Objectives: Evidence suggests that inflammation has a harmful effect on muscle strength as well as on functional disability. The purpose of the present study was to examine the association of objectively measured disease activity levels and functional disability among Iranian patients with Rheumatoid Arthritis (RA), and to analyse whether a Persian version of the health assessment questionnaire-disability index (PHAQ-DI) can distinguish between RA patients at different stages of the disease progression.
Materials & Methods: 198 RA patients were requested to complete the PHAQ-DI. The disease activity score for each patient was evaluated using the disease activity score (DAS-28). The association analysis between the PHAQ-DI scores and the four levels of disease activity was measured using a Spearman correlation coefficient. A Kruskal-Wallis analysis was utilized to determine differences in PHAQ-DI scores among the levels of disease activity. The Receiver operating characteristic (ROC) curve was also utilized to determine the PHAQ-DI total score cut-off for predicting the level of disease activity.
Results: Spearman's correlation coefficients between the PHAQ-DI and the disease activity level ranged between 0.59 and 0.75. There were significant differences in the PHAQ-DI total score among known groups with various disease activity levels (P = 0.001); However, HAQ-DI total score could not differentiate the remission phase from low disease activity levels in patients with RA (p = 0.37). The PHAQ-DI total score cut-off for distinguishing remission-low disease activity from moderate-high disease activity was 1.19, with a specificity of 0.91 and a sensitivity of 0.615.
Conclusion: The present findings provide evidence for the degree to which the PHAQ-DI measures identify and distinguish disease activity levels in patients with rheumatoid arthritis. The PHAQ-DI questionnaire, as a patient-administered, non-invasive, fast, inexpensive and available tool, can be used in the rheumatologist's office as a substitute for determining disease activity in patients with active RA.
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http://dx.doi.org/10.1016/j.jbmt.2020.02.023 | DOI Listing |
Proc Natl Acad Sci U S A
January 2025
Bioelectricity Laboratory, Department of Physiology and Biophysics, School of Medicine, University of California, Irvine, CA 92697.
Loss-of-function sequence variants in , which encodes the voltage-gated potassium channel Kv1.1, cause Episodic Ataxia Type 1 (EA1) and epilepsy. Due to a paucity of drugs that directly rescue mutant Kv1.
View Article and Find Full Text PDFProc Natl Acad Sci U S A
January 2025
Innovative Genomics Institute, University of California, Berkeley, CA 94720.
The widespread application of genome editing to treat and cure disease requires the delivery of genome editors into the nucleus of target cells. Enveloped delivery vehicles (EDVs) are engineered virally derived particles capable of packaging and delivering CRISPR-Cas9 ribonucleoproteins (RNPs). However, the presence of lentiviral genome encapsulation and replication proteins in EDVs has obscured the underlying delivery mechanism and precluded particle optimization.
View Article and Find Full Text PDFJ Pediatr Hematol Oncol
January 2025
Department of Pediatrics, West China Second University Hospital, Sichuan University, Sichuan, China.
Langerhans cell histiocytosis (LCH) is characterized genetically by diverse gene mutations of the mitogen-activated protein kinase signaling cascade. BRAFN486_T491delinsK mutation is a rare mutation that involves the β2-αC ring domain, causing activation of the mitogen-activated protein kinase pathway, and is predicted to be resistant to the chemotherapy and BRAFV600E inhibitor in adult LCH cases. Here, we report a childhood LCH case with this novel BRAF mutation and had a good response to conventional chemotherapy.
View Article and Find Full Text PDFPLoS Negl Trop Dis
January 2025
Department of Pediatrics, National School of Tropical Medicine, Baylor College of Medicine, Houston, Texas, United States of America.
Background: The antigen Na-GST-1, expressed by the hookworm Necator americanus, plays crucial biochemical roles in parasite survival. This study explores the development of mRNA vaccine candidates based on Na-GST-1, building on the success of recombinant Na-GST-1 (rNa-GST-1) protein, currently assessed as a subunit vaccine candidate, which has shown promise in preclinical and clinical studies.
Methodology/findings: By leveraging the flexible design of RNA vaccines and protein intracellular trafficking signal sequences, we developed three variants of Na-GST-1 as native (cytosolic), secretory, and plasma membrane-anchored (PM) antigens.
PLoS Comput Biol
January 2025
IRSD-Institut de Recherche en Santé Digestive, Université de Toulouse, INSERM, INRAE, ENVT, UPS, Toulouse, France.
Understanding the interplay between biology and mechanics in tissue architecture is challenging, particularly in terms of 3D tissue organization. Addressing this challenge requires a biological model enabling observations at multiple levels from cell to tissue, as well as theoretical and computational approaches enabling the generation of a synthetic model that is relevant to the biological model and allowing for investigation of the mechanical stresses experienced by the tissue. Using a monolayer human colon epithelium organoid as a biological model, freely available tools (Fiji, Cellpose, Napari, Morphonet, or Tyssue library), and the commercially available Abaqus FEM solver, we combined vertex and FEM approaches to generate a comprehensive viscoelastic finite element model of the human colon organoid and demonstrated its flexibility.
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