JCK-AP session 4 (II-JCKAP4)
Fontan

[II-JCKAP4-5] Phase 2 Open-label, Single-arm, Multi-center Clinical Trial of Camostate Mesylate in Patients with Protein-losing Enteropathy after Fontan Operation

^○Woo Young Park¹, Gi Beom Kim¹, Sang Yun Lee¹, Jae Suk Baek², Soo Jin Kim³, Jowon Jung⁴, Myung Chul Hyun⁵, Young Tae Lim⁵, Joowon Lee⁶ (1.Department of Pediatrics, Seoul National University Children's Hospital, Seoul, 2.Department of Pediatrics, Asan Medical Center Children's Hospital, Ulsan University College of Medicine, Seoul, 3.Department of Pediatrics, Sejong General Hospital, Bucheon, 4.Department of Pediatrics, Yonsei University College of Medicine, Seoul, 5.Department of Pediatrics, Kyungpook National University Hospital, Daegu, 6.Department of Pediatrics, Seoul National University Bundang Hospital, Seongnam)

Keywords:Camostat mesylate, protein-losing enteropathy, Fontan operation

ObjectiveThis study evaluates the efficacy and safety of Camostat Mesylate in treating protein-losing enteropathy (PLE) in patients post-Fontan operation.MethodsThis phase 2, multicenter, open-label, single-arm trial included patients over 4 years old diagnosed with PLE following a Fontan operation. Camostat Mesylate was added to existing treatments, with follow-up assessments at 1, 3, and 6 months, and a final evaluation one month after discontinuation. Efficacy was measured by changes in serum albumin, stool alpha-1 antitrypsin levels, and PLE symptoms such as diarrhea, edema, weight changes, and ascites.ResultsNineteen patients (average age 17.6 years; 11 males and 8 females) were enrolled. The median time between the Fontan operation and PLE diagnosis was 2.4 years. Eleven patients completed the 6-month follow-up. Serum albumin levels increased from 2.2 to 2.5 g/dL (p=0.461), and stool alpha-1 antitrypsin levels decreased significantly from 266.7 to 44.9 mg/dL (p=0.035). Particularly, patients with diarrhea at baseline showed substantial improvement in both parameters (serum albumin from 2.2 to 2.8, and stool alpha-1 antitrypsin levels from 266.7 to 27.3). No serious adverse events were reported.ConclusionsCamostat Mesylate demonstrated safety and efficacy, increasing serum albumin levels and reducing stool alpha-1 antitrypsin in PLE patients post-Fontan, especially those with diarrhea. Therefore, Camostat Mesylate could be considered as an additional treatment option for patients with PLE following Fontan operation.