Department of Nuclear Medicine, Cruces University Hospital, Plaza de Cruces s/n, 48903, Baracaldo, Vizcaya, Spain.
Department of Medical Physics and Radiation Protection, Cruces University Hospital, Plaza de Cruces s/n, 48903, Baracaldo, Vizcaya, Spain.
Eur J Nucl Med Mol Imaging. 2019 Jul;46(7):1567-1575. doi: 10.1007/s00259-019-04291-x. Epub 2019 Mar 5.
Patients with high-risk neuroblastoma have an increased risk of recurrence and relapse of disease and a very poor prognosis. I-metaiodobenzylguanidine (I-mIBG) in combination with topotecan as a radiosensitizer can be an effective and relatively well-tolerated agent for the treatment of refractory neuroblastoma. The aim of this retrospective study was to evaluate response and outcome of combined therapy with I-mIBG and topotecan.
Ten patients, between 3 and 20 years of age, were included. Nine patients had been refractory to several lines of chemotherapy and radiotherapy. One patient with a very high-risk neuroblastoma had received only induction therapy. Response was graded according to the International Neuroblastoma Staging System.
Regarding treatment response, two patients achieved complete remission, one with relapse at 16 months, five achieved a partial remission, four showed progression at between 1 and 18 months; two showed stable disease with progression at between 1 and 5 months, and one showed progressive disease. Eight of the ten patients died with overall survival between 4 and 63 months, and two patients were still alive without disease at the time of this report: 52 and 32 months (patient had received only induction therapy). Acute and subacute adverse effects were mainly haematological, and one patient developed a differentiated thyroid cancer.
In patients with high-risk refractory neuroblastoma, administration of high activities of I-mIBG in combination with topotecan was found to be an effective therapy, increasing overall survival and progression-free survival. Further studies including a larger number of patients and using I-mIBG for first-line up-front therapy are warranted.
患有高危神经母细胞瘤的患者疾病复发和转移的风险增加，预后极差。间碘苄胍（I-mIBG）联合拓扑替康作为放射增敏剂可有效治疗难治性神经母细胞瘤，且不良反应相对较轻。本回顾性研究旨在评估 I-mIBG 联合拓扑替康治疗的疗效和预后。
共纳入 10 例年龄 3 至 20 岁的患者。9 例患者对多种化疗和放疗方案耐药，1 例高危神经母细胞瘤患者仅接受诱导治疗。根据国际神经母细胞瘤分期系统评估缓解情况。
2 例患者达到完全缓解，1 例缓解后 16 个月复发，5 例部分缓解，4 例缓解后 1 至 18 个月疾病进展，2 例疾病稳定但进展，1 例疾病进展。10 例患者中 8 例死亡，总生存期为 4 至 63 个月，2 例患者仍存活且无疾病，分别为 52 个月和 32 个月（该患者仅接受诱导治疗）。急性和亚急性不良反应主要为血液学毒性，1 例患者发生分化型甲状腺癌。
对于高危难治性神经母细胞瘤患者，给予高活性 I-mIBG 联合拓扑替康治疗可有效提高总生存期和无进展生存期。需要进一步开展包括更多患者的研究，并评估 I-mIBG 作为一线治疗药物的疗效。
