1. Clinical Features and Ultrasound Parameters in Pulmonary Arterial Hypertension, in Pediatric Cardiology Author: Gáspár Hanga 1 Scientific coordinators: Prof. Dr. Rodica Tog ă nel Dr. Carmen uteu

2. Introduction 2 Mean pulmonary artery pressure at rest mPAP > 25 mmHg Pulmonary vascular resistance PVR > 3 Wood units (or 240 dyness/s/cm5) Pulmonary capillary wedge pressure PCWP < 15 mmHg Mean pulmonary artery pressure at rest mPAP > 25 mmHg Pulmonary vascular resistance PVR > 3 Wood units (or 240 dyness/s/cm5) Pulmonary capillary wedge pressure PCWP < 15 mmHg IntroductionObjective Material and method ResultsConclusion Incidence: 1-2/ 1.000.000 Progressive disease Vascular remodeling with luminal obliteration of small vessels Incidence: 1-2/ 1.000.000 Progressive disease Vascular remodeling with luminal obliteration of small vessels Pulmonary vasoconstriction and thrombosis (endothelial dysfunction: imbalance favoring vasoconstriction) Pulmonary vascular inflammation and remodelling: intimal proliferation, medial thickening Injury to endothelial cells Right ventricular hypertrophy (compensatory) Pulmonary vasoconstriction and thrombosis (endothelial dysfunction: imbalance favoring vasoconstriction) Pulmonary vascular inflammation and remodelling: intimal proliferation, medial thickening Injury to endothelial cells Right ventricular hypertrophy (compensatory)

3. Objective 3 IntroductionObjective Material and method ResultsConclusion Evaluation and 1-year follow-up of clinical manifestations and ultrasound parameters of children suffering from PAH, receiving Bosentan, Sildenafil or combination therapy.

4. IntroductionObjective Material and method ResultsConclusion Period January 2008 -March 2013 41 children enrolled 21 from Târgu Mure Emergency Institute for Cardiovascular Diseases and Transplantation 20 from the National Institute of Cardiology Gottsegen György, Budapest Gender distribution 22 male:19 female 53.7: 46.3 (%) Material and method Age children aged between 4 months-15 years mean age: 5.16 years

5. Material and method 5 IntroductionObjective Material and method ResultsConclusion symptoms functional class results of the six-minute walk test heart ultrasound parameters Doppler echocardiographyRight heart catheterization Acute vasodilator test Diagnosis of PAH Assessed at enrollment and one year layer:

6. Clinical classification* based on aethiology 6 IntroductionObjective Material and method ResultsConclusion 4 groups created based on the causative disease * according to the Dana Point classification

7. Disease targeted therapy 7 IntroductionObjective Material and method ResultsConclusion

8. Results 8 IntroductionObjective Material and method ResultsConclusion Clinical symptoms

9. Functional class 9 IntroductionObjective Material and method ResultsConclusion right- and biventricular hypertrophy had developed in 1-1 patient respectively 1 patient developed congestive cardiac insufficiency no syncopal episodes Functional classification is strongly predictive of mortality, and is an important factor in the choice of PAH therapy.

10. 6-Minute Walk Test 10 IntroductionObjective Material and method ResultsConclusion

11. Heart ultrasound 11 IntroductionObjective Material and method ResultsConclusion The increment or decrement of mPAP values in first year of therapy Mean pulmonary artery pressure improved in the BPD and the Eisenmenger groups worsened in the post-operative PAH and iPAH group Mean pulmonary artery pressure - a major diagnostic criteria Mean pulmonary artery pressure - a major diagnostic criteria iPAH Post-op ESBPD

12. Tricuspid Annular Plane Systolic Excursion 12 IntroductionObjective Material and method ResultsConclusion TAPSE (mm) Increment or decrement in TAPSE values (%) TAPSE –a useful and easily measured parameter in the evaluation of the right ventricle

13. Parameters with high prognostic value 13 IntroductionObjective Material and method ResultsConclusion Right atrial area increased in all subgroups Right atrial area (cm 2 ) Right atrial area enlargement (%)

14. Parameters with high prognostic value 14 IntroductionObjective Material and method ResultsConclusion The relative presence of pericardial effusion in each subgroup iPAH: 1 patient, showing a worsening tendency Post-op PAH: 3 patients, with stable or improving tendencies Eisenmenger: 2 patients, 1 improving, 1 worsening BPD: absent iPAH: 1 patient, showing a worsening tendency Post-op PAH: 3 patients, with stable or improving tendencies Eisenmenger: 2 patients, 1 improving, 1 worsening BPD: absent

15. Conclusions 15 IntroductionObjective Material and method ResultsConclusion Bosentan and Sildenafil are efficient in improving: life quality functional status (improvement in NYHA class) exercise capacity (significant increment in distance walked) But do not significantly improve cardiac status: right atrial area increased in all groups pericardial effusion, if present, persisted or worsened left ventricular eccentricity index did not present significant changes

16. Conclusions 16 IntroductionObjective Material and method ResultsConclusion despite their serious condition, PAH patients with bronchopulmonary dysplasia displayed improvement of most ultrasound parameters BPD group patients presented lowering tendencies only in pulmonary pressures Post-operative PAH group Differences in disease progression can be noted.

17. Conclusions 17 IntroductionObjective Material and method ResultsConclusion increased saturation and less pronounced drop during the walk test increasing TAPSE values, opposed to the decreasing tendency noted in iPAH prognosis is not as poor as in iPAH Eisenmenger patients react better to treatment than those suffering from idiopathic PAH:

18. References 18  Professor Michael A. Gatzoulis -Pulmonary Arterial Hypertension, Oxford University Press, Great Britain, 2012  Robyn J. Barst –Pulmonary Arterial Hypertension: Diagnosis and Evidence-Based Treatment, Great Britain, 2008  ATS statement: guidelines for the six-minute walk test, Am J Respir Crit Care Med, 2002, 166: 111-7.  Simonneau G, Robbins I, Beghetti M, et al. Updated clinical classification of pulmonary hypertension. J Am Coll Cardiol 2009;54:S43-S54.  Rubin LJ. Primary pulmonary hypertension. N Engl J Med 1997; 336: 111-7  Shafazand S, et al. Health-related quality of life in patients with pulmonary arterial hypertension. Chest 2004; 126: 1452-9.  McLaughlin VV, et.al. Prognosis of pulmonary arterial hypertension. Chest. 2004;126:78S-92S  Badesch DB, Abman SH, et.al. Medical therapy for pulmonary arterial hypertension. Chest. 2004;126:35S-62S  Professor Michael A. Gatzoulis -Pulmonary Arterial Hypertension, Oxford University Press, Great Britain, 2012  Robyn J. Barst –Pulmonary Arterial Hypertension: Diagnosis and Evidence-Based Treatment, Great Britain, 2008  ATS statement: guidelines for the six-minute walk test, Am J Respir Crit Care Med, 2002, 166: 111-7.  Simonneau G, Robbins I, Beghetti M, et al. Updated clinical classification of pulmonary hypertension. J Am Coll Cardiol 2009;54:S43-S54.  Rubin LJ. Primary pulmonary hypertension. N Engl J Med 1997; 336: 111-7  Shafazand S, et al. Health-related quality of life in patients with pulmonary arterial hypertension. Chest 2004; 126: 1452-9.  McLaughlin VV, et.al. Prognosis of pulmonary arterial hypertension. Chest. 2004;126:78S-92S  Badesch DB, Abman SH, et.al. Medical therapy for pulmonary arterial hypertension. Chest. 2004;126:35S-62S  Initiative to expand patient spectrum  Data gathering and processing  Assisting patient follow-up  Statistics  Presentation  Initiative to expand patient spectrum  Data gathering and processing  Assisting patient follow-up  Statistics  Presentation

19. 19 Thank you for your kind attention!