Efficacy and safety of chlorambucil in intractable noninfectious uveitis: the Massachusetts Eye and Ear Infirmary experience

Ophthalmology. 2002 Jan;109(1):137-42. doi: 10.1016/s0161-6420(01)00864-8.


Purpose: To report our experience with the use of chlorambucil for otherwise treatment-resistant uveitis and to assess its safety and efficacy.

Design: Noncomparative interventional case series.

Participants: Twenty-eight patients with intractable noninfectious uveitis.

Methods: We reviewed the records of 28 patients (56 eyes) with chronic noninfectious uveitis who were treated with chlorambucil from 1987 to 2000. Diagnoses included Adamantiades-Behçet's disease (ABD) (7 patients), juvenile rheumatoid arthritis (JRA)-associated uveitis (10 patients), pars planitis (2 patients), sympathetic ophthalmia (1 patient), idiopathic uveitis (6 patients), Crohn's disease (1 patient), and HLA-B27-associated uveitis (1 patient). All patients were refractory to other immunomodulatory therapy and systemic steroids. The median duration of treatment with chlorambucil was 12 months (range, 4-50 months), whereas the median daily dosage was 8 mg (range, 4-22 mg). Patients were followed for a median follow-up period of 46 months (range, 4-166 months) after chlorambucil treatment was begun and continued to be followed for relapse after cessation of therapy.

Main outcome measures: Visual outcome, response to treatment, treatment-related side effects, drug dosage, previous and final treatment, discontinuation of systemic corticosteroids.

Results: Chlorambucil was discontinued in seven patients because of side effects: two females had temporary amenorrhea develop, two patients had unacceptable gastrointestinal intolerance, one patient had infection, and 2 patients had progressive leukopenia. Nineteen patients (68%) showed positive clinical response to the treatment, four (14%) initially responded then relapsed after discontinuation of the drug, three patients with ABD had improvement of ocular disease but worsening of systemic symptoms, and two had persistent inflammation. Visual acuity was improved in 24 eyes (43%), stable in 22 (39%), and worsened in 10 eyes (18%). Systemic prednisone was successfully discontinued in 19 of the 28 patients (68%), and 14 patients were free of inflammation at the end of follow-up without any systemic medication.

Conclusions: Chlorambucil can be a safe and effective alternative for preserving vision in patients with otherwise treatment resistant uveitis.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antineoplastic Agents, Alkylating / adverse effects
  • Antineoplastic Agents, Alkylating / therapeutic use*
  • Child
  • Child, Preschool
  • Chlorambucil / adverse effects
  • Chlorambucil / therapeutic use*
  • Chronic Disease
  • Female
  • Humans
  • Male
  • Middle Aged
  • Safety
  • Treatment Outcome
  • Uveitis / drug therapy*
  • Visual Acuity


  • Antineoplastic Agents, Alkylating
  • Chlorambucil