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Diabetes, Metabolic Syndrome and... 2024Autonomic nervous system dysfunction (ANSD), for which presently no treatment exists, has a negative impact on prognosis in people with type 2 diabetes (T2D). Periosteal... (Clinical Trial)
Clinical Trial
New Diabetic Treatment by Alleviation of Autonomic Nervous System Dysfunction Measured as Periosteal Pressure Sensitivity at Sternum Improves Empowerment, Treatment Satisfaction, and Self-Reported Health of People with Type 2 Diabetes: A Randomized Trial.
PURPOSE
Autonomic nervous system dysfunction (ANSD), for which presently no treatment exists, has a negative impact on prognosis in people with type 2 diabetes (T2D). Periosteal pressure sensitivity (PPS) on sternum may be a measure of autonomic nervous system dysfunction (ANSD). We tested if a non-pharmacological PPS-feedback-guided treatment program based on non-noxious sensory nerve stimulation, known to reduce PPS, changed empowerment, treatment satisfaction, and quality of life in people with T2D, compared to usual treatment.
PATIENTS AND METHODS
Analysis of secondary endpoints in a single center, two-armed, parallel-group, observer-blinded, randomized controlled trial of individuals with T2D. Participants were randomized to non-pharmacological intervention as an add-on to treatment as usual. Endpoints were evaluated by five validated questionnaires: Diabetes specific Empowerment (DES-SF), Diabetes Treatment Satisfaction (DTSQ), quality of life (QOL) (WHO-5), clinical stress signs (CSS), and self-reported health (SF-36). Sample size calculation was based on the primary endpoint HbA1c.
RESULTS
We included 144 participants, 71 allocated to active intervention and 73 to the control group. Active intervention compared to control revealed improved diabetes-specific empowerment (p = 0.004), DTSQ (p = 0.001), and SF-36 self-reported health (p=0.003) and tended to improve quality of life (WHO-5) (p = 0.056). The findings were clinically relevant with a Cohen's effect size of 0.5 to 0.7.
CONCLUSION
This non-pharmacological intervention, aiming to reduce PPS, and thus ANSD, improved diabetes-specific empowerment, treatment satisfaction, and self-reported health when compared to usual treatment. The proposed intervention may be a supplement to conventional treatment for T2D.
PubMed: 38910915
DOI: 10.2147/DMSO.S455216 -
Cureus May 2024The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative was established in 2016 to assess the... (Review)
Review
The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) initiative was established in 2016 to assess the quality and standardization of patient-reported outcomes (PRO) data analysis in randomized controlled trials (RCTs) on advanced breast cancer. The initiative identified deficiencies in PRO data reporting, including nonstandardized methods for handling missing data. This study evaluated the reporting of health-related quality of life (HRQOL) in Japanese cancer RCTs to provide insights into the state of PRO reporting in Japan. The study reviewed PubMed articles published from 2010 to 2018. Eligible studies included Japanese cancer RCTs with ≥50 adult patients (≥50% were Japanese) with solid tumors receiving anticancer treatments. The evaluation criteria included clarity of the HRQOL hypotheses, multiplicity testing, primary analysis methods, and reporting of clinically meaningful differences. Twenty-seven HRQOL trials were identified. Only 15% provided a clear HRQOL hypothesis, and 63% examined multiple HRQOL domains without adjusting for multiplicity. Model-based methods were the most common statistical methods for the primary HRQOL analysis. Only 22% of the trials explicitly reported clinically meaningful differences in HRQOL. Baseline assessments were reported in most trials, but only 26% reported comparisons between the treatment groups. HRQOL analysis was based on the intention-to-treat population in 19% of the trials, and 74% reported compliance at follow-up; however, 41% did not specify how missing values were handled. Although the rates of reporting clinical hypotheses and clinically meaningful differences were relatively low, the current state of HRQOL evaluation in the Japanese cancer RCT appears comparable to that of previous studies.
PubMed: 38910767
DOI: 10.7759/cureus.60804 -
Cureus May 2024Developing a validated, standardized, and easily accessible pain assessment tool is the first step toward improving pain management. Since pain measurement is often used...
Developing a validated, standardized, and easily accessible pain assessment tool is the first step toward improving pain management. Since pain measurement is often used as a primary or secondary endpoint in daily clinical practice and clinical trials, accurate and precise pain measurement is of great importance. Therefore, there is a need for a valid, reliable, safe, and low-cost method to measure and assess pain levels more objectively. Traditional measurement tools, still considered the gold standard in clinical pain research, have significant disadvantages, including low sensitivity and higher rates of false responses. Perhaps most importantly, the assumption that general pain is a one-dimensional experience that can be measured with a single-item scale is limiting. Recently, new technologies utilizing smart devices have emerged to improve existing traditional pain outcome measures. The Visual, Auditory, and Tactile Analog Scale (VATAS) was designed to address tactile, auditory, and visual senses. By including multiple senses, it is thought that errors arising from the objectification of pain solely through a single-dimensional scale, such as Visual Analog Scale (VAS), could be eliminated, providing a more standardized and repeatable pain assessment. VATAS has the potential to complement the deficiencies of standard pain measurement methods by appealing to multiple senses. It can provide a more standardized, patient-compliant, and repeatable pain assessment. Furthermore, it can be used for evaluating and recording pain in visually impaired patients and has the potential for data tracking, allowing patients' pain to be monitored even when they are at home.
PubMed: 38910725
DOI: 10.7759/cureus.60793 -
Cureus May 2024Background Chemotherapy-induced peripheral neuropathy (CIPN) is a problematic adverse event for breast cancer patients receiving taxane antimitotic agents. We evaluated...
Background Chemotherapy-induced peripheral neuropathy (CIPN) is a problematic adverse event for breast cancer patients receiving taxane antimitotic agents. We evaluated the effectiveness of compression therapy against CIPN in the lower extremities of breast cancer patients receiving taxanes. Methods Eligible patients scheduled for perioperative treatment with taxanes for early-stage breast cancer were enrolled. Each patient wore latex-free surgical gloves and compression socks, putting on two layers of each 15 minutes before the administration of taxanes and removing them 15 minutes after administration. Peripheral neuropathy (PN) was evaluated using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 and the Patient Neurotoxicity Questionnaire (PNQ). The primary endpoint was the incidence of CTCAE version 4.0 grade 2 or higher CIPN in the lower extremities during the entire period of perioperative chemotherapy with taxanes. Results PN assessment by CTCAE in the lower extremities, the primary outcome, showed that 13.3% developed grade 2 sensory disturbances, and 8.3% developed grade 2 motor disturbances. The incidence of CTCAE grade 2 or higher PN in the hands was 26.7% for sensory disturbances and 13.3% for motor disturbances during the entire study period. No patient had grade 3 or higher PN. No adverse events due to compression therapy were observed. Conclusion Compression of the lower extremities with compression socks tended to reduce the incidence of CIPN compared to the general incidence. Compression therapy may help prevent the development of CIPN.
PubMed: 38910688
DOI: 10.7759/cureus.60998 -
Acta Oncologica (Stockholm, Sweden) Jun 2024The Precision Oncology Platform (POP) trial represents the effort of the Portuguese Oncology Institute of Porto (IPO Porto) for joining other leading European...
BACKGROUND AND PURPOSE
The Precision Oncology Platform (POP) trial represents the effort of the Portuguese Oncology Institute of Porto (IPO Porto) for joining other leading European institutions in both 'Personalised Cancer Medicine for all EU citizens' (PCM4EU), and 'PRecisIon Cancer MEdicine RepurpOsing SystEm Using Pragmatic Clinical Trials' (PRIME-ROSE) consortia, enabling the development of the Portuguese version of the Drug Rediscovery Protocol (DRUP)-like Clinical Trial (DLCT), based on the experience of the DRUP trial developed in The Netherlands.
PATIENTS/MATERIAL AND METHODS
The POP trial is a phase II, pragmatic multicentric, non-randomised, open-label study, designed entirely like the other DLCTs. Its primary objective is to describe anti-tumour activity of targeted anticancer drugs in patients with advanced malignancies harbouring actionable molecular alterations. The primary endpoint is disease control rate (DCR). Secondary endpoints encompass treatment-related grade ≥3 adverse events, objective response rate (ORR), duration of response (DOR), progression-free survival (PFS), and overall survival (OS). Exploratory objectives will assess biomarkers, resource use and costs, and patient-reported outcome measures (PROMs).
INTERPRETATION
The POP trial will offer access to innovative treatments for patients without further therapeutic options and provide evidence on efficacy and safety of molecularly-guided treatments. Methodologically, it represents a pioneer approach in Portugal, including a pay-for-performance model embedded in the clinical trial. The POP trial represents a unique opportunity to integrate clinical research within cancer care, pursuing an evidence-based precision oncology strategy, and facilitating its rational and cost-effective implementation into the Portuguese healthcare system.
Topics: Humans; Precision Medicine; Portugal; Neoplasms; Antineoplastic Agents; Medical Oncology; Clinical Trials, Phase II as Topic; Molecular Targeted Therapy
PubMed: 38910310
DOI: 10.2340/1651-226X.2023.33322 -
BMJ Open Jun 2024Although the prognosis of Langerhans cell histiocytosis (LCH) is excellent, the high recurrence rate and permanent consequences, such as central diabetes insipidus and...
INTRODUCTION
Although the prognosis of Langerhans cell histiocytosis (LCH) is excellent, the high recurrence rate and permanent consequences, such as central diabetes insipidus and LCH-associated neurodegenerative diseases, remain to be resolved. Based on previous reports that patients with high-risk multisystem LCH show elevated levels of inflammatory molecules, we hypothesised that dexamethasone would more effectively suppress LCH-associated inflammation, especially in the central nervous system (CNS). We further hypothesised that intrathecal chemotherapy would effectively reduce CNS complications. We administer zoledronate to patients with multifocal bone LCH based on an efficacy report from a small case series.
METHODS AND ANALYSIS
This phase II study (labelled the LCH-19-MSMFB study) is designed to evaluate the significance of introducing dexamethasone and intrathecal chemotherapy for multisystem disease and zoledronate for multifocal bone disease in previously untreated, newly diagnosed children, adolescents (under 20 years) and adults under 40 years. The primary endpoint is the 3-year event-free survival rate by risk group of under 20 years and the 3-year event-free survival rate of 20 years and over.
ETHICS AND DISSEMINATION
This study was approved by the Central Review Board of the National Hospital Organisation Nagoya Medical Centre (Nagoya, Japan) on 21 January 2022 and was registered in the Japan Registry of Clinical Trials (https://jrct.niph.go.jp/en-latest-detail/jRCTs041210027). Written informed consent will be obtained from all patients and/or their guardians.
TRIAL REGISTRATION NUMBER
jRCTs041210027.
Topics: Humans; Histiocytosis, Langerhans-Cell; Child; Adolescent; Japan; Adult; Dexamethasone; Young Adult; Zoledronic Acid; Male; Female; Clinical Trials, Phase II as Topic; Child, Preschool; Bone Density Conservation Agents
PubMed: 38910000
DOI: 10.1136/bmjopen-2024-084159 -
BMJ Open Jun 2024Participation in bowel cancer screening is lower in regions where there is high ethnic diversity and/or socioeconomic deprivation. Interventions, such as text message... (Randomized Controlled Trial)
Randomized Controlled Trial Comparative Study
Comparing the effectiveness and cost-effectiveness of text-message reminders and telephone patient navigation to improve the uptake of faecal immunochemical test screening among non-responders in London: a randomised controlled trial protocol.
INTRODUCTION
Participation in bowel cancer screening is lower in regions where there is high ethnic diversity and/or socioeconomic deprivation. Interventions, such as text message reminders and patient navigation (PN), have the potential to increase participation in these areas. As such, there is interest in the comparative effectiveness of these interventions to increase bowel cancer screening participation, as well as their relative cost-effectiveness.
METHODS AND ANALYSIS
This study will use a three-arm randomised controlled trial design to compare the effectiveness and cost-effectiveness of text message reminders and PN to increase the uptake of bowel cancer screening in London. Participants will be individuals who have not returned a completed faecal immunochemical test kit within 13 weeks of receiving a routine invitation from the London bowel cancer screening hub. Participants will be randomised (in a 1:1:1 ratio) to receive either (1) usual care (ie, 'no intervention'), (2) a text message reminder at 13 weeks, followed by repeated text message reminders at 15, 17 and 19 weeks (in the event of non-response) or (3) a text message reminder at 13 weeks, followed by PN telephone calls at 15, 17 and 19 weeks in the event of non-response. The primary endpoint will be participation in bowel cancer screening, defined as 'the return of a completed kit by week 24'. Statistical analysis will use multivariate logistic regression and will incorporate pairwise comparisons of all three groups, adjusted for multiple testing.
ETHICS AND DISSEMINATION
Approvals to conduct the research have been obtained from University College London's Joint Research Office (Ref: 150666), the Screening Research, Innovation and Development Advisory Committee ('RIDAC', Ref: 2223 014 BCSP Kerrison), the Health Research Authority (Ref: 22/WM/0212) and the Confidentiality Advisory Group (Ref: 22/CAG/0140). Results will be conveyed to stakeholders, notably those managing the screening programme and published in peer-reviewed journals/presented at academic conferences.
TRIAL REGISTRATION NUMBER
ISRCTN17245519.
Topics: Humans; Text Messaging; London; Cost-Benefit Analysis; Early Detection of Cancer; Reminder Systems; Occult Blood; Colorectal Neoplasms; Telephone; Patient Navigation; Randomized Controlled Trials as Topic
PubMed: 38909999
DOI: 10.1136/bmjopen-2023-079482 -
The Spine Journal : Official Journal of... Jun 2024Traumatic subaxial cervical spine fractures are a significant public health concern due to their association with spinal cord injuries (SCI). Despite being mostly caused...
BACKGROUND
Traumatic subaxial cervical spine fractures are a significant public health concern due to their association with spinal cord injuries (SCI). Despite being mostly caused by low-energy trauma, these fractures significantly contribute to morbidity and mortality. Currently, research regarding early mortality based on the choice of treatment following these fractures is limited. Identifying predictors of early mortality may aid in postoperative patient monitoring and improve outcomes.
PURPOSE
This study aimed to identify predictors of 30-days, 90-days, and one-year mortality in adults treated for subaxial fractures.
STUDY DESIGN
A retrospective review of the nationwide Swedish Fracture Register (SFR).
PATIENT SAMPLE
All adult patients in the SFR who underwent treatment for a subaxial cervical fracture (n = 1,963).
OUTCOME MEASURES
Analyzed variables included age, sex, injury mechanism, neurological function, fracture characteristics, and treatment type. The primary endpoints were 30-days, 90-days, and one-year mortality.
METHODS
1,963 patients in the SFR, treated for subaxial cervical fractures between 2013 to 2021, were analyzed. Surgical procedures included anterior, posterior, or anteroposterior approaches. Non-surgical treatment included collar treatment or medical examinations without intervention. Stepwise regression and Cox regression analysis were used to determine predictors. Model performance was tested using the area under the receiver operating characteristic curve (AUC).
RESULTS
620 patients underwent surgery and 1,343 received non-surgical treatment. Surgical cases had primarily translation fractures, with 323 (52%) displaying no neurological deficits. Mortality rates at 30 days, 90 days, and one year were 22/620 (3.5%), 35/620 (5.6%), and 53/620 (8.5%), respectively. Age and SCI were predictors of mortality. Non-surgically treated patients mostly had compression fracture, with 1,214 (90%) experiencing no neurological deficits. Mortality rates at 30 days, 90 days, and one year were 41/1,343 (3.1%), 71/1,343 (5.3%), and 118/1,343 (8.7%). Age, male sex, SCI and fractures occurring at the C3 or C6 levels were predictors of mortality. An intact neurological function was a positive predictor of survival among non-surgically treated patients (AUC > 0.78).
CONCLUSIONS
Age and SCI emerged as significant predictors of early mortality in both surgically and non-surgically treated patients. An intact neurological function served as a protective factor against early mortality in non-surgically treated patients. Fractures at C3 or C6 vertebrae may impact mortality.
PubMed: 38909908
DOI: 10.1016/j.spinee.2024.06.015 -
BMC Neurology Jun 2024Parkinson's disease (PD) is a neurodegenerative disease for which no disease-modifying therapies exist. Preclinical and clinical evidence suggest that repeated exposure... (Randomized Controlled Trial)
Randomized Controlled Trial
BACKGROUND
Parkinson's disease (PD) is a neurodegenerative disease for which no disease-modifying therapies exist. Preclinical and clinical evidence suggest that repeated exposure to intermittent hypoxia might have short- and long-term benefits in PD. In a previous exploratory phase I trial, we demonstrated that in-clinic intermittent hypoxia exposure is safe and feasible with short-term symptomatic effects on PD symptoms. The current study aims to explore the safety, tolerability, feasibility, and net symptomatic effects of a four-week intermittent hypoxia protocol, administered at home, in individuals with PD.
METHODS/DESIGN
This is a two-armed double-blinded randomized controlled trial involving 40 individuals with mild to moderate PD. Participants will receive 45 min of normobaric intermittent hypoxia (fraction of inspired oxygen 0.16 for 5 min interspersed with 5 min normoxia), 3 times a week for 4 weeks. Co-primary endpoints include nature and total number of adverse events, and a feasibility-tolerability questionnaire. Secondary endpoints include Movement Disorders Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part II and III scores, gait tests and biomarkers indicative of hypoxic dose and neuroprotective pathway induction.
DISCUSSION
This trial builds on the previous phase I trial and aims to investigate the safety, tolerability, feasibility, and net symptomatic effects of intermittent hypoxia in individuals with PD. Additionally, the study aims to explore induction of relevant neuroprotective pathways as measured in plasma. The results of this trial could provide further insight into the potential of hypoxia-based therapy as a novel treatment approach for PD.
TRIAL REGISTRATION
ClinicalTrials.gov Identifier: NCT05948761 (registered June 20th, 2023).
Topics: Humans; Parkinson Disease; Hypoxia; Double-Blind Method; Male; Middle Aged; Female; Aged; Adult
PubMed: 38909201
DOI: 10.1186/s12883-024-03702-3 -
Scientific Reports Jun 2024The number of patients with atrial fibrillation is increasing, and frailty prevalence increases with age, posing challenges for physicians in prescribing anticoagulants... (Observational Study)
Observational Study
The number of patients with atrial fibrillation is increasing, and frailty prevalence increases with age, posing challenges for physicians in prescribing anticoagulants to such patients because of possible harm. The effects of frailty on anticoagulant therapy in older Japanese patients with nonvalvular atrial fibrillation (NVAF) are unclear. Herein, we prescribed rivaroxaban to Japanese patients with NVAF and monitored for a mean of 2.0 years. The primary endpoint was stroke or systemic embolism. The secondary endpoints were all-cause or cardiovascular death, composite endpoint, and major or non-major bleeding. Frailty was assessed using the Japanese long-term care insurance system. A multiple imputation technique was used for missing data. The propensity score (PS) was obtained to estimate the treatment effect of frailty and was used to create two PS-matched groups. Overall, 5717 older patients had NVAF (mean age: 73.9 years), 485 (8.5%) were classified as frail. After PS matching, background characteristics were well-balanced between the groups. Rivaroxaban dosages were 10 and 15 mg/day for approximately 80% and the remaining patients, respectively. Frailty was not associated with the primary endpoint or secondary endpoints. In conclusion, frailty does not affect the effectiveness or safety of rivaroxaban anticoagulant therapy in older Japanese patients with NVAF.Trial registration: UMIN000019135, NCT02633982.
Topics: Humans; Atrial Fibrillation; Aged; Male; Female; Frailty; Rivaroxaban; Aged, 80 and over; Anticoagulants; Japan; Stroke; Frail Elderly; Hemorrhage; Factor Xa Inhibitors; East Asian People
PubMed: 38909144
DOI: 10.1038/s41598-024-65237-4