Author Interviews, Heart Disease, JACC, Pediatrics, Surgical Research, UCSD / 04.01.2018
Children with Heart Failure from Dilated Cardiomyopathy are Surviving Longer in the Most Recent Era
[caption id="attachment_39174" align="alignleft" width="300"]
Structural categories of cardiomyopathy Wikipedia image[/caption]
MedicalResearch.com Interview with:
Rakesh K. Singh MD, MS
Department of Pediatrics, University of California–San Diego and
Rady Children’s Hospital
San Diego, California
Steven E. Lipshultz MD
Department of Pediatrics
Wayne State University School of Medicine and
Children’s Hospital of Michigan
Detroit, Michigan
MedicalResearch.com: What is the background for this study?
Response: Dilated cardiomyopathy (DCM) is a disease characterized by dilation and dysfunction of the left ventricle of the heart. While DCM is a relatively rare disease in children, nearly 40% of children with DCM require a heart transplant or die within 2 years of diagnosis. Heart transplantation has improved the outcomes of children with DCM over the last 3 decades, but is limited by donor heart availability. Newer therapies, including advanced ICU care and artificial heart machines, are now being used to treat children with DCM.
This study published in the November 28, 2017 issue of the Journal of American College of Cardiology (JACC) sought to determine whether more children with DCM were surviving longer in the more recent era. Specifically, it investigated whether children with DCM were surviving longer without the need for heart transplantation. Rakesh Singh, MD is the first author and an Associate Professor of Pediatrics at UC San Diego/Rady Children’s Hospital, while the senior author is Steven Lipshultz, MD, Professor at Wayne State University School of Medicine/Detroit Medical Center’s Children’s Hospital of Michigan and Director of Children’s Research Center of Michigan.
The Pediatric Cardiomyopathy Registry (PCMR) is a National Heart, Lung, and Blood Institute (NHLBI) sponsored registry from 98 pediatric centers in United States and Canada created to study the outcomes of children with various heart muscle disorders known as cardiomyopathies. For this study, outcomes of 1,199 children diagnosed with DCM from 1990-1999 were compared with 754 children diagnosed with DCM from 2000-2009.
Structural categories of cardiomyopathy Wikipedia image[/caption]
MedicalResearch.com Interview with:
Rakesh K. Singh MD, MS
Department of Pediatrics, University of California–San Diego and
Rady Children’s Hospital
San Diego, California
Steven E. Lipshultz MD
Department of Pediatrics
Wayne State University School of Medicine and
Children’s Hospital of Michigan
Detroit, Michigan
MedicalResearch.com: What is the background for this study?
Response: Dilated cardiomyopathy (DCM) is a disease characterized by dilation and dysfunction of the left ventricle of the heart. While DCM is a relatively rare disease in children, nearly 40% of children with DCM require a heart transplant or die within 2 years of diagnosis. Heart transplantation has improved the outcomes of children with DCM over the last 3 decades, but is limited by donor heart availability. Newer therapies, including advanced ICU care and artificial heart machines, are now being used to treat children with DCM.
This study published in the November 28, 2017 issue of the Journal of American College of Cardiology (JACC) sought to determine whether more children with DCM were surviving longer in the more recent era. Specifically, it investigated whether children with DCM were surviving longer without the need for heart transplantation. Rakesh Singh, MD is the first author and an Associate Professor of Pediatrics at UC San Diego/Rady Children’s Hospital, while the senior author is Steven Lipshultz, MD, Professor at Wayne State University School of Medicine/Detroit Medical Center’s Children’s Hospital of Michigan and Director of Children’s Research Center of Michigan.
The Pediatric Cardiomyopathy Registry (PCMR) is a National Heart, Lung, and Blood Institute (NHLBI) sponsored registry from 98 pediatric centers in United States and Canada created to study the outcomes of children with various heart muscle disorders known as cardiomyopathies. For this study, outcomes of 1,199 children diagnosed with DCM from 1990-1999 were compared with 754 children diagnosed with DCM from 2000-2009.




Dr. Jonathan Hsu[/caption]
Jonathan Hsu, MD, MAS, FACC, FAHA, FHRS
Assistant Professor
Cardiac Electrophysiology, Division of Cardiology
University of California, San Diego (UCSD)
MedicalResearch.com: What is the background for this study?
Response: Atrial fibrillation (AF) is the most common cardiac arrhythmia worldwide and imparts significant stroke risk. In patients with AF determined to be at intermediate to high risk for thromboembolism, anticoagulation with warfarin (a vitamin K antagonist) or the newer non-vitamin K antagonist oral anticoagulants clearly reduces morbidity and mortality compared to aspirin. We sought to evaluate practice patterns of cardiovascular specialists in the United states to determine how often AF patients at risk for stroke are prescribed aspirin over oral anticoagulation, and predictors of this practice.
Dr Siddharth Singh[/caption]
Siddharth Singh, MD, MS
Postdoctoral Fellow, NLM/NIH Clinical Informatics Fellowship
Division of Biomedical Informatics
Clinical Assistant Professor of Medicine, Division of Gastroenterology, Department of Internal Medicine,
University of California
San Diego, La Jolla
MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. Singh: Over the last 4 years, four new medications have been approved for long-term use for weight loss by the FDA. We sought to evaluate the comparative effectiveness and tolerability of these medications through a systematic review and network meta-analysis. Based on 28 trials in over 29,000 overweight or obese patients, we observed that magnitude of weight loss achieved with these agents is variable, ranging from 2.6kg with orlistat to 8.8kg with phentermine-topiramate. Over 44-75% of patients are estimated to lose at least 5% body weight, and 20-54% may lose more than 10% of body weight; phentermine-topiramate was the most efficacious, whereas lorcaserin was the best tolerated.
Dr. Maria Schwaederle[/caption]
Maria Schwaederle PharmD
Clinical Research Scientist
Center for Personalized Cancer Therapy
UCSD Moores Cancer Center
La Jolla, CA 92093
MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. Schwaederle: We performed this analysis with experts in the field, including but not limited to Drs Schilsky, Lee, Mendelsohn and Kurzrock, all known for their experience in the area of precision/personalized medicine.
Historically, phase I trials (which are often first in human or highly experimental in other ways) were believed to be examining only toxicity. Our meta-analysis of 13,203 patients shows that in the era of precision medicine, this historical belief needs to be discarded. Second, it is the use of precision medicine that makes this belief outdated.
Indeed, Phase I trials that utilized a biomarker-driven approach that is the essence of precision medicine had a median response rate of about 31%, which is higher than many FDA approved drugs, and this is in spite of the fact that phase I patients are a highly refractory group having failed multiple lines of conventional therapy.
Importantly, however, it was not the use of targeted agents alone that was important. It was the biomarker-based approach where patients are matched to drugs. Without matching, response rates were dismal—about 5%.




Prof. Schmid-Schonbein[/caption]
MedicalResearch.com Interview with:
Geert W. Schmid-Schonbein, Ph.D.
Distinguished Professor and Chairman
Department of Bioengineering
Adjunct Professor in Medicine
University of California San Diego
Medical Research: What is the background for this study? What are the main findings?
Dr. Schmid-Schonbein: Most approaches to control/reduce body weight focus on reducing food quantity, improving quality and promoting daily activity. These approaches, effective in the short term, only yield modest weight control. Weight management strategies recommended in the past have not significantly diminished the current trend towards childhood and adolescence obesity.
We developed and tested an alternative approach to control weight gain in healthy individuals to reduce the risk for development of obesity and diabetes complications. The essence is to:
“Eat deliberately slow AND stop eating when you feel no longer hungry”.
The approach avoids any form of special diet, uses no drugs, can be adopted for a lifetime and used in any ethnic environment. Children in a Mexican School in Durango were instructed by a pediatrician to learn to eat deliberately slow and to stop eating when the satiety reflex sets in, i.e. the moment when the feeling of hunger has disappeared.
They were instructed to:













