Systemic chemotherapy for ocular cicatricial pemphigoid.

Area of Science:

• Ophthalmology
• Immunology
• Dermatology

Background:

• Ocular cicatricial pemphigoid (OCP) is a chronic autoimmune disease that can lead to severe visual impairment.
• Chemotherapeutic agents are used to manage OCP, but long-term efficacy and tolerance vary.
• Comparing different treatment regimens is crucial for optimizing patient outcomes.

Purpose of the Study:

• To compare the long-term efficacy, side effects, and tolerance of three chemotherapeutic agents for OCP.
• To identify the most effective initial and sequential treatment strategies for OCP.
• To provide recommendations for chemotherapy in OCP management.

Main Methods:

• Retrospective review of 105 patients treated with diaminodiphenylsulfone (DAP), cyclophosphamide (CYC), or azathioprine (AZA).
• Analysis of disease progression, treatment failures, and patient tolerance.
• Stratification of results based on disease activity and treatment duration.

Main Results:

• OCP progressed in 6% of eyes (10% of patients) over a 35-month follow-up.
• Treatment failures were more common in patients intolerant to chemotherapy.
• Initial treatment failure rates: DAP (2%), CYC (8%), AZA (9%) (p<0.05).
• Long-term single-agent failure rates (≥10 months): DAP (4%), CYC (4%), AZA (15%) (p<0.01).
• DAP was most effective for modestly active OCP; CYC for highly active OCP.

Conclusions:

• Diaminodiphenylsulfone (DAP) and cyclophosphamide (CYC) demonstrate superior long-term efficacy compared to azathioprine (AZA) for OCP.
• A sequential approach to chemotherapy, considering disease activity and agent tolerance, is recommended for OCP.
• Optimizing chemotherapy regimens can improve long-term control and reduce visual impairment in OCP patients.