Author Interviews, Hematology, Leukemia, Pediatrics / 05.12.2018

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 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. (more…)
Author Interviews, Blood Pressure - Hypertension, Duke, Heart Disease, JAMA / 05.12.2018

MedicalResearch.com Interview with: Taku Inohara MD, PhD Duke Clinical Research Institute, Duke University Medical Center, Durham, North Carolina Department of Cardiology Keio University School of Medicine, Tokyo, Japan MedicalResearch.com: What is the background for this study? What are the main findings? Response: Transcatheter aortic valve replacement (TAVR) has been increasingly used for treating patients with severe aortic stenosis. Owing to the advancement of TAVR technology, the mortality and heart failure (HF) readmission after TAVR is decreasing over time, but 4.3% experienced readmission due to HF and 23.7% died within 1 year after TAVR. Inhibition of the renin-angiotensin system (RAS) with angiotensin-converting enzyme inhibitors (ACE-Is) or angiotensin-receptor blockers (ARBs) is known to improve clinical outcomes in patients with heart failure, but there remains unknown whether a RAS inhibitor is associated with a reduction in mortality and heart failure readmission after TAVR. Using the STS/ACC TVT Registry, a nationwide TAVR Registry in the US, we analyzed 15896 propensity-matched patients who underwent TAVR and found that receiving a prescription for a RAS inhibitor at discharge, compared with no prescription, was associated with a reduced risk for mortality ( 12.5% vs 14.9%) and HF readmission (12.0% vs 13.8%). (more…)
Author Interviews, Brigham & Women's - Harvard, Social Issues / 05.12.2018

MedicalResearch.com Interview with: Professor Tyler VanderWeele Ph.D John L. Loeb and Frances Lehman Loeb Professor of Epidemiology Harvard University MedicalResearch.com: What is the background for this study? What are the key points of the paper?   Response: Several prior studies have suggested that religious service attendance is associated with lower rates of divorce. However, many of these studies have been with small samples and have not had rigorous study designs. In addition, most studies have focused on women earlier in life and there has been little research on the effects of religious service attendance on divorce later in life. While divorce rates in the United States in general has been falling, it has in fact been increasing for middle-aged groups, doubling between 1990 and 2010. In our study we found that among women in mid- to late- life, regular religious service attendance was subsequently associated with 50% lower divorce rates over the following 14 years of the study. We also found that among those who were widowed, religious service attendance was associated with a 49% increase in the likelihood of remarrying over the 14 years of the study. (more…)
Author Interviews, Infections, NIH, Ophthalmology / 05.12.2018

MedicalResearch.com Interview with: Top, retina of a control patient. Bottom, retina from a patient with CJD. Arrowheads point to abnormal prions in the outer plexiform layer (opl), and the asterisk (*) marks more diffuse prions in the inner plexiform layer (ipl).Orrù et al., mBioByron Caughey, Ph.D. Senior Investigator Chief, TSE/prion Biochemistry Section Laboratory of Persistent Viral Diseases NIH/NIAID Rocky Mountain Laboratories Hamilton, MT 59840 USA  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Corneal transplants have caused the transmission of Creutzfeldt-Jakob disease (CJD) in at least two cases, and pathological prion protein has been detected in the retinas of the eyes of sporadic CJD cases. To build on these previous indications of prions in eye tissue, we tested the distribution of prions in various components of eyes from 11 sCJD decedents. We applied a highly sensitive surrogate test for prions (RT-QuIC) that indicated that all of the sCJD cases had prions in multiple parts of their eye, including the cornea and sclera, which is the white outer surface of the eye. Retinas were usually contained the highest levels, in some cases approaching levels in the brain. Some other parts such as the cornea, lens and vitreous had much lower, but detectable, levels.  (more…)
ADHD, Author Interviews, Pediatrics, Race/Ethnic Diversity / 05.12.2018

MedicalResearch.com Interview with: George J. DuPaul, PhD Department of Education and Human Services Lehigh University   Charles Barrett. Ph.D. School Psychologist Loudon County Virginia Public Schools   MedicalResearch.com: What is the background for this study? What are the main findings? Response: Numerous studies have shown that Black children are more likely to receive ratings that are more indicative of displaying externalizing behavior difficulties, including Attention Deficit Hyperactivity Disorder (ADHD).  However, many of these studies included teachers as the informants. Consistent with most teachers in the United States, raters have typically been White females.  For this reason, it is unclear if these outcomes would exist if the rater and child shared the same racial/ethnic background. Additionally, most research in the United States that involved cross-cultural comparisons has used White and Hispanic boys.  Few empirical studies have examined differences between Black and White boys. The present study sought to address several limitations in the field.  Most notably, cross-cultural comparisons between Black and White boys were included instead of Hispanic and White children.  Next, maternal figures, rather than teachers, were included as the informants. The present study was developed using a similar methodology that examined Hispanic and White boys’ behavior from the perspective of Hispanic and White teachers (Dominguez de Ramirez & Shapiro, 2005). In sum, we sought to determine if there were differences in how Black and White maternal figures rated Black and White boys who were demonstrating the same level/type of behavior (i.e., sub-clinical levels of ADHD).  Notably, although the boys’ behaviors were the same, maternal ratings were not identical. Specifically, using the ADHD Rating Scale, Fourth Edition (ARS-4), Black mothers assigned higher ratings to both Black and White boys. (more…)
Author Interviews, Autism, Pediatrics / 05.12.2018

MedicalResearch.com Interview with: Michael Kogan, Ph.D. Director of the office of Epidemiology and Research Health Resources and Services Administration’s Maternal and Child Health Bureau MedicalResearch.com: What is the background for this study? What are the main findings? Response: This was a study led by the Health Resources and Services Administration’s Maternal and Child Health Bureau, along with researchers from the Centers from Disease Control and Prevention, Harvard, Drexel, and George Washington Universities.  We used the 2016 National Survey of Children’s Health, a nationally representative survey of over 50,000 children that examines the health and well-being of US children, to examine the prevalence, treatment, and health care experiences of children with Autism Spectrum Disorder (ASD). We found that 1 out of 40 children in the US were reported by their parents to have been diagnosed with Autism Spectrum Disorder.  We also found that children with ASD were significantly less likely to receive services like needed care coordination, referrals to other services, and mental health counseling – even compared to children with other emotional, behavioral or developmental disorders (EBDs).  Parents of children with ASD were also significantly more likely to report being usually or always frustrated in their attempts to get services, again compared to families of children with other EBDs. Finally, we looked at treatment patterns for children with ASD and found that 64% had received behavioral therapy in the year before the interview, and 27% had received medications to treat symptoms of irritability.  (more…)
Author Interviews, OBGYNE, Science / 05.12.2018

MedicalResearch.com Interview with: "38 week fetus" by Zappys Technology Solutions is licensed under CC BY 2.0Kimberley Whitehead Neuroscience, Physiology & Pharmacology University College London MedicalResearch.com: What is the background for this study? Response: Fetuses move a lot! Very similar movement patterns are seen in both pre-term and full-term newborn infants, but their function is unclear. In animals such as rats, spontaneous movement and consequent feedback from the environment during the early developmental period trigger specific patterns of electrical activity in the brain that are necessary for proper brain mapping. (more…)
Author Interviews, Education, Sleep Disorders / 04.12.2018

MedicalResearch.com Interview with: Michael K. Scullin, Ph.D. Assistant Professor of Psychology and Neuroscience Director, Sleep Neuroscience and Cognition Laboratory Baylor University Waco, TX 76798  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: There is a gap between what health behaviors individuals know they should adopt, and what those individuals actually end up doing. For example, a growing literature shows that simply educating students on the importance of sleep does not change their sleep behaviors. Thus, we need to think outside of the box for solutions. In three classes, we have now investigated a motivational solution: if students can earn extra credit on their final exam for sleeping better, will they do so even during finals week? (more…)
Author Interviews, Cost of Health Care, Hematology, J&J-Janssen, Thromboembolism / 04.12.2018

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Clots - Coagulation, Hematology, J&J-Janssen / 04.12.2018

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, FDA, JAMA, Medical Imaging / 04.12.2018

MedicalResearch.com Interview with: Aldo Badano, Ph.D. Deputy Director, Division of Imaging, Diagnostics, and Software Reliability Office of Science and Engineering Laboratories Center for Devices and Radiological Health Silver Spring, MD 20993 Aldo Badano, Ph.D. Deputy Director, Division of Imaging, Diagnostics, and Software Reliability Office of Science and Engineering Laboratories Center for Devices and Radiological Health Silver Spring, MD 20993  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Expensive and lengthy clinical trials can delay regulatory evaluation of innovative technologies, affecting patient access to high-quality medical products. Although computational modeling is increasingly being used in product development, it is rarely at the center of regulatory applications. Within this context, the VICTRE project attempted to replicate a previously conducted imaging clinical trial using only computational models. The VICTRE trial involved no human subjects and no clinicians. All trial steps were conducted in silico. The fundamental question the article addresses is whether in silico imaging trials are at a mature development stage to play a significant role in the regulatory evaluation of new medical imaging systems. The VICTRE trial consisted of in silico imaging of 2986 virtual patients comparing digital mammography (DM) and digital breast tomosynthesis (DBT) systems. The improved lesion detection performance favoring DBT for all breast sizes and lesion types was consistent with results from a comparative trial using human patients and radiologists.  (more…)
Annals Internal Medicine, Author Interviews, Heart Disease, Infections, Opiods / 04.12.2018

MedicalResearch.com Interview with: Asher Schranz, MD Division of Infectious Disease Department of Medicine UNC School of Medicine MedicalResearch.com: What is the background for this study? Response: The opioid crisis has led to several major infectious diseases concerns, including HIV and Hepatitis C. Drug use-associated infective endocarditis (DUA-IE) is a less commonly discussed consequence of the opioid epidemic. DUA-IE is an infection of one or more heart valves that occurs from injecting drugs. It can be a severe, life-threatening infection and requires a long course of intravenous antibiotics as well as, in some cases, open heart surgery to replace an infected heart valve. Several studies over the past few years have shown that DUA-IE has been increasing. Our study examined hospital discharges in North Carolina statewide from 2007 to 2017. We sought to update trends in DUA-IE and describe how much heart valve surgery was being performed for DUA-IE. We also aimed to report the demographics of persons who are undergoing heart valve surgery for DUA-IE and the charges, lengths of stay and outcomes of these hospitalizations.  (more…)
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 04.12.2018

MedicalResearch.com Interview with: 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.  (more…)
Annals Internal Medicine, Author Interviews, Lipids, Pharmacology / 04.12.2018

MedicalResearch.com Interview with: Prof. Dr. Milo Puhan Epidemiology, Biostatistics and Prevention Institute University of Zurich MedicalResearch.com: What is the background for this study? What are the main findings? Response: The use of statins for primary cardiovascular prevention is controversial and there is a debate at what risks statins provide more benefits than harms. Current guidelines recommend statins if the 10 year risk for cardiovascular events is above 7.5 to 10% and they do not distinguish between men and women, different age groups and different statins. We found in our study that the benefits of statins exceeds the harms if the 10 year risk for cardiovascular events is above 14% for middle aged mean (40-44 years) but even higher for older age groups, and women. In addition, the benefit harm balance varies substantially between different statins with atorvastatin providing the best benefit harm balance. (more…)
Author Interviews, Dermatology, Lipids / 04.12.2018

MedicalResearch.com Interview with: Wendy Bollag, PhD, FAHA Professor of Physiology VA Research Career Scientist Augusta University, Georgia MedicalResearch.com: What is the background for this study? Response: We have previously shown that the lipid (fat) phosphatidylglycerol (PG) is able to inhibit rapidly growing keratinocytes (skin cells) and promote their maturation. We also found that PG can suppress skin inflammation. Since the common skin disease psoriasis is characterized by inflammation and excessive growth and abnormal maturation of skin cells, we believed that PG might be useful as a treatment. However, the mechanism of its anti-inflammatory effect was unknown. PG in the lung has been found to inhibit inflammation induced by microbes or their components, which work by activating the innate immune system via binding to proteins called toll-like receptors (TLRs); however, psoriasis is not considered to be an infectious disease. We hypothesized that PG would also inhibit inflammation induced by anti-microbial peptides that activate TLRs. Anti-microbial peptides, produced normally by the skin to protect against infection, are known to be excessively up-regulated in psoriatic skin.  (more…)
Author Interviews, Dental Research, JAMA, Opiods, Pediatrics / 03.12.2018

MedicalResearch.com Interview with: Alan Schroeder MD Associate Chief for Research Division of pediatric hospital medicine Lucile Packard Children’s Hospital Stanford MedicalResearch.com: What is the background for this study? What are the main findings? Response: Third molar “wisdom teeth” extractions are one of the most common surgeries performed in adolescents and young adults, but an adequate appraisal of risks and benefits is lacking. Most patients who undergo this procedure are exposed to opioids post-operatively. We demonstrate that, for privately-insured opioid-naïve patients 16-25 years of age, exposure to opioids from a dental provider is associated with persistent use at 90-365 days in 7% of patients and a subsequent diagnosis relating to abuse in 6% of patients. In contrast persistent use and abuse were significantly lower in control patients not exposed to dental opioids (0.1% and 0.4%, respectively). The median number of pills dispensed for the initial prescriptions was 20. (more…)
Author Interviews, Breast Cancer, Mammograms / 03.12.2018

MedicalResearch.com Interview with: Stamatia Destounis MD, FACR, FSBI, FAIUM Elizabeth Wende Breast Care Clinical Professor University of Rochester Imaging Sciences Rochester, NY 14620  MedicalResearch.com: What is the background for this study? What are the main findings? Response:  The current breast cancer screening recommendations in the United States are unclear regarding when to stop screening. Several societies with published recommendations conflict in regard to when to discontinue screeningmammography. There is little evidence studying the benefit of annual mammography in the population of women 75 and older. Due to this, we felt that it was a very important and timely topic to investigate, with the goal of providing further guidance on why screening mammography may be beneficial in this older population. (more…)
Anemia, Author Interviews, Cancer Research, Hematology, Leukemia / 03.12.2018

MedicalResearch.com Interview with: 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.  (more…)
Author Interviews, Cancer Research, Hematology / 03.12.2018

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Cannabis, University of Pennsylvania / 02.12.2018

MedicalResearch.com Interview with: Daniel Romer PhD Annenberg Public Policy Center The University of Pennsylvania Philadelphia, PA MedicalResearch.com: What is the background for this study? What are the main findings? Response: Previous research has found some troubling relations between adolescent cannabis use and subsequent increases in conduct problems and other unhealthy consequences.  These studies were done in New Zealand in the late 90’s and we wanted to re-examine those relationships using more contemporary data in the US. We had data on 364 adolescents who were followed from age 13 to 19 in Philadelphia that could provide a more up to date picture of the effects of using cannabis on one important outcome, conduct disorder.  We also wanted to use more sensitive methods than had been used in prior research that would enable us to examine reciprocal relations between cannabis use and c (CP).  That is, it might be the case that youth with CP are prone to using cannabis and that this helps to explain why there appears to be a relation over time between cannabis use and CP rather than cannabis use leading to CP. Our findings supported that hypothesis.  There was no prospective relation between changes in cannabis use and subsequent changes in conduct problems.  Instead, changes in conduct problems were found to predict changes in use of cannabis.  Youth with conduct problems also affiliated more with peers who used cannabis, adding further to their own use.  There was also no evidence that youth who used cannabis sought out peers who used it apart from the effects of CP. Finally, both use of cannabis and  conduct problems predicted subsequent development of a mild cannabis use disorder (CUD).  (more…)
Author Interviews, Cancer Research, JAMA, OBGYNE / 02.12.2018

MedicalResearch.com Interview with: Dr. Weimin Ye, MD MSC, PhD Department of Medical Epidemiology and Biostatistics Karolinska Institue MedicalResearch.com: What is the background for this study? What are the main findings? Response: Polycystic ovary syndrome (PCOS) is the most common endocrine    disorder affecting 5-10% of women of reproductive age. Characterized by hyperandrogenism and metabolic abnormalities, PCOS is known to be related to various long-term health consequences, including diabetes, cardiovascular disease and endometrial cancer. Besides, inconsistent results have been reported for the associations between PCOS and the risk of ovarian and breast cancer. Studies addressing the risks of other cancers are scarce. Thus, we conducted a large, population-based cohort study with a long follow-up and rather sufficient confounding adjustment to explore the full picture of associations between PCOS and the risks of various cancer types. We found that PCOS is a risk factor for certain types of cancer, including cancers of the endometrium, ovary, endocrine gland, pancreas, kidney and skeletal & hematopoietic system. (more…)
Author Interviews, Hematology / 02.12.2018

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 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 (more…)
Addiction, Author Interviews, Methamphetamine, OBGYNE, Opiods / 02.12.2018

MedicalResearch.com Interview with: Lindsay Admon, MD MSc Assistant Professor, Department of Obstetrics & Gynecology Institute for Healthcare Policy and Innovation University of Michigan MedicalResearch.com: What is the background for this study? What are the main findings? In our previous work (https://journals.lww.com/greenjournal/Fulltext/2017/12000/Disparities_in_Chronic_Conditions_Among_Women.19.aspx), we identified higher rates of deliveries complicated by substance use among rural women. We knew that some of this difference would be accounted for by opioids.What we didn’t expect was that when we took a closer look, amphetamine use disorder accounted for a significant portion of this disparity as well. The main findings of this study are that, between 2008-09 and 2014-15, amphetamine and opioid use among delivering women increased disproportionately across rural compared to urban counties in three of four census regions. By 2014-15, amphetamine use disorder was identified among approximately 1% of all deliveries in the rural western United States, which was higher than the incidence of opioid use in most regions. Compared to opioid-related deliveries, amphetamine-related deliveries were associated with higher incidence of the majority of adverse gestational outcomes that we examined including pre-eclampsia, preterm delivery, and severe maternal morbidity and mortality.   (more…)
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 02.12.2018

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Cost of Health Care, Hematology, J&J-Janssen, Lymphoma / 02.12.2018

MedicalResearch.com Interview with: 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. (more…)
Allergies, AstraZeneca, Author Interviews / 01.12.2018

MedicalResearch.com Interview with: Mr. Tosh Butt Vice President, Respiratory AstraZeneca Mr. Butt discusses the recent announcement that the FDA has granted Orphan Drug Designation for Fasenra for the treatment of Eosinophilic Granulomatosis with Polyangiitis.  MedicalResearch.com: What is the background for this announcement? Can you tell us a little more about Eosinophilic Granulomatosis with Polyangiitis/Churg Strauss? How does it differ/resemble severe eosinophilic asthma?
  • The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for FASENRA™ (benralizumab) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA). The ODD application was based on epidemiology demonstrating the rarity of the disease (<200k US patients) and a scientific rationale that FASENRA may benefit patients with this condition. The core role of eosinophilia in EGPA and FASENRA’s demonstrated eosinophil-depleting properties provided this rationale and suggest it may deliver benefit to patients with EGPA.
  • EGPA is a rare autoimmune disease that can cause damage to multiple organs and tissues. EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell. All patients with EGPA have very high levels of eosinophils at some point in their disease. FASENRA induces rapid and near-complete depletion of eosinophils in the blood and has proven efficacy in severe eosinophilic asthma, which suggest it may deliver benefit to patients with EGPA. 
(more…)
Author Interviews, Compliance, JAMA / 01.12.2018

MedicalResearch.com Interview with: Dr. Andrea Gurmankin Levy, PhD MBE Department of Social Sciences Middlesex Community College, Middletown, Connecticut MedicalResearch.com: What is the background for this study? Response: It is so important for clinicians to get accurate information from their patients so that they can make accurate diagnoses and appropriate recommendations. But we know that people tend to withhold information from others, and that this is especially true when it comes to sensitive information. And in fact, in medicine, there is a long-standing conventional wisdom that clinicians need to adjust patients’ answers (e.g., doubling patients’ report of alcohol consumption) to get a more accurate picture. So we wanted to explore this. How many patients withhold medically-relevant information from their clinicians, and why do they do so?  There have been surprisingly few studies looking at this question in a comprehensive way. (more…)
Author Interviews, Dermatology, JAMA, Technology / 01.12.2018

MedicalResearch.com Interview with: Philipp Tschandl, MD PhD, Priv.Doz. Assistant Professor Department of Dermatology Medical University of Vienna MedicalResearch.com: What is the background for this study? What are the main findings? Response: Dermatoscopy is a non-invasive imaging technique, where the surface of the skin is rendered translucent and additional important morphologic features become visible from deeper layers. This is achieved through use of immersion fluid or cross-polarised light - equivalent to the effect when using a pair of goggles to look underwater, or polarised sunglasses to reduce glare on glass surfaces. After the first description of “Dermatoskopie" almost 100 years ago by a German dermatologist (Johann Saphier), this technique has evolved to a successful, low-cost, state-of-the-art technique for clinical skin cancer detection in the last decades. Convolutional neural networks (“CNN”) are powerful machine learning methods, and frequently applied to medical image data in the recent scientific literature. They are highly accurate for basic image classification tasks in experimental settings, and found to be as good as dermatologists in melanoma recognition on clinical or dermatoscopic images. In this study we trained a CNN to diagnose non-pigmented skin lesions (where melanomas are only a minority) through analysis of digital images, and compared the accuracy to >90 human readers including 62 board-certified dermatologists. This study expands knowledge in the following ways compared to previous work: - We applied the network for the detection of non-pigmented skin cancer, which is far more common in the (caucasian) population than melanoma. - We created a prediction model that combines analysis of a dermatoscopic and clinical image (“cCNN”) which is able to further increase diagnostic accuracy. - We compared accuracy not only to experts, but users with different level of experience (more…)
Author Interviews, Diabetes, Geriatrics, JAMA, Kidney Disease / 01.12.2018

MedicalResearch.com Interview with: Dr. Ziyad Al-Aly, MD Associate Chief of Staff for Research and Education Veterans Affairs St. Louis Health Care System Institute for Public Health Washington University, St. Louis MO MedicalResearch.com: What is the background for this study? Response: A lot has changed in the US over the past 15 years including aging, population growth, and increased exposure to risk factors such as obesity, elevated blood pressure, etc. With all of these changes, we wondered, how did the burden of kidney disease change in the United States over the past 15 years. (more…)
Author Interviews, Baylor College of Medicine Houston, Infections, Microbiome, OBGYNE, Vaccine Studies / 30.11.2018

MedicalResearch.com Interview with: Sasirekha Ramani, PhD Assistant Professor Molecular Virology and Microbiology Baylor College of Medicine Houston, TX MedicalResearch.com: What is the background for this study? Response: This work pertains to Rotavirus, a leading cause of diarrhea and vomiting in children under the age of 5 years. In this paper, we described our work with a rotavirus strain that almost exclusively causes neonatal infections. For many years, we have been trying to understand why this strain primarily infects newborns and why infection in some babies is associated with gastrointestinal symptoms while others are asymptomatic. A few years ago, we showed that this particular virus binds to developmentally-regulated glycans (sugars) in the gut as receptors. As the baby grows, these sugars get modified, and that potentially explains why infection with this virus is primarily restricted to neonates. However, we didn’t really have to answer to why there are differences in association with clinical presentations. (more…)