Lymphoma & Plasma Cell Disorders

Lab mouse

Scientists believe they’ve devised a way to use antimiRs as anticancer drugs by showing that a specific antimiR could treat diffuse large B-cell lymphoma (DLBCL) in mice.

In a letter to Nature, the group explained that microRNAs known as oncomiRs can play a causal role in the onset and maintenance of cancer when they are overexpressed.

So inhibiting oncomiRs using antisense oligomers, or antimiRs, has seemed a promising therapeutic strategy. But physiological and cellular barriers have prevented targeted delivery.

Frank Slack, PhD, of Beth Israel Deaconess Medical Center in Boston, Massachusetts, and his colleagues have devised a new antimiR delivery platform and shown that it can inhibit DLBCL growth in vivo.

The team created a mouse model to study miR-155, an oncomiR that, when overexpressed, leads to DLBCL.

“We hypothesized that we could inhibit the function of miR-155 by way of an antisense molecule that would bind to miR-155,” Dr Slack said. “[However,] there are a number of significant obstacles to reaching the tumor cell target. Some roadblocks are clearance through the kidneys and accumulation in the liver, which absorbs any systemically injected agent.”

“Furthermore, even if you are able to reach your targeted cells, the molecules must cross cell membranes and escape degradation from a process known as endocytosis. If you can picture our antisense molecule being a warhead, we had to find the right ‘rocket’ to actually transport it to its target.”

The “rocket” turned out to be a peptide with a low-pH induced transmembrane structure (pHLIP), meaning it inserts into cell membranes only when cells are low in pH. And tumor cells provided the ideal environment.

“When we attached our antisense warhead to the pHLIP peptide, not only did it successfully insert itself into the tumor cell, but it also dragged the antisense molecule itself into the cell,” Dr Slack said. “Now the ‘warhead’ could deploy and actually inhibit microRNA function and control cancer growth.”

In the miR-155/DLBCL mouse models, pHLIP-anti155 slowed tumor growth, suppressed the metastatic spread of neoplastic lymphocytes to other organs, reduced the onset of splenomegaly, and delayed the development of conspicuous lymphadenopathy, when compared to control mice.

Responses with pHLIP-anti155 were similar to those observed in mice that received doxorubicin or CHOP, but pHLIP-anti155 proved less toxic than the other treatments.

“With this delivery platform, we should also be able to transform other RNAs into druggable targets,” Dr Slack said, adding that low pH is also an issue in kidney disease, myocardial infarction, stroke, and infection, among other conditions. So this type of therapy could have wide applications.

Lab mouse

Scientists believe they’ve devised a way to use antimiRs as anticancer drugs by showing that a specific antimiR could treat diffuse large B-cell lymphoma (DLBCL) in mice.

In a letter to Nature, the group explained that microRNAs known as oncomiRs can play a causal role in the onset and maintenance of cancer when they are overexpressed.

So inhibiting oncomiRs using antisense oligomers, or antimiRs, has seemed a promising therapeutic strategy. But physiological and cellular barriers have prevented targeted delivery.

Frank Slack, PhD, of Beth Israel Deaconess Medical Center in Boston, Massachusetts, and his colleagues have devised a new antimiR delivery platform and shown that it can inhibit DLBCL growth in vivo.

The team created a mouse model to study miR-155, an oncomiR that, when overexpressed, leads to DLBCL.

“We hypothesized that we could inhibit the function of miR-155 by way of an antisense molecule that would bind to miR-155,” Dr Slack said. “[However,] there are a number of significant obstacles to reaching the tumor cell target. Some roadblocks are clearance through the kidneys and accumulation in the liver, which absorbs any systemically injected agent.”

“Furthermore, even if you are able to reach your targeted cells, the molecules must cross cell membranes and escape degradation from a process known as endocytosis. If you can picture our antisense molecule being a warhead, we had to find the right ‘rocket’ to actually transport it to its target.”

The “rocket” turned out to be a peptide with a low-pH induced transmembrane structure (pHLIP), meaning it inserts into cell membranes only when cells are low in pH. And tumor cells provided the ideal environment.

“When we attached our antisense warhead to the pHLIP peptide, not only did it successfully insert itself into the tumor cell, but it also dragged the antisense molecule itself into the cell,” Dr Slack said. “Now the ‘warhead’ could deploy and actually inhibit microRNA function and control cancer growth.”

In the miR-155/DLBCL mouse models, pHLIP-anti155 slowed tumor growth, suppressed the metastatic spread of neoplastic lymphocytes to other organs, reduced the onset of splenomegaly, and delayed the development of conspicuous lymphadenopathy, when compared to control mice.

Responses with pHLIP-anti155 were similar to those observed in mice that received doxorubicin or CHOP, but pHLIP-anti155 proved less toxic than the other treatments.

“With this delivery platform, we should also be able to transform other RNAs into druggable targets,” Dr Slack said, adding that low pH is also an issue in kidney disease, myocardial infarction, stroke, and infection, among other conditions. So this type of therapy could have wide applications.

Lab mouse

Scientists believe they’ve devised a way to use antimiRs as anticancer drugs by showing that a specific antimiR could treat diffuse large B-cell lymphoma (DLBCL) in mice.

In a letter to Nature, the group explained that microRNAs known as oncomiRs can play a causal role in the onset and maintenance of cancer when they are overexpressed.

So inhibiting oncomiRs using antisense oligomers, or antimiRs, has seemed a promising therapeutic strategy. But physiological and cellular barriers have prevented targeted delivery.

Frank Slack, PhD, of Beth Israel Deaconess Medical Center in Boston, Massachusetts, and his colleagues have devised a new antimiR delivery platform and shown that it can inhibit DLBCL growth in vivo.

The team created a mouse model to study miR-155, an oncomiR that, when overexpressed, leads to DLBCL.

“We hypothesized that we could inhibit the function of miR-155 by way of an antisense molecule that would bind to miR-155,” Dr Slack said. “[However,] there are a number of significant obstacles to reaching the tumor cell target. Some roadblocks are clearance through the kidneys and accumulation in the liver, which absorbs any systemically injected agent.”

“Furthermore, even if you are able to reach your targeted cells, the molecules must cross cell membranes and escape degradation from a process known as endocytosis. If you can picture our antisense molecule being a warhead, we had to find the right ‘rocket’ to actually transport it to its target.”

The “rocket” turned out to be a peptide with a low-pH induced transmembrane structure (pHLIP), meaning it inserts into cell membranes only when cells are low in pH. And tumor cells provided the ideal environment.

“When we attached our antisense warhead to the pHLIP peptide, not only did it successfully insert itself into the tumor cell, but it also dragged the antisense molecule itself into the cell,” Dr Slack said. “Now the ‘warhead’ could deploy and actually inhibit microRNA function and control cancer growth.”

In the miR-155/DLBCL mouse models, pHLIP-anti155 slowed tumor growth, suppressed the metastatic spread of neoplastic lymphocytes to other organs, reduced the onset of splenomegaly, and delayed the development of conspicuous lymphadenopathy, when compared to control mice.

Responses with pHLIP-anti155 were similar to those observed in mice that received doxorubicin or CHOP, but pHLIP-anti155 proved less toxic than the other treatments.

“With this delivery platform, we should also be able to transform other RNAs into druggable targets,” Dr Slack said, adding that low pH is also an issue in kidney disease, myocardial infarction, stroke, and infection, among other conditions. So this type of therapy could have wide applications.

Breast implant

Credit: FDA

Bacterial infection on the surface of textured breast implants may increase the risk of developing breast implant-associated anaplastic large-cell lymphoma (BIA-ALCL), according to research published in Plastic & Reconstructive Surgery.

Previous studies have shown that biofilm infection around breast implants is a major cause of capsular contracture, a painful hardening of the tissue around the implant that can cause physical deformity and pain.

Now, researchers have found that chronic infection around implants can also lead to an activation of the immune system and the patient’s lymphocytes. And long-term stimulation of lymphocytes by this infection may prompt the transformation of these cells into BIA-ALCL.

The infection was shown to be highest around textured breast implants, and this may provide an explanation as to why BIA-ALCL seems to be more common in patients with textured implants.

“Our previous research has shown that, 24 hours after bacteria come into contact with breast implants, textured implants had 72 times the number of bacteria attached to their surface as compared with the smooth implants,” said Anand Deva, MBBS, of Macquarie University in Sydney, Australia.

“This latest study has shown that the textured implants with the highest numbers of bacteria also had the highest number of activated lymphocytes around them. This finding is important and has now become even more relevant since the reporting of BIA-ALCL, as it provides us with a possible biological explanation of how this rare cancer could arise.”

To uncover these findings, Dr Devan and his colleagues first examined implants in pigs. The team inserted 12 textured and 12 smooth implants into submammary pockets in 3 adult pigs.

After a mean of 8.75 months, all of the samples were positive for bacterial biofilm. And there was a significant correlation between bacterial numbers and the grade of capsular contracture (P=0.04).

Lymphocyte numbers were significantly higher on textured implants (P<0.001), with T cells accounting for the majority of the lymphocytic infiltrate.

The researchers then examined implants in humans, collecting 57 capsules from patients with Baker grade 4 capsules over a 4-year period. The team analyzed biofilm and the surrounding lymphocytes.

As in the pigs, all of the capsules were positive for biofilm, and T cells were the predominant lymphocyte (P<0.001).

The researchers also discovered a significant linear correlation between the number of T and B cells and the number of detected bacteria (P<0.001). And there was a significantly higher number of bacteria for polyurethane implants (P<0.005).

These results suggest a possible link between bacterial biofilm and T-cell hyperplasia, a finding that may have implications for BIA-ALCL, the researchers said.

Dr Deva and his colleagues have published a 14-step guide to reduce the risk of breast implant infection, based on evidence of best practice to educate surgeons on how to reduce the risk of bacterial contamination.

A number of clinical studies have applied these principles and successfully reduced the rate of capsular contracture by a factor of 10 in their patients.

“This is a great validation of our research and a demonstration that good science in the laboratory can be translated into real benefits to patients at the bedside,” Dr Deva said.

“Now, with our greater understanding of the importance of preventing infection, we, as surgeons, can reduce the risk of capsular contracture and thereby reduce the risk of lymphocyte activation and possible transformation into BIA-ALCL.”

Breast implant

Credit: FDA

Bacterial infection on the surface of textured breast implants may increase the risk of developing breast implant-associated anaplastic large-cell lymphoma (BIA-ALCL), according to research published in Plastic & Reconstructive Surgery.

Previous studies have shown that biofilm infection around breast implants is a major cause of capsular contracture, a painful hardening of the tissue around the implant that can cause physical deformity and pain.

Now, researchers have found that chronic infection around implants can also lead to an activation of the immune system and the patient’s lymphocytes. And long-term stimulation of lymphocytes by this infection may prompt the transformation of these cells into BIA-ALCL.

The infection was shown to be highest around textured breast implants, and this may provide an explanation as to why BIA-ALCL seems to be more common in patients with textured implants.

“Our previous research has shown that, 24 hours after bacteria come into contact with breast implants, textured implants had 72 times the number of bacteria attached to their surface as compared with the smooth implants,” said Anand Deva, MBBS, of Macquarie University in Sydney, Australia.

“This latest study has shown that the textured implants with the highest numbers of bacteria also had the highest number of activated lymphocytes around them. This finding is important and has now become even more relevant since the reporting of BIA-ALCL, as it provides us with a possible biological explanation of how this rare cancer could arise.”

To uncover these findings, Dr Devan and his colleagues first examined implants in pigs. The team inserted 12 textured and 12 smooth implants into submammary pockets in 3 adult pigs.

After a mean of 8.75 months, all of the samples were positive for bacterial biofilm. And there was a significant correlation between bacterial numbers and the grade of capsular contracture (P=0.04).

Lymphocyte numbers were significantly higher on textured implants (P<0.001), with T cells accounting for the majority of the lymphocytic infiltrate.

The researchers then examined implants in humans, collecting 57 capsules from patients with Baker grade 4 capsules over a 4-year period. The team analyzed biofilm and the surrounding lymphocytes.

As in the pigs, all of the capsules were positive for biofilm, and T cells were the predominant lymphocyte (P<0.001).

The researchers also discovered a significant linear correlation between the number of T and B cells and the number of detected bacteria (P<0.001). And there was a significantly higher number of bacteria for polyurethane implants (P<0.005).

These results suggest a possible link between bacterial biofilm and T-cell hyperplasia, a finding that may have implications for BIA-ALCL, the researchers said.

Dr Deva and his colleagues have published a 14-step guide to reduce the risk of breast implant infection, based on evidence of best practice to educate surgeons on how to reduce the risk of bacterial contamination.

A number of clinical studies have applied these principles and successfully reduced the rate of capsular contracture by a factor of 10 in their patients.

“This is a great validation of our research and a demonstration that good science in the laboratory can be translated into real benefits to patients at the bedside,” Dr Deva said.

“Now, with our greater understanding of the importance of preventing infection, we, as surgeons, can reduce the risk of capsular contracture and thereby reduce the risk of lymphocyte activation and possible transformation into BIA-ALCL.”

Breast implant

Credit: FDA

Bacterial infection on the surface of textured breast implants may increase the risk of developing breast implant-associated anaplastic large-cell lymphoma (BIA-ALCL), according to research published in Plastic & Reconstructive Surgery.

Previous studies have shown that biofilm infection around breast implants is a major cause of capsular contracture, a painful hardening of the tissue around the implant that can cause physical deformity and pain.

Now, researchers have found that chronic infection around implants can also lead to an activation of the immune system and the patient’s lymphocytes. And long-term stimulation of lymphocytes by this infection may prompt the transformation of these cells into BIA-ALCL.

The infection was shown to be highest around textured breast implants, and this may provide an explanation as to why BIA-ALCL seems to be more common in patients with textured implants.

“Our previous research has shown that, 24 hours after bacteria come into contact with breast implants, textured implants had 72 times the number of bacteria attached to their surface as compared with the smooth implants,” said Anand Deva, MBBS, of Macquarie University in Sydney, Australia.

“This latest study has shown that the textured implants with the highest numbers of bacteria also had the highest number of activated lymphocytes around them. This finding is important and has now become even more relevant since the reporting of BIA-ALCL, as it provides us with a possible biological explanation of how this rare cancer could arise.”

To uncover these findings, Dr Devan and his colleagues first examined implants in pigs. The team inserted 12 textured and 12 smooth implants into submammary pockets in 3 adult pigs.

After a mean of 8.75 months, all of the samples were positive for bacterial biofilm. And there was a significant correlation between bacterial numbers and the grade of capsular contracture (P=0.04).

Lymphocyte numbers were significantly higher on textured implants (P<0.001), with T cells accounting for the majority of the lymphocytic infiltrate.

The researchers then examined implants in humans, collecting 57 capsules from patients with Baker grade 4 capsules over a 4-year period. The team analyzed biofilm and the surrounding lymphocytes.

As in the pigs, all of the capsules were positive for biofilm, and T cells were the predominant lymphocyte (P<0.001).

The researchers also discovered a significant linear correlation between the number of T and B cells and the number of detected bacteria (P<0.001). And there was a significantly higher number of bacteria for polyurethane implants (P<0.005).

These results suggest a possible link between bacterial biofilm and T-cell hyperplasia, a finding that may have implications for BIA-ALCL, the researchers said.

Dr Deva and his colleagues have published a 14-step guide to reduce the risk of breast implant infection, based on evidence of best practice to educate surgeons on how to reduce the risk of bacterial contamination.

A number of clinical studies have applied these principles and successfully reduced the rate of capsular contracture by a factor of 10 in their patients.

“This is a great validation of our research and a demonstration that good science in the laboratory can be translated into real benefits to patients at the bedside,” Dr Deva said.

“Now, with our greater understanding of the importance of preventing infection, we, as surgeons, can reduce the risk of capsular contracture and thereby reduce the risk of lymphocyte activation and possible transformation into BIA-ALCL.”

Doctor and patient

Credit: CDC

Patients with advanced cancer receive less aggressive care and have lower healthcare costs during their last year of life if they use hospice care, according to research published in JAMA.

Patients who entered hospice had significantly lower rates of hospitalization, intensive care unit admissions, and invasive procedures, compared to patients who did not enter hospice.

Furthermore, patients who chose hospice were about 5 times less likely to die in hospitals and nursing homes.

“Our study shows very clearly that hospice matters,” said Ziad Obermeyer, MD, of Brigham and Women’s Hospital in Boston.

“Hospice and non-hospice patients had very similar patterns of healthcare utilization right up until the week of hospice enrollment. Then, the care started to look very different. Patients who didn’t enroll in hospice ended up with far more aggressive care in their last year of life, most of it related to acute complications like infections and organ failure, and not directly related to their cancer diagnosis.”

To conduct this study, Dr Obermeyer and his colleagues used data from Medicare beneficiaries with poor-prognosis cancers, including hematologic malignancies. The study included a nationally representative 20% sample of Medicare fee-for-service beneficiaries who died in 2011.

Among 86,851 patients, 51,924 (60%) entered hospice before death. Matching patients based on various criteria produced hospice and non-hospice groups, each with 18,165 patients. The median hospice duration was 11 days.

The researchers found that non-hospice patients had significantly greater healthcare utilization, largely for acute conditions not directly related to cancer.

This included rates of hospitalization (65% vs 42%), intensive care unit admissions (36% vs 15%), invasive procedures (51% vs 27%), and death in a hospital or nursing facility (74% vs 14%).

The costs of care for hospice and non-hospice patients were not significantly different before hospice care began. But they diverged sharply thereafter, contributing to a significant difference in total costs of $8697 over the last year of life—$71,517 for non-hospice patients and $62,819 for hospice patients.

“These findings highlight the importance of honest discussions between doctors and patients about our patients’ goals of their care at the end of life, relating to treatment decisions and quality of life,” Dr  Obermeyer said. “This is of particular importance now, in light of the ongoing policy discussions around reimbursing providers for advance-care planning.”

Doctor and patient

Credit: CDC

Patients with advanced cancer receive less aggressive care and have lower healthcare costs during their last year of life if they use hospice care, according to research published in JAMA.

Patients who entered hospice had significantly lower rates of hospitalization, intensive care unit admissions, and invasive procedures, compared to patients who did not enter hospice.

Furthermore, patients who chose hospice were about 5 times less likely to die in hospitals and nursing homes.

“Our study shows very clearly that hospice matters,” said Ziad Obermeyer, MD, of Brigham and Women’s Hospital in Boston.

“Hospice and non-hospice patients had very similar patterns of healthcare utilization right up until the week of hospice enrollment. Then, the care started to look very different. Patients who didn’t enroll in hospice ended up with far more aggressive care in their last year of life, most of it related to acute complications like infections and organ failure, and not directly related to their cancer diagnosis.”

To conduct this study, Dr Obermeyer and his colleagues used data from Medicare beneficiaries with poor-prognosis cancers, including hematologic malignancies. The study included a nationally representative 20% sample of Medicare fee-for-service beneficiaries who died in 2011.

Among 86,851 patients, 51,924 (60%) entered hospice before death. Matching patients based on various criteria produced hospice and non-hospice groups, each with 18,165 patients. The median hospice duration was 11 days.

The researchers found that non-hospice patients had significantly greater healthcare utilization, largely for acute conditions not directly related to cancer.

This included rates of hospitalization (65% vs 42%), intensive care unit admissions (36% vs 15%), invasive procedures (51% vs 27%), and death in a hospital or nursing facility (74% vs 14%).

The costs of care for hospice and non-hospice patients were not significantly different before hospice care began. But they diverged sharply thereafter, contributing to a significant difference in total costs of $8697 over the last year of life—$71,517 for non-hospice patients and $62,819 for hospice patients.

“These findings highlight the importance of honest discussions between doctors and patients about our patients’ goals of their care at the end of life, relating to treatment decisions and quality of life,” Dr  Obermeyer said. “This is of particular importance now, in light of the ongoing policy discussions around reimbursing providers for advance-care planning.”

Doctor and patient

Credit: CDC

Patients with advanced cancer receive less aggressive care and have lower healthcare costs during their last year of life if they use hospice care, according to research published in JAMA.

Patients who entered hospice had significantly lower rates of hospitalization, intensive care unit admissions, and invasive procedures, compared to patients who did not enter hospice.

Furthermore, patients who chose hospice were about 5 times less likely to die in hospitals and nursing homes.

“Our study shows very clearly that hospice matters,” said Ziad Obermeyer, MD, of Brigham and Women’s Hospital in Boston.

“Hospice and non-hospice patients had very similar patterns of healthcare utilization right up until the week of hospice enrollment. Then, the care started to look very different. Patients who didn’t enroll in hospice ended up with far more aggressive care in their last year of life, most of it related to acute complications like infections and organ failure, and not directly related to their cancer diagnosis.”

To conduct this study, Dr Obermeyer and his colleagues used data from Medicare beneficiaries with poor-prognosis cancers, including hematologic malignancies. The study included a nationally representative 20% sample of Medicare fee-for-service beneficiaries who died in 2011.

Among 86,851 patients, 51,924 (60%) entered hospice before death. Matching patients based on various criteria produced hospice and non-hospice groups, each with 18,165 patients. The median hospice duration was 11 days.

The researchers found that non-hospice patients had significantly greater healthcare utilization, largely for acute conditions not directly related to cancer.

This included rates of hospitalization (65% vs 42%), intensive care unit admissions (36% vs 15%), invasive procedures (51% vs 27%), and death in a hospital or nursing facility (74% vs 14%).

The costs of care for hospice and non-hospice patients were not significantly different before hospice care began. But they diverged sharply thereafter, contributing to a significant difference in total costs of $8697 over the last year of life—$71,517 for non-hospice patients and $62,819 for hospice patients.

“These findings highlight the importance of honest discussions between doctors and patients about our patients’ goals of their care at the end of life, relating to treatment decisions and quality of life,” Dr  Obermeyer said. “This is of particular importance now, in light of the ongoing policy discussions around reimbursing providers for advance-care planning.”

Doctor consults with cancer

patient and her father

Credit: Rhoda Baer

A new tool can identify patients with limited cancer health literacy, according to research published in the Journal of Health Communications.

The study authors noted that cancer patients are expected to play an active role in their care by adhering to medication regimens, distinguishing between scientifically credible medical evidence and misconceptions, and communicating with their providers about treatment decisions, risks, and survival rates.

However, patients with limited cancer health literacy can struggle with these responsibilities and potentially jeopardize their health by making uninformed decisions.

So Levent Dumenci, PhD, of Virginia Commonwealth University in Richmond, and his colleagues developed a tool to assess health literacy among cancer patients.

“Before now, there were only instruments that measured a particular aspect of general health literacy,” Dr Dumenci said. “It is important to have a tool that is specific to cancer because of the complex treatment options that cancer patients face, along with the increased demand for self-care.”

To meet that need, he and his colleagues developed the Cancer Health Literacy Test (CHLT)-30. The test, which is given via a touch-screen device, includes 30

questions about cancer treatment, medication side effects, and other cancer-related issues.

Six of the questions (CHLT-6) were specifically designed to quickly identify individuals with limited cancer health literacy.

The researchers tested the CHLT-30 in 1306 African American and Caucasian patients who were treated at oncology clinics in Virginia.

The team said the test accurately measured literacy and quickly identified patients with limited cancer health literacy. Eighteen percent of cancer patients had limited literacy, with an overrepresentation of African American, undereducated, and low-income patients.

“Using this tool, it takes just 1 to 2 minutes in the doctor’s office waiting room to identify patients with limited [cancer health literacy],” Dr Dumenci said. “Then, this information can be digitally communicated to the doctors prior to seeing the patients, so that they are prepared to talk with the patients in terms they can understand. This simple change could lead to big improvements in health outcomes.”

In future studies, the researchers hope to evaluate the cancer health literacy of Hispanic patients because prior research has pointed to disparities in this population’s health literacy.

Doctor consults with cancer

patient and her father

Credit: Rhoda Baer

A new tool can identify patients with limited cancer health literacy, according to research published in the Journal of Health Communications.

The study authors noted that cancer patients are expected to play an active role in their care by adhering to medication regimens, distinguishing between scientifically credible medical evidence and misconceptions, and communicating with their providers about treatment decisions, risks, and survival rates.

However, patients with limited cancer health literacy can struggle with these responsibilities and potentially jeopardize their health by making uninformed decisions.

So Levent Dumenci, PhD, of Virginia Commonwealth University in Richmond, and his colleagues developed a tool to assess health literacy among cancer patients.

“Before now, there were only instruments that measured a particular aspect of general health literacy,” Dr Dumenci said. “It is important to have a tool that is specific to cancer because of the complex treatment options that cancer patients face, along with the increased demand for self-care.”

To meet that need, he and his colleagues developed the Cancer Health Literacy Test (CHLT)-30. The test, which is given via a touch-screen device, includes 30

questions about cancer treatment, medication side effects, and other cancer-related issues.

Six of the questions (CHLT-6) were specifically designed to quickly identify individuals with limited cancer health literacy.

The researchers tested the CHLT-30 in 1306 African American and Caucasian patients who were treated at oncology clinics in Virginia.

The team said the test accurately measured literacy and quickly identified patients with limited cancer health literacy. Eighteen percent of cancer patients had limited literacy, with an overrepresentation of African American, undereducated, and low-income patients.

“Using this tool, it takes just 1 to 2 minutes in the doctor’s office waiting room to identify patients with limited [cancer health literacy],” Dr Dumenci said. “Then, this information can be digitally communicated to the doctors prior to seeing the patients, so that they are prepared to talk with the patients in terms they can understand. This simple change could lead to big improvements in health outcomes.”

In future studies, the researchers hope to evaluate the cancer health literacy of Hispanic patients because prior research has pointed to disparities in this population’s health literacy.

Doctor consults with cancer

patient and her father

Credit: Rhoda Baer

A new tool can identify patients with limited cancer health literacy, according to research published in the Journal of Health Communications.

The study authors noted that cancer patients are expected to play an active role in their care by adhering to medication regimens, distinguishing between scientifically credible medical evidence and misconceptions, and communicating with their providers about treatment decisions, risks, and survival rates.

However, patients with limited cancer health literacy can struggle with these responsibilities and potentially jeopardize their health by making uninformed decisions.

So Levent Dumenci, PhD, of Virginia Commonwealth University in Richmond, and his colleagues developed a tool to assess health literacy among cancer patients.

“Before now, there were only instruments that measured a particular aspect of general health literacy,” Dr Dumenci said. “It is important to have a tool that is specific to cancer because of the complex treatment options that cancer patients face, along with the increased demand for self-care.”

To meet that need, he and his colleagues developed the Cancer Health Literacy Test (CHLT)-30. The test, which is given via a touch-screen device, includes 30

questions about cancer treatment, medication side effects, and other cancer-related issues.

Six of the questions (CHLT-6) were specifically designed to quickly identify individuals with limited cancer health literacy.

The researchers tested the CHLT-30 in 1306 African American and Caucasian patients who were treated at oncology clinics in Virginia.

The team said the test accurately measured literacy and quickly identified patients with limited cancer health literacy. Eighteen percent of cancer patients had limited literacy, with an overrepresentation of African American, undereducated, and low-income patients.

“Using this tool, it takes just 1 to 2 minutes in the doctor’s office waiting room to identify patients with limited [cancer health literacy],” Dr Dumenci said. “Then, this information can be digitally communicated to the doctors prior to seeing the patients, so that they are prepared to talk with the patients in terms they can understand. This simple change could lead to big improvements in health outcomes.”

In future studies, the researchers hope to evaluate the cancer health literacy of Hispanic patients because prior research has pointed to disparities in this population’s health literacy.

Drugs in vials

Credit: Bill Branson

The US Food and Drug Administration (FDA) has granted fast track designation to two hematology drugs: the monoclonal antibody MOR208 to treat relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and the antifibrotic agent PRM-151 to treat myelofibrosis (MF).

The FDA’s fast track program aims to expedite the development and review of drugs that have the potential to fill an unmet medical need in serious or life-threatening conditions.

MOR208

MOR208 is a humanized monoclonal antibody targeting CD19. It is under development by MorphoSys AG to treat B-cell malignancies. The program is in phase 2 clinical development in chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma.

Preclinical research with MOR208 revealed that it can trigger natural killer cell-mediated lysis of ALL cells. The drug had lytic activity against ALL cells from both adult and pediatric patients.

In a phase 1 study, MOR208 exhibited preliminary efficacy in patients with high-risk, heavily pretreated CLL, prompting responses in 67% of patients. Researchers said toxicity was acceptable, but infusion reactions were common.

“First results of our ongoing phase 2 trial, which we will present at this year’s ASH conference in December, have helped to identify diffuse large B-cell lymphoma as a valuable development opportunity for MOR208,” said Arndt Schottelius, chief development officer of MorphoSys AG.

“We are therefore delighted to have received the fast track designation for further development of MOR208 in DLBCL. The more frequent interactions with the FDA that this enables will help us to expedite the development of MOR208 in this particular subset of non-Hodgkin’s lymphoma patients.”

PRM-151

PRM-151 is a recombinant form of an endogenous human protein, pentraxin-2, that is specifically active at the site of tissue damage. PRM-151 is an agonist that acts as a monocyte/macrophage differentiation factor to prevent and potentially reverse fibrosis.

The drug has shown broad anti-fibrotic activity in preclinical models of fibrotic disease, including pulmonary fibrosis, acute and chronic nephropathy, liver fibrosis, and age-related macular degeneration.

PRM-151 has orphan designation in the US for MF and in both the US and European Union for the treatment of idiopathic pulmonary fibrosis.

The FDA’s fast track designation for PRM-151 covers primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF.

“This designation validates our perspective that there is a clear and compelling need for a novel mechanism for the treatment of myelofibrosis that specifically targets the underlying fibrotic processes of the disease,” said Beth Tréhu, MD, FACP, chief medical officer of Promedior Inc., the company developing PRM-151.

“We will continue to work expeditiously to advance this program through the clinic and look forward to presenting the full data set from the first stage of our phase 2 study later this year.”

Preliminary data from the phase 2 study of PRM-151 demonstrated benefits across all clinically relevant measures of MF, including decreases in bone marrow fibrosis, symptom responses, improvements in hemoglobin and platelets, and reductions in spleen size.

The drug also appeared to be well-tolerated and did not prompt myelosuppression.

Drugs in vials

Credit: Bill Branson

The US Food and Drug Administration (FDA) has granted fast track designation to two hematology drugs: the monoclonal antibody MOR208 to treat relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and the antifibrotic agent PRM-151 to treat myelofibrosis (MF).

The FDA’s fast track program aims to expedite the development and review of drugs that have the potential to fill an unmet medical need in serious or life-threatening conditions.

MOR208

MOR208 is a humanized monoclonal antibody targeting CD19. It is under development by MorphoSys AG to treat B-cell malignancies. The program is in phase 2 clinical development in chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma.

Preclinical research with MOR208 revealed that it can trigger natural killer cell-mediated lysis of ALL cells. The drug had lytic activity against ALL cells from both adult and pediatric patients.

In a phase 1 study, MOR208 exhibited preliminary efficacy in patients with high-risk, heavily pretreated CLL, prompting responses in 67% of patients. Researchers said toxicity was acceptable, but infusion reactions were common.

“First results of our ongoing phase 2 trial, which we will present at this year’s ASH conference in December, have helped to identify diffuse large B-cell lymphoma as a valuable development opportunity for MOR208,” said Arndt Schottelius, chief development officer of MorphoSys AG.

“We are therefore delighted to have received the fast track designation for further development of MOR208 in DLBCL. The more frequent interactions with the FDA that this enables will help us to expedite the development of MOR208 in this particular subset of non-Hodgkin’s lymphoma patients.”

PRM-151

PRM-151 is a recombinant form of an endogenous human protein, pentraxin-2, that is specifically active at the site of tissue damage. PRM-151 is an agonist that acts as a monocyte/macrophage differentiation factor to prevent and potentially reverse fibrosis.

The drug has shown broad anti-fibrotic activity in preclinical models of fibrotic disease, including pulmonary fibrosis, acute and chronic nephropathy, liver fibrosis, and age-related macular degeneration.

PRM-151 has orphan designation in the US for MF and in both the US and European Union for the treatment of idiopathic pulmonary fibrosis.

The FDA’s fast track designation for PRM-151 covers primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF.

“This designation validates our perspective that there is a clear and compelling need for a novel mechanism for the treatment of myelofibrosis that specifically targets the underlying fibrotic processes of the disease,” said Beth Tréhu, MD, FACP, chief medical officer of Promedior Inc., the company developing PRM-151.

“We will continue to work expeditiously to advance this program through the clinic and look forward to presenting the full data set from the first stage of our phase 2 study later this year.”

Preliminary data from the phase 2 study of PRM-151 demonstrated benefits across all clinically relevant measures of MF, including decreases in bone marrow fibrosis, symptom responses, improvements in hemoglobin and platelets, and reductions in spleen size.

The drug also appeared to be well-tolerated and did not prompt myelosuppression.

Drugs in vials

Credit: Bill Branson

The US Food and Drug Administration (FDA) has granted fast track designation to two hematology drugs: the monoclonal antibody MOR208 to treat relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and the antifibrotic agent PRM-151 to treat myelofibrosis (MF).

The FDA’s fast track program aims to expedite the development and review of drugs that have the potential to fill an unmet medical need in serious or life-threatening conditions.

MOR208

MOR208 is a humanized monoclonal antibody targeting CD19. It is under development by MorphoSys AG to treat B-cell malignancies. The program is in phase 2 clinical development in chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma.

Preclinical research with MOR208 revealed that it can trigger natural killer cell-mediated lysis of ALL cells. The drug had lytic activity against ALL cells from both adult and pediatric patients.

In a phase 1 study, MOR208 exhibited preliminary efficacy in patients with high-risk, heavily pretreated CLL, prompting responses in 67% of patients. Researchers said toxicity was acceptable, but infusion reactions were common.

“First results of our ongoing phase 2 trial, which we will present at this year’s ASH conference in December, have helped to identify diffuse large B-cell lymphoma as a valuable development opportunity for MOR208,” said Arndt Schottelius, chief development officer of MorphoSys AG.

“We are therefore delighted to have received the fast track designation for further development of MOR208 in DLBCL. The more frequent interactions with the FDA that this enables will help us to expedite the development of MOR208 in this particular subset of non-Hodgkin’s lymphoma patients.”

PRM-151

PRM-151 is a recombinant form of an endogenous human protein, pentraxin-2, that is specifically active at the site of tissue damage. PRM-151 is an agonist that acts as a monocyte/macrophage differentiation factor to prevent and potentially reverse fibrosis.

The drug has shown broad anti-fibrotic activity in preclinical models of fibrotic disease, including pulmonary fibrosis, acute and chronic nephropathy, liver fibrosis, and age-related macular degeneration.

PRM-151 has orphan designation in the US for MF and in both the US and European Union for the treatment of idiopathic pulmonary fibrosis.

The FDA’s fast track designation for PRM-151 covers primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF.

“This designation validates our perspective that there is a clear and compelling need for a novel mechanism for the treatment of myelofibrosis that specifically targets the underlying fibrotic processes of the disease,” said Beth Tréhu, MD, FACP, chief medical officer of Promedior Inc., the company developing PRM-151.

“We will continue to work expeditiously to advance this program through the clinic and look forward to presenting the full data set from the first stage of our phase 2 study later this year.”

Preliminary data from the phase 2 study of PRM-151 demonstrated benefits across all clinically relevant measures of MF, including decreases in bone marrow fibrosis, symptom responses, improvements in hemoglobin and platelets, and reductions in spleen size.

The drug also appeared to be well-tolerated and did not prompt myelosuppression.

 

 

Sodas and other sweetened

beverages at the supermarket

 

Consuming aspartame and drinking sweetened beverages do not increase a person’s risk of developing non-Hodgkin lymphoma (NHL), according to research published in the Journal of Nutrition.

Investigators analyzed information from more than 100,000 men and women in the US.

The results suggested that neither aspartame intake nor the consumption of sugar-sweetened or artificially sweetened beverages were associated with an increased risk of NHL.

Marjorie L. McCullough, SCD, RD, of the American Cancer Society in Atlanta, Georgia, and her colleagues conducted this research, analyzing data from the nutrition cohort of the Cancer Prevention Study II, an assessment of cancer incidence

and mortality in the US.

Study subjects first completed a questionnaire in 1992, noting information related to diet and other lifestyle factors. They completed follow-up questionnaires in 1999 and 2003, which included questions related to the consumption of sugar-sweetened and artificially sweetened beverages, as well as tabletop sweeteners containing aspartame.

Among the 100,442 adult men and women who provided information on diet and lifestyle factors in 1999, there were 1196 NHL cases verified during a 10-year follow-up period.

The investigators assessed the risk of NHL associated with sweetened beverage and aspartame consumption, adjusted for the subjects’ smoking status, body mass index, and history of diabetes.

The analysis revealed that, in women and men combined, there was no association between NHL risk and the consumption of 1 or more servings (355 mL) of artificially sweetened beverages. Compared to nondrinkers, subjects who drank artificially sweetened beverages had a risk ratio (RR) of 0.92 (P=0.14).

Similarly, there was no association between NHL risk and sugar-sweetened beverages. Compared to nondrinkers, the RR for sugar-sweetened beverage drinkers was 1.10 (P=0.62).

Furthermore, subjects’ overall aspartame intake, which was estimated from artificially sweetened carbonated beverage consumption and the use of aspartame packets, was not associated with NHL risk.

The RR was 1.02 (P=0.69) for the top quintile (which had a median aspartame intake of 145 mg per day) vs the bottom quintile (which had a median aspartame intake of 0 mg per day).

The investigators also found that associations between disease and sweetened beverage consumption or aspartame intake were generally null for specific NHL subtypes, including multiple myeloma, diffuse large B-cell lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma, follicular lymphoma, and other B-cell lymphomas.

“The study supports the decades of research that have continued to find that aspartame is safe for use in foods and beverages,” said Haley Stevens, PhD, President of the Calorie Control Council. “It also supports the conclusions of the National Cancer Institute, who have determined that aspartame does not increase a person’s risk of developing cancer.”

 

 

Sodas and other sweetened

beverages at the supermarket

 

Consuming aspartame and drinking sweetened beverages do not increase a person’s risk of developing non-Hodgkin lymphoma (NHL), according to research published in the Journal of Nutrition.

Investigators analyzed information from more than 100,000 men and women in the US.

The results suggested that neither aspartame intake nor the consumption of sugar-sweetened or artificially sweetened beverages were associated with an increased risk of NHL.

Marjorie L. McCullough, SCD, RD, of the American Cancer Society in Atlanta, Georgia, and her colleagues conducted this research, analyzing data from the nutrition cohort of the Cancer Prevention Study II, an assessment of cancer incidence

and mortality in the US.

Study subjects first completed a questionnaire in 1992, noting information related to diet and other lifestyle factors. They completed follow-up questionnaires in 1999 and 2003, which included questions related to the consumption of sugar-sweetened and artificially sweetened beverages, as well as tabletop sweeteners containing aspartame.

Among the 100,442 adult men and women who provided information on diet and lifestyle factors in 1999, there were 1196 NHL cases verified during a 10-year follow-up period.

The investigators assessed the risk of NHL associated with sweetened beverage and aspartame consumption, adjusted for the subjects’ smoking status, body mass index, and history of diabetes.

The analysis revealed that, in women and men combined, there was no association between NHL risk and the consumption of 1 or more servings (355 mL) of artificially sweetened beverages. Compared to nondrinkers, subjects who drank artificially sweetened beverages had a risk ratio (RR) of 0.92 (P=0.14).

Similarly, there was no association between NHL risk and sugar-sweetened beverages. Compared to nondrinkers, the RR for sugar-sweetened beverage drinkers was 1.10 (P=0.62).

Furthermore, subjects’ overall aspartame intake, which was estimated from artificially sweetened carbonated beverage consumption and the use of aspartame packets, was not associated with NHL risk.

The RR was 1.02 (P=0.69) for the top quintile (which had a median aspartame intake of 145 mg per day) vs the bottom quintile (which had a median aspartame intake of 0 mg per day).

The investigators also found that associations between disease and sweetened beverage consumption or aspartame intake were generally null for specific NHL subtypes, including multiple myeloma, diffuse large B-cell lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma, follicular lymphoma, and other B-cell lymphomas.

“The study supports the decades of research that have continued to find that aspartame is safe for use in foods and beverages,” said Haley Stevens, PhD, President of the Calorie Control Council. “It also supports the conclusions of the National Cancer Institute, who have determined that aspartame does not increase a person’s risk of developing cancer.”

 

 

Sodas and other sweetened

beverages at the supermarket

 

Consuming aspartame and drinking sweetened beverages do not increase a person’s risk of developing non-Hodgkin lymphoma (NHL), according to research published in the Journal of Nutrition.

Investigators analyzed information from more than 100,000 men and women in the US.

The results suggested that neither aspartame intake nor the consumption of sugar-sweetened or artificially sweetened beverages were associated with an increased risk of NHL.

Marjorie L. McCullough, SCD, RD, of the American Cancer Society in Atlanta, Georgia, and her colleagues conducted this research, analyzing data from the nutrition cohort of the Cancer Prevention Study II, an assessment of cancer incidence

and mortality in the US.

Study subjects first completed a questionnaire in 1992, noting information related to diet and other lifestyle factors. They completed follow-up questionnaires in 1999 and 2003, which included questions related to the consumption of sugar-sweetened and artificially sweetened beverages, as well as tabletop sweeteners containing aspartame.

Among the 100,442 adult men and women who provided information on diet and lifestyle factors in 1999, there were 1196 NHL cases verified during a 10-year follow-up period.

The investigators assessed the risk of NHL associated with sweetened beverage and aspartame consumption, adjusted for the subjects’ smoking status, body mass index, and history of diabetes.

The analysis revealed that, in women and men combined, there was no association between NHL risk and the consumption of 1 or more servings (355 mL) of artificially sweetened beverages. Compared to nondrinkers, subjects who drank artificially sweetened beverages had a risk ratio (RR) of 0.92 (P=0.14).

Similarly, there was no association between NHL risk and sugar-sweetened beverages. Compared to nondrinkers, the RR for sugar-sweetened beverage drinkers was 1.10 (P=0.62).

Furthermore, subjects’ overall aspartame intake, which was estimated from artificially sweetened carbonated beverage consumption and the use of aspartame packets, was not associated with NHL risk.

The RR was 1.02 (P=0.69) for the top quintile (which had a median aspartame intake of 145 mg per day) vs the bottom quintile (which had a median aspartame intake of 0 mg per day).

The investigators also found that associations between disease and sweetened beverage consumption or aspartame intake were generally null for specific NHL subtypes, including multiple myeloma, diffuse large B-cell lymphoma, chronic lymphocytic leukemia/small lymphocytic lymphoma, follicular lymphoma, and other B-cell lymphomas.

“The study supports the decades of research that have continued to find that aspartame is safe for use in foods and beverages,” said Haley Stevens, PhD, President of the Calorie Control Council. “It also supports the conclusions of the National Cancer Institute, who have determined that aspartame does not increase a person’s risk of developing cancer.”

Monoclonal antibodies

Credit: Linda Bartlett

The UK’s National Institute for Health and Care Excellence (NICE) has issued a preliminary draft guidance recommending ofatumumab (Arzerra) for use in patients with chronic lymphocytic leukemia (CLL).

The agency is recommending the anti-CD20 monoclonal antibody in combination with chlorambucil for patients with untreated CLL who are ineligible for treatment with fludarabine combination therapy and for whom bendamustine is unsuitable.

NICE believes ofatumumab is a cost-effective use of National Health Service (NHS) resources for this patient population, as GlaxoSmithKline, the company developing ofatumumab, has agreed to provide the drug at a reduced price.

The company has agreed with the Department of Health that the size of the discount be confidential.

Clinical effectiveness

“The information provided by GlaxoSmithKline, who market the drug, showed that ofatumumab with chlorambucil is a clinically effective treatment option for those people unable to take fludarabine combination therapy or bendamustine,” said Sir Andrew Dillon, NICE chief executive.

In the phase 3 COMPLEMENT 1 trial, ofatumumab plus chlorambucil improved progression-free survival compared to chlorambucil alone. The median times were 22.4 months and 13.1 months, respectively, and the hazard ratio was 0.57 (P<0.001).

A NICE advisory committee also considered the use of ofatumumab in combination with bendamustine. But the committee said that, due to limited clinical evidence and the absence of cost-effectiveness estimates, it could not make a recommendation on this combination.

In a phase 2 trial known as OMB115991, ofatumumab plus bendamustine elicited an overall response rate of 95% and a complete response rate of 43%.

Cost-effectiveness

Ofatumumab’s list price is £182 for a 100 mg vial and £1820 for a 1000 mg vial. Assuming 6 cycles and no drug wastage, the mean cost of a treatment course for ofatumumab at its list price is £11,466 for 6300 mg.

GlaxoSmithKline has agreed to a patient access scheme with the Department of Health that makes ofatumumab available with a discount on the list price, though the exact amount is confidential.

The NICE advisory committee said the most plausible cost-effectiveness estimate for ofatumumab plus chlorambucil compared with chlorambucil alone using the

ofatumumab patient access scheme price was £26,000 per quality-adjusted life year gained.

Consultees, including GlaxoSmithKline, healthcare professionals, and members of the public, can now comment on NICE’s preliminary draft guidance. It will be available for public consultation until November 25, 2014.

Until the final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations.

Monoclonal antibodies

Credit: Linda Bartlett

The UK’s National Institute for Health and Care Excellence (NICE) has issued a preliminary draft guidance recommending ofatumumab (Arzerra) for use in patients with chronic lymphocytic leukemia (CLL).

The agency is recommending the anti-CD20 monoclonal antibody in combination with chlorambucil for patients with untreated CLL who are ineligible for treatment with fludarabine combination therapy and for whom bendamustine is unsuitable.

NICE believes ofatumumab is a cost-effective use of National Health Service (NHS) resources for this patient population, as GlaxoSmithKline, the company developing ofatumumab, has agreed to provide the drug at a reduced price.

The company has agreed with the Department of Health that the size of the discount be confidential.

Clinical effectiveness

“The information provided by GlaxoSmithKline, who market the drug, showed that ofatumumab with chlorambucil is a clinically effective treatment option for those people unable to take fludarabine combination therapy or bendamustine,” said Sir Andrew Dillon, NICE chief executive.

In the phase 3 COMPLEMENT 1 trial, ofatumumab plus chlorambucil improved progression-free survival compared to chlorambucil alone. The median times were 22.4 months and 13.1 months, respectively, and the hazard ratio was 0.57 (P<0.001).

A NICE advisory committee also considered the use of ofatumumab in combination with bendamustine. But the committee said that, due to limited clinical evidence and the absence of cost-effectiveness estimates, it could not make a recommendation on this combination.

In a phase 2 trial known as OMB115991, ofatumumab plus bendamustine elicited an overall response rate of 95% and a complete response rate of 43%.

Cost-effectiveness

Ofatumumab’s list price is £182 for a 100 mg vial and £1820 for a 1000 mg vial. Assuming 6 cycles and no drug wastage, the mean cost of a treatment course for ofatumumab at its list price is £11,466 for 6300 mg.

GlaxoSmithKline has agreed to a patient access scheme with the Department of Health that makes ofatumumab available with a discount on the list price, though the exact amount is confidential.

The NICE advisory committee said the most plausible cost-effectiveness estimate for ofatumumab plus chlorambucil compared with chlorambucil alone using the

ofatumumab patient access scheme price was £26,000 per quality-adjusted life year gained.

Consultees, including GlaxoSmithKline, healthcare professionals, and members of the public, can now comment on NICE’s preliminary draft guidance. It will be available for public consultation until November 25, 2014.

Until the final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations.

Monoclonal antibodies

Credit: Linda Bartlett

The UK’s National Institute for Health and Care Excellence (NICE) has issued a preliminary draft guidance recommending ofatumumab (Arzerra) for use in patients with chronic lymphocytic leukemia (CLL).

The agency is recommending the anti-CD20 monoclonal antibody in combination with chlorambucil for patients with untreated CLL who are ineligible for treatment with fludarabine combination therapy and for whom bendamustine is unsuitable.

NICE believes ofatumumab is a cost-effective use of National Health Service (NHS) resources for this patient population, as GlaxoSmithKline, the company developing ofatumumab, has agreed to provide the drug at a reduced price.

The company has agreed with the Department of Health that the size of the discount be confidential.

Clinical effectiveness

“The information provided by GlaxoSmithKline, who market the drug, showed that ofatumumab with chlorambucil is a clinically effective treatment option for those people unable to take fludarabine combination therapy or bendamustine,” said Sir Andrew Dillon, NICE chief executive.

In the phase 3 COMPLEMENT 1 trial, ofatumumab plus chlorambucil improved progression-free survival compared to chlorambucil alone. The median times were 22.4 months and 13.1 months, respectively, and the hazard ratio was 0.57 (P<0.001).

A NICE advisory committee also considered the use of ofatumumab in combination with bendamustine. But the committee said that, due to limited clinical evidence and the absence of cost-effectiveness estimates, it could not make a recommendation on this combination.

In a phase 2 trial known as OMB115991, ofatumumab plus bendamustine elicited an overall response rate of 95% and a complete response rate of 43%.

Cost-effectiveness

Ofatumumab’s list price is £182 for a 100 mg vial and £1820 for a 1000 mg vial. Assuming 6 cycles and no drug wastage, the mean cost of a treatment course for ofatumumab at its list price is £11,466 for 6300 mg.

GlaxoSmithKline has agreed to a patient access scheme with the Department of Health that makes ofatumumab available with a discount on the list price, though the exact amount is confidential.

The NICE advisory committee said the most plausible cost-effectiveness estimate for ofatumumab plus chlorambucil compared with chlorambucil alone using the

ofatumumab patient access scheme price was £26,000 per quality-adjusted life year gained.

Consultees, including GlaxoSmithKline, healthcare professionals, and members of the public, can now comment on NICE’s preliminary draft guidance. It will be available for public consultation until November 25, 2014.

Until the final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations.