A Proposal for the IMACS (ISHLT Mechanically Assisted Circulatory Support) Registry James K. Kirklin April 2011

Historical Development of ISHLT MCS Registries First MCS Registry. Ill-fated attempt to stage collection of tier 1 (demographics) and tier 2 (outcomes) data. Registry discontinued in consortium of ISHLT stakeholders in MCS therapy asked me and colleagues at UAB to lead response to NIH RFP for US database in MCS. Result- INTERMACS, which earlier this year was awarded a 5 year renewal.

Historical Development of ISHLT MCS Registries Subsequent efforts for an international MCS database focused on the Tx Registry model (collect incompletely validated data from all possible centers) Efforts within ISHLT unsuccessful in a landscape of polarized views about US vs European location of database. No traction from EuroMacs or EuroTransplant in establishing a viable database – Revised ISHLT mission statement established MCS at equal priority level as Hrt and Lung Tx

Historical Development of ISHLT MCS Registries Dec, 2010 – the Board reaffirmed its interest in establishing an MCS registry to further position ISHLT as the home for MCS clinical science and practice. Further, emanating from the strategic planning process, the ISHLT Board pledged to reject international politics as an agenda for decision- making in favor of merit/quality-based decisions regarding initiatives of global ISHLT interest. Extending this principle to the MCS registry initiative, the board invited me to provide a formal proposal at this Board meeting.

MCS Registry Proposal The novel concept I presented in December with agreement of the Board was the development of an ISHLT MCS Registry that focuses on collecting accurate, complete, and verifiable data from any non-US centers willing to commit to our data quality process. (This contrasts with the previous principle of getting any possible data, regardless of quality). This data would be combined with the INTERMACS data set to provide scientific quality analyses to seek truths in the global application of durable MCS, all under the ISHLT brand. It is with the spirit of this historical evolution in the current ISHLT mission that I make the following proposal.

UMACS Established in 2009 at the recommendation of NHLBI, UMACS is a not-for-profit group based at UAB for the sole purpose of contracting with national and international entities for the collection and/or analysis of data about outcomes in MCS therapy. UMACS is NOT supported by the NHLBI and is not a part of INTERMACS. This proposal is from UMACS. UNOS would be subcontracted by UMACS (by prior agreement) only for data collection. Center interactions, data management, and analyses would be performed by UMACS.

Intl Years Inst Pts Congenital Heart Surgeons Society Registry (CHSS) Y ,750 Cardiac Transplant Research Database (CTRD) N ,000 Pediatric Heart Transplant Study (PHTS) Y ,000 ISHLT MCSD Registry (participated in the analysis) Y Inter-agency Registry for Mechanically Assisted Circulatory N ,500 Assisted Circulatory Devices (INTERMACS) BeneMACS (Benelux countries with Thoratec) Y MedaMACS (End stage heart disease: medical treatment) N UABs Experience with Registries (Why you should trust us) IMACS Registry

Annual InstitutionsPatientsCost ($) ISHLT Tx202,000 Registry mechanics46 (35 US) 170 Analysis ISHLT MCSD ,000 INTERMACS1201, million Costs of Registries IMACS Registry

Governance ISHLT Board of Directors IMACS Registry Director UMACS UNOS IMACS Registry

Hospital Enrollment During the last five years we have been contacted by a number of non-US hospitals desiring to either join INTERMACS or join an MCSD registry that had similar definitions and major data elements as INTERMACS. We have also been contacted by groups of hospitals usually at the national level. We would create, in collaboration with ISHLT, a proactive plan for hospital enrollment. IMACS Registry

Inclusion Criteria All approved MCSDs (country by country) Exclusion Criteria None IMACS Registry

Adverse EventsMajor Events Device malfunctionDeath Infection episodeTransplant Bleeding episodeRecovery Neurological eventDevice exchange IMACS Registry

Data Structure and Elements Pre-implant Form Implant Form Discharge Form Follow-up (every 6 months) Form Adverse Events will be captured in the discharge form and the follow-up forms. Note: Exchange of an LVAD or TAH will terminate the first record and will start a new record. IMACS Registry

Data Entry: Two Mechanisms Direct Entry: The hospital enters data into the ISHLT VAD Registry. Indirect Entry: The hospital enters data into another database (a national database or consortium database: known as a collective). The data from the collective is imported into the ISHLT VAD Registry according to a precise technical protocol that will be created by UNOS. The collective would generate the export file that meets the UNOS specifications. This would be similar to the data transfers for the Tx registry. IMACS Registry

Deliverables A registry Quarterly progress reports to ISHLT Statistical Reports to ISHLT, hospitals, collectives, countries Data Quality: create and implement a plan Disciplined definitions of Outcome Events Science/Research Web Site (an addendum to the current ISHLT website) IMACS Registry

Collective Enrollment During the first year of registry Identify and contact any existing MCSD registries (e.g. JMACS, BeneMACS) Create technical document to specify the parameters for export files (collective to the IMACS Registry) Implement the download IMACS Registry

First YearYear 2Year 3 UMACS$ $50,000 $50,000 UNOS$133,500 $16,000$16,000 Total$196,000 $66,000$66,000 *excludes collective downloads Cost for the Registry* IMACS Registry

First YearYear 2Year 3 UMACS$ $94,000$94,000 UNOS$133,500 $16,000$16,000 Total $255,000$110,000$110,000 * excludes collective downloads Cost for the Registry and Statistical Report and Analyses* IMACS Registry

First YearYear 2Year 3 UMACS$61,500 $110,000$94,000 UNOS$133,500 $16,000$16,000 Total$195,000$126,000$110,000 * excludes collective downloads Cost for the Registry If Statistical Report and Analyses Begin in Year 2* IMACS Registry

Guiding Principles High Quality Database Create and implement the registry Match implants at a hospital with implants from industry Require > 90% compliance with follow-up Respond to queries about data inconsistencies or errors Conduct training sessions Provide Benefits to ISHLT Own and direct an international high quality MCSD registry Will complement the ISHLT transplant registry as ISHLT emphasizes its role in MCSD research Provides Statistical Reports and Research Analyses to ISHLT Researchers Provide Benefits to the hospital Statistical summaries of their experience Benchmark against the international experience Participate in device research IMACS Registry

RECOMMENDATIONS Select proposal with data analyses and hospital reports beginning in year ($195, ,000 – 110,000) Commit to a 3 year trial period to fully explore this concept, after which the Board reassesses all aspects before going further. I would act as IMACS registry director for years I through 3 to make sure things are running well and that UMACS is maximally effective An MCS committee would be formed to outline initial expectations and enrollment goals as well as opportunities for collective data downloads in MCS. IMACS Registry

Registries for Evaluating Patient Outcomes: A Users Guide Second Edition 347 pages Publication: September 2010 Agency for Healthcare Research and Quality Advancing Excellence in Health Care ISHLT MACS Registry