Long-term follow-up shows durability of chemotherapy for ablation and resolution of mucinous pancreatic cysts
A decade ago, a team of researchers at Penn State Health Milton S. Hershey Medical Center began developing a safer and minimally invasive approach to treating mucinous (precancerous) pancreatic cystic lesions. About 2% of all Americans have these pre-cancerous cysts, which lead to 15% to 20% of all cases of pancreatic cancer. Endoscopic ultrasound (EUS)-guided pancreatic cyst ablation is an innovative and emerging technique of eliminating mucinous pancreatic cysts without the need for surgery in appropriately selected patients.
Results of Penn State Health’s Chemotherapy for Ablation and Resolution of Mucinous pancreatic cysts (CHARM) trial, published in August 2017, showed that using an innovative cocktail of paclitaxel, gemcitabine and alcohol-free saline essentially eliminated associated adverse events when compared with previously used, alcohol-based ablation protocols (which carried a 3% to 10% risk of serious adverse events).¹ This significantly improved the safety of the procedure, paving the way for broader adoption of this approach internationally.
The CHARM study sparked a second multicenter clinical trial, CHARM II, funded by the National Institutes of Health.² While CHARM II will continue through 2023, in the interim, researchers at the Milton S. Hershey Medical Center wanted to evaluate the long-term durability of this innovative, alcohol free chemoablation treatment.
“With surgery, we know a pre-cancerous cyst is gone, but with ablation, we didn’t have objective evidence on whether the cysts are completely eradicated without long-term follow-up analysis,” said gastroenterologist Dr. Matthew T. Moyer with Hershey Medical Center.
To conduct that analysis, Moyer and his research team recorded the baseline, 12-month and long-term follow-up radiology images for 36 of the 39 patients treated in the initial CHARM trial. The results, published in April 2021 in Clinical Gastroenterology and Hepatology, proved the durability of the new treatment. A total of 20 of the 23 complete responders (87%) treated with EUS-guided chemoablation maintained resolution at three to five years of follow-up. The results also suggested the possibility of protracted treatment effects because 31% of patients who did not achieve complete response initially continued toward resolution.³
“Patients who received a complete response stayed in complete response long term, and not a single patient — whether or not they received a complete response initially — developed any worrisome tumors or high-grade pathology long term,” Moyer said.
Moyer hopes the results lead to more widespread acceptance of this alcohol-free, EUS-guided chemoablation protocol. “We’re essentially taking the concept of colonoscopy for cancer screening and prevention and applying it to pancreatic cancer,” Moyer said. “Eliminating these cystic tumors from progressing to pancreatic cancer through chemoablation is minimally invasive, cost effective and tremendously more effective than waiting for cancer to develop.”
Members of the multidisciplinary Liver, Pancreas and Foregut Committee at Penn State Cancer Institute currently use the alcohol-free ablation protocol in clinical practice for appropriately selected patients. “It’s an exciting, minimally invasive treatment option for patients who have pre-cancerous cysts that are large enough to be worrisome, but haven’t yet progressed to have overt signs of pancreatic cancer.” Other centers have reached out to Moyer and his team to learn how to set up a programs at their institutions.
Moyer’s co-authors on the durability study were Dr. Abraham Mathew, Dr. John Levenick, certified physician assistant Brandon Headlee, Dr. Jennifer Maranki and Dr. Charles Dye, all from the Division of Gastroenterology and Hepatology; Dr. Leonard Walsh and Dr. Kayla Hartz with the Division of General Internal Medicine at the Medical Center; Heather Heisey, Penn State Investigational Drug Services Pharmacy; Dr. James Birkholz, Department of Radiology; and Dr. Matthew Dixon with the Division of Surgical Oncology.
“I’d also like to recognize Dr. Christopher Sciamanna of the Division of General Internal Medicine, who helped us write and successfully obtain NIH funding for the CHARM II trial; Dr. John Dewitt of the Indiana University School of Medicine; our original research coordinator, Brooke Ancrile, PhD; and several others who deserve recognition for making the CHARM series of research trials so successful.”
Matthew T. Moyer, MD, MS
Professor, Department of Medicine, Division of Gastroenterology and Hepatology, Penn State Cancer Institute
Fellowship: Gastroenterology, Penn State Health Milton S. Hershey Medical Center, Hershey, Pa.
Residency: Internal Medicine, Penn State Health Milton S. Hershey Medical Center, Hershey, Pa.
Medical School: Penn State College of Medicine, Hershey, Pa.
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- Moyer MT, Sharzehi S, Mathew A, et al. The Safety and Efficacy of an Alcohol-Free Pancreatic Cyst Ablation Protocol. Gastroenterology. 2017;153(5):1295-1303. doi:10.1053/j.gastro.2017.08.009
- CHARM II: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts (CHARM). National Institutes of Health U.S. National Library of Medicine ClinicalTrials.gov website. https://clinicaltrials.gov/ct2/show/NCT03085004. Updated March 26, 2021. Accessed July 16, 2021.
- Lester C, Walsh L, Hartz KM, et al. The Durability of EUS-Guided Chemoablation of Mucinous Pancreatic Cysts: A Long-Term Follow-Up of the CHARM trial [published online ahead of print, 2021 Apr 1]. Clin Gastroenterol Hepatol. 2021;S1542-3565(21)00351-7. doi:10.1016/j.cgh.2021.03.041