Therapeutics and Clinical Risk Management最新文献

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Diffusion Tensor Imaging for Evaluating Postoperative Outcomes of Supratentorial Glioma in the Motor Function Area [Letter]. 弥散张量成像评价幕上胶质瘤术后运动功能区预后[j]。
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-07-10 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S428676
Theo Moraes Teixeira, Luis Filipe de Souza Godoy, Wellingson Silva Paiva
{"title":"Diffusion Tensor Imaging for Evaluating Postoperative Outcomes of Supratentorial Glioma in the Motor Function Area [Letter].","authors":"Theo Moraes Teixeira, Luis Filipe de Souza Godoy, Wellingson Silva Paiva","doi":"10.2147/TCRM.S428676","DOIUrl":"10.2147/TCRM.S428676","url":null,"abstract":"","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"579-580"},"PeriodicalIF":2.3,"publicationDate":"2023-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/38/80/tcrm-19-579.PMC10348315.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9823643","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Emerging Treatment Options for Myelofibrosis: Focus on Anemia. 骨髓纤维化的新兴治疗选择:关注贫血。
IF 2.8 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-06-28 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S386802
Dahniel Sastow, Douglas Tremblay
{"title":"Emerging Treatment Options for Myelofibrosis: Focus on Anemia.","authors":"Dahniel Sastow,&nbsp;Douglas Tremblay","doi":"10.2147/TCRM.S386802","DOIUrl":"10.2147/TCRM.S386802","url":null,"abstract":"<p><p>Myelofibrosis (MF) is a hematologic malignancy characterized by abnormal proliferation of myeloid cells and the release of pro-inflammatory cytokines, leading to progressive bone marrow dysfunction. The introduction of ruxolitinib just over a decade ago marked a significant advancement in MF therapy, with JAK inhibitors now being the first-line treatment for reducing spleen size and managing symptoms. However, early JAK inhibitors (ruxolitinib and fedratinib) are often associated with cytopenias, particularly thrombocytopenia and anemia, which limit their tolerability. To address these complications, pacritinib has been developed and recently approved for patients with thrombocytopenia, while momelotinib is in development for those with anemia. Although JAK inhibitors have significantly improved the quality of life of MF patients, they have not demonstrated the ability to reduce leukemic transformation and their impact on survival is debated. Numerous drugs are currently being developed and investigated in clinical trials, both as standalone therapy and in combination with JAK inhibitors, with promising results enhancing the benefits of JAK inhibitors. In the near future, MF treatment strategies will involve selecting the most suitable JAK inhibitor based on individual patient characteristics and prior therapy. Ongoing and future clinical trials are crucial for advancing the field and expanding therapeutic options for MF patients.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"535-547"},"PeriodicalIF":2.8,"publicationDate":"2023-06-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0e/b4/tcrm-19-535.PMC10315142.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10159492","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Factors Affecting Radial Artery Occlusion After Right Transradial Artery Catheterization for Coronary Intervention and Procedures. 右侧经桡动脉导管冠状动脉介入和手术后桡动脉闭塞的影响因素。
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-06-24 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S403410
Dujuan Xu, Ying Liu, Chao Xu, Xuekui Liu, Ye Chen, Chunguang Feng, Nan Lyu
{"title":"Factors Affecting Radial Artery Occlusion After Right Transradial Artery Catheterization for Coronary Intervention and Procedures.","authors":"Dujuan Xu, Ying Liu, Chao Xu, Xuekui Liu, Ye Chen, Chunguang Feng, Nan Lyu","doi":"10.2147/TCRM.S403410","DOIUrl":"10.2147/TCRM.S403410","url":null,"abstract":"<p><strong>Objective: </strong>To determine the factors influencing proximal radial artery occlusion (PRAO) right radial artery after coronary intervention.</p><p><strong>Methods: </strong>This is a single-center prospective observational study. A total of 460 patients were selected to undergo coronary angiography (CAG) or percutaneous coronary intervention (PCI) via the proximal transradial approach (PTRA) or distal transradial approach (DTRA). The 6F sheath tube were received by all patients. Radial artery ultrasound was performed 1 day before procedure and 1-4 days after procedure. Patients were divided into the PRAO group (42 cases) and the non-PRAO group (418 cases). General clinical data and preoperative radial artery ultrasound indexes of the two groups were compared to analyze related factors leading to PRAO.</p><p><strong>Results: </strong>The total incidence of PRAO was 9.1%, including 3.8% for DTAR and 12.7% for PTRA. The PRAO rate of DTRA was significantly lower than that of PTRA (<i>p</i> < 0.05). Female, low body weight, low body mass index (BMI) and CAG patients were more likely to develop PRAO after procedure (<i>p</i> < 0.05). The internal diameter and cross-sectional area of the distal radial artery and proximal radial artery were smaller in the PRAO group than in the non-PRAO group, and the differences were statistically significant (<i>p</i> < 0.05). Multifactorial model analysis showed that the puncture approach, radial artery diameter and procedure type were predictive factors of PRAO, and the receiver operating characteristic curve showed a good predictive value.</p><p><strong>Conclusion: </strong>A larger radial artery diameter and DTRA may reduce the incidence of PRAO. Preoperative radial artery ultrasound can guide the clinical selection of appropriate arterial sheath and puncture approach.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"525-533"},"PeriodicalIF":2.3,"publicationDate":"2023-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b9/e3/tcrm-19-525.PMC10305768.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9737563","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Innovations in the Treatment of Dystrophic Epidermolysis Bullosa (DEB): Current Landscape and Prospects. 治疗萎缩性表皮松解症(DEB)的创新:当前形势与前景。
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-06-14 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S386923
Ping-Chen Hou, Nathalie Del Agua, Su M Lwin, Chao-Kai Hsu, John A McGrath
{"title":"Innovations in the Treatment of Dystrophic Epidermolysis Bullosa (DEB): Current Landscape and Prospects.","authors":"Ping-Chen Hou, Nathalie Del Agua, Su M Lwin, Chao-Kai Hsu, John A McGrath","doi":"10.2147/TCRM.S386923","DOIUrl":"10.2147/TCRM.S386923","url":null,"abstract":"<p><p>Dystrophic epidermolysis bullosa (DEB) is one of the major types of EB, a rare hereditary group of trauma-induced blistering skin disorders. DEB is caused by inherited pathogenic variants in the <i>COL7A1</i> gene, which encodes type VII collagen, the major component of anchoring fibrils which maintain adhesion between the outer epidermis and underlying dermis. DEB can be subclassified into dominant (DDEB) and recessive (RDEB) forms. Generally, DDEB has a milder phenotype, while RDEB patients often have more extensive blistering, chronic inflammation, skin fibrosis, and a propensity for squamous cell carcinoma development, collectively impacting on daily activities and life expectancy. At present, best practice treatments are mostly supportive, and thus there is a considerable burden of disease with unmet therapeutic need. Over the last 20 years, considerable translational research efforts have focused on either trying to cure DEB by direct correction of the <i>COL7A1</i> gene pathology, or by modifying secondary inflammation to lessen phenotypic severity and improve patient symptoms such as poor wound healing, itch, and pain. In this review, we provide an overview and update on various therapeutic innovations for DEB, including gene therapy, cell-based therapy, protein therapy, and disease-modifying and symptomatic control agents. We outline the progress and challenges for each treatment modality and identify likely prospects for future clinical impact.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"455-473"},"PeriodicalIF":2.3,"publicationDate":"2023-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d2/0a/tcrm-19-455.PMC10277004.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9660429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Clinical Utility of Deucravacitinib for the Management of Moderate to Severe Plaque Psoriasis. Deucravacitinib治疗中重度斑块型银屑病的临床应用
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-05-18 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S388324
Joy Q Jin, Riley K Spencer, Vidhatha Reddy, Tina Bhutani, Wilson Liao
{"title":"Clinical Utility of Deucravacitinib for the Management of Moderate to Severe Plaque Psoriasis.","authors":"Joy Q Jin, Riley K Spencer, Vidhatha Reddy, Tina Bhutani, Wilson Liao","doi":"10.2147/TCRM.S388324","DOIUrl":"10.2147/TCRM.S388324","url":null,"abstract":"<p><strong>Introduction: </strong>Psoriasis is a chronic, immune-mediated skin condition with significant detriments to physical/mental health. While systemic therapies are available for the treatment of moderate-to-severe psoriasis, patients can experience therapeutic failure, loss of efficacy, or medical contraindications that require other therapeutic options.</p><p><strong>Objective: </strong>With the recent approval of deucravacitinib, a first-in-class TYK2 small molecule inhibitor administered orally for psoriasis patients, we reviewed data from randomized controlled trials (RCTs) to synthesize its clinical utility. To our knowledge, this is the first systematic review and meta-analysis of deucravacitinib comparing its clinical efficacy to placebo in psoriasis.</p><p><strong>Methods: </strong>A literature search was conducted in PubMed (MEDLINE), Embase, and the Cochrane Central Register of Controlled Trials to identify RCTs studying deucravacitinib in human patients with moderate-to-severe psoriasis.</p><p><strong>Results: </strong>One placebo-controlled Phase II RCT and two placebo-controlled/active-comparator Phase III RCTs were included for review. Patients (N=1953) treated with deucravacitinib 6 mg daily showed marked improvement in disease severity (Psoriasis Area and Severity Index (PASI), static Physician Global Assessment (sPGA) and quality-of-life outcomes compared to patients administered comparator (apremilast) and placebo. Clinical improvement given deucravacitinib was noted for scalp psoriasis but not fingernail psoriasis. Meta-analysis (deucravacitinib, n=888; placebo, n=466) comparing rates of clearance (sPGA 0/1) demonstrated superior efficacy of deucravacitinib compared to placebo (odds ratio, 12.87; 95% confidence interval, 8.97-18.48; <i>χ</i><sup>2</sup>=4.08, I<sup>2</sup>=51%). Deucravacitinib was well-tolerated, with similar rate of occurrence and type of adverse events reported among patients treated with placebo or apremilast at Week 12-16. No cardiovascular events, serious infections, or lab abnormalities were noted.</p><p><strong>Conclusion: </strong>Deucravacitinib possesses good efficacy, with no report of safety concerns associated with prior JAK inhibitors used for psoriasis. Meta-analysis demonstrated deucravacitinib's superiority compared to placebo, indicating its promising clinical utility. Further studies are needed to observe long-term safety and efficacy, and to compare deucravacitinib to existing treatments.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"413-423"},"PeriodicalIF":2.3,"publicationDate":"2023-05-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/88/58/tcrm-19-413.PMC10202110.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9518474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Effect of Tricalcium Phosphate on Healing of Non-Unions: An Observational Study of over 400 Non-Unions. 磷酸三钙对非溃疡愈合的影响:对 400 多例非溃疡患者的观察研究。
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-05-12 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S409119
Thomas Ferbert, Christina Münch, Sebastian Findeisen, William Pauly, Matthias Miska, Tobias Grossner, Michael C Tanner, Gerhard Schmidmaier, Lars Helbig
{"title":"Effect of Tricalcium Phosphate on Healing of Non-Unions: An Observational Study of over 400 Non-Unions.","authors":"Thomas Ferbert, Christina Münch, Sebastian Findeisen, William Pauly, Matthias Miska, Tobias Grossner, Michael C Tanner, Gerhard Schmidmaier, Lars Helbig","doi":"10.2147/TCRM.S409119","DOIUrl":"10.2147/TCRM.S409119","url":null,"abstract":"<p><strong>Purpose: </strong>A central aspect of the treatment of non-unions is the filling of bone defects. The quantity of available autologous bone for this purpose is limited. Alternatively, or additionally, bone substitutes may be used. The aim of this retrospective, single-center study including 404 non-unions in 393 patients is to investigate the effect of tricalcium phosphate (TCP) on the healing of non-unions. Furthermore, the influence of gender, age, smoking status, comorbidities, type of surgical procedure, presence of infection, and length of treatment was investigated.</p><p><strong>Methods: </strong>We evaluated three groups of patients. Group 1 received TCP + BG, group 2 received BG alone and group 3 received no augmentation. Bone stability was assessed 1 and 2 years after non-union revision surgery through analysis of radiographs using the Lane Sandhu Score. Scores ≥3 were rated as stable Other influencing factors were collected from the electronic medical record.</p><p><strong>Results: </strong>In 224 non-unions, bone defects were filled with autologous bone and TCP (TCP+BG). In 137 non-unions, bone defects were filled with autologous bone (BG), and in 43 non-unions presenting non-relevant defects, neither autologous bone nor TCP were used (NBG). After 2 years, 72.7% of the TCP+BG patients, 90.1% of the BG patients and 84.4% of the NBG patients achieved a consolidation score ≥3. Advanced age, presence of comorbidities and longer treatment period had a significantly negative effect on consolidation 1 year after surgery. Longer treatment periods also showed a negative significant effect after 2 years. It is notable that larger defects, mainly treated with the combination of autologous bone and TCP, showed similar healing rates to that of smaller defects after 2 years.</p><p><strong>Conclusion: </strong>The combination of TCP and autologous bone-grafts shows good results in the reconstruction of complicated bone-defects, but patience is required since the healing period exceeds 1 year in most patients.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"395-404"},"PeriodicalIF":2.3,"publicationDate":"2023-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d5/1f/tcrm-19-395.PMC10187654.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9490166","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Discontinuation of Antidepressants in Older Adults: A Literature Review. 老年人停用抗抑郁药:文献综述。
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-03-28 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S395449
Ahmed Romdhani, Stephanie Lehmann, Joël Schlatter
{"title":"Discontinuation of Antidepressants in Older Adults: A Literature Review.","authors":"Ahmed Romdhani, Stephanie Lehmann, Joël Schlatter","doi":"10.2147/TCRM.S395449","DOIUrl":"10.2147/TCRM.S395449","url":null,"abstract":"<p><p>Polypharmacy increases the risk of unbearable side effects, drug-drug interactions, and hospitalizations in geriatric patients. The iatrogenic risk of inadequate management of antidepressants is very important in this population. Therefore, primary care physicians and geriatricians have the responsibility of the optimization of antidepressants prescriptions. Our work is a literature review of the European and the international guidelines regarding the management of antidepressants. We reviewed the PubMed database and Google scholar for articles and reviews from 2015. We also screened relevant articles for more references and searched the web for available European guidelines relevant to our topic. We divided our findings into four main inquiries that are Indication, effectiveness, tolerability, and iatrogenic risks. Poor or absence of effectiveness should lead to a readjustment of the treatment plan. In case of unbearable side effects, antidepressants should be stopped, and alternative non-pharmacological therapies should be proposed. Doctors should look out for drug-drug interaction risks in this population and constantly adjust the prescription. Prescription of antidepressants is not always evidence based which leads to heavy iatrogenic consequences. We suggest a simple 4-questions-algorithm that aims to remind doctors of the basics of good practice and helps in the process of deprescribing an antidepressant in older adults.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"291-299"},"PeriodicalIF":2.3,"publicationDate":"2023-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/1e/2a/tcrm-19-291.PMC10066696.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9248514","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Predictive Value of Preoperative Diffusion Tensor Imaging for Evaluating Postoperative Outcomes of Supratentorial Glioma in the Motor Function Area. 术前弥散张量成像对评估运动功能区脑室上胶质瘤术后疗效的预测价值
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-03-14 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S402622
Nguyen Duy Hung, Nguyen Duy Linh, Nguyen Ha Vi, Nguyen Thi Van Anh, Nguyen Dinh Hieu, Duong Dai Ha, Nguyen Minh Duc
{"title":"Predictive Value of Preoperative Diffusion Tensor Imaging for Evaluating Postoperative Outcomes of Supratentorial Glioma in the Motor Function Area.","authors":"Nguyen Duy Hung, Nguyen Duy Linh, Nguyen Ha Vi, Nguyen Thi Van Anh, Nguyen Dinh Hieu, Duong Dai Ha, Nguyen Minh Duc","doi":"10.2147/TCRM.S402622","DOIUrl":"10.2147/TCRM.S402622","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to assess the predictive value of preoperative diffusion tensor imaging (DTI) data for surgical outcomes of patients with supratentorial glioma in the motor function area.</p><p><strong>Patients and methods: </strong>This is a retrospective study of 43 patients receiving navigation-guided surgery for histopathologically demonstrated supratentorial glioma in the motor function area. All patients underwent preoperative 3 Tesla magnetic resonance imaging examinations with conventional and DTI sequences. Data on preoperative imaging and pre- and postoperative clinical characteristics of patients were retrospectively collected. Univariate and multivariate linear regressions were applied to analyze the relationships between preoperative parameters and pre- and postoperative muscle strength and the Karnofsky Performance Status (KPS) score.</p><p><strong>Results: </strong>Fourteen patients had low-grade gliomas and 29 had high-grade gliomas. Although the corticospinal tract (CST) score did not differ significantly between tumor grades, edema and deviation were common in low-grade gliomas (64.3%), while destroyed and infiltrated lesions were common in high-grade gliomas (58.6%). Muscle strength improved after surgery in the deviated tract group (40%) more than in the infiltrated tract group (33.3%). Two independent indices, preoperative muscle strength (<i>p</i> = 0.000) and glioma-to-CST distance (<i>p</i> = 0.001), were linearly related to postoperative muscle strength. The preoperative KPS score was the only indicator that affected the postoperative KPS score (<i>p</i> = 0.000).</p><p><strong>Conclusion: </strong>DTI should be considered in surgical management of supratentorial gliomas in the motor function area to determine the appropriate surgical strategy and predict the nature of the tumor and postoperative motor function.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"269-278"},"PeriodicalIF":2.3,"publicationDate":"2023-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/3d/86/tcrm-19-269.PMC10024488.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9156229","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Congenital Athymia: Unmet Needs and Practical Guidance. 先天性腹胀:未满足的需求和实用指导。
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-03-13 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S379673
Evey Howley, E Graham Davies, Alexandra Y Kreins
{"title":"Congenital Athymia: Unmet Needs and Practical Guidance.","authors":"Evey Howley, E Graham Davies, Alexandra Y Kreins","doi":"10.2147/TCRM.S379673","DOIUrl":"10.2147/TCRM.S379673","url":null,"abstract":"<p><p>Inborn errors of thymic stromal cell development and function which are associated with congenital athymia result in life-threatening immunodeficiency with susceptibility to infections and autoimmunity. Athymic patients can be treated by thymus transplantation using cultured donor thymus tissue. Outcomes in patients treated at Duke University Medical Center and Great Ormond Street Hospital (GOSH) over the past three decades have shown that sufficient T-cell immunity can be recovered to clear and prevent infections, but post-treatment autoimmune manifestations are relatively common. Whilst thymus transplantation offers the chance of long-term survival, significant challenges remain to optimise the outcomes for the patients. In this review, we will discuss unmet needs and offer practical guidance based on the experience of the European Thymus Transplantation programme at GOSH. Newborn screening (NBS) for severe combined immunodeficiency (SCID) and routine use of next-generation sequencing (NGS) platforms have improved early recognition of congenital athymia and increasing numbers of patients are being referred for thymus transplantation. Nevertheless, there remain delays in diagnosis, in particular when the cause is genetically undefined, and treatment accessibility needs to be improved. The majority of athymic patients have syndromic features with acute and chronic complex health issues, requiring life-long multidisciplinary and multicentre collaboration to optimise their medical and social care. Comprehensive follow up after thymus transplantation including monitoring of immunological results, management of co-morbidities and patient and family quality-of-life experience, is vital to understanding long-term outcomes for this rare cohort of patients. Alongside translational research into improving strategies for thymus replacement therapy, patient-focused clinical research will facilitate the design of strategies to improve the overall care for athymic patients.</p>","PeriodicalId":48769,"journal":{"name":"Therapeutics and Clinical Risk Management","volume":"19 ","pages":"239-254"},"PeriodicalIF":2.3,"publicationDate":"2023-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/15/e4/tcrm-19-239.PMC10022451.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9143060","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
LncRNA AFAP1-AS1 Induces Gefitinib Resistance of Lung Adenocarcinoma Through the miR-653-5p/AGR2 Axis. LncRNA AFAP1-AS1 通过 miR-653-5p/AGR2 轴诱导吉非替尼对肺腺癌的耐药性
IF 2.3 3区 医学
Therapeutics and Clinical Risk Management Pub Date : 2023-01-05 eCollection Date: 2023-01-01 DOI: 10.2147/TCRM.S374162
Tao Zuo, Ping Jiang, Jun Fu, Yongjian Zhang
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