To assess the efficacy of low-dose methotrexate (MTX) given long-term for t
he treatment of autoimmune hearing loss, we performed a prospective open-la
bel study of 11 patients with treatment-refractory autoimmune hearing loss.
All patients had ongoing episodic worsening of hearing in 1 or both ears b
efore enrollment despite traditional medical therapy. The MTX dose was 7.5
to 17.5 mg/wk. Hearing loss and vertigo were evaluated at baseline and at c
ompletion of the study. Hearing improvement was defined as an improvement i
n the pure tone threshold (PT) average of >10 dB or an increase in speech d
iscrimination (SD) of >15%, whereas worsening was defined as a worsening of
>10 dB in PT or a decrease of >15% in SD in at least 1 ear. The MTX was we
ll tolerated. Among the 6 patients with Meniere's disease, 4 had improvemen
t or resolution of vertigo, while 2 had no improvement. Disequilibrium impr
oved in all 3 patients with Cogan's syndrome. According to the parameters d
efined above, hearing improved in 9 patients (82%), was unchanged in 1 pati
ent (9%), and worsened in 1 patient (9%). Long-term low-dose MTX therapy ma
y be a useful therapy for some patients who have hearing loss with a presum
ptively autoimmune-mediated component that is refractory to traditional the
rapies.