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Multidisciplinary, Specialized Care for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a progressive, autosomal recessive neuromuscular disease caused by survival motor neuron protein deficiency. SMA has four phenotypes, characterized by varying levels of severity and age of onset. Types 1 and 2 are the most common and the most deadly, affecting children from birth to age 2 years. SMA is the leading genetic cause of infant death.
In this ReCAP, neurologist Emma Ciafaloni, MD, explains how novel gene therapies for SMA disrupt the pathophysiology of the disease. Use of these agents should be left to a specialist who has expertise in monitoring progression of SMA. Dr Ciafaloni is director of the Muscular Dystrophy Association Clinic at Strong Memorial Hospital in Rochester, New York, where she leads a multidisciplinary SMA care team that includes a pulmonologist, pediatrician, physical therapist, orthopedist, and dietitian.
She recommends that pediatricians stay abreast of their state's position on newborn screening for SMA because research shows that early diagnosis and treatment can improve outcomes, including life expectancy and quality of life.
Professor of Neurology, Pediatrics, and Obstetrics and Gynecology, Department of Neurology, University of Rochester; Director, The Muscular Dystrophy Association Clinic; Program Director, Neuromuscular Medicine Fellowship, Strong Memorial Hospital, Rochester, New York
-- Emma Ciafaloni, MD, has disclosed the following relevant financial relationships:
Serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for: Biogen; AveXis; Ra Pharmaceuticals, Inc.; Wave Pharma; Sarepta Therapeutics; Viela Bio; PTC Therapeutics; Strongbridge Biopharma
Serve(d) as a speaker or a member of a speakers bureau for: Biogen
Received research grant from: Santhera Pharmaceuticals; Sarepta Therapeutics; Orphazyme; PTC Therapeutics; US Food and Drug Administration; Parent Project Muscular Dystrophy; Cure SMA; Centers for Disease Control and Prevention
Spinal muscular atrophy (SMA) is a progressive, autosomal recessive neuromuscular disease caused by survival motor neuron protein deficiency. SMA has four phenotypes, characterized by varying levels of severity and age of onset. Types 1 and 2 are the most common and the most deadly, affecting children from birth to age 2 years. SMA is the leading genetic cause of infant death.
In this ReCAP, neurologist Emma Ciafaloni, MD, explains how novel gene therapies for SMA disrupt the pathophysiology of the disease. Use of these agents should be left to a specialist who has expertise in monitoring progression of SMA. Dr Ciafaloni is director of the Muscular Dystrophy Association Clinic at Strong Memorial Hospital in Rochester, New York, where she leads a multidisciplinary SMA care team that includes a pulmonologist, pediatrician, physical therapist, orthopedist, and dietitian.
She recommends that pediatricians stay abreast of their state's position on newborn screening for SMA because research shows that early diagnosis and treatment can improve outcomes, including life expectancy and quality of life.
Professor of Neurology, Pediatrics, and Obstetrics and Gynecology, Department of Neurology, University of Rochester; Director, The Muscular Dystrophy Association Clinic; Program Director, Neuromuscular Medicine Fellowship, Strong Memorial Hospital, Rochester, New York
-- Emma Ciafaloni, MD, has disclosed the following relevant financial relationships:
Serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for: Biogen; AveXis; Ra Pharmaceuticals, Inc.; Wave Pharma; Sarepta Therapeutics; Viela Bio; PTC Therapeutics; Strongbridge Biopharma
Serve(d) as a speaker or a member of a speakers bureau for: Biogen
Received research grant from: Santhera Pharmaceuticals; Sarepta Therapeutics; Orphazyme; PTC Therapeutics; US Food and Drug Administration; Parent Project Muscular Dystrophy; Cure SMA; Centers for Disease Control and Prevention
Spinal muscular atrophy (SMA) is a progressive, autosomal recessive neuromuscular disease caused by survival motor neuron protein deficiency. SMA has four phenotypes, characterized by varying levels of severity and age of onset. Types 1 and 2 are the most common and the most deadly, affecting children from birth to age 2 years. SMA is the leading genetic cause of infant death.
In this ReCAP, neurologist Emma Ciafaloni, MD, explains how novel gene therapies for SMA disrupt the pathophysiology of the disease. Use of these agents should be left to a specialist who has expertise in monitoring progression of SMA. Dr Ciafaloni is director of the Muscular Dystrophy Association Clinic at Strong Memorial Hospital in Rochester, New York, where she leads a multidisciplinary SMA care team that includes a pulmonologist, pediatrician, physical therapist, orthopedist, and dietitian.
She recommends that pediatricians stay abreast of their state's position on newborn screening for SMA because research shows that early diagnosis and treatment can improve outcomes, including life expectancy and quality of life.
Professor of Neurology, Pediatrics, and Obstetrics and Gynecology, Department of Neurology, University of Rochester; Director, The Muscular Dystrophy Association Clinic; Program Director, Neuromuscular Medicine Fellowship, Strong Memorial Hospital, Rochester, New York
-- Emma Ciafaloni, MD, has disclosed the following relevant financial relationships:
Serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for: Biogen; AveXis; Ra Pharmaceuticals, Inc.; Wave Pharma; Sarepta Therapeutics; Viela Bio; PTC Therapeutics; Strongbridge Biopharma
Serve(d) as a speaker or a member of a speakers bureau for: Biogen
Received research grant from: Santhera Pharmaceuticals; Sarepta Therapeutics; Orphazyme; PTC Therapeutics; US Food and Drug Administration; Parent Project Muscular Dystrophy; Cure SMA; Centers for Disease Control and Prevention
Optimizing Severe Asthma Treatment: Challenges and Approaches to Care
Asthma is a complex, heterogeneous disease with unmet treatment needs. Patients with severe asthma generally continue to have severe disease despite being on controller therapies.
Uncontrolled asthma can result in unnecessary suffering and interfere with daily activities. It also increases the risk for exacerbations and places substantial burden on the healthcare system.
In this ReCAP, Drs Sandhya Khurana and Steve N. Georas, from the Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care in Rochester, New York, discuss current challenges in the management of severe asthma, and how advances in the understanding of phenotypes and therapeutic options guide their approaches to asthma management.
They review key indicators of severe asthma, tools to assess asthma control, approaches to phenotyping patients, treatment options for type 2 and non–type 2 asthma, and emerging agents.
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Sandhya Khurana, MD is a Professor, Department of Medicine, University of Rochester, Director, Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care, Rochester, New York.
Sandhya Khurana, MD, has disclosed the following relevant financial relationships: Received research grant from: GlaxoSmithKline.
Steve N. Georas, MD is a Professor, Department of Medicine, University of Rochester; Walter & Carmina Mary Parkes Family Endowed Professor; Director, Pulmonary Function Labs, Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care, Rochester, New York.
Steve N. Georas, MD, has disclosed no relevant financial relationships.
Asthma is a complex, heterogeneous disease with unmet treatment needs. Patients with severe asthma generally continue to have severe disease despite being on controller therapies.
Uncontrolled asthma can result in unnecessary suffering and interfere with daily activities. It also increases the risk for exacerbations and places substantial burden on the healthcare system.
In this ReCAP, Drs Sandhya Khurana and Steve N. Georas, from the Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care in Rochester, New York, discuss current challenges in the management of severe asthma, and how advances in the understanding of phenotypes and therapeutic options guide their approaches to asthma management.
They review key indicators of severe asthma, tools to assess asthma control, approaches to phenotyping patients, treatment options for type 2 and non–type 2 asthma, and emerging agents.
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Sandhya Khurana, MD is a Professor, Department of Medicine, University of Rochester, Director, Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care, Rochester, New York.
Sandhya Khurana, MD, has disclosed the following relevant financial relationships: Received research grant from: GlaxoSmithKline.
Steve N. Georas, MD is a Professor, Department of Medicine, University of Rochester; Walter & Carmina Mary Parkes Family Endowed Professor; Director, Pulmonary Function Labs, Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care, Rochester, New York.
Steve N. Georas, MD, has disclosed no relevant financial relationships.
Asthma is a complex, heterogeneous disease with unmet treatment needs. Patients with severe asthma generally continue to have severe disease despite being on controller therapies.
Uncontrolled asthma can result in unnecessary suffering and interfere with daily activities. It also increases the risk for exacerbations and places substantial burden on the healthcare system.
In this ReCAP, Drs Sandhya Khurana and Steve N. Georas, from the Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care in Rochester, New York, discuss current challenges in the management of severe asthma, and how advances in the understanding of phenotypes and therapeutic options guide their approaches to asthma management.
They review key indicators of severe asthma, tools to assess asthma control, approaches to phenotyping patients, treatment options for type 2 and non–type 2 asthma, and emerging agents.
--
Sandhya Khurana, MD is a Professor, Department of Medicine, University of Rochester, Director, Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care, Rochester, New York.
Sandhya Khurana, MD, has disclosed the following relevant financial relationships: Received research grant from: GlaxoSmithKline.
Steve N. Georas, MD is a Professor, Department of Medicine, University of Rochester; Walter & Carmina Mary Parkes Family Endowed Professor; Director, Pulmonary Function Labs, Mary H. Parkes Center for Asthma, Allergy, and Pulmonary Care, Rochester, New York.
Steve N. Georas, MD, has disclosed no relevant financial relationships.
Therapeutic Approaches in Advanced Breast Cancer
More than 280,000 women in the United States will be diagnosed with invasive breast cancer this year. For those with metastatic breast cancer with distant spread, the 5-year survival rate is approximately 28%. Whether advanced disease is discovered at initial diagnosis or in relapsed disease, it is imperative to understand the molecular characteristics of the metastatic tumor.
Dr Susan Domchek, from the University of Pennsylvania, discusses the importance of retesting for estrogen receptor, progesterone receptor, and HER2/neu on a metastatic tumor focus in order to identify potential discordance between the primary cancer and metastatic disease.
Additionally, Dr Domchek discusses the importance of molecular testing for targetable mutations, including P13K and germline BRCA1/2, for which approved therapies have shown survival benefit.
The list of targetable mutations in breast cancer continues to expand. In the tumor-agnostic studies, pembrolizumab has shown survival benefit in tumors that have mismatch repair deficiency and microsatellite instability, and TRK inhibitors have shown efficacy in tumors positive for NTRK fusions. Numerous clinical trials are available looking at additional molecular-based therapies.
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Susan M. Domchek, MD, Basser Professor, Department of Oncology; Executive Director, Basser Center for BRCA, Abramson Cancer Center, University of Pennsylvania, Philadelphia.
Susan M. Domchek, MD, has disclosed the following relevant financial relationships: Received income in an amount equal to or greater than $250 from: AstraZeneca; Clovis; Bristol Myers Squibb.
More than 280,000 women in the United States will be diagnosed with invasive breast cancer this year. For those with metastatic breast cancer with distant spread, the 5-year survival rate is approximately 28%. Whether advanced disease is discovered at initial diagnosis or in relapsed disease, it is imperative to understand the molecular characteristics of the metastatic tumor.
Dr Susan Domchek, from the University of Pennsylvania, discusses the importance of retesting for estrogen receptor, progesterone receptor, and HER2/neu on a metastatic tumor focus in order to identify potential discordance between the primary cancer and metastatic disease.
Additionally, Dr Domchek discusses the importance of molecular testing for targetable mutations, including P13K and germline BRCA1/2, for which approved therapies have shown survival benefit.
The list of targetable mutations in breast cancer continues to expand. In the tumor-agnostic studies, pembrolizumab has shown survival benefit in tumors that have mismatch repair deficiency and microsatellite instability, and TRK inhibitors have shown efficacy in tumors positive for NTRK fusions. Numerous clinical trials are available looking at additional molecular-based therapies.
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Susan M. Domchek, MD, Basser Professor, Department of Oncology; Executive Director, Basser Center for BRCA, Abramson Cancer Center, University of Pennsylvania, Philadelphia.
Susan M. Domchek, MD, has disclosed the following relevant financial relationships: Received income in an amount equal to or greater than $250 from: AstraZeneca; Clovis; Bristol Myers Squibb.
More than 280,000 women in the United States will be diagnosed with invasive breast cancer this year. For those with metastatic breast cancer with distant spread, the 5-year survival rate is approximately 28%. Whether advanced disease is discovered at initial diagnosis or in relapsed disease, it is imperative to understand the molecular characteristics of the metastatic tumor.
Dr Susan Domchek, from the University of Pennsylvania, discusses the importance of retesting for estrogen receptor, progesterone receptor, and HER2/neu on a metastatic tumor focus in order to identify potential discordance between the primary cancer and metastatic disease.
Additionally, Dr Domchek discusses the importance of molecular testing for targetable mutations, including P13K and germline BRCA1/2, for which approved therapies have shown survival benefit.
The list of targetable mutations in breast cancer continues to expand. In the tumor-agnostic studies, pembrolizumab has shown survival benefit in tumors that have mismatch repair deficiency and microsatellite instability, and TRK inhibitors have shown efficacy in tumors positive for NTRK fusions. Numerous clinical trials are available looking at additional molecular-based therapies.
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Susan M. Domchek, MD, Basser Professor, Department of Oncology; Executive Director, Basser Center for BRCA, Abramson Cancer Center, University of Pennsylvania, Philadelphia.
Susan M. Domchek, MD, has disclosed the following relevant financial relationships: Received income in an amount equal to or greater than $250 from: AstraZeneca; Clovis; Bristol Myers Squibb.
Tardive dyskinesia: The role of targeted therapy
Tardive dyskinesia is a movement disorder characterized by involuntary and repetitive movements of the tongue, lips, face, trunk, and extremities. It is caused by the use of dopamine receptor-blocking drugs, most often antipsychotics, but has also been associated with treatments for some gastrointestinal disorders.
Dr. Peter LeWitt, Sastry Foundation Endowed Chair in Neurology at Wayne State University School of Medicine in Detroit, MI, walks through some of the known causes of tardive dyskinesia as well as steps physicians can take in making a differential diagnosis.
Next, Dr. LeWitt dives into the role of targeted therapy in the management of tardive dyskinesia. VMAT2 (vesicular monoamine transporter type 2) inhibitors are emerging as a treatment class of choice that help suppress tardive dyskinesia symptoms by depleting presynaptic dopamine, while other patients may benefit more from benzodiazepines, botulinum toxin, or deep brain stimulation.
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Dr. Peter LeWitt, Sastry Foundation Endowed Chair in Neurology at Wayne State University School of Medicine in Detroit, MI.
Peter A. LeWitt reports advisory roles for: Abide, Acorda Therapeutics, Adamas, Biogen, Cavion, Denali, Intec Pharma, Jazz Pharmaceuticals, Lundbeck, Neurocrine, Mitsubishi NeuroDerm Ltd, Prexton, Revance, Sage, and US WorldMeds.
Lecture fees from: Acorda, American Academy of Neurology, Kyowa Hakko Kirin, and US WorldMeds.
Research grant support from: Abide, Acorda, Amneal, Lundbeck, Michael J. Fox Foundation for Parkinson's Research, Mitsubishi NeuroDerm Ltd, Parkinson Study Group; Pharma 2B, Revance, Hoffmann-La Roche; Sunovion, Sun Pharma, and US WorldMeds.
Tardive dyskinesia is a movement disorder characterized by involuntary and repetitive movements of the tongue, lips, face, trunk, and extremities. It is caused by the use of dopamine receptor-blocking drugs, most often antipsychotics, but has also been associated with treatments for some gastrointestinal disorders.
Dr. Peter LeWitt, Sastry Foundation Endowed Chair in Neurology at Wayne State University School of Medicine in Detroit, MI, walks through some of the known causes of tardive dyskinesia as well as steps physicians can take in making a differential diagnosis.
Next, Dr. LeWitt dives into the role of targeted therapy in the management of tardive dyskinesia. VMAT2 (vesicular monoamine transporter type 2) inhibitors are emerging as a treatment class of choice that help suppress tardive dyskinesia symptoms by depleting presynaptic dopamine, while other patients may benefit more from benzodiazepines, botulinum toxin, or deep brain stimulation.
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Dr. Peter LeWitt, Sastry Foundation Endowed Chair in Neurology at Wayne State University School of Medicine in Detroit, MI.
Peter A. LeWitt reports advisory roles for: Abide, Acorda Therapeutics, Adamas, Biogen, Cavion, Denali, Intec Pharma, Jazz Pharmaceuticals, Lundbeck, Neurocrine, Mitsubishi NeuroDerm Ltd, Prexton, Revance, Sage, and US WorldMeds.
Lecture fees from: Acorda, American Academy of Neurology, Kyowa Hakko Kirin, and US WorldMeds.
Research grant support from: Abide, Acorda, Amneal, Lundbeck, Michael J. Fox Foundation for Parkinson's Research, Mitsubishi NeuroDerm Ltd, Parkinson Study Group; Pharma 2B, Revance, Hoffmann-La Roche; Sunovion, Sun Pharma, and US WorldMeds.
Tardive dyskinesia is a movement disorder characterized by involuntary and repetitive movements of the tongue, lips, face, trunk, and extremities. It is caused by the use of dopamine receptor-blocking drugs, most often antipsychotics, but has also been associated with treatments for some gastrointestinal disorders.
Dr. Peter LeWitt, Sastry Foundation Endowed Chair in Neurology at Wayne State University School of Medicine in Detroit, MI, walks through some of the known causes of tardive dyskinesia as well as steps physicians can take in making a differential diagnosis.
Next, Dr. LeWitt dives into the role of targeted therapy in the management of tardive dyskinesia. VMAT2 (vesicular monoamine transporter type 2) inhibitors are emerging as a treatment class of choice that help suppress tardive dyskinesia symptoms by depleting presynaptic dopamine, while other patients may benefit more from benzodiazepines, botulinum toxin, or deep brain stimulation.
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Dr. Peter LeWitt, Sastry Foundation Endowed Chair in Neurology at Wayne State University School of Medicine in Detroit, MI.
Peter A. LeWitt reports advisory roles for: Abide, Acorda Therapeutics, Adamas, Biogen, Cavion, Denali, Intec Pharma, Jazz Pharmaceuticals, Lundbeck, Neurocrine, Mitsubishi NeuroDerm Ltd, Prexton, Revance, Sage, and US WorldMeds.
Lecture fees from: Acorda, American Academy of Neurology, Kyowa Hakko Kirin, and US WorldMeds.
Research grant support from: Abide, Acorda, Amneal, Lundbeck, Michael J. Fox Foundation for Parkinson's Research, Mitsubishi NeuroDerm Ltd, Parkinson Study Group; Pharma 2B, Revance, Hoffmann-La Roche; Sunovion, Sun Pharma, and US WorldMeds.
Common Comorbidities of Chronic Rhinosinusitis With Nasal Polyps
The majority of patients with chronic rhinosinusitis with nasal polyps (CRSwNP) have both upper and lower airway comorbidities such as asthma, allergic rhinosinusitis, and GERD. Recent data show that more than 50% of patients with CRSwNP have concurrent asthma.
Comorbidities in CRSwNP increase the risk for refractory disease, lead to repeated surgical procedures, and adversely affect patients' quality of life.
Dr Anju Peters, from Northwestern University Feinberg School of Medicine, discusses the importance of identifying comorbidities in patients with CRSwNP in order to create a more effective and personalized treatment plan.
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Anju T. Peters, MD, MSci, Professor, Department of Medicine, Division of Allergy-Immunology, Northwestern University Feinberg School of Medicine; Director of Clinical Research, Associate Chief of Research, Education, and Clinical Affairs, Division of Allergy-Immunology, Northwestern Medicine, Chicago, Illinois.
Anju T. Peters, MD, MSci, has disclosed the following relevant financial relationships:
Serve(d) on the advisory board for: Sanofi Regeneron; Optinose; AstraZeneca. Received research grant from: AstraZeneca; Optinose.
The majority of patients with chronic rhinosinusitis with nasal polyps (CRSwNP) have both upper and lower airway comorbidities such as asthma, allergic rhinosinusitis, and GERD. Recent data show that more than 50% of patients with CRSwNP have concurrent asthma.
Comorbidities in CRSwNP increase the risk for refractory disease, lead to repeated surgical procedures, and adversely affect patients' quality of life.
Dr Anju Peters, from Northwestern University Feinberg School of Medicine, discusses the importance of identifying comorbidities in patients with CRSwNP in order to create a more effective and personalized treatment plan.
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Anju T. Peters, MD, MSci, Professor, Department of Medicine, Division of Allergy-Immunology, Northwestern University Feinberg School of Medicine; Director of Clinical Research, Associate Chief of Research, Education, and Clinical Affairs, Division of Allergy-Immunology, Northwestern Medicine, Chicago, Illinois.
Anju T. Peters, MD, MSci, has disclosed the following relevant financial relationships:
Serve(d) on the advisory board for: Sanofi Regeneron; Optinose; AstraZeneca. Received research grant from: AstraZeneca; Optinose.
The majority of patients with chronic rhinosinusitis with nasal polyps (CRSwNP) have both upper and lower airway comorbidities such as asthma, allergic rhinosinusitis, and GERD. Recent data show that more than 50% of patients with CRSwNP have concurrent asthma.
Comorbidities in CRSwNP increase the risk for refractory disease, lead to repeated surgical procedures, and adversely affect patients' quality of life.
Dr Anju Peters, from Northwestern University Feinberg School of Medicine, discusses the importance of identifying comorbidities in patients with CRSwNP in order to create a more effective and personalized treatment plan.
---
Anju T. Peters, MD, MSci, Professor, Department of Medicine, Division of Allergy-Immunology, Northwestern University Feinberg School of Medicine; Director of Clinical Research, Associate Chief of Research, Education, and Clinical Affairs, Division of Allergy-Immunology, Northwestern Medicine, Chicago, Illinois.
Anju T. Peters, MD, MSci, has disclosed the following relevant financial relationships:
Serve(d) on the advisory board for: Sanofi Regeneron; Optinose; AstraZeneca. Received research grant from: AstraZeneca; Optinose.
Optimal Treatment for HR+/HER2- Breast Cancer Patients With Visceral Metastasis
In the past, chemotherapy was the go-to treatment for patients with HR+/HER2- breast cancer with visceral metastases, which most commonly involve lung and liver. Dr Adam M. Brufsky, of the University of Pittsburgh School of Medicine, reports that the advent of CDK4/6 inhibitors offers an opportunity for several different courses of action appropriate to subgroups within this population of patients.
Three CDK4/6 inhibitors are FDA approved for clinical practice. Dr Brufsky summarizes results from three studies of CDK4/6 inhibitors — abemaciclib, ribociclib, and palbociclib — where these agents were used as second-line therapy after progression on a nonsteroidal aromatase inhibitor. In combination with fulvestrant, all three offered significant benefits in progression-free survival (PFS) and two in overall survival (OS). Among those who derived survival benefit were patients who had visceral metatases, who constituted 56%, 27% and 60% of the respective studies.
For patients who show progression on CDK4/6 inhibitors, additional treatment can be determined by genomic sequencing. Combination therapy with alpelisib and fulvestrant has been shown to lengthen PFS in patients whose tumors have PIK3 mutations. Patients without PIK3 mutations can be treated with fulvestrant or a combination of fulvestrant and everolimus.
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Adam M. Brufsky, MD, PhD, Professor, Department of Medicine, Division of Hematology-Oncology, University of Pittsburgh School of Medicine; Associate Chief, Department of Medicine, Division of Hematology-Oncology, UPMC-Hillman Cancer Center, Pittsburgh, Pennsylvania.
Adam M. Brufsky, MD, PhD, has disclosed the following relevant financial relationships:
Received income in an amount equal to or greater than $250 from: Seattle Genetics; Roche; AstraZeneca; Puma; Daiichi Sankyo.
In the past, chemotherapy was the go-to treatment for patients with HR+/HER2- breast cancer with visceral metastases, which most commonly involve lung and liver. Dr Adam M. Brufsky, of the University of Pittsburgh School of Medicine, reports that the advent of CDK4/6 inhibitors offers an opportunity for several different courses of action appropriate to subgroups within this population of patients.
Three CDK4/6 inhibitors are FDA approved for clinical practice. Dr Brufsky summarizes results from three studies of CDK4/6 inhibitors — abemaciclib, ribociclib, and palbociclib — where these agents were used as second-line therapy after progression on a nonsteroidal aromatase inhibitor. In combination with fulvestrant, all three offered significant benefits in progression-free survival (PFS) and two in overall survival (OS). Among those who derived survival benefit were patients who had visceral metatases, who constituted 56%, 27% and 60% of the respective studies.
For patients who show progression on CDK4/6 inhibitors, additional treatment can be determined by genomic sequencing. Combination therapy with alpelisib and fulvestrant has been shown to lengthen PFS in patients whose tumors have PIK3 mutations. Patients without PIK3 mutations can be treated with fulvestrant or a combination of fulvestrant and everolimus.
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Adam M. Brufsky, MD, PhD, Professor, Department of Medicine, Division of Hematology-Oncology, University of Pittsburgh School of Medicine; Associate Chief, Department of Medicine, Division of Hematology-Oncology, UPMC-Hillman Cancer Center, Pittsburgh, Pennsylvania.
Adam M. Brufsky, MD, PhD, has disclosed the following relevant financial relationships:
Received income in an amount equal to or greater than $250 from: Seattle Genetics; Roche; AstraZeneca; Puma; Daiichi Sankyo.
In the past, chemotherapy was the go-to treatment for patients with HR+/HER2- breast cancer with visceral metastases, which most commonly involve lung and liver. Dr Adam M. Brufsky, of the University of Pittsburgh School of Medicine, reports that the advent of CDK4/6 inhibitors offers an opportunity for several different courses of action appropriate to subgroups within this population of patients.
Three CDK4/6 inhibitors are FDA approved for clinical practice. Dr Brufsky summarizes results from three studies of CDK4/6 inhibitors — abemaciclib, ribociclib, and palbociclib — where these agents were used as second-line therapy after progression on a nonsteroidal aromatase inhibitor. In combination with fulvestrant, all three offered significant benefits in progression-free survival (PFS) and two in overall survival (OS). Among those who derived survival benefit were patients who had visceral metatases, who constituted 56%, 27% and 60% of the respective studies.
For patients who show progression on CDK4/6 inhibitors, additional treatment can be determined by genomic sequencing. Combination therapy with alpelisib and fulvestrant has been shown to lengthen PFS in patients whose tumors have PIK3 mutations. Patients without PIK3 mutations can be treated with fulvestrant or a combination of fulvestrant and everolimus.
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Adam M. Brufsky, MD, PhD, Professor, Department of Medicine, Division of Hematology-Oncology, University of Pittsburgh School of Medicine; Associate Chief, Department of Medicine, Division of Hematology-Oncology, UPMC-Hillman Cancer Center, Pittsburgh, Pennsylvania.
Adam M. Brufsky, MD, PhD, has disclosed the following relevant financial relationships:
Received income in an amount equal to or greater than $250 from: Seattle Genetics; Roche; AstraZeneca; Puma; Daiichi Sankyo.
Bridging the Gap: Multidisciplinary Management of NSCLC
Numerous advances in diagnosis, staging, and treatment have evolved over the past several years for non-small cell lung cancer (NSCLC) patients. Because of the new developments, input from a team of specialists is required to treat each patient. Many hospitals have implemented a multidisciplinary team approach to treat these complex cases.
In this ReCAP, Dr Nicole Tanner, a pulmonary critical care specialist from the Medical University of South Carolina, hosts thoracic oncologist Dr Carrie Lee and thoracic surgeon Dr Jason Long, colleagues from the UNC Lineberger Comprehensive Cancer Center, in a discussion of the advantages of multidisciplinary teams in the treatment of NSCLC patients.
Their discussion ranges from identifying key multidisciplinary team members, individual practitioner responsibilities, benefits of multidisciplinary teams to patients and clinicians alike, and the advantages of incorporating telehealth visits into standard practice after the pandemic.
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Jason M. Long, MD, MPH, FCCP, Associate Professor, Department of Surgery, University of North Carolina School of Medicine; Staff Physician, Department of Surgery, UNC Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina
Jason M. Long, MD, MPH, FCCP, has disclosed no relevant financial relationships.
Carrie B. Lee, MD, MPH, Associate Professor, Department of Medicine, University of North Carolina, Chapel Hill; Medical Director, Clinical Protocol Office, UNC Lineberger Comprehensive Cancer Center, North Carolina Cancer Hospital, Chapel Hill, North Carolina
Carrie B. Lee, MD, MPH, has disclosed the following relevant financial relationships:
Serve(d) as the chair of the Data and Safety Monitoring Board for: Delcath, Inc.
Nichole T. Tanner, MD, MSCR, FCCP, Associate Professor, Department of Medicine, Medical University of South Carolina, Charleston, South Carolina
Nichole T. Tanner, MD, MSCR, FCCP, has disclosed no relevant financial relationships.
Numerous advances in diagnosis, staging, and treatment have evolved over the past several years for non-small cell lung cancer (NSCLC) patients. Because of the new developments, input from a team of specialists is required to treat each patient. Many hospitals have implemented a multidisciplinary team approach to treat these complex cases.
In this ReCAP, Dr Nicole Tanner, a pulmonary critical care specialist from the Medical University of South Carolina, hosts thoracic oncologist Dr Carrie Lee and thoracic surgeon Dr Jason Long, colleagues from the UNC Lineberger Comprehensive Cancer Center, in a discussion of the advantages of multidisciplinary teams in the treatment of NSCLC patients.
Their discussion ranges from identifying key multidisciplinary team members, individual practitioner responsibilities, benefits of multidisciplinary teams to patients and clinicians alike, and the advantages of incorporating telehealth visits into standard practice after the pandemic.
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Jason M. Long, MD, MPH, FCCP, Associate Professor, Department of Surgery, University of North Carolina School of Medicine; Staff Physician, Department of Surgery, UNC Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina
Jason M. Long, MD, MPH, FCCP, has disclosed no relevant financial relationships.
Carrie B. Lee, MD, MPH, Associate Professor, Department of Medicine, University of North Carolina, Chapel Hill; Medical Director, Clinical Protocol Office, UNC Lineberger Comprehensive Cancer Center, North Carolina Cancer Hospital, Chapel Hill, North Carolina
Carrie B. Lee, MD, MPH, has disclosed the following relevant financial relationships:
Serve(d) as the chair of the Data and Safety Monitoring Board for: Delcath, Inc.
Nichole T. Tanner, MD, MSCR, FCCP, Associate Professor, Department of Medicine, Medical University of South Carolina, Charleston, South Carolina
Nichole T. Tanner, MD, MSCR, FCCP, has disclosed no relevant financial relationships.
Numerous advances in diagnosis, staging, and treatment have evolved over the past several years for non-small cell lung cancer (NSCLC) patients. Because of the new developments, input from a team of specialists is required to treat each patient. Many hospitals have implemented a multidisciplinary team approach to treat these complex cases.
In this ReCAP, Dr Nicole Tanner, a pulmonary critical care specialist from the Medical University of South Carolina, hosts thoracic oncologist Dr Carrie Lee and thoracic surgeon Dr Jason Long, colleagues from the UNC Lineberger Comprehensive Cancer Center, in a discussion of the advantages of multidisciplinary teams in the treatment of NSCLC patients.
Their discussion ranges from identifying key multidisciplinary team members, individual practitioner responsibilities, benefits of multidisciplinary teams to patients and clinicians alike, and the advantages of incorporating telehealth visits into standard practice after the pandemic.
--
Jason M. Long, MD, MPH, FCCP, Associate Professor, Department of Surgery, University of North Carolina School of Medicine; Staff Physician, Department of Surgery, UNC Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina
Jason M. Long, MD, MPH, FCCP, has disclosed no relevant financial relationships.
Carrie B. Lee, MD, MPH, Associate Professor, Department of Medicine, University of North Carolina, Chapel Hill; Medical Director, Clinical Protocol Office, UNC Lineberger Comprehensive Cancer Center, North Carolina Cancer Hospital, Chapel Hill, North Carolina
Carrie B. Lee, MD, MPH, has disclosed the following relevant financial relationships:
Serve(d) as the chair of the Data and Safety Monitoring Board for: Delcath, Inc.
Nichole T. Tanner, MD, MSCR, FCCP, Associate Professor, Department of Medicine, Medical University of South Carolina, Charleston, South Carolina
Nichole T. Tanner, MD, MSCR, FCCP, has disclosed no relevant financial relationships.
Combination Therapy for Severe Asthma
Patients with severe asthma often experience symptoms and exacerbations that can interfere with daily life and further compromise lung function.
These patients often need combination therapy to achieve optimal control. This typically includes a low-dose inhaled corticosteroid (ICS) plus a long-acting beta-agonist (LABA).
For some patients, however, adherence to these therapies will not result in optimal outcomes.
Dr Monica Kraft, of the University of Arizona Health Sciences Center, discusses additional therapeutic options for these patients, which include increasing the dose of ICS or adding an oral corticosteroid such as prednisone or methylprednisolone.
When treatment-adherent patients are still unable to maintain control of their asthma symptoms, it may be optimal to move to biologic therapy.
There are currently five available biologics that work against IgE, IL-4/IL-13, IL-5, and IL-5R. Biomarker testing for blood eosinophils, exhaled nitric oxide, and serum IgE can help determine which biologic is best suited to each individual patient.
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Robert and Irene Flinn Professor, Department of Medicine, Banner University Medical Center, North Campus; Chair, Department of Medicine, University of Arizona Health Sciences Center, Tucson, Arizona.
Monica Kraft, MD, has disclosed the following relevant financial relationships:
Serve(d) as a consultant for: AstraZeneca; Genentech; Chiesi; Sanofi
Serve(d) as Chief Medical Officer for: RaeSedo, LLC
Received research grant from: National Institutes of Health; American Lung Association; Sanofi; AstraZeneca; Chiesi
received income in an amount equal to or greater than $250 from: AstraZeneca; Genentech; Chiesi; Sanof.
Patients with severe asthma often experience symptoms and exacerbations that can interfere with daily life and further compromise lung function.
These patients often need combination therapy to achieve optimal control. This typically includes a low-dose inhaled corticosteroid (ICS) plus a long-acting beta-agonist (LABA).
For some patients, however, adherence to these therapies will not result in optimal outcomes.
Dr Monica Kraft, of the University of Arizona Health Sciences Center, discusses additional therapeutic options for these patients, which include increasing the dose of ICS or adding an oral corticosteroid such as prednisone or methylprednisolone.
When treatment-adherent patients are still unable to maintain control of their asthma symptoms, it may be optimal to move to biologic therapy.
There are currently five available biologics that work against IgE, IL-4/IL-13, IL-5, and IL-5R. Biomarker testing for blood eosinophils, exhaled nitric oxide, and serum IgE can help determine which biologic is best suited to each individual patient.
--
Robert and Irene Flinn Professor, Department of Medicine, Banner University Medical Center, North Campus; Chair, Department of Medicine, University of Arizona Health Sciences Center, Tucson, Arizona.
Monica Kraft, MD, has disclosed the following relevant financial relationships:
Serve(d) as a consultant for: AstraZeneca; Genentech; Chiesi; Sanofi
Serve(d) as Chief Medical Officer for: RaeSedo, LLC
Received research grant from: National Institutes of Health; American Lung Association; Sanofi; AstraZeneca; Chiesi
received income in an amount equal to or greater than $250 from: AstraZeneca; Genentech; Chiesi; Sanof.
Patients with severe asthma often experience symptoms and exacerbations that can interfere with daily life and further compromise lung function.
These patients often need combination therapy to achieve optimal control. This typically includes a low-dose inhaled corticosteroid (ICS) plus a long-acting beta-agonist (LABA).
For some patients, however, adherence to these therapies will not result in optimal outcomes.
Dr Monica Kraft, of the University of Arizona Health Sciences Center, discusses additional therapeutic options for these patients, which include increasing the dose of ICS or adding an oral corticosteroid such as prednisone or methylprednisolone.
When treatment-adherent patients are still unable to maintain control of their asthma symptoms, it may be optimal to move to biologic therapy.
There are currently five available biologics that work against IgE, IL-4/IL-13, IL-5, and IL-5R. Biomarker testing for blood eosinophils, exhaled nitric oxide, and serum IgE can help determine which biologic is best suited to each individual patient.
--
Robert and Irene Flinn Professor, Department of Medicine, Banner University Medical Center, North Campus; Chair, Department of Medicine, University of Arizona Health Sciences Center, Tucson, Arizona.
Monica Kraft, MD, has disclosed the following relevant financial relationships:
Serve(d) as a consultant for: AstraZeneca; Genentech; Chiesi; Sanofi
Serve(d) as Chief Medical Officer for: RaeSedo, LLC
Received research grant from: National Institutes of Health; American Lung Association; Sanofi; AstraZeneca; Chiesi
received income in an amount equal to or greater than $250 from: AstraZeneca; Genentech; Chiesi; Sanof.
Chronic Rhinosinusitis With Nasal Polyposis: Type 2 Inflammation
Medical management of chronic rhinosinusitis with nasal polyposis (CRSwNP) typically has focused on controlling tissue inflammation. Approximately 85% of patients will exhibit a type 2 inflammatory pattern, and recent studies have examined the implications of this process for the treatment of CRSwNP.
As in other respiratory diseases, emerging CRSwNP endotypes are helping researchers identify actionable targets and develop targeted biologic treatments. In fact, therapies targeting cytokines IL-4 and IL-13, which contribute to type 2 inflammation in CRSwNP, recently have been approved by the FDA.
Dr Stella Lee from the University of Pittsburgh discusses the use of new biologic therapies in clinical practice as well as strategies to test for potential drivers of type 2 inflammation.
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Stella Lee, MD, Chief, Sinonasal Disorder and Allergy; Assistant Professor, Otolaryngology, Department of Head & Neck Surgery, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania .
Stella Lee, MD, has disclosed the following relevant financial relationships:
Received research grant from: sanofi-aventis; Regeneron Pharmaceuticals Inc; GlaxoSmithKline; AstraZeneca Pharmaceuticals LP; Genentech.
Medical management of chronic rhinosinusitis with nasal polyposis (CRSwNP) typically has focused on controlling tissue inflammation. Approximately 85% of patients will exhibit a type 2 inflammatory pattern, and recent studies have examined the implications of this process for the treatment of CRSwNP.
As in other respiratory diseases, emerging CRSwNP endotypes are helping researchers identify actionable targets and develop targeted biologic treatments. In fact, therapies targeting cytokines IL-4 and IL-13, which contribute to type 2 inflammation in CRSwNP, recently have been approved by the FDA.
Dr Stella Lee from the University of Pittsburgh discusses the use of new biologic therapies in clinical practice as well as strategies to test for potential drivers of type 2 inflammation.
---
Stella Lee, MD, Chief, Sinonasal Disorder and Allergy; Assistant Professor, Otolaryngology, Department of Head & Neck Surgery, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania .
Stella Lee, MD, has disclosed the following relevant financial relationships:
Received research grant from: sanofi-aventis; Regeneron Pharmaceuticals Inc; GlaxoSmithKline; AstraZeneca Pharmaceuticals LP; Genentech.
Medical management of chronic rhinosinusitis with nasal polyposis (CRSwNP) typically has focused on controlling tissue inflammation. Approximately 85% of patients will exhibit a type 2 inflammatory pattern, and recent studies have examined the implications of this process for the treatment of CRSwNP.
As in other respiratory diseases, emerging CRSwNP endotypes are helping researchers identify actionable targets and develop targeted biologic treatments. In fact, therapies targeting cytokines IL-4 and IL-13, which contribute to type 2 inflammation in CRSwNP, recently have been approved by the FDA.
Dr Stella Lee from the University of Pittsburgh discusses the use of new biologic therapies in clinical practice as well as strategies to test for potential drivers of type 2 inflammation.
---
Stella Lee, MD, Chief, Sinonasal Disorder and Allergy; Assistant Professor, Otolaryngology, Department of Head & Neck Surgery, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania .
Stella Lee, MD, has disclosed the following relevant financial relationships:
Received research grant from: sanofi-aventis; Regeneron Pharmaceuticals Inc; GlaxoSmithKline; AstraZeneca Pharmaceuticals LP; Genentech.
Targeted Treatment in Chronic Rhinosinusitis With Nasal Polyps
Treatments in chronic rhinosinusitis with nasal polyps (CRSwNP) focus on reducing chronic type 2 inflammation. When intranasal corticosteroids, nasal saline irrigation, antibiotics, and oral steroids fail, patients move to surgery. Unfortunately, 40% of patients experience polyp regrowth following surgery.
New research in biologic therapy is showing promise. Inflammatory mediators IL-4, IL-5, IL-13, and IgE play key roles in CRSwNP. Targeting these specific inflammatory pathways may provide effective treatment options for refractory CRSwNP patients.
Dr Anju Peters discusses dupilumab, a biologic currently approved for CRSwNP, as well as other targeted biologics in development.
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Anju T. Peters, MD, MSci, Professor, Department of Medicine, Division of Allergy-Immunology, Northwestern University Feinberg School of Medicine; Director of Clinical Research, Associate Chief of Research, Education, and Clinical Affairs, Division of Allergy-Immunology, Northwestern Medicine, Chicago, Illinois.
Anju T. Peters, MD, MSci, has disclosed the following relevant financial relationships:
Serve(d) on the advisory board for: Sanofi Regeneron; Optinose; AstraZeneca. Received research grant from: AstraZeneca; Optinose.
Treatments in chronic rhinosinusitis with nasal polyps (CRSwNP) focus on reducing chronic type 2 inflammation. When intranasal corticosteroids, nasal saline irrigation, antibiotics, and oral steroids fail, patients move to surgery. Unfortunately, 40% of patients experience polyp regrowth following surgery.
New research in biologic therapy is showing promise. Inflammatory mediators IL-4, IL-5, IL-13, and IgE play key roles in CRSwNP. Targeting these specific inflammatory pathways may provide effective treatment options for refractory CRSwNP patients.
Dr Anju Peters discusses dupilumab, a biologic currently approved for CRSwNP, as well as other targeted biologics in development.
---
Anju T. Peters, MD, MSci, Professor, Department of Medicine, Division of Allergy-Immunology, Northwestern University Feinberg School of Medicine; Director of Clinical Research, Associate Chief of Research, Education, and Clinical Affairs, Division of Allergy-Immunology, Northwestern Medicine, Chicago, Illinois.
Anju T. Peters, MD, MSci, has disclosed the following relevant financial relationships:
Serve(d) on the advisory board for: Sanofi Regeneron; Optinose; AstraZeneca. Received research grant from: AstraZeneca; Optinose.
Treatments in chronic rhinosinusitis with nasal polyps (CRSwNP) focus on reducing chronic type 2 inflammation. When intranasal corticosteroids, nasal saline irrigation, antibiotics, and oral steroids fail, patients move to surgery. Unfortunately, 40% of patients experience polyp regrowth following surgery.
New research in biologic therapy is showing promise. Inflammatory mediators IL-4, IL-5, IL-13, and IgE play key roles in CRSwNP. Targeting these specific inflammatory pathways may provide effective treatment options for refractory CRSwNP patients.
Dr Anju Peters discusses dupilumab, a biologic currently approved for CRSwNP, as well as other targeted biologics in development.
---
Anju T. Peters, MD, MSci, Professor, Department of Medicine, Division of Allergy-Immunology, Northwestern University Feinberg School of Medicine; Director of Clinical Research, Associate Chief of Research, Education, and Clinical Affairs, Division of Allergy-Immunology, Northwestern Medicine, Chicago, Illinois.
Anju T. Peters, MD, MSci, has disclosed the following relevant financial relationships:
Serve(d) on the advisory board for: Sanofi Regeneron; Optinose; AstraZeneca. Received research grant from: AstraZeneca; Optinose.