Developing the Polish Educational Needs Assessment Tool (Pol-ENAT) in rheumatoid arthritis and systemic sclerosis: a cross-cultural validation study using Rasch analysis

Objectives: To undertake cross-cultural adaptation and validation of the educational needs assessment tool (ENAT) for use with people with rheumatoid arthritis (RA) and systemic sclerosis (Ssc) in Poland. Methods: The study involved two main phases: (i) cross-cultural adaptation of the ENAT from English into Polish and (ii) Cross-cultural validation of Pol-ENAT. The first phase followed an established process of cross-cultural adaptation of self-report measures. The second phase involved completion of the Pol-ENAT by patients and subjecting the data to Rasch analysis to assess the construct validity, reliability and cross-cultural invariance. Results: An adequate conceptual equivalence was achieved following the adaptation process. The dataset for validation comprised a total of 278 patients, 237 (85.3%) of which were female. In each disease group (145, RA and 133, SSC), the 7 domains of the Pol-ENAT were found to fit the Rasch model, 2(df)=16.953(14), p=0.259 and 8.132(14), p=0.882 for RA and SSc respectively. Reliability (person separation index, PSI>0.85) was high. Cross-cultural differential item functioning (DIF) was detected in some subscales and DIF-adjusted conversion tables were calibrated to enable cross-cultural comparison of data between Poland and the UK. Conclusion: Pol-ENAT is a robust measure of educational needs for people with RA and SSc in Poland. The tool has been shown to have sufficient cross-cultural validity to enable data pooling and comparisons between Poland and the UK

Factors associated with quality of life in systemic sclerosis: a cross-sectional study

© 2019, The Author(s). Introduction: Systemic sclerosis (SSc) is a connective tissue disease characterized by progressive fibrosis of the skin and internal organs, leading to their failure and disturbances in the morphology and function of blood vessels. The disease affects people in different ways, and identifying how the difficulties and limitations are related to quality of life may contribute to designing helpful interventions. The aim of this study was to identify factors associated with quality of life in people with SSc. Methods: This was a cross-sectional study conducted in 11 rheumatic centres in Poland. Patients diagnosed with SSc were included. Quality of life was measured using the SSc Quality of Life Questionnaire (SScQoL). The following candidate factors were entered in preliminary multivariable analysis: age, place of residence, marital status, occupational status, disease type, disease duration, pain, fatigue, intestinal problems, breathing problems, Raynaud’s symptoms, finger ulcerations, disease severity, functional disability, anxiety and depression. Factors that achieved statistical significance at the 10% level were then entered into a final multivariable model. Factors achieving statistical significance at the 5% level in the final model were considered to be associated with quality of life in SSc. Results: In total, 231 participants were included. Mean age (SD) was 55.82 (12.55) years, disease duration 8.39 (8.18) years and 198 (85.7%) were women. Factors associated with quality of life in SSc were functional disability (β = 2.854, p < 0.001) and anxiety (β = 0.404, p < 0.001). This model with two factors (functional disability and anxiety) explained 56.7% of the variance in patients with diffuse SSc and 73.2% in those with localized SSc. Conclusions: Functional disability and anxiety are significantly associated with quality of life in SSc. Interventions aimed at improving either of these factors may contribute towards improving the quality of life of people with SSc

Targeted correction of a thalassemia-associated β-globin mutation induced by pseudo-complementary peptide nucleic acids

β-Thalassemia is a genetic disorder caused by mutations in the β-globin gene. Triplex-forming oligonucleotides and triplex-forming peptide nucleic acids (PNAs) have been shown to stimulate recombination in mammalian cells via site-specific binding and creation of altered helical structures that provoke DNA repair. However, the use of these molecules for gene targeting requires homopurine tracts to facilitate triple helix formation. Alternatively, to achieve binding to mixed-sequence target sites for the induced gene correction, we have used pseudo-complementary PNAs (pcPNAs). Due to steric hindrance, pcPNAs are unable to form pcPNA–pcPNA duplexes but can bind to complementary DNA sequences via double duplex-invasion complexes. We demonstrate here that pcPNAs, when co-transfected with donor DNA fragments, can promote single base pair modification at the start of the second intron of the beta-globin gene. This was detected by the restoration of proper splicing of transcripts produced from a green fluorescent protein-beta globin fusion gene. We also demonstrate that pcPNAs are effective in stimulating recombination in human fibroblast cells in a manner dependent on the nucleotide excision repair factor, XPA. These results suggest that pcPNAs can be effective tools to induce heritable, site-specific modification of disease-related genes in human cells without purine sequence restriction

Effects of needs-based patient education on self-efficacy and health outcomes in people with rheumatoid arthritis: A multicentre, single blind, randomised controlled trial

Objectives The Educational Needs Assessment Tool (ENAT) is a self-completed questionnaire, which allows patients with arthritis to prioritise their educational needs. The aim of this study was to evaluate the effects of needs-based patient education on self-efficacy, health outcomes and patient knowledge in people with rheumatoid arthritis (RA). Methods Patients with RA were enrolled into this multicentre, single-blind, parallel-group, pragmatic randomised controlled trial. Patients were randomised to either the intervention group (IG) where patients completed ENAT, responses of which were used by the clinical nurse specialist to guide patient education; or control group (CG) in which they received patient education without the use of ENAT. Patients were seen at weeks 0, 16 and 32. The primary outcome was selfefficacy (Arthritis Self Efficacy Scale (ASES)-Pain and ASES-Other symptoms). Secondary outcomes were health status (short form of Arthritis Impact Measurement Scale 2, AIMS2-SF) and patient knowledge questionnaire-RA. We investigated between-group differences using analysis of covariance, adjusting for baseline variables. Results A total of 132 patients were recruited (IG=70 and CG=62). Their mean (SD) age was 54 (12.3) years, 56 (13.3) years and disease duration 5.2 (4.9) years, 6.7 (8.9) years for IG and CG, respectively. There were significant between-group differences, in favour of IG at week 32 in the primary outcomes, ASES-Pain, mean difference (95% CI) -4.36 (1.17 to 7.55), t=-2.72, p=0.008 and ASES-Other symptoms, mean difference (95% CI) -5.84 (2.07 to 9.62), t=-3.07, p=0.003. In secondary outcomes, the between-group differences favoured IG in AIMS2-SF Symptoms and AIMS2-SF Affect. There were no between-group differences in other secondary outcomes. Conclusions The results suggest that needs-based education helps improve patients' self-efficacy and some aspects of health status

Deciphering Diseases and Biological Targets for Environmental Chemicals using Toxicogenomics Networks

Exposure to environmental chemicals and drugs may have a negative effect on human health. A better understanding of the molecular mechanism of such compounds is needed to determine the risk. We present a high confidence human protein-protein association network built upon the integration of chemical toxicology and systems biology. This computational systems chemical biology model reveals uncharacterized connections between compounds and diseases, thus predicting which compounds may be risk factors for human health. Additionally, the network can be used to identify unexpected potential associations between chemicals and proteins. Examples are shown for chemicals associated with breast cancer, lung cancer and necrosis, and potential protein targets for di-ethylhexyl-phthalate, 2,3,7,8-tetrachlorodibenzo-p-dioxin, pirinixic acid and permethrine. The chemical-protein associations are supported through recent published studies, which illustrate the power of our approach that integrates toxicogenomics data with other data types

Utility of the Care Dependency Scale in predicting care needs and health risks of elderly patients admitted to a geriatric unit: a cross-sectional study of 200 consecutive patients

Halina Doroszkiewicz,1 Matylda Sierakowska,2 Marta Muszalik3 1Department of Geriatrics, Medical University of Bialystok, Bialystok, Poland; 2Department of Integrated Medical Care, Medical University of Bialystok, Bialystok, Poland; 3Department and Clinic of Geriatrics, Nicolaus Copernicus University, Collegium Medicum in Bydgoszcz, Bydgoszcz, Poland Objective: The aim of the study was to evaluate the usefulness of the Polish version of the Care Dependency Scale (CDS) in predicting care needs and health risks of elderly patients admitted to a geriatric unit.Methods: This was a cross-sectional study of 200 geriatric patients aged &ge;60&nbsp;years, chronologically admitted to a geriatrics unit in Poland. The study was carried out using the Polish version of the CDS questionnaire to evaluate biopsychosocial needs and the level of care dependency.Results: The mean age of the participating geriatric patients was 81.8&plusmn;6.6. The mean result of the sum of the CDS index for all the participants was 55.3&plusmn;15.1. Detailed analysis of the results of evaluation of the respondents&rsquo; functional condition showed statistically significant differences in the levels of care dependency. Evaluation of the patients&rsquo; physical performance in terms of the ability to do basic activities of daily living (ADL) and instrumental ADL (I-ADL) showed statistically significant differences between the levels of care dependency. Patients with high dependency were more often prone to pressure ulcers &ndash; 13.1&plusmn;3.3, falls (87.2%), poorer emotional state &ndash; 6.9&plusmn;3.6, mental function &ndash; 5.1&plusmn;2.8, and more often problems with locomotion, vision, and hearing. The results showed that locomotive disability, depression, advanced age, and problem with vision and hearing are connected with increasing care dependency.Conclusion: CDS evaluation of each admitted geriatric patient enables us to predict the care needs and health risks that need to be reduced and the disease states to be improved. CDS evaluation should be accompanied by the use of other instruments and assessments to evaluate pressure ulcer risk, fall risk, and actions toward the improvement of subjective well-being, as well as correction of vision and hearing problems where possible and assistive devices for locomotion. Keywords: Care Dependency Scale, care needs, health risks, elderl