• Written By: Shahzad I. Mian, MD
    Cornea/External Disease

    This retrospective, noncomparative study provides long-term follow-up results on the treatment of patients with a difficult-to-manage rare disease that can have devastating visual complications. The authors report on their experience managing with the stepladder approach 223 eyes with ocular cicatricial pemphigoid in 115 patients with at least six months of follow-up over an 11 ½-year period. Also known as ocular mucous membrane pemphigoid (MMP), the condition is a systemic cicatricial autoimmune disease that involves the eyes, mouth, and skin. Without treatment, the disease progresses to result in significant scarring and neovascularization of the cornea and eventual blindness.

    Systemic immunosuppression is necessary to reduce the risk of vision-threatening complications. Traditionally, treatment is initiated with sulfa derivative drugs, including dapsone, sulfasalazine, or sulfapyridine. Methotrexate, azathioprine, cyclophosphamide, corticosteroids, and intravenous immunoglobulin may be used in a stepladder therapeutic approach. Mycophenolate mofetil can also be used in the management of mild to moderate disease.

    In the current study, the authors used a step-up approach for patients with mild or moderate inflammation that involved initial treatment with dapsone or sulfapyridine. Azathioprine was added for patients with an incomplete response or substituted in those with no response after two to three months. Patients who were intolerant of or not responsive to azathioprine were given mycophenolate mofetil.

    Those with severe inflammatory disease received cyclophosphamide and, during the initial six weeks until optimal effects are reached, prednisolone, sometimes in combination with intravenous pulses of methylprednisolone. Since safe use of cyclophosphamide is limited to 12 months, treatment was stepped down to azathioprine, mycophenolate mofetil, methotrexate, or dapsone. Resistant cases received combination therapy including a sulfa drug (dapsone or sulfapyridine) and a myelosuppressive agent (azathioprine, mycophenolate, or cyclophosphamide), the addition of a maintenance dose or tapering six- to eight-week course of prednisolone, or both.

    Inflammation was controlled in 70 percent of patients. The authors concluded that mycophenolate was effective and well-tolerated compared with standard therapy in patients with mild to moderate levels of MMP. Their results indicated that inflammation was best controlled with cyclophosphamide, which was followed in order of effectiveness by mycophenolate, azathioprine, dapsone, and sulfapyridine. Side effects necessitated a change in therapy most frequently in patients given azathioprine, which occurred in 40 percent of those who received this treatment, compared with 15 percent for mycophenolate, the treatment with the fewest side effects.


    Financial Disclosures
    Dr. Mian has no financial interests to disclose.