Gaziev Javid, Marziali Simone, Paciaroni Katia, Isgrò Antonella, Di Giuliano Francesca, Rossi Giorgia, Marziali Marco, De Angelis Gioia, Alfieri Cecilia, Ribersani Michela, Andreani Marco, Palmieri Maria Giuseppina, Placidi Fabio, Romigi Andrea, Izzi Francesca, Floris Roberto, Mercuri Nicola Biagio
International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinico Tor Vergata, Rome, Italy.
Department of Biomedicine and Prevention, University of Rome Tor Vergata, Department of Diagnostic and Molecular Imaging, Interventional Radiology, Radiotherapy and Neuroradiology Unità Operativa Complessa, Fondazione PTV Policlinico Tor Vergata, Rome, Italy.
Biol Blood Marrow Transplant. 2017 Sep;23(9):1531-1540. doi: 10.1016/j.bbmt.2017.05.033. Epub 2017 Jul 3.
Posterior reversible encephalopathy syndrome (PRES) is a serious adverse event associated with calcineurin inhibitors used for graft-versus-host disease (GVHD) prophylaxis. We compared the incidence of PRES in children with thalassemia (n = 222, 1.4 to 17.8 years old) versus sickle cell disease (SCD; n = 59, 2 to 17 years old) who underwent hematopoietic cell transplantation from HLA-matched siblings or alternative donors and analyzed the risk factors for PRES. Overall, 31 children developed calcineurin inhibitor-related PRES (11%), including 30 patients with seizures and 1 patient without seizures. PRES incidence was significantly higher in SCD patients (22%; 95% confidence interval [CI], 10% to 32%) than in thalassemia patients (8%; 95% CI, 5% to 12%;P = .002). In multivariate analysis, factors associated with PRES were hypertension (hazard ratio [HR], 5.87; 95% CI, 2.57 to 13.43; P = .0001), SCD (HR, 2.49; 95% CI, 1.25 to 4.99; P = .009), and acute GVHD (HR 2.27; 95% CI, 1.06 to 4.85; P= .031). In the entire cohort overall survival (OS) was significantly higher in patients without versus with PRES (90% versus 77%; P = .02). In a subgroup analysis that including matched sibling transplants, OS and disease-free survival (DFS) were similar in thalassemia patients without PRES (92% and 88%, respectively) and with PRES (82% and 73%, respectively), whereas SCD patients with PRES had significantly lower OS (67%) and DFS (67%) than patients without PRES (94% and 94%, respectively; P = .008). Thus, SCD patients had a significantly higher incidence of PRES than thalassemia patients, and hypertension and GVHD were the 2 main risk factors for PRES in patients with hemoglobinopathies. Although PRES did not significantly influence survival in patients with thalassemia, patients with SCD had significantly lower survival after PRES.
后部可逆性脑病综合征（PRES）是一种与用于预防移植物抗宿主病（GVHD）的钙调神经磷酸酶抑制剂相关的严重不良事件。我们比较了接受来自 HLA 匹配同胞或替代供体的造血细胞移植的地中海贫血患儿（n = 222，年龄 1.4 至 17.8 岁）与镰状细胞病（SCD；n = 59，年龄 2 至 17 岁）中 PRES 的发生率，并分析了 PRES 的危险因素。总体而言，31 名儿童发生了与钙调神经磷酸酶抑制剂相关的 PRES（11%），其中包括 30 例有癫痫发作的患者和 1 例无癫痫发作的患者。SCD 患者中 PRES 的发生率（22%；95%置信区间[CI]，10%至 32%）显著高于地中海贫血患者（8%；95%CI，5%至 12%；P = 0.002）。在多变量分析中，与 PRES 相关的因素有高血压（风险比[HR]，5.87；95%CI，2.57 至 13.43；P = 0.0001）、SCD（HR，2.49；95%CI，1.25 至 4.99；P = 0.009）和急性 GVHD（HR 2.27；95%CI，1.06 至 4.85；P = 0.031）。在整个队列中，无 PRES 的患者总体生存率（OS）显著高于有 PRES 的患者（90%对 77%；P = 0.02）。在一项包括匹配同胞移植的亚组分析中，地中海贫血无 PRES 的患者（分别为 92%和 88%）和有 PRES 的患者（分别为 82%和 73%）的 OS 和无病生存率（DFS）相似，而 SCD 有 PRES 的患者的 OS（67%）和 DFS（67%）显著低于无 PRES 的患者（分别为 94%和 94%；P = 0.008）。因此，SCD 患者 PRES 的发生率显著高于地中海贫血患者，高血压和 GVHD 是血红蛋白病患者 PRES 的两个主要危险因素。尽管 PRES 对地贫患者的生存没有显著影响，但 SCD 患者发生 PRES 后的生存率显著降低。
