Su Meng, Gao Yi-Jin, Pan Ci, Chen Jing, Tang Jing-Yan
a Key Laboratory of Pediatric Hematology & Oncology, Department of Hematology and Oncology, Shanghai Children's Medical Center , Shanghai Jiao Tong University School of Medicine , Shanghai , China.
Pediatr Hematol Oncol. 2018 Oct-Nov;35(7-8):385-392. doi: 10.1080/08880018.2018.1545814. Epub 2019 Jan 29.
This is a descriptive review of the clinical patterns and outcomes of children with Langerhans cell histiocytosis and single-system involvement (SS-LCH) treated at Shanghai Children's Medical Center.
60 evaluable newly diagnosed patients (37 boys, 23 girls) with a median age of 3.9 years (range: 0.3-15.3 years) and histiopathology-confirmed SS-LCH were enrolled from 2010 to 2014. All patients received systemic chemotherapy using either the DAL HX-83 or LCH-II protocol as determined by the physician.
Bone was the most frequently affected organ (56/60, 93.3%). Of the 56 patients suffering from SS-bone disease, 35 (62.5%) had unifocal disease and 21 (37.5%) had multifocal disease. CNS-risk lesions were seen in nine patients (16.1%, 9/56) at diagnosis. Thirty-two patients were treated with the LCH-II protocol and 28 received the DAL HX-83 protocol. No patient received intralesional steroid injection at the time of surgery. CNS-risk lesion correlated with an inferior event-free survival (EFS) for patients with bone disease (62.5 ± 17.1% vs. 90.7 ± 4.5%; p = 0.039). The difference in the 5-year EFS between patients with unifocal and multifocal SS-bone LCH did not reach the statistical significance (93.8 ± 4.3% vs. 75.0 ± 9.7%; p = 0.074). No deaths were observed, leading to a 5-year OS of 100% in the present cohort of patients. Permanent consequences and secondary malignancies were not observed but were also limited by short follow-up.
Optimal therapy for patients with SS-bone LCH has not been established. Less toxic therapeutic approaches should be considered for these patients and tested in prospective trials.
这是一项对在上海儿童医学中心接受治疗的朗格汉斯细胞组织细胞增多症单系统受累（SS-LCH）患儿的临床模式和治疗结果的描述性综述。
2010年至2014年纳入了60例可评估的新诊断患者（37例男孩，23例女孩），中位年龄为3.9岁（范围：0.3 - 15.3岁），组织病理学确诊为SS-LCH。所有患者均根据医生的决定采用DAL HX - 83或LCH-II方案接受全身化疗。
骨骼是最常受累的器官（56/60，93.3%）。在56例患有SS-骨病的患者中，35例（62.5%）为单病灶疾病，21例（37.5%）为多病灶疾病。诊断时9例患者（16.1%，9/56）出现中枢神经系统风险病变。32例患者采用LCH-II方案治疗，28例接受DAL HX - 83方案治疗。手术时无患者接受病灶内类固醇注射。中枢神经系统风险病变与骨病患者较差的无事件生存率（EFS）相关（62.5±17.1%对90.7±4.5%；p = 0.039）。单病灶和多病灶SS-骨LCH患者的5年EFS差异未达到统计学意义（93.8±4.3%对75.0±9.7%；p = 0.074）。未观察到死亡病例，本队列患者的5年总生存率为100%。未观察到永久性后果和继发性恶性肿瘤，但随访时间短也存在局限性。
SS-骨LCH患者的最佳治疗方案尚未确立。应考虑为这些患者采用毒性较小的治疗方法，并在前瞻性试验中进行测试。
