Evaluating the Safety and Efficacy of Cinobufacini in Patients with Oral Potentially Malignant Disorders: An Open-Label Pilot Study

Objective Herein, we collected preliminary data on the effects of cinobufacini, a Chinese medicine extracted from Venenum Bufonis, to assess its safety and efficacy in patients with potentially malignant disorders (OPMDs). Methods Patients with histologically confirmed OPMDs were enrolled in this open-label uncontrolled pilot clinical study for a four-week treatment. The main outcome measures were efficacy (lesion size, activity score, and numerical rating scale) and safety (blood cell count, general biochemistry, liver and adrenal function tests, and adverse effects). Results A fifteen-patient cohort (9 females and 6 males; average age, 53.67 ± 11.69 years) with various OPMD types successfully completed the four-week cinobufacini treatment. Among them, only two patient (13.33%) exhibited partial clinical response (lesion size reduction, ≥ 50%). It was observed that the lesion size decreased in 10 participants (66.67%), the mean area score showed a significant reduction from 6.53 ± 2.16 to 4.50 ± 1.60 (P < 0.05). When assessed using the activity scoring system, 9 participants (73.33%) demonstrated a decrease in their activity score, with a significant decrease in mean activity score from 5.40 ± 1.48 to 3.13 ± 0.68 (P < 0.05). The mean numerical rating scale (NRS) score showed a significant reduction from 3.23 ± 1.73 to 1.73 ± 0.92 (P < 0.05). Remarkably, four patients had a histopathological complete response after extending treatment to 12 weeks. Overall, most patients had no adverse effects except four cases reporting mild gastrointestinal discomfort. Conclusions The findings from this open-label pilot study highlight the potential of cinobufacini as a promising therapy for OPMDs, underscoring the need for larger and longer-term trials. The trial registration number (TRN): IRB-YJ-2022005；The date of registration:01/06/2022