title: Analysis of a novel protocol of pulsed intravenous cyclophosphamide for recalcitrant or severe ocular inflammatory disease.
abstract: OBJECTIVE
  To analyze the success rate of pulsed intravenous (IV) cyclophosphamide (CyP) for noninfectious ocular inflammatory disease and to identify risk factors for failure of therapy.
  
  
  METHODS
  Retrospective, interventional, noncomparative cohort study.
  
  
  METHODS
  One hundred ten eyes of 65 patients.
  
  
  METHODS
  Through a computer search of the Massachusetts Eye Research and Surgery Institution's database, we identified patients who were treated with IV CyP between May 2005 and April 2012. We obtained demographic and clinical information through review of the electronic health record of each patient.
  
  
  METHODS
  Clinical response, corticosteroid-sparing effect, recurrence rate, calculated "risk factors" for failure, visual acuity, and adverse reactions.
  
  
  RESULTS
  Pulsed IV CyP achieved complete remission of inflammation (for ≥ 2 visits) in 54 patients (84.4%). Sustained remission of inflammation occurred in 70% of patients within 3 months, 86.6% of patients within 6 months, and 91.7% within 9 months. The mean time to achieving quiescence was 3.5 months. The success rate in reducing corticosteroid to prednisone ≤ 10 mg/d within 6 months, while maintaining control of ocular inflammation, was 89.7% (95% confidence interval [CI], 81.1-93.5%). The mean duration of clinical remission for those patients who had a positive response to CyP was 32.67 months (95% CI, 25.91-39.43). Relapse of vasculitis was observed in 1 patient (1.5%) after completing the course of therapy. Early initiation of therapy during the course of the disease was correlated with a lesser rate of recurrence (P = 0.028). The most common adverse effects were nausea (29%) and transient lymphopenia (26%). The mean best-corrected visual acuity (BCVA) improved from 0.59 ± 0.66 at baseline to 0.30 ± 0.54 at 6 months of follow-up (P<0.001). The mean follow-up period was 31.61 ± 20.47 months.
  
  
  CONCLUSIONS
  Pulsed IV CyP employing our protocol results in an extremely high rate of sustained complete remission in patients with recalcitrant and fulminant, vision-threatening ocular inflammatory disorders, with an excellent safety profile in the hands of physicians trained and skilled in the art of this therapy. It also allows tapering and discontinuing corticosteroids in most patients. Early initiation of therapy may decrease the risk of relapses.
  
  
  BACKGROUND
  The authors have no proprietary or commercial interest in any materials discussed in this article.
authors.affiliation: Massachusetts Eye Research and Surgery Institution, Cambridge, Massachusetts, USA.
authors.forename: Ana M
authors.initials: AM
authors.lastname: Suelves
authors.forename: Cheryl A
authors.initials: CA
authors.lastname: Arcinue
authors.forename: Jesús María
authors.initials: JM
authors.lastname: González-Martín
authors.forename: Jonathan N
authors.initials: JN
authors.lastname: Kruh
authors.forename: C Stephen
authors.initials: CS
authors.lastname: Foster
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002985:Clinical Protocols; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005451:Fluorescein Angiography; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000009:adverse effects; D007262:Infusions, Intravenous; D008297:Male; D008875:Middle Aged; D010390:Pemphigoid, Benign Mucous Membrane / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D020551:Pulse Therapy, Drug; D012008:Recurrence; D031300:Retinal Vasculitis / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D012189:Retrospective Studies; D012307:Risk Factors; D015423:Scleritis / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D017211:Treatment Failure; D014605:Uveitis / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D014792:Visual Acuity / Q000502:physiology; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide