Rituximab in the treatment of ocular cicatricial pemphigoid: a retrospective cohort study.
OBJECTIVE
The purpose was to evaluate the effectiveness and safety of rituximab (RTX) for the treatment of patients with aggressive ocular cicatricial pemphigoid (OCP).


METHODS
A review of patient records at a tertiary referral center with biopsy confirmed OCP who presented between 2006 and 2016. Sixty-one eyes of 32 patients with symptomatic OCP who received treatment with RTX monotherapy or RTX in combination with additional immunomodulatory treatment (IMT) were evaluated. Main outcomes included clinically evident remission of disease, the percentage of corticosteroid sparing patients, stage of OCP (Foster), best corrected visual acuity, and treatment complications. Remission was defined as absence of progressive scarring and active ocular inflammation for ≥ 2 months. Partial remission/responding was defined as disease control and clinical improvement for ≥ 2 months.


RESULTS
Mean age at the initiation of RTX treatment was 59.1 years (range, 24-80 years) with a median follow-up time after RTX initiation of 32 months (range, 14 to 127 months). Twenty-six patients achieved clinical remission with an average sustained remission of 24.5 months (from 9 months to 84 months). RTX monotherapy was used in six patients, RTX in combination with intravenous immunoglobulin in 14 patients, and RTX with intravenous immunoglobulin and/or with other IMT agent in six patients. Seven eyes (11.5%) of six patients had favorable response to RTX and achieved response and partial remission, while inflammation remained active in the other seven eyes (11.5%) of four patients though there was no progressive scarring. At the last visit, three patients (9.4%) were on topical corticosteroid, three patients (9.4%) were treated with systemic corticosteroid treatments, and the other 26 patients (81.2%) achieved corticosteroid sparing therapy. Five eyes (8.2%) progressed one Foster stage. No other cicatrization progression or worsening of LogMAR visual acuity (p = 0.641) was observed during the follow-up period. Adverse events included leukopenia in three patients (9.4%), anemia in two patients (6.2%), liver enzyme elevation in three patients (9.4%) who were also on another concomitant IMT drug, and Epstein-Barr Virus infection and sinus infection in one patient each (3.1%). No other severe adverse events were noted during the follow-up period.


CONCLUSIONS
These retrospective data suggest that RTX is efficacious and well tolerated when included for the treatment of OCP. Controlled studies are necessary to identify the role of this IMT agent in the therapeutic arsenal, especially its optimum dose and duration of administration.
Massachusetts Eye Research and Surgery Institution (MERSI), 1440 Main Street, Suite 201, Waltham, MA, 02451, USA.|Ocular Immunology and Uveitis Foundation, 348 Glen Road, Weston, MA, 02493, USA.|Department of Ophthalmology, Tianjin Medical University General Hospital, Tianjin, China, 300052.
Caiyun
C
You
Massachusetts Eye Research and Surgery Institution (MERSI), 1440 Main Street, Suite 201, Waltham, MA, 02451, USA.|Ocular Immunology and Uveitis Foundation, 348 Glen Road, Weston, MA, 02493, USA.
Neerav
N
Lamba
Massachusetts Eye Research and Surgery Institution (MERSI), 1440 Main Street, Suite 201, Waltham, MA, 02451, USA.|Ocular Immunology and Uveitis Foundation, 348 Glen Road, Weston, MA, 02493, USA.
Andres F
AF
Lasave
Massachusetts Eye Research and Surgery Institution (MERSI), 1440 Main Street, Suite 201, Waltham, MA, 02451, USA.|Ocular Immunology and Uveitis Foundation, 348 Glen Road, Weston, MA, 02493, USA.
Lina
L
Ma
Massachusetts Eye Research and Surgery Institution (MERSI), 1440 Main Street, Suite 201, Waltham, MA, 02451, USA.|Ocular Immunology and Uveitis Foundation, 348 Glen Road, Weston, MA, 02493, USA.
Mikhail Hernandez
MH
Diaz
Massachusetts Eye Research and Surgery Institution (MERSI), 1440 Main Street, Suite 201, Waltham, MA, 02451, USA. sfoster@mersi.com.|Ocular Immunology and Uveitis Foundation, 348 Glen Road, Weston, MA, 02493, USA. sfoster@mersi.com.|Department of Ophthalmology, Harvard Medical School, 25 Shattuck Street, Boston, MA, 02115, USA. sfoster@mersi.com.
C Stephen
CS
Foster
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001706:Biopsy; D003228:Conjunctiva / Q000473:pathology*; D004305:Dose-Response Relationship, Drug; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage; D008297:Male; D008875:Middle Aged; D010390:Pemphigoid, Benign Mucous Membrane / Q000175:diagnosis / Q000188:drug therapy*; D012189:Retrospective Studies; D000069283:Rituximab / Q000008:administration & dosage*; D013997:Time Factors; D016896:Treatment Outcome; D014792:Visual Acuity; D055815:Young Adult
D007155:Immunologic Factors; D000069283:Rituximab
Corticosteroid sparing; Ocular cicatricial pemphigoid; Remission; Rituximab