Clinical therapeutics最新文献

{"title":"Evaluating the clinical and economic impact of ceramide-infused skin barriers in patients with Intestinal and urinary stomas: A systematic review and meta-analysis.","authors":"Rosario Caruso, Silvia Belloni, Beniamino Schiavone, Gianluca Conte, Cristina Di Pasquale, Arianna Magon, Cristina Arrigoni, Giuseppe Candilio, Francesco Stanzione, Alessandro Stievano, Gennaro Rocco, Maddalena De Maria","doi":"10.1016/j.clinthera.2025.02.001","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.02.001","url":null,"abstract":"<p><strong>Purpose: </strong>Ceramide-infused skin barriers (CIBs) applied to stoma care hold potential benefits, which are thus far not summarized. This study aims to summarize the literature on CIBs in patients with intestinal and urinary stomas and to quantitatively compare the clinical, economic, and well-being outcomes of CIBs against the standard of care (SOC) in these patients.</p><p><strong>Methods: </strong>Systematic review and random-effect meta-analysis following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement, including meta-regression analyses to explore sources of heterogeneity. PubMed, CINAHL, Scopus, Web of Science, Embase, Google Scholar, and clinicaltrials.gov were searched for studies published up to November 2024. Studies involving patients of any age with intestinal or urinary stomas treated with CIBs or SOC. Outcomes included peristomal skin complications (PSCs), cost-effectiveness, and quality-adjusted life days (QALDs).</p><p><strong>Findings: </strong>CIBs increased the odds of preventing PSCs by 77% compared to SOC (OR = 1.77, 95% CI: 1.40, 2.23). Cost savings averaged -140,000 USD per patient (95% CI: -142,000 USD, -139,000 USD), although cost-effectiveness varied significantly (I² = 100%, P < 0.001). Meta-regression identified gross domestic product (GDP) per capita (β = -7.31, P = 0.010) and healthcare expenditure per capita (β = -169.33, P < 0.001) as key contributors to cost variability. CIBs also improved QALDs (MD = 0.35, 95% CI: 0.33, 0.37), enhancing patient quality of life.</p><p><strong>Implications: </strong>CIBs reduce PSCs, generate cost savings, and improve QALDs, demonstrating potential for widespread clinical adoption. However, economic benefits vary across healthcare systems, warranting further research into their long-term impact and country-specific cost-effectiveness.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143584887","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Effect of Ulinastatin on Sepsis Outcomes: An Umbrella Review of Meta-Analysis.","authors":"Sheng Cao, Ping Han","doi":"10.1016/j.clinthera.2025.01.013","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.013","url":null,"abstract":"<p><strong>Objectives: </strong>Sepsis, a multifaceted disorder, emerges from dysregulated host response to infection, culminating in organ dysfunction and heightened risk of mortality. Present umbrella systematic review was conducted to impart accurate data regarding the effect of urinary trypsin inhibitor (UTI) alone, UTI in combination with thymosin α1, and UTI in combination with Xuebijing on sepsis and inflammation, 28-day mortality rate survival day, time of mechanical ventilation, length of intensive care unit stay, and acute physiology and chronic health evaluation (APACHE II) score.</p><p><strong>Methods: </strong>Relevant studies were searched in international databases, including PubMed, Scopus, EMBASE, Web of Science, and Cochrane Central Library up to March 2024. Our study included meta-analyses that evaluated the effects of ulinastatin (UTI) alone, or in combination with thymosin α1 or Xuebijing, on sepsis and inflammatory biomarkers.</p><p><strong>Results: </strong>Nine studies were deemed relevant and subsequently included in the study. The age of the study's participants was between 42.3 and 55.7 years. In total, the dose varied between 166 and 570 KIU/12 h. Moreover, the duration varied between 3 and 8.5 days.</p><p><strong>Conclusion: </strong>A comprehensive assessment of ulinastatin's overall efficacy necessitates a careful consideration of the combined effects of ulinastatin with other interventions. Future research is warranted to disentangle the specific contributions of ulinastatin in combination therapies and to enhance our understanding of its independent effects in clinical settings.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143584910","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Randomized Controlled Trial Assessing the Efficacy and Safety of a Liposomal Carrier for Low-Dose Dual Antiplatelet Therapy (Clopidogrel and Aspirin) in Coronary Heart Disease Patients.","authors":"Lu Yang, Jiaqi Mei, Fang Qiao, Shiyao Chen, Congcong Xu","doi":"10.1016/j.clinthera.2025.01.018","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.018","url":null,"abstract":"<p><strong>Background: </strong>Coronary heart disease (CHD) is a leading cause of global mortality, and antiplatelet drugs are crucial in its treatment. Traditional therapy, however, often faces issues like inconsistent efficacy, frequent dosing, and high complication rates.</p><p><strong>Purpose: </strong>This study aimed to develop a liposomal carrier for low-dose dual antiplatelet drugs (clopidogrel and aspirin) using nanotechnology and evaluate its efficacy and safety in CHD patients.</p><p><strong>Methods: </strong>The study first prepared drug carriers for clopidogrel and aspirin using a liposomal approach, and the characteristics and in vitro drug release properties of these carriers were evaluated using various techniques. Subsequently, a randomized controlled trial was conducted with 270 patients diagnosed with CHD, who were divided into the control group (receiving 75 mg of clopidogrel and 100 mg of aspirin daily) and the treatment group (receiving the same regimen as the control group, with the addition of a nanoparticle drug delivery system), with 135 patients in each group. The efficacy and safety of the two interventions were then evaluated.</p><p><strong>Findings: </strong>The liposomal carriers demonstrated high drug encapsulation efficiency and sustained release. Clinical trials showed superior efficacy and fewer complications with the nanoparticle drug delivery system compared to traditional antiplatelet therapy.</p><p><strong>Implications: </strong>The nanoparticle drug delivery system for low-dose dual antiplatelet drugs shows promise as a novel therapeutic strategy for CHD patients. Further validation through larger sample sizes and long-term follow-up studies is necessary.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143566204","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic Value of Enhanced Monofocal Intraocular Lenses for Cataract Surgery in Italy.","authors":"Carla Rognoni, Ilaria Giabbani, Marco Balestrieri, Giacomo Costa, Eleonora Favuzza, Rosa Giglio, Rita Mencucci, Giovanni Staurenghi, Leonardo Taroni, Daniele Tognetto, Rosanna Tarricone","doi":"10.1016/j.clinthera.2025.02.002","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.02.002","url":null,"abstract":"<p><strong>Aim: </strong>Cataract is a prevalent health condition, primarily caused by aging, affecting approximately 95 million individuals worldwide. The only effective treatment currently involves surgically removing and replacing the crystalline lens with an artificial intraocular lens (IOL). Various IOLs are available, each with distinct characteristics, costs, and outcomes. This study aimed to assess the value of an enhanced monofocal IOL for cataract surgery, which has been shown to improve intermediate vision and reduce the need for spectacles during intermediate tasks, compared to a conventional monofocal IOL (standard of care), through a cost-utility analysis from both the National Healthcare Service (NHS) and societal perspectives in Italy.</p><p><strong>Methods: </strong>A cost-utility model was developed incorporating both healthcare and nonhealthcare costs, as well as productivity losses, using data from a socio-economic questionnaire administered at three clinical centers in Italy. The questionnaire included the EuroQol 5D-5L to assess quality of life. National Healthcare Service costs were based on reimbursement tariffs.</p><p><strong>Results: </strong>Over a 10-year horizon, estimated costs were 15,723 € (16,643 USD) for the standard IOL group and 11,190 € (11,845 USD) for the enhanced monofocal IOL group from the societal perspective. Since no significant differences in patients' quality of life were observed between the two groups, the innovative IOL may be considered a cost-saving option compared to standard monofocal IOL. From the NHS perspective, only the intervention for lens implantation was considered, resulting in costs of 940 € (994.99 USD) and 900 € (952.65 USD) for enhanced monofocal IOL and standard IOL, respectively. In this perspective, enhanced monofocal IOL was dominated (more costly with the same QALYs) by standard IOL.</p><p><strong>Conclusions: </strong>This study fills a literature gap by evaluating the cost-utility of enhanced monofocal IOLs for cataract surgery compared to standard IOLs. While enhanced monofocal IOL is dominated from the NHS perspective due to slightly higher direct healthcare costs, it is cost-saving from a societal perspective by reducing the overall economic burden with comparable patients' quality of life. The broader benefits, including reduced reliance on corrective measures, visits and exams, formal and informal assistance, emphasize its societal value. This highlights the need for a holistic healthcare approach that balances long-term societal savings with short-term healthcare costs.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143566203","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of SOX Plus Bevacizumab Versus SOX Plus Cetuximab for First-Line Treatment of KRAS Wild-Type Metastatic Colorectal Cancer in Japan.","authors":"Takashi Morimoto, Kaori Fujito, Rei Goto","doi":"10.1016/j.clinthera.2025.01.019","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.019","url":null,"abstract":"<p><strong>Purpose: </strong>In this study, we aimed to evaluate the cost-effectiveness of S-1 and oxaliplatin (SOX) plus bevacizumab (Bmab group) compared with SOX plus cetuximab (Cmab group) as a first-line treatment for patients with Kirsten rat sarcoma virus (KRAS) wild-type metastatic colorectal cancer (mCRC) in Japan from the perspective of healthcare payers.</p><p><strong>Methods: </strong>A partitioned survival model was developed using data from the randomized phase II Osaka Multicenter Study Group on Colorectal Cancer-1107 study, which included overall survival, progression-free survival, and treatment regimens for the Bmab and Cmab groups. Treatment costs were estimated from the Japanese medical claims database and the National Health Insurance drug price list. The utilities were derived from the literature. Outcomes were reported as incremental cost, incremental quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). The willingness-to-pay (WTP) threshold was set at 7.5 million JPY per QALY. The time horizon of the model was set to 20 years. Sensitivity analyses were conducted to assess the uncertainty of the model for various parameters.</p><p><strong>Findings: </strong>Compared with the Cmab group, the Bmab group had an incremental cost of 911,373 JPY (6,528 USD), an incremental effectiveness of 0.79 QALY, and an ICER of 1146,745 JPY (8,215 USD) per QALY. One-way sensitivity analysis showed that the cost of progressive disease treatment in the Bmab group had the greatest impact on the ICER. According to the probabilistic sensitivity analysis, the Bmab group had a 94.9% probability of being cost-effective compared with the Cmab group.</p><p><strong>Implications: </strong>Considering a WTP threshold of 7.5 million JPY (approximately 53,700 USD) per QALY, Bmab might be a cost-effective treatment option for patients with KRAS wild-type mCRC in Japan. Further studies on economic evaluations based on personalized drugs and patient selection based on clinical and genetic information are warranted.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143556186","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Febuxostat, a Urate-Lowering Drug Bridging From Adults to Pediatrics; A Brief Report.","authors":"Toktam Faghihi, Farahnak Assadi","doi":"10.1016/j.clinthera.2025.01.017","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.017","url":null,"abstract":"<p><strong>Purpose: </strong>Febuxostat, a nonpurine selective inhibitor of xanthine oxidase has elucidated an effective urate-lowering in adults. However, data on febuxostat utility in children is limited. The present study summarizes the current knowledge on the efficacy and safety of febuxostat in children with hyperuricemia.</p><p><strong>Methods: </strong>We searched PubMed, Google Scholar, Embase, and Web of Science from inception to September 2024 for studies assessing febuxostat or comparing febuxostat with allopurinol in children and adolescents with hyperuricemia with diverse etiologies. Randomized controlled trials, prospective observational, and systematic reviews, meta-analyses, and retrospective studies were included.</p><p><strong>Findings: </strong>Search results illuminated three studies assessing febuxostat in children with hyperuricemia of different etiologies; including tumor lysis syndrome (TLS), asymptomatic hyperuricemia, and gout. An open-label observational study and one-retrospective study assessed febuxostat efficacy for asymptomatic hyperuricemia and gout, respectively. There was one retrospective study that compared febuxostat to allopurinol for TLS prevention.</p><p><strong>Implications: </strong>Febuxostat is a promising medication that is effective in attaining the desired outcomes in children with hyperuricemia. However, existing evidence does not permit any conclusion regarding the comparative efficacy and safety of febuxostat with allopurinol in children. Future randomized clinical trials evaluating its effectiveness and safety are needed.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536693","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Vagus Nerve Stimulation for the Treatment of Drug-resistant Epilepsy on Patterns of Use and Cost of Healthcare Services and Pharmacotherapy Among Medicare Enrollees: Findings From Analyses of Healthcare Claims From the Centers of Medicare and Medicaid Services.","authors":"Kathryn Evans, Qian Li, Yuliya Halchenko, Lu Zhang, Vanessa Danielson, Reginald Lassagne, Bronwyn Do Rego, Ariel Berger","doi":"10.1016/j.clinthera.2025.01.015","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.015","url":null,"abstract":"<p><strong>Purpose: </strong>To examine the expected impact of vagus nerve stimulation (VNS) on patterns of utilization and cost of healthcare services and prescription pharmacotherapies among Medicare enrollees with drug-resistant epilepsy (DRE) versus continued use of antiseizure medications (ASMs) alone.</p><p><strong>Methods: </strong>This was a retrospective, observational, cohort study that used healthcare claims data from the US Centers for Medicare and Medicaid Services. All Medicare enrollees who underwent VNS implantation between January 1, 2011 and December 31, 2020 were selected. Individuals without at least 24 months of continuous enrollment before implantation (index date) and at least 1 month of enrollment immediately thereafter were excluded. Patients without a diagnosis of epilepsy on the index date, and those without ASM claims during the 1-year period before that date, were also excluded. Observed patterns of utilization and cost of healthcare services and pharmacotherapies during the 2-year period prior to VNS were used to develop regression models to predict these outcomes during the 2-year period following the index date. Predicted monthly outcomes from these models during each month of the 24-month follow-up period were compared with corresponding outcomes observed in the database, with differences (observed minus expected) attributed to VNS implantation.</p><p><strong>Findings: </strong>A total of 16,223 Medicare enrollees had a procedure code for VNS between January 1, 2011, and December 31, 2020, of whom 19.4% (n = 3155) met all other selection criteria. Expected composite rates of hospitalizations and emergency department (ED) visits were higher than observed for all-cause (38.95 events per 100 person-months [PMs] vs 23.15 per 100 PMs) and epilepsy-related (33.46 per 100 PMs vs 15.97 per 100 PMs) events (P < 0.001 for both comparisons). Following the index month, mean monthly observed all-cause costs were $1286 lower than expected; epilepsy-related costs were $1351 lower. Differences between predicted and observed all-cause costs (including costs related to implantation) did not differ significantly by month 20, indicating an expectation that VNS \"breaks even\" within 2 years of implantation.</p><p><strong>Implications: </strong>VNS implantation was associated with 41% and 52% reductions in all-cause and epilepsy-related hospitalizations and ED visits, respectively (both vs expected), for Medicare patients with DRE, and its implantation may be cost-neutral within 2 years of the procedure. These results are similar in direction and magnitude to those observed in a previous study of commercially insured patients with DRE. Additional research is needed to better understand the impacts of neuromodulator implantation on other important outcomes, such as health-related quality of life.</p>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143514756","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Individual, Social and Environmental Factors Influencing Medication-Taking Among Adults of Vietnamese Heritage With Type 2 Diabetes Living in Australia: A Qualitative Study.","authors":"Olumuyiwa Omonaiye, Elizabeth Holmes-Truscott, Bodil Rasmussen, Peter S Hamblin, Kevin Mc Namara, Jane Tran, Cheryl Steele, Jerry Lai, Elizabeth Manias","doi":"10.1016/j.clinthera.2025.01.012","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.012","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Purpose: &lt;/strong&gt;To explore factors influencing diabetes medication-taking among adults of Vietnamese heritage with type 2 diabetes mellitus (T2DM) residing in Australia. Barriers to and enablers of optimal medication use, as perceived by those with diabetes and health professionals working with this community, were explored via the Theoretical Domains Framework (TDF).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This qualitative study was conducted between November 2021 - March 2023 with input from an advisory group consisting of 4 individuals of Vietnamese heritage (a person living with T2DM, a credentialed diabetes care and education specialist, a General Practitioner, and Nephrologist). Data were collected using semistructured interviews with people with T2DM (adults, living in Australia, Vietnamese country of birth and/or language spoken at home) and focus group discussions with health professionals involved in the care of people with T2DM from Vietnamese background. Recruitment of participants was from a national diabetes registry and/or a tertiary hospital. The 14 domains of the TDF informed the development of the study aim, guided data collection, and thematic analysis. The TDF is a comprehensive framework that can be used to identify barriers and facilitators that influence health behaviors.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Twenty-three interviews were conducted with adults with T2DM (n = 14 women; median [IQR] age = 60 [16] years; n = 15 insulin-treated; all Vietnamese born, with n = 15 reporting Vietnamese as primary language). One focus group was undertaken with each group of health professionals (n = 7 doctors - 5 endocrinologists and 2 advanced endocrinology physician trainees, n = 6 credentialed diabetes care and education specialists, and n=3 pharmacists). A wide range of themes about the barriers and enablers [determinants] of medication taking were generated and mapped on 13 of 14 Theoretical Domains Framework domains, only excluding the domain of ``goals.'' The most important (determined through frequency and richness) domains that influenced medication-taking were: Environmental Context and Resources-access to subsidized medications is facilitated via the Australian Pharmaceutical Benefits Scheme, but high costs remained a significant barrier for many. Emotion-participants reported anxiety about diabetes complications as a motivator for medication-taking, while fears about long-term side effects created barriers. Social Influences-family support was an enabler of medication-taking. However, lack of support and pressure to use alternative treatments posed barriers for some participants. Beliefs About Consequences- belief in the negative outcomes of missed doses motivated medication-taking, while a lack of immediate side effects from missed doses reinforced perceptions that skipping medication was harmless. Memory, attention, and decision making-participants prioritized certain medications, sometimes neglecting others they viewed","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476422","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tacrolimus-Induced Fever in a Lung Transplant Recipient: A Case-Report.","authors":"Robin Thirionet, Charline Leclercq, Michel Dumonceaux, Thomas Planté-Bordeneuve, François M Carlier","doi":"10.1016/j.clinthera.2025.01.016","DOIUrl":"https://doi.org/10.1016/j.clinthera.2025.01.016","url":null,"abstract":"","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143472392","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effects of Thalidomide on Metabolism and Lifespan of Red Blood Cell in Patients With β-Thalassemia Major: A Post Hoc Analysis of a Randomized Controlled Trial","authors":"Huanju Yang ,&nbsp;Sichong Han ,&nbsp;Jianquan Xu ,&nbsp;Sheng He ,&nbsp;Qiyang Lu ,&nbsp;Tianying Luo ,&nbsp;Shuying Chen ,&nbsp;Lujie Dang ,&nbsp;Guizhen Wang ,&nbsp;Jinyan Li ,&nbsp;Minjie Huang ,&nbsp;Yangdong Liao ,&nbsp;Yanfang He ,&nbsp;Ning Cai ,&nbsp;Lan Huang ,&nbsp;Meiguang Zhou ,&nbsp;Yongquan Mo ,&nbsp;Weijian Zhu ,&nbsp;Zhengwei Wu MD ,&nbsp;Guangbiao Zhou PhD ,&nbsp;Jiangming Chen MD","doi":"10.1016/j.clinthera.2025.01.008","DOIUrl":"10.1016/j.clinthera.2025.01.008","url":null,"abstract":"<div><h3>Purpose</h3><div>Recent studies have shown the thalidomide's therapeutic potential in treatment of patients with β-thalassemia major. However, the effect of thalidomide on metabolism and lifespan of red blood cells (RBCs) is rarely reported.</div></div><div><h3>Methods</h3><div>This study was a post hoc analysis of a randomized controlled trial (Chinese Clinical Trial Registry, ChiCTR1800015702). One hundred patients with β-thalassemia major were randomly assigned 1:1 to treatment with a placebo or thalidomide. The primary outcomes were the differences in RBC lifespan, reticulocyte count, and peripheral nucleated RBC count of patients after treatment of 12 weeks. Other indicators of hemolytic reaction were also analyzed.</div></div><div><h3>Findings</h3><div>Compared with the placebo group after treatment of 12 weeks, the thalidomide group showed a longer RBC lifespan (16.29 ± 6.42 vs 12.90 ± 4.98 days; <em>P</em> = 0.004), smaller mean corpuscular volume (68.34 ± 7.79 vs 78.01 ± 6.33 fl; <em>P</em> &lt; 0.001), smaller mean corpuscular hemoglobin (21.62 ± 2.85 vs 24.68 ± 2.69 pg; <em>P</em> &lt; 0.001), and lower lactate dehydrogenase (190.00 [148.00 - 305.00] vs 251.00 [199.20 - 327.80]; <em>P</em> = 0.014). Meanwhile, thalidomide significantly increased the RBC lifespan at 24 weeks (21.24 ± 8.30 days; <em>P</em> &lt; 0.001) and 48 weeks (23.21 ± 8.42 days; <em>P</em> &lt; 0.001) when compared with baseline (12.8 ± 6.0 days).</div></div><div><h3>Implications</h3><div>Thalidomide increases the RBC lifespan and reduces hemolytic reactions in patients with β-thalassemia major. Chinese Clinical Trial Registry identifier: ChiCTR1800015702.</div></div>","PeriodicalId":10699,"journal":{"name":"Clinical therapeutics","volume":"47 4","pages":"Pages 252-260"},"PeriodicalIF":3.2,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143457179","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}