Risk Factors for Transplant-Associated Thrombotic Microangiopathy after Autologous Hematopoietic Cell Transplant in High-Risk Neuroblastoma.
Risk Factors for Transplant-Associated Thrombotic Microangiopathy after Autologous Hematopoietic Cell Transplant in High-Risk Neuroblastoma.
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DOI：
10.1016/j.bbmt.2019.06.006
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发表时间：
2019-10
影响因子：
4.3
通讯作者：
Vo, Kieuhoa T.
中科院分区：
文献类型：
--
作者：
Tolbert, Vanessa P.;Dvorak, Christopher C.;Golden, Carla;Vissa, Madhav;El-Haj, Nura;Perwad, Farzana;Matthay, Katherine K.;Vo, Kieuhoa T.
关键词：
高危神经母细胞瘤预后不良，研究表明，增加治疗强度可改善预后。自体造血细胞移植（aHCT）作为巩固治疗具有显著的生存优势，但伴随着显著的发病率。移植相关血栓性微血管病（TA-TMA）是一种由内皮损伤引起的危及生命的并发症，常导致溶血性贫血、微血栓性血小板消耗和肾损伤。在此，我们调查了高危神经母细胞瘤患者TA-TMA的发病率、潜在危险因素和后遗症。我们对2000-2017年在我们机构接受aHCT的所有神经母细胞瘤患者（n=141）进行了回顾性图表审查。10例患者（7%）发生TA-TMA。TA-TMA组的患者在人口统计学、疾病负担、既往治疗、肾功能和移植时间方面与其余受试者相似。预处理方案的类型是唯一具有统计学意义的移植前变量（p<0.001）。15例患者中有6例（40%）打算接受串联移植（环磷酰胺/塞替派，然后卡铂/依托泊苷/美法仑（CEM）），68例接受单CEM预处理的患者中有4例（6%），56例接受白消安/美法仑的患者中无一例被诊断为TA-TMA。TA-TMA患者更可能需要ICU转移，住院时间更长，并且在随后的治疗中经历延迟或改变。在我们的队列中，移植后延迟治疗的患者的总体生存率似乎更差，尽管差异无统计学意义。由于这种高发病率和显著的发病率，我们在移植期间和移植后实施了TA-TMA的标准化筛查。我们预计，筛查将导致早期干预和降低疾病的严重程度。
High-risk neuroblastoma has a poor prognosis and research studies have shown that increasing the intensity of therapy improves outcomes. Autologous hematopoietic cell transplant (aHCT) as consolidation therapy confers a significant survival advantage but is accompanied by significant morbidity. Transplant-associated thrombotic microangiopathy (TA-TMA) is a life-threatening complication caused by endothelial injury that often leads to hemolytic anemia, microthrombotic platelet consumption, and renal injury. Here we investigated the incidence, potential risk-factors, and sequelae of TA-TMA in patients with high-risk neuroblastoma. We conducted a retrospective chart review of all patients (n=141) with neuroblastoma in our institutions who underwent aHCT from 2000-2017. Ten patients (7%) developed TA-TMA. The patients in the TA-TMA group were similar to the rest of the subjects in demographics, disease burden, prior therapies, renal function, and timing of transplant. The type of conditioning regimen was the only statistically significant pre-transplant variable (p<0.001). Six of the 15 (40%) patients intended to receive tandem transplants (cyclophosphamide/thiotepa, then carboplatin/etoposide/melphalan (CEM)), four of the 68 (6%) patients who received conditioning with single CEM, and none of the 56 who received busulfan/melphalan were diagnosed with TA-TMA. Patients with TA-TMA were more likely to require ICU transfer, have a longer length of stay in the hospital, and experience a delay or change in their subsequent therapy. In our cohort overall, patients with a delay in therapy following transplant appeared to have a worse overall survival, although the difference was not statistically significant. Due to this high incidence and significant morbidity, we have implemented standardized screening for TA-TMA during and after transplant. We anticipate that screening will lead to earlier intervention and decreased severity of disease.
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影响因子：
6.2
作者：
de Fontbrune, Flore Sicre;Galambrun, Claire;de Latour, Regis Peffaut
通讯作者：
de Latour, Regis Peffaut
影响因子：
45.3
作者：
BRODEUR, GM;PRITCHARD, J;VOUTE, PA
通讯作者：
VOUTE, PA
影响因子：
20.3
作者：
Jodele, Sonata;Davies, Stella M.;Laskin, Benjamin L.
通讯作者：
Laskin, Benjamin L.
影响因子：
45.3
作者：
Grupp, SA;Stern, JW;Diller, L
通讯作者：
Diller, L
影响因子：
20.3
作者：
Jodele, Sonata;Zhang, Kejian;Davies, Stella M.
通讯作者：
Davies, Stella M.