Drugs in Context最新文献

{"title":"Pegvisomant in severe, treatment-resistant thyroid eye disease: a case report.","authors":"Laura Köröskényi, Noémi Szkibák, Zita Steiber, Sándor Barna, Edit B Nagy, Zsolt Karányi, József Balla, Miklós Bodor, Endre V Nagy, Annamária Erdei","doi":"10.7573/dic.2026-7-5","DOIUrl":"10.7573/dic.2026-7-5","url":null,"abstract":"<p><p>Teprotumumab, a monoclonal antibody against the IGF1 receptor, has proven effective in the treatment of thyroid eye disease (TED). Pegvisomant reduces IGF1 levels through selective inhibition of growth hormone receptors. Here, we present a hypothesis-generating case of a 69-year-old patient with progressive TED. Pegvisomant 20 mg was administered daily for 90 days. IGF1 levels declined and remained low-normal during treatment. Clinical activity scores and VISA (vision, inflammation, strabismus and appearance) scores improved rapidly. MRI assessment showed that water content of both extraocular muscles and orbital connective tissue decreased after treatment. ^99mTc-DTPA SPECT confirmed the decrease in disease activity. Further studies may confirm the efficacy of pegvisomant in TED.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674976/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149215192","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A review of dextromethorphan and bupropion (Auvelity™) for the treatment of agitation in Alzheimer's disease.","authors":"Rajesh R Tampi, Kayla Murphy, Julia Golden, Haley Schuster, Robin Conyers","doi":"10.7573/dic.2026-6-4","DOIUrl":"10.7573/dic.2026-6-4","url":null,"abstract":"<p><p>Dextromethorphan hydrobromide and bupropion hydrochloride extended-release tablets (Auvelity^TM) became the second medication to be approved by the FDA for the treatment of agitation in Alzheimer's disease dementia after brexpiprazole. It also became the first non-antipsychotic medication to be approved for this specific indication. In this Editorial, the available data regarding the efficacy and adverse effects of this medication in the treatment of agitation in Alzheimer's disease dementia are reviewed.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674975/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149215079","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Biochemical and clinical changes during enzymatic replacement therapy in hypophosphatasia: three real-world cases in Spain.","authors":"Mercedes Marbán Calzón, María Pilar Aguado Acín, Juan Luis Carrillo-Linares, Alicia González Jimeno, José Rioja Villodres, Carolina Tornero Marín","doi":"10.7573/dic.2026-5-14","DOIUrl":"10.7573/dic.2026-5-14","url":null,"abstract":"<p><p>Hypophosphatasia (HPP) is a rare recessive or dominant autosomal disease caused by loss-of-function variants in the <i>ALPL</i> gene, which encodes tissue-nonspecific alkaline phosphatase (TNSALP). Molecules involved in the pathogenesis of HPP are alkaline phosphatase (ALP), inorganic pyrophosphate (PPi; an inhibitor of bone mineralization), pyridoxal-5'-phosphate (PLP; responsible for the neurological complications) and phosphoethanolamine. Asfotase alfa is a recombinant, bone-targeted, human TNSALP approved to treat patients with HPP. Its clinical effectiveness has been demonstrated in real-world settings across international HPP registries. However, evidence from routine clinical practice in Spain is lacking. Here, we report three Spanish cases of HPP treated successfully with asfotase alfa: a girl diagnosed at 3 months; a woman diagnosed at age 47 years; and a 27-year-old woman with paediatric-onset symptoms. In all cases, asfotase alfa treatment was associated with an increase in serum ALP, marked reductions in plasma PPi and PLP, and marked improvements in symptoms and physical performance. Our series of HPP cases confirms the beneficial effects of asfotase alfa on biochemical and clinical outcomes, consistent with evidence from randomized clinical trials.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674974/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149215178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Corrigendum: SGLT2 inhibitor role in cardio-metabolic-renal diseases: a narrative review of recent evidence and their pharmacological, clinical and economic implications.","authors":"","doi":"10.7573/dic.2026-8-5","DOIUrl":"https://doi.org/10.7573/dic.2026-8-5","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.7573/dic.2026-1-1.].</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674977/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149215240","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Standalone NF1: guiding the transition from paediatric to adult care.","authors":"Héctor Salvador, Álvaro Lassaletta, Juan Carlos López-Gutiérrez, Marc Cucurull, Ignacio Blanco","doi":"10.7573/dic.2026-6-6","DOIUrl":"https://doi.org/10.7573/dic.2026-6-6","url":null,"abstract":"<p><p>This report summarizes expert and patient-representative perspectives from the meeting entitled 'Standalone NF1: guiding the transition from paediatric to adult care', held in Madrid on October 2, 2025. Its primary objective was to examine clinical, organizational and patient-related challenges during transition and identify practical measures to improve continuity of care. Discussions focused on multidisciplinary co-ordination, case management, patient autonomy and structured transition pathways. NF1 characteristics, plexiform neurofibroma burden, surgery, and the currently approved MEK inhibitors selumetinib and mirdametinib, are summarized only for clinical context; selumetinib is discussed in greater detail because it was the treatment available at that time and the one addressed during the meeting.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13626555/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149105311","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Local carboxymethyl β-glucan and polycarbophil in women positive for human papillomavirus: a prospective observational study.","authors":"Carmen Pingarrón Santofimia, Ysmael Álvarez Espinosa, Juan José Hernández Aguado, Ana Isabel López Castejón, Maria Soledad Martínez García, Carlos Blanco-Soler, Cristina Del Valle Rubido, Maria Del Pilar Robles Ruiz, Janet Abanto Alvarez, Maria Del Carmen Chicharro Cassuso, Amira Alkourdi Martinez, Monica de Vera Gomis, Silvia Tamarit Bordes, Laura Rubert Torro, Caterina Cortés Alaguero, Irene Vela Sampedro, Manuel David Menor Almagro, Stefanie Redon Fitzl, Laura Lopez Prada, Montserrat Albarrán Gómez","doi":"10.7573/dic.2026-5-1","DOIUrl":"10.7573/dic.2026-5-1","url":null,"abstract":"<p><strong>Background: </strong>High-risk human papillomavirus (HR-HPV) infections with normal or low-grade cervical cytology are generally managed through observation because many infections clear spontaneously. However, persistent infection, particularly in women over 35 years of age, may increase the risk of progression and lead to prolonged surveillance, repeated examinations and patient anxiety. This study evaluates the effectiveness of local treatment with carboxymethyl β-glucan and polycarbophil (Colpofix^®) in achieving negativization of high-risk (HR) human papillomavirus (HPV) in a Spanish population of women with HR-HPV positivity.</p><p><strong>Methods: </strong>This prospective, non-randomized controlled, multi-centre study enrolled 533 women aged 25-65 with HR-HPV and normal cytology, atypical squamous cells of undetermined significance or low-grade squamous intraepithelial lesions. Conducted in real clinical settings, it involved a 3-month treatment or 'wait-and-see' approach, with final follow-up at 6 months. Statistical analysis included various tests such as the Mann-Whitney test, Fisher's exact test and Pearson's χ² test.</p><p><strong>Results: </strong>At 6 months, HR-HPV negativity was observed in 50.1% of treated patients compared with 24.0% in the observation group (<i>p</i><0.0001). Higher clearance rates were recorded across all HR-HPV genotypes. For HPV16, negativization occurred in 50.7% of treated patients <i>versus</i> 9.2% of controls (<i>p</i>=0.0291). Resolution of cervical ectopy was more frequent in the treatment group (42.6% <i>versus</i> 11.5%, <i>p</i><0.0001). No adverse events were reported.</p><p><strong>Conclusions: </strong>Local treatment with carboxymethyl β-glucan and polycarbophil was associated with higher rates of HR-HPV negativization and ectopy resolution compared with observation alone at 6-month follow-up, supporting its use as a management option, particularly in women over 35 years of age.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13585142/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148988578","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cardiovascular effects of doxazosin in the treatment of prostatic hyperplasia.","authors":"Qianhui Fan, Libin Fan, Shihang Kuang, Huafu Li","doi":"10.7573/dic.2026-4-12","DOIUrl":"10.7573/dic.2026-4-12","url":null,"abstract":"<p><p>Doxazosin, a selective α1-receptor antagonist, is widely used to treat lower urinary tract symptoms associated with benign prostatic hyperplasia (BPH). However, its cardiovascular effects remain clinically relevant, particularly in older patients. This systematic review and meta-analysis evaluated blood pressure, heart rate, headache and vertigo during doxazosin treatment. PubMed/MEDLINE, the Cochrane Library, CNKI, Wanfang Data, Google Scholar and ScienceDirect were searched using Chinese and English terms related to doxazosin, BPH, prostate and cardiovascular outcomes. Sixteen evidence reports were included: 12 primary studies, 2 pooled analyses and 2 secondary review sources that supplied aggregate trial data. The pooled analyses showed no statistically significant difference in systolic blood pressure (MD 1.44, 95% CI -4.17 to 7.06; <i>p</i>=0.61), diastolic blood pressure (MD -0.33, 95% CI -3.68 to 3.03; <i>p=</i>0.85), headache (OR 0.99, 95% CI 0.73-1.34; <i>p</i>=0.95) or heart rate (MD 0.98, 95% CI -0.89 to 2.84; <i>p</i>=0.30). Vertigo was more frequent with doxazosin than with control treatment (OR 1.93, 95% CI 1.50-2.49; <i>p</i><0.00001). Risk-of-bias assessment identified 2 reports at low risk, 10 with some concerns, 1 at high risk, 1 observational study of moderate quality, and 2 secondary reviews for which Cochrane Risk of Bias 2 and the Newcastle-Ottawa Scale were not applicable. Doxazosin was not associated with statistically significant average changes in blood pressure or heart rate, but the increased occurrence of vertigo warrants counselling and monitoring, particularly in older patients at risk of falls.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13585143/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148988650","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multidisciplinary expert consensus on treatment intensification in metastatic androgen pathway modulation-sensitive prostate cancer: a case-based RAND/UCLA analysis.","authors":"Ángel Borque-Fernando, Teresa Alonso-Gordoa, María José Juan-Fita, Fernando Lopez Campos, Daniel Adolfo Pérez-Fentes, Antoni Vilaseca, Cristina Moretones Agut, Paola Usán, Pablo Maroto Rey","doi":"10.7573/dic.2026-6-2","DOIUrl":"10.7573/dic.2026-6-2","url":null,"abstract":"<p><p>Metastatic androgen pathway modulation (mAPM)-sensitive prostate cancer, formerly termed metastatic hormone-sensitive prostate cancer, is a clinically heterogeneous disease in which treatment intensification decisions are guided by metastatic volume and timing of presentation. However, increasing use of advanced imaging and molecular profiling has revealed prognostic scenarios that are not adequately addressed by current algorithms. This study presents a structured multidisciplinary expert consensus exercise, rather than primary clinical research, applying the RAND/UCLA Appropriateness Method to evaluate systemic treatment intensification strategies in mAPM-sensitive prostate cancer. Four anonymized real-world clinical cases representing key prognostic constellations - metachronous/synchronous disease, low/high metastatic burden - were assessed. Panellists rated the appropriateness of doublet therapy <i>versus</i> triplet therapy, including tailored 'what-if' variations, through two rounds of anonymous voting with structured discussion. The resulting assessments reflect structured expert judgement integrating available evidence with clinical experience. The panel identified metastatic pattern, particularly the presence and type of visceral involvement, disease dynamics, patient fitness and imaging context as dominant drivers of treatment selection. These findings should be interpreted as consensus-based appropriateness assessments rather than evidence-based treatment recommendations. They support a personalized, multidisciplinary approach to mAPM-sensitive prostate cancer management, emphasizing expert integration of prognostic factors beyond volume-based classifications in clinical scenarios where high-level evidence is limited or not directly applicable.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13574230/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148967280","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of an Ayurvedic Menohelp syrup in the management of heavy menstrual bleeding (menorrhagia): an open-label clinical study.","authors":"Kamini Dhiman, Pradeep Kumar Prajapati, Deepika Gopal, Ashutosh Gautam, Rajat Singal","doi":"10.7573/dic.2026-3-1","DOIUrl":"10.7573/dic.2026-3-1","url":null,"abstract":"<p><strong>Background: </strong>Heavy menstrual bleeding (HMB) is a common gynaecological condition that significantly affects the quality of life and daily functioning amongst women of reproductive age. In Ayurveda, this condition is described under the category of Asrigdara. Menohelp Syrup, an Ashoka (<i>Saraca asoca</i>)-based Ayurvedic formulation, is traditionally used for the management of HMB. This open-label study evaluated the efficacy and safety of Menohelp Syrup in women with HMB. The study aimed to assess reductions in menstrual blood loss and associated symptoms along with improvement in quality of life.</p><p><strong>Methods: </strong>This open-label clinical study included 228 non-pregnant women with HMB aged 18-45 years. Participants received Menohelp Syrup at a dose of 15 mL twice daily for 90 days. The primary outcome was menstrual blood loss assessed using the Pictorial Blood Assessment Chart. Secondary outcomes included bleeding duration, cycle regularity, pain-related symptoms, haemoglobin and haematological parameters, endometrial thickness, and quality of life assessed using the Menorrhagia Impact Questionnaire.</p><p><strong>Results: </strong>Treatment with Menohelp Syrup resulted in a substantial reduction in menstrual blood loss, with a 54% decrease in estimated menstrual blood loss and a 58% reduction in Pictorial Blood Assessment Chart scores from baseline to day 90. Improvement was also observed in bleeding duration, absorbent product usage, clot passage and bleeding-related urgency. Menstrual pain symptoms improved considerably, with 68.06% of participants reporting complete resolution of dysmenorrhoea at the end of treatment. Mean haemoglobin levels showed modest improvement, whilst endometrial thickness remained stable throughout the study period. No clinically significant adverse events were reported, indicating a favourable safety profile.</p><p><strong>Conclusions: </strong>Menohelp Syrup was well tolerated and was associated with clinically meaningful improvement in HMB, bleeding-related symptom burden and quality-of-life outcomes in women with HMB. These findings support the potential role of Menohelp Syrup as a safe, non-invasive therapeutic option for the management of HMB.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13574237/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148967305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world safety assessment of MET-targeted therapies: a pharmacovigilance analysis using the FAERS database.","authors":"Ning Li, Minghua Cong, Jincheng Yang","doi":"10.7573/dic.2026-4-7","DOIUrl":"10.7573/dic.2026-4-7","url":null,"abstract":"<p><strong>Background: </strong>Globally, lung cancer is the foremost contributor to cancer-associated fatalities. Of its various histological sub-types, non-small-cell lung cancer (NSCLC) constitutes the vast majority, accounting for more than 85% of diagnoses. A key driver in this pathology is the mesenchymal-epithelial transition (MET) receptor tyrosine kinase, whose biological activity is governed by the hepatocyte growth factor (HGF) ligand. MET plays a pivotal role in regulating tumour cell proliferation and migration, and aberrant activation of MET signalling is linked to the pathogenesis of NSCLC.</p><p><strong>Methods: </strong>A retrospective analysis of the FAERS database (2016-2025) was conducted to obtain adverse event data pertaining to four MET-targeted therapeutic agents, namely amivantamab, savolitinib, capmatinib and tepotinib. A systematic disproportionality analysis was performed to identify adverse events.</p><p><strong>Results: </strong>Statistically significant differences were observed across most baseline clinical characteristics. Patients taking tepotinib were significantly older (mean 74.7 years) and experience a higher mortality (21%) than those taking amivantamab, savolitinib or capmatinib. In signal detection, amivantamab caused severe cutaneous/mucosal and infusion-related toxicities. The three c-Met inhibitors led to oedema, with savolitinib distinguished by haematological risks, capmatinib by severe oedema and tepotinib by pulmonary/renal toxicities. In time-to-onset analysis, amivantamab's infusion-related reaction occurred earliest with the highest incidence; savolitinib (pyrexia/hepatic abnormalities) and capmatinib (nausea) appeared early with relatively high incidences; tepotinib's peripheral swelling was early but had a low incidence. High death-associated adverse drug reactions included dyspnoea, peripheral oedema, decreased appetite and increased creatinine, warranting further investigation. In cluster analysis, key adverse drug reactions (e.g. infusion-related reactions for amivantamab, hepatotoxicity for savolitinib, peripheral swelling for capmatinib and peripheral oedema for tepotinib) primarily occur as isolated events with limited co-occurrence of multiple toxicities.</p><p><strong>Conclusion: </strong>These findings support individualized monitoring and evidence-based treatment selection for older patients, those with comorbidities or patients at high risk with MET-altered NSCLC.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"15 ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2026-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13574236/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148967295","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}