Cost-effectiveness analysis of ocriplasmin versus watchful waiting for treatment of symptomatic vitreomacular adhesion in the US.

Aim: Evaluate the cost-effectiveness of ocriplasmin in symptomatic vitreomacular adhesion (VMA) with or without full-thickness macular hole ≤400 µm versus standard of care. Methods: A state-transition model simulated a cohort through disease health states; assignment of utilities to health states reflected the distribution of visual acuity. Efficacy of ocriplasmin was derived from logistic regression models using Ocriplasmin for Treatment for Symptomatic Vitreomacular Adhesion Including Macular Hole trial data. Model inputs were extracted from Phase III trials and published literature. The analysis was conducted from a US Medicare perspective. Results: Lifetime incremental cost-effectiveness ratio was US$4887 per quality-adjusted life year gained in the total population, US$4255 and US$10,167 in VMA subgroups without and with full-thickness macular hole, respectively. Conclusion: Ocriplasmin was cost effective compared with standard of care in symptomatic VMA.

A Patient-Reported Outcomes Analysis Of Lanreotide In The Treatment Of NETs Patients With Carcinoid Syndrome: Evidence From The ELECT Trial.

PURPOSE: The purpose of this analysis of patient-reported outcomes from the ELECT (Evaluation of Lanreotide Depot/Autogel Efficacy and Safety as a Carcinoid Syndrome Treatment) trial (NCT00774930) was to explore the effect of lanreotide on symptoms of carcinoid syndrome. Specifically, this post hoc analysis was designed to identify the most important patient-reported outcomes for patients in ELECT. METHODS: The post hoc analysis of ELECT, a placebo-controlled study of lanreotide in patients with neuroendocrine tumors, evaluated patient-reported outcomes during the double-blind phase of the trial, specifically daily diarrhea and flushing symptoms, octreotide rescue use, and the EORTC QLQ-C30 and QLQ-GINET21 questionnaires at baseline and week 12. Principal component (PC) analysis was applied on baseline data to identify independent variable clusters and clinically meaningful summary measures that highly correlated to these PCs. From those, the minimum clinical important differences were derived so to perform a responder analysis. RESULTS: The three largest PCs captured 42.9% of the variation among baseline variables. The C30 summary score (C30-SS), diarrhea burden, and flushing burden were highly correlated with PC1, PC2, and PC3, respectively. Lanreotide patients were more likely to experience an improvement on the C30-SS (risk ratio [RR] 2.42; P=0.023), diarrhea burden (RR 2.85; P=0.005), and flushing burden (RR 1.39; P=0.31) compared to placebo patients. Lanreotide-treated patients have a higher probability of being a responder on at least one of the three domains of C30-SS, diarrhea burden, or flushing burden compared to placebo patients (RR 1.48; P=0.06). CONCLUSION: The higher response rates in the diarrhea burden are consistent with the previously reported effects of lanreotide on octreotide rescue medication use, while the findings of a greater efficacy of lanreotide vs placebo in the quality-of-life domains represent a novel aspect in the benefits of lanreotide. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT00774930.

Visual Function Response to Ocriplasmin for the Treatment of Vitreomacular Traction and Macular Hole: The OASIS Study.

Purpose: To assess the effect of ocriplasmin on visual function response (VFR) measured using visual acuity (VA) and vision-related quality of life, and to quantify the association between release of vitreomacular adhesion (VMA) at day 28 and VFR. Methods: Prespecified analysis of secondary endpoints from a randomized controlled trial. Of 220 participants with symptomatic VMA/vitreomacular traction (VMT), including VMT associated with a macular hole up to 400 µm, 146 received a single intravitreal injection of 125 µg ocriplasmin and 74 a sham injection. Based on principal components analysis results, a VFR was defined as either a VA improvement of ≥2 lines or an improvement exceeding the minimal clinically important difference (MCID) in the composite or the mental health subscale scores of the Visual Function Questionnaire (VFQ-25). The MCID was estimated using the standard error of measurement approach. The main outcome measure was the VFR at month 6, with further assessments at months 12 and 24. Results: The MCID was estimated at 3.71 points for the VFQ-25 composite score and 10.71 for the VFQ-25 mental health subscale score. A VFR occurred in 51.0% of ocriplasmin versus 23.3% of sham participants (P = 0.0001). The VFR was maintained through months 12 and 24: 53.1% and 50.3% in ocriplasmin versus 21.9% and 20.5% in sham participants, respectively (P < 0.0001). Resolution of VMA at day 28 significantly increased the odds of a VFR at each assessment period. Conclusions: Treatment with ocriplasmin compared with sham resulted in a significant improvement in VFR. The 6-month treatment effect was sustained at months 12 and 24.

Visual function response to ocriplasmin for the treatment of vitreomacular traction and macular hole.

PURPOSE: To assess the effect of an intravitreal ocriplasmin injection on visual function, measured using visual acuity (VA) and vision-related quality of life. METHODS: Post hoc analysis of prespecified secondary end-points in two multicentre, randomized, double-masked, phase 3 clinical trials. A total of 652 participants with symptomatic vitreomacular adhesion were enrolled, of whom 464 received a single intravitreal injection of 125 µg ocriplasmin and 188 received a single intravitreal placebo injection. Based on principal components analysis results, visual function response (VFR) was defined as either a VA improvement of ≥2 lines; or an improvement in the composite score of the National Eye Institute Visual Function Questionnaire (VFQ-25) exceeding the minimal clinically important difference (MCID), estimated using the standard error of measurement approach; or an improvement in the VFQ-25 driving subscale score exceeding the MCID. The main outcome measure was VFR at 6 months. RESULTS: A VFR occurred in 55.1% of the ocriplasmin group versus 34.2% of the placebo injection group (p < 0.0001). This comprised 23.7% versus 11.2% (p = 0.0003) with a ≥ 2-line VA improvement, 35.9% versus 22.7% (p = 0.0016) for the VFQ-25 composite score, and 10.2% versus 6.2% (p = 0.1697) for the driving subscale. CONCLUSION: Ocriplasmin produces a clinically meaningful visual function benefit.

Non-interventional study AI463-12 of real-world chronic HBV infection management--baseline characteristics and treatment patterns of Polish patients kohort.

AIM: This longitudinal non-interventional study aims to describe the demographics data disease characteristics and clinical management of a cross-sectional CHB patient population in Poland treated in regional medical centers. MATERIAL AND METHODS: [corrected] Between March 2008 and December 2010 we observed patients with HBV related liver disease from 5 medical centers in Poland, both sexes, > 18 years old. At baseline, we used a case report form to extract data from patient charts, comprising: sociodemographic data; disease characteristics, HBeAg/ antiHBeAg status, genotype HBV; co-morbidities; viral load, liver biopsy and ALT levels in previous 12 months; treatment history in previous 12 months; current CHB treatment; changes in disease characteristics and CHB management; time from diagnosis to the therapy and resource utilization and any reasons for termination of follow-up. Written informed consent was obtained from all participants RESULTS: The analysis population included 253 patients (94 treated and 159 non-treated at baseline) mostly male (69.1 vs. 56.6). Patients in treated group compared with untreated group were: significantly older (mean 42.6 vs. 37.5 years respectively, p < 0.001), observed longer since diagnosis(3.9 vs.2.9 years), with higher rate of HBeAg(+)(42.6% vs.5.1%), lower ALT activity, and higher VL HBV DNA PCR. Of the 53% of treated patients, the most frequently prescribed anti-HBV drugs were: Lamivudine (53%), Entecavir (23.7%), Pegylated IFN-alfa2a (23.7%), Adefovir (11.1%). During 24 months of follow-up in treated group 13(36.1%) patients underwent a treatment switch to another nucleosi(-ti)de analogue, in one (2.8%) patient another analogue was added, and in 25 (69.4%)patients the therapy was stopped. The proportion of all patients treated with monotherapy at the end of follow-up was 99.4%, unfortunately mostly with Lamivudine-49.3%. SUMMARY: 1. Despite the several methodological limitations usually associated with this type of observation, the collected data does characterize the demographics of polish patients chronically infected with HBV well, provides some insights into the determinants of treatment initiation and the clinical management of patients in real-word settings. 2. These results indicate that in clinical practice in 5 medical non-academic centers in Poland, European guidelines regarding the qualification to HBV treatment were followed, but there were discrepancies between the initial treatment decisions in real-life current clinical practice and guideline recommendations

The burden of viral hepatitis C in Europe: a propensity analysis of patient outcomes.

OBJECTIVE: Hepatitis C virus (HCV) affects 170 million patients worldwide and is the leading cause of liver cirrhosis and hepatocellular carcinoma. The aim of the current study is to examine the burden of HCV in the European Union (EU) from a patient perspective. METHODS: Using data from the 2010 EU National Health and Wellness Survey, patients who reported a diagnosis of HCV (n=332) were compared with a propensity-score-matched non-HCV control group (n=332) on measures of quality of life (using the SF-12v2), work productivity, and healthcare resource utilization in the past 6 months. All analyses applied sampling weights to project to the respective country populations. RESULTS: Projected prevalence estimates of HCV were 0.59% in France, 0.44% in Germany, 1.42% in Italy, 0.82% in Spain, and 0.35% in the UK. HCV patients reported significantly lower levels of emotional role limitations (means=66.4 vs. 70.6, P=0.040), physical functioning (means=63.8 vs. 71.9, P=0.001), general health (means=48.3 vs. 54.4, P=0.004), bodily pain (means=64.3 vs. 70.8, P=0.002), and physical component summary scores (means=42.9 vs. 45.3, P=0.002) than the matched controls. Patients with HCV also reported significantly higher levels of presenteeism (means=27.1 vs. 21.0%, P=0.044) and a greater number of physician visits in the past 6 months (means=9.9 vs. 6.7, P<0.001). CONCLUSION: Using a population-based survey methodology and a propensity-score matching analysis, these results add to the literature by documenting the significant effect that HCV has on a variety of both humanistic and economic outcomes in the EU.