Treatment Strategies for Recurrent Oral Aphthous Ulcers


Am J Health Syst Pharm. 2001;58(1) 

In This Article


A tablet formulation of amlexanox has been marketed in Japan since 1987 for the long-term treatment of asthma.[29,30] In the United States, amlexanox is available only as a 5% topical paste (Aphthasol, Block) for use in the treatment of RAU. Amlexanox is a potent inhibitor of the formation and release of inflammatory mediators from mast cells, neutrophils, and mononuclear cells. Given topically, amlexanox could be expected to facilitate the healing of aphthous ulcers but not to reduce the frequency of RAU episodes.[30]

Four vehicle-controlled, randomized, double-blind, multicenter trials assessed the efficacy of amlexanox with respect to ulcer healing and pain resolution in 1335 patients with minor RAU.[31] All the trials excluded patients using concurrent medications to treat RAU, patients with medical conditions posing a risk for study involvement and patients with alternative causes of RAU, including irritable-bowel disease, Behçet's syndrome, and anemia. The patients applied small dabs of amlexanox oral paste four times daily directly to the ulcers for 4-10 days. The first trial established the efficacy of 5% amlexanox paste relative to either 1% amlexanox paste or the paste vehicle. The 5% amlexanox paste produced significantly greater reductions in ulcer size than the vehicle and greater improvement in the rate of healing and erythema than either the vehicle or 1% amlexanox paste. The results of the remaining three trials comparing 5% amlexanox paste with the paste vehicle were presented as a meta-analysis examining rates of complete healing of ulcers and resolution of pain. After six days of therapy, 74% of the patients receiving amlexanox paste had complete healing of ulcers, versus 54% of those using the vehicle (p < 0.001). Similarly, 83% of the patients treated with amlexanox paste had complete resolution of pain, compared with 73% of those receiving the vehicle (p < 0.001). The self-limiting nature of aphthous ulcers, combined with the protective effects of the paste vehicle, produced high rates of healing and pain resolution independent of amlexanox. Patients may benefit from earlier application of amlexanox during the prodromal stage of an RAU episode.

In a review of clinical trials and premarketing studies involving 991 subjects, only 2.1% of those using 5% amlexanox paste reported adverse events.[29] Almost 70% of these events were associated with the topical application of amlexanox and included stinging, dryness, bumps on the lips, and mucositis. All of the adverse events were transient, and none resulted in early discontinuation of amlexanox therapy.


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