泰国高危神经母细胞瘤患者大剂量化疗加干细胞救援的治疗结果
Treatment outcomes of high-dose chemotherapy plus stem cell rescue in high-risk neuroblastoma patients in Thailand.
作者信息
Suwannaying Kunanya, Techavichit Piti, Komvilaisak Patcharee, Laoaroon Napat, Narkbunnam Nattee, Sanpakit Kleebsabai, Chiengthong Kanhatai, Chotsampancharoen Thirachit, Sathitsamitphong Lalita, Santong Chalongpon, Seksarn Panya, Hongeng Suradej, Wiangnon Surapon
机构信息
Division of Hematology-Oncology, Department of Pediatrics, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand.
Integrative and Innovative Hematology/Oncology Research Unit, Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.
出版信息
Clin Exp Pediatr. 2022 Sep;65(9):453-458. doi: 10.3345/cep.2022.00437. Epub 2022 May 24.
BACKGROUND
In 2013, the Thai Pediatric Oncology Group (ThaiPOG) introduced a national protocol in which high-dose chemotherapy plus stem cell rescue is performed without immunotherapy.
METHODS
This study aimed to elucidate the outcomes of high-risk neuroblastoma (HR-NB) patients treated with the ThaiPOG protocol. This retrospective cohort review included 48 patients (30 males, 18 females) with a median age of 3 years (range, 8 months to 18 years) who were treated at 5 ThaiPOG treatment centers in Thailand in 2000-2018.
RESULTS
Eight of the 48 patients showed MYCN amplification. Twenty-three patients (48%) received 131I-meta-iodobenzylguanidine prior to high-dose chemotherapy and stem cell rescue. The majority of patients achieved a complete or very good response prior to consolidation treatment. The 5-year overall survival (OS) and event-free survival (EFS) rates were 45.1% and 40.4%, respectively. Patients aged >2 years had a nonsignificantly higher mortality risk (hazard ratio [HR], 2.66; 95% confidence interval [CI], 0.92-7.68; P=0.07). The MYCN amplification group had lower OS and EFS rates than the MYCN nonamplification group, but the difference was not statistically significant (45% OS and 37.5% EFS vs. 33.3% OS and 16.6% EFS; P=0.67 and P=0.67, respectively). Cis-retinoic acid treatment for 12 months was a strong prognostic factor that could reduce mortality rates among HR-NB patients (HR, 0.27; 95% CI, 0.09-0.785; P=0.01).
CONCLUSION
High-dose chemotherapy plus stem cell rescue followed by cis-retinoic acid for 12 months was well tolerated and could improve the survival rates of patients with HR-NB.
背景
2013年，泰国儿科肿瘤学组（ThaiPOG）推出了一项全国性方案，即进行高剂量化疗加干细胞救援，不进行免疫治疗。
方法
本研究旨在阐明接受ThaiPOG方案治疗的高危神经母细胞瘤（HR-NB）患者的治疗结果。这项回顾性队列研究纳入了2000年至2018年期间在泰国5个ThaiPOG治疗中心接受治疗的48例患者（30例男性，18例女性），中位年龄为3岁（范围为8个月至18岁）。
结果
48例患者中有8例显示MYCN扩增。23例患者（48%）在高剂量化疗和干细胞救援前接受了131I-间碘苄胍治疗。大多数患者在巩固治疗前达到了完全缓解或非常好的缓解。5年总生存率（OS）和无事件生存率（EFS）分别为45.1%和40.4%。年龄>2岁的患者死亡风险略高（风险比[HR]，2.66；95%置信区间[CI]，0.92-7.68；P=0.07）。MYCN扩增组的OS和EFS率低于MYCN非扩增组，但差异无统计学意义（OS分别为45%和37.5%，EFS分别为33.3%和16.6%；P分别为0.67和0.67）。12个月的顺式维甲酸治疗是一个强有力的预后因素，可降低HR-NB患者的死亡率（HR，0.27；95%CI，0.09-0.785；P=0.01）。
结论
高剂量化疗加干细胞救援，随后进行12个月的顺式维甲酸治疗，耐受性良好，可提高HR-NB患者的生存率。
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