The effect of treatment and its related side effects in patients with severe ocular cicatricial pemphigoid.
OBJECTIVE
To determine the clinical outcome of patients with ocular-cicatricial pemphigoid (OCP) and the influence of systemic treatment on clinical progression.


METHODS
Noncomparative interventional case series.


METHODS
Sixty-one patients with biopsy-proven OCP.


METHODS
Patients with documented disease progression treated with chemotherapy and/or corticosteroids were followed between 1985 and 2000. The parameters evaluated were ocular stage at presentation, visual acuity, ocular complications, disease progression, control of ocular inflammation, and presence of extraocular involvement. Systemic treatment and related side effects were analyzed.


METHODS
Visual acuity, ocular complications, extraocular involvement, disease progression, clinical outcome, systemic treatment, and related side effects.


RESULTS
Sixty-one patients (32 female; 29 male) with a mean age of 67 years were studied. Extraocular involvement was present in 50% of patients. Sixty percent of eyes were initially seen with stage III (advanced cicatrizing) disease at first evaluation. Seven percent of involved eyes at first visit and 21% at the end of follow-up were legally blind. The most common ocular complications encountered were dry eye, corneal abnormalities, and glaucoma. Dapsone was the most commonly used drug (51 patients), followed by methotrexate (24 patients), azathioprine (23 patients), and cyclophosphamide (15 patients); prednisone, always given as adjunctive treatment, was used in 17 patients. Control of ocular inflammation (total or partial) was achieved in 90% of patients, but 46% of them needed continuation of systemic treatment to avoid disease recurrences, and 10% progressed despite different drugs used. Two agents were required in 32% of cases to control disease activity. The most common treatment-related side effects were hematologic complications (n = 34) followed by gastrointestinal (n = 17), cardiovascular (n = 15), and urinary complications (n = 11). Dapsone was responsible for the greatest number of side effects (n = 43); methotrexate caused the least trouble (n = 6). Corticosteroid-related complications (n = 34) were mostly cardiovascular and endocrinologic.


CONCLUSIONS
Ocular-cicatricial pemphigoid is an autoimmune disease that, untreated, progresses to conjunctival scarring and blindness; systemic immunosuppression is required to control it. Long-term systemic treatment and more than one drug are frequently necessary to avoid recurrences, exposing elderly patients to a higher risk of drug toxicity. The most frequently encountered treatment-related side effects were anemia, leukopenia, liver toxicity, and hypertension.
Miserocchi
Elisabetta
E
Immunology and Uveitis Service, Department of Ophthalmology, Massachusetts Eye and Ear Infirmary, Harvard Medical School, 243 Charles St., Boston, MA 02114, USA.
Baltatzis
Stefanos
S
Roque
Manolette R
MR
Ahmed
A Razzaque
AR
Foster
C Stephen
CS
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000009:adverse effects* / Q000627:therapeutic use; D001327:Autoimmune Diseases / Q000188:drug therapy* / Q000503:physiopathology; D001379:Azathioprine / Q000009:adverse effects / Q000627:therapeutic use; D003231:Conjunctivitis / Q000188:drug therapy* / Q000503:physiopathology; D003520:Cyclophosphamide / Q000009:adverse effects / Q000627:therapeutic use; D003622:Dapsone / Q000009:adverse effects / Q000627:therapeutic use; D018450:Disease Progression; D004342:Drug Hypersensitivity / Q000209:etiology; D005260:Female; D005938:Glucocorticoids / Q000009:adverse effects* / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects* / Q000627:therapeutic use; D008297:Male; D008727:Methotrexate / Q000009:adverse effects / Q000627:therapeutic use; D008875:Middle Aged; D010390:Pemphigoid, Benign Mucous Membrane / Q000188:drug therapy* / Q000503:physiopathology; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use; D014792:Visual Acuity
D000894:Anti-Inflammatory Agents, Non-Steroidal; D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D003622:Dapsone; D001379:Azathioprine; D011241:Prednisone; D008727:Methotrexate