Treprostinil Improves Severity of PH-associated CDH in Infants, but May Cause Other Problems, Study Finds

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by Diogo Pinto |

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pediatric study of Revatio

Intravenous infusions of treprostinil can improve disease severity in children with pulmonary hypertension (PH) associated with congenital diaphragmatic hernia (CDH), a birth defect that occurs when the diaphragm does not form properly, a new study shows.

However, these babies have a greater likelihood of needing extracorporeal respiratory support, longer periods on mechanical ventilation, and longer hospital stays.

The findings were published in a study titled “Treprostinil Improves Persistent Pulmonary Hypertension Associated with Congenital Diaphragmatic Hernia,” in The Journal of Pediatrics.

Treprostinil is a vasodilator approved for the treatment of pulmonary arterial hypertension in adults. It is marketed as Remodulin (by United Therapeutics) to be administered intravenously in adult patients.

CDH is a major birth defect affecting 1 in every 3,000 to 4,000 live births. Despite advances in treatments, the mortality rate ranges from 20-30% at tertiary care centers.

The severity and persistence of PH is known to be one of the major causes of morbidity and mortality in infants with CDH. Ventilators and other extracorporeal support are often needed.

Researchers have investigated the effect of continuous treprostinil use on the severity of PH-associated CDH in infants. The team also assessed the therapy’s safety and tolerability in these young patients.

Seventeen babies were treated with treprostinil for a median of 54.5 days and were compared to a control group of 147 children with CDH only and who did not receive treprostinil.

PH severity was measured by echocardiogram and serum B-type natriuretic peptide (BNP) levels, a hormone produced by the heart in response to heart failure. These measurements were done before the start of treatment, and 24 hours, one week, and one month after treatment was initiated.

Echocardiogram results showed a significant improvement in PH severity one month after treprostinil was started, and a reduction in blood pressure. Also, BNP levels were seen to be reduced one week after the start of treprostinil therapy (from 1,439 to 393 picograms per milliliter of blood), and after one month (242 pg/mL).

But despite these improvements, babies treated with treprostinil were more likely to need extracorporeal respiratory support compared to the control group (76.5% vs. 25.2%, respectively), to have a longer duration on mechanical ventilation (76.5 days vs. 30.9 days), and a longer hospital stay (144 days vs. 60 days).

No adverse events were reported related to treprostinil treatment, but the overall mortality rate remained high (35%) in these infants.

“We found that treprostinil improved pulmonary hypertension severity assessed by echocardiogram and BNP in a cohort of critically ill infants with CDH and persistent pulmonary hypertension,” researchers wrote.

“Larger, prospective studies are needed to elucidate ideal patient selection and timing of initiation of therapy, as well as effect on mortality related to pulmonary hypertension,” they added.