Hematology Times

Patient receives chemotherapy

through a central line

Credit: Rhoda Baer

Healthcare-associated infections (HAIs) are on the decline in the US, according to a report by the Centers for Disease Control and Prevention (CDC).

The data show that most types of HAIs have decreased in recent years, with a particularly large decrease in the rate of central line-associated bloodstream infections (CLABSIs).

The National and State Healthcare-associated Infection Progress Report is a snapshot of how each state and the country are doing in eliminating the infections that hospitals are required to report to the CDC.

The report summarizes data submitted to the CDC’s National Healthcare Safety Network, the nation’s HAI tracking system, which is used by more than 14,500 healthcare facilities across all 50 states, Washington, DC, and Puerto Rico.

“Hospitals have made real progress to reduce some types of healthcare-associated infections; it can be done,” said CDC Director Tom Frieden, MD.

“The key is for every hospital to have rigorous infection control programs to protect patients and healthcare workers, and for health care facilities and others to work together to reduce the many types of infections that haven’t decreased enough.”

On the national level, the report showed a 46% decrease in CLABSIs between 2008 and 2013. It also revealed a 19% decrease in surgical site infections related to the 10 procedures tracked in the report between 2008 and 2013.

There was an 8% decrease in methicillin-resistant Staphylococcus aureus (MRSA) bloodstream infections between 2011 and 2013 and a 10% decrease in Clostridium difficile infections between 2011 and 2013.

However, there was a 6% increase in catheter-associated urinary tract infections since 2009.

Not all states reported or had enough data to calculate valid infection information on every infection in the report. But the CDC compared the number of infections reported to a national baseline.

And they found that 26 states performed better than the nation on at least 2 of the infection types. Sixteen states performed better than the nation on 3 or more infections, including 6 states performing better on 4 infections.

But 19 states performed worse than the nation on 2 infections, with 8 states performing worse on at least 3 infections.

The national baseline for HAIs will be reset at the end of 2015. Starting in 2016, HAI prevention progress from 2016 to 2020 will be measured in comparison to infection data from 2015.

Patient receives chemotherapy

through a central line

Credit: Rhoda Baer

Healthcare-associated infections (HAIs) are on the decline in the US, according to a report by the Centers for Disease Control and Prevention (CDC).

The data show that most types of HAIs have decreased in recent years, with a particularly large decrease in the rate of central line-associated bloodstream infections (CLABSIs).

The National and State Healthcare-associated Infection Progress Report is a snapshot of how each state and the country are doing in eliminating the infections that hospitals are required to report to the CDC.

The report summarizes data submitted to the CDC’s National Healthcare Safety Network, the nation’s HAI tracking system, which is used by more than 14,500 healthcare facilities across all 50 states, Washington, DC, and Puerto Rico.

“Hospitals have made real progress to reduce some types of healthcare-associated infections; it can be done,” said CDC Director Tom Frieden, MD.

“The key is for every hospital to have rigorous infection control programs to protect patients and healthcare workers, and for health care facilities and others to work together to reduce the many types of infections that haven’t decreased enough.”

On the national level, the report showed a 46% decrease in CLABSIs between 2008 and 2013. It also revealed a 19% decrease in surgical site infections related to the 10 procedures tracked in the report between 2008 and 2013.

There was an 8% decrease in methicillin-resistant Staphylococcus aureus (MRSA) bloodstream infections between 2011 and 2013 and a 10% decrease in Clostridium difficile infections between 2011 and 2013.

However, there was a 6% increase in catheter-associated urinary tract infections since 2009.

Not all states reported or had enough data to calculate valid infection information on every infection in the report. But the CDC compared the number of infections reported to a national baseline.

And they found that 26 states performed better than the nation on at least 2 of the infection types. Sixteen states performed better than the nation on 3 or more infections, including 6 states performing better on 4 infections.

But 19 states performed worse than the nation on 2 infections, with 8 states performing worse on at least 3 infections.

The national baseline for HAIs will be reset at the end of 2015. Starting in 2016, HAI prevention progress from 2016 to 2020 will be measured in comparison to infection data from 2015.

Patient receives chemotherapy

through a central line

Credit: Rhoda Baer

Healthcare-associated infections (HAIs) are on the decline in the US, according to a report by the Centers for Disease Control and Prevention (CDC).

The data show that most types of HAIs have decreased in recent years, with a particularly large decrease in the rate of central line-associated bloodstream infections (CLABSIs).

The National and State Healthcare-associated Infection Progress Report is a snapshot of how each state and the country are doing in eliminating the infections that hospitals are required to report to the CDC.

The report summarizes data submitted to the CDC’s National Healthcare Safety Network, the nation’s HAI tracking system, which is used by more than 14,500 healthcare facilities across all 50 states, Washington, DC, and Puerto Rico.

“Hospitals have made real progress to reduce some types of healthcare-associated infections; it can be done,” said CDC Director Tom Frieden, MD.

“The key is for every hospital to have rigorous infection control programs to protect patients and healthcare workers, and for health care facilities and others to work together to reduce the many types of infections that haven’t decreased enough.”

On the national level, the report showed a 46% decrease in CLABSIs between 2008 and 2013. It also revealed a 19% decrease in surgical site infections related to the 10 procedures tracked in the report between 2008 and 2013.

There was an 8% decrease in methicillin-resistant Staphylococcus aureus (MRSA) bloodstream infections between 2011 and 2013 and a 10% decrease in Clostridium difficile infections between 2011 and 2013.

However, there was a 6% increase in catheter-associated urinary tract infections since 2009.

Not all states reported or had enough data to calculate valid infection information on every infection in the report. But the CDC compared the number of infections reported to a national baseline.

And they found that 26 states performed better than the nation on at least 2 of the infection types. Sixteen states performed better than the nation on 3 or more infections, including 6 states performing better on 4 infections.

But 19 states performed worse than the nation on 2 infections, with 8 states performing worse on at least 3 infections.

The national baseline for HAIs will be reset at the end of 2015. Starting in 2016, HAI prevention progress from 2016 to 2020 will be measured in comparison to infection data from 2015.

Vibrio vulnificus bacteria

Credit: Paul A. Gulig

Researchers say they’ve determined why patients with hereditary hemochromatosis are so vulnerable to severe illness from Vibrio vulnificus infection.

Patients with hereditary hemochromatosis have a deficiency of the iron-regulating hormone hepcidin and therefore develop excess iron in their blood and tissue, providing prime growth conditions for Vibrio vulnificus.

The researchers also showed that minihepcidin, a medicinal form of the hormone hepcidin that lowers iron levels in blood, could cure the infection by restricting bacterial growth.

The findings appear in Cell Host and Microbe.

“This is the first time that the association of hepcidin deficiency and susceptibility to Vibrio vulnificus infection was tested,” said study author Yonca Bulut, MD, of the David Geffen School of Medicine at UCLA in Los Angeles.

“The dramatic effectiveness of the new treatment, even after the infection was established, was impressive.”

To conduct this study, Dr Bulut and her colleagues compared the fatality of Vibrio vulnificus infection in healthy mice with mice that lacked hepcidin, modeling human hereditary hemochromatosis.

The infection was much more lethal in hepcidin-deficient mice because their bodies could not decrease iron levels in the blood in response to infection, a process mediated by hepcidin in healthy mice.

Giving minihepcidin to hepcidin-deficient mice prevented infection if the hormone was given before Vibrio vulnificus was introduced. And mice given minihepcidin 3 hours after the bacterium was introduced were cured of any infection.

“We found that hepcidin is required for resistance to a Vibrio vulnificus infection,” said study author Joao Arezes, a graduate student from the University of Porto in Portugal.

“The development of the treatment tested in mouse models could reduce the high mortality rate of this disease.”

The next stage of this research is to investigate why Vibrio vulnificus bacteria become so lethal when iron levels are high, and to learn which other microbes respond similarly to excess iron.

Vibrio vulnificus bacteria

Credit: Paul A. Gulig

Researchers say they’ve determined why patients with hereditary hemochromatosis are so vulnerable to severe illness from Vibrio vulnificus infection.

Patients with hereditary hemochromatosis have a deficiency of the iron-regulating hormone hepcidin and therefore develop excess iron in their blood and tissue, providing prime growth conditions for Vibrio vulnificus.

The researchers also showed that minihepcidin, a medicinal form of the hormone hepcidin that lowers iron levels in blood, could cure the infection by restricting bacterial growth.

The findings appear in Cell Host and Microbe.

“This is the first time that the association of hepcidin deficiency and susceptibility to Vibrio vulnificus infection was tested,” said study author Yonca Bulut, MD, of the David Geffen School of Medicine at UCLA in Los Angeles.

“The dramatic effectiveness of the new treatment, even after the infection was established, was impressive.”

To conduct this study, Dr Bulut and her colleagues compared the fatality of Vibrio vulnificus infection in healthy mice with mice that lacked hepcidin, modeling human hereditary hemochromatosis.

The infection was much more lethal in hepcidin-deficient mice because their bodies could not decrease iron levels in the blood in response to infection, a process mediated by hepcidin in healthy mice.

Giving minihepcidin to hepcidin-deficient mice prevented infection if the hormone was given before Vibrio vulnificus was introduced. And mice given minihepcidin 3 hours after the bacterium was introduced were cured of any infection.

“We found that hepcidin is required for resistance to a Vibrio vulnificus infection,” said study author Joao Arezes, a graduate student from the University of Porto in Portugal.

“The development of the treatment tested in mouse models could reduce the high mortality rate of this disease.”

The next stage of this research is to investigate why Vibrio vulnificus bacteria become so lethal when iron levels are high, and to learn which other microbes respond similarly to excess iron.

Vibrio vulnificus bacteria

Credit: Paul A. Gulig

Researchers say they’ve determined why patients with hereditary hemochromatosis are so vulnerable to severe illness from Vibrio vulnificus infection.

Patients with hereditary hemochromatosis have a deficiency of the iron-regulating hormone hepcidin and therefore develop excess iron in their blood and tissue, providing prime growth conditions for Vibrio vulnificus.

The researchers also showed that minihepcidin, a medicinal form of the hormone hepcidin that lowers iron levels in blood, could cure the infection by restricting bacterial growth.

The findings appear in Cell Host and Microbe.

“This is the first time that the association of hepcidin deficiency and susceptibility to Vibrio vulnificus infection was tested,” said study author Yonca Bulut, MD, of the David Geffen School of Medicine at UCLA in Los Angeles.

“The dramatic effectiveness of the new treatment, even after the infection was established, was impressive.”

To conduct this study, Dr Bulut and her colleagues compared the fatality of Vibrio vulnificus infection in healthy mice with mice that lacked hepcidin, modeling human hereditary hemochromatosis.

The infection was much more lethal in hepcidin-deficient mice because their bodies could not decrease iron levels in the blood in response to infection, a process mediated by hepcidin in healthy mice.

Giving minihepcidin to hepcidin-deficient mice prevented infection if the hormone was given before Vibrio vulnificus was introduced. And mice given minihepcidin 3 hours after the bacterium was introduced were cured of any infection.

“We found that hepcidin is required for resistance to a Vibrio vulnificus infection,” said study author Joao Arezes, a graduate student from the University of Porto in Portugal.

“The development of the treatment tested in mouse models could reduce the high mortality rate of this disease.”

The next stage of this research is to investigate why Vibrio vulnificus bacteria become so lethal when iron levels are high, and to learn which other microbes respond similarly to excess iron.

Researchers in the lab

Credit: Rhoda Baer

An analysis of countries in North America, Europe, and Asia-Oceania showed that the US had the slowest annual growth in medical research funding from 2004 to 2011.

Nevertheless, the US was the leading sponsor of global medical research in 2011, accounting for 44% of the $265 billion spent in all the regions studied.

Hamilton Moses III, MD, of the Alerion Institute and Alerion Advisors LLC, in North Garden, Virginia, and his colleagues reported these discoveries in JAMA.

The researchers examined developments over the past 2 decades in the pattern of who conducts and who supports medical research, as well as resulting patents, publications, and new drug and device approvals.

The group compiled publicly available data from 1994 to 2012, showing trends in US and international research funding, productivity, and disease burden by source and industry type. Patents and publications (1981-2011) were evaluated using citation rates and impact factors.

International research funding

The researchers included data from the major countries of North America (US and Canada), Europe (including the 10 largest European countries in the

Organisation for Economic Co-operation and Development), and Asia-Oceania (Australia, China, India, Japan, Singapore, and South Korea).

Of these regions, the US had the lowest rate of annual growth in funding from 2004 to 2011 (1%). The rate was 4.1% in Europe, 4.5% in Canada, 6.8% in Japan, 9.3% in Australia, 16.9% in China, and 20.8% in the other Asian countries.

Still, in 2011, the US invested $117.2 billion (44%) of the $265 billion spent in all the regions studied. Europe spent $88.6 billion (33%), Japan spent $37.8 billion (14%), China spent $4.9 billion (1.2%), other Asian countries spent $9.7 billion (4%), Australia spent $3.8 billion (1.4%), and Canada spent $3.1 billion (1.2%).

Research outcomes

Dr Moses and his colleagues also compared other aspects of medical research among the regions, such as patent applications, research articles, and drug approvals.

They found that China filed 30% of global life science patent applications in 2011, while the US filed 24%. Japan filed the fewest applications of all the

regions analyzed.

The US and the European Union were neck-and-neck with regard to the share of biomedical research articles published in all regions in 2009—33.4% and 32.8%, respectively. China’s share was only 5%, but the country had the greatestgrowth in contribution from 2000 through 2009, at 18.7%.

And the European Medicines Agency (EMA) outstripped the US Food and Drug Administration (FDA) when it came to drug approvals. In 2013, the EMA approved 57 new molecular entities and biologics, compared to the FDA’s 27. From 2003 to2013, the FDA averaged 26 approvals per year, and the EMA averaged 42.

Researchers in the lab

Credit: Rhoda Baer

An analysis of countries in North America, Europe, and Asia-Oceania showed that the US had the slowest annual growth in medical research funding from 2004 to 2011.

Nevertheless, the US was the leading sponsor of global medical research in 2011, accounting for 44% of the $265 billion spent in all the regions studied.

Hamilton Moses III, MD, of the Alerion Institute and Alerion Advisors LLC, in North Garden, Virginia, and his colleagues reported these discoveries in JAMA.

The researchers examined developments over the past 2 decades in the pattern of who conducts and who supports medical research, as well as resulting patents, publications, and new drug and device approvals.

The group compiled publicly available data from 1994 to 2012, showing trends in US and international research funding, productivity, and disease burden by source and industry type. Patents and publications (1981-2011) were evaluated using citation rates and impact factors.

International research funding

The researchers included data from the major countries of North America (US and Canada), Europe (including the 10 largest European countries in the

Organisation for Economic Co-operation and Development), and Asia-Oceania (Australia, China, India, Japan, Singapore, and South Korea).

Of these regions, the US had the lowest rate of annual growth in funding from 2004 to 2011 (1%). The rate was 4.1% in Europe, 4.5% in Canada, 6.8% in Japan, 9.3% in Australia, 16.9% in China, and 20.8% in the other Asian countries.

Still, in 2011, the US invested $117.2 billion (44%) of the $265 billion spent in all the regions studied. Europe spent $88.6 billion (33%), Japan spent $37.8 billion (14%), China spent $4.9 billion (1.2%), other Asian countries spent $9.7 billion (4%), Australia spent $3.8 billion (1.4%), and Canada spent $3.1 billion (1.2%).

Research outcomes

Dr Moses and his colleagues also compared other aspects of medical research among the regions, such as patent applications, research articles, and drug approvals.

They found that China filed 30% of global life science patent applications in 2011, while the US filed 24%. Japan filed the fewest applications of all the

regions analyzed.

The US and the European Union were neck-and-neck with regard to the share of biomedical research articles published in all regions in 2009—33.4% and 32.8%, respectively. China’s share was only 5%, but the country had the greatestgrowth in contribution from 2000 through 2009, at 18.7%.

And the European Medicines Agency (EMA) outstripped the US Food and Drug Administration (FDA) when it came to drug approvals. In 2013, the EMA approved 57 new molecular entities and biologics, compared to the FDA’s 27. From 2003 to2013, the FDA averaged 26 approvals per year, and the EMA averaged 42.

Researchers in the lab

Credit: Rhoda Baer

An analysis of countries in North America, Europe, and Asia-Oceania showed that the US had the slowest annual growth in medical research funding from 2004 to 2011.

Nevertheless, the US was the leading sponsor of global medical research in 2011, accounting for 44% of the $265 billion spent in all the regions studied.

Hamilton Moses III, MD, of the Alerion Institute and Alerion Advisors LLC, in North Garden, Virginia, and his colleagues reported these discoveries in JAMA.

The researchers examined developments over the past 2 decades in the pattern of who conducts and who supports medical research, as well as resulting patents, publications, and new drug and device approvals.

The group compiled publicly available data from 1994 to 2012, showing trends in US and international research funding, productivity, and disease burden by source and industry type. Patents and publications (1981-2011) were evaluated using citation rates and impact factors.

International research funding

The researchers included data from the major countries of North America (US and Canada), Europe (including the 10 largest European countries in the

Organisation for Economic Co-operation and Development), and Asia-Oceania (Australia, China, India, Japan, Singapore, and South Korea).

Of these regions, the US had the lowest rate of annual growth in funding from 2004 to 2011 (1%). The rate was 4.1% in Europe, 4.5% in Canada, 6.8% in Japan, 9.3% in Australia, 16.9% in China, and 20.8% in the other Asian countries.

Still, in 2011, the US invested $117.2 billion (44%) of the $265 billion spent in all the regions studied. Europe spent $88.6 billion (33%), Japan spent $37.8 billion (14%), China spent $4.9 billion (1.2%), other Asian countries spent $9.7 billion (4%), Australia spent $3.8 billion (1.4%), and Canada spent $3.1 billion (1.2%).

Research outcomes

Dr Moses and his colleagues also compared other aspects of medical research among the regions, such as patent applications, research articles, and drug approvals.

They found that China filed 30% of global life science patent applications in 2011, while the US filed 24%. Japan filed the fewest applications of all the

regions analyzed.

The US and the European Union were neck-and-neck with regard to the share of biomedical research articles published in all regions in 2009—33.4% and 32.8%, respectively. China’s share was only 5%, but the country had the greatestgrowth in contribution from 2000 through 2009, at 18.7%.

And the European Medicines Agency (EMA) outstripped the US Food and Drug Administration (FDA) when it came to drug approvals. In 2013, the EMA approved 57 new molecular entities and biologics, compared to the FDA’s 27. From 2003 to2013, the FDA averaged 26 approvals per year, and the EMA averaged 42.

Warfarin tablets

SAN FRANCISCO—Monitoring warfarin using a modified prothrombin time (PT) test can improve anticoagulation stability and long-term clinical outcomes, result of the Fiix trial suggest.

With Fiix-PT—a test that only measures the activity of coagulation factors II and X—the effect of warfarin fluctuated less than with standard PT.

Fiix-PT also proved superior in reducing long-term, recurrent thromboembolism (TE), and it did not increase bleeding, despite the fact that the test omits factor VII activity.

However, most of these effects occurred only after the 6-month mark.

Pall T. Onundarson, MD, of Landspitali University Hospital and University of Iceland School of Medicine in Reykjavik, presented these results at the 2014 ASH Annual Meeting (abstract 347).

Dr Onundarson is a co-inventor of the Fiix-PT test and has stock in Fiix Diagnostics, a startup company with the two inventors of the test as majority shareholders.

“Why would anyone still be interested in warfarin in 2014,” Dr Onundarson asked the audience at ASH. “Even if warfarin is very efficacious . . ., managing warfarin is trouble. The dose varies between patients, and the main problem is that we have a fluctuating or unstable effect—namely, a fluctuating INR—and this leads to frequent dose adjustments and frequent testing.”

Dr Onundarson said the common wisdom is that the effect of warfarin fluctuates due to food interactions and drug interactions. But he and his colleagues speculated that the fluctuation is partly caused by conventional PT.

They noted that experiments have suggested the anticoagulation effect of warfarin is mainly influenced by a reduction in FII and FX activity, and FVII may have relatively little effect. Due to the short half-life of FVII and its strong influence on the PT, monitoring warfarin using PT may confound dosing.

With that in mind, the Fiix-PT test was designed to measure only the activity of FII and FX. And the researchers conducted the Fiix trial to compare Fiix-PT and conventional PT.

Their study included 1148 patients—573 in the Fiix-PT arm and 575 in the PT arm. Overall, about 69% of patients had atrial fibrillation, and 21% had venous thromboembolism. The median monitoring time was 1.7 years per patient in both arms.

The researchers noted that most outcomes were not significantly different between the 2 arms in the first 6 months, but, after that point, things changed. So they examined outcomes after 6 months in a secondary analysis.

From day 1 to 180, there was no significant difference between the Fiix-PT arm and the PT arm in the number of dose changes per patient year (5.3 and 6.5, respectively, P=0.08). However, from day 181 to 720, there were significantly fewer dose changes in the Fiix-PT arm (4.1 and 5.0, respectively, P=0.01).

The researchers assessed the median time within target range (TTR) during 4 periods of the study, and, although results fluctuated, there were significant differences in favor of Fiix-PT.

The median TTR was 85% in the Fiix-PT arm and 81% in the PT arm for days 1 to 180 (P=0.013); 85% and 90%, respectively, for days 181 to 360 (P<0.0003); 80% and 81%, respectively, for days 361 to 540 (P=0.34); and 87% and 79%, respectively, for days 541 to 720 (P<0.005).

Overall, there was no significant difference in the rate of TE or major bleeding between the Fiix-PT and PT arms. However, when the researchers excluded the first 6 months of observation, they noted a significant difference in the rate of TE in favor of Fiix-PT.

In the primary analysis (encompassing days 1 to 720), the TE rate per patient year was 1.2% in the Fiix-PT arm and 2.3% in the PT arm (P=0.09 for superiority, P<0.001 for non-inferiority). The major bleeding rates were 2.2% and 2.5%, respectively (P=0.8 for superiority, P<0.001 for non-inferiority).

In the secondary analysis (from day 181 to 720), Fiix-PT led to a superior long-term reduction in TE compared to conventional PT—1.1% and 2.2%, respectively (P=0.03 for superiority). But there was no significant difference in the rates of major bleeding—1.5% and 2.3%, respectively (P=0.5 for superiority).

“Compared to high-quality PT-INR monitoring, Fiix-PT increased the stability of warfarin anticoagulation,” Dr Onundarson said in closing. “Fiix-PT was clinically at least non-inferior to the INR in the primary analysis, and Fiix-PT led to superior long-term reduction in TE in the secondary analysis. Fiix-PT did not increase major bleeding, despite lowering the long-term thromboembolic rate and despite not being affected by FVII in the test sample.”

“So my overall conclusion is that a fluctuating INR during warfarin treatment is partly a confounding side effect of the PT itself. The data suggests that, if the PT is replaced with a monitoring test that is not affected by FVII, warfarin may become more stable than was previously assumed.”

Warfarin tablets

SAN FRANCISCO—Monitoring warfarin using a modified prothrombin time (PT) test can improve anticoagulation stability and long-term clinical outcomes, result of the Fiix trial suggest.

With Fiix-PT—a test that only measures the activity of coagulation factors II and X—the effect of warfarin fluctuated less than with standard PT.

Fiix-PT also proved superior in reducing long-term, recurrent thromboembolism (TE), and it did not increase bleeding, despite the fact that the test omits factor VII activity.

However, most of these effects occurred only after the 6-month mark.

Pall T. Onundarson, MD, of Landspitali University Hospital and University of Iceland School of Medicine in Reykjavik, presented these results at the 2014 ASH Annual Meeting (abstract 347).

Dr Onundarson is a co-inventor of the Fiix-PT test and has stock in Fiix Diagnostics, a startup company with the two inventors of the test as majority shareholders.

“Why would anyone still be interested in warfarin in 2014,” Dr Onundarson asked the audience at ASH. “Even if warfarin is very efficacious . . ., managing warfarin is trouble. The dose varies between patients, and the main problem is that we have a fluctuating or unstable effect—namely, a fluctuating INR—and this leads to frequent dose adjustments and frequent testing.”

Dr Onundarson said the common wisdom is that the effect of warfarin fluctuates due to food interactions and drug interactions. But he and his colleagues speculated that the fluctuation is partly caused by conventional PT.

They noted that experiments have suggested the anticoagulation effect of warfarin is mainly influenced by a reduction in FII and FX activity, and FVII may have relatively little effect. Due to the short half-life of FVII and its strong influence on the PT, monitoring warfarin using PT may confound dosing.

With that in mind, the Fiix-PT test was designed to measure only the activity of FII and FX. And the researchers conducted the Fiix trial to compare Fiix-PT and conventional PT.

Their study included 1148 patients—573 in the Fiix-PT arm and 575 in the PT arm. Overall, about 69% of patients had atrial fibrillation, and 21% had venous thromboembolism. The median monitoring time was 1.7 years per patient in both arms.

The researchers noted that most outcomes were not significantly different between the 2 arms in the first 6 months, but, after that point, things changed. So they examined outcomes after 6 months in a secondary analysis.

From day 1 to 180, there was no significant difference between the Fiix-PT arm and the PT arm in the number of dose changes per patient year (5.3 and 6.5, respectively, P=0.08). However, from day 181 to 720, there were significantly fewer dose changes in the Fiix-PT arm (4.1 and 5.0, respectively, P=0.01).

The researchers assessed the median time within target range (TTR) during 4 periods of the study, and, although results fluctuated, there were significant differences in favor of Fiix-PT.

The median TTR was 85% in the Fiix-PT arm and 81% in the PT arm for days 1 to 180 (P=0.013); 85% and 90%, respectively, for days 181 to 360 (P<0.0003); 80% and 81%, respectively, for days 361 to 540 (P=0.34); and 87% and 79%, respectively, for days 541 to 720 (P<0.005).

Overall, there was no significant difference in the rate of TE or major bleeding between the Fiix-PT and PT arms. However, when the researchers excluded the first 6 months of observation, they noted a significant difference in the rate of TE in favor of Fiix-PT.

In the primary analysis (encompassing days 1 to 720), the TE rate per patient year was 1.2% in the Fiix-PT arm and 2.3% in the PT arm (P=0.09 for superiority, P<0.001 for non-inferiority). The major bleeding rates were 2.2% and 2.5%, respectively (P=0.8 for superiority, P<0.001 for non-inferiority).

In the secondary analysis (from day 181 to 720), Fiix-PT led to a superior long-term reduction in TE compared to conventional PT—1.1% and 2.2%, respectively (P=0.03 for superiority). But there was no significant difference in the rates of major bleeding—1.5% and 2.3%, respectively (P=0.5 for superiority).

“Compared to high-quality PT-INR monitoring, Fiix-PT increased the stability of warfarin anticoagulation,” Dr Onundarson said in closing. “Fiix-PT was clinically at least non-inferior to the INR in the primary analysis, and Fiix-PT led to superior long-term reduction in TE in the secondary analysis. Fiix-PT did not increase major bleeding, despite lowering the long-term thromboembolic rate and despite not being affected by FVII in the test sample.”

“So my overall conclusion is that a fluctuating INR during warfarin treatment is partly a confounding side effect of the PT itself. The data suggests that, if the PT is replaced with a monitoring test that is not affected by FVII, warfarin may become more stable than was previously assumed.”

Warfarin tablets

SAN FRANCISCO—Monitoring warfarin using a modified prothrombin time (PT) test can improve anticoagulation stability and long-term clinical outcomes, result of the Fiix trial suggest.

With Fiix-PT—a test that only measures the activity of coagulation factors II and X—the effect of warfarin fluctuated less than with standard PT.

Fiix-PT also proved superior in reducing long-term, recurrent thromboembolism (TE), and it did not increase bleeding, despite the fact that the test omits factor VII activity.

However, most of these effects occurred only after the 6-month mark.

Pall T. Onundarson, MD, of Landspitali University Hospital and University of Iceland School of Medicine in Reykjavik, presented these results at the 2014 ASH Annual Meeting (abstract 347).

Dr Onundarson is a co-inventor of the Fiix-PT test and has stock in Fiix Diagnostics, a startup company with the two inventors of the test as majority shareholders.

“Why would anyone still be interested in warfarin in 2014,” Dr Onundarson asked the audience at ASH. “Even if warfarin is very efficacious . . ., managing warfarin is trouble. The dose varies between patients, and the main problem is that we have a fluctuating or unstable effect—namely, a fluctuating INR—and this leads to frequent dose adjustments and frequent testing.”

Dr Onundarson said the common wisdom is that the effect of warfarin fluctuates due to food interactions and drug interactions. But he and his colleagues speculated that the fluctuation is partly caused by conventional PT.

They noted that experiments have suggested the anticoagulation effect of warfarin is mainly influenced by a reduction in FII and FX activity, and FVII may have relatively little effect. Due to the short half-life of FVII and its strong influence on the PT, monitoring warfarin using PT may confound dosing.

With that in mind, the Fiix-PT test was designed to measure only the activity of FII and FX. And the researchers conducted the Fiix trial to compare Fiix-PT and conventional PT.

Their study included 1148 patients—573 in the Fiix-PT arm and 575 in the PT arm. Overall, about 69% of patients had atrial fibrillation, and 21% had venous thromboembolism. The median monitoring time was 1.7 years per patient in both arms.

The researchers noted that most outcomes were not significantly different between the 2 arms in the first 6 months, but, after that point, things changed. So they examined outcomes after 6 months in a secondary analysis.

From day 1 to 180, there was no significant difference between the Fiix-PT arm and the PT arm in the number of dose changes per patient year (5.3 and 6.5, respectively, P=0.08). However, from day 181 to 720, there were significantly fewer dose changes in the Fiix-PT arm (4.1 and 5.0, respectively, P=0.01).

The researchers assessed the median time within target range (TTR) during 4 periods of the study, and, although results fluctuated, there were significant differences in favor of Fiix-PT.

The median TTR was 85% in the Fiix-PT arm and 81% in the PT arm for days 1 to 180 (P=0.013); 85% and 90%, respectively, for days 181 to 360 (P<0.0003); 80% and 81%, respectively, for days 361 to 540 (P=0.34); and 87% and 79%, respectively, for days 541 to 720 (P<0.005).

Overall, there was no significant difference in the rate of TE or major bleeding between the Fiix-PT and PT arms. However, when the researchers excluded the first 6 months of observation, they noted a significant difference in the rate of TE in favor of Fiix-PT.

In the primary analysis (encompassing days 1 to 720), the TE rate per patient year was 1.2% in the Fiix-PT arm and 2.3% in the PT arm (P=0.09 for superiority, P<0.001 for non-inferiority). The major bleeding rates were 2.2% and 2.5%, respectively (P=0.8 for superiority, P<0.001 for non-inferiority).

In the secondary analysis (from day 181 to 720), Fiix-PT led to a superior long-term reduction in TE compared to conventional PT—1.1% and 2.2%, respectively (P=0.03 for superiority). But there was no significant difference in the rates of major bleeding—1.5% and 2.3%, respectively (P=0.5 for superiority).

“Compared to high-quality PT-INR monitoring, Fiix-PT increased the stability of warfarin anticoagulation,” Dr Onundarson said in closing. “Fiix-PT was clinically at least non-inferior to the INR in the primary analysis, and Fiix-PT led to superior long-term reduction in TE in the secondary analysis. Fiix-PT did not increase major bleeding, despite lowering the long-term thromboembolic rate and despite not being affected by FVII in the test sample.”

“So my overall conclusion is that a fluctuating INR during warfarin treatment is partly a confounding side effect of the PT itself. The data suggests that, if the PT is replaced with a monitoring test that is not affected by FVII, warfarin may become more stable than was previously assumed.”

Prescription medications

Credit: Steven Harbour

The National Health Service (NHS) has increased the budget for England’s Cancer Drugs Fund (CDF) and  added a new drug to treat 2 hematologic malignancies, but 5 other blood cancer drugs will be removed from the fund in March.

The budget for the CDF will grow from £200 million in 2013/14 to £280 million in 2014/15.

However, 16 drugs (for 25 different indications) will no longer be offered through the fund as of March 12, 2015.

Still, the NHS said it has taken steps to ensure patients can receive appropriate treatment.

Review leads to cuts

A national panel of oncologists, pharmacists, and patient representatives independently reviewed the drug indications currently available through the CDF, plus new applications.

They evaluated the clinical benefit, survival, quality of life, toxicity, and safety associated with each treatment, as well as the level of unmet need and the median cost per patient. In cases where the high cost of a drug would lead to its exclusion from CDF, manufacturers were given an opportunity to reduce prices.

The result of the review is that 59 of the 84 most effective currently approved indications of drugs will rollover into the CDF next year, creating room for new drug indications that will be funded for the first time.

These are panitumumab for bowel cancer, ibrutinib for mantle cell lymphoma, and ibrutinib for chronic lymphocytic leukemia.

However, 16 drugs, including 5 blood cancer drugs—bendamustine, bortezomib, bosutinib, dasatinib, and ofatumumab—will no longer be offered through the CDF.

Following these changes, the NHS will put 4 measures in place to ensure patients can receive appropriate treatment. First, any patient currently receiving a drug through the CDF will continue to receive it, regardless of whether it remains in the CDF.

Second, drugs that are the only therapy for the cancer in question will remain available through the CDF. Third, if the CDF panel removes a drug for a particular indication, some patients may instead be able to receive it in another line of therapy or receive an alternative CDF-approved drug.

And finally, clinicians can apply for their patient to receive a drug not available through the CDF on an exceptional basis.

Cuts to blood cancer drugs

The full list of cuts to the CDF is available on the NHS website, but the following list includes all drugs for hematologic malignancies that will no longer be available. These drugs will still be available for other indications, however.

1. Bendamustine for the treatment of low-grade lymphoma that is refractory to rituximab alone or in combination.
2. Bortezomib for the treatment of:

   • relapsed/refractory mantle cell lymphoma after 1 or more prior chemotherapies or stem cell transplant
   • relapsed multiple myeloma patients with a previous partial response or complete response of 6 months or more with bortezomib
   • relapsed Waldenstrom’s macroglobulinemia patients who received previous treatment with alkylating agents and purine analogues.

3. Bosutinib for the treatment of:

   • blast crisis chronic myeloid leukemia (CML) that is refractory to nilotinib or dasatinib if dasatinib was accessed via a clinical trial or via its current approved CDF indication
   • blast crisis CML where there is treatment intolerance, specifically, significant intolerance to dasatinib (grade 3 or 4 adverse events) if dasatinib was accessed via its current approved CDF indication.

4. Dasatinib for the treatment of lymphoid, blast crisis CML that is refractory to, significantly intolerant of, or resistant to prior therapy including imatinib (grade 3 or 4 adverse events); also when used as the 2nd- or 3rd-line treatment.
5. Ofatumumab for the treatment of CML as the 2nd- or 3rd-line indication and if the patient is refractory to treatment with fludarabine in combination and/or alemtuzumab or if treatment with fludarabine in combination and/or alemtuzumab is contraindicated.

More about the CDF and the NHS

The CDF—set up in 2010 and currently due to run until March 2016—is money the government has set aside to pay for cancer drugs that haven’t been approved by the National Institute for Health and Care Excellence (NICE) and aren’t available within the NHS in England. Most cancer drugs are routinely funded outside of the CDF.

NHS England said it is working with cancer charities, the pharmaceutical industry, and NICE to create a sustainable model for the commissioning of chemotherapy. The agency has also updated its procedures for evaluating drugs in the CDF, in an effort to ensure sustainability.

In addition, NHS England has set up an appeals process by which pharmaceutical companies can challenge the decision-making process.

And a newly assembled national taskforce, headed by Harpal Kumar, chief executive of Cancer Research UK, is set to produce a refreshed, 5-year cancer plan for the NHS.

Prescription medications

Credit: Steven Harbour

The National Health Service (NHS) has increased the budget for England’s Cancer Drugs Fund (CDF) and  added a new drug to treat 2 hematologic malignancies, but 5 other blood cancer drugs will be removed from the fund in March.

The budget for the CDF will grow from £200 million in 2013/14 to £280 million in 2014/15.

However, 16 drugs (for 25 different indications) will no longer be offered through the fund as of March 12, 2015.

Still, the NHS said it has taken steps to ensure patients can receive appropriate treatment.

Review leads to cuts

A national panel of oncologists, pharmacists, and patient representatives independently reviewed the drug indications currently available through the CDF, plus new applications.

They evaluated the clinical benefit, survival, quality of life, toxicity, and safety associated with each treatment, as well as the level of unmet need and the median cost per patient. In cases where the high cost of a drug would lead to its exclusion from CDF, manufacturers were given an opportunity to reduce prices.

The result of the review is that 59 of the 84 most effective currently approved indications of drugs will rollover into the CDF next year, creating room for new drug indications that will be funded for the first time.

These are panitumumab for bowel cancer, ibrutinib for mantle cell lymphoma, and ibrutinib for chronic lymphocytic leukemia.

However, 16 drugs, including 5 blood cancer drugs—bendamustine, bortezomib, bosutinib, dasatinib, and ofatumumab—will no longer be offered through the CDF.

Following these changes, the NHS will put 4 measures in place to ensure patients can receive appropriate treatment. First, any patient currently receiving a drug through the CDF will continue to receive it, regardless of whether it remains in the CDF.

Second, drugs that are the only therapy for the cancer in question will remain available through the CDF. Third, if the CDF panel removes a drug for a particular indication, some patients may instead be able to receive it in another line of therapy or receive an alternative CDF-approved drug.

And finally, clinicians can apply for their patient to receive a drug not available through the CDF on an exceptional basis.

Cuts to blood cancer drugs

The full list of cuts to the CDF is available on the NHS website, but the following list includes all drugs for hematologic malignancies that will no longer be available. These drugs will still be available for other indications, however.

1. Bendamustine for the treatment of low-grade lymphoma that is refractory to rituximab alone or in combination.
2. Bortezomib for the treatment of:

   • relapsed/refractory mantle cell lymphoma after 1 or more prior chemotherapies or stem cell transplant
   • relapsed multiple myeloma patients with a previous partial response or complete response of 6 months or more with bortezomib
   • relapsed Waldenstrom’s macroglobulinemia patients who received previous treatment with alkylating agents and purine analogues.

3. Bosutinib for the treatment of:

   • blast crisis chronic myeloid leukemia (CML) that is refractory to nilotinib or dasatinib if dasatinib was accessed via a clinical trial or via its current approved CDF indication
   • blast crisis CML where there is treatment intolerance, specifically, significant intolerance to dasatinib (grade 3 or 4 adverse events) if dasatinib was accessed via its current approved CDF indication.

4. Dasatinib for the treatment of lymphoid, blast crisis CML that is refractory to, significantly intolerant of, or resistant to prior therapy including imatinib (grade 3 or 4 adverse events); also when used as the 2nd- or 3rd-line treatment.
5. Ofatumumab for the treatment of CML as the 2nd- or 3rd-line indication and if the patient is refractory to treatment with fludarabine in combination and/or alemtuzumab or if treatment with fludarabine in combination and/or alemtuzumab is contraindicated.

More about the CDF and the NHS

The CDF—set up in 2010 and currently due to run until March 2016—is money the government has set aside to pay for cancer drugs that haven’t been approved by the National Institute for Health and Care Excellence (NICE) and aren’t available within the NHS in England. Most cancer drugs are routinely funded outside of the CDF.

NHS England said it is working with cancer charities, the pharmaceutical industry, and NICE to create a sustainable model for the commissioning of chemotherapy. The agency has also updated its procedures for evaluating drugs in the CDF, in an effort to ensure sustainability.

In addition, NHS England has set up an appeals process by which pharmaceutical companies can challenge the decision-making process.

And a newly assembled national taskforce, headed by Harpal Kumar, chief executive of Cancer Research UK, is set to produce a refreshed, 5-year cancer plan for the NHS.

Prescription medications

Credit: Steven Harbour

The National Health Service (NHS) has increased the budget for England’s Cancer Drugs Fund (CDF) and  added a new drug to treat 2 hematologic malignancies, but 5 other blood cancer drugs will be removed from the fund in March.

The budget for the CDF will grow from £200 million in 2013/14 to £280 million in 2014/15.

However, 16 drugs (for 25 different indications) will no longer be offered through the fund as of March 12, 2015.

Still, the NHS said it has taken steps to ensure patients can receive appropriate treatment.

Review leads to cuts

A national panel of oncologists, pharmacists, and patient representatives independently reviewed the drug indications currently available through the CDF, plus new applications.

They evaluated the clinical benefit, survival, quality of life, toxicity, and safety associated with each treatment, as well as the level of unmet need and the median cost per patient. In cases where the high cost of a drug would lead to its exclusion from CDF, manufacturers were given an opportunity to reduce prices.

The result of the review is that 59 of the 84 most effective currently approved indications of drugs will rollover into the CDF next year, creating room for new drug indications that will be funded for the first time.

These are panitumumab for bowel cancer, ibrutinib for mantle cell lymphoma, and ibrutinib for chronic lymphocytic leukemia.

However, 16 drugs, including 5 blood cancer drugs—bendamustine, bortezomib, bosutinib, dasatinib, and ofatumumab—will no longer be offered through the CDF.

Following these changes, the NHS will put 4 measures in place to ensure patients can receive appropriate treatment. First, any patient currently receiving a drug through the CDF will continue to receive it, regardless of whether it remains in the CDF.

Second, drugs that are the only therapy for the cancer in question will remain available through the CDF. Third, if the CDF panel removes a drug for a particular indication, some patients may instead be able to receive it in another line of therapy or receive an alternative CDF-approved drug.

And finally, clinicians can apply for their patient to receive a drug not available through the CDF on an exceptional basis.

Cuts to blood cancer drugs

The full list of cuts to the CDF is available on the NHS website, but the following list includes all drugs for hematologic malignancies that will no longer be available. These drugs will still be available for other indications, however.

1. Bendamustine for the treatment of low-grade lymphoma that is refractory to rituximab alone or in combination.
2. Bortezomib for the treatment of:

   • relapsed/refractory mantle cell lymphoma after 1 or more prior chemotherapies or stem cell transplant
   • relapsed multiple myeloma patients with a previous partial response or complete response of 6 months or more with bortezomib
   • relapsed Waldenstrom’s macroglobulinemia patients who received previous treatment with alkylating agents and purine analogues.

3. Bosutinib for the treatment of:

   • blast crisis chronic myeloid leukemia (CML) that is refractory to nilotinib or dasatinib if dasatinib was accessed via a clinical trial or via its current approved CDF indication
   • blast crisis CML where there is treatment intolerance, specifically, significant intolerance to dasatinib (grade 3 or 4 adverse events) if dasatinib was accessed via its current approved CDF indication.

4. Dasatinib for the treatment of lymphoid, blast crisis CML that is refractory to, significantly intolerant of, or resistant to prior therapy including imatinib (grade 3 or 4 adverse events); also when used as the 2nd- or 3rd-line treatment.
5. Ofatumumab for the treatment of CML as the 2nd- or 3rd-line indication and if the patient is refractory to treatment with fludarabine in combination and/or alemtuzumab or if treatment with fludarabine in combination and/or alemtuzumab is contraindicated.

More about the CDF and the NHS

The CDF—set up in 2010 and currently due to run until March 2016—is money the government has set aside to pay for cancer drugs that haven’t been approved by the National Institute for Health and Care Excellence (NICE) and aren’t available within the NHS in England. Most cancer drugs are routinely funded outside of the CDF.

NHS England said it is working with cancer charities, the pharmaceutical industry, and NICE to create a sustainable model for the commissioning of chemotherapy. The agency has also updated its procedures for evaluating drugs in the CDF, in an effort to ensure sustainability.

In addition, NHS England has set up an appeals process by which pharmaceutical companies can challenge the decision-making process.

And a newly assembled national taskforce, headed by Harpal Kumar, chief executive of Cancer Research UK, is set to produce a refreshed, 5-year cancer plan for the NHS.

Acute myeloid leukemia

In a phase 2 trial, an investigational drug conferred a significant improvement in survival when used as salvage therapy in poor-risk patients with acute myeloid leukemia (AML).

On the other hand, the drug did not provide any significant improvements over control treatment (investigator’s choice) in the entire population of AML patients in first relapse.

The drug, CPX-351, is a fixed-ratio combination of cytarabine and daunorubicin inside a lipid vesicle.

Researchers reported these results with CPX-351 in Cancer. The study was funded by Celator Pharmaceuticals, the company developing CPX-351, and the Leukemia and Lymphoma Society.

“Patients with first-relapse AML have generally a poor prognosis, with a limited likelihood of response following salvage treatment,” said study author Jorge Cortes, MD, of the MD Anderson Cancer Center in Houston, Texas.

“This is particularly true for patients classified by the EPI [European Prognostic Index] as poor-risk upon entering the trial.”

For this trial, Dr Cortes and his colleagues evaluated 125 patients, ages 18 to 65, from 35 centers in the US, Canada, and Europe. Patients had AML in first relapse after an initial complete remission lasting a month or longer.

They were stratified per the EPI into favorable-, intermediate-, and poor-risk groups based on the duration of their first complete remission, cytogenetics, age, and transplant history. Most patients (68%) were in the poor-risk group.

Patients were randomized 2:1 to receive CPX-351 (100 units/m² on days 1, 3, and 5 by 90-minute infusion) or the investigators’ choice of first salvage chemotherapy. The control treatment was usually based on cytarabine and an anthracycline, often with one or more additional agents.

Patient characteristics were largely well-balanced between the treatment arms. However, the CPX-351 group had a younger median age (52 years vs 56 years), more patients with secondary AML (12.3% vs 6.8%), and a higher rate of prior transplant (27.2% vs 15.9%).

Response and survival

Response rates were higher in the CPX-351 arm than in the control arm. The rates of complete response were 37% and 31.8%, respectively. And the rates of complete response with incomplete count recovery were 12.3% and 9.1%, respectively.

However, there was no significant difference in event-free survival (EFS) or overall survival (OS) between the treatment arms.

The median EFS was 4 months in the CPX-351 arm and 1.4 months in the control arm (hazard ratio[HR]=0.66, P=0.08). And the median OS was 8.5 months and 6.3 months, respectively (HR=0.75, P=0.19).

In the poor-risk population, there was no significant improvement in EFS with CPX-351, but there was a significant improvement in OS.

The median EFS was 1.8 months in the CPX-351 arm and 1.2 months in the control arm (HR=0.63, P=0.08). And the median OS was 6.5 months and 4.2 months, respectively (HR=0.55, P=0.02).

Safety and early mortality

The early mortality rate was similar between the treatment arms at 30 days—7.4% in the CPX-351 arm and 4.5% in the control arm—and at 60 days—14.8% and 15.9%, respectively. However, at 90 days, deaths were more frequent in the control arm—21.4% and 37.9%.

Patients in the CPX-351 arm had slower neutrophil recovery than those in the control arm—42 days and 34 days, respectively. The same was true for platelet recovery—45 days and 35 days, respectively.

Delayed hematologic recovery was associated with more infection-related events, such as febrile neutropenia, bacteremia, pneumonia, sepsis, urinary tract infection, pyrexia, and cellulitis.

The researchers believe these results, along with the previously published results from a phase 2 trial of CPX-351 in patients newly diagnosed with AML, support the phase 3 study of CPX-351 as a first-line therapy in older patients with high-risk (secondary) AML.

“We were very pleased to see promising response rates in this difficult-to-treat population,” Dr Cortes said. “And we eagerly await results from Celator’s pivotal phase 3 study of CPX-351, which could fulfill a considerable unmet need in AML.”

Acute myeloid leukemia

In a phase 2 trial, an investigational drug conferred a significant improvement in survival when used as salvage therapy in poor-risk patients with acute myeloid leukemia (AML).

On the other hand, the drug did not provide any significant improvements over control treatment (investigator’s choice) in the entire population of AML patients in first relapse.

The drug, CPX-351, is a fixed-ratio combination of cytarabine and daunorubicin inside a lipid vesicle.

Researchers reported these results with CPX-351 in Cancer. The study was funded by Celator Pharmaceuticals, the company developing CPX-351, and the Leukemia and Lymphoma Society.

“Patients with first-relapse AML have generally a poor prognosis, with a limited likelihood of response following salvage treatment,” said study author Jorge Cortes, MD, of the MD Anderson Cancer Center in Houston, Texas.

“This is particularly true for patients classified by the EPI [European Prognostic Index] as poor-risk upon entering the trial.”

For this trial, Dr Cortes and his colleagues evaluated 125 patients, ages 18 to 65, from 35 centers in the US, Canada, and Europe. Patients had AML in first relapse after an initial complete remission lasting a month or longer.

They were stratified per the EPI into favorable-, intermediate-, and poor-risk groups based on the duration of their first complete remission, cytogenetics, age, and transplant history. Most patients (68%) were in the poor-risk group.

Patients were randomized 2:1 to receive CPX-351 (100 units/m² on days 1, 3, and 5 by 90-minute infusion) or the investigators’ choice of first salvage chemotherapy. The control treatment was usually based on cytarabine and an anthracycline, often with one or more additional agents.

Patient characteristics were largely well-balanced between the treatment arms. However, the CPX-351 group had a younger median age (52 years vs 56 years), more patients with secondary AML (12.3% vs 6.8%), and a higher rate of prior transplant (27.2% vs 15.9%).

Response and survival

Response rates were higher in the CPX-351 arm than in the control arm. The rates of complete response were 37% and 31.8%, respectively. And the rates of complete response with incomplete count recovery were 12.3% and 9.1%, respectively.

However, there was no significant difference in event-free survival (EFS) or overall survival (OS) between the treatment arms.

The median EFS was 4 months in the CPX-351 arm and 1.4 months in the control arm (hazard ratio[HR]=0.66, P=0.08). And the median OS was 8.5 months and 6.3 months, respectively (HR=0.75, P=0.19).

In the poor-risk population, there was no significant improvement in EFS with CPX-351, but there was a significant improvement in OS.

The median EFS was 1.8 months in the CPX-351 arm and 1.2 months in the control arm (HR=0.63, P=0.08). And the median OS was 6.5 months and 4.2 months, respectively (HR=0.55, P=0.02).

Safety and early mortality

The early mortality rate was similar between the treatment arms at 30 days—7.4% in the CPX-351 arm and 4.5% in the control arm—and at 60 days—14.8% and 15.9%, respectively. However, at 90 days, deaths were more frequent in the control arm—21.4% and 37.9%.

Patients in the CPX-351 arm had slower neutrophil recovery than those in the control arm—42 days and 34 days, respectively. The same was true for platelet recovery—45 days and 35 days, respectively.

Delayed hematologic recovery was associated with more infection-related events, such as febrile neutropenia, bacteremia, pneumonia, sepsis, urinary tract infection, pyrexia, and cellulitis.

The researchers believe these results, along with the previously published results from a phase 2 trial of CPX-351 in patients newly diagnosed with AML, support the phase 3 study of CPX-351 as a first-line therapy in older patients with high-risk (secondary) AML.

“We were very pleased to see promising response rates in this difficult-to-treat population,” Dr Cortes said. “And we eagerly await results from Celator’s pivotal phase 3 study of CPX-351, which could fulfill a considerable unmet need in AML.”

Acute myeloid leukemia

In a phase 2 trial, an investigational drug conferred a significant improvement in survival when used as salvage therapy in poor-risk patients with acute myeloid leukemia (AML).

On the other hand, the drug did not provide any significant improvements over control treatment (investigator’s choice) in the entire population of AML patients in first relapse.

The drug, CPX-351, is a fixed-ratio combination of cytarabine and daunorubicin inside a lipid vesicle.

Researchers reported these results with CPX-351 in Cancer. The study was funded by Celator Pharmaceuticals, the company developing CPX-351, and the Leukemia and Lymphoma Society.

“Patients with first-relapse AML have generally a poor prognosis, with a limited likelihood of response following salvage treatment,” said study author Jorge Cortes, MD, of the MD Anderson Cancer Center in Houston, Texas.

“This is particularly true for patients classified by the EPI [European Prognostic Index] as poor-risk upon entering the trial.”

For this trial, Dr Cortes and his colleagues evaluated 125 patients, ages 18 to 65, from 35 centers in the US, Canada, and Europe. Patients had AML in first relapse after an initial complete remission lasting a month or longer.

They were stratified per the EPI into favorable-, intermediate-, and poor-risk groups based on the duration of their first complete remission, cytogenetics, age, and transplant history. Most patients (68%) were in the poor-risk group.

Patients were randomized 2:1 to receive CPX-351 (100 units/m² on days 1, 3, and 5 by 90-minute infusion) or the investigators’ choice of first salvage chemotherapy. The control treatment was usually based on cytarabine and an anthracycline, often with one or more additional agents.

Patient characteristics were largely well-balanced between the treatment arms. However, the CPX-351 group had a younger median age (52 years vs 56 years), more patients with secondary AML (12.3% vs 6.8%), and a higher rate of prior transplant (27.2% vs 15.9%).

Response and survival

Response rates were higher in the CPX-351 arm than in the control arm. The rates of complete response were 37% and 31.8%, respectively. And the rates of complete response with incomplete count recovery were 12.3% and 9.1%, respectively.

However, there was no significant difference in event-free survival (EFS) or overall survival (OS) between the treatment arms.

The median EFS was 4 months in the CPX-351 arm and 1.4 months in the control arm (hazard ratio[HR]=0.66, P=0.08). And the median OS was 8.5 months and 6.3 months, respectively (HR=0.75, P=0.19).

In the poor-risk population, there was no significant improvement in EFS with CPX-351, but there was a significant improvement in OS.

The median EFS was 1.8 months in the CPX-351 arm and 1.2 months in the control arm (HR=0.63, P=0.08). And the median OS was 6.5 months and 4.2 months, respectively (HR=0.55, P=0.02).

Safety and early mortality

The early mortality rate was similar between the treatment arms at 30 days—7.4% in the CPX-351 arm and 4.5% in the control arm—and at 60 days—14.8% and 15.9%, respectively. However, at 90 days, deaths were more frequent in the control arm—21.4% and 37.9%.

Patients in the CPX-351 arm had slower neutrophil recovery than those in the control arm—42 days and 34 days, respectively. The same was true for platelet recovery—45 days and 35 days, respectively.

Delayed hematologic recovery was associated with more infection-related events, such as febrile neutropenia, bacteremia, pneumonia, sepsis, urinary tract infection, pyrexia, and cellulitis.

The researchers believe these results, along with the previously published results from a phase 2 trial of CPX-351 in patients newly diagnosed with AML, support the phase 3 study of CPX-351 as a first-line therapy in older patients with high-risk (secondary) AML.

“We were very pleased to see promising response rates in this difficult-to-treat population,” Dr Cortes said. “And we eagerly await results from Celator’s pivotal phase 3 study of CPX-351, which could fulfill a considerable unmet need in AML.”

From left: Drs Alex Delbridge,

Stephanie Grabow, Liz Valente,

and Andreas Strasser

Photo courtesy of the

Walter and Eliza Hall Insitute

Targeting a cell survival protein could overcome treatment resistance in T-cell lymphomas, according to preclinical research published in Blood.

Investigators found that removing the pro-survival protein MCL-1 prompted the death of lymphoma cells that had become resistant to conventional treatments.

The team noted that half of all cancers become resistant to chemotherapy and radiotherapy by acquiring mutations in the tumor-suppressing p53 protein.

And their research showed that MCL-1 helps these cancer cells survive by subverting the normal process of apoptosis.

“There are several pro-survival proteins that promote the sustained survival of cancer cells; the challenge is to identify which one is the most important in keeping each type of cancer cell alive,” said Stephanie Grabow, PhD, of the Walter and Eliza Hall Institute of Medical Research in Victoria, Australia.

“When we removed MCL-1 in models of T-cell lymphoma that had ‘lost’ the tumor-suppressing protein p53, cancers could not develop, demonstrating that MCL-1 is critical for the development of T-cell lymphomas.”

“Previous work from our colleagues at the institute has shown that MCL-1 is also critical for the survival and therapy-resistance of other blood cancers, including B-cell lymphoma and acute myeloid leukemia, indicating that is a very important target for potential new anticancer treatments.”

So the new finding reinforces the need to develop compounds that specifically target MCL-1, said Andreas Strasser, PhD, also of the Walter and Eliza Hall Institute.

“Investigating the role of MCL-1 and other proteins involved in controlling apoptosis has shown that MCL-1 is a critical protein in the survival of many types of cancer cells,” he said. “Targeting MCL-1 could therefore allow us to develop new, urgently needed therapies to treat cancers that have stopped responding to other anticancer drugs.”

Dr Grabow said the researchers will continue to investigate the role of MCL-1 in the development and progression of other cancers.

“Finding new treatment targets is crucial if we are to reduce the impact of these diseases,” she concluded.

From left: Drs Alex Delbridge,

Stephanie Grabow, Liz Valente,

and Andreas Strasser

Photo courtesy of the

Walter and Eliza Hall Insitute

Targeting a cell survival protein could overcome treatment resistance in T-cell lymphomas, according to preclinical research published in Blood.

Investigators found that removing the pro-survival protein MCL-1 prompted the death of lymphoma cells that had become resistant to conventional treatments.

The team noted that half of all cancers become resistant to chemotherapy and radiotherapy by acquiring mutations in the tumor-suppressing p53 protein.

And their research showed that MCL-1 helps these cancer cells survive by subverting the normal process of apoptosis.

“There are several pro-survival proteins that promote the sustained survival of cancer cells; the challenge is to identify which one is the most important in keeping each type of cancer cell alive,” said Stephanie Grabow, PhD, of the Walter and Eliza Hall Institute of Medical Research in Victoria, Australia.

“When we removed MCL-1 in models of T-cell lymphoma that had ‘lost’ the tumor-suppressing protein p53, cancers could not develop, demonstrating that MCL-1 is critical for the development of T-cell lymphomas.”

“Previous work from our colleagues at the institute has shown that MCL-1 is also critical for the survival and therapy-resistance of other blood cancers, including B-cell lymphoma and acute myeloid leukemia, indicating that is a very important target for potential new anticancer treatments.”

So the new finding reinforces the need to develop compounds that specifically target MCL-1, said Andreas Strasser, PhD, also of the Walter and Eliza Hall Institute.

“Investigating the role of MCL-1 and other proteins involved in controlling apoptosis has shown that MCL-1 is a critical protein in the survival of many types of cancer cells,” he said. “Targeting MCL-1 could therefore allow us to develop new, urgently needed therapies to treat cancers that have stopped responding to other anticancer drugs.”

Dr Grabow said the researchers will continue to investigate the role of MCL-1 in the development and progression of other cancers.

“Finding new treatment targets is crucial if we are to reduce the impact of these diseases,” she concluded.

From left: Drs Alex Delbridge,

Stephanie Grabow, Liz Valente,

and Andreas Strasser

Photo courtesy of the

Walter and Eliza Hall Insitute

Targeting a cell survival protein could overcome treatment resistance in T-cell lymphomas, according to preclinical research published in Blood.

Investigators found that removing the pro-survival protein MCL-1 prompted the death of lymphoma cells that had become resistant to conventional treatments.

The team noted that half of all cancers become resistant to chemotherapy and radiotherapy by acquiring mutations in the tumor-suppressing p53 protein.

And their research showed that MCL-1 helps these cancer cells survive by subverting the normal process of apoptosis.

“There are several pro-survival proteins that promote the sustained survival of cancer cells; the challenge is to identify which one is the most important in keeping each type of cancer cell alive,” said Stephanie Grabow, PhD, of the Walter and Eliza Hall Institute of Medical Research in Victoria, Australia.

“When we removed MCL-1 in models of T-cell lymphoma that had ‘lost’ the tumor-suppressing protein p53, cancers could not develop, demonstrating that MCL-1 is critical for the development of T-cell lymphomas.”

“Previous work from our colleagues at the institute has shown that MCL-1 is also critical for the survival and therapy-resistance of other blood cancers, including B-cell lymphoma and acute myeloid leukemia, indicating that is a very important target for potential new anticancer treatments.”

So the new finding reinforces the need to develop compounds that specifically target MCL-1, said Andreas Strasser, PhD, also of the Walter and Eliza Hall Institute.

“Investigating the role of MCL-1 and other proteins involved in controlling apoptosis has shown that MCL-1 is a critical protein in the survival of many types of cancer cells,” he said. “Targeting MCL-1 could therefore allow us to develop new, urgently needed therapies to treat cancers that have stopped responding to other anticancer drugs.”

Dr Grabow said the researchers will continue to investigate the role of MCL-1 in the development and progression of other cancers.

“Finding new treatment targets is crucial if we are to reduce the impact of these diseases,” she concluded.

Lab mouse

Regulatory T cells (Tregs) need T-cell receptors to fulfill their protective functions, according to research published in Immunity.

The researchers knew that Tregs need T-cell receptors to develop properly, but they were unsure of the receptors’ role after that.

To find out, the team deactivated T-cell receptors on mature Tregs in genetically modified mice.

They found these defective Tregs were not able to carry out their protective function without the T-cell receptors.

Furthermore, the Treg pool fell significantly, as these cells were no longer multiplying.

However, the researchers also discovered that two of Tregs’ most well-known central molecular properties—the production of Foxp3 protein and specific chemical changes to DNA—were still present in the defective T cells.

“Without their receptor, the Tregs are still clearly identifiable as Tregs,” said study author Christoph Vahl, PhD, of the Max Planck Institute of Biochemistry in Martinsried, Germany.

“However, they lose a large part of their cellular identity. They also lose their special ability to suppress excessive immune reactions.”

“The Tregs obviously need continuous contact with their environment to function correctly. This is presumably the reason why they need a receptor that recognizes endogenous substances and continuously sends signals.”

“During the course of our research, we uncovered a very important mechanism for suppressing excessive responses and responses targeted against the human body,” added Marc Schmidt-Supprian, PhD, also of the Max Planck Institute.

“These findings could be relevant for situations where it would be beneficial to weaken the control of Tregs over immune responses—for example, in the treatment of cancer.”

Lab mouse

Regulatory T cells (Tregs) need T-cell receptors to fulfill their protective functions, according to research published in Immunity.

The researchers knew that Tregs need T-cell receptors to develop properly, but they were unsure of the receptors’ role after that.

To find out, the team deactivated T-cell receptors on mature Tregs in genetically modified mice.

They found these defective Tregs were not able to carry out their protective function without the T-cell receptors.

Furthermore, the Treg pool fell significantly, as these cells were no longer multiplying.

However, the researchers also discovered that two of Tregs’ most well-known central molecular properties—the production of Foxp3 protein and specific chemical changes to DNA—were still present in the defective T cells.

“Without their receptor, the Tregs are still clearly identifiable as Tregs,” said study author Christoph Vahl, PhD, of the Max Planck Institute of Biochemistry in Martinsried, Germany.

“However, they lose a large part of their cellular identity. They also lose their special ability to suppress excessive immune reactions.”

“The Tregs obviously need continuous contact with their environment to function correctly. This is presumably the reason why they need a receptor that recognizes endogenous substances and continuously sends signals.”

“During the course of our research, we uncovered a very important mechanism for suppressing excessive responses and responses targeted against the human body,” added Marc Schmidt-Supprian, PhD, also of the Max Planck Institute.

“These findings could be relevant for situations where it would be beneficial to weaken the control of Tregs over immune responses—for example, in the treatment of cancer.”

Lab mouse

Regulatory T cells (Tregs) need T-cell receptors to fulfill their protective functions, according to research published in Immunity.

The researchers knew that Tregs need T-cell receptors to develop properly, but they were unsure of the receptors’ role after that.

To find out, the team deactivated T-cell receptors on mature Tregs in genetically modified mice.

They found these defective Tregs were not able to carry out their protective function without the T-cell receptors.

Furthermore, the Treg pool fell significantly, as these cells were no longer multiplying.

However, the researchers also discovered that two of Tregs’ most well-known central molecular properties—the production of Foxp3 protein and specific chemical changes to DNA—were still present in the defective T cells.

“Without their receptor, the Tregs are still clearly identifiable as Tregs,” said study author Christoph Vahl, PhD, of the Max Planck Institute of Biochemistry in Martinsried, Germany.

“However, they lose a large part of their cellular identity. They also lose their special ability to suppress excessive immune reactions.”

“The Tregs obviously need continuous contact with their environment to function correctly. This is presumably the reason why they need a receptor that recognizes endogenous substances and continuously sends signals.”

“During the course of our research, we uncovered a very important mechanism for suppressing excessive responses and responses targeted against the human body,” added Marc Schmidt-Supprian, PhD, also of the Max Planck Institute.

“These findings could be relevant for situations where it would be beneficial to weaken the control of Tregs over immune responses—for example, in the treatment of cancer.”

AML cells

Credit: Lance Liotta

Researchers have discovered that interactions between two molecules—STAT3 and PRL-3—may provide a therapeutic target for acute myeloid leukemia (AML).

The team found evidence to suggest that the STAT3-PRL-3 regulatory loop contributes to the development of AML.

Chng Wee Joo, MB ChB, PhD, of the National University Cancer Institute in Singapore, and his colleagues reported these findings in Experimental Hematology.

The researchers discovered that STAT3, a transcription factor, binds and promotes the production of PRL-3 in cells. A decrease in STAT3 levels led to a corresponding decrease in the levels of PRL-3 and diminished the malignant properties of leukemic cells.

The team therefore concluded that a disruption of this regulatory loop may offer an attractive anti-AML therapeutic strategy. Furthermore, PRL-3 has the potential to be used as a biomarker in personalized therapy for AML patients.

The group was the first to report that the PRL-3 protein is overexpressed in 47% of bone marrow samples from AML patients. In addition, cellular levels of STAT3 were found to be elevated in about 50% of AML cases.

The researchers created a core STAT3 signature by analyzing datasets in the scientific literature. And they found that STAT3 core signature was significantly enriched in AML cases with high PRL-3 expression.

“Earlier studies on PRL-3 have been conducted in other cancers, but only in recent years has attention been turned to the significance of PRL-3 in blood cancer,” Dr Chng said.

“Previously, the mechanism by which PRL-3 is regulated in AML has also not been fully elucidated. This study reveals a novel connection between these two important oncogenes for the first time and also shows that the STAT3-PRL-3 regulatory loop contributes to the pathogenesis of AML.”

The researchers are now looking into methods to target the STAT3-PRL-3 pathway in AML, which could open up new avenues to treat AML patients with high expression of PRL-3 and offer an attractive anti-leukemia therapeutic strategy.

AML cells

Credit: Lance Liotta

Researchers have discovered that interactions between two molecules—STAT3 and PRL-3—may provide a therapeutic target for acute myeloid leukemia (AML).

The team found evidence to suggest that the STAT3-PRL-3 regulatory loop contributes to the development of AML.

Chng Wee Joo, MB ChB, PhD, of the National University Cancer Institute in Singapore, and his colleagues reported these findings in Experimental Hematology.

The researchers discovered that STAT3, a transcription factor, binds and promotes the production of PRL-3 in cells. A decrease in STAT3 levels led to a corresponding decrease in the levels of PRL-3 and diminished the malignant properties of leukemic cells.

The team therefore concluded that a disruption of this regulatory loop may offer an attractive anti-AML therapeutic strategy. Furthermore, PRL-3 has the potential to be used as a biomarker in personalized therapy for AML patients.

The group was the first to report that the PRL-3 protein is overexpressed in 47% of bone marrow samples from AML patients. In addition, cellular levels of STAT3 were found to be elevated in about 50% of AML cases.

The researchers created a core STAT3 signature by analyzing datasets in the scientific literature. And they found that STAT3 core signature was significantly enriched in AML cases with high PRL-3 expression.

“Earlier studies on PRL-3 have been conducted in other cancers, but only in recent years has attention been turned to the significance of PRL-3 in blood cancer,” Dr Chng said.

“Previously, the mechanism by which PRL-3 is regulated in AML has also not been fully elucidated. This study reveals a novel connection between these two important oncogenes for the first time and also shows that the STAT3-PRL-3 regulatory loop contributes to the pathogenesis of AML.”

The researchers are now looking into methods to target the STAT3-PRL-3 pathway in AML, which could open up new avenues to treat AML patients with high expression of PRL-3 and offer an attractive anti-leukemia therapeutic strategy.

AML cells

Credit: Lance Liotta

Researchers have discovered that interactions between two molecules—STAT3 and PRL-3—may provide a therapeutic target for acute myeloid leukemia (AML).

The team found evidence to suggest that the STAT3-PRL-3 regulatory loop contributes to the development of AML.

Chng Wee Joo, MB ChB, PhD, of the National University Cancer Institute in Singapore, and his colleagues reported these findings in Experimental Hematology.

The researchers discovered that STAT3, a transcription factor, binds and promotes the production of PRL-3 in cells. A decrease in STAT3 levels led to a corresponding decrease in the levels of PRL-3 and diminished the malignant properties of leukemic cells.

The team therefore concluded that a disruption of this regulatory loop may offer an attractive anti-AML therapeutic strategy. Furthermore, PRL-3 has the potential to be used as a biomarker in personalized therapy for AML patients.

The group was the first to report that the PRL-3 protein is overexpressed in 47% of bone marrow samples from AML patients. In addition, cellular levels of STAT3 were found to be elevated in about 50% of AML cases.

The researchers created a core STAT3 signature by analyzing datasets in the scientific literature. And they found that STAT3 core signature was significantly enriched in AML cases with high PRL-3 expression.

“Earlier studies on PRL-3 have been conducted in other cancers, but only in recent years has attention been turned to the significance of PRL-3 in blood cancer,” Dr Chng said.

“Previously, the mechanism by which PRL-3 is regulated in AML has also not been fully elucidated. This study reveals a novel connection between these two important oncogenes for the first time and also shows that the STAT3-PRL-3 regulatory loop contributes to the pathogenesis of AML.”

The researchers are now looking into methods to target the STAT3-PRL-3 pathway in AML, which could open up new avenues to treat AML patients with high expression of PRL-3 and offer an attractive anti-leukemia therapeutic strategy.

Doctor consults with cancer

patient and her father

Credit: Rhoda Baer

New research shows that, even decades after being cured, many cancer survivors face challenges resulting from their disease and its treatment.

A survey of more than 1500 cancer survivors revealed 16 themes of challenges or unmet needs, such as physical dysfunction, financial problems, a lack of education about cancer survival, and anxiety about cancer recurrence.

Mary Ann Burg, PhD, of the University of Central Florida in Orlando, and her colleagues reported these findings in Cancer.

To assess the unmet needs of cancer survivors, the researchers evaluated responses from an American Cancer Society survey in which subjects responded to the open-ended question, “Please tell us about any needs you have now as a cancer survivor that are not being met to your satisfaction.”

There were a total of 1514 respondents who were 2, 5, or 10 years from cancer diagnosis. They were 24 to 97 years of age, 65.4% were female, and 24.8% were racial/ethnic minorities (black and Hispanic/Latino).

“This study was unique in that it gave a very large sample of cancer survivors a real voice to express their needs and concerns,” Dr Burg said.

The researchers found that the number and type of challenges/unmet needs were not associated with a subject’s time since cancer treatment, although older cancer survivors tended to report fewer unmet needs than younger survivors.

Sixteen themes of challenges/unmet needs emerged from respondents’ answers, with physical issues being the most common. About 38% of respondents reported physical issues, such as pain, symptoms, and sexual dysfunction.

About 20% reported financial problems, such as issues with insurance and the affordability of needed services and products. About 20% also said they had needs related to unanswered questions and a lack of knowledge about what to expect as a cancer survivor, including guidance on follow-up care and cancer risks, causes, and prevention.

About 16% of respondents cited issues relating to personal control (a lack of physical and social autonomy). And about 16% described flaws and constraints in the healthcare system that affected early detection, diagnosis, treatment, follow-up care, and continuity of care.

About 14% of respondents reported a lack of resources (such as supplies, equipment, and medications), and about 14% cited emotional and mental health issues (such as fear of cancer recurrence, depression, and anxiety).

About 13% of respondents said they lacked social support (such as access to support groups), and 10% reported issues relating to societal perceptions of cancer survivors (such as discrimination and misinformation).

About 9% of respondents expressed the need to talk about or explain the cancer experience with their physician, friends, and family. And about 9% cited a lack of trust in healthcare providers.

Other themes included the wish for more effective cancer treatments (3.5%), body image issues such as feeling unattractive or losing trust in the body (3.5%), issues with the “survivor” identity (3.1%), trouble obtaining or maintaining appropriate employment (2.3%), and existential issues, such as finding meaning in the cancer experience (0.6%).

“Overall, we found that cancer survivors are often caught off guard by the lingering problems they experience after cancer treatment,” Dr Burg said. “In the wake of cancer, many survivors feel they have lost a sense of personal control, have reduced quality of life, and are frustrated that these problems are not sufficiently addressed within the medical care system.”

She added that this study points to several areas in which we might work to improve the situation, including raising public awareness of cancer survivors’ problems, promoting honest professional communication about the side effects of cancer and its treatment, and coordinating medical care resources to help survivors and their families cope with lingering challenges.

Doctor consults with cancer

patient and her father

Credit: Rhoda Baer

New research shows that, even decades after being cured, many cancer survivors face challenges resulting from their disease and its treatment.

A survey of more than 1500 cancer survivors revealed 16 themes of challenges or unmet needs, such as physical dysfunction, financial problems, a lack of education about cancer survival, and anxiety about cancer recurrence.

Mary Ann Burg, PhD, of the University of Central Florida in Orlando, and her colleagues reported these findings in Cancer.

To assess the unmet needs of cancer survivors, the researchers evaluated responses from an American Cancer Society survey in which subjects responded to the open-ended question, “Please tell us about any needs you have now as a cancer survivor that are not being met to your satisfaction.”

There were a total of 1514 respondents who were 2, 5, or 10 years from cancer diagnosis. They were 24 to 97 years of age, 65.4% were female, and 24.8% were racial/ethnic minorities (black and Hispanic/Latino).

“This study was unique in that it gave a very large sample of cancer survivors a real voice to express their needs and concerns,” Dr Burg said.

The researchers found that the number and type of challenges/unmet needs were not associated with a subject’s time since cancer treatment, although older cancer survivors tended to report fewer unmet needs than younger survivors.

Sixteen themes of challenges/unmet needs emerged from respondents’ answers, with physical issues being the most common. About 38% of respondents reported physical issues, such as pain, symptoms, and sexual dysfunction.

About 20% reported financial problems, such as issues with insurance and the affordability of needed services and products. About 20% also said they had needs related to unanswered questions and a lack of knowledge about what to expect as a cancer survivor, including guidance on follow-up care and cancer risks, causes, and prevention.

About 16% of respondents cited issues relating to personal control (a lack of physical and social autonomy). And about 16% described flaws and constraints in the healthcare system that affected early detection, diagnosis, treatment, follow-up care, and continuity of care.

About 14% of respondents reported a lack of resources (such as supplies, equipment, and medications), and about 14% cited emotional and mental health issues (such as fear of cancer recurrence, depression, and anxiety).

About 13% of respondents said they lacked social support (such as access to support groups), and 10% reported issues relating to societal perceptions of cancer survivors (such as discrimination and misinformation).

About 9% of respondents expressed the need to talk about or explain the cancer experience with their physician, friends, and family. And about 9% cited a lack of trust in healthcare providers.

Other themes included the wish for more effective cancer treatments (3.5%), body image issues such as feeling unattractive or losing trust in the body (3.5%), issues with the “survivor” identity (3.1%), trouble obtaining or maintaining appropriate employment (2.3%), and existential issues, such as finding meaning in the cancer experience (0.6%).

“Overall, we found that cancer survivors are often caught off guard by the lingering problems they experience after cancer treatment,” Dr Burg said. “In the wake of cancer, many survivors feel they have lost a sense of personal control, have reduced quality of life, and are frustrated that these problems are not sufficiently addressed within the medical care system.”

She added that this study points to several areas in which we might work to improve the situation, including raising public awareness of cancer survivors’ problems, promoting honest professional communication about the side effects of cancer and its treatment, and coordinating medical care resources to help survivors and their families cope with lingering challenges.

Doctor consults with cancer

patient and her father

Credit: Rhoda Baer

New research shows that, even decades after being cured, many cancer survivors face challenges resulting from their disease and its treatment.

A survey of more than 1500 cancer survivors revealed 16 themes of challenges or unmet needs, such as physical dysfunction, financial problems, a lack of education about cancer survival, and anxiety about cancer recurrence.

Mary Ann Burg, PhD, of the University of Central Florida in Orlando, and her colleagues reported these findings in Cancer.

To assess the unmet needs of cancer survivors, the researchers evaluated responses from an American Cancer Society survey in which subjects responded to the open-ended question, “Please tell us about any needs you have now as a cancer survivor that are not being met to your satisfaction.”

There were a total of 1514 respondents who were 2, 5, or 10 years from cancer diagnosis. They were 24 to 97 years of age, 65.4% were female, and 24.8% were racial/ethnic minorities (black and Hispanic/Latino).

“This study was unique in that it gave a very large sample of cancer survivors a real voice to express their needs and concerns,” Dr Burg said.

The researchers found that the number and type of challenges/unmet needs were not associated with a subject’s time since cancer treatment, although older cancer survivors tended to report fewer unmet needs than younger survivors.

Sixteen themes of challenges/unmet needs emerged from respondents’ answers, with physical issues being the most common. About 38% of respondents reported physical issues, such as pain, symptoms, and sexual dysfunction.

About 20% reported financial problems, such as issues with insurance and the affordability of needed services and products. About 20% also said they had needs related to unanswered questions and a lack of knowledge about what to expect as a cancer survivor, including guidance on follow-up care and cancer risks, causes, and prevention.

About 16% of respondents cited issues relating to personal control (a lack of physical and social autonomy). And about 16% described flaws and constraints in the healthcare system that affected early detection, diagnosis, treatment, follow-up care, and continuity of care.

About 14% of respondents reported a lack of resources (such as supplies, equipment, and medications), and about 14% cited emotional and mental health issues (such as fear of cancer recurrence, depression, and anxiety).

About 13% of respondents said they lacked social support (such as access to support groups), and 10% reported issues relating to societal perceptions of cancer survivors (such as discrimination and misinformation).

About 9% of respondents expressed the need to talk about or explain the cancer experience with their physician, friends, and family. And about 9% cited a lack of trust in healthcare providers.

Other themes included the wish for more effective cancer treatments (3.5%), body image issues such as feeling unattractive or losing trust in the body (3.5%), issues with the “survivor” identity (3.1%), trouble obtaining or maintaining appropriate employment (2.3%), and existential issues, such as finding meaning in the cancer experience (0.6%).

“Overall, we found that cancer survivors are often caught off guard by the lingering problems they experience after cancer treatment,” Dr Burg said. “In the wake of cancer, many survivors feel they have lost a sense of personal control, have reduced quality of life, and are frustrated that these problems are not sufficiently addressed within the medical care system.”

She added that this study points to several areas in which we might work to improve the situation, including raising public awareness of cancer survivors’ problems, promoting honest professional communication about the side effects of cancer and its treatment, and coordinating medical care resources to help survivors and their families cope with lingering challenges.