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Purpose Ophthalmologists increasingly depend on new drugs to advance their treatment options. These options are limited by restraints on reimbursements for new and expensive drugs. These restraints are put in place through health policy decisions based on cost-effectiveness analyses (CEA). Cost-effectiveness analyses need to be valid and of good quality to support correct decisions to create new treatment opportunities. In this study, we report the quality, validity and usefulness of CEAs for therapies for nAMD. Methods A systematic review in PubMed, EMBASE and Cochrane was performed to include CEAs. Quality and validity assessment was based on current general quality criteria and on elements that are specific to the field of ophthalmology. Results Forty-eight CEAs were included in the review. Forty-four CEAs did not meet four basic model quality and validity criteria specific to CEAs in the field of ophthalmology (both eyes analysed instead of one; a time horizon extending beyond 4 years; extrapolating VA and treatment intervals beyond trial data realistically; and including the costs of low-vision). Four CEAs aligned with the quality and validity criteria. In two of these CEAs bevacizumab as-needed (PRN) was more cost-effective than bevacizumab monthly; aflibercept (VIEW); or ranibizumab monthly or PRN. In two CEAs, ranibizumab (PRN or treat and extent) was dominant over aflibercept. In two other CEAs, aflibercept was either more cost-effective or dominant over ranibizumab monthly or PRN. Conclusion Two of the CEAs of sufficient quality and validity show that bevacizumab PRN is the most cost-effective treatment. Comparing ranibizumab and aflibercept, either treatment can be more cost-effective depending on the assumptions used for drug prices and treatment frequencies. The majority of the published CEAs are of insufficient quality and validity. They wrongly inform decision-makers at the cost of opportunities for ophthalmologists to treat patients. As such, they may negatively influence overall patient outcomes and societal costs. For future ophthalmic treatments, CEAs need to be improved and only published when they are of sufficient quality and validity.

Purpose: To evaluate the effect of topical instillation of pegaptanib sodium upon inflammatory angiogenesis induced in the rabbit cornea by alkaline cauterization. Methods: Inflammatory angiogenesi...

Purpose Ophthalmologists increasingly depend on new drugs to advance their treatment options. These options are limited by restraints on reimbursements for new and expensive drugs. These restraints are put in place through health policy decisions based on cost-effectiveness analyses (CEA). Cost-effectiveness analyses need to be valid and of good quality to support correct decisions to create new treatment opportunities. In this study, we report the quality, validity and usefulness of CEAs for therapies for nAMD. Methods A systematic review in PubMed, EMBASE and Cochrane was performed to include CEAs. Quality and validity assessment was based on current general quality criteria and on elements that are specific to the field of ophthalmology. Results Forty-eight CEAs were included in the review. Forty-four CEAs did not meet four basic model quality and validity criteria specific to CEAs in the field of ophthalmology (both eyes analysed instead of one; a time horizon extending beyond 4 years; extrapolating VA and treatment intervals beyond trial data realistically; and including the costs of low-vision). Four CEAs aligned with the quality and validity criteria. In two of these CEAs bevacizumab as-needed (PRN) was more cost-effective than bevacizumab monthly; aflibercept (VIEW); or ranibizumab monthly or PRN. In two CEAs, ranibizumab (PRN or treat and extent) was dominant over aflibercept. In two other CEAs, aflibercept was either more cost-effective or dominant over ranibizumab monthly or PRN. Conclusion Two of the CEAs of sufficient quality and validity show that bevacizumab PRN is the most cost-effective treatment. Comparing ranibizumab and aflibercept, either treatment can be more cost-effective depending on the assumptions used for drug prices and treatment frequencies. The majority of the published CEAs are of insufficient quality and validity. They wrongly inform decision-makers at the cost of opportunities for ophthalmologists to treat patients. As such, they may negatively influence overall patient outcomes and societal costs. For future ophthalmic treatments, CEAs need to be improved and only published when they are of sufficient quality and validity.

To evaluate the results of a 3-year follow-up of intravitreal pegaptanib sodium injection as maintenance therapy for the treatment of neovascular age-related macular degeneration (AMD) in Japanese patients.In this prospective, uncontrolled interventional study, 20 eyes of 19 patients with treatment-naïve AMD who had received 3 consecutive monthly injections of 0.5 mg/0.05 mL ranibizumab as the induction treatment and had shown clinical/anatomical improvement were enrolled. An intravitreal injection of 0.3 mg/0.09 mL pegaptanib sodium was administered as the maintenance therapy every 6 weeks. Booster treatments using ranibizumab were allowed if clinical deterioration was judged to be present. The primary outcome measures were the best-corrected visual acuity (BCVA) and the central foveal thickness (CFT) as evaluated using spectral-domain optical coherence tomography.Sixteen of the 20 eyes (80 %) were assessed at the 3-year follow-up. The mean logMAR BCVA improved significantly from 0.56 ± 0.31 before the induction treatment to 0.24 ± 0.25 at baseline (P0.001) and was well maintained at 156 weeks (0.25 ± 0.28, P = 0.938). Moreover, the mean CFT also decreased significantly from 346 ± 111 μm before the induction treatment to 232 ± 54 μm at baseline (P0.001) and was well preserved at 156 weeks (210 ± 59 μm, P = 0.278). Thirteen eyes (81.3 %) received an unscheduled booster treatment, and no severe systemic or ocular side effects occurred during follow-up.Intravitreal pegaptanib sodium injection as the maintenance therapy was effective in stabilizing the vision of patients with AMD in whom induction treatment led to improved BCVA, as evaluated at the 3-year follow-up.

Anthony S Basile,1 Matthew M Hutmacher,2 Kenneth G Kowalski,2 Kuan Y Gandelman,3 Dana J Nickens1 1Clinical Pharmacology, Specialty Care Business Unit, Pfizer Inc, San Diego, CA, USA; 2Ann Arbor Pharmacometrics Group, Ann Arbor, MI, USA; 3Clinical Pharmacology, World Wide Biopharmaceuticals, Pfizer Inc, New York, NY, USA Objective: Population pharmacokinetic modeling of pegaptanib was undertaken to determine influence of renal function on apparent clearance. Methods: In a randomized, double-masked multicenter trial, intravitreal pegaptanib (0.3, 1.0, or 3.0 mg/eye) was administered in patients with diabetic macular edema every 6 weeks for 12–30weeks. A one-compartment model with first-order absorption, distribution volume, and clearance was used to characterize the pegaptanib plasma concentration–time profile. Results: In 58 patients, increases in area under the concentration–time curve (AUC) to end of the dosing interval (AUC0–tau) and maximum concentration with repeat doses were 68 years) relative to younger patients due to decreases in creatinine clearance (CRCL), a significant predictor of clearance. Pegaptanib clearance was reduced by 29% when CRCL decreased by 50%. The change in exposure with CRCL (range, 0–190 mL/minute) was < 10-fold with 0.3–3.0 mg doses. Conclusion: While pegaptanib clearance and AUC were significantly influenced by CRCL, the predicted exposure in patients with renal insufficiency or renal failure shows no evidence that a dose adjustment is warranted, given the tenfold margin of safety observed over the dose range of 0.3–3.0 mg. Keywords: clearance, diabetic macular edema, pegaptanib, population pharmacokinetics, renal function&nbsp

The management of ocular neovascular diseases received a major shot in the arm with the advent of anti-VEGF (vascular endothelial growth factor) therapy at the turn of the millennium. The anti-VEGF triumvirate – bevacizumab (Avastin), ranibizumab (Lucentis) and Pegaptanib sodium (Macugen), especially the former two, constitute the bulk of therapy for exudative and neovascular retinal pathologies.1-6 The use of these agents have brought along with them challenges and conundrums of their own. We have a few such challenging clinical scenarios with eminent experts providing their take and therapeutic strategies.

Objective Spinal epidural fibrosis is a clinical condition that develops after laminectomy and can compress the spine. Many agents have been tried for the treatment, but none has entered clinical use at present. Pegaptanib sodium is an antiangiogenetic drug that prevents the development of new vessels and thus adhesion by inhibiting the effect of VEGF. Material and method 20 Wistar rats were used in this study. The rats were divided into 2 different groups as the control and pegaptanib sodium group. Three levels of laminectomy were performed. Only laminectomy was performed in the control group. A cotton ball soaked with 3 mg/kg Pegaptanib sodium diluted 1: 10 with 0.9 % NaCl was topically applied to the dura in the surgical field for 5 minutes in the pegaptanib sodium group. The rats were sacrificed 3 weeks later and histopathologically examined. The epidural fibrosis was graded. Results The epidural fibrosis grade in the pegaptanib sodium was significantly lower than in the control group c2 = 11,65; (p = 0.004)CONCLUSION: Pegaptanib sodium blocked the VEGF through its anti-VEGF effect and decreased spinal epidural fibrosis in rats that had undergone laminectomy (Tab. 2, Fig. 3, Ref. 53).

Systemic VEGF inhibition disrupts endothelial homeostasis and accelerates the atherogenesis, suggesting that these events contribute to the clinical cardiovascular adverse events of VEGF-inhibiting therapies. The objective of the current study was to analyze the effect of anti-VEGF therapy on cardiovascular risk factors in patients with exudative age related macular degeneration. A total of 73 patients with exudative age related macular degeneration (without previous anti-VEGF therapy) were treated with two anti-VEGF: Ranibizumab and Pegaptanib sodium. The follow up was 6 months. The following parameters were determined before and after treatment: homocysteine, lipids (total cholesterol, triglycerides, HDL-c, LDL-c), C-Reactive Protein and fibrinogen. There were not statistically significant differences in parameters studied before and after treatment with both Pegaptanib sodium and Ranibizumab, except C-Reactive Protein. Of all patients analyzed, only 3 of them have initially C-Reactive Protein levels above normal levels and after antiangiogenic therapy, there was a significant increase in C-Reactive Protein. We have not found results in our study who to suspect that treatment with anti-VEGF in the patients with exudative age related macular degeneration increases cardiovascular risk predictors. However, after therapy was increased the CRP and fibrinogen may mean that anti-VEGF contribute an alteration of endothelial homeostasis in exudative AMD.

Sobha Sivaprasad,1 Richard C Browning,2 Carla Starita2 1Consultant Ophthalmologist, King’s College Hospital, Denmark Hill, London, 2Pfizer Ltd, Walton Oaks, Dorking Road, Tadworth, Surrey, UK Background: The purpose of this study was to evaluate the safety and tolerability of pegaptanib in patients with diabetic macular edema. Methods: An open-label, multicenter, noncomparative, one-year study of approximately 500patients was planned. Recruitment was terminated after enrollment of 46 patients. Enrolled patients were fully informed and reconsented; 12 patients elected to complete the study. Patients received intravitreal injections of pegaptanib 0.3 mg once every 6 weeks or less frequently, as determined by the investigator. Clinical benefit was evaluated after the patient received two or more injections. Ocular and nonocular adverse events were closely monitored throughout the study. Results: Compared with baseline, mean best-corrected visual acuity increased by week 6. Ten patients reported ocular-related adverse events, none of which were severe, and eight patients reported nonocular adverse events, two of which were severe but unrelated to study treatment. Three serious adverse events, unrelated to study treatment, were reported.Conclusion: In this limited set of patients with diabetic macular edema, pegaptanib appeared to be well tolerated with evidence of efficacy. Keywords: pegaptanib, diabetic macular edema, safety, tolerability

A 28-year-old man presented with bilateral vision loss. His best-corrected visual acuity (BCVA) was 0.3 in the right eye (OD) and 0.6 in the left eye (OS). Fundoscopy and fluorescein angiography showed angioid streaks encircling the optic discs of both eyes (OU). Spectral Domain Optical Coherence Tomography (SD-OCT) showed bilateral macular serous detachment. Systemic and ocular screening tests showed no specific cause for the angioid streaks. The patient had previously received pegaptanib sodium injection on three occasions, photodynamic therapy in OS, and no treatment in OD. Upon intravitreal injection of ranibizumab (twice in OU), subretinal fluid was nearly eliminated in OU. BCVA increased to 0.6 in OD and 0.9 in OS, and remained improved until 6 months after treatment.

We describe the case of an infant with a retinitis due to a postnatally acquired Cytomegalovirus, successfully treated at six months of life with three cycles of intravitreal injections of pegaptanib sodium. The therapy was effective in modulating the retinal disease activity in this immunocompetent child, without any side effect observed at a long-term follow up.

To evaluate the effect of 2 vascular endothelial growth factor (VEGF) isoforms (121 and 165) and 2 anti-VEGF compounds (ranibizumab and pegaptanib sodium) on the permeability of human retinal pigment epithelium (RPE) cells in vitro. The RPE permeability was assessed on ARPE19 cells grown onto inserts of polytetrafluoroethylene previously treated with ammonia gas plasma. Paracellular permeability to ions was measured by mean of transepithelial electrical resistance (TEER). Permeability to non-ionic molecules was gathered by the amount of fluorescein dextran (FD) passing across the monolayer within 2 hours. Only VEGF165 applied at the apical side of the monolayer induced a statistically significant decrease of TEER (p0.001). No changes in TEER were observed when pegaptanib sodium or ranibizumab were apically administered together with VEGF165. Both VEGF isoforms significantly increased permeability to 4 kDa dextran (p0.01). Apical administration of ranibizumab or pegaptanib sodium as well as coadministration of pegaptanib sodium with VEGF121 or VEGF165 induced a statistically significant increase of permeability to 4 kDa FD. Both VEGF isoforms and anti-VEGF compounds exert an effect on human RPE permeability in vitro.

To explore the efficacy and safety of pegaptanib sodium as maintenance therapy in Japanese patients with neovascular, age-related macular degeneration (AMD) after induction therapy (LEVEL-J study).A multi-center, prospective study was conducted at 21 medical institutions between 2009 and 2011. Of Japanese neovascular AMD patients with choroidal neovascularization who showed improvement in visual acuity (VA) with induction therapy, those who were scheduled for intravitreal injections of pegaptanib as maintenance therapy were recruited. LogMAR VA was assessed. Booster treatment (unscheduled treatment with other agents) was allowed during the study period if symptoms were judged to have worsened. Safety was assessed by monitoring adverse events and intraocular pressure (IOP).Of 75 patients included in the analysis, 80 % completed the 54-week study period. Their mean age was 74.7 ± 6.9 years, and 54 patients (72.0 %) were men. The mean number of pegaptanib injections was 5.7 ± 2.6. Booster treatment was not required in 40 eyes (53.3 %). Mean logMAR VA was 0.61 ± 0.31 before induction therapy, 0.26 ± 0.24 before maintenance therapy, and 0.29 ± 0.28 at 54 weeks. No notable change in VA was observed during maintenance therapy. Adverse events were reported in 4 patients (5.3 %), including increased intraocular pressure, cancer, gallstones and recurrence of breast cancer, but mean IOP remained stable during maintenance therapy.The results of this exploratory study suggest that maintenance therapy with pegaptanib is potentially an effective and well-tolerated option in Japanese patients with neovascular AMD in whom induction therapy has been successful.

Aims. To determine the effectiveness of second line treatments in patients with neovascular AMD who did not respond adequately to primary treatment. Methods. Retrospective, multicentre assessment. The frequency of primary treatment failure and outcomes of subsequent secondary treatment were assessed according to the type of primary treatment, type of CNV and change in BCVA over a 12 month period. Results. At the time of assessment 750 entries (750 treated eyes, 725 treated patients) had follow-up longer than 12 months. A treatment change required 7.7% subjects treated with ranibizumab, 20.5% with pegaptanib and 22% with PDT and verteporfin. Average BCVA of all patients at the beginning of primary treatment was 50.7 ± 3 letters and 43 ± 3.5 letters in 12 th month (P

WHAT IS ALREADY KNOWN ABOUT THIS SUBJECT • Diabetic macular oedema can develop at all stages of diabetic retinopathy (DR), and its prevalence increases with the duration of diabetes, affecting approximately 30% of diabetic patients after 25–30 years of disease. • Elevated concentrations of VEGF have been identified in aqueous humour and vitreous of patients with DR, and the severity of macular oedema is correlated with its vitreous concentrations. • Intravitreal anti-VEGFs exert a key role in the treatment of diabetic macular oedema. WHAT THIS STUDY ADDS • The present study, performed on 30 eyes of 30 patients, shows that intravitreal pegaptanib injections induced a significant reduction in mean central retinal thickness, with a parallel improvement in visual acuity. • For the first time a correlation between retinal morphologic and functional parameters in diabetic patients with macular oedema has been ascertained. • Our study demonstrates that the selective inhibition of VEGF-165 isoform by pegaptanib represents an effective treatment for diabetic macular oedema, as assessed throughout a 48 week follow-up. AIMS To study whether morphologic (foveal thickness, FT) variations of clinically significant macular oedema (CMO) in patients suffering from diabetes following intravitreal pegaptanib sodium (IVP) injection were associated with functional [macular sensitivity (MS) and colour discrimination (CD)] changes. METHODS A longitudinal, interventional, non-randomized study was performed. FT was assessed by optical coherence tomography (OCT), MS by microperimetry, best-corrected visual acuity (BCVA) by early treatment diabetic retinopathy study charts (ETDRS) and CD by Farnswoth-Munsell test. The treatment protocol consisted of three consecutive injections (0.3 mg/0.05 ml; baseline, week 6 and week 12). Follow-up checks were scheduled at 18, 24, 36 and 48 weeks, after injections. RESULTS Thirty eyes of 30 patients with clinically significant CMO were included for analysis. After IVP a significant decrease of FT occurred with a mean reduction from baseline of 56.9% (P= 0.0001). An improvement of functional parameters was recorded in all patients (BCVA from 18.2 ± 8.5 letters to 25.5 ± 8.4 letters, P < 0.005, MS from 8.6 ± 2.16 dB to 10.6 ± 2.61 dB, P < 0.001, colour analysis from 376.1 ± 125.6 TES to 116 ± 34.6 TES, P= 0.0001). A statistically significant correlation between FT and BCVA as well as MS and CD was also found. Neither ocular nor systemic adverse events were reported. CONCLUSIONS Intravitreal pegaptanib significantly reduced FT, with a concomitant improvement of MS and CD. This association emphasizes the efficacy of IVP in the treatment of CMO.

Based on the success of intravitreal therapies targeting vascular endothelial growth factor,1–3 some of which suggested efficacy independent of lesion subtype,3 we investigated the benefit and safety of pegaptanib sodium in patients with early neovascular age-related macular degeneration (NV-AMD) versus those with established lesions.4 PERSPECTIVES (NCT00327470) was a 102-week, open-label trial. Participants aged ≥50 years with evidence of NV-AMD in at least one eye, clear ocular media and a visual acuity >25 Early Treatment of Diabetic Retinopathy Study (ETDRS) letters, and a lesion

Purpose: Comparison of efficacy and safety of combination therapy of photodynamic therapy (PDT) with Ranibizumab and Pegaptanib sodium respectively for subfoveal choroidal neovascularization in age-related macular degeneration. Design: Prospective, comparative, interventional case series. Materials and methods: Twenty patients received either PDT with 0.5 mg Ranibizumab (group 1; n = 10) or PDT with 0.3 mg Pegaptanib sodium (group 2; n = 10). Three doses of anti-VEGF injections were given monthly followed by reinjections pro re nata basis in both groups. Data recorded included visual acuity (VA), central macular thickness (CMT) by optical coherence tomography and greatest linear dimension (GLD) by angiography at 1 week, 1, 2, 3 and 6 months follow-up. Results: At 6 months follow-up, groups 1 and 2 showed 13.7 ± 10.25 and 2.8 ± 6.87 letters gain (p = 0.020), CMT decreased to 78.2 ± 61.28 μm and 13 ± 23.83 μm (p = 0.011) and decrease in GLD from baseline was 640 ± 687.6 μm and 584.0 ± 565.15 μm respectively (p = 0.971).

Age-related macular degeneration (AMD) is the leading cause of severe visual loss and blindness over the age of 50 in developed countries. Vascular endothelial growth factor (VEGF) is considered as a critical molecule in the pathogenesis of choroidal neovascularization (CNV), which characterizes the neovascular AMD. Anti-VEGF agents are considered the most promising way of effectively inhibition of the neovascular AMD process. VEGF is a heparin-binding glycoprotein with potent angiogenic, mitogenic and vascular permeability-enhancing activities specific for endothelial cells. Two anti-VEGF agents have been approved by the US Food and Drug Administration (FDA) for the treatment of neovascular AMD. Pegaptanib sodium, which is an aptamer and ranibizumab, which is a monoclonal antibody fragment. Another humanized monoclonal antibody is currently off-label used, bevacizumab. This paper aims to discuss in details the effectiveness, the efficacy and safety of these three anti-VEGF agents. New anti-VEGF compounds which are recently investigated for their clinical usage (VEGF-trap, small interfering RNA) are also discussed for their promising outcomes.

The neovascular form of age-related macular degeneration (AMD), called wet-AMD or choroidal neovascularisation, begins with damage to the outer retinal cells and retinal pigment epithelium (RPE), which elicits a cascade of inflammatory and angiogenic responses leading to neovascularisation under the macula. Studies showed that oxidative damage, chronic inflammation of the RPE and complement misregulation work at different steps of this disease. After established neovascularisation, several pro- and antiangiogenic agents start to play an important role. Vascular endothelial growth factors (VEGFs) are the most specific and potent regulators of angiogenesis, which are inhibited by intravitreal injections of ranibizumab, bevacizumab, VEGF Trap, pegaptanib sodium and other agents under investigation. Pigment epithelium-derived factor, on the other hand, shows neuroprotective and antiangiogenic activities. Hepatocyte growth factor (HGF) has a mitogenic effect on a wide range of epithelial and endothelial cells, and it is inhibited by an anti-HGF monoclonal antibody. Platelet-derived growth factor is a potent chemoattractant and mitogen for both fibroblasts and retinal RPE cells, which has been inhibited experimentally by VEGF Trap and human anti-platelet-derived growth factor-D monoclonal antibody. Fibroblast growth factor-2 has pleiotropic effects in different cell and organ systems, and it is blocked by anti-FGF antibodies, with a greater benefit regarding antiangiogenesis when combined treatment with anti-VEGF is performed. Tumour necrosis factor alpha is expressed in the retina and the choroid, and its blockade in choroidal neovascularisation includes the use of monoclonals such as infliximab. This paper reviews the most important cytokines involved in the pathogenesis of wet-AMD, with emphasis on potential combined therapies for disease control.

Purpose: To investigate the effectiveness of intravitreal pegaptanib sodium injection in patients with choroidal neovascularization (CNV) secondary to angioid streaks.Methods: Five eyes of four patients with angioid streaks with CNV underwent uneventful intravitreal injection of pegaptanib sodium (0.3 mg/90 µL). Patients were followed up with Snellen visual acuity testing, optical coherence tomography, and fundus fluorescein angiography.Results: The median follow-up time was 18 months (range: 15 to 24 months). Visual acuity improved in two eyes, and stabilized in three out of five eyes. At final examination, CNV regressed with resolution of subretinal fluid in all but one patient with bilateral CNV from angioid streaks.Conclusion: Intravitreal pegaptanib sodium for CNV associated with angioid streaks led to inactivation of most of the CNV lesions, stabilizing or improving visual acuity in all eyes.

Purpose The purpose of this study was to determine the incidence of infectious endophthalmitis after intravitreal injection of antivascular endothelial growth factor medications using 2% topical lidocaine gel anesthesia. Methods Retrospective chart review of 4690 consecutive intravitreal injections of antivascular endothelial growth factor medications using 2% topical lidocaine gel anesthesia. All patients had at least 6 weeks of follow-up. Results A total of 608 patients underwent intravitreal injection of antivascular endothelial growth factor medications during the study period. There were 428 injections of pegaptanib sodium, 1841 injections of bevacizumab, and 2421 injections of ranibizumab. There were no cases of infectious endophthalmitis. The per-injection infection rate was 0.0% (95% confidence interval, 0.0-0.06%). Conclusion The incidence of infectious endophthalmitis after intravitreal injection of antivascular endothelial growth factor medications using 2% topical lidocaine gel anesthesia is low.

Aim To assess the efficacy of pegaptanib as maintenance therapy in neovascular age-related macular degeneration (NV-AMD) patients after induction therapy. Methods A phase IV, prospective, open-label, uncontrolled exploratory study including subjects with subfoveal NV-AMD who had had one to three induction treatments 30–120 days before entry and showed investigator-determined clinical/anatomical NV-AMD improvement. Lesions in the study eye were: any subtype, 12 or fewer disc areas; postinduction centre point thickness (CPT) 275 μm or less or thinning of 100 μm or more (optical coherence tomography); visual acuity (VA) 20/20–20/400. Intravitreal pegaptanib 0.3 mg was administered as maintenance every 6 weeks for 48 weeks with follow-up to week 54. Booster treatment additional unscheduled treatment for wet age-related macular degeneration, was allowed in the study eye at the investigators9 discretion for clinical deterioration. Results Of 568 enrolled subjects, 86% completed 1 year of pegaptanib. Mean VA improvement during induction (49.6 to 65.5 letters) was well preserved (54-week mean 61.8 letters). Mean CPT was relatively stable during maintenance (20 μm increase during the study). Fifty per cent did not receive unscheduled booster treatment to week 54; 46% did have one such booster (mean 147 days after maintenance initiation). Conclusions An induction-maintenance strategy, using non-selective then selective vascular endothelial growth factor (VEGF) inhibitors, could be considered for NV-AMD. This approach may have particular relevance for patients with systemic comorbidities who require long-term anti-VEGF therapy for NV-AMD.

The purpose of this study was to report the successful management of a case of choroidal neovascular membrane secondary to persistent fetal vasculature syndrome in an infant using intravitreal pegaptanib sodium (Macugen).A 14-month-old male infant developed a peripapillary choroidal neovascular membrane 10 months after lens-sparing vitrectomy that he underwent for persistent fetal vasculature syndrome and confirmed on angiography and optical coherence tomography. A single dose of intravitreal pegaptanib sodium (0.3 mg) was administered after informed consent was obtained. Angiography and optical coherence tomography scans were performed at 6 and 14 weeks after the injection to determine the result.Six weeks after the single injection, the lesion reduced in size and exhibited less leakage on angiography. At 14 weeks, leakage was absent, and the lesion had involuted.Choroidal neovascular membrane in a case of persistent fetal vasculature syndrome is rare. This report shows that intravitreal pegaptanib sodium is highly effective in treating this lesion even in an infant.

The study aim was to evaluate the short-term safety and efficacy of pegaptanib sodium injections (Macugen, Eyetech Pharmaceuticals, Inc., New York, NY) in the compassionate-use therapy of choroidal neovascularization (CNV) secondary to age-related macular degeneration (AMD). Intravitreal pegaptanib was used to treat 41 eyes in 40 patients with CNV. Injections were given every 6 weeks, and a minimum of three injections were planned. The mean change in BCVA for all lesions was a loss of 0.03 Snellen lines. Seven eyes (17.1%) gained more than 3 lines, three (7.31%) lost 6 lines or more, and in 75.6% the BCVA stabilized or improved.

To evaluate the short-term toxic effects of pegaptanib sodium on retinal function. At present, intraocular anti-vascular endothelial growth factor (VEGF) therapy is the primary choice of treatment for neovascular maculopathy. The isoform VEGF(165) is specifically inhibited by pegaptanib sodium. Therefore, since VEGF(165) has neuroprotective effects against apoptosis of neuronal cells, blockage of VEGF(165) by pegaptanib could induce retinal dysfunction. In the present study, we used an electrophysiological technique for testing retinal toxicity in order to evaluate the short-term toxic effects of pegaptanib sodium on retinal function in a model of isolated perfused vertebrate retina.Isolated bovine retinas were perfused with an oxygenated, pre-incubated nutrient solution. Electroretinograms (ERGs) were recorded as trans-retinal potentials using Ag/AgCl electrodes. Pegaptanib sodium (0.006, 0.06, or 0.2 mg/ml) and solvent carrier were added to the nutrient solution for 45 min. ERGs were monitored before, during, and after exposure.No significant reductions of b-wave (p = 0.357, p = 0.31, and p = 0.11, respectively) or a-wave (p = 0.189, p = 0.46, and p = 0.23, respectively) amplitudes were detected during application of pegaptanib (0.006, 0.06, or 0.2 mg/ml). The solvent carrier alone had no effect on ERG b- or a-waves (p = 0.98 and p = 0.42, respectively). During washout, ERG amplitudes of all test series remained unchanged.Results suggest that both pegaptanib sodium and its solvent carrier have good safety profiles. Intraocular application of 0.3 mg pegaptanib sodium induced no significant changes in ERGs in our ex vivo model and, thus, appears to be safe.

Purpose: To report the functional and anatomical outcomes resulting from the use of intravitreal pegaptanib sodium (Macugen®) in patients with diabetic macular oedema (DMO). Methods: We conducted a retrospective outcome analysis, by optical coherence tomography (OCT) and best-corrected visual acuity (BCVA), of eyes with DMO treated with intravitreal pegaptanib sodium. Moreover, we evaluated the foveal transverse photoreceptor (PR) band integrity in the OCT images at the time of the last follow-up visit. Results: Sixty-three eyes of 48 patients with a minimum of 6 months of follow-up were included for analysis. Intravitreal pegaptanib was found to produce significant improvements in mean BCVA (p = 0.019) and reductions in mean central macular thickness (CMT) (p

Background: To compare the efficacy of intravitreal pegaptanib (IVP) versus panretinal laser photocoagulation (PRP) in the treatment of active proliferative diabetic retinopathy (PDR). Methods: A prospective, randomized, controlled, open-label, exploratory study. Twenty subjects with active PDR were randomly assigned at a 1:1 ratio to receive treatment in one eye either with IVP (0.3 mg) every 6 weeks for 30 weeks, or with PRP laser. Efficacy endpoints included regression of retinal neovascularisation (NV), and changes from baseline in best-corrected visual acuity (BCVA), and foveal thickness. Safety outcomes included observed and reported adverse events. Results: In 90% of randomized eyes to IVP, retinal NV showed regression by week 3. By week 12, all IVP-eyes were completely regressed, and was maintained through week 36. In the PRP-treated group, at week 36, two eyes demonstrated complete regression, two showed partial regression, and four showed persistent active PDR. Mean change in BCVA at 36 weeks was +5.8 letters in pegaptanib-treated eyes and -6.0 letters in PRP-treated eyes. Only mild to moderate transient ocular adverse events were reported with pegaptanib. Conclusions: IVP produces short-term marked and rapid regression of diabetic retinal NV. Regression of NV was maintained throughout the study and at the final visit.