Pedimacs. Quarterly Statistical Report. Pediatric Registry for Mechanically Assisted Circulatory Support

Transcription

1 1 #HHSN C Alfred M Pedimacs Pediatric Registry for Mechanically Assisted Circulatory Support Quarterly Statistical Report nd Quarter Implant and event dates: September 19, 2012 to June 30, /25/2017 Prepared by: The Data and Clinical Coordinating Center University of Alabama at Birmingham For questions or comments contact: James K. Kirklin, MD Betsy Blume, MD Craig Collum, MPH Nick Timkovich Kathryn Hollifield, RN Maceo Cleggett

2 2 #HHSN C Pedimacs Quarterly Report Coverage: September 19, 2012 to June 30, 2017 The Interagency Registry for Mechanically Assisted Circulatory Support (INTERMACS) is a North American registry established in 2005 for patients who are receiving mechanical circulatory support device therapy to treat advanced heart failure. Intermacs was established as a joint effort of the National Heart, Lung and Blood Institute (NHLBI), the Centers for Medicare and Medicaid Services (CMS), the Food and Drug Administration (FDA), clinicians, scientists and industry representatives in conjunction with Dr. James K. Kirklin and the University of Alabama at Birmingham. Pedimacs is the pediatric portion of Intermacs which was launched on 9/19/2012. While Intermacs has always included durable devices implanted in pediatric patients, Pedimacs has been developed to focus on capturing data elements unique to pediatric patients. Pedimacs evaluates special issues in the pediatric population receiving Mechanical Circulatory Support Device (MCSD) therapy, differences in devices available, and the particular pediatric population for whom this therapy may be most effective. Elizabeth Blume, M.D. from the Children's Hospital Boston, is the Pedimacs Chair. This quarterly report includes clinical information from 471 pediatric patients receiving prospective implants between September 19, 2012 and June 30, Table of Contents Exhibit 1: Pedimacs Participating Hospitals Exhibit 2: Pedimacs Enrollment Counts Exhibit 3: Pedimacs Patients - Temporary vs Durable Devices Exhibit 4: Pedimacs Patients Receiving Durable Devices - Demographics Exhibit 5: Pedimacs Patients Receiving Durable Devices - Device Type Exhibit 6: Pedimacs Patients Receiving Durable Devices - Patient Profile Exhibit 7: Pedimacs Patients Receiving Durable Devices - Device Strategy Exhibit 8: Pedimacs Patients Receiving Durable Devices - Patient Profile by Device Strategy Exhibit 9: Pedimacs Patients Receiving Durable Devices - Patient Status by Pre-Implant Device Strategy Exhibit 10: Pedimacs Patients Receiving Durable Devices - Primary Cause of Death Exhibit 11: Kaplan-Meier Survival for Durable Devices Exhibit 12: Kaplan-Meier Survival for Continuous Durable Devices Exhibit 13: Kaplan-Meier Survival for Pulsatile Durable Devices Exhibit 14: Competing Outcomes for Durable Devices Exhibit 15: Competing Outcomes for Continuous Durable Devices Exhibit 16: Competing Outcomes for Pulsatile Durable Devices Glossary

3 3 Exhibit 1: Pedimacs Participating Hospitals As of February 16, 2017 there are 77 hospitals enrolled in Pedimacs of whom 45 contributed data used in this report. SITE HOSPITAL NAME CITY STATE MNAN Abbott Northwestern Hospital Minneapolis MN ILCM Ann & Robert H. Lurie Children s Hospital of Chica Chicago IL ARCH Arkansas Children's Hospital Little Rock AR AZUA Banner Univ. Medical Center (Univ. of Arizona) Tucson AZ TNBM Baptist Memorial Hospital - Memphis Memphis TN MOBH Barnes-Jewish Hospital St. Louis MO NCCM Carolinas Medical Center Charlotte NC CACS Cedars Sinai Medical Center Los Angeles CA GAEH Children's Healthcare of Atlanta Atlanta GA MACH Children's Hospital Boston Boston MA WICH Children's Hospital of Wisconsin Milwaukee WI TXCM Children's Medical Center Dallas TX MOCM Children's Mercy Hospital Kansas City MO DCCH Children's National Medical Center Washington DC OHCM Cincinnati Children's Hospital Medical Center cincinnati OH OHCC Cleveland Clinic Cleveland OH NYCP Columbia Presbyterian - Children's Hospital of New New York NY NYCP Columbia University Medical Center-NY Presbyterian New York NY NCDU Duke University Medical Center Durham NC PAUP Hospital of the University of Pennsylvania Philadelphi PA TXMH Houston Methodist Hospital Houston TX WASH Inland Northwest Thoracic Organ Transplant Program Spokane WA FLJM Jackson Memorial Health System/University of Miami Miami FL CAKPX Kaiser Permanente Santa Clara Medical Center Santa Clara CA CAUH Keck Hospital of USC Los Angeles CA INLH Lutheran Hospital of Indiana Fort Wayne IN AZMC Mayo Clinic Hospital Phoenix AZ FLSL Mayo Clinic Jacksonville Jacksonvill FL MNSM Mayo Clinic Rochester MN Rochester MN TXHD Medical City Dallas Hospital Dallas TX SCMU Medical University of South Carolina Medical Cente Charleston SC INIM Methodist Hospital Indianapoli IN TXHS Methodist Specialty and Transplant Hospital San Antonio TX MOLH Mid America Heart Institute of Saint Luke's Hospit Kansas City MO NYMA Montefiore Medical Center Bronx NY

4 4 Exhibit 1: Pedimacs Participating Hospitals SITE HOSPITAL NAME CITY STATE NYMS Mount Sinai Medical New York NY OHCH Nationwide Children's Hospital Columbus OH DEAI Nemours/A.I. dupont Hospital for Children Wilmington DE NCBG North Carolina Baptist Hospital Winston Sal NC LAOF Ochsner Medical Center New Orleans LA ORUO Oregon Health & Science University Portland OR PAPPX Penn Presbyterian Medical Center Philadelphi PA PAHE Penn State Milton S. Hershey Medical Center Hershey PA CACH Rady Children's Hospital, San Diego San Diego CA WACH Seattle Children's Hospital Seattle WA VANG Sentara Norfolk General Hospital Norfolk VA FLUF Shands at the University of Florida Gainesville FL MOCH St. Louis Children's Hospital St. Louis MO INSV St. Vincent Hospital and Health Care Center Indianapoli IN CASU Stanford University Medical Center Stanford CA CASG Sutter Memorial Hospital Sacramento CA TXTC Texas Children's Hospital Houston TX PACP The Children's Hospital of Philadelphia Philadelphi PA COCH The Childrens Hospital Denver CO MDJH The Johns Hopkins Hospital Baltimore MD CAUC UCLA Medical Center Los Angeles CA ALUA University of Alabama at Birmingham Hospital Birmingham AL CASD University of California, San Diego Medical Center San Diego CA ILUC University of Chicago Hospitals Chicago IL IAIV University of Iowa Hospitals and Clinics Iowa City IA KYUK University of Kentucky Chandler Medical Center Lexington KY MDUM University of Maryland Medical Center Baltimore MD MIUM University of Michigan Health Systems Ann Arbor MI MNUM University of Minnesota Medical Center-Fairview Minneapolis MN NECH University of Nebraska Children's Hospital Omaha NE NCMH University of North Carolina Hospitals Chapel Hill NC PAPT University of Pittsburgh Medical Center Pittsbugh PA UTMC University of Utah Hospital Salt Lake C UT VAUV University of Virginia Health System Charlottesv VA DCWH Washington Hospital Center Washington DC WICH Children's Hospital of Wisconsin Milwaukee W TXMH Houston Methodist Hospital Houston T

5 5 Exhibit 1: Pedimacs Participating Hospitals SITE HOSPITAL NAME CITY STATE WASH Inland Northwest Thoracic Organ Transplant Progra Spokane W WACH Seattle Children's Hospital Seattle W VANG Sentara Norfolk General Hospital Norfolk V TXTC Texas Children's Hospital Houston T VAUV University of Virginia Health System Charlottes V

6 6 Exhibit 2: Pedimacs Enrollment Counts As of June 30, 2017 there were 77 hospitals participating in Pedimacs of whom 45 contributed prospective data used in this report.

7 7 Exhibit 3: Pedimacs Patients - Temporary vs Durable Devices Exhibits that are displayed after this exhibit are based on each patients first durable device.

8 8 Exhibit 4: Pedimacs Patients Receiving Durable Devices - Demographics The following tables present demographic characteristics for patients at the time of their first durable device implant (September 19, 2012 to June 30, 2017). Age Group AGE Pulsatile Continuous All n % n % n % <1 yr yr yr yr yr TOTAL Gender GENDER Pulsatile Continuous All n % n % n % Female Male TOTAL Race RACE Pulsatile Continuous All n % n % n % White African American Other TOTAL

9 9 Exhibit 5: Pedimacs Patients Receiving Durable Devices - Device Type First Durable Device Type GENDER Pulsatile Continuous All n % n % n % LVAD RVAD BiVAD TAH TOTAL

10 10 Exhibit 6: Pedimacs Patients Receiving Durable Devices - Patient Profile Patient profile status provides a general clinical description of the patients at the time of implantation. PRE-IMPLANT PATIENT PROFILE Pulsatile Continuous All n % n % n % 1. Critical Cardiogenic Shock Progressive Decline Stable but Inotrope Dependent Resting Symptoms or Less Sick TOTAL Critical cardiogenic shock describes a patient who is 'crashing and burning', in which a patient has life-threatening hypotension and rapidily escalating inotropic pressor support. 2. Progressive decline describes a patient who has been demonstrated 'dependent' on inotropic support, but nonetheless shows signs of continuing deterioration. 3. Stable but inotrope dependent describes a patient who is clinically stable on mild-moderate doses of intravenous inotropes. 4. Resting symptoms describe a patient who is at home on oral therapy, but frequently has symptoms of congestion at rest or with ADL. 5. Exertion Intolerant describes a patient who is comfortable at rest, but unable to engage in any activity, living predominantly within the house or household. 6. Exertion Limited also describes a patient who is comfortable at rest without evidence of fluid overload, but who is able to do some mild activity. 7. Advanced NYHA Class 3 describes a patient who is clinically stable with a reasonable level of comfortable activity, despite history of previous decompensation that is not recent.

11 11 Exhibit 7: Pedimacs Patients Receiving Durable Devices - Device Strategy Device strategy is determined in conjunction with the heart failure cardiologist and surgeon at the time of the implant. PRE-IMPLANT DEVICE STRATEGY Pulsatile Continuous All n % n % n % Bridge to Recovery Bridge to Transplant (patient currently listed for transplant) Possible Bridge to Transplant - Likely to be eligible Possible Bridge to Transplant - Moderate likelihood of becoming eligible Possible Bridge to Transplant - Unlikely to become eligible Destination Therapy (patient definitely not eligible for transplant) Rescue Therapy TOTAL Bridge to Transplant (BTT) Listed - patient already listed for transplant or listed within 24 hours before device implantation. 2. Bridge to Transplant (BTT) Likely - patient in whom the transplant evaluation has not been completed, but no contra-indications are anticipated, or in whom a current contra-indication is anticipated to resolve rapidly. 3. Bridge to Transplant (BTT) Moderate - patient in whom the transplant evaluation has not been completed, but with some potential concerns that might prevent eligibility. 4. Bridge to Transplant (BTT) Unlikely - patient in whom major concerns that might prevent eligibility have already been identified. 5. Destination Therapy - the patient is definitely not eligible for transplant. 6. Bridge to Recovery (BTR) - use of a durable device to allow recovery from chronic cardiac failure (at least 3 months in duration). 7. Rescue Therapy - use of a durable device to support resolution from an acute event without major previous cardiac dysfunction. 8. Other.

12 12 Exhibit 8: Pedimacs Patients Receiving Durable Devices - Patient Profile by Device Strategy The following tables present patient profile status by the device strategy for different flow types. Type=Pulsatile PATIENT PROFILE STATUS. Bridge to Recovery Pre-Implant Device Strategy Bridge to Transplant - Listed Bridge to Candidacy Destination Therapy Rescue Therapy TOTAL n % n % n % n % n % n % n % % % % % 1. Critical Cardiogenic Shock % % % % % % 2. Progressive Decline % % % % % 3. Stable but Inotrope Dependent % % % 4-7. Resting Symptoms or Less Sick % % % TOTAL % % % % % % % Type=Continuous Pre-Implant Device Strategy PATIENT PROFILE STATUS Bridge to Recovery Bridge to Transplant - Listed Bridge to Candidacy Destination Therapy TOTAL n % n % n % n % n % % % % 1. Critical Cardiogenic Shock % % % % % 2. Progressive Decline % % % % % 3. Stable but Inotrope Dependent % % % 4-7. Resting Symptoms or Less Sick % % % % TOTAL % % % % %

13 13 Exhibit 9: Pedimacs Patients Receiving Durable Devices - Patient Status by Pre-Implant Device Strategy The following tables present patient status as of June 30, 2017 by the device strategy for different time periods. Patient status is defined as the first of the following events: Alive (device in place): patients that were alive on a device at the end of this follow-up period. Transplant: patients that have received a transplant during this follow-up period. Recovery: patients that were explanted due to recovery at or before the end of this follow-up period. Dead: patients who died during this follow-up period. Type=Pulsatile Patient Status (June 30, 2017) PRE-IMPLANT DEVICE STRATEGY 1. Alive on a device 2. Transplanted 3. Death 4. Explant due to recovery TOTAL N N N N N Bridge to Recovery Bridge to Transplant (patient currently listed for transplant) Possible Bridge to Transplant - Likely to be eligible Possible Bridge to Transplant - Moderate likelihood of becoming eligible Possible Bridge to Transplant - Unlikely to become eligible Destination Therapy (patient definitely not eligible for transplant) Rescue Therapy TOTAL Type=Continuous Patient Status (June 30, 2017) PRE-IMPLANT DEVICE STRATEGY 1. Alive on a device 2. Transplanted 3. Death 4. Explant due to recovery TOTAL N N N N N Bridge to Recovery Bridge to Transplant (patient currently listed for transplant) Possible Bridge to Transplant - Likely to be eligible Possible Bridge to Transplant - Moderate likelihood of becoming eligible Possible Bridge to Transplant - Unlikely to become eligible Destination Therapy (patient definitely not eligible for transplant) TOTAL

14 14 Exhibit 10: Pedimacs Patients Receiving Durable Devices - Primary Cause of Death PRIMARY CAUSE OF DEATH Pulsatile Continuous All n % n % n % Circulatory: Other, Specify Device Malfunction Major Infection Respiratory: Respiratory Failure Circulatory: Sudden Unexplained Death Circulatory: Major Bleeding Circulatory: CHF Circulatory: End Stage Cardiomyopathy Nervous System: Neurological Dysfunction Multisystem Organ Failure (MSOF) Withdrawal of Support, specify Other, specify TOTAL Note: 0 patients have a missing primary cause of death.

15 15 Exhibit 11: Kaplan-Meier Survival for Durable Devices Percent Survival [% (70% CI)] Months after Device Implant Pedimacs Durable Dev % (91.3%-94.1%) % (82.0%-86.3%) % (75.0%-80.8%) % (71.4%-78.4%) % (48.0%-63.0%) % (48.0%-63.0%) % (48.0%-63.0%)

16 16 Exhibit 12: Kaplan-Meier Survival for Continuous Durable Devices Percent Survival [% (70% CI)] Months after Device Implant Durable CF % (97.4%-99.2%) % (88.9%-93.4%) % (83.4%-89.7%) % (80.2%-87.9%) % (55.8%-72.7%) % (55.8%-72.7%) % (55.8%-72.7%)

17 17 Exhibit 13: Kaplan-Meier Survival for Pulsatile Durable Devices Percent Survival [% (70% CI)] Months after Device Implant Durable PF % (80.9%-87.1%) % (69.0%-77.2%) % (59.4%-69.8%) % (55.2%-67.1%)

18 18 Exhibit 14: Competing Outcomes for Durable Devices Number of Patients at Risk Month

19 19 Exhibit 15: Competing Outcomes for Continuous Durable Devices Number of Patients at Risk Month

20 20 Exhibit 16: Competing Outcomes for Pulsatile Durable Devices Number of Patients at Risk Month

21 21 Exhibit 17: Follow-up Compliance Site compliance is determined by the percentage of all follow-up forms that are due during this reporting period that have been completed. This calculation has been updated to include ALL follow-up forms expected for ALL patients and ALL devices. Only sites that have at least 5 follow-up forms expected are included in this figure. Intermacs has defined required compliance as 90%.

22 22 Glossary BiVAD: BiVentricular Assist Device BMI: Body Mass Index BP: Blood Pressure BSA: Body Surface Area BTC: Bridge to Candidacy BTT: Bridge to Transplant BUN: Blood Urea Nitrogen CF: Continuous CMS: Centers for Medicare and Medicaid COPD: Chronic Obstructive Pulmonary Disease CNS: Central Nervous System CRP: C - Reactive Protein CVA: Cerebrovascular Accident DCC: Data Coordinating Center DT: Destination Therapy ECMO: Extracorporeal-membrane Oxygenation EQ-5D: Euro Quality of Life FDA: Federal Drug Administration HF: Heart Failure IABP: Intra-Aortic Balloon Pump IgG: Immunoglobulin G INR: International Normalized Ratio Intermacs: Interagency Registry for Mechanically Assisted Circulatory Support LVAD: Left Ventricular Assist Device LVEF: Left Ventricular Ejection Fraction LVEDD: Left Ventricular End Diastolic Dysfunction LVSF: Left Ventricular Shortening Fraction MCSD: Mechanically Circulatory Support Device NHLBI: National Heart Lung and Blood Institute NIH: National Institute of Health NT pro brain natriuretic peptide: N-Terminal pro brain Natriuretic peptide NYHA: New York Heart Association OR: Operating Room Pedimacs: Pediatric Registry for Mechanically Assisted Circulatory Support PF: Pulsatile Regurg: Regurgitation RVAD: Right Ventricular Assist Device RVEF: Right Ventricular Ejection Fraction SAE: Serious Adverse Event SGOT-AST: Serum Glutamic Oxaloacetic Transaminase SGPT-ALT: Serum Glutamic Pyruvic Transaminase TAH: Total Artificial Heart TIA: Transient Ischemic Attack VAD: Ventricular Assist Device VAS: Visual Analog Scale