Jennifer Smith

New research suggests several factors are associated with failure to discontinue immune suppression after allogeneic hematopoietic cell transplant (HCT).

Patients older than 50 years, those with advanced stage disease, patients with a mismatched unrelated donor, and those who received peripheral blood stem cells from an unrelated donor were less likely to discontinue immune suppression successfully, Joseph Pidala, MD, PhD, of Moffitt Cancer Center in Tampa, Fla., and colleagues reported in JAMA Oncology.

The researchers analyzed data from 827 patients in two national Blood and Marrow Transplant Clinical Trial Network studies (NCT00075816 and NCT00406393). These randomized, phase 3 trials enrolled patients with hematologic malignancies who received myeloablative conditioning before allogeneic HCT.

The patients’ median age at HCT was 44 years (range, less than 1 to 67 years), and 55.1% were male. The median follow-up was 72 months (range, 11-124 months).

At 5 years, 20% of patients (n = 168) had successfully discontinued immune suppression and were still alive. A total of 342 patients (41.4%) were able to stop immune suppression, but 127 of them had to resume it after developing graft-versus-host disease (GVHD). There were an additional 47 patients who died or relapsed after stopping immune suppression.

The researchers identified several factors that were significantly associated with lower odds of discontinuing immune suppression and being free of GVHD, including:
 

• Being older than 50 years versus younger than 30 years (adjusted odds ratio [aOR], 0.27; 99% confidence interval [CI], 0.14-0.50; P less than .001).
• Having a mismatched unrelated donor versus having a matched sibling (aOR, 0.37; 99% CI, 0.14-0.97; P = .008).
• Receiving peripheral blood stem cells versus bone marrow, from unrelated donors only (aOR, 0.46; 99% CI, 0.26-0.82; P less than .001).
• Having advanced stage disease versus early disease (aOR, 0.45; 99%CI, 0.23-0.86; P = .002).

The researchers also found that discontinuing immune suppression was not significantly associated with a decreased risk of relapse, with a hazard ratio (HR) of 1.95 (99% CI, 0.88-4.31; P = .03).

There was no significant association between acute GVHD–related variables and discontinuation of immune suppression. However, there were a few factors significantly associated with a lower likelihood of discontinuation after chronic GVHD, including:
 

• Current skin involvement (HR, 0.33; 99% CI, 0.14-0.80; P = .001).
• Unrelated well-matched donor versus matched sibling donor (HR, 0.29; 99% CI, 0.10-0.79; P = .001).
• Unrelated mismatched donor versus matched sibling donor (HR, 0.17; 99% CI, 0.03-0.95; P = .008).

In total, 127 patients had to resume immune suppression because of GVHD. Such failed attempts at discontinuing immune suppression were associated with receiving peripheral blood stem cells from an unrelated donor versus bone marrow from an unrelated donor, with an HR of 2.62 (99% CI, 1.30-5.29; P less than .001).

A history of acute or chronic GVHD was associated with failure to discontinue immune suppression, the researchers noted.

Lastly, the researchers developed dynamic prediction models for the probability of freedom from immune suppression and GVHD at 1, 3, and 5 years in the future. The team found that graft type, donor type, age, state history, and timing of immune suppression discontinuation were all associated with the likelihood of being free from immune suppression and GVHD at all three time points. Disease risk was only associated with freedom from immune suppression and GVHD at the 1-year mark.

The researchers said their findings must be validated in an independent cohort of patients and, after that, should be tested in a prospective trial.

The current study was funded by the National Heart Lung and Blood Institute. Two of the researchers reported relationships with more than 30 pharmaceutical companies.

[email protected]

SOURCE: Pidala J et al. JAMA Oncol. 2019 Sep 26. doi: 10.1001/jamaoncol.2019.2974.

New research suggests several factors are associated with failure to discontinue immune suppression after allogeneic hematopoietic cell transplant (HCT).

Patients older than 50 years, those with advanced stage disease, patients with a mismatched unrelated donor, and those who received peripheral blood stem cells from an unrelated donor were less likely to discontinue immune suppression successfully, Joseph Pidala, MD, PhD, of Moffitt Cancer Center in Tampa, Fla., and colleagues reported in JAMA Oncology.

The researchers analyzed data from 827 patients in two national Blood and Marrow Transplant Clinical Trial Network studies (NCT00075816 and NCT00406393). These randomized, phase 3 trials enrolled patients with hematologic malignancies who received myeloablative conditioning before allogeneic HCT.

The patients’ median age at HCT was 44 years (range, less than 1 to 67 years), and 55.1% were male. The median follow-up was 72 months (range, 11-124 months).

At 5 years, 20% of patients (n = 168) had successfully discontinued immune suppression and were still alive. A total of 342 patients (41.4%) were able to stop immune suppression, but 127 of them had to resume it after developing graft-versus-host disease (GVHD). There were an additional 47 patients who died or relapsed after stopping immune suppression.

The researchers identified several factors that were significantly associated with lower odds of discontinuing immune suppression and being free of GVHD, including:
 

• Being older than 50 years versus younger than 30 years (adjusted odds ratio [aOR], 0.27; 99% confidence interval [CI], 0.14-0.50; P less than .001).
• Having a mismatched unrelated donor versus having a matched sibling (aOR, 0.37; 99% CI, 0.14-0.97; P = .008).
• Receiving peripheral blood stem cells versus bone marrow, from unrelated donors only (aOR, 0.46; 99% CI, 0.26-0.82; P less than .001).
• Having advanced stage disease versus early disease (aOR, 0.45; 99%CI, 0.23-0.86; P = .002).

The researchers also found that discontinuing immune suppression was not significantly associated with a decreased risk of relapse, with a hazard ratio (HR) of 1.95 (99% CI, 0.88-4.31; P = .03).

There was no significant association between acute GVHD–related variables and discontinuation of immune suppression. However, there were a few factors significantly associated with a lower likelihood of discontinuation after chronic GVHD, including:
 

• Current skin involvement (HR, 0.33; 99% CI, 0.14-0.80; P = .001).
• Unrelated well-matched donor versus matched sibling donor (HR, 0.29; 99% CI, 0.10-0.79; P = .001).
• Unrelated mismatched donor versus matched sibling donor (HR, 0.17; 99% CI, 0.03-0.95; P = .008).

In total, 127 patients had to resume immune suppression because of GVHD. Such failed attempts at discontinuing immune suppression were associated with receiving peripheral blood stem cells from an unrelated donor versus bone marrow from an unrelated donor, with an HR of 2.62 (99% CI, 1.30-5.29; P less than .001).

A history of acute or chronic GVHD was associated with failure to discontinue immune suppression, the researchers noted.

Lastly, the researchers developed dynamic prediction models for the probability of freedom from immune suppression and GVHD at 1, 3, and 5 years in the future. The team found that graft type, donor type, age, state history, and timing of immune suppression discontinuation were all associated with the likelihood of being free from immune suppression and GVHD at all three time points. Disease risk was only associated with freedom from immune suppression and GVHD at the 1-year mark.

The researchers said their findings must be validated in an independent cohort of patients and, after that, should be tested in a prospective trial.

The current study was funded by the National Heart Lung and Blood Institute. Two of the researchers reported relationships with more than 30 pharmaceutical companies.

[email protected]

SOURCE: Pidala J et al. JAMA Oncol. 2019 Sep 26. doi: 10.1001/jamaoncol.2019.2974.

New research suggests several factors are associated with failure to discontinue immune suppression after allogeneic hematopoietic cell transplant (HCT).

Patients older than 50 years, those with advanced stage disease, patients with a mismatched unrelated donor, and those who received peripheral blood stem cells from an unrelated donor were less likely to discontinue immune suppression successfully, Joseph Pidala, MD, PhD, of Moffitt Cancer Center in Tampa, Fla., and colleagues reported in JAMA Oncology.

The researchers analyzed data from 827 patients in two national Blood and Marrow Transplant Clinical Trial Network studies (NCT00075816 and NCT00406393). These randomized, phase 3 trials enrolled patients with hematologic malignancies who received myeloablative conditioning before allogeneic HCT.

The patients’ median age at HCT was 44 years (range, less than 1 to 67 years), and 55.1% were male. The median follow-up was 72 months (range, 11-124 months).

At 5 years, 20% of patients (n = 168) had successfully discontinued immune suppression and were still alive. A total of 342 patients (41.4%) were able to stop immune suppression, but 127 of them had to resume it after developing graft-versus-host disease (GVHD). There were an additional 47 patients who died or relapsed after stopping immune suppression.

The researchers identified several factors that were significantly associated with lower odds of discontinuing immune suppression and being free of GVHD, including:
 

• Being older than 50 years versus younger than 30 years (adjusted odds ratio [aOR], 0.27; 99% confidence interval [CI], 0.14-0.50; P less than .001).
• Having a mismatched unrelated donor versus having a matched sibling (aOR, 0.37; 99% CI, 0.14-0.97; P = .008).
• Receiving peripheral blood stem cells versus bone marrow, from unrelated donors only (aOR, 0.46; 99% CI, 0.26-0.82; P less than .001).
• Having advanced stage disease versus early disease (aOR, 0.45; 99%CI, 0.23-0.86; P = .002).

The researchers also found that discontinuing immune suppression was not significantly associated with a decreased risk of relapse, with a hazard ratio (HR) of 1.95 (99% CI, 0.88-4.31; P = .03).

There was no significant association between acute GVHD–related variables and discontinuation of immune suppression. However, there were a few factors significantly associated with a lower likelihood of discontinuation after chronic GVHD, including:
 

• Current skin involvement (HR, 0.33; 99% CI, 0.14-0.80; P = .001).
• Unrelated well-matched donor versus matched sibling donor (HR, 0.29; 99% CI, 0.10-0.79; P = .001).
• Unrelated mismatched donor versus matched sibling donor (HR, 0.17; 99% CI, 0.03-0.95; P = .008).

In total, 127 patients had to resume immune suppression because of GVHD. Such failed attempts at discontinuing immune suppression were associated with receiving peripheral blood stem cells from an unrelated donor versus bone marrow from an unrelated donor, with an HR of 2.62 (99% CI, 1.30-5.29; P less than .001).

A history of acute or chronic GVHD was associated with failure to discontinue immune suppression, the researchers noted.

Lastly, the researchers developed dynamic prediction models for the probability of freedom from immune suppression and GVHD at 1, 3, and 5 years in the future. The team found that graft type, donor type, age, state history, and timing of immune suppression discontinuation were all associated with the likelihood of being free from immune suppression and GVHD at all three time points. Disease risk was only associated with freedom from immune suppression and GVHD at the 1-year mark.

The researchers said their findings must be validated in an independent cohort of patients and, after that, should be tested in a prospective trial.

The current study was funded by the National Heart Lung and Blood Institute. Two of the researchers reported relationships with more than 30 pharmaceutical companies.

[email protected]

SOURCE: Pidala J et al. JAMA Oncol. 2019 Sep 26. doi: 10.1001/jamaoncol.2019.2974.

Hearn Jay Cho, MD, PhD, has been appointed chief medical officer of the Multiple Myeloma Research Foundation (MMRF). In this role, Dr. Cho will develop a clinical research strategy and accelerate drug development programs for the MMRF. He will also lead the Multiple Myeloma Research Consortium, a group of 25 research centers focused on multiple myeloma.

Dr. Cho will continue on as an associate professor of medicine at the Icahn School of Medicine at Mt. Sinai in New York, and as an attending physician with the multiple myeloma service at the Mt. Sinai Tisch Cancer Institute. He will also continue to manage his lab at Mt. Sinai.

Michael Jay Styler, MD, has joined the department of hematology/oncology as an associate professor in the academic clinician track as part of the bone marrow transplant program at Fox Chase Cancer Center in Philadelphia.

Dr. Styler was previously medical director of the transplant center, apheresis center, and collection center at Hahnemann University Hospital in Philadelphia, as well as medical director of the stem cell transplant program and the clinical service chief of hematology/oncology. Dr. Styler was also an associate professor at Drexel University, Philadelphia, where he served as division director of hematology/oncology and associate fellowship director of the hematology/oncology program.

Jaspreet Chahal, MD, has joined the Oncology Institute of Hope and Innovation, which has locations in Arizona, California, and Nevada. Dr. Chahal will serve patients in Tucson and Green Valley, Ariz.

Dr. Chahal has a doctor of medicine degree from St. George’s University in West Indies, Grenada. She completed her internship in hematology and oncology at Hofstra University, Hempstead, N.Y., and her residency in internal medicine at the University of Arizona in Tucson.

Marcin Chwistek, MD, has been appointed editor in chief of AAHPM Quarterly, which is published by the American Academy of Hospice and Palliative Medicine. Dr. Chwistek is an associate professor in the department of hematology/oncology at Fox Chase Cancer Center in Philadelphia, where he is also director of the pain and palliative care program.

As editor in chief of AAHPM Quarterly, Dr. Chwistek will work with other members of the editorial board to develop content related to hospice care and palliative medicine. Dr. Chwistek previously served as the associate editor in chief of AAHPM Quarterly and writes a column that appears in every issue.
 

Movers in Medicine highlights career moves and personal achievements by hematologists and oncologists. Did you switch jobs, take on a new role, climb a mountain? Tell us all about it at [email protected] , and you could be featured in Movers in Medicine.

[email protected]

Hearn Jay Cho, MD, PhD, has been appointed chief medical officer of the Multiple Myeloma Research Foundation (MMRF). In this role, Dr. Cho will develop a clinical research strategy and accelerate drug development programs for the MMRF. He will also lead the Multiple Myeloma Research Consortium, a group of 25 research centers focused on multiple myeloma.

Dr. Cho will continue on as an associate professor of medicine at the Icahn School of Medicine at Mt. Sinai in New York, and as an attending physician with the multiple myeloma service at the Mt. Sinai Tisch Cancer Institute. He will also continue to manage his lab at Mt. Sinai.

Michael Jay Styler, MD, has joined the department of hematology/oncology as an associate professor in the academic clinician track as part of the bone marrow transplant program at Fox Chase Cancer Center in Philadelphia.

Dr. Styler was previously medical director of the transplant center, apheresis center, and collection center at Hahnemann University Hospital in Philadelphia, as well as medical director of the stem cell transplant program and the clinical service chief of hematology/oncology. Dr. Styler was also an associate professor at Drexel University, Philadelphia, where he served as division director of hematology/oncology and associate fellowship director of the hematology/oncology program.

Jaspreet Chahal, MD, has joined the Oncology Institute of Hope and Innovation, which has locations in Arizona, California, and Nevada. Dr. Chahal will serve patients in Tucson and Green Valley, Ariz.

Dr. Chahal has a doctor of medicine degree from St. George’s University in West Indies, Grenada. She completed her internship in hematology and oncology at Hofstra University, Hempstead, N.Y., and her residency in internal medicine at the University of Arizona in Tucson.

Marcin Chwistek, MD, has been appointed editor in chief of AAHPM Quarterly, which is published by the American Academy of Hospice and Palliative Medicine. Dr. Chwistek is an associate professor in the department of hematology/oncology at Fox Chase Cancer Center in Philadelphia, where he is also director of the pain and palliative care program.

As editor in chief of AAHPM Quarterly, Dr. Chwistek will work with other members of the editorial board to develop content related to hospice care and palliative medicine. Dr. Chwistek previously served as the associate editor in chief of AAHPM Quarterly and writes a column that appears in every issue.
 

Movers in Medicine highlights career moves and personal achievements by hematologists and oncologists. Did you switch jobs, take on a new role, climb a mountain? Tell us all about it at [email protected] , and you could be featured in Movers in Medicine.

[email protected]

Hearn Jay Cho, MD, PhD, has been appointed chief medical officer of the Multiple Myeloma Research Foundation (MMRF). In this role, Dr. Cho will develop a clinical research strategy and accelerate drug development programs for the MMRF. He will also lead the Multiple Myeloma Research Consortium, a group of 25 research centers focused on multiple myeloma.

Dr. Cho will continue on as an associate professor of medicine at the Icahn School of Medicine at Mt. Sinai in New York, and as an attending physician with the multiple myeloma service at the Mt. Sinai Tisch Cancer Institute. He will also continue to manage his lab at Mt. Sinai.

Michael Jay Styler, MD, has joined the department of hematology/oncology as an associate professor in the academic clinician track as part of the bone marrow transplant program at Fox Chase Cancer Center in Philadelphia.

Dr. Styler was previously medical director of the transplant center, apheresis center, and collection center at Hahnemann University Hospital in Philadelphia, as well as medical director of the stem cell transplant program and the clinical service chief of hematology/oncology. Dr. Styler was also an associate professor at Drexel University, Philadelphia, where he served as division director of hematology/oncology and associate fellowship director of the hematology/oncology program.

Jaspreet Chahal, MD, has joined the Oncology Institute of Hope and Innovation, which has locations in Arizona, California, and Nevada. Dr. Chahal will serve patients in Tucson and Green Valley, Ariz.

Dr. Chahal has a doctor of medicine degree from St. George’s University in West Indies, Grenada. She completed her internship in hematology and oncology at Hofstra University, Hempstead, N.Y., and her residency in internal medicine at the University of Arizona in Tucson.

Marcin Chwistek, MD, has been appointed editor in chief of AAHPM Quarterly, which is published by the American Academy of Hospice and Palliative Medicine. Dr. Chwistek is an associate professor in the department of hematology/oncology at Fox Chase Cancer Center in Philadelphia, where he is also director of the pain and palliative care program.

As editor in chief of AAHPM Quarterly, Dr. Chwistek will work with other members of the editorial board to develop content related to hospice care and palliative medicine. Dr. Chwistek previously served as the associate editor in chief of AAHPM Quarterly and writes a column that appears in every issue.
 

Movers in Medicine highlights career moves and personal achievements by hematologists and oncologists. Did you switch jobs, take on a new role, climb a mountain? Tell us all about it at [email protected] , and you could be featured in Movers in Medicine.

[email protected]

BOSTON – The all-oral combination of selinexor, lenalidomide, and dexamethasone has demonstrated efficacy in patients with relapsed/refractory multiple myeloma, particularly those who are lenalidomide naive.

Jennifer Smith/MDedge News

In the phase 1/2 STOMP trial (NCT02343042), selinexor plus lenalidomide and dexamethasone produced an overall response rate (ORR) of 60% in all evaluable patients and an ORR of 92% in lenalidomide-naive patients.

Darrell White, MD, of Dalhousie University in Halifax, N.S., presented these results at the International Myeloma Workshop held by the International Myeloma Society.

Dr. White discussed results in myeloma patients who had received at least one prior line of therapy. This arm of the STOMP trial has enrolled 24 patients. Their median age at baseline was 67 years (range, 49-84 years), and their median time from diagnosis was 4.5 years (range, less than 1-22 years).

The patients had received a median of 1 (range, 1-8) prior therapies. Half of patients had received a transplant. All patients had received a proteasome inhibitor (65% refractory), and 38% had received prior lenalidomide (21% refractory).

Dosing

Patients received selinexor at 60 mg once or twice weekly or at 80 mg once weekly on a 28-day cycle. They received dexamethasone at 20 mg twice weekly or 40 mg once weekly and lenalidomide at 25 mg once daily every 21 days.

The recommended phase 2 dosing schedule was selinexor at 60 mg once weekly plus lenalidomide at 25 mg daily and dexamethasone at 40 mg once weekly. Half of patients (n = 12) received this dosing schedule.

There were no dose-limiting toxicities (DLTs) at the recommended phase 2 dose. When selinexor was given at 60 mg twice weekly, DLTs included grade 3 fatigue, grade 3 anorexia and weight loss, and grade 4 thrombocytopenia (n = 2). When selinexor was given at 80 mg once weekly, the DLTs were grade 4 thrombocytopenia (n = 2).

Safety

“The side-effect profile was as expected, with mainly grade 3/4 toxicity being hematologic and primarily thrombocytopenia and neutropenia,” Dr. White said. “Frequent gastrointestinal side effects [were] expected. Investigators were able to manage that with appropriate supportive care and antiemetics in particular.”

Among patients who received the recommended phase 2 dose, the grade 4 treatment-related adverse events (AEs) were thrombocytopenia (n = 4) and neutropenia (n = 4). Grade 3 treatment-related AEs were thrombocytopenia (n = 3), neutropenia (n = 4), anemia (n = 1), nausea (n = 1), asthenia (n = 1), fatigue (n = 2), and dehydration (n = 1). Common grade 1/2 treatment-related AEs in patients who received the recommended phase 2 dose were nausea (n = 6), anorexia (n = 5), weight loss (n = 5), vomiting (n = 4), diarrhea (n = 4), and fatigue (n = 4).

Efficacy

In the 20 patients evaluable for response, the ORR was 60% (n = 12). One patient achieved a stringent complete response, three had a very good partial response, and eight had a partial response. The median time to response was 1 month.

The ORR was 92% (11/12) in lenalidomide-naive patients and 13% (1/8) in lenalidomide-exposed patients. The single responder in the lenalidomide-exposed group achieved a partial response.

Among lenalidomide-naive patients who received the recommended phase 2 dose, the median time on treatment was 12 months. The patient who achieved a stringent complete response remained on treatment at last follow-up, as did one partial responder and one patient with a very good partial response.

These results suggest the combination of selinexor, lenalidomide, and dexamethasone “is active, relatively well tolerated, and could warrant further investigation,” Dr. White said.

The STOMP trial is sponsored by Karyopharm Therapeutics. Dr. White disclosed relationships with Karyopharm, Amgen, Celgene, Janssen, Sanofi, and Takeda.

[email protected]

SOURCE: White D et al. IMW 2019, Abstract OAB-083.

BOSTON – The all-oral combination of selinexor, lenalidomide, and dexamethasone has demonstrated efficacy in patients with relapsed/refractory multiple myeloma, particularly those who are lenalidomide naive.

Jennifer Smith/MDedge News

In the phase 1/2 STOMP trial (NCT02343042), selinexor plus lenalidomide and dexamethasone produced an overall response rate (ORR) of 60% in all evaluable patients and an ORR of 92% in lenalidomide-naive patients.

Darrell White, MD, of Dalhousie University in Halifax, N.S., presented these results at the International Myeloma Workshop held by the International Myeloma Society.

Dr. White discussed results in myeloma patients who had received at least one prior line of therapy. This arm of the STOMP trial has enrolled 24 patients. Their median age at baseline was 67 years (range, 49-84 years), and their median time from diagnosis was 4.5 years (range, less than 1-22 years).

The patients had received a median of 1 (range, 1-8) prior therapies. Half of patients had received a transplant. All patients had received a proteasome inhibitor (65% refractory), and 38% had received prior lenalidomide (21% refractory).

Dosing

Patients received selinexor at 60 mg once or twice weekly or at 80 mg once weekly on a 28-day cycle. They received dexamethasone at 20 mg twice weekly or 40 mg once weekly and lenalidomide at 25 mg once daily every 21 days.

The recommended phase 2 dosing schedule was selinexor at 60 mg once weekly plus lenalidomide at 25 mg daily and dexamethasone at 40 mg once weekly. Half of patients (n = 12) received this dosing schedule.

There were no dose-limiting toxicities (DLTs) at the recommended phase 2 dose. When selinexor was given at 60 mg twice weekly, DLTs included grade 3 fatigue, grade 3 anorexia and weight loss, and grade 4 thrombocytopenia (n = 2). When selinexor was given at 80 mg once weekly, the DLTs were grade 4 thrombocytopenia (n = 2).

Safety

“The side-effect profile was as expected, with mainly grade 3/4 toxicity being hematologic and primarily thrombocytopenia and neutropenia,” Dr. White said. “Frequent gastrointestinal side effects [were] expected. Investigators were able to manage that with appropriate supportive care and antiemetics in particular.”

Among patients who received the recommended phase 2 dose, the grade 4 treatment-related adverse events (AEs) were thrombocytopenia (n = 4) and neutropenia (n = 4). Grade 3 treatment-related AEs were thrombocytopenia (n = 3), neutropenia (n = 4), anemia (n = 1), nausea (n = 1), asthenia (n = 1), fatigue (n = 2), and dehydration (n = 1). Common grade 1/2 treatment-related AEs in patients who received the recommended phase 2 dose were nausea (n = 6), anorexia (n = 5), weight loss (n = 5), vomiting (n = 4), diarrhea (n = 4), and fatigue (n = 4).

Efficacy

In the 20 patients evaluable for response, the ORR was 60% (n = 12). One patient achieved a stringent complete response, three had a very good partial response, and eight had a partial response. The median time to response was 1 month.

The ORR was 92% (11/12) in lenalidomide-naive patients and 13% (1/8) in lenalidomide-exposed patients. The single responder in the lenalidomide-exposed group achieved a partial response.

Among lenalidomide-naive patients who received the recommended phase 2 dose, the median time on treatment was 12 months. The patient who achieved a stringent complete response remained on treatment at last follow-up, as did one partial responder and one patient with a very good partial response.

These results suggest the combination of selinexor, lenalidomide, and dexamethasone “is active, relatively well tolerated, and could warrant further investigation,” Dr. White said.

The STOMP trial is sponsored by Karyopharm Therapeutics. Dr. White disclosed relationships with Karyopharm, Amgen, Celgene, Janssen, Sanofi, and Takeda.

[email protected]

SOURCE: White D et al. IMW 2019, Abstract OAB-083.

BOSTON – The all-oral combination of selinexor, lenalidomide, and dexamethasone has demonstrated efficacy in patients with relapsed/refractory multiple myeloma, particularly those who are lenalidomide naive.

Jennifer Smith/MDedge News

In the phase 1/2 STOMP trial (NCT02343042), selinexor plus lenalidomide and dexamethasone produced an overall response rate (ORR) of 60% in all evaluable patients and an ORR of 92% in lenalidomide-naive patients.

Darrell White, MD, of Dalhousie University in Halifax, N.S., presented these results at the International Myeloma Workshop held by the International Myeloma Society.

Dr. White discussed results in myeloma patients who had received at least one prior line of therapy. This arm of the STOMP trial has enrolled 24 patients. Their median age at baseline was 67 years (range, 49-84 years), and their median time from diagnosis was 4.5 years (range, less than 1-22 years).

The patients had received a median of 1 (range, 1-8) prior therapies. Half of patients had received a transplant. All patients had received a proteasome inhibitor (65% refractory), and 38% had received prior lenalidomide (21% refractory).

Dosing

Patients received selinexor at 60 mg once or twice weekly or at 80 mg once weekly on a 28-day cycle. They received dexamethasone at 20 mg twice weekly or 40 mg once weekly and lenalidomide at 25 mg once daily every 21 days.

The recommended phase 2 dosing schedule was selinexor at 60 mg once weekly plus lenalidomide at 25 mg daily and dexamethasone at 40 mg once weekly. Half of patients (n = 12) received this dosing schedule.

There were no dose-limiting toxicities (DLTs) at the recommended phase 2 dose. When selinexor was given at 60 mg twice weekly, DLTs included grade 3 fatigue, grade 3 anorexia and weight loss, and grade 4 thrombocytopenia (n = 2). When selinexor was given at 80 mg once weekly, the DLTs were grade 4 thrombocytopenia (n = 2).

Safety

“The side-effect profile was as expected, with mainly grade 3/4 toxicity being hematologic and primarily thrombocytopenia and neutropenia,” Dr. White said. “Frequent gastrointestinal side effects [were] expected. Investigators were able to manage that with appropriate supportive care and antiemetics in particular.”

Among patients who received the recommended phase 2 dose, the grade 4 treatment-related adverse events (AEs) were thrombocytopenia (n = 4) and neutropenia (n = 4). Grade 3 treatment-related AEs were thrombocytopenia (n = 3), neutropenia (n = 4), anemia (n = 1), nausea (n = 1), asthenia (n = 1), fatigue (n = 2), and dehydration (n = 1). Common grade 1/2 treatment-related AEs in patients who received the recommended phase 2 dose were nausea (n = 6), anorexia (n = 5), weight loss (n = 5), vomiting (n = 4), diarrhea (n = 4), and fatigue (n = 4).

Efficacy

In the 20 patients evaluable for response, the ORR was 60% (n = 12). One patient achieved a stringent complete response, three had a very good partial response, and eight had a partial response. The median time to response was 1 month.

The ORR was 92% (11/12) in lenalidomide-naive patients and 13% (1/8) in lenalidomide-exposed patients. The single responder in the lenalidomide-exposed group achieved a partial response.

Among lenalidomide-naive patients who received the recommended phase 2 dose, the median time on treatment was 12 months. The patient who achieved a stringent complete response remained on treatment at last follow-up, as did one partial responder and one patient with a very good partial response.

These results suggest the combination of selinexor, lenalidomide, and dexamethasone “is active, relatively well tolerated, and could warrant further investigation,” Dr. White said.

The STOMP trial is sponsored by Karyopharm Therapeutics. Dr. White disclosed relationships with Karyopharm, Amgen, Celgene, Janssen, Sanofi, and Takeda.

[email protected]

SOURCE: White D et al. IMW 2019, Abstract OAB-083.

BOSTON — Daratumumab plus bortezomib, lenalidomide, and dexamethasone (D-RVd) may be a new standard of care for transplant-eligible patients with newly diagnosed multiple myeloma, according to a speaker at the International Myeloma Workshop.

Jennifer Smith/MDedge News

In the phase 2 GRIFFIN trial, adding daratumumab to RVd deepened responses at all time points and improved rates of stringent complete response and minimal residual disease (MRD) negativity post consolidation.

These results might convince “early adopters of therapy” to change their practice, said Peter M. Voorhees, MD, of Levine Cancer Institute at Atrium Health in Charlotte, N.C., who presented results from GRIFFIN as a late-breaking abstract at the workshop, which is held by the International Myeloma Society.

“But I think you do have to be careful,” Dr. Voorhees added. “We’ll have to see how these patients do over time. Is the MRD sustained, and does that MRD negativity improve progression-free survival?”

Dr. Voorhees presented data on 207 adults with transplant-eligible, newly diagnosed multiple myeloma who were enrolled in the GRIFFIN trial. The patients received RVd, with or without daratumumab, for induction (cycles 1-4). They received granulocyte colony stimulating factor, with or without plerixafor, for stem cell mobilization, and melphalan for conditioning prior to transplant.

Patients received consolidation with D-RVd or RVd (cycles 5-6) and maintenance with lenalidomide alone or in combination with daratumumab (cycles 7-32). Patients could continue maintenance with lenalidomide alone beyond cycle 32.

The D-RVd arm comprised 104 patients, and the RVd arm comprised 103 patients. Baseline characteristics were well balanced between the treatment arms. The median age was 59 years (range, 29-70 years) in the D-RVd arm and 61 years (range, 40-70 years) in the RVd arm.

Most patients had stage I (47% in the D-RVd arm and 49% in the RVd arm) or stage II disease (39% and 36%, respectively) according to the International Staging System. And most patients had standard risk cytogenetics (84% and 86%, respectively).

Response, MRD, and engraftment

The study’s primary endpoint was stringent complete response by the end of consolidation, which was achieved by 42.4% of patients in the D-RVd arm and 32.0% in the RVd arm (odds ratio [OR] = 1.57; P = .068). The overall response rate at that time point was 99.0% and 91.8%, respectively (P = .0160).

Responses deepened over time, and response rates were greater for D-RVd than for RVd at all time points. The complete response rate was 19.2% in the D-RVd arm and 13.4% in the RVd arm at the end of induction; 27.3% and 19.6%, respectively, at the end of transplant; 51.5% and 42.3%, respectively, at the end of consolidation; and 62.6% and 47.4%, respectively, at the clinical cutoff.

D-RVd also improved MRD negativity (10^-5) rates at the end of consolidation. MRD negativity was 44.2% in the D-RVd arm and 14.6% in the RVd arm (OR = 4.70; P less than .0001). The rate of MRD negativity in patients with a complete response or better was 28.8% and 9.7%, respectively (OR = 3.73; P = .0007).

Dr. Voorhees noted that D-RVd was favored across all subgroups for MRD negativity and stringent compete response, except among patients with high-risk cytogenetics and stage III disease.

He also pointed out that stem cell mobilization was “feasible” in the D-RVd arm, and daratumumab did not impact engraftment. The median time to neutrophil engraftment was 12 days in both treatment arms. The median time to platelet engraftment was 13 days in the D-RVd arm and 12 days in the RVd arm.

Safety

“The adverse events are what you would expect,” Dr. Voorhees said. “Grade 3 and 4 neutropenia and thrombocytopenia were seen more often in the dara arm of the trial compared to the RVd arm.”

The most common grade 3/4 treatment-emergent adverse events (in the D-RVd and RVd arms, respectively) were neutropenia (32% and 15%), lymphopenia (23% in both), thrombocytopenia (16% and 8%), and leukopenia (15% and 7%).

“Nonhematologic toxicities were generally equal between the two groups, but I do want to stress that there was a higher rate of infection in the dara arm,” Dr. Voorhees noted.

The incidence of infection was 82% in the D-RVd arm and 55% in the RVd arm, but the rate of grade 3/4 infection was 17% in both arms. The rate of pneumonia was 10% in the D-RVd arm and 9% in the RVd arm.

All-grade infusion-related reactions occurred in 41% of patients in the D-RVd arm, and grade 3/4 infusion-related reactions occurred in 5%.

The trial was sponsored by Janssen. Dr. Voorhees reported relationships with Janssen and several other companies.

[email protected]

SOURCE: Voorhees PM et al. IMW 2019. Abstract OAB-087.

BOSTON — Daratumumab plus bortezomib, lenalidomide, and dexamethasone (D-RVd) may be a new standard of care for transplant-eligible patients with newly diagnosed multiple myeloma, according to a speaker at the International Myeloma Workshop.

Jennifer Smith/MDedge News

In the phase 2 GRIFFIN trial, adding daratumumab to RVd deepened responses at all time points and improved rates of stringent complete response and minimal residual disease (MRD) negativity post consolidation.

These results might convince “early adopters of therapy” to change their practice, said Peter M. Voorhees, MD, of Levine Cancer Institute at Atrium Health in Charlotte, N.C., who presented results from GRIFFIN as a late-breaking abstract at the workshop, which is held by the International Myeloma Society.

“But I think you do have to be careful,” Dr. Voorhees added. “We’ll have to see how these patients do over time. Is the MRD sustained, and does that MRD negativity improve progression-free survival?”

Dr. Voorhees presented data on 207 adults with transplant-eligible, newly diagnosed multiple myeloma who were enrolled in the GRIFFIN trial. The patients received RVd, with or without daratumumab, for induction (cycles 1-4). They received granulocyte colony stimulating factor, with or without plerixafor, for stem cell mobilization, and melphalan for conditioning prior to transplant.

Patients received consolidation with D-RVd or RVd (cycles 5-6) and maintenance with lenalidomide alone or in combination with daratumumab (cycles 7-32). Patients could continue maintenance with lenalidomide alone beyond cycle 32.

The D-RVd arm comprised 104 patients, and the RVd arm comprised 103 patients. Baseline characteristics were well balanced between the treatment arms. The median age was 59 years (range, 29-70 years) in the D-RVd arm and 61 years (range, 40-70 years) in the RVd arm.

Most patients had stage I (47% in the D-RVd arm and 49% in the RVd arm) or stage II disease (39% and 36%, respectively) according to the International Staging System. And most patients had standard risk cytogenetics (84% and 86%, respectively).

Response, MRD, and engraftment

The study’s primary endpoint was stringent complete response by the end of consolidation, which was achieved by 42.4% of patients in the D-RVd arm and 32.0% in the RVd arm (odds ratio [OR] = 1.57; P = .068). The overall response rate at that time point was 99.0% and 91.8%, respectively (P = .0160).

Responses deepened over time, and response rates were greater for D-RVd than for RVd at all time points. The complete response rate was 19.2% in the D-RVd arm and 13.4% in the RVd arm at the end of induction; 27.3% and 19.6%, respectively, at the end of transplant; 51.5% and 42.3%, respectively, at the end of consolidation; and 62.6% and 47.4%, respectively, at the clinical cutoff.

D-RVd also improved MRD negativity (10^-5) rates at the end of consolidation. MRD negativity was 44.2% in the D-RVd arm and 14.6% in the RVd arm (OR = 4.70; P less than .0001). The rate of MRD negativity in patients with a complete response or better was 28.8% and 9.7%, respectively (OR = 3.73; P = .0007).

Dr. Voorhees noted that D-RVd was favored across all subgroups for MRD negativity and stringent compete response, except among patients with high-risk cytogenetics and stage III disease.

He also pointed out that stem cell mobilization was “feasible” in the D-RVd arm, and daratumumab did not impact engraftment. The median time to neutrophil engraftment was 12 days in both treatment arms. The median time to platelet engraftment was 13 days in the D-RVd arm and 12 days in the RVd arm.

Safety

“The adverse events are what you would expect,” Dr. Voorhees said. “Grade 3 and 4 neutropenia and thrombocytopenia were seen more often in the dara arm of the trial compared to the RVd arm.”

The most common grade 3/4 treatment-emergent adverse events (in the D-RVd and RVd arms, respectively) were neutropenia (32% and 15%), lymphopenia (23% in both), thrombocytopenia (16% and 8%), and leukopenia (15% and 7%).

“Nonhematologic toxicities were generally equal between the two groups, but I do want to stress that there was a higher rate of infection in the dara arm,” Dr. Voorhees noted.

The incidence of infection was 82% in the D-RVd arm and 55% in the RVd arm, but the rate of grade 3/4 infection was 17% in both arms. The rate of pneumonia was 10% in the D-RVd arm and 9% in the RVd arm.

All-grade infusion-related reactions occurred in 41% of patients in the D-RVd arm, and grade 3/4 infusion-related reactions occurred in 5%.

The trial was sponsored by Janssen. Dr. Voorhees reported relationships with Janssen and several other companies.

[email protected]

SOURCE: Voorhees PM et al. IMW 2019. Abstract OAB-087.

BOSTON — Daratumumab plus bortezomib, lenalidomide, and dexamethasone (D-RVd) may be a new standard of care for transplant-eligible patients with newly diagnosed multiple myeloma, according to a speaker at the International Myeloma Workshop.

Jennifer Smith/MDedge News

In the phase 2 GRIFFIN trial, adding daratumumab to RVd deepened responses at all time points and improved rates of stringent complete response and minimal residual disease (MRD) negativity post consolidation.

These results might convince “early adopters of therapy” to change their practice, said Peter M. Voorhees, MD, of Levine Cancer Institute at Atrium Health in Charlotte, N.C., who presented results from GRIFFIN as a late-breaking abstract at the workshop, which is held by the International Myeloma Society.

“But I think you do have to be careful,” Dr. Voorhees added. “We’ll have to see how these patients do over time. Is the MRD sustained, and does that MRD negativity improve progression-free survival?”

Dr. Voorhees presented data on 207 adults with transplant-eligible, newly diagnosed multiple myeloma who were enrolled in the GRIFFIN trial. The patients received RVd, with or without daratumumab, for induction (cycles 1-4). They received granulocyte colony stimulating factor, with or without plerixafor, for stem cell mobilization, and melphalan for conditioning prior to transplant.

Patients received consolidation with D-RVd or RVd (cycles 5-6) and maintenance with lenalidomide alone or in combination with daratumumab (cycles 7-32). Patients could continue maintenance with lenalidomide alone beyond cycle 32.

The D-RVd arm comprised 104 patients, and the RVd arm comprised 103 patients. Baseline characteristics were well balanced between the treatment arms. The median age was 59 years (range, 29-70 years) in the D-RVd arm and 61 years (range, 40-70 years) in the RVd arm.

Most patients had stage I (47% in the D-RVd arm and 49% in the RVd arm) or stage II disease (39% and 36%, respectively) according to the International Staging System. And most patients had standard risk cytogenetics (84% and 86%, respectively).

Response, MRD, and engraftment

The study’s primary endpoint was stringent complete response by the end of consolidation, which was achieved by 42.4% of patients in the D-RVd arm and 32.0% in the RVd arm (odds ratio [OR] = 1.57; P = .068). The overall response rate at that time point was 99.0% and 91.8%, respectively (P = .0160).

Responses deepened over time, and response rates were greater for D-RVd than for RVd at all time points. The complete response rate was 19.2% in the D-RVd arm and 13.4% in the RVd arm at the end of induction; 27.3% and 19.6%, respectively, at the end of transplant; 51.5% and 42.3%, respectively, at the end of consolidation; and 62.6% and 47.4%, respectively, at the clinical cutoff.

D-RVd also improved MRD negativity (10^-5) rates at the end of consolidation. MRD negativity was 44.2% in the D-RVd arm and 14.6% in the RVd arm (OR = 4.70; P less than .0001). The rate of MRD negativity in patients with a complete response or better was 28.8% and 9.7%, respectively (OR = 3.73; P = .0007).

Dr. Voorhees noted that D-RVd was favored across all subgroups for MRD negativity and stringent compete response, except among patients with high-risk cytogenetics and stage III disease.

He also pointed out that stem cell mobilization was “feasible” in the D-RVd arm, and daratumumab did not impact engraftment. The median time to neutrophil engraftment was 12 days in both treatment arms. The median time to platelet engraftment was 13 days in the D-RVd arm and 12 days in the RVd arm.

Safety

“The adverse events are what you would expect,” Dr. Voorhees said. “Grade 3 and 4 neutropenia and thrombocytopenia were seen more often in the dara arm of the trial compared to the RVd arm.”

The most common grade 3/4 treatment-emergent adverse events (in the D-RVd and RVd arms, respectively) were neutropenia (32% and 15%), lymphopenia (23% in both), thrombocytopenia (16% and 8%), and leukopenia (15% and 7%).

“Nonhematologic toxicities were generally equal between the two groups, but I do want to stress that there was a higher rate of infection in the dara arm,” Dr. Voorhees noted.

The incidence of infection was 82% in the D-RVd arm and 55% in the RVd arm, but the rate of grade 3/4 infection was 17% in both arms. The rate of pneumonia was 10% in the D-RVd arm and 9% in the RVd arm.

All-grade infusion-related reactions occurred in 41% of patients in the D-RVd arm, and grade 3/4 infusion-related reactions occurred in 5%.

The trial was sponsored by Janssen. Dr. Voorhees reported relationships with Janssen and several other companies.

[email protected]

SOURCE: Voorhees PM et al. IMW 2019. Abstract OAB-087.

BOSTON – Adding elotuzumab to lenalidomide and dexamethasone can prolong overall survival in patients with relapsed/refractory multiple myeloma, according to final results from the ELOQUENT-2 trial.

At a minimum follow-up of 6 years, elotuzumab plus lenalidomide/dexamethasone (ELd) reduced the risk of death by 18% and prolonged the median overall survival by 8.7 months when compared to treatment with lenalidomide/dexamethasone (Ld).

“The combination of elotuzumab with lenalidomide and dexamethasone demonstrated a statistically significant and clinically meaningful 18% reduction in the risk of death,” said Meletios A. Dimopoulos, MD, PhD, of the National and Kapodistrian University of Athens. “This treatment combination is the only approved antibody-based regimen shown to prolong overall survival significantly in patients with relapsed or refractory myeloma in the context of a large, prospective, randomized trial.”

Dr. Dimopoulos presented results from this phase 3 trial at the International Myeloma Workshop, held by the International Myeloma Society.

The final analysis of ELOQUENT-2 included 646 patients with relapsed/refractory multiple myeloma who had received one to three prior lines of therapy at baseline. There were 321 patients randomized to ELd and 325 randomized to Ld. Baseline characteristics were well balanced between the treatment arms.

At the data cutoff of Oct. 3, 2018, 319 patients in the ELd arm and 316 in the Ld arm had received their assigned treatment. Ten percent (n = 33) of patients in the ELd arm and 4% (n = 14) in the Ld arm were still receiving their assigned treatment at the cutoff date.

The median number of treatment cycles was 19 (range, 9-42) in the ELd arm and 14 (range, 6-25) in the Ld arm. Most patients discontinued treatment due to disease progression (56% in the ELd arm and 57% in the Ld arm) or treatment-related toxicity (12% and 14%, respectively).
 

Survival

At a minimum follow-up of 71 months, the median overall survival was 48.3 months in the ELd arm and 39.6 months in the Ld arm. The hazard ratio was 0.82 (P = .0408).

The overall survival advantage with ELd was observed in all prespecified patient subgroups, Dr. Dimopoulos said. For example, overall survival favored ELd in patients aged 75 years and older (HR, 0.69), patients with International Staging System stage III disease at enrollment (HR, 0.74), those who had received two to three prior lines of therapy (HR, 0.71), and patients with del(17p) (HR, 0.71).

There were 212 deaths in the ELd arm and 225 in the Ld arm (67% and 71%, respectively). The most common causes of death were disease progression (41% in the ELd arm and 45% in the Ld arm), infection (9% and 6%, respectively), and “other” or unknown causes (7% and 9%, respectively). Two percent of patients in each arm (n = 7 in both) died from study treatment–related toxicity.

Dr. Dimopoulos pointed out that there were no imbalances in subsequent therapies between the ELd and Ld arms. The most common subsequent therapies (in the ELd and Ld arms, respectively) were bortezomib (38% and 42%), cyclophosphamide (30% in both arms), pomalidomide (26% and 29%), and lenalidomide (18% and 22%).

Safety

“Most of the adverse events which occurred throughout the study were due to the known side effects of lenalidomide and dexamethasone,” Dr. Dimopoulos said. “The contribution of elotuzumab to any kind of toxicity was minimal.”

Nearly all patients in both arms (99%) experienced adverse events. Serious adverse events were observed in 75% of patients in the ELd arm and 61% in the Ld arm. Adverse events leading to treatment discontinuation occurred in 36% and 33%, respectively, and grade 3-4 events leading to discontinuation occurred in 21% and 20%, respectively.

Grade 3-4 adverse events of special interest (in the ELd and Ld arms, respectively) were infections (35% and 27%), renal and urinary disorders (5% in both), cardiac disorders (6% and 8%), and lymphopenia (8% and 4%). Second primary malignancies occurred in 12% of patients in the ELd arm and 9% in the Ld arm.

This trial was sponsored by Bristol-Myers Squibb in collaboration with AbbVie. Dr. Dimopoulos reported relationships with Bristol-Myers Squibb, Amgen, Celgene, Janssen, and Takeda.

[email protected]

SOURCE: Dimopoulos MA et al. IMW 2019, Abstract OAB-021.

BOSTON – Adding elotuzumab to lenalidomide and dexamethasone can prolong overall survival in patients with relapsed/refractory multiple myeloma, according to final results from the ELOQUENT-2 trial.

At a minimum follow-up of 6 years, elotuzumab plus lenalidomide/dexamethasone (ELd) reduced the risk of death by 18% and prolonged the median overall survival by 8.7 months when compared to treatment with lenalidomide/dexamethasone (Ld).

“The combination of elotuzumab with lenalidomide and dexamethasone demonstrated a statistically significant and clinically meaningful 18% reduction in the risk of death,” said Meletios A. Dimopoulos, MD, PhD, of the National and Kapodistrian University of Athens. “This treatment combination is the only approved antibody-based regimen shown to prolong overall survival significantly in patients with relapsed or refractory myeloma in the context of a large, prospective, randomized trial.”

Dr. Dimopoulos presented results from this phase 3 trial at the International Myeloma Workshop, held by the International Myeloma Society.

The final analysis of ELOQUENT-2 included 646 patients with relapsed/refractory multiple myeloma who had received one to three prior lines of therapy at baseline. There were 321 patients randomized to ELd and 325 randomized to Ld. Baseline characteristics were well balanced between the treatment arms.

At the data cutoff of Oct. 3, 2018, 319 patients in the ELd arm and 316 in the Ld arm had received their assigned treatment. Ten percent (n = 33) of patients in the ELd arm and 4% (n = 14) in the Ld arm were still receiving their assigned treatment at the cutoff date.

The median number of treatment cycles was 19 (range, 9-42) in the ELd arm and 14 (range, 6-25) in the Ld arm. Most patients discontinued treatment due to disease progression (56% in the ELd arm and 57% in the Ld arm) or treatment-related toxicity (12% and 14%, respectively).
 

Survival

At a minimum follow-up of 71 months, the median overall survival was 48.3 months in the ELd arm and 39.6 months in the Ld arm. The hazard ratio was 0.82 (P = .0408).

The overall survival advantage with ELd was observed in all prespecified patient subgroups, Dr. Dimopoulos said. For example, overall survival favored ELd in patients aged 75 years and older (HR, 0.69), patients with International Staging System stage III disease at enrollment (HR, 0.74), those who had received two to three prior lines of therapy (HR, 0.71), and patients with del(17p) (HR, 0.71).

There were 212 deaths in the ELd arm and 225 in the Ld arm (67% and 71%, respectively). The most common causes of death were disease progression (41% in the ELd arm and 45% in the Ld arm), infection (9% and 6%, respectively), and “other” or unknown causes (7% and 9%, respectively). Two percent of patients in each arm (n = 7 in both) died from study treatment–related toxicity.

Dr. Dimopoulos pointed out that there were no imbalances in subsequent therapies between the ELd and Ld arms. The most common subsequent therapies (in the ELd and Ld arms, respectively) were bortezomib (38% and 42%), cyclophosphamide (30% in both arms), pomalidomide (26% and 29%), and lenalidomide (18% and 22%).

Safety

“Most of the adverse events which occurred throughout the study were due to the known side effects of lenalidomide and dexamethasone,” Dr. Dimopoulos said. “The contribution of elotuzumab to any kind of toxicity was minimal.”

Nearly all patients in both arms (99%) experienced adverse events. Serious adverse events were observed in 75% of patients in the ELd arm and 61% in the Ld arm. Adverse events leading to treatment discontinuation occurred in 36% and 33%, respectively, and grade 3-4 events leading to discontinuation occurred in 21% and 20%, respectively.

Grade 3-4 adverse events of special interest (in the ELd and Ld arms, respectively) were infections (35% and 27%), renal and urinary disorders (5% in both), cardiac disorders (6% and 8%), and lymphopenia (8% and 4%). Second primary malignancies occurred in 12% of patients in the ELd arm and 9% in the Ld arm.

This trial was sponsored by Bristol-Myers Squibb in collaboration with AbbVie. Dr. Dimopoulos reported relationships with Bristol-Myers Squibb, Amgen, Celgene, Janssen, and Takeda.

[email protected]

SOURCE: Dimopoulos MA et al. IMW 2019, Abstract OAB-021.

BOSTON – Adding elotuzumab to lenalidomide and dexamethasone can prolong overall survival in patients with relapsed/refractory multiple myeloma, according to final results from the ELOQUENT-2 trial.

At a minimum follow-up of 6 years, elotuzumab plus lenalidomide/dexamethasone (ELd) reduced the risk of death by 18% and prolonged the median overall survival by 8.7 months when compared to treatment with lenalidomide/dexamethasone (Ld).

“The combination of elotuzumab with lenalidomide and dexamethasone demonstrated a statistically significant and clinically meaningful 18% reduction in the risk of death,” said Meletios A. Dimopoulos, MD, PhD, of the National and Kapodistrian University of Athens. “This treatment combination is the only approved antibody-based regimen shown to prolong overall survival significantly in patients with relapsed or refractory myeloma in the context of a large, prospective, randomized trial.”

Dr. Dimopoulos presented results from this phase 3 trial at the International Myeloma Workshop, held by the International Myeloma Society.

The final analysis of ELOQUENT-2 included 646 patients with relapsed/refractory multiple myeloma who had received one to three prior lines of therapy at baseline. There were 321 patients randomized to ELd and 325 randomized to Ld. Baseline characteristics were well balanced between the treatment arms.

At the data cutoff of Oct. 3, 2018, 319 patients in the ELd arm and 316 in the Ld arm had received their assigned treatment. Ten percent (n = 33) of patients in the ELd arm and 4% (n = 14) in the Ld arm were still receiving their assigned treatment at the cutoff date.

The median number of treatment cycles was 19 (range, 9-42) in the ELd arm and 14 (range, 6-25) in the Ld arm. Most patients discontinued treatment due to disease progression (56% in the ELd arm and 57% in the Ld arm) or treatment-related toxicity (12% and 14%, respectively).
 

Survival

At a minimum follow-up of 71 months, the median overall survival was 48.3 months in the ELd arm and 39.6 months in the Ld arm. The hazard ratio was 0.82 (P = .0408).

The overall survival advantage with ELd was observed in all prespecified patient subgroups, Dr. Dimopoulos said. For example, overall survival favored ELd in patients aged 75 years and older (HR, 0.69), patients with International Staging System stage III disease at enrollment (HR, 0.74), those who had received two to three prior lines of therapy (HR, 0.71), and patients with del(17p) (HR, 0.71).

There were 212 deaths in the ELd arm and 225 in the Ld arm (67% and 71%, respectively). The most common causes of death were disease progression (41% in the ELd arm and 45% in the Ld arm), infection (9% and 6%, respectively), and “other” or unknown causes (7% and 9%, respectively). Two percent of patients in each arm (n = 7 in both) died from study treatment–related toxicity.

Dr. Dimopoulos pointed out that there were no imbalances in subsequent therapies between the ELd and Ld arms. The most common subsequent therapies (in the ELd and Ld arms, respectively) were bortezomib (38% and 42%), cyclophosphamide (30% in both arms), pomalidomide (26% and 29%), and lenalidomide (18% and 22%).

Safety

“Most of the adverse events which occurred throughout the study were due to the known side effects of lenalidomide and dexamethasone,” Dr. Dimopoulos said. “The contribution of elotuzumab to any kind of toxicity was minimal.”

Nearly all patients in both arms (99%) experienced adverse events. Serious adverse events were observed in 75% of patients in the ELd arm and 61% in the Ld arm. Adverse events leading to treatment discontinuation occurred in 36% and 33%, respectively, and grade 3-4 events leading to discontinuation occurred in 21% and 20%, respectively.

Grade 3-4 adverse events of special interest (in the ELd and Ld arms, respectively) were infections (35% and 27%), renal and urinary disorders (5% in both), cardiac disorders (6% and 8%), and lymphopenia (8% and 4%). Second primary malignancies occurred in 12% of patients in the ELd arm and 9% in the Ld arm.

This trial was sponsored by Bristol-Myers Squibb in collaboration with AbbVie. Dr. Dimopoulos reported relationships with Bristol-Myers Squibb, Amgen, Celgene, Janssen, and Takeda.

[email protected]

SOURCE: Dimopoulos MA et al. IMW 2019, Abstract OAB-021.

BOSTON – CT103A, a chimeric antigen receptor (CAR) T-cell therapy, is “active and effective” in patients with relapsed/refractory multiple myeloma, according to a speaker at the International Myeloma Workshop, held by the International Myeloma Society.

Jennifer Smith/MDedge News

The anti–B-cell maturation antigen (BCMA) CAR T-cell therapy produced a 100% response rate in patients with heavily pretreated multiple myeloma, and three of four patients who had failed a prior CAR T-cell therapy achieved a stringent complete response after CT103A.

Chunrui Li, MD, PhD, of Tongji Hospital and Tongji Medical College, Huazhong University of Science, Wuhan, China, presented these results at the workshop.

Dr. Li noted that anti-BCMA CAR T-cell therapy has produced responses in myeloma patients, but approximately half of patients typically relapse in about a year. CAR T-cell infusions after relapse have not been effective in these patients.

In an effort to change that, Dr. Li and his colleagues developed CT103A, a lentiviral vector containing a CAR structure with a fully human single-chain fragment variant; CD8a hinger; and transmembrane, 4-1BB co-stimulatory, and CD3z activation domains.

Dr. Li and his colleagues evaluated CT103A in a phase 0 trial (ChiCTR1800018137) of 18 patients who had received at least three prior lines of therapy and had disease refractory to a proteasome inhibitor and an immunomodulatory agent.

The patients’ median age was 53.3 years (range, 38-66 years), and their median time since diagnosis was 32 months (range, 8-92 months). They had received a median of 4 (range, 3-6) prior therapies. All had received prior bortezomib and lenalidomide, seven had undergone a transplant, and four had been treated on a trial of murine anti-BCMA CAR T-cell therapy.

For the current trial, patients received lymphodepletion with cyclophosphamide and fludarabine, followed by CT103A at 1x10⁶, 3x10⁶, or 6x10⁶ CAR T cells/kg.

There was one dose-limiting toxicity at the highest dose level – grade 4 cytokine release syndrome (CRS) in a patient who died at day 19 after CT103A infusion.

In all, 17 patients developed CRS, four with grade 1, eight with grade 2, four with grade 3, and one with grade 4 CRS. None of the patients developed neurologic toxicity.

Serious adverse events related to lymphodepletion and/or CT103A included prolonged cytopenia (n = 3), pulmonary infection (n = 2), herpes zoster (n = 1), pleuritis (n = 1), and hypoxemia (n = 1).

There were 17 patients evaluable for efficacy, and all of them achieved a response at some point. In eight patients, responses have lasted more than 200 days.

At the data cutoff, there were 10 stringent complete responses, two complete responses, and three very good partial responses. One patient progressed after achieving a very good partial response, and one patient achieved a partial response but ultimately died (likely of respiratory failure attributable to a lung infection).

Of the four patients who had previously received murine CAR T-cell therapy, one progressed, and three achieved a stringent complete response.

This study was funded by Nanjing Iaso Biotherapeutics. Dr. Li did not disclose any conflicts of interest.

[email protected]

SOURCE: Li C et al. IMW 2019, Abstract OAB-033.

BOSTON – CT103A, a chimeric antigen receptor (CAR) T-cell therapy, is “active and effective” in patients with relapsed/refractory multiple myeloma, according to a speaker at the International Myeloma Workshop, held by the International Myeloma Society.

Jennifer Smith/MDedge News

The anti–B-cell maturation antigen (BCMA) CAR T-cell therapy produced a 100% response rate in patients with heavily pretreated multiple myeloma, and three of four patients who had failed a prior CAR T-cell therapy achieved a stringent complete response after CT103A.

Chunrui Li, MD, PhD, of Tongji Hospital and Tongji Medical College, Huazhong University of Science, Wuhan, China, presented these results at the workshop.

Dr. Li noted that anti-BCMA CAR T-cell therapy has produced responses in myeloma patients, but approximately half of patients typically relapse in about a year. CAR T-cell infusions after relapse have not been effective in these patients.

In an effort to change that, Dr. Li and his colleagues developed CT103A, a lentiviral vector containing a CAR structure with a fully human single-chain fragment variant; CD8a hinger; and transmembrane, 4-1BB co-stimulatory, and CD3z activation domains.

Dr. Li and his colleagues evaluated CT103A in a phase 0 trial (ChiCTR1800018137) of 18 patients who had received at least three prior lines of therapy and had disease refractory to a proteasome inhibitor and an immunomodulatory agent.

The patients’ median age was 53.3 years (range, 38-66 years), and their median time since diagnosis was 32 months (range, 8-92 months). They had received a median of 4 (range, 3-6) prior therapies. All had received prior bortezomib and lenalidomide, seven had undergone a transplant, and four had been treated on a trial of murine anti-BCMA CAR T-cell therapy.

For the current trial, patients received lymphodepletion with cyclophosphamide and fludarabine, followed by CT103A at 1x10⁶, 3x10⁶, or 6x10⁶ CAR T cells/kg.

There was one dose-limiting toxicity at the highest dose level – grade 4 cytokine release syndrome (CRS) in a patient who died at day 19 after CT103A infusion.

In all, 17 patients developed CRS, four with grade 1, eight with grade 2, four with grade 3, and one with grade 4 CRS. None of the patients developed neurologic toxicity.

Serious adverse events related to lymphodepletion and/or CT103A included prolonged cytopenia (n = 3), pulmonary infection (n = 2), herpes zoster (n = 1), pleuritis (n = 1), and hypoxemia (n = 1).

There were 17 patients evaluable for efficacy, and all of them achieved a response at some point. In eight patients, responses have lasted more than 200 days.

At the data cutoff, there were 10 stringent complete responses, two complete responses, and three very good partial responses. One patient progressed after achieving a very good partial response, and one patient achieved a partial response but ultimately died (likely of respiratory failure attributable to a lung infection).

Of the four patients who had previously received murine CAR T-cell therapy, one progressed, and three achieved a stringent complete response.

This study was funded by Nanjing Iaso Biotherapeutics. Dr. Li did not disclose any conflicts of interest.

[email protected]

SOURCE: Li C et al. IMW 2019, Abstract OAB-033.

BOSTON – CT103A, a chimeric antigen receptor (CAR) T-cell therapy, is “active and effective” in patients with relapsed/refractory multiple myeloma, according to a speaker at the International Myeloma Workshop, held by the International Myeloma Society.

Jennifer Smith/MDedge News

The anti–B-cell maturation antigen (BCMA) CAR T-cell therapy produced a 100% response rate in patients with heavily pretreated multiple myeloma, and three of four patients who had failed a prior CAR T-cell therapy achieved a stringent complete response after CT103A.

Chunrui Li, MD, PhD, of Tongji Hospital and Tongji Medical College, Huazhong University of Science, Wuhan, China, presented these results at the workshop.

Dr. Li noted that anti-BCMA CAR T-cell therapy has produced responses in myeloma patients, but approximately half of patients typically relapse in about a year. CAR T-cell infusions after relapse have not been effective in these patients.

In an effort to change that, Dr. Li and his colleagues developed CT103A, a lentiviral vector containing a CAR structure with a fully human single-chain fragment variant; CD8a hinger; and transmembrane, 4-1BB co-stimulatory, and CD3z activation domains.

Dr. Li and his colleagues evaluated CT103A in a phase 0 trial (ChiCTR1800018137) of 18 patients who had received at least three prior lines of therapy and had disease refractory to a proteasome inhibitor and an immunomodulatory agent.

The patients’ median age was 53.3 years (range, 38-66 years), and their median time since diagnosis was 32 months (range, 8-92 months). They had received a median of 4 (range, 3-6) prior therapies. All had received prior bortezomib and lenalidomide, seven had undergone a transplant, and four had been treated on a trial of murine anti-BCMA CAR T-cell therapy.

For the current trial, patients received lymphodepletion with cyclophosphamide and fludarabine, followed by CT103A at 1x10⁶, 3x10⁶, or 6x10⁶ CAR T cells/kg.

There was one dose-limiting toxicity at the highest dose level – grade 4 cytokine release syndrome (CRS) in a patient who died at day 19 after CT103A infusion.

In all, 17 patients developed CRS, four with grade 1, eight with grade 2, four with grade 3, and one with grade 4 CRS. None of the patients developed neurologic toxicity.

Serious adverse events related to lymphodepletion and/or CT103A included prolonged cytopenia (n = 3), pulmonary infection (n = 2), herpes zoster (n = 1), pleuritis (n = 1), and hypoxemia (n = 1).

There were 17 patients evaluable for efficacy, and all of them achieved a response at some point. In eight patients, responses have lasted more than 200 days.

At the data cutoff, there were 10 stringent complete responses, two complete responses, and three very good partial responses. One patient progressed after achieving a very good partial response, and one patient achieved a partial response but ultimately died (likely of respiratory failure attributable to a lung infection).

Of the four patients who had previously received murine CAR T-cell therapy, one progressed, and three achieved a stringent complete response.

This study was funded by Nanjing Iaso Biotherapeutics. Dr. Li did not disclose any conflicts of interest.

[email protected]

SOURCE: Li C et al. IMW 2019, Abstract OAB-033.

BOSTON – Melflufen plus dexamethasone is active in patients with relapsed/refractory multiple myeloma, whether or not they have extramedullary disease (EMD), a phase 2 trial suggests.

Jennifer Smith/MDedge News

In the HORIZON trial, melflufen-dexamethasone produced an overall response rate of 23% in patients with EMD and 27% in those without EMD.

Paul G. Richardson, MD, of Harvard Medical School and the Dana-Farber Cancer Institute, both in Boston, presented these results as a late-breaking abstract at the International Myeloma Workshop, held by the International Myeloma Society.

As of July 30, 2019, 136 patients had been treated on the HORIZON trial. The trial is enrolling patients with relapsed/refractory multiple myeloma refractory to pomalidomide, an anti-CD38 monoclonal antibody (mAb), or both. The patients must have received at least two prior lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD).

Dr. Richardson presented results for 130 patients, 44 with EMD and 86 without it. The median age at baseline was 64 years in the EMD and non-EMD groups (overall range, 35-86 years). More than half of patients had high-risk cytogenetics (52% in the EMD group and 57% in the non-EMD group).

The median number of prior therapies was five in both the EMD and non-EMD groups. Most patients had received at least one prior transplant (73% in the EMD group and 69% in the non-EMD group). Most patients in both groups were refractory to an anti-CD38 mAb (93% EMD and 72% non-EMD); an IMiD and a PI (93% EMD and 90% non-EMD); an IMiD, a PI, and an anti-CD38 mAb (91% EMD and 63% non-EMD); and their last therapy (100% EMD and 95% non-EMD).

Dr. Richardson pointed out that the incidence of EMD in this trial is higher than has been reported previously. Of the 44 EMD patients, 26 had soft-tissue EMD, and 18 had bone-related EMD. Five patients had CNS involvement.

Another key finding, according to Dr. Richardson, was that EMD appeared to be associated with prior anti-CD38 therapy. Specifically, 40% of patients exposed to an anti-CD38 mAb had EMD, compared with 11% of patients who had not received an anti-CD38 mAb (P = .01).

“I don’t for a minute want to say that CD38-targeted therapy engenders extramedullary disease,” Dr. Richardson said. “I think what we can say, though, is that, once CD38 treatment fails a patient, extramedullary disease … is a very real challenge. Therefore, we need rationally targeted approaches, ideally in combination, to meet that challenge.”

The overall response rate was similar for EMD and non-EMD patients – 23% and 27%, respectively. The median duration of response was 3.4 months in the EMD patients and 4.4 months in the non-EMD group. The clinical benefit rate was 30% and 45%, respectively.

In the EMD group, the overall response rate was 19% in patients with soft-tissue EMD and 28% in bone-related EMD. None of the patients with CNS disease responded.

The median progression-free survival was 2.9 months for patients with EMD and 4.6 months for those without EMD. The median overall survival was 5.8 month and 11.6 months, respectively.

The median overall survival was 18.5 months in EMD responders and 17.2 months in non-EMD responders. The median overall survival was 5.1 months in EMD nonresponders and 8.5 months in non-EMD nonresponders.

In all, 54% of patients received subsequent therapy. There were no significant differences in outcomes between EMD and non-EMD patients.

The safety profiles were similar for EMD and non-EMD patients, Dr. Richardson said. Melflufen-dexamethasone was considered well tolerated overall, and there were no treatment-related deaths.

The most common treatment-emergent adverse events (grade 3 and 4, respectively) were thrombocytopenia (22% and 46%), neutropenia (32% and 35%), anemia (35% and 1%), white blood cell count decrease (10% and 7%), and pneumonia (7% and 1%).

This trial is sponsored by Oncopeptides. Dr. Richardson reported an advisory role and research funding from Oncopeptides.

[email protected]

SOURCE: Richardson PG et al. IMW 2019, Abstract OAB-086.

BOSTON – Melflufen plus dexamethasone is active in patients with relapsed/refractory multiple myeloma, whether or not they have extramedullary disease (EMD), a phase 2 trial suggests.

Jennifer Smith/MDedge News

In the HORIZON trial, melflufen-dexamethasone produced an overall response rate of 23% in patients with EMD and 27% in those without EMD.

Paul G. Richardson, MD, of Harvard Medical School and the Dana-Farber Cancer Institute, both in Boston, presented these results as a late-breaking abstract at the International Myeloma Workshop, held by the International Myeloma Society.

As of July 30, 2019, 136 patients had been treated on the HORIZON trial. The trial is enrolling patients with relapsed/refractory multiple myeloma refractory to pomalidomide, an anti-CD38 monoclonal antibody (mAb), or both. The patients must have received at least two prior lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD).

Dr. Richardson presented results for 130 patients, 44 with EMD and 86 without it. The median age at baseline was 64 years in the EMD and non-EMD groups (overall range, 35-86 years). More than half of patients had high-risk cytogenetics (52% in the EMD group and 57% in the non-EMD group).

The median number of prior therapies was five in both the EMD and non-EMD groups. Most patients had received at least one prior transplant (73% in the EMD group and 69% in the non-EMD group). Most patients in both groups were refractory to an anti-CD38 mAb (93% EMD and 72% non-EMD); an IMiD and a PI (93% EMD and 90% non-EMD); an IMiD, a PI, and an anti-CD38 mAb (91% EMD and 63% non-EMD); and their last therapy (100% EMD and 95% non-EMD).

Dr. Richardson pointed out that the incidence of EMD in this trial is higher than has been reported previously. Of the 44 EMD patients, 26 had soft-tissue EMD, and 18 had bone-related EMD. Five patients had CNS involvement.

Another key finding, according to Dr. Richardson, was that EMD appeared to be associated with prior anti-CD38 therapy. Specifically, 40% of patients exposed to an anti-CD38 mAb had EMD, compared with 11% of patients who had not received an anti-CD38 mAb (P = .01).

“I don’t for a minute want to say that CD38-targeted therapy engenders extramedullary disease,” Dr. Richardson said. “I think what we can say, though, is that, once CD38 treatment fails a patient, extramedullary disease … is a very real challenge. Therefore, we need rationally targeted approaches, ideally in combination, to meet that challenge.”

The overall response rate was similar for EMD and non-EMD patients – 23% and 27%, respectively. The median duration of response was 3.4 months in the EMD patients and 4.4 months in the non-EMD group. The clinical benefit rate was 30% and 45%, respectively.

In the EMD group, the overall response rate was 19% in patients with soft-tissue EMD and 28% in bone-related EMD. None of the patients with CNS disease responded.

The median progression-free survival was 2.9 months for patients with EMD and 4.6 months for those without EMD. The median overall survival was 5.8 month and 11.6 months, respectively.

The median overall survival was 18.5 months in EMD responders and 17.2 months in non-EMD responders. The median overall survival was 5.1 months in EMD nonresponders and 8.5 months in non-EMD nonresponders.

In all, 54% of patients received subsequent therapy. There were no significant differences in outcomes between EMD and non-EMD patients.

The safety profiles were similar for EMD and non-EMD patients, Dr. Richardson said. Melflufen-dexamethasone was considered well tolerated overall, and there were no treatment-related deaths.

The most common treatment-emergent adverse events (grade 3 and 4, respectively) were thrombocytopenia (22% and 46%), neutropenia (32% and 35%), anemia (35% and 1%), white blood cell count decrease (10% and 7%), and pneumonia (7% and 1%).

This trial is sponsored by Oncopeptides. Dr. Richardson reported an advisory role and research funding from Oncopeptides.

[email protected]

SOURCE: Richardson PG et al. IMW 2019, Abstract OAB-086.

BOSTON – Melflufen plus dexamethasone is active in patients with relapsed/refractory multiple myeloma, whether or not they have extramedullary disease (EMD), a phase 2 trial suggests.

Jennifer Smith/MDedge News

In the HORIZON trial, melflufen-dexamethasone produced an overall response rate of 23% in patients with EMD and 27% in those without EMD.

Paul G. Richardson, MD, of Harvard Medical School and the Dana-Farber Cancer Institute, both in Boston, presented these results as a late-breaking abstract at the International Myeloma Workshop, held by the International Myeloma Society.

As of July 30, 2019, 136 patients had been treated on the HORIZON trial. The trial is enrolling patients with relapsed/refractory multiple myeloma refractory to pomalidomide, an anti-CD38 monoclonal antibody (mAb), or both. The patients must have received at least two prior lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD).

Dr. Richardson presented results for 130 patients, 44 with EMD and 86 without it. The median age at baseline was 64 years in the EMD and non-EMD groups (overall range, 35-86 years). More than half of patients had high-risk cytogenetics (52% in the EMD group and 57% in the non-EMD group).

The median number of prior therapies was five in both the EMD and non-EMD groups. Most patients had received at least one prior transplant (73% in the EMD group and 69% in the non-EMD group). Most patients in both groups were refractory to an anti-CD38 mAb (93% EMD and 72% non-EMD); an IMiD and a PI (93% EMD and 90% non-EMD); an IMiD, a PI, and an anti-CD38 mAb (91% EMD and 63% non-EMD); and their last therapy (100% EMD and 95% non-EMD).

Dr. Richardson pointed out that the incidence of EMD in this trial is higher than has been reported previously. Of the 44 EMD patients, 26 had soft-tissue EMD, and 18 had bone-related EMD. Five patients had CNS involvement.

Another key finding, according to Dr. Richardson, was that EMD appeared to be associated with prior anti-CD38 therapy. Specifically, 40% of patients exposed to an anti-CD38 mAb had EMD, compared with 11% of patients who had not received an anti-CD38 mAb (P = .01).

“I don’t for a minute want to say that CD38-targeted therapy engenders extramedullary disease,” Dr. Richardson said. “I think what we can say, though, is that, once CD38 treatment fails a patient, extramedullary disease … is a very real challenge. Therefore, we need rationally targeted approaches, ideally in combination, to meet that challenge.”

The overall response rate was similar for EMD and non-EMD patients – 23% and 27%, respectively. The median duration of response was 3.4 months in the EMD patients and 4.4 months in the non-EMD group. The clinical benefit rate was 30% and 45%, respectively.

In the EMD group, the overall response rate was 19% in patients with soft-tissue EMD and 28% in bone-related EMD. None of the patients with CNS disease responded.

The median progression-free survival was 2.9 months for patients with EMD and 4.6 months for those without EMD. The median overall survival was 5.8 month and 11.6 months, respectively.

The median overall survival was 18.5 months in EMD responders and 17.2 months in non-EMD responders. The median overall survival was 5.1 months in EMD nonresponders and 8.5 months in non-EMD nonresponders.

In all, 54% of patients received subsequent therapy. There were no significant differences in outcomes between EMD and non-EMD patients.

The safety profiles were similar for EMD and non-EMD patients, Dr. Richardson said. Melflufen-dexamethasone was considered well tolerated overall, and there were no treatment-related deaths.

The most common treatment-emergent adverse events (grade 3 and 4, respectively) were thrombocytopenia (22% and 46%), neutropenia (32% and 35%), anemia (35% and 1%), white blood cell count decrease (10% and 7%), and pneumonia (7% and 1%).

This trial is sponsored by Oncopeptides. Dr. Richardson reported an advisory role and research funding from Oncopeptides.

[email protected]

SOURCE: Richardson PG et al. IMW 2019, Abstract OAB-086.

Can receiving all posttransplant care at home benefit patients undergoing hematopoietic stem cell transplant (HSCT)? Researchers are conducting phase 2 trials to find out.

Nelson Chao, MD, and colleagues at Duke University in Durham, N.C., completed a phase 1 trial that suggested post-HSCT care at home was feasible and safe (Blood. 2017;130:745).

Now, the team is conducting phase 2 trials – NCT01725022 and NCT02218151 – comparing patients who receive all posttransplant care at home with patients treated in the hospital or in the outpatient setting with daily visits to the clinic.

The main goal is to determine if allogeneic HSCT recipients treated at home can maintain their normal microbiome and, as a result, have a lower risk of graft-versus-host disease (GVHD). The researchers are also looking at other outcomes such as quality of life, treatment-related morbidities and mortality, and the cost of care for both allogeneic and autologous transplant recipients.

To be eligible for home care after HSCT, a patient must live within a 90-minute driving distance of Duke and have a caregiver available at home. The patient’s home must pass an inspection, showing it to be free of sources for potential infection, such as mold or pets that sleep in the patient’s bed.

When the time comes for treatment, the patient receives conditioning at the hospital but can return home the day before or the day of transplant. After discharge, the patient is visited by a nurse practitioner or physician assistant each morning for a physical examination and blood draw.

In the afternoon, the patient is visited by a clinic nurse who brings any necessary supplies or treatments, such as blood products or intravenous antibiotics. The patient also has daily video calls with an attending physician and can be admitted to the hospital for any events that cannot be managed in the home setting.

Patients can have visitors and spend time away from home, but precautions are necessary. Friends or family who are sick should not be allowed to visit, and patients should avoid crowds when they go out.

Vidyard Video

Initial findings

The Duke team has treated 41 HSCT recipients at home so far. Dr. Chao said it’s still too early to draw any conclusions about differences in outcomes between home care and inpatient/outpatient HSCT.

However, a preliminary analysis of costs suggests home care is cheaper than inpatient HSCT. The researchers found that, for the first several transplants, at day 60, the cost of home care was roughly half that of inpatient HSCT.

In addition, patients seem to be happy with posttransplant care at home.

“The patients love being at home, in their own environment, with their families,” Dr. Chao said. “Almost every single patient [in the phase 1 trial] said that he or she liked it much better. There was one patient in the phase 1 that felt a little isolated, and I can see why because we say, ‘You can stay home, but don’t have a whole lot of people in.’ ”

 

 

One patient’s experience

Beth Vanderkin said it was “a blessing” to receive care at home after undergoing HSCT at Duke.

Ms. Vanderkin was diagnosed with diffuse large B-cell lymphoma in 2014. After two chemotherapy regimens failed to shrink the tumor in her chest, she underwent radiotherapy and responded well. When a PET scan revealed the tumor had gone completely, she proceeded to transplant.

She received a haploidentical HSCT using cells donated by her eldest daughter, Hannah Eichhorst. Ms. Vanderkin received the transplant in the hospital, and for 2 weeks after that, she made daily visits to the transplant clinic.

After those 2 weeks, Ms. Vanderkin continued her treatment at home. Like other patients eligible for home care, Ms. Vanderkin lived close to Duke, had a caregiver available, and had passed a home inspection. The Duke team shipped the needed medical supplies to her house and arranged twice-daily visits from nurses and daily video calls with a doctor.

Ms. Vanderkin said receiving care at home was “a game changer.” She derived comfort from recovering in her own environment, could spend more time with her family, and didn’t have to miss special events. While receiving care at home, Ms. Vanderkin attended the homecoming event where her son, Josiah, was part of the court. Wearing a face mask and carrying a portable pump in her purse, Ms. Vanderkin joined other mothers in escorting their children onto the football field.

“I got to escort my son out onto the field, and he was crowned king that night,” Ms. Vanderkin said. “I didn’t do a lot of things [while receiving care at home], but there were things I didn’t have to miss because I was at home and not in the hospital.”

Ms. Vanderkin said home care was also beneficial for her husband, who was her caregiver. Thomas Vanderkin was able to work from home while caring for his wife, and the daily nurses’ visits allowed him to run errands without having to leave Ms. Vanderkin alone.

Since her experience with home care, Ms. Vanderkin has spent many more days in the hospital and clinic. She experienced a relapse after the transplant and went on to receive more chemotherapy as well as ipilimumab. She responded to that treatment and has now been cancer-free for 3 years.

The ipilimumab did cause side effects, including intestinal problems that resulted in the need for parenteral nutrition. This side effect was made more bearable, Ms. Vanderkin said, because she was able to receive the parenteral nutrition at home. She and her husband were comfortable with additional home care because of their positive experience with posttransplant care.

“I think we’re conditioned to think that, to receive the best care, we have to be sitting in a hospital room or a clinic, but I think there’s a lot of things we can probably do at home,” Ms. Vanderkin said. “And we might fare a lot better as patients if we’re in an environment that we feel comfortable in.”

 

 

Experience at other centers

The team at Duke is not the first to study HSCT care at home. In fact, researchers in Sweden have been studying posttransplant home care since 1998.

A pilot trial the group published in 2000 suggested that home care was safe and, in some ways, superior to inpatient HSCT (Bone Marrow Transplant. 2000 Nov;26[10]:1057-60). Patients treated at home had a lower rate of bacteremia, fewer days of total parenteral nutrition, fewer erythrocyte transfusions, and fewer days on antibiotics and analgesics. Rates of fever, engraftment time, and acute GVHD were similar between the inpatient and home-care groups.

A study published by the same researchers in 2002 showed that patients who received home care had lower rates of grade 2-4 acute GVHD and transplant-related mortality compared to inpatients (Blood. 2002 Dec 15;100[13]:4317-24). Two-year overall survival was superior with home care as well.

On the other hand, a study the group published in 2013 showed no significant differences in 5-year survival, transplant-related mortality, relapse, or chronic GVHD between inpatients and those who received care at home (Biol Blood Marrow Transplant. 2013. doi: 10.1016/j.bbmt.2012.11.5189).

The phase 2 trials at Duke should provide more insight into patient outcomes, but results probably won’t be available for 2 more years, Dr. Chao said.

In the meantime, other U.S. researchers are studying home care as well. Memorial Sloan Kettering Cancer Center is conducting a pilot study to determine if HSCT care at home is feasible (NCT02671448).

Dr. Chao said home care should be possible for other centers, particularly those that already perform outpatient HSCT.

“Having the outpatient infrastructure to support these patients is a big step,” he said. “And I think we were able to do that mainly because we do most of our transplants in the outpatient setting already. So that jump to the home is a little less compared to a center that does no outpatient transplants.”

He added, “There’s a certain amount of inertia to overcome and a certain amount of apprehension from the caregivers initially because [patients aren’t] sitting in your unit all the time, but I don’t see this as a huge barrier.”

In fact, Dr. Chao said, if results with home care are favorable, it could potentially replace inpatient HSCT for certain patients.

Dr. Chao’s research is supported by Duke University, and he reported having no relevant financial disclosures.

[email protected]

Can receiving all posttransplant care at home benefit patients undergoing hematopoietic stem cell transplant (HSCT)? Researchers are conducting phase 2 trials to find out.

Nelson Chao, MD, and colleagues at Duke University in Durham, N.C., completed a phase 1 trial that suggested post-HSCT care at home was feasible and safe (Blood. 2017;130:745).

Now, the team is conducting phase 2 trials – NCT01725022 and NCT02218151 – comparing patients who receive all posttransplant care at home with patients treated in the hospital or in the outpatient setting with daily visits to the clinic.

The main goal is to determine if allogeneic HSCT recipients treated at home can maintain their normal microbiome and, as a result, have a lower risk of graft-versus-host disease (GVHD). The researchers are also looking at other outcomes such as quality of life, treatment-related morbidities and mortality, and the cost of care for both allogeneic and autologous transplant recipients.

To be eligible for home care after HSCT, a patient must live within a 90-minute driving distance of Duke and have a caregiver available at home. The patient’s home must pass an inspection, showing it to be free of sources for potential infection, such as mold or pets that sleep in the patient’s bed.

When the time comes for treatment, the patient receives conditioning at the hospital but can return home the day before or the day of transplant. After discharge, the patient is visited by a nurse practitioner or physician assistant each morning for a physical examination and blood draw.

In the afternoon, the patient is visited by a clinic nurse who brings any necessary supplies or treatments, such as blood products or intravenous antibiotics. The patient also has daily video calls with an attending physician and can be admitted to the hospital for any events that cannot be managed in the home setting.

Patients can have visitors and spend time away from home, but precautions are necessary. Friends or family who are sick should not be allowed to visit, and patients should avoid crowds when they go out.

Vidyard Video

Initial findings

The Duke team has treated 41 HSCT recipients at home so far. Dr. Chao said it’s still too early to draw any conclusions about differences in outcomes between home care and inpatient/outpatient HSCT.

However, a preliminary analysis of costs suggests home care is cheaper than inpatient HSCT. The researchers found that, for the first several transplants, at day 60, the cost of home care was roughly half that of inpatient HSCT.

In addition, patients seem to be happy with posttransplant care at home.

“The patients love being at home, in their own environment, with their families,” Dr. Chao said. “Almost every single patient [in the phase 1 trial] said that he or she liked it much better. There was one patient in the phase 1 that felt a little isolated, and I can see why because we say, ‘You can stay home, but don’t have a whole lot of people in.’ ”

 

 

One patient’s experience

Beth Vanderkin said it was “a blessing” to receive care at home after undergoing HSCT at Duke.

Ms. Vanderkin was diagnosed with diffuse large B-cell lymphoma in 2014. After two chemotherapy regimens failed to shrink the tumor in her chest, she underwent radiotherapy and responded well. When a PET scan revealed the tumor had gone completely, she proceeded to transplant.

She received a haploidentical HSCT using cells donated by her eldest daughter, Hannah Eichhorst. Ms. Vanderkin received the transplant in the hospital, and for 2 weeks after that, she made daily visits to the transplant clinic.

After those 2 weeks, Ms. Vanderkin continued her treatment at home. Like other patients eligible for home care, Ms. Vanderkin lived close to Duke, had a caregiver available, and had passed a home inspection. The Duke team shipped the needed medical supplies to her house and arranged twice-daily visits from nurses and daily video calls with a doctor.

Ms. Vanderkin said receiving care at home was “a game changer.” She derived comfort from recovering in her own environment, could spend more time with her family, and didn’t have to miss special events. While receiving care at home, Ms. Vanderkin attended the homecoming event where her son, Josiah, was part of the court. Wearing a face mask and carrying a portable pump in her purse, Ms. Vanderkin joined other mothers in escorting their children onto the football field.

“I got to escort my son out onto the field, and he was crowned king that night,” Ms. Vanderkin said. “I didn’t do a lot of things [while receiving care at home], but there were things I didn’t have to miss because I was at home and not in the hospital.”

Ms. Vanderkin said home care was also beneficial for her husband, who was her caregiver. Thomas Vanderkin was able to work from home while caring for his wife, and the daily nurses’ visits allowed him to run errands without having to leave Ms. Vanderkin alone.

Since her experience with home care, Ms. Vanderkin has spent many more days in the hospital and clinic. She experienced a relapse after the transplant and went on to receive more chemotherapy as well as ipilimumab. She responded to that treatment and has now been cancer-free for 3 years.

The ipilimumab did cause side effects, including intestinal problems that resulted in the need for parenteral nutrition. This side effect was made more bearable, Ms. Vanderkin said, because she was able to receive the parenteral nutrition at home. She and her husband were comfortable with additional home care because of their positive experience with posttransplant care.

“I think we’re conditioned to think that, to receive the best care, we have to be sitting in a hospital room or a clinic, but I think there’s a lot of things we can probably do at home,” Ms. Vanderkin said. “And we might fare a lot better as patients if we’re in an environment that we feel comfortable in.”

 

 

Experience at other centers

The team at Duke is not the first to study HSCT care at home. In fact, researchers in Sweden have been studying posttransplant home care since 1998.

A pilot trial the group published in 2000 suggested that home care was safe and, in some ways, superior to inpatient HSCT (Bone Marrow Transplant. 2000 Nov;26[10]:1057-60). Patients treated at home had a lower rate of bacteremia, fewer days of total parenteral nutrition, fewer erythrocyte transfusions, and fewer days on antibiotics and analgesics. Rates of fever, engraftment time, and acute GVHD were similar between the inpatient and home-care groups.

A study published by the same researchers in 2002 showed that patients who received home care had lower rates of grade 2-4 acute GVHD and transplant-related mortality compared to inpatients (Blood. 2002 Dec 15;100[13]:4317-24). Two-year overall survival was superior with home care as well.

On the other hand, a study the group published in 2013 showed no significant differences in 5-year survival, transplant-related mortality, relapse, or chronic GVHD between inpatients and those who received care at home (Biol Blood Marrow Transplant. 2013. doi: 10.1016/j.bbmt.2012.11.5189).

The phase 2 trials at Duke should provide more insight into patient outcomes, but results probably won’t be available for 2 more years, Dr. Chao said.

In the meantime, other U.S. researchers are studying home care as well. Memorial Sloan Kettering Cancer Center is conducting a pilot study to determine if HSCT care at home is feasible (NCT02671448).

Dr. Chao said home care should be possible for other centers, particularly those that already perform outpatient HSCT.

“Having the outpatient infrastructure to support these patients is a big step,” he said. “And I think we were able to do that mainly because we do most of our transplants in the outpatient setting already. So that jump to the home is a little less compared to a center that does no outpatient transplants.”

He added, “There’s a certain amount of inertia to overcome and a certain amount of apprehension from the caregivers initially because [patients aren’t] sitting in your unit all the time, but I don’t see this as a huge barrier.”

In fact, Dr. Chao said, if results with home care are favorable, it could potentially replace inpatient HSCT for certain patients.

Dr. Chao’s research is supported by Duke University, and he reported having no relevant financial disclosures.

[email protected]

Can receiving all posttransplant care at home benefit patients undergoing hematopoietic stem cell transplant (HSCT)? Researchers are conducting phase 2 trials to find out.

Nelson Chao, MD, and colleagues at Duke University in Durham, N.C., completed a phase 1 trial that suggested post-HSCT care at home was feasible and safe (Blood. 2017;130:745).

Now, the team is conducting phase 2 trials – NCT01725022 and NCT02218151 – comparing patients who receive all posttransplant care at home with patients treated in the hospital or in the outpatient setting with daily visits to the clinic.

The main goal is to determine if allogeneic HSCT recipients treated at home can maintain their normal microbiome and, as a result, have a lower risk of graft-versus-host disease (GVHD). The researchers are also looking at other outcomes such as quality of life, treatment-related morbidities and mortality, and the cost of care for both allogeneic and autologous transplant recipients.

To be eligible for home care after HSCT, a patient must live within a 90-minute driving distance of Duke and have a caregiver available at home. The patient’s home must pass an inspection, showing it to be free of sources for potential infection, such as mold or pets that sleep in the patient’s bed.

When the time comes for treatment, the patient receives conditioning at the hospital but can return home the day before or the day of transplant. After discharge, the patient is visited by a nurse practitioner or physician assistant each morning for a physical examination and blood draw.

In the afternoon, the patient is visited by a clinic nurse who brings any necessary supplies or treatments, such as blood products or intravenous antibiotics. The patient also has daily video calls with an attending physician and can be admitted to the hospital for any events that cannot be managed in the home setting.

Patients can have visitors and spend time away from home, but precautions are necessary. Friends or family who are sick should not be allowed to visit, and patients should avoid crowds when they go out.

Vidyard Video

Initial findings

The Duke team has treated 41 HSCT recipients at home so far. Dr. Chao said it’s still too early to draw any conclusions about differences in outcomes between home care and inpatient/outpatient HSCT.

However, a preliminary analysis of costs suggests home care is cheaper than inpatient HSCT. The researchers found that, for the first several transplants, at day 60, the cost of home care was roughly half that of inpatient HSCT.

In addition, patients seem to be happy with posttransplant care at home.

“The patients love being at home, in their own environment, with their families,” Dr. Chao said. “Almost every single patient [in the phase 1 trial] said that he or she liked it much better. There was one patient in the phase 1 that felt a little isolated, and I can see why because we say, ‘You can stay home, but don’t have a whole lot of people in.’ ”

 

 

One patient’s experience

Beth Vanderkin said it was “a blessing” to receive care at home after undergoing HSCT at Duke.

Ms. Vanderkin was diagnosed with diffuse large B-cell lymphoma in 2014. After two chemotherapy regimens failed to shrink the tumor in her chest, she underwent radiotherapy and responded well. When a PET scan revealed the tumor had gone completely, she proceeded to transplant.

She received a haploidentical HSCT using cells donated by her eldest daughter, Hannah Eichhorst. Ms. Vanderkin received the transplant in the hospital, and for 2 weeks after that, she made daily visits to the transplant clinic.

After those 2 weeks, Ms. Vanderkin continued her treatment at home. Like other patients eligible for home care, Ms. Vanderkin lived close to Duke, had a caregiver available, and had passed a home inspection. The Duke team shipped the needed medical supplies to her house and arranged twice-daily visits from nurses and daily video calls with a doctor.

Ms. Vanderkin said receiving care at home was “a game changer.” She derived comfort from recovering in her own environment, could spend more time with her family, and didn’t have to miss special events. While receiving care at home, Ms. Vanderkin attended the homecoming event where her son, Josiah, was part of the court. Wearing a face mask and carrying a portable pump in her purse, Ms. Vanderkin joined other mothers in escorting their children onto the football field.

“I got to escort my son out onto the field, and he was crowned king that night,” Ms. Vanderkin said. “I didn’t do a lot of things [while receiving care at home], but there were things I didn’t have to miss because I was at home and not in the hospital.”

Ms. Vanderkin said home care was also beneficial for her husband, who was her caregiver. Thomas Vanderkin was able to work from home while caring for his wife, and the daily nurses’ visits allowed him to run errands without having to leave Ms. Vanderkin alone.

Since her experience with home care, Ms. Vanderkin has spent many more days in the hospital and clinic. She experienced a relapse after the transplant and went on to receive more chemotherapy as well as ipilimumab. She responded to that treatment and has now been cancer-free for 3 years.

The ipilimumab did cause side effects, including intestinal problems that resulted in the need for parenteral nutrition. This side effect was made more bearable, Ms. Vanderkin said, because she was able to receive the parenteral nutrition at home. She and her husband were comfortable with additional home care because of their positive experience with posttransplant care.

“I think we’re conditioned to think that, to receive the best care, we have to be sitting in a hospital room or a clinic, but I think there’s a lot of things we can probably do at home,” Ms. Vanderkin said. “And we might fare a lot better as patients if we’re in an environment that we feel comfortable in.”

 

 

Experience at other centers

The team at Duke is not the first to study HSCT care at home. In fact, researchers in Sweden have been studying posttransplant home care since 1998.

A pilot trial the group published in 2000 suggested that home care was safe and, in some ways, superior to inpatient HSCT (Bone Marrow Transplant. 2000 Nov;26[10]:1057-60). Patients treated at home had a lower rate of bacteremia, fewer days of total parenteral nutrition, fewer erythrocyte transfusions, and fewer days on antibiotics and analgesics. Rates of fever, engraftment time, and acute GVHD were similar between the inpatient and home-care groups.

A study published by the same researchers in 2002 showed that patients who received home care had lower rates of grade 2-4 acute GVHD and transplant-related mortality compared to inpatients (Blood. 2002 Dec 15;100[13]:4317-24). Two-year overall survival was superior with home care as well.

On the other hand, a study the group published in 2013 showed no significant differences in 5-year survival, transplant-related mortality, relapse, or chronic GVHD between inpatients and those who received care at home (Biol Blood Marrow Transplant. 2013. doi: 10.1016/j.bbmt.2012.11.5189).

The phase 2 trials at Duke should provide more insight into patient outcomes, but results probably won’t be available for 2 more years, Dr. Chao said.

In the meantime, other U.S. researchers are studying home care as well. Memorial Sloan Kettering Cancer Center is conducting a pilot study to determine if HSCT care at home is feasible (NCT02671448).

Dr. Chao said home care should be possible for other centers, particularly those that already perform outpatient HSCT.

“Having the outpatient infrastructure to support these patients is a big step,” he said. “And I think we were able to do that mainly because we do most of our transplants in the outpatient setting already. So that jump to the home is a little less compared to a center that does no outpatient transplants.”

He added, “There’s a certain amount of inertia to overcome and a certain amount of apprehension from the caregivers initially because [patients aren’t] sitting in your unit all the time, but I don’t see this as a huge barrier.”

In fact, Dr. Chao said, if results with home care are favorable, it could potentially replace inpatient HSCT for certain patients.

Dr. Chao’s research is supported by Duke University, and he reported having no relevant financial disclosures.

[email protected]

Faculty members at Fox Chase Cancer Center have received grants to promote education about liver cancer, study pancreatic and breast cancer, and examine burnout among physician assistants (PAs).

Eric D. Tetzlaff, a PA at Fox Chase in Philadelphia, has received a 3-year grant from the Association of Physician Assistants in Oncology. With this $15,000 grant, Mr. Tetzlaff plans to conduct a longitudinal study that will explore burnout among PAs working in oncology.

The goals of his study are to “understand the impact of the attitudes of oncology PAs regarding teamwork, expectations for their professional role, type of collaborative practice, organizational context of the job environment, and moral distress, on burnout and career satisfaction,” according to Fox Chase.

Jaye Gardiner, PhD, a postdoctoral researcher in the Edna Cukierman laboratory at Fox Chase, has received a $163,500 grant from the American Cancer Society. With this grant, Dr. Gardiner will investigate the role of tumor stroma in pancreatic cancer.

Dr. Gardiner plans to explore how cancer-associated fibroblasts in the pancreatic stroma “communicate with one another and how this communication is altered in tumor-promoting versus tumor-restricting conditions,” according to Fox Chase.

Dietmar J. Kappes, PhD, a professor of blood cell development and cancer and director of the Transgenic Mouse Facility at Fox Chase, has received a 5-year grant from the National Institutes of Health. With this $626,072 grant, Dr. Kappes will investigate the role of the transcription factor ThPOK in breast cancer.

Dr. Kappes and colleagues previously found a link between high cytoplasmic levels of ThPOK and poor outcomes in breast cancer. Now, Dr. Kappes plans to “further elucidate the role of ThPOK in breast cancer by combining novel animal models and molecular approaches,” according to Fox Chase.

Evelyn González, senior director of the Fox Chase’s Office of Community Outreach, and Shannon Lynch, PhD, who is with the Cancer Prevention and Control program, have received a 2-year grant from the Pennsylvania Department of Human Services.
 

The pair will use this $125,000 grant to provide liver cancer and hepatitis education to communities in the Philadelphia area with the greatest burden of liver cancer and related risk factors. Dr. Lynch will find these at-risk communities, and the Office of Community Outreach will work with partner groups in those areas to provide bilingual education about hepatitis and how it relates to liver cancer.

[email protected]

Faculty members at Fox Chase Cancer Center have received grants to promote education about liver cancer, study pancreatic and breast cancer, and examine burnout among physician assistants (PAs).

Eric D. Tetzlaff, a PA at Fox Chase in Philadelphia, has received a 3-year grant from the Association of Physician Assistants in Oncology. With this $15,000 grant, Mr. Tetzlaff plans to conduct a longitudinal study that will explore burnout among PAs working in oncology.

The goals of his study are to “understand the impact of the attitudes of oncology PAs regarding teamwork, expectations for their professional role, type of collaborative practice, organizational context of the job environment, and moral distress, on burnout and career satisfaction,” according to Fox Chase.

Jaye Gardiner, PhD, a postdoctoral researcher in the Edna Cukierman laboratory at Fox Chase, has received a $163,500 grant from the American Cancer Society. With this grant, Dr. Gardiner will investigate the role of tumor stroma in pancreatic cancer.

Dr. Gardiner plans to explore how cancer-associated fibroblasts in the pancreatic stroma “communicate with one another and how this communication is altered in tumor-promoting versus tumor-restricting conditions,” according to Fox Chase.

Dietmar J. Kappes, PhD, a professor of blood cell development and cancer and director of the Transgenic Mouse Facility at Fox Chase, has received a 5-year grant from the National Institutes of Health. With this $626,072 grant, Dr. Kappes will investigate the role of the transcription factor ThPOK in breast cancer.

Dr. Kappes and colleagues previously found a link between high cytoplasmic levels of ThPOK and poor outcomes in breast cancer. Now, Dr. Kappes plans to “further elucidate the role of ThPOK in breast cancer by combining novel animal models and molecular approaches,” according to Fox Chase.

Evelyn González, senior director of the Fox Chase’s Office of Community Outreach, and Shannon Lynch, PhD, who is with the Cancer Prevention and Control program, have received a 2-year grant from the Pennsylvania Department of Human Services.
 

The pair will use this $125,000 grant to provide liver cancer and hepatitis education to communities in the Philadelphia area with the greatest burden of liver cancer and related risk factors. Dr. Lynch will find these at-risk communities, and the Office of Community Outreach will work with partner groups in those areas to provide bilingual education about hepatitis and how it relates to liver cancer.

[email protected]

Faculty members at Fox Chase Cancer Center have received grants to promote education about liver cancer, study pancreatic and breast cancer, and examine burnout among physician assistants (PAs).

Eric D. Tetzlaff, a PA at Fox Chase in Philadelphia, has received a 3-year grant from the Association of Physician Assistants in Oncology. With this $15,000 grant, Mr. Tetzlaff plans to conduct a longitudinal study that will explore burnout among PAs working in oncology.

The goals of his study are to “understand the impact of the attitudes of oncology PAs regarding teamwork, expectations for their professional role, type of collaborative practice, organizational context of the job environment, and moral distress, on burnout and career satisfaction,” according to Fox Chase.

Jaye Gardiner, PhD, a postdoctoral researcher in the Edna Cukierman laboratory at Fox Chase, has received a $163,500 grant from the American Cancer Society. With this grant, Dr. Gardiner will investigate the role of tumor stroma in pancreatic cancer.

Dr. Gardiner plans to explore how cancer-associated fibroblasts in the pancreatic stroma “communicate with one another and how this communication is altered in tumor-promoting versus tumor-restricting conditions,” according to Fox Chase.

Dietmar J. Kappes, PhD, a professor of blood cell development and cancer and director of the Transgenic Mouse Facility at Fox Chase, has received a 5-year grant from the National Institutes of Health. With this $626,072 grant, Dr. Kappes will investigate the role of the transcription factor ThPOK in breast cancer.

Dr. Kappes and colleagues previously found a link between high cytoplasmic levels of ThPOK and poor outcomes in breast cancer. Now, Dr. Kappes plans to “further elucidate the role of ThPOK in breast cancer by combining novel animal models and molecular approaches,” according to Fox Chase.

Evelyn González, senior director of the Fox Chase’s Office of Community Outreach, and Shannon Lynch, PhD, who is with the Cancer Prevention and Control program, have received a 2-year grant from the Pennsylvania Department of Human Services.
 

The pair will use this $125,000 grant to provide liver cancer and hepatitis education to communities in the Philadelphia area with the greatest burden of liver cancer and related risk factors. Dr. Lynch will find these at-risk communities, and the Office of Community Outreach will work with partner groups in those areas to provide bilingual education about hepatitis and how it relates to liver cancer.

[email protected]

Researchers are leading several programs designed to serve the sickle cell community in the United States and sub-Saharan Africa, officials at the National Heart, Lung, and Blood Institute (NHLBI) said during a recent webinar.

Dr_Microbe/Thinkstock

One program based in the United States is focused on building a registry for patients with sickle cell disease (SCD) and conducting studies designed to improve SCD care. Another program involves building “an information-sharing network and patient-powered registry” in the United States.

The programs in sub-Saharan Africa were designed to establish a database of SCD patients, optimize the use of hydroxyurea in children with SCD, and aid genomic studies of SCD.

W. Keith Hoots, MD, director of the Division of Blood Diseases and Resources at NHLBI, began the webinar with an overview of the programs in sub-Saharan Africa. He described four programs with sites in nine countries (Angola, Cameroon, Democratic Republic of Congo, Ghana, Kenya, Nigeria, South Africa, Tanzania, and Uganda).

SPARCO and SADaCC

Dr. Hoots outlined the scope the Sickle Pan-African Research Consortium (SPARCO) and the Sickle Africa Data Coordinating Center (SADaCC), both part of the Sickle In Africa consortium.

A major goal of SPARCO and SADaCC is to create a Research Electronic Data Capture database that encompasses SCD patients in sub-Saharan Africa. As of April 2019, the database included 6,578 patients. The target is 13,000 patients.

Other goals of SPARCO and SADaCC are to “harmonize” SCD phenotype definitions and ontologies, create clinical guidelines for SCD management in sub-Saharan Africa, plan future cohort studies, and develop programs for newborn screening, infection prevention, and increased use of hydroxyurea.

“So far, they’re well along in establishing a registry and a database system,” Dr. Hoots said. “They’ve agreed on the database elements, phenotype definitions, and ontologies, they’ve developed some regionally appropriate clinical management guidelines, and they’ve begun skills development on the ground at all respective sites.”

REACH

Another program Dr. Hoots discussed is Realizing Effectiveness Across Continents With Hydroxyurea (REACH), a phase 1/2 pilot study of hydroxyurea in children (aged 1-10 years) with SCD in sub-Saharan Africa.

The goals of REACH are to determine the optimal dose of hydroxyurea in this population; teach African physicians how to administer hydroxyurea; assess the safety, feasibility, and benefits of hydroxyurea; study variability in response to hydroxyurea; gather data for the Research Electronic Data Capture database; and establish a research infrastructure for future collaborations.

Results from more than 600 children enrolled in REACH were presented at the 2018 annual meeting of the American Society of Hematology and simultaneously published in the New England Journal of Medicine (N Engl J Med. 2019 Jan 10; 380[2]:121-31).

SickleGenAfrica

SickleGenAfrica is part of the H3Africa consortium and aims to “build capacity for genomic research in Africa,” Dr. Hoots said.

Under this program, researchers will conduct three studies to test the hypothesis that genetic variation affects the defense against hemolysis and organ damage in patients with SCD. The researchers will study existing cohorts of SCD patients including children and adults.

Other goals of SickleGenAfrica are to establish a molecular hematology and sickle cell mouse core, an SCD biorepository core, a bioinformatics core, and an administrative core for the coordination of activities. The program will also be used to train “future science leaders” in SCD research, Dr. Hoots said.
 

SCDIC

Cheryl Anne Boyce, PhD, chief of the Implementation Science Branch at the Center for Translation Research and Implementation Science at NHLBI, discussed the United States–based Sickle Cell Disease Implementation Consortium (SCDIC).

“The goals of the consortium are to develop a registry in collaboration with other centers and the NHLBI, as well as a needs-based community assessment of the barriers to care for subjects with sickle cell disease,” Dr. Boyce said. “We also wanted to design implementation research studies that address the identified barriers to care.”

Dr. Boyce said the SCDIC’s registry is open to patients aged 15-45 years who have a confirmed SCD diagnosis, speak English, and are able to consent to and complete a survey. The registry has enrolled almost 2,400 patients from eight centers over 18 months.

The SCDIC has also performed a needs assessment that prompted the development of three implementation research studies. The first study involves using mobile health interventions to, ideally, increase patient adherence to hydroxyurea and improve provider knowledge of hydroxyurea.

With the second study, researchers aim to improve the care of SCD patients in the emergency department by using an inpatient portal. The goals of the third study are to establish a standard definition for unaffiliated patients, conduct a needs assessment for this group, and develop an intervention that can provide these patients with guideline-based SCD care.

Get Connected

Kim Smith-Whitley, MD, director of the Comprehensive Sickle Cell Center at the Children’s Hospital of Philadelphia and a board member of the Sickle Cell Disease Association of America (SCDAA), described Get Connected, “an information-sharing network and patient-powered registry” created by SCDAA.

Dr. Smith-Whitley said one purpose of Get Connected is to provide a network that facilitates “the distribution of information related to clinical care, research, health services, health policy, and advocacy.”

The network is open to families living with SCD and sickle cell trait, SCDAA member organizations, health care providers, clinical researchers, and community-based organizations.

Get Connected also includes a registry for SCD patients that stores information on their diagnosis and treatment, as well as online communities that can be used to share information and provide psychosocial support.

Thus far, Get Connected has enrolled 6,329 individuals. This includes 5,100 children and adults with SCD, 652 children and adults with sickle cell trait, and 577 nonpatients.

The webinar presenters did not disclose any conflicts of interest.

[email protected]

Researchers are leading several programs designed to serve the sickle cell community in the United States and sub-Saharan Africa, officials at the National Heart, Lung, and Blood Institute (NHLBI) said during a recent webinar.

Dr_Microbe/Thinkstock

One program based in the United States is focused on building a registry for patients with sickle cell disease (SCD) and conducting studies designed to improve SCD care. Another program involves building “an information-sharing network and patient-powered registry” in the United States.

The programs in sub-Saharan Africa were designed to establish a database of SCD patients, optimize the use of hydroxyurea in children with SCD, and aid genomic studies of SCD.

W. Keith Hoots, MD, director of the Division of Blood Diseases and Resources at NHLBI, began the webinar with an overview of the programs in sub-Saharan Africa. He described four programs with sites in nine countries (Angola, Cameroon, Democratic Republic of Congo, Ghana, Kenya, Nigeria, South Africa, Tanzania, and Uganda).

SPARCO and SADaCC

Dr. Hoots outlined the scope the Sickle Pan-African Research Consortium (SPARCO) and the Sickle Africa Data Coordinating Center (SADaCC), both part of the Sickle In Africa consortium.

A major goal of SPARCO and SADaCC is to create a Research Electronic Data Capture database that encompasses SCD patients in sub-Saharan Africa. As of April 2019, the database included 6,578 patients. The target is 13,000 patients.

Other goals of SPARCO and SADaCC are to “harmonize” SCD phenotype definitions and ontologies, create clinical guidelines for SCD management in sub-Saharan Africa, plan future cohort studies, and develop programs for newborn screening, infection prevention, and increased use of hydroxyurea.

“So far, they’re well along in establishing a registry and a database system,” Dr. Hoots said. “They’ve agreed on the database elements, phenotype definitions, and ontologies, they’ve developed some regionally appropriate clinical management guidelines, and they’ve begun skills development on the ground at all respective sites.”

REACH

Another program Dr. Hoots discussed is Realizing Effectiveness Across Continents With Hydroxyurea (REACH), a phase 1/2 pilot study of hydroxyurea in children (aged 1-10 years) with SCD in sub-Saharan Africa.

The goals of REACH are to determine the optimal dose of hydroxyurea in this population; teach African physicians how to administer hydroxyurea; assess the safety, feasibility, and benefits of hydroxyurea; study variability in response to hydroxyurea; gather data for the Research Electronic Data Capture database; and establish a research infrastructure for future collaborations.

Results from more than 600 children enrolled in REACH were presented at the 2018 annual meeting of the American Society of Hematology and simultaneously published in the New England Journal of Medicine (N Engl J Med. 2019 Jan 10; 380[2]:121-31).

SickleGenAfrica

SickleGenAfrica is part of the H3Africa consortium and aims to “build capacity for genomic research in Africa,” Dr. Hoots said.

Under this program, researchers will conduct three studies to test the hypothesis that genetic variation affects the defense against hemolysis and organ damage in patients with SCD. The researchers will study existing cohorts of SCD patients including children and adults.

Other goals of SickleGenAfrica are to establish a molecular hematology and sickle cell mouse core, an SCD biorepository core, a bioinformatics core, and an administrative core for the coordination of activities. The program will also be used to train “future science leaders” in SCD research, Dr. Hoots said.
 

SCDIC

Cheryl Anne Boyce, PhD, chief of the Implementation Science Branch at the Center for Translation Research and Implementation Science at NHLBI, discussed the United States–based Sickle Cell Disease Implementation Consortium (SCDIC).

“The goals of the consortium are to develop a registry in collaboration with other centers and the NHLBI, as well as a needs-based community assessment of the barriers to care for subjects with sickle cell disease,” Dr. Boyce said. “We also wanted to design implementation research studies that address the identified barriers to care.”

Dr. Boyce said the SCDIC’s registry is open to patients aged 15-45 years who have a confirmed SCD diagnosis, speak English, and are able to consent to and complete a survey. The registry has enrolled almost 2,400 patients from eight centers over 18 months.

The SCDIC has also performed a needs assessment that prompted the development of three implementation research studies. The first study involves using mobile health interventions to, ideally, increase patient adherence to hydroxyurea and improve provider knowledge of hydroxyurea.

With the second study, researchers aim to improve the care of SCD patients in the emergency department by using an inpatient portal. The goals of the third study are to establish a standard definition for unaffiliated patients, conduct a needs assessment for this group, and develop an intervention that can provide these patients with guideline-based SCD care.

Get Connected

Kim Smith-Whitley, MD, director of the Comprehensive Sickle Cell Center at the Children’s Hospital of Philadelphia and a board member of the Sickle Cell Disease Association of America (SCDAA), described Get Connected, “an information-sharing network and patient-powered registry” created by SCDAA.

Dr. Smith-Whitley said one purpose of Get Connected is to provide a network that facilitates “the distribution of information related to clinical care, research, health services, health policy, and advocacy.”

The network is open to families living with SCD and sickle cell trait, SCDAA member organizations, health care providers, clinical researchers, and community-based organizations.

Get Connected also includes a registry for SCD patients that stores information on their diagnosis and treatment, as well as online communities that can be used to share information and provide psychosocial support.

Thus far, Get Connected has enrolled 6,329 individuals. This includes 5,100 children and adults with SCD, 652 children and adults with sickle cell trait, and 577 nonpatients.

The webinar presenters did not disclose any conflicts of interest.

[email protected]

Researchers are leading several programs designed to serve the sickle cell community in the United States and sub-Saharan Africa, officials at the National Heart, Lung, and Blood Institute (NHLBI) said during a recent webinar.

Dr_Microbe/Thinkstock

One program based in the United States is focused on building a registry for patients with sickle cell disease (SCD) and conducting studies designed to improve SCD care. Another program involves building “an information-sharing network and patient-powered registry” in the United States.

The programs in sub-Saharan Africa were designed to establish a database of SCD patients, optimize the use of hydroxyurea in children with SCD, and aid genomic studies of SCD.

W. Keith Hoots, MD, director of the Division of Blood Diseases and Resources at NHLBI, began the webinar with an overview of the programs in sub-Saharan Africa. He described four programs with sites in nine countries (Angola, Cameroon, Democratic Republic of Congo, Ghana, Kenya, Nigeria, South Africa, Tanzania, and Uganda).

SPARCO and SADaCC

Dr. Hoots outlined the scope the Sickle Pan-African Research Consortium (SPARCO) and the Sickle Africa Data Coordinating Center (SADaCC), both part of the Sickle In Africa consortium.

A major goal of SPARCO and SADaCC is to create a Research Electronic Data Capture database that encompasses SCD patients in sub-Saharan Africa. As of April 2019, the database included 6,578 patients. The target is 13,000 patients.

Other goals of SPARCO and SADaCC are to “harmonize” SCD phenotype definitions and ontologies, create clinical guidelines for SCD management in sub-Saharan Africa, plan future cohort studies, and develop programs for newborn screening, infection prevention, and increased use of hydroxyurea.

“So far, they’re well along in establishing a registry and a database system,” Dr. Hoots said. “They’ve agreed on the database elements, phenotype definitions, and ontologies, they’ve developed some regionally appropriate clinical management guidelines, and they’ve begun skills development on the ground at all respective sites.”

REACH

Another program Dr. Hoots discussed is Realizing Effectiveness Across Continents With Hydroxyurea (REACH), a phase 1/2 pilot study of hydroxyurea in children (aged 1-10 years) with SCD in sub-Saharan Africa.

The goals of REACH are to determine the optimal dose of hydroxyurea in this population; teach African physicians how to administer hydroxyurea; assess the safety, feasibility, and benefits of hydroxyurea; study variability in response to hydroxyurea; gather data for the Research Electronic Data Capture database; and establish a research infrastructure for future collaborations.

Results from more than 600 children enrolled in REACH were presented at the 2018 annual meeting of the American Society of Hematology and simultaneously published in the New England Journal of Medicine (N Engl J Med. 2019 Jan 10; 380[2]:121-31).

SickleGenAfrica

SickleGenAfrica is part of the H3Africa consortium and aims to “build capacity for genomic research in Africa,” Dr. Hoots said.

Under this program, researchers will conduct three studies to test the hypothesis that genetic variation affects the defense against hemolysis and organ damage in patients with SCD. The researchers will study existing cohorts of SCD patients including children and adults.

Other goals of SickleGenAfrica are to establish a molecular hematology and sickle cell mouse core, an SCD biorepository core, a bioinformatics core, and an administrative core for the coordination of activities. The program will also be used to train “future science leaders” in SCD research, Dr. Hoots said.
 

SCDIC

Cheryl Anne Boyce, PhD, chief of the Implementation Science Branch at the Center for Translation Research and Implementation Science at NHLBI, discussed the United States–based Sickle Cell Disease Implementation Consortium (SCDIC).

“The goals of the consortium are to develop a registry in collaboration with other centers and the NHLBI, as well as a needs-based community assessment of the barriers to care for subjects with sickle cell disease,” Dr. Boyce said. “We also wanted to design implementation research studies that address the identified barriers to care.”

Dr. Boyce said the SCDIC’s registry is open to patients aged 15-45 years who have a confirmed SCD diagnosis, speak English, and are able to consent to and complete a survey. The registry has enrolled almost 2,400 patients from eight centers over 18 months.

The SCDIC has also performed a needs assessment that prompted the development of three implementation research studies. The first study involves using mobile health interventions to, ideally, increase patient adherence to hydroxyurea and improve provider knowledge of hydroxyurea.

With the second study, researchers aim to improve the care of SCD patients in the emergency department by using an inpatient portal. The goals of the third study are to establish a standard definition for unaffiliated patients, conduct a needs assessment for this group, and develop an intervention that can provide these patients with guideline-based SCD care.

Get Connected

Kim Smith-Whitley, MD, director of the Comprehensive Sickle Cell Center at the Children’s Hospital of Philadelphia and a board member of the Sickle Cell Disease Association of America (SCDAA), described Get Connected, “an information-sharing network and patient-powered registry” created by SCDAA.

Dr. Smith-Whitley said one purpose of Get Connected is to provide a network that facilitates “the distribution of information related to clinical care, research, health services, health policy, and advocacy.”

The network is open to families living with SCD and sickle cell trait, SCDAA member organizations, health care providers, clinical researchers, and community-based organizations.

Get Connected also includes a registry for SCD patients that stores information on their diagnosis and treatment, as well as online communities that can be used to share information and provide psychosocial support.

Thus far, Get Connected has enrolled 6,329 individuals. This includes 5,100 children and adults with SCD, 652 children and adults with sickle cell trait, and 577 nonpatients.

The webinar presenters did not disclose any conflicts of interest.

[email protected]