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儿童异基因造血干细胞移植后静脉闭塞性疾病诊断前启动支持性治疗的作用

Role of Initiating Supportive Care Preceding Veno-occlusive Disease Diagnosis Following Allogeneic Hematopoietic Stem Cell Transplantation in Children.

作者信息

Rayner Patricia, Spruit Jessica L, Chu Roland, Yankelevich Maxim, Henry Meret, Ravindranath Yaddanapudi, Savaşan Süreyya

机构信息

Pediatric Blood and Marrow Transplantation Program, Division of Hematology/Oncology, Children's Hospital of Michigan.

Barbara Ann Karmanos Cancer Center.

出版信息

J Pediatr Hematol Oncol. 2019 Aug;41(6):e395-e401. doi: 10.1097/MPH.0000000000001455.

Abstract

Severe veno-occlusive disease (VOD) following hematopoietic stem cell transplantation has a high mortality rate. The clinical course of VOD, role of preemptive and aggressive supportive care, and outcomes were investigated in a retrospective study from 2007 to 2014. Defibrotide was not available in all but one case with VOD at our center during the study. Forty-nine allogeneic transplants with intravenous busulfan-based or total body irradiation-based myeloablative conditioning were included. The median after hematopoietic stem cell transplantation day for suspicion of developing VOD (pre-VOD phase) was 6 due to weight gain, hepatomegaly, and/or mild increase in total bilirubin without fulfilling the modified Seattle criteria in 22 cases (45%). Despite fluid restriction, aggressive diuresis, and fresh frozen plasma infusions, 16 patients (33%) developed VOD by +10 days. Five cases (31%) had severe, 9 (56%) moderate, and 2 (13%) mild VOD. Eight cases (50%) required transfer to intensive care. One patient was given defibrotide, which was later discontinued due to concerns of adverse effects. Day +100 survival was 100% with complete resolution of VOD. Preemptive and aggressive supportive care could help achieve favorable outcomes in VOD and may have ameliorated the severity. This approach may be combined with other measures in the prevention/treatment of VOD.

摘要

造血干细胞移植后发生的严重静脉闭塞性疾病(VOD)死亡率很高。在一项2007年至2014年的回顾性研究中,对VOD的临床病程、抢先和积极支持治疗的作用以及预后进行了调查。在研究期间,除了1例VOD患者外,我们中心所有患者均未使用去纤苷。纳入了49例接受基于静脉注射白消安或全身照射的清髓性预处理的异基因移植患者。由于体重增加、肝肿大和/或总胆红素轻度升高,但未达到改良西雅图标准,22例(45%)患者在造血干细胞移植后怀疑发生VOD(VOD前期)的中位天数为6天。尽管采取了液体限制、积极利尿和输注新鲜冰冻血浆等措施,仍有16例患者(33%)在移植后10天内发生了VOD。5例(31%)为重度VOD,9例(56%)为中度VOD,2例(13%)为轻度VOD。8例(50%)患者需要转入重症监护病房。1例患者接受了去纤苷治疗,但后来因担心不良反应而停药。VOD完全缓解,移植后100天生存率为100%。抢先和积极的支持治疗有助于VOD取得良好预后,可能减轻了疾病严重程度。这种方法可与其他预防/治疗VOD的措施相结合。

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