The Key to Success: INTERMACS Hospitals Site Utilization of INTERMACS Data and Reports for local QI Quality Assurance and data quality Evaluation of Site Data: Audits, Complete Enrollment, Complete Data, etc. Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

The Key to Success: INTERMACS Hospitals! Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

The Key to Success: INTERMACS Hospitals Site Utilization of INTERMACS Data and Reports for local QINaftel Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Hospitals What services do the hospitals receive for their participation fee? Services Meets CMS/Joint Commission requirement for Destination Therapy Certification Meets FDA required submission of Medical Device Reports (MDRs) by hospitals Provides clinical summaries of patients Provides quality assurance reports Provides electronic data transfer Provides standardized datasets Provides benchmarking Provides training and continuing education units INTERMACS Annual Meeting March 2012

Hospitals (Continued) What benefits do the hospitals receive for their participation fee? Benefits Fulfills CMS DT Certification requirement Become part of the national dialogue on the evaluation and evolution of MCSDs Invited to participate in the INTERMACS Annual Meeting Invited to join the INTERMACS Committees Coordinators Council and other committees Select Hospital Administrators will have the opportunity to serve on the Business Advisory Committee INTERMACS Annual Meeting March 2012

The Key to Success: INTERMACS Hospitals Quality Assurance and data qualityNaftel Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Hospital Perspective – Deliverables Coordinators Clinical Team Quality Assurance Officer Office of Risk Management Financial Officers / Administrators Office of Accreditation Researchers Fifth Annual Meeting, April 12, 2011 INTERMACS Annual Meeting March 2012

A Workshop for Researchers and INTERMACS Hospitals QA Report 8INTERMACS Data Workshop – April 12, 2011 INTERMACS Annual Meeting March 2012

A Workshop for Researchers and INTERMACS Hospitals QA Report 9INTERMACS Data Workshop – April 12, 2011 INTERMACS Annual Meeting March 2012

A Workshop for Researchers and INTERMACS Hospitals 10INTERMACS Data Workshop – April 12, 2011 INTERMACS Annual Meeting March 2012

A Workshop for Researchers and INTERMACS Hospitals 11INTERMACS Data Workshop – April 12, 2011 INTERMACS Annual Meeting March 2012

The Key to Success: INTERMACS Hospitals Evaluation of Site Data: Audits, Complete Enrollment, Complete Data, etc. Naftel Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

1. Regulatory Requirements Regulatory requirements must be met. Assessment: UNOS collects and the DCC evaluates all regulatory documents Goal: 100% of participating hospitals meet all regulatory requirements Minimal Standard: 100% Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

133 Activated Sites (currently 131) 1,705 INTERMACS Personnel 1,267 IRB Approvals since ,590 Informed Consent, HIPAA, Revoke Authorization, Transfer, Blood / Tissue documents 1,490 Human Subjects Training Certificates 1,805 Financial Disclosure / Conflict of Interest Coordinator Training, March 11, 2012 INTERMACS Annual Meeting March 2012

2. Timely Follow-up data Assessment: will focus on submission of follow-up forms. Goal: 100% of follow-up forms submitted within 30 days of the date of expected follow-up. Minimal Standard: 90% of follow-up forms must be submitted within 30 days of the date of expected follow- up Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 Overview of Data EntryNaftel 16 INTERMACS Annual Meeting March 2012

3. All Device Implants Complete Accounting of all eligible device implants. Assessment: Matching hospital enrollment to industry counts. Goal: 100% of eligible devices enrolled. Minimal Standard: 90% of eligible devices enrolled. Note1: All eligible DT devices must be entered. Note2: Informed consent is a barrier to the minimal standard. Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Days from Implant to Entry into INTERMACS Jun 2006 – Dec 2011 Sixth Annual Meeting, March 12, 2012 Median (days) n % < % 30 – % 60 – % 90 – % > % Total hospitals125100% INTERMACS Annual Meeting March 2012

Days from Implant to Entry into INTERMACS Jun 2006 – Dec 2011 Sixth Annual Meeting, March 12, 2012 Median (days) n % < % 30 – % 60 – % 90 – % > % Total hospitals125100% INTERMACS Annual Meeting March 2012

4. Completeness of data elements Assessment: The web-based application requires that all elements be addressed (either a data value entered or “not done” selected) before the form can be submitted. The proportion of captured data values will be calculated. Goal: 100% completion of data elements. Minimal Standard: To be determined after consultation with HSC. Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Completeness of Quality of Life Data will be addressed by Dr. Grady Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

The Key to Success: INTERMACS Hospitals Risk adjustment: Will require an in-depth, collaborative effort among the INTERMACS Collaborators including hospital representatives. The approach will be modeled after the SRTR evaluation of post heart transplant survival. Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Update on New INTERMACS/NIH Initiatives MedaMACS PumpKIN PediMACS IMACS Revive-IT Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

Update on New INTERMACS/NIH Initiatives MedaMACS (15 min)Stewart Sixth Annual Meeting, March 12, 2012 INTERMACS Annual Meeting March 2012

MEDAMACS Update 2012: Medical Arm of Mechanical Circulatory Support INTERMACS - 6 th Annual Meeting March 12, 2012 INTERMACS Annual Meeting March 2012

The MEDAMACS Mission Map terrain of ambulatory advanced heart failure Improve selection and timing of MCS by studying outcomes with medical therapy Support IOM mandate for patient-centered care and shared decision making Design integrated endpoints that move beyond survival alone Define a broader context for the next generation of MCS clinical trials INTERMACS Annual Meeting March 2012

Evolution of MEDAMACS INTERMACS Specific Aim 2006, 2011 MEDAMACSStudy n= centers 2 yrs follow-up Screening Pilot n= centers 1 yr follow-up INTERMACS Annual Meeting March 2012

Brigham and Women’sU. of Alabama Birm.U. of Pittsburgh Cedars-SinaiU. of ColoradoU. of Pennsylvania Cleveland ClinicU. of Iowa U. of South Florida DukeU. of MichiganU. of Texas Southwestern MEDAMACS Study Sites INTERMACS Annual Meeting March 2012

MEDAMACS ROADMAP 1 REVIVE-IT 2 Size350100~50 Design Prospective observational, OMM only Prospective observational, Thoratec HM2 vs. OMM Randomized trial, Heartware HVAD vs. OMM Inclusion Criteria HF symptoms >12m NYHA III-IV Optimal med rx ≥1 HF admission in 12m AND one 1.) Additional HF Hosp 2.) Peak VO2 <16 men, or <14 women 3.) BNP > ) 6MW <300m 5.) SHF Score ≥ 1.5 EF ≤35% Can be eligible for transplant, but not listed NYHA IIIB-IV ≥1 HF hospitalization in 12m, or 2 unscheduled EW visits for HF 6MW <300m EF ≤25% Not currently listed, not planned in 12m HF symptoms >12m NYHA III for 3m Optimal med rx ≤1 HF hosp within 6m, no admit in 30d CRT if QRS >120 Peak VO2 < 16men, <14women 6MW <350m SHF Score ≥ 1.5 EF ≤35% Not a transplant candidate LVAD candidate by CMS criteria 1.clinicaltrials.gov/NCT clinicaltrials.gov/NCT INTERMACS Annual Meeting March 2012

MEDAMACS ROADMAP 1 REVIVE-IT 2 Exclusion criteria Non-cardiac diagnosis limits prognosis ≤2 yrs Current inotrope use Listed for transplant QRS >120 and planned CRT, or CRT within 3m Other condition that limits survival to ≤2 yrs Inotrope in 30d Listed for transplant Inability to perform 6MW Severe illness limiting prognosis to ≤2 yrs STEMI within 3m Inotrope within 3m GFR <30, cerebrovasc dz A transplant candidate Plt <100K, alb <3, PVD CRT if QRS >120 must have been >90d previously Liver, pulm dysfunction Mechanical AVR PVR >6 Wood units Endpoints Primary at 1 yr Integrated endpoint of survival, requirement for advanced therapies, functional capacity, QOL, patient preferences Primary at 1yr Survival at 1yr with improvement of at least 75m in 6MW Actuarial survival and survival free of stroke Primary at 2 yrs Composite of survival, freedom from moderately disabling stroke, and 6MW improvement >75m 1.clinicaltrials.gov/NCT clinicaltrials.gov/NCT INTERMACS Annual Meeting March 2012

6MW Gait speed Euroqol+KCCQ VAD Survey Events Risk Scores Treatments Baseline B MEDAMACS Encounters Inpt or outpt 1 Month Re-Look Baseline 6 mos Phone Interview 1 Yr Face-to-face 18m 2 Yrs Phone Interview outpt AB C Time Zero D Telephone Contact 6MW Gait speed Euroqol+KCCQ VAD Survey Risk Scores 6MW Gait speed Euroqol+KCCQ VAD Survey Events Treatments Baseline A 6MW Gait speed Euroqol+KCCQ VAD Survey Events Risk Scores Treatments Study Site Phone Calls 6 and 18 mos Events (hosp, stroke, transplant, vad, inotropes, death) Meds, Euroqol, NYHA/INTERMACS profile One month Consent 1mo. 12mos 24mos Face-to-face encounters End 1-Yr Visit C 2-Yr Visit D INTERMACS Annual Meeting March 2012

MedaMACS Pilot Enrollment N=350 x 12sites Design MedaMACS OSMB Review & DCC IRB (11-12/2011) 6M Telephone 18M Telephone 1 Year Visit 2 Year Visit & Closeout WBDE Programming Double Baselines Completed DATABASE CLOSED 1 Yr Data Complete IRB STUDY SITE DCC + STEERING MEDAMACS Timeline Pilot Complete Late-Breaking Submission Baseline Data ISHLT/ACC Final Pilot Analysis Spring Training Annual Annual Annual Late-Breaking Submission 6M Data Complete Estimated # Active Pts ISHLT INTERMACS Annual Meeting March 2012

MEDAMACS at ISHLT 2012: Screening Pilot Data High Event Rates in Medically Managed Advanced Heart Failure Patients Followed at VAD Centers (Stewart et al.) Prediction of Events in Patients with Advanced Heart Failure: Application of the Seattle Heart Failure Model to the Medamacs Population (Teuteberg et al.) Patients with INTERMACS 4-7 Heart Failure Have Reduced Quality of Life (Patel et al.) Uric Acid Elevation is Associated with Severity of Congestion in Advanced Heart Failure (Guglin et al.) Tricuspid Regurgitation is a Measure of Right Heart Dysfunction and is Associated with Event Free Survival in Stage D Heart Failure (Testani et al.) INTERMACS Annual Meeting March 2012

Event-Free Survival Months since Enrollment Death Death, Transplant, or VAD Death, Transplant, VAD or Inotrope 68% 64% 84% Event-Free Survival in Ambulatory Advanced Heart Failure Event of Interest Stewart et al. ISHLT 2012 INTERMACS Annual Meeting March 2012

Survival Free of VAD or Transplant Months since Enrollment INTERMACS Profiles Risk Stratify Ambulatory Advanced Heart Failure Patients INTERMACS 6/7 INTERMACS 5 INTERMACS 4 P<0.001 Stewart et al. ISHLT 2012 INTERMACS Annual Meeting March 2012

Event-free survival (%) 0369 Months post-enrollment SHFM < 1.5SHFM SHFM > 2.5 p = by Seattle HFM category Event-free Survival Teuteberg et al. ISHLT 2012 REVIVE-IT INTERMACS Annual Meeting March 2012

Euroqol VAS Score by HF Severity INTERMACS; Grady K, et al J Heart Lung Trans 2009;28:S269. MEDAMACS: Patel P, et al. ISHLT HF Action; Flynn K, et al. Am Heart J 2009;158: INTERMACS Annual Meeting March 2012

InvestigatorSite Amrut Ambardekar U Colorado Chetan Patel Duke Eddie Rame U Penn Frances Johnson U Iowa Garrick Stewart Brigham and Women's Jeffrey Testani U Penn Jeffrey Teuteberg U Pitt Jennifer M. Cowger U Michigan JoAnn Lindenfeld U Colorado Jon Kobashigawa Cedars-Sinai Joseph Rogers Duke Keith Aaronson U Michigan Leslie Miller U South Florida Lynne Stevenson - PI Brigham and Women's Maria Mountis Cleveland Clinic Mariell Jessup U Penn Mark Drazner U Texas Southwestern Maya Guglin U South Florida Michele Hamilton Cedars-Sinai Michelle Kittleson Cedars-Sinai Parag Patel U Texas Southwestern Randy Starling Cleveland Clinic Salpy Pamboukhian U Alabama Birmingham Team MEDAMACS UAB/DCC David Naftel Mary Lynne Clark Susan Myers Craig Collum Kathryn HollifieldNHLBI/NIH Monica Shah Tim Baldwin Marissa Miller Thoratec Corporation INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 pediMACS David C. Naftel, PhD 39 INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 INTERMACS Re-Launch Status  Our Goals :  Reduce the number of elements and forms  Streamline the data entry process  Clarify elements that were confusing in the past  Examine the AE definitions for current clinical relevance 40 It became clear that the pediatric VAD patients needed a different web-based data entry system. INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 pediMACS Launch Status  Pediatric Committee  Betsy Blume, MD- Chair  David Morales, MD  David Rosenthal, MD  Peter Wearden, MD  Christopher Almond, MD  Robert Jaquiss, MD  Jonathan Chen, MD  Dee Dee Epstein, RN  Heidi Moses, MEd, CCRA  David Naftel, PhD  Tim Baldwin, PhD 41 INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 pediMACS Launch Status 42  We (Pediatric Committee, NIH, INTERMACS Co-PIs) have spent 1 year reviewing the WBDE in all aspects:  AE definitions and other definitions  Screens / Forms  Data Elements  Patient Flow through the WBDE INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 pediMACS Launch Status 43  pediMACS will follow the structure of INTERMACS  A few important changes from INTERMACS:  Pediatric patients (< 19 yrs. at time of implant)  Includes both durable and temporary support MCSDs  Modifications of AE definitions  Possible expansion of quality of life instruments INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 pediMACS Launch Status 44  Target Live test site: July 1, 2012  Testing by INTERMACS Nurse Monitors  Testing by the DCC Data Managers  Testing by the INTERMACS Co-PIs  Testing by 3 Hospitals (Beta Sites)  Testing by Pediatric Committee  Target Launch Date: August 1, 2012  Online training will be available  Training Session in September 2012 Target: Live Test Site July 1, 2012 Target: LAUNCH Date Aug 1, 2012 Jul 1 - Jul 31 INTERMACS Annual Meeting March 2012

Coordinator Training Session: March 11, 2012 pediMACS Launch Status 45  Training Session for pediMACS  “Mechanical Cardiac Support in Pediatric Heart Disease – State of the Art 2012”: September 20-22, 2012  The St. Louis Children’s and Washington University Heart Center INTERMACS Annual Meeting March 2012