Progress in Pediatric Cardiology 43 (2016) 12
Contents lists available at ScienceDirect
Progress in Pediatric Cardiology
journal homepage: www.elsevier.com/locate/ppedcard
Preface
Proceedings of 2015 Innovations in Pediatric Heart Failure Symposium
Heart failure in children is a rare disease that often leads to poor clin-
ical outcomes. For example, the incidence of dilated cardiomyopathy in
childhood is relatively small as compared to other childhood diseases,
estimated at 0.57 cases per 100,000 per year in the US. However, nearly
half of these children (46%) will die or require heart transplantation
within ve years of presentation [1]. With the improvement in out-
comes for children with congenital heart disease, the management of
heart failure in survivors of congenital heart surgery has become a sig-
nicant burden for many pediatric and adult congenital cardiac centers.
Finally, with the explosion of new technology over the last 2 decades,
the use of newer medical devices such as dual-chamber pacemakers
and fully-implantable ventricular assist devices have changed the land-
scape of heart failure management in children.
The Innovations in Pediatric Heart Failure Symposium was held in San
Diego from December 3-5, 2015 to address and highlight some of these
issues. The symposium was jointly presented by Rady Childrens Hospi-
tal San Diego, UC San Diego School of Medicine, and Childrens Hospital
of Orange County. Over 50 internationally recognized faculty members
from over 20 institutions covered challenging heart failure topics relat-
ed to cardiomyopathies, end stage congenital heart disease (including
the failing Fontan), heart transplantation, ventricular assist devices,
interventional cardiology, cardiac ICU management, nursing care, geno-
mics, cardiac imaging, pharmacotherapeutics, electrophysiology and
more.
In this issue, we present 14 papers based upon the symposium talks
and related research on various topics in congenital heart disease, heart
failure and transplantation. The rst manuscript provides an important
roadmap of the evolution of medical therapy for children with heart fail-
ure. Dr. Yuk Law and his colleagues provide a strong argument that chil-
dren and congenital heart disease are not the same as adults and
ischemic/dilated cardiomyopathy, respectively.
The second paper, from Drs. Daphne Hsu and Robert Shaddy, re-
views the advantages, disadvantages, and alternatives to using adult
heart failure clinical trials to guide pediatric heart failure therapy.
They discuss the relevant adult and pediatric heart failure clinical trials
that potentially offer insight into disease management for the clinician.
They nish with a discussion about how signicant differences in pa-
tient population, underlying anatomy, predicted outcomes, and phar-
macokinetics and pharmacodynamics preclude the widespread
adoption of adult therapies in children with heart failure based on the
results of adult heart failure trials alone.
The third manuscript, from Drs. Scott Auerbach and Shelley
Miyamoto, provides a state-of-the-art review of current biomarkers in
pediatric heart failure. They describe the standard biomarkers used cur-
rently in clinical practice, along with emerging biomarkers being tested
primarily in a research setting.
http://dx.doi.org/10.1016/j.ppedcard.2016.11.002
1058-9813/ 2016 Published by Elsevier Ireland Ltd.
The fourth paper, from Drs. Anthony McCanta and James Perry, dis-
cusses the current state of cardiac resynchronization therapy (CRT) in
children with heart failure. CRT is a widely utilized and effective therapy
for adults with ventricular dysfunction and some degree of ventricular
conduction delay. While few studies exist regarding CRT in children,
this paper describes the potential benet of CRT and challenges associ-
ated with this heterogeneous population. The paper concludes with dis-
cussions on the future of cardiac resynchronization therapy in children,
including new technology to better understand interventricular ow
patterns and myocardial efciency before and after CRT.
The fth manuscript describes the current diagnosis and treatment
strategies for children with myocarditis. Dr. Rakesh Singh and his col-
leagues provide a review of diagnosis and presentation of these patients
before providing an extensive discussion of treatment strategies that are
data-driven. Mechanical circulatory support options and immunomod-
ulatory treatments are discussed along with their potential benets and
pitfalls. Given the heterogeneity of pediatric patients with myocarditis,
the authors conclude with a plea for smaller, focused clinical trials en-
rolling children with persistent inammatory or viral infection.
The sixth paper provides an extensive, contemporary review of me-
chanical circulatory support in children. Dr. Shriprasad Deshpande and
colleagues describe the challenges and opportunities associated with
extracorporeal membrane oxygenation (ECMO) and ventricular assist
device (VAD) use in children. They provide an important update of
emerging technologies and upcoming clinical trials.
The seventh manuscript reviews the important role of advanced
practice nursing in pediatric heart failure models of care and is based
on the talks given during the nursing breakout sessions at the sympo-
sium. Mrs. Gonzalez and her heart failure nurse practitioner colleagues
describe the current and evolving therapies for pediatric heart failure,
including home inotropic therapy and outpatient ventricular device
support. Special advanced practice nursing topics, such as psychosocial
considerations and development of a heart failure program, are
discussed in detail in this manuscript.
The eighth and ninth papers are from the Fontan Failure to Success
session at the symposium. Dr. Vaughn and her colleagues review the
current medical, surgical and catheter-based interventions for the fail-
ing Fontan patient. Drs. Sanjeet Hegde and Albert Hsiao describe
newer imaging techniques for improving pre-surgical planning and
post-operative management of these complex Fontan patients. Novel
technologies described include advanced MRI, computer aided design,
three-dimensional printing, virtual surgery and computational uid
dynamics.
The tenth manuscript describes an atlas-based ventricular shape
analysis for better understanding congenital heart disease (CHD).
Dr. Andrew McCulloch and his colleagues describe their novel
2 Preface
techniques for associating abnormal ventricular shape with functional
defects in myocardial deformation in eight adolescent or adult single-
ventricle CHD patients with tricuspid atresia and Fontans. These nd-
ings suggest that atlas-based ventricular shape analysis may be a useful
new tool in the management of patients with CHD who are at risk of im-
paired ventricular wall mechanics and chamber remodeling.
The eleventh paper is a comprehensive review of right sided heart
failure and pulmonary hypertension. Dr. Alejos and colleagues describe
the pathogenesis of pulmonary hypertension (PH) and right ventricular
(RV) dysfunction, along with current therapies available to target PH
and RV failure. They conclude with an extensive discussion of newer
treatment options, including many still undergoing research testing.
The development of HLA and other antibodies against the allograft is
one of the most challenging areas in pediatric heart transplantation
today. The twelfth manuscript describes the recent advances in heart
transplant immunology. Dr. OConner and colleagues review the
advancements in the characterization and understanding of these anti-
bodies, along with the optimal management of patients with anti-HLA
antibodies before, during, and after transplant.
The thirteenth paper highlights the current knowledge in the new
eld of cell-based regenerative therapies and the potential these strate-
gies offer children with severe heart disease. Dr. Nelson and colleagues
summarize prior and ongoing clinical and pre-clinical studies that offer
the most promise for pediatric heart failure patients.
The nal manuscript looks to the future of pediatric cardiology by
reviewing advances in the use of big data in medicine. Dr. Anthony
Chang discusses the expansion of biomedical big data and the potential
for articial intelligence to harvest meaningful medical data to improve
quality and outcome.
We hope that these manuscripts will stimulate discussion in our
community on ways to collaborate on future research priorities that
may ultimately lead to advanced diagnostic capabilities, improved clin-
ical outcomes, and better quality of life for our patients and their
families.
Reference
[1] Towbin JA, Lowe AM, Colan SD, et al. Incidence, causes, and outcomes of dilated car-
diomyopathy in children. JAMA 2006;296:186776.
Rakesh K. Singh
Anthony C. Chang
Jeffrey A. Towbin
Steven E. Lipshultz