Development of the CDISC Pediatrics User Guide: a CDISC and conect4children collaboration.

Introduction: The conect4children (c4c) project aims to facilitate efficient planning and delivery of paediatric clinical trials. One objective of c4c is data standardization and reuse. Interoperability and reusability of paediatric clinical trial data is challenging due to a lack of standardization. The Clinical Data Interchange Standards Consortium (CDISC) standards that are required or recommended for regulatory submissions in several countries lack paediatric specificity with limited awareness within academic institutions. To address this, c4c and CDISC collaborated to develop the Pediatrics User Guide (PUG) consisting of cross-cutting data items that are routinely collected in paediatric clinical trials, factoring in all paediatric age ranges. Methods and Results: The development of the PUG consisted of six stages. During the scoping phase, subtopics (each containing several clinically relevant concepts) were suggested and debated for inclusion in the PUG. Ninety concepts were selected for the modelling phase. Concept maps describing the Research Topic and representation procedure were developed for the 19 concepts that had no (or partial) previous modelling in CDISC. Next, metadata and implementation examples were developed for concepts. This was followed by a CDISC internal review and a public review. For both these review stages, the feedback comments were either implemented or rejected based on budget, timelines, expert review, and scope. The PUG was published on the CDISC website on February 23, 2023. Discussion: The PUG is a first step in bridging the lack of child specific CDISC standards, particularly within academia. Several academic and industrial partners were involved in the development of the PUG, and c4c has undertaken multiple steps to publicize the PUG within its academic partner organizations - in particular, the European Reference Networks (ERNs) that are developing registries and dictionaries in 24 disease areas. In the long term, continued use of the PUG in paediatric clinical trials will enable the pooling of data from multiple trials, which is particularly important for medical domains with small populations.

Bile Duct Tumor as the Presenting Manifestation of Colon Cancer: A Case Report.

Painless obstructive jaundice is a well-recognized clinical sign of hepatocellular pathology or obstruction of the biliary system. Bile duct tumors are a known etiology of painless obstructive jaundice. Here, we present a case of obstructive jaundice, which was initially thought be caused by cholangiocarcinoma based on computerized tomography imaging and endoscopic retrograde cholangiopancreatography but was later found to be hilar metastasis from an undiscovered colon cancer.

Laryngopharyngeal reflux: Comparing improvements in reflux symptom index with reflux finding score.

PURPOSE: The objective of this study was to determine the correlation of reported symptom improvement in laryngopharyngeal reflux (LPR) with physical findings on laryngoscopic examination following medical therapy. MATERIALS AND METHODS: This IRB-approved, retrospective, single-institution study included all patients who were diagnosed and medically treated for LPR from Jan. 1, 2015-October 21st, 2019. Only patients who received at least six weeks of treatment with a proton pump inhibitor and those with pre- and post-treatment Reflux Symptom Index; RSI (n = 91) and Reflux Finding Score; RFS (n = 33) were included in the study. RESULTS: A total of 91 patients were included in the analysis (61.54% female). There was a 19.99% improvement in total RSI (p = 0.0034) and a 25.20% improvement in total RFS (p = 0.0011) following at least six weeks of treatment (average = 253 ± 213 SD days). RSI symptoms were significantly decreased between pre- and post-treatment for hoarseness (p = 0.0005), clearing of the throat (p = 0.0066), excess throat mucus or postnasal drip (p = 0.0004), troublesome cough (p = 0.0231), and heartburn/chest pain (p = 0.0053). RFS demonstrated a statistically significant decrease in only subglottic edema (p < 0.0001) and ventricular obliteration (p = 0.0295). Pearson's correlation test did not demonstrate a relationship between RSI and RFS in the pre- and post-treatment analyses alone, but did demonstrate a statistically significant relationship when analyzed across all captured pre and post data (r = 0.265, p = 0.006). CONCLUSIONS: RSI and RFS are complimentary to one another when assessing for treatment response, but improvement in RSI does not correlate with that in RFS.

Vocalizations convey sex, seasonal phenotype, and aggression in a seasonal mammal.

Seasonal variation in social behavior is often accompanied by seasonal variation in communication. In mammals, how seasonal environmental cues influence aggressive vocalizations remains underexplored. Photoperiod is the primary cue coordinating seasonal responses in most temperate zone animals, including Siberian hamsters (Phodopus sungorus), a species that undergoes reproductive inhibition and increased aggression in winter. During same-sex aggressive encounters, hamsters emit both broadband calls (BBCs) and ultrasonic vocalizations (USVs) that indicate aggression and the vocalizer's sex, respectively; however, it is not known whether these rodents adjust specific elements of their vocal repertoire to reflect their photoperiod-induced seasonal phenotypes. To address this, we recorded vocalizations emitted during dyadic interactions between male or female pairs of hamsters housed in long or short photoperiods and measured serum testosterone levels. USV emission rate remained stable across photoperiods, but proportional use of USV subtypes varied in novel ways: 'jump' USVs were sensitive to seasonal phenotype, but not the vocalizer's sex, whereas 'plain' USVs were sensitive only to the sex of the vocalizer. BBC emission rate varied with seasonal phenotype; short-day non-reproductive hamsters produced more BBCs and demonstrated increased aggression compared with reproductive hamsters. Testosterone, however, was not related to vocalization rates. Collectively, these findings demonstrate that changes in the vocal repertoire of Siberian hamsters reflect sex, aggression, and seasonal phenotype, suggesting that both BBCs and USVs are important signals used during same-sex social encounters.