ISHLT iPHF REGISTRY Status and Update April 2017 Based on data as of March 15, 2017 2017

Goals of the Registry To collect and analyze clinically relevant data on pediatric heart failure patients in order to better understand the natural history and response to current and future disease treatment regimes. 2017

Enrollment Overview First patient enrolled: April 4, 2016 16 Centers 81 patients 2017

Participating Institutions (N=16) * Institutions enrolled as of March 15, 2017 2017

In Progress Boston Children’s Cincinnati 2017

Potential Centres (have expressed interest / been in contact) Newcastle Singapore Rady Children’s (San Diego) Arkansas Washington Kentucky Utah Delaware Montefiore Osaka, Japan La Paz, Spain Oslo, Norway Michigan ?Others? 2017

Enrollment Activities As of March 15, 2017 Number of centers: 16 Number of patients: 81 2017

Age Distribution at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=81 2017

Race Distribution at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=75 2017

Gender Distribution at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=81 z scores 2017

Weight Distribution at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=74 Z scores 2017

Diagnosis at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=81 2017

Medical Condition at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=75 2017

Life Support at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=75 2017

Functional Status at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=73 2017

Academic Progress at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=74 2017

Academic Activity Level at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=73 2017

Cognitive Development at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=72 2017

Motor Development at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=72 2017

LVEF at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=64 2017

LVEDD at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=46 2017

MR at Enrollment 2017 (Patients diagnosed during 1/1/2016 – 3/3/2017)

Qualitative RV Function at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=71 2017

Qualitative RV Size at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=71 2017

Heart Catheterization at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=72 2017

Creatinine at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=74 2017

BNP at Enrollment 2017 (Patients diagnosed during 1/1/2016 – 3/3/2017)

nt-pro BNP at Enrollment (Patients diagnosed during 1/1/2016 – 3/3/2017) N=28 2017

Number of Hospitalizations (Patients diagnosed during 1/1/2016 – 3/3/2017) N=64 2017

Reasons for Hospitalization (Patients diagnosed during 1/1/2016 – 3/3/2017) N=62 2017

Length of First Hospitalization (Patients diagnosed during 1/1/2016 – 3/3/2017) N=61 2017

Interventions Initiated on First Hospitalization * (Patients diagnosed during 1/1/2016 – 3/3/2017) N=60 * Only most common interventions are displayed; Patients may be reported with more than one intervention. 2017

Outcomes of First Hospitalization (Patients diagnosed during 1/1/2016 – 3/3/2017) N=64 2017

Why Participate? Benefits to Centers Statistical summaries of hospital experience Participate in heart failure research Generate volume and trend data to facilitate short and long term manpower and financial planning 2017

Why Participate? Benefits to pediatric heart failure community Improved outcomes Facilitate accelerated evaluation of new therapies (drugs and devices) Development of international standards for pediatric heart failure 2017

Challenges: IRB and Data Sharing Agreements Templates online Consents online – including Spanish consents Some non-NA centers having challenges (e.g. Europe) Data sharing agreement with UNOS Draft online 2017

Challenges: Data Entry Feedback: Not difficult (data elements clear; web-based data entry very user friendly) Time Enrollment: 10-30 mins Hospitalization form: 10-30 mins 2017

Challenges: Data Entry Human Resources*** MD – fastest but workload and time Clinical APN or RN – have knowledge base but workload and time Research APN or RN Research coordinator – education Seems to be easiest if incorporated into patient care workflow 2017

Why should we persist? Funding – ISHLT Infrastructure – The database Collaboration – All of Us Unique – there is no other way to get this data to address gaps in knowledge Very Unique – merger projects with PEDIMACS/IMACS and the Transplant Registries (ISHLT, PHTS, and UNOS) 2017

iPHFR Patient Destinations Recovery Stable HF MCS (Pedimacs, iMacs) Listing (PHTS, UNOS) Tx (ISHLT, PHTS, UNOS) And the ability to understand better recovery and stability 2017

Steering Committee Anne Dipchand (Chair) Richard Kirk Chris Almond Christina VanderPluym Angela Lorts Yuk Law Elfi Pahl Estela Azeka Robert Gajarski Scott Auerbach Warren Zuckerman Wida Cherikh (UNOS) 2017

Institutional enrollment information available at... www.ishlt.org/registries 2017

Questions? anne.dipchand@sickkids.ca 2017