Long-term follow-up of patients treated with short-term high-dose chlorambucil for sight-threatening ocular inflammation.
OBJECTIVE
To determine the effectiveness of short-term high-dose chlorambucil in the treatment of sight-threatening uveitis and to ascertain the incidence of severe side effects, particularly late malignancy.


METHODS
Retrospective, noncomparative interventional case series.


METHODS
Fifty-three patients treated at the University of Illinois at Chicago Eye and Ear Infirmary and the private office of one of the authors for severe sight-threatening uveitis.


METHODS
Treatment with short-term high-dose chlorambucil (2-9 months of therapy).


METHODS
Visual acuity and degree of inflammation were assessed at every visit. The development of systemic side effects, including malignancy, was assessed using a detailed questionnaire.


RESULTS
Total cumulative dose of chlorambucil ranged from 392 to 5200 mg with an average of 1429 mg. The maximum daily dose ranged from 10 to 30 mg with an average of 20 mg. Average duration of treatment was 16 weeks with a range of 7 to 40 weeks. Seventy-seven percent of patients treated were in remission with an average follow-up of 4 years (range: 6 months to 24 years). Forty-seven percent had at least two lines of improvement in Snellen visual acuity after treatment, with an average gain of 3.5 lines. Adverse effects include secondary amenorrhea, nonophthalmic herpes zoster, testicular atrophy, and erectile dysfunction. None of the patients had developed a malignancy as of their last follow-up.


CONCLUSIONS
Short-term high-dose chlorambucil therapy may be a reasonable option in patients with intractable sight-threatening uveitis.
Goldstein
Debra A
DA
Eye and Ear Infirmary, University of Illinois at Chicago, Chicago, Illinois 60612, USA.
Fontanilla
Fernandino A
FA
Kaul
Suruchi
S
Sahin
Ozlem
O
Tessler
Howard H
HH
D000293:Adolescent; D000328:Adult; D000368:Aged; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D002699:Chlorambucil / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome; D014605:Uveitis / Q000188:drug therapy*; D014792:Visual Acuity
D018906:Antineoplastic Agents, Alkylating; D002699:Chlorambucil