Treatment of Ocular Mucous Membrane Pemphigoid
Systemic immunosuppressive therapy is required to control ocular mucous membrane pemphigoid (OMMP), as topical corticosteroids alone are insufficient to prevent disease progression and blindness. 1
Treatment Algorithm Based on Disease Severity
Mild to Moderate Disease (Slowly Progressive)
For patients with mild and slowly progressive OMMP, first-line systemic therapy includes: 1
- Mycophenolate mofetil (typically 1-2 g daily)
- Dapsone (with mandatory G6PD screening prior to initiation) 1
- Azathioprine
- Methotrexate
Dapsone should be avoided in patients with glucose-6-phosphate dehydrogenase (G6PD) deficiency due to risk of severe hemolytic anemia. 1
Among these agents, mycophenolate mofetil and methotrexate demonstrate lower discontinuation rates due to side effects compared to other immunosuppressants, making them preferred choices when selecting initial therapy. 2
Severe or Refractory Disease
For severe inflammation or disease unresponsive to first-line agents, cyclophosphamide should be considered. 1
Cyclophosphamide is the most potent immunosuppressive agent for ocular MMP but carries significant toxicity risks including hematologic complications, cardiovascular effects, and malignancy risk, requiring careful patient selection and monitoring. 3, 2
Additional Therapeutic Options
Other effective therapies that can be used alone or in combination include: 1
- Oral tetracycline and niacinamide 1
- Sulfasalazine 1
- Intravenous immunoglobulin (IVIg) - particularly for patients with contraindications to conventional therapy, significant adverse effects, or rapidly progressive disease 1, 2
- Biologics (rituximab, anti-TNF agents) 1
Refractory cases may benefit from combination intravenous immunoglobulin and rituximab. 1
Role of Corticosteroids
Systemic corticosteroids may be indicated initially to rapidly control acute inflammation, but they must be weaned as other immunosuppressive therapy becomes effective to avoid complications of chronic corticosteroid use. 1
Topical corticosteroid therapy may aid in controlling acute conjunctival inflammation but is never sufficient as monotherapy - systemic immunosuppression is mandatory to inhibit inflammation, prevent keratopathy, and prevent progression of conjunctival scarring. 1
Critical Management Considerations
A physician with expertise in immunosuppressive therapy should administer and monitor treatment to minimize and manage side effects. 1
Long-term systemic treatment is frequently necessary, with approximately 46% of patients requiring continuation of therapy to avoid disease recurrences. 3 Control of inflammation (total or partial) can be achieved in approximately 90% of patients with appropriate systemic immunosuppression. 3
Two or more agents may be required in approximately 32% of cases to adequately control disease activity. 3
Common Pitfalls and Monitoring
The most frequently encountered treatment-related side effects include: 3
- Hematologic complications (anemia, leukopenia) - most common with dapsone
- Gastrointestinal complications
- Cardiovascular complications - particularly with corticosteroids
- Hepatotoxicity
Dapsone causes the highest number of side effects overall, while methotrexate demonstrates the most favorable side effect profile among conventional agents. 3
Adjunctive Ocular Management
Associated dry eye syndrome should be treated aggressively with preservative-free lubricants. 1 Trichiasis, distichiasis, and entropion should be managed nonsurgically when possible, as surgical intervention can exacerbate disease activity. 1
Any ocular reconstructive surgery is considered high risk and should only be performed when disease is quiescent with adequate immunosuppression. 1
Long-Term Outcomes
Prolonged remission off therapy can be achieved in approximately one-third of patients, though lifelong follow-up is mandatory as relapse occurs in another third of cases. 4 Patients who relapse typically regain disease control readily upon reinstitution of therapy without progression to more advanced cicatrization. 4