Related Experiment Video
Updated: Apr 8, 2026

Increasing Pulmonary Artery Pulsatile Flow Improves Hypoxic Pulmonary Hypertension in Piglets
Published on: May 11, 2015
Sildenafil in pediatric pulmonary arterial hypertension
1Department of Pediatrics, Topiwala National Medical College and BYL Nair Charitable Hospital, Mumbai, Maharashtra, India.
Insights
Pulmonary arterial hypertension (PAH) treatment in children faces challenges due to a USFDA warning on sildenafil. This review examines sildenafil
Area of Science:
- Pediatric Cardiology
- Pulmonology
- Pharmacology
Background:
- Pulmonary arterial hypertension (PAH) is a severe condition with limited curative options.
- Advances in understanding PAH pathophysiology and diagnosis have improved patient outlook.
- Sildenafil is a widely used off-label drug for pediatric PAH management.
Purpose of the Study:
- To review the use of sildenafil in treating pediatric pulmonary arterial hypertension.
- To address the implications of the 2012 USFDA warning on sildenafil use in children with PAH.
Main Methods:
- Literature review of studies on sildenafil for pediatric PAH.
- Analysis of regulatory guidance and clinical practice trends.
- Examination of the efficacy and safety data of sildenafil in children.
Main Results:
- Sildenafil has been extensively used off-label for pediatric PAH.
- A 2012 USFDA warning discouraged high-dose sildenafil use in children with PAH.
- This has created a therapeutic gap, with limited approved treatments.
Conclusions:
- The use of sildenafil in pediatric PAH requires careful consideration due to regulatory concerns.
- Further research and approved therapies are needed for effective pediatric PAH management.
- This review highlights the complexities of managing pediatric PAH with available, albeit restricted, therapeutic options.
Abstract:
Pulmonary arterial hypertension (PAH) is a life-threatening disease of varied etiologies. Although PAH has no curative treatment, a greater understanding of pathophysiology, technological advances resulting in early diagnosis, and the availability of several newer drugs have improved the outlook for patients with PAH. Sildenafil is one of the therapeutic agents used extensively in the treatment of PAH in children, as an off-label drug. In 2012, the United States Food and Drug Administration (USFDA) issued a warning regarding the of use high-dose sildenafil in children with PAH. This has led to a peculiar situation where there is a paucity of approved therapies for the management of PAH in children and the use of the most extensively used drug being discouraged by the regulator. This article provides a review of the use of sildenafil in the treatment of PAH in children.
More Related Videos
Related Concept Videos
Treatment for Pulmonary Arterial Hypertension: Phosphodiesterase Inhibitors
Among the PDE5 inhibitors, sildenafil (Revatio) stands out as a competitive and selective inhibitor. It operates by elevating cellular levels of cGMP and augmenting signaling through the cGMP-PKG pathway, promoting vasodilation. Upon oral...
Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
Pulmonary Hypertension: Classification and Pathogenesis
There are various classifications for PH, each relating to different underlying causes and also...
Pharmacokinetics in Pediatric Patients: Drug Metabolism
Pharmacokinetics in Pediatric Patients: Drug Excretion

