Sedky Mohamed S, Hamouda Asmaa, Taha Hala, Zaky Iman, Hassanain Omayma, El Hemaly Ahmed, ElHaddad Alaa
Departments of Pediatric Oncology.
Department of Pediatrics, National Research Centre.
J Pediatr Hematol Oncol. 2019 Nov;41(8):635-643. doi: 10.1097/MPH.0000000000001314.
High-risk multisystem organ (RO+) Langerhans cell histiocytosis (LCH) has the least survival. We present the outcome of RO+ LCH in a pediatric single center. Fifty RO+ LCH patients, treated between 07/2007 and 07/2015, were retrospectively analyzed. Induction vinblastine (VBL) and prednisone (PRED) with intermediate-dose methotrexate (idMTX) was adopted until 2012 (n=20) wherein idMTX was omitted (n=30). The 3-year overall survival (OS) of MTX and non-MTX groups was 75% and 63%, respectively, P=0.537, while the event-free survival (EFS) was 36.9% and 13.2%, respectively, P=0.005. At week 12 of induction, "better status" was obtained in 80% of those receiving MTX, and 55% of those who were not. The statistically significant factors associated with both poor OS and EFS were trihemopoietic cytopenias, hepatic dysfunction, tri RO+ combination, and single induction. The factors associated with disease progression (DP) on induction were trihemopoietic cytopenias, hepatic dysfunction, and lack of idMTX, while those for disease reactivations (REA), the season of autumn/winter, lung disease, male sex, and idMTX were the associated factors. The 1-year OS was remarkably affected with the occurrence of DP versus REA versus none, wherein it was 47%, 93%, and 95%, respectively, P=0.001. In conclusion, idMTX is associated with better EFS. DP on induction remains of dismal prognosis in relation to disease REA afterwards. Risk stratification should highlight the role of trihemopoietic cytopenias, hepatic dysfunction, tri RO+, central nervous system risk site, and lung association.
高危多系统器官（RO+）朗格汉斯细胞组织细胞增多症（LCH）的生存率最低。我们展示了一家儿科单中心RO+ LCH的治疗结果。对2007年7月至2015年7月期间治疗的50例RO+ LCH患者进行回顾性分析。2012年之前采用长春花碱（VBL）、泼尼松（PRED）联合中剂量甲氨蝶呤（idMTX）进行诱导治疗（n = 20），之后不再使用idMTX（n = 30）。MTX组和非MTX组的3年总生存率（OS）分别为75%和63%，P = 0.537，而无事件生存率（EFS）分别为36.9%和13.2%，P = 0.005。在诱导治疗第12周时，接受MTX治疗的患者中有80%达到“较好状态”，未接受MTX治疗的患者中这一比例为55%。与OS和EFS较差均相关的统计学显著因素包括三系血细胞减少、肝功能障碍、三联RO+组合和单次诱导。与诱导期疾病进展（DP）相关的因素包括三系血细胞减少症、肝功能障碍和未使用idMTX，而与疾病复发（REA）相关的因素包括秋冬季节、肺部疾病、男性性别和使用idMTX。DP、REA与无上述情况相比，1年OS受到显著影响，其中分别为47%、93%和95%，P = 0.001。总之，idMTX与更好的EFS相关。诱导期的DP相较于之后的疾病REA，预后仍然很差。风险分层应突出三系血细胞减少、肝功能障碍、三联RO+、中枢神经系统风险部位和肺部关联的作用。
