Dr. Sagar[/caption]
Manish Sagar, MD
Infectious Disease Physician at Boston Medical Center
Boston MA
MedicalResearch.com: What is the background for this study?
Response: Women compromise the majority of new infections in the world and most of them acquire the virus after sexual exposure. The goal of the study was to understand how HIV establishes initial infection in the female genital tract. We obtained discarded vaginal tissue and isolated cells present in the outermost layer that contact the virus during exposure.
Dr. Davidge[/caption]
Sandra T. Davidge, PhD, FCAHS
Executive Director, Women and Children's Health Research Institute
Canada Research Chair in Maternal and Perinatal Cardiovascular Health
Professor, Depts. of Ob/Gyn and Physiology
University of Alberta
Edmonton, Alberta
Canada
MedicalResearch.com: What is the background for this study?
Response: This research contributes to the growing body of literature that developmental programming of adult onset cardiovascular disease originates in the womb.
Our study is among the first to discover that maternal age may be considered a ‘prenatal stress’ in certain circumstances.
Dr. Boessen[/caption]
Dr. Lars Boesen MD PhD
Department of Urology
Herlev Hospital
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: The current standard of care in prostate cancer diagnosis includes untargeted transrectal ultrasound-guided biopsies for all biopsy-naïve men with clinically suspicion of prostate cancer. However, this strategy that practically has remained unchanged for decades has limited diagnostic accuracy as significant cancers are missed or under-graded and insignificant cancers are unintendedly detected by the random sampling leading to possible overtreatment.
Multiparametric MRI in the diagnosis of prostate cancer has been studied extensively in recent years and has improved detection, localization, staging and risk stratification. It has been suggested that if multiparametric MRIs were used as a triage test prior to biopsies, a significant proportion of men might safely avoid prostate biopsies and the diagnostic ratio of significant vs. insignificant cancer could be improved compared to performing standard biopsies in all men. However, multiparametric MRIs are generally time-consuming (~40 min scan time), expensive and include intravenous contrast media. This reduces its feasibility for widespread clinical implementation in larger patient populations in the western community with its high PCa prevalence.
The development of a simpler and faster (~15 min) biparametric MRI protocol using less scan sequences and circumvents intravenous contrast-media seems to preserve adequate diagnostic accuracy in a detection setting and could facilitate dissemination of prostate MRI as a triage test before any biopsy.
Here we present a large prospective study that assesses the diagnostic accuracy of a novel biparametric MRI to rule out significant prostate cancer in N=1020 biopsy-naive men with clinically suspicion of prostate cancer.
We found that a low suspicion biparametric MRI had a very high negative predictive value (97%) for ruling out significant cancer on confirmatory biopsies. Furthermore, bpMRI suspicion scores were strongly associated with prostate cancer detection rates and restricting biopsies (targeted plus standard) to men with suspicious biparametric MRIs meant 30% could avoid prostate biopsies, improved significant prostate cancer diagnosis by 11%, and reduced insignificant prostate cancer diagnosis by 40% compared to our current diagnostic approach – standard biopsies for all men with clinically suspicion of prostate cancer.
Dr. Lekoubou[/caption]
Dr. Alain Lekoubou Looti, MD Msc
Clinical Neurophysiology
Department of Neurosciences
College of Medicine
Medical University of South Carolina
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Hypertension is strongly associated with stroke. Individuals who suffer a stroke are more likely to have another stroke. They also die at a rate twice as high as those who experience a first event. We have evidence that treating hypertension reduces the risk of recurrence stroke among stroke survivors.
Prior hypertension guidelines defined hypertension for a systolic blood pressure (top number) equal or greater than 140 and a diastolic blood pressure (lower number) equal or greater than 90. The American college of cardiology/American heart association have published a new guideline to help healthcare providers identify and treat blood pressure including among stroke survivors. The threshold to define blood pressure has been lowered to 130 for the top number and 80 for the lower number for everyone. Unlike the general population, pharmacological treatment for stroke survivors is now recommended for systolic blood pressure greater than or equal than 130 and diastolic blood pressure greater than or equal to 80. In the same line achieving a blood pressure of less than 130/80 mmHg in stroke survivors is now recommended. In the stroke community, there has been mounting evidence to suggest that achieving lower blood pressure goal was desirable. When the new guidelines were published, we could not wait any longer to see the impact of the new guidelines on the proportion of stroke survivors with hypertension, recommended pharmacological treatment, and above blood pressure target. We were also curious to see how the new guidelines would potentially affect mortality among stroke survivors.
We have found that the new guidelines would result in a nearly 67% (from 29.9% to 49.8%) to relative increase in the proportion of U.S. stroke survivors diagnosed with hypertension and 54% (from 36.3% to 56%) relative increase in those not within the recommended BP target. We have also found that if the new guidelines were applied, this would result in a 33% relative drop in mortality.
Dr. Wei Bao[/caption]
Wei Bao, MD, PhD
Assistant Professor, Epidemiology
College of Public Health
University of Iowa
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Previous studies indicated a possible link between immunologic dysfunction and autism. The current study, based on nationally representative large-scale surveys, showed that food allergy, respiratory allergy, and skin allergy, all relevant to immunological dysfunction, were associated with autism spectrum disorder among US children.
Dr. Avidor-Reiss[/caption]
Tomer Avidor-Reiss, Ph.D.
Professor, College of Natural Sciences and Mathematics
Department of Biological Sciences
University of Toledo
Toledo, OH 43606
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Most dividing cells in the body need exactly two centrioles for normal cell division and development. Abnormalities in centriole number can cause cancer and devastating developmental defects. Because of this importance of centriole number and because all cells originate from the zygote – the product of the sperm and egg – it makes sense that the zygote should possess two centrioles, as well.
However, the egg does not contain any centrioles, so the sperm is the sole contributor of the centrioles; and yet, it is currently thought that the sperm contains only one centriole. This is problematic because supposedly that leaves the zygote with only one centriole, even though it must propagate cells with two centrioles. In the past, we found that insect sperm have an atypical centriole that escaped discovery because it is so different. We therefore hypothesized that humans may also have an atypical sperm centriole.
Our new paper shows that in human sperm there exists, in addition to the known centriole, a second centriole that deviates from the typical structural and composition that is expected from a centriole. Although it looks very different from any centriole ever described, we found that it functions in an in vitro functional assay. Furthermore, during fertilization, it performs the functions traditionally associated with centrioles. Together, this resolves a 50-year-long debate regarding the centrioles of human sperm; the sperm contains two functional centrioles, despite that one is atypical.
David Haussler PhD
Investigator, Howard Hughes Medical Institute
Distinguished Professor, Biomolecular Engineering
Scientific Director, UC Santa Cruz Genomics Institute
Scientific Co-Director, California Institute for Quantitative Biosciences and
Sofie Salama, PhD
Research Scientist in BIomolecular Engineering
Howard Hughes Medical Institute Senior Scientist
MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. Haussler: Researchers specializing in this area are interested understanding which evolutionary changes in our genome underlie human-specific brain features including our large (3X greater than chimpanzee) brain.
It has been my personal dream to peer into human evolution at the level of individual genes and gene functions.
Michelle Nguyen
Senior Director, Consumer Marketing Lead Shire
Ophthalmics
MedicalResearch.com: What is the background for this initiative? How does screen time affect eye health?
Response: Screen responsibly is an educational initiative about the intersection between our screen use and eye health. This initiative is underscored by new national survey data and aimed at increasing our dialogue about how to love both our screens and our eyes. Screen responsibly is an extension of the eyelove campaign, which Shire launched in 2016 to elevate the importance of eye health and raise awareness of the signs and symptoms of Chronic Dry Eye, commonly referred to as Dry Eye. Since being glued to your screen can trigger Dry Eye symptoms, it is important that people are aware of ways to screen responsibly.
[caption id="attachment_42278" align="alignleft" width="125"]
Michelle Nguyen[/caption]
MedicalResearch.com: How common is the problem of Dry Eye? How is it related to excessive screen time?
Response: In the United States, nearly 30 million adults report symptoms consistent with Dry Eye. Dry Eye symptoms include a burning, itchy, stinging or gritty feeling in the eyes, episodes of blurred vision, eye redness and watery eyes. Shire is committed to addressing unmet needs within the Dry Eye patient community, and to do so, we sought to better understand their current behaviors, perceptions and knowledge around a common trigger of Dry Eye symptoms – intense screen usage.
Naoto Tada Ueno, M.D., Ph.D., F.A.C.P.
Executive Director, Morgan Welch Inflammatory Breast Cancer Research Program and Clinic
Section Chief, Section of Translational Breast Cancer Research, Department of Breast Medical Oncology
Division of Cancer Medicine
The University of Texas MD Anderson Cancer Center
Houston, TX
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: The best outcome of inflammatory breast cancer (IBC) is dependent on achieving a pathological completed response after neoadjuvant chemotherapy for primary inflammatory breast cancer, which is the most aggressive type of breast cancer.
We have conducted extensive preclinical work, which showed that EGFR is a potential therapeutic targets of IBC.
Based on this preclinical data, we have conducted a phase II study to determine the pathological complete response rate of panitumumab plus neoadjuvant chemotherapy for HER2 negative primary inflammatory breast cancer.
Atrial Fibrillation-
Ali Khashan, PhD
Senior Lecturer in Epidemiology
School of Public Health & INFANT Centre
University College Cork
Cork, Ireland
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: There is some evidence to suggest an increased likelihood of neurodevelopmental disorders in relation to hypertensive disorders in pregnancy, however consensus is lacking. Considering hypertensive disorders in pregnancy are among the most common prenatal complication, we decided to synthesise the published literature on this topic by conducting a comprehensive systematic review and meta-analysis.
Our main findings suggest that hypertensive disorders in pregnancy are associated with about 30% increase in the likelihood of autism spectrum disorders (ASD) and ADHD in the offspring, compared to offspring not exposed to hypertensive disorders in pregnancy.
Rebecca Ivy Hartman, M.D
Instructor in Dermatology
Brigham and Women's Hospital
Boston MA 02115
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Organ transplant recipients (OTR) are at 100-fold higher risk to develop certain skin cancers compared to the general population due to immunosuppression, and thus preventing skin cancer in this population is critical.
Our study found that in a high-risk Australian OTR population, only half of patients practiced multiple measures of sun protection regularly.
However, after participating in a research study that required dermatology visits, patients were over 4-times more likely to report using multiple measures of sun protection regularly. Patients were more likely to have a positive behavioral change if they did not already undergo annual skin cancer screening prior to study participation.
Dr. Witkiewitz[/caption]
Dr. Katie Witkiewitz PhD
Professor, Department of Psychology
University of New Mexico
MedicalResearch.com: What are the main findings?
Response: The main findings from our study indicate that individuals with alcohol dependence who misused opioids (e.g., used without a prescription or not as prescribed) had a significantly higher likelihood of relapse to heavy drinking during alcohol treatment and were drinking more alcohol during and following alcohol treatment.
Prof. Mäki[/caption]
Markku Mäki, MD, PhD
Professor (emeritus) at the University of Tampere and
Presently research director at the
Tampere University Hospital
Tampere, Finland
MedicalResearch.com: What is the background for this study?
Response: The only treatment for this life-long gluten-induced autoimmune systemic disease is a strict avoidance of wheat, rye and barley, the food cereals which contain gluten, the environmental trigger and driving force in celiac disease. Gluten causes intestinal
inflammation, usually with (but sometimes without) gastrointestinal or
nutritional symptoms or signs, and with frequent extra-intestinal
diseases. However, it is impossible for celiac disease patients to
avoid gluten entirely and indefinitely and a third of patients report
symptoms on a strict gluten-free diet. Gut mucosal healing is not
optimal in half of the patients, and inflammation and injury is
detected for years after starting the diet, presumably due to
contamination with gluten in the diet. This is why patients are
requesting, and academia and industry are looking for novel adjunct
therapies for celiac disease. Initially, these therapies are tested to
prevent the consequences of hidden gluten; the ultimate goal being
that also celiacs could one day eat safely wheat, barley and rye
products. Some 20 novel experimental therapies are at present actively
being investigated (modifying wheat or different drugs, devices and
vaccines/immunotherapy).
The present study investigated whether blocking interleukin 15, an
important mediator of celiac disease, reduces or prevents
gluten-driven ill health, both the inflammation and injury at the
small intestinal mucosal level and gluten-induced symptoms. The
experimental drug used was Amgen’s AMG 714, a human monoclonal
antibody, used at a low and high dose, in the presence or absence of a
high-dose gluten challenge.
Dr. Corpechot[/caption]
Dr Christophe Corpechot
Centre de Référence Maladie Rares: Maladies Inflammatoires des Voies Biliaires et Hépatites Auto-immunes (MIVB-H)
Filière Maladies Rares: Maladies Rares du Foie de l’Adulte et de l’Enfant
Hôpital Saint-Antoine (APHP) et Sorbonne Universités
Paris
MedicalResearch.com: What is the background for this study?
Response: Primary biliary cholangitis (PBC, previously known as "primary biliary cirrhosis") is a rare, chronic, slowly progressive liver disease of unknown cause, mainly affecting women of middle age. It is characterized by serum marks of autoimmunity (specific auto-antibodies), chronic inflammation and destruction of small intra-hepatic bile ducts, and consequent bile secretion impairment (chronic cholestasis) leading to the progressive development of cirrhosis and liver failure. Ursodeoxycholic acid (UDCA) is the only first-line approved treatment for PBC. It improves the biochemical measures of cholestasis and prolongs survival without liver transplantation. However, 30% to 40% of UDCA-treated patients continue to have clinically significant abnormalities of their biochemical liver tests and those patients remain at high risk of developing end-stage liver disease complications.
Recently (2016), obeticholic acid (OCA) in association with UDCA has been conditionally approved in patients with an inadequate response to UDCA. This approval (FDA, EMA) was based one the results of a 1-year randomized, double-blind, placebo-controlled trial of OCA in patients with an incomplete response or intolerance to UDCA (POISE trial). In this trial, OCA was shown to improve the biochemical features of cholestasis (alkaline phosphatase (ALP) level < 1.67 times the upper limit of the normal range and a reduction of at least 15% from baseline) but was associated with a significant increase of pruritus, a characteristic, potentially debilitating symptom of PBC. BEZURSO is the first ever placebo-controlled phase 3 trial of a fibrate (a class of drugs known to be agonists of the peroxisome proliferator-activated receptors alpha) in PBC. In this 2-year randomized double-blind trial, 100 patients with an incomplete response to UDCA were assigned to bezafibrate 400 mg/day (n=50) or placebo (n=50), all in association with continued UDCA therapy.
Dr. Le Cesne[/caption]
Axel Le Cesne, MD
Institute de Cancerologie Gustave-Roussy
Villejuif, France
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: With the exception of a study in translocation-related sarcomas (TRS) (Kawai, TLO 2015), trabectedin was never compared to best supportive care (BSC) in a randomized study in patients with all STS histotypes. This trial required by French Health Authorities in 2014.
The tumor control rate after 6 cycles of trabectedin is similar (30%) to previous study in French referral centers (T-DIS trial, Le Cesne, Lancet Oncol 2015) evaluating trabectedin in all subtypes of STS. As already reported, trabectedin was well tolerated with no cumulative toxicities
This study met its first endpoint as a preplanned PFS analysis showed a significant improvement in median PFS with trabectedin over BSC in patients with pretreated ASTS including multiple histologies (HR: 0.39). A major impact of trabectedin was observed in the L-STS cohort (liposarcomas and leiomyosarcomas) with a median PFS of 1.4 months in the BSC arm and 5.13 m (HR: 0.29, p<0.0001) in the trabectedin arm. respectively). The benefit observed with trabectedin in the L-STS cohort of patients is similar to those observed in the US trial in the same population (4.2 vs 1.5m for DTIC) (Demetri, JCO 2016) and in the Japanese trial mentioned above (5.8 vs 0.9m for BSC) (Kawai, TLO 2015)
After the crossing over allowed by the protocol (trabectedin for patients progressing in the BSC arm), safety and efficacy profiles of trabectedin remains similar. We did not observe a difference in terms of OS between the two arms, probably due to the cross-over planned by the protocol.
Dr. Sumner[/caption]
Rick Sumner, PhD, FAAA
The Mary Lou Bell McGrew Presidential Professor for Medical Research
Chair, Department of Cell & Molecular Medicine (formerly, Anatomy and Cell Biology)
Rush University Medical Center
Chicago, IL 60612
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: The main cause of failure for total hip replacements is implant loosening which is often a consequence of particle-induced peri-implant osteolysis. Unfortunately, this condition is usually not diagnosed until it has progressed to the point of needing a revision surgery.
We discovered two biomarkers that may be useful for identifying at risk patients much earlier than is currently possible.
Dr. Bardia[/caption]
Dr. Aditya Bardia MD, MPH
Assistant Professor, Medicine
Harvard Medical School
Attending Physician, Medical Oncology
Massachusetts General Hospital
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Hormone receptor-positive (HR+)/ and human epidermal growth factor receptor 2-negative (HER2-) breast cancer is the most common sub-type of breast cancer. While metastatic HR+/HER2- breast cancer is initially treated with endocrine therapy-based combinations, including CDK 4/6 inhibitors, patients eventually have disease progression, but the response rate to standard chemotherapy is low (~10-15 percent, post-taxane setting). In particular, patients with visceral disease have a poor prognosis.
In this trial, we evaluated the efficacy of sacituzumab govitecan in patients with metastatic HR+/HER2- breast cancer, who had measurable disease and had received prior therapies for metastatic breast cancer. We observed an overall response rate of 31 percent in a heavily pre-treated population (prior number of therapies for metastatic breast cancer = 5; number of patients with prior CDK 4/6 inhibitor use = 69 percent). The responses were durable (median duration of response = 7.4 months). Neutropenia was the main adverse event noted (grade 3 neutropenia = 42 percent), and two patients (3.7 percent) discontinued the clinical trial due to adverse events. The response rate in patients with visceral metastaseswas 27 percent.
Dr. Raed[/caption]
Anas Raed, MD
Section of General Internal Medicine
Augusta University
MedicalResearch.com: What is the background for this study?
Response: Colorectal cancer (CRC) incidence and mortality rates have been decreasing in the US since mid 1980s, however, recent evidence shows that incidence and mortality rates of CRC in patients younger than 50 years have been increasing significantly.
In spite of the increasing trend of colorectal cancer, routine screening of this population has not been addressed due to lack of evidence and cost-effectiveness. Administering screening colonoscopy for all individuals younger than 50 years might not be feasible and, therefore routine screening colonoscopy for specific age groups might reduce the disparity of the incidence in this disease.
Dr. Ishida[/caption]
Dr. Julie H. Ishida MD
Department of Medicine, Division of Nephrology
University of California, San Francisco and
San Francisco Veterans Affairs Medical Center
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Gabapentin and pregabalin are used for the management of symptoms such as neuropathic pain, itching, and restless leg syndrome in patients receiving hemodialysis. However, hemodialysis patients may be particularly vulnerable to adverse events related to these agents, which are cleared by the kidney, but there is limited data evaluating their risk in this population.
Gabapentin and pregabalin use were associated with risk for altered mental status, fall, and fracture, and in some cases, even at doses that would be considered safe for use in this population.
Dr. Maggiolini[/caption]
Alfio Maggiolini, MD
Minotauro, Milan
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Antisocial behaviour is common during adolescence and it incurs significant costs both for society and for the young people themselves. Persistent antisocial behaviour places a heavy burden on the community, the justice system and the public health system. Responses to juvenile crime have always seen a tension between a focus on the understanding and the rehabilitation of the youth and the need to enforce discipline and public safety through punishment and threat.
The treatment of young offenders was traditionally deemed particularly difficult, and often ineffective. In recent years, therapeutic nihilism has given way to cautious optimism. While punitive-based approaches, at all levels, are hardly ever effective in the long term, the most popular and effective programs tend to focus on behaviour control. Common core elements of such programs include positive reinforcement, problem solving skills training and role playing, as behavioral problems are often assumed somewhat inherently wrong, or a “lack of something”, the programs aims at improving or changing.
The study presents a developmental approach that understands behavioral problems as the result of intentions, values and goals that need to be taken in full consideration and that are usually legitimate, even though carried out in ways that prove dysfunctional for both the young person and society. In other words, we consider antisocial behaviors as maladaptive responses to legitimate developmental tasks, a deviant way of meeting positive goals and taking control of one’s life. In the program we describe, a developmental understanding is combined with a psychoanalytically informed perspective on treatment and delivered in multi-modal terms. It has been carried out in Italy for the past 20 years, with positive outcomes, both in private practice and within the juvenile justice services.
Mícheál de Barra, PhD
Lecturer in Psychology
Brunel University London
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Disgust has been called the "intuitive microbiologist" - it tracks the sources of infection in our environment. But so far, there has been little attempt to link the sources of disgust to the sources of infectious disease in a comprehensive way. So we developed a method for developing stimuli based on a random sample illness.
We basically asked ourselves what the kinds of cues that might be associated with that kind of disease risk and asked people to rate disgust responses. The main motive for this was to contribute to a debate in the literature about if there are "kinds of disgust" and if so, how many. I results were a little ambiguous there I'm afraid.
Dr. Del Priore[/caption]
Dr. Giuseppe Del Priore, MD, MPH
Chief Medical Officer of Tyme Inc.
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Metastatic breast cancer, sometimes also called “stage IV” or “advanced breast cancer,” is the most extensive stage of breast cancer. It is an invasive cancer that has spread to other parts of the body, most often bones, lungs, liver, and brain. The current standard of care for metastatic breast cancer is systemic drug therapies, such as hormone therapy, chemotherapy, targeted drugs or a combination of these. Because they reach every cell in the body, they have side effects that can worsen the patient’s quality of life. Existing treatments cannot cure metastatic breast cancer and are palliative in intent. This presents a great unmet need and challenge in treating patients with metastatic breast cancer.
SM-88 is a novel relatively non-toxic combination therapy that harnesses cancer’s unique cell metabolism and oxidative stress to selectively drive cancer cell death. Earlier studies with SM-88 therapy demonstrated its potential efficacy in breast and other metastatic cancers. In this current report, we assessed the efficacy of SM-88 in patients with metastatic breast cancer from the first in human “Phase 1” and compassionate use programs from 2012 to 2017. Data demonstrated the potential efficacy of SM-88 in metastatic breast cancer with favorable safety and quality of life profiles. In addition, there were no indications of cross-resistance based on hormone profile, previous treatments or metastatic site. This is an extremely important finding since most cancer deaths are due to resistance to subsequent therapies. As predicted by the SM-88 mechanism of action, we could not detect this problem with SM-88 use.
This image depicts the gross appearance of a cutaneous pigmented lesion, which had been diagnosed as superficial spreading malignant melanoma (SSMM). Note the roughened edges of this mole, and its heterogeneous, mottled, multicolored appearance, which are all characteristics that should evoke suspicions about its classification.
Dr. 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.
MedicalResearch.com Interview with: Hilary Tindle, MD, MPH Associate Professor of Medicine and theWilliam Anderson Spickard, Jr., MD Chair in Medicine Founding Director of ViTAL, the Vanderbilt Center for Tobacco, Addiction and Lifestyle Division of Internal Medicine & Public Health and Vanderbilt Ingram Cancer Center (VICC) MedicalResearch.com: What is the background for this study? Response: Lung cancer is the most common...
Dr. Ryerson[/caption]
Christopher J. Ryerson, M.D.
Assistant Professor
Centre for Heart Lung Innovation
University of British Columbia
Vancouver, Canada
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: A new Idiopathic pulmonary fibrosis (IPF) mortality analysis presented at the American Thoracic Society’s 2018 annual conference suggests that treatment with nintedanib may be associated with reduced risk of death in patients with the rare lung disease idiopathic pulmonary fibrosis (IPF).
Pooled data from the two Phase II INPULSIS trials and the Phase II TOMORROW study compared the number of deaths observed versus the number predicted based on GAP stage over one year. GAP stage is used to predict IPF prognosis and is based on gender, age and lung function (as measured by forced vital capacity [FVC] decline predicted and DLco % predicted). Higher stages of GAP are associated with an increased risk of death.
Across the population in the analysis (n=1,228), there were fewer deaths observed in each treatment group than predicted based on GAP stage at baseline (nintedanib: 42 vs. 89.9; placebo: 41 vs. 64.2). In the treated group, the number of observed deaths was 46.7% of the number predicted based on GAP stage, while in the placebo group the number of observed deaths was 63.9% of the number predicted. Based on these observations, the analysis suggests that nintedanib may be associated with a 26.8% relative reduction in the risk of death compared with placebo over one year.