Background and study aims: Endoscopic ultrasound (EUS)-guided ethanol lavage with paclitaxel injection has been
shown to be effective for the treatment of pancreatic cystic neoplasms; however, the
evidence for effectiveness is based primarily on cyst resolution on imaging. The aim
of this study was to evaluate changes in pancreatic cyst fluid DNA following EUS-guided
pancreatic cyst ablation (PCA) with ethanol and paclitaxel.
Patients and methods: In a single-center, prospective study, patients with suspected benign pancreatic
cysts (15 – 50 mm in diameter; ≤ 5 compartments) underwent EUS-PCA with ethanol and
paclitaxel followed 3 months later by repeat EUS-FNA, cyst aspiration for repeat DNA
analysis, and possible repeat EUS-PCA. Abdominal imaging was repeated 3 – 4 months
and 12 months after the second EUS. Changes in baseline pancreatic cyst fluid DNA,
procedural complications, and radiographic changes in cyst volume were evaluated.
Results: A total of 22 patients (median age 67 years; 15 women) with cysts in the head or
uncinate (n = 10), body or neck (n = 8), and tail (n = 4), measuring a median diameter
of 25 mm (range 15 – 43 mm), underwent one (n = 22) or two (n = 9) EUS-PCA procedures.
Baseline cyst DNA included mutations in 11 patients (50 %). Postablation cyst fluid
(n = 19) showed elimination of all baseline mutations in eight patients, new mutations
in three, and no changes in eight without a baseline mutation. The largest per-protocol
postablation image-defined volume change (n = 20) from either of the follow-up abdominal
imaging studies (n = 20) demonstrated complete response ( < 5 % original volume) in
10 patients (50 %), partial response (5 % – 25 % original volume) in 5 (25 %), and
a persistent cyst (> 25 % original volume) in 5 (25 %). During a median follow-up
of 27 months (range 17 – 42 months), adverse events from all EUS-PCAs (n = 31) included
abdominal pain alone in four patients (13 %), pancreatitis in three (10 %), peritonitis
in one (3 %), and gastric wall cyst in one (3 %). The adverse events were classified
as moderately severe in four patients (three with pancreatitis, one with peritonitis).
Conclusion: EUS-PCA with ethanol and paclitaxel may possibly eliminate mutant DNA in neoplastic
pancreatic cysts. This technique leads to complete or partial image-defined resolution
in 75 % of cysts but may lead to rare adverse events.
Clinical trial registration: ClinicalTrials.gov (NCT01643460)