Changes in vision- and health-related quality of life in patients with diabetic macular edema treated with pegaptanib sodium or sham

Invest Ophthalmol Vis Sci. 2011 Sep 29;52(10):7498-505. doi: 10.1167/iovs.11-7613.


Purpose: To compare vision function and self-reported quality of life (QoL) in patients with diabetic macular edema (DME) treated with intravitreous pegaptanib 0.3 mg or sham injection.

Methods: This randomized (1:1), controlled, multicenter trial included subjects with DME (center point thickness on OCT, ≥ 250 μm) and visual acuity (VA) ≤ 65 letters and ≥ 35 letters. In year 1, pegaptanib or sham was administered every 6 weeks with focal/grid photocoagulation at investigator discretion after week 18. Subjects received injections as often as every 6 weeks per pre-specified criteria in year 2. Primary efficacy endpoint: proportion gaining ≥10 letters of VA from baseline to week 54. Change in QoL from baseline to weeks 54 and 102 was assessed with the 25-item National Eye Institute-Visual Function Questionnaire (NEI-VFQ 25) and the EQ-5D.

Results: One hundred thirty-three pegaptanib- and 127 sham-treated subjects were in the year 1 intent-to-treat population. From baseline to week 54, ≥ 10 letter gains seen in 49 (36.8%) pegaptanib- and 25 (19.7%) sham-treated subjects (odds ratio [95% CI]: 2.38 [1.32-4.30]; P = 0.0047). At 2 years, the VA trend favored pegaptanib. The NEI-VFQ 25 domains of Near Vision, Distance Vision, and Social Functioning (week 54) and Distance Vision, Social Functioning, Mental Health, and Composite Score (week 102) demonstrated clinically meaningful (>5-point between-group difference) and statistically significant (P < 0.05) benefits favoring pegaptanib. No significant difference in the mean change in generic EQ-5D-weighted utility scores was seen.

Conclusions: The VA improvement from pegaptanib treatment versus sham is reflected by improved vision-related QoL as reported by the DME patient ( number, NCT00605280).

Publication types

  • Comparative Study
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Aptamers, Nucleotide / therapeutic use*
  • Diabetic Retinopathy / drug therapy*
  • Diabetic Retinopathy / physiopathology
  • Double-Blind Method
  • Female
  • Humans
  • Macular Edema / drug therapy*
  • Macular Edema / physiopathology
  • Male
  • Middle Aged
  • Quality of Life*
  • Sickness Impact Profile*
  • Surveys and Questionnaires
  • Tomography, Optical Coherence
  • Treatment Outcome
  • Vision, Ocular / physiology*
  • Visual Acuity / physiology*
  • Young Adult


  • Aptamers, Nucleotide
  • pegaptanib

Associated data