Pub Date : 2020-08-04DOI: 10.1080/23808993.2020.1804865
P. Cavalcante, R. Mantegazza, P. Bernasconi
ABSTRACT Introduction Inter-individual variation in drug efficacy and tolerability in myasthenia gravis (MG), an autoimmune disorder mostly treated by chronic immunosuppression, highlights the need of specific, safe and tailored precision medicine approaches. By targeting the disease-effector molecules, biological drugs are the most promising therapeutic agents to treat MG. A number of pharmacogenetic biomarkers associated with response to immunosuppressive drugs in MG have been identified, and microRNAs (miRNAs) are emerging as reliable markers for predicting or monitoring treatment response in individual patients. Areas covered This review provides an overview of pharmacogenetic and pharmaco-miR biomarkers associated with immunosuppressive treatment response in MG and other autoimmune diseases, pointing out the need of pharmacogenetic/-miR profiling for the recently developed biological drugs as an important step toward precision medicine. Expert opinion Extension of pharmacogenetic and pharmaco-miR data, and their entry into the clinical practice, hold the promise to greatly revolutionize MG therapy with clinically relevant drugs, including conventional and biological drugs, or their combination. High-throughput technologies, covering DNA and RNA, have the potential to disclose valuable pharmacogenomic/-miRNomic profiles able to guide the choice of the different drugs in individual patients, or biomarker-defined patients’ subgroups, thus significantly improving MG treatment in a cost/effective manner.
{"title":"Pharmacogenetic and pharmaco-miR biomarkers for tailoring and monitoring myasthenia gravis treatments","authors":"P. Cavalcante, R. Mantegazza, P. Bernasconi","doi":"10.1080/23808993.2020.1804865","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804865","url":null,"abstract":"ABSTRACT Introduction Inter-individual variation in drug efficacy and tolerability in myasthenia gravis (MG), an autoimmune disorder mostly treated by chronic immunosuppression, highlights the need of specific, safe and tailored precision medicine approaches. By targeting the disease-effector molecules, biological drugs are the most promising therapeutic agents to treat MG. A number of pharmacogenetic biomarkers associated with response to immunosuppressive drugs in MG have been identified, and microRNAs (miRNAs) are emerging as reliable markers for predicting or monitoring treatment response in individual patients. Areas covered This review provides an overview of pharmacogenetic and pharmaco-miR biomarkers associated with immunosuppressive treatment response in MG and other autoimmune diseases, pointing out the need of pharmacogenetic/-miR profiling for the recently developed biological drugs as an important step toward precision medicine. Expert opinion Extension of pharmacogenetic and pharmaco-miR data, and their entry into the clinical practice, hold the promise to greatly revolutionize MG therapy with clinically relevant drugs, including conventional and biological drugs, or their combination. High-throughput technologies, covering DNA and RNA, have the potential to disclose valuable pharmacogenomic/-miRNomic profiles able to guide the choice of the different drugs in individual patients, or biomarker-defined patients’ subgroups, thus significantly improving MG treatment in a cost/effective manner.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"317 - 329"},"PeriodicalIF":1.2,"publicationDate":"2020-08-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804865","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46297234","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-08-03DOI: 10.1080/23808993.2020.1804864
N. Tidbury, J. Preston, W. Ding, J. Rivera‐Caravaca, F. Marín, G. Lip
ABSTRACT Introduction Atrial fibrillation is the most common sustained cardiac rhythm disorder, which currently affects 1–2% of the global population. Furthermore, the incidence and prevalence of atrial fibrillation is rising. Biomarkers have the potential to improve clinical management of patients and therefore reduce the burden on health systems in the future. Areas covered A variety of pathways and mechanisms have been associated with atrial fibrillation. This paper provides an overview of a range of blood-based, imaging and genetic biomarkers that are associated with mechanisms and outcomes in atrial fibrillation and their potential use in a clinical setting. Expert commentary Atrial fibrillation is becoming increasingly prevalent. Current biomarkers associated with atrial fibrillation such as those involved in myocardial stress, inflammation, hemostasis and fibrosis do not currently provide much additional practical value beyond recommended scores based only on clinical risk factors.
{"title":"Utilizing biomarkers associated with cardiovascular events in atrial fibrillation: informing a precision medicine response","authors":"N. Tidbury, J. Preston, W. Ding, J. Rivera‐Caravaca, F. Marín, G. Lip","doi":"10.1080/23808993.2020.1804864","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804864","url":null,"abstract":"ABSTRACT Introduction Atrial fibrillation is the most common sustained cardiac rhythm disorder, which currently affects 1–2% of the global population. Furthermore, the incidence and prevalence of atrial fibrillation is rising. Biomarkers have the potential to improve clinical management of patients and therefore reduce the burden on health systems in the future. Areas covered A variety of pathways and mechanisms have been associated with atrial fibrillation. This paper provides an overview of a range of blood-based, imaging and genetic biomarkers that are associated with mechanisms and outcomes in atrial fibrillation and their potential use in a clinical setting. Expert commentary Atrial fibrillation is becoming increasingly prevalent. Current biomarkers associated with atrial fibrillation such as those involved in myocardial stress, inflammation, hemostasis and fibrosis do not currently provide much additional practical value beyond recommended scores based only on clinical risk factors.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"331 - 345"},"PeriodicalIF":1.2,"publicationDate":"2020-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804864","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48062056","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-08-03DOI: 10.1080/23808993.2020.1804866
M. Frantzi, E. Gómez-Gómez, H. Mischak
ABSTRACT Introduction Prostate cancer (PCa) is one of the most frequently diagnosed malignancies worldwide and is associated with high mortality. Broad screening through prostate-specific antigen analysis, along with an aging and growing population has resulted in a vast increase in PCa incidence. As not all PCa forms are life threatening, personalized management is of paramount importance to preserve survival and quality of life for the diagnosed patients. Owing to the complexity of PCa, noninvasive biomarkers for diagnosis, stratification and monitoring, are essential to tailor intervention among patients with different disease manifestations. Areas covered In this article, we aim to provide a critical assessment of the reported noninvasive biomarkers for PCa and their applicability according to the targeted clinical context. For this purpose, a systematic review of the literature published within the last five years was performed, focusing on noninvasive biomarkers to guide initial and repeated biopsies, stratify for active surveillance, monitor biochemical recurrence and metastasis, and adjust treatment for metastatic castration resistant PCa. Expert’s opinion Evidence from clinical trials on novel drugs and latest technological advancements, indicate several clinical applications for biomarkers to tailor intervention throughout PCa progression, toward a more personalized medicine approach in PCa clinical management.
{"title":"Noninvasive biomarkers to guide intervention: toward personalized patient management in prostate cancer","authors":"M. Frantzi, E. Gómez-Gómez, H. Mischak","doi":"10.1080/23808993.2020.1804866","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804866","url":null,"abstract":"ABSTRACT Introduction Prostate cancer (PCa) is one of the most frequently diagnosed malignancies worldwide and is associated with high mortality. Broad screening through prostate-specific antigen analysis, along with an aging and growing population has resulted in a vast increase in PCa incidence. As not all PCa forms are life threatening, personalized management is of paramount importance to preserve survival and quality of life for the diagnosed patients. Owing to the complexity of PCa, noninvasive biomarkers for diagnosis, stratification and monitoring, are essential to tailor intervention among patients with different disease manifestations. Areas covered In this article, we aim to provide a critical assessment of the reported noninvasive biomarkers for PCa and their applicability according to the targeted clinical context. For this purpose, a systematic review of the literature published within the last five years was performed, focusing on noninvasive biomarkers to guide initial and repeated biopsies, stratify for active surveillance, monitor biochemical recurrence and metastasis, and adjust treatment for metastatic castration resistant PCa. Expert’s opinion Evidence from clinical trials on novel drugs and latest technological advancements, indicate several clinical applications for biomarkers to tailor intervention throughout PCa progression, toward a more personalized medicine approach in PCa clinical management.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"383 - 400"},"PeriodicalIF":1.2,"publicationDate":"2020-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804866","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43225406","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-07-31DOI: 10.1080/23808993.2020.1804867
I. Miyagawa, S. Kubo, Yoshiya Tanaka
ABSTRACT Introduction The strategy of precision medicine is achieved by the stratification of patients and targeted therapy. It has been shown that the pathological conditions of patients who are classified or diagnosed with a single disease are actually diverse in almost all autoimmune diseases. Therefore, the practice of precision medicine is important for the treatment of patients with autoimmune diseases. Areas covered At present, precision medicine has not been achieved in any autoimmune disease. This article reviewed the attempted use of precision medicine for the treatment of systemic lupus erythematosus and psoriatic arthritis. Expert opinion Although multiple molecular-targeted therapies are available for the treatment of rheumatoid arthritis, the response to each treatment strategy often differs markedly among patients, and it is unclear how they are differentially selected for each patient. The establishment of precision medicine is especially important in heterogenous autoimmune diseases (e.g., systemic lupus erythematosus, psoriatic arthritis). Various symptoms must be improved at the same time, but the treatment options are limited. Therefore, the selection of the optimal treatment for individual patients is complex. Although some predictors of the treatment response are reported, precision medicine has not been sufficiently well evaluated in real clinical practice, especially for autoimmune diseases.
{"title":"A wide perspective of targeted therapies for precision medicine in autoimmune diseases","authors":"I. Miyagawa, S. Kubo, Yoshiya Tanaka","doi":"10.1080/23808993.2020.1804867","DOIUrl":"https://doi.org/10.1080/23808993.2020.1804867","url":null,"abstract":"ABSTRACT Introduction The strategy of precision medicine is achieved by the stratification of patients and targeted therapy. It has been shown that the pathological conditions of patients who are classified or diagnosed with a single disease are actually diverse in almost all autoimmune diseases. Therefore, the practice of precision medicine is important for the treatment of patients with autoimmune diseases. Areas covered At present, precision medicine has not been achieved in any autoimmune disease. This article reviewed the attempted use of precision medicine for the treatment of systemic lupus erythematosus and psoriatic arthritis. Expert opinion Although multiple molecular-targeted therapies are available for the treatment of rheumatoid arthritis, the response to each treatment strategy often differs markedly among patients, and it is unclear how they are differentially selected for each patient. The establishment of precision medicine is especially important in heterogenous autoimmune diseases (e.g., systemic lupus erythematosus, psoriatic arthritis). Various symptoms must be improved at the same time, but the treatment options are limited. Therefore, the selection of the optimal treatment for individual patients is complex. Although some predictors of the treatment response are reported, precision medicine has not been sufficiently well evaluated in real clinical practice, especially for autoimmune diseases.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"447 - 453"},"PeriodicalIF":1.2,"publicationDate":"2020-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1804867","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46728653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-07-28DOI: 10.1080/23808993.2020.1792286
F. Pasquier, C. Chahine, C. Marzac, S. de Botton
ABSTRACT Introduction Management of acute myeloid leukemia (AML) remains challenging, especially for relapsed or refractory (R/R) AML patients who display poor prognosis with conventional therapies. This underlined the need for new treatments in this population. Areas covered This review will focus on ivosidenib, an oral inhibitor of mutated isocitrate dehydrogenase 1 (IDH1) enzyme. Mechanisms of IDH1 mutations and their inhibition by ivosidenib will be cover, as well as clinical efficacy and safety of ivosidenib in R/R AML. Ivosidenib has been approved by the FDA for R/R AML patients with IDH1 mutation in July 2018 and for unfit AML patients with IDH1 mutation as first line treatment in May 2019. Expert commentary Ivosidenib induces impressive response rates in R/R AML, a population of bad prognosis. Nevertheless, primary and acquired resistances to ivosidenib have been recently described, underlining importance of the ongoing clinical trials with ivosidenib in combination with standard chemotherapy, hypomethylating agents or other targeted therapies.
{"title":"Ivosidenib for the treatment of relapsed or refractory acute myeloid leukemia with an IDH1 mutation","authors":"F. Pasquier, C. Chahine, C. Marzac, S. de Botton","doi":"10.1080/23808993.2020.1792286","DOIUrl":"https://doi.org/10.1080/23808993.2020.1792286","url":null,"abstract":"ABSTRACT Introduction Management of acute myeloid leukemia (AML) remains challenging, especially for relapsed or refractory (R/R) AML patients who display poor prognosis with conventional therapies. This underlined the need for new treatments in this population. Areas covered This review will focus on ivosidenib, an oral inhibitor of mutated isocitrate dehydrogenase 1 (IDH1) enzyme. Mechanisms of IDH1 mutations and their inhibition by ivosidenib will be cover, as well as clinical efficacy and safety of ivosidenib in R/R AML. Ivosidenib has been approved by the FDA for R/R AML patients with IDH1 mutation in July 2018 and for unfit AML patients with IDH1 mutation as first line treatment in May 2019. Expert commentary Ivosidenib induces impressive response rates in R/R AML, a population of bad prognosis. Nevertheless, primary and acquired resistances to ivosidenib have been recently described, underlining importance of the ongoing clinical trials with ivosidenib in combination with standard chemotherapy, hypomethylating agents or other targeted therapies.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"429 - 438"},"PeriodicalIF":1.2,"publicationDate":"2020-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1792286","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43294350","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-07-16DOI: 10.1080/23808993.2020.1792285
Louis Williams, J. Caro, B. Razzo, E. Boyle, G. Morgan
ABSTRACT Introduction Multiple myeloma (MM) is plasma cell dyscrasia with marked variability in its clinical presentation and outcome, both of which are dictated by its underlying genetics. Next-generation sequencing techniques (NGS) have become essential to understanding the genomics of multiple myeloma. The exploitation of these advances in the clinic will require new clinical trial designs with endpoints that facilitate rapid readouts of success or failure and capture the impact of rare mutational events on outcomes. An understanding of NGS and its applications in multiple myeloma is therefore significant for both researchers and clinicians alike. Areas covered In this review, we summarize the significant advances that NGS has yielded in our understanding of the prognosis, clonal evolution, treatment and response assessment of multiple myeloma and its precursor conditions. We synthesize the relevant literature related to both genomics and the clinical management of MM, with articles selected based on our experience in the field. Expert opinion In the opinion of these authors, NGS will play a significant role in the future of precision medicine in multiple myeloma, especially as disease-specific panels and response adapted approaches to therapy gain traction. In the process, challenges related to cost, quality control, and standardization will need to be overcome.
{"title":"Deep sequencing as an approach to understanding the complexity and improving the treatment of multiple myeloma","authors":"Louis Williams, J. Caro, B. Razzo, E. Boyle, G. Morgan","doi":"10.1080/23808993.2020.1792285","DOIUrl":"https://doi.org/10.1080/23808993.2020.1792285","url":null,"abstract":"ABSTRACT Introduction Multiple myeloma (MM) is plasma cell dyscrasia with marked variability in its clinical presentation and outcome, both of which are dictated by its underlying genetics. Next-generation sequencing techniques (NGS) have become essential to understanding the genomics of multiple myeloma. The exploitation of these advances in the clinic will require new clinical trial designs with endpoints that facilitate rapid readouts of success or failure and capture the impact of rare mutational events on outcomes. An understanding of NGS and its applications in multiple myeloma is therefore significant for both researchers and clinicians alike. Areas covered In this review, we summarize the significant advances that NGS has yielded in our understanding of the prognosis, clonal evolution, treatment and response assessment of multiple myeloma and its precursor conditions. We synthesize the relevant literature related to both genomics and the clinical management of MM, with articles selected based on our experience in the field. Expert opinion In the opinion of these authors, NGS will play a significant role in the future of precision medicine in multiple myeloma, especially as disease-specific panels and response adapted approaches to therapy gain traction. In the process, challenges related to cost, quality control, and standardization will need to be overcome.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"363 - 370"},"PeriodicalIF":1.2,"publicationDate":"2020-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1792285","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49370900","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-06-19DOI: 10.1080/23808993.2020.1776107
M. Francke, B. D. de Winter, L. Elens, N. Lloberas, D. Hesselink
Twenty-five years after its approval by the FDA and EMA, tacrolimus remains the cornerstone of immunosuppressive treatment following solid organ transplantation [1,2]. The drug is highly effective ...
{"title":"The pharmacogenetics of tacrolimus and its implications for personalized therapy in kidney transplant recipients","authors":"M. Francke, B. D. de Winter, L. Elens, N. Lloberas, D. Hesselink","doi":"10.1080/23808993.2020.1776107","DOIUrl":"https://doi.org/10.1080/23808993.2020.1776107","url":null,"abstract":"Twenty-five years after its approval by the FDA and EMA, tacrolimus remains the cornerstone of immunosuppressive treatment following solid organ transplantation [1,2]. The drug is highly effective ...","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"313 - 316"},"PeriodicalIF":1.2,"publicationDate":"2020-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1776107","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45737291","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-06-16DOI: 10.1080/23808993.2020.1777853
Y. Garcilazo-Reyes, M. Ibañez-Juliá, I. Hernández-Verdin, L. Nguyen-Them, N. Younan, C. Houillier, K. Hoang-Xuan, A. Alentorn
ABSTRACT Introduction Primary central nervous system lymphoma (PCNSL) is a rare extra-nodal non-Hodgkin lymphoma that in the vast majority of cases belongs to diffuse large B-cell lymphoma (DLBCL) histology. The standard first-line treatment is based on high-dose methotrexate (HD-MTX) regimens. However, the majority of patients will relapse, leading to a poor prognosis of the disease. Areas covered Reviewed are the potential new therapeutic approaches in PCNSL. With the advent of tailored treatment, immunomodulators and immunotherapies are appearing as new promising therapeutic approaches for this orphan disease. This review seeks to summarize the novel approaches currently under evaluation. Expert opinion The therapeutic management of PCNSL is rapidly evolving with the description of PCNSL molecular alterations. However, due to the rarity of this disease, phase III clinical trials using new therapeutic drugs are still lacking. In addition, the vast majority of newly diagnosed PCNSL affect elderly patients, and specific and adapted clinical trials for this fragile population are warranted. Currently, the use of targeted therapies or immune-mediated treatments is only studied in relapsed/refractory (R/R) PCNSL, but the use of these approaches as a first-line treatment (compared with HD-MTX) could also be used as new promising approaches to decrease the toxicity associated with MTX regimens.
{"title":"Treating central nervous system lymphoma in the era of precision medicine","authors":"Y. Garcilazo-Reyes, M. Ibañez-Juliá, I. Hernández-Verdin, L. Nguyen-Them, N. Younan, C. Houillier, K. Hoang-Xuan, A. Alentorn","doi":"10.1080/23808993.2020.1777853","DOIUrl":"https://doi.org/10.1080/23808993.2020.1777853","url":null,"abstract":"ABSTRACT Introduction Primary central nervous system lymphoma (PCNSL) is a rare extra-nodal non-Hodgkin lymphoma that in the vast majority of cases belongs to diffuse large B-cell lymphoma (DLBCL) histology. The standard first-line treatment is based on high-dose methotrexate (HD-MTX) regimens. However, the majority of patients will relapse, leading to a poor prognosis of the disease. Areas covered Reviewed are the potential new therapeutic approaches in PCNSL. With the advent of tailored treatment, immunomodulators and immunotherapies are appearing as new promising therapeutic approaches for this orphan disease. This review seeks to summarize the novel approaches currently under evaluation. Expert opinion The therapeutic management of PCNSL is rapidly evolving with the description of PCNSL molecular alterations. However, due to the rarity of this disease, phase III clinical trials using new therapeutic drugs are still lacking. In addition, the vast majority of newly diagnosed PCNSL affect elderly patients, and specific and adapted clinical trials for this fragile population are warranted. Currently, the use of targeted therapies or immune-mediated treatments is only studied in relapsed/refractory (R/R) PCNSL, but the use of these approaches as a first-line treatment (compared with HD-MTX) could also be used as new promising approaches to decrease the toxicity associated with MTX regimens.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"275 - 281"},"PeriodicalIF":1.2,"publicationDate":"2020-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1777853","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"44387843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-06-09DOI: 10.1080/23808993.2020.1764346
M. Frías, A. Rivero-Juarez, I. Machuca, Á. Camacho, A. Rivero
ABSTRACT Introduction This review focuses on the milestones and challenges of precision medicine in the natural history of HCV infection. Areas covered The manuscript summarizes the genetic and viral factors identified as predictive of outcome in HCV patients and discusses opportunities for a precision medicine approach to the management of patients with HCV infection. Special emphasis will be placed on the factors associated with response to treatment as well as other factors that can help the clinician in the management of patients after sustained virological response. Expert opinion The establishment of DAAs in recent years has led to a paradigmatic change in the natural history of hepatitis C virus infection. However, there are still challenges that precision medicine must solve, especially in the management of patients at high risk of liver complications after sustained virological response.
{"title":"The outlook for precision medicine for the treatment of chronic hepatitis C infection: challenges and opportunities","authors":"M. Frías, A. Rivero-Juarez, I. Machuca, Á. Camacho, A. Rivero","doi":"10.1080/23808993.2020.1764346","DOIUrl":"https://doi.org/10.1080/23808993.2020.1764346","url":null,"abstract":"ABSTRACT Introduction This review focuses on the milestones and challenges of precision medicine in the natural history of HCV infection. Areas covered The manuscript summarizes the genetic and viral factors identified as predictive of outcome in HCV patients and discusses opportunities for a precision medicine approach to the management of patients with HCV infection. Special emphasis will be placed on the factors associated with response to treatment as well as other factors that can help the clinician in the management of patients after sustained virological response. Expert opinion The establishment of DAAs in recent years has led to a paradigmatic change in the natural history of hepatitis C virus infection. However, there are still challenges that precision medicine must solve, especially in the management of patients at high risk of liver complications after sustained virological response.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"253 - 263"},"PeriodicalIF":1.2,"publicationDate":"2020-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1764346","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43337561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-06-07DOI: 10.1080/23808993.2020.1776106
A. Vatrella, A. Maglio, S. Pellegrino, C. Pelaia, C. Stellato, G. Pelaia, C. Vitale
ABSTRACT Introduction Asthma is a complex disease with heterogeneity in etiology, triggers, clinical characteristics and different responses to pharmacological therapies. Areas covered Patients with severe uncontrolled asthma constitute a relatively small percentage, ranging from 5 to 10% of all asthmatics. These patients can benefit from targeted biological therapies developed in recent years. In fact, a better understanding of the etiopathological mechanisms of different phenotypes and endotypes of severe asthma have led to the availability of innovative biological therapies, able to modify the natural history of the disease by targeting molecules involved in airway inflammation. Several phenotypes based on clinical and physiologic variables and on inflammatory markers have been reported. Expert opinion The priority is to define the molecular process underlying the disease. In this context the recognition of T2 and non T2 inflammatory pathways, so called molecular phenotypes, represents the most reliable approach to drive the use of novel biological therapies. For this purpose, several biomarkers have been validated for identifying severe asthma phenotypes and for guiding the choice of the most appropriate treatment. The purpose of this review is to discuss the current knowledge about the molecular phenotypes of severe asthma, as well as the rationale underlying the use of existing biological drugs.
{"title":"Phenotyping severe asthma: a rationale for biologic therapy","authors":"A. Vatrella, A. Maglio, S. Pellegrino, C. Pelaia, C. Stellato, G. Pelaia, C. Vitale","doi":"10.1080/23808993.2020.1776106","DOIUrl":"https://doi.org/10.1080/23808993.2020.1776106","url":null,"abstract":"ABSTRACT Introduction Asthma is a complex disease with heterogeneity in etiology, triggers, clinical characteristics and different responses to pharmacological therapies. Areas covered Patients with severe uncontrolled asthma constitute a relatively small percentage, ranging from 5 to 10% of all asthmatics. These patients can benefit from targeted biological therapies developed in recent years. In fact, a better understanding of the etiopathological mechanisms of different phenotypes and endotypes of severe asthma have led to the availability of innovative biological therapies, able to modify the natural history of the disease by targeting molecules involved in airway inflammation. Several phenotypes based on clinical and physiologic variables and on inflammatory markers have been reported. Expert opinion The priority is to define the molecular process underlying the disease. In this context the recognition of T2 and non T2 inflammatory pathways, so called molecular phenotypes, represents the most reliable approach to drive the use of novel biological therapies. For this purpose, several biomarkers have been validated for identifying severe asthma phenotypes and for guiding the choice of the most appropriate treatment. The purpose of this review is to discuss the current knowledge about the molecular phenotypes of severe asthma, as well as the rationale underlying the use of existing biological drugs.","PeriodicalId":12124,"journal":{"name":"Expert Review of Precision Medicine and Drug Development","volume":"5 1","pages":"265 - 274"},"PeriodicalIF":1.2,"publicationDate":"2020-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/23808993.2020.1776106","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"44896154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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