Division of Hematology-Oncology, Department of Pediatrics, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand.
Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and Phramongkutklao College of Medicine, Bangkok, Thailand.
Pediatr Blood Cancer. 2022 Sep;69(9):e29757. doi: 10.1002/pbc.29757. Epub 2022 May 13.
Neuroblastoma is the most common extracranial malignant solid tumor during childhood. Despite intensified treatment, patients with high-risk neuroblastoma (HR-NBL) still carry a dismal prognosis. The Thai Pediatric Oncology Group (ThaiPOG) proposed the use of a multimodality treatment to improve outcomes of HR-NBL in non-immunotherapy settings.
Patients with HR-NBL undergoing ThaiPOG protocols (ThaiPOG-NB-13HR or -18HR) between 2013 and 2019 were retrospectively reviewed. Patient demographic data, treatment modalities, outcomes, and prognostic factors were evaluated and analyzed.
A total of 183 patients with HR-NBL undergoing a topotecan containing induction regimen were enrolled in this study. During the consolidation phase (n = 169), 116 patients (68.6%) received conventional chemotherapy, while 53 patients (31.4%) underwent hematopoietic stem cell transplantation (HSCT). The 5-year overall survival (OS) and event-free survival (EFS) were 41.2% and 22.8%, respectively. Patients who underwent HSCT had more superior 5-year EFS (36%) than those who received chemotherapy (17.1%) (p = .041), although they both performed similarly in 5-year OS (48.7% vs. 39.8%, p = .17). The variation of survival outcomes was observed depending on the number of treatment modalities. HSCT combined with metaiodobenzylguanidine (MIBG) treatment and maintenance with 13-cis-retinoic acid (cis-RA) demonstrated a desirable 5-year OS and EFS of 65.6% and 58.3%, respectively. Poorly or undifferentiated tumor histology and cis-RA administration were independent factors associated with relapse and survival outcomes, respectively (p < .05).
A combination of HSCT and cis-RA successfully improved the outcomes of patients with HR-NBL in immunotherapy inaccessible settings.
神经母细胞瘤是儿童期最常见的颅外恶性实体瘤。尽管强化治疗，高危神经母细胞瘤（HR-NBL）患者的预后仍较差。泰国儿科肿瘤学组（ThaiPOG）提出采用多模式治疗以改善非免疫治疗环境中 HR-NBL 的结局。
回顾性分析 2013 年至 2019 年期间接受 ThaiPOG 方案（ThaiPOG-NB-13HR 或 -18HR）治疗的 HR-NBL 患者。评估并分析患者的人口统计学数据、治疗方式、结局和预后因素。
共纳入 183 例接受拓扑替康含诱导方案治疗的 HR-NBL 患者。在巩固阶段（n=169），116 例（68.6%）患者接受常规化疗，53 例（31.4%）患者接受造血干细胞移植（HSCT）。5 年总生存率（OS）和无事件生存率（EFS）分别为 41.2%和 22.8%。接受 HSCT 的患者 5 年 EFS 优于接受化疗的患者（36% vs. 17.1%，p=0.041），尽管两组患者 5 年 OS 相似（48.7% vs. 39.8%，p=0.17）。生存结局的变化取决于治疗方式的数量。HSCT 联合间碘苄胍（MIBG）治疗和 13-顺式维甲酸（cis-RA）维持治疗显示出令人满意的 5 年 OS 和 EFS，分别为 65.6%和 58.3%。较差或未分化的肿瘤组织学和 cis-RA 给药是与复发和生存结局相关的独立因素（p<0.05）。
HSCT 和 cis-RA 的联合成功改善了免疫治疗不可及环境中 HR-NBL 患者的结局。
