ASH18: RNA Sequencing Identifies More Subtypes of Childhood Leukemia

MedicalResearch.com Interview with:

Charles G. Mullighan, MBBS (Hons), MSc, MD Member, St. Jude Faculty Co-Leader, Hematological Malignancies Program Medical Director, St. Jude Biorepository William E. Evans Endowed Chair St. Judes Children’s Research Hospital Memphis, TN

Dr. Mullighan

Charles G. Mullighan, MBBS (Hons), MSc, MD
Member, St. Jude Faculty
Co-Leader, Hematological Malignancies Program
Medical Director, St. Jude Biorepository
William E. Evans Endowed Chair
St. Judes Children’s Research Hospital
Memphis, TN

MedicalResearch.com: What is the background for this study?

 

Response: B-lineage acute lymphoblastic leukemia (B-ALL) is the commonest form of ALL, and the commonest childhood tumor. It is a leading cause of childhood cancer death. It consists of multiple subtypes defined by genetic alterations. These are often chromosomal translocations that deregulate oncogenes or form fusion proteins. These alterations are disease initiating events and are associated with distinct patterns of leukemic cell gene expression. Most subtypes also have additional mutations that are important for cells to become fully leukemic.

Identifying these initiating genetic changes is very important to identify patients that are likely to respond or do poorly with conventional therapy (multiagent chemotherapy). Also, some identify new opportunities for targeted therapy. However, using standard genetic testing approaches such as chromosomal cytogenetics, about 30% of B-ALL patients don’t have a subtype classifying alteration.

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Healthcare Costs in Patients with Cancer Rise with Increasing Risk of Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Despite being largely preventable, venous thromboembolism (VTE) is the second leading cause of death in people with cancer. The risk of VTE is five times greater in people with cancer than those without cancer, and that risk is magnified in those receiving certain types of chemotherapy, in the newly diagnosed and in those with more advanced, metastatic disease. This 6,194-patient study examined economic burden associated with VTE, and found patients newly diagnosed with cancer who are at a higher risk of a VTE had significantly higher all-cause and VTE-related health care costs compared to patients with a lower risk of VTE. Continue reading

Lower Overall Costs with Rivaroxaban (XARELTO® ) vs Warfarin Among Morbidly Obese Patients with Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Treatment of venous thromboembolism (VTE) is complicated among morbidly obese patients. Current guidelines do not recommend use of Factor Xa inhibitors in these patients due to limited clinical data available. That’s why Janssen undertook this study to examine XARELTO® (rivaroxaban) in these patients. In this 5,780-patient retrospective study, results found patients treated with XARELTO® had a similar risk of recurrent VTE and major bleeding compared to those taking warfarin.

However, treatment with XARELTO® was associated with less all-cause health care resource utilization (HCRU) (e.g., inpatient hospitalizations and outpatient visits) and reduced total medical costs compared to warfarin. Of note, patients taking XARELTO® had an average $2,829 lower total medical costs per patient per year (PPPY) than those taking warfarin, which was mainly driven by lower hospitalization costs. Continue reading

How Do Patients With Multiple Myeloma Weight Treatment Options?

MedicalResearch.com Interview with:

Caroline McKay, PhD Real World Value & Evidence, Oncology Janssen Scientific Affairs

Dr. McKay

Caroline McKay, PhD
Real World Value & Evidence, Oncology
Janssen Scientific Affairs

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Considering patient preferences in treatment decision-making in oncology is growing in importance. While recent introduction of new treatments for multiple myeloma have improved survival and the possibility of sustaining longer remission periods, regimen options still vary with respect to efficacy, safety, and dosing.

Therefore, patients and providers must consider tradeoffs inherent in making treatment decisions that are growing in complexity. Despite this, there is a lack of research describing patient preferences within the context of currently available treatment regimens. To address this gap, this study examined how multiple myeloma patients evaluate, or weigh, treatment options. Key findings from the research are that treatment preferences do not appear to be static, but instead suggest that the relative importance of treatment attributes may change over time and treatment history.

Further, patients place higher importance on overall survival and progression-free survival than other treatment attributes, and may be willing to accept an increase in the risk of serious side effects and reduced convenience in exchange for greater efficacy; however, when efficacy is comparable, patients appear to place greater weight on dosing frequency than on the duration of treatment administration, i.e., more frequent dosing appears to be less preferable to patients than longer administration/infusion time.  Continue reading

The Medalist Trial: Luspatercept Reduced Transfusion Need in Some Myelodysplastic Syndromes

MedicalResearch.com Interview with:

Dr. Alan List MD President and Chief Executive Officer Moffitt Cancer Center Tampa, FL

Dr. List

Dr. Alan List MD
President and Chief Executive Officer
Moffitt Cancer Center
Tampa, FL

MedicalResearch.com: What is the background for this study?  

Response: In patients with lower risk Myelodysplastic Syndromes (MDS), which accounts for the vast majority of patients with MDS overall, the most common symptomatic cytopenia is anemia. These patients, overtime, become dependent upon red blood cell transfusions and with that, they face a risk of iron loading as well as complications that occur with it. The standard first line therapy that we consider for these patients is erythropoietin-stimulating agents (ESAs). Patients who are transfusion dependent have a low response rate to ESAs, and responses are of short duration. There limited effective limited treatment options for those patients unresponsive or lose response to ESAs.

For years, we’ve known that the transforming growth factor (TGF)-β pathway play an important pathogenetic role in suppressing red cell maturation and cell survival.

Luspatercept is an agent that acts as an erythroid maturation agent by inhibiting the TGF-β signaling pathway by neutralizing a select group of TGF-β superfamily ligands.  Continue reading

Alcyone Trial: DaraVMP New Standard for Transplant-Ineligible Newly Diagnosed Multiple Myeloma

MedicalResearch.com Interview with:

Maria-Victoria Mateos, MD, PhD Associate Professor of Medicine  University of Salamanca Salamanca, Spain

Dr. Mateos

Maria-Victoria Mateos, MD, PhD
Associate Professor of Medicine
University of Salamanca
Salamanca, Spain

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Alcyone trial is a phase 3 trial in which Daratumumab, the CD38 mAb has been added to a standard of care for elderly newly diagnosed myeloma patients, VMP, and compared with VMP. The main finding is that the addition of dara to VMP resulted into a significant benefit in PFS with a 57% reduction in the risk of progression and/or death. In addition, the benefit was also reported in terms of ORR and CR rate and 45% of patients receiving Dara VMP achieved CR. Minimal residual disease was evaluated and was undetectable in 27% of the patients what it is relevant because a 5% increase was observed in comparison with the publication one year ago. This means that Daratumumab as maintenance after the first 9 cycles daraVMP was able to upgrade the quality of response. Toxicity profile was acceptable and no new safety signals were reported. Continue reading

Experimental Luspatercept Reduced Need For Transfusions in Beta Thalassemia

MedicalResearch.com Interview with:

Maria Domenica Cappellini, M.D. Principal Investigator, BELIEVE Clinical Trial Fondazione IRCCS Ca’ Granda Policlinico Hospital University of Milan, Milan, Italy

Dr. Cappellini

Maria Domenica Cappellini, M.D.
Principal Investigator, BELIEVE Clinical Trial
Fondazione IRCCS Ca’ Granda Policlinico Hospital
University of Milan, Milan, Italy

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Beta thalassemia is a severe, inherited hemoglobinophathy due to a reduced production of hemoglobin. Standard of care is blood transfusion and iron chelation to remove the accumulated iron by transfusion. This is a very demanding treatment.

Therefore, there is the need to find new approaches for treating these patients. The BELIEVE study showed that the experimental drug luspatercept significantly reduced the need for blood transfusions in patients with beta thalassemia. More than 70 percent of patients who received luspatercept were able to cut blood transfusions by at least one-third over any consecutive 12-week period Continue reading

ASH18: CENTAURUS Study Evaluates Dosing Schedule of DARZALEX® (daratumumab) for High Risk Smoldering Multiple Myeloma

MedicalResearch.com Interview with:

Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health

Dr. Vorhees

Peter Voorhees, MD
Plasma Cell Disorders Program
Department of Hematologic Oncology and Blood Disorders
Levine Cancer Institute
Atrium Health

MedicalResearch.com: What is the background for this study?

Response: All multiple myeloma arises from its precursor conditions, monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). Although the rate of progression to multiple myeloma for patients with MGUS is low (~5% over 5 years), patients with SMM have a ~50% likelihood of requiring therapy for their multiple myeloma within the first 5 years of diagnosis. For those at intermediate to high risk of disease progression, early intervention to delay progression of disease, thereby averting disease-related morbidities related and potentially changing the natural course of the disease, is highly desirable.

On the other hand, given the fact that these patients are by definition asymptomatic and would otherwise be monitored off treatment, it is critical that any intervention applied in this group of patients is well tolerated. Daratumumab is a highly attractive candidate in this particular space, because it has single agent activity in heavily-pretreated relapsed/refractory multiple myeloma and a favorable side effect profile relative to many other myeloma therapeutics. Additionally, given the importance of impaired immune surveillance in multiple myeloma, the immuno-stimulatory effects of daratumumab in the bone marrow microenvironment could potentially reawaken robust T cell responses to the disease.

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CLL: Overall Treatment Savings With Ibrutinib (Imbruvica) Despite Higher Prescription Costs

MedicalResearch.com Interview with:

Dr. Sundaram

Murali Sundaram, MBA, Ph.D.
Director of Real World Value and Evidence
Oncology, Janssen

MedicalResearch.com: What is the background for this study?

Response: Ibrutinib is a novel Bruton’s tyrosine kinase (BTK) inhibitor approved for the treatment of patients with newly diagnosed chronic lymphocytic leukemia (CLL).

Ibrutinib is administered orally while standard of care (CD20 monoclonal antibody-based chemoimmunotherapy [CIT]) is administered intravenously. This difference in route of administration impacts what type of benefit covers these treatments (i.e., pharmacy benefit for oral ibrutinib and medical benefit for intravenous CIT).

Previous studies evaluating the costs burden of patients treated with ibrutinib versus CIT did not include the full spectrum of real-world healthcare costs.

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Iron Deficiency: The Causes, Detection, and Treatment

spinach-bundlesIron Deficiency: The Causes, Detection, and Treatment

Background:

IDA or iron deficiency anemia is a result of a lack of iron in the human body. This causes a complication of hemoglobin, resulting in the body being unable to obtain enough oxygen. IDA can be caused by a few different occurrences, such as not receiving an adequate level of iron through intake, internal bleeding, or being unable to fully absorb iron into the body. Whatever the cause, research throughout the years has furthered the knowledge available on treatments for IDA, as well as how it can be detected, its symptoms, and how it affects the human body.

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Subcutaneous Emicizumab Reduces Number of Injections Needed to Control Hemophilia

MedicalResearch.com Interview with:

Dr. Johnny Mahlangu  MBBCh Faculty of Health Sciences University of the Witwatersrand and National Health Laboratory Service Johannesburg, South Africa

Dr. Mahlangu

Dr. Johnny Mahlangu  MBBCh
Faculty of Health Sciences
University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Current unmet needs in patients with haemophilia without inhibitors are the high disease burden imposed by the frequent injections which have to be given intravensously .

Emicizumab which is given subcutaneously weekly or fortnightly aims to address these unmet needs.

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Plitidepsin Evaluated for Refractory Multiple Myeloma

MedicalResearch.com Interview with:
PharmaMarDr. Javier Gómez García
Senior Manager. Biostatistics and Data Management
PharmaMar
Madrid Area, Spain

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Multiple myeloma accounts for approximately 1 percent of all cancers and slightly more than 10 percent of all hematologic malignancies. At present, more than 80,000 new cases are reported worldwide each year, and the disease prevalence is increasing.

Plitidepsin is a synthetic cyclic depsipeptide isolated from the marine tunicate Aplidium albicans targeting the proto-oncogene eEF1A2, which is over-expressed in multiple myeloma cells.

In ADMYRE trial 255 relapsed and refractory multiple myeloma patients with at least three but not more than six prior regimens, including at least bortezomib and lenalidomide/thalidomide, were randomized at 2:1 ratio to receive plitidepsin 5 mg/m2 D1 and 15 plus DXM 40 mg D1, 8, 15 and 22 (P+DXM), or DXM 40 mg D1,8,15 and 22 (DXM) every four weeks.

P+DXM met the primary endpoint, progression-free survival (PFS) assessed by an Independent Review Committee, showing a 35% risk reduction in the probability of progression or death. Indeed, a 20% risk reduction in the probability of death was also observed in spite of the fact that 44% of patients from control arm (DXM) switched to P+DXM arm after progression. Therefore, survival in the control arm might have been extended by the effect of plitidepsin, increasing the post progression survival and overestimating the survival observed in the control arm and consequently underestimating the actual difference between treatment arms.

Several methods were used to assess the impact of crossover and the robustness of the results even when penalizations were applied; PharmaMar believes that strong evidence in terms of survival benefit in favour of P+DXM has been established.  Continue reading

One Blood Type Is a Risk For Bleeding Out After Trauma

MedicalResearch.com Interview with:
Dr. Wataru Takayama
Tokyo Medical and Dental University
Department of Emergency and Disaster Medicine
Tokyo, Japan

MedicalResearch.com: What is the background for this study?

Response: ABO blood type is a potential risk of various diseases and various conditions. Furthermore, ABO blood type has a profound influence on hemostasis. Hemorrhage is the leading cause of death in patients with trauma, we assessed the association between the difference in blood types and the outcomes of death.

MedicalResearch.com: What are the main findings?

Response: Blood type O was the independent risk factor for all-cause in-hospital mortality and death due to exsanguination, TBI, and other causes after adjusting for potential confounders. This is the first study to report the association between ABO blood types and mortality in patients with severe trauma.

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Is Sickle Cell Really a Risk Factor for Stroke?

MedicalResearch.com Interview with :

Dr. Hyacinth I Hyacinth MD Aflac Cancer and Blood Disorder Center, Emory Children’s Center, Department of Pediatrics, Emory University School of Medicine Atlanta, GA 30322

Dr. Hyacinth

Dr. Hyacinth I Hyacinth MD
Aflac Cancer and Blood Disorder Center, Emory Children’s Center, Department of Pediatrics, Emory University School of Medicine
Atlanta, GA 30322

MedicalResearch.com: What is the background for this study?

This study was conducted against the backdrop of a significantly higher risk for stroke among African Americans compared to non-Hispanic Whites, despite adjusting for traditional risk factors. Also, sickle cell disease is a well-known genetic risk factor for stroke and recent studies show that sickle cell trait is a risk factor for chronic kidney disease, venous thromboembolism and pulmonary embolism, all of which are potential risk factors for stroke.

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TXA Increasingly Use in Shoulder Surgery To Reduce Transfusion Risk and Complications

MedicalResearch.com Interview with:

Shawn Anthony, MD, MBA Assistant Professor of Orthopaedics Icahn School of Medicine at Mount Sinai

Dr. Anthony

Shawn Anthony, MD, MBA
Assistant Professor of Orthopaedics
Icahn School of Medicine at Mount Sinai

MedicalResearch.com: What is the background for this study? What are the main findings? 

Response: Rates of total shoulder arthroplasty are increasing, especially with an aging population.  Blood loss requiring transfusion is less common than in total hip or knee replacements but still required in some patients.  Tranexamic acid (TXA) is increasingly used to reduce blood loss in lower extremity arthroplasty but limited data exists for its effectiveness and safety in patients undergoing shoulder arthroplasty. We aimed to utilize national data to assess frequency of use and effectiveness of TXA in shoulder arthroplasty patients.

While utilization of TXA has become very common in total hip and knee arthroplasty, TXA is still used in less than 50% of patients undergoing shoulder arthroplasty as of 2016.  TXA use was associated with a 36% decrease in transfusion risk and a 35% decreased risk for combined complications. Moreover, TXA use was associated with 6.2% shorter hospital stay.

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Investigational Imaging Test Can Help Determine Success or Failure of Bone Marrow Transplant

MedicalResearch.com Interview with:

Kirsten Williams, M.D. Blood and marrow transplant specialist Children’s National Health System

Dr. Williams

Kirsten Williams, M.D.
Blood and marrow transplant specialist
Children’s National Health System 

MedicalResearch.com: What is the background for this study? What are the main findings? 

Response: This study addressed a life-threatening complication of bone marrow transplantation called bone marrow failure. Bone marrow transplantation has provided a cure for patients with aggressive leukemias or acquired or genetic marrow dysfunction. The process of bone marrow transplantation involves giving chemotherapy and/or radiation, which removes the diseased blood cells from the bone marrow. After this, new bone marrow stem cells are infused from a healthy individual. They travel to the bone marrow and start the slow process of remaking the blood system. Because these new cells start from infancy, it takes upwards of four to five weeks for new mature healthy cells to emerge into the blood, where they can be identified. Historically, there has been no timely way to determine if the new cells have successfully repopulated unless they can be seen in the blood compartment. This condition of bone marrow failure is life-threatening, because patients don’t have white blood cells to protect them from infection. Once bone marrow failure is diagnosed, a second new set of stem cells are infused, often after more chemotherapy is given. However, for many individuals this re-transplantation is too late, because severe infections can be fatal while waiting cells to recover.

We were the first group to use a new imaging test to understand how the newly infused bone marrow cells develop inside the patient. We have recently published a way to detect the new bone marrow cell growth as early as five days after the cells are given. We used an investigational nuclear medicine test to reveal this early cell growth, which could be detected weeks before the cells appear in the blood. This radiology test is safe, does not cause any problems and is not invasive. It is called FLT (18F-fluorothymidine) and the contrast is taken up by dividing hematopoietic stem cells. The patients could even see the growth of their new cells inside the bone marrow (which they very much enjoyed while waiting to see recovery of the cells in their blood). We could use the brightness of the image (called SUV) to determine approximately how many weeks remained before the cells were visible in the blood.

Finally, we actually could see where the new cells went after they were infused, tracking their settling in various organs and bones. Through this, we could see that cells did not travel directly to all of the bones right away as was previously thought, but rather first went to the liver and spleen, then to the mid-spine (thorax), then to the remainder of the spine and breastplate, and finally to the arms and legs. This pattern of bone marrow development is seen in healthy developing fetuses. In this case, it occurs in a similar pattern in adults undergoing bone marrow transplant.

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Aspirin or Rivaroxaban for VTE Prophylaxis after Hip or Knee Arthroplasty?

MedicalResearch.com Interview with:

Dr. David R. Anderson, MD, FRCPC, FACP Faculty of Medicine Dean, Professor Dean, Faculty of Medicine Division of Hematology, Department of Medicine  & Nova Scotia Health Authority

Dr. Anderson

Dr. David R. Anderson, MD, FRCPC, FACP
Faculty of Medicine Dean, Professor
Dean, Faculty of Medicine
Division of Hematology, Department of Medicine
& Nova Scotia Health Authority

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Blood clots in the lungs (pulmonary embolism) and veins of the legs (deep vein thrombosis) are well recognized complications following total hip and knee arthroplasty surgeries.  Prior to the routine use of antithrombotic prophylaxis, pulmonary embolism was the most common cause of death following these procedures.  Oral anticoagulants such as rivaroxaban are commonly prescribed for the indication of preventing blood clots following total hip or knee arthroplasty.  For maximal benefit these agents are continued following surgery for up to five weeks following total hip arthroplasty and for two weeks following total knee arthroplasty.

There is evidence that aspirin has some benefit for the prevention of deep vein thrombosis and pulmonary embolism following total hip or knee arthroplasty.  However there is less evidence for its benefit than for oral anticoagulants.  We reasoned that aspirin would potentially be an attractive alternative for extended out of hospital prophylaxis following total hip or knee arthroplasty for patients who received a short course (5 days )of rivaroxaban following surgery.  Aspirin would be attractive for this indication because of its low cost, ease of use, and low rates of side effects.

Our study demonstrated that in a randomized controlled trial involving a large group (over 3400) of patients undergoing total hip or knee arthroplasty that extended therapy with aspirin was comparable to rivaroxaban for the prevention of deep vein thrombosis and pulmonary embolism following surgery.  Low rates of complications (< 1%) were observed with both treatment arms.  We also found that rates of clinically important bleeding complications (the most common side effect with antithrombotic drugs) were uncommon and similar with the two agents.

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Hemophilia B: Rebinyn® Now Available To Treat Acute Bleeds and Surgical Bleeding

MedicalResearch.com Interview with:

Stephanie Seremetis, M.D. Corporate Vice President and Chief Medical Officer Biopharmaceuticals at Novo Nordisk

Dr. Stephanie Seremetis

Stephanie Seremetis, M.D.
Corporate Vice President and Chief Medical Officer
Biopharmaceuticals at Novo Nordisk


MedicalResearch.com:
What is the background for this announcement?

Response: We’re proud and excited to make Rebinyn® (Coagulation Factor IX (Recombinant), GlycoPEGylated) available as a new extended half-life treatment for hemophilia B management.

Rebinyn® is an injectable medicine used to treat and control bleeding in adults and children with hemophilia B. It can be used to treat bleeds when they occur and to manage bleeding during surgery. Rebinyn® is not used for routine prophylaxis or for immune tolerance induction in patients with hemophilia B.

Hemophilia B is a serious, chronic, inherited bleeding disease that affects about 5,000 people in the U.S. People living with hemophilia B have low levels of clotting Factor IX protein in the blood, often resulting in prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.  Continue reading

Phase 3 Darzalex Trial Demonstrated Meaningful Improvement in Patients with Newly Diagnosed Multiple Myeloma

MedicalResearch.com Interview with:
janseen-oncologyMaria-Victoria Mateos, MD, PhD

University Hospital of Salamanca/IBSAL
Salamanca, Spain

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: The Phase 3 ALCYONE study data showed DARZALEX (daratumumab) in combination with bortezomib, melphalan, and prednisone (VMP) significantly improved clinical outcomes, including reducing the risk of disease progression or death by 50 percent, in newly diagnosed patients with multiple myeloma who are ineligible for autologous stem cell transplantation (ASCT) at a median follow-up of 16.5 months (Hazard Ratio [HR] = 0.50; 95 percent CI [0.38-0.65], p<0.0001).

The median progression-free survival (PFS) for DARZALEX-VMP had not yet been reached, compared to an estimated median PFS of 18.1 months for patients who received VMP alone. In addition to reducing the risk of disease progression or death, DARZALEX significantly improved the overall response rate (ORR) as compared to VMP alone, including more than doubling rates of stringent complete response, significantly improved rates of very good partial response or better and complete response or better (CR).

The most common (≥10 percent) Grade 3/4 treatment-emergent adverse events (TEAEs) for DARZALEX-VMP vs. VMP were neutropenia (40 percent vs. 39 percent), thrombocytopenia (34 percent vs. 38 percent), anemia (16 percent vs. 20 percent) and pneumonia (11 percent vs. 4 percent). One patient in each arm discontinued treatment due to pneumonia, and 0.9 percent of patients discontinued DARZALEX due to an infection. Twenty-eight percent of patients experienced infusion reactions (IRs) due to DARZALEX.. In the DARZALEX-VMP arm, 42 percent of patients experienced a serious adverse event (SAE), compared to 33 percent in the VMP arm.

The study findings were as a late-breaking abstract (Abstract #LBA-4) at the 59th American Society of Hematology (ASH) Annual Meeting in Atlanta, and simultaneously published in the New England Journal of Medicine (NEJM).

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Breakthrough Treatment With Prometic’s IV Plasminogen Treats Rare Disabling Disorder

MedicalResearch.com Interview with:
Dr. Charles T. Nakar, MD

Indiana Hemophilia and Thrombosis Center Pediatrics
Indianapolis, IN  

MedicalResearch.com: What is the background for this study?

Response: Congenital plasminogen deficiency is a rare genetic disorder that is caused by mutations in the PLG gene. Mutations in this gene lead to clinical manifestations such as fibrinous deposits on mucous membranes leading to disruption of tissue or organ function. These symptoms, when untreated, lead to significant morbidity and decreased quality of life. Life-threatening episodes may occur especially when the respiratory system is affected. There is currently no established approach to treatment of type 1 plasminogen deficiency and the available topical and systemic therapies (e.g. FFP, corticosteroids, immunomodulatory drugs, anticoagulants, amongst others) lack consistent efficacy. Patients may undergo multiple surgeries to remove lesions, but this approach typically leads to regrowth of lesions. Prometic’s intravenous plasminogen replacement therapy represents the first direct treatment for this serious disorder.

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Multiple Myeloma: Phase 3 Study of DARZALEX + VMP Reduced Risk of Disease Progression and Mortality

MedicalResearch.com Interview with:

Dr. Meletios A. Dimopoulos MD Professor and Chairman Department of Clinical Therapeutics University Athens School of Medicine Athens, Greece

Dr. Dimopoulos

DrMeletios A. Dimopoulos MD
Professor and Chairman
Department of Clinical Therapeutics
University Athens School of Medicine
Athens, Greece

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Updated data from the Phase 3 POLLUX trials showed DARZALEX, in combination with lenalidomide and dexamethasone, reduced the risk of disease progression or death by 56 percent, compared to lenalidomide and dexamethasone alone (Hazard Ratio [HR]=0.44; 95 percent CI [0.34-0.55], p<0.0001). After a median follow-up of 32.9 months, the median progression-free survival (PFS) in the DARZALEX arm has not been reached, compared with a median PFS of 17.5 months for patients who received lenalidomide and dexamethasone alone.

DARZALEX in combination with lenalidomide and dexamethasone also significantly increased the overall response rate (ORR) compared to lenalidomide and dexamethasone alone (93 percent vs. 76 percent, p<0.0001), including rates of complete response (CR) or better (55 percent vs. 23 percent, p<0.0001). DARZALEX also showed significantly higher (>3-fold) MRD-negative rates compared to lenalidomide and dexamethasone alone. These data were featured as an oral presentation (Abstract #739) at the 59th American Society of Hematology (ASH) Annual Meeting in early December.

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Daratumumab Monotherapy for Patients with Intermediate or High-Risk Smoldering Multiple Myeloma

MedicalResearch.com Interview with:

Craig C. Hofmeister, MD, MPH The Ohio State University 

Dr. Hofmeister

Craig CHofmeisterMD, MPH
The Ohio State University 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Preliminary data presented from the randomized, open-label, Phase 2 CENTAURUS (SMM2001) study evaluated three dosing schedules for DARZALEX monotherapy in patients with intermediate or high-risk smoldering multiple myeloma. A total of 123 patients were enrolled, with a median time since initial smoldering multiple myeloma diagnosis of 6.83 months (0.4-56). Patients were randomized to one of three treatment arms receiving DARZALEX 16 mg/kg intravenously in 8-week cycles: 1.) a long-intense dosing schedule (LONG) where DARZALEX was administered weekly in Cycle 1, every other week in Cycle 2-3, every 4 weeks in Cycle 4-7, and every 8 weeks up to Cycle 20; 2.) an intermediate dosing schedule (INT), where DARZALEX was given weekly for 1 cycle, and every 8 weeks up to Cycle 20 and; 3.) a short intense dosing schedule (SHORT), where DARZALEX was given weekly for 1 cycle. Results from the study showed DARZALEX monotherapy had a tolerable safety profile in patients with intermediate or high-risk smoldering multiple myeloma, with the most common treatment-emergent adverse events (TEAEs) being fatigue, cough, headache and insomnia. The efficacy endpoints included overall response rate, progression free survival, time to next treatment, and overall survival rate at 4 years. These study results serve as the basis for a Phase 3 study for DARZALEX in smoldering multiple myeloma, which is actively enrolling. These findings demonstrated DARZALEX had a manageable safety profile in patients with intermediate or high-risk smoldering multiple myeloma.

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Cost and Side Effects Influence Patients’ Preferences for Leukemia Medications

MedicalResearch.com Interview with:

Carol Mansfield, PhD, Senior Research Economist Health Preference Assessment RTI Health Solutions www.rtihs.org

Dr. Mansfield

Carol Mansfield, PhD,
Senior Research Economist

Health Preference Assessment
RTI Health Solutions
www.rtihs.org 

MedicalResearch.com: What is the background for this study?

Response: As the most prevalent form of leukemia, chronic lymphocytic leukemia (CLL) affects approximately 130,000 people in the United States. More than 20,000 new cases are diagnosed each year. In recent years, more treatment options–each with its own associated benefits, side effects, and price tag–have been approved. This leaves patients and physicians with a variety of factors they must consider when choosing a treatment plan.

While every patient wants the most effective drug with the fewest side effects, most people don’t have that option available. By asking patients to make tradeoffs and rank their preferences, we can form an understanding of how patients approach their treatment.

This study showed that patients with CLL value medicines that provide the longest progression-free survival, but are willing to trade some benefits for a lower risk of serious adverse events. Additionally, we found that cost clearly has an impact on which treatment a patient would choose. When patients get prescribed something they can’t afford, they are forced to make very difficult choices.

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Transfusions From Previously Pregnant Donors Add Risk To Younger Male Recipients

MedicalResearch.com Interview with:

Rutger Middelburg, PhD Assistant Professor in clinical epidemiology Sanquin Research and LUMC

Dr. Middelburg

Rutger Middelburg, PhD
Assistant Professor in clinical epidemiology
Sanquin Research and LUMC 

MedicalResearch.com: What is the background for this study?

Response: Six years ago we found transfusions from female donor to be associated with increased mortality among male recipients, especially under 50 years of age. This was an unexpected observation and we considered the probability of a false positive finding (i.e. a chance association) to be relatively high. We therefore immediately started a follow-up study with two main objectives. First, we wanted to confirm our findings in an independent and much larger cohort. Second, since some complications of blood transfusion are known to be related to pregnancy history of the donor, we wanted to study a possible relationship with previous pregnancy of the blood donors.

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Emergency Transfusion of Patients with Unknown Blood Type with Blood Group O Rhesus D Positive Blood

MedicalResearch.com Interview with:
Dr. med. Kathleen Selleng, OÄ, QB Hämotherapie

Universitätsmedizin Greifswald
Institut für Immunologie und Transfusionsmedizin,
Abt. Transfusionsmedizin
Sauerbruchstraße
Greifswald Deutschland

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Red blood cell concentrates (RBCs) of blood group O RhD negative are frequently used as universal blood for emergency transfusions in patients with unknown blood type. This leads to an over-proportional use of these red blood cell concentrates and regular shortages of O RhD negative RBCs.

Due to these shortages, patients with known RhD negative blood type sometimes have to be transfused with RhD positive RBCs.

The present study shows that the overall risk to induce an anti-D by transfusing all emergency patients with unknown blood type with O RhD positive RBCs is in the range of 3 to 6%, while this risk is much higher (20-30%) in RhD negative patients which have to be transfused with RhD positive RBCs due to RhD negative RBC shortages.

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