Department of Pediatric Oncology/Hematology, Erasmus MC-Sophia Children's Hospital, Rotterdam, The Netherlands.
Pediatr Blood Cancer. 2011 May;56(5):733-7. doi: 10.1002/pbc.22922. Epub 2010 Dec 22.
Survival data of malignant rhabdoid tumour of the kidney (MRTK) registered in SIOP trials, advocating preoperative chemotherapy, are not available.
To evaluate characteristics, response and survival of MRTK patients registered in recent SIOP protocols.
An evaluation of all MRTK patients treated from 1993 to 2005 (SIOP trials 93-01 and 2001) was performed. Data were obtained from study specific case record forms and entered centrally in a database.
Hundred and seven patients were identified (57 male), with a median age at diagnosis of 13 months (interquartile range 6-27 months), and a median follow-up time of 60 months. Left and right kidneys were equally affected. Tumour stage distribution was stage I (6%), stage II (22%), stage III (43%), stage IV (22%) and stage V (3%). Stage IV patients included 17 with pulmonary metastasis (8 lung-only) and 12 with multiple organ metastases (bone, brain and liver). Primary surgery was the upfront treatment approach in 22/107 patients (21%), by which 19 patients reached a complete remission (CR). Median difference in tumour volume before and after preoperative chemotherapy was 69 ml (interquartile range: 4.5-158.0, P < 0.0001), indicating marked chemosensitivity. The 5-year event-free survival (EFS) of the total group was 22% (95% CI: 15-33) and overall survival 26% (95% CI: 18-37). Most events (86%) occurred within the first 2 years after diagnosis. Younger age at diagnosis was an important adverse prognostic factors for survival. In contrast, tumour volume at diagnosis, nor volume reduction was associated with outcome.
MRTK has a poor outcome especially in young and advanced-stage disease patients. Neither tumour volume at diagnosis, nor pre-operative chemosensitivity are prognostic factors for survival.
在 SIOP 试验中，登记的恶性横纹肌样瘤（MRTK）的生存数据提倡术前化疗，但目前尚无此类数据。
评估最近 SIOP 方案中登记的 MRTK 患者的特征、反应和生存情况。
对 1993 年至 2005 年（SIOP 试验 93-01 和 2001 年）期间治疗的所有 MRTK 患者进行评估。数据来自特定的研究病例记录，并集中输入数据库。
共确定了 107 例患者（57 例男性），中位诊断年龄为 13 个月（四分位间距 6-27 个月），中位随访时间为 60 个月。左右肾脏均受影响。肿瘤分期分布为 I 期（6%）、II 期（22%）、III 期（43%）、IV 期（22%）和 V 期（3%）。IV 期患者包括 17 例肺转移（8 例仅肺转移）和 12 例多器官转移（骨、脑和肝）。22/107 例（21%）患者直接进行了初始手术，其中 19 例患者达到完全缓解（CR）。术前化疗前后肿瘤体积的中位差异为 69ml（四分位间距：4.5-158.0，P<0.0001），表明化疗敏感性高。总组的 5 年无事件生存率（EFS）为 22%（95%CI：15-33），总生存率为 26%（95%CI：18-37）。大多数事件（86%）发生在诊断后 2 年内。诊断时年龄较小是生存的一个重要不良预后因素。相比之下，诊断时的肿瘤体积以及体积减少均与预后无关。
MRTK 的预后较差，尤其是在年轻和晚期疾病患者中。诊断时的肿瘤体积和术前化疗敏感性均不是生存的预后因素。
