Kushner Brian H, Kramer Kim, LaQuaglia Michael P, Modak Shakeel, Cheung Nai-Kong V
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, New York 10021, USA.
Pediatr Blood Cancer. 2006 Mar;46(3):278-84. doi: 10.1002/pbc.20564.
We reviewed clinical and biologic findings in a series of infants with neuroblastoma (NB) in liver. The aim was to gain insights into improving therapy.
Among 19 newly or recently diagnosed infants with NB in liver, 1987-2002, those with stage 4 involving bone received chemotherapy, while those without bone or extensive bone marrow (BM) involvement were observed or received limited treatment if NB caused life-threatening symptoms. We assessed results in the context of NB treatment risk stratification, which is based on age, stage, and selected biologic features (MYCN, ploidy, histology).
Six of eight infants with bone involvement became long-term event-free survivors including 1/2 with MYCN amplification and four who received only 4-6 cycles of chemotherapy; at the end of treatment, four infants had abnormalities in liver +/- the primary site, but these resolved. All 11 infants without bone lesions became long-term survivors with either no cytotoxic therapy or only one cycle of chemotherapy (+/- radiotherapy to liver), including four who had stage 4 and one stage 4S patient who still had NB in BM at age 15 months.
Treatment reduction should be considered for subsets of infants with non-MYCN-amplified widespread NB: stage 4 without bone or extensive BM involvement may not require cytotoxic therapy, stage 4S with symptomatic hepatomegaly may not require multiple cycles of chemotherapy, and classic stage 4 may do well with limited chemotherapy. Persistent liver abnormalities post-treatment may not require continued therapy to achieve a radiologic complete remission.
我们回顾了一系列肝脏神经母细胞瘤（NB）婴儿的临床和生物学发现。目的是深入了解如何改进治疗方法。
在1987年至2002年间新诊断或近期诊断的19例肝脏NB婴儿中，4期累及骨骼的患儿接受化疗，而无骨骼或广泛骨髓（BM）受累的患儿，如果NB引起危及生命的症状，则进行观察或接受有限治疗。我们在基于年龄、分期和选定生物学特征（MYCN、倍性、组织学）的NB治疗风险分层背景下评估结果。
8例有骨骼受累的婴儿中有6例成为长期无事件幸存者，其中包括1/2例MYCN扩增患儿和4例仅接受4 - 6周期化疗的患儿；治疗结束时，4例婴儿肝脏及/或原发部位有异常，但这些异常均已消退。所有11例无骨骼病变的婴儿均成为长期幸存者，他们要么未接受细胞毒性治疗，要么仅接受一个周期的化疗（肝脏±放疗），其中包括4例4期患儿和1例4S期患儿，该4S期患儿在15个月时骨髓中仍有NB。
对于非MYCN扩增的广泛NB婴儿亚组，应考虑减少治疗：4期无骨骼或广泛BM受累可能不需要细胞毒性治疗，4S期有症状性肝肿大可能不需要多个周期的化疗，经典4期采用有限化疗可能效果良好。治疗后持续存在的肝脏异常可能不需要继续治疗以实现影像学完全缓解。
