Pediatric pulmonary arterial hypertension and the pediatrician

Lewis Romer, MD, professor of Anesthesiology and Critical Care Medicine at Johns Hopkins Medicine and principal investigator of the Mono vs. Dual Therapy for Pediatric Pulmonary Arterial Hypertension (MoD PAH) trial, recently discussed the vital role of pediatricians in identifying and managing pulmonary arterial hypertension (PAH) in children. This progressive and rare disease can present with nonspecific symptoms, making early recognition critical.

“When one is a general pediatrician, one is never sure who's going to walk into one's office,” Romer said. “Some disease processes kind of sneak up on people slowly over time, and they don’t even realize... this is a child who can’t climb up into a chair in the doctor’s office.” These subtle signs, such as fatigue or not keeping up with peers on the playground, Romer used as an example, can be early indicators of PAH, emphasizing the need for vigilance among primary care providers.

The MoD PAH trial, a Phase 3 study, is evaluating the safety and efficacy of sildenafil monotherapy versus combination therapy with sildenafil and bosentan in children newly diagnosed with PAH. Currently recruiting participants, the trial includes 15 centers across the United States to ensure accessibility. The goal is to determine the best first-line treatment for children with World Health Organization functional class II or III symptoms.

Click here for more information on the MoD PAH trial, as explained by Romer in a previous Contemporary Pediatrics video interview.

Romer highlighted the importance of referral to specialized centers for accurate diagnosis and care. “If [pediatricians] are suspicious of pulmonary hypertension, referral to a pulmonary hypertension center, if feasible, is the very best thing to do as quickly as possible,” he explained. These centers are equipped to make precise diagnoses efficiently and safely, providing the foundation for effective treatment planning, stated Romer.

Despite a child’s participation in a specialized trial, the pediatrician remains a cornerstone of their care team. Romer emphasized the significance of ongoing collaboration: “The pediatrician continues to be the first contact for things that are not right, for things that are unusual.”

The MoD PAH trial also integrates routine follow-up, including monthly calls and quarterly visits, to foster a supportive relationship with families. However, Romer reiterated that pediatricians are the consistent presence in a child’s life, offering continuity of care and a trusted partnership in navigating this challenging disease.

Reference:

Fitch, J. Investigating mono vs dual therapy for pediatric pulmonary arterial hypertension. Contemporary Pediatrics. January 13, 2024. Accessed January 20, 2025. https://mjh.sanity.studio/contpeds/structure/article;edb2ab89-ff82-480b-84eb-0646387bb76c