Faculty of Medicine, Dalhousie Medical School, Dalhousie University, Halifax, Canada.
Department of Biostatistics, University of Florida, Gainesville, Florida.
Pediatr Blood Cancer. 2019 Jan;66(1):e27430. doi: 10.1002/pbc.27430. Epub 2018 Aug 30.
Wilms tumor (WT) is the most common renal tumor in children. We describe the outcomes for patients with WT that metastasized to bone (WTBM) to assist in decision making for these uncommon patients.
We retrospectively reviewed the research records of patients identified with WTBM from the National Wilms Tumor Study (NWTS 1-5) database. We then related overall survival (OS) to histology, chemotherapy, radiation therapy to bone, location of metastasis, and when bone metastasis presented.
Thirty-eight of 8609 patients enrolled on NWTS 1-5 (0.44%) developed bone metastasis. Bone metastasis most commonly first occurred at progression or relapse (29/38, 76%). Five of thirty-eight survived (13%) with the 5-year OS following presentation of bone metastasis of 14.3% (95% CI: 2.7-25.8%). The primary cause of death was tumor (29/33, 88%). Of those who died, the median survival time was 10.9 months (range 0.49-61.4). Four of nine (44%) patients presenting at diagnosis and 3% (1/29) of patients presenting in progression or relapse survived (P = 0.0075). Nineteen percent (5/26) of patients with favorable histology and 0% (0/12) with anaplastic histology survived (P = 0.16). Of the five survivors, median follow-up was 14 years (range 6.7-23.8). Radiation to metastatic bone sites was recorded in three of five survivors. No consistent chemotherapeutic approach appeared to be associated with disease outcome.
Bone metastasis is rare in patients with WT, occurring more commonly in progression or relapse than at initial diagnosis. Patients with WTBM have poor prognosis. We could not identify a consistent chemotherapeutic strategy associated with survival.
Wilms 瘤（WT）是儿童中最常见的肾肿瘤。我们描述了发生骨转移的 WT 患者（WTBM）的结局，以帮助这些罕见患者做出决策。
我们回顾性地审查了国家 Wilms 肿瘤研究（NWTS 1-5）数据库中确定的 WTBM 患者的研究记录。然后，我们将总生存（OS）与组织学、化疗、骨放疗、转移部位以及骨转移发生的时间相关联。
NWTS 1-5 中登记的 8609 例患者中有 38 例（0.44%）发生骨转移。骨转移最常发生在进展或复发时（29/38，76%）。38 例中有 5 例（13%）存活，骨转移后 5 年 OS 为 14.3%（95%CI：2.7-25.8%）。死亡的主要原因是肿瘤（29/33，88%）。死亡者的中位生存时间为 10.9 个月（范围 0.49-61.4）。4 例（44%）在诊断时出现转移，29 例（3%）在进展或复发时出现转移的患者中存活 1 例（P=0.0075）。组织学表现良好的患者中有 19%（5/26）存活，组织学表现为间变性的患者中无存活者（0%，0/12）（P=0.16）。5 例幸存者中，中位随访时间为 14 年（范围 6.7-23.8）。在 3 例幸存者中记录了对转移骨部位的放疗。没有一致的化疗方法似乎与疾病结局相关。
WT 患者中骨转移罕见，在进展或复发时比初始诊断时更常见。WTBM 患者预后差。我们无法确定与生存相关的一致化疗策略。
