Author Interviews, Pediatrics, Smoking, Tobacco, Tobacco Research, University Texas / 10.07.2018

MedicalResearch.com Interview with: “hookah” by Ksenia M is licensed under CC BY 2.0Cheryl L. Perry, Ph.D. Professor of Health Promotion and Behavioral Sciences The Rockwell Distinguished Chair in Society and Health University of Texas Health Science Center at Houston (UTHealth) School of Public Health, Austin, Texas MedicalResearch.com: What is the background for this study? What are the main findings? Response: There have been large changes in the social environment over the past 10 years that have affected tobacco use among youth and young adults. These include social media, e-cigarettes, and new regulations aimed at preventing use among youth. Historically, nearly all onset of tobacco use, particularly cigarettes, occurred prior to high school graduation by age 18. Some recent national cross-sectional data suggested that onset might be occurring among young adults. We decided to explore, with national and Texas data, whether onset of tobacco use was more likely to occur among young adults. We did this by analyzing data from 3 studies over one year. (more…)
Author Interviews, Cancer Research, Lancet / 10.07.2018

MedicalResearch.com Interview with: Paul Lyon DPhil, MRCS Academic Clinical Fellow in Radiology Oxford University Hospitals NHS Foundation Trust Oxford, UK MedicalResearch.com: What is the background for this study? What are the main findings? Response: Delivering therapeutic doses of systemic chemotherapy to solid tumours, whilst ensuring side effects remain tolerable, has a presented a long-standing and unsolved challenge in oncology. With the advent of smart nanomedicines for clinical use, such as Lyso-Thermosensitive Liposomal Doxorubicin (LTLD, ThermoDox®, Celsion, USA), which has been formulated to release its doxorubicin content at 2.5°C above body temperature, there is now opportunity for targeted tumour therapy in combination with therapeutic devices. Much like a magnifying glass can focus energy from the sun to burn a hole in paper, ultrasound can be focused deep within the body to induce therapeutic effects in tumours, including ablation, hyperthermia and other bioeffects. Since its inception in the 1940s, focused (or therapeutic) ultrasound has evolved and is now FDA-approved for a variety of indications including ablation of several tumour types, virtue of being safe, non-invasive and non-ionising. Building on decades of preclinical research efforts worldwide, the TARDOX study is the first clinical trial to attempt triggered drug delivery to a target tumour non-invasively using an external focused ultrasound device. This phase 1 study which ran between March 2015-March 2017 in Oxford, UK, treated 10 patients with inoperable primary or secondary liver tumours which were either stable or refractory to previous chemotherapies. In each patient, a single intervention under general anaesthetic was performed during which a selected liver tumour was targeted and gently heated with focused ultrasound following an intravenous infusion of LTLD. Biopsies were used to determine the quantity of intratumoral doxorubicin before and after the ultrasound exposure.  (more…)
Author Interviews, Infections / 10.07.2018

MedicalResearch.com Interview with: Thomas A Russo, MD, CM The Departments of Medicine, and Microbiology and Immunology The Witebsky Center for Microbial Pathogenesis University at Buffalo-State University of New York, and the Veterans Administration Western New York Healthcare System Buffalo, New York MedicalResearch.com: What is the background for this study? What is Klebsiella pneumoniae? Response: K. pneumoniae is an important bacterial pathogen that cause a number of different infections. Presently, two pathotypes exist that behave very differently. Classical K. pneumoniae, which is most common in North America and Europe primarily causes infections in the healthcare setting, usually in patients with co-morbidities. Also, it is becoming increasingly antimicrobial resistant, making treatment challenging. Hypervirulent K. pneumoniae, which is more common in the Asian Pacific Rim,  can cause infections in otherwise healthy individuals, often causes infection in multiple sites, and these sites are usually not infected by classical K. pneumonia, such as the eye, brain, and aggressive soft-tissue infection (necrotizing fasciitis). Hypervirulent K. pneumonia strains are also becoming antimicrobial resistant, albeit at a slower rate than classical K. pneumoniae at this time. There are some differences how infections due to these two pathotypes are managed. It would also be ideal to track the prevalence and relative antimicrobial resistance of these two pathotypes, but up until now this could not be reliably done because there was not a validated test that could differentiate them. The goal of this study was to identify biomarkers that could accurately differentiate classical from hypervirulent K. pneumoniae.  (more…)
Aging, Author Interviews, Genetic Research / 09.07.2018

MedicalResearch.com Interview with: “siblings” by Katina Rogers is licensed under CC BY 2.0Stacy L. Andersen, PhD Assistant Professor of Medicine Project Manager New England Centenarian Study Long Life Family Study Boston University School of Medicine Boston Medical Center Boston, MA 02118 MedicalResearch.com: What is the background for this study? Response: Exceptional longevity appears to run in families. Previous studies have found that people who have siblings who live into their 90s or who reach 100 years of age have a greater chance themselves of living longer than the general population. Yet it is supercentenarians, those who reach the age of 110 years, who represent the true extreme of the human lifespan.  We wanted to determine whether the parents and siblings of supercentenarians were more likely to reach very old ages than family members of younger centenarians. We collected family tree information for 29 participants of the New England Centenarian Study aged 110-119 years. Proof of age documents and familial reconstruction methods were used to validate ages and dates of birth and death of the supercentenarian as well as his or her parents and siblings. Mean age at death was compared to birth year and sex-specific US and Swedish cohort life table estimates conditional on survival to age 20 for siblings to omit deaths due to nonheritable factors such as infectious disease or accidents and survival to age 50 (the approximate age at which women are no longer able to reproduce) for parents.  (more…)
Author Interviews, Columbia, Neurology / 09.07.2018

MedicalResearch.com Interview with: Andrew Goldman PhD Laboratory for Intelligent Imaging and Neural Computing Department of Biomedical Engineering, Columbia University Presidential Scholar in Society and Neuroscience, Columbia University Andrew Goldman PhD Laboratory for Intelligent Imaging and Neural Computing Department of Biomedical Engineering, Columbia University Presidential Scholar in Society and Neuroscience, Columbia University  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Many Western musicians have difficulty improvising, despite having extensive training and experience. These musicians learn about and use similar musical structures in their playing (like chords, scales, rhythmic patterns, etc.) as experienced improvisers, but they may know about them in different ways. In other words, different musicians have different ways of knowing and learning about similar musical structures. To understand which ways of knowing facilitate the ability to improvise contributes to an understanding of how people are able to use knowledge creatively. Western music provides an important opportunity to compare these different ways of knowing because in other improvisatory domains of behavior (like speaking), it is difficult to find people who know how to do it but cannot improvise with it (e.g., if you know a language, you can very likely improvise with that language). In order to advance our understanding of these improvisatory ways of knowing, we compared musicians with varying degrees of improvisation experience in a task that tested how they categorized musical chords. In Western music, different chords are theorized to have similar “functions.” For example, on a guitar, there are different ways to play a C chord, and you could often substitute one for the other. You might even play another chord in place of the C chord and have it sound similar, or lead to a similar subsequent harmony. Improvisers often use notation that specifies classes of chords rather than specific realizations (versions) of a chord whereas those who do not typically improvise use notation that specifies the full realization of the chord. By analogy, one chef might use a recipe that calls for “citrus” (in music, a class of musical chord) while another chef’s recipe might specifically call for “lemon” (in music, a specific realization of a functional class of chords). We tested whether improvisers categorize similar-functioning harmonies as more similar to each other than different-functioning harmonies, and compared how less experienced improvisers categorize the same harmonies. Our task required the musicians to listen to a series of repeating harmonies (the “standard” stimuli) and pick out occasional chords that were different in any way (the “deviant” stimuli). Some deviant stimuli were different versions of the standard chord (like limes in place of lemons) and some deviant stimuli were chords with different musical functions (like bananas instead of lemons). The more experienced improvisers were better at detecting the function deviants than the exemplar deviants whereas the less experienced improvisers showed little difference in their ability to detect the two types of deviants. In other words, because improvisers categorize the different versions of the same chord as similar, they have a relatively harder time picking out the similarly functioning harmonies. This was measured using behavioral data, and electroencephalography (EEG), which can be used to provide a neural measure of how different stimuli are perceived to be from each other. (more…)
Author Interviews, JAMA, Opiods / 09.07.2018

MedicalResearch.com Interview with: Tyler Winkelman MD, MSc  Clinician-Investigator Division of General Internal Medicine, Hennepin Healthcare Center for Patient and Provider Experience, Hennepin Healthcare Research Institute Assistant Professor Departments of Medicine & Pediatrics University of Minnesota MedicalResearch.com: What is the background for this study? Response: Opioid overdose deaths continue to escalate, and there have been reports that jails and prisons are bearing the brunt of the opioid epidemic. However, it wasn’t known, nationally, how many people who use opioids were involved in the criminal justice system. We also didn’t have recent estimates of common physical and mental health conditions among people with different levels of opioid use. We used two years of national survey data to understand these associations, which are critical in developing a public health response to the opioid epidemic. (more…)
Author Interviews, Colon Cancer, JNCI, NIH, Vitamin D / 09.07.2018

MedicalResearch.com Interview with: Stephanie J. Weinstein, M.S., Ph.D.  Metabolic Epidemiology Branch, Division of Cancer Epidemiology and Genetics National Cancer Institute, NIH   MedicalResearch.com: What is the background for this study?   Response: Vitamin D, known for its role in maintaining bone health, is hypothesized to lower colorectal cancer risk via several pathways related to cell growth and regulation. Previous prospective studies have reported inconsistent results for whether higher concentrations of circulating 25-hydroxyvitamin D, the accepted measure of vitamin D status, are linked to lower risk of colorectal cancer. The few randomized clinical trials of vitamin D supplementation and colorectal cancer completed thus far have not shown an effect; but study size, relatively short supplementation duration, and only moderate compliance may have contributed to their null findings. To address inconsistencies in prior studies on vitamin D, and to investigate associations in population subgroups, we harmonized and analyzed participant-level data from over 5,700 colorectal cancer cases who had blood collected before colorectal cancer diagnosis, and 7,100 matched cancer-free controls. Study participants were drawn from 17 prospective cohorts from the United States, Europe, and Asia and were followed for an average of 5.5 years (range: 1 – 25 years). We used a single, widely accepted assay and laboratory for new vitamin D measurements and calibrated existing vitamin D measurements. In the past, substantial differences between assays made it difficult to integrate vitamin D data from different studies. Our novel calibration approach enabled us to explore risk systematically over the broad range of vitamin D levels seen internationally.  (more…)
Author Interviews, Beth Israel Deaconess, HIV, Lancet, Vaccine Studies / 08.07.2018

MedicalResearch.com Interview with: Dan Barouch, M.D., Ph.D. Professor of Medicine Harvard Medical School Ragon Institute of MGH, MIT, and Harvard Director, Center for Virology and Vaccine Research Beth Israel Deaconess Medical Center Boston, MA 02215 MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study demonstrates that the mosaic Ad26/Env HIV vaccine candidate induced robust and comparable immune responses in humans and monkeys. Moreover, the vaccine provided 67% protection against viral challenge in monkeys.    (more…)
Author Interviews, Cost of Health Care, Geriatrics / 07.07.2018

MedicalResearch.com Interview with: Jonathan H. Watanabe, PharmD, PhD, BCGP Associate Professor of Clinical Pharmacy National Academy of Medicine Anniversary Fellow in Pharmacy Division of Clinical Pharmacy | Skaggs School of Pharmacy and Pharmaceutical Sciences | University of California San Diego La Jolla, CA  Jonathan H. Watanabe, PharmD, PhD, BCGP Associate Professor of Clinical Pharmacy National Academy of Medicine Anniversary Fellow in Pharmacy Division of Clinical Pharmacy | Skaggs School of Pharmacy and Pharmaceutical Sciences | University of California San Diego La Jolla, CA MedicalResearch.com: What is the background for this study? What are the main findings?  Response: As a clinician in older adult care and as a health economist, I’ve been following the news and research studies on older patients unable to pay for their medications and consequently not getting the treatment they require. Our goal was to measure how spending on the medications Part D spends the most on, has been increasing over time and to figure out what prices patients are facing out-of-pocket to get these medications. In 2015 US dollars, Medicare Part D spent on the ten highest spend medications increased from $21.5 billion in 2011 to $28.4 billion in 2015.  The number of patients that received one of the ten highest spend medications dropped from 12,913,003 in 2011 to 8,818,471--- a 32% drop in that period. A trend of spending more tax dollars on fewer patients already presents societal challenges, but more troubling is that older adults are spending much more of their own money out-of-pocket on these medications.  For patients without a federal low income subsidy, the average out-of-pocket cost share for one of the ten highest spend medications increased from $375 in 2011 to $1,366 in 2015.  This represented a 264% increase and an average 66% increase per year.  For patients receiving the low income subsidy, the average out-of-pocket cost share grew from $29 in 2011 to $44 in 2015 an increase of 51% and an average increase of 12.7% per year.  This may not sound like much, but for those living close to the federal poverty level this can be the difference between foregoing necessities to afford your medications or choosing not to take your medications.   (more…)
Author Interviews, Kidney Disease, Social Issues / 07.07.2018

MedicalResearch.com Interview with: Dr. Ann M. O’Hare, MD Professor,Division of Nephrology University of Washington Investigator, VA HSR&D Center of Excellence Affiliate Investigator, Group Health Research Institute Seattle, WA MedicalResearch.com: What is the background for this study? What are the main findings? Response: We set out to conduct a qualitative study among patients with advanced kidney disease to learn about their thoughts and experience with advance care planning. Our questions, especially at the beginning of the interview were quite broad and asked patients more generally about their experiences of illness and care. Although we did not ask patients about the emotional impact of illness and care, this came across as a strong theme when we analyzed the interviews, and that is what we describe here. (more…)
Alzheimer's - Dementia, Author Interviews, Obstructive Sleep Apnea, Sleep Disorders / 07.07.2018

MedicalResearch.com Interview with: “Woman sleeping” by Timothy Krause is licensed under CC BY 2.0Nathan E. Cross PhD, first author School of Psychology. Sharon L. Naismith, PhD, senior author Leonard P Ullman Chair in Psychology Brain and Mind Centre Neurosleep, NHMRC Centre of Research Excellence The University of Sydney, Australia  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Between 30 to 50% of the risk for dementia is due to modifiable risk factors such depression, hypertension, physical inactivity, obesity, diabetes and smoking. In recent years, multiple longitudinal cohort studies have observed a link between sleep apnoea and a greater risk (1.85 to 2.6 times more likely) of developing cognitive decline and dementia.  Furthermore, one study in over 8000 people also indicated that the presence of obstructive sleep apnoea (OSA) in older adults was associated with an earlier age of cognitive decline, and that treatment of OSA may delay the onset of cognitive impairment. This study reveals important insights into how sleep disorders such as OSA may impact the brain in older adults, as it is associated with widespread structural alterations in diverse brain regions. We found that reduced blood oxygen levels during sleep are related to reduced thickness of the brain's cortex in both the left and right temporal areas - regions that are important in memory and are early sites of injury in Alzheimer's disease. Indeed, reduced thickness in these regions was associated with poorer ability to learn new information, thereby being the first to link this structural change to memory decline. (more…)
Author Interviews, BMJ, Clots - Coagulation, Heart Disease / 07.07.2018

MedicalResearch.com Interview with: Yana Vinogradova, PhD, Research Fellow Division of Primary Care, School of Medicine University of Nottingham Nottingham MedicalResearch.com: What is the background for this study? What are the main findings? Response: Anticoagulants are prescribed for treatment and prevention of thrombosis and stroke but may lead to major bleeding.  Unlike the older drug warfarin, newer direct oral anticoagulants do not require regular blood tests but their safety was shown only in selected patients and in trial conditions. The study found that Direct Oral AntiCoagulants (DOACs) are safer than warfarin in terms of bleeding risks with apixaban being the safest.  (more…)
Author Interviews, Brigham & Women's - Harvard, Electronic Records, JAMA / 07.07.2018

MedicalResearch.com Interview with: Li Zhou, MD, PhD, FACMI Associate Professor of Medicine Division of General Internal Medicine and Primary Care Brigham and Women’s Hospital, Harvard Medical School Somerville, MA 02145 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Documentation is one of the most time-consuming and costly aspects of electronic health record (EHR) use. Speech recognition (SR) technology, the automatic translation of voice to text, has been increasingly adopted to help clinicians complete their documentation in an efficient and cost-effective manner. One way in which SR can assist this process is commonly known as “back-end” SR, in which the clinician dictates into the telephone, the recorded audio is automatically transcribed to text by an speech recognition engine, and the text is edited by a professional medical transcriptionist and sent back to the EHR for the clinician to review and sign. In this study, we analyzed errors at different processing stages of clinical documents collected from 2 health care institutions using the same back-end SR vendor. We defined a comprehensive schema to systematically classify and analyze these errors, focusing particularly on clinically significant errors (errors that could plausibly affect a patient’s future care). We found an average of 7 errors per 100 words in raw  speech recognition transcriptions, and about 6% of those errors were clinically significant. 96.3% of the raw speech recognition transcriptions evaluated contained at least one error, and 63.6% had at least one clinically significant error. However, the rate of errors fell significantly after review by a medical transcriptionist, and it fell further still after the clinician reviewed the edited transcript. (more…)
Accidents & Violence, Author Interviews, Sleep Disorders / 07.07.2018

MedicalResearch.com Interview with: “Driving...” by Stig Nygaard is licensed under CC BY 2.0Prof. Stephen R Robinson PhD Discipline Leader, Psychology School of Health and Biomedical Sciences RMIT University Australia MedicalResearch.com: What is the background for this study? Response: Around the world, driver drowsiness and fatigue are estimated to contribute to 250,000 deaths on the road per year. Current research in this area has focused on detecting when drivers become drowsy, by examining their eye movements or steering patterns, and then alerting the driver with a warning tone or vibration of the steering wheel. Rather than this reactive approach, we are interested in helping to prevent drivers from becoming drowsy in the first place. (more…)
Author Interviews, Prostate, Prostate Cancer, Urology / 06.07.2018

MedicalResearch.com Interview with: Prof. Hashim Ahmed Professor and Chair of Urology Imperial College London MedicalResearch.com: What is the background for this study? What are the main findings? Response: Men with localised clinically significant prostate cancer currently undergo radical (whole gland) surgery or radiotherapy. These treatments are effective but can cause urine leakage in 5-30% and erectile dysfunction in 30-60%. Radiotherapy can cause rectal problems in 5%. So, although there is benefit in treating the cancer in these men, the side effects significantly affect the quality of life.  (more…)
Author Interviews, Breast Cancer, Cancer Research, Cost of Health Care, JAMA / 06.07.2018

MedicalResearch.com Interview with:

Dr Nora Pashayan PhD

Clinical Reader in Applied Health Research

University College London

Dept of Applied Health Research

London 

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

Response: Not all women have the same risk of developing breast cancer and not all women have the same potential to benefit from screening.

 

If the screening programme takes into account the individual variation in risk, then evidence from different studies indicate that this could improve the efficiency of the screening programme. However, questions remain on what is the best risk-stratified screening strategy, does risk-stratified screening add value for money, and what are benefit and harm trade-offs.

(more…)
Author Interviews, Cost of Health Care, End of Life Care, Medicare, Science / 06.07.2018

MedicalResearch.com Interview with: Amy Finkelstein PhD John & Jennie S. MacDonald Professor of Economics MIT Department of Economics National Bureau of Economic Research Cambridge MA 02139  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Although only 5% of Medicare beneficiaries die in a given year, they account for almost 25% of Medciare spending. This fact about high end of life spending has been constantly used to refer to inefficiency of the US healthcare system. A natural observation is that the fact is retrospective, and it motivated us to explore a prospective analog, which would take as an input the probability of someone dying in a given year rather than her realized outcome. We therefore used machine learning techniques to predict death, and somewhat to our surprise we found that at least using standardized and detailed claims-level data, predicting death is difficult, and there are only a tiny fraction of Medicare beneficiaries for whom we can predict death (within a year) with near certainty. Those who end up dying are obviously sicker, and our study finds that up to half of the higher spending on those who die could be attributed to the fact that those who die are sicker and sick individuals are associated with higher spending.   (more…)
Author Interviews, JAMA, Mental Health Research, Nutrition, OBGYNE / 05.07.2018

MedicalResearch.com Interview with: Joshua L. Roffman, MD Department of Psychiatry Mass General Hospital MedicalResearch.com: What is the background for this study? What are the main findings? Response: Autism, schizophrenia, and other serious mental illness affecting young people are chronic, debilitating, and incurable at present.  Recent public health studies have associated prenatal exposure to folic acid, a B-vitamin, with reduced subsequent risk of these illnesses.  However, until this point, biological evidence supporting a causal relationship between prenatal folic acid exposure and reduced psychiatric risk has remained elusive. We leveraged the rollout of government-mandated folic acid fortification of grain products in the U.S. from 1996-98 as a "natural experiment" to determine whether increased prenatal folic acid exposure influenced subsequent brain development.  This intervention, implemented to reduce risk of spina bifida and other disabling neural tube defects in infants, rapidly doubled blood folate levels among women of childbearing age in surveillance studies. Across two large, independent cohorts of youths age 8 to 18 who received MRI scans, we observed increased cortical thickness, and a delay in age-related cortical thinning, in brain regions associated with schizophrenia risk among individuals who were born during or after the fortification rollout, compared to those born just before it.  Further, delayed cortical thinning also predicted reduced risk of psychosis spectrum symptoms, a finding that suggests biological plausibility in light of previous work demonstrating early and accelerated cortical thinning among school-aged individuals with autism or psychosis. (more…)
Accidents & Violence, Author Interviews, JAMA, Pediatrics, University of Pittsburgh / 05.07.2018

MedicalResearch.com Interview with: Alison J. Culyba, MD, PhD, MPH Assistant Professor of Pediatrics UPMC Children’s Hospital of Pittsburgh University of Pittsburgh School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: Homicide is the third leading cause of death among adolescents, and disproportionately affects minority youth in under-resourced urban communities. Most research on youth violence focuses on risk factors, such as weapon carrying and substance abuse. We know much less about factors that protect youth from violence. Future orientation, defined as hopes and plans for the future, is linked to many important positive outcomes for youth, including doing well in school and avoiding illicit substances. However, there has been very little research to examine whether future orientation may also protect youth from violence. To study links between future orientation and violence perpetration, we surveyed over 850 male youth in lower resource neighborhoods in Pittsburgh as part of a community-based sexual violence prevention study. We found that youth with positive future orientation were significantly less likely to report threatening someone with a weapon or injuring someone with a weapon in the past nine months. (more…)
Author Interviews, Social Issues / 05.07.2018

MedicalResearch.com Interview with: Jessica Wood, MSc PhD Candidate, Applied Social Psychology Department of Psychology University of Guelph MedicalResearch.com: What is the background for this study? Response: We are at time in history where we expect more from our romantic partners than at any point in our recent past (e.g., love, emotional and financial support, sexual excitement/fulfillment, friendship etc.). This can place pressure on relationships and make it difficult for each person to have their needs fulfilled. Some choose to opt out of of relationships altogether to avoid disappointment, and some even purchase a real sex doll for fulfilment. Another option is consensually non-monogamous (CNM) relationships, where sexual and emotional needs are dispersed among multiple partners, potentially decreasing pressures placed on a primary relationship. However, CNM relationships are stigmatized and often viewed as less stable or satisfying. In our study, we assessed the legitimacy of this perception by comparing relational outcomes among CNM and monogamous individuals. We also examined whether the motives a person reports for engaging in sex was important to how fulfilled a person was in the relationship, and how this was linked to relational outcomes (such as relationship and sexual satisfaction). That is, having sex for more intrinsic/autonomous motives (e.g., pleasure, intimacy, valuing sex) has been associated with higher relationship quality. In contrast, having sex for more extrinsic reasons (e.g., feeling pressured, wanting to manage feelings of guilt or shame), has been linked to lower relational quality. (more…)
Author Interviews, Neurology / 05.07.2018

MedicalResearch.com Interview with: Prof. David Adams Head of the French National Reference Centre for Familial Amyloidotic Polyneuropathy (NNERF)/APHP/INSERM Paris FranceProf. David Adams Head of the French National Reference Centre for Familial Amyloidotic Polyneuropathy (NNERF)/APHP/INSERM Paris France MedicalResearch.com:  What is the background for this study? Response: Hereditary transthyretin amyloidosis is an autosomal dominant, multisystemic, progressive, life-threatening disease caused by mutations in the gene encoding transthyretin (TTR ). The liver is the primary source of circulating tetrameric TTR protein. In hereditary transthyretin amyloidosis, both mutant and wild-type transthyretin deposit as amyloid in peripheral nerves and the heart, kidney, resulting in polyneuropathy and cardiomyopathy. Neuropathic changes result in profound sensorimotor disturbances, with deterioration in activities of daily living and ambulation, hypotension, diarrhea, impotence, and bladder disturbances. Until now,  only few patients have access to anti-amyloid therapy : Liver Transplantation or TTR stabilizers which are able only to slow progression of the disease at very early stage of the disease. Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin and is specifically addressed to the liver by lipid nanoparticle (LNP) formulation. This study carried out a multicenter, international, randomized, double-blind, placebo-controlled, phase 3 trial of patisiran in patients with hereditary transthyretin amyloidosis with polyneuropathy. (more…)
Author Interviews, Infections, NEJM / 05.07.2018

MedicalResearch.com Interview with: Dr. Dennis E. Hruby PhD Chief Science Officer of SIGA Technologies Corvallis, OR MedicalResearch.com: What is the background for this study?  Response: Naturally occurring smallpox was declared eradicated in 1980 following coordinated decades-long global vaccination campaigns. However, there is a significant concern that smallpox, which is both highly contagious and highly lethal, could be used as a potential bioweapon. DNA synthesis technology and the possibility of unaccounted for smallpox stocks pose significant risks. While there are two publicly acknowledged stocks of smallpox virus held by the United States and Russia, some believe that additional stores of the virus could be in the hands of governments or organizations that might use them to cause harm. The DNA sequence of the smallpox genome is in the public domain and could potentially be synthesized in a laboratory from scratch or created by genetically modifying a similar virus. Currently, there are no therapies approved for the treatment of smallpox infection. A smallpox bioterror attack could be especially damaging because the majority of today’s population is not immune to the virus, as routine vaccination ended in the 1970s. It is estimated that without vaccination or treatment, each person infected with smallpox would infect 5 - 7 others. Rapid spread from person-to-person can occur through speaking, breathing or touching. Smallpox also can be transmitted by direct contact with infected fluids and contaminated objects. Furthermore, vaccination must occur within 3-5 days of exposure to smallpox, when patients are still asymptomatic, to be effective. These limitations underscore the need for an effective smallpox antiviral therapy, in addition to any available vaccine. (more…)
Author Interviews, Cancer Research, JAMA, OBGYNE / 04.07.2018

MedicalResearch.com Interview with: Dr. Gina Ogilvie | MD MSc FCFP DrPH Professor | Faculty of Medicine | University of British Columbia Canada Research Chair | Global control of HPV related disease and cancer Senior Public Health Scientist | BC Centre for Disease Control Senior Research Advisor | BC Women's Hospital and Health Centre BC Women's Hospital and Health Centre Vancouver, BC MedicalResearch.com: What is the background for this study? What are the main findings? Response: HPV is known to be the cause of 99% of cervcial cancers. In this study, we compared the routine screening test for cervical cancer, Pap test, to HPV testing. We found that by using HPV testing, women were significantly more likely to have cervical pre-cancers detected earlier. In addition, women with negative HPV tests were significantly less likely to have pre-cancers 48 months later. (more…)
Author Interviews, BMJ, Diabetes, Occupational Health / 03.07.2018

MedicalResearch.com Interview with: Mahée Gilbert-Ouimet, PhD Postdoctoral fellow/Chercheure postdoctorale Institute for Work & Health Hôpital du St-Sacrement,  Québec  MedicalResearch.com: What is the background for this study?  Response: Diabetes is one of the primary causes of death worldwide, in addition to being a major risk factor for several other chronic diseases including cardiovascular diseases. Considering the rapid and substantial increase of diabetes prevalence, identifying modifiable risk factors is of major importance. In this regard, long work hours have recently been linked with diabetes, but more high-quality prospective studies are needed. Our study evaluated the relationship between long work hours and the incidence of diabetes among 7065 workers over a 12-year period in Ontario, Canada. (more…)
Annals Internal Medicine, Author Interviews, Blood Pressure - Hypertension, Brigham & Women's - Harvard, Heart Disease, OBGYNE / 03.07.2018

MedicalResearch.com Interview with: Jennifer J. Stuart, ScD Postdoctoral Research Fellow in Reproductive & Cardiovascular Epidemiology Department of Epidemiology Harvard T.H. Chan School of Public Health Division of Women's Health Brigham and Women's Hospital and Harvard Medical School  MedicalResearch.com: What is the background for this study? Response: Preeclampsia and gestational hypertension are common pregnancy complications involving high blood pressure that develops for the first time during pregnancy and returns to normal after delivery. Approximately 10 to 15% of all women who have given birth have a history of either preeclampsia or gestational hypertension. Previous studies have shown that women with a history of high blood pressure in pregnancy are more likely to develop cardiovascular disease events like heart attack and stroke later in life when compared to women with normal blood pressure in pregnancy. However, what is less clear is to what extent these women are more likely to develop chronic hypertension, diabetes, and high cholesterol and when these risk factors begin to emerge after pregnancy. We examined this question in a cohort of nearly 60,000 American women who we were able to follow for up to 50 years after their first pregnancy. Previous studies have been limited by small numbers, short follow-up, or a lack of information on shared risk factors, such as pre-pregnancy body mass index, smoking, and family history. This research was conducted within the Nurses’ Health Study II, which collected data on these pre-pregnancy factors in tens of thousands of women over several decades. (more…)
AACR, Author Interviews, Cancer Research, Pancreatic / 02.07.2018

MedicalResearch.com Interview with: Michael J. Pishvaian, MD, PhD Phase I Program Director Assistant Professor Lombardi Comprehensive Cancer Center Washington, DC 20007 MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Pancreatic cancer is a deadly disease and will soon be the second leading cause of cancer-related death. We have made some progress in the last few years....but despite this, patients with advanced, inoperable pancreatic cancer (which represents about 80% of pancreatic cancer patients) still only live 12 months, on average. We desperately need new therapies, AND to think "outside the box" for the treatment of pancreatic cancer. In that context, we have been learning that there are subgroups of patients with cancer whose tumors are particularly susceptible to certain therapies - either new therapies, or in some cases, approved therapies that would have not normally been used for that disease.  These specific patient subgroups with "actionable" findings have been identified through extensive genetic and molecular characterization of a patient's tumor. In the past there was a cynical perspective that pancreatic cancer did not harbor any "actionable" molecular abnormalities. We have now demonstrated that: 1) There are clearly and undeniably patients with pancreatic cancer whose tumors do indeed harbor "actionable" findings.  This represents at least 27% of pancreatic cancer patients, but may represent up to 50% as new therapies evolve.  These percentages are also highly consistent with similar publications in the pancreatic cancer field over the last few years; and 2) Importantly, we have been following our patients longitudinally for outcomes, and while it is still early, there is a statistically significant improvement in progression-free survival when a patient with a specific actionable molecular abnormality is treated with the appropriately "targeted" therapy.  This finding is also consistent with findings that have been observed in other cancer types.   (more…)
Author Interviews, Cost of Health Care, Hospital Acquired, Medicare / 02.07.2018

MedicalResearch.com Interview with: Michael S. Calderwood, MD, MPH, FIDSA Regional Hospital Epidemiologist Assistant Professor of Medicine Infectious Disease & International Health MedicalResearch.com: What is the background for this study?   Response: Prior work by Lee et al. (N Engl J Med 2012;367:1428–1437) found that the 2008 CMS Hospital-Acquired Conditions (HAC) policy did not impact already declining national rates of central line-associated bloodstream infections (CLABSIs) or catheter-associated urinary tract infections (CAUTIs). We studied why this policy did not have its intended impact by looking at coding practices and the impact of the policy on the diagnosis-related group (DRG) assignment for Medicare hospitalizations. The DRG assignment determines reimbursement for inpatient hospitalizations. (more…)
Author Interviews, Cancer Research, Endocrinology, JAMA, Pediatrics / 02.07.2018

MedicalResearch.com Interview with: “Cancer awareness” by Susan Roberts is licensed under CC BY 2.0Mette Vestergaard Jensen, MD Danish Cancer Society Research Center MedicalResearch.com: What is the background for this study? Response: Cancer survival rates have improved and it is necessary to explore the long-term consequences of cancer treatment. Adolescents and young adults with cancer are at risk for several therapy-related late effects; however, these have not been studied extensively. We investigatet the lifetime risks of endocrine late effects of cancer and cancer treatment in adolescent and young adult cancer s (more…)
Annals Internal Medicine, Author Interviews, Cancer Research, Kidney Disease, Radiology, Surgical Research / 30.06.2018

MedicalResearch.com Interview with: Adam Talenfeld, M.D Assistant Professor of Radiology Weill Cornell Medical College Assistant Attending Radiologist New York-Presbyterian Hospital. MedicalResearch.com: What is the background for this study? What are the main findings? Response: We know that renal function decreases as we age, and we know that decreased renal function is independently associated with increased mortality. This is why medical society guidelines recommend partial nephrectomy, which preserves kidney tissue and function, over radical nephrectomy for the treatment of the smallest kidney cancers, stage T1a tumors, which are under 4 cm diameter. Paradoxically, though, we know older patients are more likely than younger patients to receive radical nephrectomy for these smallest tumors, probably because it’s a simpler surgery than partial nephrectomy. Percutaneous ablation, focal tissue destruction using heat or cold emanating from the tip of a needle, is a newer, image-guided, minimally-invasive, tissue-sparing treatment for solid organ tumors. We wanted to test how well percutaneous ablation would compare to partial nephrectomy and radical nephrectomy for these smallest kidney cancers. We found that percutaneous ablation was associated with similar 5-year overall and cancer-specific survival compared to radical nephrectomy. At the same time, ablation was associated with significantly lower rates of new-onset chronic renal insufficiency and one-fifth as many serious non-urological complications than radical nephrectomy within 30 days of treatment. These were complications, such as deep venous thrombosis or pneumonia, that resulted in emergency department visits or new hospital admissions. The outcomes of percutaneous ablation compared with partial nephrectomy were somewhat less clear, though ablation was again associated with fewer perioperative complications. (more…)