探索现实生活中胰腺导管腺癌化疗的外部有效性。

IF 4 3区医学 Q1 GASTROENTEROLOGY & HEPATOLOGY

Digestive and Liver Disease Pub Date : 2025-01-01 DOI:10.1016/j.dld.2024.06.025

Michele Reni , Guido Giordano , Marco Audisio , Giulia Orsi , Marina Macchini , Stefania Maria Gobba , Ilario Rapposelli , Antonio Lucenti , Giovanna Luchena , Luca Faloppi , Fable Zustovich , Vincenzo Ricci , Massimiliano Cergnul , Vincenzo Formica , Letizia Procaccio , Valeria Baccolini , Assunta Briccolani , Stefano Cascinu , Umberto Peretti

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引用次数: 0

摘要

简介：一项针对胰腺导管腺癌（PDAC）的单机构 II 期试验（PACT-19）评估了顺铂、纳布-紫杉醇、卡培他滨和吉西他滨（PAXG）方案的活性。PACT-31研究探讨了PACT-19结果的外部有效性：年龄≥18岁和≤75岁、KPS≥70、确诊为PDAC且在参与机构接受PAXG治疗的患者均符合条件，并按以下方式分类：A）PACT-19；B）PACT-31-HSR；C）PACT-31-non-HSR。假设转移性患者的目标 1 年总生存率为 60%，非转移性患者的目标 1 年总生存率为 80%，则 1 年 OS 达到 95% CI，试验即为成功：检索了 68 名 PACT-19 和 168 名 PACT-31 患者的数据。124 例事件后，转移性患者的 1 年生存率为 52.5%（95 %CI：44.6-60.4%），非转移性患者的 1 年生存率为 80.5%（95 %CI：71.9-89.1%）。PACT-19 和 PACT-31-HSR 的生存期重叠（中位数分别为 17.6 个月和 17.4 个月，p = 0.21），而 PACT-31-non-HSR 的生存期明显较短（中位数为 11.3 个月；p = 0.03）。PACT-31-HSR与非HSR在剂量强度、维持治疗的使用以及进展后的治疗方面存在差异：讨论：PACT-19 结果具有外部有效性。HSR中心和非HSR中心之间的结果差异表明，有必要建立一个旨在共享罕见病专业知识的中心辐射网络。

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Exploring external validity of chemotherapy for pancreatic ductal adenocarcinoma in real life

Introduction

Cisplatin, nab-paclitaxel, capecitabine, and gemcitabine (PAXG) regimen activity was assessed in a single institution phase II trial (PACT-19) on pancreatic ductal adenocarcinoma (PDAC). The PACT-31 study explored the external validity of PACT-19 results.

Materials and Methods

Patients aged ≥18 and ≤75 years with KPS ≥70, and PDAC diagnosis receiving PAXG in the participating institutions were eligible and categorized as follows: A) PACT-19; B) PACT-31-HSR; C) PACT-31-non-HSR. With a sample of 175 patients, assuming a target 1-year overall survival of 60 % for metastatic and of 80 % for non-metastatic patients, the trial will be considered successful with the 1-year OS falling into the 95 % CI.

Results

Data from 68 PACT-19 and 168 PACT-31 patients were retrieved. After 124 events, 1yOS was 52.5 % (95 %CI: 44.6–60.4 %) for metastatic and 80.5 % (95 %CI: 71.9–89.1 %) for non-metastatic patients. Survival overlapped between PACT-19 and PACT-31-HSR (median 17.6 and 17.4 months, p = 0.21) and was significantly shorter in PACT-31-non-HSR (median 11.3 months; p = 0.03). Differences of dose-intensity, use of maintenance therapy, and treatment after progression between PACT-31-HSR and non-HSR were evidenced.

Discussion

PACT-19 results have external validity. The outcome difference between HSR and non-HSR centers endorses the need of creating a hub-and-spoke network aimed at sharing the expertise on rare-diseases.

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来源期刊

Digestive and Liver Disease 医学-胃肠肝病学

CiteScore

6.10

自引率

2.20%

发文量

632

审稿时长

19 days

期刊介绍： Digestive and Liver Disease is an international journal of Gastroenterology and Hepatology. It is the official journal of Italian Association for the Study of the Liver (AISF); Italian Association for the Study of the Pancreas (AISP); Italian Association for Digestive Endoscopy (SIED); Italian Association for Hospital Gastroenterologists and Digestive Endoscopists (AIGO); Italian Society of Gastroenterology (SIGE); Italian Society of Pediatric Gastroenterology and Hepatology (SIGENP) and Italian Group for the Study of Inflammatory Bowel Disease (IG-IBD). Digestive and Liver Disease publishes papers on basic and clinical research in the field of gastroenterology and hepatology. Contributions consist of: Original Papers Correspondence to the Editor Editorials, Reviews and Special Articles Progress Reports Image of the Month Congress Proceedings Symposia and Mini-symposia.