Objective: The aim of the study was to identify and characterize outcome measures for objective and subjective assessment in persons with Rett syndrome (RS).
Methods: A systematic review was conducted consulting the EBSCO, Cochrane, Web of Science, Scielo, MEDLINE and PsycINFO databases for published studies describing the use of patient-reported outcome measures (PROMs) and other outcome measures in persons with RS. Validation studies and observational studies were included. The PROMs were first described, and then the measurement properties were evaluated using predefined criteria according to the COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN). The outcome measures were then grouped according to the International Classification of Functioning, Disability and Health (ICF) to establish a relationship between outcome measures and ICF domains.
Results: Twenty out of 2327 articles were appraised, and seventeen different outcome measures were identified and described. Ten outcome measures corresponded to evaluation questionnaires, while the remaining seven assessed functional outcomes: walking distance, physical activity level and ability to interact visually. A relation between these outcome measures that assess RS and the ICF allows understanding that most of the instruments (fifteen) include the assessment of activity limitations.
Conclusions: The findings of this study seem to be promising for their use by clinicians and researchers, although they have methodological limitations. The accuracy and quality of these individual outcome measures should continue to be assessed in an attempt to gather a consensus on the best tools used in RS.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.ejpn.2022.06.003 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
The clinical significance of T-cell regulation in hypertension treatment.
Front Immunol
March 2025
Sinopharm Dongfeng General Hospital (Hubei Clinical Research Center of Hypertension), Hubei Key Laboratory of Wudang Local Chinese Medicine Research, Hubei University of Medicine, Shiyan, Hubei, China.
Hypertension, a globally prevalent condition, is closely associated with T cell-mediated inflammatory responses. Studies have shown that T cells, by secreting pro-inflammatory cytokines such as interferon-gamma (IFN-γ), Interleukin-17 (IL-17), and Tumor necrosis factor-alpha (TNF-α), directly lead to vascular dysfunction and elevated blood pressure. The activation of Th1 and Th17 cell subsets, along with the dysfunction of regulatory T cells (Tregs), is a critical mechanism in the onset and progression of hypertension.
View Article and Find Full Text PDFPhase 2 study of chidamide in combination with CAG and venetoclax-azacitidine in older patients with newly diagnosed acute myeloid leukemia.
Front Immunol
March 2025
State Key Laboratory of Experimental Hematology, Senior Department of Hematology, The Fifth Medical Center of Chinese PLA General Hospital, Beijing, China.
Introduction: Older patients with acute myeloid leukemia (AML) respond poorly to standard induction therapy. DNA methyltransferases (DNMTs) and histone-deacetylases (HDACs) are key regulators of gene expression in cells and have been investigated as important therapeutic targets. However, their effects remains unclear as induction therapy for AML.
View Article and Find Full Text PDFVirological response and predictive factors for antiviral treatment in chronic HBV-related liver disease with low ALT and high HBV DNA.
Front Immunol
March 2025
Center of Liver Diseases Division 3, Beijing Ditan Hospital, Capital Medical University, Beijing, China.
Objective: To investigate virological response and predictive factors for antiviral treatment in chronic HBV patients with low ALT and high HBV DNA.
Methods: A retrospective study grouped chronic HBV patients by baseline ALT: ALT > 80 U/L (significantly elevated group, SAG), 40-80 U/L (mildly elevated group, MAG), and ≤ 40 U/L (normal group, NG). Inverse probability treatment weighting balanced confounding factors.
Immune repertoire sequencing reveals differences in treatment response to camrelizumab plus platinum-based chemotherapy in advanced ESCC.
Front Immunol
March 2025
Department of Oncology, Changzhi People's Hospital Affiliated to Changzhi Medical College, Changzhi, Shanxi, China.
This study evaluated the efficacy and safety of camrelizumab combined with platinum-based chemotherapy (taxanes [T] or fluorouracil agents [F] plus platinum [P] drugs) as the first-line treatment in advanced esophageal squamous cell carcinoma (ESCC), using immune repertoire sequencing (IRS) to explore treatment response mechanism. In this multi-center, prospective cohort study, 88 patients received camrelizumab plus TP or FP, achieving a 1-year progression-free survival of 56.8% and overall survival of 68.
View Article and Find Full Text PDFCAR-T cells in the treatment of multiple myeloma: an encouraging cell therapy.
Front Immunol
March 2025
Department of Orthopaedic Medical Center, The Second Norman Bethune Hospital of Jilin University, Changchun, Jilin, China.
Multiple myeloma (MM) is a malignant disease of plasma cells that accounts for approximately 10% of all hematological malignancies and is characterized by a clonal proliferation of malignant plasma cells in the bone marrow. Numerous therapeutic strategies, including proteasome inhibitors, immunomodulators, monoclonal antibodies against CD38 and autologous stem cell transplantation, have prolonged the median survival of MM patients. Nevertheless, almost all MM patients suffer disease relapses due to drug resistance and eventually die from MM or MM-related complications.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!