Multiple Myeloma

Peripheral blood is a major stem cell source for hematopoietic stem cell transplantation in many parts of the world – even though bone marrow is preferred – likely due to costs, the need for patient hospitalization, and a lack of trained physicians and necessary equipment for performing bone marrow transplant, Dr. Ayami Yoshimi, and colleagues reported for the Worldwide Network of Blood and Marrow Transplantation.

Peripheral blood stem cells are readily available at transplant centers, as they are collected routinely for other indications, and they offer short-term cost savings due to rapid engraftment, the network reported in a research letter (JAMA. 2016;315[2]:198-200. doi:10.1001/jama.2015.13706).

In patients with nonmalignant disorders, use of peripheral blood stem cells in HSCT has a higher rate of graft-vs.-host disease and lower survival rates.

In the network’s retrospective survey, which they estimate covered 90% of transplants performed in 2009 and 2010, 114,217 HSCTs were reported by 1,482 transplant teams and 3,282 allogeneic HSCTs were performed for bone marrow failure. Use of peripheral blood stem cells in HSCT varied from 80% in countries with low and middle incomes to 50% in those with high-middle incomes to 36% in those with high incomes (P less than .001). For the 3,282 allogeneic HSCTs performed for bone marrow failure worldwide, stem cell sources included bone marrow (1,766, 54%), peripheral blood stem cells (1,336, 41%), and cord blood (180, 5%).

Use of unrelated donors was highest in Europe (515/1107; 47%); use of matched sibling donors was highest in the Eastern Mediterranean region and Africa (249/274; 91%).

Bone marrow was used most commonly in the Americas (631/843; 75%) and in Europe (632/1057; 60%), but not in the Eastern Mediterranean region and Africa (123/266; 46%) and in the Asia Pacific region (380/936; 41%; excluding Japan, 19%).

“National and international transplant organizations and authorities should foster regional accredited bone marrow harvest centers for patients with nonmalignant disorders and provide resources to establish such infrastructures. Unrelated donor registries should provide information on the necessity of bone marrow donation for patients with bone marrow failure,” wrote Dr. Yoshimi of the University of Freiburg, Germany, and colleagues.

Funding for the study was indirectly provided by the Worldwide Network of Blood and Marrow Transplantation. Dr. Yoshimi reported having no disclosures.

Peripheral blood is a major stem cell source for hematopoietic stem cell transplantation in many parts of the world – even though bone marrow is preferred – likely due to costs, the need for patient hospitalization, and a lack of trained physicians and necessary equipment for performing bone marrow transplant, Dr. Ayami Yoshimi, and colleagues reported for the Worldwide Network of Blood and Marrow Transplantation.

Peripheral blood stem cells are readily available at transplant centers, as they are collected routinely for other indications, and they offer short-term cost savings due to rapid engraftment, the network reported in a research letter (JAMA. 2016;315[2]:198-200. doi:10.1001/jama.2015.13706).

In patients with nonmalignant disorders, use of peripheral blood stem cells in HSCT has a higher rate of graft-vs.-host disease and lower survival rates.

In the network’s retrospective survey, which they estimate covered 90% of transplants performed in 2009 and 2010, 114,217 HSCTs were reported by 1,482 transplant teams and 3,282 allogeneic HSCTs were performed for bone marrow failure. Use of peripheral blood stem cells in HSCT varied from 80% in countries with low and middle incomes to 50% in those with high-middle incomes to 36% in those with high incomes (P less than .001). For the 3,282 allogeneic HSCTs performed for bone marrow failure worldwide, stem cell sources included bone marrow (1,766, 54%), peripheral blood stem cells (1,336, 41%), and cord blood (180, 5%).

Use of unrelated donors was highest in Europe (515/1107; 47%); use of matched sibling donors was highest in the Eastern Mediterranean region and Africa (249/274; 91%).

Bone marrow was used most commonly in the Americas (631/843; 75%) and in Europe (632/1057; 60%), but not in the Eastern Mediterranean region and Africa (123/266; 46%) and in the Asia Pacific region (380/936; 41%; excluding Japan, 19%).

“National and international transplant organizations and authorities should foster regional accredited bone marrow harvest centers for patients with nonmalignant disorders and provide resources to establish such infrastructures. Unrelated donor registries should provide information on the necessity of bone marrow donation for patients with bone marrow failure,” wrote Dr. Yoshimi of the University of Freiburg, Germany, and colleagues.

Funding for the study was indirectly provided by the Worldwide Network of Blood and Marrow Transplantation. Dr. Yoshimi reported having no disclosures.

Peripheral blood is a major stem cell source for hematopoietic stem cell transplantation in many parts of the world – even though bone marrow is preferred – likely due to costs, the need for patient hospitalization, and a lack of trained physicians and necessary equipment for performing bone marrow transplant, Dr. Ayami Yoshimi, and colleagues reported for the Worldwide Network of Blood and Marrow Transplantation.

Peripheral blood stem cells are readily available at transplant centers, as they are collected routinely for other indications, and they offer short-term cost savings due to rapid engraftment, the network reported in a research letter (JAMA. 2016;315[2]:198-200. doi:10.1001/jama.2015.13706).

In patients with nonmalignant disorders, use of peripheral blood stem cells in HSCT has a higher rate of graft-vs.-host disease and lower survival rates.

In the network’s retrospective survey, which they estimate covered 90% of transplants performed in 2009 and 2010, 114,217 HSCTs were reported by 1,482 transplant teams and 3,282 allogeneic HSCTs were performed for bone marrow failure. Use of peripheral blood stem cells in HSCT varied from 80% in countries with low and middle incomes to 50% in those with high-middle incomes to 36% in those with high incomes (P less than .001). For the 3,282 allogeneic HSCTs performed for bone marrow failure worldwide, stem cell sources included bone marrow (1,766, 54%), peripheral blood stem cells (1,336, 41%), and cord blood (180, 5%).

Use of unrelated donors was highest in Europe (515/1107; 47%); use of matched sibling donors was highest in the Eastern Mediterranean region and Africa (249/274; 91%).

Bone marrow was used most commonly in the Americas (631/843; 75%) and in Europe (632/1057; 60%), but not in the Eastern Mediterranean region and Africa (123/266; 46%) and in the Asia Pacific region (380/936; 41%; excluding Japan, 19%).

“National and international transplant organizations and authorities should foster regional accredited bone marrow harvest centers for patients with nonmalignant disorders and provide resources to establish such infrastructures. Unrelated donor registries should provide information on the necessity of bone marrow donation for patients with bone marrow failure,” wrote Dr. Yoshimi of the University of Freiburg, Germany, and colleagues.

Funding for the study was indirectly provided by the Worldwide Network of Blood and Marrow Transplantation. Dr. Yoshimi reported having no disclosures.

HSCT preparation

Photo by Chad McNeeley

Tandem autologous/allogeneic hematopoietic stem cell transplant (HSCT) may cure multiple myeloma (MM), according to a phase 2 study.

The study included newly diagnosed patients who received induction therapy followed by an autograft and a non-myeloablative allograft from a matched sibling donor.

The patients have been followed for a median of 8.8 years.

The probability of progression-free survival at 10 years is 41%, and the overall survival is 62%.

The rate of chronic graft-vs-host disease (GVHD) is high, but the rate of non-relapse mortality is low.

“In many hospitals, doctors have abandoned the use of allografts for multiple myeloma due to the risk of toxicity and relapse,” said study author Jean Roy, MD, of the Maisonneuve-Rosemont Hospital and University of Montreal in Quebec, Canada.

“Our results, on the other hand, have led us to offer the treatment to more patients, especially younger patients and those with poorer prognoses.”

Dr Roy and his colleagues reported these results in Bone Marrow Transplantation.

The researchers assessed 92 patients newly diagnosed with MM between 2001 and 2010. Their median age was 52 (range, 39-64), and 97% had Durie–Salmon stage II or III disease.

Patients received an induction regimen consisting of vincristine, doxorubicin, and dexamethasone (2001–2007, n=75) or a bortezomib-based regimen (2008–2010, n=17).

After induction, patients underwent autologous HSCT using melphalan 200 mg/m².

A median of 4 months later (range, 2-13), after complete clinical recovery, the patients received an allogeneic transplant from a 6/6 HLA-matched sibling donor. Fifty-seven percent of patients had achieved at least a very good partial response before the second transplant.

The allogeneic transplant was performed on an outpatient basis. The graft consisted of G-CSF-mobilized peripheral blood stem cells (target dose ≥ 4×10⁶ CD34⁺cells/kg).

The conditioning regimen consisted of fludarabine at 30 mg/m² and cyclophosphamide at 300 mg/m² for 5 days. GVHD prophylaxis was oral tacrolimus and mycophenolate mofetil.

Results

At a median follow-up of 8.8 years, 56 patients were still alive (and 36 had died).

Forty patients had no evidence of progression, including 11 patients who were still taking systemic immunosuppressive drugs for GVHD. The remaining 16 patients who were still alive had relapsed after allogeneic HSCT.

A total of 45 patients relapsed. Thirty-nine went on to receive treatment with at least 1 new drug (thalidomide, lenalidomide, bortezomib, or pomalidomide).

They received a median of 2 lines of therapy (range, 1–6), and their 5-year overall survival from first relapse was 49%. Ten of these patients were in very good partial response or greater at last follow-up.

Ten patients died from causes other than refractory MM—6 from refractory GVHD, 3 from lung adenocarcinoma (1 smoker), and 1 from invasive aspergillosis.

The cumulative incidence of extensive chronic GVHD was 79%. The cumulative incidence of grade 2-4 acute GVHD at 6 months was 9%. And 3 patients developed grade 3-4 acute GVHD.

Among the 56 patients who were still alive at last follow-up, the probability of being on any systemic immunosuppressive treatment for GVHD is 38% at 5 years and 22% at 10 years.

The probability of overall survival at 10 years is 62%, the probability of progression-free survival is 41%, and the cumulative incidence of non-relapse mortality is 10%.

HSCT preparation

Photo by Chad McNeeley

Tandem autologous/allogeneic hematopoietic stem cell transplant (HSCT) may cure multiple myeloma (MM), according to a phase 2 study.

The study included newly diagnosed patients who received induction therapy followed by an autograft and a non-myeloablative allograft from a matched sibling donor.

The patients have been followed for a median of 8.8 years.

The probability of progression-free survival at 10 years is 41%, and the overall survival is 62%.

The rate of chronic graft-vs-host disease (GVHD) is high, but the rate of non-relapse mortality is low.

“In many hospitals, doctors have abandoned the use of allografts for multiple myeloma due to the risk of toxicity and relapse,” said study author Jean Roy, MD, of the Maisonneuve-Rosemont Hospital and University of Montreal in Quebec, Canada.

“Our results, on the other hand, have led us to offer the treatment to more patients, especially younger patients and those with poorer prognoses.”

Dr Roy and his colleagues reported these results in Bone Marrow Transplantation.

The researchers assessed 92 patients newly diagnosed with MM between 2001 and 2010. Their median age was 52 (range, 39-64), and 97% had Durie–Salmon stage II or III disease.

Patients received an induction regimen consisting of vincristine, doxorubicin, and dexamethasone (2001–2007, n=75) or a bortezomib-based regimen (2008–2010, n=17).

After induction, patients underwent autologous HSCT using melphalan 200 mg/m².

A median of 4 months later (range, 2-13), after complete clinical recovery, the patients received an allogeneic transplant from a 6/6 HLA-matched sibling donor. Fifty-seven percent of patients had achieved at least a very good partial response before the second transplant.

The allogeneic transplant was performed on an outpatient basis. The graft consisted of G-CSF-mobilized peripheral blood stem cells (target dose ≥ 4×10⁶ CD34⁺cells/kg).

The conditioning regimen consisted of fludarabine at 30 mg/m² and cyclophosphamide at 300 mg/m² for 5 days. GVHD prophylaxis was oral tacrolimus and mycophenolate mofetil.

Results

At a median follow-up of 8.8 years, 56 patients were still alive (and 36 had died).

Forty patients had no evidence of progression, including 11 patients who were still taking systemic immunosuppressive drugs for GVHD. The remaining 16 patients who were still alive had relapsed after allogeneic HSCT.

A total of 45 patients relapsed. Thirty-nine went on to receive treatment with at least 1 new drug (thalidomide, lenalidomide, bortezomib, or pomalidomide).

They received a median of 2 lines of therapy (range, 1–6), and their 5-year overall survival from first relapse was 49%. Ten of these patients were in very good partial response or greater at last follow-up.

Ten patients died from causes other than refractory MM—6 from refractory GVHD, 3 from lung adenocarcinoma (1 smoker), and 1 from invasive aspergillosis.

The cumulative incidence of extensive chronic GVHD was 79%. The cumulative incidence of grade 2-4 acute GVHD at 6 months was 9%. And 3 patients developed grade 3-4 acute GVHD.

Among the 56 patients who were still alive at last follow-up, the probability of being on any systemic immunosuppressive treatment for GVHD is 38% at 5 years and 22% at 10 years.

The probability of overall survival at 10 years is 62%, the probability of progression-free survival is 41%, and the cumulative incidence of non-relapse mortality is 10%.

HSCT preparation

Photo by Chad McNeeley

Tandem autologous/allogeneic hematopoietic stem cell transplant (HSCT) may cure multiple myeloma (MM), according to a phase 2 study.

The study included newly diagnosed patients who received induction therapy followed by an autograft and a non-myeloablative allograft from a matched sibling donor.

The patients have been followed for a median of 8.8 years.

The probability of progression-free survival at 10 years is 41%, and the overall survival is 62%.

The rate of chronic graft-vs-host disease (GVHD) is high, but the rate of non-relapse mortality is low.

“In many hospitals, doctors have abandoned the use of allografts for multiple myeloma due to the risk of toxicity and relapse,” said study author Jean Roy, MD, of the Maisonneuve-Rosemont Hospital and University of Montreal in Quebec, Canada.

“Our results, on the other hand, have led us to offer the treatment to more patients, especially younger patients and those with poorer prognoses.”

Dr Roy and his colleagues reported these results in Bone Marrow Transplantation.

The researchers assessed 92 patients newly diagnosed with MM between 2001 and 2010. Their median age was 52 (range, 39-64), and 97% had Durie–Salmon stage II or III disease.

Patients received an induction regimen consisting of vincristine, doxorubicin, and dexamethasone (2001–2007, n=75) or a bortezomib-based regimen (2008–2010, n=17).

After induction, patients underwent autologous HSCT using melphalan 200 mg/m².

A median of 4 months later (range, 2-13), after complete clinical recovery, the patients received an allogeneic transplant from a 6/6 HLA-matched sibling donor. Fifty-seven percent of patients had achieved at least a very good partial response before the second transplant.

The allogeneic transplant was performed on an outpatient basis. The graft consisted of G-CSF-mobilized peripheral blood stem cells (target dose ≥ 4×10⁶ CD34⁺cells/kg).

The conditioning regimen consisted of fludarabine at 30 mg/m² and cyclophosphamide at 300 mg/m² for 5 days. GVHD prophylaxis was oral tacrolimus and mycophenolate mofetil.

Results

At a median follow-up of 8.8 years, 56 patients were still alive (and 36 had died).

Forty patients had no evidence of progression, including 11 patients who were still taking systemic immunosuppressive drugs for GVHD. The remaining 16 patients who were still alive had relapsed after allogeneic HSCT.

A total of 45 patients relapsed. Thirty-nine went on to receive treatment with at least 1 new drug (thalidomide, lenalidomide, bortezomib, or pomalidomide).

They received a median of 2 lines of therapy (range, 1–6), and their 5-year overall survival from first relapse was 49%. Ten of these patients were in very good partial response or greater at last follow-up.

Ten patients died from causes other than refractory MM—6 from refractory GVHD, 3 from lung adenocarcinoma (1 smoker), and 1 from invasive aspergillosis.

The cumulative incidence of extensive chronic GVHD was 79%. The cumulative incidence of grade 2-4 acute GVHD at 6 months was 9%. And 3 patients developed grade 3-4 acute GVHD.

Among the 56 patients who were still alive at last follow-up, the probability of being on any systemic immunosuppressive treatment for GVHD is 38% at 5 years and 22% at 10 years.

The probability of overall survival at 10 years is 62%, the probability of progression-free survival is 41%, and the cumulative incidence of non-relapse mortality is 10%.

Patient receiving chemotherapy

Photo by Rhoda Baer

The US Food and Drug Administration (FDA) has approved a supplemental new drug application for single-dose fosaprepitant dimeglumine (Emend) for injection.

The agency approved the substance P/neurokinin-1 (NK1) receptor antagonist for use in combination with other anti-emetic medicines to prevent delayed nausea and vomiting in adults receiving initial and repeat courses of moderately emetogenic chemotherapy (MEC).

This makes fosaprepitant dimeglumine the first intravenous NK1 receptor antagonist approved in the US for patients receiving either highly emetogenic chemotherapy or MEC.

Fosaprepitant dimeglumine has not been studied for the treatment of established nausea and vomiting.

The FDA’s latest approval of fosaprepitant dimeglumine is supported by data from a phase 3 study published in the Annals of Oncology.

Patients receiving MEC were given ondansetron and dexamethasone (n=498) or ondansetron and dexamethasone plus a single intravenous infusion of fosaprepitant dimeglumine (n=502).

The primary endpoint was complete response (CR)—defined as no vomiting and no use of rescue therapy—in the delayed phase of chemotherapy-induced nausea and vomiting, which is 25 to 120 hours after the initiation of chemotherapy.

Secondary endpoints included CR in the overall and acute phases—0 to 120 and 0 to 24 hours after MEC initiation, respectively—and no vomiting in the overall phase.

The fosaprepitant regimen improved CR significantly in the delayed and overall phases but not in the acute phase.

In the delayed phase, the CR rate was 78.9% with the fosaprepitant regimen and 68.5% with the control regimen (P<0.001). In the acute phase, the CR rate was 93.2% and 91.0%, respectively (P=0.184). Overall, the CR rate was 77.1% and 66.9%, respectively (P<0.001).

In the overall phase, the proportion of subjects with no vomiting was 82.7% with the fosaprepitant regimen and 72.9% with the control regimen (P<0.001). The proportion of patients with no significant nausea was 83.2% and 77.9%, respectively (P=0.030).

The most common adverse events reported in the fosaprepitant and control arms, respectively, were fatigue (15% vs 13%), diarrhea (13% vs 11%), neutropenia (8% vs 7%), asthenia (4% vs 3%), anemia (3% vs 2%), peripheral neuropathy (3% vs 2%), leukopenia (2% vs 1%), dyspepsia (2% vs 1%), urinary tract infection (2% vs 1%), and pain in extremity (2% vs 1%).

Fosaprepitant dimeglumine is a product of Merck. For more details on the drug, see the prescribing information.

Patient receiving chemotherapy

Photo by Rhoda Baer

The US Food and Drug Administration (FDA) has approved a supplemental new drug application for single-dose fosaprepitant dimeglumine (Emend) for injection.

The agency approved the substance P/neurokinin-1 (NK1) receptor antagonist for use in combination with other anti-emetic medicines to prevent delayed nausea and vomiting in adults receiving initial and repeat courses of moderately emetogenic chemotherapy (MEC).

This makes fosaprepitant dimeglumine the first intravenous NK1 receptor antagonist approved in the US for patients receiving either highly emetogenic chemotherapy or MEC.

Fosaprepitant dimeglumine has not been studied for the treatment of established nausea and vomiting.

The FDA’s latest approval of fosaprepitant dimeglumine is supported by data from a phase 3 study published in the Annals of Oncology.

Patients receiving MEC were given ondansetron and dexamethasone (n=498) or ondansetron and dexamethasone plus a single intravenous infusion of fosaprepitant dimeglumine (n=502).

The primary endpoint was complete response (CR)—defined as no vomiting and no use of rescue therapy—in the delayed phase of chemotherapy-induced nausea and vomiting, which is 25 to 120 hours after the initiation of chemotherapy.

Secondary endpoints included CR in the overall and acute phases—0 to 120 and 0 to 24 hours after MEC initiation, respectively—and no vomiting in the overall phase.

The fosaprepitant regimen improved CR significantly in the delayed and overall phases but not in the acute phase.

In the delayed phase, the CR rate was 78.9% with the fosaprepitant regimen and 68.5% with the control regimen (P<0.001). In the acute phase, the CR rate was 93.2% and 91.0%, respectively (P=0.184). Overall, the CR rate was 77.1% and 66.9%, respectively (P<0.001).

In the overall phase, the proportion of subjects with no vomiting was 82.7% with the fosaprepitant regimen and 72.9% with the control regimen (P<0.001). The proportion of patients with no significant nausea was 83.2% and 77.9%, respectively (P=0.030).

The most common adverse events reported in the fosaprepitant and control arms, respectively, were fatigue (15% vs 13%), diarrhea (13% vs 11%), neutropenia (8% vs 7%), asthenia (4% vs 3%), anemia (3% vs 2%), peripheral neuropathy (3% vs 2%), leukopenia (2% vs 1%), dyspepsia (2% vs 1%), urinary tract infection (2% vs 1%), and pain in extremity (2% vs 1%).

Fosaprepitant dimeglumine is a product of Merck. For more details on the drug, see the prescribing information.

Patient receiving chemotherapy

Photo by Rhoda Baer

The US Food and Drug Administration (FDA) has approved a supplemental new drug application for single-dose fosaprepitant dimeglumine (Emend) for injection.

The agency approved the substance P/neurokinin-1 (NK1) receptor antagonist for use in combination with other anti-emetic medicines to prevent delayed nausea and vomiting in adults receiving initial and repeat courses of moderately emetogenic chemotherapy (MEC).

This makes fosaprepitant dimeglumine the first intravenous NK1 receptor antagonist approved in the US for patients receiving either highly emetogenic chemotherapy or MEC.

Fosaprepitant dimeglumine has not been studied for the treatment of established nausea and vomiting.

The FDA’s latest approval of fosaprepitant dimeglumine is supported by data from a phase 3 study published in the Annals of Oncology.

Patients receiving MEC were given ondansetron and dexamethasone (n=498) or ondansetron and dexamethasone plus a single intravenous infusion of fosaprepitant dimeglumine (n=502).

The primary endpoint was complete response (CR)—defined as no vomiting and no use of rescue therapy—in the delayed phase of chemotherapy-induced nausea and vomiting, which is 25 to 120 hours after the initiation of chemotherapy.

Secondary endpoints included CR in the overall and acute phases—0 to 120 and 0 to 24 hours after MEC initiation, respectively—and no vomiting in the overall phase.

The fosaprepitant regimen improved CR significantly in the delayed and overall phases but not in the acute phase.

In the delayed phase, the CR rate was 78.9% with the fosaprepitant regimen and 68.5% with the control regimen (P<0.001). In the acute phase, the CR rate was 93.2% and 91.0%, respectively (P=0.184). Overall, the CR rate was 77.1% and 66.9%, respectively (P<0.001).

In the overall phase, the proportion of subjects with no vomiting was 82.7% with the fosaprepitant regimen and 72.9% with the control regimen (P<0.001). The proportion of patients with no significant nausea was 83.2% and 77.9%, respectively (P=0.030).

The most common adverse events reported in the fosaprepitant and control arms, respectively, were fatigue (15% vs 13%), diarrhea (13% vs 11%), neutropenia (8% vs 7%), asthenia (4% vs 3%), anemia (3% vs 2%), peripheral neuropathy (3% vs 2%), leukopenia (2% vs 1%), dyspepsia (2% vs 1%), urinary tract infection (2% vs 1%), and pain in extremity (2% vs 1%).

Fosaprepitant dimeglumine is a product of Merck. For more details on the drug, see the prescribing information.

Researcher in the lab

Photo by Daniel Sone

The European Hematology Association (EHA) has created a “roadmap” for hematology research in Europe.

This guidance document summarizes the current status of basic, translational, and clinical hematology research and identifies areas of unmet scientific and medical need in Europe.

It is intended to help European and national policy makers, funding agencies, charities, research institutes, and researchers make decisions on initiating, funding, or developing research.

The guidance, “The European Hematology Association Roadmap for European Hematology Research: A Consensus Document,” is published in this month’s issue of haematologica.

“For the first time, hematologists in Europe came together to develop a roadmap to guide hematology research in Europe” said Andreas Engert, MD, chair of the EHA Research Roadmap Task Force.

“Hematology in Europe has achieved a lot, but the discipline must focus and collaborate to be efficient and remain successful in improving patient outcomes. The roadmap does just that and will determine the research agenda in Europe in the coming years.”

Roughly 300 experts from more than 20 countries—including clinicians, basic researchers, and patients—contributed to the roadmap. Stakeholders such as national hematology societies, patient organizations, hematology trial groups, and other European organizations were consulted to comment on the final draft version.

The final roadmap has 9 sections: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation.

The roadmap lists priorities and needs in these areas, including the need for targeted therapies based on genomic profiling and chemical biology, the need to eradicate minimal residual disease, and the need for treatments that are better tolerated by elderly patients.

“Now’s the time for Europe to pay attention,” said Ulrich Jäger, MD, chair of the EHA European Affairs Committee.

“With an aging population, the slow recovery from the financial and Euro crises, costly medical breakthroughs and innovations—quite a few of which involve hematology researchers—Europe faces increased health expenditures while budgets are limited.”

“Policy makers are rightfully cautious when spending the taxpayers’ money. So it is our responsibility to provide the policy makers with the information and evidence they need to decide where their support impacts knowledge and health most efficiently, to the benefit of patients and society. The Research Roadmap delivers on that. Now, it is up to the policy makers in the EU to deliver too.”

Researcher in the lab

Photo by Daniel Sone

The European Hematology Association (EHA) has created a “roadmap” for hematology research in Europe.

This guidance document summarizes the current status of basic, translational, and clinical hematology research and identifies areas of unmet scientific and medical need in Europe.

It is intended to help European and national policy makers, funding agencies, charities, research institutes, and researchers make decisions on initiating, funding, or developing research.

The guidance, “The European Hematology Association Roadmap for European Hematology Research: A Consensus Document,” is published in this month’s issue of haematologica.

“For the first time, hematologists in Europe came together to develop a roadmap to guide hematology research in Europe” said Andreas Engert, MD, chair of the EHA Research Roadmap Task Force.

“Hematology in Europe has achieved a lot, but the discipline must focus and collaborate to be efficient and remain successful in improving patient outcomes. The roadmap does just that and will determine the research agenda in Europe in the coming years.”

Roughly 300 experts from more than 20 countries—including clinicians, basic researchers, and patients—contributed to the roadmap. Stakeholders such as national hematology societies, patient organizations, hematology trial groups, and other European organizations were consulted to comment on the final draft version.

The final roadmap has 9 sections: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation.

The roadmap lists priorities and needs in these areas, including the need for targeted therapies based on genomic profiling and chemical biology, the need to eradicate minimal residual disease, and the need for treatments that are better tolerated by elderly patients.

“Now’s the time for Europe to pay attention,” said Ulrich Jäger, MD, chair of the EHA European Affairs Committee.

“With an aging population, the slow recovery from the financial and Euro crises, costly medical breakthroughs and innovations—quite a few of which involve hematology researchers—Europe faces increased health expenditures while budgets are limited.”

“Policy makers are rightfully cautious when spending the taxpayers’ money. So it is our responsibility to provide the policy makers with the information and evidence they need to decide where their support impacts knowledge and health most efficiently, to the benefit of patients and society. The Research Roadmap delivers on that. Now, it is up to the policy makers in the EU to deliver too.”

Researcher in the lab

Photo by Daniel Sone

The European Hematology Association (EHA) has created a “roadmap” for hematology research in Europe.

This guidance document summarizes the current status of basic, translational, and clinical hematology research and identifies areas of unmet scientific and medical need in Europe.

It is intended to help European and national policy makers, funding agencies, charities, research institutes, and researchers make decisions on initiating, funding, or developing research.

The guidance, “The European Hematology Association Roadmap for European Hematology Research: A Consensus Document,” is published in this month’s issue of haematologica.

“For the first time, hematologists in Europe came together to develop a roadmap to guide hematology research in Europe” said Andreas Engert, MD, chair of the EHA Research Roadmap Task Force.

“Hematology in Europe has achieved a lot, but the discipline must focus and collaborate to be efficient and remain successful in improving patient outcomes. The roadmap does just that and will determine the research agenda in Europe in the coming years.”

Roughly 300 experts from more than 20 countries—including clinicians, basic researchers, and patients—contributed to the roadmap. Stakeholders such as national hematology societies, patient organizations, hematology trial groups, and other European organizations were consulted to comment on the final draft version.

The final roadmap has 9 sections: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation.

The roadmap lists priorities and needs in these areas, including the need for targeted therapies based on genomic profiling and chemical biology, the need to eradicate minimal residual disease, and the need for treatments that are better tolerated by elderly patients.

“Now’s the time for Europe to pay attention,” said Ulrich Jäger, MD, chair of the EHA European Affairs Committee.

“With an aging population, the slow recovery from the financial and Euro crises, costly medical breakthroughs and innovations—quite a few of which involve hematology researchers—Europe faces increased health expenditures while budgets are limited.”

“Policy makers are rightfully cautious when spending the taxpayers’ money. So it is our responsibility to provide the policy makers with the information and evidence they need to decide where their support impacts knowledge and health most efficiently, to the benefit of patients and society. The Research Roadmap delivers on that. Now, it is up to the policy makers in the EU to deliver too.”

Cancer patient receiving

chemotherapy

Photo by Rhoda Baer

Loved ones of cancer patients often turn to the Internet for further information about the disease, but they are less inclined to seek emotional support from social media forums, according to a study published in Computers, Informatics, Nursing.

It is fairly common for loved ones of cancer patients to develop depression or anxiety disorders as a result of the diagnosis, but there aren’t many studies focusing specifically on cancer patients’ caregivers and family members, said study author Carolyn Lauckner, PhD, of the University of Georgia in Athens.

“I think, sometimes, the loved ones and caregivers get forgotten about,” she said. “And that’s why I wanted to research this population to see if there are ways that we can better support these individuals.”

Dr Lauckner surveyed 191 people whose loved ones were diagnosed with cancer in the past year or who were currently acting as caregivers to someone with cancer. The motivation behind the research was personal for Dr Lauckner.

“I went through a period of time where I had 3 loved ones diagnosed within a short amount of time,” she said. “I had these experiences where I heard about the diagnosis and I would go online to look it up, and then I would immediately become terrified and freak out about all the stuff I read online.”

Like Dr Lauckner, more than 75% of the subjects she surveyed searched online for information on a loved one’s disease. Most looked for treatment options, prevention strategies and risk factors, and prognosis information.

“I was pleasantly surprised by the amount of people who said that they were looking for prevention information online and detection information,” Dr Lauckner said. “[T]hat shows that not only are they concerned for their loved one but they’re also concerned about how they themselves can avoid cancer, which, from a public health perspective, is great.”

Respondents were less inclined to view blogs or go online to hear about others’ cancer experiences. These kinds of sites were linked to negative emotions for participants, such as fear, sadness, and anger.

“A lot of people, especially in the cancer realm, they will use blogs or discussion posts to vent and to talk about the harsh realities of living with an illness,” Dr Lauckner said.

“And while I think that that is beneficial for both the person who is writing it and potentially for some people who want an idea of what to expect, when someone is dealing with the prospect of their loved one having to go through that experience, it can be extremely distressing.”

The most commonly visited websites were those of charitable organizations like the American Cancer Society, which were associated with positive emotions. Dr Lauckner said she found this information encouraging because it shows that participants were consulting reliable sources of information and not being swayed by personal accounts as much.

Dr Lauckner ultimately wants to build on the information gleaned in this study to determine the most effective use of social media and technology to distribute cancer prevention and risk reduction messages to the public.

Cancer patient receiving

chemotherapy

Photo by Rhoda Baer

Loved ones of cancer patients often turn to the Internet for further information about the disease, but they are less inclined to seek emotional support from social media forums, according to a study published in Computers, Informatics, Nursing.

It is fairly common for loved ones of cancer patients to develop depression or anxiety disorders as a result of the diagnosis, but there aren’t many studies focusing specifically on cancer patients’ caregivers and family members, said study author Carolyn Lauckner, PhD, of the University of Georgia in Athens.

“I think, sometimes, the loved ones and caregivers get forgotten about,” she said. “And that’s why I wanted to research this population to see if there are ways that we can better support these individuals.”

Dr Lauckner surveyed 191 people whose loved ones were diagnosed with cancer in the past year or who were currently acting as caregivers to someone with cancer. The motivation behind the research was personal for Dr Lauckner.

“I went through a period of time where I had 3 loved ones diagnosed within a short amount of time,” she said. “I had these experiences where I heard about the diagnosis and I would go online to look it up, and then I would immediately become terrified and freak out about all the stuff I read online.”

Like Dr Lauckner, more than 75% of the subjects she surveyed searched online for information on a loved one’s disease. Most looked for treatment options, prevention strategies and risk factors, and prognosis information.

“I was pleasantly surprised by the amount of people who said that they were looking for prevention information online and detection information,” Dr Lauckner said. “[T]hat shows that not only are they concerned for their loved one but they’re also concerned about how they themselves can avoid cancer, which, from a public health perspective, is great.”

Respondents were less inclined to view blogs or go online to hear about others’ cancer experiences. These kinds of sites were linked to negative emotions for participants, such as fear, sadness, and anger.

“A lot of people, especially in the cancer realm, they will use blogs or discussion posts to vent and to talk about the harsh realities of living with an illness,” Dr Lauckner said.

“And while I think that that is beneficial for both the person who is writing it and potentially for some people who want an idea of what to expect, when someone is dealing with the prospect of their loved one having to go through that experience, it can be extremely distressing.”

The most commonly visited websites were those of charitable organizations like the American Cancer Society, which were associated with positive emotions. Dr Lauckner said she found this information encouraging because it shows that participants were consulting reliable sources of information and not being swayed by personal accounts as much.

Dr Lauckner ultimately wants to build on the information gleaned in this study to determine the most effective use of social media and technology to distribute cancer prevention and risk reduction messages to the public.

Cancer patient receiving

chemotherapy

Photo by Rhoda Baer

Loved ones of cancer patients often turn to the Internet for further information about the disease, but they are less inclined to seek emotional support from social media forums, according to a study published in Computers, Informatics, Nursing.

It is fairly common for loved ones of cancer patients to develop depression or anxiety disorders as a result of the diagnosis, but there aren’t many studies focusing specifically on cancer patients’ caregivers and family members, said study author Carolyn Lauckner, PhD, of the University of Georgia in Athens.

“I think, sometimes, the loved ones and caregivers get forgotten about,” she said. “And that’s why I wanted to research this population to see if there are ways that we can better support these individuals.”

Dr Lauckner surveyed 191 people whose loved ones were diagnosed with cancer in the past year or who were currently acting as caregivers to someone with cancer. The motivation behind the research was personal for Dr Lauckner.

“I went through a period of time where I had 3 loved ones diagnosed within a short amount of time,” she said. “I had these experiences where I heard about the diagnosis and I would go online to look it up, and then I would immediately become terrified and freak out about all the stuff I read online.”

Like Dr Lauckner, more than 75% of the subjects she surveyed searched online for information on a loved one’s disease. Most looked for treatment options, prevention strategies and risk factors, and prognosis information.

“I was pleasantly surprised by the amount of people who said that they were looking for prevention information online and detection information,” Dr Lauckner said. “[T]hat shows that not only are they concerned for their loved one but they’re also concerned about how they themselves can avoid cancer, which, from a public health perspective, is great.”

Respondents were less inclined to view blogs or go online to hear about others’ cancer experiences. These kinds of sites were linked to negative emotions for participants, such as fear, sadness, and anger.

“A lot of people, especially in the cancer realm, they will use blogs or discussion posts to vent and to talk about the harsh realities of living with an illness,” Dr Lauckner said.

“And while I think that that is beneficial for both the person who is writing it and potentially for some people who want an idea of what to expect, when someone is dealing with the prospect of their loved one having to go through that experience, it can be extremely distressing.”

The most commonly visited websites were those of charitable organizations like the American Cancer Society, which were associated with positive emotions. Dr Lauckner said she found this information encouraging because it shows that participants were consulting reliable sources of information and not being swayed by personal accounts as much.

Dr Lauckner ultimately wants to build on the information gleaned in this study to determine the most effective use of social media and technology to distribute cancer prevention and risk reduction messages to the public.

Osteoclast culture

New research has revealed single-nucleotide polymorphisms (SNPs) that may increase the risk of bone disease in patients with multiple myeloma (MM).

One of these SNPs affects a gene encoding osteoprotegerin (OPG), a signaling protein that helps regulate the density of bones.

In patients with MM bone disease (MBD), bones are broken down faster than they can be repaired, resulting in fractures and lesions.

OPG is known to keep the numbers of osteoclasts in check, so it follows that mutations adversely affecting the gene may contribute to excessive bone breakdown.

Richard Houlston, MD, PhD, of The Institute of Cancer Research in London, UK, and his colleagues reported these findings in Leukemia.

The researchers analyzed the DNA of 3774 MM patients and used imaging techniques to detect which of these patients had MBD and which did not. The team used statistical analysis to compare sequencing data from the group of patients who developed MBD with those who did not.

The analysis revealed 9 SNPs that showed an association with MBD and reached genome-wide significance. They were all located in the same region at 8q24.12 and were in strong linkage disequilibrium (LD).

The strongest association at 8q24.12 was observed with the SNP rs4407910, which localizes 19Kb 3’ to the gene encoding TNFRSF11B, also known as OPG (odds ratio=1.38, P=4.02×10⁻⁹).

The researchers also found a “promising association” for MBD with the SNP rs74676832 at 19q13.43, which is located within a 29 kb region of LD intergenic to ZNF444 and GALP (odds ratio=1.97, P=9.33×10^–7).

The team said these findings demonstrate that germline variation influences MBD, and they highlight the importance of the RANK/RANKL/OPG pathway in MBD development.

Furthermore, the work could enable scientists to develop new strategies to prevent MBD development and identify which patients might benefit from treatment with existing bone therapies such as bisphosphonates.

“It is really important to understand why some patients with myeloma are severely affected by bone disease,” Dr Houlston said. “This study tells us that at least part of the answer is in the patient’s DNA, greatly improving our understanding of the disease.”

“We want to be able to identify which patients are most at risk and which treatments are most likely to help them. This study gives us clues that can begin to help us do this. In the longer term, understanding the factors that contributed to bone breakdown could help us find new treatments for the disease.”

Osteoclast culture

New research has revealed single-nucleotide polymorphisms (SNPs) that may increase the risk of bone disease in patients with multiple myeloma (MM).

One of these SNPs affects a gene encoding osteoprotegerin (OPG), a signaling protein that helps regulate the density of bones.

In patients with MM bone disease (MBD), bones are broken down faster than they can be repaired, resulting in fractures and lesions.

OPG is known to keep the numbers of osteoclasts in check, so it follows that mutations adversely affecting the gene may contribute to excessive bone breakdown.

Richard Houlston, MD, PhD, of The Institute of Cancer Research in London, UK, and his colleagues reported these findings in Leukemia.

The researchers analyzed the DNA of 3774 MM patients and used imaging techniques to detect which of these patients had MBD and which did not. The team used statistical analysis to compare sequencing data from the group of patients who developed MBD with those who did not.

The analysis revealed 9 SNPs that showed an association with MBD and reached genome-wide significance. They were all located in the same region at 8q24.12 and were in strong linkage disequilibrium (LD).

The strongest association at 8q24.12 was observed with the SNP rs4407910, which localizes 19Kb 3’ to the gene encoding TNFRSF11B, also known as OPG (odds ratio=1.38, P=4.02×10⁻⁹).

The researchers also found a “promising association” for MBD with the SNP rs74676832 at 19q13.43, which is located within a 29 kb region of LD intergenic to ZNF444 and GALP (odds ratio=1.97, P=9.33×10^–7).

The team said these findings demonstrate that germline variation influences MBD, and they highlight the importance of the RANK/RANKL/OPG pathway in MBD development.

Furthermore, the work could enable scientists to develop new strategies to prevent MBD development and identify which patients might benefit from treatment with existing bone therapies such as bisphosphonates.

“It is really important to understand why some patients with myeloma are severely affected by bone disease,” Dr Houlston said. “This study tells us that at least part of the answer is in the patient’s DNA, greatly improving our understanding of the disease.”

“We want to be able to identify which patients are most at risk and which treatments are most likely to help them. This study gives us clues that can begin to help us do this. In the longer term, understanding the factors that contributed to bone breakdown could help us find new treatments for the disease.”

Osteoclast culture

New research has revealed single-nucleotide polymorphisms (SNPs) that may increase the risk of bone disease in patients with multiple myeloma (MM).

One of these SNPs affects a gene encoding osteoprotegerin (OPG), a signaling protein that helps regulate the density of bones.

In patients with MM bone disease (MBD), bones are broken down faster than they can be repaired, resulting in fractures and lesions.

OPG is known to keep the numbers of osteoclasts in check, so it follows that mutations adversely affecting the gene may contribute to excessive bone breakdown.

Richard Houlston, MD, PhD, of The Institute of Cancer Research in London, UK, and his colleagues reported these findings in Leukemia.

The researchers analyzed the DNA of 3774 MM patients and used imaging techniques to detect which of these patients had MBD and which did not. The team used statistical analysis to compare sequencing data from the group of patients who developed MBD with those who did not.

The analysis revealed 9 SNPs that showed an association with MBD and reached genome-wide significance. They were all located in the same region at 8q24.12 and were in strong linkage disequilibrium (LD).

The strongest association at 8q24.12 was observed with the SNP rs4407910, which localizes 19Kb 3’ to the gene encoding TNFRSF11B, also known as OPG (odds ratio=1.38, P=4.02×10⁻⁹).

The researchers also found a “promising association” for MBD with the SNP rs74676832 at 19q13.43, which is located within a 29 kb region of LD intergenic to ZNF444 and GALP (odds ratio=1.97, P=9.33×10^–7).

The team said these findings demonstrate that germline variation influences MBD, and they highlight the importance of the RANK/RANKL/OPG pathway in MBD development.

Furthermore, the work could enable scientists to develop new strategies to prevent MBD development and identify which patients might benefit from treatment with existing bone therapies such as bisphosphonates.

“It is really important to understand why some patients with myeloma are severely affected by bone disease,” Dr Houlston said. “This study tells us that at least part of the answer is in the patient’s DNA, greatly improving our understanding of the disease.”

“We want to be able to identify which patients are most at risk and which treatments are most likely to help them. This study gives us clues that can begin to help us do this. In the longer term, understanding the factors that contributed to bone breakdown could help us find new treatments for the disease.”

Elotuzumab (Empliciti)

Photo courtesy of

Bristol-Myers Squibb

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of the immunostimulatory antibody elotuzumab (Empliciti).

The CHMP is recommending the drug be approved for use in combination with lenalidomide and dexamethasone to treat patients with multiple myeloma (MM) who have received at least 1 prior therapy.

The CHMP’s recommendation will be reviewed by the European Commission, which usually follows the CHMP’s advice and is expected to deliver its final decision on elotuzumab within 3 months.

The CHMP’s positive opinion of elotuzumab is based on data from the phase 3 ELOQUENT-2 trial, which were presented at ASCO 2015 and published in NEJM.

The trial included 646 MM patients who had received 1 to 3 prior therapies. Baseline patient demographics and disease characteristics were well balanced between treatment arms.

Patients were randomized 1:1 to receive either elotuzumab at 10 mg/kg in combination with lenalidomide and dexamethasone (len-dex) or len-dex alone in 4-week cycles until disease progression or unacceptable toxicity.

The minimum follow-up for all study subjects was 24 months. The co-primary endpoints were progression-free survival (PFS) and overall response rate.

The overall response rate was 78.5% in the elotuzumab arm and 65.5% in the len-dex arm (P=0.0002).

The median PFS was 19.4 months in the elotuzumab arm and 14.9 months in the len-dex arm (P=0.0004). At 1 year, the PFS was 68% in the elotuzumab arm and 57% in the len-dex arm. At 2 years, the PFS was 41% and 27%, respectively.

The most common adverse events in the elotuzumab arm and len-dex arm, respectively, were fatigue (61.6% vs 51.7%), diarrhea (46.9% vs 36.0%), pyrexia (37.4% vs 24.6%), constipation (35.5% vs 27.1%), cough (34.3% vs 18.9%), peripheral neuropathy (26.7% vs 20.8%), nasopharyngitis (24.5% vs 19.2%), upper respiratory tract infection (22.6% vs 17.4%), decreased appetite (20.8% vs 12.6%), and pneumonia (20.1% vs 14.2%).

Serious adverse events occurred in 65.4% of patients in the elotuzumab arm and 56.5% in the len-dex arm. The most frequent events were pneumonia, pyrexia, respiratory tract infection, anemia, pulmonary embolism, and acute renal failure.

Bristol-Myers Squibb and AbbVie are co-developing elotuzumab as Empliciti, with Bristol-Myers Squibb solely responsible for commercial activities.

Elotuzumab (Empliciti)

Photo courtesy of

Bristol-Myers Squibb

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of the immunostimulatory antibody elotuzumab (Empliciti).

The CHMP is recommending the drug be approved for use in combination with lenalidomide and dexamethasone to treat patients with multiple myeloma (MM) who have received at least 1 prior therapy.

The CHMP’s recommendation will be reviewed by the European Commission, which usually follows the CHMP’s advice and is expected to deliver its final decision on elotuzumab within 3 months.

The CHMP’s positive opinion of elotuzumab is based on data from the phase 3 ELOQUENT-2 trial, which were presented at ASCO 2015 and published in NEJM.

The trial included 646 MM patients who had received 1 to 3 prior therapies. Baseline patient demographics and disease characteristics were well balanced between treatment arms.

Patients were randomized 1:1 to receive either elotuzumab at 10 mg/kg in combination with lenalidomide and dexamethasone (len-dex) or len-dex alone in 4-week cycles until disease progression or unacceptable toxicity.

The minimum follow-up for all study subjects was 24 months. The co-primary endpoints were progression-free survival (PFS) and overall response rate.

The overall response rate was 78.5% in the elotuzumab arm and 65.5% in the len-dex arm (P=0.0002).

The median PFS was 19.4 months in the elotuzumab arm and 14.9 months in the len-dex arm (P=0.0004). At 1 year, the PFS was 68% in the elotuzumab arm and 57% in the len-dex arm. At 2 years, the PFS was 41% and 27%, respectively.

The most common adverse events in the elotuzumab arm and len-dex arm, respectively, were fatigue (61.6% vs 51.7%), diarrhea (46.9% vs 36.0%), pyrexia (37.4% vs 24.6%), constipation (35.5% vs 27.1%), cough (34.3% vs 18.9%), peripheral neuropathy (26.7% vs 20.8%), nasopharyngitis (24.5% vs 19.2%), upper respiratory tract infection (22.6% vs 17.4%), decreased appetite (20.8% vs 12.6%), and pneumonia (20.1% vs 14.2%).

Serious adverse events occurred in 65.4% of patients in the elotuzumab arm and 56.5% in the len-dex arm. The most frequent events were pneumonia, pyrexia, respiratory tract infection, anemia, pulmonary embolism, and acute renal failure.

Bristol-Myers Squibb and AbbVie are co-developing elotuzumab as Empliciti, with Bristol-Myers Squibb solely responsible for commercial activities.

Elotuzumab (Empliciti)

Photo courtesy of

Bristol-Myers Squibb

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of the immunostimulatory antibody elotuzumab (Empliciti).

The CHMP is recommending the drug be approved for use in combination with lenalidomide and dexamethasone to treat patients with multiple myeloma (MM) who have received at least 1 prior therapy.

The CHMP’s recommendation will be reviewed by the European Commission, which usually follows the CHMP’s advice and is expected to deliver its final decision on elotuzumab within 3 months.

The CHMP’s positive opinion of elotuzumab is based on data from the phase 3 ELOQUENT-2 trial, which were presented at ASCO 2015 and published in NEJM.

The trial included 646 MM patients who had received 1 to 3 prior therapies. Baseline patient demographics and disease characteristics were well balanced between treatment arms.

Patients were randomized 1:1 to receive either elotuzumab at 10 mg/kg in combination with lenalidomide and dexamethasone (len-dex) or len-dex alone in 4-week cycles until disease progression or unacceptable toxicity.

The minimum follow-up for all study subjects was 24 months. The co-primary endpoints were progression-free survival (PFS) and overall response rate.

The overall response rate was 78.5% in the elotuzumab arm and 65.5% in the len-dex arm (P=0.0002).

The median PFS was 19.4 months in the elotuzumab arm and 14.9 months in the len-dex arm (P=0.0004). At 1 year, the PFS was 68% in the elotuzumab arm and 57% in the len-dex arm. At 2 years, the PFS was 41% and 27%, respectively.

The most common adverse events in the elotuzumab arm and len-dex arm, respectively, were fatigue (61.6% vs 51.7%), diarrhea (46.9% vs 36.0%), pyrexia (37.4% vs 24.6%), constipation (35.5% vs 27.1%), cough (34.3% vs 18.9%), peripheral neuropathy (26.7% vs 20.8%), nasopharyngitis (24.5% vs 19.2%), upper respiratory tract infection (22.6% vs 17.4%), decreased appetite (20.8% vs 12.6%), and pneumonia (20.1% vs 14.2%).

Serious adverse events occurred in 65.4% of patients in the elotuzumab arm and 56.5% in the len-dex arm. The most frequent events were pneumonia, pyrexia, respiratory tract infection, anemia, pulmonary embolism, and acute renal failure.

Bristol-Myers Squibb and AbbVie are co-developing elotuzumab as Empliciti, with Bristol-Myers Squibb solely responsible for commercial activities.

Carfilzomib (Kyprolis)

Photo courtesy of Amgen

Health Canada has approved the proteasome inhibitor carfilzomib (Kyprolis) for use in combination with lenalidomide and dexamethasone to treat patients with relapsed multiple myeloma (MM) who have received 1 to 3 prior lines of therapy.

Carfilzomib, a product of Amgen Canada, is also approved for use in the US, the European Union, Argentina, Israel, Kuwait, Mexico, Thailand, and Colombia.

Health Canada’s approval is based on results of the phase 3 ASPIRE trial, which were presented at ASH 2014 and published in NEJM.

The trial enrolled 792 patients with relapsed or refractory MM who had received 1 to 3 prior lines of therapy. The patients were randomized (1:1) to receive carfilzomib plus lenalidomide and dexamethasone (KRd) or just lenalidomide and dexamethasone (Rd) for 18 cycles.

Lenalidomide and dexamethasone were continued thereafter until disease progression. There was no planned cross-over from the control arm to treatment with carfilzomib.

The study’s primary endpoint was progression-free survival. The median progression-free survival was significantly longer in the KRd arm than the Rd arm—26.3 months and 17.6 months, respectively (hazard ratio=0.69, P=0.0001).

At the time of analysis, the difference in overall survival did not reach the prespecified boundary for statistical significance.

The overall response rate was 87% in the KRd arm and 67% in the Rd arm. The median duration of response was 28.6 months and 21.2 months, respectively.

The rates of death due to adverse events (AEs) within 30 days of the last dose were similar between the treatment arms.

The most common causes of death not due to progressive disease occurring in patients in the KRd arm and the Rd arm, respectively, were cardiac disorders (3% vs 2%), infection (2% vs 3%), renal events (0% vs less than 1%), and other AEs (2% vs 3%).

Serious AEs were reported in 60% of patients in the KRd arm and 54% in the Rd arm. The most common serious AEs reported in the KRd arm and the Rd arm, respectively, were pneumonia (14% vs 11%), respiratory tract infection (4% vs 2%), pyrexia (4% vs 2%), and pulmonary embolism (3% vs 2%).

Carfilzomib (Kyprolis)

Photo courtesy of Amgen

Health Canada has approved the proteasome inhibitor carfilzomib (Kyprolis) for use in combination with lenalidomide and dexamethasone to treat patients with relapsed multiple myeloma (MM) who have received 1 to 3 prior lines of therapy.

Carfilzomib, a product of Amgen Canada, is also approved for use in the US, the European Union, Argentina, Israel, Kuwait, Mexico, Thailand, and Colombia.

Health Canada’s approval is based on results of the phase 3 ASPIRE trial, which were presented at ASH 2014 and published in NEJM.

The trial enrolled 792 patients with relapsed or refractory MM who had received 1 to 3 prior lines of therapy. The patients were randomized (1:1) to receive carfilzomib plus lenalidomide and dexamethasone (KRd) or just lenalidomide and dexamethasone (Rd) for 18 cycles.

Lenalidomide and dexamethasone were continued thereafter until disease progression. There was no planned cross-over from the control arm to treatment with carfilzomib.

The study’s primary endpoint was progression-free survival. The median progression-free survival was significantly longer in the KRd arm than the Rd arm—26.3 months and 17.6 months, respectively (hazard ratio=0.69, P=0.0001).

At the time of analysis, the difference in overall survival did not reach the prespecified boundary for statistical significance.

The overall response rate was 87% in the KRd arm and 67% in the Rd arm. The median duration of response was 28.6 months and 21.2 months, respectively.

The rates of death due to adverse events (AEs) within 30 days of the last dose were similar between the treatment arms.

The most common causes of death not due to progressive disease occurring in patients in the KRd arm and the Rd arm, respectively, were cardiac disorders (3% vs 2%), infection (2% vs 3%), renal events (0% vs less than 1%), and other AEs (2% vs 3%).

Serious AEs were reported in 60% of patients in the KRd arm and 54% in the Rd arm. The most common serious AEs reported in the KRd arm and the Rd arm, respectively, were pneumonia (14% vs 11%), respiratory tract infection (4% vs 2%), pyrexia (4% vs 2%), and pulmonary embolism (3% vs 2%).

Carfilzomib (Kyprolis)

Photo courtesy of Amgen

Health Canada has approved the proteasome inhibitor carfilzomib (Kyprolis) for use in combination with lenalidomide and dexamethasone to treat patients with relapsed multiple myeloma (MM) who have received 1 to 3 prior lines of therapy.

Carfilzomib, a product of Amgen Canada, is also approved for use in the US, the European Union, Argentina, Israel, Kuwait, Mexico, Thailand, and Colombia.

Health Canada’s approval is based on results of the phase 3 ASPIRE trial, which were presented at ASH 2014 and published in NEJM.

The trial enrolled 792 patients with relapsed or refractory MM who had received 1 to 3 prior lines of therapy. The patients were randomized (1:1) to receive carfilzomib plus lenalidomide and dexamethasone (KRd) or just lenalidomide and dexamethasone (Rd) for 18 cycles.

Lenalidomide and dexamethasone were continued thereafter until disease progression. There was no planned cross-over from the control arm to treatment with carfilzomib.

The study’s primary endpoint was progression-free survival. The median progression-free survival was significantly longer in the KRd arm than the Rd arm—26.3 months and 17.6 months, respectively (hazard ratio=0.69, P=0.0001).

At the time of analysis, the difference in overall survival did not reach the prespecified boundary for statistical significance.

The overall response rate was 87% in the KRd arm and 67% in the Rd arm. The median duration of response was 28.6 months and 21.2 months, respectively.

The rates of death due to adverse events (AEs) within 30 days of the last dose were similar between the treatment arms.

The most common causes of death not due to progressive disease occurring in patients in the KRd arm and the Rd arm, respectively, were cardiac disorders (3% vs 2%), infection (2% vs 3%), renal events (0% vs less than 1%), and other AEs (2% vs 3%).

Serious AEs were reported in 60% of patients in the KRd arm and 54% in the Rd arm. The most common serious AEs reported in the KRd arm and the Rd arm, respectively, were pneumonia (14% vs 11%), respiratory tract infection (4% vs 2%), pyrexia (4% vs 2%), and pulmonary embolism (3% vs 2%).

Doctor using a smartphone

Photo by Daniel Sone

Understanding and integrating patients’ cultural beliefs into cancer treatment plans may help improve their acceptance of and adherence to treatment in multicultural settings, according to research published in the Journal of Global Oncology.

Researchers examined traditional Maya healers’ understanding of cancer and found that, although there are key differences between Maya and Western medicine perspectives, they also share many fundamental concepts.

“Maya healers understand cancer in remarkably similar ways to Western doctors,” said lead study author Mónica Berger-González, PhD, of the Institute for Environmental Decisions at ETH Zurich in Switzerland.

“Recognizing this is the first step to bridging the gap between cultures and ultimately providing better, more effective services for indigenous populations.”

Nearly half of the population in Guatemala (approximately 5.4 million people) relies on Maya medicine. Traditional healers have practiced in Guatemala for more than 2000 years, with the healing tradition passed down orally and through apprenticeship.

According to the authors, this is one of the first studies to explore the subject of Maya healers and cancer across several ethno-linguistic groups, and limited data exist on survival outcomes.

Dr Berger-González and her colleagues conducted in-depth interviews with 67 healers across various ethnic and language groups in Guatemala, exploring how its indigenous people define and treat cancer.

Of the Maya healers interviewed, 46% were illiterate. Although only 36% were able to define the word cancer, most (85%) were familiar with the term and identified malignancy as a core characteristic of the disease, explaining the concept of metastasis clearly.

The analysis also revealed that Maya healers understand the origins of cancer in ways that align closely with Western medical concepts.

When asked to identify the physical causes of cancer, 10 of 17 causes provided correlated directly with cancer risk factors as understood in Western medicine. Healers cited causes such as the consumption of harmful foods (46.3%), hereditary conditions (29.6%), and lifestyle factors such as smoking or working with toxic substances (29.6%).

One notable difference identified between the 2 perspectives is that Maya healers’ view of cancer is not limited to the physical body, but rather includes a complex imbalance of the emotions, mind, and spirit.

The Maya treatment of cancer is consequently holistic and seeks to restore that balance. This is achieved through a combination of methods—such as regulating diet, plant therapy, detoxifying baths—as well as social, psychological, and spiritual methods, the latter of which, the authors note, is harder to grasp in Western medicine.

“If healthcare professionals do not understand indigenous peoples’ conception of cancer, these patients are far less likely to accept and adhere to treatment in the public healthcare system,” Dr Berger-González said.

Many indigenous people in Guatemala do not have access to Western medicine services, cannot afford them, or prefer Maya medicine even when Western medical treatment is available. Yet Western medicine practitioners have little to no training in multicultural management or traditional indigenous medicine.

The authors offer 3 key recommendations to help address the challenges of providing care in multicultural settings:

• Adequate training of healthcare professionals on cultural and social perceptions of cancer
• Increasing evidence-based research on traditional medicine
• Establishing national regulations on integrating traditional and Western medicine—following other countries like Peru, Brazil, and Ecuador, which have successfully incorporated these aspects of care.

The authors plan to continue their transdisciplinary research with the goal of providing biomedical evidence that advances different aspects of Maya medicine.

Doctor using a smartphone

Photo by Daniel Sone

Understanding and integrating patients’ cultural beliefs into cancer treatment plans may help improve their acceptance of and adherence to treatment in multicultural settings, according to research published in the Journal of Global Oncology.

Researchers examined traditional Maya healers’ understanding of cancer and found that, although there are key differences between Maya and Western medicine perspectives, they also share many fundamental concepts.

“Maya healers understand cancer in remarkably similar ways to Western doctors,” said lead study author Mónica Berger-González, PhD, of the Institute for Environmental Decisions at ETH Zurich in Switzerland.

“Recognizing this is the first step to bridging the gap between cultures and ultimately providing better, more effective services for indigenous populations.”

Nearly half of the population in Guatemala (approximately 5.4 million people) relies on Maya medicine. Traditional healers have practiced in Guatemala for more than 2000 years, with the healing tradition passed down orally and through apprenticeship.

According to the authors, this is one of the first studies to explore the subject of Maya healers and cancer across several ethno-linguistic groups, and limited data exist on survival outcomes.

Dr Berger-González and her colleagues conducted in-depth interviews with 67 healers across various ethnic and language groups in Guatemala, exploring how its indigenous people define and treat cancer.

Of the Maya healers interviewed, 46% were illiterate. Although only 36% were able to define the word cancer, most (85%) were familiar with the term and identified malignancy as a core characteristic of the disease, explaining the concept of metastasis clearly.

The analysis also revealed that Maya healers understand the origins of cancer in ways that align closely with Western medical concepts.

When asked to identify the physical causes of cancer, 10 of 17 causes provided correlated directly with cancer risk factors as understood in Western medicine. Healers cited causes such as the consumption of harmful foods (46.3%), hereditary conditions (29.6%), and lifestyle factors such as smoking or working with toxic substances (29.6%).

One notable difference identified between the 2 perspectives is that Maya healers’ view of cancer is not limited to the physical body, but rather includes a complex imbalance of the emotions, mind, and spirit.

The Maya treatment of cancer is consequently holistic and seeks to restore that balance. This is achieved through a combination of methods—such as regulating diet, plant therapy, detoxifying baths—as well as social, psychological, and spiritual methods, the latter of which, the authors note, is harder to grasp in Western medicine.

“If healthcare professionals do not understand indigenous peoples’ conception of cancer, these patients are far less likely to accept and adhere to treatment in the public healthcare system,” Dr Berger-González said.

Many indigenous people in Guatemala do not have access to Western medicine services, cannot afford them, or prefer Maya medicine even when Western medical treatment is available. Yet Western medicine practitioners have little to no training in multicultural management or traditional indigenous medicine.

The authors offer 3 key recommendations to help address the challenges of providing care in multicultural settings:

• Adequate training of healthcare professionals on cultural and social perceptions of cancer
• Increasing evidence-based research on traditional medicine
• Establishing national regulations on integrating traditional and Western medicine—following other countries like Peru, Brazil, and Ecuador, which have successfully incorporated these aspects of care.

The authors plan to continue their transdisciplinary research with the goal of providing biomedical evidence that advances different aspects of Maya medicine.

Doctor using a smartphone

Photo by Daniel Sone

Understanding and integrating patients’ cultural beliefs into cancer treatment plans may help improve their acceptance of and adherence to treatment in multicultural settings, according to research published in the Journal of Global Oncology.

Researchers examined traditional Maya healers’ understanding of cancer and found that, although there are key differences between Maya and Western medicine perspectives, they also share many fundamental concepts.

“Maya healers understand cancer in remarkably similar ways to Western doctors,” said lead study author Mónica Berger-González, PhD, of the Institute for Environmental Decisions at ETH Zurich in Switzerland.

“Recognizing this is the first step to bridging the gap between cultures and ultimately providing better, more effective services for indigenous populations.”

Nearly half of the population in Guatemala (approximately 5.4 million people) relies on Maya medicine. Traditional healers have practiced in Guatemala for more than 2000 years, with the healing tradition passed down orally and through apprenticeship.

According to the authors, this is one of the first studies to explore the subject of Maya healers and cancer across several ethno-linguistic groups, and limited data exist on survival outcomes.

Dr Berger-González and her colleagues conducted in-depth interviews with 67 healers across various ethnic and language groups in Guatemala, exploring how its indigenous people define and treat cancer.

Of the Maya healers interviewed, 46% were illiterate. Although only 36% were able to define the word cancer, most (85%) were familiar with the term and identified malignancy as a core characteristic of the disease, explaining the concept of metastasis clearly.

The analysis also revealed that Maya healers understand the origins of cancer in ways that align closely with Western medical concepts.

When asked to identify the physical causes of cancer, 10 of 17 causes provided correlated directly with cancer risk factors as understood in Western medicine. Healers cited causes such as the consumption of harmful foods (46.3%), hereditary conditions (29.6%), and lifestyle factors such as smoking or working with toxic substances (29.6%).

One notable difference identified between the 2 perspectives is that Maya healers’ view of cancer is not limited to the physical body, but rather includes a complex imbalance of the emotions, mind, and spirit.

The Maya treatment of cancer is consequently holistic and seeks to restore that balance. This is achieved through a combination of methods—such as regulating diet, plant therapy, detoxifying baths—as well as social, psychological, and spiritual methods, the latter of which, the authors note, is harder to grasp in Western medicine.

“If healthcare professionals do not understand indigenous peoples’ conception of cancer, these patients are far less likely to accept and adhere to treatment in the public healthcare system,” Dr Berger-González said.

Many indigenous people in Guatemala do not have access to Western medicine services, cannot afford them, or prefer Maya medicine even when Western medical treatment is available. Yet Western medicine practitioners have little to no training in multicultural management or traditional indigenous medicine.

The authors offer 3 key recommendations to help address the challenges of providing care in multicultural settings:

• Adequate training of healthcare professionals on cultural and social perceptions of cancer
• Increasing evidence-based research on traditional medicine
• Establishing national regulations on integrating traditional and Western medicine—following other countries like Peru, Brazil, and Ecuador, which have successfully incorporated these aspects of care.

The authors plan to continue their transdisciplinary research with the goal of providing biomedical evidence that advances different aspects of Maya medicine.

Medicare will cover allogeneic hematopoietic stem cell transplantation (HSCT) for beneficiaries with multiple myeloma, myelofibrosis, or sickle cell disease in the context of approved, prospective clinical trials, the Centers for Medicare & Medicaid Services announced in a final decision memo Jan. 27.

Approvable studies must examine whether Medicare beneficiaries who receive allogeneic HSCT have improved outcomes, compared with patients who do not receive allogeneic HSCT as measured by graft vs. host disease, other transplant-related adverse events, overall survival, and, optionally, quality of life measures.

In multiple myeloma, allogeneic HSCT will be covered only for Medicare beneficiaries who have Durie-Salmon Stage II or III multiple myeloma, or International Staging System (ISS) Stage II or Stage III multiple myeloma, and are participating in an approved prospective clinical study. Such studies must control for selection bias and potential confounding by age, duration of diagnosis, disease classification, International Myeloma Working Group (IMWG) classification, ISS staging, Durie-Salmon staging, comorbid conditions, type of preparative/conditioning regimen, graft vs. host disease (GVHD) prophylaxis, donor type, and cell source, the CMS said in its memo.

In myelofibrosis, allogeneic HSCT will be covered by Medicare in an approved prospective study only for beneficiaries with Dynamic International Prognostic Scoring System (DIPSS plus) intermediate-2 or high primary or secondary myelofibrosis. Studies must be controlled for selection bias and potential confounding by age, duration of diagnosis, disease classification, DIPSS plus score, comorbid conditions, type of preparative/conditioning regimen, GVHD prophylaxis, donor type, and cell source.

In sickle cell disease, allogeneic HSCT will be covered by Medicare only for beneficiaries who have severe, symptomatic disease. Approvable studies must control for selection bias and potential confounding by age, duration of diagnosis, comorbid conditions, type of preparative/conditioning regimen, GVHD prophylaxis, donor type, and cell source.

[email protected]

On Twitter @maryjodales

Medicare will cover allogeneic hematopoietic stem cell transplantation (HSCT) for beneficiaries with multiple myeloma, myelofibrosis, or sickle cell disease in the context of approved, prospective clinical trials, the Centers for Medicare & Medicaid Services announced in a final decision memo Jan. 27.

Approvable studies must examine whether Medicare beneficiaries who receive allogeneic HSCT have improved outcomes, compared with patients who do not receive allogeneic HSCT as measured by graft vs. host disease, other transplant-related adverse events, overall survival, and, optionally, quality of life measures.

In multiple myeloma, allogeneic HSCT will be covered only for Medicare beneficiaries who have Durie-Salmon Stage II or III multiple myeloma, or International Staging System (ISS) Stage II or Stage III multiple myeloma, and are participating in an approved prospective clinical study. Such studies must control for selection bias and potential confounding by age, duration of diagnosis, disease classification, International Myeloma Working Group (IMWG) classification, ISS staging, Durie-Salmon staging, comorbid conditions, type of preparative/conditioning regimen, graft vs. host disease (GVHD) prophylaxis, donor type, and cell source, the CMS said in its memo.

In myelofibrosis, allogeneic HSCT will be covered by Medicare in an approved prospective study only for beneficiaries with Dynamic International Prognostic Scoring System (DIPSS plus) intermediate-2 or high primary or secondary myelofibrosis. Studies must be controlled for selection bias and potential confounding by age, duration of diagnosis, disease classification, DIPSS plus score, comorbid conditions, type of preparative/conditioning regimen, GVHD prophylaxis, donor type, and cell source.

In sickle cell disease, allogeneic HSCT will be covered by Medicare only for beneficiaries who have severe, symptomatic disease. Approvable studies must control for selection bias and potential confounding by age, duration of diagnosis, comorbid conditions, type of preparative/conditioning regimen, GVHD prophylaxis, donor type, and cell source.

[email protected]

On Twitter @maryjodales

Medicare will cover allogeneic hematopoietic stem cell transplantation (HSCT) for beneficiaries with multiple myeloma, myelofibrosis, or sickle cell disease in the context of approved, prospective clinical trials, the Centers for Medicare & Medicaid Services announced in a final decision memo Jan. 27.

Approvable studies must examine whether Medicare beneficiaries who receive allogeneic HSCT have improved outcomes, compared with patients who do not receive allogeneic HSCT as measured by graft vs. host disease, other transplant-related adverse events, overall survival, and, optionally, quality of life measures.

In multiple myeloma, allogeneic HSCT will be covered only for Medicare beneficiaries who have Durie-Salmon Stage II or III multiple myeloma, or International Staging System (ISS) Stage II or Stage III multiple myeloma, and are participating in an approved prospective clinical study. Such studies must control for selection bias and potential confounding by age, duration of diagnosis, disease classification, International Myeloma Working Group (IMWG) classification, ISS staging, Durie-Salmon staging, comorbid conditions, type of preparative/conditioning regimen, graft vs. host disease (GVHD) prophylaxis, donor type, and cell source, the CMS said in its memo.

In myelofibrosis, allogeneic HSCT will be covered by Medicare in an approved prospective study only for beneficiaries with Dynamic International Prognostic Scoring System (DIPSS plus) intermediate-2 or high primary or secondary myelofibrosis. Studies must be controlled for selection bias and potential confounding by age, duration of diagnosis, disease classification, DIPSS plus score, comorbid conditions, type of preparative/conditioning regimen, GVHD prophylaxis, donor type, and cell source.

In sickle cell disease, allogeneic HSCT will be covered by Medicare only for beneficiaries who have severe, symptomatic disease. Approvable studies must control for selection bias and potential confounding by age, duration of diagnosis, comorbid conditions, type of preparative/conditioning regimen, GVHD prophylaxis, donor type, and cell source.

[email protected]

On Twitter @maryjodales