Visser, M. de (Marjanne) (2018) Clinical and echocardiographic assessment of treatment initiation and add-on therapy in pediatric pulmonary arterial hypertension. thesis, Medicine.
Full text available on request.Abstract
Introduction: To evaluate treatment effect in children with pulmonary arterial hypertension (PAH) echocardiography is routinely done. However, research on treatment-induced echocardiographic changes is limited. The aim of this study was to see whether PAH-targeted therapy can improve both the clinical status and the echocardiographic parameters in patients after treatment initiation. Besides, the effect of add-on therapy on these parameters was studied. Materials and methods: Data of patients from the prospective clinical registry of the Dutch National Network for Pediatric Pulmonary Hypertension were studied. Both clinical and echocardiographic parameters were studied. The clinical parameters included heartrate, World Health Organization functional class (WHO-FC), 6-minute-walk-distance (6MWD), N-terminal pro-B-type natriuretic peptide (NT-proBNP), uric acid and creatinine. The investigated echocardiographic variables were predefined parameters from the standard echocardiographic protocol of the University Medical Center Groningen (UMCG) with some additional recently suggested predictive variables. Both the clinical and echocardiographic parameters were collected and assessed before and after treatment initiation and before and after add-on therapy. The treatment induced changes were studied in the total PAH-group and for patients with idiopathic or hereditary PAH (IPAH/HPAH) and patients with PAH associated with congenital heart disease (PAH-CHD) separately. Results: Seventy-eight treatment naïve patients started on treatment, thirty-eight patients started first add-on therapy and fourteen patients started second add-on therapy. Significant improvements were seen with treatment initiation for the clinical parameters WHO-FC, 6MWD, 6MWD Z-score and NT-proBNP and for echocardiographic left ventricle (LV) dimensions and right ventricle (RV) functional parameters (p<0.05). Add-on therapy showed no improvement. The clinical parameter 6MWD and the echocardiographic LV and RV dimensions even worsened. Conclusion: The initiation of PAH-targeted therapy can improve the clinical status and echocardiographic parameters, especially the LV parameters, of treatment naïve pediatric PAH-patients. This study suggests that the clinical and echocardiographic parameters could not be improved by add-on therapy, because no improvement was seen, but rather a worsening in the clinical parameters and echocardiographic LV and RV dimensions.
| Item Type: | Thesis (Thesis) |
|---|---|
| Supervisor name: | Faculty supervisor: and Berger, prof. R.M.F. and Second supervisor: and Haarman, M.G. MSc and University Medical Center Groningen; Beatrix Children’s Hosp |
| Faculty: | Medical Sciences |
| Date Deposited: | 25 Jun 2020 10:53 |
| Last Modified: | 25 Jun 2020 10:53 |
| URI: | https://umcg.studenttheses.ub.rug.nl/id/eprint/1403 |
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