1. Sildenafil add-on therapy in paediatric pulmonary arterial hypertension, experiences of a national referral centre.
- Author
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Douwes JM, Roofthooft MT, Van Loon RL, Ploegstra MJ, Bartelds B, Hillege HL, and Berger RM
- Subjects
- Adolescent, Age Factors, Bosentan, Child, Cohort Studies, Drug Therapy, Combination, Familial Primary Pulmonary Hypertension, Female, Humans, Hypertension, Pulmonary diagnosis, Hypertension, Pulmonary mortality, Male, Netherlands, Purines administration & dosage, Secondary Care Centers, Sildenafil Citrate, Treatment Outcome, Antihypertensive Agents administration & dosage, Hypertension, Pulmonary drug therapy, Piperazines administration & dosage, Sulfonamides administration & dosage, Sulfones administration & dosage, Vasodilator Agents administration & dosage
- Abstract
Objective: In paediatric pulmonary arterial hypertension (PAH), the effectiveness of add-on combination PAH-therapy has not yet been systematically studied. The purpose of this study was to determine the effect of sildenafil add-on therapy in paediatric PAH patients treated with bosentan., Methods: In this observational study within a national paediatric patient cohort, follow-up data of 24 consecutive paediatric PAH patients initially treated with bosentan monotherapy and prospectively followed at the Dutch national referral centre for paediatric PAH in 2007-2013, were reviewed. Patients received add-on sildenafil therapy in case of clinical worsening., Results: Fifteen children received add-on sildenafil therapy; nine remained on bosentan monotherapy. Patient characteristics, 6-minute walk distance (6MWD), WHO functional class (WHO-FC), N-terminal pro-Brain Natriuretic Peptide (NT-proBNP), and haemodynamic measurements at bosentan start were similar in both patient groups. In children with clinical worsening, sildenafil add-on therapy improved 6MWD at 5, 10, 15 and 21 months of follow-up, improved WHO-FC at 10, 15 and 21 months and stabilised NT-proBNP. Patients who received add-on sildenafil therapy had more advanced disease progression during bosentan monotherapy. Despite that, they had better or, at least, no worse survival compared to patients who remained on bosentan monotherapy., Conclusions: In children with PAH, sildenafil add-on therapy indicated by clinical deterioration on bosentan monotherapy, was associated with a significant improvement of WHO-functional class and 6MWD. Despite clinical deterioration on bosentan monotherapy, patients receiving sildenafil add-on therapy, had either better or, at least, no worse survival than patients remaining on bosentan monotherapy.
- Published
- 2014
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