3/8/2019 Epidemiology, Risk Factors, and Outcomes of Pediatric PVD: • LESSONS learned from the Spanish Registry: Lessons Learned from the Registries o Is It possible (and worthy) to do a national registry? Lessons learned from the o Epidemiology. SPANISH REGISTRY OF PEDIATRIC PULMONARY HYPERTENSION o Phenotyping : Clinical characterization & Comorbidities o Use of Diagnostic Tests in Pediatrics PULMONARY CIRCULATION WORKING GROUP o Outcomes and risk factors. SECPCC o Use of targeted therapies. Maria Jesus del Cerro Ramón y Cajal University Hospital • Tools for research . Madrid, Spain Started: January 2010 • LESSONS LEARNED: After 9 years… - Participation of 28 centers o Is It possible to do a national registry in a - Inclusion of 568 patients country witouth centralized PH care? Financiation: unrestricted grants from Pharmaceutical companies - Problems to face: • Dispersion of patients in 22 centers with cardiac 2010 2011 2011 2013 2014 2015 2016 2017 2018 2019 surgery program…!!!! 70.000 € 48.000 35.000 27.700 22.700 18.435 18.000 18.000 18.000 18.000 • Intellectual property Intellectual property (promotor) • Sponsorship 1
3/8/2019 568 PATIENTS • LESSONS LEARNED: • Design of the registry… - what kind of patients should we include? - Just PAH? - starting point…? Centros con 2 pacientes incluidos (0,34%) Centros con 1 paciente incluido (0,17%) Jan 2009 Jan 2011 Jan 2013 Jan 2015 Jan 2017 385 patients Jan 2014 Survival from diagnosis Jan 2010 Jan 2012 Jan 2016 Jan 2018 PULMONARY HYPERTENSION Biventricular Circulation Diagnosed before 2009 2009 2019 Diagnosed after 2009 Historical (n= 398) 70% CASES: Diagnosed after January 2009 (Dx and finished before 2009) CASES Diagnosed before 2009 25% (n= 141) 25% HISTORICAL CASES: Diagnosed and Finished (Exitus /tx) before 2009 5% 70% (n= 27) 5% 2
3/8/2019 Etiologic Group Nice 385 patients Inclusion of patients 2009-2018 Classification PULMONARY HYPERTENSION Biventricular Circulation PH biventricular Univentricular P 600 500 400 300 Lung Disease 200 Left Heart D PAH 100 0 2010 2011 2012 2013 2014 2015 2016 2017 2018 Data: January 2019 NATIONAL REGISTRIES • LESSONS LEARNED: o Epidemiology of Pediatric PH and Comparison with Adults Epidemiology Cerro et al. AJRCCM 2014 3
3/8/2019 NATIONAL REGISTRIES SPAIN UK FRANCE Netherands Ajrccm 2014 Heart 2009 Arch Cardiov Dis 2010 Circulation 2011 nº pts PH 264 216 50 3263/ 572 Pulmonary 156 161 154 50 Nº pts PAH ARTERIAL Hypertension 2.56 3.7 3 PAH incidence 2,8% 3,8% 1,7% 4,7% IDIOPATHIC 14 20 PAH prevalence 7,0% IDIOPATHIC 2% 36% 6,2% 15% ) 0.49 0.48 0.7 IPAH Incidence 19% 2.9 2.07 2.2 4.4 IPAH prevalenc. 19% CONNECTIVE 82% TISSUE DISEASE 18,0% %Idiopathic connective TD CONGENITAL HEART DEFECT CHD HIV Portal hypert TOS Venoclus Other 2 or more etiology REHIPED REHAP PEDIATRIC REGISTRY ADULT REGISTRY SPANISH SPANISH PEDIATRIC REGISTRY ADULT REGISTRY eisenmenger. 23% MULTIFACTORIAL 20% 31% 2.8% small defect, 33% Etiology high PVR 5% big defect, high 68% 10% 5% PVR ph after defect 33% closure Abstract presented at the 6th Pediatric Cardiology World Congress, Cape Town 2013 4
3/8/2019 385 patients • Lessons learned: PULMONARY HYPERTENSION in Biventricular Circulation mPAP > 25 mmHg o Phenotyping : and PVRI > 3 WU.m 2 o Clinical characterization & Comorbidities N= 385 (67,8%) Data: January 2019 385 patients 385 patients AGE at diagnosis PULMONARY HYPERTENSION PULMONARY HYPERTENSION Biventricular Circulation Biventricular Circulation Mean: 3,60 + 4,58 years GENDER Male 196 189 (50,9%) (49,1%) Female 5
3/8/2019 Etiologic Group Nice 385 patients AJRCCM 2014 Classification PULMONARY HYPERTENSION Biventricular Circulation COMORBIDITIES % Lung Disease % Left Heart D PAH % % 385 patients PULMONARY HYPERTENSION Biventricular Circulation PREMATURITY AND PULMONARY HYPERTENSION CHROMOSOMOPATHY In 2012: 21,8% Prematurity < 34 weeks GA PERSONAL HISTORY 2018 6
3/8/2019 169 patients 240 patients Congenital Heart Disease - PAH P ULMONARY A RTERIAL H YPERTENSION GROUP I GROUP III 42 patients 82 patients GROUP II PULMONARY HYPERTENSION PH LUNG DISEASE / HYPOXEMIA LEFT HEART DISEASES Bronchopulmonary Dysplasia (n=43; 52%) Other Developmental Lung Disorders (n=27; 33%) Cardiomyopathies (n=16; 38%) Chronic Obstructive Pulmonary Disease (n=6; 7%) Mitral / aortic valve disease (n=22; 52%) Interstitial Lung disease (n=13; 16%) OSAS (n=4; 5%) Pulmonary vein stenosis (n=9; 21%) Cystic fibrosis (n=1; 1%) 7
3/8/2019 2 patients 19 patients GROUP IV GROUP V PULMONARY HYPERTENSION PULMONARY HYPERTENSION CHRONIC THROMBOEMBOLIC DISEASE “UNKNOWN” ETIOLOGY Metabolic Diseases (n=19, 79%) There are 2 Patients with other etiology who have Chronic Thromboembolic Disease, –Thyroid disorders (n=1) 1 of group I and 1 of group II. –Non-ketotic hyperglycinemia (n=12) –Mitochondrial diseases (n=8) CTEPH prevalence: –NFU Gene Mutation (n=2) 0.36 cases/million younger than 18 years –Lipoic Acid deficit (n=1) Autosomal inheritance diseases –Neonatal Hemochromatosis (n=1) High lethality Correct diagnosis of the affected child allows prenatal diagnosis. ADULT REGISTRY CTEPH prevalence: 3.2 cases/million 385 patients Time PULMONARY HYPERTENSION Biventricular Circulation interval Lessons learned … (months) between symptoms and - Are we achieving early diagnosis? PH - Use of Diagnostic Tests diagnosis > 18 years 2,6 + 5,4 YEARS in adult population with HP of I and IV etiology groups. 8
3/8/2019 385 patients 385 patients FUNCTIONAL CLASS (NYHA) DIAGNOSTIC TESTs PULMONARY HYPERTENSION PULMONARY HYPERTENSION at diagnosis Biventricular Circulation Biventricular Circulation 52,7% CHEST 94,3% synus rythm CT / MRI 50% PATIENTS 12,5% PULMONARY diagnosed in HYPOPLASIA FC III/IV 11,9% PULMONARY VEIN STENOSIS 80,0% 17,7% 2012: Abnormal chest X-ray V/Q LUNG SCAN 6,2% TOPP registry Pediatric Registries: 385 patients Use of Beguetti M, et al Eur Respir J 2013; 42: 689–700 PULMONARY HYPERTENSION Biventricular Circulation Diagnostic Tests CATHETERIZATION DATA TOPP REHIPED ERJ 2013 AJRCCM 2014 Lung Function Tests 27% 16% Lung Scintigraphy 23% 24% Overnight O2 Sat 28% Polisomnography 15% CT scan 41% 52% CT/MRI MRI 9% 52% CT/MRI CPET 7% 2012: pts catheterized Cerro Mj, et al. AJRCCM 2014 9
3/8/2019 480 catheterizations 480 catheterizations in 326 patients Acute Vasoreactivity TEST 326 patients PULMONARY HYPERTENSION in 277 catheterizations (58%) Biventricular circulation PULMONARY HYPERTENSION Biventricular circulation CATHETERIZATION COMPLICATIONS 2019: 326 pts 2012: 195 pts SEVERE SEVERE 2012 reported in AMRCC 2014 COMPLICATIONS COMPLICATIONS (4,6%) (5,1%) EXITUS EXITUS 3 (1,5%) (0,8%) PEDIATRIC PHVD REGISTRIES CATHETERIZATION 480 catheterizations in 326 patients SPAIN UK FRANCE REVEAL (USA) Dutch TOPP PULMONARY HYPERTENSION AJRCCM 2014 CONDITIONS Circulation 2011 Heart 2009 Arch Cardiov Dis Circulation Lancet 2012 Biventricular circulation 2010 2011 JACC 2016 nº pts PH 264 216 50 216 3263/ 356 572 nº pts PAH 161 154 50 317 156 % IPAH 6% 7.4% 11% 19% Sitbon 15% Sitbon Responders Barst or Sitbon 35% Barst 30% Barst PAH criteria Prospective 45 % 30% 14% 30% patients DIFFERENCES IN THE WAY THE Catheterization 8% sedation 50% sedation conditions 87% general 50% general VASOREACTIVITITY TESTING WAS DONE? anesthesia anesthesia TOPP: “There were significantly more responders in the patients tested under procedural sedation compared to patients under general anesthesia.” 10
3/8/2019 268 patients catheterized at diagnosis 268 patients catheterized at diagnosis PULMONARY HYPERTENSION PULMONARY HYPERTENSION Biventricular circulation Biventricular circulation HEMODYNAMIC DATA HEMODYNAMIC DATA AND ETHIOLOGY PEDIATRIC REGISTRY Systolic Pulmonary CI mPAP PWP PVR 81,9 ± 27,3 pressure/Systolic Aortic pressure (l/min/m 2 ) (mmHg) (mmHg) (WU.m2) ADULT REGISTRY Mean Pulmonary Pressure GRUPO I 3,76 46,6 11,34 84,7% 13,39 82% 44,4 ± 16,8 49,3 + 15,1 (MPAP) GRUPO II 4,50 41,8 18,57 80,4% 10,32 73% Cardiac Index 4,2 ± 1,9 2,5 + 0,8 PVRI 12,4 ± 9,5 GRUPO III 5,21 35,8 11,28 66,3% 9,21 66% 11.5 + 7,7 PVRI/ SVRI 0,78 ± 0,50 GRUPO IV 2,40 37,00 6,00 85,2% 26,87 59% 9,2 ± 8,0 RAP GRUPO V 5,90 45,0 8,50 93,6% 9,89 82% PWP 12,0 ± 6,0 Grad.Transpulmonar 33,0 ± 17,1 Assesing Pulmonary Hypertensive Vascular Disease in Childhood: Data from the Spanish Registry Am J resp crit care med 2014 • LESSONS LEARNED: Heart 2008 o Outcomes and risk factors . GROUP III LUNG DISEASE Data about management & survival of PHVD in lung disease, left heart disease, … Are still needed 11
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