Changes in Vision- and Health-Related Quality of Life in Patients with Diabetic Macular Edema Treated with Pegaptanib Sodium or Sham

Abstract

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...

Patient-Reported Visual Function Outcomes Improve After Ranibizumab Treatment in Patients With Vision Impairment Due to Diabetic Macular Edema

“…These outcomes were similar, but of greater magnitude, to an analysis of randomized controlled trial data for pegaptanib treatment. 18 In the RESTORE trial, approximately half of the patients achieved at least a 5-point improvement in the NEI VFQ-25 composite score with ranibizumab monotherapy and 43% with ranibizumab plus laser at the 12-month follow-up visit compared with 31% with laser alone. Greater improvements from baseline for ranibizumab monotherapy compared with laser alone were also noted on the subscales for near activities (visual activities, including reading ordinary print in a newspaper and close-up work or hobbies, such as cooking or using hand tools) and general vision (perception of how good their eyesight is).…”

Changes in vision related quality of life in patients with diabetic macular edema: Ranibizumab or laser treatment?

“…There are several therapeutic modalities for DME, such as focal laser photocoagulation, intravitreal or subtenon's injection of triamcinolone acetonide, intravitreal bevacizumab, and pars plana vitrectomy (Bressler et al, 2014;Hariprasad et al, 2008;Loftus et al, 2011;Nguyen et al, 2010;Okamoto et al, 2014). The Early Treatment for Diabetic Retinopathy Study (ETDRS) has reported the beneficial effect of laser photocoagulation for patients with clinically significant macular edema (CSME) (Brown et al, 2013;Tranos et al, 2004).…”

Current intravitreal pharmacologic therapies for diabetic macular edema

“…A trial reported in 2011 showed administration of intravitreal pegaptanib every 6 weeks for 1 year resulted in statistically significant improvement compared to sham in both visual acuity as measured by ‡10-letter gains (p = 0.0047) and patient quality of life as measured by a >5-point difference in the National Eye Institute Visual Functioning Questionnaire 25 [59].…”