The role of mycophenolate mofetil in autoimmune inner ear disease AIED has never been evaluated.

1
Treatment of autoimmune sensorineural hearing
loss with mycophenolate mofetil
a retrospective study
Samar Charabaty, MD¹ Victoria Shanmugam, MD¹
Sean Whelton, MD¹ Anthony Turkiewicz, MD¹ Ari
Rubinfled, MD² H. Jeffrey Kim, MD² Thomas Cupp
s, MD¹
1 Department of Rheumatology, Allergy and
Immunology, ²Department of Otolaryngology,
Georgetown University Hospital, 3800 Reservoir
Rd, N.W., Washington DC 20007
Georgetown University
Abstract
Results
The role of mycophenolate mofetil in autoimmune
inner ear disease ( AIED) has never been
evaluated. We report a series of 10 patients with
autoimmune sensorineural hearing loss (SNHL)
where the use of mycophenolate mofetil (MMF) was
successful in 80 of the cases, in preventing
further hearing loss after induction with high
dose oral or intravenous steroid and/or after
failing methotrexate.

• Patient demographics are summarized in table 1.

Table 1

• The diagram on the right hand side summarizes
  the distribution of patients in each category.

• All 3 patients who got switched from MTX to MMF
  showed stable or improved hearing.

Introduction
Autoimmune sensorineural hearing loss (SNHL) was
originally described in 19791 but its
pathophysiology remains poorly understood. The
diagnosis is a clinical one, based on rapidly
progressive (over a course of weeks to months)
sensorineural hearing loss, often bilateral and
asymmetric, that responds to corticosteroids.
Although the mainstay of treatment is
corticosteroids, maintenance therapy with steroid
sparing agents has never been validated. In a
randomized controlled trial, methotrexate (MTX)
offered no significant benefit 2.

• Pure-tone air conduction thresholds remained
  stable or improved in 80 of the patients who
  received MMF compared to only 56 of the
  patients who received MTX (Graph 1).
• WDS were stable or improved in 80 of the
  patients who received MMF compared to 50 in the
  MTX group (Graph 2).
  
• Mean follow-up was 42.5 months for the MTX group
  and 29.7 months for the MMF group.
  
• P values were trending without however reaching
  statistical significance.

Graph 1 Pure-tone threshold response
Graph 2 WDS response
Methods
A retrospective chart review was completed on
patients evaluated in the Georgetown University
Hospital Division of Rheumatology between 1998
and 2007, with the diagnosis of autoimmune SNHL
based on evaluation by an Otolaryngologist and a
Rheumatologist. Failure of treatment was defined
as the deterioration in the pure-tone air
conduction threshold by 15 dB at an individual
frequency or by 10 dB at two consecutive
frequencies, and/or worsening of the word
discrimination score (WDS) by 12, compared to
the hearing achieved after the initial steroid
therapy. 3 patients with Cogans syndrome in who
m SNHL were thought to be immune mediated were
included in the analysis. Clinical hallmarks of
this chronic inflammatory disorder are
interstitial keratitis and vestibuloauditory
dysfunction. Statistical analysis was performed
using the Fishers exact test.
Conclusion
In patients with autoimmune SNHL, mycophenolate
mofetil appears to be effective in preventing
further deterioration after induction with
corticosteroids.
Discussion
The natural history of AIED is poorly understood.
While some patients have a brief, self-limited
process which responds to corticosteroids, many
experience relapses after the initial response.
The goal of long term immunosuppression is to
prevent further worsening of the hearing.
This data suggests that mycophenolate mofetil may
have a benefit as a steroid sparing agent in
autoimmune SNHL. A randomized placebo-controlled
trial is needed to better understand its role in
autoimmune inner ear disease.
References
1- McCabe BF Autoimmune sensorineural hearing
loss. Ann Otol Rhinol Laryngol 1979 88
585-589. 2- Harris JP et al. Treatment of Cortic
osteroid- Responsive Autoimmune Inner Ear Disease
with Methotrexate. JAMA. 2003 290 1875-1883.