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Burnout Is Rampant, But Oncologists Can Turn the Tide
SAN DIEGO—Before the pandemic, an estimated one-third of oncologists worldwide suffered a high level of burnout. Cancer physicians face many of the same risk factors as their colleagues—high workloads, lack of autonomy, and no support—along with the added pressure of working in a medical field where patients often die. Then COVID-19 hit, and the burnout crisis got even worse.
This tide can be reversed with a focus on best practices and resilience, a mental health researcher told cancer professionals at the September 2022 annual meeting of the Association of VA Hematology/Oncology. Assessments, long-term interventions, and communication are all key, said Fay J. Hlubocky, PhD, MA, a clinical health psychologist and ethicist at the University of Chicago.
Even simple actions like taking time for “mindful moments” and checking in with a colleague can make a difference, she said. But institutions must act, she said. “Long-term tailored strategies are incredibly important to promote well-being.”
Hlubocky, who led an American Society of Clinical Oncology committee on burnout prior to the pandemic, noted that statistics about burnout in American medicine and oncology specifically, are grim. In 2017, a systematic review and meta-analysis found that significant numbers of oncologists suffered from high burnout (32%), high psychiatric morbidity (27%), depression (at least 12%), and alcohol misuse (as many as 30%).
The pandemic piled on more stressors. In the second half of 2020, researchers interviewed 25 American oncologists in focus groups and found that their “underlying oncologist burnout exacerbated stressors associated with disruptions in care, education, research, financial practice health, and telemedicine. Many feared delays in cancer screening, diagnosis, and treatment [and] strongly considered working part-time or taking early retirement.”
As one participant put it, “everyone is seeing a lot of death and heartache and social isolation and anger that they’re not used to encountering and in very new and different ways.”
Major contributors to oncologist burnout, Hlubocky said, include moral distress, moral injury, and compassion fatigue. “Moral distress occurs when that individual believes he or she knows the right thing to do, but institutional constraints make it really difficult to do what is right,” Hlubocky said. “The individual is aware of the moral problem, acknowledges and takes moral responsibility, makes some moral judgments, but yet—as a result of these constraints — participates in perceived moral wrongdoing.”
Moral injury refers to the damage that can be caused by moral distress or by witnessing acts that violate morals, such as during military service. Compassion fatigue, meanwhile, is defined by the American Stress Institute as “a low level, chronic clouding of caring and concern for others in your life.”
What can be done? Hlubocky highlighted multiple interventions, such as adjustment of work patterns, cognitive behavioral therapy, and training in mindfulness, relaxation, and communication. One strategy is to adopt multiple in-person interventions simultaneously.
But first it’s crucial for administrators to understand the problem in a specific workplace: “You have to know what’s going on in your organization to intervene on it,” she said. “There are multiple tools that have been validated in other health care fields and can be used on a regular basis over time to measure burnout, satisfaction, and engagement.”
For individuals, other strategies include daily check-ins with colleagues to catch signs of stress, she said, as Toronto oncologists started doing amid the pandemic. The check-ins can include simple questions like: How are you doing? How are you feeling? Are you sleeping, eating and exercising? Do you need help?
As for resilience, Hlubocky said it must grow at the individual level. “We can't rely so much on the organization. We need to develop our personal resilience in order for professional resilience to flourish again, and we have to do a lot to protect ourselves. It’s about focusing on the strength of the individual—that empowerment to rise above adversity, that vitality, that engagement, that self-efficacy. It supports health and enhances coping, and it is the key element of physician and clinician well-being.”
Research into resilience offers guidance about how to achieve it, she said. A 2013 German study of 200 physicians found that the most resilient physicians change their attitudes and behaviors, take time off, set boundaries, spend time with family and friends, and ask colleagues for help. And they gained resilience, the study found, by getting older and becoming more experienced.
Hlubocky pointed to several useful resources for burned-out medical professionals, including mindfulness, cognitive behavioral therapy and breathing apps: She highlighted Breathe2Relax, Headspace, MoodGYM, Stress Gym, and guided audio files from the University of California at San Diego. And she said ASCO has resources on combatting burnout and promoting well-being.
Hlubocky has no relevant disclosures.
SAN DIEGO—Before the pandemic, an estimated one-third of oncologists worldwide suffered a high level of burnout. Cancer physicians face many of the same risk factors as their colleagues—high workloads, lack of autonomy, and no support—along with the added pressure of working in a medical field where patients often die. Then COVID-19 hit, and the burnout crisis got even worse.
This tide can be reversed with a focus on best practices and resilience, a mental health researcher told cancer professionals at the September 2022 annual meeting of the Association of VA Hematology/Oncology. Assessments, long-term interventions, and communication are all key, said Fay J. Hlubocky, PhD, MA, a clinical health psychologist and ethicist at the University of Chicago.
Even simple actions like taking time for “mindful moments” and checking in with a colleague can make a difference, she said. But institutions must act, she said. “Long-term tailored strategies are incredibly important to promote well-being.”
Hlubocky, who led an American Society of Clinical Oncology committee on burnout prior to the pandemic, noted that statistics about burnout in American medicine and oncology specifically, are grim. In 2017, a systematic review and meta-analysis found that significant numbers of oncologists suffered from high burnout (32%), high psychiatric morbidity (27%), depression (at least 12%), and alcohol misuse (as many as 30%).
The pandemic piled on more stressors. In the second half of 2020, researchers interviewed 25 American oncologists in focus groups and found that their “underlying oncologist burnout exacerbated stressors associated with disruptions in care, education, research, financial practice health, and telemedicine. Many feared delays in cancer screening, diagnosis, and treatment [and] strongly considered working part-time or taking early retirement.”
As one participant put it, “everyone is seeing a lot of death and heartache and social isolation and anger that they’re not used to encountering and in very new and different ways.”
Major contributors to oncologist burnout, Hlubocky said, include moral distress, moral injury, and compassion fatigue. “Moral distress occurs when that individual believes he or she knows the right thing to do, but institutional constraints make it really difficult to do what is right,” Hlubocky said. “The individual is aware of the moral problem, acknowledges and takes moral responsibility, makes some moral judgments, but yet—as a result of these constraints — participates in perceived moral wrongdoing.”
Moral injury refers to the damage that can be caused by moral distress or by witnessing acts that violate morals, such as during military service. Compassion fatigue, meanwhile, is defined by the American Stress Institute as “a low level, chronic clouding of caring and concern for others in your life.”
What can be done? Hlubocky highlighted multiple interventions, such as adjustment of work patterns, cognitive behavioral therapy, and training in mindfulness, relaxation, and communication. One strategy is to adopt multiple in-person interventions simultaneously.
But first it’s crucial for administrators to understand the problem in a specific workplace: “You have to know what’s going on in your organization to intervene on it,” she said. “There are multiple tools that have been validated in other health care fields and can be used on a regular basis over time to measure burnout, satisfaction, and engagement.”
For individuals, other strategies include daily check-ins with colleagues to catch signs of stress, she said, as Toronto oncologists started doing amid the pandemic. The check-ins can include simple questions like: How are you doing? How are you feeling? Are you sleeping, eating and exercising? Do you need help?
As for resilience, Hlubocky said it must grow at the individual level. “We can't rely so much on the organization. We need to develop our personal resilience in order for professional resilience to flourish again, and we have to do a lot to protect ourselves. It’s about focusing on the strength of the individual—that empowerment to rise above adversity, that vitality, that engagement, that self-efficacy. It supports health and enhances coping, and it is the key element of physician and clinician well-being.”
Research into resilience offers guidance about how to achieve it, she said. A 2013 German study of 200 physicians found that the most resilient physicians change their attitudes and behaviors, take time off, set boundaries, spend time with family and friends, and ask colleagues for help. And they gained resilience, the study found, by getting older and becoming more experienced.
Hlubocky pointed to several useful resources for burned-out medical professionals, including mindfulness, cognitive behavioral therapy and breathing apps: She highlighted Breathe2Relax, Headspace, MoodGYM, Stress Gym, and guided audio files from the University of California at San Diego. And she said ASCO has resources on combatting burnout and promoting well-being.
Hlubocky has no relevant disclosures.
SAN DIEGO—Before the pandemic, an estimated one-third of oncologists worldwide suffered a high level of burnout. Cancer physicians face many of the same risk factors as their colleagues—high workloads, lack of autonomy, and no support—along with the added pressure of working in a medical field where patients often die. Then COVID-19 hit, and the burnout crisis got even worse.
This tide can be reversed with a focus on best practices and resilience, a mental health researcher told cancer professionals at the September 2022 annual meeting of the Association of VA Hematology/Oncology. Assessments, long-term interventions, and communication are all key, said Fay J. Hlubocky, PhD, MA, a clinical health psychologist and ethicist at the University of Chicago.
Even simple actions like taking time for “mindful moments” and checking in with a colleague can make a difference, she said. But institutions must act, she said. “Long-term tailored strategies are incredibly important to promote well-being.”
Hlubocky, who led an American Society of Clinical Oncology committee on burnout prior to the pandemic, noted that statistics about burnout in American medicine and oncology specifically, are grim. In 2017, a systematic review and meta-analysis found that significant numbers of oncologists suffered from high burnout (32%), high psychiatric morbidity (27%), depression (at least 12%), and alcohol misuse (as many as 30%).
The pandemic piled on more stressors. In the second half of 2020, researchers interviewed 25 American oncologists in focus groups and found that their “underlying oncologist burnout exacerbated stressors associated with disruptions in care, education, research, financial practice health, and telemedicine. Many feared delays in cancer screening, diagnosis, and treatment [and] strongly considered working part-time or taking early retirement.”
As one participant put it, “everyone is seeing a lot of death and heartache and social isolation and anger that they’re not used to encountering and in very new and different ways.”
Major contributors to oncologist burnout, Hlubocky said, include moral distress, moral injury, and compassion fatigue. “Moral distress occurs when that individual believes he or she knows the right thing to do, but institutional constraints make it really difficult to do what is right,” Hlubocky said. “The individual is aware of the moral problem, acknowledges and takes moral responsibility, makes some moral judgments, but yet—as a result of these constraints — participates in perceived moral wrongdoing.”
Moral injury refers to the damage that can be caused by moral distress or by witnessing acts that violate morals, such as during military service. Compassion fatigue, meanwhile, is defined by the American Stress Institute as “a low level, chronic clouding of caring and concern for others in your life.”
What can be done? Hlubocky highlighted multiple interventions, such as adjustment of work patterns, cognitive behavioral therapy, and training in mindfulness, relaxation, and communication. One strategy is to adopt multiple in-person interventions simultaneously.
But first it’s crucial for administrators to understand the problem in a specific workplace: “You have to know what’s going on in your organization to intervene on it,” she said. “There are multiple tools that have been validated in other health care fields and can be used on a regular basis over time to measure burnout, satisfaction, and engagement.”
For individuals, other strategies include daily check-ins with colleagues to catch signs of stress, she said, as Toronto oncologists started doing amid the pandemic. The check-ins can include simple questions like: How are you doing? How are you feeling? Are you sleeping, eating and exercising? Do you need help?
As for resilience, Hlubocky said it must grow at the individual level. “We can't rely so much on the organization. We need to develop our personal resilience in order for professional resilience to flourish again, and we have to do a lot to protect ourselves. It’s about focusing on the strength of the individual—that empowerment to rise above adversity, that vitality, that engagement, that self-efficacy. It supports health and enhances coping, and it is the key element of physician and clinician well-being.”
Research into resilience offers guidance about how to achieve it, she said. A 2013 German study of 200 physicians found that the most resilient physicians change their attitudes and behaviors, take time off, set boundaries, spend time with family and friends, and ask colleagues for help. And they gained resilience, the study found, by getting older and becoming more experienced.
Hlubocky pointed to several useful resources for burned-out medical professionals, including mindfulness, cognitive behavioral therapy and breathing apps: She highlighted Breathe2Relax, Headspace, MoodGYM, Stress Gym, and guided audio files from the University of California at San Diego. And she said ASCO has resources on combatting burnout and promoting well-being.
Hlubocky has no relevant disclosures.
Worldwide trial seeks to revolutionize pediatric leukemia care
While great strides have been made in children’s leukemia care during the past 50 years, statistics have remained grim. For acute myeloid leukemia (AML), the most common type, 5-year survival rates were just 69% for children younger than 15 between 2009 and 2015. Patients who do survive past adolescence face high risks of future complications.
Specialists say the challenges hindering more progress include a lack of clinical research, an emphasis on competition over cooperation, and sparse insight into how best to adjust adult leukemia treatments to children.
“Our project aims to find better treatments, more targeted treatments, that will leave children with fewer long-term health problems as adults. We want them to not just survive but thrive,” Gwen Nichols, MD, chief medical officer of LLS, said in an interview. “What we’ve had was not working for anybody. So we have to try a different approach.”
The LLS Pediatric Acute Leukemia (PedAL) Master Trial launched in spring of 2022. Seventy-five study locations from Nova Scotia to Hawaii are now recruiting patients up to age 22 with known or suspected relapsed/refractory AML, mixed phenotype acute leukemia, or relapsed acute lymphoblastic leukemia (ALL).
The 5-year trial expects to recruit 960 participants in the United States and Canada. Clinics in Europe, Australia, and New Zealand also are taking part.
“Pediatric oncologists should know that PedAL, for the first time, is providing a cooperative, seamless way to interrogate [the genomics of] a child’s leukemia,” hematologist/oncologist Todd Cooper, DO, section chief of pediatric oncology at Seattle Children’s Cancer and Blood Disorders Center, said in an interview. “It is also providing a seamless and efficient way for children to be assigned to clinical trials that are going to be tailored towards a particular child’s leukemia. This is something that’s never been done.”
In North America, all trial participants with relapsed AML will undergo genetic sequencing for free as part of the screening process. Clinics “can’t always access genomic screening for their patients,” Dr. Nichols said. “We’re providing that even if they don’t participate in any other part of the trial, even if they go and get another available therapy or go on a different trial. We want them to know that this is available, and they will get the results. And if they’re looking for a trial when they get those results, we have trained oncology nurses who will help them navigate and find clinical trials.”
In PedAL itself, one subtrial is now in progress: An open-label phase 3 randomized multicenter analysis of whether the oral leukemia drug venetoclax combined with the intensive infused chemotherapy treatment FLA+GO (fludarabine, high-dose cytarabine, and gemtuzumab ozogamicin) will improve overall survival compared to FLA+GO alone. Ninety-eight subjects are expected to join the 5-year subtrial.
“We expect within the next year to open three or four different subtrials of targeted therapies for specific groups of patients,” E. Anders Kolb, MD, chief of oncology and hematology at Nemours Children’s Health in Delaware and cochair of the PedAL trial, said in an interview. “Over the course of the next few years, we’re going to learn a lot about the natural history of relapsed leukemia – we don’t have a ton of data on that – and then how targeted therapies may alter some of those outcomes.”
Discussions with multiple drugmakers are in progress regarding the potential subtrials, he said.
The PedAL strategy addresses the lack of new drugs for children with AML, Seattle Children’s Dr. Cooper said. One main reason for the gap is that childhood leukemia is much less common than the adult form, he said, so a lot of drug development is geared toward adults. As a result, he said, new drugs “are geared towards adults whose leukemia is not as aggressive. Whereas in children, the acute leukemias, especially AML, are quite aggressive and need therapies that are often more intense.”
In addition, he said, “we have only recently become aware of how AML is biologically much different than in adults.”
In AML, Delaware’s Dr. Kolb explained, “there are many different phenotypes – ways that these cells can look and behave. But we treat them with a single regimen. What I like to tell families is that we’ve got a few tools in our toolbox, but they all happen to be sledgehammers. The key to the challenge in AML is that it is a molecular disease, but we’re treating it with therapies that were developed 40-50 years ago.”
In PedAL, the goal is to figure out the best ways to target therapy for the specific types that patients have. On this front, the genomic screening in the trial is crucial because it will identify which patients express certain targets and allow them to be assigned to appropriate sub-trials, Dr. Coooper said.
What’s next? “LLS has planned for this to be ongoing for the next 5 to 7 years, so that we can get a number of studies up and running,” Dr. Nichols said. “After that, those studies will continue. We will hope that most of them can be self-funded by then.”
As for cost, she noted that the PedAL trial is part of the society’s Dare to Dream Project, formerly known as the Children’s Initiative, which focuses on pediatric blood cancers. The project, with a fundraising goal of $175 million, focuses on research, patient services and survivorship.
”We have a whole range of services, travel assistance, copay programs and educational resources that doctors may want to use as a valid source of information,” she said. ‘When I was in practice, patients were always asking me, ‘Do you have anything I can read or take home to give my son something about his disease?’ LLS has good-quality, patient-level information for patients. We welcome people contacting us or going to our website and taking advantage of that for free.”
Dr. Nichols and Dr. Kolb report no disclosures. Dr. Cooper reports academic funding from LLS.
While great strides have been made in children’s leukemia care during the past 50 years, statistics have remained grim. For acute myeloid leukemia (AML), the most common type, 5-year survival rates were just 69% for children younger than 15 between 2009 and 2015. Patients who do survive past adolescence face high risks of future complications.
Specialists say the challenges hindering more progress include a lack of clinical research, an emphasis on competition over cooperation, and sparse insight into how best to adjust adult leukemia treatments to children.
“Our project aims to find better treatments, more targeted treatments, that will leave children with fewer long-term health problems as adults. We want them to not just survive but thrive,” Gwen Nichols, MD, chief medical officer of LLS, said in an interview. “What we’ve had was not working for anybody. So we have to try a different approach.”
The LLS Pediatric Acute Leukemia (PedAL) Master Trial launched in spring of 2022. Seventy-five study locations from Nova Scotia to Hawaii are now recruiting patients up to age 22 with known or suspected relapsed/refractory AML, mixed phenotype acute leukemia, or relapsed acute lymphoblastic leukemia (ALL).
The 5-year trial expects to recruit 960 participants in the United States and Canada. Clinics in Europe, Australia, and New Zealand also are taking part.
“Pediatric oncologists should know that PedAL, for the first time, is providing a cooperative, seamless way to interrogate [the genomics of] a child’s leukemia,” hematologist/oncologist Todd Cooper, DO, section chief of pediatric oncology at Seattle Children’s Cancer and Blood Disorders Center, said in an interview. “It is also providing a seamless and efficient way for children to be assigned to clinical trials that are going to be tailored towards a particular child’s leukemia. This is something that’s never been done.”
In North America, all trial participants with relapsed AML will undergo genetic sequencing for free as part of the screening process. Clinics “can’t always access genomic screening for their patients,” Dr. Nichols said. “We’re providing that even if they don’t participate in any other part of the trial, even if they go and get another available therapy or go on a different trial. We want them to know that this is available, and they will get the results. And if they’re looking for a trial when they get those results, we have trained oncology nurses who will help them navigate and find clinical trials.”
In PedAL itself, one subtrial is now in progress: An open-label phase 3 randomized multicenter analysis of whether the oral leukemia drug venetoclax combined with the intensive infused chemotherapy treatment FLA+GO (fludarabine, high-dose cytarabine, and gemtuzumab ozogamicin) will improve overall survival compared to FLA+GO alone. Ninety-eight subjects are expected to join the 5-year subtrial.
“We expect within the next year to open three or four different subtrials of targeted therapies for specific groups of patients,” E. Anders Kolb, MD, chief of oncology and hematology at Nemours Children’s Health in Delaware and cochair of the PedAL trial, said in an interview. “Over the course of the next few years, we’re going to learn a lot about the natural history of relapsed leukemia – we don’t have a ton of data on that – and then how targeted therapies may alter some of those outcomes.”
Discussions with multiple drugmakers are in progress regarding the potential subtrials, he said.
The PedAL strategy addresses the lack of new drugs for children with AML, Seattle Children’s Dr. Cooper said. One main reason for the gap is that childhood leukemia is much less common than the adult form, he said, so a lot of drug development is geared toward adults. As a result, he said, new drugs “are geared towards adults whose leukemia is not as aggressive. Whereas in children, the acute leukemias, especially AML, are quite aggressive and need therapies that are often more intense.”
In addition, he said, “we have only recently become aware of how AML is biologically much different than in adults.”
In AML, Delaware’s Dr. Kolb explained, “there are many different phenotypes – ways that these cells can look and behave. But we treat them with a single regimen. What I like to tell families is that we’ve got a few tools in our toolbox, but they all happen to be sledgehammers. The key to the challenge in AML is that it is a molecular disease, but we’re treating it with therapies that were developed 40-50 years ago.”
In PedAL, the goal is to figure out the best ways to target therapy for the specific types that patients have. On this front, the genomic screening in the trial is crucial because it will identify which patients express certain targets and allow them to be assigned to appropriate sub-trials, Dr. Coooper said.
What’s next? “LLS has planned for this to be ongoing for the next 5 to 7 years, so that we can get a number of studies up and running,” Dr. Nichols said. “After that, those studies will continue. We will hope that most of them can be self-funded by then.”
As for cost, she noted that the PedAL trial is part of the society’s Dare to Dream Project, formerly known as the Children’s Initiative, which focuses on pediatric blood cancers. The project, with a fundraising goal of $175 million, focuses on research, patient services and survivorship.
”We have a whole range of services, travel assistance, copay programs and educational resources that doctors may want to use as a valid source of information,” she said. ‘When I was in practice, patients were always asking me, ‘Do you have anything I can read or take home to give my son something about his disease?’ LLS has good-quality, patient-level information for patients. We welcome people contacting us or going to our website and taking advantage of that for free.”
Dr. Nichols and Dr. Kolb report no disclosures. Dr. Cooper reports academic funding from LLS.
While great strides have been made in children’s leukemia care during the past 50 years, statistics have remained grim. For acute myeloid leukemia (AML), the most common type, 5-year survival rates were just 69% for children younger than 15 between 2009 and 2015. Patients who do survive past adolescence face high risks of future complications.
Specialists say the challenges hindering more progress include a lack of clinical research, an emphasis on competition over cooperation, and sparse insight into how best to adjust adult leukemia treatments to children.
“Our project aims to find better treatments, more targeted treatments, that will leave children with fewer long-term health problems as adults. We want them to not just survive but thrive,” Gwen Nichols, MD, chief medical officer of LLS, said in an interview. “What we’ve had was not working for anybody. So we have to try a different approach.”
The LLS Pediatric Acute Leukemia (PedAL) Master Trial launched in spring of 2022. Seventy-five study locations from Nova Scotia to Hawaii are now recruiting patients up to age 22 with known or suspected relapsed/refractory AML, mixed phenotype acute leukemia, or relapsed acute lymphoblastic leukemia (ALL).
The 5-year trial expects to recruit 960 participants in the United States and Canada. Clinics in Europe, Australia, and New Zealand also are taking part.
“Pediatric oncologists should know that PedAL, for the first time, is providing a cooperative, seamless way to interrogate [the genomics of] a child’s leukemia,” hematologist/oncologist Todd Cooper, DO, section chief of pediatric oncology at Seattle Children’s Cancer and Blood Disorders Center, said in an interview. “It is also providing a seamless and efficient way for children to be assigned to clinical trials that are going to be tailored towards a particular child’s leukemia. This is something that’s never been done.”
In North America, all trial participants with relapsed AML will undergo genetic sequencing for free as part of the screening process. Clinics “can’t always access genomic screening for their patients,” Dr. Nichols said. “We’re providing that even if they don’t participate in any other part of the trial, even if they go and get another available therapy or go on a different trial. We want them to know that this is available, and they will get the results. And if they’re looking for a trial when they get those results, we have trained oncology nurses who will help them navigate and find clinical trials.”
In PedAL itself, one subtrial is now in progress: An open-label phase 3 randomized multicenter analysis of whether the oral leukemia drug venetoclax combined with the intensive infused chemotherapy treatment FLA+GO (fludarabine, high-dose cytarabine, and gemtuzumab ozogamicin) will improve overall survival compared to FLA+GO alone. Ninety-eight subjects are expected to join the 5-year subtrial.
“We expect within the next year to open three or four different subtrials of targeted therapies for specific groups of patients,” E. Anders Kolb, MD, chief of oncology and hematology at Nemours Children’s Health in Delaware and cochair of the PedAL trial, said in an interview. “Over the course of the next few years, we’re going to learn a lot about the natural history of relapsed leukemia – we don’t have a ton of data on that – and then how targeted therapies may alter some of those outcomes.”
Discussions with multiple drugmakers are in progress regarding the potential subtrials, he said.
The PedAL strategy addresses the lack of new drugs for children with AML, Seattle Children’s Dr. Cooper said. One main reason for the gap is that childhood leukemia is much less common than the adult form, he said, so a lot of drug development is geared toward adults. As a result, he said, new drugs “are geared towards adults whose leukemia is not as aggressive. Whereas in children, the acute leukemias, especially AML, are quite aggressive and need therapies that are often more intense.”
In addition, he said, “we have only recently become aware of how AML is biologically much different than in adults.”
In AML, Delaware’s Dr. Kolb explained, “there are many different phenotypes – ways that these cells can look and behave. But we treat them with a single regimen. What I like to tell families is that we’ve got a few tools in our toolbox, but they all happen to be sledgehammers. The key to the challenge in AML is that it is a molecular disease, but we’re treating it with therapies that were developed 40-50 years ago.”
In PedAL, the goal is to figure out the best ways to target therapy for the specific types that patients have. On this front, the genomic screening in the trial is crucial because it will identify which patients express certain targets and allow them to be assigned to appropriate sub-trials, Dr. Coooper said.
What’s next? “LLS has planned for this to be ongoing for the next 5 to 7 years, so that we can get a number of studies up and running,” Dr. Nichols said. “After that, those studies will continue. We will hope that most of them can be self-funded by then.”
As for cost, she noted that the PedAL trial is part of the society’s Dare to Dream Project, formerly known as the Children’s Initiative, which focuses on pediatric blood cancers. The project, with a fundraising goal of $175 million, focuses on research, patient services and survivorship.
”We have a whole range of services, travel assistance, copay programs and educational resources that doctors may want to use as a valid source of information,” she said. ‘When I was in practice, patients were always asking me, ‘Do you have anything I can read or take home to give my son something about his disease?’ LLS has good-quality, patient-level information for patients. We welcome people contacting us or going to our website and taking advantage of that for free.”
Dr. Nichols and Dr. Kolb report no disclosures. Dr. Cooper reports academic funding from LLS.
Drug combo holds promise of better AML outcomes
Adding venetoclax (Venclexta) to a gilteritinib (Xospata) regimen appeared to improve outcomes in refractory/relapsed FLT3-mutated acute myeloid leukemia (AML), a new industry-funded phase 1b study reported.
“.
Outcomes in AML are poor. As the study notes, most patients relapse and face a median overall survival of 4-7 months even with standard chemotherapy. Gilteritinib, a selective oral FLT3 inhibitor, is Food and Drug Administration–approved for the 30% of relapsed/refractory patients with AML who have FLT3 mutations.
“The general sentiment is that, although some patients have great benefit from gilteritinib monotherapy, there is room to improve the quality, frequency, and duration of responses with combinations,” said hematologist Andrew Brunner, MD, of Massachusetts General Hospital in Boston, in an interview. He was not involved with the study research.
For the new open-label, dose-escalation/dose-expansion study, led by hematologist Naval Daver, MD, of the University of Texas MD Anderson Cancer Center, Houston, researchers enrolled 61 patients (56 with FLT3 mutations) from 2018 to 2020. The median age was 63 years (range 21-85).
The subjects were assigned to get a recommended phase 2 dose of 400 mg venetoclax once daily and 120 mg gilteritinib once daily.
Over a median follow-up of 17.5 months, the median remission time was 4.9 months (95% confidence interval, 3.4-6.6), and the patients with FLT3 mutations survived a median of 10 months.
“The combination of venetoclax and gilteritinib was tolerable at standard doses of each drug, generated remarkably high response rates, and markedly reduced FLT3-internal tandem duplications mutation burden. … Early mortality was similar to gilteritinib monotherapy,” the authors wrote.
Eighty percent of patients experienced cytopenias, and “adverse events prompted venetoclax and gilteritinib dose interruptions in 51% and 48%, respectively.”
About 60% of patients who went on to receive allogeneic hematopoietic stem cell transplantation were alive at the end of follow-up, “suggesting that VenGilt [the combo treatment] could be an effective bridge to transplant in young/fit patients with relapsed FLT3mut AML,” the researchers wrote.
All patients withdrew from the study by November 2021 for several reasons such as death (n=42), adverse events (n=10), and disease progression (29); some had multiple reasons.
Dr. Brunner said the study is “an important step toward evaluating a new potential regimen.”
The remission duration, FLT3 molecular response, and median overall survival “seem quite encouraging for a severe disease like AML in relapse,” he said. However, he added that the drug combo “would need to be evaluated in a randomized and, ideally, placebo-controlled setting to know if this is a significant improvement.”
He also highlighted the high number of severe cyptopenias with associated complications such as death. “Whether this is acceptable depends on the patient and circumstances,” he said. “But it does suggest that this regimen would potentially be for more robust patients, particularly since the group that did best were those who went to transplant later.”
Pending more research, Dr. Brunner said, “I am not sure I would use [the combination treatment] over gilteritinib monotherapy, for instance. But there may be settings where no other options are available, and this could be considered, particularly if a transplant option is a next step.”
The study was funded by AbbVie, Genentech, and Astellas. The study authors report multiple disclosures; some are employed by Astellas, AbbVie, and Genentech/Roche.
Dr. Bronner reports running clinical trials, advisory board service and/or consultation for Acceleron, Agios, Abbvie, BMS/Celgene, Keros Therapeutics, Novartis, Takeda, GSK, AstraZeneca, Janssen, and Gilead.
Adding venetoclax (Venclexta) to a gilteritinib (Xospata) regimen appeared to improve outcomes in refractory/relapsed FLT3-mutated acute myeloid leukemia (AML), a new industry-funded phase 1b study reported.
“.
Outcomes in AML are poor. As the study notes, most patients relapse and face a median overall survival of 4-7 months even with standard chemotherapy. Gilteritinib, a selective oral FLT3 inhibitor, is Food and Drug Administration–approved for the 30% of relapsed/refractory patients with AML who have FLT3 mutations.
“The general sentiment is that, although some patients have great benefit from gilteritinib monotherapy, there is room to improve the quality, frequency, and duration of responses with combinations,” said hematologist Andrew Brunner, MD, of Massachusetts General Hospital in Boston, in an interview. He was not involved with the study research.
For the new open-label, dose-escalation/dose-expansion study, led by hematologist Naval Daver, MD, of the University of Texas MD Anderson Cancer Center, Houston, researchers enrolled 61 patients (56 with FLT3 mutations) from 2018 to 2020. The median age was 63 years (range 21-85).
The subjects were assigned to get a recommended phase 2 dose of 400 mg venetoclax once daily and 120 mg gilteritinib once daily.
Over a median follow-up of 17.5 months, the median remission time was 4.9 months (95% confidence interval, 3.4-6.6), and the patients with FLT3 mutations survived a median of 10 months.
“The combination of venetoclax and gilteritinib was tolerable at standard doses of each drug, generated remarkably high response rates, and markedly reduced FLT3-internal tandem duplications mutation burden. … Early mortality was similar to gilteritinib monotherapy,” the authors wrote.
Eighty percent of patients experienced cytopenias, and “adverse events prompted venetoclax and gilteritinib dose interruptions in 51% and 48%, respectively.”
About 60% of patients who went on to receive allogeneic hematopoietic stem cell transplantation were alive at the end of follow-up, “suggesting that VenGilt [the combo treatment] could be an effective bridge to transplant in young/fit patients with relapsed FLT3mut AML,” the researchers wrote.
All patients withdrew from the study by November 2021 for several reasons such as death (n=42), adverse events (n=10), and disease progression (29); some had multiple reasons.
Dr. Brunner said the study is “an important step toward evaluating a new potential regimen.”
The remission duration, FLT3 molecular response, and median overall survival “seem quite encouraging for a severe disease like AML in relapse,” he said. However, he added that the drug combo “would need to be evaluated in a randomized and, ideally, placebo-controlled setting to know if this is a significant improvement.”
He also highlighted the high number of severe cyptopenias with associated complications such as death. “Whether this is acceptable depends on the patient and circumstances,” he said. “But it does suggest that this regimen would potentially be for more robust patients, particularly since the group that did best were those who went to transplant later.”
Pending more research, Dr. Brunner said, “I am not sure I would use [the combination treatment] over gilteritinib monotherapy, for instance. But there may be settings where no other options are available, and this could be considered, particularly if a transplant option is a next step.”
The study was funded by AbbVie, Genentech, and Astellas. The study authors report multiple disclosures; some are employed by Astellas, AbbVie, and Genentech/Roche.
Dr. Bronner reports running clinical trials, advisory board service and/or consultation for Acceleron, Agios, Abbvie, BMS/Celgene, Keros Therapeutics, Novartis, Takeda, GSK, AstraZeneca, Janssen, and Gilead.
Adding venetoclax (Venclexta) to a gilteritinib (Xospata) regimen appeared to improve outcomes in refractory/relapsed FLT3-mutated acute myeloid leukemia (AML), a new industry-funded phase 1b study reported.
“.
Outcomes in AML are poor. As the study notes, most patients relapse and face a median overall survival of 4-7 months even with standard chemotherapy. Gilteritinib, a selective oral FLT3 inhibitor, is Food and Drug Administration–approved for the 30% of relapsed/refractory patients with AML who have FLT3 mutations.
“The general sentiment is that, although some patients have great benefit from gilteritinib monotherapy, there is room to improve the quality, frequency, and duration of responses with combinations,” said hematologist Andrew Brunner, MD, of Massachusetts General Hospital in Boston, in an interview. He was not involved with the study research.
For the new open-label, dose-escalation/dose-expansion study, led by hematologist Naval Daver, MD, of the University of Texas MD Anderson Cancer Center, Houston, researchers enrolled 61 patients (56 with FLT3 mutations) from 2018 to 2020. The median age was 63 years (range 21-85).
The subjects were assigned to get a recommended phase 2 dose of 400 mg venetoclax once daily and 120 mg gilteritinib once daily.
Over a median follow-up of 17.5 months, the median remission time was 4.9 months (95% confidence interval, 3.4-6.6), and the patients with FLT3 mutations survived a median of 10 months.
“The combination of venetoclax and gilteritinib was tolerable at standard doses of each drug, generated remarkably high response rates, and markedly reduced FLT3-internal tandem duplications mutation burden. … Early mortality was similar to gilteritinib monotherapy,” the authors wrote.
Eighty percent of patients experienced cytopenias, and “adverse events prompted venetoclax and gilteritinib dose interruptions in 51% and 48%, respectively.”
About 60% of patients who went on to receive allogeneic hematopoietic stem cell transplantation were alive at the end of follow-up, “suggesting that VenGilt [the combo treatment] could be an effective bridge to transplant in young/fit patients with relapsed FLT3mut AML,” the researchers wrote.
All patients withdrew from the study by November 2021 for several reasons such as death (n=42), adverse events (n=10), and disease progression (29); some had multiple reasons.
Dr. Brunner said the study is “an important step toward evaluating a new potential regimen.”
The remission duration, FLT3 molecular response, and median overall survival “seem quite encouraging for a severe disease like AML in relapse,” he said. However, he added that the drug combo “would need to be evaluated in a randomized and, ideally, placebo-controlled setting to know if this is a significant improvement.”
He also highlighted the high number of severe cyptopenias with associated complications such as death. “Whether this is acceptable depends on the patient and circumstances,” he said. “But it does suggest that this regimen would potentially be for more robust patients, particularly since the group that did best were those who went to transplant later.”
Pending more research, Dr. Brunner said, “I am not sure I would use [the combination treatment] over gilteritinib monotherapy, for instance. But there may be settings where no other options are available, and this could be considered, particularly if a transplant option is a next step.”
The study was funded by AbbVie, Genentech, and Astellas. The study authors report multiple disclosures; some are employed by Astellas, AbbVie, and Genentech/Roche.
Dr. Bronner reports running clinical trials, advisory board service and/or consultation for Acceleron, Agios, Abbvie, BMS/Celgene, Keros Therapeutics, Novartis, Takeda, GSK, AstraZeneca, Janssen, and Gilead.
FROM JOURNAL OF CLINICAL ONCOLOGY
Family affair: OncBrothers host oncology hangout online
It’s hard out there for a small-town cancer doctor. Just ask Wederson M. Claudino, MD, who serves the town of Paducah in far western Kentucky. The nearest cities with significant numbers of hematologist/oncologists are hours away in cities like St. Louis and Nashville, Tenn., too far to go to talk shop over coffee, drinks, or lunch.
“It’s very challenging in a rural or small community,” he said. “I miss the opportunity to elaborate on a case.”
Now Dr. Claudino and hundreds of colleagues have discovered that useful cancer conversations are just a click away.
Urban and rural oncologists gather there to discuss new research, compare notes about challenging cases, and get to know each other.
“Following their Twitter feed and the comments and discussions make me feel like part of a bigger community,” Dr. Claudino said. And @OncBrothers is indeed a bustling Internet destination: The account’s 4,300 followers include hundreds who participate in discussions and offer perspective.
For instance, the brothers recently posted a poll asking followers how they’d treat a 55-year-old patient with non–small cell lung cancer. Nearly 250 people responded with their preferred approaches, and the survey thread included comments from oncologists from the City of Hope National Medical Center, the University of Florida, UC San Diego, and elsewhere.
In an interview, the Gosain brothers said the Twitter account is an outgrowth of their phone conversations in recent years, as they trained and settled into their early careers as general medical oncologists in smaller communities.
“After our clinic days, we’ll jump on the phone for 30-45 minutes. We’d talk about patients, how he would treat a case, and what I would do,” Rahul said. “We realized that we were living in a bubble, but we also thought that there are a lot more people in the same boat. They might jump at being able to do the same thing.”
Rahul recently became medical director at the new Wilmot Webster Cancer Center in Rochester, N.Y., after working in Corning, a tiny New York town just north of the Pennsylvania border. His brother Rohit is chair of hematology and oncology at the University of Pittsburgh’s Hillman Cancer Center in Jamestown, a small town at the western edge of New York.
“When we initially kicked off the Twitter account in August 2021, we didn’t realize the traction it would get,” Rohit said. “Now we realize that there really is a need for this.”
On an ordinary day, the @OncBrothers account may highlight research presented at a oncology conference, retweet posts by other oncologists about new guidelines or FDA drug approvals, and ask followers to consider how they’d handle a difficult case.
The brothers are especially thrilled when posts spawn discussions that draw voices from leading medical institutions who normally don’t interact much with community oncologists. “You’ll have someone from Sloan Kettering or Dana-Farber saying ‘This is what would do,’ ” Rahul said. “You have the brightest minds pitching in, and we get to learn from them.”
The Gosain brothers were both born in India and immigrated as children to Toronto. They each went to medical school in the Caribbean – for Rohit, it was after a stint as a computer engineer – and they each embraced oncology. “For me, it was about having the right mentors while I was doing my clinical rotations as a medical student and as a resident,” Rahul said. In addition, he said, “this field was moving and is still moving so fast. It really intrigued and excited me and made me want to be at the forefront of it.”
The fast-moving nature of oncology, in fact, was one of the drivers behind the daily conversations between the brothers and the subsequent creation of the @OncBrothers account. “Just last year, in 2021, there were 40 new drugs that were indicated for hematology-oncology,” Rahul said. “To stay on top of that is very, very hard.”
It’s especially difficult to figure out treatment plans when multiple options exist. A 2022 thread on @OncBrothers revealed a wide divergence of opinions about triple therapy in prostate cancer: The 322 respondents to a Twitter poll were sharply divided about the best three-drug combination from these options – docetaxel, daratumumab, abiraterone, androgen deprivation therapy, and alpha-reductase inhibitors.
To make things more challenging, community oncologists often are generalists who treat patients with a wide variety of cancers from prostate and lung to breast and colon. As a result, these oncologists must keep up on developments across the entire cancer field. Rohit highlighted a 2022 thread that polled users about the approach they’d take to another patient with non–small cell lung cancer; 474 people responded. The accompanying discussion emphasized the need for the next-generation sequencing (NGS).
“A significant portion of community oncologists are not even doing NGS testing, which is FDA-approved,” Rohit said. “There’s a huge gap that still exists, and we weren’t even aware of it. We continue to learn from these conversations.”
The brothers contend that there’s a crucial need for education among community oncologists in light of evidence suggesting that some cancer outcomes are worse than those in urban areas.
In fact, Rohit led a 2019 study published in the journal Cancer that found that overall survival in rural patients with neuroendocrine tumors trended toward worse outcomes than in urban patients.
“There are many factors such as financial burden, lack of education, and rural patients not willing to travel to the city,” Rohit said. “We need to be more creative and ask, ‘How can we equip our medical oncologist in rural settings to continue to do better?’ ”
What’s next for the OncBrothers? The Gosains have created a website (www.oncbrothers.com) that highlights their social media work, and they’re exploring options such as podcasts and short videos. “Our goal is to focus on how to continue to keep general medical oncologists up to date, informed, and educated so patients can get the best care close to home,” Rahul said. “We need to do better.”
It’s hard out there for a small-town cancer doctor. Just ask Wederson M. Claudino, MD, who serves the town of Paducah in far western Kentucky. The nearest cities with significant numbers of hematologist/oncologists are hours away in cities like St. Louis and Nashville, Tenn., too far to go to talk shop over coffee, drinks, or lunch.
“It’s very challenging in a rural or small community,” he said. “I miss the opportunity to elaborate on a case.”
Now Dr. Claudino and hundreds of colleagues have discovered that useful cancer conversations are just a click away.
Urban and rural oncologists gather there to discuss new research, compare notes about challenging cases, and get to know each other.
“Following their Twitter feed and the comments and discussions make me feel like part of a bigger community,” Dr. Claudino said. And @OncBrothers is indeed a bustling Internet destination: The account’s 4,300 followers include hundreds who participate in discussions and offer perspective.
For instance, the brothers recently posted a poll asking followers how they’d treat a 55-year-old patient with non–small cell lung cancer. Nearly 250 people responded with their preferred approaches, and the survey thread included comments from oncologists from the City of Hope National Medical Center, the University of Florida, UC San Diego, and elsewhere.
In an interview, the Gosain brothers said the Twitter account is an outgrowth of their phone conversations in recent years, as they trained and settled into their early careers as general medical oncologists in smaller communities.
“After our clinic days, we’ll jump on the phone for 30-45 minutes. We’d talk about patients, how he would treat a case, and what I would do,” Rahul said. “We realized that we were living in a bubble, but we also thought that there are a lot more people in the same boat. They might jump at being able to do the same thing.”
Rahul recently became medical director at the new Wilmot Webster Cancer Center in Rochester, N.Y., after working in Corning, a tiny New York town just north of the Pennsylvania border. His brother Rohit is chair of hematology and oncology at the University of Pittsburgh’s Hillman Cancer Center in Jamestown, a small town at the western edge of New York.
“When we initially kicked off the Twitter account in August 2021, we didn’t realize the traction it would get,” Rohit said. “Now we realize that there really is a need for this.”
On an ordinary day, the @OncBrothers account may highlight research presented at a oncology conference, retweet posts by other oncologists about new guidelines or FDA drug approvals, and ask followers to consider how they’d handle a difficult case.
The brothers are especially thrilled when posts spawn discussions that draw voices from leading medical institutions who normally don’t interact much with community oncologists. “You’ll have someone from Sloan Kettering or Dana-Farber saying ‘This is what would do,’ ” Rahul said. “You have the brightest minds pitching in, and we get to learn from them.”
The Gosain brothers were both born in India and immigrated as children to Toronto. They each went to medical school in the Caribbean – for Rohit, it was after a stint as a computer engineer – and they each embraced oncology. “For me, it was about having the right mentors while I was doing my clinical rotations as a medical student and as a resident,” Rahul said. In addition, he said, “this field was moving and is still moving so fast. It really intrigued and excited me and made me want to be at the forefront of it.”
The fast-moving nature of oncology, in fact, was one of the drivers behind the daily conversations between the brothers and the subsequent creation of the @OncBrothers account. “Just last year, in 2021, there were 40 new drugs that were indicated for hematology-oncology,” Rahul said. “To stay on top of that is very, very hard.”
It’s especially difficult to figure out treatment plans when multiple options exist. A 2022 thread on @OncBrothers revealed a wide divergence of opinions about triple therapy in prostate cancer: The 322 respondents to a Twitter poll were sharply divided about the best three-drug combination from these options – docetaxel, daratumumab, abiraterone, androgen deprivation therapy, and alpha-reductase inhibitors.
To make things more challenging, community oncologists often are generalists who treat patients with a wide variety of cancers from prostate and lung to breast and colon. As a result, these oncologists must keep up on developments across the entire cancer field. Rohit highlighted a 2022 thread that polled users about the approach they’d take to another patient with non–small cell lung cancer; 474 people responded. The accompanying discussion emphasized the need for the next-generation sequencing (NGS).
“A significant portion of community oncologists are not even doing NGS testing, which is FDA-approved,” Rohit said. “There’s a huge gap that still exists, and we weren’t even aware of it. We continue to learn from these conversations.”
The brothers contend that there’s a crucial need for education among community oncologists in light of evidence suggesting that some cancer outcomes are worse than those in urban areas.
In fact, Rohit led a 2019 study published in the journal Cancer that found that overall survival in rural patients with neuroendocrine tumors trended toward worse outcomes than in urban patients.
“There are many factors such as financial burden, lack of education, and rural patients not willing to travel to the city,” Rohit said. “We need to be more creative and ask, ‘How can we equip our medical oncologist in rural settings to continue to do better?’ ”
What’s next for the OncBrothers? The Gosains have created a website (www.oncbrothers.com) that highlights their social media work, and they’re exploring options such as podcasts and short videos. “Our goal is to focus on how to continue to keep general medical oncologists up to date, informed, and educated so patients can get the best care close to home,” Rahul said. “We need to do better.”
It’s hard out there for a small-town cancer doctor. Just ask Wederson M. Claudino, MD, who serves the town of Paducah in far western Kentucky. The nearest cities with significant numbers of hematologist/oncologists are hours away in cities like St. Louis and Nashville, Tenn., too far to go to talk shop over coffee, drinks, or lunch.
“It’s very challenging in a rural or small community,” he said. “I miss the opportunity to elaborate on a case.”
Now Dr. Claudino and hundreds of colleagues have discovered that useful cancer conversations are just a click away.
Urban and rural oncologists gather there to discuss new research, compare notes about challenging cases, and get to know each other.
“Following their Twitter feed and the comments and discussions make me feel like part of a bigger community,” Dr. Claudino said. And @OncBrothers is indeed a bustling Internet destination: The account’s 4,300 followers include hundreds who participate in discussions and offer perspective.
For instance, the brothers recently posted a poll asking followers how they’d treat a 55-year-old patient with non–small cell lung cancer. Nearly 250 people responded with their preferred approaches, and the survey thread included comments from oncologists from the City of Hope National Medical Center, the University of Florida, UC San Diego, and elsewhere.
In an interview, the Gosain brothers said the Twitter account is an outgrowth of their phone conversations in recent years, as they trained and settled into their early careers as general medical oncologists in smaller communities.
“After our clinic days, we’ll jump on the phone for 30-45 minutes. We’d talk about patients, how he would treat a case, and what I would do,” Rahul said. “We realized that we were living in a bubble, but we also thought that there are a lot more people in the same boat. They might jump at being able to do the same thing.”
Rahul recently became medical director at the new Wilmot Webster Cancer Center in Rochester, N.Y., after working in Corning, a tiny New York town just north of the Pennsylvania border. His brother Rohit is chair of hematology and oncology at the University of Pittsburgh’s Hillman Cancer Center in Jamestown, a small town at the western edge of New York.
“When we initially kicked off the Twitter account in August 2021, we didn’t realize the traction it would get,” Rohit said. “Now we realize that there really is a need for this.”
On an ordinary day, the @OncBrothers account may highlight research presented at a oncology conference, retweet posts by other oncologists about new guidelines or FDA drug approvals, and ask followers to consider how they’d handle a difficult case.
The brothers are especially thrilled when posts spawn discussions that draw voices from leading medical institutions who normally don’t interact much with community oncologists. “You’ll have someone from Sloan Kettering or Dana-Farber saying ‘This is what would do,’ ” Rahul said. “You have the brightest minds pitching in, and we get to learn from them.”
The Gosain brothers were both born in India and immigrated as children to Toronto. They each went to medical school in the Caribbean – for Rohit, it was after a stint as a computer engineer – and they each embraced oncology. “For me, it was about having the right mentors while I was doing my clinical rotations as a medical student and as a resident,” Rahul said. In addition, he said, “this field was moving and is still moving so fast. It really intrigued and excited me and made me want to be at the forefront of it.”
The fast-moving nature of oncology, in fact, was one of the drivers behind the daily conversations between the brothers and the subsequent creation of the @OncBrothers account. “Just last year, in 2021, there were 40 new drugs that were indicated for hematology-oncology,” Rahul said. “To stay on top of that is very, very hard.”
It’s especially difficult to figure out treatment plans when multiple options exist. A 2022 thread on @OncBrothers revealed a wide divergence of opinions about triple therapy in prostate cancer: The 322 respondents to a Twitter poll were sharply divided about the best three-drug combination from these options – docetaxel, daratumumab, abiraterone, androgen deprivation therapy, and alpha-reductase inhibitors.
To make things more challenging, community oncologists often are generalists who treat patients with a wide variety of cancers from prostate and lung to breast and colon. As a result, these oncologists must keep up on developments across the entire cancer field. Rohit highlighted a 2022 thread that polled users about the approach they’d take to another patient with non–small cell lung cancer; 474 people responded. The accompanying discussion emphasized the need for the next-generation sequencing (NGS).
“A significant portion of community oncologists are not even doing NGS testing, which is FDA-approved,” Rohit said. “There’s a huge gap that still exists, and we weren’t even aware of it. We continue to learn from these conversations.”
The brothers contend that there’s a crucial need for education among community oncologists in light of evidence suggesting that some cancer outcomes are worse than those in urban areas.
In fact, Rohit led a 2019 study published in the journal Cancer that found that overall survival in rural patients with neuroendocrine tumors trended toward worse outcomes than in urban patients.
“There are many factors such as financial burden, lack of education, and rural patients not willing to travel to the city,” Rohit said. “We need to be more creative and ask, ‘How can we equip our medical oncologist in rural settings to continue to do better?’ ”
What’s next for the OncBrothers? The Gosains have created a website (www.oncbrothers.com) that highlights their social media work, and they’re exploring options such as podcasts and short videos. “Our goal is to focus on how to continue to keep general medical oncologists up to date, informed, and educated so patients can get the best care close to home,” Rahul said. “We need to do better.”
Royal family affliction or not, porphyria is treatable
European royal families may be enormously rich, but being a blueblood doesn’t always mean your blood is pristine. Queen Victoria’s DNA is famously believed to have silently bequeathed hemophilia to many of her descendants, including a great-grandson whose severe illness played a tragic role in spurring the Russian Revolution.
And that’s not all.
There’s plenty of skepticism about this theory, which seeks to explain the “madness” of King George III. But one thing is clear. If porphyria does indeed haunt the imperial bloodline that stretches to a new generation – the late Queen Elizabeth II’s great-grandchildren – any royal who’s afflicted going forward is likely to benefit mightily from modern treatment. While this disease may require lifelong vigilance, experts said in interviews that porphyria can often be controlled.
“If patients know they have the diagnosis, and they do the right things and avoid alcohol and risky drugs, most people will have few acute attacks,” said gastroenterologist Herbert Lloyd Bonkovsky, MD, of Wake Forest University, Winston-Salem, N.C., a leading porphyria specialist.
Heme infusions can also be helpful, he added, and the revolutionary new drug givosiran is available for those who suffer recurrent attacks. And “if all else fails, a successful liver transplant is curative” – as long as the transplanted liver doesn’t have porphyria, as happened in at least one case.
But, Dr. Bonkovsky cautioned, the diagnosis is often missed, in some cases for 15 years or more.
Diagnosing porphyria: Awareness and tests are crucial
Porphyria is caused when porphyins – essential components of hemoglobin – build up in the body, disrupting systems such as the nerves, skin, and gut. The urine can turn purplish, hence the condition’s name. (Porphyrus is the Greek word for purple.)
According to hematologist Danielle Nance, MD, of Banner MD Anderson Cancer Center in Gilbert, Ariz., acute intermittent porphyria “should be suspected in persons who have recurrent severe attacks of abdominal pain requiring strong pain medication to control symptoms, and there is no obvious physical cause.”
In such cases, practitioners should send out blood and urine for porphobilinogen (PBG) and delta-aminolevulinic acid (dALA or Delta-ALA) testing, Dr. Nance said. “These are almost always elevated, even between attacks, in persons with diagnoses of acute intermittent porphyria. Other types of porphyria, such as erythropoietic porphyria, may require additional testing. Genetic testing should be offered when a patient is suspected of having porphyria, as this can speed the diagnosis.”
The typical patient is a woman from age 18 to 55, often a young woman with recurrent abdominal pain that may occur during the second half of the menstrual cycle, Wake Forest’s Dr. Bonkovsky said. Constipation is common.
“She keeps coming to the clinic or emergency department, and no one knows what’s going on. Eventually, she tends to undergo an appendectomy, often a cholecystectomy, or sometimes gynecologic procedures without cure of the disease. Only after this long and arduous road of misdiagnosis does someone think it’s porphyria and do the correct tests.”
Dr. Bonkovsky led a 2014 study of 108 subjects (81% female) with acute porphyrias and found that the average time to a correct diagnosis was a whopping 15 years. Pain in the abdomen was the most common symptom (74%), followed by nausea/vomiting (73%), weakness (63%), and constipation (60%).
While underdiagnosis is common, porphyrias can also be overdiagnosed. According to Dr. Bonkovsky, a mild increase in urinary porphyrins is often misdiagnosed as porphyria when it may be a sign of liver disease or alcohol use, instead.
Hematologist Kleber Y. Fertrin, MD, PhD, of the University of Washington, Seattle, emphasized the importance of ordering the correct tests. “Urinary porphyrins are often inappropriately ordered because of their name. They are not diagnostic for acute hepatic porphyrias and may be nonspecifically elevated. It is paramount to get the labs from a reliable lab test experienced at performing diagnostic testing for porphyrias and make sure the urine sample needed is correctly obtained and preserved.”
New drug can control attacks, but it’s costly
Treatment should begin right away if porphyria is suspected, without waiting for confirmation, Dr. Nance said. “There are porphyria experts in the U.S. and in many other countries who can help practitioners interpret symptoms and testing results if there is uncertainty or if treatment options are unclear.”
Patients are counseled to avoid attack triggers and eat healthy diets, she said. “Persons with porphyria are encouraged to have a plan for home management of an attack and when to come to the hospital for symptoms that don’t respond to home therapy,” she said.
Dr. Fertrin said “the typical treatment for an acute porphyric attack is the use of intravenous glucose, as well as intravenous hemin infusions, along with pain medications and fluids as needed. Some patients may need medication for high blood pressure. For patients with recurrent attacks, prophylactic doses of hemin can be used.”
Another option is givosiran (Givlaari), the biggest recent advance in porphyria treatment. It was approved by the Food and Drug Administration for acute hepatic porphyria in 2019. “It’s an inhibitory mRNA that shuts down heme synthesis by downregulating aminolevulinic acid synthase,” said hematologist Thomas DeLoughery, MD, of Oregon Health & Science University, Portland.
A 2020 study coauthored by Dr. Bonkovsky found that the drug reduced the mean annualized attack rate in acute intermittent porphyria by 74%, compared with placebo (P < .001). “Givosiran led to lower levels of urinary ALA and porphobilinogen, fewer days of hemin use, and better daily scores for pain than placebo,” the study authors write. “Key adverse events that were observed more frequently in the givosiran group were elevations in serum aminotransferase levels, changes in serum creatinine levels and the estimated glomerular filtration rate, and injection-site reactions.”
Dr. Bonkovsky said the drug is appropriate in patients whose disease is not controlled by lifestyle interventions regarding alcohol, smoking, and substance use. However, while givosiran is helpful, “it’s not a cure,” he said. “Probably about 40% of patients have found the side effects such as nausea, fatigue, headaches, and liver injury to be too adverse and have not decided to take it in the long term.”
In addition, the drug costs about $500,000 a year, and insurers may balk at the expense. Still, specialists say it can be a good option. “Before givosiran, the standard therapy was hematin, which decreased the activity of the ALA synthase,” Dr. DeLoughery said. “This is a nasty drug that can inflame the blood vessels and is not all that effective.”
The royal family may be able to breathe easy
There are other forms of porphyria besides the acute type. Cutaneous porphryias can cause blistering from sunlight exposure, and treatment is avoidance of such triggers, Dr. Fertin said. “For a rare form called erythropoietic protoporphyria, there is a subcutaneous implant with a medication called afamelanotide that increases tolerance to sunlight.”
There’s another type: variegate porphyria, which UpToDate says is “characterized by cutaneous blistering and/or acute neurovisceral attacks.” In 1966, a mother-and-son psychiatrist team theorized that Britain’s King George III suffered from this type of porphyria.
However, researchers have sharply criticized the porphyria theory with regard to that king, and a recent theory now suggests that he actually had bipolar disorder. Dr. Fertin is among the skeptics, noting the high prevalence of porphyria in affected families.
“Since King George III would now have a living number of relatives of about 900 people, we would expect around 180 cases of variegate porphyria to be connected to him, and those are nowhere to be found,” he said. “The idea that it could cause progressive psychosis and dementia is also a misconception. We do not find that porphyria is the underlying cause of psychiatric conditions on their own. Many atypical symptoms of the king were attributed to variegate porphyria, and the few more typical symptoms he reportedly had, of abdominal pain and urine discoloration, can be more easily explained by common diseases, such as kidney stones or gallstones. Finally, attempts at obtaining genetic or biochemical evidence of porphyria from relatives of King George III have been negative or inconclusive.”
That’s good news for the royals, who have many other concerns these days.
Dr. Nance discloses research study funding from Alnylam (maker of Givlaari) and payments to participate in educational activities for patients and practitioners. Dr. Bonkovsky discloses relationships with drugmakers who have developed products to treat porphyria including Alnylam, Mitsubishi Tanabe, Recordati, and Disc. Dr. Fertin is subinvestigator in a clinical trial for a novel treatment for erythropoietic protoporphyria sponsored by Mitsubishi Tanabe. Dr. DeLoughery reports no disclosures.
European royal families may be enormously rich, but being a blueblood doesn’t always mean your blood is pristine. Queen Victoria’s DNA is famously believed to have silently bequeathed hemophilia to many of her descendants, including a great-grandson whose severe illness played a tragic role in spurring the Russian Revolution.
And that’s not all.
There’s plenty of skepticism about this theory, which seeks to explain the “madness” of King George III. But one thing is clear. If porphyria does indeed haunt the imperial bloodline that stretches to a new generation – the late Queen Elizabeth II’s great-grandchildren – any royal who’s afflicted going forward is likely to benefit mightily from modern treatment. While this disease may require lifelong vigilance, experts said in interviews that porphyria can often be controlled.
“If patients know they have the diagnosis, and they do the right things and avoid alcohol and risky drugs, most people will have few acute attacks,” said gastroenterologist Herbert Lloyd Bonkovsky, MD, of Wake Forest University, Winston-Salem, N.C., a leading porphyria specialist.
Heme infusions can also be helpful, he added, and the revolutionary new drug givosiran is available for those who suffer recurrent attacks. And “if all else fails, a successful liver transplant is curative” – as long as the transplanted liver doesn’t have porphyria, as happened in at least one case.
But, Dr. Bonkovsky cautioned, the diagnosis is often missed, in some cases for 15 years or more.
Diagnosing porphyria: Awareness and tests are crucial
Porphyria is caused when porphyins – essential components of hemoglobin – build up in the body, disrupting systems such as the nerves, skin, and gut. The urine can turn purplish, hence the condition’s name. (Porphyrus is the Greek word for purple.)
According to hematologist Danielle Nance, MD, of Banner MD Anderson Cancer Center in Gilbert, Ariz., acute intermittent porphyria “should be suspected in persons who have recurrent severe attacks of abdominal pain requiring strong pain medication to control symptoms, and there is no obvious physical cause.”
In such cases, practitioners should send out blood and urine for porphobilinogen (PBG) and delta-aminolevulinic acid (dALA or Delta-ALA) testing, Dr. Nance said. “These are almost always elevated, even between attacks, in persons with diagnoses of acute intermittent porphyria. Other types of porphyria, such as erythropoietic porphyria, may require additional testing. Genetic testing should be offered when a patient is suspected of having porphyria, as this can speed the diagnosis.”
The typical patient is a woman from age 18 to 55, often a young woman with recurrent abdominal pain that may occur during the second half of the menstrual cycle, Wake Forest’s Dr. Bonkovsky said. Constipation is common.
“She keeps coming to the clinic or emergency department, and no one knows what’s going on. Eventually, she tends to undergo an appendectomy, often a cholecystectomy, or sometimes gynecologic procedures without cure of the disease. Only after this long and arduous road of misdiagnosis does someone think it’s porphyria and do the correct tests.”
Dr. Bonkovsky led a 2014 study of 108 subjects (81% female) with acute porphyrias and found that the average time to a correct diagnosis was a whopping 15 years. Pain in the abdomen was the most common symptom (74%), followed by nausea/vomiting (73%), weakness (63%), and constipation (60%).
While underdiagnosis is common, porphyrias can also be overdiagnosed. According to Dr. Bonkovsky, a mild increase in urinary porphyrins is often misdiagnosed as porphyria when it may be a sign of liver disease or alcohol use, instead.
Hematologist Kleber Y. Fertrin, MD, PhD, of the University of Washington, Seattle, emphasized the importance of ordering the correct tests. “Urinary porphyrins are often inappropriately ordered because of their name. They are not diagnostic for acute hepatic porphyrias and may be nonspecifically elevated. It is paramount to get the labs from a reliable lab test experienced at performing diagnostic testing for porphyrias and make sure the urine sample needed is correctly obtained and preserved.”
New drug can control attacks, but it’s costly
Treatment should begin right away if porphyria is suspected, without waiting for confirmation, Dr. Nance said. “There are porphyria experts in the U.S. and in many other countries who can help practitioners interpret symptoms and testing results if there is uncertainty or if treatment options are unclear.”
Patients are counseled to avoid attack triggers and eat healthy diets, she said. “Persons with porphyria are encouraged to have a plan for home management of an attack and when to come to the hospital for symptoms that don’t respond to home therapy,” she said.
Dr. Fertrin said “the typical treatment for an acute porphyric attack is the use of intravenous glucose, as well as intravenous hemin infusions, along with pain medications and fluids as needed. Some patients may need medication for high blood pressure. For patients with recurrent attacks, prophylactic doses of hemin can be used.”
Another option is givosiran (Givlaari), the biggest recent advance in porphyria treatment. It was approved by the Food and Drug Administration for acute hepatic porphyria in 2019. “It’s an inhibitory mRNA that shuts down heme synthesis by downregulating aminolevulinic acid synthase,” said hematologist Thomas DeLoughery, MD, of Oregon Health & Science University, Portland.
A 2020 study coauthored by Dr. Bonkovsky found that the drug reduced the mean annualized attack rate in acute intermittent porphyria by 74%, compared with placebo (P < .001). “Givosiran led to lower levels of urinary ALA and porphobilinogen, fewer days of hemin use, and better daily scores for pain than placebo,” the study authors write. “Key adverse events that were observed more frequently in the givosiran group were elevations in serum aminotransferase levels, changes in serum creatinine levels and the estimated glomerular filtration rate, and injection-site reactions.”
Dr. Bonkovsky said the drug is appropriate in patients whose disease is not controlled by lifestyle interventions regarding alcohol, smoking, and substance use. However, while givosiran is helpful, “it’s not a cure,” he said. “Probably about 40% of patients have found the side effects such as nausea, fatigue, headaches, and liver injury to be too adverse and have not decided to take it in the long term.”
In addition, the drug costs about $500,000 a year, and insurers may balk at the expense. Still, specialists say it can be a good option. “Before givosiran, the standard therapy was hematin, which decreased the activity of the ALA synthase,” Dr. DeLoughery said. “This is a nasty drug that can inflame the blood vessels and is not all that effective.”
The royal family may be able to breathe easy
There are other forms of porphyria besides the acute type. Cutaneous porphryias can cause blistering from sunlight exposure, and treatment is avoidance of such triggers, Dr. Fertin said. “For a rare form called erythropoietic protoporphyria, there is a subcutaneous implant with a medication called afamelanotide that increases tolerance to sunlight.”
There’s another type: variegate porphyria, which UpToDate says is “characterized by cutaneous blistering and/or acute neurovisceral attacks.” In 1966, a mother-and-son psychiatrist team theorized that Britain’s King George III suffered from this type of porphyria.
However, researchers have sharply criticized the porphyria theory with regard to that king, and a recent theory now suggests that he actually had bipolar disorder. Dr. Fertin is among the skeptics, noting the high prevalence of porphyria in affected families.
“Since King George III would now have a living number of relatives of about 900 people, we would expect around 180 cases of variegate porphyria to be connected to him, and those are nowhere to be found,” he said. “The idea that it could cause progressive psychosis and dementia is also a misconception. We do not find that porphyria is the underlying cause of psychiatric conditions on their own. Many atypical symptoms of the king were attributed to variegate porphyria, and the few more typical symptoms he reportedly had, of abdominal pain and urine discoloration, can be more easily explained by common diseases, such as kidney stones or gallstones. Finally, attempts at obtaining genetic or biochemical evidence of porphyria from relatives of King George III have been negative or inconclusive.”
That’s good news for the royals, who have many other concerns these days.
Dr. Nance discloses research study funding from Alnylam (maker of Givlaari) and payments to participate in educational activities for patients and practitioners. Dr. Bonkovsky discloses relationships with drugmakers who have developed products to treat porphyria including Alnylam, Mitsubishi Tanabe, Recordati, and Disc. Dr. Fertin is subinvestigator in a clinical trial for a novel treatment for erythropoietic protoporphyria sponsored by Mitsubishi Tanabe. Dr. DeLoughery reports no disclosures.
European royal families may be enormously rich, but being a blueblood doesn’t always mean your blood is pristine. Queen Victoria’s DNA is famously believed to have silently bequeathed hemophilia to many of her descendants, including a great-grandson whose severe illness played a tragic role in spurring the Russian Revolution.
And that’s not all.
There’s plenty of skepticism about this theory, which seeks to explain the “madness” of King George III. But one thing is clear. If porphyria does indeed haunt the imperial bloodline that stretches to a new generation – the late Queen Elizabeth II’s great-grandchildren – any royal who’s afflicted going forward is likely to benefit mightily from modern treatment. While this disease may require lifelong vigilance, experts said in interviews that porphyria can often be controlled.
“If patients know they have the diagnosis, and they do the right things and avoid alcohol and risky drugs, most people will have few acute attacks,” said gastroenterologist Herbert Lloyd Bonkovsky, MD, of Wake Forest University, Winston-Salem, N.C., a leading porphyria specialist.
Heme infusions can also be helpful, he added, and the revolutionary new drug givosiran is available for those who suffer recurrent attacks. And “if all else fails, a successful liver transplant is curative” – as long as the transplanted liver doesn’t have porphyria, as happened in at least one case.
But, Dr. Bonkovsky cautioned, the diagnosis is often missed, in some cases for 15 years or more.
Diagnosing porphyria: Awareness and tests are crucial
Porphyria is caused when porphyins – essential components of hemoglobin – build up in the body, disrupting systems such as the nerves, skin, and gut. The urine can turn purplish, hence the condition’s name. (Porphyrus is the Greek word for purple.)
According to hematologist Danielle Nance, MD, of Banner MD Anderson Cancer Center in Gilbert, Ariz., acute intermittent porphyria “should be suspected in persons who have recurrent severe attacks of abdominal pain requiring strong pain medication to control symptoms, and there is no obvious physical cause.”
In such cases, practitioners should send out blood and urine for porphobilinogen (PBG) and delta-aminolevulinic acid (dALA or Delta-ALA) testing, Dr. Nance said. “These are almost always elevated, even between attacks, in persons with diagnoses of acute intermittent porphyria. Other types of porphyria, such as erythropoietic porphyria, may require additional testing. Genetic testing should be offered when a patient is suspected of having porphyria, as this can speed the diagnosis.”
The typical patient is a woman from age 18 to 55, often a young woman with recurrent abdominal pain that may occur during the second half of the menstrual cycle, Wake Forest’s Dr. Bonkovsky said. Constipation is common.
“She keeps coming to the clinic or emergency department, and no one knows what’s going on. Eventually, she tends to undergo an appendectomy, often a cholecystectomy, or sometimes gynecologic procedures without cure of the disease. Only after this long and arduous road of misdiagnosis does someone think it’s porphyria and do the correct tests.”
Dr. Bonkovsky led a 2014 study of 108 subjects (81% female) with acute porphyrias and found that the average time to a correct diagnosis was a whopping 15 years. Pain in the abdomen was the most common symptom (74%), followed by nausea/vomiting (73%), weakness (63%), and constipation (60%).
While underdiagnosis is common, porphyrias can also be overdiagnosed. According to Dr. Bonkovsky, a mild increase in urinary porphyrins is often misdiagnosed as porphyria when it may be a sign of liver disease or alcohol use, instead.
Hematologist Kleber Y. Fertrin, MD, PhD, of the University of Washington, Seattle, emphasized the importance of ordering the correct tests. “Urinary porphyrins are often inappropriately ordered because of their name. They are not diagnostic for acute hepatic porphyrias and may be nonspecifically elevated. It is paramount to get the labs from a reliable lab test experienced at performing diagnostic testing for porphyrias and make sure the urine sample needed is correctly obtained and preserved.”
New drug can control attacks, but it’s costly
Treatment should begin right away if porphyria is suspected, without waiting for confirmation, Dr. Nance said. “There are porphyria experts in the U.S. and in many other countries who can help practitioners interpret symptoms and testing results if there is uncertainty or if treatment options are unclear.”
Patients are counseled to avoid attack triggers and eat healthy diets, she said. “Persons with porphyria are encouraged to have a plan for home management of an attack and when to come to the hospital for symptoms that don’t respond to home therapy,” she said.
Dr. Fertrin said “the typical treatment for an acute porphyric attack is the use of intravenous glucose, as well as intravenous hemin infusions, along with pain medications and fluids as needed. Some patients may need medication for high blood pressure. For patients with recurrent attacks, prophylactic doses of hemin can be used.”
Another option is givosiran (Givlaari), the biggest recent advance in porphyria treatment. It was approved by the Food and Drug Administration for acute hepatic porphyria in 2019. “It’s an inhibitory mRNA that shuts down heme synthesis by downregulating aminolevulinic acid synthase,” said hematologist Thomas DeLoughery, MD, of Oregon Health & Science University, Portland.
A 2020 study coauthored by Dr. Bonkovsky found that the drug reduced the mean annualized attack rate in acute intermittent porphyria by 74%, compared with placebo (P < .001). “Givosiran led to lower levels of urinary ALA and porphobilinogen, fewer days of hemin use, and better daily scores for pain than placebo,” the study authors write. “Key adverse events that were observed more frequently in the givosiran group were elevations in serum aminotransferase levels, changes in serum creatinine levels and the estimated glomerular filtration rate, and injection-site reactions.”
Dr. Bonkovsky said the drug is appropriate in patients whose disease is not controlled by lifestyle interventions regarding alcohol, smoking, and substance use. However, while givosiran is helpful, “it’s not a cure,” he said. “Probably about 40% of patients have found the side effects such as nausea, fatigue, headaches, and liver injury to be too adverse and have not decided to take it in the long term.”
In addition, the drug costs about $500,000 a year, and insurers may balk at the expense. Still, specialists say it can be a good option. “Before givosiran, the standard therapy was hematin, which decreased the activity of the ALA synthase,” Dr. DeLoughery said. “This is a nasty drug that can inflame the blood vessels and is not all that effective.”
The royal family may be able to breathe easy
There are other forms of porphyria besides the acute type. Cutaneous porphryias can cause blistering from sunlight exposure, and treatment is avoidance of such triggers, Dr. Fertin said. “For a rare form called erythropoietic protoporphyria, there is a subcutaneous implant with a medication called afamelanotide that increases tolerance to sunlight.”
There’s another type: variegate porphyria, which UpToDate says is “characterized by cutaneous blistering and/or acute neurovisceral attacks.” In 1966, a mother-and-son psychiatrist team theorized that Britain’s King George III suffered from this type of porphyria.
However, researchers have sharply criticized the porphyria theory with regard to that king, and a recent theory now suggests that he actually had bipolar disorder. Dr. Fertin is among the skeptics, noting the high prevalence of porphyria in affected families.
“Since King George III would now have a living number of relatives of about 900 people, we would expect around 180 cases of variegate porphyria to be connected to him, and those are nowhere to be found,” he said. “The idea that it could cause progressive psychosis and dementia is also a misconception. We do not find that porphyria is the underlying cause of psychiatric conditions on their own. Many atypical symptoms of the king were attributed to variegate porphyria, and the few more typical symptoms he reportedly had, of abdominal pain and urine discoloration, can be more easily explained by common diseases, such as kidney stones or gallstones. Finally, attempts at obtaining genetic or biochemical evidence of porphyria from relatives of King George III have been negative or inconclusive.”
That’s good news for the royals, who have many other concerns these days.
Dr. Nance discloses research study funding from Alnylam (maker of Givlaari) and payments to participate in educational activities for patients and practitioners. Dr. Bonkovsky discloses relationships with drugmakers who have developed products to treat porphyria including Alnylam, Mitsubishi Tanabe, Recordati, and Disc. Dr. Fertin is subinvestigator in a clinical trial for a novel treatment for erythropoietic protoporphyria sponsored by Mitsubishi Tanabe. Dr. DeLoughery reports no disclosures.
Biden’s Cancer Moonshot turns its focus to early-detection blood tests
There’s big buzz about the hot prospects for blood tests designed to detect multiple kinds of cancer. President Biden highlighted them in a speech about the Cancer Moonshot program on Sept. 12, just a day after study results touted an experimental test’s ability to detect dozens of kinds of cancer. Meanwhile, the federal government is heralding an upcoming trial that will eventually enroll as many as 225,000 subjects.
There are plenty of reasons to be cautious, however. And if these tests become standard, the oncology field will need to figure out how to navigate a thicket of new challenges.
“Our friends in internal medicine and primary care will be looking to us for guidance. We need to make sure that we’re coming at this without too much optimism before we really have the data,” said Jyoti D. Patel, MD, medical director of thoracic oncology and assistant director for clinical research at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago.
Dr. Patel is a member of the communications workgroup of the Multicancer Early Detection Consortium, a nonprofit, public-private organization that’s providing insight and guidance into the development of screening tests. The consortium published a position paper earlier this year.
According to Dr. Patel, early cancer screening today can detect only five types of cancer: prostate, breast, lung, cervical, and colon. The Cancer Moonshot program has prioritized research into greatly expanding this number. President Biden referred to this goal in his Sept. 12 speech: “Imagine a simple blood test during an annual physical that could detect cancer early, where the chances of a cure are best.”
Biden said the National Cancer Institute is launching a major trial as part of the Cancer Moonshot program. The Vanguard Study on Multi-Cancer Detection plans to enlist 25,000 healthy women and men between 45 and 70 years old in 2024, then later enroll as many as 225,000 people.
Meanwhile, researchers reported on Sept. 11 that the Galleri multicancer detection blood test found positive cancer signals in 1.4% of 6,621 healthy subjects, and cancer was ultimately confirmed in 38% of those in that group. Nineteen solid tumors and 17 hematologic cancers were diagnosed; 26 of these were cancer types that don’t have routine screening available.
The Galleri test is widely available in the United States, although the $950 cost is not covered by insurance.
While the data is exciting, the high false-positive rate is worrisome, Dr. Patel said. “Are there ways that we can further define that by cancer-risk assessment or by having better captures in our technology that reflect RNA methylation or epigenetic changes that may lead to susceptibility to cancers?”
Additional research is essential
Ernest Hawk, MD, vice president and division head of cancer prevention and population sciences at the University of Texas MD Anderson Cancer Center, Houston, said it’s “absolutely essential” that research into screening tests clearly demonstrates improved patient outcomes over time.
“We need to have much longer follow-up of all participants – whether the screening results are positive or negative – and mitigate the potential risks of such testing,” said Dr. Hawk, who’s worked with the Multicancer Early Detection Consortium.
On another front, Northwestern University’s Dr. Patel highlighted that while easy-to-access cancer screening could create tremendous opportunities to treat early cancer and shrink disparities in care, it may produce “an onslaught of patients with early-stage disease. Do we have the workforce to help us?” Also, she said, “if we find a patient with early-stage disease, how are we going to risk-stratify their follow-up and adjuvant therapy? Are there ways to prognosticate with more granularity than we do now?”
What’s next? “Multicancer early-detection tests could truly revolutionize cancer care if they work as we hope they will, but only time, extensive participation in research, and hard work will prove whether that is true or not,” said MD Anderson’s Dr. Hawk. “I anticipate that we’ll have reasonable answers within the next decade, given the pace of existing company-sponsored research and NCI’s planned involvement in testing various technologies available.”
For her part, Dr. Patel said oncologists should be aware that multicancer screening tests are available and be ready to address questions about them. “Think about how you can advise patients in the absence of data,” she said.
Dr. Patel and Dr. Hawk have no relevant disclosures.
There’s big buzz about the hot prospects for blood tests designed to detect multiple kinds of cancer. President Biden highlighted them in a speech about the Cancer Moonshot program on Sept. 12, just a day after study results touted an experimental test’s ability to detect dozens of kinds of cancer. Meanwhile, the federal government is heralding an upcoming trial that will eventually enroll as many as 225,000 subjects.
There are plenty of reasons to be cautious, however. And if these tests become standard, the oncology field will need to figure out how to navigate a thicket of new challenges.
“Our friends in internal medicine and primary care will be looking to us for guidance. We need to make sure that we’re coming at this without too much optimism before we really have the data,” said Jyoti D. Patel, MD, medical director of thoracic oncology and assistant director for clinical research at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago.
Dr. Patel is a member of the communications workgroup of the Multicancer Early Detection Consortium, a nonprofit, public-private organization that’s providing insight and guidance into the development of screening tests. The consortium published a position paper earlier this year.
According to Dr. Patel, early cancer screening today can detect only five types of cancer: prostate, breast, lung, cervical, and colon. The Cancer Moonshot program has prioritized research into greatly expanding this number. President Biden referred to this goal in his Sept. 12 speech: “Imagine a simple blood test during an annual physical that could detect cancer early, where the chances of a cure are best.”
Biden said the National Cancer Institute is launching a major trial as part of the Cancer Moonshot program. The Vanguard Study on Multi-Cancer Detection plans to enlist 25,000 healthy women and men between 45 and 70 years old in 2024, then later enroll as many as 225,000 people.
Meanwhile, researchers reported on Sept. 11 that the Galleri multicancer detection blood test found positive cancer signals in 1.4% of 6,621 healthy subjects, and cancer was ultimately confirmed in 38% of those in that group. Nineteen solid tumors and 17 hematologic cancers were diagnosed; 26 of these were cancer types that don’t have routine screening available.
The Galleri test is widely available in the United States, although the $950 cost is not covered by insurance.
While the data is exciting, the high false-positive rate is worrisome, Dr. Patel said. “Are there ways that we can further define that by cancer-risk assessment or by having better captures in our technology that reflect RNA methylation or epigenetic changes that may lead to susceptibility to cancers?”
Additional research is essential
Ernest Hawk, MD, vice president and division head of cancer prevention and population sciences at the University of Texas MD Anderson Cancer Center, Houston, said it’s “absolutely essential” that research into screening tests clearly demonstrates improved patient outcomes over time.
“We need to have much longer follow-up of all participants – whether the screening results are positive or negative – and mitigate the potential risks of such testing,” said Dr. Hawk, who’s worked with the Multicancer Early Detection Consortium.
On another front, Northwestern University’s Dr. Patel highlighted that while easy-to-access cancer screening could create tremendous opportunities to treat early cancer and shrink disparities in care, it may produce “an onslaught of patients with early-stage disease. Do we have the workforce to help us?” Also, she said, “if we find a patient with early-stage disease, how are we going to risk-stratify their follow-up and adjuvant therapy? Are there ways to prognosticate with more granularity than we do now?”
What’s next? “Multicancer early-detection tests could truly revolutionize cancer care if they work as we hope they will, but only time, extensive participation in research, and hard work will prove whether that is true or not,” said MD Anderson’s Dr. Hawk. “I anticipate that we’ll have reasonable answers within the next decade, given the pace of existing company-sponsored research and NCI’s planned involvement in testing various technologies available.”
For her part, Dr. Patel said oncologists should be aware that multicancer screening tests are available and be ready to address questions about them. “Think about how you can advise patients in the absence of data,” she said.
Dr. Patel and Dr. Hawk have no relevant disclosures.
There’s big buzz about the hot prospects for blood tests designed to detect multiple kinds of cancer. President Biden highlighted them in a speech about the Cancer Moonshot program on Sept. 12, just a day after study results touted an experimental test’s ability to detect dozens of kinds of cancer. Meanwhile, the federal government is heralding an upcoming trial that will eventually enroll as many as 225,000 subjects.
There are plenty of reasons to be cautious, however. And if these tests become standard, the oncology field will need to figure out how to navigate a thicket of new challenges.
“Our friends in internal medicine and primary care will be looking to us for guidance. We need to make sure that we’re coming at this without too much optimism before we really have the data,” said Jyoti D. Patel, MD, medical director of thoracic oncology and assistant director for clinical research at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago.
Dr. Patel is a member of the communications workgroup of the Multicancer Early Detection Consortium, a nonprofit, public-private organization that’s providing insight and guidance into the development of screening tests. The consortium published a position paper earlier this year.
According to Dr. Patel, early cancer screening today can detect only five types of cancer: prostate, breast, lung, cervical, and colon. The Cancer Moonshot program has prioritized research into greatly expanding this number. President Biden referred to this goal in his Sept. 12 speech: “Imagine a simple blood test during an annual physical that could detect cancer early, where the chances of a cure are best.”
Biden said the National Cancer Institute is launching a major trial as part of the Cancer Moonshot program. The Vanguard Study on Multi-Cancer Detection plans to enlist 25,000 healthy women and men between 45 and 70 years old in 2024, then later enroll as many as 225,000 people.
Meanwhile, researchers reported on Sept. 11 that the Galleri multicancer detection blood test found positive cancer signals in 1.4% of 6,621 healthy subjects, and cancer was ultimately confirmed in 38% of those in that group. Nineteen solid tumors and 17 hematologic cancers were diagnosed; 26 of these were cancer types that don’t have routine screening available.
The Galleri test is widely available in the United States, although the $950 cost is not covered by insurance.
While the data is exciting, the high false-positive rate is worrisome, Dr. Patel said. “Are there ways that we can further define that by cancer-risk assessment or by having better captures in our technology that reflect RNA methylation or epigenetic changes that may lead to susceptibility to cancers?”
Additional research is essential
Ernest Hawk, MD, vice president and division head of cancer prevention and population sciences at the University of Texas MD Anderson Cancer Center, Houston, said it’s “absolutely essential” that research into screening tests clearly demonstrates improved patient outcomes over time.
“We need to have much longer follow-up of all participants – whether the screening results are positive or negative – and mitigate the potential risks of such testing,” said Dr. Hawk, who’s worked with the Multicancer Early Detection Consortium.
On another front, Northwestern University’s Dr. Patel highlighted that while easy-to-access cancer screening could create tremendous opportunities to treat early cancer and shrink disparities in care, it may produce “an onslaught of patients with early-stage disease. Do we have the workforce to help us?” Also, she said, “if we find a patient with early-stage disease, how are we going to risk-stratify their follow-up and adjuvant therapy? Are there ways to prognosticate with more granularity than we do now?”
What’s next? “Multicancer early-detection tests could truly revolutionize cancer care if they work as we hope they will, but only time, extensive participation in research, and hard work will prove whether that is true or not,” said MD Anderson’s Dr. Hawk. “I anticipate that we’ll have reasonable answers within the next decade, given the pace of existing company-sponsored research and NCI’s planned involvement in testing various technologies available.”
For her part, Dr. Patel said oncologists should be aware that multicancer screening tests are available and be ready to address questions about them. “Think about how you can advise patients in the absence of data,” she said.
Dr. Patel and Dr. Hawk have no relevant disclosures.
VA Launches Virtual Tumor Board
SAN DIEGO – The US Department of Veterans Affairs (VA) TeleOncology program has rolled out a virtual tumor board that brings medical professionals together to offer insight and guidance about challenging hematology cases. Over the past 6 months the board has held 10 sessions and reviewed about 20 cases. A small survey found that participants think the meetings are beneficial.
“Virtual tumor boards help to connect experts across the country to leverage the expertise within the VA,” he-matologist/oncologist Thomas Rodgers, MD, of the Duke Cancer Institute and Durham Veterans Affairs Medical Center, told Federal Practitioner in an interview. He is the lead author of a poster about the program that was pre-sented here at the annual meeting of the Association of VA Hematology/Oncology (AVAHO).
As Dr. Rodgers noted, tumor boards are already in place at some VA centers. However, “they are not available at every VA and often are not set up to cover every cancer type.”
The VA National TeleOncology program created the virtual tumor board program as part of its mission to ex-tend hematology/oncology services across the system. “Cancer care has become increasingly complex. Beyond ad-vancing therapeutics, patient care often involves multiple specialties and medical disciplines,” Dr. Rodgers said. “A tumor board offers a forum for these specialists to communicate with each other in real time, not only to help estab-lish the correct diagnosis and stage of cancer but also to form a consensus on the most fitting treatment option. Think of it as getting all of the people involved in a person’s care in the same room.”
Currently, he said, the virtual tumor boards cover patients with malignant hematology diagnoses such as leuke-mia, multiple myeloma, and lymphomas. “We welcome submissions. If a provider is interested in submitting a case, they can email us and will be provided with a short intake form. Once submitted, we will collect necessary imaging and pathology for review. The provider will then present the patient case on the day of the tumor board.”
Typically, more than 30 medical professionals participate in the virtual tumor boards, Dr. Rodgers said, repre-senting medical oncology/hematology, pathology, radiology, palliative care, pharmacy, social work, and die-tary/nutrition.
According to the poster presented at AVAHO, 9 participants responded to a survey after 4 tumor board sessions. All found the boards to be beneficial or somewhat beneficial, and 55% reported that they were “highly applicable” to their practice.
Pathologist Claudio A. Mosse, MD, PhD, of Vanderbilt University Medical Center and VA Tennessee Valley Healthcare System, praised the virtual tumor board program. “It’s been incredibly useful from my end as a pathologist as it shows me which diagnoses are most challenging for my colleagues,” Dr. Mosse said in an inter-view. “Reviewing and then presenting these challenging cases forces me to go into the published literature to come to a unitary diagnosis based on the patient history, radiology, various laboratory tests, and the biopsy I was asked to review.”
He added that “as a pathologist, I learn so much from the hematologists as they discuss the possible therapeutic options, and that strengthens my ability as a pathologist because I have to understand how one diagnosis versus an-other affects their therapeutic decision tree.”
What’s next for the virtual tumor board program? The next step is to expand to solid tumors, said VA Pittsburgh Healthcare System hematologist/oncologist Vida Almario Passero, MD, MBA, chief medical officer of National TeleOncology, in an interview.
No disclosures were reported.
SAN DIEGO – The US Department of Veterans Affairs (VA) TeleOncology program has rolled out a virtual tumor board that brings medical professionals together to offer insight and guidance about challenging hematology cases. Over the past 6 months the board has held 10 sessions and reviewed about 20 cases. A small survey found that participants think the meetings are beneficial.
“Virtual tumor boards help to connect experts across the country to leverage the expertise within the VA,” he-matologist/oncologist Thomas Rodgers, MD, of the Duke Cancer Institute and Durham Veterans Affairs Medical Center, told Federal Practitioner in an interview. He is the lead author of a poster about the program that was pre-sented here at the annual meeting of the Association of VA Hematology/Oncology (AVAHO).
As Dr. Rodgers noted, tumor boards are already in place at some VA centers. However, “they are not available at every VA and often are not set up to cover every cancer type.”
The VA National TeleOncology program created the virtual tumor board program as part of its mission to ex-tend hematology/oncology services across the system. “Cancer care has become increasingly complex. Beyond ad-vancing therapeutics, patient care often involves multiple specialties and medical disciplines,” Dr. Rodgers said. “A tumor board offers a forum for these specialists to communicate with each other in real time, not only to help estab-lish the correct diagnosis and stage of cancer but also to form a consensus on the most fitting treatment option. Think of it as getting all of the people involved in a person’s care in the same room.”
Currently, he said, the virtual tumor boards cover patients with malignant hematology diagnoses such as leuke-mia, multiple myeloma, and lymphomas. “We welcome submissions. If a provider is interested in submitting a case, they can email us and will be provided with a short intake form. Once submitted, we will collect necessary imaging and pathology for review. The provider will then present the patient case on the day of the tumor board.”
Typically, more than 30 medical professionals participate in the virtual tumor boards, Dr. Rodgers said, repre-senting medical oncology/hematology, pathology, radiology, palliative care, pharmacy, social work, and die-tary/nutrition.
According to the poster presented at AVAHO, 9 participants responded to a survey after 4 tumor board sessions. All found the boards to be beneficial or somewhat beneficial, and 55% reported that they were “highly applicable” to their practice.
Pathologist Claudio A. Mosse, MD, PhD, of Vanderbilt University Medical Center and VA Tennessee Valley Healthcare System, praised the virtual tumor board program. “It’s been incredibly useful from my end as a pathologist as it shows me which diagnoses are most challenging for my colleagues,” Dr. Mosse said in an inter-view. “Reviewing and then presenting these challenging cases forces me to go into the published literature to come to a unitary diagnosis based on the patient history, radiology, various laboratory tests, and the biopsy I was asked to review.”
He added that “as a pathologist, I learn so much from the hematologists as they discuss the possible therapeutic options, and that strengthens my ability as a pathologist because I have to understand how one diagnosis versus an-other affects their therapeutic decision tree.”
What’s next for the virtual tumor board program? The next step is to expand to solid tumors, said VA Pittsburgh Healthcare System hematologist/oncologist Vida Almario Passero, MD, MBA, chief medical officer of National TeleOncology, in an interview.
No disclosures were reported.
SAN DIEGO – The US Department of Veterans Affairs (VA) TeleOncology program has rolled out a virtual tumor board that brings medical professionals together to offer insight and guidance about challenging hematology cases. Over the past 6 months the board has held 10 sessions and reviewed about 20 cases. A small survey found that participants think the meetings are beneficial.
“Virtual tumor boards help to connect experts across the country to leverage the expertise within the VA,” he-matologist/oncologist Thomas Rodgers, MD, of the Duke Cancer Institute and Durham Veterans Affairs Medical Center, told Federal Practitioner in an interview. He is the lead author of a poster about the program that was pre-sented here at the annual meeting of the Association of VA Hematology/Oncology (AVAHO).
As Dr. Rodgers noted, tumor boards are already in place at some VA centers. However, “they are not available at every VA and often are not set up to cover every cancer type.”
The VA National TeleOncology program created the virtual tumor board program as part of its mission to ex-tend hematology/oncology services across the system. “Cancer care has become increasingly complex. Beyond ad-vancing therapeutics, patient care often involves multiple specialties and medical disciplines,” Dr. Rodgers said. “A tumor board offers a forum for these specialists to communicate with each other in real time, not only to help estab-lish the correct diagnosis and stage of cancer but also to form a consensus on the most fitting treatment option. Think of it as getting all of the people involved in a person’s care in the same room.”
Currently, he said, the virtual tumor boards cover patients with malignant hematology diagnoses such as leuke-mia, multiple myeloma, and lymphomas. “We welcome submissions. If a provider is interested in submitting a case, they can email us and will be provided with a short intake form. Once submitted, we will collect necessary imaging and pathology for review. The provider will then present the patient case on the day of the tumor board.”
Typically, more than 30 medical professionals participate in the virtual tumor boards, Dr. Rodgers said, repre-senting medical oncology/hematology, pathology, radiology, palliative care, pharmacy, social work, and die-tary/nutrition.
According to the poster presented at AVAHO, 9 participants responded to a survey after 4 tumor board sessions. All found the boards to be beneficial or somewhat beneficial, and 55% reported that they were “highly applicable” to their practice.
Pathologist Claudio A. Mosse, MD, PhD, of Vanderbilt University Medical Center and VA Tennessee Valley Healthcare System, praised the virtual tumor board program. “It’s been incredibly useful from my end as a pathologist as it shows me which diagnoses are most challenging for my colleagues,” Dr. Mosse said in an inter-view. “Reviewing and then presenting these challenging cases forces me to go into the published literature to come to a unitary diagnosis based on the patient history, radiology, various laboratory tests, and the biopsy I was asked to review.”
He added that “as a pathologist, I learn so much from the hematologists as they discuss the possible therapeutic options, and that strengthens my ability as a pathologist because I have to understand how one diagnosis versus an-other affects their therapeutic decision tree.”
What’s next for the virtual tumor board program? The next step is to expand to solid tumors, said VA Pittsburgh Healthcare System hematologist/oncologist Vida Almario Passero, MD, MBA, chief medical officer of National TeleOncology, in an interview.
No disclosures were reported.
New Research Supports a Changing Approach to Peripheral Artery Disease
SAN DIEGO–A cardiologist/vascular medicine specialist urged hematologist and oncologists within the US Department of Veterans Affairs system to think beyond the guidelines–at least until they’re updated–when they consider how to treat peripheral artery disease (PAD).
The 2016 American College of Cardiology/American Heart Association guidelines for PAD care are due for an update and don’t reflect recent positive research into the role that the blood thinner rivaroxaban can play in certain patients, said Geoffrey Barnes, MD, MSc, of the University of Michigan Health System, in a presentation here at the annual meeting of the Association of VA Hematology/Oncology (AVAHO).
Recent research has “really got us excited about the potential for this drug in this particular patient population,” Barnes said, although he cautioned that it’s most appropriate for patients at highest risk of PAD.
Research has found that patients with PAD are more likely to develop cancer, apparently because of common risk factors, and there’s discussion about whether they should undergo special screening. Cancer treatment may also boost the risk of PAD, according to a 2021 US study that tracked 248 patients with both breast cancer and PAD. “Of all patients, 48% were on statins and 54% were on antiplatelet therapies,” the study found, although the 2016 guidelines recommend both (statins for all patients with PAD, antiplatelets for those with symptoms).
In his presentation, Barnes noted that the 2016 guidelines specifically recommend aspirin (75-325 mg daily) or clopidogrel (75 mg) in patients with symptomatic PAD. Treatment is especially important, he said, because the risk of cardiovascular mortality in PAD is high. A 2020 study found that 9.1% of 13,885 patients died over a median 30-month follow-up.
The good news about treatment Brand said, came in a 2020 industry-funded study of patients with PAD who had undergone revascularization. Various outcomes such as amputation, heart attack, and death from cardiovascular causes—the primary efficacy outcome—were less common in subjects who took 2.5 mg twice daily of rivaroxaban plus aspirin or placebo plus aspirin (hazard ratio, 0.85, 95% CI, 0.76-0.96; P = .009).
So who should go on rivaroxaban? As Brand noted, a 2019 study found that patients with no high-risk features didn’t benefit much in terms of risk of vascular events, but those with high-risk features did. In higher-risk patients, the study found, “rivaroxaban and aspirin prevented 33 serious vascular events, whereas in lower-risk patients, rivaroxaban and aspirin treatment led to the avoidance of 10 events per 1,000 patients treated for 30 months.”
Per the study, patients at higher risk are those with heart failure, at least 2 vascular beds affected, renal insufficiency, or diabetes.
Brand supports the use of rivaroxaban in these patients. However, he cautioned colleagues not to switch out the drug with apixaban, another blood thinner. “These are not interchangeable,” he said. “You do need to stick with rivaroxaban. And you do need to remember that you’re going to use 2.5 milligrams twice a day—very different than many of the other ways we are using rivaroxaban.”
Brand discloses consulting fees (Pfizer/Bristol-Myers Squib, Janssen, Acelis Connected Health, Boston Scientific, Abbott Vascular), grant funding (Boston Scientific) and board of directors service (Anticoagulation Forum).
SAN DIEGO–A cardiologist/vascular medicine specialist urged hematologist and oncologists within the US Department of Veterans Affairs system to think beyond the guidelines–at least until they’re updated–when they consider how to treat peripheral artery disease (PAD).
The 2016 American College of Cardiology/American Heart Association guidelines for PAD care are due for an update and don’t reflect recent positive research into the role that the blood thinner rivaroxaban can play in certain patients, said Geoffrey Barnes, MD, MSc, of the University of Michigan Health System, in a presentation here at the annual meeting of the Association of VA Hematology/Oncology (AVAHO).
Recent research has “really got us excited about the potential for this drug in this particular patient population,” Barnes said, although he cautioned that it’s most appropriate for patients at highest risk of PAD.
Research has found that patients with PAD are more likely to develop cancer, apparently because of common risk factors, and there’s discussion about whether they should undergo special screening. Cancer treatment may also boost the risk of PAD, according to a 2021 US study that tracked 248 patients with both breast cancer and PAD. “Of all patients, 48% were on statins and 54% were on antiplatelet therapies,” the study found, although the 2016 guidelines recommend both (statins for all patients with PAD, antiplatelets for those with symptoms).
In his presentation, Barnes noted that the 2016 guidelines specifically recommend aspirin (75-325 mg daily) or clopidogrel (75 mg) in patients with symptomatic PAD. Treatment is especially important, he said, because the risk of cardiovascular mortality in PAD is high. A 2020 study found that 9.1% of 13,885 patients died over a median 30-month follow-up.
The good news about treatment Brand said, came in a 2020 industry-funded study of patients with PAD who had undergone revascularization. Various outcomes such as amputation, heart attack, and death from cardiovascular causes—the primary efficacy outcome—were less common in subjects who took 2.5 mg twice daily of rivaroxaban plus aspirin or placebo plus aspirin (hazard ratio, 0.85, 95% CI, 0.76-0.96; P = .009).
So who should go on rivaroxaban? As Brand noted, a 2019 study found that patients with no high-risk features didn’t benefit much in terms of risk of vascular events, but those with high-risk features did. In higher-risk patients, the study found, “rivaroxaban and aspirin prevented 33 serious vascular events, whereas in lower-risk patients, rivaroxaban and aspirin treatment led to the avoidance of 10 events per 1,000 patients treated for 30 months.”
Per the study, patients at higher risk are those with heart failure, at least 2 vascular beds affected, renal insufficiency, or diabetes.
Brand supports the use of rivaroxaban in these patients. However, he cautioned colleagues not to switch out the drug with apixaban, another blood thinner. “These are not interchangeable,” he said. “You do need to stick with rivaroxaban. And you do need to remember that you’re going to use 2.5 milligrams twice a day—very different than many of the other ways we are using rivaroxaban.”
Brand discloses consulting fees (Pfizer/Bristol-Myers Squib, Janssen, Acelis Connected Health, Boston Scientific, Abbott Vascular), grant funding (Boston Scientific) and board of directors service (Anticoagulation Forum).
SAN DIEGO–A cardiologist/vascular medicine specialist urged hematologist and oncologists within the US Department of Veterans Affairs system to think beyond the guidelines–at least until they’re updated–when they consider how to treat peripheral artery disease (PAD).
The 2016 American College of Cardiology/American Heart Association guidelines for PAD care are due for an update and don’t reflect recent positive research into the role that the blood thinner rivaroxaban can play in certain patients, said Geoffrey Barnes, MD, MSc, of the University of Michigan Health System, in a presentation here at the annual meeting of the Association of VA Hematology/Oncology (AVAHO).
Recent research has “really got us excited about the potential for this drug in this particular patient population,” Barnes said, although he cautioned that it’s most appropriate for patients at highest risk of PAD.
Research has found that patients with PAD are more likely to develop cancer, apparently because of common risk factors, and there’s discussion about whether they should undergo special screening. Cancer treatment may also boost the risk of PAD, according to a 2021 US study that tracked 248 patients with both breast cancer and PAD. “Of all patients, 48% were on statins and 54% were on antiplatelet therapies,” the study found, although the 2016 guidelines recommend both (statins for all patients with PAD, antiplatelets for those with symptoms).
In his presentation, Barnes noted that the 2016 guidelines specifically recommend aspirin (75-325 mg daily) or clopidogrel (75 mg) in patients with symptomatic PAD. Treatment is especially important, he said, because the risk of cardiovascular mortality in PAD is high. A 2020 study found that 9.1% of 13,885 patients died over a median 30-month follow-up.
The good news about treatment Brand said, came in a 2020 industry-funded study of patients with PAD who had undergone revascularization. Various outcomes such as amputation, heart attack, and death from cardiovascular causes—the primary efficacy outcome—were less common in subjects who took 2.5 mg twice daily of rivaroxaban plus aspirin or placebo plus aspirin (hazard ratio, 0.85, 95% CI, 0.76-0.96; P = .009).
So who should go on rivaroxaban? As Brand noted, a 2019 study found that patients with no high-risk features didn’t benefit much in terms of risk of vascular events, but those with high-risk features did. In higher-risk patients, the study found, “rivaroxaban and aspirin prevented 33 serious vascular events, whereas in lower-risk patients, rivaroxaban and aspirin treatment led to the avoidance of 10 events per 1,000 patients treated for 30 months.”
Per the study, patients at higher risk are those with heart failure, at least 2 vascular beds affected, renal insufficiency, or diabetes.
Brand supports the use of rivaroxaban in these patients. However, he cautioned colleagues not to switch out the drug with apixaban, another blood thinner. “These are not interchangeable,” he said. “You do need to stick with rivaroxaban. And you do need to remember that you’re going to use 2.5 milligrams twice a day—very different than many of the other ways we are using rivaroxaban.”
Brand discloses consulting fees (Pfizer/Bristol-Myers Squib, Janssen, Acelis Connected Health, Boston Scientific, Abbott Vascular), grant funding (Boston Scientific) and board of directors service (Anticoagulation Forum).
Time to pull back on postsurgery radiation in breast cancer?
A new study suggests that oncologists can safely pull back on standard locoregional radiotherapy (RT) in select patients with cT1-2N1 breast cancer who are treated with primary chemotherapy prior to surgery. The key is to divide patients by risk level and treat them according to the study’s guidelines, the researchers reported.
lead study author Sabine de Wild, MD, a PhD student at Maastricht (the Netherlands) University Medical Center, said in an interview.
The study, published in The Lancet Oncology, was intended to provide insight into which breast cancer patients need adjuvant locoregional radiotherapy following postchemotherapy surgery, coauthor Liesbeth Boersma, MD, PhD, a radiation oncologist at Maastricht University Medical Center, said in an interview. “It is not yet known which of these patients would benefit from adjuvant locoregional radiotherapy and to what extent the response of the tumor to the chemotherapy should be taken into account.”
For the study, believed to be the first prospective analysis tackling this topic, researchers tracked 838 patients in The Netherlands who were treated for cT1-2N1 breast cancer with primary chemotherapy and surgery of the breast and axilla from 2011-2015. Tumors were less than 5 cm and metastases were one to three axillary nodes.
The subjects were divided into groups based on risk of locoregional recurrence, and each group underwent different therapies.
- Low-risk group: no metastases were present in the nodes (n = 291). “We omitted regional radiotherapy, and we omitted RT of the chest wall in case of a mastectomy. After breast conserving surgery, regular RT of the breast was recommended,” Dr. de Wild said.
- Intermediate-risk group, one to three metastases were still present (n = 370). “We omitted regional radiotherapy, but irradiated the chest wall or breast,” she said.
- High-risk group, three metastases were present (n = 177). “We did not de-escalate, and all patients were treated with locoregional RT,” she said.
According to the study, “the 5-year locoregional recurrence rate in all patients was 2.2% (95% confidence interval, 1.4-3.4). The 5-year locoregional recurrence rate was 2.1% (95% CI, 0.9-4.3) in the low-risk group, 2.2% (95% CI, 1.0-4.1) in the intermediate-risk group, and 2.3% (95% CI, 0.8-5.5) in the high-risk group.”
In 26% of cases, patients received more radiotherapy than the study guidelines suggested. “Remarkably,” the researchers wrote, “this did not seem to affect locoregional recurrence rate, recurrence-free interval, and overall survival in a statistically significant or clinically relevant way.”
As for limitations, the authors noted that, “in each risk group, the actual sample size treated according to the study guideline was smaller than required based on the power calculation. Nevertheless, when performing the analyses in the subset of patients treated according to the study guideline, the upper limit of 95% CI of 5-year locoregional recurrence rate did not exceed 7.8%.”
The study authors wrote that, “in the future, the results of this study might lead to more frequent omission of locoregional radiotherapy, which could result in lower morbidity and a better quality of life for patients with breast cancer who are receiving primary chemotherapy.”
However, Dr. de Wild said randomized trials are necessary “to investigate how treatment can be individualized further, i.e., by taking into account specific tumor characteristics.” Also, most patients in the study underwent axillary lymph node dissection, “while patients in daily practice may instead undergo targeted axillary dissection. Future studies are needed to determine if less radiotherapy is also safe in patients in whom axillary lymph node dissection is omitted.”
The study was funded by the Dutch Cancer Society. One coauthor reported a pending patent plus grants from AstraZeneca, Eurocept Plaza, Roche, Genentech, Gilead Sciences, Tesaro, Novartis, Dutch Cancer Society, ZonMw, and A Sister’s Hope; as well as consulting fees and other financial support from a variety of pharmaceutical companies. The other authors had no disclosures.
A new study suggests that oncologists can safely pull back on standard locoregional radiotherapy (RT) in select patients with cT1-2N1 breast cancer who are treated with primary chemotherapy prior to surgery. The key is to divide patients by risk level and treat them according to the study’s guidelines, the researchers reported.
lead study author Sabine de Wild, MD, a PhD student at Maastricht (the Netherlands) University Medical Center, said in an interview.
The study, published in The Lancet Oncology, was intended to provide insight into which breast cancer patients need adjuvant locoregional radiotherapy following postchemotherapy surgery, coauthor Liesbeth Boersma, MD, PhD, a radiation oncologist at Maastricht University Medical Center, said in an interview. “It is not yet known which of these patients would benefit from adjuvant locoregional radiotherapy and to what extent the response of the tumor to the chemotherapy should be taken into account.”
For the study, believed to be the first prospective analysis tackling this topic, researchers tracked 838 patients in The Netherlands who were treated for cT1-2N1 breast cancer with primary chemotherapy and surgery of the breast and axilla from 2011-2015. Tumors were less than 5 cm and metastases were one to three axillary nodes.
The subjects were divided into groups based on risk of locoregional recurrence, and each group underwent different therapies.
- Low-risk group: no metastases were present in the nodes (n = 291). “We omitted regional radiotherapy, and we omitted RT of the chest wall in case of a mastectomy. After breast conserving surgery, regular RT of the breast was recommended,” Dr. de Wild said.
- Intermediate-risk group, one to three metastases were still present (n = 370). “We omitted regional radiotherapy, but irradiated the chest wall or breast,” she said.
- High-risk group, three metastases were present (n = 177). “We did not de-escalate, and all patients were treated with locoregional RT,” she said.
According to the study, “the 5-year locoregional recurrence rate in all patients was 2.2% (95% confidence interval, 1.4-3.4). The 5-year locoregional recurrence rate was 2.1% (95% CI, 0.9-4.3) in the low-risk group, 2.2% (95% CI, 1.0-4.1) in the intermediate-risk group, and 2.3% (95% CI, 0.8-5.5) in the high-risk group.”
In 26% of cases, patients received more radiotherapy than the study guidelines suggested. “Remarkably,” the researchers wrote, “this did not seem to affect locoregional recurrence rate, recurrence-free interval, and overall survival in a statistically significant or clinically relevant way.”
As for limitations, the authors noted that, “in each risk group, the actual sample size treated according to the study guideline was smaller than required based on the power calculation. Nevertheless, when performing the analyses in the subset of patients treated according to the study guideline, the upper limit of 95% CI of 5-year locoregional recurrence rate did not exceed 7.8%.”
The study authors wrote that, “in the future, the results of this study might lead to more frequent omission of locoregional radiotherapy, which could result in lower morbidity and a better quality of life for patients with breast cancer who are receiving primary chemotherapy.”
However, Dr. de Wild said randomized trials are necessary “to investigate how treatment can be individualized further, i.e., by taking into account specific tumor characteristics.” Also, most patients in the study underwent axillary lymph node dissection, “while patients in daily practice may instead undergo targeted axillary dissection. Future studies are needed to determine if less radiotherapy is also safe in patients in whom axillary lymph node dissection is omitted.”
The study was funded by the Dutch Cancer Society. One coauthor reported a pending patent plus grants from AstraZeneca, Eurocept Plaza, Roche, Genentech, Gilead Sciences, Tesaro, Novartis, Dutch Cancer Society, ZonMw, and A Sister’s Hope; as well as consulting fees and other financial support from a variety of pharmaceutical companies. The other authors had no disclosures.
A new study suggests that oncologists can safely pull back on standard locoregional radiotherapy (RT) in select patients with cT1-2N1 breast cancer who are treated with primary chemotherapy prior to surgery. The key is to divide patients by risk level and treat them according to the study’s guidelines, the researchers reported.
lead study author Sabine de Wild, MD, a PhD student at Maastricht (the Netherlands) University Medical Center, said in an interview.
The study, published in The Lancet Oncology, was intended to provide insight into which breast cancer patients need adjuvant locoregional radiotherapy following postchemotherapy surgery, coauthor Liesbeth Boersma, MD, PhD, a radiation oncologist at Maastricht University Medical Center, said in an interview. “It is not yet known which of these patients would benefit from adjuvant locoregional radiotherapy and to what extent the response of the tumor to the chemotherapy should be taken into account.”
For the study, believed to be the first prospective analysis tackling this topic, researchers tracked 838 patients in The Netherlands who were treated for cT1-2N1 breast cancer with primary chemotherapy and surgery of the breast and axilla from 2011-2015. Tumors were less than 5 cm and metastases were one to three axillary nodes.
The subjects were divided into groups based on risk of locoregional recurrence, and each group underwent different therapies.
- Low-risk group: no metastases were present in the nodes (n = 291). “We omitted regional radiotherapy, and we omitted RT of the chest wall in case of a mastectomy. After breast conserving surgery, regular RT of the breast was recommended,” Dr. de Wild said.
- Intermediate-risk group, one to three metastases were still present (n = 370). “We omitted regional radiotherapy, but irradiated the chest wall or breast,” she said.
- High-risk group, three metastases were present (n = 177). “We did not de-escalate, and all patients were treated with locoregional RT,” she said.
According to the study, “the 5-year locoregional recurrence rate in all patients was 2.2% (95% confidence interval, 1.4-3.4). The 5-year locoregional recurrence rate was 2.1% (95% CI, 0.9-4.3) in the low-risk group, 2.2% (95% CI, 1.0-4.1) in the intermediate-risk group, and 2.3% (95% CI, 0.8-5.5) in the high-risk group.”
In 26% of cases, patients received more radiotherapy than the study guidelines suggested. “Remarkably,” the researchers wrote, “this did not seem to affect locoregional recurrence rate, recurrence-free interval, and overall survival in a statistically significant or clinically relevant way.”
As for limitations, the authors noted that, “in each risk group, the actual sample size treated according to the study guideline was smaller than required based on the power calculation. Nevertheless, when performing the analyses in the subset of patients treated according to the study guideline, the upper limit of 95% CI of 5-year locoregional recurrence rate did not exceed 7.8%.”
The study authors wrote that, “in the future, the results of this study might lead to more frequent omission of locoregional radiotherapy, which could result in lower morbidity and a better quality of life for patients with breast cancer who are receiving primary chemotherapy.”
However, Dr. de Wild said randomized trials are necessary “to investigate how treatment can be individualized further, i.e., by taking into account specific tumor characteristics.” Also, most patients in the study underwent axillary lymph node dissection, “while patients in daily practice may instead undergo targeted axillary dissection. Future studies are needed to determine if less radiotherapy is also safe in patients in whom axillary lymph node dissection is omitted.”
The study was funded by the Dutch Cancer Society. One coauthor reported a pending patent plus grants from AstraZeneca, Eurocept Plaza, Roche, Genentech, Gilead Sciences, Tesaro, Novartis, Dutch Cancer Society, ZonMw, and A Sister’s Hope; as well as consulting fees and other financial support from a variety of pharmaceutical companies. The other authors had no disclosures.
FROM THE LANCET ONCOLOGY
Oncologists often misinterpret posttreatment HNSCC scan details
Patient outcomes could be threatened because of misinterpretation by oncologic surgeons of free-form posttreatment radiological reports in head and neck squamous cell carcinoma (HNSCC), a new study finds.
“Clinician perception of patient response from the post-RT [radiation treament] PET/CT free-form report is unreliable and does not consistently reflect the radiologist’s intended meaning, which was strongly associated with survival,” researchers wrote in a study published Aug. 18 in JAMA Otolaryngology-Head & Neck Surgery. They found “minimal agreement between clinicians’ consensus perspective on the patient’s response status derived from free-form imaging reports and the criterion standard response category assigned by a nuclear medicine specialist after PET/CT image review.”
According to radiation oncologist Ryan T. Hughes, MD, and colleagues at Wake Forest University, Winston-Salem, N.C., it’s common for patients with HNSCC to get PET, CT, or PET/CT imaging following treatment in order to assess how patients responded. Accurate communication about the results is essential to determining next steps, they write.
However, they write, “to our knowledge there is no universally accepted standardized method for communicating results,” such as whether there’s been a complete or partial response. Discrepancies between a radiological posttreatment report and an oncologist’s perception of the findings “may contribute to unnecessary patient care complexities, including elevated patient anxiety, unnecessary follow-up testing/procedures, and failure to recognize and adequately treat residual, recurrent, or progressive disease,” the researchers write.
For the new study, the authors tracked 171 patients (26.3% women, median age 61 years, ethnicity not provided), mainly (87%) with stage III-IV disease. Most (89%) received concurrent chemotherapy, and 30% received radiotherapy following operations.
Four oncologists reviewed free-form radiologic reports and determined whether the patient had a complete, indeterminate or partial response, or progressive disease. “Next, the group conferred to assign a consensus clinician MDS [modified Deauville score] and associated response category to assess the percentage of agreement with the criterion standard nuclear medicine physician MDS response derived from PET/CT image review.”
The researchers found that “interrater reliability of clinician-perceived post-RT PET/CT response was moderate [k = 0.680; 95% confidence interval, 0.638-0.721], and there was minimal reliability and low rate of agreement between clinician perception and radiologist-intended PET/CT response [63.7%; k = 0.365; 95% CI, 0.251-0.478).”
The clinicians were more likely to perceive patients as having an indeterminate response (28.1%), compared with the radiologists (9.3%). “There were 16 instances of significant discordance: 7 patients for whom the clinician perception MDS was 1 to 2 and nuclear medicine MDS 3 to 4, and 9 patients for whom the clinician perception MDS was 3 to 4 and nuclear medicine MDS 1 to 2.”
Due to statistical limitations, the researchers were unable to link the MDS scores to prognoses. The researchers suggest it’s time to further standardize the assessment of posttreatment responses to therapy. They add that “the decision to use a standardized interpretation and reporting system rather than free-form reporting is more important than the specific system selected.”
As for next steps, the researchers report that “prospective studies of post-RT PET/CT standardized reporting among patients with HNSCC are warranted, and a prospective implementation study of this workflow is planned at our institution.”
The study was funded by the National Center for Advancing Translational Sciences and National Institutes of Health. The authors had no disclosures.
Patient outcomes could be threatened because of misinterpretation by oncologic surgeons of free-form posttreatment radiological reports in head and neck squamous cell carcinoma (HNSCC), a new study finds.
“Clinician perception of patient response from the post-RT [radiation treament] PET/CT free-form report is unreliable and does not consistently reflect the radiologist’s intended meaning, which was strongly associated with survival,” researchers wrote in a study published Aug. 18 in JAMA Otolaryngology-Head & Neck Surgery. They found “minimal agreement between clinicians’ consensus perspective on the patient’s response status derived from free-form imaging reports and the criterion standard response category assigned by a nuclear medicine specialist after PET/CT image review.”
According to radiation oncologist Ryan T. Hughes, MD, and colleagues at Wake Forest University, Winston-Salem, N.C., it’s common for patients with HNSCC to get PET, CT, or PET/CT imaging following treatment in order to assess how patients responded. Accurate communication about the results is essential to determining next steps, they write.
However, they write, “to our knowledge there is no universally accepted standardized method for communicating results,” such as whether there’s been a complete or partial response. Discrepancies between a radiological posttreatment report and an oncologist’s perception of the findings “may contribute to unnecessary patient care complexities, including elevated patient anxiety, unnecessary follow-up testing/procedures, and failure to recognize and adequately treat residual, recurrent, or progressive disease,” the researchers write.
For the new study, the authors tracked 171 patients (26.3% women, median age 61 years, ethnicity not provided), mainly (87%) with stage III-IV disease. Most (89%) received concurrent chemotherapy, and 30% received radiotherapy following operations.
Four oncologists reviewed free-form radiologic reports and determined whether the patient had a complete, indeterminate or partial response, or progressive disease. “Next, the group conferred to assign a consensus clinician MDS [modified Deauville score] and associated response category to assess the percentage of agreement with the criterion standard nuclear medicine physician MDS response derived from PET/CT image review.”
The researchers found that “interrater reliability of clinician-perceived post-RT PET/CT response was moderate [k = 0.680; 95% confidence interval, 0.638-0.721], and there was minimal reliability and low rate of agreement between clinician perception and radiologist-intended PET/CT response [63.7%; k = 0.365; 95% CI, 0.251-0.478).”
The clinicians were more likely to perceive patients as having an indeterminate response (28.1%), compared with the radiologists (9.3%). “There were 16 instances of significant discordance: 7 patients for whom the clinician perception MDS was 1 to 2 and nuclear medicine MDS 3 to 4, and 9 patients for whom the clinician perception MDS was 3 to 4 and nuclear medicine MDS 1 to 2.”
Due to statistical limitations, the researchers were unable to link the MDS scores to prognoses. The researchers suggest it’s time to further standardize the assessment of posttreatment responses to therapy. They add that “the decision to use a standardized interpretation and reporting system rather than free-form reporting is more important than the specific system selected.”
As for next steps, the researchers report that “prospective studies of post-RT PET/CT standardized reporting among patients with HNSCC are warranted, and a prospective implementation study of this workflow is planned at our institution.”
The study was funded by the National Center for Advancing Translational Sciences and National Institutes of Health. The authors had no disclosures.
Patient outcomes could be threatened because of misinterpretation by oncologic surgeons of free-form posttreatment radiological reports in head and neck squamous cell carcinoma (HNSCC), a new study finds.
“Clinician perception of patient response from the post-RT [radiation treament] PET/CT free-form report is unreliable and does not consistently reflect the radiologist’s intended meaning, which was strongly associated with survival,” researchers wrote in a study published Aug. 18 in JAMA Otolaryngology-Head & Neck Surgery. They found “minimal agreement between clinicians’ consensus perspective on the patient’s response status derived from free-form imaging reports and the criterion standard response category assigned by a nuclear medicine specialist after PET/CT image review.”
According to radiation oncologist Ryan T. Hughes, MD, and colleagues at Wake Forest University, Winston-Salem, N.C., it’s common for patients with HNSCC to get PET, CT, or PET/CT imaging following treatment in order to assess how patients responded. Accurate communication about the results is essential to determining next steps, they write.
However, they write, “to our knowledge there is no universally accepted standardized method for communicating results,” such as whether there’s been a complete or partial response. Discrepancies between a radiological posttreatment report and an oncologist’s perception of the findings “may contribute to unnecessary patient care complexities, including elevated patient anxiety, unnecessary follow-up testing/procedures, and failure to recognize and adequately treat residual, recurrent, or progressive disease,” the researchers write.
For the new study, the authors tracked 171 patients (26.3% women, median age 61 years, ethnicity not provided), mainly (87%) with stage III-IV disease. Most (89%) received concurrent chemotherapy, and 30% received radiotherapy following operations.
Four oncologists reviewed free-form radiologic reports and determined whether the patient had a complete, indeterminate or partial response, or progressive disease. “Next, the group conferred to assign a consensus clinician MDS [modified Deauville score] and associated response category to assess the percentage of agreement with the criterion standard nuclear medicine physician MDS response derived from PET/CT image review.”
The researchers found that “interrater reliability of clinician-perceived post-RT PET/CT response was moderate [k = 0.680; 95% confidence interval, 0.638-0.721], and there was minimal reliability and low rate of agreement between clinician perception and radiologist-intended PET/CT response [63.7%; k = 0.365; 95% CI, 0.251-0.478).”
The clinicians were more likely to perceive patients as having an indeterminate response (28.1%), compared with the radiologists (9.3%). “There were 16 instances of significant discordance: 7 patients for whom the clinician perception MDS was 1 to 2 and nuclear medicine MDS 3 to 4, and 9 patients for whom the clinician perception MDS was 3 to 4 and nuclear medicine MDS 1 to 2.”
Due to statistical limitations, the researchers were unable to link the MDS scores to prognoses. The researchers suggest it’s time to further standardize the assessment of posttreatment responses to therapy. They add that “the decision to use a standardized interpretation and reporting system rather than free-form reporting is more important than the specific system selected.”
As for next steps, the researchers report that “prospective studies of post-RT PET/CT standardized reporting among patients with HNSCC are warranted, and a prospective implementation study of this workflow is planned at our institution.”
The study was funded by the National Center for Advancing Translational Sciences and National Institutes of Health. The authors had no disclosures.
FROM JAMA OTOLARYNGOLOGY–HEAD & NECK SURGERY
