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, Ն250 m) and visual acuity (VA) Յ65 letters and Ն35 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 Ն10 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 shamtreated subjects were in the year 1 intent-to-treat population. (QoL) among DME patients is greatly affected at all stages of disease, from preliminary symptoms to diagnosis to vision loss.1-4 Treatment of DME may improve vision-related QoL in these patients.
3,5The importance of identifying safe and effective treatments for DME that result in anatomic, functional, and QoL improvements is underscored by the rising prevalence of diabetesestimated to affect more than 350 million individuals worldwide by 2030.6 Among adults with diabetes, approximately 1 in 10 experiences vision-threatening diabetic retinopathy; half (5.7% of all diabetics) go on to develop DME, 7 a condition responsible for 4.8% of blindness worldwide.8 Until recently, laser photocoagulation has been the standard treatment for DME, but vascular endothelial growth factor inhibitors have produced impressive gains in visual acuity during up to 2 years of follow-up in treating this condition (Radhakrishnan R, et al. IOVS 2010;52:ARVO E-Abstract 5042).9 -15 A recent study 14 found intravitreal (IVT) injections of pegaptanib sodium 0.3 mg to be a well-tolerated and effective treatment for DME. The study demonstrated a statistically significant difference in favor of pegaptanib over sham injection in the proportion of subjects with Ն10 letters (or 2 lines) visual acuity (VA) improvement at week 54 (primary endpoint). Improvement of at least 10 letters from baseline to week 54 was achieved by 36.8% (49/133) of subjects treated with pegaptanib compared with 19.7% (25/ 127) of sham-treated subjects (odds ratio [95% CI]: 2.38 [1.32-4.30]; P ϭ 0.0047). The clinical benefit of pegaptanib treatment for DME was further supported by statistically significant differences favoring pegaptanib with regard to several secondary endpoints (e.g., degree of retinopathy, use of focal/grid laser treatment). As part of the pivotal trial of pegaptanib in DME, changes in QoL over 54 and 102 weeks were evaluated by using the condition-specific 25-item National Eye Institute Visual Function Questionnaire (NEI-VFQ 25) and the EuroQol five-dimension (EQ-5D) health status measure. Results of thos...