Fuchs Juri, Murtha-Lemekhova Anastasia, Kessler Markus, Günther Patrick, Fichtner Alexander, Pfeiffenberger Jan, Probst Pascal, Hoffmann Katrin
Department of General, Visceral and Transplantation Surgery, University Hospital Heidelberg, Heidelberg, Germany.
Department of General, Visceral and Transplantation Surgery, Division of Pediatric Surgery, University Hospital Heidelberg, Heidelberg, Germany.
Front Oncol. 2021 Sep 30;11:701400. doi: 10.3389/fonc.2021.701400. eCollection 2021.
The biliary tree is a rare location of pediatric rhabdomyosarcoma. Due to the low incidence, there is a lack of evidence concerning therapeutic guidelines for this tumor location. In particular, the impact of surgery is discussed controversially.
Objective is to generate evidence-based treatment guidelines for pediatric biliary rhabdomyosarcoma (BRMS). All available published data on therapeutic regimens and important prognostic factors are investigated with a focus on the role of surgery.
A systematic literature search of MEDLINE, Web of Science, and CENTRAL was performed. Patient data were entered individually. Data was pooled and qualitative and quantitative analyses of demographic data, therapy, postoperative/interventional outcomes, relapse, and survival were conducted. In an individual patient data analysis, cox regression was applied to identify key factors predicting the outcome of patients with BRMS.
65 studies met the inclusion criteria, providing data on 176 patients with BRMS. Individual patient data analysis showed a 5-year overall survival and progression-free survival of 51% and 50% for the total study population. For patients treated after 2000, 5-year OS and PFS was 65% and 59%, respectively. Absence of surgical tumor resection was an independent risk factor for death (Hazard ratio 8.9, 95%-CI 1.8-43.6, = 0.007) and significantly associated with recurrent disease and disease-related death.
This analysis provides comprehensive information on the largest number of patients hitherto reported in the literature. BRMS is still associated with high morbidity and mortality. Surgical tumor resection is essential for appropriate oncological treatment of BRMS. International cooperation studies are needed to enhance evidence and improve the outcome of this orphan disease.
PROSPERO (CRD42021228911) https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42021228911.
胆管树是小儿横纹肌肉瘤的罕见发病部位。由于发病率低，缺乏关于该肿瘤部位治疗指南的证据。特别是，手术的影响存在争议。
目的是为小儿胆管横纹肌肉瘤（BRMS）制定基于证据的治疗指南。研究所有已发表的关于治疗方案和重要预后因素的可用数据，重点关注手术的作用。
对MEDLINE、科学网和CENTRAL进行系统文献检索。分别输入患者数据。汇总数据并对人口统计学数据、治疗、术后/介入结果、复发和生存进行定性和定量分析。在个体患者数据分析中，应用cox回归确定预测BRMS患者预后的关键因素。
65项研究符合纳入标准，提供了176例BRMS患者的数据。个体患者数据分析显示，整个研究人群的5年总生存率和无进展生存率分别为51%和50%。对于2000年后接受治疗的患者，5年总生存率和无进展生存率分别为65%和59%。未进行手术肿瘤切除是死亡的独立危险因素（风险比8.9，95%置信区间1.8 - 43.6，P = 0.007），并与疾病复发和疾病相关死亡显著相关。
该分析提供了文献中迄今报道的最大数量患者的全面信息。BRMS仍与高发病率和死亡率相关。手术肿瘤切除对于BRMS的适当肿瘤治疗至关重要。需要开展国际合作研究以加强证据并改善这种罕见病的治疗结果。
PROSPERO（CRD42021228911）https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42021228911
