Bazarbashi Shouki, Aseafan Mohamed, Elshenawy Mahmoud, Alzahrani Ahmed, Aljubran Ali H, Almugbel Fahad, Alzannan Noura, Elhassan Tusneem
Oncology, Cancer Centre of Excellence, King Faisal Specialist Hospital and Research Centre, Riyadh, SAU.
Medical Oncology, Security Forces Hospital Program, Riyadh, SAU.
Cureus. 2024 Jan 21;16(1):e52656. doi: 10.7759/cureus.52656. eCollection 2024 Jan.
Biliary tract cancers (BTCs), characterized by poor prognosis and limited treatment options, are increasingly prevalent malignancies with a five-year survival rate of less than 20% for advanced-stage disease. The standard first-line chemotherapy combining gemcitabine and cisplatin offers modest survival benefits, necessitating the exploration of more effective therapies. This study reports the results of a single-arm, open-label, phase 2 trial assessing the efficacy and safety of fluorouracil, leucovorin, oxaliplatin, and irinotecan (FOLFIRINOX) as a first-line treatment for metastatic or locally advanced unresectable BTC.
Patients aged ≥18 with measurable disease and adequate organ function were enrolled, receiving biweekly FOLFIRINOX for up to 12 cycles with follow-up imaging every four cycles. The primary endpoint was the overall response rate (ORR), with progression-free survival (PFS), overall survival (OS), and safety as secondary endpoints.
Thirteen patients were enrolled from December 2016 to September 2021 before early termination due to slow accrual and the emergence of immunotherapy. The ORR was 54%, with a disease control rate of 77%. Median PFS and OS were 6.8 and 19.25 months, respectively. Grade 3/4 toxicities were predominantly hematologic, with neutropenia being the most common severe adverse event.
The trial suggests that FOLFIRINOX is a potentially effective first-line therapy for unresectable or metastatic BTC with a manageable safety profile. However, the early termination of the study and the introduction of immunotherapy warrant further research to confirm these findings.
胆道癌（BTC）预后较差且治疗选择有限，是一种日益常见的恶性肿瘤，晚期疾病的五年生存率低于20%。吉西他滨和顺铂联合的标准一线化疗仅带来适度的生存获益，因此需要探索更有效的治疗方法。本研究报告了一项单臂、开放标签的2期试验结果，评估氟尿嘧啶、亚叶酸钙、奥沙利铂和伊立替康（FOLFIRINOX）作为转移性或局部晚期不可切除BTC一线治疗的疗效和安全性。
纳入年龄≥18岁、具有可测量病灶且器官功能良好的患者，每两周接受一次FOLFIRINOX治疗，最多12个周期，每四个周期进行一次随访影像学检查。主要终点为总缓解率（ORR），无进展生存期（PFS）、总生存期（OS）和安全性为次要终点。
2016年12月至2021年9月共纳入13例患者，后因入组缓慢和免疫治疗的出现而提前终止试验。ORR为54%，疾病控制率为77%。中位PFS和OS分别为6.8个月和19.25个月。3/4级毒性主要为血液学毒性，中性粒细胞减少是最常见的严重不良事件。
该试验表明，FOLFIRINOX对于不可切除或转移性BTC是一种潜在有效的一线治疗方法，安全性可控。然而，研究的提前终止和免疫治疗的引入需要进一步研究以证实这些发现。
