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Pediatric Cardiomyopathy Registry

Pediatric Cardiomyopathy Registry. Outcome Predictors in Pediatric Hypertrophic Cardiomyopathy Steven Lipshultz, M.D. University of Miami School of Medicine Funded by NHLBI R01 HL 53392, DHHS. Presenter Disclosure Information. No relationships exist related to this presentation.

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Pediatric Cardiomyopathy Registry

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  1. Pediatric Cardiomyopathy Registry Outcome Predictors in Pediatric Hypertrophic Cardiomyopathy Steven Lipshultz, M.D. University of Miami School of Medicine Funded by NHLBI R01 HL 53392, DHHS

  2. Presenter Disclosure Information No relationships exist related to this presentation.

  3. Background In pediatric hypertrophic cardiomyopathy (HCM), there is limited understanding of patient factors measured at the time of diagnosis that affect the subsequent risk of death or heart transplant.

  4. Methods The NHLBI Pediatric Cardiomyopathy Registry (PCMR) collected longitudinal data on 882 children with HCM diagnosed with cardiomyopathy from 1990 to 2003: • 781 HCM • 57 HCM with dilated CM (DCM) • 44 HCM with restrictive CM (RCM) • or other CM

  5. Methods The 781 children with pure HCM were further divided into 6 subgroups by age and etiology: • Errors of Metabolism • Malformation Syndrome • Neuromuscular • Familial • Idiopathic, diagnosed at age <1 year • Idiopathic, diagnosed at age ≥1 year

  6. Methods We examined the combined outcome of time to death or heart transplant • We compared the outcome between the HCM subgroups using log-rank tests and Kaplan-Meier curves • Using demographic, clinical and echocardiographic data from the time of diagnosis, we identified predictors of outcome using proportional hazards regression

  7. Patient Characteristics at Diagnosis of HCM Pure HCM HCM/DCM HCM/RCM Mean Age (yrs) 5.6 (6.2) 3.7 (5.8) 6.0 (5.7) Mean BSA (m2) .83 (.64) .59 (.54) .80 (.57) Weight Z-Score .02 (1.76) -1.05 (1.4) -.74 (1.9) Female 33% 46% 45% CHF 15% 65% 43%

  8. Echocardiographic Z Scores at Diagnosis of HCM Pure HCM HCM/DCM HCM/RCM Fractional Shortening 3.3 (5.4) -7.2 (4.2) -.51 (4.3) ED Wall Thickness: Septal 3.3 (2.6) 1.7 (2.7) 2.6 (2.8) Posterior 2.2 (3.0) 2.7 (3.8) 2.1 (3.1) ED Dimension -2.0 (3.6) 2.2 (2.4) -.46 (2.6)

  9. Mortality and Transplant No. of No. of No. of Transplants & Children Deaths (%) Deaths (%) Pure HCM 781 110 (14%) 128 (16%) Errors of Metabolism 62 33 (53%) 36 (58%) Malformation Syndrome 49 12 (24%) 12 (24%) Neuromuscular 51 3 ( 6%) 3 ( 6%) Familial 79 4 ( 5%) 7 ( 9%) Idiopathic 540 58 (11%) 70 (13%) Age < 1 year 220 42 (19%) 48 (22%) Age ≥ 1 year 320 16 ( 5%) 22 ( 7%)

  10. Mortality and Transplant No. of No. of No. of Transplants & Children Deaths (%) Deaths (%) HCM/DCM 57 18 (32%) 23 (40%) HCM/RCM 44 11 (25%) 18 (41%)

  11. Multivariable Modeling for Death or Transplant Results from a multivariable regression model for time to death or transplant in HCM patients, stratified for HCM subgroup: Predictor Hazard Ratio P value BSA .93 [.50, 1.7] .82 CHF 1.63 [.96, 2.8] .07 Black vs. White 2.24 [1.3, 3.9] .01 Hispanic vs. White 1.55 [.85, 2.8] .16 Female vs. Male 1.23 [.81, 1.9] .34 ED Posterior Wall Z score 1.14 [1.1, 1.2] <.001 Fractional shortening Z score .92 [.87, .96] <.001 EDD Z score .89 [.82, .97] .01

  12. 100 90 80 70 60 Death or Transplant-free (%) 50 40 30 Pure HCM: Idiopathic Age ≥1 year (n=320) 20 Pure HCM: Idiopathic Age <1 year (n=220) 10 Pure HCM: Malformation Syndrome (n=49) 0 0 2 4 6 8 10 12 14 Years from Diagnosis Death or Transplant-free Survival by HCM Subgroup

  13. Kaplan-Meier Estimates by HCM Subgroup Idiopathic Age ≥ 1 year Idiopathic Age < 1 year Malformation Syndrome Actual Events/Sample Size (%) 22/320 (7%) 48/220 (22%) 12/49 (25%) Estimated 1-Yr. Death/ Transplant (se) 99% (.005) 81% (.03) 80% (.06) 2-Yr. Death/ Transplant (se) 96% (.01) 77% (.03) 75% (.07)

  14. Death or Transplant-free Survival by HCM Subgroup 100 90 80 70 60 Death or Transplant-free (%) 50 40 30 20 Pure HCM: Errors of Metabolism (n=62) 10 HCM with DCM (n=57) HCM with RCM/Other (n=44) 0 0 2 4 6 8 10 12 14 Years from Diagnosis

  15. Kaplan-Meier Estimates by HCM Subgroup Errors of Metabolism HCM/ DCM HCM/ RCM Actual Events/Sample Size (%) 36/62 (58%) 23/57 (40%) 18/44 (41%) Estimated 1-Yr. Death/ Transplant (se) 49% (.07) 57% (.07) 69% (.07) 2-Yr. Death/ Transplant (se) 39% (.07) 55% (.07) 61% (.08)

  16. Conclusions • Prognosis is worse when pediatric HCM patients present with inborn errors of metabolism or with HCM in combination with other types of CM. • The best outcomes were observed in children presenting at 1 year of age or older with idiopathic HCM.

  17. Conclusions • Increased ED posterior wall thickness, reduced LV fractional shortening, and reduced ED dimension at presentation were associated with a greater risk of subsequent death or transplant. • In subgroups of HCM, reduced BSA and the presence of CHF were also associated with a greater risk of death or transplant.

  18. Conclusions • Female and black children were also at risk for poor outcomes in some subgroups of HCM

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