RESUMEN
A patient with alveolar capillary dysplasia has survived more than 56 months with medical therapy. Intrauterine exposure to metformin potentially modified the severity of disease. In combination with other agents, endothelin receptor antagonists and amlodipine have been key medications in lowering pulmonary arterial pressure and managing right heart failure. (Level of Difficulty: Beginner.).
RESUMEN
The pulmonary arterial pressure of a child with severe pulmonary arterial hypertension immediately normalized while breathing nitric oxide during heart catheterization at 8 years of age. Her acute pulmonary vascular response to nitric oxide has persisted throughout her life. Her acute response to other medications has been similar to her long-term response to medications in the same class. Acute vasodilator testing with inhaled nitric oxide and other medications may be an opportunity to refine study design and advance precision care for patients with pulmonary hypertension.
RESUMEN
Pulmonary arterial hypertension is a progressive, incurable disease that occurs in adults and children alike. Therapeutic options for children are limited and infrequently described, including newer agents such as treprostinil, an oral prostanoid. Herein, we describe the pooled pediatric experience in 28 patients from four pediatric pulmonary hypertension programs over two years. This descriptive, observational study describes the various methods of initiation of oral treprostinil in both prostanoid-naïve patients and those transitioning from parenteral or inhaled prostanoids. The youngest patient was four years old and the smallest weighed 16 kg. We describe adverse reactions and their management. Most patients in this study (27/28) were able to successfully initiate therapy. However, gastrointestinal adverse reactions were common; half of the patients started on this therapy had discontinued it within the two-year study period.