Colchicine is an effective treatment for most pediatric patients with complex aphthous stomatitis (CAS), results from a small retrospective study showed.
"Complex aphthous stomatitis in children is typically treated with topical supportive care, which is often not effective," one of the study investigators, Ananya Shah, a third-year medical student at the University of Rochester School of Medicine & Dentistry, Rochester, New York, told this news organization following the Society for Pediatric Dermatology (SPD) 2024 Annual Meeting, where the study was presented during a poster session. "There is limited research on CAS and its treatment in children. Colchicine is often used for treatment of CAS in adults, but its use in children has not been studied."
Shah, in collaboration with Hilary Kunkel, MD, Nessa Aghazadeh, MD, and Megha Tollefson, MD, of the Department of Dermatology, Mayo Clinic, Rochester, Minnesota, retrospectively reviewed the charts of 20 children diagnosed with CAS who were treated with colchicine, an antiinflammatory drug often used to treat gout, at the clinic between 2000 and 2023. Treatment responses were defined as no response, partial response, and complete response. Half of the patients were girls, and their median age at presentation was 5 years.
Ulcers were most commonly located in the buccal mucosa (80%), followed by the gingiva (50%), the mucosal lip (50%), and the palate (40%). Nearly all patients (95%) reported that the CAS caused difficulties with eating or drinking. Other effects on their quality of life included weight loss (35%), bleeding (30%), and difficulty brushing teeth (25%). "I was surprised by how much CAS impacts pediatric patients' quality of life," Shah said. "Almost all of the patients experienced trouble with basic activities of daily living, including eating and drinking. Additionally, CAS negatively impacted mental health and led to missed school for patients."
The researchers had follow-up data on responses to colchicine for 14 of the 20 patients. Of these, 12 (86%) had symptom improvement, five (36%) had a complete response, eight (57%) had a partial response, and one (7%) did not respond. Nine patients (64%) experienced side effects. Of these, six had diarrhea, two had nausea, and one had constipation.
"Colchicine should be considered as a treatment in pediatric patients who have refractory complex aphthous stomatitis as it is generally well tolerated with minimal side effects," Shah said. She acknowledged certain limitations of the study, including its single-center, retrospective design.
The researchers reported having no relevant disclosures.