Long-term outcomes in patients with stage IV neuroblastoma
Long-term outcomes in patients with stage IV neuroblastoma
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DOI：
10.1016/j.jpedsurg.2005.11.032
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发表时间：
2006-02-01
影响因子：
2.4
通讯作者：
Rescorla, FJ
中科院分区：
文献类型：
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作者：
Escobar, MA;Grosfeld, JL;Rescorla, FJ
研究目的：IV期神经母细胞瘤(NB)幸存者的长期预后研究很少。这篇综述评估了IV期NB幸存者的远期并发症和长期结果。主要结果：在153例IV期NB患者中，52例(34%)存活(男性-女性，26：26)。幸存者确诊时的年龄为29.1+/-31.7个月。18例小于或等于1岁，34例大于1岁，而死亡10例小于等于1岁，91例大于1岁(P=.0003，Fisher‘s精确检验)。原发灶位于肾上腺(35例)、腹膜后(11例)、纵隔(3例)、盆腔(2例)，未发现原发肿瘤转移(1例)。存活的10例组织学良好，16例组织学不佳，而死亡的患者中1例良好，18例无效(P=0.01)。4名幸存者有MYCN扩增(=10拷贝)，以及11)和11q的2个缺失。幸存者和死亡者的转移部位相似。存活患者接受手术治疗51例(75%[39/51])，化疗50例，放射治疗17例，干细胞移植20例，骨髓移植1例。6例肿瘤复发但仍存活(平均9.3+/-8.3年；范围6个月~24年)。复发包括局部复发(1例)、远处复发(2例)和两者均复发(3例)，并接受了切除、化疗和放射治疗。存活年龄2~34岁，平均12.4±8.3岁。在所有IV期病例中，无事件存活率为30%，总存活率为34%。远期并发症23例(44%)，包括内分泌紊乱(7例)、骨科(5例)、白内障(2例)、粘连性肠梗阻(2例)、高血压(1例)、毛细支气管炎(1例)、失明(1例)、周围神经病变(1例)、肾功能不全(1例)、胆结石(1例)、甲状腺结节(1例)。年龄小于1岁、良好的病理、CTR和无复发是唯一具有统计学意义的有利于生存的因素。44%的幸存者经历了晚期发病率，52人中有6人(11.5%)肿瘤复发。应该监测患者的肿瘤复发和长期后遗症。需要新的治疗方法才能取得更好的结果。(C)2006 Elsevier Inc.保留所有权利。
Aim of Study: Long-term outcome Studies in survivors with stage IV neuroblastoma (NB) are sparse. This review evaluates late complications and long-term outcomes in stage IV NB survivors.Methods: A retrospective review of stage IV NB survivors. was performed to analyze outcomes, including long-term morbidity, recurrence, and survival.Main Results: Of 153 patients with stage IV NB, 52 (34%) survived (male-female, 26:26). Age at diagnosis was 29.1 +/- 3 1.7 months in Survivors. Eighteen were 1 year or Younger and 34 were older than 1 year compared with 10 nonsurvivors 1 year or younger and 91 older than 1 year (P = .0003, Fisher's Exact test). Primary turner Sites were adrenal (35), retroperitoneal (11), mediastinal (3), pelvic (2), and no primary with tumor metastases identified (1). Ten survivors had favorable and 16 had unfavorable histology compared with 1 favorable and 18 unfavorable in nonsurvivors (P = .01). Four survivors had MYCN amplification (>= 10 copies) and 2 deletions of 11) and 11q. Sites of metastasis in survivors and nonsurvivors Were similar. Treatment in Survivors included surgery in 51 (75% [39/51] complete tumor resection [CTR]); chemotherapy, 50; radiation, 17; stern cell transplantation, 20; and bone marrow transplant, 1. In nonsurvivors, 13 (25%) of 53 (P < .0001) had CTR, 18 stern cell transplantation, and 12 bone marrow transplant. Six patients had tumor recurrence but survived (mean, 9.3 +/- 8.3 years; range, 6 months-24 years). Recurrence was local (1), distant (2), and both (3) and was treated by resection, chemotherapy, and radiation. The mean age of survivors was 12.4 +/- 8.3 years (range, 2-34 years). In all stage IV cases, event-free survival was 30% and overall Survival was 34%. Long-term complications occurred in 23 (44%) survivors, including endocrine disturbances (7), orthopedic (5), cataracts (2), adhesive bowel obstruction (2), hypertension (1), bronchiolitis (1), blindness (1), peripheral neuropathy (1), nonfunctioning kidney (1), cholelithiasis (1), and thyroid nodule (1).Conclusion: Only 34% of patients with stage IV NB survived despite aggressive multimodal therapy. Age of younger than I year, favorable pathology, CTR, and no recurrence were the only statistically significant factors that favored survival. Forty-four percent of survivors experienced late morbidity, and tumor recurred in 6 (11.5%) of 52. Patients should be monitored for tumor recurrence and long-term sequelae. New methods of treatment are required to achieve better outcomes. (c) 2006 Elsevier Inc. All rights reserved.