Subcutaneous Treprostinil Used to Treat PAH Infants

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by Maureen Newman |

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infant PAH

infant PAHAlthough pulmonary hypertension and chronic lung disease are usually associated with adults, some cases develop in infancy. Dina Ferdman, MD, from Columbia University Medical Center, is finding a way to treat infant patients, who were premature and have severe pulmonary arterial hypertension (PAH) and chronic lung disease, using a second-line therapy known as subcutaneous treprostinil (SCTre).

According to Dr. Ferdman’s study published in Pediatrics, “There is a need for improved delivery of targeted PAH therapies for this subgroup of patients who have severe and persistent PAH despite standard respiratory care for chronic lung disease.” The research team, led by Dr. Ferdman and principal investigator Usha Krishnan, MD, also from the Department of Pediatrics at Columbia University Medical Center, conducted a case report of five infants continuously infused with SCTre, a long-acting prostanoid with hemodynamic effects similar to those of traditional intravenous epoprostenol.

All five infants tolerated SCTre and showed improved right ventricular function, estimated pulmonary hypertension, and required respiratory support. None of the patients experienced bleeding, bruising, or infection, which can be complications of intravenous therapy and are complications of SCTre in adults.

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pulmonary arterial hypertension in infantsThe case at hand builds on a report of eight children with congenital heart defects and idiopathic PAH who were infused with SCTre. “The authors have added to the small but growing literature on ScTre use in previously premature infants.,” said Brian Hanna, MDCM, PhD Director, in the Section of Pulmonary Hypertension at The Children’s Hosptital of Philadelphia, in a news report from Medscape Medical News. “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.”

Premature infants are predisposed for severe lung injury and chronic lung disease because low birth weight increases the risk for hypoalveolarization and prolonged ventilation. SCTre may soon be added to inhaled nitric oxide and intravenous prostacyclin as targeted PAH therapies for children.

However, Dr. Hanna cautions, “SCTre 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.” While SCTre is a reasonable alternative to current treatments, follow-up reports of greater numbers of patients are necessary.

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