Jennifer L Cohen1, Shannon N Nees1, Gerson A Valencia1, Erika B Rosenzweig1, Usha S Krishnan2. 1. Division of Pediatric Cardiology, Department of Pediatrics, Columbia University Vagelos College of Physicians and Surgeons, New York Presbyterian Hospital, New York, NY. 2. Division of Pediatric Cardiology, Department of Pediatrics, Columbia University Vagelos College of Physicians and Surgeons, New York Presbyterian Hospital, New York, NY. Electronic address: usk1@cumc.columbia.edu.
Abstract
OBJECTIVE: To assess the demographics, treatment algorithm, and outcomes in a large cohort of children treated with sildenafil. STUDY DESIGN: A retrospective cohort study of children with pulmonary hypertension (PH) treated with sildenafil at a single institution between 2004 and 2015. Baseline and follow-up data collected by chart review. RESULTS: There were 269 children included in this study: 47 with idiopathic pulmonary arterial hypertension, 53 with congenital heart disease, 135 with bronchopulmonary dysplasia, 24 with congenital diaphragmatic hernia, and 7 with other causes. Sildenafil was initial monotherapy in 84.8% and add-on therapy in 15.2%. Median follow-up time was 3.1 years (2 weeks-12.4 years). On follow-up, 99 (37%) remained on sildenafil or transitioned to tadalafil, 93 (35%) stopped sildenafil for improvement in PH, 54 (20%) died, and 20 (7%) were lost to follow-up. PH was most likely to improve in those with bronchopulmonary dysplasia, allowing for the discontinuation of sildenafil in 45%. Eighteen deaths were related to PH and 36 from other systemic causes. Two patients stopped sildenafil owing to airway spasm with desaturation. Overall survival was significantly lower in World Health Organization group 3 PH (bronchopulmonary dysplasia and congenital diaphragmatic hernia) vs group 1 (idiopathic pulmonary arterial hypertension and congenital heart disease), P = .02. CONCLUSIONS: In this retrospective experience in children with mainly World Health Organization groups 1 and 3 PH, low-dose sildenafil was well-tolerated, safe, and had an acceptable side effect profile. Although patients with group 3 PH have high mortality, survivors have a high likelihood of PH improving. Published by Elsevier Inc.
OBJECTIVE: To assess the demographics, treatment algorithm, and outcomes in a large cohort of children treated with sildenafil. STUDY DESIGN: A retrospective cohort study of children with pulmonary hypertension (PH) treated with sildenafil at a single institution between 2004 and 2015. Baseline and follow-up data collected by chart review. RESULTS: There were 269 children included in this study: 47 with idiopathic pulmonary arterial hypertension, 53 with congenital heart disease, 135 with bronchopulmonary dysplasia, 24 with congenital diaphragmatic hernia, and 7 with other causes. Sildenafil was initial monotherapy in 84.8% and add-on therapy in 15.2%. Median follow-up time was 3.1 years (2 weeks-12.4 years). On follow-up, 99 (37%) remained on sildenafil or transitioned to tadalafil, 93 (35%) stopped sildenafil for improvement in PH, 54 (20%) died, and 20 (7%) were lost to follow-up. PH was most likely to improve in those with bronchopulmonary dysplasia, allowing for the discontinuation of sildenafil in 45%. Eighteen deaths were related to PH and 36 from other systemic causes. Two patients stopped sildenafil owing to airway spasm with desaturation. Overall survival was significantly lower in World Health Organization group 3 PH (bronchopulmonary dysplasia and congenital diaphragmatic hernia) vs group 1 (idiopathic pulmonary arterial hypertension and congenital heart disease), P = .02. CONCLUSIONS: In this retrospective experience in children with mainly World Health Organization groups 1 and 3 PH, low-dose sildenafil was well-tolerated, safe, and had an acceptable side effect profile. Although patients with group 3 PH have high mortality, survivors have a high likelihood of PH improving. Published by Elsevier Inc.
Authors: Christian Apitz; Janette T Reyes; Helen Holtby; Tilman Humpl; Andrew N Redington Journal: J Am Coll Cardiol Date: 2010-04-06 Impact factor: 24.094
Authors: Hyo Soon An; Eun Jung Bae; Gi Beom Kim; Bo Sang Kwon; Jae Suk Beak; Ee Kyung Kim; Han Suk Kim; Jung-Hwan Choi; Chung Il Noh; Yong Soo Yun Journal: Korean Circ J Date: 2010-03-24 Impact factor: 3.243
Authors: E Spaggiari; J J Stirnemann; P Sonigo; N Khen-Dunlop; L De Saint Blanquat; Y Ville Journal: Ultrasound Obstet Gynecol Date: 2015-05 Impact factor: 7.299
Authors: Paul J Critser; Nara S Higano; Jean A Tkach; Emilia S Olson; David R Spielberg; Paul S Kingma; Robert J Fleck; Sean M Lang; Ryan A Moore; Michael D Taylor; Jason C Woods Journal: Am J Respir Crit Care Med Date: 2020-01-01 Impact factor: 21.405
Authors: Paul J Critser; Nara S Higano; Sean M Lang; Paul S Kingma; Robert J Fleck; Russel Hirsch; Michael D Taylor; Jason C Woods Journal: J Cardiovasc Magn Reson Date: 2020-07-23 Impact factor: 5.364
Authors: Dunbar Ivy; Damien Bonnet; Rolf M F Berger; Gisela M B Meyer; Simin Baygani; Baohui Li Journal: Pulm Circ Date: 2021-06-23 Impact factor: 3.017