Garaventa A, De Bernardi B, Pianca C, Donfrancesco A, Cordero di Montezemolo L, Di Tullio M T, Bagnulo S, Mancini A, Carli M, Pession A, Arrighini A, Di Cataldo A, Tamaro P, Iasonni V, Taccone A, Rogers D, Boni L
Department of Hematology-Oncology, Giannina Gaslini Children's Hospital, Genova, Italy.
J Clin Oncol. 1993 Sep;11(9):1770-9. doi: 10.1200/JCO.1993.11.9.1770.
To define factors that influence outcome in children with localized but unresectable neuroblastoma by retrospective investigation of response to therapy and outcome in 21 Italian institutions.
Of 145 assessable children diagnosed between 1979 and 1990, 77 were treated between 1979 and 1984 with three consecutive standard-dose (SD) protocols, and 68 between 1985 and 1990 with a high-dose (HD) protocol. All protocols included chemotherapy, followed by resection of primary tumor if feasible. If at least partial resection was achieved, consolidation therapy followed, except that from 1985 onward, patients considered disease-free following surgery received no further treatment.
Ninety-four of 145 patients (65%) achieved a complete response (CR) or partial response (PR) with chemotherapy and 75 (52%) subsequently underwent complete resection of the primary tumor. Eighty-one patients are alive (73 without disease, eight with disease), 63 have died, and one is lost to follow-up. The 5-year overall survival (OS) rate is 55% and progression-free survival (PFS) rate 50%. Both OS and PFS correlated with response to chemotherapy, removal of primary tumor, HD therapy, and serum lactate dehydrogenase (LDH) levels. Infants (< 1 year), independent of primary tumor site, and children aged 1 to 15 years with a nonabdominal primary tumor, did better compared with children aged 1 to 15 years with an abdominal primary tumor (PFS, 72% and 64% v 30%; P < .001 and < .01, respectively). Outcome of this last group improved with the HD protocol (PFS, 40% v 23%; P = .01).
In children with unresectable neuroblastoma, risk categories can be defined by age and primary tumor site. HD chemotherapy should be investigated for the poor-risk category age 1 to 15 years with an abdominal primary tumor.
通过对意大利21家机构中局部但无法切除的神经母细胞瘤患儿的治疗反应和预后进行回顾性研究，确定影响其预后的因素。
在1979年至1990年间确诊的145例可评估患儿中，77例在1979年至1984年间接受了三个连续的标准剂量（SD）方案治疗，68例在1985年至1990年间接受了高剂量（HD）方案治疗。所有方案均包括化疗，若可行则随后切除原发肿瘤。若至少实现了部分切除，则进行巩固治疗，但从1985年起，术后被认为无疾病的患者不再接受进一步治疗。
145例患者中有94例（65%）通过化疗达到完全缓解（CR）或部分缓解（PR），75例（52%）随后接受了原发肿瘤的完全切除。81例患者存活（73例无疾病，8例有疾病），63例死亡，1例失访。5年总生存率（OS）为55%，无进展生存率（PFS）为50%。OS和PFS均与化疗反应、原发肿瘤切除、HD治疗及血清乳酸脱氢酶（LDH）水平相关。1岁以下婴儿（无论原发肿瘤部位）以及1至15岁非腹部原发肿瘤的儿童，与1至15岁腹部原发肿瘤的儿童相比，预后更好（PFS分别为72%和64%对30%；P分别<.001和<.01）。最后一组患者采用HD方案后预后改善（PFS为40%对23%；P = .01）。
在无法切除的神经母细胞瘤患儿中，风险类别可根据年龄和原发肿瘤部位来定义。对于1至
