Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts (CHARM) Trial: Initial Proof-of-concept Index Case for this Prospective, Randomized, Double-blinded, Single-center Study

Abstract

Purpose: An initial report of the safety and feasibility of the recently opened CHARM trial, evaluating EUS-guided fine-needle infusion (EUS-FNI) of a chemotherapeutic cocktail, following ethanol or normal saline lavage, for mucinous pancreatic cyst ablation. We hypothesize that: 1) EUS-guided lavage of these premalignant pancreatic cysts with normal saline (rather than ethanol) will result in similar efficacy and a lower rate of complications; 2) EUS-FNI of a combination of paclitaxel and gemcitabine will be safe and more effective than previously used ablative agents (paclitaxel or ethanol alone) for the elimination of such lesions. Methods: Adult subjects with mucinous or indeterminate pancreatic cysts who meet inclusion criteria are randomized to undergo single-session EUS-guided lavage of the cyst with 80% ethanol followed by EUS-FNI of a chemotherapeutic cocktail of 3 mg/mL of paclitaxel and 19 mg/mL of gemcitabine (control group) or alternatively, EUS-guided normal saline lavage followed by EUS-FNI of the same chemotherapeutic agents (study group). Patients are then monitored for procedure-related complications, and the success of cyst ablation is assessed by follow-up CT or MRI at 3, 6, and 12 months post-procedure. We aim to enroll 78 subjects (39 in each arm) over a period of 4 years. Results: EUS-guided lavage of a pancreatic mucinous cyst followed by EUS-FNI chemoablation has been successfully performed in the initial patient enrolled in the CHARM trial (78-year-old male with coronary artery disease). The procedure required 21 minutes, and the patient developed no complications. A follow-up CT 3 months post-procedure showed a marked reduction of the lesion from 21 mm to 8 mm (Figure).Figure: [237]Conclusion: Ablation of mucinous pancreatic cysts using EUS-FNI with a chemotherapeutic cocktail of paclitaxel and gemcitabine (with or without ethanol lavage) may represent a safe and effective option in selected cases for elimination of these lesions. Furthermore, management of pancreatic mucinous cysts using EUS-FNI in selected cases offers a minimally invasive alternative to surgery. This initial case of the CHARM trial suggests that EUS-FNI ablation of pancreatic mucinous or indeterminate cysts using a combination of paclitaxel and gemcitabine is feasible and safe. Progress report of this trial will be presented at future meetings.