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PloS One 2024Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the evidence regarding efficacy and safety.
OBJECTIVE
Conduct a meta-analysis to assess the efficacy and safety of onasemnogene abeparvovec in patients diagnosed with SMA type 1, based on the available evidence.
METHODS
This article results from searches conducted on databases up to November 2022. Outcomes of interest were global survival and event-free survival, improvement in motor function and treatment-related adverse events. Risk of bias assessment and certainty of evidence were performed for each outcome. Proportional meta-analysis models were performed when applicable.
RESULTS
Four reports of three open-label, non-comparative clinical trials covering 67 patients were included. Meta-analyses of data available in a 12-month follow-up estimate a global survival of 97.56% (95%CI: 92.55 to 99.86, I2 = 0%, n = 67), an event-free survival of 96.5% (95%CI: 90.76 to 99.54, I2 = 32%, n = 66) and a CHOP-INTEND score ≥ 40 points proportion of 87.28% (95%CI: 69.81 to 97.83, I2 = 69%, n = 67). Proportion of 52.64% (95%CI: 27.11 to 77.45, I2 = 78%, n = 67) of treatment-related adverse events was estimated.
CONCLUSION
The results indicate a potential change in the natural history of type 1 SMA, but the methodological limitations of the studies make the real extent of the technology's long-term benefits uncertain.
Topics: Humans; Spinal Muscular Atrophies of Childhood; Biological Products; Treatment Outcome; Recombinant Fusion Proteins
PubMed: 38713659
DOI: 10.1371/journal.pone.0302860 -
Supportive Care in Cancer : Official... May 2024The impact of sarcopenia in oncology is increasingly recognized, yet little is known about its clinical implications in breast cancer. This systematic review and... (Meta-Analysis)
Meta-Analysis Review
PURPOSE
The impact of sarcopenia in oncology is increasingly recognized, yet little is known about its clinical implications in breast cancer. This systematic review and meta-analysis estimates the overall prevalence of sarcopenia in breast cancer, quantifies skeletal muscle index (SMI), and comprehensively evaluates sarcopenia's impact on clinical outcomes.
METHODS
We systematically searched primary original research published before June 2023 in four databases: the Cochrane Library via Wiley, CINAHL Plus with Full Text, Embase via Elsevier Excerpta Medica, and Medline via Ovid. Standardized mean SMI and 95% confidence interval (CI) were calculated by applying the random-effects model. The methodological quality of the included studies was assessed using the National Institutes of Health quality assessment checklist.
RESULTS
The systematic review included 17 studies with a total of 9863 patients; the meta-analysis included 12 of these studies. The mean prevalence of sarcopenia in breast cancer (stages I-III) was 32.5%. The mean SMI assessed by CT was 43.94 cm/m (95% CI 42.87, 45.01; p < .01). Overall, low muscle mass was associated with chemotherapy toxicities, dose reductions, dose delays, or treatment discontinuation. Low muscle mass was generally associated with poor survival, but in some studies, this association was not significant or reversed direction.
CONCLUSION
Sarcopenia is not just a state of muscle mass loss, but an influencing factor on therapeutic effects and survival rates in oncology. It is thus necessary to recognize the risk of sarcopenia throughout the trajectory of cancer treatment, identify low muscle mass early, and manage it from a prehabilitation perspective.
Topics: Humans; Sarcopenia; Breast Neoplasms; Prevalence; Female
PubMed: 38702479
DOI: 10.1007/s00520-024-08532-0 -
Heliyon Apr 2024Magnetic resonance imaging (MRI) techniques, such as quantitative susceptibility mapping (QSM) and susceptibility-weighted imaging (SWI), can detect iron deposition in...
Parkinson's disease and Parkinsonism syndromes: Evaluating iron deposition in the putamen using magnetic susceptibility MRI techniques - A systematic review and literature analysis.
Magnetic resonance imaging (MRI) techniques, such as quantitative susceptibility mapping (QSM) and susceptibility-weighted imaging (SWI), can detect iron deposition in the brain. Iron accumulation in the putamen (PUT) can contribute to the pathogenesis of Parkinson's disease (PD) and atypical Parkinsonian disorders. This systematic review aimed to synthesize evidence on iron deposition in the PUT assessed by MRI susceptibility techniques in PD and Parkinsonism syndromes. The PubMed and Scopus databases were searched for relevant studies. Thirty-four studies from January 2007 to October 2023 that used QSM, SWI, or other MRI susceptibility methods to measure putaminal iron in PD, progressive supranuclear palsy (PSP), multiple system atrophy (MSA), and healthy controls (HCs) were included. Most studies have found increased putaminal iron levels in PD patients versus HCs based on higher quantitative susceptibility. Putaminal iron accumulation correlates with worse motor scores and cognitive decline in patients with PD. Evidence regarding differences in susceptibility between PD and atypical Parkinsonism is emerging, with several studies showing greater putaminal iron deposition in PSP and MSA than in PD patients. Alterations in putaminal iron levels help to distinguish these disorders from PD. Increased putaminal iron levels appear to be associated with increased disease severity and progression. Thus, magnetic susceptibility MRI techniques can detect abnormal iron accumulation in the PUT of patients with Parkinsonism. Moreover, quantifying putaminal susceptibility may serve as an MRI biomarker to monitor motor and cognitive changes in PD and aid in the differential diagnosis of Parkinsonian disorders.
PubMed: 38689949
DOI: 10.1016/j.heliyon.2024.e27950 -
Journal of Integrative Neuroscience Apr 2024Motor neuron diseases (MNDs) are progressive neurodegenerative disorders characterized by motor impairment and non-motor symptoms. The involvement of the thalamus in...
BACKGROUND
Motor neuron diseases (MNDs) are progressive neurodegenerative disorders characterized by motor impairment and non-motor symptoms. The involvement of the thalamus in MNDs, especially in conditions such as amyotrophic lateral sclerosis (ALS), and its interaction with frontotemporal dementia (FTD), has garnered increasing research interest. This systematic review analyzed magnetic resonance imaging (MRI) studies that focused on thalamic alterations in MNDs to understand the significance of these changes and their correlation with clinical outcomes.
METHODS
Following PRISMA 2020 guidelines, the PubMed and Scopus databases were searched from inception to June 2023 for studies related to MRI findings in the thalamus of patients with MNDs. Eligible studies included adult patients diagnosed with ALS or other forms of MND who underwent brain MRI, with outcomes related to thalamic alterations. Studies were evaluated for risk of bias using the Newcastle-Ottawa scale.
RESULTS
A total of 52 studies (including 3009 MND patients and 2181 healthy controls) used various MRI techniques, including volumetric analysis, diffusion tensor imaging, and functional MRI, to measure thalamic volume, connectivity, and other alterations. This review confirmed significant thalamic changes in MNDs, such as atrophy and microstructural degradation, which are associated with disease severity, progression, and functional disability. Thalamic involvement varies across different MND subtypes and is influenced by the presence of cognitive impairment and mutations in genes including chromosome 9 open reading frame 72 (). The synthesis of findings across studies indicates that thalamic pathology is a prevalent early biomarker of MNDs that contributes to motor and cognitive deficits. The thalamus is a promising target for monitoring as its dysfunction underpins a variety of clinical symptoms in MNDs.
CONCLUSIONS
Thalamic alterations provide valuable insights into the pathophysiology and progression of MNDs. Multimodal MRI techniques are potent tools for detecting dynamic thalamic changes, indicating structural integrity, connectivity disruption, and metabolic activity.
Topics: Humans; Thalamus; Motor Neuron Disease; Magnetic Resonance Imaging; Amyotrophic Lateral Sclerosis
PubMed: 38682227
DOI: 10.31083/j.jin2304077 -
Children (Basel, Switzerland) Mar 2024The effect of pediatric inguinal hernia repair (IHR) on testicular vascularization remains unclear. Manipulating the spermatic cord during surgery may reduce blood flow... (Review)
Review
BACKGROUND
The effect of pediatric inguinal hernia repair (IHR) on testicular vascularization remains unclear. Manipulating the spermatic cord during surgery may reduce blood flow due to edema and vasoconstriction. This can lead to testicular atrophy. The study aims to review current knowledge of testicular vascular impairment following IHR in children.
METHODS
A systematic literature search was conducted in PubMed/Medline, Embase, Cochrane Library, and Web of Science. Methodological quality was assessed using validated tools. Data were extracted, and a pooled data analysis was performed.
RESULTS
Ten studies were included in the systematic review. Six of these studies were eligible for meta-analysis. This revealed a significant decrease in testicular vascularization during the short-term follow-up (1 day-1 week) after IHR using the open surgical approach. This decrease was not present after laparoscopic intervention. There was no more increased resistance in the vessels at long-term follow-up (1 month-6 months), suggesting that the impaired vascularity is only temporary.
CONCLUSIONS
There seems to be a short-term transient vascular impairment of the testis after open IHR in children. This might be of clinical relevance to prefer the laparoscopic approach for IHR in children, even though the open approach is the gold standard, in contrast to adult IHR. The impact on testicular function and sperm quality later in life remains unclear. Comparative studies of both techniques are needed to determine if there is a significant difference in testicular vascularity. Long-term studies are necessary to assess the impact of transiently reduced vascularity on sperm quality and fertility later in life.
PubMed: 38671625
DOI: 10.3390/children11040409 -
Value in Health Regional Issues Jul 2024This study aims to systematically collect data on cost-effectiveness analyses that assess technologies to treat type I and II spinal muscular atrophy and evaluate their...
OBJECTIVES
This study aims to systematically collect data on cost-effectiveness analyses that assess technologies to treat type I and II spinal muscular atrophy and evaluate their recommendations.
METHODS
A structured electronic search was conducted in 4 databases. Additionally, a complementary manual search was conducted. Complete economic studies that evaluated nusinersen, risdiplam, onasemnogene abeparvovec (OA), and the best support therapy (BST) from the health system's perspective were selected. The incremental cost-effectiveness ratios were compared with various thresholds for the analysis. The review was registered a priori in PROSPERO (CRD42022365391).
RESULTS
Twenty studies were included in the analyses. They were all published between 2017 and 2022 and represent the recommendations in 8 countries. Most studies adopted 5, 6, or 10-state Markov models. Some authors took part in multiple studies. Four technologies were evaluated: BST (N = 14), nusinersen (N = 19), risdiplam (N = 5), and OA (N = 9). OA, risdiplam, and nusinersen were considered inefficient compared with the BST. Risdiplam and OA were generally regarded as cost-effective when compared with nusinersen. Because nusinersen is not a cost-effective drug, no recommendation can be derived from this result. Risdiplam and OA were compared in 2 studies that presented opposite results.
CONCLUSIONS
Nusinersen, risdiplam, and OA are being adopted worldwide as a treatment for spinal muscular atrophy. Despite that, the pharmacoeconomic analyses show that the technologies are not cost-effective compared with the BST. The lack of controlled studies for risdiplam and OA hamper any conclusions about their face-to-face comparison.
Topics: Humans; Cost-Benefit Analysis; Muscular Atrophy, Spinal; Oligonucleotides; Aptamers, Nucleotide; Azo Compounds; Pyrimidines
PubMed: 38669792
DOI: 10.1016/j.vhri.2024.02.002 -
Archives of Gerontology and Geriatrics Aug 2024Sarcopenic obesity significantly burdens health and autonomy. Strategies to intervene in or prevent sarcopenic obesity generally focus on losing body fat and building or...
Analysis of behavioral change techniques used in exercise and nutritional interventions targeting adults around retirement age with sarcopenic obesity in a systematic review.
BACKGROUND
Sarcopenic obesity significantly burdens health and autonomy. Strategies to intervene in or prevent sarcopenic obesity generally focus on losing body fat and building or maintaining muscle mass and function. For a lifestyle intervention, it is important to consider psychological aspects such as behavioral change techniques (BCTs) to elicit a long-lasting behavioral change.
PURPOSE
The study was carried out to analyze BCTs used in exercise and nutritional interventions targeting community-dwelling adults around retirement age with sarcopenic obesity.
METHODS
We conducted an analysis of articles cited in an existing systematic review on the effectiveness of exercise and nutritional interventions on physiological outcomes in community-dwelling adults around retirement age with sarcopenic obesity. We identified BCTs used in these studies by applying a standardized taxonomy.
RESULTS
Only nine BCTs were identified. Most BCTs were not used intentionally (82 %), and those used derived from the implementation of lifestyle components, such as exercise classes ("instructions on how to perform a behavior," "demonstration of the behavior," "behavioral practice/rehearsal," and "body changes"). Only two studies used BCTs intentionally to reinforce adherence in their interventions.
CONCLUSIONS
Few studies integrated BCTs in lifestyle interventions for community-dwelling persons around retirement age with sarcopenic obesity. Future studies on interventions to counteract sarcopenic obesity should include well-established BCTs to foster adherence and, therefore, their effectiveness.
Topics: Aged; Female; Humans; Male; Middle Aged; Behavior Therapy; Exercise; Independent Living; Life Style; Obesity; Retirement; Sarcopenia
PubMed: 38653002
DOI: 10.1016/j.archger.2024.105437 -
BMC Geriatrics Apr 2024Sarcopenic obesity emerges as a risk factor for adverse clinical outcomes in non-hospitalized older adults, including physical disabilities, metabolic diseases, and even... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Sarcopenic obesity emerges as a risk factor for adverse clinical outcomes in non-hospitalized older adults, including physical disabilities, metabolic diseases, and even mortality. In this systematic review and meta-analysis, we investigated the overall SO prevalence in non-hospitalized adults aged ≥ 65 years and assessed the sociodemographic, clinicobiological, and lifestyle factors related to SO.
METHODS
We searched the PubMed, Embase, Cochrane Library, and Web of Science databases for studies reporting the prevalence of SO from database inception to October 2023. Two researchers independently screened the literature, evaluated the study quality, and extracted the data. Both fixed- and random-effects models were used in the meta-analysis to estimate the pooled SO prevalence and perform subgroup analyses. Publication and sensitivity bias analyses were performed to test the robustness of the associations.
RESULTS
Among 46 studies eligible for review and a total of 71,757 non-hospitalized older adults, the combined prevalence of SO was 14% (95% CI:11-17%, I = 99.5%, P < 0.01). Subgroup analysis according to lifestyle factors demonstrated that the SO prevalence was 17% (95% CI: 8-29%, I = 99.5%, P < 0.01) in older adults without exercise habits. Regarding clinicobiological factors, older adults with a history of falls (15% [95% CI: 10-22%, I = 82%, P < 0.01]), two or more chronic diseases (19% [95% CI: 10-29%, I = 97%, P < 0.01]), functional impairment (33% [95% CI: 29-37%, I = 0%, P = 0.95]), cognitive impairment (35% [95% CI: 9-65%, I = 83%, P = 0.02]), osteoporosis (20% [95% CI: 8-35%, I = 96%, P < 0.01]), high fasting glucose level (17% [95% CI: 1-49%, I = 98%, P < 0.01]), or the use of antipsychotics (13% [95% CI: 2-28%, I = 0%, P = 0.32]) exhibited a higher SO prevalence.
CONCLUSION
SO prevalence is high among non-hospitalized older adults, especially those with functional and cognitive impairments. Thus, SO is a potential problem for the aging population; implementation of planned interventions in the community is needed to reduce the prevalence and adverse outcomes of SO.
Topics: Humans; Aged; Prevalence; Sarcopenia; Obesity; Risk Factors; Aged, 80 and over
PubMed: 38649825
DOI: 10.1186/s12877-024-04952-z -
Tzu Chi Medical Journal 2024Age-related macular degeneration (AMD) is a chronic and degenerative disease of the retina that leads to irreversible blindness. There is no proven effective treatment...
OBJECTIVES
Age-related macular degeneration (AMD) is a chronic and degenerative disease of the retina that leads to irreversible blindness. There is no proven effective treatment for early AMD and advanced AMD. Mediterranean diet (MD) has been linked to reducing the risk or delaying the progression of AMD. Therefore, in this study, we aim to investigate the potential of MD as a modifiable risk factor for AMD.
MATERIALS AND METHODS
A systematic search was performed in three databases: PubMed, EBSCO host, and Proquest. We search for studies that determine the association of MD in AMD. Then, we pooled the data for meta-analysis.
RESULTS
Eight studies were included in our systematic review. Seven studies were included for meta-analysis. Subjects with medium-high (hazard ratio [HR] 0.82; 95% confidence interval [CI]: 0.75-0.90) adherence to the MD showed a reduced risk of developing AMD. Moreover, medium adherence AMD shows a significant and inverse relationship with the progression to advanced AMD (HR: 0.87; 95% CI: 0.81-0.93). Although it is still inconsistent, the reduction appears stronger for geographic atrophy than for neovascular AMD.
CONCLUSION
Adhering to the MD, particularly at a medium to high level, appears to confer a protective effect against AMD. The sub-analysis demonstrates even that there is a protective effect associated with moderate adherence against advanced AMD. The presence of considerable heterogeneity within the results warrants cautious interpretation. Further research is needed to enhance our understanding.
PubMed: 38645781
DOI: 10.4103/tcmj.tcmj_153_23 -
Journal of Shoulder and Elbow Surgery Apr 2024To identify and quantify the factors associated with the reparability of rotator cuff tears (RCTs). (Review)
Review
PURPOSE
To identify and quantify the factors associated with the reparability of rotator cuff tears (RCTs).
METHODS
PubMed, Scopus, and Web of Science databases were searched for clinical studies published in English focusing on RCT reparability by using the keywords "rotator cuff tear" and "reparability". A meta-analysis was conducted if ≥3 studies examined the same factor and provided enough data to assess RCT reparability. Quality assessment was completed using the QUADAS-2 tool.
RESULTS
Eighteen studies (2700 patients) were enrolled, and 26 factors were included in the meta-analysis. The dichotomous variables associated with irreparability were Patte stage 3 (Odds ratio (OR): 8.0, 95% CI: 4.3-14.9), massive tear versus large tear (OR: 3.1, 95% CI: 1.3-7.2), Goutallier stage for each tendon, and tangent sign (OR: 11.1, 95% CI: 4.3-28.4). The continuous variables associated with irreparability were age (Mean difference (MD): 3.25, 95% CI: 1.4-5.1), mediolateral tear size (MD: 12.3, 95% CI: 5.8-18.9), anteroposterior tear size (MD: 10.4, 95% CI: 5.2-15.6), acromiohumeral distance on X-ray (MD: -2.3, 95% CI: -3.0 to -1.6) and magnetic resonance imaging (MD: -1.8, 95% CI: -2.8 to -0.9), and inferior glenohumeral distance on MRI (MD: 2.2, 95% CI: 1.4-3.0).
CONCLUSION
This study revealed that older age, larger tear size, severe fatty infiltration, muscle atrophy, and advanced superior migration of the humeral head were strongly associated with irreparable RCTs. Conversely, clinical symptoms provided limited information for predicting reparability. Additionally, the tangent sign emerged as a powerful and simple tool for individual prediction, and several quantitative scoring systems also proved useful.
PubMed: 38642872
DOI: 10.1016/j.jse.2024.03.014