Epidemiology and Biostatistics Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Oncology Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy.
Pediatr Blood Cancer. 2021 May;68(5):e28904. doi: 10.1002/pbc.28904. Epub 2021 Jan 18.
Stage 4S neuroblastoma, a tumor affecting infants, is characterized by the capacity to regress spontaneously and high cure rate. About a third of these infants undergo tumor progression requiring antitumor treatment and 10-15% eventually die. In case of metastatic progression, it may occur either at 4S sites (mainly liver) or sites characterizing stage 4 (mainly bone). Aim of this study was to estimate incidence, presenting features and outcome of infants who progressed to stage 4S or stage 4 sites.
Of 280 Italian infants diagnosed with stage 4S neuroblastoma between 1979 and 2013 and registered in the Italian Neuroblastoma Registry, 268 were evaluable for this study, of whom 57 developed metastatic progression.
Progression to stage 4S sites occurred in 29/268 infants (10.8%) (Group A) and to stage 4 in 28/268 (10.4%) (Group B). No significant difference was observed between the two groups at the time of diagnosis. At the time of progression, Group A infants were younger (7.3 vs 14.4 months, P = .001) and had a shorter interval from diagnosis to progression (3.8 vs 9.6 months, P = .001). Survival after progression was worse for Group B infants (45% vs 69%, P = .058) and was associated with age at diagnosis lower than 2 months (P = .005) and adrenal primary tumor site (P = .008). Survival rates increased for both groups along the study period.
Infants who progressed to stage 4 did worse, possibly in relation to older age at progression and longer interval between diagnosis and progression. Large prospective studies of these patients may lead to more effective treatments.
4S 期神经母细胞瘤是一种影响婴儿的肿瘤，其特征是具有自发消退的能力和较高的治愈率。大约三分之一的婴儿会出现肿瘤进展，需要进行抗肿瘤治疗，而 10-15%的婴儿最终会死亡。在发生转移进展的情况下，可能发生在 4S 部位（主要是肝脏）或 4 期部位（主要是骨骼）。本研究的目的是评估进展为 4S 期或 4 期部位的婴儿的发病率、表现特征和结局。
1979 年至 2013 年间，意大利神经母细胞瘤登记处登记了 280 例诊断为 4S 期神经母细胞瘤的意大利婴儿，其中 268 例可用于本研究，其中 57 例发生转移性进展。
268 例婴儿中有 29 例（10.8%）（A 组）进展为 4S 期部位，28 例（10.4%）（B 组）进展为 4 期。两组在诊断时无显著差异。在进展时，A 组婴儿年龄更小（7.3 个月与 14.4 个月，P=0.001），从诊断到进展的时间间隔更短（3.8 个月与 9.6 个月，P=0.001）。B 组婴儿的进展后生存情况较差（45%与 69%，P=0.058），与诊断时年龄小于 2 个月（P=0.005）和肾上腺原发肿瘤部位（P=0.008）相关。随着研究时间的推移，两组的生存率均有所提高。
进展为 4 期的婴儿预后较差，可能与进展时年龄较大和从诊断到进展的时间间隔较长有关。对这些患者进行大型前瞻性研究可能会导致更有效的治疗方法。
