Author Interviews, Connective Tissue Disease, Pulmonary Disease / 29.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41845" align="alignleft" width="125"]Pierre Laurin, CEO Prometic Pierre Laurin[/caption] Pierre Laurin, CEO Prometic Life Sciences MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by idiopathic pulmonary fibrosis? Response: Idiopathic pulmonary fibrosis (IPF) is a chronic, devastating, and ultimately fatal disease characterized by a progressive decline in lung function. It is a specific type of interstitial lung disease in which the small air sacs of the lung, the “alveoli,” gradually become replaced by fibrotic (scar) tissue and is the cause of worsening dyspnea (shortness of breath). The 5-year mortality rate for patients with IPF is estimated to range from 50% to 70%. Small molecule candidate PBI-4050’s anti-fibrotic activity has been observed in various fibrosis models in different organs: lung, kidney, heart, liver, and pancreas. PBI-4050 has been shown to improve forced vital capacity (FVC) in an open-label Phase 2 study in IPF. The main objective of this exploratory study was to determine whether treatment with PBI-4050 alters the level of key biomarkers in patients with IPF. Subjects with a confirmed diagnosis of IPF received daily oral doses of 800 mg PBI-4050 with or without nintedanib or pirfenidone for 12 weeks. The biomarkers chosen for measurement can be divided into two main groups: cytokines and matrix metalloproteinases associated with fibrosis and inflammation.
Author Interviews, Connective Tissue Disease, Genetic Research, Rheumatology / 20.07.2015

MedicalResearch.com Interview with: Dr. Changfu Kuo MD PhD Division of Rheumatology, Orthopaedics, and Dermatology School of Medicine, University of Nottingham, Nottingham, England Division of Rheumatology, Allergy, and Immunology Chang Gung Memorial Hospital, Taoyuan, Taiwan Medical Research: What is the background for this study? What are the main findings? Dr. Kuo: Systemic lupus erythematosus (SLE) is a prototype of autoimmune disease with features like autoantibody production and multiple target organ damage. SLE can affect any part of the body and the course of the disease is highly diverse and unpredictable. SLE can occur at any age and affect both females and males with a sex ratio of 9 to 1. Familial predisposition has been recognised as a risk factor previously and heritability of SLE has been estimated to be 66%. However, previous reports are often based on less robust sampling strategies and case ascertainment which generally depend on hospital records, self-reported diagnosis and disease registries, therefore limiting generalisability. The previous estimates of heritability are overestimated, due to a lack of consideration of shared environmental contribution. This study utilised a unique health insurance database that provides information on the whole population of Taiwan and permits determination of spouse and first-degree relatives. Over 23 million people were included in this study. Furthermore, through inclusion of SLE status of the spouse in our analyses the study is also able to examine how much of familial clustering results from genetic versus shared environmental factors. Overall the familial relative risk is 16.92. The genetic contribution to SLE susceptibility is estimated to be 44%. In addition to SLE, other autoimmune diseases are also more prevalent in individuals with a family history of SLE.
AHA Journals, Author Interviews, Connective Tissue Disease, Heart Disease, NYU / 08.07.2015

Mohamed Boutjdir, PhD, FAHADirector of the Cardiovascular Research Program VA New York Harbor Healthcare System Professor, Depts of Medicine, Cell Biology and Pharmacology, State University of New York Downstate Medical Center and NYU School of Medicine, New York, NYMedicalResearch.com Interview with: Mohamed Boutjdir, PhD, FAHA Director of the Cardiovascular Research Program VA New York Harbor Healthcare System Professor, Depts of Medicine, Cell Biology and Pharmacology, State University of New York Downstate Medical Center and NYU School of Medicine, New York, NY Medical Research: What is the background for this study? What are the main findings? Dr. Boutjdir: Patients with autoimmune diseases including Sjogren’s syndrome, systemic lupus erythematosus and other connective tissue diseases who are seropositive for anti-SSA/Ro antibodies may present with corrected QTc prolongation on the surface ECG. This QTc prolongation can be arrhythmogenic and lead to Torsades de Pointes fatal arrhythmia. In our study, we established for the first time an animal model for this autoimmune associated QTc prolongation that is reminiscent of the clinical long QT2 syndrome. We also demonstrated the functional and molecular mechanisms by which the presence of the anti-SSA/Ro antibodies causes QTc prolongation by a direct cross-reactivity and then block of the hERG channel (Human ether-a-go-go-related gene). This hERG channel is responsible for cardiac repolarization and its inhibition causes QTc prolongation. We were able to pinpoint to the target epitope at the extracellular pore forming loop between segment 5 and segment 6 of the hERG channel.
Author Interviews, Connective Tissue Disease, Emory, Kidney Disease, Race/Ethnic Diversity / 20.02.2015

Laura Plantinga, PhD Assistant Professor Division of Renal Medicine, Department of Medicine Emory University School of Medicine Atlanta, GA 30322MedicalResearch.com Interview with: Laura Plantinga, PhD Assistant Professor Division of Renal Medicine, Department of Medicine Emory University School of Medicine Atlanta, GA 30322 Medical Research: What is the background for this study? What are the main findings? Dr. Plantinga: Quality of care for end-stage renal disease (ESRD), which is treated with dialysis or kidney transplantation, is a high priority for the U.S. healthcare system, given universal coverage of these services. However, quality of ESRD care remains relatively unexplored in lupus patients, who have multiple providers and may have greater access to care. We found that, overall, nearly three-quarters of U.S. ESRD patients with lupus had pre-ESRD nephrology care and about 20% of lupus patients on dialysis were waitlisted for kidney transplant per year; however, fewer than one-quarter of those who started on dialysis had a permanent vascular access in place, which is associated with better outcomes than a temporary catheter. Furthermore, patients who were black or Hispanic were nearly a third less likely to have pre-ESRD care and were also less likely to be placed on the kidney transplant waitlist in the first year of dialysis than white patients. Having Medicaid or no insurance at the start of ESRD were both associated with lower likelihood of quality ESRD care by all measures, despite universal Medicare coverage after the start of ESRD. While there was geographic variation in quality of ESRD care, patterns were not consistent across quality measures.
Author Interviews, Connective Tissue Disease, Transplantation / 03.07.2014

MedicalResearch.com Interview with: ProfProf. Dr. Jacob M. van Laar Professor and Chair Dept of Rheumatology & Clinical Immunology University Medical Center Utrecht . Dr. Jacob M. van Laar Professor and Chair Dept of Rheumatology & Clinical Immunology University Medical Center Utrecht MedicalResearch: What are the main findings of the study? Prof. van Laar: The results of the ASTIS-trial demonstrate that stem cell transplantation in selected patients with early, diffuse cutaneous systemic sclerosis, a rare, autoimmune connective tissue disease, prolongs long-term survival and improves clinical manifestations (skin, lung) and quality of life, when compared to monthly infusions with cyclophosphamide. The benefits must be weighed against the risks which include early  treatment-related mortality (10% in the ASTIS-trial) and viral infections.
Author Interviews, Connective Tissue Disease / 17.05.2014

MedicalResearch.com Interview with: Dr Renee Martin Anthera Pharmaceuticals, Inc. 25801 Industrial Blvd, Suite B., Hayward, CA 94545, MedicalResearch: What are the main findings of the study? Dr. Martin: The PEARL-SC study identified many key elements that inform the further development of blisibimod for treatment of SLE: i.         That patients with severely active disease responded well to blisibimod, ii.         That a dose of 200mg blisibimod administered subcutaneously once per week improved measures of SLE disease activity, iii.         That greater treatment effect was observed when the SRI-7 and SRI-8 endpoints were evaluated, presumably because the criteria for these endpoints (including 7- or 8- point improvements in SELENA-SLEDAI score, respectively, along with no new BILAG A or 2B scores, and no worsening of physician’s global assessment score) demand substantial improvement in disease activity compared with baseline, and are unlikely to be met by chance (e.g. in the placebo group), and iv.         That blisibimod was safe and well-tolerated over 24-52 weeks of continuous therapy.
Author Interviews, Biomarkers, Connective Tissue Disease, NEJM / 19.12.2013

Prof. Dr. T.R.D.J. Radstake, MD, PhD Staff Rheumatologist / head translational Immunology Department of Rheumatology & Clinical Immunology EULAR Center of Excellence Director, UMC Utrecht Infection and Immunity FOCIS Center of Excellence University Medical Center Utrecht, The NetherlandsMedicalResearch.com Interview with: Prof. Dr. T.R.D.J. Radstake, MD, PhD Staff Rheumatologist / head translational Immunology Department of Rheumatology & Clinical Immunology EULAR Center of Excellence Director, UMC Utrecht Infection and Immunity FOCIS Center of Excellence University Medical Center Utrecht, The Netherlands MedicalResearch.com: What are the main findings of the study? Prof. Radstake: We observed that the chemokine CXCL4 is highly produced by so-called plasmacytoid dendritic cells in systemic sclerosis (Ssc). CXCL4 is associated with the progression and clinical phenotype of Ssc and thus provides a tool for clinicians to identify those patients in need for aggressive therapy and on the other hand, avoid unnecessary side-effects for those who have mild disease. Moreover, the identified roles for CXCL4 in SSc sparks our knowledge on the pathogenic pathways at hand in this terrible conditions. Now, we and other groups will have to further unravel the precise roles for CXCL4 in SSc and possibly other fibrotic and immune mediated conditions that cover the spectrum of medicine.
Author Interviews, Compliance, Connective Tissue Disease, UCSF / 25.11.2013

Dr. Jinoos Yazdany MD, MPH Assistant Professor in Residence UCSF School of MedicineMedicalResearch.com Interview with: Dr. Jinoos Yazdany MD, MPH Assistant Professor in Residence UCSF School of Medicine MedicalResearch.com: What are the main findings of the study? Answer: For almost all of the drugs we examined, we found that less than half of patients adhered to treatment.  For some drugs, less than one-third of individuals were adherent. The average medication possession ratios were low across all drugs. We found that several factors played an important part in adherence.  Younger individuals were less likely to adhere to treatment for several drugs, and we also found racial/ethnic differences, with Black, Hispanic and Native populations having lower adherence.  We also found geographic variation in adherence, with individuals in the Northeast being the most likely to adhere to treatment.