肾恶性横纹肌样瘤的大剂量治疗：无生存改善证据——德国儿科肿瘤与血液学会（GPOH）的经验
High-dose treatment for malignant rhabdoid tumor of the kidney: No evidence for improved survival-The Gesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH) experience.
作者信息
Furtwängler Rhoikos, Kager Leo, Melchior Patrick, Rübe Christian, Ebinger Martin, Nourkami-Tutdibi Nasenien, Niggli Felix, Warmann Steven, Hubertus Jochen, Amman Gabriele, Leuschner Ivo, Vokuhl Christian, Graf Norbert, Frühwald Michael C
机构信息
Department of Pediatric Hematology and Oncology, Saarland University Hospital, Homburg/Saar, Germany.
Department of Pediatrics, St. Anna Children's Hospital, Medical University Vienna, Vienna, Austria.
出版信息
Pediatr Blood Cancer. 2018 Jan;65(1). doi: 10.1002/pbc.26746. Epub 2017 Aug 26.
BACKGROUND
Malignant rhabdoid tumor of the kidney (MRTK) is the most aggressive childhood renal tumor with overall survival (OS) rates ranging from 22% to 42%. Whether high-dose chemotherapy with autologous stem-cell transplantation (HDSCT) in an intensive first-line treatment offers additional benefit is an ongoing discussion.
METHODS
A retrospective analysis of all 58 patients with MRTK from Austria, Switzerland, and Germany treated in the framework of consecutive, prospective renal/rhabdoid tumor studies SIOP9/GPO, SIOP93-01/GPOH (where SIOP is International Society of Pediatric Oncology and GPOH is German Society of Pediatric Oncology and Hematology), SIOP2001/GPOH, and European Rhabdoid Tumor Registry from 1991 to 2014.
RESULTS
Median age at diagnosis was 11 months. Fifty percent of patients had metastases or multifocal disease at diagnosis (Stage IV). Local stage distribution was as follows: not done/I/II/III-1/6/11/40. Fifteen (26%) patients underwent upfront surgery. Thirty-seven (64%) patients achieved a complete remission, 17 (29%) relapsed, 34 (59%) died of disease progression, and two (3%) died of treatment-related complication. Mean time to the first event was 3.5 months. Two-year EFS/OS (where EFS is event-free survival) for the whole group was 37 ± 6%/38 ± 6%. Metastases/multifocal disease, younger age, and local stage III were associated with significantly inferior survival. Eleven (19%) patients underwent HDSCT (carboplatin + thiotepa, n = 6; carboplatin + etoposide + melphalan, n = 4; others, n = 1); 2-year OS in this group was 60 ± 15% compared to 34 ± 8% in the non-HDSCT group (P = 0.064). However, the time needed from radiologic to histologic diagnosis, stem-cell harvest, and HDSCT must also be taken into account to avoid selection bias by excluding the highest risk group with early progression (<90 days). Thus, 2-year EFS only for patients without progression until day 90 was 60 ± 16% consolidated by HDSCT compared to 62 ± 11% without (P = 0.8).
CONCLUSION
Our retrospective analysis suggests comparable outcomes for patients with and without HDSCT, if adjusted for early disease progression.
背景
肾恶性横纹肌样瘤（MRTK）是最具侵袭性的儿童肾肿瘤，总生存率（OS）为22%至42%。强化一线治疗中采用高剂量化疗联合自体干细胞移植（HDSCT）是否能带来额外益处仍在讨论中。
方法
对1991年至2014年期间在连续的前瞻性肾/横纹肌样瘤研究SIOP9/GPO、SIOP93 - 01/GPOH（其中SIOP是国际儿科肿瘤学会，GPOH是德国儿科肿瘤与血液学会）、SIOP2001/GPOH以及欧洲横纹肌样瘤登记处框架下接受治疗的来自奥地利、瑞士和德国的58例MRTK患者进行回顾性分析。
结果
诊断时的中位年龄为11个月。50%的患者在诊断时有转移或多灶性疾病（IV期）。局部分期分布如下：未评估/I/II/III - 1/6/11/40。15例（26%）患者接受了 upfront 手术。37例（64%）患者实现完全缓解，17例（29%）复发，34例（59%）死于疾病进展，2例（3%）死于治疗相关并发症。首次事件的平均时间为3.5个月。整个组的两年无事件生存率（EFS）/总生存率（OS）为37 ± 6%/38 ± 6%。转移/多灶性疾病、年龄较小和局部III期与显著较差的生存率相关。11例（19%）患者接受了HDSCT（卡铂 + 噻替派，n = 6；卡铂 + 依托泊苷 + 美法仑，n = 4；其他，n = 1）；该组的两年OS为60 ± 15%，而非HDSCT组为34 ± 8%（P = 0.064）。然而，还必须考虑从影像学诊断到组织学诊断、干细胞采集和HDSCT所需的时间，以避免因排除早期进展（<90天）的最高风险组而产生选择偏倚。因此，仅对于直到第90天无进展的患者，接受HDSCT巩固后的两年EFS为60 ± 16%，未接受的为62 ± 11%（P = 0.8）。
结论
我们的回顾性分析表明，如果对早期疾病进展进行调整，接受和未接受HDSCT的患者预后相当。
Zotero 插件