Idiopathic inflammatory demyelinating diseases (IIDDs) of the central nervous system (CNS) are rare but serious neurological complications of haploidentical hematopoietic stem cell transplantation (haplo-HSCT). However, the risk factors and a method to predict the prognosis of post-transplantation CNS IIDDs are not available. This retrospective study first reviewed data from 4532 patients who received haplo-HSCT during 2008–2019 in our center, and 184 patients (4.1%) with IIDDs after haplo-HSCT were identified. Grades II to IV acute graft-versus-host disease (aGVHD) (p < 0.001) and chronic GVHD (cGVHD) (p = 0.009) were identified as risk factors for developing IIDDs after haplo-HSCT. We then divided the 184 IIDD patients into a derivation cohort and validation cohort due to transplantation time to develop and validate a model for predicting the prognosis of IIDDs. In the multivariate analysis of the derivation cohort, four candidate predictors were entered into the final prognostic model: cytomegalovirus (CMV) infection, Epstein–Barr virus (EBV) infection, IgG synthesis (IgG-syn) and spinal cord lesions. The prognostic model had an area under the receiver operating characteristic curve of 0.864 (95% CI: 0.803–0.925) in the internal validation cohort and 0.871 (95% CI: 0.806–0.931) in the external validation cohort. The calibration plots showed a high agreement between the predicted and observed outcomes. Decision curve analysis indicated that IIDD patients could benefit from the clinical application of the prognostic model. The identification of IIDD patients after allo-HSCT who have a poor prognosis might allow timely treatment and improve patient survival and outcomes.

中文翻译：

---

血液系统恶性肿瘤患者半相合造血干细胞移植后特发性炎性脱髓鞘疾病的临床危险因素和预后模型

中枢神经系统 (CNS) 的特发性炎性脱髓鞘疾病 (IIDD) 是单倍体相合造血干细胞移植 (haplo-HSCT) 的罕见但严重的神经系统并发症。然而，目前尚无预测移植后 CNS IIDDs 预后的风险因素和方法。这项回顾性研究首先回顾了 2008-2019 年我们中心接受 haplo-HSCT 的 4532 名患者的数据，并确定了 184 名（4.1%）在 haplo-HSCT 后患有 IIDD 的患者。II 至 IV 级急性移植物抗宿主病 (aGVHD) ( p  < 0.001) 和慢性 GVHD (cGVHD) ( p = 0.009) 被确定为 haplo-HSCT 后发生 IIDD 的风险因素。然后，我们根据移植时间将 184 名 IIDD 患者分为派生队列和验证队列，以开发和验证预测 IIDD 预后的模型。在衍生队列的多变量分析中，将四个候选预测因子输入到最终的预后模型中：巨细胞病毒 (CMV) 感染、爱泼斯坦-巴尔病毒 (EBV) 感染、IgG 合成 (IgG-syn) 和脊髓损伤。预后模型的受试者操作特征曲线下面积在内部验证队列中为 0.864（95% CI：0.803-0.925），在外部验证队列中为 0.871（95% CI：0.806-0.931）。校准图显示预测结果和观察结果之间高度一致。决策曲线分析表明 IIDD 患者可以从预后模型的临床应用中受益。鉴别异基因造血干细胞移植后预后不良的 IIDD 患者可能允许及时治疗并改善患者的生存和结果。