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Cocaine-associated non-occlusive ischemic colitis mimicking appendicitis in a young woman: a case report. 年轻女性可卡因相关性非闭塞性缺血性结肠炎模拟阑尾炎1例报告。
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-10-10 DOI: 10.1080/20565623.2025.2570011
Haythem Elaskri, Sadri Ben Abid, Alaa Oueslati, Sondes Bizid, Sonia Ben Ali, Omar Beskri, Arine Aouadi, Issam Msakni, Hatem Ben Abdallah, Iheb Labbene

Cocaine can trigger severe splanchnic vasoconstriction and non-occlusive mesenteric ischemia, occasionally mimicking common surgical emergencies. A previously healthy 27-year-old woman presented with 5 days of worsening right lower quadrant pain, nausea, and fever. CT suggested appendicitis. Laparoscopy revealed a normal appendix but ischemic ileum and cecal gangrene, requiring conversion to laparotomy and ileocecal resection with stoma. Histopathology showed transmural ischemic necrosis without vascular occlusion. Postoperatively, the patient disclosed recent cocaine use. Cocaine-related ischemia often affects young adults without vascular risk factors, may be radiologically subtle, and carries higher mortality when diagnosis is delayed. In young adults with unexplained acute abdomen, clinicians should consider cocaine-associated ischemia and obtain toxicology screening when the etiology is unclear, enabling timely management and improved outcomes.

可卡因可以引发严重的内脏血管收缩和非闭塞性肠系膜缺血,偶尔会模仿常见的外科急诊。先前健康的27岁女性表现为5天恶化的右下腹疼痛,恶心和发烧。CT提示阑尾炎。腹腔镜检查显示阑尾正常,但回肠缺血性和盲肠坏疽,需要转开腹手术和回盲切除并造口。组织病理学表现为无血管闭塞的全壁缺血性坏死。术后,患者透露近期使用可卡因。可卡因相关性缺血通常影响没有血管危险因素的年轻人,可能在放射学上很微妙,如果诊断延迟,死亡率会更高。对于患有不明原因急腹症的年轻人,当病因不明时,临床医生应考虑可卡因相关性缺血,并进行毒理学筛查,以便及时处理并改善预后。
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引用次数: 0
Development of a reverse-phase HPLC method for the simultaneous determination of curcumin and dexamethasone in polymeric micelles. 建立反相高效液相色谱法同时测定聚合物胶束中姜黄素和地塞米松的含量。
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-10-26 DOI: 10.1080/20565623.2025.2577618
Dana Haj-Ali, Hanan Azzam, Khaled Aiedeh, Walhan Alshaer

Background: A rapid, simple, accurate, and robust reverse-phase high-performance liquid chromatography (RP-HPLC) method was developed to quantify curcumin and dexamethasone in polymeric micelle nanoparticle formulations simultaneously.

Methods: The optimized chromatographic conditions involved a Universal HS C18 column, isocratic elution with a methanol: acidic water (pH 3.5, 80:20, v/v) mobile phase, and detection wavelengths of 425 nm for curcumin and 254 nm for dexamethasone. The developed method was subsequently validated according to ICH guidelines, and demonstrated excellent linearity (R2 > 0.999), precision (RSD% < 2%), and accuracy (mean recovery was 98.7% for curcumin and 101.7% for dexamethasone).

Results: The limits of detection (LOD) were 0.0035 mg/mL for curcumin and 0.0029 mg/mL for dexamethasone, while limits of quantification (LOQ) were 0.0106 mg/mL for curcumin and 0.0088 mg/mL for dexamethasone, respectively. The method was applied to evaluate the encapsulation efficiency (EE%) of curcumin and dexamethasone into polymeric micelle nanoparticles formulated using Soluplus® and DOPE in a 1:10 molar ratio. EE% values were 78.84 ± 0.05% for curcumin and 54.33 ± 0.05% for dexamethasone.

Conclusions: the current developed method indicates suitability for the simultaneous determination of curcumin and dexamethasone, thereby facilitating the quality control and optimization of such advanced drug delivery systems.

背景:建立了一种快速、简便、准确、可靠的反相高效液相色谱(RP-HPLC)方法,用于同时定量聚合物胶束纳米颗粒制剂中的姜黄素和地塞米松。方法:采用通用HS C18色谱柱,甲醇:酸性水(pH 3.5, 80:20, v/v)流动相等密度洗脱,姜黄素检测波长425 nm,地塞米松检测波长254 nm。结果表明,该方法具有良好的线性关系(R2 = 0.999)、精密度(RSD% < 2%)和准确度(姜黄素的平均回收率为98.7%,地塞米松的平均回收率为101.7%)。结果:姜黄素的检出限为0.0035 mg/mL,地塞米松的检出限为0.0029 mg/mL,定量限为0.0106 mg/mL,地塞米松的定量限为0.0088 mg/mL。用该方法评价了姜黄素和地塞米松以1:10的摩尔比与Soluplus®和DOPE配制的聚合物胶束纳米颗粒的包封率(EE%)。姜黄素和地塞米松的EE%分别为78.84±0.05%和54.33±0.05%。结论:本方法适用于姜黄素和地塞米松的同时测定,为此类先进给药系统的质量控制和优化提供了便利。
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引用次数: 0
Clinical practice perspectives on adipose-derived stem cells and platelet-rich plasma for female infertility treatments. 脂肪源性干细胞和富血小板血浆治疗女性不孕症的临床实践观点。
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-10-29 DOI: 10.1080/20565623.2025.2580233
Zaher Merhi

Infertility poses a significant global health burden, especially in cases where diminished ovarian reserve and endometrial injury limit the success of standard assisted reproductive technology treatments. Platelet-Rich Plasma (PRP) have gained attention as novel regenerative tool and adipose-derived stem cells (ADSCs) has recently gained lots of interest owing to their easy availability, multipotent characteristics, and paracrine activity. The combination of PRP and ADSCs acts better than each one individually and acts synergistically to promote tissue regeneration. This review addresses studies on the application of PRP and ADSCs in reproductive medicine, specifically targeting ovaries and uterus. In ovaries, ADSCs and PRP demonstrated potential for functional recovery in premature ovarian insufficiency, early menopause, and chemotherapy-induced ovarian damage, showing menstrual restoration, hormonal normalization, and stimulation of follicle growth. ADSC-derived exosomes and conditioned medium promoted oocyte maturation, lowered oxidative stress, improved blastocyst development, and increased embryo survival. Additionally, findings indicate that intrauterine delivery of ADSCs and PRP enhances endometrial thickness, angiogenesis, and receptivity, with reports of improved implantation and pregnancy outcomes in women with thin endometrial lining or Asherman's syndrome. Collectively, these results underscore the regenerative promise of ADSCs and PRP in overcoming various infertility causes.

不孕症是一个重大的全球健康负担,特别是在卵巢储备减少和子宫内膜损伤限制了标准辅助生殖技术治疗成功的情况下。富血小板血浆(PRP)作为一种新的再生工具而受到关注,而脂肪源性干细胞(ADSCs)由于其易于获得、多能性和旁分泌活性而受到广泛关注。PRP与ADSCs联合使用对组织再生的促进作用优于单独使用,并具有协同作用。本文综述了PRP和ADSCs在生殖医学中的应用研究,特别是针对卵巢和子宫。在卵巢中,ADSCs和PRP在卵巢功能不全、早期绝经和化疗引起的卵巢损伤中显示出功能恢复的潜力,表现出月经恢复、激素正常化和刺激卵泡生长。adsc衍生的外泌体和条件培养基促进卵母细胞成熟,降低氧化应激,改善囊胚发育,提高胚胎存活率。此外,研究结果表明,宫内输送ADSCs和PRP可增强子宫内膜厚度、血管生成和容受性,有报道称,子宫内膜薄或Asherman综合征的妇女可改善植入和妊娠结局。总的来说,这些结果强调了ADSCs和PRP在克服各种不孕原因方面的再生前景。
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引用次数: 0
Emerging threats of monkeypox virus in 2024: current insights and future directions. 2024年猴痘病毒的新威胁:目前的见解和未来的方向。
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-10-31 DOI: 10.1080/20565623.2025.2577069
Dhivya L S, Divya R, Monisha A, Harikrishnan N, Rukaiah Fatma Begum, Nirenjen S, Manisha M, Ankul Singh S

Monkeypox has reemerged as a major global public health threat, with 2024 witnessing the rise of Clade Ib, a new, more transmissible and potentially severe variant. This review analyzes recent outbreaks, uncovering how Clade Ib has accelerated human-to-human and zoonotic spread, increasing case counts across both endemic and non-endemic regions. The study synthesizes current evidence on the virus's evolving epidemiology, diagnostic advances such as CRISPR and PCR techniques, and progress in vaccine development, focusing on JYNNEOS and ACAM2000. Despite these advancements, significant challenges persist, including timely diagnosis and equitable vaccine access, particularly in low-resource settings. Our findings underscore the importance of sustained genomic surveillance, integrative One Health strategies that unite human, animal, and environmental health data, and robust global collaboration. Addressing these gaps is vital for curbing monkeypox and preparing for future zoonotic threats.

猴痘已重新成为一项重大的全球公共卫生威胁,2024年将出现一种新的、传染性更强、可能更严重的变种——b支猴痘。本综述分析了最近的疫情,揭示了进化枝Ib如何加速人与人之间和人畜共患的传播,增加了流行和非流行地区的病例数。该研究综合了关于该病毒不断演变的流行病学的现有证据、CRISPR和PCR技术等诊断进展以及疫苗开发的进展,重点是JYNNEOS和ACAM2000。尽管取得了这些进展,但仍然存在重大挑战,包括及时诊断和公平获得疫苗,特别是在资源匮乏的环境中。我们的研究结果强调了持续的基因组监测、整合人类、动物和环境健康数据的一体化“同一个健康”战略以及强大的全球合作的重要性。解决这些差距对于遏制猴痘和为未来的人畜共患威胁做好准备至关重要。
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引用次数: 0
Using machine learning to develop Iraqi-specific spirometric reference equations. 利用机器学习开发伊拉克特有的肺活量测量参考方程。
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-11-05 DOI: 10.1080/20565623.2025.2582429
Walid Al-Qerem, Alaa Alsajri, Anan Jarab, Judith Eberhardt

Background: Spirometry is essential for diagnosing and managing respiratory diseases. Accurate interpretation relies on reference equations that reflect population-specific lung function. Global equations, such as those from the Global Lung Function Initiative (GLI), may not suit all populations, including Iraqis.

Objective: To develop sex-specific spirometric reference equations for healthy Iraqi adults using machine learning (ML) and compare their performance with the GLI 2012 and 2022 equations.

Methods: This cross-sectional study included 3,959 healthy, nonsmoking Iraqi adults aged ≥18 years. Spirometry was performed per ATS/ERS guidelines. Five ML models (linear regression, random forest, support vector machine, gradient boosting machine (GBM), and k-nearest neighbors) were trained using age and height. Data were split into training (70%) and validation (30%) sets. Performance was assessed using RMSE, R2, and z-score calibration. GBM was selected as the best model.

Results: GBM outperformed all other models and GLI equations. In females, R2 was 0.4473 for FEV1 and 0.4519 for FVC; in males, 0.3509 and 0.3674, respectively. GLI equations underestimated lung volumes, while GBM predictions were well calibrated with mean z-scores near zero.

Conclusion: GBM-derived equations show improved accuracy and calibration over GLI standards for Iraqi adults, offering a more suitable tool for spirometry interpretation.

背景:肺量测定是诊断和治疗呼吸系统疾病的必要手段。准确的解释依赖于反映人群特异性肺功能的参考方程。全球肺功能倡议(Global Lung Function Initiative, GLI)的全球方程式可能并不适合所有人群,包括伊拉克人。目的:利用机器学习(ML)为健康的伊拉克成年人建立性别特异性的肺活量测定参考方程,并将其与GLI 2012和2022方程的性能进行比较。方法:本横断面研究纳入3,959名年龄≥18岁的健康、不吸烟的伊拉克成年人。肺活量测定按照ATS/ERS指南进行。使用年龄和身高训练5个ML模型(线性回归、随机森林、支持向量机、梯度增强机(GBM)和k近邻)。数据分为训练集(70%)和验证集(30%)。使用RMSE、R2和z-score校准评估性能。选择GBM作为最佳模型。结果:GBM优于所有其他模型和GLI方程。雌性FEV1和FVC的R2分别为0.4473和0.4519;男性分别为0.3509和0.3674。GLI方程低估了肺容量,而GBM预测则很好地校准了平均z分数接近于零。结论:与GLI标准相比,gbm导出的方程在伊拉克成年人中显示出更高的准确性和校准性,为肺活量测定解释提供了更合适的工具。
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引用次数: 0
GLM-DM: language model boosted neural networks for HbA1c trend prediction in diabetes mellitus. GLM-DM:语言模型增强神经网络用于糖尿病HbA1c趋势预测。
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-11-11 DOI: 10.1080/20565623.2025.2567166
Yikun Ban, Xinrui He, Patricia M Verona, Curtiss B Cook, Jingrui He

Aim: Predict Hemoglobin A1c (HbA1c) trends, a key metric in diabetes mellitus (DM) management, using readily available patient variables and language models (LMs).

Methods: We propose GLM (Language Model Boosted Neural Network) -DM, which leverages data augmentation and language model-driven feature encoding to predict HbA1c trends using easily accessible patient-level variables. Our model captures complex relationships among patient characteristics and enhances predictive performance through Generative Adversarial Networks (GANs) for synthetic data augmentation and LMs for feature embedding. By transforming patient profiles into rich latent representations, our approach enables a more comprehensive analysis of how patient-level variables correlate with HbA1c trends over time.

Results: Using clinical data from 257 DM patients, GLM-DM achieves 70.2% accuracy of HbA1c trend prediction, outperforming classic classifiers and transformer-based models. Ablation studies confirm the effectiveness of GAN-based augmentation and LM-driven embedding. Our model achieves 68.2% prediction accuracy for Type 1 DM and 72.7% for Type 2 DM.

Conclusion: Proposed approach learns the underlying complex function of HbA1c using clinical variables easily available at the patient visit and leveraging the power of LMs to accurately predict the trend of HbA1c in a period. The model can enhance patient advisories for daily diabetes management without the need for continuous glucose monitoring.

目的:利用现成的患者变量和语言模型(LMs)预测糖化血红蛋白(HbA1c)趋势,这是糖尿病(DM)管理的关键指标。方法:我们提出了GLM(语言模型增强神经网络)-DM,它利用数据增强和语言模型驱动的特征编码来预测HbA1c趋势,使用易于获取的患者级变量。我们的模型捕捉了患者特征之间的复杂关系,并通过生成对抗网络(GANs)增强合成数据,通过LMs增强特征嵌入,提高了预测性能。通过将患者资料转化为丰富的潜在表征,我们的方法能够更全面地分析患者水平变量与HbA1c随时间变化趋势的相关性。结果:使用257例糖尿病患者的临床数据,GLM-DM对HbA1c趋势预测的准确率达到70.2%,优于经典分类器和基于变压器的模型。消融研究证实了基于gan的增强和lm驱动的嵌入的有效性。我们的模型对1型糖尿病的预测准确率为68.2%,对2型糖尿病的预测准确率为72.7%。结论:本文提出的方法利用患者就诊时容易获得的临床变量学习HbA1c的潜在复杂功能,并利用LMs的力量准确预测一段时间内HbA1c的趋势。该模型可以在不需要连续血糖监测的情况下,提高患者对日常糖尿病管理的建议。
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引用次数: 0
Triple autoimmune overlap syndrome of the liver: a rare case of concomitant PBC, PSC, and AIH. 肝脏三重自身免疫重叠综合征:罕见的PBC、PSC和AIH合并病例
IF 2.1 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-12-08 DOI: 10.1080/20565623.2025.2601633
Houssem Hassen, Wafa Dahmani, Ghassen Bedoui, Safia Sakly, Emna Souilem, Yosr Ghdiri, Hanene Jaziri, Aida Ben Slema, Ahlem Brahem, Nour Elleuch, Mehdi Ksiaa

Triple overlap syndrome involving autoimmune hepatitis (AIH), primary biliary cholangitis (PBC), and primary sclerosing cholangitis (PSC) is an exceptionally rare autoimmune hepatopathy, posing major diagnostic and therapeutic challenges. A 51-year-old woman initially diagnosed with AIH-PBC overlap presented persistent hepatocellular cytolysis despite corticosteroids and ursodeoxycholic acid. Autoimmune screening revealed ANA, anti-M2, and anti-gp210 positivity. Repeat liver biopsy showed interface hepatitis, ductopenic lymphocytic cholangitis, and periductal fibrosis. Magnetic resonance cholangiography demonstrated an attenuated intrahepatic biliary tree with a "pruned-tree" appearance, confirming PSC features. The diagnosis of AIH-PBC-PSC triple overlap was established. Due to azathioprine intolerance and hepatotoxicity, therapy was switched to mycophenolate mofetil with UDCA and low-dose corticosteroids, resulting in biochemical stabilization. This case represents one of the few well-documented instances of AIH-PBC-PSC overlap. It underscores the importance of integrating serology, histology, and advanced imaging for accurate diagnosis and individualized therapy. In the absence of standardized guidelines, management relies on tailored immunosuppression and supportive care to prevent progression toward liver failure.

三重重叠综合征包括自身免疫性肝炎(AIH)、原发性胆道炎(PBC)和原发性硬化性胆管炎(PSC),是一种非常罕见的自身免疫性肝病,对诊断和治疗提出了重大挑战。一名最初诊断为AIH-PBC重叠的51岁女性,尽管使用皮质类固醇和熊去氧胆酸,但仍出现持续的肝细胞溶解。自身免疫筛查显示ANA、抗m2和抗gp210阳性。重复肝活检显示界面肝炎,导管减少性淋巴细胞性胆管炎和管周纤维化。磁共振胆管造影显示肝内胆道树减弱,呈“修剪树”状,证实PSC的特征。建立AIH-PBC-PSC三重重叠诊断。由于硫唑嘌呤不耐受和肝毒性,治疗转为霉酚酸酯加UDCA和低剂量皮质类固醇,导致生化稳定。该病例是少有的AIH-PBC-PSC重叠病例之一。它强调了整合血清学、组织学和先进影像学对准确诊断和个体化治疗的重要性。在缺乏标准化指南的情况下,管理依赖于量身定制的免疫抑制和支持性护理,以防止肝衰竭的进展。
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引用次数: 0
Computer vision syndrome: a comprehensive literature review. 计算机视觉综合征:综合文献综述。
IF 2.4 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-03-08 DOI: 10.1080/20565623.2025.2476923
Fares Kahal, Ahmad Al Darra, André Torbey

Computer Vision Syndrome is a growing health concern in the digital age, with a reported prevalence of 69.0%. It is caused by screen-related, environmental, ergonomic, and physiological factors, affecting diverse demographics. The COVID-19 pandemic significantly amplified CVS due to increased screen time for remote work, online learning, and social media use, with studies reporting symptoms in up to 74% of individuals. Unique visual challenges from digital screens, including reduced clarity and glare, exacerbate symptoms like dry eyes and discomfort, especially in those with uncorrected vision. Understanding CVS is crucial for mitigating its impact through effective prevention and management strategies. This study explores the causes, diagnosis, management, and prevention strategies of CVS by synthesizing recent findings from optometry, occupational health, digital health, and ergonomics. It also highlights emerging trends such as AI, wearables, and augmented reality while providing practical management strategies. A narrative review of literature from 2014 to 2024 was conducted, focusing on PubMed-indexed, peer-reviewed articles, including meta-analyses and systematic reviews, with priority given to recent, highly cited studies.

在数字时代,计算机视觉综合症是一个日益严重的健康问题,据报道患病率为69.0%。它是由屏幕相关、环境、人体工程学和生理因素引起的,影响着不同的人口统计学。由于远程工作、在线学习和社交媒体使用的屏幕时间增加,COVID-19大流行显著放大了CVS,研究报告多达74%的人出现了症状。数字屏幕带来的独特视觉挑战,包括清晰度降低和眩光,加剧了眼睛干涩和不适等症状,特别是在那些视力未矫正的人身上。了解CVS对于通过有效的预防和管理策略减轻其影响至关重要。本研究通过综合验光、职业健康、数字健康和人体工程学的最新研究成果,探讨了CVS的原因、诊断、管理和预防策略。它还强调了人工智能、可穿戴设备、增强现实等新兴趋势,并提供了实用的管理策略。对2014年至2024年的文献进行了叙述性回顾,重点关注pubmed索引的同行评议文章,包括荟萃分析和系统综述,优先考虑最近的高引用研究。
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引用次数: 0
Polymeric nanoparticles-based targeted delivery of drugs and bioactive compounds for arthritis management. 基于聚合物纳米颗粒的靶向递送药物和生物活性化合物用于关节炎管理。
IF 2.4 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-02-20 DOI: 10.1080/20565623.2025.2467591
Tanu Dixit, Anuradha Vaidya, Selvan Ravindran

This review explores the potential of polymeric nanoparticles (PNPs) as targeted drug delivery systems for arthritic treatment, overcoming the limitations of the present therapy. A thorough literature search was conducted on the databases PubMed, Scopus, and Web of Science to find published articles on the use of polymeric nanoparticles in the treatment of arthritis. This includes synthesis methods, mechanisms in drug delivery, and applications of PNPs. Polymeric nanoparticles showed excellent promise in the management of arthritis through enhanced stability of drugs, controlled and sustained drug release, and reduced systemic side effects. Some of the highlighted biocompatible and targeting capabilities of natural and synthetic polymers include chitosan, hyaluronic acid, and PLGA. Bioactive compounds such as curcumin and resveratrol delivered by PNPs enhanced therapeutic efficacy in preclinical arthritis models. Despite their promise, challenges such as rapid clearance and manufacturing scalability remain critical barriers. Polymeric nanoparticles offer a transformative approach to arthritis management by enabling targeted, sustained, and safe drug delivery. Translation into clinical applications would thus require developments in nanoparticle design, personalized medicine, and scalable production techniques.

这篇综述探讨了聚合物纳米颗粒(PNPs)作为治疗关节炎的靶向药物传递系统的潜力,克服了目前治疗的局限性。我们对PubMed、Scopus和Web of Science数据库进行了全面的文献检索,以找到有关使用聚合纳米颗粒治疗关节炎的已发表文章。这包括合成方法、药物传递机制和pnp的应用。聚合物纳米颗粒通过增强药物的稳定性,控制和持续的药物释放,减少全身副作用,在关节炎的治疗中显示出良好的前景。一些突出的生物相容性和靶向性的天然和合成聚合物包括壳聚糖,透明质酸和PLGA。通过PNPs传递的姜黄素和白藜芦醇等生物活性化合物增强了临床前关节炎模型的治疗效果。尽管前景看好,但诸如快速清除和制造可扩展性等挑战仍然是关键障碍。聚合物纳米颗粒通过靶向、持续和安全的给药,为关节炎治疗提供了一种变革性的方法。因此,转化为临床应用将需要纳米颗粒设计、个性化医疗和可扩展的生产技术的发展。
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引用次数: 0
CAR-T cell therapies: patient access and affordability solutions. CAR-T细胞疗法:患者可及性和可负担性解决方案。
IF 2.4 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2025-12-01 Epub Date: 2025-03-29 DOI: 10.1080/20565623.2025.2483613
Elisabete Gonçalves

Chimeric Antigen Receptor (CAR)-T cell therapies, as potentially curative treatments, are a group of immunotherapy agents that are changing the paradigm for the treatment of hematologic malignancies. Ongoing research on CAR-T cell therapy is expected to expand the currently approved indications, which, given the high prices of these innovative therapeutic solutions, will increase the pressure on the sustainability of health systems, enhancing the need to establish adjusted financial solutions and promote the implementation of post-marketing monitoring procedures. This study examines the specific challenges in the development of robust clinical evidence to support the value measurement and cost-effectiveness assessment of CAR-T cell therapies and in the selection of adequate financing solutions. Managed Entry Agreements, which create mechanisms in which the risk associated with the uncertainty in long-term outcomes of these therapies is shared between the manufacturer and the payer, have emerged as preferred solutions in several European Union countries. The access barriers to CAR-T cell therapies are described, and recommendations on potential solutions to address affordability concerns using a framework of a life cycle approach to value assessment involving different stakeholders and adapted financing tools are proposed.

嵌合抗原受体(CAR)-T细胞疗法作为一种潜在的治疗方法,是一组正在改变血液系统恶性肿瘤治疗模式的免疫疗法。正在进行的CAR-T细胞疗法研究预计将扩大目前批准的适应症,鉴于这些创新治疗方案的高昂价格,将增加卫生系统可持续性的压力,加强建立调整后的财务解决方案和促进实施上市后监测程序的必要性。本研究探讨了在开发强有力的临床证据以支持CAR-T细胞疗法的价值测量和成本效益评估以及选择适当的融资解决方案方面的具体挑战。管理准入协议创造了一种机制,在这种机制中,与这些疗法长期结果的不确定性相关的风险由制造商和付款人共同承担,这已成为一些欧盟国家的首选解决方案。本文描述了CAR-T细胞疗法的获取障碍,并提出了使用涉及不同利益相关者和适应融资工具的生命周期方法框架来解决可负担性问题的潜在解决方案。
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引用次数: 0
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