Treat to Target: A Proposed New Paradigm for the Management of Crohn's Disease.

International audience: The traditional management of CD, based on progressive, step-wise treatment intensification with re-evaluation of response according to symptoms, does not improve long-term outcomes of CD and places patients at risk for bowel damage. The introduction of novel therapies and the development of new approaches to treatment in rheumatoid arthritis led to better outcomes for patients. Prominent among these is a "treat to target" strategy that is based on regular assessment of disease activity using objective clinical and biological outcome measures and the subsequent adjustment of treatments. This approach is complementary to the concept of early intervention in high risk patients. This review evaluates current literature on this topic and proposes a definition for the concept treating to targets for Crohn's disease

Baseline body mass index among children and adults undergoing allogeneic hematopoietic cell transplantation: clinical characteristics and outcomes

Obesity is an important public health problem that may influence the outcomes of hematopoietic cell transplantation (HCT). We studied 898 children and adults receiving first-time allogeneic hematopoietic stem cell transplants between 2004 and 2012. Pre-transplant body mass index (BMI) was classified as underweight, normal weight, overweight, or obese using the WHO classification, or age-adjusted BMI percentiles for children. The study population was predominantly Caucasian, and the median age was 51 years (5 months – 73 years). The cumulative 3-year incidence of non-relapse mortality (NRM) in underweight, normal weight, overweight, and obese patients was 20%, 19%, 20%, and 33%, respectively. Major causes of NRM were acute and chronic graft-versus-host disease (GVHD). The corresponding incidence of relapse was 30%, 41%, 37%, and 30%, respectively. Three-year overall survival was 59%, 48%, 47%, and 43%, respectively. Multivariate analysis showed that obesity was associated with higher NRM (HR 1.43, p=0.04), and lower relapse (HR 0.65, p=0.002). Pre-transplant plasma levels of ST2 and TNFR1 biomarkers were significantly higher in obese compared with normal weight patients (p=0.04 and p=0.05, respectively). The increase in NRM observed in obese patients was partially offset by lower incidence of relapse with no difference in overall survival

Gerstmann-Straussler-Scheinker disease in an Alsatian family: clinical and genetic studies

The clinical progression of Gerstmann-Straussler-Scheinker disease in a family of Alsatian origin is reported. The age of onset and the duration of evolution were variable. The clinical picture became more complex over the generations: in the first generations, isolated dementia and in later generations a triad of pyramidal, pseudobulbar syndromes and dementia associated with spinal cord and cerebellar features. Prion gene analysis showed that four surviving patients carry double missense changes at codons 117 and 129, identical to those found in one case at necropsy and 10 other healthy members of the family. The missense changes were not found in 100 controls. No member of the family had modification of condons 102, 178, or 200. The lod score suggests linkage between the missense change at codon 117 and Gerstmann- Straussler-Scheinker disease in this family

Outcome measurement in clinical trials for Ulcerative Colitis: towards standardisation

Clinical trials on novel drug therapies require clear criteria for patient selection and agreed definitions of disease remission. This principle has been successfully applied in the field of rheumatology where agreed disease scoring systems have allowed multi-centre collaborations and facilitated audit across treatment centres. Unfortunately in ulcerative colitis this consensus is lacking. Thirteen scoring systems have been developed but none have been properly validated. Most trials choose different endpoints and activity indices, making comparison of results from different trials extremely difficult. International consensus on endoscopic, clinical and histological scoring systems is essential as these are the key components used to determine entry criteria and outcome measurements in clinical trials on ulcerative colitis. With multiple new therapies under development, there is a pressing need for consensus to be reached

Complexities, variations, and errors of numbering within clinical notes: the potential impact on information extraction and cohort-identification

Abstract Background Numbers and numerical concepts appear frequently in free text clinical notes from electronic health records. Knowledge of the frequent lexical variations of these numerical concepts, and their accurate identification, is important for many information extraction tasks. This paper describes an analysis of the variation in how numbers and numerical concepts are represented in clinical notes. Methods We used an inverted index of approximately 100 million notes to obtain the frequency of various permutations of numbers and numerical concepts, including the use of Roman numerals, numbers spelled as English words, and invalid dates, among others. Overall, twelve types of lexical variants were analyzed. Results We found substantial variation in how these concepts were represented in the notes, including multiple data quality issues. We also demonstrate that not considering these variations could have substantial real-world implications for cohort identification tasks, with one case missing > 80% of potential patients. Conclusions Numbering within clinical notes can be variable, and not taking these variations into account could result in missing or inaccurate information for natural language processing and information retrieval tasks.https://deepblue.lib.umich.edu/bitstream/2027.42/148519/1/12911_2019_Article_784.pd

Transition and Reflection in the Use of Health Information: The Case of Pediatric Bone Marrow Transplant Caregivers

ABSTRACT The impact of health information on caregivers is of increasing interest to HCI/CSCW in designing systems to support the social and emotional dimensions of managing health. Drawing on an interview study, as well as corroborating data including a multi-year ethnography, we detail the practices of caregivers (particularly parents) in a bone marrow transplant (BMT) center. We examine the interconnections between information and emotion work performed by caregivers through a liminal lens, highlighting the BMT experience as a time of transition and reflection in which caregivers must quickly adapt to the new social world of the hospital and learn to manage a wide range of patient needs. The transition from parent to 'caregiver' is challenging, placing additional emotional burdens on the intensive information work for managing BMT. As a time of reflection, the BMT experience also provides an occasion for generative thinking and alternative approaches to health management. Our study findings call for health systems that reflect a design paradigm focused on 'transforming lives' rather than 'transferring information.' Autho

Complexities, variations, and errors of numbering within clinical notes: the potential impact on information extraction and cohort-identification

Background Numbers and numerical concepts appear frequently in free text clinical notes from electronic health records. Knowledge of the frequent lexical variations of these numerical concepts, and their accurate identification, is important for many information extraction tasks. This paper describes an analysis of the variation in how numbers and numerical concepts are represented in clinical notes. Methods We used an inverted index of approximately 100 million notes to obtain the frequency of various permutations of numbers and numerical concepts, including the use of Roman numerals, numbers spelled as English words, and invalid dates, among others. Overall, twelve types of lexical variants were analyzed. Results We found substantial variation in how these concepts were represented in the notes, including multiple data quality issues. We also demonstrate that not considering these variations could have substantial real-world implications for cohort identification tasks, with one case missing > 80% of potential patients. Conclusions Numbering within clinical notes can be variable, and not taking these variations into account could result in missing or inaccurate information for natural language processing and information retrieval tasks

Purification of Nanoparticles by Size and Shape

Producing monodisperse nanoparticles is essential to ensure consistency in biological experiments and to enable a smooth translation into the clinic. Purification of samples into discrete sizes and shapes may not only improve sample quality, but also provide us with the tools to understand which physical properties of nanoparticles are beneficial for a drug delivery vector. In this study, using polymersomes as a model system, we explore four techniques for purifying pre-formed nanoparticles into discrete fractions based on their size, shape or density. We show that these techniques can successfully separate polymersomes into monodisperse fractions