Expert Opinion on Emerging Drugs最新文献_第10页

Antibody-based therapy for acute myeloid leukemia: a review of phase 2 and 3 trials. 基于抗体的急性髓性白血病治疗:2期和3期试验回顾。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-06-01 Epub Date: 2022-07-04 DOI: 10.1080/14728214.2022.2094365

Xavier Thomas, Mohamed Elhamri, Alexandre Deloire, Maël Heiblig

{"title":"Antibody-based therapy for acute myeloid leukemia: a review of phase 2 and 3 trials.","authors":"Xavier Thomas, Mohamed Elhamri, Alexandre Deloire, Maël Heiblig","doi":"10.1080/14728214.2022.2094365","DOIUrl":"https://doi.org/10.1080/14728214.2022.2094365","url":null,"abstract":"Introduction: Despite recent advances in the treatment of adult acute myeloid leukemia (AML), the clinical outcome of patients continues to be unsatisfactory especially among older patients, those with a high-risk profile, and in the relapsed/refractory setting. For this reason, recent clinical trials have explored novel therapeutic agents either used alone or in combination with intensive chemotherapy or low-intensity treatments.Areas covered: The current paper reviews the clinical development of monoclonal antibody-based therapies in AML, their current status and phases 2 and 3 prospective trials.Expert opinion: Monoclonal antibody-based therapies demonstrated efficacy and tolerability in several clinical trials, especially when used in combination either with '3 + 7' chemotherapy or with low-intensity treatments. Additional studies are needed to determine new antigens for antibody-based therapies that target leukemia stem cells and spare normal hematopoiesis. Phase 2 and 3 additional clinical trial data are needed to assess the promise of first trials, especially regarding chimeric antigen receptor T cells redirected against myeloid antigens and immune checkpoint inhibitor therapies.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"169-185"},"PeriodicalIF":3.4,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40396454","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1

Emerging drugs for antibody-mediated rejection after kidney transplantation: a focus on phase II & III trials. 新兴药物治疗肾移植后抗体介导的排斥反应:II期和III期试验的重点。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-06-01 Epub Date: 2022-06-23 DOI: 10.1080/14728214.2022.2091131

Katharina A Mayer, Klemens Budde, Bernd Jilma, Konstantin Doberer, Georg A Böhmig

{"title":"Emerging drugs for antibody-mediated rejection after kidney transplantation: a focus on phase II & III trials.","authors":"Katharina A Mayer, Klemens Budde, Bernd Jilma, Konstantin Doberer, Georg A Böhmig","doi":"10.1080/14728214.2022.2091131","DOIUrl":"https://doi.org/10.1080/14728214.2022.2091131","url":null,"abstract":"Introduction: Antibody-mediated rejection (ABMR) is a leading cause of kidney allograft failure. Its therapy continues to be challenge, and no treatment has been approved for the market thus far.Areas covered: In this article, we discuss the pathophysiology and phenotypic presentation of ABMR, the current level of evidence to support the use of available therapeutic strategies, and the emergence of tailored drugs now being evaluated in systematic clinical trials. We searched PubMed, Clinicaltrials.gov and Citeline's Pharmaprojects for pertinent information on emerging anti-rejection strategies, laying a focus on phase II and III trials.Expert opinion: Currently, we rely on the use of apheresis for alloantibody depletion and intravenous immunoglobulin (referred to as standard of care), preferentially in early active ABMR. Recent systematic trials have questioned the benefits of using the CD20 antibody rituximab or the proteasome inhibitor bortezomib. However, there are now several promising treatment approaches in the pipeline, which are being trialed in phase II and III studies. These include interleukin-6 antagonism, CD38-targeting antibodies, and selective inhibitors of complement. On the basis of the information that has emerged so far, it seems that innovative treatment strategies for clinical use in ABMR may be available within the next 5-10 years.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"151-167"},"PeriodicalIF":3.4,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39986305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 11

Treatment for acute respiratory distress syndrome in adults: a narrative review of phase 2 and 3 trials. 成人急性呼吸窘迫综合征的治疗:2期和3期试验的叙述性回顾。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-06-01 Epub Date: 2022-07-28 DOI: 10.1080/14728214.2022.2105833

Denise Battaglini, Chiara Robba, Paolo Pelosi, Patricia R M Rocco

{"title":"Treatment for acute respiratory distress syndrome in adults: a narrative review of phase 2 and 3 trials.","authors":"Denise Battaglini, Chiara Robba, Paolo Pelosi, Patricia R M Rocco","doi":"10.1080/14728214.2022.2105833","DOIUrl":"https://doi.org/10.1080/14728214.2022.2105833","url":null,"abstract":"Introduction: Ventilatory management and general supportive care of acute respiratory distress syndrome (ARDS) in the adult population have led to significant clinical improvements, but morbidity and mortality remain high. Pharmacologic strategies acting on the coagulation cascade, inflammation, oxidative stress, and endothelial cell injury have been targeted in the last decade for patients with ARDS, but only a few of these have shown potential benefits with a meaningful clinical response and improved patient outcomes. The lack of availability of specific pharmacologic treatments for ARDS can be attributed to its complex pathophysiology, different risk factors, huge heterogeneity, and difficult classification into specific biological phenotypes and genotypes.Areas covered: In this narrative review, we briefly discuss the relevance and current advances in pharmacologic treatments for ARDS in adults and the need for the development of new pharmacological strategies.Expert opinion: Identification of ARDS phenotypes, risk factors, heterogeneity, and pathophysiology may help to design clinical trials personalized according to ARDS-specific features, thus hopefully decreasing the rate of failed clinical pharmacologic trials. This concept is still under clinical investigation and needs further development.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"187-209"},"PeriodicalIF":3.4,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40530017","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 5

Emerging drugs for the treatment of hereditary angioedema due to C1-inhibitor deficiency. 治疗c1抑制剂缺乏引起的遗传性血管性水肿的新药物。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-06-01 Epub Date: 2022-07-26 DOI: 10.1080/14728214.2022.2105834

Andrea Zanichelli, Vincenzo Montinaro, Massimo Triggiani, Francesco Arcoleo, Debora Visigalli, Mauro Cancian

{"title":"Emerging drugs for the treatment of hereditary angioedema due to C1-inhibitor deficiency.","authors":"Andrea Zanichelli, Vincenzo Montinaro, Massimo Triggiani, Francesco Arcoleo, Debora Visigalli, Mauro Cancian","doi":"10.1080/14728214.2022.2105834","DOIUrl":"https://doi.org/10.1080/14728214.2022.2105834","url":null,"abstract":"Introduction: Hereditary angioedema due to C1-inhibitor (C1-INH-HAE) is a rare disease characterized by unpredictable swelling attacks that may be life-threatening when affecting the upper airways. Understanding the pathophysiology of HAE and the mechanism of bradykinin-mediated angioedema allowed the development of new therapies for the treatment of HAE: clinical trials are ongoing to expand the number of drugs available for on-demand treatment and prophylaxis.Areas covered: Authors discuss the products that have been used to treat this disease for many years and present the most recently marketed products and those which are under development.Expert opinion: Significant therapeutic progress has been made in HAE. In particular, drugs targeting specific molecules involved in the angioedema formation were developed and studies with new drugs are ongoing. In the coming years, more effective therapies with easier administration route options for on-demand treatment and long-term prophylaxis will be available to treat this disease and the variety of patients. Gene therapy strategies may offer a definitive treatment. High costs of current and new drugs may be a limiting factor for their availability, especially in developing countries.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"103-110"},"PeriodicalIF":3.4,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40621487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Emerging drugs for the treatment of sickle cell disease: a review of phase II/III trials. 用于治疗镰状细胞病的新兴药物:II/III期试验综述

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-06-01 Epub Date: 2022-08-01 DOI: 10.1080/14728214.2022.2105835

Jules M Ross, Stéphanie Forté, Denis Soulières

{"title":"Emerging drugs for the treatment of sickle cell disease: a review of phase II/III trials.","authors":"Jules M Ross, Stéphanie Forté, Denis Soulières","doi":"10.1080/14728214.2022.2105835","DOIUrl":"https://doi.org/10.1080/14728214.2022.2105835","url":null,"abstract":"Introduction: The substitution of glutamic acid by valine on the ß-globin chain produces the hemoglobin S variant responsible for sickle cell disease (SCD), a disorder that affects millions of people worldwide and leads to acute and cumulative organ damage. Even though life expectancy has significantly improved where the best medical care is available, there are still few therapeutic options for SCD and those are limited by their availability, cost, and individual toxicities.Areas covered: This review summarizes the clinical data on current treatments for SCD and emerging therapies studied in the acute setting as well as potential disease-modifying agents, with an emphasis on the FDA-approved agents.Expert opinion: Hydroxyurea has been a gold standard for two decades, showing benefits in acute complications and overall survival in sickle cell anemia, although data is lacking for certain genotypes such as hemoglobin SC. As progress is made in our understanding of the pathophysiological networks characterizing SCD, numerous pathways appear to be targetable, with L-glutamine, crizanlizumab and voxelotor now approved by the FDA. Pursuing a multi-agent approach could alter the disease course in a more effective fashion and provide an alternative option to curative therapies, but longer clinical studies are needed.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"211-224"},"PeriodicalIF":3.4,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40662674","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1

Epidermal growth factor receptor-targeted therapy for the treatment of non-small cell lung cancer: a review of phase II and III trials 表皮生长因子受体靶向治疗非小细胞肺癌:II期和III期试验综述

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-04-03 DOI: 10.1080/14728214.2022.2063836

Hong-Lian Lu, G. Jie, Yi‐Long Wu

{"title":"Epidermal growth factor receptor-targeted therapy for the treatment of non-small cell lung cancer: a review of phase II and III trials","authors":"Hong-Lian Lu, G. Jie, Yi‐Long Wu","doi":"10.1080/14728214.2022.2063836","DOIUrl":"https://doi.org/10.1080/14728214.2022.2063836","url":null,"abstract":"ABSTRACT Introduction Epidermal growth factor receptor (EGFR) is one of the most common driver gene mutations in non-small-cell lung cancer (NSCLC). EGFR-tyrosine kinase inhibitors (TKIs) monotherapy and EGFR-TKI combined with chemotherapy or anti-angiogenesis drugs have significantly prolonged the survival of patients with EGFR-mutant NSCLC. However, disease progression caused by acquired resistance to EGFR-TKIs is inevitable. And patients with EGFR exon 20ins showed limited efficacy to EGFR-TKIs. Areas covered In this review, we initially evaluated the efficacy of existing treatments for EGFR-mutant NSCLC. Second, we reviewed the ongoing phase II and III clinical trials, provided the latest results, discussed the scientific rationale of these trials and the potential development issues. Expert opinion The application of EGFR-TKIs has greatly changed the therapeutic strategies for advanced and resected NSCLC with EGFR mutations, and the 5-year overall survival (OS) rate for advanced NSCLC was close to 40%. The current research direction for the treatment of patients with EGFR mutations focuses on the following three aspects: uncommon EGFR mutation subtypes, brain metastases, and EGFR TKI-based combination therapy. Future studies on EGFR-mutant NSCLC therapy will focus on overcoming EGFR-TKI-related resistance, preventing drug resistance in advance, and developing bispecific antibody drugs.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"111 - 126"},"PeriodicalIF":3.4,"publicationDate":"2022-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47773104","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1

Assessing the developing pharmacotherapeutic landscape in hepatitis B treatment: a spotlight on drugs in phase II clinical trials 评估乙型肝炎治疗药物治疗的发展前景：II期临床试验中药物的关注

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-04-03 DOI: 10.1080/14728214.2022.2074977

R. Hui, L. Mak, W. Seto, M. Yuen

{"title":"Assessing the developing pharmacotherapeutic landscape in hepatitis B treatment: a spotlight on drugs in phase II clinical trials","authors":"R. Hui, L. Mak, W. Seto, M. Yuen","doi":"10.1080/14728214.2022.2074977","DOIUrl":"https://doi.org/10.1080/14728214.2022.2074977","url":null,"abstract":"ABSTRACT Introduction Functional cure, defined as sustained HBsAg seroclearance, is associated with favorable outcomes in chronic hepatitis B (CHB). While nucleos(t)ide analogues (NAs) are effective in suppressing HBV replication, NAs are unable to induce functional cure at high rates. A range of novel HBV antivirals, aiming to induce functional cure, are currently under development. Areas covered This article covered novel hepatitis B virus (HBV) antivirals that have entered phase II trials. Virus-directing agents covered include entry inhibitors, transcription inhibitors, RNA silencers, core protein allosteric modulators, noncompetitive polymerase inhibitors, and viral protein export inhibitors. Immunomodulators covered include innate immune stimulators, T-cell modulators, therapeutic vaccines, and monoclonal antibodies. Upcoming developmental directions would also be discussed. Expert opinion Among novel HBV antivirals, RNA silencers, viral protein export inhibitors (with pegylated interferon), and entry inhibitors (with pegylated interferon) appear to be effective in suppressing HBsAg and may even induce functional cure. The other virus-targeting agents have variable effects on HBV DNA, HBsAg, HBeAg, and HBcrAg. Immunomodulators have modest effects on HBsAg but may have important roles in combination therapy. Upcoming trials will answer important questions on ideal dosing, long-term drug effects, and efficacy of combination regimens.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"127 - 140"},"PeriodicalIF":3.4,"publicationDate":"2022-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41594487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 5

Emerging drugs for the treatment of hepatocellular carcinoma 治疗肝细胞癌的新兴药物

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-04-03 DOI: 10.1080/14728214.2022.2083107

W. Ayoub, Patricia D. Jones, J. D. Yang, Paul M. Martin

{"title":"Emerging drugs for the treatment of hepatocellular carcinoma","authors":"W. Ayoub, Patricia D. Jones, J. D. Yang, Paul M. Martin","doi":"10.1080/14728214.2022.2083107","DOIUrl":"https://doi.org/10.1080/14728214.2022.2083107","url":null,"abstract":"ABSTRACT Introduction Hepatocellular carcinoma (HCC) remains a leading cause of liver-related mortality. Cirrhosis of any etiology is the major risk factor although HCC can develop in its absence in patients with hepatitis B and increasingly in those with nonalcoholic fatty liver disease. When detected at an early stage, curative options include surgical resection, liver transplantation, and/or ablative therapies. Unfortunately, most cases of HCC are recognized at an advanced stage when options are limited and noncurative. However, new systemic therapies with tyrosine kinase inhibitors and immunotherapy have expanded therapeutic options in advanced HCC. Advances in systemic therapy have given patients with advanced HCC hope and prolonged their survival. Areas covered We discuss recent data and ongoing research efforts to improve the treatment of hepatocellular carcinoma with discussion of current and upcoming systemic therapies combining agents of different classes. Expert opinion Systemic therapy for HCC is in evolution. The inclusion of immunotherapy to systemic therapy has revolutionized the field of HCC treatment. Identification of the appropriate combination and sequence of systemic therapy coupled with discovery of reliable HCC biomarkers will lead to improved survival and individualized HCC therapy.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"141 - 149"},"PeriodicalIF":3.4,"publicationDate":"2022-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45627778","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 4

Epilepsy: expert opinion on emerging drugs in phase 2/3 clinical trials. 癫痫:专家对2/3期临床试验新兴药物的意见

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-03-01 Epub Date: 2022-04-01 DOI: 10.1080/14728214.2022.2059464

Amanda W Pong, Jonathan Ross, Ivana Tyrlikova, Alexander J Giermek, Maya P Kohli, Yousef A Khan, Roger D Salgado, Pavel Klein

{"title":"Epilepsy: expert opinion on emerging drugs in phase 2/3 clinical trials.","authors":"Amanda W Pong, Jonathan Ross, Ivana Tyrlikova, Alexander J Giermek, Maya P Kohli, Yousef A Khan, Roger D Salgado, Pavel Klein","doi":"10.1080/14728214.2022.2059464","DOIUrl":"10.1080/14728214.2022.2059464","url":null,"abstract":"Introduction: Despite the existence of over 30 anti-seizure medications (ASM), including 20 over the last 30 years, a third of patients with epilepsy remain refractory to treatment, with no disease-modifying or preventive therapies until very recently. The development of new ASMs with new mechanisms of action is therefore critical. Recent clinical trials of new treatments have shifted focus from traditional common epilepsies to rare, genetic epilepsies with known mechanistic targets for treatment and disease-specific animal models.Areas covered: ASMs in phase 2a/b-3 clinical trials target cholesterol, serotonin, sigma-1 receptors, potassium channels and metabotropic glutamate receptors. Neuroinflammation, protein misfolding, abnormal thalamocortical firing, and molecular deficiencies are among the targeted pathways. Clinically, the current phase 2a/b-3 agents hold promise for variety of epilepsy conditions, from developmental epileptic encephalopathies (Dravet Syndrome, Lennox-Gastaut syndrome, CDKL5 and PCDH19, Rett's Syndrome), infantile spasms, tuberous sclerosis as well as focal and idiopathic generalized epilepsies and acute rescue therapy for cluster seizures.Expert opinion: New delivery mechanisms increase potency and site-specificity of existing drugs. Novel mechanisms of action involve cholesterol degradation, mitochondrial pathways, anti-inflammation, and neuro-regeneration. Earlier identification of genetic conditions through genetic testing will allow for earlier use of disease specific and disease-modifying therapies.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"75-90"},"PeriodicalIF":2.7,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48611252","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Emerging FLT3 inhibitors for the treatment of acute myeloid leukemia. 新出现的FLT3抑制剂治疗急性髓性白血病。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-03-01 Epub Date: 2022-01-25 DOI: 10.1080/14728214.2021.2009800

Antonio Solana-Altabella, Octavio Ballesta-López, Juan Eduardo Megías-Vericat, David Martínez-Cuadrón, Pau Montesinos

{"title":"Emerging FLT3 inhibitors for the treatment of acute myeloid leukemia.","authors":"Antonio Solana-Altabella, Octavio Ballesta-López, Juan Eduardo Megías-Vericat, David Martínez-Cuadrón, Pau Montesinos","doi":"10.1080/14728214.2021.2009800","DOIUrl":"https://doi.org/10.1080/14728214.2021.2009800","url":null,"abstract":"Introduction: The FMS-like tyrosine kinase 3 (FLT3) gene is mutated in one-third of patients with Acute Myeloid Leukemia (AML). Midostaurin, quizartinib, and gilteritinib have been approved in the last years for the treatment of AML, and more Tyrosine Kinase Inhibitors (TKIs) targeting FLT3 are being developed such as crenolanib.Areas covered: In this systematic review, we will analyze the available clinical data on FLT3 inhibitors in development and describe the potential role that these FLT3-TKIs may play in the future management of FLT3-mutated (FLT3mut) AML.Expert opinion: Although several aspects may challenge the use of FLT3 inhibitors in AML (resistance mechanisms, on- and off-target toxicities or drug-drug interactions), these drugs are generally well tolerated, particularly if we compare their safety profile with classical chemotherapy agents or even with newer immunotherapies, thus enabling their use in fit and unfit AML patients, alone or combined. As AML is a polyclonal disease and FLT3 mutations are a late leukemogenic event, combinations of these FLT3 inhibitors with other antileukemic agents (like venetoclax or hypomethylating agents) seem a necessary research pathway.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-18"},"PeriodicalIF":3.4,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39947123","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 5