Additional file 1: of The impact of lipoprotein lipase deficiency on health-related quality of life: a detailed, structured, qualitative study

HRQoL during the worst attack of pancreatitis and at the interview, assessed by EQ-5D-3L VAS. Score of 0 = worst state imaginable; score of 100 = best state imaginable. EQ-5D-3L EuroQoL 5 domains, 3 levels; HRQoL health-related quality of life; VAS visual analogue scale. (PDF 838 kb

The effect of volanesorsen treatment on the burden associated with familial chylomicronemia syndrome: the results of the ReFOCUS study

<p><b>Background</b>: Volanesorsen, an investigational inhibitor of apoC-III synthesis, significantly reduced triglyceride levels in clinical trials in patients with familial chylomicronemia syndrome (FCS), a rare genetic disorder characterized by marked chylomicronemia leading to a spectrum of symptoms, including recurrent abdominal pain and episodes of potentially fatal acute pancreatitis (AP).</p> <p><b>Objective</b>: To determine the effect of volanesorsen on burden of disease on patients with FCS</p> <p><b>Methods</b>: ReFOCUS was a retrospective global web-based survey open to patients with FCS who received volanesorsen for ≥3 months in an open-label extension study. The survey included questions about patients’ experiences before and after volanesorsen treatment.</p> <p><b>Results</b>: Twenty-two respondents had received volanesorsen for a median of 222 days. Volanesorsen significantly reduced the number of symptoms per patient across physical, emotional, and cognitive domains. Significant reductions from baseline were reported for steatorrhea, pancreatic pain, and constant worry about an attack of pain/AP. Respondents reported that volanesorsen improved overall management of symptoms and reduced interference of FCS with work/school responsibilities. Reductions in the negative impact of FCS on personal, social, and professional life were also reported.</p> <p><b>Conclusions</b>: Treatment with volanesorsen has the potential to reduce disease burden in patients with FCS through modulation of multiple symptom domains.</p

Diabetes Dyslipidemia

<p><strong>Article full text</strong></p> <p><br> The full text of this article can be found <a href="https://link.springer.com/article/10.1007/s13300-016-0167-x"><b>here</b>.</a><br> <br> <strong>Provide enhanced digital features for this article</strong><br> If you are an author of this publication and would like to provide additional enhanced digital features for your article then please contact <u>[email protected]</u>.<br> <br> The journal offers a range of additional features designed to increase visibility and readership. All features will be thoroughly peer reviewed to ensure the content is of the highest scientific standard and all features are marked as ‘peer reviewed’ to ensure readers are aware that the content has been reviewed to the same level as the articles they are being presented alongside. Moreover, all sponsorship and disclosure information is included to provide complete transparency and adherence to good publication practices. This ensures that however the content is reached the reader has a full understanding of its origin. No fees are charged for hosting additional open access content.<br> <br> Other enhanced features include, but are not limited to:<br> • Slide decks<br> • Videos and animations<br> • Audio abstracts<br> • Audio slides<u></u></p> <p> </p> <p> </p

Summary plot of ICC values for all experiments in relation to reliability of assessment of CCM parameters.

<p>Summary plot of ICC values for all experiments in relation to reliability of assessment of CCM parameters.</p

Examples of CCM images.

<p>A) a high quality CCM image of sub basal nerves from the central cornea (nerves are vertical); B) A CCM image with a pressure line (indicated with an arrow); C) A CCM image of sub basal nerves from the peripheral cornea (oblique nerves); D) a CCM image with overlapping corneal layers (stroma and sub-basal nerve plexus) as can be seen with the keratocytes in the inferior and peripheral zone; E) a low contrast CCM image; F) inferior whorl of the cornea.</p

Corneal nerve parameters expressed as mean ±SD for each observer with significant difference and ICC value with significance.

<p>Corneal nerve parameters expressed as mean ±SD for each observer with significant difference and ICC value with significance.</p

Intra class correlation coefficient (ICC) and the mean± SD of differences between different observers for corneal nerve fibre density (CNFD), corneal nerve branch density (CNBD) and corneal nerve fibre length (CNFL).

<p>Intra class correlation coefficient (ICC) and the mean± SD of differences between different observers for corneal nerve fibre density (CNFD), corneal nerve branch density (CNBD) and corneal nerve fibre length (CNFL).</p

On-Treatment Adverse Events Reported in at Least 10% of Mipomersen Patients.

a<p>This patient also had another acute myocardial infarction occurring during post-treatment follow-up, which was fatal.</p>b<p>Events not related to study drug in the opinion of the investigator.</p>c<p>3 of 4 events of angina pectoris in the mipomersen group were considered drug-related. The 4^th event and the event in the placebo group were considered not drug-related.</p>d<p>Considered possibly drug-related.</p

Baseline Characteristics.

<p>Abbreviations: BMI, body mass index; Max, maximum; Min, minimum; SD, standard deviation.</p>a<p>Metabolic syndrome determined according to the American Heart Association and the National Heart, Lung, and Blood Institute definition.</p

Laboratory Abnormalities of Interest.

<p>Abbreviations: ALT, alanine aminotransferase; ULN, upper limit of normal range.</p>a<p>Not all laboratory abnormalities were reported as AEs.</p>b<p>The numbers after ‘n  = ’ are the total numbers of male/female patients in the placebo group and in the mipomersen group, respectively.</p>c<p>Percentages are calculated out of the total number of treated male or female patients, respectively, for the particular treatment group.</p