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1.
Brain Sci ; 14(10)2024 Oct 06.
Article in English | MEDLINE | ID: mdl-39452022

ABSTRACT

Background: Traumatic brain injuries (TBIs) are a common consequence of experiencing intimate partner violence (IPV). IPV-related TBI contributes to adverse health outcomes among women, but it is unknown whether a history of IPV-related TBI negatively impacts safety outcomes following healthcare-based interventions for IPV. Methods: Using data from a larger randomized clinical trial, we explored the impact of IPV-related TBI status on safety-related outcomes in two healthcare-based IPV interventions. At baseline, 35% (n = 21) of the sample screened positive for IPV-related TBI history. We used repeated measures ANOVAs to examine differences in safety outcomes at post-treatment and 1-month follow-up based on treatment condition and IPV-related TBI status. Results: Significant interaction effects were found for safety outcomes, such that women with IPV-related TBI history experienced larger reductions in the frequency of physical IPV and similar reductions in sexual IPV across both treatment conditions compared to women without IPV-TBI (F(2, 102) = 10.88, p < 0.001; F(2, 98) = 3.93, p = 0.036). Conclusions: Findings suggest that brief healthcare-based IPV interventions may result in improvements in safety outcomes for women with a history of IPV-TBI. This highlights the continued need for implementation of promising IPV-focused interventions to promote safety and protect women from experiencing further IPV.

2.
Value Health ; 2024 Oct 08.
Article in English | MEDLINE | ID: mdl-39389353

ABSTRACT

OBJECTIVES: This study examines the impact of slippage in hazard ratios (tending towards the null over subsequent datacuts) for overall survival for combination treatment with a PD-(L)-1 inhibitor and a tyrosine kinase inhibitor (TKI) in advanced renal cell carcinoma (RCC). METHODS: Four trials' Kaplan Meier curves were digitized over several datacuts and fitted with standard parametric curves. Accuracy and consistency of early data projections were calculated versus observed restricted mean survival time (RMST) and fitted lifetime survival from the longest follow-up datacut. The change in economically justifiable price (eJP) was calculated fitting the same curve to both arms, using an assumed average utility of 0.7 and willingness-to-pay threshold of £30,000 per QALY. The eJP represents the lifetime justifiable price increment for the new treatment, including differences in drug, administration and disease-related costs. RESULTS: Slippage in hazard ratios was observed in trials with longer follow-up, potentially influenced by subsequent PD-(L)-1 use after TKI monotherapy, early stoppage of PD-(L)-1 and development of resistance. Lognormal and log-logistic curves were more likely to over-predict the observed result; Gompertz and gamma under-predicted. Statistical measures for goodness of fit did not reliably predict the RMST. Large differences in incremental mean life years were observed between even the penultimate and final datacuts for the majority of the fitted curves, meaningfully impacting the eJP. CONCLUSIONS: This work demonstrates the challenge in predicting treatment benefits with novel therapies using immature data. Incorporating information on the impact of subsequent treatment is likely to play a key role in improving predictions.

3.
OTJR (Thorofare N J) ; : 15394492241277470, 2024 Oct 22.
Article in English | MEDLINE | ID: mdl-39437008

ABSTRACT

As the literature states, interoception is important in health and wellness. Disruption of this system can negatively impact occupational performance. We completed a descriptive study to understand how occupational therapy practitioners working with adults address interoceptive awareness. A researcher-developed survey gathered data on knowledge acquisition of interoception and perceived practice patterns related to assessment, intervention, and outcomes. Participants (N = 44) reported acquiring knowledge of interoception through professional websites, browsing databases, and continuing education as the most common information sources. Client self-report (56.8%) and clinical observation (54.5%) were the most commonly reported assessments. Rehabilitation practitioners (n = 8) reported no use of formal assessments. Most participants (63.89%) reported interoceptive awareness interventions to be effective. Further research is needed on best practices for interoception assessment and intervention related to occupational performance.


Interoception, Sensing and Knowing What Is Happening in the Body: Understanding the Knowledge and Practice Patterns of Occupational Therapy Practitioners in Evaluating and Treating Interoception with the Adult Population.Interoception is an advanced sensory system that provides sensory information from the inside of the body to help with the regulation of emotions and support homeostasis. A person's ability to effectively utilize interoceptive awareness routinely has been implicated with supporting overall health and well-being. The authors completed a study to better understand how occupational therapy practitioners (OTPs) working with adult clients with physical and mental health concerns evaluate and use interoceptive awareness interventions to promote health and wellness. Information was collected from 44 OTPs working with adults regarding their knowledge and practices on the topic of interoception. The study conducted by the authors revealed that the practitioners mainly gained knowledge about interoception from browsing databases and professional websites. Therapists reported evaluation of interoceptive awareness through client observation, client report, and limited formal testing was used. A substantial number of participants (63.89%) felt addressing interoceptive awareness effectively achieved health, well-being, and occupational participation. Further studies are needed to help inform the occupational therapy community about the importance of evaluating and treating interoception. Supportive data from research can guide educators and practitioners in creating a uniform way to address interoception taught in academic settings and used as best practice to achieve desired clinical outcomes and support holistic well-being for the adult population.

4.
Int J Mol Sci ; 25(17)2024 Sep 09.
Article in English | MEDLINE | ID: mdl-39273698

ABSTRACT

Fabry disease (FD, OMIM #301500) is caused by pathogenic GLA gene (OMIM #300644) variants, resulting in a deficiency of the α-galactosidase A enzyme with accumulation of its substrate globotriaosylceramide and its derivatives. The phenotype of FD is highly variable, with distinctive disease features and course in classical male patients but more diverse and often nonspecific features in non-classical and female patients. FD-specific therapies have been available for approximately two decades, yet establishing robust evidence for long-term effectiveness remains challenging. This review aims to identify the factors contributing to this lack of robust evidence for the treatment of FD with enzyme replacement therapy (ERT) (agalsidase-alfa and -beta and pegunigalsidase alfa) and chaperone therapy (migalastat). Major factors that have been identified are study population heterogeneity (concerning sex, age, phenotype, disease stage) and differences in study design (control groups, outcomes assessed), as well as the short duration of studies. To address these challenges, we advocate for patient matching to improve control group compatibility in future FD therapy studies. We recommend international collaboration and harmonization, facilitated by an independent FD registry. We propose a stepwise approach for evaluating the effectiveness of novel treatments, including recommendations for surrogate outcomes and required study duration.


Subject(s)
Enzyme Replacement Therapy , Fabry Disease , alpha-Galactosidase , Fabry Disease/therapy , Fabry Disease/genetics , Fabry Disease/drug therapy , Humans , Enzyme Replacement Therapy/methods , alpha-Galactosidase/genetics , alpha-Galactosidase/therapeutic use , Treatment Outcome , Female , 1-Deoxynojirimycin/analogs & derivatives , 1-Deoxynojirimycin/therapeutic use , Male , Isoenzymes/genetics , Recombinant Proteins/therapeutic use
5.
HCA Healthc J Med ; 5(4): 405-413, 2024.
Article in English | MEDLINE | ID: mdl-39290488

ABSTRACT

Background: While the reported prevalence of polyneuropathies is 1%-3%, the incidence of hereditary transthyretin amyloidosis in the United States is estimated to be 1 in 100 000 individuals. Polyneuropathies are known to be difficult to treat and lead to significant morbidity. The aim of pain management is symptomatic treatment, with varying approaches to progression prevention being based on the causative pathophysiology.We assessed the prevalence of hereditary amyloid transthyretin variant (ATTRv) amyloidosis, a progressive autosomal dominant multisystem disease caused by the abnormal formation and extracellular deposition of transthyretin protein fibrils in various tissues, in an idiopathic polyneuropathy population by using genetic analysis. Methods: Individuals aged 18 and over with an established diagnosis of polyneuropathy, via electromyography testing that was deemed to be idiopathic, at a large, urban neurology clinic consented to an institutional review board-approved protocol for genetic testing. No further exclusions were made regarding age of onset, family history, axonal neuropathy subtype, comorbidities suggestive of ATTRv amyloidosis, etc. Clinical genetic testing was performed on 134 participants via an 81-gene panel associated with inherited neuromuscular disorders or targeted TTR gene sequencing with deletion and duplication analysis. Results: Within our cohort, 38.06% had at least one reportable finding in one of 38 distinct genes, for a total of 76 reported alterations. Four individuals were identified as having a single pathogenic alteration in an autosomal recessive gene, consistent with carrier status for the 4 following disorders: congenital insensitivity to pain with anhidrosis (NTRK1), Charcot-Marie-Tooth disease type IIP (LRSAM1), Brown-Vialetto-Van Laere syndrome type II (SLC52A2), hereditary sensory and autonomic neuropathy type III (IKBKAP). One individual was found to have a variant of uncertain significance (VUS) (p.G103D) in the TTR gene. Conclusion: Precision medicine on the molecular level with genetic testing in the identification of specific neuropathies may provide clinicians with more detailed information for developing a more direct therapeutic and treatment modality for better-targeted management. Further investigation is needed to expand on the knowledge and understanding of the clinical relevance surrounding the alterations found in the genetic evaluation of idiopathic neuropathy.

6.
HCA Healthc J Med ; 5(4): 397-404, 2024.
Article in English | MEDLINE | ID: mdl-39290490

ABSTRACT

Description Spinal epidural abscess (SEA), a critical surgical emergency, demands prompt recognition and intervention to prevent severe complications and fatalities. The incidence of SEA is notably increasing, particularly among individuals with diabetes, intravenous drug use, or a history of invasive spinal procedures. Although SEA can manifest through various clinical symptoms, the presence of its classic triad-back pain, fever, and neurological deficits-is noteworthy despite its occurrence in only 10% to 13% of cases. Identifying this triad is vital due to its high specificity for SEA, which is essential to guiding swift diagnostic and therapeutic actions in a condition where early intervention is critical. Magnetic resonance imaging is pivotal in diagnosing SEA, offering unmatched sensitivity and specificity compared to other imaging techniques. Immediate empirical antibiotic therapy and timely neurosurgical consultation, when required, form the foundation of SEA treatment. The prognosis significantly depends on the patient's initial neurological status, underlying health conditions, and the timeliness of their presentation, diagnosis, and treatment initiation. Given the complexity of SEA and the high risk of diagnostic delays, managing this condition involves substantial medicolegal considerations. Enhanced comprehension of SEA is imperative for improving patient outcomes and reducing health care resource burdens. Prompt and accurate diagnosis and appropriate interventions are essential for effectively managing this urgent condition.

7.
J Pharm Bioallied Sci ; 16(Suppl 3): S2770-S2772, 2024 Jul.
Article in English | MEDLINE | ID: mdl-39346471

ABSTRACT

Background: Because of its ease and visual attractiveness, clear aligner therapy has grown in popularity as an orthodontic treatment option. On the other hand, the results of therapy may be impacted by attachment wear. Comparing attachment wear in three distinct clear aligner systems-System A, System B, and System C-was the goal of this investigation. Methods: The research comprised 60 individuals receiving clear aligners as part of their orthodontic treatment. Depending on whatever clear aligner system was being utilised, the patients were split into three groups. Attachment wear was evaluated using a standardised scoring method regularly. The various systems' attachment wear was compared using statistical analysis. Results: Compared to Systems B and C, System A showed less attachment wear. There were notable variations in attachment wear ratings between the systems. System C's attachments had the most wear, whereas System A's attachments had the least. Conclusion: Different clear aligner systems showed varying degrees of attachment wear, with System A exhibiting the least amount of wear. To maximise treatment procedures and guarantee treatment efficacy and longevity in clear aligner therapy, it is vital to comprehend attachment wear patterns.

8.
Lung Cancer ; 195: 107919, 2024 Sep.
Article in English | MEDLINE | ID: mdl-39197358

ABSTRACT

INTRODUCTION: With multiple targeted therapies approved for anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), it is increasingly important to understand outcomes with various sequences of next-generation ALK tyrosine kinase inhibitors (TKIs). We describe contemporary sequencing patterns and treatment effectiveness of first-line (1L) and second-line (2L) treatments in patients who received second-generation ALK TKIs in the 1L treatment of ALK-positive NSCLC in the United States. METHODS: A cohort of adults with ALK-positive advanced NSCLC who initiated treatment with 1L alectinib or brigatinib between June 2017 and April 2021 in the Flatiron Health electronic health record-derived de-identified database were followed through April 2023. Time to treatment discontinuation (TTD) in 1L and 2L, TTD on 1L plus 2L sequential therapy (TTD2), and total time on sequential ALK TKI therapy (including beyond 2L) were evaluated. RESULTS: Patients (N=273) were followed up for a median duration of 28.9 months. Among patients who discontinued 1L therapy, 22% died after 1L discontinuation (median time from discontinuation to death, 4.0 months) without receiving 2L therapy. Median (95% confidence interval [CI]) TTD was 21.9 (15.2-25.8) and 7.3 (5.3-10.2) months in 1L and 2L, respectively. Median (95% CI) TTD2 was 29.4 (25.1-36.1) months and total time on sequential ALK TKI treatment was 28.0 (23.6-32.9) months. CONCLUSIONS: In this large real-world study, TTD2 and the total time on sequential ALK TKIs was approximately 2.5 years. The high attrition rate from 1L to 2L and the longest clinical benefit observed with 1L therapy support using the drug with the longest 1L effectiveness up front in patients with ALK-positive advanced NSCLC.


Subject(s)
Anaplastic Lymphoma Kinase , Carcinoma, Non-Small-Cell Lung , Lung Neoplasms , Protein Kinase Inhibitors , Humans , Carcinoma, Non-Small-Cell Lung/drug therapy , Carcinoma, Non-Small-Cell Lung/mortality , Carcinoma, Non-Small-Cell Lung/genetics , Carcinoma, Non-Small-Cell Lung/pathology , Lung Neoplasms/drug therapy , Lung Neoplasms/mortality , Lung Neoplasms/genetics , Lung Neoplasms/pathology , Male , Anaplastic Lymphoma Kinase/genetics , Anaplastic Lymphoma Kinase/antagonists & inhibitors , Protein Kinase Inhibitors/therapeutic use , Female , Middle Aged , Aged , Adult , Aged, 80 and over , Treatment Outcome , Follow-Up Studies , Retrospective Studies , Piperidines/therapeutic use , Carbazoles , Organophosphorus Compounds , Pyrimidines
9.
Influenza Other Respir Viruses ; 18(6): e13311, 2024 Jun.
Article in English | MEDLINE | ID: mdl-38840301

ABSTRACT

In September 2023, France was one of the first countries that started a national immunisation campaign with nirsevimab, a new monoclonal antibody against respiratory syncytial virus (RSV). Using data from a network of paediatric intensive care units (PICUs), we aimed to estimate nirsevimab effectiveness against severe cases of RSV bronchiolitis in France. We conducted a case-control study based on the test-negative design and included 288 infants reported by 20 PICUs. We estimated nirsevimab effectiveness at 75.9% (48.5-88.7) in the main analysis and 80.6% (61.6-90.3) and 80.4% (61.7-89.9) in two sensitivity analyses. These real-world estimates confirmed the efficacy observed in clinical studies.


Subject(s)
Hospitalization , Intensive Care Units, Pediatric , Respiratory Syncytial Virus Infections , Humans , France/epidemiology , Respiratory Syncytial Virus Infections/drug therapy , Infant , Intensive Care Units, Pediatric/statistics & numerical data , Case-Control Studies , Male , Female , Hospitalization/statistics & numerical data , Respiratory Syncytial Virus, Human/drug effects , Antibodies, Monoclonal, Humanized/therapeutic use , Antiviral Agents/therapeutic use , Bronchiolitis/drug therapy , Bronchiolitis/virology , Bronchiolitis, Viral/drug therapy , Bronchiolitis, Viral/virology , Treatment Outcome
10.
Rheumatol Ther ; 11(4): 989-999, 2024 Aug.
Article in English | MEDLINE | ID: mdl-38858318

ABSTRACT

INTRODUCTION: With an increasing number of biologic/targeted synthetic disease-modifying antirheumatic drug options available for the treatment of active ankylosing spondylitis (AS), also known as radiographic axial spondyloarthritis, it is of clinical interest to determine the comparative efficacy of these advanced therapies among populations with differing prior advanced therapy exposure. This study aimed to assess the comparative efficacy of approved advanced therapies for AS in tumor necrosis factor inhibitor (TNFi)-naïve and, separately, in TNFi inadequate responder/intolerant (-IR) populations. METHODS: A systematic literature review was conducted to identify randomized clinical trials for TNFis, interleukin-17A inhibitors, and Janus kinase inhibitors used as advanced therapies for active AS. Clinical efficacy was considered by the Ankylosing Spondylitis Disease Activity Score low disease activity (ASDAS LDA) criteria, defined as ASDAS score less than 2.1, among approved therapies. Comparative efficacy in the TNFi-naïve population was assessed utilizing network meta-analysis, while comparative efficacy in the TNFi-IR population was assessed utilizing matching-adjusted indirect comparison. Odds ratios were calculated, from which absolute rates and numbers needed to treat were calculated. Safety in the form of trial-reported and placebo-adjusted rates of discontinuation due to adverse events (AEs) was reviewed. RESULTS: Among the TNFi-naïve population, the estimated ASDAS LDA rate between week 12 and 16 was highest for patients treated with upadacitinib (52.8%) and lowest for patients treated with placebo (11.6%). Among the TNFi-IR population, the estimated ASDAS LDA rate was 41.3% for patients treated with upadacitinib and 17.5% for patients treated with ixekizumab. The trial-reported and placebo-adjusted rates of discontinuation due to AEs were generally low across included advanced therapies. CONCLUSIONS: Relative to other assessed therapies, upadacitinib demonstrated greater clinical efficacy per ASDAS LDA in the treatment of active AS in both TNFi-naïve and TNFi-IR populations. Head-to-head and real-world data comparisons are warranted to both validate these findings and aid medical decision makers.

11.
Eur J Radiol ; 177: 111555, 2024 Aug.
Article in English | MEDLINE | ID: mdl-38880053

ABSTRACT

PURPOSE: Three-dimensional contrast-enhanced magnetic resonance imaging (3D-Ce-MRI) is a most powerful tool for evaluation of neoadjuvant chemotherapy (NAC). However, the use of contrast agent is invasive, expensive, and time consuming, Thus, contrast agent-free imaging is preferable. We aimed to investigate the tumor volume change after NAC using maximum intensity projection diffusion-weighted image (MIP-DWI) and 3D-Ce-MRI. METHOD: We finally enrolled 55 breast cancer patients who underwent NAC in 2018. All MRI analyses were performed using SYNAPSE VINCENT® medical imaging system (Fujifilm Medical, Tokyo, Japan). We evaluated the tumor volumes before, during, and after NAC. Tumor volume before NAC on 3D-Ce-MRI was termed Pre-CE and those during and after NAC were termed Post-CE. The observer raised the lower end of the window width until the tumor was clearly visible and then manually deleted the non-tumor tissues. A month thereafter, the same observer who was blinded to the 3D-Ce-MRI results randomly evaluated the tumor volumes (Pre-DWI and Post-DWI) using MIP-DWI with the same method. Tumor volume change between ΔCE (Pre-CE - Post-CE/Pre-CE) and ΔDWI (Pre-DWI - Post-DWI/Pre-DWI) and the processing time for both methods (Time-DWI and Time-CE) were compared. RESULTS: We enrolled 55 patients. Spearman's rho between ΔDWI and ΔCE for pure mass lesions, and non-mass enhancement (NME) was 0.89 (p < 0.01), 0.63(p < 0.01) respectively. Time-DWI was significantly shorter than Time-CE (41.3 ± 21.2 and 199.5 ± 98.3 respectively, p < 0.01). CONCLUSIONS: Non-contrast-enhanced Breast MRI enables appropriate and faster evaluation of tumor volume change after NAC than 3D-Ce-MRI especially for mass lesions.


Subject(s)
Breast Neoplasms , Imaging, Three-Dimensional , Magnetic Resonance Imaging , Neoadjuvant Therapy , Tumor Burden , Humans , Breast Neoplasms/diagnostic imaging , Breast Neoplasms/drug therapy , Female , Neoadjuvant Therapy/methods , Middle Aged , Imaging, Three-Dimensional/methods , Adult , Aged , Magnetic Resonance Imaging/methods , Chemotherapy, Adjuvant , Diffusion Magnetic Resonance Imaging/methods , Contrast Media
12.
Front Immunol ; 15: 1438587, 2024.
Article in English | MEDLINE | ID: mdl-38895125

ABSTRACT

[This corrects the article DOI: 10.3389/fimmu.2024.1368749.].

13.
J Dermatol ; 51(8): 1060-1067, 2024 Aug.
Article in English | MEDLINE | ID: mdl-38895831

ABSTRACT

Autologous cultured pure melanocyte transplantation (CMT) can be utilized to treat stable vitiligo cases, but clinical data are insufficient to improve its efficacy. To evaluate the influence of various factors on the therapeutic effect of CMT, this single-center retrospective study enrolled stable vitiligo patients who underwent CMT between 2009 and 2020. Univariate and multivariable analysis were used to determine the factors affecting the outcome of repigmentation. The study included 491 patients with long-term follow-up data (6-120 months). It was found that 69.7% of patients achieved an excellent re-color effect and 18.4% achieved a good re-color effect. There were statistically significant differences in pigmentation between patients with stable disease course, vitiligo type, and lesion site. Overall, a significant positive correlation between the target area treatment ratio of varied lesions and the percentage of repigmentation was found. CMT is effective and well tolerated in the treatment of stable vitiligo. Various factors, especially the target area treatment ratio of varied lesions, should be carefully assessed before using CMT. As the target area treatment ratio of varied lesions could further improve the post-operative repigmentation other than type of vitiligo. This clinic trial was approved by Hangzhou Third People's Hospital (number 2023KA015, national clinical record number MR-33-23-034502).


Subject(s)
Melanocytes , Skin Pigmentation , Transplantation, Autologous , Vitiligo , Humans , Vitiligo/therapy , Retrospective Studies , Female , Male , Adult , Melanocytes/transplantation , Young Adult , Adolescent , Middle Aged , Treatment Outcome , Child , Follow-Up Studies , Cells, Cultured , Child, Preschool
14.
Front Psychol ; 15: 1369487, 2024.
Article in English | MEDLINE | ID: mdl-38770259

ABSTRACT

The prevalence and impact of chronic pain in individuals worldwide necessitate effective management strategies. This narrative review specifically aims to assess the effectiveness of neurofeedback, an emerging non-pharmacological intervention, on the management of chronic pain. The methodology adopted for this review involves a meticulous search across various scientific databases. The search was designed to capture a broad range of studies related to neurofeedback and chronic pain management. To ensure the quality and relevance of the included studies, strict inclusion and exclusion criteria were applied. These criteria focused on the study design, population, intervention type, and reported outcomes. The review synthesizes the findings from a diverse array of studies, including randomized controlled trials, observational studies, and case reports. Key aspects evaluated include the types of neurofeedback used (such as EEG biofeedback), the various chronic pain conditions addressed (like fibromyalgia, neuropathic pain, and migraines), and the methodologies employed in these studies. The review highlights the underlying mechanisms by which neurofeedback may influence pain perception and management, exploring theories related to neural plasticity, pain modulation, and psychological factors. The results of the review reveal a positive correlation between neurofeedback interventions and improved pain management. Several studies report significant reductions on pain intensity, improved quality of life, and decreased reliance on medication following neurofeedback therapy. The review also notes variations in the effectiveness of different neurofeedback protocols and individual responses to treatment. Despite the promising results, the conclusion of the review emphasizes the need for further research. It calls for larger, well-designed clinical trials to validate the findings, to understand the long-term implications of neurofeedback therapy, and to optimize treatment protocols for individual patients.

15.
Nervenarzt ; 95(7): 651-655, 2024 Jul.
Article in German | MEDLINE | ID: mdl-38753178

ABSTRACT

Antidepressive pharmacotherapy has undergone various phases in its history. The euphoria of the early years on the relief of depressive symptoms was followed by a long period of clinical experience and intensive scientific work resulting in a more balanced perspective. Current debates circle around the actual effectiveness, especially with respect to long-term treatment, the prevention of suicide and the sequelae of discontinuation of an antidepressant. The evaluation of antidepressants as a group and often also the risk-benefit ratio of an individual treatment change over time. Antidepressants are typical for many forms of psychiatric treatment which, in a term from Hanfried Helmchen, are just as Janus-faced as psychiatry in a general sense is as a science and as a clinical discipline.


Subject(s)
Antidepressive Agents , Humans , Antidepressive Agents/therapeutic use , Antidepressive Agents/adverse effects , Risk Assessment , Suicide Prevention , Depressive Disorder/drug therapy , Treatment Outcome , Evidence-Based Medicine , Psychiatry
16.
Future Oncol ; 20(22): 1553-1563, 2024.
Article in English | MEDLINE | ID: mdl-38629593

ABSTRACT

Aim: To assess real-world clinical outcomes with standard therapies for advanced non-small-cell lung cancer (aNSCLC) with METexon14 skipping mutation (METex14).Methods: In an oncologists-led retrospective review of medical records, data were abstracted and analyzed for patients initiating first-line (1L) systemic therapy after 1 January 2017.Results: In total 287 aNSCLC patients with METex14, the real-world best overall response rate was 73.4% for capmatinib (n = 146), 68.8% for immunotherapy (IO) monotherapy (n = 48), 52.0% for chemotherapy (CT, n = 30), and 54.8% for IO + CT (n = 63). As compared with capmatinib, patients receiving IO (hazard ratio [HR]: 1.57; 95% CI: 0.77-3.20; p = 0.220), CT (HR: 2.41; 95% CI: 1.19-4.85; p = 0.014) and IO + CT (HR: 2.33; 95% CI: 1.35-4.04; p = 0.003) had higher rates of progression. Further, patients receiving CT (HR: 4.43; 95% CI: 1.54-12.75; p = 0.006) and IO + CT (HR: 3.53, 95% CI: 1.41-8.85; p = 0.007) had higher rates of mortality than patients receiving capmatinib.Conclusion: The study showed better clinical outcomes with capmatinib than other standard therapies in 1L setting for aNSCLC harboring METex14.


Real-world study that investigated the outcomes of different therapies used to treat non-small-cell lung cancer patients with mesenchymal-epithelial transition exon 14 skipping mutationWhat is this article about? A real-world study that investigated clinical outcomes in patients with diagnosis of advanced non-small-cell lung cancer (aNSCLC) with mesenchymal-epithelial transition exon 14 (METex14) skipping­a rare form of genetic mutation­who received treatment with one of the commonly used therapies for this disease: immunotherapy, chemotherapy, immunotherapy + chemotherapy combination and capmatinib, which is a highly selective inhibitor of MET tyrosine kinase protein involved in the growth of cancer cells.What were the results? The study showed that, in general, patients treated with capmatinib as the frontline therapy more frequently achieved a clinical response in the form of complete tumor resolution or tumor shrinkage, had a lower risk of disease worsening and lived longer than patients who were treated with immunotherapy, chemotherapy or immunotherapy + chemotherapy combination.What do the results of the study mean? This study suggests that capmatinib is effective in treating patients with aNSCLC with METex14 skipping who have not been treated with another anticancer therapy previously. It provides evidence to support the use of capmatinib in the frontline setting and may inform clinical decision-making in routine practice.


Subject(s)
Carcinoma, Non-Small-Cell Lung , Lung Neoplasms , Mutation , Proto-Oncogene Proteins c-met , Humans , Carcinoma, Non-Small-Cell Lung/genetics , Carcinoma, Non-Small-Cell Lung/drug therapy , Carcinoma, Non-Small-Cell Lung/mortality , Carcinoma, Non-Small-Cell Lung/therapy , Carcinoma, Non-Small-Cell Lung/pathology , Lung Neoplasms/genetics , Lung Neoplasms/drug therapy , Lung Neoplasms/mortality , Lung Neoplasms/pathology , Lung Neoplasms/therapy , Female , Male , Middle Aged , Retrospective Studies , Aged , Proto-Oncogene Proteins c-met/genetics , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Adult , Treatment Outcome , Immunotherapy/methods , Aged, 80 and over , Benzamides , Imidazoles , Triazines
17.
Magn Reson Imaging ; 110: 149-160, 2024 Jul.
Article in English | MEDLINE | ID: mdl-38621553

ABSTRACT

Phototherapy, also known as photobiological therapy, is a non-invasive and highly effective physical treatment method. Its broad use in clinics has led to significant therapeutic results. Phototherapy parameters, such as intensity, wavelength, and duration, can be adjusted to create specific therapeutic effects for various medical conditions. Meanwhile, Magnetic Resonance Imaging (MRI), with its diverse imaging sequences and excellent soft-tissue contrast, provides a valuable tool to understand the therapeutic effects and mechanisms of phototherapy. This review explores the clinical applications of commonly used phototherapy techniques, gives a brief overview of how phototherapy impacts different diseases, and examines MRI's role in various phototherapeutic scenarios. We argue that MRI is crucial for precise targeting, treatment monitoring, and prognosis assessment in phototherapy. Future research and applications will focus on personalized diagnosis and monitoring of phototherapy, expanding its applications in treatment and exploring multimodal imaging technology to enhance diagnostic and therapeutic precision and effectiveness.


Subject(s)
Magnetic Resonance Imaging , Phototherapy , Humans , Magnetic Resonance Imaging/methods , Phototherapy/methods , Treatment Outcome
18.
Front Med (Lausanne) ; 11: 1362192, 2024.
Article in English | MEDLINE | ID: mdl-38576716

ABSTRACT

Introduction: This study aims to discuss and assess the impact of three prevalent methodological biases: competing risks, immortal-time bias, and confounding bias in real-world observational studies evaluating treatment effectiveness. We use a demonstrative observational data example of COVID-19 patients to assess the impact of these biases and propose potential solutions. Methods: We describe competing risks, immortal-time bias, and time-fixed confounding bias by evaluating treatment effectiveness in hospitalized patients with COVID-19. For our demonstrative analysis, we use observational data from the registry of patients with COVID-19 who were admitted to the Bellvitge University Hospital in Spain from March 2020 to February 2021 and met our predefined inclusion criteria. We compare estimates of a single-dose, time-dependent treatment with the standard of care. We analyze the treatment effectiveness using common statistical approaches, either by ignoring or only partially accounting for the methodological biases. To address these challenges, we emulate a target trial through the clone-censor-weight approach. Results: Overlooking competing risk bias and employing the naïve Kaplan-Meier estimator led to increased in-hospital death probabilities in patients with COVID-19. Specifically, in the treatment effectiveness analysis, the Kaplan-Meier estimator resulted in an in-hospital mortality of 45.6% for treated patients and 59.0% for untreated patients. In contrast, employing an emulated trial framework with the weighted Aalen-Johansen estimator, we observed that in-hospital death probabilities were reduced to 27.9% in the "X"-treated arm and 40.1% in the non-"X"-treated arm. Immortal-time bias led to an underestimated hazard ratio of treatment. Conclusion: Overlooking competing risks, immortal-time bias, and confounding bias leads to shifted estimates of treatment effects. Applying the naïve Kaplan-Meier method resulted in the most biased results and overestimated probabilities for the primary outcome in analyses of hospital data from COVID-19 patients. This overestimation could mislead clinical decision-making. Both immortal-time bias and confounding bias must be addressed in assessments of treatment effectiveness. The trial emulation framework offers a potential solution to address all three methodological biases.

19.
Int J Neurosci ; : 1-8, 2024 Apr 16.
Article in English | MEDLINE | ID: mdl-38597661

ABSTRACT

OBJECTIVE: To analyze the effectiveness of stent retriever mechanical thrombectomy combined with tirofiban in treating acute ischemic stroke. METHODS: Markedly effective is defined as an SIS score of over 90, effective is indicated by an SIS score of between 50-90, and a score of below 50 suggests ineffective treatment results. RESULTS: ①The treatment's overall effectiveness in the observation group (91.30%) was significantly higher than that in the control group (75.56%) (p < 0.05). ②The vascular recanalization rate in the observation group (89.13%) was significantly higher than that in the control group (71.11%) (p < 0.05). ③The stent retrieval operation count (2.41 ± 0.23) was significantly lower in the observation group than in the control group (1.29 ± 0.16) (p < 0.05). ④ After treatment, the platelet aggregation rate (10.74 ± 3.95) and NIHSS scores (6.58 ± 1.04) were significantly lower, and the Barthel index (77.86 ± 7.21) was significantly higher in the observation group compared to the control group (26.47 ± 5.12, 7.75 ± 2.36, 68.12 ± 6.15) (p < 0.05). All platelet aggregation rate, NIHSS scores and Barthel Index showed significant improvement after treatment when compared to those before treatment (p < 0.05). CONCLUSION: The combined application of stent retriever mechanical thrombectomy and tirofiban in acute ischemic stroke treatment shows promising effectiveness. Compared to stent retriever alone, tirofiban adjunctive therapy enhances vascular recanalization, reduces retrieval procedures, shortens treatment duration, inhibits platelet aggregation, and improves neurological function recovery, daily living activities, and prognosis. Moreover, it doesn't significantly increase symptom-related risks.

20.
J Clin Med ; 13(8)2024 Apr 18.
Article in English | MEDLINE | ID: mdl-38673626

ABSTRACT

Background: There is evidence suggesting the existence of sex differences in the effectiveness of specific drug classes for rheumatoid arthritis (RA). Our study stands as the first to elucidate sex-related differences in the effectiveness of Janus kinase (JAK) inhibitors. Methods: The study involved 150 RA patients treated with tofacitinib, baricitinib, upadacitinib, or filgotinib between September 2017 and October 2023. Sex differences in achieving remission and low disease activity (LDA) were identified through logistic regression analyses. Sex disparities in treatment effectiveness survival were evaluated through the Kaplan-Meier estimate, employing the log-rank test for comparison. The Cox model was applied to analyze the variable sex as a potential factor that could influence the maintenance of the JAK inhibitor treatment effectiveness. Results: Concerning the achievement of remission and LDA, no differences were observed between sexes in terms of the 28-joint Disease Activity Score (DAS28) C-reactive protein (CRP), the Clinical Disease Activity Index (CDAI), and the Simplified Disease Activity Index (SDAI). With respect to the DAS28-erythrocyte sedimentation rate (ESR), female patients, compared to males, possessed 70% lower odds of achieving remission (p = 0.018) and 66% lower odds of achieving LDA (p = 0.023). No differences were observed in treatment effectiveness survival between sexes (p = 0.703). Sex was not found to influence the survival of JAK inhibitor treatment effectiveness (p = 0.704). Conclusions: Being a female or male patient does not entail differences in the effectiveness of the JAK inhibitor treatment. Our findings encourage the consideration of a global pool of composite indices (DAS28-ESR/CRP, CDAI, SDAI) to measure RA disease activity, thus individualizing the target value as advocated by the treat-to-target strategy.

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