Evidence of quality professional development:a study in childhood practice

Peer reviewedPostprin

Detect, Retrieve, Comprehend: A Flexible Framework for Zero-Shot Document-Level Question Answering

Researchers produce thousands of scholarly documents containing valuable technical knowledge. The community faces the laborious task of reading these documents to identify, extract, and synthesize information. To automate information gathering, document-level question answering (QA) offers a flexible framework where human-posed questions can be adapted to extract diverse knowledge. Finetuning QA systems requires access to labeled data (tuples of context, question and answer). However, data curation for document QA is uniquely challenging because the context (i.e. answer evidence passage) needs to be retrieved from potentially long, ill-formatted documents. Existing QA datasets sidestep this challenge by providing short, well-defined contexts that are unrealistic in real-world applications. We present a three-stage document QA approach: (1) text extraction from PDF; (2) evidence retrieval from extracted texts to form well-posed contexts; (3) QA to extract knowledge from contexts to return high-quality answers -- extractive, abstractive, or Boolean. Using QASPER for evaluation, our detect-retrieve-comprehend (DRC) system achieves a +7.19 improvement in Answer-F1 over existing baselines while delivering superior context selection. Our results demonstrate that DRC holds tremendous promise as a flexible framework for practical scientific document QA

Kidney transplant graft outcomes in 379 257 recipients on 3 continents

Peer Reviewedhttps://deepblue.lib.umich.edu/bitstream/2027.42/145422/1/ajt14694_am.pdfhttps://deepblue.lib.umich.edu/bitstream/2027.42/145422/2/ajt14694.pd

Increasing Incidence of Clostridium difficile-associated Disease, Singapore

10.3201/eid1409.070043Emerging Infectious Diseases1491487-148

The care of patients with Duchenne, Becker and other muscular dystrophies in the COVID-19 pandemic

The corona virus disease 2019 (COVID-19) pandemic has resulted in the reorganization of healthcare settings affecting clinical care delivery to patients with Duchenne and Becker muscular dystrophy (DBMD) as well as other inherited muscular dystrophies. The magnitude of the impact of this public health emergency on the care of patients with DBMD is unclear as they are suspected of having an increased risk for severe manifestations of COVID-19. In this paper, the authors discuss their consensus recommendations pertaining to care of these patients during the pandemic. We address issues surrounding corticosteroid and exon skipping treatments, cardiac medications, hydroxychloroquine use, emergency/respiratory care, rehabilitation management, and the conduct of clinical trials. We highlight the importance of collaborative treatment decisions between the patient, family, and health care provider, considering any geographic or institution-specific policies and precautions for COVID-19. We advocate for continuing multidisciplinary care for these patients using telehealth

DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials

BACKGROUND AND OBJECTIVES: Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients to untreated patients with the same DMD genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing challenges for trial enrollment in this already rare disease. To evaluate the suitability of genotypically unmatched controls in DMD, we quantified effects of genotype class on 1-year changes in motor function endpoints used in clinical trials. METHODS: Over 1,600 patient-years of follow-up (>700 patients) were studied from six real-world/natural history data sources (UZ Leuven, PRO-DMD-01 shared by CureDuchenne, iMDEX, North Star UK, Cincinnati Children's Hospital Medical Center, and the DMD Italian Group), with genotypes classified as amenable to skipping exons 44, 45, 51 or 53, other skippable, nonsense, and other mutations. Associations between genotype class and 1-year changes in North Star Ambulatory Assessment total score (ΔNSAA) and in 10-meter walk/run velocity (Δ10MWR) were studied in each data source with and without adjustment for baseline prognostic factors. RESULTS: The studied genotype classes accounted for approximately 2% of variation in ΔNSAA outcomes after 12 months, whereas other prognostic factors explained >30% of variation in large data sources. Based on a meta-analysis across all data sources, pooled effect estimates for the studied skip-amenable mutation classes were all small in magnitude (<2 units in ΔNSAA total score in 1-year follow up), smaller than clinically important differences in NSAA, and were precisely estimated with standard errors <1 unit after adjusting for non-genotypic prognostic factors. DISCUSSION: These findings suggest viability of trial designs incorporating genotypically mixed or unmatched controls for up to 12 months in duration for motor function outcomes, which would ease recruitment challenges and reduce numbers of patients assigned to placebos. Such trial designs, including multi-genotype platform trials and hybrid designs, should ensure baseline balance between treatment and control groups for the most important prognostic factors, while accounting for small remaining genotype effects quantified in the present study