Patient & Survivor Care

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

A version of this article first appeared on Medscape.com.

TOPLINE:

Adults receiving cancer treatment who use cannabis perceived benefits regarding pain, sleep, nausea, and other factors but also reported worse physical and psychological symptoms.

METHODOLOGY:

• Participants included 267 adults (mean age, 58 years; 70% women; 88% White) undergoing treatment for cancer, most commonly breast (47%) and ovarian (29%).
• Participants completed online surveys to characterize cannabis use, reasons for using it, perceived benefits and harms, and physical/psychological symptoms.
• Participants who had used cannabis for more than 1 day during the previous 30 days were compared with those who had not.

TAKEAWAY:

• Overall, 26% of respondents reported cannabis use in the past 30 days, most often edibles (65%) or smoked cannabis (51%).
• Cannabis users were more likely to be younger, male, Black, to have lower income, worse physical/psychological symptoms, and to be disabled or unable to work in comparison with nonusers.
• Cannabis was used to treat pain, cancer, sleep problems, anxiety, nausea, and poor appetite; perceived benefits were greatest with respect to sleep, nausea, pain, muscle spasms, and anxiety.
• Despite perceived benefits, cannabis users reported worse overall distress, anxiety, sleep disturbances, appetite, nausea, fatigue, and pain.

IN PRACTICE:

“The study findings indicate that patients with cancer perceived benefits to using cannabis for many symptoms” but also revealed that “those who used cannabis in the past 30 days had significantly worse symptom profiles overall than those who did not use cannabis,” the authors wrote.

SOURCE:

The study, led by Desiree R. Azizoddin, PsyD, University of Oklahoma Health Science Center, Oklahoma City, was published online in Cancer.

LIMITATIONS:

It’s not known whether adults who used cannabis had significantly worse symptoms at the outset, which may have prompted cannabis use, or whether cannabis use may have exacerbated their symptoms.

DISCLOSURES:

Funding for the study was provided by grants from the National Cancer Institute and the Oklahoma Tobacco Settlement Endowment Trust. Nine of the 10 authors have disclosed no relevant conflicts of interest. One author has relationships with various pharmaceutical companies involved in oncology.

A version of this article first appeared on Medscape.com.

TOPLINE:

Adults receiving cancer treatment who use cannabis perceived benefits regarding pain, sleep, nausea, and other factors but also reported worse physical and psychological symptoms.

METHODOLOGY:

• Participants included 267 adults (mean age, 58 years; 70% women; 88% White) undergoing treatment for cancer, most commonly breast (47%) and ovarian (29%).
• Participants completed online surveys to characterize cannabis use, reasons for using it, perceived benefits and harms, and physical/psychological symptoms.
• Participants who had used cannabis for more than 1 day during the previous 30 days were compared with those who had not.

TAKEAWAY:

• Overall, 26% of respondents reported cannabis use in the past 30 days, most often edibles (65%) or smoked cannabis (51%).
• Cannabis users were more likely to be younger, male, Black, to have lower income, worse physical/psychological symptoms, and to be disabled or unable to work in comparison with nonusers.
• Cannabis was used to treat pain, cancer, sleep problems, anxiety, nausea, and poor appetite; perceived benefits were greatest with respect to sleep, nausea, pain, muscle spasms, and anxiety.
• Despite perceived benefits, cannabis users reported worse overall distress, anxiety, sleep disturbances, appetite, nausea, fatigue, and pain.

IN PRACTICE:

“The study findings indicate that patients with cancer perceived benefits to using cannabis for many symptoms” but also revealed that “those who used cannabis in the past 30 days had significantly worse symptom profiles overall than those who did not use cannabis,” the authors wrote.

SOURCE:

The study, led by Desiree R. Azizoddin, PsyD, University of Oklahoma Health Science Center, Oklahoma City, was published online in Cancer.

LIMITATIONS:

It’s not known whether adults who used cannabis had significantly worse symptoms at the outset, which may have prompted cannabis use, or whether cannabis use may have exacerbated their symptoms.

DISCLOSURES:

Funding for the study was provided by grants from the National Cancer Institute and the Oklahoma Tobacco Settlement Endowment Trust. Nine of the 10 authors have disclosed no relevant conflicts of interest. One author has relationships with various pharmaceutical companies involved in oncology.

A version of this article first appeared on Medscape.com.

TOPLINE:

Adults receiving cancer treatment who use cannabis perceived benefits regarding pain, sleep, nausea, and other factors but also reported worse physical and psychological symptoms.

METHODOLOGY:

• Participants included 267 adults (mean age, 58 years; 70% women; 88% White) undergoing treatment for cancer, most commonly breast (47%) and ovarian (29%).
• Participants completed online surveys to characterize cannabis use, reasons for using it, perceived benefits and harms, and physical/psychological symptoms.
• Participants who had used cannabis for more than 1 day during the previous 30 days were compared with those who had not.

TAKEAWAY:

• Overall, 26% of respondents reported cannabis use in the past 30 days, most often edibles (65%) or smoked cannabis (51%).
• Cannabis users were more likely to be younger, male, Black, to have lower income, worse physical/psychological symptoms, and to be disabled or unable to work in comparison with nonusers.
• Cannabis was used to treat pain, cancer, sleep problems, anxiety, nausea, and poor appetite; perceived benefits were greatest with respect to sleep, nausea, pain, muscle spasms, and anxiety.
• Despite perceived benefits, cannabis users reported worse overall distress, anxiety, sleep disturbances, appetite, nausea, fatigue, and pain.

IN PRACTICE:

“The study findings indicate that patients with cancer perceived benefits to using cannabis for many symptoms” but also revealed that “those who used cannabis in the past 30 days had significantly worse symptom profiles overall than those who did not use cannabis,” the authors wrote.

SOURCE:

The study, led by Desiree R. Azizoddin, PsyD, University of Oklahoma Health Science Center, Oklahoma City, was published online in Cancer.

LIMITATIONS:

It’s not known whether adults who used cannabis had significantly worse symptoms at the outset, which may have prompted cannabis use, or whether cannabis use may have exacerbated their symptoms.

DISCLOSURES:

Funding for the study was provided by grants from the National Cancer Institute and the Oklahoma Tobacco Settlement Endowment Trust. Nine of the 10 authors have disclosed no relevant conflicts of interest. One author has relationships with various pharmaceutical companies involved in oncology.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration recently released final guidance on a voluntary pilot program to help reduce the risks associated with certain diagnostic biomarker tests used to guide cancer treatment decisions for patients.

These laboratory-developed tests were designed to detect cancer biomarkers to help clinicians find the most appropriate cancer treatments for their patients. But the agency explained it has “become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA-authorized tests.”

Currently, in most circumstances, an in vitro companion diagnostic would be granted marketing authorization alongside the approval of a corresponding cancer therapy. Under limited circumstances, however, the FDA may decide to approve a cancer therapy that requires a diagnostic test, which has not yet received marketing authorization. In these instances, “the benefits from the use of the therapeutic product are so pronounced as to outweigh the risks from the lack of an [in vitro companion diagnostic] with marketing authorization,” the FDA explained in 2014 in a guidance titled, In Vitro Companion Diagnostic Devices .

The new pilot program now aims to “address concerns and questions around the use of unauthorized diagnostics” and help improve cancer care for patients, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press announcement.

The voluntary program will seek to provide greater transparency surrounding performance recommendations for these diagnostic tests. More specifically, the FDA will ask drug manufacturers to provide performance information for tests used to enroll patients in clinical trials that support drug approval. The agency will assess the performance information and establish the minimum performance criteria recommended for similar tests used to select cancer treatments for patients. The results, posted to the FDA’s website, may be used by laboratories to guide their development of diagnostic tests.

The FDA plans to evaluate no more than nine drug sponsors for the pilot program. This initial phase of the program is anticipated to last up to a year.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health, said in a statement.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration recently released final guidance on a voluntary pilot program to help reduce the risks associated with certain diagnostic biomarker tests used to guide cancer treatment decisions for patients.

These laboratory-developed tests were designed to detect cancer biomarkers to help clinicians find the most appropriate cancer treatments for their patients. But the agency explained it has “become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA-authorized tests.”

Currently, in most circumstances, an in vitro companion diagnostic would be granted marketing authorization alongside the approval of a corresponding cancer therapy. Under limited circumstances, however, the FDA may decide to approve a cancer therapy that requires a diagnostic test, which has not yet received marketing authorization. In these instances, “the benefits from the use of the therapeutic product are so pronounced as to outweigh the risks from the lack of an [in vitro companion diagnostic] with marketing authorization,” the FDA explained in 2014 in a guidance titled, In Vitro Companion Diagnostic Devices .

The new pilot program now aims to “address concerns and questions around the use of unauthorized diagnostics” and help improve cancer care for patients, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press announcement.

The voluntary program will seek to provide greater transparency surrounding performance recommendations for these diagnostic tests. More specifically, the FDA will ask drug manufacturers to provide performance information for tests used to enroll patients in clinical trials that support drug approval. The agency will assess the performance information and establish the minimum performance criteria recommended for similar tests used to select cancer treatments for patients. The results, posted to the FDA’s website, may be used by laboratories to guide their development of diagnostic tests.

The FDA plans to evaluate no more than nine drug sponsors for the pilot program. This initial phase of the program is anticipated to last up to a year.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health, said in a statement.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration recently released final guidance on a voluntary pilot program to help reduce the risks associated with certain diagnostic biomarker tests used to guide cancer treatment decisions for patients.

These laboratory-developed tests were designed to detect cancer biomarkers to help clinicians find the most appropriate cancer treatments for their patients. But the agency explained it has “become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA-authorized tests.”

Currently, in most circumstances, an in vitro companion diagnostic would be granted marketing authorization alongside the approval of a corresponding cancer therapy. Under limited circumstances, however, the FDA may decide to approve a cancer therapy that requires a diagnostic test, which has not yet received marketing authorization. In these instances, “the benefits from the use of the therapeutic product are so pronounced as to outweigh the risks from the lack of an [in vitro companion diagnostic] with marketing authorization,” the FDA explained in 2014 in a guidance titled, In Vitro Companion Diagnostic Devices .

The new pilot program now aims to “address concerns and questions around the use of unauthorized diagnostics” and help improve cancer care for patients, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press announcement.

The voluntary program will seek to provide greater transparency surrounding performance recommendations for these diagnostic tests. More specifically, the FDA will ask drug manufacturers to provide performance information for tests used to enroll patients in clinical trials that support drug approval. The agency will assess the performance information and establish the minimum performance criteria recommended for similar tests used to select cancer treatments for patients. The results, posted to the FDA’s website, may be used by laboratories to guide their development of diagnostic tests.

The FDA plans to evaluate no more than nine drug sponsors for the pilot program. This initial phase of the program is anticipated to last up to a year.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health, said in a statement.

A version of this article first appeared on Medscape.com.

In women who have locally advanced breast cancer (LABC), staging defines the extent of the disease and guides therapy.

Researchers have found in the first large, randomized, controlled study on the subject that ¹⁸ F-labeled fluorodeoxyglucose positron emission tomography–computed tomography (PET-CT) finds more distant metastases and allows more accurate staging than usual staging, which is determined by a bone scan and computed tomography (CT) of the thorax/abdomen and pelvis.

Findings of the study, led by Ian S. Dayes, MD, MSc, with the department of oncology at McMaster University in Hamilton, Ont., were published online in the Journal of Clinical Oncology.
 

Scans indicate less aggressive treatment strategy

The authors of the new study wrote that women with LABC, who are at high risk of metastatic disease, have large tumors that “can involve the chest wall or skin, clinically fixed axillary lymph nodes, or infraclavicular, supraclavicular, or internal mammary lymphadenopathy.”

If staging does not detect metastases, treatment is centered on combined modality therapy with curative intent (neoadjuvant chemotherapy and surgery, followed by regional radiation). If metastases are found, the treatment goal changes to controlling the disease.

In this study, twice as many women saw their stage increase from stage IIB or III to stage IV when PET-CT was used instead of conventional staging, guiding their treatment toward less aggressive care to control, rather than attempt to cure, the disease.

The women included in this study had histological evidence of invasive ductal carcinoma of the breast and TNM stage III or IIb (T3N0, but not T2N1).
 

Methods and results

Between December 2016 and April 2022, consenting patients from six regional cancer centers in Ontario were randomly assigned to one of two groups: 184 patients were randomly assigned to whole-body PET-CT and 185 patients to conventional staging.

Overall, the authors wrote, 43 (23%) of PET-CT patients “were upstaged” to stage IV compared with 21 (11%) of the conventionally staged patients (relative risk, 2.4; 95% confidence interval [CI], 1.4-4.2, P = .002).

There were 33 patients in a subset with inflammatory breast cancer and, among them, 4 of 16 (25%) PET-CT patients were upstaged to stage IV compared with 4 of 17 (24%) conventionally staged patients.

In the patients who did not have inflammatory breast cancer, 39 of 168 (23%) PET-CT patients were upstaged compared with 17 (10%) of 168 in the conventionally staged group.

Journal of Clinical Oncology (JCO) Senior Deputy Editor Kathy D. Miller, MD, said that, “PET/CT staging identifies distant disease in more patients and changes goals of therapy. Further research is needed to determine the impact on patient outcome.”
 

Findings have already changed practice

Senior author, Mark Levine, MD, MSc, also with McMaster, said in an interview that the results of this study have already changed practice in Canada, and he expects the United States to follow suit.

Dr. Levine said the study is important “in terms of helping plan therapy and being very open and honest with patients as to their prognosis.”

The findings constitute level 1 evidence in favor of PET-CT. Already, in Canada, “because of the results of the study, people with stage III breast cancer can get a PET scan,” he said.

Dr. Levine said he expects this evidence also to clarify “wishy-washy” National Comprehensive Cancer Network guidelines on using PET scans for LABC in the United States when the guidelines are next updated.

“That will make it easier for payers in the United States,” he added.

Cost effectiveness, Dr. Levine said, is complicated, because on one hand PET scans are quite costly. But its use would lead to more women getting less aggressive and expensive therapy and surgery.

Dr. Levine noted that his team will be analyzing cost-effectiveness over the next year.
 

New questions with more in stage IV

In an editorial, Lajos Pusztai, MD, DPhil, scientific codirector of the breast center at Yale University in New Haven, Conn., noted that, “all good studies raise new questions” and this one is no exception.

He pointed out that the number of women with stage IV metastatic breast cancer (MBC) has been increasing over the past 2 decades because of more sensitive staging methods. At the same time the number of women with recurrent metastatic disease is decreasing, because adjuvant therapies have improved.
 

Findings highlight need for stage IV treatment studies

Dr. Pusztai noted that the patients who have de novo oligometastatic stage IV disease “are a unique subset among patients with MBC,” and the best treatment [for them] has not been established in randomized, controlled trials.

“Almost all randomized trials that targeted oligometastatic patients accrued mostly recurrent metastatic cancers; many included various cancer types, and none have tested the value of systemic multidrug regimens administered with curative intent,” he wrote.

If the health care systems adopt PET-CT for routine staging of locally advanced breast cancer, that will increase the diagnosis of de novo oligometastatic stage IV breast cancer, Dr. Pusztai said. That “underlines the importance of conducting studies for this unique subset of patients to establish level 1 evidence-based treatment strategies.”

Dr. Dayes has received honoraria from Verity Pharmaceuticals. One coauthor is employed by Point Biopharma. Other coauthors reported ties with AbbVie, Agendia, Genomic Health, InMode and Lutronic. Dr. Pusztai’s institution has received research funding from Merck, Genentech, Seagen, AstraZeneca, Bristol Myers Squibb, and Pfizer. He has received honoraria and travel expenses and has served in a consulting role for several pharmaceutical companies. Full disclosures are available on Open Payments.

In women who have locally advanced breast cancer (LABC), staging defines the extent of the disease and guides therapy.

Researchers have found in the first large, randomized, controlled study on the subject that ¹⁸ F-labeled fluorodeoxyglucose positron emission tomography–computed tomography (PET-CT) finds more distant metastases and allows more accurate staging than usual staging, which is determined by a bone scan and computed tomography (CT) of the thorax/abdomen and pelvis.

Findings of the study, led by Ian S. Dayes, MD, MSc, with the department of oncology at McMaster University in Hamilton, Ont., were published online in the Journal of Clinical Oncology.
 

Scans indicate less aggressive treatment strategy

The authors of the new study wrote that women with LABC, who are at high risk of metastatic disease, have large tumors that “can involve the chest wall or skin, clinically fixed axillary lymph nodes, or infraclavicular, supraclavicular, or internal mammary lymphadenopathy.”

If staging does not detect metastases, treatment is centered on combined modality therapy with curative intent (neoadjuvant chemotherapy and surgery, followed by regional radiation). If metastases are found, the treatment goal changes to controlling the disease.

In this study, twice as many women saw their stage increase from stage IIB or III to stage IV when PET-CT was used instead of conventional staging, guiding their treatment toward less aggressive care to control, rather than attempt to cure, the disease.

The women included in this study had histological evidence of invasive ductal carcinoma of the breast and TNM stage III or IIb (T3N0, but not T2N1).
 

Methods and results

Between December 2016 and April 2022, consenting patients from six regional cancer centers in Ontario were randomly assigned to one of two groups: 184 patients were randomly assigned to whole-body PET-CT and 185 patients to conventional staging.

Overall, the authors wrote, 43 (23%) of PET-CT patients “were upstaged” to stage IV compared with 21 (11%) of the conventionally staged patients (relative risk, 2.4; 95% confidence interval [CI], 1.4-4.2, P = .002).

There were 33 patients in a subset with inflammatory breast cancer and, among them, 4 of 16 (25%) PET-CT patients were upstaged to stage IV compared with 4 of 17 (24%) conventionally staged patients.

In the patients who did not have inflammatory breast cancer, 39 of 168 (23%) PET-CT patients were upstaged compared with 17 (10%) of 168 in the conventionally staged group.

Journal of Clinical Oncology (JCO) Senior Deputy Editor Kathy D. Miller, MD, said that, “PET/CT staging identifies distant disease in more patients and changes goals of therapy. Further research is needed to determine the impact on patient outcome.”
 

Findings have already changed practice

Senior author, Mark Levine, MD, MSc, also with McMaster, said in an interview that the results of this study have already changed practice in Canada, and he expects the United States to follow suit.

Dr. Levine said the study is important “in terms of helping plan therapy and being very open and honest with patients as to their prognosis.”

The findings constitute level 1 evidence in favor of PET-CT. Already, in Canada, “because of the results of the study, people with stage III breast cancer can get a PET scan,” he said.

Dr. Levine said he expects this evidence also to clarify “wishy-washy” National Comprehensive Cancer Network guidelines on using PET scans for LABC in the United States when the guidelines are next updated.

“That will make it easier for payers in the United States,” he added.

Cost effectiveness, Dr. Levine said, is complicated, because on one hand PET scans are quite costly. But its use would lead to more women getting less aggressive and expensive therapy and surgery.

Dr. Levine noted that his team will be analyzing cost-effectiveness over the next year.
 

New questions with more in stage IV

In an editorial, Lajos Pusztai, MD, DPhil, scientific codirector of the breast center at Yale University in New Haven, Conn., noted that, “all good studies raise new questions” and this one is no exception.

He pointed out that the number of women with stage IV metastatic breast cancer (MBC) has been increasing over the past 2 decades because of more sensitive staging methods. At the same time the number of women with recurrent metastatic disease is decreasing, because adjuvant therapies have improved.
 

Findings highlight need for stage IV treatment studies

Dr. Pusztai noted that the patients who have de novo oligometastatic stage IV disease “are a unique subset among patients with MBC,” and the best treatment [for them] has not been established in randomized, controlled trials.

“Almost all randomized trials that targeted oligometastatic patients accrued mostly recurrent metastatic cancers; many included various cancer types, and none have tested the value of systemic multidrug regimens administered with curative intent,” he wrote.

If the health care systems adopt PET-CT for routine staging of locally advanced breast cancer, that will increase the diagnosis of de novo oligometastatic stage IV breast cancer, Dr. Pusztai said. That “underlines the importance of conducting studies for this unique subset of patients to establish level 1 evidence-based treatment strategies.”

Dr. Dayes has received honoraria from Verity Pharmaceuticals. One coauthor is employed by Point Biopharma. Other coauthors reported ties with AbbVie, Agendia, Genomic Health, InMode and Lutronic. Dr. Pusztai’s institution has received research funding from Merck, Genentech, Seagen, AstraZeneca, Bristol Myers Squibb, and Pfizer. He has received honoraria and travel expenses and has served in a consulting role for several pharmaceutical companies. Full disclosures are available on Open Payments.

In women who have locally advanced breast cancer (LABC), staging defines the extent of the disease and guides therapy.

Researchers have found in the first large, randomized, controlled study on the subject that ¹⁸ F-labeled fluorodeoxyglucose positron emission tomography–computed tomography (PET-CT) finds more distant metastases and allows more accurate staging than usual staging, which is determined by a bone scan and computed tomography (CT) of the thorax/abdomen and pelvis.

Findings of the study, led by Ian S. Dayes, MD, MSc, with the department of oncology at McMaster University in Hamilton, Ont., were published online in the Journal of Clinical Oncology.
 

Scans indicate less aggressive treatment strategy

The authors of the new study wrote that women with LABC, who are at high risk of metastatic disease, have large tumors that “can involve the chest wall or skin, clinically fixed axillary lymph nodes, or infraclavicular, supraclavicular, or internal mammary lymphadenopathy.”

If staging does not detect metastases, treatment is centered on combined modality therapy with curative intent (neoadjuvant chemotherapy and surgery, followed by regional radiation). If metastases are found, the treatment goal changes to controlling the disease.

In this study, twice as many women saw their stage increase from stage IIB or III to stage IV when PET-CT was used instead of conventional staging, guiding their treatment toward less aggressive care to control, rather than attempt to cure, the disease.

The women included in this study had histological evidence of invasive ductal carcinoma of the breast and TNM stage III or IIb (T3N0, but not T2N1).
 

Methods and results

Between December 2016 and April 2022, consenting patients from six regional cancer centers in Ontario were randomly assigned to one of two groups: 184 patients were randomly assigned to whole-body PET-CT and 185 patients to conventional staging.

Overall, the authors wrote, 43 (23%) of PET-CT patients “were upstaged” to stage IV compared with 21 (11%) of the conventionally staged patients (relative risk, 2.4; 95% confidence interval [CI], 1.4-4.2, P = .002).

There were 33 patients in a subset with inflammatory breast cancer and, among them, 4 of 16 (25%) PET-CT patients were upstaged to stage IV compared with 4 of 17 (24%) conventionally staged patients.

In the patients who did not have inflammatory breast cancer, 39 of 168 (23%) PET-CT patients were upstaged compared with 17 (10%) of 168 in the conventionally staged group.

Journal of Clinical Oncology (JCO) Senior Deputy Editor Kathy D. Miller, MD, said that, “PET/CT staging identifies distant disease in more patients and changes goals of therapy. Further research is needed to determine the impact on patient outcome.”
 

Findings have already changed practice

Senior author, Mark Levine, MD, MSc, also with McMaster, said in an interview that the results of this study have already changed practice in Canada, and he expects the United States to follow suit.

Dr. Levine said the study is important “in terms of helping plan therapy and being very open and honest with patients as to their prognosis.”

The findings constitute level 1 evidence in favor of PET-CT. Already, in Canada, “because of the results of the study, people with stage III breast cancer can get a PET scan,” he said.

Dr. Levine said he expects this evidence also to clarify “wishy-washy” National Comprehensive Cancer Network guidelines on using PET scans for LABC in the United States when the guidelines are next updated.

“That will make it easier for payers in the United States,” he added.

Cost effectiveness, Dr. Levine said, is complicated, because on one hand PET scans are quite costly. But its use would lead to more women getting less aggressive and expensive therapy and surgery.

Dr. Levine noted that his team will be analyzing cost-effectiveness over the next year.
 

New questions with more in stage IV

In an editorial, Lajos Pusztai, MD, DPhil, scientific codirector of the breast center at Yale University in New Haven, Conn., noted that, “all good studies raise new questions” and this one is no exception.

He pointed out that the number of women with stage IV metastatic breast cancer (MBC) has been increasing over the past 2 decades because of more sensitive staging methods. At the same time the number of women with recurrent metastatic disease is decreasing, because adjuvant therapies have improved.
 

Findings highlight need for stage IV treatment studies

Dr. Pusztai noted that the patients who have de novo oligometastatic stage IV disease “are a unique subset among patients with MBC,” and the best treatment [for them] has not been established in randomized, controlled trials.

“Almost all randomized trials that targeted oligometastatic patients accrued mostly recurrent metastatic cancers; many included various cancer types, and none have tested the value of systemic multidrug regimens administered with curative intent,” he wrote.

If the health care systems adopt PET-CT for routine staging of locally advanced breast cancer, that will increase the diagnosis of de novo oligometastatic stage IV breast cancer, Dr. Pusztai said. That “underlines the importance of conducting studies for this unique subset of patients to establish level 1 evidence-based treatment strategies.”

Dr. Dayes has received honoraria from Verity Pharmaceuticals. One coauthor is employed by Point Biopharma. Other coauthors reported ties with AbbVie, Agendia, Genomic Health, InMode and Lutronic. Dr. Pusztai’s institution has received research funding from Merck, Genentech, Seagen, AstraZeneca, Bristol Myers Squibb, and Pfizer. He has received honoraria and travel expenses and has served in a consulting role for several pharmaceutical companies. Full disclosures are available on Open Payments.

A new scientific statement from the American Heart Association focuses on equity in cardio-oncology care and research.

A “growing body of evidence” suggests that women and people from underrepresented patient groups experience disproportionately higher cardiovascular effects from new and emerging anticancer therapies, the writing group, led by Daniel Addison, MD, with the Ohio State University, Columbus, pointed out.

For example, women appear to be at higher risk of immune checkpoint inhibitor–related toxicities, whereas Black patients with cancer face up to a threefold higher risk of cardiotoxicity with anticancer therapies.

With reduced screening and delayed preventive measures, Hispanic patients have more complex heart disease, cancer is diagnosed at later stages, and they receive more cardiotoxic regimens because of a lack of eligibility for novel treatments. Ultimately, this contributes to a higher incidence of treatment complications, cardiac dysfunction, and adverse patient outcomes for this patient group, they write.

Although no studies have specifically addressed cardio-oncology disparities in the LGBTQIA+ population, such disparities can be inferred from known cardiovascular disease and oncology disparities, the writing group noted.

These disparities are supported by “disparately high” risk of death after a cancer diagnosis among women and individuals from underrepresented groups, even after accounting for socioeconomic and behavioral patterns, they pointed out.

The scientific statement was published online in Circulation.
 

Evidence gaps and the path forward

“Despite advances in strategies to limit the risks of cardiovascular events among cancer survivors, relatively limited guidance is available to address the rapidly growing problem of disparate cardiotoxic risks among women and underrepresented patient populations,” the writing group said.

Decentralized and sporadic evaluations have led to a lack of consensus on the definitions, investigations, and potential optimal strategies to address disparate cardiotoxicity with contemporary cancer immunotherapy, as well as biologic and cytotoxic therapies, they noted.

They said caution is needed when interpreting clinical trial data about cardiotoxicity and in generalizing the results because people from diverse racial and ethnic groups have not been well represented in many trials.

The writing group outlined key evidence gaps and future research directions for addressing cardio-oncology disparities, as well as strategies to improve equity in cardio-oncology care and research.

These include the following:

• Identifying specific predictive factors of long-term cardiotoxic risk with targeted and immune-based cancer therapies in women and underrepresented populations.
• Investigating biological mechanisms that may underlie differences in cardiotoxicities between different patient groups.
• Developing personalized cardioprotection strategies that integrate biological, genetic, and social determinant markers.
• Intentionally diversifying clinical trials and identifying optimal strategies to improve representation in cancer clinical trials.
• Determining the role of technology, such as artificial intelligence, in improving cardiotoxicity disparities.

“Conscientiously leveraging technology and designing trials with outcomes related to these issues in practice (considering feasibility and cost) will critically accelerate the field of cardio-oncology in the 21st century. With tangible goals, we can improve health inequities in cardio-oncology,” the writing group said.

The research had no commercial funding. No conflicts of interest were reported.

A version of this article originally appeared on Medscape.com.

A new scientific statement from the American Heart Association focuses on equity in cardio-oncology care and research.

A “growing body of evidence” suggests that women and people from underrepresented patient groups experience disproportionately higher cardiovascular effects from new and emerging anticancer therapies, the writing group, led by Daniel Addison, MD, with the Ohio State University, Columbus, pointed out.

For example, women appear to be at higher risk of immune checkpoint inhibitor–related toxicities, whereas Black patients with cancer face up to a threefold higher risk of cardiotoxicity with anticancer therapies.

With reduced screening and delayed preventive measures, Hispanic patients have more complex heart disease, cancer is diagnosed at later stages, and they receive more cardiotoxic regimens because of a lack of eligibility for novel treatments. Ultimately, this contributes to a higher incidence of treatment complications, cardiac dysfunction, and adverse patient outcomes for this patient group, they write.

Although no studies have specifically addressed cardio-oncology disparities in the LGBTQIA+ population, such disparities can be inferred from known cardiovascular disease and oncology disparities, the writing group noted.

These disparities are supported by “disparately high” risk of death after a cancer diagnosis among women and individuals from underrepresented groups, even after accounting for socioeconomic and behavioral patterns, they pointed out.

The scientific statement was published online in Circulation.
 

Evidence gaps and the path forward

“Despite advances in strategies to limit the risks of cardiovascular events among cancer survivors, relatively limited guidance is available to address the rapidly growing problem of disparate cardiotoxic risks among women and underrepresented patient populations,” the writing group said.

Decentralized and sporadic evaluations have led to a lack of consensus on the definitions, investigations, and potential optimal strategies to address disparate cardiotoxicity with contemporary cancer immunotherapy, as well as biologic and cytotoxic therapies, they noted.

They said caution is needed when interpreting clinical trial data about cardiotoxicity and in generalizing the results because people from diverse racial and ethnic groups have not been well represented in many trials.

The writing group outlined key evidence gaps and future research directions for addressing cardio-oncology disparities, as well as strategies to improve equity in cardio-oncology care and research.

These include the following:

• Identifying specific predictive factors of long-term cardiotoxic risk with targeted and immune-based cancer therapies in women and underrepresented populations.
• Investigating biological mechanisms that may underlie differences in cardiotoxicities between different patient groups.
• Developing personalized cardioprotection strategies that integrate biological, genetic, and social determinant markers.
• Intentionally diversifying clinical trials and identifying optimal strategies to improve representation in cancer clinical trials.
• Determining the role of technology, such as artificial intelligence, in improving cardiotoxicity disparities.

“Conscientiously leveraging technology and designing trials with outcomes related to these issues in practice (considering feasibility and cost) will critically accelerate the field of cardio-oncology in the 21st century. With tangible goals, we can improve health inequities in cardio-oncology,” the writing group said.

The research had no commercial funding. No conflicts of interest were reported.

A version of this article originally appeared on Medscape.com.

A new scientific statement from the American Heart Association focuses on equity in cardio-oncology care and research.

A “growing body of evidence” suggests that women and people from underrepresented patient groups experience disproportionately higher cardiovascular effects from new and emerging anticancer therapies, the writing group, led by Daniel Addison, MD, with the Ohio State University, Columbus, pointed out.

For example, women appear to be at higher risk of immune checkpoint inhibitor–related toxicities, whereas Black patients with cancer face up to a threefold higher risk of cardiotoxicity with anticancer therapies.

With reduced screening and delayed preventive measures, Hispanic patients have more complex heart disease, cancer is diagnosed at later stages, and they receive more cardiotoxic regimens because of a lack of eligibility for novel treatments. Ultimately, this contributes to a higher incidence of treatment complications, cardiac dysfunction, and adverse patient outcomes for this patient group, they write.

Although no studies have specifically addressed cardio-oncology disparities in the LGBTQIA+ population, such disparities can be inferred from known cardiovascular disease and oncology disparities, the writing group noted.

These disparities are supported by “disparately high” risk of death after a cancer diagnosis among women and individuals from underrepresented groups, even after accounting for socioeconomic and behavioral patterns, they pointed out.

The scientific statement was published online in Circulation.
 

Evidence gaps and the path forward

“Despite advances in strategies to limit the risks of cardiovascular events among cancer survivors, relatively limited guidance is available to address the rapidly growing problem of disparate cardiotoxic risks among women and underrepresented patient populations,” the writing group said.

Decentralized and sporadic evaluations have led to a lack of consensus on the definitions, investigations, and potential optimal strategies to address disparate cardiotoxicity with contemporary cancer immunotherapy, as well as biologic and cytotoxic therapies, they noted.

They said caution is needed when interpreting clinical trial data about cardiotoxicity and in generalizing the results because people from diverse racial and ethnic groups have not been well represented in many trials.

The writing group outlined key evidence gaps and future research directions for addressing cardio-oncology disparities, as well as strategies to improve equity in cardio-oncology care and research.

These include the following:

• Identifying specific predictive factors of long-term cardiotoxic risk with targeted and immune-based cancer therapies in women and underrepresented populations.
• Investigating biological mechanisms that may underlie differences in cardiotoxicities between different patient groups.
• Developing personalized cardioprotection strategies that integrate biological, genetic, and social determinant markers.
• Intentionally diversifying clinical trials and identifying optimal strategies to improve representation in cancer clinical trials.
• Determining the role of technology, such as artificial intelligence, in improving cardiotoxicity disparities.

“Conscientiously leveraging technology and designing trials with outcomes related to these issues in practice (considering feasibility and cost) will critically accelerate the field of cardio-oncology in the 21st century. With tangible goals, we can improve health inequities in cardio-oncology,” the writing group said.

The research had no commercial funding. No conflicts of interest were reported.

A version of this article originally appeared on Medscape.com.

Patients with head and neck cancer and overweight saw better treatment response and survival after chemoradiation, compared with patients with the same type of cancer but a normal weight, a new study finds.

The findings, published in JAMA Network Open, are the latest to parse the complex relationship between body mass index (BMI) and treatment in cancers that is sometimes called the “obesity paradox.” The researchers compared outcomes among patients with normal weight, overweight, and obesity.

While higher BMI is an established risk factor for many types of cancer and for cancer-specific mortality overall, studies in some cancers have shown that patients with higher BMI do better, possibly because excess BMI acts as a nutrient reserve against treatment-associated weight loss.
 

Methods and results

For their research, Sung Jun Ma, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., and colleagues looked at records for 445 patients (84% men, median age 61) at Dr. Ma’s institution with nonmetastatic head and neck cancer who underwent chemoradiotherapy between 2005 and 2021. Patients were followed up for a median 48 months, and those with underweight at treatment initiation were excluded.

The researchers found that overweight BMI (25-29.9 kg/m²) was associated with improved overall survival at 5 years (71% vs. 58% of patients with normal weight), as well as 5-year progression-free survival (68% vs. 51%). No overall or progression-free survival benefit link was seen in patients with a BMI of 30 or higher, in contrast to some previous studies of patients with head and neck cancers. BMI was not associated with improved survival outcomes among human papillomavirus–positive patients.

Both overweight and obesity were associated with complete response on follow-up PET-CT, with nearly 92% of patients with overweight and 91% of patients with obesity (defined as having a BMI of 30 or higher) seeing a complete metabolic response, compared with 74% of patients with normal weight.

Having an overweight BMI was also associated with improvements in tumor recurrence, with fewer of patients with this type of BMI experiencing 5-year locoregional failure than patients with normal weight (7% vs 26%). Having an obese BMI was not associated with improvements in recurrence. All the reported differences reached statistical significance.

The study authors surmised that the discrepancies between outcomes for patients with overweight and obesity “may be due to a nonlinear association between BMI and survival, with the highest survival seen in the overweight BMI range.”

It was important to note that this study saw no differences in treatment interruptions between the BMI groups that could account for differences in outcomes. Only three patients in the cohort saw their radiotherapy treatment interrupted, Dr. Ma said in an interview.

“If we felt that the obesity paradox happens because people with normal BMI lose too much weight during the treatment course, treatment gets interrupted, and they get worse outcomes from suboptimal treatments, then we would have seen more treatment interruptions among those with normal BMI. However, that was not the case in our study,” he said. Rather, the results point to “a complex interaction among cancer, [a person’s build], and nutritional status.”

Clinicians should be aware, Dr. Ma added, “that the same head and neck cancer may behave more aggressively among patients with normal BMI, compared to others with overweight BMI. Patients with normal BMI may need to be monitored more closely and carefully for potentially worse outcomes.” 

The investigators acknowledged several weaknesses of their study, including its retrospective design, the measure of BMI using cutoffs rather than a continuum, and the collection of BMI information at a single time point. While 84% of patients in the study received cisplatin, the study did not contain information on cumulative cisplatin dose.
 

Importance of nutritional support during treatment highlighted

In an interview, Ari Rosenberg, MD, of the University of Chicago Medicine, commented that the findings highlighted the importance of expert nutritional supportive care during treatment and monitoring for patients with advanced head and neck cancers undergoing chemoradiation.

“Nutritional status is very important both at baseline and during treatment,” Dr. Rosenberg said. “Even small changes in weight or BMI can be a key indicator of supportive care during chemoradiation and represent a biomarker to guide supportive management. ... The take home message is that patients should be treated at centers that have a high volume of advanced head and neck cancer patients, which have all the supportive components and expertise to optimize treatment delivery and maximize survival.”

Dr. Ma and colleagues’ study was funded by the National Cancer Institute Cancer Center. None of its authors declared financial conflicts of interest. Dr. Rosenberg disclosed receiving consulting fees from EMD Serono related to head and neck cancer treatment.
 

Patients with head and neck cancer and overweight saw better treatment response and survival after chemoradiation, compared with patients with the same type of cancer but a normal weight, a new study finds.

The findings, published in JAMA Network Open, are the latest to parse the complex relationship between body mass index (BMI) and treatment in cancers that is sometimes called the “obesity paradox.” The researchers compared outcomes among patients with normal weight, overweight, and obesity.

While higher BMI is an established risk factor for many types of cancer and for cancer-specific mortality overall, studies in some cancers have shown that patients with higher BMI do better, possibly because excess BMI acts as a nutrient reserve against treatment-associated weight loss.
 

Methods and results

For their research, Sung Jun Ma, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., and colleagues looked at records for 445 patients (84% men, median age 61) at Dr. Ma’s institution with nonmetastatic head and neck cancer who underwent chemoradiotherapy between 2005 and 2021. Patients were followed up for a median 48 months, and those with underweight at treatment initiation were excluded.

The researchers found that overweight BMI (25-29.9 kg/m²) was associated with improved overall survival at 5 years (71% vs. 58% of patients with normal weight), as well as 5-year progression-free survival (68% vs. 51%). No overall or progression-free survival benefit link was seen in patients with a BMI of 30 or higher, in contrast to some previous studies of patients with head and neck cancers. BMI was not associated with improved survival outcomes among human papillomavirus–positive patients.

Both overweight and obesity were associated with complete response on follow-up PET-CT, with nearly 92% of patients with overweight and 91% of patients with obesity (defined as having a BMI of 30 or higher) seeing a complete metabolic response, compared with 74% of patients with normal weight.

Having an overweight BMI was also associated with improvements in tumor recurrence, with fewer of patients with this type of BMI experiencing 5-year locoregional failure than patients with normal weight (7% vs 26%). Having an obese BMI was not associated with improvements in recurrence. All the reported differences reached statistical significance.

The study authors surmised that the discrepancies between outcomes for patients with overweight and obesity “may be due to a nonlinear association between BMI and survival, with the highest survival seen in the overweight BMI range.”

It was important to note that this study saw no differences in treatment interruptions between the BMI groups that could account for differences in outcomes. Only three patients in the cohort saw their radiotherapy treatment interrupted, Dr. Ma said in an interview.

“If we felt that the obesity paradox happens because people with normal BMI lose too much weight during the treatment course, treatment gets interrupted, and they get worse outcomes from suboptimal treatments, then we would have seen more treatment interruptions among those with normal BMI. However, that was not the case in our study,” he said. Rather, the results point to “a complex interaction among cancer, [a person’s build], and nutritional status.”

Clinicians should be aware, Dr. Ma added, “that the same head and neck cancer may behave more aggressively among patients with normal BMI, compared to others with overweight BMI. Patients with normal BMI may need to be monitored more closely and carefully for potentially worse outcomes.” 

The investigators acknowledged several weaknesses of their study, including its retrospective design, the measure of BMI using cutoffs rather than a continuum, and the collection of BMI information at a single time point. While 84% of patients in the study received cisplatin, the study did not contain information on cumulative cisplatin dose.
 

Importance of nutritional support during treatment highlighted

In an interview, Ari Rosenberg, MD, of the University of Chicago Medicine, commented that the findings highlighted the importance of expert nutritional supportive care during treatment and monitoring for patients with advanced head and neck cancers undergoing chemoradiation.

“Nutritional status is very important both at baseline and during treatment,” Dr. Rosenberg said. “Even small changes in weight or BMI can be a key indicator of supportive care during chemoradiation and represent a biomarker to guide supportive management. ... The take home message is that patients should be treated at centers that have a high volume of advanced head and neck cancer patients, which have all the supportive components and expertise to optimize treatment delivery and maximize survival.”

Dr. Ma and colleagues’ study was funded by the National Cancer Institute Cancer Center. None of its authors declared financial conflicts of interest. Dr. Rosenberg disclosed receiving consulting fees from EMD Serono related to head and neck cancer treatment.
 

Patients with head and neck cancer and overweight saw better treatment response and survival after chemoradiation, compared with patients with the same type of cancer but a normal weight, a new study finds.

The findings, published in JAMA Network Open, are the latest to parse the complex relationship between body mass index (BMI) and treatment in cancers that is sometimes called the “obesity paradox.” The researchers compared outcomes among patients with normal weight, overweight, and obesity.

While higher BMI is an established risk factor for many types of cancer and for cancer-specific mortality overall, studies in some cancers have shown that patients with higher BMI do better, possibly because excess BMI acts as a nutrient reserve against treatment-associated weight loss.
 

Methods and results

For their research, Sung Jun Ma, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., and colleagues looked at records for 445 patients (84% men, median age 61) at Dr. Ma’s institution with nonmetastatic head and neck cancer who underwent chemoradiotherapy between 2005 and 2021. Patients were followed up for a median 48 months, and those with underweight at treatment initiation were excluded.

The researchers found that overweight BMI (25-29.9 kg/m²) was associated with improved overall survival at 5 years (71% vs. 58% of patients with normal weight), as well as 5-year progression-free survival (68% vs. 51%). No overall or progression-free survival benefit link was seen in patients with a BMI of 30 or higher, in contrast to some previous studies of patients with head and neck cancers. BMI was not associated with improved survival outcomes among human papillomavirus–positive patients.

Both overweight and obesity were associated with complete response on follow-up PET-CT, with nearly 92% of patients with overweight and 91% of patients with obesity (defined as having a BMI of 30 or higher) seeing a complete metabolic response, compared with 74% of patients with normal weight.

Having an overweight BMI was also associated with improvements in tumor recurrence, with fewer of patients with this type of BMI experiencing 5-year locoregional failure than patients with normal weight (7% vs 26%). Having an obese BMI was not associated with improvements in recurrence. All the reported differences reached statistical significance.

The study authors surmised that the discrepancies between outcomes for patients with overweight and obesity “may be due to a nonlinear association between BMI and survival, with the highest survival seen in the overweight BMI range.”

It was important to note that this study saw no differences in treatment interruptions between the BMI groups that could account for differences in outcomes. Only three patients in the cohort saw their radiotherapy treatment interrupted, Dr. Ma said in an interview.

“If we felt that the obesity paradox happens because people with normal BMI lose too much weight during the treatment course, treatment gets interrupted, and they get worse outcomes from suboptimal treatments, then we would have seen more treatment interruptions among those with normal BMI. However, that was not the case in our study,” he said. Rather, the results point to “a complex interaction among cancer, [a person’s build], and nutritional status.”

Clinicians should be aware, Dr. Ma added, “that the same head and neck cancer may behave more aggressively among patients with normal BMI, compared to others with overweight BMI. Patients with normal BMI may need to be monitored more closely and carefully for potentially worse outcomes.” 

The investigators acknowledged several weaknesses of their study, including its retrospective design, the measure of BMI using cutoffs rather than a continuum, and the collection of BMI information at a single time point. While 84% of patients in the study received cisplatin, the study did not contain information on cumulative cisplatin dose.
 

Importance of nutritional support during treatment highlighted

In an interview, Ari Rosenberg, MD, of the University of Chicago Medicine, commented that the findings highlighted the importance of expert nutritional supportive care during treatment and monitoring for patients with advanced head and neck cancers undergoing chemoradiation.

“Nutritional status is very important both at baseline and during treatment,” Dr. Rosenberg said. “Even small changes in weight or BMI can be a key indicator of supportive care during chemoradiation and represent a biomarker to guide supportive management. ... The take home message is that patients should be treated at centers that have a high volume of advanced head and neck cancer patients, which have all the supportive components and expertise to optimize treatment delivery and maximize survival.”

Dr. Ma and colleagues’ study was funded by the National Cancer Institute Cancer Center. None of its authors declared financial conflicts of interest. Dr. Rosenberg disclosed receiving consulting fees from EMD Serono related to head and neck cancer treatment.
 

An expert panel of pain management clinicians has released what they say are the first international guidelines for general practitioners on opioid analgesic deprescribing in adults.

The recommendations describe best practices for stopping opioid therapy and emphasize slow tapering and individualized deprescribing plans tailored to each patient.

Developed by general practitioners, pain specialists, addiction specialists, pharmacists, registered nurses, consumers, and physiotherapists, the guidelines note that deprescribing may not be appropriate for every patient and that stopping abruptly can be associated with an increased risk of overdose.

“Internationally, we were seeing significant harms from opioids but also significant harms from unsolicited and abrupt opioid cessation,” said lead author Aili Langford, PhD, who conducted the study as a doctoral student at the University of Sydney. “It was clear that recommendations to support safe and person-centered opioid deprescribing were required.”

The findings were published online  in the Medical Journal of Australia.
 

Deprescribing plan

The consensus guidelines include 11 recommendations for deprescribing in adult patients who take at least one opioid for any type of pain.

Recommendations include implementing a deprescribing plan when opioids are first prescribed and gradual and individualized deprescribing, with regular monitoring and review.

Clinicians should consider opioid deprescribing in patients who experience no clinically meaningful improvement in function, quality of life, or pain at high risk with opioid therapy, they note. Patients who are at high risk for opioid-related harm are also good candidates for deprescribing.

Stopping opioid therapy is not recommended for patients with severe opioid use disorder (OUD). In those patients, medication-assisted OUD treatment and other evidence-based interventions are recommended.

“Opioids can be effective in pain management,” co-author Carl Schneider, PhD, an associate professor of pharmacy at the University of Sydney, said in a press release. “However, over the longer term, the risk of harms may outweigh the benefits.”
 

A ‘global problem’

Commenting on the guidelines, Orman Trent Hall, DO, assistant professor of addiction medicine, department of psychiatry and behavioral health at the Ohio State University Wexner Medical Center, Columbus, said they are similar to recommendations published by the U.S. Centers for Disease Control and Prevention in 2016 and 2022 but offer additional information that could be helpful.

“This new guideline provides more explicit advice about tapering and withdrawal management, which may be useful to practitioners. The opioid crisis is a global problem, and while individual countries may require local solutions, the new international guideline may offer a framework for approaching this issue,” he said.

The guideline’s emphasis on the potential risks of deprescribing in some patients is also key, Dr. Hall added. Patients who are tapering off opioid therapy may have worsening pain and loss of function that can affect their quality of life.

“Patients may also experience psychological harm and increased risk of opioid use disorder and death by suicide following opioid deprescribing,” Dr. Hall said. “Therefore, it is important for providers to carefully weigh the risks of prescribing and deprescribing and engage patients with person-centered communication and shared decision-making.”

The work was funded by grants from the University of Sydney and the National Health and Medical Research Council. Full disclosures are available in the original article. Dr. Hall has provided expert opinion to the health care consultancy firm Lumanity and Emergent BioSolutions regarding the overdose crisis.

A version of this article originally appeared on Medscape.com.

An expert panel of pain management clinicians has released what they say are the first international guidelines for general practitioners on opioid analgesic deprescribing in adults.

The recommendations describe best practices for stopping opioid therapy and emphasize slow tapering and individualized deprescribing plans tailored to each patient.

Developed by general practitioners, pain specialists, addiction specialists, pharmacists, registered nurses, consumers, and physiotherapists, the guidelines note that deprescribing may not be appropriate for every patient and that stopping abruptly can be associated with an increased risk of overdose.

“Internationally, we were seeing significant harms from opioids but also significant harms from unsolicited and abrupt opioid cessation,” said lead author Aili Langford, PhD, who conducted the study as a doctoral student at the University of Sydney. “It was clear that recommendations to support safe and person-centered opioid deprescribing were required.”

The findings were published online  in the Medical Journal of Australia.
 

Deprescribing plan

The consensus guidelines include 11 recommendations for deprescribing in adult patients who take at least one opioid for any type of pain.

Recommendations include implementing a deprescribing plan when opioids are first prescribed and gradual and individualized deprescribing, with regular monitoring and review.

Clinicians should consider opioid deprescribing in patients who experience no clinically meaningful improvement in function, quality of life, or pain at high risk with opioid therapy, they note. Patients who are at high risk for opioid-related harm are also good candidates for deprescribing.

Stopping opioid therapy is not recommended for patients with severe opioid use disorder (OUD). In those patients, medication-assisted OUD treatment and other evidence-based interventions are recommended.

“Opioids can be effective in pain management,” co-author Carl Schneider, PhD, an associate professor of pharmacy at the University of Sydney, said in a press release. “However, over the longer term, the risk of harms may outweigh the benefits.”
 

A ‘global problem’

Commenting on the guidelines, Orman Trent Hall, DO, assistant professor of addiction medicine, department of psychiatry and behavioral health at the Ohio State University Wexner Medical Center, Columbus, said they are similar to recommendations published by the U.S. Centers for Disease Control and Prevention in 2016 and 2022 but offer additional information that could be helpful.

“This new guideline provides more explicit advice about tapering and withdrawal management, which may be useful to practitioners. The opioid crisis is a global problem, and while individual countries may require local solutions, the new international guideline may offer a framework for approaching this issue,” he said.

The guideline’s emphasis on the potential risks of deprescribing in some patients is also key, Dr. Hall added. Patients who are tapering off opioid therapy may have worsening pain and loss of function that can affect their quality of life.

“Patients may also experience psychological harm and increased risk of opioid use disorder and death by suicide following opioid deprescribing,” Dr. Hall said. “Therefore, it is important for providers to carefully weigh the risks of prescribing and deprescribing and engage patients with person-centered communication and shared decision-making.”

The work was funded by grants from the University of Sydney and the National Health and Medical Research Council. Full disclosures are available in the original article. Dr. Hall has provided expert opinion to the health care consultancy firm Lumanity and Emergent BioSolutions regarding the overdose crisis.

A version of this article originally appeared on Medscape.com.

An expert panel of pain management clinicians has released what they say are the first international guidelines for general practitioners on opioid analgesic deprescribing in adults.

The recommendations describe best practices for stopping opioid therapy and emphasize slow tapering and individualized deprescribing plans tailored to each patient.

Developed by general practitioners, pain specialists, addiction specialists, pharmacists, registered nurses, consumers, and physiotherapists, the guidelines note that deprescribing may not be appropriate for every patient and that stopping abruptly can be associated with an increased risk of overdose.

“Internationally, we were seeing significant harms from opioids but also significant harms from unsolicited and abrupt opioid cessation,” said lead author Aili Langford, PhD, who conducted the study as a doctoral student at the University of Sydney. “It was clear that recommendations to support safe and person-centered opioid deprescribing were required.”

The findings were published online  in the Medical Journal of Australia.
 

Deprescribing plan

The consensus guidelines include 11 recommendations for deprescribing in adult patients who take at least one opioid for any type of pain.

Recommendations include implementing a deprescribing plan when opioids are first prescribed and gradual and individualized deprescribing, with regular monitoring and review.

Clinicians should consider opioid deprescribing in patients who experience no clinically meaningful improvement in function, quality of life, or pain at high risk with opioid therapy, they note. Patients who are at high risk for opioid-related harm are also good candidates for deprescribing.

Stopping opioid therapy is not recommended for patients with severe opioid use disorder (OUD). In those patients, medication-assisted OUD treatment and other evidence-based interventions are recommended.

“Opioids can be effective in pain management,” co-author Carl Schneider, PhD, an associate professor of pharmacy at the University of Sydney, said in a press release. “However, over the longer term, the risk of harms may outweigh the benefits.”
 

A ‘global problem’

Commenting on the guidelines, Orman Trent Hall, DO, assistant professor of addiction medicine, department of psychiatry and behavioral health at the Ohio State University Wexner Medical Center, Columbus, said they are similar to recommendations published by the U.S. Centers for Disease Control and Prevention in 2016 and 2022 but offer additional information that could be helpful.

“This new guideline provides more explicit advice about tapering and withdrawal management, which may be useful to practitioners. The opioid crisis is a global problem, and while individual countries may require local solutions, the new international guideline may offer a framework for approaching this issue,” he said.

The guideline’s emphasis on the potential risks of deprescribing in some patients is also key, Dr. Hall added. Patients who are tapering off opioid therapy may have worsening pain and loss of function that can affect their quality of life.

“Patients may also experience psychological harm and increased risk of opioid use disorder and death by suicide following opioid deprescribing,” Dr. Hall said. “Therefore, it is important for providers to carefully weigh the risks of prescribing and deprescribing and engage patients with person-centered communication and shared decision-making.”

The work was funded by grants from the University of Sydney and the National Health and Medical Research Council. Full disclosures are available in the original article. Dr. Hall has provided expert opinion to the health care consultancy firm Lumanity and Emergent BioSolutions regarding the overdose crisis.

A version of this article originally appeared on Medscape.com.

Younger colorectal cancer patients with metastatic disease had distinct patterns of adverse events and worse survival than older patients with the same type of cancer, results of a new study indicate.

The incidence of metastatic colorectal cancer (mCRC) among adults younger than 50 years has been increasing, and although younger patients are treated with aggressive regimens similarly to older patients, outcomes data, including incidence of toxic effects, across age groups are limited, wrote Lingbin Meng, MD, of H. Lee Moffitt Cancer Center & Research Institute, Tampa, Fla., and colleagues, in their paper on the new research.

“Studies on the age-related disparity ... provided mixed findings,” said corresponding author Hao Xie, MD, of the Mayo Clinic in Rochester, Minn., in an interview.

According to the paper, published in JAMA Network Open, the researchers sought to evaluate the association between age and mCRC treatment-related adverse events and survival.

The study population included 1,223 mCRC patients who underwent first-line treatment with fluorouracil and oxaliplatin therapy in three clinical trials. An additional 736 patients with mCRC from the Moffitt Cancer Center were used to assess genomic alterations and serve as an external validation cohort.
 

Methods and results

Patients were divided into three age groups: younger than 50 years, 50-65 years, and 65 years and older. Early onset was defined as younger than 50 years. Approximately 58% of the study population was male.

The primary outcomes were treatment-related adverse events and survival rates. Overall survival (OS) and progression-free survival (PFS) were was significantly shorter in the early-onset group, compared with the 50-65 years group (hazard ratios, 1.48 and 1.46, respectively, P < .001 for both) in a multivariate analysis. The shorter OS in the early-onset group was confirmed in the validation cohort.

The early-onset patients had significantly higher incidence of nausea and vomiting, severe abdominal pain, severe anemia, and severe rash, compared with patients in both the 50- to 65-year-old group and the older than 65 years group. In addition, abdominal pain and severe liver toxicity effects were associated with shorter survival in the early-onset patients.

Genomic data from the Moffitt cohort showed a higher prevalence of CTNNB1 mutation among patients younger than 50 years, compared with the 50- to 65-year-old group and the older than 65 years group (6.6%, 3.1%, and 2.3%, respectively; P = .047), as well as ERBB2 amplification (5.1%, 0.6%, and 2.3%, respectively; P = .005), and CREBBP mutation (3.1%, 0.9%, and 0.5%; P = .05), although the prevalence of BRAF mutation was significantly lower in the younger patients, compared with patients in the older groups (7.7%, 8.5%, and 16.7%, respectively; P = .002).

These data suggest that distinct genomic profiles may play a role in the worse outcomes for patients with early-onset mCRC, the researchers said.

The findings were limited by several factors, including the timing of the trials prior to the use of biologics as standard first-line therapy, the researchers noted. Other limitations include a lack of data on treatment adherence and intensity and the location and number of metastases, and potential limited generalizability to other populations given that the majority of the participants were white, they said.
 

Data support individualized treatment

“We were surprised to find that patients with early-onset metastatic colorectal cancer had worse survival outcome, compared to older patients with metastatic colorectal cancer,” Dr. Xie said, in an interview. “On the other hand, we were not surprised to find unique adverse-event patterns in patients with early-onset metastatic colorectal cancer.”

For clinicians, “The take home message is that we should adopt individualized management approaches [regarding] cancer-direct treatments and treatment-related side effects in patients with early-onset metastatic colorectal cancer,” said Dr. Xie. However, more research is needed in the form of prospective and interventional studies to address treatment-related side effects and to develop novel and personalized therapeutics for patients with early-onset metastatic colorectal cancer, Dr. Xie added.
 

Early-onset cancer concerns persist

“The increasing shift in early colon cancers demands better understanding, in particular as we attempt a more patient-focused approach to treatments,” said David A. Johnson, MD, chief of gastroenterology at Eastern Virginia Medical School, Norfolk, in an interview. “Clearly, genetic amplifications and oncogene mutations play an essential role in tumorigenesis and tumor progression, but data on specifics are needed.”

With regard to the current study, “it makes sense that the pathways to CRC development and progression at least in part play a role in age-related cancers,” said Dr. Johnson, who was not involved in the study.

The clinical implications from the study are that early-onset CRC “can be aggressive and progressive,” Dr. Johnson said. “Younger patients need to recognize the earlier ages for beginning CRC screening, age 45 years for those at average risk, and certainly report any new sign or symptom to their care provider, in particular blood in the stool.”

As for additional research, “The oncogenetic markers will be helpful in guiding treatment approaches to be more individual specific, rather than just disease focused,” Dr. Johnson said. “The role of the gut microbiome will need evaluation as it relates to these oncogenetic factors,” he noted. Considerations include not only the potential influence of the gut microbiome on the expression of these factors, but also the impact of the gut microbiome on the chemotherapeutic response, as has been evident with checkpoint inhibitors, he added.

The study was supported by a Moffitt Support Grant to Dr. Xie and the University of South Florida Continuing Medical Education Funding. The researchers had no financial conflicts to disclose. Dr. Johnson had no relevant financial conflicts to disclose.

Younger colorectal cancer patients with metastatic disease had distinct patterns of adverse events and worse survival than older patients with the same type of cancer, results of a new study indicate.

The incidence of metastatic colorectal cancer (mCRC) among adults younger than 50 years has been increasing, and although younger patients are treated with aggressive regimens similarly to older patients, outcomes data, including incidence of toxic effects, across age groups are limited, wrote Lingbin Meng, MD, of H. Lee Moffitt Cancer Center & Research Institute, Tampa, Fla., and colleagues, in their paper on the new research.

“Studies on the age-related disparity ... provided mixed findings,” said corresponding author Hao Xie, MD, of the Mayo Clinic in Rochester, Minn., in an interview.

According to the paper, published in JAMA Network Open, the researchers sought to evaluate the association between age and mCRC treatment-related adverse events and survival.

The study population included 1,223 mCRC patients who underwent first-line treatment with fluorouracil and oxaliplatin therapy in three clinical trials. An additional 736 patients with mCRC from the Moffitt Cancer Center were used to assess genomic alterations and serve as an external validation cohort.
 

Methods and results

Patients were divided into three age groups: younger than 50 years, 50-65 years, and 65 years and older. Early onset was defined as younger than 50 years. Approximately 58% of the study population was male.

The primary outcomes were treatment-related adverse events and survival rates. Overall survival (OS) and progression-free survival (PFS) were was significantly shorter in the early-onset group, compared with the 50-65 years group (hazard ratios, 1.48 and 1.46, respectively, P < .001 for both) in a multivariate analysis. The shorter OS in the early-onset group was confirmed in the validation cohort.

The early-onset patients had significantly higher incidence of nausea and vomiting, severe abdominal pain, severe anemia, and severe rash, compared with patients in both the 50- to 65-year-old group and the older than 65 years group. In addition, abdominal pain and severe liver toxicity effects were associated with shorter survival in the early-onset patients.

Genomic data from the Moffitt cohort showed a higher prevalence of CTNNB1 mutation among patients younger than 50 years, compared with the 50- to 65-year-old group and the older than 65 years group (6.6%, 3.1%, and 2.3%, respectively; P = .047), as well as ERBB2 amplification (5.1%, 0.6%, and 2.3%, respectively; P = .005), and CREBBP mutation (3.1%, 0.9%, and 0.5%; P = .05), although the prevalence of BRAF mutation was significantly lower in the younger patients, compared with patients in the older groups (7.7%, 8.5%, and 16.7%, respectively; P = .002).

These data suggest that distinct genomic profiles may play a role in the worse outcomes for patients with early-onset mCRC, the researchers said.

The findings were limited by several factors, including the timing of the trials prior to the use of biologics as standard first-line therapy, the researchers noted. Other limitations include a lack of data on treatment adherence and intensity and the location and number of metastases, and potential limited generalizability to other populations given that the majority of the participants were white, they said.
 

Data support individualized treatment

“We were surprised to find that patients with early-onset metastatic colorectal cancer had worse survival outcome, compared to older patients with metastatic colorectal cancer,” Dr. Xie said, in an interview. “On the other hand, we were not surprised to find unique adverse-event patterns in patients with early-onset metastatic colorectal cancer.”

For clinicians, “The take home message is that we should adopt individualized management approaches [regarding] cancer-direct treatments and treatment-related side effects in patients with early-onset metastatic colorectal cancer,” said Dr. Xie. However, more research is needed in the form of prospective and interventional studies to address treatment-related side effects and to develop novel and personalized therapeutics for patients with early-onset metastatic colorectal cancer, Dr. Xie added.
 

Early-onset cancer concerns persist

“The increasing shift in early colon cancers demands better understanding, in particular as we attempt a more patient-focused approach to treatments,” said David A. Johnson, MD, chief of gastroenterology at Eastern Virginia Medical School, Norfolk, in an interview. “Clearly, genetic amplifications and oncogene mutations play an essential role in tumorigenesis and tumor progression, but data on specifics are needed.”

With regard to the current study, “it makes sense that the pathways to CRC development and progression at least in part play a role in age-related cancers,” said Dr. Johnson, who was not involved in the study.

The clinical implications from the study are that early-onset CRC “can be aggressive and progressive,” Dr. Johnson said. “Younger patients need to recognize the earlier ages for beginning CRC screening, age 45 years for those at average risk, and certainly report any new sign or symptom to their care provider, in particular blood in the stool.”

As for additional research, “The oncogenetic markers will be helpful in guiding treatment approaches to be more individual specific, rather than just disease focused,” Dr. Johnson said. “The role of the gut microbiome will need evaluation as it relates to these oncogenetic factors,” he noted. Considerations include not only the potential influence of the gut microbiome on the expression of these factors, but also the impact of the gut microbiome on the chemotherapeutic response, as has been evident with checkpoint inhibitors, he added.

The study was supported by a Moffitt Support Grant to Dr. Xie and the University of South Florida Continuing Medical Education Funding. The researchers had no financial conflicts to disclose. Dr. Johnson had no relevant financial conflicts to disclose.

Younger colorectal cancer patients with metastatic disease had distinct patterns of adverse events and worse survival than older patients with the same type of cancer, results of a new study indicate.

The incidence of metastatic colorectal cancer (mCRC) among adults younger than 50 years has been increasing, and although younger patients are treated with aggressive regimens similarly to older patients, outcomes data, including incidence of toxic effects, across age groups are limited, wrote Lingbin Meng, MD, of H. Lee Moffitt Cancer Center & Research Institute, Tampa, Fla., and colleagues, in their paper on the new research.

“Studies on the age-related disparity ... provided mixed findings,” said corresponding author Hao Xie, MD, of the Mayo Clinic in Rochester, Minn., in an interview.

According to the paper, published in JAMA Network Open, the researchers sought to evaluate the association between age and mCRC treatment-related adverse events and survival.

The study population included 1,223 mCRC patients who underwent first-line treatment with fluorouracil and oxaliplatin therapy in three clinical trials. An additional 736 patients with mCRC from the Moffitt Cancer Center were used to assess genomic alterations and serve as an external validation cohort.
 

Methods and results

Patients were divided into three age groups: younger than 50 years, 50-65 years, and 65 years and older. Early onset was defined as younger than 50 years. Approximately 58% of the study population was male.

The primary outcomes were treatment-related adverse events and survival rates. Overall survival (OS) and progression-free survival (PFS) were was significantly shorter in the early-onset group, compared with the 50-65 years group (hazard ratios, 1.48 and 1.46, respectively, P < .001 for both) in a multivariate analysis. The shorter OS in the early-onset group was confirmed in the validation cohort.

The early-onset patients had significantly higher incidence of nausea and vomiting, severe abdominal pain, severe anemia, and severe rash, compared with patients in both the 50- to 65-year-old group and the older than 65 years group. In addition, abdominal pain and severe liver toxicity effects were associated with shorter survival in the early-onset patients.

Genomic data from the Moffitt cohort showed a higher prevalence of CTNNB1 mutation among patients younger than 50 years, compared with the 50- to 65-year-old group and the older than 65 years group (6.6%, 3.1%, and 2.3%, respectively; P = .047), as well as ERBB2 amplification (5.1%, 0.6%, and 2.3%, respectively; P = .005), and CREBBP mutation (3.1%, 0.9%, and 0.5%; P = .05), although the prevalence of BRAF mutation was significantly lower in the younger patients, compared with patients in the older groups (7.7%, 8.5%, and 16.7%, respectively; P = .002).

These data suggest that distinct genomic profiles may play a role in the worse outcomes for patients with early-onset mCRC, the researchers said.

The findings were limited by several factors, including the timing of the trials prior to the use of biologics as standard first-line therapy, the researchers noted. Other limitations include a lack of data on treatment adherence and intensity and the location and number of metastases, and potential limited generalizability to other populations given that the majority of the participants were white, they said.
 

Data support individualized treatment

“We were surprised to find that patients with early-onset metastatic colorectal cancer had worse survival outcome, compared to older patients with metastatic colorectal cancer,” Dr. Xie said, in an interview. “On the other hand, we were not surprised to find unique adverse-event patterns in patients with early-onset metastatic colorectal cancer.”

For clinicians, “The take home message is that we should adopt individualized management approaches [regarding] cancer-direct treatments and treatment-related side effects in patients with early-onset metastatic colorectal cancer,” said Dr. Xie. However, more research is needed in the form of prospective and interventional studies to address treatment-related side effects and to develop novel and personalized therapeutics for patients with early-onset metastatic colorectal cancer, Dr. Xie added.
 

Early-onset cancer concerns persist

“The increasing shift in early colon cancers demands better understanding, in particular as we attempt a more patient-focused approach to treatments,” said David A. Johnson, MD, chief of gastroenterology at Eastern Virginia Medical School, Norfolk, in an interview. “Clearly, genetic amplifications and oncogene mutations play an essential role in tumorigenesis and tumor progression, but data on specifics are needed.”

With regard to the current study, “it makes sense that the pathways to CRC development and progression at least in part play a role in age-related cancers,” said Dr. Johnson, who was not involved in the study.

The clinical implications from the study are that early-onset CRC “can be aggressive and progressive,” Dr. Johnson said. “Younger patients need to recognize the earlier ages for beginning CRC screening, age 45 years for those at average risk, and certainly report any new sign or symptom to their care provider, in particular blood in the stool.”

As for additional research, “The oncogenetic markers will be helpful in guiding treatment approaches to be more individual specific, rather than just disease focused,” Dr. Johnson said. “The role of the gut microbiome will need evaluation as it relates to these oncogenetic factors,” he noted. Considerations include not only the potential influence of the gut microbiome on the expression of these factors, but also the impact of the gut microbiome on the chemotherapeutic response, as has been evident with checkpoint inhibitors, he added.

The study was supported by a Moffitt Support Grant to Dr. Xie and the University of South Florida Continuing Medical Education Funding. The researchers had no financial conflicts to disclose. Dr. Johnson had no relevant financial conflicts to disclose.

CHICAGO – Clinical trials are so White. Only a small percentage of eligible patients participate in clinical trials in the first place, and very few come from racial and ethnic minority groups.

For example, according to the Food and Drug Administration, in trials that resulted in drug approvals from 2017 to 2020, only 2%-5% of participants were Black patients.

When clinical trials lack diverse patient populations, those who are left out have fewer opportunities to get new therapies. Moreover, the scope of the research is limited by smaller phenotypic and genotypic samples, and the trial results are applicable only to more homogeneous patient groups.

There has been a push to include more underrepresented patients in clinical trials. One group reported its success in doing so here at the annual meeting of the American Society of Clinical Oncology.

Researchers from the Alliance for Clinical Trials in Oncology explained how a multifaceted approach resulted in a 75% relative improvement in trial enrollment from 2014 to 2022, a period that included a pandemic-induced hiatus in clinical trials in general.

Alliance member Electra D. Paskett, PhD, from the College of Public Health at the Ohio State University in Columbus, presented accrual data from 117 trials led by the Alliance from 2014 to 2022.

During this period, accrual of racial and ethnic minority patients increased from 13.6% to 25.3% for cancer treatment trials and from 13% to 21.5% for cancer control trials.

Overall, the recruitment program resulted in an absolute increase from 13.5 % to 23.6% of underrepresented populations, which translated into a relative 74.8% improvement.

“We’re focusing now on monitoring accrual of women, rural populations, younger AYAs [adolescents and young adults] and older patients, and we’ll see what strategies we need to implement,” Dr. Packett told this news organization.

The Alliance has implemented a real-time accrual dashboard on its website that allows individual sites to review accrual by trial and overall for all of the identified underrepresented populations, she noted.
 

Program to increase underrepresented patient accrual

The impetus for the program to increase enrollment of underrepresented patients came from the goal set by Monica M. Bertagnolli, MD, group chair of the Alliance from 2011 to 2022 and currently the director of the U.S. National Cancer Institute.

“Our leader, Dr. Bertagnolli, set out a group-wide goal for accrual of underrepresented minorities to our trials of 20%, and that gave us permission to implement a whole host of new strategies,” Dr. Paskett said in an interview.

“These strategies follow the Accrual of Clinical Trials framework, which essentially says that the interaction between the patient and the provider for going on a clinical trial is not just an interaction between the patient and provider but recognizes, for example, that the provider has coworkers and they have norms and beliefs and attitudes, and the patient comes from a family with their own values. And then there are system-level barriers, and there are community barriers that all relate to this interaction about going on a trial,” Dr. Packett said.
 

What works?

The study was presented as a poster at the meeting. During the poster discussion session, comoderator Victoria S. Blinder, MD, from Memorial Sloan Kettering Cancer Center in New York, asked Dr. Paskett, “If you had a certain amount of money and you really wanted to use that resource to focus on one area, where would you put that resource?”

“I’m going to violate the rules of your question,” Dr. Paskett replied.

“You cannot change this problem by focusing on one thing, and that’s what we showed in our Alliance poster, and what I’ve said is based on over 30 years of work in this area,” she said.

She cited what she considered as the two most important components for improving accrual of underrepresented populations: a commitment by leadership to a recruitment goal, and the development of protocols with specific accrual goals for minority populations.

Still, those are only two components of a comprehensive program that includes the aforementioned accrual goal set by Dr. Bertagnolli, as well as the following:

• Funding of minority junior investigators and research that focuses on issues of concern to underrepresented populations.
• Establishment of work groups that focus on specific populations with the Alliance health disparities committee.
• Translation of informational materials for patients.
• Opening studies at National Cancer Institute Community. Oncology Research Program–designated minority underserved sites.
• Real-time monitoring of accrual demographics by the Alliance and at the trial site.
• Closing protocol enrollment to majority populations.
• Increasing the study sample sizes to enroll additional minority participants and to allow for subgroup analyses.

The study was funded by the National Institutes of Health. Dr. Packett and Dr. Blinder reported no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

CHICAGO – Clinical trials are so White. Only a small percentage of eligible patients participate in clinical trials in the first place, and very few come from racial and ethnic minority groups.

For example, according to the Food and Drug Administration, in trials that resulted in drug approvals from 2017 to 2020, only 2%-5% of participants were Black patients.

When clinical trials lack diverse patient populations, those who are left out have fewer opportunities to get new therapies. Moreover, the scope of the research is limited by smaller phenotypic and genotypic samples, and the trial results are applicable only to more homogeneous patient groups.

There has been a push to include more underrepresented patients in clinical trials. One group reported its success in doing so here at the annual meeting of the American Society of Clinical Oncology.

Researchers from the Alliance for Clinical Trials in Oncology explained how a multifaceted approach resulted in a 75% relative improvement in trial enrollment from 2014 to 2022, a period that included a pandemic-induced hiatus in clinical trials in general.

Alliance member Electra D. Paskett, PhD, from the College of Public Health at the Ohio State University in Columbus, presented accrual data from 117 trials led by the Alliance from 2014 to 2022.

During this period, accrual of racial and ethnic minority patients increased from 13.6% to 25.3% for cancer treatment trials and from 13% to 21.5% for cancer control trials.

Overall, the recruitment program resulted in an absolute increase from 13.5 % to 23.6% of underrepresented populations, which translated into a relative 74.8% improvement.

“We’re focusing now on monitoring accrual of women, rural populations, younger AYAs [adolescents and young adults] and older patients, and we’ll see what strategies we need to implement,” Dr. Packett told this news organization.

The Alliance has implemented a real-time accrual dashboard on its website that allows individual sites to review accrual by trial and overall for all of the identified underrepresented populations, she noted.
 

Program to increase underrepresented patient accrual

The impetus for the program to increase enrollment of underrepresented patients came from the goal set by Monica M. Bertagnolli, MD, group chair of the Alliance from 2011 to 2022 and currently the director of the U.S. National Cancer Institute.

“Our leader, Dr. Bertagnolli, set out a group-wide goal for accrual of underrepresented minorities to our trials of 20%, and that gave us permission to implement a whole host of new strategies,” Dr. Paskett said in an interview.

“These strategies follow the Accrual of Clinical Trials framework, which essentially says that the interaction between the patient and the provider for going on a clinical trial is not just an interaction between the patient and provider but recognizes, for example, that the provider has coworkers and they have norms and beliefs and attitudes, and the patient comes from a family with their own values. And then there are system-level barriers, and there are community barriers that all relate to this interaction about going on a trial,” Dr. Packett said.
 

What works?

The study was presented as a poster at the meeting. During the poster discussion session, comoderator Victoria S. Blinder, MD, from Memorial Sloan Kettering Cancer Center in New York, asked Dr. Paskett, “If you had a certain amount of money and you really wanted to use that resource to focus on one area, where would you put that resource?”

“I’m going to violate the rules of your question,” Dr. Paskett replied.

“You cannot change this problem by focusing on one thing, and that’s what we showed in our Alliance poster, and what I’ve said is based on over 30 years of work in this area,” she said.

She cited what she considered as the two most important components for improving accrual of underrepresented populations: a commitment by leadership to a recruitment goal, and the development of protocols with specific accrual goals for minority populations.

Still, those are only two components of a comprehensive program that includes the aforementioned accrual goal set by Dr. Bertagnolli, as well as the following:

• Funding of minority junior investigators and research that focuses on issues of concern to underrepresented populations.
• Establishment of work groups that focus on specific populations with the Alliance health disparities committee.
• Translation of informational materials for patients.
• Opening studies at National Cancer Institute Community. Oncology Research Program–designated minority underserved sites.
• Real-time monitoring of accrual demographics by the Alliance and at the trial site.
• Closing protocol enrollment to majority populations.
• Increasing the study sample sizes to enroll additional minority participants and to allow for subgroup analyses.

The study was funded by the National Institutes of Health. Dr. Packett and Dr. Blinder reported no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

CHICAGO – Clinical trials are so White. Only a small percentage of eligible patients participate in clinical trials in the first place, and very few come from racial and ethnic minority groups.

For example, according to the Food and Drug Administration, in trials that resulted in drug approvals from 2017 to 2020, only 2%-5% of participants were Black patients.

When clinical trials lack diverse patient populations, those who are left out have fewer opportunities to get new therapies. Moreover, the scope of the research is limited by smaller phenotypic and genotypic samples, and the trial results are applicable only to more homogeneous patient groups.

There has been a push to include more underrepresented patients in clinical trials. One group reported its success in doing so here at the annual meeting of the American Society of Clinical Oncology.

Researchers from the Alliance for Clinical Trials in Oncology explained how a multifaceted approach resulted in a 75% relative improvement in trial enrollment from 2014 to 2022, a period that included a pandemic-induced hiatus in clinical trials in general.

Alliance member Electra D. Paskett, PhD, from the College of Public Health at the Ohio State University in Columbus, presented accrual data from 117 trials led by the Alliance from 2014 to 2022.

During this period, accrual of racial and ethnic minority patients increased from 13.6% to 25.3% for cancer treatment trials and from 13% to 21.5% for cancer control trials.

Overall, the recruitment program resulted in an absolute increase from 13.5 % to 23.6% of underrepresented populations, which translated into a relative 74.8% improvement.

“We’re focusing now on monitoring accrual of women, rural populations, younger AYAs [adolescents and young adults] and older patients, and we’ll see what strategies we need to implement,” Dr. Packett told this news organization.

The Alliance has implemented a real-time accrual dashboard on its website that allows individual sites to review accrual by trial and overall for all of the identified underrepresented populations, she noted.
 

Program to increase underrepresented patient accrual

The impetus for the program to increase enrollment of underrepresented patients came from the goal set by Monica M. Bertagnolli, MD, group chair of the Alliance from 2011 to 2022 and currently the director of the U.S. National Cancer Institute.

“Our leader, Dr. Bertagnolli, set out a group-wide goal for accrual of underrepresented minorities to our trials of 20%, and that gave us permission to implement a whole host of new strategies,” Dr. Paskett said in an interview.

“These strategies follow the Accrual of Clinical Trials framework, which essentially says that the interaction between the patient and the provider for going on a clinical trial is not just an interaction between the patient and provider but recognizes, for example, that the provider has coworkers and they have norms and beliefs and attitudes, and the patient comes from a family with their own values. And then there are system-level barriers, and there are community barriers that all relate to this interaction about going on a trial,” Dr. Packett said.
 

What works?

The study was presented as a poster at the meeting. During the poster discussion session, comoderator Victoria S. Blinder, MD, from Memorial Sloan Kettering Cancer Center in New York, asked Dr. Paskett, “If you had a certain amount of money and you really wanted to use that resource to focus on one area, where would you put that resource?”

“I’m going to violate the rules of your question,” Dr. Paskett replied.

“You cannot change this problem by focusing on one thing, and that’s what we showed in our Alliance poster, and what I’ve said is based on over 30 years of work in this area,” she said.

She cited what she considered as the two most important components for improving accrual of underrepresented populations: a commitment by leadership to a recruitment goal, and the development of protocols with specific accrual goals for minority populations.

Still, those are only two components of a comprehensive program that includes the aforementioned accrual goal set by Dr. Bertagnolli, as well as the following:

• Funding of minority junior investigators and research that focuses on issues of concern to underrepresented populations.
• Establishment of work groups that focus on specific populations with the Alliance health disparities committee.
• Translation of informational materials for patients.
• Opening studies at National Cancer Institute Community. Oncology Research Program–designated minority underserved sites.
• Real-time monitoring of accrual demographics by the Alliance and at the trial site.
• Closing protocol enrollment to majority populations.
• Increasing the study sample sizes to enroll additional minority participants and to allow for subgroup analyses.

The study was funded by the National Institutes of Health. Dr. Packett and Dr. Blinder reported no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

CHICAGO – One-third of patients with cancer also experience anxiety or depression, and an estimated 70% of the 18 million patients with cancer and cancer survivors in the US experience emotional symptoms, including fear of recurrence.

Despite many having these symptoms, few patients with cancer have access to psycho-oncologic support.

A digital cognitive-behavioral stress management (CBSM) application may help to ease some of the burden, reported Allison Ramiller, MPH, of Blue Note Therapeutics in San Francisco, which developed the app version of the program.

In the randomized controlled RESTORE study, use of the cell phone–based CBSM app was associated with significantly greater reduction in symptoms of anxiety and depression compared with a digital health education control app.

In addition, patients assigned to the CBSM app were twice as likely as control persons to report that their symptoms were “much” or “very much” improved after using the app for 12 weeks, Ms. Ramiller reported at an oral abstract session at the annual meeting of the American Society of Clinical Oncology (ASCO).

However, the investigators did not report baseline characteristics of patients in each of the study arms, which might have helped to clarify the depth of the effects they saw.

The CBSM program was developed by Michael H. Antoni, PhD, and colleagues in the University of Miami Health System. It is based on cognitive-behavioral therapy but also includes stress management and relaxation techniques to help patients cope with cancer-specific stress.

“”It has been clinically validated and shown to benefit patients with cancer,” Ms. Ramiller said. “However, access is a problem,” she said.

“There aren’t enough qualified, trained providers for the need, and patients with cancer encounter barriers to in-person participation, including things like transportation or financial barriers. So to overcome this, we developed a digitized version of CBSM,” she explained.
 

Impressive and elegant

“Everything about [the study] I thought was very impressive, very elegant, very nicely done,” said invited discussant Raymond U. Osarogiagbon, MBBS, FACP, chief scientist at Baptist Memorial Health Care Corp in Memphis, Tenn.

“They showed efficacy, they showed safety – very nice – user friendliness – very good. Certainly they look like they’re trying to address a highly important, unmet need in a very elegant way. Certainly, they pointed out it needs longer follow-up to see sustainability. We need to see will this work in other settings. Will this be cost-effective? You’ve gotta believe it probably will be,” he said.

CBSM has previously been shown to help patients with cancer reduce stress, improve general and cancer-specific quality of life at various stages of treatment, reduce symptom burden, and improve coping skills, Ms. Ramiller said.

To see whether these benefits could be conveyed digitally rather than in face-to-face encounters, Ms. Ramiller and colleagues worked with Dr. Antoni to develop the CBSM app.

Patients using the app received therapeutic content over 10 sessions with audio, video, and interactive tools that mimicked the sessions they would have received during in-person interventions.

They then compared the app against the control educational app in the randomized, decentralized RESTORE study.
 

High-quality control

Ms. Ramiller said that the control app set “a high bar.”

“The control also offered 10 interactive self-guided sessions. Both treatment apps were professionally designed and visually similar in styling, and they were presented as digital therapeutic-specific for cancer patients. And they were also in a match condition, meaning they received the same attention from study staff and cadence of reminders, but importantly, only the intervention app was based on CBSM,” she explained.

A total of 449 patients with cancers of stage I–III who were undergoing active systemic treatment or were planning to undergo such treatment within 6 months were randomly assigned to the CBSM app or the control app.

The CBSM app was superior to the control app for the primary outcome of anxiety reduction over baseline, as measured at 4, 8 and 12 weeks by the Patient-Reported Outcomes Measurement Information System Anxiety Scale (PROMIS-A) (beta = -.03; P = .019).

CBSM was also significantly better than the control app for the secondary endpoints of reducing symptoms of depression, as measured by the PROMIS-D scale (beta = -.02, P = .042), and also at increasing the percentage of patients who reported improvement in anxiety and depression symptoms on the Patient Global Impression of Change instrument (P < .001)

An extension study of the durability of the effects at 3 and 6 months is underway.

The investigators noted that the incremental cost of management of anxiety or depression is greater than $17,000 per patient per year.

“One of the big promises of a digital therapeutic like this is that it could potentially reduce costs,” Ms. Ramiller told the audience, but she acknowledged, “More work is really needed, however, to directly test the potential savings.”

The RESTORE study is funded by Blue Note Therapeutics. Dr. Osarogiagbon owns stock in Gilead, Lilly, and Pfizer, has received honoraria from Biodesix and Medscape, and has a consulting or advisory role for the American Cancer Society AstraZeneca, Genentech/Roche, LUNGevity, National Cancer Institute, and Triptych Health Partners.
 

A version of this article originally appeared on Medscape.com.

CHICAGO – One-third of patients with cancer also experience anxiety or depression, and an estimated 70% of the 18 million patients with cancer and cancer survivors in the US experience emotional symptoms, including fear of recurrence.

Despite many having these symptoms, few patients with cancer have access to psycho-oncologic support.

A digital cognitive-behavioral stress management (CBSM) application may help to ease some of the burden, reported Allison Ramiller, MPH, of Blue Note Therapeutics in San Francisco, which developed the app version of the program.

In the randomized controlled RESTORE study, use of the cell phone–based CBSM app was associated with significantly greater reduction in symptoms of anxiety and depression compared with a digital health education control app.

In addition, patients assigned to the CBSM app were twice as likely as control persons to report that their symptoms were “much” or “very much” improved after using the app for 12 weeks, Ms. Ramiller reported at an oral abstract session at the annual meeting of the American Society of Clinical Oncology (ASCO).

However, the investigators did not report baseline characteristics of patients in each of the study arms, which might have helped to clarify the depth of the effects they saw.

The CBSM program was developed by Michael H. Antoni, PhD, and colleagues in the University of Miami Health System. It is based on cognitive-behavioral therapy but also includes stress management and relaxation techniques to help patients cope with cancer-specific stress.

“”It has been clinically validated and shown to benefit patients with cancer,” Ms. Ramiller said. “However, access is a problem,” she said.

“There aren’t enough qualified, trained providers for the need, and patients with cancer encounter barriers to in-person participation, including things like transportation or financial barriers. So to overcome this, we developed a digitized version of CBSM,” she explained.
 

Impressive and elegant

“Everything about [the study] I thought was very impressive, very elegant, very nicely done,” said invited discussant Raymond U. Osarogiagbon, MBBS, FACP, chief scientist at Baptist Memorial Health Care Corp in Memphis, Tenn.

“They showed efficacy, they showed safety – very nice – user friendliness – very good. Certainly they look like they’re trying to address a highly important, unmet need in a very elegant way. Certainly, they pointed out it needs longer follow-up to see sustainability. We need to see will this work in other settings. Will this be cost-effective? You’ve gotta believe it probably will be,” he said.

CBSM has previously been shown to help patients with cancer reduce stress, improve general and cancer-specific quality of life at various stages of treatment, reduce symptom burden, and improve coping skills, Ms. Ramiller said.

To see whether these benefits could be conveyed digitally rather than in face-to-face encounters, Ms. Ramiller and colleagues worked with Dr. Antoni to develop the CBSM app.

Patients using the app received therapeutic content over 10 sessions with audio, video, and interactive tools that mimicked the sessions they would have received during in-person interventions.

They then compared the app against the control educational app in the randomized, decentralized RESTORE study.
 

High-quality control

Ms. Ramiller said that the control app set “a high bar.”

“The control also offered 10 interactive self-guided sessions. Both treatment apps were professionally designed and visually similar in styling, and they were presented as digital therapeutic-specific for cancer patients. And they were also in a match condition, meaning they received the same attention from study staff and cadence of reminders, but importantly, only the intervention app was based on CBSM,” she explained.

A total of 449 patients with cancers of stage I–III who were undergoing active systemic treatment or were planning to undergo such treatment within 6 months were randomly assigned to the CBSM app or the control app.

The CBSM app was superior to the control app for the primary outcome of anxiety reduction over baseline, as measured at 4, 8 and 12 weeks by the Patient-Reported Outcomes Measurement Information System Anxiety Scale (PROMIS-A) (beta = -.03; P = .019).

CBSM was also significantly better than the control app for the secondary endpoints of reducing symptoms of depression, as measured by the PROMIS-D scale (beta = -.02, P = .042), and also at increasing the percentage of patients who reported improvement in anxiety and depression symptoms on the Patient Global Impression of Change instrument (P < .001)

An extension study of the durability of the effects at 3 and 6 months is underway.

The investigators noted that the incremental cost of management of anxiety or depression is greater than $17,000 per patient per year.

“One of the big promises of a digital therapeutic like this is that it could potentially reduce costs,” Ms. Ramiller told the audience, but she acknowledged, “More work is really needed, however, to directly test the potential savings.”

The RESTORE study is funded by Blue Note Therapeutics. Dr. Osarogiagbon owns stock in Gilead, Lilly, and Pfizer, has received honoraria from Biodesix and Medscape, and has a consulting or advisory role for the American Cancer Society AstraZeneca, Genentech/Roche, LUNGevity, National Cancer Institute, and Triptych Health Partners.
 

A version of this article originally appeared on Medscape.com.

CHICAGO – One-third of patients with cancer also experience anxiety or depression, and an estimated 70% of the 18 million patients with cancer and cancer survivors in the US experience emotional symptoms, including fear of recurrence.

Despite many having these symptoms, few patients with cancer have access to psycho-oncologic support.

A digital cognitive-behavioral stress management (CBSM) application may help to ease some of the burden, reported Allison Ramiller, MPH, of Blue Note Therapeutics in San Francisco, which developed the app version of the program.

In the randomized controlled RESTORE study, use of the cell phone–based CBSM app was associated with significantly greater reduction in symptoms of anxiety and depression compared with a digital health education control app.

In addition, patients assigned to the CBSM app were twice as likely as control persons to report that their symptoms were “much” or “very much” improved after using the app for 12 weeks, Ms. Ramiller reported at an oral abstract session at the annual meeting of the American Society of Clinical Oncology (ASCO).

However, the investigators did not report baseline characteristics of patients in each of the study arms, which might have helped to clarify the depth of the effects they saw.

The CBSM program was developed by Michael H. Antoni, PhD, and colleagues in the University of Miami Health System. It is based on cognitive-behavioral therapy but also includes stress management and relaxation techniques to help patients cope with cancer-specific stress.

“”It has been clinically validated and shown to benefit patients with cancer,” Ms. Ramiller said. “However, access is a problem,” she said.

“There aren’t enough qualified, trained providers for the need, and patients with cancer encounter barriers to in-person participation, including things like transportation or financial barriers. So to overcome this, we developed a digitized version of CBSM,” she explained.
 

Impressive and elegant

“Everything about [the study] I thought was very impressive, very elegant, very nicely done,” said invited discussant Raymond U. Osarogiagbon, MBBS, FACP, chief scientist at Baptist Memorial Health Care Corp in Memphis, Tenn.

“They showed efficacy, they showed safety – very nice – user friendliness – very good. Certainly they look like they’re trying to address a highly important, unmet need in a very elegant way. Certainly, they pointed out it needs longer follow-up to see sustainability. We need to see will this work in other settings. Will this be cost-effective? You’ve gotta believe it probably will be,” he said.

CBSM has previously been shown to help patients with cancer reduce stress, improve general and cancer-specific quality of life at various stages of treatment, reduce symptom burden, and improve coping skills, Ms. Ramiller said.

To see whether these benefits could be conveyed digitally rather than in face-to-face encounters, Ms. Ramiller and colleagues worked with Dr. Antoni to develop the CBSM app.

Patients using the app received therapeutic content over 10 sessions with audio, video, and interactive tools that mimicked the sessions they would have received during in-person interventions.

They then compared the app against the control educational app in the randomized, decentralized RESTORE study.
 

High-quality control

Ms. Ramiller said that the control app set “a high bar.”

“The control also offered 10 interactive self-guided sessions. Both treatment apps were professionally designed and visually similar in styling, and they were presented as digital therapeutic-specific for cancer patients. And they were also in a match condition, meaning they received the same attention from study staff and cadence of reminders, but importantly, only the intervention app was based on CBSM,” she explained.

A total of 449 patients with cancers of stage I–III who were undergoing active systemic treatment or were planning to undergo such treatment within 6 months were randomly assigned to the CBSM app or the control app.

The CBSM app was superior to the control app for the primary outcome of anxiety reduction over baseline, as measured at 4, 8 and 12 weeks by the Patient-Reported Outcomes Measurement Information System Anxiety Scale (PROMIS-A) (beta = -.03; P = .019).

CBSM was also significantly better than the control app for the secondary endpoints of reducing symptoms of depression, as measured by the PROMIS-D scale (beta = -.02, P = .042), and also at increasing the percentage of patients who reported improvement in anxiety and depression symptoms on the Patient Global Impression of Change instrument (P < .001)

An extension study of the durability of the effects at 3 and 6 months is underway.

The investigators noted that the incremental cost of management of anxiety or depression is greater than $17,000 per patient per year.

“One of the big promises of a digital therapeutic like this is that it could potentially reduce costs,” Ms. Ramiller told the audience, but she acknowledged, “More work is really needed, however, to directly test the potential savings.”

The RESTORE study is funded by Blue Note Therapeutics. Dr. Osarogiagbon owns stock in Gilead, Lilly, and Pfizer, has received honoraria from Biodesix and Medscape, and has a consulting or advisory role for the American Cancer Society AstraZeneca, Genentech/Roche, LUNGevity, National Cancer Institute, and Triptych Health Partners.
 

A version of this article originally appeared on Medscape.com.