Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension
Abstract The aim of this single‐centre retrospective observational study was to evaluate the safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension. Children aged over 5...
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| Format: | Article |
| Language: | English |
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Wiley
2024-10-01
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| Series: | Pulmonary Circulation |
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| Online Access: | https://doi.org/10.1002/pul2.70011 |
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| author | Cara Morgan Nikmah Idris Kathy Elterefi Luca Di Ienno Andrew Constantine Sadia Quyam Roberta Bini Shahin Moledina |
| author_facet | Cara Morgan Nikmah Idris Kathy Elterefi Luca Di Ienno Andrew Constantine Sadia Quyam Roberta Bini Shahin Moledina |
| author_sort | Cara Morgan |
| collection | DOAJ |
| description | Abstract The aim of this single‐centre retrospective observational study was to evaluate the safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension. Children aged over 5 years who were established on sildenafil plus bosentan were offered to undergo a therapy switch from May 2014 to May 2021 and, if remaining in the service, followed up to May 2024. Children with Eisenmenger syndrome, open intra or extra‐cardiac shunt, or with pulmonary hypertension‐associated lung disease were excluded. As part of a structured clinical program children were assessed via walk test, echocardiography, cardiac magnetic resonance imaging (CMRI), cardiopulmonary exercise testing, and serum biomarkers. Fifty‐two children were included, 33 in the switch group and 19 in the control group. Clinical characteristics at diagnosis and baseline assessments did not differ between groups. All children tolerated the medication switch. Over a median 13.0 [12.0,13.7] week follow‐up in the switch group there was a significant improvement in World Health Organization functional class (WHO FC, p < 0.001); reduction in estimated right ventricular systolic pressure by echocardiography of 7 mmHg (p = 0.03) and a 2% increase (p = 0.03) in right ventricular ejection fraction on CMRI. There was a sustained improvement in WHO FC (p < 0.01) in the switch group at medium‐term follow‐up of 40.9 [35.2,49.3] weeks. Long‐term outcome of transplant‐ or Potts shunt‐free survival was comparable between the two groups. |
| format | Article |
| id | doaj-art-4f1bf5f9192f4f36b6f0e6c687e8e6bc |
| institution | Kabale University |
| issn | 2045-8940 |
| language | English |
| publishDate | 2024-10-01 |
| publisher | Wiley |
| record_format | Article |
| series | Pulmonary Circulation |
| spelling | doaj-art-4f1bf5f9192f4f36b6f0e6c687e8e6bc2024-12-27T05:54:35ZengWileyPulmonary Circulation2045-89402024-10-01144n/an/a10.1002/pul2.70011Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertensionCara Morgan0Nikmah Idris1Kathy Elterefi2Luca Di Ienno3Andrew Constantine4Sadia Quyam5Roberta Bini6Shahin Moledina7UK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKUK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKUK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKUK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKDepartment of Adult Congenital Cardiology Queen Elizabeth Hospital Birmingham UKUK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKUK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKUK Service for Pulmonary Hypertension in Children, Great Ormond Street Hospital for Children London UKAbstract The aim of this single‐centre retrospective observational study was to evaluate the safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension. Children aged over 5 years who were established on sildenafil plus bosentan were offered to undergo a therapy switch from May 2014 to May 2021 and, if remaining in the service, followed up to May 2024. Children with Eisenmenger syndrome, open intra or extra‐cardiac shunt, or with pulmonary hypertension‐associated lung disease were excluded. As part of a structured clinical program children were assessed via walk test, echocardiography, cardiac magnetic resonance imaging (CMRI), cardiopulmonary exercise testing, and serum biomarkers. Fifty‐two children were included, 33 in the switch group and 19 in the control group. Clinical characteristics at diagnosis and baseline assessments did not differ between groups. All children tolerated the medication switch. Over a median 13.0 [12.0,13.7] week follow‐up in the switch group there was a significant improvement in World Health Organization functional class (WHO FC, p < 0.001); reduction in estimated right ventricular systolic pressure by echocardiography of 7 mmHg (p = 0.03) and a 2% increase (p = 0.03) in right ventricular ejection fraction on CMRI. There was a sustained improvement in WHO FC (p < 0.01) in the switch group at medium‐term follow‐up of 40.9 [35.2,49.3] weeks. Long‐term outcome of transplant‐ or Potts shunt‐free survival was comparable between the two groups.https://doi.org/10.1002/pul2.70011pulmonary artery hypertensionpulmonary hypertensionrare pediatric lung diseasetreatment |
| spellingShingle | Cara Morgan Nikmah Idris Kathy Elterefi Luca Di Ienno Andrew Constantine Sadia Quyam Roberta Bini Shahin Moledina Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension Pulmonary Circulation pulmonary artery hypertension pulmonary hypertension rare pediatric lung disease treatment |
| title | Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension |
| title_full | Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension |
| title_fullStr | Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension |
| title_full_unstemmed | Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension |
| title_short | Safety, tolerability, and efficacy of an in‐class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension |
| title_sort | safety tolerability and efficacy of an in class combination therapy switch from bosentan plus sildenafil to ambrisentan plus tadalafil in children with pulmonary arterial hypertension |
| topic | pulmonary artery hypertension pulmonary hypertension rare pediatric lung disease treatment |
| url | https://doi.org/10.1002/pul2.70011 |
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