PMID- 33547769 OWN - NLM STAT- MEDLINE DCOM- 20210701 LR - 20210701 IS - 2055-5822 (Electronic) IS - 2055-5822 (Linking) VI - 8 IP - 2 DP - 2021 Apr TI - Predicting outcome in children with dilated cardiomyopathy: the use of repeated measurements of risk factors for outcome. PG - 1472-1481 LID - 10.1002/ehf2.13233 [doi] AB - AIMS: We aimed to determine whether in children with dilated cardiomyopathy repeated measurement of known risk factors for death or heart transplantation (HTx) during disease progression can identify children at the highest risk for adverse outcome. METHODS AND RESULTS: Of 137 children we included in a prospective cohort, 36 (26%) reached the study endpoint (SE: all-cause death or HTx), 15 (11%) died at a median of 0.09 years [inter-quartile range (IQR) 0.03-0.7] after diagnosis, and 21 (15%) underwent HTx at a median of 2.9 years [IQR 0.8-6.1] after diagnosis. Median follow-up was 2.1 years [IQR 0.8-4.3]. Twenty-three children recovered at a median of 0.6 years [IQR 0.5-1.4] after diagnosis, and 78 children had ongoing disease at the end of the study. Children who reached the SE could be distinguished from those who did not, based on the temporal evolution of four risk factors: stunting of length growth (-0.42 vs. -0.02 length Z-score per year, P < 0.001), less decrease in N-terminal pro-B-type natriuretic peptide (NT-proBNP) (-0.26 vs. -1.06 2log pg/mL/year, P < 0.01), no decrease in left ventricular internal diastolic dimension (LVIDd; 0.24 vs. -0.60 Boston Z-score per year, P < 0.01), and increase in New York University Pediatric Heart Failure Index (NYU PHFI; 0.49 vs. -1.16 per year, P < 0.001). When we compared children who reached the SE with those with ongoing disease (leaving out the children who recovered), we found similar results, although the effects were smaller. In univariate analysis, NT-proBNP, length Z-score, LVIDd Z-score, global longitudinal strain (%), NYU PHFI, and age >6 years at presentation (all P < 0.001) were predictive of adverse outcome. In multivariate analysis, NT-proBNP appeared the only independent predictor for adverse outcome, a two-fold higher NT-proBNP was associated with a 2.8 times higher risk of the SE (hazard ratio 2.78, 95% confidence interval 1.81-3.94, P < 0.001). CONCLUSIONS: The evolution over time of NT-proBNP, LVIDd, length growth, and NYU PHFI identified a subgroup of children with dilated cardiomyopathy at high risk for adverse outcome. In this sample, with a limited number of endpoints, NT-proBNP was the strongest independent predictor for adverse outcome. CI - (c) 2021 The Authors. ESC Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology. FAU - van der Meulen, Marijke AU - van der Meulen M AD - Department of Pediatric Cardiology, Erasmus MC, University Medical Center Rotterdam, Dr Molewaterplein 60, PO Box 2060, Rotterdam, 3000 CB, The Netherlands. FAU - den Boer, Susanna AU - den Boer S AD - Department of Pediatric Cardiology, Erasmus MC, University Medical Center Rotterdam, Dr Molewaterplein 60, PO Box 2060, Rotterdam, 3000 CB, The Netherlands. FAU - du Marchie Sarvaas, Gideon J AU - du Marchie Sarvaas GJ AD - Department of Pediatric Cardiology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands. FAU - Blom, Nico AU - Blom N AD - Leiden University Medical Center, Department of Pediatric Cardiology, University of Leiden, Leiden, The Netherlands. AD - Academic Medical Center, Department of Pediatric Cardiology, University of Amsterdam, Amsterdam, The Netherlands. FAU - Ten Harkel, Arend D J AU - Ten Harkel ADJ AD - Leiden University Medical Center, Department of Pediatric Cardiology, University of Leiden, Leiden, The Netherlands. FAU - Breur, Hans M P J AU - Breur HMPJ AD - Department of Pediatric Cardiology, University of Utrecht, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands. FAU - Rammeloo, Lukas A J AU - Rammeloo LAJ AD - Department of Pediatric Cardiology, Free University of Amsterdam, Free University Medical Center, Amsterdam, The Netherlands. FAU - Tanke, Ronald AU - Tanke R AD - Department of Pediatric Cardiology, Radboud University Medical Center, Nijmegen, The Netherlands. FAU - Bogers, Ad J J C AU - Bogers AJJC AD - Department of Cardiothoracic Surgery, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands. FAU - Helbing, Willem A AU - Helbing WA AD - Department of Pediatric Cardiology, Erasmus MC, University Medical Center Rotterdam, Dr Molewaterplein 60, PO Box 2060, Rotterdam, 3000 CB, The Netherlands. AD - Department of Pediatric Cardiology, Radboud University Medical Center, Nijmegen, The Netherlands. FAU - Boersma, Eric AU - Boersma E AD - Department of Cardiology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands. FAU - Dalinghaus, Michiel AU - Dalinghaus M AD - Department of Pediatric Cardiology, Erasmus MC, University Medical Center Rotterdam, Dr Molewaterplein 60, PO Box 2060, Rotterdam, 3000 CB, The Netherlands. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20210205 PL - England TA - ESC Heart Fail JT - ESC heart failure JID - 101669191 RN - 0 (Biomarkers) RN - 0 (Peptide Fragments) RN - 114471-18-0 (Natriuretic Peptide, Brain) SB - IM MH - Biomarkers MH - *Cardiomyopathy, Dilated/diagnosis/epidemiology MH - Child MH - *Heart Failure/epidemiology MH - Humans MH - Natriuretic Peptide, Brain MH - Peptide Fragments MH - Prospective Studies MH - Risk Factors PMC - PMC8006605 OTO - NOTNLM OT - Dilated cardiomyopathy OT - Paediatric cardiology OT - Risk factors COIS- None declared. EDAT- 2021/02/07 06:00 MHDA- 2021/07/02 06:00 PMCR- 2021/02/05 CRDT- 2021/02/06 05:48 PHST- 2021/01/18 00:00 [revised] PHST- 2020/02/21 00:00 [received] PHST- 2021/01/23 00:00 [accepted] PHST- 2021/02/07 06:00 [pubmed] PHST- 2021/07/02 06:00 [medline] PHST- 2021/02/06 05:48 [entrez] PHST- 2021/02/05 00:00 [pmc-release] AID - EHF213233 [pii] AID - 10.1002/ehf2.13233 [doi] PST - ppublish SO - ESC Heart Fail. 2021 Apr;8(2):1472-1481. doi: 10.1002/ehf2.13233. Epub 2021 Feb 5.