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Technology has changed the way we approach medical care health data is constantly being generated, medical discoveries are progressing more rapidly, and individuals are more connected across the world than ever before. Backpack Health is a global personal health record platform that harnesses the power of technology to connect users to their primary health data sources, the medical community, and researchers. By syncing with existing patient portals, health data can be stored on the Backpack Health platform and easily accessed and controlled by users in one connected interface. Individuals manage and collate their current and past conditions, genetic test results, symptoms, medications, procedures, labs, and other health data. Users are empowered to disseminate their information to clinicians, researchers, foundations, and pharmaceutical and biotechnology companies they connect with through the Backpack Health application. Here, we describe how two rare disease advocacy groups, The Marfan Foundation and Project Alive, utilize Backpack Health to connect with their target populations. Through secure transfer of pseudonymized data, groups can query their members to improve understanding of clinical features and to facilitate meaningful research. Responses to the groups' surveys show strong member engagement with high completion rates and increases in new Backpack Health users when surveys are deployed. Data from these surveys have been published and used to better inform clinical outcomes for treatment trials. By connecting users directly to the foundations, clinicians, researchers, and industry partners working on their condition, Backpack Health is instrumental in fast-tracking medical discoveries and treatment for rare diseases.Direct-acting antiviral (DAA) therapy has transformed the management of human immunodeficiency virus (HIV) and hepatitis C (HCV) coinfected patients with advanced liver disease. STOP-Coinfection was a multicenter prospective and retrospective, open-label study using sofosbuvir-based DAA therapy to treat HIV/HCV-coinfected participants pre- or post-liver transplant (LT). Sixty-eight participants with end-stage liver disease (Child-Turcotte-Pugh score ≥7 and Model for End-Stage Liver Disease score 6-29) were enrolled, 26 had hepatocellular carcinoma. Forty-two participants were treated pre-LT and 26 post-LT. All participants completed therapy without need for dose reduction or transfusion; eight required two or more courses of therapy. Ninety-three percent achieved a sustained virologic response and DAA therapy was well tolerated. Despite HCV cure, 12 end-stage liver disease participants required subsequent LT, 7 for decompensated liver disease. Thirteen participants died, 10 with decompensated liver disease pre-LT and three post-LT. Overall, transplant free survival was 42.8% at 4 years and post-LT survival was 87.9% at 5 years. We conclude that sofosbuvir-based DAA therapy is safe and highly effective in HCV-HIV patients with decompensated liver disease and post-LT, with post-LT survival rates comparable to other indications. This removes one of the last barriers to liver transplantation in this challenging cohort of recipients.

Upper airway dyspnoea is a challenging condition in which assessing the discomfort experienced by the patient is essential. There are three patient-reported outcome (PRO) instruments developed particularly for this patient group, none of which is available in Swedish. The aim of this study was to translate the Dyspnea Index (DI) into Swedish and validate the instrument for use in the Swedish-speaking population by investigating its basic psychometric properties.

A prospective instrument validation study.

Tertiary referral centre.

Fifty-three (n=53) patients with upper airway dyspnoea and 19 healthy controls.

The questionnaire was translated into Swedish (swDI) with a forward-backward method. Reliability, repeatability, responsiveness and construct validity were assessed by asking the subjects to complete the swDI, a visual analog scale (VAS) at exertion and at rest and the Voice Handicap Index (VHI).

The swDI showed excellent internal consistency (Cronbach's α 0.85) and repeatability (interclass correlation coefficient 0.87 and Pearson's r .89) in the patient group. No ceiling effect was observed (maximum score achieved was 39; 85% of the patients scored≤36). SwDI scores moderately correlated with VAS at exertion (r .59) and at rest (r .42), yet poorly with the VHI (r .36). The effect size (ES) was 3.8.

The swDI is a valid, robust and reliable questionnaire for self-assessment in Swedish-speaking patients with upper airway obstruction. A future anchor-based longitudinal study is needed to assess the smallest detectable change (SDC) and minimal important change (MIC) that were not estimated in our study.

The swDI is a valid, robust and reliable questionnaire for self-assessment in Swedish-speaking patients with upper airway obstruction. SHR-3162 clinical trial A future anchor-based longitudinal study is needed to assess the smallest detectable change (SDC) and minimal important change (MIC) that were not estimated in our study.Tetraspanin CD63 has been widely implicated in tumour progression of human malignancies. However, its role in the tumorigenesis and metastasis of hepatocellular carcinoma (HCC) remains unclear yet. In the present study, we aimed to investigate the specific function and underlying mechanisms of CD63 in HCC progression. CD63 expression in HCC tissues was detected using immunohistochemistry and quantitative real-time PCR analyses; effects of CD63 on HCC cell proliferation and migration were investigated by CCK-8 assay, colony formation assay, transwell assay and a xenograft model of nude mice. RNA-sequencing, bioinformatics analysis, dual-luciferase reporter assay and Western blot analysis were performed to explore the underlying molecular mechanisms. Results of our experiments showed that CD63 expression was frequently reduced in HCC tissues compared with adjacent normal tissues, and decreased CD63 expression was significantly associated with larger tumour size, distant site metastasis and higher tumour stages of HCC.