Drugs in ContextPub Date : 2024-12-18eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-10-6
Hema Sundaram, Beatriz Molina, Editta Buttura da Prato, Gabriel Siquier-Dameto, Michela Zazzaron, Clara Cigni, Franco Grimolizzi
{"title":"Good scientific practice of using worldwide post-marketing surveillance data to ensure safety with HA<sub>ALI</sub> BDDE cross-linked hyaluronic acid fillers.","authors":"Hema Sundaram, Beatriz Molina, Editta Buttura da Prato, Gabriel Siquier-Dameto, Michela Zazzaron, Clara Cigni, Franco Grimolizzi","doi":"10.7573/dic.2024-10-6","DOIUrl":"10.7573/dic.2024-10-6","url":null,"abstract":"<p><strong>Background: </strong>Aliaxin fillers (HA<sub>ALI</sub>), produced by IBSA Farmaceutici Italia SrL (Italy), are biodegradable, non-pyrogenic, 1,4-butanediol diglycidyl ether cross-linked hyaluronic acid (HA) hydrogels. The formulations are tailored for different clinical indications, ensuring precise and natural outcomes. Their cohesivity and tissue integration capabilities are associated with relatively few adverse events (AEs), supporting their widespread use in aesthetic treatments. This article examines the real-world safety profile of HA<sub>ALI</sub> fillers derived from worldwide post-marketing surveillance data.</p><p><strong>Methods: </strong>Post-marketing surveillance was registered by the manufacturer from January 2018 to September 2023. During this period, product complaints were globally gathered from healthcare practitioners and consumers, relating to technical issues or safety and product-related adverse events.</p><p><strong>Results: </strong>No discernible trend or substantial escalation in AEs across the entire product range were observed during the surveillance period (<i>p</i>>0.05). No statistically significant increases (<i>p</i>>0.05) in the frequency or severity of safety incidents and AEs were observed. The most frequently observed AEs were oedema (26%) and swelling (19%).</p><p><strong>Conclusion: </strong>The analysed data further support and confirm the high safety profile of the HA<sub>ALI</sub> fillers for different approaches in aesthetic medicine. This evaluation also highlights the importance of post-marketing analysis by continuing to foster a robust understanding of products currently used in daily clinical practice.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11666269/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142881249","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}
Drugs in ContextPub Date : 2024-11-28eCollection Date: 2024-01-01DOI: 10.7573/dic.2023-12-3
Joel C Marrs, Sarah L Anderson
{"title":"Inclisiran for the treatment of hypercholesterolaemia.","authors":"Joel C Marrs, Sarah L Anderson","doi":"10.7573/dic.2023-12-3","DOIUrl":"10.7573/dic.2023-12-3","url":null,"abstract":"<p><p>Inclisiran is a synthetic small interfering RNA (siRNA) that inhibits the production of proprotein convertase subtilisin/kexin 9 (PCSK9) in hepatocytes by silencing the translation of PCSK9 mRNA. The result of this mechanism is a decrease in PCSK9 synthesis resulting in decreased degradation of the LDL receptor, leading to more LDL receptors being available to clear LDL cholesterol (LDL-C) from the circulation. Inclisiran received FDA approval in 2021 and EMA approval in 2020. The indication for inclisiran use is as an adjunct to diet and statin therapy for the treatment of adults with primary hyperlipidaemia, including those with heterozygous familial hypercholesterolaemia to reduce LDL-C. Inclisiran has demonstrated consistent LDL-C lowering in the range of 44-54%. Furthermore, inclisiran has been demonstrated to be a safe medication with indications of significant or serious adverse events when compared to placebo. Inclisiran is given as an initial subcutaneous dose followed by a repeat dose at 3 months and every 6 months thereafter. The 2022 American College of Cardiology Expert Consensus Decision Pathway includes inclisiran as an option for non-statin therapy in addition to maximally tolerated statin therapy in those at very high risk of atherosclerotic cardiovascular disease or those with LDL-C >190 mg/dL. The ORION-4, VICTORION-1 PREVENT and VICTORION-2 PREVENT trials are ongoing and designed to evaluate the ability of inclisiran to reduce major cardiovascular events in addition to LDL-C lowering but will not be completed for a few years.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11619601/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142784573","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}
Drugs in ContextPub Date : 2024-11-21eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-6-5
Marta Matrone, Alessandro Cuomo, Sergio De Filippis, Andrea Fagiolini, Mario Amore
{"title":"Liquid antipsychotics in the management of psychomotor agitation: a focus on promazine.","authors":"Marta Matrone, Alessandro Cuomo, Sergio De Filippis, Andrea Fagiolini, Mario Amore","doi":"10.7573/dic.2024-6-5","DOIUrl":"https://doi.org/10.7573/dic.2024-6-5","url":null,"abstract":"<p><p>Psychomotor agitation (PMA) is a prominent clinical issue frequently observed in various psychiatric and neurological conditions, including schizophrenia, bipolar disorder, Parkinson disease, dementia and substance use disorder. Characterized by motor restlessness, anxiety and irritability, PMA can rapidly escalate into aggression and violence, necessitating prompt intervention to ensure patient and caregiver safety. The prevalence of PMA in psychiatric emergency settings ranges from 4.3% to 10%, imposing a substantial burden on healthcare systems. Despite the critical nature of PMA, there is a lack of standardized treatment protocols, particularly concerning the use of liquid formulations of antipsychotics such as liquid promazine, which may offer unique advantages in emergency care. This review aims to provide a comprehensive analysis of the existing literature on the efficacy, safety and tolerability of liquid antipsychotics, with a particular focus on promazine, in the management of PMA. An extensive literature search was conducted across publicly available databases with no time limitations to ensure the inclusion of all relevant articles. The findings suggest that liquid promazine offers several benefits, including ease of administration, rapid onset of action and improved patient compliance, making it a valuable option in acute PMA management. However, the review also highlights the need for future research, particularly long-term studies and head-to-head comparisons with other antipsychotics, to better establish the clinical utility of liquid promazine. Future research should focus on expanding the evidence base for liquid antipsychotic formulations, which will contribute to improved clinical outcomes in the management of PMA.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11610565/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142767510","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}
Drugs in ContextPub Date : 2024-11-18eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-7-1
Gema Ariceta, Simón Lalanza, Catalina Peña, Marta Martínez Montero, Carlos Bezos Daleske, Laura Acuña Álvarez, Elisa Giner
{"title":"Patient journey in cystinosis: focus on non-adherence and disease management.","authors":"Gema Ariceta, Simón Lalanza, Catalina Peña, Marta Martínez Montero, Carlos Bezos Daleske, Laura Acuña Álvarez, Elisa Giner","doi":"10.7573/dic.2024-7-1","DOIUrl":"https://doi.org/10.7573/dic.2024-7-1","url":null,"abstract":"<p><strong>Background: </strong>Few studies have assessed patient-reported experience measures in nephropathic cystinosis. This study uses patient reports focused on the impact of cystinosis, cysteamine treatment-associated problems, and therapeutic adherence and suggests potential actions for improvement.</p><p><strong>Methods: </strong>In March 2022, six patients with nephropathic cystinosis treated with cysteamine, aged between 12 and 40 years as well as two caregivers, underwent standardized online interviews. Further, in April 2022, two online workshops were organized, each one with the participation of an advisory board consisting of up to four patients and six caregivers. As a result, the first patient journey mapping was developed considering pre-diagnosis, diagnosis and post-diagnosis steps, prescription of treatment, laboratory tests and daily life for patients, categorized by age (children, teenagers, adults). A patient support programme was also considered.</p><p><strong>Results: </strong>Patients were not completely aware of the risks associated with non-adherence. The main factors explaining poor adherence were impaired sleep and chronic fatigue, both related to cysteamine night dosing and prominent gastrointestinal symptoms. These factors have a negative impact on the daily lives of patients. Opportunities for improvement in disease management and therapeutic adherence in nephropathic cystinosis were highlighted. Consequently, a series of lines of action and suggestions were made.</p><p><strong>Conclusion: </strong>This qualitative study offers insights on nephropathic cystinosis from the point of view of patients and parents/caregivers. The critical steps during patient journey and the pitfalls for therapeutic adherence have been highlighted, opening ways to improve not only disease management but also the quality of life of patients with cystinosis.A lay summary is provided as supplementary material; available at: https://www.drugsincontext.com/wp-content/uploads/2024/10/dic.2024-7-1-Suppl-Lay-Summary.pdf.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11610563/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142766145","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":"Pharmacovigilance monitoring and treatment adherence in patients on antihypertensive drugs at a tertiary care centre.","authors":"Pooja Agrawal, Shilpa Patrick, Meenu Thomas, Dhyuti Gupta, Prithpal Singh Matreja, Preeti Singh, Shaneela Zafar","doi":"10.7573/dic.2024-5-2","DOIUrl":"10.7573/dic.2024-5-2","url":null,"abstract":"<p><strong>Background: </strong>Hypertension is one of the main factors contributing to the global burden of non-communicable diseases. Previous research has revealed that stress, bad lifestyle choices and a lack of knowledge about the disease are the main causes of hypertension that can be controlled. The key cause behind the prevalence of the condition is the lack of medication adherence by patients. This study aims to evaluate medication adherence in patients with hypertension through the Morisky Medication Adherence Scale (MMAS) and to observe any adverse drug reaction leading to non-adherence of medications.</p><p><strong>Methods: </strong>A descriptive, cross-sectional study was conducted on 124 patients who attended the outpatient department of medicine. The descriptive tools were MMAS and causality scales for adverse drug reactions.</p><p><strong>Result: </strong>The mean MMAS score was 5.20±1.29. Amongst the demographic profile, age, sex, comorbidities and duration of disease were significantly associated with decreased mean MMAS scores. Forty-two patients experienced drug reactions and only four patients were adherent to their medications.</p><p><strong>Conclusion: </strong>Our study suggests that patients were poorly adherent to their medications. Effective interventions should be considered to improve adherence in patients. Monitoring for adverse drug reactions can lead to improved patient outcomes, whilst interventions to improve adherence can lead to better blood pressure control and reduced risk of cardiovascular events.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11514578/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142521312","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}
Drugs in ContextPub Date : 2024-10-10eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-6-1
Silvia Alberti Violetti, Marco Ardigò, Paolo Fava, Giuseppe Gritti, Erika Morsia, Francesco Onida, Marco Paulli, Alessandro Pileri, Pietro Quaglino, Serena Rupoli, Miriam Teoli, Pamela Vezzoli
{"title":"Multidisciplinary and personalized approach to the management of mycosis fungoides with chlormethine gel: a collection of clinical experiences.","authors":"Silvia Alberti Violetti, Marco Ardigò, Paolo Fava, Giuseppe Gritti, Erika Morsia, Francesco Onida, Marco Paulli, Alessandro Pileri, Pietro Quaglino, Serena Rupoli, Miriam Teoli, Pamela Vezzoli","doi":"10.7573/dic.2024-6-1","DOIUrl":"https://doi.org/10.7573/dic.2024-6-1","url":null,"abstract":"<p><p>Topical chlormethine (CL) gel formulation was approved by the EMA in 2017 for the treatment of adult patients with mycosis fungoides (MF). To expand the knowledge on the management of MF, this paper provides an overview of clinical practice evidence about the MF diagnostic phase and a collection of clinical experiences to better characterize the use of CL gel in daily practice. Collected cases underline the importance of the concomitant biopsy and clinical evaluation in the diagnostic phase, with the contribution of a multidisciplinary team, and support the use of CL gel as a first-line or adjuvant treatment in selected patients.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11482090/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142460554","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}
Drugs in ContextPub Date : 2024-10-09eCollection Date: 2024-01-01DOI: 10.7573/dic.2023-11-5
Anna Campanati, Maria Esposito, Giacomo Caldarola, Sara Cacciapuoti, Gabriella Fabbrocini
{"title":"Fixed-dose combination of calcipotriene/betamethasone dipropionate foam for the management of mild-to-moderate psoriasis in daily clinical practice: a collection of clinical experiences.","authors":"Anna Campanati, Maria Esposito, Giacomo Caldarola, Sara Cacciapuoti, Gabriella Fabbrocini","doi":"10.7573/dic.2023-11-5","DOIUrl":"https://doi.org/10.7573/dic.2023-11-5","url":null,"abstract":"<p><p>A fixed-dose combination of calcipotriene 0.005%/betamethasone dipropionate 0.064% (Cal/BD) aerosol foam (Enstilar, LEO Pharma) is the only topical therapy approved for the acute (reactive) and proactive management of psoriasis. Although treatment with Cal/BD foam has been characterized in a clinical context, further evidence is needed to determine its optimal use in clinical practice. A group of experts discussed the value of the Cal/BD foam as a topical treatment for mild-to-moderate psoriasis in combination with systemic treatments. The reported experiences support effectiveness of the Cal/BD foam in daily clinical practice, with an improvement in patient quality of life.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11482089/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142460553","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}
Drugs in ContextPub Date : 2024-09-23eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-7-2
Annamaria Bosi, Carlo Lombardi, Cristiano Caruso, Marcello Cottini, Ilaria Baglivo, Stefania Colantuono, Francesco Menzella
{"title":"Clinical remission and control in severe asthma: agreements and disagreements.","authors":"Annamaria Bosi, Carlo Lombardi, Cristiano Caruso, Marcello Cottini, Ilaria Baglivo, Stefania Colantuono, Francesco Menzella","doi":"10.7573/dic.2024-7-2","DOIUrl":"https://doi.org/10.7573/dic.2024-7-2","url":null,"abstract":"<p><p>Over the last two decades, we have witnessed great advancements in our understanding of the immunological pathways of asthma, leading to the development of targeted therapies, such as biologic drugs, that have radically and definitively changed the clinical outcomes of severe asthma. Despite the numerous therapeutic options available, ~4-10% of all people with asthma have severe or uncontrolled asthma, associated with an increased risk of developing chronic oral corticosteroid use, fixed airflow limitation, exacerbations, hospitalization and, finally, increased healthcare costs. The new concept of disease modification in asthma comes from the evolution of asthma management, which encompasses phenotyping patients with different inflammatory endotypes characterizing the disease, followed by the advent of more effective therapies capable of targeting the proximal factors of airway inflammation. This treat-to-target approach aims to achieve remission of the disease. Because the novel treatment paradigm for severe asthma with the advent of biologic therapies is no longer clinical control but rather clinical remission - a step closer to the concept of cure - a deeper and more accurate understanding of the critical causal mechanisms and endotypes of asthma is necessary to achieve the goal of clinical remission, which has the potential to generate real life-changing benefits for patients. This review aims to frame the evolution of the debated concept of clinical remission and provide clinicians with insights that may be helpful in achieving remission in the greatest number of patients.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11430537/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142343712","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}
Drugs in ContextPub Date : 2024-09-05eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-6-3
Nicoletta Bernardini, Nevena Skroza, Laura Atzori, Cristina Mugheddu, Matteo Megna, Sara Cacciapuoti, Michela Ortoncelli, Maria A Montesu, Antonio Carpentieri, Martino Carriero, Maria G Atzori, Gianmario Addis, Riccardo Balestri, Giulia Rech, Pierluigi Bruni, Manuela Papini, Concetta Potenza
{"title":"Rapid Efficacy of riSankizumab in pretibial psoriasis invOLVEment: RESOLVE.","authors":"Nicoletta Bernardini, Nevena Skroza, Laura Atzori, Cristina Mugheddu, Matteo Megna, Sara Cacciapuoti, Michela Ortoncelli, Maria A Montesu, Antonio Carpentieri, Martino Carriero, Maria G Atzori, Gianmario Addis, Riccardo Balestri, Giulia Rech, Pierluigi Bruni, Manuela Papini, Concetta Potenza","doi":"10.7573/dic.2024-6-3","DOIUrl":"https://doi.org/10.7573/dic.2024-6-3","url":null,"abstract":"<p><strong>Background: </strong>Despite extraordinary improvements in the management of psoriasis in recent times, some areas of the body, such as the pretibial area, still show an unsatisfactory response and a more significant impact on patient quality of life. This multicentre study focuses on psoriasis affecting sensitive areas (particularly the pretibial area), its impact on quality of life and the therapeutic response to risankizumab.</p><p><strong>Methods: </strong>This multicentre prospective observational study recruited patients with moderate-to-severe psoriasis with pretibial area involvement. All patients underwent treatment with risankizumab (150 mg every 3 weeks), and efficacy was assessed after 24 weeks.</p><p><strong>Results: </strong>The study included 128 patients with a mean age of 51 years, suffering from moderate-to-severe psoriasis with involvement of the pretibial area with median total Psoriasis Area Severity Index score of 17.05 and Dermatology Life Quality Index of 16.27. The group was further divided into two sub-groups: the 'mother patch' group, in whom the very first psoriatic plaque appeared in the pretibial region (45 patients), and the 'non-mother patch' group, in whom the psoriatic lesion in the pretibial region was present but not as the first manifestation (83 patients). In order to better assess the involvement of psoriasis in the pretibial area, the pretibial plaque lesion severity index was also calculated at baseline in all patients: extent 2.75, erythema 2.64, infiltration 2.45 and desquamation 2.38. All participants in this study showed a good therapeutic response, with a reduction in all scores.</p><p><strong>Conclusions: </strong>The pretibial area is becoming an object of therapeutic interest due to some resistance to clearance and the consequent impairment of patient quality of life. This study showed that risankizumab can give favourable therapeutic results not only in patients with moderate-to-severe psoriasis with involvement of the difficult-to-treat areas but particularly in patients with recalcitrant plaques in the pretibial area.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11389876/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142282055","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}
Drugs in ContextPub Date : 2024-08-20eCollection Date: 2024-01-01DOI: 10.7573/dic.2024-3-1
Renata Tj Fazoli, Luciano F Drager, Roberto Kalil-Filho, Giuliano Generoso
{"title":"RNA interference therapy in cardiology: will new targets improve therapeutic goals?","authors":"Renata Tj Fazoli, Luciano F Drager, Roberto Kalil-Filho, Giuliano Generoso","doi":"10.7573/dic.2024-3-1","DOIUrl":"10.7573/dic.2024-3-1","url":null,"abstract":"<p><p>The discovery of RNA interference in 1998 opened avenues for the manipulation of gene expression, leading to the development of small interfering RNA (siRNA) drugs. Patisiran, the first FDA-approved siRNA medication, targets hereditary transthyretin amyloidosis with polyneuropathy. Givosiran, lumasiran and nedosiran further expand siRNA applications in treating rare genetic diseases, demonstrating positive outcomes. In cardiology, inclisiran, approved for hypercholesterolaemia, showcases sustained reductions in LDL cholesterol levels. However, ongoing research aims to establish its impact on cardiovascular outcomes. Lipoprotein(a), an independent risk factor for atherosclerotic cardiovascular disease, has become a focus of siRNA therapies, precipitating the development of specific siRNA drugs like olpasiran, zerlasiran and lepodisiran, with promising reductions in lipoprotein(a) levels. Research to assess the effectiveness of these medications in reducing events is currently under way. Zodasiran and plozasiran address potential risk factors for cardiovascular diseases, targeting triglyceride-rich lipoproteins. Zilebesiran, which targets hepatic angiotensinogen mRNA, has demonstrated a dose-related reduction in serum angiotensinogen levels, thereby lowering blood pressure in patients with systemic arterial hypertension. The evolving siRNA methodology presents a promising future in cardiology, with ongoing studies assessing its effectiveness in various conditions. In the future, larger studies will provide insights into improvements in cardiovascular outcomes, long-term safety and broader applications in the general population. This review highlights the historical timeline of the development of siRNA-based drugs, their clinical indications, potential side-effects and future perspectives.</p>","PeriodicalId":11362,"journal":{"name":"Drugs in Context","volume":"13 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11346576/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142072342","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}