A study in high-risk, maximally pretreated patients to determine the potential use of PCSK9 inhibitors at various thresholds of total and LDL cholesterol levels.

PURPOSE OF THE STUDY: Statins and ezetimibe reduce low-density lipoprotein cholesterol (LDL-c) and cardiovascular disease (CVD) risk. Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors lower LDL-c by 50%-70% and might be useful in refractory patients. The National Institute for Health and Care Excellence (NICE) technology appraisal guidance (TAG) recommends use of these drugs in secondary prevention and familial hypercholesterolaemia (FH) at differing LDL-c thresholds. We have estimated the proportion of patients in whom this third-line drug might be useful. STUDY DESIGN: We used data from a lipid-lowering audit programme to study 72 with FH and/or CVD of 271 patients referred over 12 months who failed to achieve target total cholesterol (TC) and LDL-c levels. All 72 patients were treated with ezetimibe, and 69 cases also received statins. We used LDL-c thresholds 1.5-5.5 mmol/L to estimate how many of these refractory patients could benefit from PCSK9 inhibitors. RESULTS: In 72 patients, TC and LDL-c targets were not met by 64 and 53 patients, respectively. We judged using the NICE TAG that only one patient (1.4% ezetimibe requiring and 0.4% total referrals) required a PCSK9 inhibitor. CONCLUSIONS: We determined that the proportion of patients eligible for a PCSK9 inhibitor at various TC and LDL-c levels is modest. This may reflect the use of all available statins in UK lipid clinics often at non-daily frequency. We suggest that cost-effective use of PCSK9 inhibitors requires prescribing being restricted to clinicians working in specialised lipid clinics.

Childhood blindness in a rural population of southern India: prevalence and etiology.

PURPOSE: To determine the prevalence and etiology of childhood blindness in a rural population in southern India through a population based study. METHODS: A cross sectional, house-to-house survey to screen for childhood blindness included 14,423 children < 16 years. Blindness was defined as best corrected visual acuity < 3/60 in the better eye. The first stage of screening for blindness was done by interns under supervision of ophthalmology residents. Senior residents examined the referred cases from the first stage. Those detected to be blind were brought to a tertiary care hospital for detailed examination. RESULTS: Fifty-four children were referred after first stage of screening of 13,241 children. Of these 14 were bilaterally blind giving a prevalence of 1.06/1000 (95% confidence interval (CI), 0.50 to 1.61); 6 (42.9%) had lens and related complications, 4 (28.6%) had globe anomalies (2 Microphthalmos and 2 Anophthalmos), 2 (14.3%) had retinal dystrophy and 1 (7.1%) each of glaucoma and optic atrophy. Among the parents of blind children, 71.4% (p = 0.002) had consanguineous marriage (83.3% in cataract blind children). CONCLUSIONS: More than half of the blindness detected was potentially avoidable. Genetic counseling, early identification and access to tertiary care would reduce the burden of childhood blindness in the local community.