Congenital paraesophageal hernia with gastric outlet obstruction in a neonate with Cornelia de Lange Syndrome.

We describe a case of a newborn being treated for encephalopathy and seizures, whose radiographs since the first day of life demonstrate a persistent ovoid lucency over the central lower chest. A CT performed confirmed a type IV hiatal hernia, which is defined as a paraesophageal type hernia containing a portion of the abdominal viscera. This infant's hernia included the distal stomach, pylorus, and proximal duodenum. There was no volvulus or ischemic change at surgery. The patient underwent successful reduction, fundoplication, and gastrostomy placement with hospital discharge after further stabilization of additional medical problems. Genetic testing later confirmed Cornelia de Lange Syndrome Type V, which has been associated with gastrointestinal manifestations and congenital diaphragmatic hernias.

Technology-Enabled Clinical Trials: Transforming Medical Evidence Generation.

The complexity and costs associated with traditional randomized, controlled trials have increased exponentially over time, and now threaten to stifle the development of new drugs and devices. Nevertheless, the growing use of electronic health records, mobile applications, and wearable devices offers significant promise for transforming clinical trials, making them more pragmatic and efficient. However, many challenges must be overcome before these innovations can be implemented routinely in randomized, controlled trial operations. In October of 2018, a diverse stakeholder group convened in Washington, DC, to examine how electronic health record, mobile, and wearable technologies could be applied to clinical trials. The group specifically examined how these technologies might streamline the execution of clinical trial components, delineated innovative trial designs facilitated by technological developments, identified barriers to implementation, and determined the optimal frameworks needed for regulatory oversight. The group concluded that the application of novel technologies to clinical trials provided enormous potential, yet these changes needed to be iterative and facilitated by continuous learning and pilot studies.

Building clinical trials around patients: Evaluation and comparison of decentralized and conventional site models in patients with low back pain.

Clinical trials are slow and costly, built around the research centers that study local participants. Building clinical trials around patients in their homes and community through remote visits and monitoring could enhance recruitment and increase convenience for participants. This study evaluated different trial settings, a decentralized arm via telemedicine center (virtual study conduct), a conventional arm via health clinic (onsite study conduct) and a mixed model arm. Acute low-back pain patients (20-65 years) were recruited to this non-interventional trial in Switzerland. The study consisted of a screening period and a 2-week data collection period using direct data capture (eSource), electronic informed consent form (eICF), electronic diary (eDiary) and wearable actigraphy sensor. A higher number of patients were enrolled in the decentralized arm (N = 18) compared to the conventional arm (N = 5) and none in the mixed model arm. The decentralized arm consisted of a diverse population with increased participation from rural areas. In the decentralized arm 89% of enrolled patients completed the study compared to 60% in the conventional arm. All the patients reported satisfaction with the use of eICF, eDiary and remote visits; whereas patients reported a lower level of satisfaction with the wearable sensor. The decentralized setting was operationally feasible and well accepted by patients. Faster recruitment and improved access to patients was observed in the decentralized arm. This study supports broader adoption of the decentralized model in clinical trials, though further investigations in larger interventional trials are needed to confirm the benefits from this patient-centric approach.

Measurement Equivalence of Patient-Reported Outcome Measure Response Scale Types Collected Using Bring Your Own Device Compared to Paper and a Provisioned Device: Results of a Randomized Equivalence Trial.

OBJECTIVES: The aim of this study was to assess the measurement equivalence of individual response scale types by using a patient reported outcome measure (PROM) collected on paper and migrated into electronic format for use on the subject's own mobile device (BYOD) and on a provisioned device (site device). METHODS: Subjects suffering from chronic health conditions causing daily pain or discomfort were invited to participate in this single-site, single visit, three-way crossover study. Association between individual item and instrument subscale scores was assessed by using the intraclass correlation coefficient (ICC) and its CI. Participant attitudes toward the use of BYOD in a clinical trial were assessed through use of a questionnaire. RESULTS: In this study, 155 subjects (females 83 [54%]; males 72 [46%]) ages 19 to 69 years (mean ± SD: 48.6 ± 13.1) were recruited. High association between the modes of administration (paper, BYOD, site device) was shown with analysis of ICCs (0.79-0.98) for each response scale type, including visual analogue scale, numeric rating scale, verbal response scale, and Likert scale. Of the subjects, 94% (146 of 155) stated that they would definitely or probably be willing to download an app onto their own mobile device for a forthcoming clinical trial. Forty-five percent of subjects felt BYOD would be more convenient compared with 15% preferring a provisioned device (40% had no preference). CONCLUSIONS: This study provides strong evidence supporting the use of BYOD for PROM collection in terms of the conservation of instrument measurement equivalence across the most widely used response scale types, and high patient acceptance of the approach.

Standards for Instrument Migration When Implementing Paper Patient-Reported Outcome Instruments Electronically: Recommendations from a Qualitative Synthesis of Cognitive Interview and Usability Studies.

OBJECTIVES: To synthesize the findings of cognitive interview and usability studies performed to assess the measurement equivalence of patient-reported outcome (PRO) instruments migrated from paper to electronic formats (ePRO), and make recommendations regarding future migration validation requirements and ePRO design best practice. METHODS: We synthesized findings from all cognitive interview and usability studies performed by a contract research organization between 2012 and 2015: 53 studies comprising 68 unique instruments and 101 instrument evaluations. We summarized study findings to make recommendations for best practice and future validation requirements. RESULTS: Five studies (9%) identified minor findings during cognitive interview that may possibly affect instrument measurement properties. All findings could be addressed by application of ePRO best practice, such as eliminating scrolling, ensuring appropriate font size, ensuring suitable thickness of visual analogue scale lines, and providing suitable instructions. Similarly, regarding solution usability, 49 of the 53 studies (92%) recommended no changes in display clarity, navigation, operation, and completion without help. Reported usability findings could be eliminated by following good product design such as the size, location, and responsiveness of navigation buttons. CONCLUSIONS: With the benefit of accumulating evidence, it is possible to relax the need to routinely conduct cognitive interview and usability studies when implementing minor changes during instrument migration. Application of design best practice and selecting vendor solutions with good user interface and user experience properties that have been assessed in a representative group may enable many instrument migrations to be accepted without formal validation studies by instead conducting a structured expert screen review.