Khan Saadiya, AlSayyad Khulood, Siddiqui Khawar, AlAnazi Awatif, AlSeraihy Amal, AlAhmari Ali, ElSolh Hassan, Ghemlas Ibrahim, AlSaedi Hawazen, AlJefri Abdullah, Ali Afshan, AlFawaz Ibrahim, AlKofide Amani, Ayas Mouhab
Department of Pediatric Hematology / Oncology, MBC 53, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
Int J Pediatr Adolesc Med. 2021 Dec;8(4):253-257. doi: 10.1016/j.ijpam.2021.02.006. Epub 2021 Mar 3.
Neuroblastoma is the most common extracranial solid tumor found in pediatric patients. High-risk neuroblastoma (HR-NBL) can be characterized by metastasis, age, and other tumor characteristics that result in an adverse outlook for this patient cohort. The standard of care includes induction chemotherapy, surgery, followed by stem cell autologous transplant (ASCT), and later, antidisialoganglioside (anti-GD2) antibodies. In this study, we provide the survival and toxicity data of our HR-NBL patients treated with a single ASCT.
We retrospectively analyzed pediatric HR-NBL patients treated with single ASCT after a carboplatin, etoposide, and melphalan (CEM) regimen in our institution between January 1993 and December 2014.
There were 99 evaluable patients with male predominance. The median age at diagnosis was 3 years. Most of our HR-NBL patients were stage 4 (88%). All patients received ASCT. Peripheral blood was the graft source in 58% of the patients. Time for hematological count recovery with bone marrow as a graft source was prolonged but not statistically significant when compared with PBSCs. Of all the patients, 58% received radiation therapy to residual disease. Overt secondary leukemia was not seen in any of these patients. Three-year overall survival (OS) was 68.5% ± 5.2% and the 3-year event-free survival (EFS) was (48.3% ± 5.2%).
Our HR-NBL patients tolerated high-dose chemotherapy well followed by single autologous stem cell transplant. Tandem transplant is a feasible option in our patient cohort. Apart from secondary solid tumors, there were no long-term complications seen.
神经母细胞瘤是儿科患者中最常见的颅外实体瘤。高危神经母细胞瘤（HR-NBL）的特征包括转移、年龄及其他肿瘤特征，这些因素导致该患者群体预后不良。治疗标准包括诱导化疗、手术，随后进行自体干细胞移植（ASCT），以及后期使用抗二唾液酸神经节苷脂（抗GD2）抗体。在本研究中，我们提供了接受单次ASCT治疗的HR-NBL患者的生存和毒性数据。
我们回顾性分析了1993年1月至2014年12月期间在我院接受卡铂、依托泊苷和美法仑（CEM）方案治疗后进行单次ASCT的儿科HR-NBL患者。
有99例可评估患者，男性居多。诊断时的中位年龄为3岁。我们的大多数HR-NBL患者为4期（88%）。所有患者均接受了ASCT。58%的患者使用外周血作为移植物来源。与外周血干细胞相比，以骨髓作为移植物来源时血液学计数恢复时间延长，但无统计学意义。所有患者中，58%接受了针对残留病灶的放射治疗。这些患者中均未出现明显的继发性白血病。三年总生存率（OS）为68.5%±5.2%，三年无事件生存率（EFS）为（48.3%±5.2%）。
我们的HR-NBL患者对大剂量化疗耐受性良好，随后进行单次自体干细胞移植。在我们的患者群体中，序贯移植是一种可行的选择。除继发性实体瘤外，未观察到长期并发症。
