Rubie H, Plantaz D, Coze C, Michon J, Frappaz D, Baranzelli M C, Chastagner P, Peyroulet M C, Hartmann O
Unité d'Hemato-Oncologie Pediatrique, Hĵpital des Enfants, Toulouse, France.
Med Pediatr Oncol. 2001 Jan;36(1):247-50. doi: 10.1002/1096-911X(20010101)36:1<247::AID-MPO1061>3.0.CO;2-Z.
Infants with neuroblastoma (NB) were assessed according to INSS recommendations, including MIBG scan and extensive bone marrow staging to eliminate metastatic spread. Patients with unresectable tumour received chemotherapy, including two courses of carboplatin-etoposide (CE) and two of vincristinecyclophosphamide-doxorubicin (CAdO). Post-operative treatment was to be given only in infants with MYCN amplification. Between 1990 and 1994, 52 consecutive children were registered.
Among the 44 patients who received CE as a first course, the response rate was (66%) and the primary could be removed in all children but one, who was in remission. The toxicity was mainly haematological and was always manageable. The 5 year overall survival (OS) and event-free survival (EFS) were 94 and 90 +/- 8%, respectively, with a median follow-up of 48 months. The outcome of infants with no MYCN amplification was excellent; OS and EFS were, respectively, 97 and 94%.
Chemotherapy allows surgical excision and excellent outcome in infants with localised and unresectable NB. Less intensive Chemotherapy should be investigated in such patients.
根据国际神经母细胞瘤分期系统（INSS）的建议对神经母细胞瘤（NB）患儿进行评估，包括进行间碘苄胍（MIBG）扫描和广泛的骨髓分期以排除转移扩散。无法切除肿瘤的患者接受化疗，包括两个疗程的卡铂 - 依托泊苷（CE）以及两个疗程的长春新碱 - 环磷酰胺 - 阿霉素（CAdO）。仅对MYCN基因扩增的婴儿进行术后治疗。1990年至1994年期间，连续登记了52名儿童。
在44例接受CE作为第一疗程的患者中，缓解率为66%，除一名处于缓解期的儿童外，所有儿童的原发肿瘤均可切除。毒性主要为血液学毒性，且始终可控。5年总生存率（OS）和无事件生存率（EFS）分别为94%和90±8%，中位随访时间为48个月。无MYCN基因扩增的婴儿预后极佳；OS和EFS分别为97%和94%。
化疗可使局限性和无法切除的NB婴儿实现手术切除并获得良好预后。对此类患者应研究强度较低的化疗方案。
