Table 2.
Studies that employed topical chemoprevention agents to treat oral leukoplakia.
| Reference/design study | Chemopreventive protocol/sample (n) | Outcomes/biomarkers | Main results | Side effects | Follow-up |
|---|---|---|---|---|---|
| Wong et al., (39) CT | Bleomycin 0.5% (n = 5) Bleomycin 1% (n = 7) 1x day for 2 weeks |
Clinical and histological | CR: 1% - 3 cases complete 0.5% - 2 cases partial HR: 0.5% - no improvement 1% - 3 cases complete |
Burning, irritation, and erosion | 23 months |
| Epstein et al., (40) RCT | Bleomycin 1% (n = 10) Placebo (n = 12) 1x day for 2 weeks |
Clinical and histological | CR: Bleomycin 50% Placebo 8.3% (p = 0.001) HR: Bleomycin 60% Placebo 16.6% (p = 0.094) |
Burning, pain, erythema with erosion | 22 months |
| Piattelli et al., (41) CT | Isotretinoin 0,1% gel (n = 6) Placebo (n = 3) 3x day for 4 months |
Clinical Protein biomarker: bcl-2 and Apoptotic analysis |
CR: 10% bcl-2 (p = 0.134) Apoptotic bodies (p = 0.0193) |
None | 4 months |
| Tetê et al., (42) CT | Isotretinoin 0,1% gel Placebo (n = 15) 3x day for 4 months |
Clinical Protein biomarker: bcl-2 and Apoptotic analysis |
CR: 3 cases bcl-2 (p = 0.132) Apoptotic bodies (p = 0.0193) |
Not reported | Not reported |
| Rudin et al., (13) CS | Mouthwash ONYX-015 for p53-mutant cells 1 Cohort: 12 cycles (daily for 5 days) intervals 4 weeks (n = 7) 2 Cohort: 1x week for 24 weeks (n = 12) 3 Cohort: 5days + 1x week for 5 weeks (n = 3) |
Histological Protein biomarker: p53, Ki67 and cyclin D1 |
HR: 37% p53 (p = 0.027). |
Stomatitis, diarrhea, fatigue, pain, fever, flu symptoms, dysphagia, vertigo, and infection | 30 months |
| Mulshine et al., (44) RCT | Ketorolac rinse solution 0.1% 10 ml (n = 38) Placebo (n = 19) 2x day for 33 days |
Clinical and histological | CR: Ketorolac 10.8% Placebo 5.2% (p = 0.88) HR: Ketorolac (p = 0.76) Placebo (p = 0.80) |
Pain (n = 1) | 3 months |
| Scardina et al., (43) CT | Isotretinoin 0,18% gel (n = 40) 2x day for 3 months |
Clinical | CR: 85% | Transient erythema and xerostomia | 120 months |
| Mallery et al., (45) RCT | Dried black raspberries (DBR) 10% gel (n = 21) Placebo (n = 19) for 3 months |
Clinical, histological Molecular biomarkers: LOH |
CR: DBR 76% (p = 0.0019) Placebo 10% (p = 0.0395) HR: DBR 40.9% (p = 0.0488) Placebo 38.9% (p = 0.4961) LOH: DBR 45.5% (p = 0.002) Placebo 38.9% (p = 0.1602) |
None | 3 months |
CS, case series; CT, clinical trial; RCT, randomized clinical trial; CT, clinical trial; CR, clinical response; HR, histological response; LOH, loss of heterozygosity.