n=1) and polyserositis (n=3). Patients with TA-TMA were unable to complete cis-RA therapy after auto-SCT. We suggest that careful attention to blood pressure and urinalysis will assist in the early diagnosis of TA-TMA, whereas serum creatinine seems to be an insensitive marker for this condition.

摘要

接受自体造血干细胞移植治疗神经母细胞瘤的患者是研究与移植相关的血栓性微血管病(TA-TMA)的独特人群,这是由于标准化疗以及随后暴露于辐射和维甲酸(cis-RA)。我们回顾性分析了 20 例自体造血干细胞移植后的患者,以评估 TA-TMA 的早期临床指标。共有 6 例患者(30%的患病率)发生 TA-TMA,并与 14 例对照进行比较。6 例患者中有 4 例在自体造血干细胞移植后 25 天内被诊断为 TA-TMA。与对照组相比,TA-TMA 患者在大剂量化疗期间的平均收缩压和舒张压水平更高,并在自体造血干细胞移植后第 13 天发生高血压。蛋白尿是 TA-TMA 的一个显著标志物,而输血和血小板输注需求不是。移植后两组之间的血清肌酐无差异。然而,与无 TA-TMA 的患者相比,TA-TMA 患者的肾功能核肾小球滤过率基线下降了 60%,而无 TA-TMA 的患者下降了 25%(P=0.001)。没有与 TA-TMA 相关的死亡。严重并发症包括终末期肾病(n=1)和多发性浆膜炎(n=3)。发生 TA-TMA 的患者在自体造血干细胞移植后无法完成 cis-RA 治疗。我们建议,密切关注血压和尿液分析将有助于早期诊断 TA-TMA,而血清肌酐似乎是该疾病的不敏感标志物。

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