Pulmonary Hypertension in Infants: Subcutaneous Treprostinil?

Lara C. Pullen, PhD

July 08, 2014

Subcutaneous treprostinil (SCTre) is an effective second-line therapy for former premature infants who have chronic lung disease and severe pulmonary hypertension, according to Dina J. Ferdman, MD, from the Division of Pediatric Cardiology, Department of Pediatrics, Columbia University Medical Center, College of Physicians and Surgeons, New York City, and colleagues. They published the results of their case series of 5 infants online June 30 and in the July issue of Pediatrics. They found SCTre to be safe, efficacious, and well-tolerated.

"In our single-center experience, all 5 patients tolerated initiation of SCTre and demonstrated clinical and echocardiographic improvement. All patients were former premature infants who were critically ill and refractory to conventional therapies. Because of the novel administration of the medication, extra training was required for the intensive care nursing staff," the authors write in their discussion.

SCTre has been used as a treatment in both children and adults. The current case series builds on another report of 8 children with congenital heart defects and idiopathic pulmonary arterial hypertension who appeared to benefit from SCTre. In adults, however, SCTre can cause severe site erythema, bleeding, bruising, and/or infection.

"The authors have added to the small but growing literature on subcutaneous treprostinil use in previously premature infants. With a follow-up time [of] from 3 to 30 weeks, they report the expected efficacy, similar to [intravenous] administration, and no obvious side effects or site limitation. However, I caution that subcutaneous treprostinil is associated with a loss of viable infusion sites in older children and adults, and this case series does not have sufficient follow-up to deal with this potential long-term complication," Brian Hanna, MDCM, PhD Director, Section of Pulmonary Hypertension at The Children's Hospital of Philadelphia, Pennsylvania, explained to Medscape Medical News.

Infants who are born at very low birth weight are at risk for hypoalveolarization and prolonged ventilation and, consequently, severe lung injury and chronic lung disease. The authors predict that neonatal intensive care centers will be treating more such patients with the increasing survival of extremely premature infants.

Inhaled nitric oxide and intravenous prostacyclin were the first targeted pulmonary arterial hypertension therapies that were shown to be effective in children. The case series suggests SCTre may soon be added to the list of reasonable therapies, especially for infants with central line complications.

"This case series does not have sufficient follow-up or numbers to recommend subcutaneous treprostinil as an early first-line [pulmonary arterial hypertension]-specific therapy in all previously premature infants with [bronchopulmonary dysplasia]-associated PAH. This report lends credence to the suggestion that in infants with increased risk of central line complications, subcutaneous treprostinil is a reasonable alternative," explained Dr. Hanna.

The authors have disclosed no relevant financial relationships. Dr. Hanna is the institutional primary investigator for industry-sponsored grants to the Children's Hospital of Philadelphia from United Therapeutics, Gilead, Eli Lily, and Actelion.

Pediatrics. 2014;134:e274-e278. Abstract

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