Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas.
Department of Pediatrics, Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas.
Biol Blood Marrow Transplant. 2019 Jan;25(1):157-162. doi: 10.1016/j.bbmt.2018.08.016. Epub 2018 Aug 23.
Transplantation-associated thrombotic microangiopathy (TA-TMA) is a complication of hematopoietic stem cell transplant (HSCT) that causes severe multiorgan injury. The kidneys are almost universally affected. There is no proven therapy, but therapeutic plasma exchange (TPE) is commonly used to treat TA-TMA at Texas Children's Hospital (TCH). To date, there have been no studies assessing the long-term efficacy of TPE in preventing the development of chronic kidney disease (CKD) in TA-TMA patients. In this study we retrospectively analyzed the incidence of CKD in TA-TMA pediatric patients treated with TPE to determine if this treatment modality improves renal morbidity. We reviewed records between January 2007 and June 2017 of pediatric HSCT patients diagnosed with TA-TMA, identified through an internal database maintained at TCH. To be included patients must have completed a course of TPE per the "TPE in TA-TMA" institutional protocol at TCH. CKD was defined as kidney damage for at least 3 months and stratified into stages 1 through 5 according to estimated glomerular filtration rate. Stages 4 and 5 were considered "severe CKD." In the 10-year timeframe 15 patients with TA-TMA completed a course of TPE per our institutional protocol and were subsequently followed for a median of 963 days. Fourteen patients developed CKD, and 5 of these 14 patients developed severe CKD. The cumulative incidence of severe CKD development was 33% (95% confidence interval. 11% to 57%). 6 patients required dialysis, and 2 patients received a renal transplant. 5 patients received eculizumab in addition to TPE. In our patients a TPE course of at least 7 weeks (and up to 25 weeks) was not effective in the prevention of CKD. Our data indicate a need for alternative therapeutic measures to prevent the development of CKD in TA-TMA patients.
移植相关血栓性微血管病（TA-TMA）是造血干细胞移植（HSCT）的并发症，可导致严重的多器官损伤。肾脏几乎普遍受到影响。目前尚无特效疗法，但治疗性血浆置换（TPE）常用于治疗德克萨斯儿童医院（TCH）的 TA-TMA。迄今为止，尚无研究评估 TPE 在预防 TA-TMA 患者发生慢性肾脏病（CKD）方面的长期疗效。在这项研究中，我们回顾性分析了接受 TPE 治疗的 TA-TMA 儿科患者的 CKD 发生率，以确定这种治疗方式是否能改善肾脏发病率。我们回顾了 2007 年 1 月至 2017 年 6 月期间在 TCH 通过内部数据库确诊为 TA-TMA 的儿科 HSCT 患者的记录。纳入标准为患者必须按照 TCH 的“TPE 在 TA-TMA 中的应用”机构方案完成 TPE 疗程。CKD 的定义为至少 3 个月的肾脏损伤，并根据估计肾小球滤过率分为 1 至 5 期。4 期和 5 期被认为是“严重 CKD”。在 10 年的时间里，15 例 TA-TMA 患者按照我们的机构方案完成了 TPE 疗程，随后中位随访 963 天。14 例患者出现 CKD，其中 14 例患者发展为严重 CKD。严重 CKD 发展的累积发生率为 33%（95%置信区间为 11%至 57%）。6 例患者需要透析，2 例患者接受了肾移植。5 例患者除 TPE 外还接受了依库珠单抗治疗。在我们的患者中，至少 7 周（最长 25 周）的 TPE 疗程并不能有效预防 CKD。我们的数据表明，需要替代治疗措施来预防 TA-TMA 患者发生 CKD。
