Treatment of serpiginous choroiditis with chlorambucil; efficacy and outcome

Abstract

Purpose : Concerns exist regarding the safety of alkylating agents in the treatment of inflammatory conditions. The safety and efficacy of chlorambucil in the treatment of serpiginous choroiditis was evaluated with a retrospective observational clinical study that examined adverse effects, recurrence, rate of new choroid neovascularization (CNVM), and visual acuity.

Methods : Institutional review board approval was attained for this study protocol. Patient records from the Massachusetts Eye Research and Surgery Institution (MERSI) were reviewed from over the past 10 years. Seventeen patients with serpiginous choroiditis treated with chlorambucil with at least 5 months of follow-up were identified. Quantiferon gold was negative in all patients. Chlorambucil was started at 0.15 mg/kg and dosage titrated up using weekly white blood cell (WBC) count to achieve a target of cell number of 3.0-4.5 x 10⁹ cells/L . The goal of therapy was to maintain this value for 9-12 months. Chlorambucil was stopped if there were any signs of adverse events including rash, subjective side effects, white blood cell (WBC) count below 2.4 x 10⁹ cells/L, platelet count below 125 × 10⁹/L and or abnormalities on liver function tests.

Results : Mean age of patients was 48 years and sixteen patients (42.1%) were male. Mean duration of follow up was 40 months with a mean duration of treatment of 11 months. None of the patients developed cancer or persistent side effects. Twelve patients (70.5%) achieved drug free remission in their last follow up ranging from 7-120 months. Visual acuity remained within 2 lines of snellen acuity in 26 eyes (76.5%), deteriorated in five eyes (14.7%), and improved in three eyes (8.8%). Three patients had disease recurrence 20, 44 and 6 months after completion of therapy. Two patients had to stop treatment after four months, prior to achieving remission, due to low WBC in one patient and fever in the other. Leukopenia was the most common side effect which was reversible in all cases. No patient developed new CNVM while on treatment but one patient with pre-existing CNVM underwent multiple intravitreal anti-VEGF injections while on chlorambucil.

Conclusions : Chlorambucil with escalating dose guided by weekly WBC was well tolerated as well as effective in preventing recurrence and maintaining vision.

This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.

 

upper image : Treated with methotrexate and prednisone
lower image: Four years after treatment with chlorambucil