Safety and Efficacy of Oxaliplatin Pressurized Intraperitoneal Aerosolized Chemotherapy (PIPAC) in Colorectal and Appendiceal Cancer with Peritoneal Metastases - Results of a Multicenter Phase I Trial in the USA

Sep 24, 2026

Uploaded by Mabel

Background: Pressurized intraperitoneal aerosolized chemotherapy (PIPAC) is a laparoscopic locoregional therapy for peritoneal metastases from colorectal or appendiceal cancer in patients unable to undergo cytoreductive surgery. In the USA, efficacy and safety required evaluation. Methods: A multicenter phase I trial assessed 90 mg/m² oxaliplatin alone (cycle 1) and oxaliplatin after systemic fluorouracil plus leucovorin (cycles 2–3). Primary endpoint was treatment-related adverse events; secondary endpoints included survival and response by laparoscopic, histologic, and radiographic criteria. Results: Twelve patients received treatment; completion occurred in half.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages11

Reading Time28 min

AccessFree to download

Abstract

Background

Patients and Methods

Results

Conclusions

Introduction

Clinical context of peritoneal metastases

PIPAC delivery rationale

Prior studies and dosing trials

Ann Surg Oncol (2023) 30:7814–7824 [https://doi.org/10.1245/s10434-023-13941-2](https://doi.org/10.1245/s10434-023-13941-2) ORIGINAL ARTICLE – PERITONEAL SURFACE MALIGNANCY Safety and Efficacy of Oxaliplatin Pressurized Intraperitoneal Aerosolized Chemotherapy (PIPAC) in Colorectal and Appendiceal Cancer with Peritoneal Metastases: Results of a Multicenter Phase I Trial in the USA Mustafa Raoof, MD1, Richard L. Whelan, MD2, Kevin M. Sullivan, MD1, Christopher Ruel, MS3, Paul H. Frankel, PhD3, Sarah E. Cole, PhD4, Raechelle Tinsley, BS, CCRP5, Melissa Eng, CRN6, Marwan Fakih, MD6, Joseph Chao, MD6, Dean Lim, MD6, Yanghee Woo, MD1, Isaac Benjamin Paz, MD1, Michael Lew, MD7, Michaela Cristea, MD6, Lorna Rodriguez‑Rodriguez, MD1, Yuman Fong, MD1, Rebecca Meera Thomas, MD8, Sue Chang, MD9, Danielle Deperalta, MD2, Amit Merchea, MD10, and Thanh H. Dellinger, MD1 1Department of Surgery, City of Hope National Medical Center, Duarte, CA; 2Department of Surgery, Northwell Health, New York, NY; 3Department of Computation and Quantitative Medicine, City of Hope National Medical Center, Duarte, CA; 4Department of Clinical Protocol Development, City of Hope National Medical Center, Duarte, CA; 5Clinical Trials Office, City of Hope National Medical Center, Duarte, CA; 6Department of Medical Oncology and Therapeutics Research, City of Hope National Medical Center, Duarte, CA; 7Department of Anesthesiology, City of Hope National Medical Center, Duarte, CA; 8Department of Pathology, Norwell Health, New York, NY; 9Department of Pathology, City of Hope National Medical Center, Duarte, CA; 10Department of Surgery, Mayo Clinic, Jacksonville, FL ABSTRACT Background. Pressurized intraperitoneal aerosolized chemotherapy (PIPAC) is a laparoscopic locoregional treatment for peritoneal metastases (PM) from colorectal cancer (CRC) or appendiceal cancer (AC) in patients who cannot undergo cytoreductive surgery (CRS) . While PIPAC has been studied in Europe and Asia, it has not been investigated in the USA. © The Author(s) 2023 First Received: 28 April 2023 Accepted: 28 June 2023 Published online: 27 July 2023 M. Raoof, MD[e-mail: mraoof@coh.org](e-mail: mraoof@coh.org) [R. L. Whelan](R. L. Whelan), MD e-mail: [rwhelan1@northwell.edu](rwhelan1@northwell.edu) A. Merchea, MD[e-mail: merchea.amit@mayo.edu](e-mail: merchea.amit@mayo.edu) [T. H. Dellinger](T. H. Dellinger), MD e-mail: [tdellinger@coh.org](tdellinger@coh.org) Patients and Methods. We evaluated PIPAC with 90 mg/ m2 oxaliplatin alone (cycle 1) and preceded by systemic chemotherapy with fluorouracil (5-FU) and leucovorin (LV) (cycle 2–3) as a multicenter prospective phase I clinical trial (NCT04329494) . The primary endpoint was treatment-related adverse events (AEs) . Secondary endpoints included survival and laparoscopic, histologic, and radiographic response. Results. 12 patients were included: 8 with CRC and 4 with AC. Median prior chemotherapy cycles was 2 (interquartile range (IQR) 2–3) . All patients were refractory to systemic oxaliplatin-based chemotherapy. Median peritoneal carcinomatosis index (PCI) was 28 (IQR 19–32) . Six (50%) of twelve patients completed three PIPAC cycles. No surgical complications or dose-limiting toxicities were observed. Two patients developed grade 3 treatment-related toxicities (one abdominal pain and one anemia) . Median overall survival (OS) was 12.0 months, and median progression-free survival (PFS) was 2.9 months. OS was correlated with stable disease by Response Evaluation Criteria in Solid Tumors (RECIST) criteria but not with laparoscopic response by PCI or histologic response by peritoneal regression grading system (PRGS) . Conclusions. This phase I trial in the USA demonstrated safety, feasibility, and early efficacy signal of PIPAC with oxaliplatin and chemotherapy in patients with PM from AC or CRC who are refractory to standard lines of systemic chemotherapy. Peritoneal metastases (PM) from colorectal c

FAQs

What was the primary endpoint in this multicenter phase I trial?

The primary endpoint was treatment-related adverse events (AEs).

How was PIPAC administered across the trial cycles?

Cycle 1 used 90 mg/m² oxaliplatin alone, and cycles 2–3 used oxaliplatin preceded by systemic chemotherapy with fluorouracil (5-FU) and leucovorin (LV).

What did the study report about safety and feasibility?

No surgical complications or dose-limiting toxicities were observed, and two patients developed grade 3 treatment-related toxicities.

Sep 24, 2026

Uploaded by Mabel